"Internal Identifier"~"FDA Center"~"Application Type"~"Application Number"~"Applicant Name"~"Product Name"~"Original Application Approval Date"~"Submission Type"~"Submission Number"~"PMR/PMC Unique Identifier"~"PMR or PMC"~"PMR/PMC Type"~"CDER PMR/PMC Set Number"~"PMR/PMC Number"~"PMR/PMC Description"~"PMR/PMC Status"~"PMR/PMC Status Explanation"~"PMR/PMC Original Final Report Due Date"~"Last Annual Status Report Received Date"
383243~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Supplement"~656~"125742/656-3"~"PMC"~"506B PMC"~~3~"Prospectively designed study to evaluate safety and immunogenicity of COMIRNATY (COVID-19 Vaccine, mRNA) (2025-2026 Formula) in participants 5 years through 11 years of age with at least one underlying condition that puts them at high risk for severe outcomes from COVID-19."~"Ongoing"~~9/30/2026 0:00:00~10/17/2025 0:00:00
383244~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Other"~686~"125742/686-3"~"PMR"~"Pediatric Research Equity Act"~~3~"Deferred pediatric Study C4591048 Substudy E to evaluate the safety and effectiveness of a single dose of COMIRNATY in children 5 through 11 years of age."~"Fulfilled"~"Per FDA letter dated August 27, 2025, this PMR has been fulfilled."~4/30/2025 0:00:00~10/17/2025 0:00:00
383245~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Supplement"~276~"125742/276-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred Study C4591048 Substudy A to evaluate the safety and effectiveness of a single dose of COMIRNATY in children 6 months through 4 years of age. "~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~9/30/2026 0:00:00~10/17/2025 0:00:00
383246~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Supplement"~656~"125742/656-1"~"PMC"~"506B PMC"~~1~"Prospectively designed study to evaluate safety and immunogenicity of COMIRNATY (COVID-19 Vaccine, mRNA) (2025-2026 Formula) in participants 65 years of age and older and in participants 12 years through 64 years of age with at least one underlying condition that puts them at high risk for severe outcomes from COVID-19."~"Pending"~~9/30/2026 0:00:00~10/17/2025 0:00:00
383247~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Supplement"~656~"125742/656-2"~"PMC"~"506B PMC"~~2~"A randomized, double-blind, placebo-controlled clinical study evaluating the safety and efficacy of a BNT162b2 variant-adapted vaccine in adults 50 years through 64 years of age without high-risk conditions for severe COVID-19."~"Pending"~~5/31/2027 0:00:00~10/17/2025 0:00:00
383248~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Supplement"~656~"125742/656-4"~"PMC"~"506B PMC"~~4~"A prospective, exploratory, placebo-controlled, randomized study to evaluate detection of circulating SARS-COV-2 spike antigen and self-reported symptoms of post-COVID-19 vaccination syndrome or Long COVID symptoms at Month 1, 3, 6, and 12 in vaccine and control arms."~"Pending"~~10/31/2027 0:00:00~10/17/2025 0:00:00
383249~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Original"~0~"125742/0-10"~"PMC"~"506B PMC"~~10~"Study C4591022, entitled ""Pfizer-BioNTech COVID-19 Vaccine Exposure during Pregnancy: A Non-Interventional Post-Approval Safety Study of Pregnancy and Infant Outcomes in the Organization of Teratology Information Specialists (OTIS)/MotherToBaby Pregnancy Registry."""~"Submitted"~~12/31/2025 0:00:00~10/17/2025 0:00:00
383250~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Original"~0~"125742/0-4"~"PMR"~"505 (o)(3)"~~4~"Study C4591009, entitled ""A Non-Interventional Post-Approval Safety Study of the Pfizer-BioNTech COVID-19 mRNA Vaccine in the United States,"" to evaluate the occurrence of myocarditis and pericarditis following administration of COMIRNATY."~"Submitted"~~10/31/2025 0:00:00~10/17/2025 0:00:00
383251~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Original"~0~"125742/0-5"~"PMR"~"505 (o)(3)"~~5~"Study C4591021, entitled ""Post Conditional Approval Active Surveillance Study Among Individuals in Europe Receiving the Pfizer-BioNTech Coronavirus Disease 2019 (COVID-19) Vaccine,"" to evaluate the occurrence of myocarditis and pericarditis following administration of COMIRNATY."~"Submitted"~~9/30/2024 0:00:00~10/17/2025 0:00:00
383252~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Original"~0~"125742/0-6"~"PMR"~"505 (o)(3)"~~6~"Study C4591021 substudy to describe the natural history of myocarditis and pericarditis following administration of COMIRNATY."~"Submitted"~~9/30/2024 0:00:00~10/17/2025 0:00:00
383253~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Supplement"~276~"125742/276-3"~"PMC"~"506B PMC"~~3~"Study C4591054, Substudy B, to evaluate immune responses following a single dose of COMIRNATY (2023-2024 Formula) in individuals 12 years and older who are not previously vaccinated with a COVID-19 vaccine."~"Fulfilled"~~12/31/2024 0:00:00~10/17/2025 0:00:00
383254~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Supplement"~276~"125742/276-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric Study C4591048 Substudy E to evaluate the safety and effectiveness of a single dose of COMIRNATY in children 2 through 11 years of age."~"Released"~"Per FDA letter dated August 15, 2025, this PMR has been released."~4/30/2025 0:00:00~10/17/2025 0:00:00
383199~"CBER"~"BLA"~125039.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Aralast; Aralast NP/Alpha-1-Proteinase Inhibitor (Human)"~12/23/2002 0:00:00~"Supplement"~69~"125039/69-4"~"PMC"~"506B PMC"~~4~"Contingent on the outcome of the pilot trial described in commitment 3,  conduct and report  the results of an adequately-powered study of clinically meaningful endpoints(s).  Based on the results of the pilot study and the available scientific data at the time that this study is being designed,  work with entities maintaining registries of alpha1-proteinase inhibitor deficient patients and with the National Institutes of Health (NIH) to design and conduct an adequately-powered study of a clinically meaningful endpoint(s). The study design could involve a single product or could potentially involve a cooperative simultaneous study of multiple products in parallel arms, using a factorial design."~"Delayed"~"The applicant has not submitted an acceptable protocol."~7/31/2024 0:00:00~2/10/2026 0:00:00
383200~"CBER"~"BLA"~125039.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Aralast; Aralast NP/Alpha-1-Proteinase Inhibitor (Human)"~12/23/2002 0:00:00~"Supplement"~69~"125039/69-3"~"PMC"~"506B PMC"~~3~"Conduct and report the results of a pilot trial to determine the effect of regular administration of the product on one or more clinically meaningful endpoint(s).  Examples of acceptable endpoints include pulmonary exacerbations, serial pulmonary functions, and serial quantitative computerized axial tomographic (CT) lung scans."~"Delayed"~"The applicant has not submitted an acceptable protocol."~7/31/2017 0:00:00~2/10/2026 0:00:00
383201~"CBER"~"BLA"~125325.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Glassia/Alpha-1-Proteinase Inhibitor (Human)"~7/1/2010 0:00:00~"Supplement"~242~"125325/242-1"~"PMC"~"506B PMC"~~1~"Kamada Ltd. commits to conduct and report the results of a Human Factors study, to evaluate the types of problems that patients/caregivers may encounter in self-infusing. This information will be used to revise, as needed, the training program and educational materials for self-infusion/non-healthcare provider caregiver infusion of GLASSIA."~"Delayed"~"The final protocol due date has passed with no final protocol received"~8/31/2018 0:00:00~8/26/2025 0:00:00
383202~"CBER"~"BLA"~125325.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Glassia/Alpha-1-Proteinase Inhibitor (Human)"~7/1/2010 0:00:00~"Original"~0~"125325/0-4"~"PMC"~"506B PMC"~~4~"Conduct a postmarketing clinical program for GLASSIA Investigating the Safety and Efficacy of GLASSIA vs. Placebo and another (Higher) dose of GLASSIA I.V. by Weekly Administration in Alpha1-Antitrypsin Deficient Patients with emphysema and design and conduct a fully statistically powered efficacy study for augmentation therapy with Alpha-1-Proteinase Inhibitor (Human)."~"Delayed"~"The study completion date has passed and the study has not yet been completed"~7/7/2025 0:00:00~8/26/2025 0:00:00
383203~"CBER"~"BLA"~125106.00~"GlaxoSmithKline Biologicals"~"Boostrix/Tetanus Toxoid, Reduced Diphtheria Toxoid and Acellular Pertussis Vaccine, Adsorbed"~5/3/2005 0:00:00~"Supplement"~1469~"125106/1469-1"~"PMC"~"506B PMC"~~1~"To conduct Study EPI-PERTUSSIS-075 VS US PR, an observational, exposure cohort study to evaluate pregnancy outcomes in individuals exposed to Boostrix as of the 1st day of the 27th week of gestation compared to pregnancy outcomes in individuals who do not receive any Tetanus Toxoid, Reduced Diphtheria Toxoid and Acellular Pertussis Vaccine Adsorbed (Tdap) during pregnancy. The registry will continue for at least 4 years, to prospectively collect data on 3000 pregnant individuals, including 1,500 exposed to Boostrix during pregnancy and 1,500 unexposed to any Tdap vaccine throughout pregnancy."~"Ongoing"~~1/31/2027 0:00:00~12/8/2025 0:00:00
383204~"CBER"~"BLA"~125265.00~"GlaxoSmithKline Biologicals"~"Rotarix/Rotavirus Vaccine, Live, Oral"~4/3/2008 0:00:00~"Supplement"~645~"125265/645-1"~"PMC"~"506B PMC"~~1~"To conduct a prospective observational postmarketing study (study 212329, protocol EPI-ROTA-070 VS LAT) to evaluate intussusception following administration of ROTARIX liquid formulation in Latin America"~"Pending"~~7/31/2028 0:00:00~5/28/2026 0:00:00
383205~"CBER"~"BLA"~125775.00~"GlaxoSmithKline Biologicals"~"AREXVY/Respiratory Syncytial Virus Vaccine, Adjuvanted"~5/3/2023 0:00:00~"Original"~0~"125775/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA (Study RSV OA=ADJ-015) to evaluate the safety and effectiveness in children and adolescents 2 to 18 years of age."~"Pending"~"Deferral Extension Requested on February 24, 2026. Deferral Extension Granted per FDA letter dated April 9, 2026."~12/30/2031 0:00:00~6/29/2026 0:00:00
383206~"CBER"~"BLA"~125775.00~"GlaxoSmithKline Biologicals"~"AREXVY/Respiratory Syncytial Virus Vaccine, Adjuvanted"~5/3/2023 0:00:00~"Original"~0~"125775/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric study under PREA (Study RSV OA=ADJ-016) to evaluate the safety and effectiveness in children and adolescents 2 to 18 years of age."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~5/30/2028 0:00:00~6/29/2026 0:00:00
383207~"CBER"~"BLA"~125775.00~"GlaxoSmithKline Biologicals"~"AREXVY/Respiratory Syncytial Virus Vaccine, Adjuvanted"~5/3/2023 0:00:00~"Supplement"~132~"125775/132-1"~"PMR"~"505 (o)(3)"~~1~"EPI-RSV-041 VS US DB (220149), to evaluate Guillain-Barre syndrome (GBS) and acute disseminated encephalomyelitis (ADEM) in adults 50 years of age and older vaccinated with AREXVY in the United States. Using a self-controlled risk interval (SCRI) design, the study will be conducted in the Sentinel System, and evaluate 1.9 million individuals vaccinated with AREXVY.
"~"Released"~~12/31/2031 0:00:00~6/29/2026 0:00:00
383208~"CBER"~"BLA"~125775.00~"GlaxoSmithKline Biologicals"~"AREXVY/Respiratory Syncytial Virus Vaccine, Adjuvanted"~5/3/2023 0:00:00~"Supplement"~132~"125775/132-2"~"PMC"~"506B PMC"~~2~"EPI-RSV-041 VS US DB (220149), to evaluate atrial fibrillation in adults 50 years of age and older vaccinated with AREXVY in the United States. Using a self-controlled risk interval (SCRI) design, the study will be conducted in the Sentinel System."~"Released"~~12/31/2031 0:00:00~6/29/2026 0:00:00
383209~"CBER"~"BLA"~125775.00~"GlaxoSmithKline Biologicals"~"AREXVY/Respiratory Syncytial Virus Vaccine, Adjuvanted"~5/3/2023 0:00:00~"Supplement"~414~"125775/414-1"~"PMR"~"505 (o)(3)"~~1~"EPI-RSV-041 VS US DB (220149), to evaluate Guillain-Barr  syndrome (GBS) and acute disseminated encephalomyelitis (ADEM) in adults 18 years of age and older vaccinated with AREXVY in the United States. Using a self-controlled risk interval (SCRI) design, the study will be conducted in the Sentinel System and evaluate 4.8 million individuals vaccinated with AREXVY."~"Pending"~~12/31/2031 0:00:00~6/29/2026 0:00:00
383210~"CBER"~"BLA"~125775.00~"GlaxoSmithKline Biologicals"~"AREXVY/Respiratory Syncytial Virus Vaccine, Adjuvanted"~5/3/2023 0:00:00~"Supplement"~414~"125775/414-2"~"PMC"~"506B PMC"~~2~"EPI-RSV-041 VS US DB (220149), to evaluate atrial fibrillation in adults 18 years of age and older vaccinated with AREXVY in the United States. Using a self-controlled risk interval (SCRI) design, the study will be conducted in the Sentinel System."~"Pending"~~12/31/2031 0:00:00~6/29/2026 0:00:00
383211~"CBER"~"BLA"~125546.00~"GlaxoSmithKline Biologicals"~"BEXSERO/Meningococcal Group B Vaccine"~1/23/2015 0:00:00~"Original"~0~"125546/0-3"~"PMR"~"Pediatric Research Equity Act"~~3~"A deferred pediatric study (V72_57) under PREA to evaluate the safety and immunogenicity of BEXSERO in North American infants 6 weeks through 12 months of age for the prevention of invasive group B meningococcal disease.

"~"Submitted"~"The final report was submitted to FDA on January 30, 2026."~6/30/2026 0:00:00~3/20/2026 0:00:00
383212~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Original"~0~"125769/0-5"~"PMR"~"505 (o)(3)"~~5~"A postmarketing retrospective cohort study utilizing Centers for Medicare and Medicaid Services (CMS) claims data, to evaluate the serious risk of Guillain-Barre syndrome (GBS) among approximately 1.5 million older adults vaccinated with ABRYSVO in the United States (Study C3671031)."~"Pending"~~5/31/2030 0:00:00~7/28/2025 0:00:00
383213~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Original"~0~"125769/0-3"~"PMR"~"Pediatric Research Equity Act"~~3~"Deferred pediatric study under PREA (Study C3671018), to evaluate safety and effectiveness in seropositive, then seronegative infants <2 years of age"~"Pending"~"The study has not been imitated but does not meet the criterion for delayed."~6/30/2028 0:00:00~7/28/2025 0:00:00
383214~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Other"~447~"125769/447-1"~"PMR"~"505 (o)(3)"~~1~"A Post-Marketing Study to Evaluate the Safety of ABRYSVO on the Risk of Guillain-Barr  Syndrome (GBS) in Pregnancy (Protocol C3671081)."~"Pending"~~3/31/2035 0:00:00~7/28/2025 0:00:00
383215~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Original"~0~"125769/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric study under PREA (Study C3671017), to evaluate safety and effectiveness in high-risk immunocompromised children 2 to <18 years of age"~"Delayed"~"The study completion date has passed, and the study has not yet been completed."~6/30/2026 0:00:00~7/28/2025 0:00:00
383216~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Original"~0~"125769/0-7"~"PMC"~"506B PMC"~~7~"Complete the ongoing Phase 3 study to Evaluate the Efficacy, Immunogenicity, and Safety of ABRYSVO in Adults (Study C3671013) and to evaluate the safety and effectiveness of revaccination."~"Delayed"~"The final report due date has passed with no final report received. "~9/30/2025 0:00:00~7/28/2025 0:00:00
383217~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Original"~0~"125769/0-4"~"PMR"~"Pediatric Research Equity Act"~~4~"Deferred nonclinical program under PREA to evaluate vaccine-associated enhanced respiratory disease"~"Submitted"~"The final report was submitted to FDA on May 6, 2025."~6/30/2025 0:00:00~7/28/2025 0:00:00
383218~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Original"~0~"125769/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA (Study C3671016), to evaluate safety and effectiveness in children and adolescents 2 to <18 years of age"~"Submitted"~"The final report was submitted to FDA on 6/5/2025."~6/30/2025 0:00:00~7/28/2025 0:00:00
383219~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Supplement"~225~"125769/225-1"~"PMR"~"505 (o)(3)"~~1~"A postmarketing study utilizing electronic health record (EHR) and claims data across FDA Sentinel System research partners to evaluate Guillain-Barre syndrome (GBS) following ABRYSVO vaccination in adults aged 18 through 59 years at increased risk of RSV-LRTD (Study C3671072)."~"Pending"~~1/31/2030 0:00:00~7/28/2025 0:00:00
383220~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Original"~0~"125769/0-10"~"PMR"~"505 (o)(3)"~~10~"A Post-Marketing Safety Study to Evaluate the Safety of Respiratory Syncytial Virus Vaccine (ABRYSVO) Exposure During Pregnancy in an Integrated Healthcare System in the United States (Protocol C3671042). This retrospective non-interventional cohort study using electronic healthcare data from a real-world healthcare system in the United States will evaluate preterm birth and hypertensive disorders of pregnancy in at least 4,712 ABRYSVO-exposed pregnant women in comparison to a group of women not exposed to ABRYSVO."~"Pending"~~8/31/2030 0:00:00~7/28/2025 0:00:00
383221~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Supplement"~225~"125769/225-2"~"PMC"~"506B PMC"~~2~"A post-marketing active surveillance safety study of atrial fibrillation following ABRYSVO among older adults and adults ages 18 through 59 years who are at increased risk for LRTD caused by RSV in the Veterans Affairs health system (Study C3671037)."~"Ongoing"~~5/26/2028 0:00:00~7/28/2025 0:00:00
383222~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Original"~0~"125769/0-11"~"PMR"~"505 (o)(3)"~~11~"Safety of respiratory syncytial virus stabilized prefusion F subunit vaccine (RSVpreF) in pregnant women and their offspring in a real world setting in Europe  (Protocol C3671026). This retrospective cohort study using electronic healthcare data from the Vaccine Monitoring Collaboration for Europe (VAC4EU) will evaluate preterm birth and hypertensive disorders of pregnancy in ABRYSVO-exposed pregnant women compared to pregnant women not exposed to ABRYSVO."~"Ongoing"~~9/30/2029 0:00:00~7/28/2025 0:00:00
383223~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Original"~0~"125769/0-8"~"PMR"~"505 (o)(3)"~~8~"A Rapid Surveillance and Cohort Post-Marketing Safety Study to Evaluate the Safety of Respiratory Syncytial Virus Vaccine (ABRYSVO) Exposure During Pregnancy in the United States (Protocol C3671027). This postmarketing database study will utilize Sentinel System claims data, including Medicaid claims data, to conduct near real-time monitoring and evaluate preterm birth and hypertensive disorders of pregnancy among approximately 80,000 pregnant women vaccinated with ABRYSVO in the United States compared to a cohort of pregnant women not exposed to ABRYSVO. "~"Ongoing"~~2/28/2029 0:00:00~7/28/2025 0:00:00
383224~"CBER"~"BLA"~125769.00~"Pfizer Inc."~"ABRYSVO/Respiratory Syncytial Virus Vaccine"~5/31/2023 0:00:00~"Original"~0~"125769/0-9"~"PMR"~"505 (o)(3)"~~9~"Post-Marketing Safety Study Using a Pregnancy Registry to Evaluate the Safety of Respiratory Syncytial Virus Vaccine (ABRYSVO) Exposure During Pregnancy (Protocol C3671041). This prospective, non-interventional pregnancy registry will evaluate preterm birth and hypertensive disorders of pregnancy in approximately 1,854 pregnant women (including 927 pregnant women exposed to ABRYSVO compared to a group of 927 pregnant women not exposed to ABRYSVO)."~"Ongoing"~~9/30/2031 0:00:00~7/28/2025 0:00:00
383225~"CBER"~"BLA"~125692.00~"Seqirus Inc."~"AUDENZ/Influenza A (H5N1) Monovalent Vaccine, Adjuvanted"~1/31/2020 0:00:00~"Original"~0~"125692/0-3"~"PMR"~"Pediatric Research Equity Act"~~3~"Deferred pediatric study V89_19 under PREA to evaluate the safety and immunogenicity of AUDENZ when administered to healthy infants 0 to < 6 months of age."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~~4/6/2024 0:00:00
383226~"CBER"~"BLA"~125692.00~"Seqirus Inc."~"AUDENZ/Influenza A (H5N1) Monovalent Vaccine, Adjuvanted"~1/31/2020 0:00:00~"Original"~0~"125692/0-4"~"PMC"~"506B PMC"~~4~"To establish a pregnancy registry in the U.S. that is able to prospectively collect data on an actively recruited cohort to study the safety of Audenz Vaccine during pregnancy. A draft protocol for this pregnancy registry will be prepared under the assumption that the vaccine would be distributed to the general population in the U.S. in an officially-declared H5N1 influenza virus pandemic. Once the circumstances of vaccine usage in an officially-declared H5N1 influenza virus pandemic are determined by the U.S. Government, Seqirus will work with the FDA, in coordination with BARDA (Biomedical Advanced Research and Development Authority) and CDC (Center for Disease Control), to finalize the protocol and initiate the registry."~"Pending"~~~4/6/2024 0:00:00
383227~"CBER"~"BLA"~125510.00~"Seqirus Inc."~"FLUAD; FLUAD QUADRIVALENT/Influenza Vaccine, Adjuvanted"~11/24/2015 0:00:00~"Original"~0~"125510/0-1"~"PMR"~"Accelerated Approval"~~1~" To conduct the confirmatory study (V118_18) to assess efficacy of Fluad aQIV as compared to an active control Tdap vaccine (tetanus toxoid, reduced diphtheria toxoid and acellular pertussis vaccine, adsorbed [Boostrix]) in adults 65 years of age and older"~"Submitted"~"The final report was submitted to FDA on January 18, 2019."~8/31/2019 0:00:00~1/24/2024 0:00:00
383228~"CBER"~"BLA"~125510.00~"Seqirus Inc."~"FLUAD; FLUAD QUADRIVALENT/Influenza Vaccine, Adjuvanted"~11/24/2015 0:00:00~"Supplement"~143~"125510/143-1"~"PMR"~"Accelerated Approval"~~1~"To conduct Study V118_24, a clinical disease endpoint trial in subjects 65 years of age and older with Fluad Quadrivalent."~"Delayed"~"As per the 180-day AA PMR Progress Report submitted July 21, 2025, the study is behind the original schedule because the study was modified to a relative vaccine efficacy design which required enrollment of significantly higher number of subjects to achieve study objectives. This report was found to be complete."~9/30/2024 0:00:00~1/24/2024 0:00:00
383229~"CBER"~"BLA"~125722.00~"PTC Therapeutics"~"KEBILIDI/eladocagene exuparvovec-tneq"~11/13/2024 0:00:00~"Original"~0~"125722/0-1"~"PMR"~"Accelerated Approval"~~1~"Submit the clinical reports and datasets of clinical studies conducted in patients with AADC deficiency in the United States treated with eladocagene exuparvovec -tneq to verify and describe its clinical benefit. Such studies should, at minimum, evaluate the product's effects on the serious manifestations of AADC deficiency in adult and pediatric patients, including but not limited to motor function."~"Ongoing"~"As per the 180-day PMR submitted on 1/16/2026, the study is progressing as per the original schedule milestones. There are currently 13  of 13 planned participants enrolled. This report was found to be complete."~9/30/2029 0:00:00~
383230~"CBER"~"BLA"~125280.00~"Valneva Austria GmbH"~"Ixiaro/Japanese Encephalitis Vaccine, Inactivated, Adsorbed"~3/30/2009 0:00:00~"Supplement"~125~"125280/125-1"~"PMC"~"506B PMC"~~1~"Conduct post-marketing assessments of human factors issues that may affect preparation and administration of the 0.25 mL dose."~"Submitted"~~3/31/2014 0:00:00~5/28/2026 0:00:00
383231~"CBER"~"BLA"~125603.00~"Vericel Corporation"~"MACI/autologous cultured chondrocytes on porcine collagen membrane"~12/13/2016 0:00:00~"Original"~0~"125603/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA for the treatment of symptomatic, single or multiple full-thickness cartilage defects of the knee with or without bone involvement in pediatric patients ages 10 to 17"~"Submitted"~"The study is ongoing"~12/31/2025 0:00:00~2/11/2026 0:00:00
383232~"CBER"~"BLA"~125770.00~"Pfizer Ireland Pharmaceuticals Unlimited Company"~"PENBRAYA/Meningococcal Groups A, B, C, W, and Y Vaccine"~10/20/2023 0:00:00~"Original"~0~"125770/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA (Study B1971067) to evaluate the safety and immunogenicity of MenABCWY in individuals 1 year to <10 years of age."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~5/31/2027 0:00:00~12/17/2025 0:00:00
383233~"CBER"~"BLA"~125770.00~"Pfizer Ireland Pharmaceuticals Unlimited Company"~"PENBRAYA/Meningococcal Groups A, B, C, W, and Y Vaccine"~10/20/2023 0:00:00~"Original"~0~"125770/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric study under PREA (Study C3511005) to evaluate the safety and immunogenicity of MenABCWY in individuals 1 year to <10 years of age."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~11/30/2030 0:00:00~12/17/2025 0:00:00
383234~"CBER"~"BLA"~125770.00~"Pfizer Ireland Pharmaceuticals Unlimited Company"~"PENBRAYA/Meningococcal Groups A, B, C, W, and Y Vaccine"~10/20/2023 0:00:00~"Original"~0~"125770/0-3"~"PMC"~"506B PMC"~~3~"A Pregnancy Registry Study to Evaluate the Safety of PENBRAYA Meningococcal Vaccine Exposure During Pregnancy."~"Ongoing"~~4/30/2033 0:00:00~12/17/2025 0:00:00
383235~"CBER"~"BLA"~125643.00~"Kite Pharma, Inc."~"YESCARTA/axicabtagene ciloleucel"~10/18/2017 0:00:00~"Supplement"~248~"125643/248-2"~"PMR"~"505 (o)(3)"~~2~"A postmarketing multicenter, prospective, observational study to assess the long-term safety of axicabtagene ciloleucel and the risk of secondary malignancies occurring after treatment with axicabtagene ciloleucel.  The study will include at least 300 adult patients with relapsed or refractory follicular lymphoma; the enrolled patients will be followed for 15 years after the product administration. "~"Ongoing"~~6/30/2042 0:00:00~12/8/2023 0:00:00
383236~"CBER"~"BLA"~125643.00~"Kite Pharma, Inc."~"YESCARTA/axicabtagene ciloleucel"~10/18/2017 0:00:00~"Supplement"~248~"125643/248-1"~"PMR"~"Accelerated Approval"~~1~"A randomized phase 3 trial of axicabtagene ciloleucel in patients with relapsed or refractory follicular lymphoma.  Patients will be randomized to axicabtagene ciloleucel or to an investigator's choice of regimens consistent with the standard of care. The primary endpoint will be progression-free survival, with secondary endpoints that include objective response rate and overall survival. 
"~"Ongoing"~"Study is Ongoing."~9/30/2027 0:00:00~12/8/2023 0:00:00
383237~"CBER"~"BLA"~125643.00~"Kite Pharma, Inc."~"YESCARTA/axicabtagene ciloleucel"~10/18/2017 0:00:00~"Original"~0~"125643/0-1"~"PMR"~"505 (o)(3)"~~1~"A post-marketing, prospective, multi-center, observational study to assess the long-term safety of axicabtagene ciloleucel and the risk of secondary malignancies occurring after treatment with axicabtagene ciloleucel. The study will include at least 1500 adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy; the enrolled patients will be followed for 15 years after the product administration. "~"Ongoing"~~12/22/2038 0:00:00~12/8/2023 0:00:00
383238~"CBER"~"BLA"~125703.00~"Kite Pharma, Inc."~"TECARTUS/brexucabtagene autoleucel"~7/24/2020 0:00:00~"Supplement"~91~"125703/91-1"~"PMR"~"505 (o)(3)"~~1~"A postmarketing, prospective, multicenter, observational study to assess the long-term safety of brexucabtagene autoleucel and the risk of all secondary malignancies occurring after treatment with brexucabtagene autoleucel. The study will include at least 500 adult patients with r/r B-cell precursor ALL, who will be followed for 15 years after the product administration."~"Ongoing"~~10/31/2042 0:00:00~9/19/2024 0:00:00
383239~"CBER"~"BLA"~125703.00~"Kite Pharma, Inc."~"TECARTUS/brexucabtagene autoleucel"~7/24/2020 0:00:00~"Original"~0~"125703/0-3"~"PMR"~"505 (o)(3)"~~3~"A post-marketing, prospective, multi-center, observational study to assess the long-term safety of brexucabtagene autoleucel and the risk of secondary malignancies occurring after treatment with brexucabtagene autoleucel. The study will include at least 500 adult patients with relapsed or refractory mantle cell lymphoma after two or more lines of systemic therapy; the enrolled patients will be followed for 15 years after the product administration.
"~"Ongoing"~~8/31/2041 0:00:00~9/19/2024 0:00:00
383240~"CBER"~"BLA"~125703.00~"Kite Pharma, Inc."~"TECARTUS/brexucabtagene autoleucel"~7/24/2020 0:00:00~"Original"~0~"125703/0-2"~"PMR"~"Accelerated Approval"~~2~"Conduct a study of brexucabtagene autoleucel treatment of subjects with relapsed or refractory mantle cell lymphoma who have not been exposed to a Bruton tyrosine kinase (BTK) inhibitor. A cohort of subjects naive to BTK inhibitor therapy will be added to the ongoing ZUMA-2 study to fulfill this requirement. Eighty-six subjects will be enrolled. The primary efficacy endpoint will be objective response rate with a supportive efficacy endpoint of duration of response based on a minimum follow-up of 18 months after first objective disease response.
"~"Fulfilled"~"PMR was fulfilled on April 1, 2026"~10/31/2025 0:00:00~9/19/2024 0:00:00
383241~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Other"~541~"125742/541-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study C4591067 to evaluate the safety and effectiveness of COMIRANTY in infants 6 weeks to <6 months of age."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed. "~1/31/2028 0:00:00~10/17/2025 0:00:00
383242~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Original"~0~"125742/0-7"~"PMR"~"505 (o)(3)"~~7~"Study C4591036, a prospective cohort study with at least 5 years of follow-up for potential long-term sequelae of myocarditis after vaccination (in collaboration with Pediatric Heart Network)."~"Ongoing"~~5/31/2027 0:00:00~10/17/2025 0:00:00
383255~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Original"~0~"125742/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric Study C4591007 to evaluate the safety and effectiveness of COMIRNATY in infants and children 6 months to <12 years of age."~"Released"~"Per FDA letter dated August 15, 2025, this PMR has been released."~12/31/2024 0:00:00~10/17/2025 0:00:00
383256~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Supplement"~564~"125742/564-1"~"PMC"~"506B PMC"~~1~"Study C4591054, Substudy C, to evaluate immune responses following a single dose of COMIRNATY (2024-2025 Formula) in individuals 18 years of age and older."~"Submitted"~~8/31/2025 0:00:00~10/17/2025 0:00:00
383257~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Other"~686~"125742/686-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric Study C4591007 to evaluate the safety and effectiveness of COMIRNATY in infants and children 6 months through 4 years of age."~"Submitted"~"The final report was submitted to FDA on May 23, 2024."~5/31/2024 0:00:00~10/17/2025 0:00:00
383258~"CBER"~"BLA"~125742.00~"BioNTech Manufacturing GmbH"~"COMIRNATY/COVID-19 Vaccine, mRNA"~8/23/2021 0:00:00~"Other"~686~"125742/686-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric Study C4591007 to evaluate the safety and effectiveness of COMIRNATY in children 5 years through 11 years of age."~"Fulfilled"~"Per FDA Letter dated August 27, 2025 this PMR has been fulfilled."~5/31/2024 0:00:00~10/17/2025 0:00:00
383259~"CBER"~"BLA"~125743.00~"GC Biopharma Corp."~"Alyglo/immune globulin intravenous, human-stwk"~12/15/2023 0:00:00~"Original"~0~"125743/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"An Open-Label, Single-Arm, Historically Controlled, Prospective, Multicenter Phase III Study to Evaluate the Pharmacokinetics and Safety of Immuno- Globulin Intravenous (Human) 10% GC5107 in Pediatric Subjects 2 years to <17 Years of Age with Primary Humoral Immunodeficiency."~"Pending"~"This study is Pending and will begin in the future. "~11/30/2026 0:00:00~
383260~"CBER"~"BLA"~125706.00~"Mesoblast, Inc."~"RYONCIL/remestemcel-L-rknd"~12/18/2024 0:00:00~"Original"~0~"125706/0-1"~"PMR"~"505 (o)(3)"~~1~"Mesoblast will complete the assessment of simulated leachables safety study for remestemcel-L through its manufacturing process and storage. This study mustinclude an assessment of total leachables presented in DP that accumulate throughout the manufacturing process and storage over the shelf-life. Such
assessment should be performed in a real-time study using maximal hold times
and temperatures at respective steps.
A toxicological risk assessment of the overall leachables in the final drug product and final study report will be provided"~"Pending"~~10/31/2030 0:00:00~
383261~"CBER"~"BLA"~125737.00~"VBI Vaccines (Delaware), Inc."~"Trehebri; Prehebrio; Prehevbrio/Hepatitis B Vaccine (Recombinant)"~11/30/2021 0:00:00~"Original"~0~"125737/0-1"~"PMC"~"506B PMC"~~1~"To establish a pregnancy registry to prospectively collect data on reported exposures to PREHEVBRIO during pregnancy and evaluate pregnancy outcomes. The registry will collect information from 120 pregnant women."~"Pending"~~12/1/2032 0:00:00~2/3/2025 0:00:00
383262~"CBER"~"BLA"~125738.00~"Gamida Cell Ltd."~"OMISIRGE/omidubicel-onlv"~4/17/2023 0:00:00~"Supplement"~69~"125738/69-1"~"PMR"~"505 (o)(3)"~~1~"A postmarketing, prospective, multi-center, observational study to assess and characterize the risk of autoimmune cytopenia following treatment with omidubicel-onlv for severe aplastic anemia. The study will include at least 24 adult and pediatric patients with severe aplastic anemia who received omidubicel-onlv following reduced intensity conditioning and each enrolled patient will be followed for 2 years after product administration."~"Pending"~~1/31/2032 0:00:00~
383263~"CBER"~"BLA"~125738.00~"Gamida Cell Ltd."~"OMISIRGE/omidubicel-onlv"~4/17/2023 0:00:00~"Supplement"~69~"125738/69-2"~"PMC"~"506B PMC"~~2~"Gamida Cell Ltd. commits to complete Study 17-H-0091,  Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS using CordIn , Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome  to collect additional safety and efficacy data with omidubicel-onlv.
"~"Pending"~~9/30/2028 0:00:00~
383264~"CBER"~"BLA"~125807.00~"Abeona Therapeutics Inc."~"ZEVASKYN/Prademagene zamikeracel"~4/28/2025 0:00:00~"Original"~0~"125807/0-1"~"PMR"~"505 (o)(3)"~~1~~"Pending"~~7/31/2046 0:00:00~
383265~"CBER"~"BLA"~125832.00~"Precigen, Inc."~"PAPZIMEOS/zopapogene imadenovec-drba"~8/14/2025 0:00:00~"Original"~0~"125832/0-1"~"PMR"~"505 (o)(3)"~~1~"Precigen, Inc. will conduct a safety study to address the virus-detection-related safety concerns for the analytical method used to detect adventitious viral contamination in the harvest during the zopapogene imadenovec-drba drug substance manufacturing process. This study will include: (1) validation of assay specificity and detection limits for control viruses; and (2) verification of method suitability."~"Pending"~~2/28/2026 0:00:00~
383266~"CBER"~"BLA"~125832.00~"Precigen, Inc."~"PAPZIMEOS/zopapogene imadenovec-drba"~8/14/2025 0:00:00~"Original"~0~"125832/0-3"~"PMC"~"506B PMC"~~3~"Precigen, Inc. commits to conduct viral shedding studies following administration of zopapogene imadenovec-drba in adult patients with recurrent respiratory papillomatosis."~"Pending"~~12/31/2026 0:00:00~
383267~"CBER"~"BLA"~125832.00~"Precigen, Inc."~"PAPZIMEOS/zopapogene imadenovec-drba"~8/14/2025 0:00:00~"Original"~0~"125832/0-4"~"PMC"~"506B PMC"~~4~"Precigen, Inc. commits to conduct a prospective, single arm, open label study of the safety and efficacy of zopapogene imadenovec-drba in pediatric patients with recurrent respiratory papillomatosis."~"Pending"~~12/31/2028 0:00:00~
383268~"CBER"~"BLA"~125832.00~"Precigen, Inc."~"PAPZIMEOS/zopapogene imadenovec-drba"~8/14/2025 0:00:00~"Original"~0~"125832/0-2"~"PMC"~"506B PMC"~~2~"Precigen, Inc. commits to complete and submit the study report and dataset for study PRGN-2012-201, a single-arm, open-label Phase 1/2 study conducted in adult patients requiring 3 or more surgical interventions for management of RRP in the 12 months prior to treatment."~"Ongoing"~~12/31/2026 0:00:00~
383269~"CBER"~"BLA"~125846.00~"Fondazione Telethon Ets"~"WASKYRA/etuvetidigene autotemcel"~12/9/2025 0:00:00~"Original"~0~"125846/0-1"~"PMR"~"505 (o)(3)"~~1~"A postmarketing, prospective, observational study to assess and characterize the risk of secondary malignancies and long-term safety following treatment with etuvetidigene autotemcel. This study will enroll 14 patients with Wiskott- Aldrich Syndrome (WAS) who received treatment with etuvetidigene autotemcel. The enrolled patients will be followed for 15 years after product administration.
"~"Pending"~~12/31/2046 0:00:00~
383270~"CBER"~"BLA"~125846.00~"Fondazione Telethon Ets"~"WASKYRA/etuvetidigene autotemcel"~12/9/2025 0:00:00~"Original"~0~"125846/0-2"~"PMR"~"505 (o)(3)"~~2~"An adequate leachables safety assessment for the TLT003 drug product (DP) through its manufacturing process, storage, and in-use conditions. The assessment must include both elemental and organic leachables from the formulation, storage and in-use preparation product-contacting components appearing cumulatively in final DP. The leachables study can be conducted without active ingredient bysimulating the DP manufacturing process from the Wash 2 step through in-use preparation steps of the simulated DP. Such study should use maximal hold times and temperatures at respective manufacturing process steps to assess cumulative leachables in the DP from the cell wash through product freezing, shelf-life, storage, thawing, and in-use processing. A final study report and toxicological risk assessment should be provided.
"~"Pending"~~12/31/2026 0:00:00~
383271~"CBER"~"BLA"~125781.00~"Sarepta Therapeutics, Inc."~"ELEVIDYS/delandistrogene moxeparvovec-rokl"~6/22/2023 0:00:00~"Supplement"~189~"125781/189-1"~"PMR"~"505 (o)(3)"~~1~"Final Protocol Submission: February 28, 2026
Study Completion Date: October 31, 2029
Final Report Submission: April 30, 2030"~"Pending"~~~6/20/2025 0:00:00
383272~"CBER"~"BLA"~125781.00~"Sarepta Therapeutics, Inc."~"ELEVIDYS/delandistrogene moxeparvovec-rokl"~6/22/2023 0:00:00~"Original"~0~"125781/0-2"~"PMC"~"506B PMC"~~2~"Sarepta commits to conducting adequate analytical and clinical validation testing that can be used to identify patients with DMD who may benefit from delandistrogene moxeparvovec-rokl therapy. The results of the validation study are intended to inform product labeling. The clinical validation should be supported by a clinical bridging study. "~"Fulfilled"~~~6/20/2025 0:00:00
383273~"CBER"~"BLA"~125781.00~"Sarepta Therapeutics, Inc."~"ELEVIDYS/delandistrogene moxeparvovec-rokl"~6/22/2023 0:00:00~"Supplement"~34~"125781/34-1"~"PMR"~"Accelerated Approval"~~1~"Conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of delandistrogene moxeparvovec-rokl in patients with Duchenne's muscular dystrophy, who are non-ambulatory and have a confirmed mutation in the DMD gene. The trial should evaluate the effects of delandistrogene moxeparvovec-rokl on an endpoint that denotes clinical benefit."~"Released"~"As of November 14, 2025, FDA removed  the indication for non-ambulatory patients with DMD and therefore the confirmatory study was released."~11/30/2027 0:00:00~6/20/2025 0:00:00
383274~"CBER"~"BLA"~125868.00~"Orca Biosystems Inc."~"TREGZI/allogeneic regulatory T cell immunotherapy with HSPC and T cells-vldq"~6/30/2026 0:00:00~"Original"~0~"125868/0-1"~"PMC"~"506B PMC"~~1~"Orca Biosystems Inc. commits to complete and submit the final report and datasets for the Phase 3 component of Precision-T study with final analysis and formal hypothesis testing of the secondary endpoints including overall survival and GVHD- and relapse free survival (GRFS) as outlined in the statistical analysis plan."~"Pending"~~9/30/2030 0:00:00~
383275~"CBER"~"BLA"~125717.00~"Genetix Biotherapeutics Inc."~"ZYNTEGLO/betibeglogene autotemcel"~8/17/2022 0:00:00~"Original"~0~"125717/0-1"~"PMR"~"505 (o)(3)"~~1~"A postmarketing, prospective, multi-center, observational study to assess the long-term safety of betibeglogene autotemcel and the risk of secondary malignancies occurring after treatment with betibeglogene autotemcel. The study will include at least 150 B-thalassemia patients and the enrolled patients will be followed for 15 years after product administration. The study design will include monitoring (at pre-specified intervals) for clonal expansion with adequate testing strategies.

"~"Ongoing"~~3/31/2044 0:00:00~10/8/2025 0:00:00
383276~"CBER"~"BLA"~125788.00~"Genetix Biotherapeutics Inc."~"LYFGENIA/lovotibeglogene autotemcel"~12/8/2023 0:00:00~"Original"~0~"125788/0-2"~"PMR"~"505 (o)(3)"~~2~"A study to evaluate leachables of the bag over the duration of the shelf-life of lovotibeglogene autotemcel. This evaluation will also include a full toxicological risk assessment for the identified leachables and extractables."~"Fulfilled"~~3/30/2025 0:00:00~3/11/2026 0:00:00
383277~"CBER"~"BLA"~125788.00~"Genetix Biotherapeutics Inc."~"LYFGENIA/lovotibeglogene autotemcel"~12/8/2023 0:00:00~"Original"~0~"125788/0-1"~"PMR"~"505 (o)(3)"~~1~"A postmarketing, prospective, multi-center, observational study, to assess and characterize the risk of secondary malignancies after treatment with 
lovotibeglogene autotemcel and to assess the long-term safety of
lovotibeglogene autotemcel (Study REG-503). The study will include 250 
patients with sickle cell disease who received lovotibeglogene autotemcel, and each enrolled patient will be followed for 15 years after product administration. The study design will include monitoring (at pre-specified intervals) for clonalexpansion with adequate testing strategies.
"~"Submitted"~~12/31/2044 0:00:00~3/11/2026 0:00:00
383278~"CBER"~"BLA"~125755.00~"Genetix Biotherapeutics Inc."~"SKYSONA/elivaldogene autotemcel"~9/16/2022 0:00:00~"Original"~0~"125755/0-3"~"PMR"~"505 (o)(3)"~~3~"A postmarketing, prospective, multi-center, observational study to assess the long-term safety of elivaldogene autotemcel and the risk of secondary malignancies occurring after treatment with elivaldogene autotemcel. The study will include at least 120 cerebral adrenoleukodystrophy patients and the enrolled patients will be followed for 15 years after product administration. The study design will include monitoring (at pre-specified intervals) for clonal expansion with adequate testing strategies.
"~"Ongoing"~~4/30/2048 0:00:00~
383279~"CBER"~"BLA"~125755.00~"Genetix Biotherapeutics Inc."~"SKYSONA/elivaldogene autotemcel"~9/16/2022 0:00:00~"Original"~0~"125755/0-1"~"PMR"~"Accelerated Approval"~~1~"Follow all subjects who received elivaldogene autotemcel in Studies ALD-102 and ALD-104 to assess event-free survival (i.e., alive without Major Functional Disability (MFD) or need for hematopoietic stem cell transplant (HSCT)) for a minimum of ten years following administration of elivaldogene autotemcel.
"~"Ongoing"~"As per the 180-day AA PMR Progress Report submitted on November 13, 2025, the study/trial is progressing as per the original schedule of milestones. There are currently 64 of 64 planned participants enrolled. This report was found to be complete."~7/31/2032 0:00:00~
383280~"CBER"~"BLA"~125755.00~"Genetix Biotherapeutics Inc."~"SKYSONA/elivaldogene autotemcel"~9/16/2022 0:00:00~"Original"~0~"125755/0-2"~"PMR"~"Accelerated Approval"~~2~"Investigate event-free survival for at least five years post-treatment in 24 boys with more advanced early active, cerebral adrenoleukodystrophy (CALD) [(based on baseline Loes scores and Neurologic Function Score (NFS)] who will be newly treated with elivaldogene autotemcel (SKYSONA"~"Ongoing"~"As per the 180-day AA PMR Progress Report submitted on November 13, 2025, the study/trial is progressing as per the original schedule of milestones. However, the number of participants enrolled is lagging. There are currently 1 of 24 planned participants enrolled. This report was found to be complete."~12/31/2038 0:00:00~
383281~"CBER"~"BLA"~125806.00~"Rocket Pharmaceuticals Inc"~"KRESLADI/marnetegragene autotemcel"~3/26/2026 0:00:00~"Original"~0~"125806/0-1"~"PMR"~"Accelerated Approval"~~1~"Submit analyses of clinical outcomes including, at a minimum, overall survival, allogeneic HSCT-free survival, and infectious outcomes, as well as biomarker changes (e.g., neutrophil CD18 expression, CD11a expression) in: 1) all treated patients with severeLAD-I currently enrolled in Study RP-L201-0121-LTFU with each patient followed to at least 10 years of age; and 2) at least 4 newly treated pediatric patients with ITGB2-associated, severe LAD-I who are =1 year of age at the 
time of marnetegragene autotemcel administration with each patient followed to at least 2 years of age. All endpoints should be well defined, and data collected systematically and consistently considering potential confounding variables to enable data interpretation, e.g., antibiotic use in relation to incidence of serious infections, etc. Biomarker assessments should be based on appropriately validated bioanalytical assays (as assessed in PMR 2). All analyses should include 
comparisons to a suitable comparator for purposes of verifying and describing the clinical benefit of marnetegragene autotemcel in
ITGB2-associated severe LAD-I.
"~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~6/30/2034 0:00:00~
383282~"CBER"~"BLA"~125806.00~"Rocket Pharmaceuticals Inc"~"KRESLADI/marnetegragene autotemcel"~3/26/2026 0:00:00~"Original"~0~"125806/0-2"~"PMR"~"Accelerated Approval"~~2~"Submit data from supplemental validation studies performed on the LAD-1 Flow Cytometry assay described in SOP-778817. This validation is needed to enable data interpretation of your confirmatory study and specifically evaluate the performance of the CD18 (6p7) and CD11a assays  throughout the complete analytical range, including low cell surface expression levels, and should  include assessments of repeatability, linearity, accuracy, intermediate precision, and
specificity.

"~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~2/27/2027 0:00:00~
383283~"CBER"~"BLA"~125806.00~"Rocket Pharmaceuticals Inc"~"KRESLADI/marnetegragene autotemcel"~3/26/2026 0:00:00~"Original"~0~"125806/0-3"~"PMR"~"505 (o)(3)"~~3~"Submit analyses of safety data from a postmarketing, prospective, longitudinal, observational study assessing and characterizing the long-term safety risks of marnetegragene autotemcel in patients 
with severe LAD-I including the risk of secondary malignancies. The study will enroll a minimum of  10 patients with severe LAD-I who receive marnetegragene autotemcel. Safety data will be
collected for each patient for at least 15 years after product administration.
"~"Pending"~~12/31/2047 0:00:00~
383284~"CBER"~"BLA"~125806.00~"Rocket Pharmaceuticals Inc"~"KRESLADI/marnetegragene autotemcel"~3/26/2026 0:00:00~"Original"~0~"125806/0-4"~"PMR"~"505 (o)(3)"~~4~"An adequate leachables safety assessment for the KRESLADI drug product (DP) through its manufacturing process, storage, and in-use conditions. This assessment must include the following:
a. Assessment of both organic and elemental extractables from the high-risk for leachables manufacturing/storage components of final DP (i.e. cumulative leachables in DP).
b. The leachables study can be conducted by simulating the DP manufacturing process from the step with high-risk for leachables components ( Spin 3 and supernatant removal , i.e. cell wash, 
concentration, followed by formulation), and may include simulation of respective buffers and excipients (such as human serum albumin),
performed using maximal hold times and temperatures through the process, product freezing, shelf-life storage, thawing, and in-use
processing.
c. A full toxicological risk assessment for the identified leachables. Since the drug product is specifically intended for pediatric use, the permitted daily exposure (PDE) or comparator values should be calculated using the worst-case body weight assumption (e.g., 5-kg) for pediatric subjects administered the LV-RP-L201 drug product for the leachables toxicological risk assessment.

"~"Pending"~~5/31/2027 0:00:00~
383285~"CBER"~"BLA"~125546.00~"GlaxoSmithKline Biologicals"~"BEXSERO/Meningococcal Group B Vaccine"~1/23/2015 0:00:00~"Original"~0~"125546/0-5"~"PMC"~"506B PMC"~~5~"To conduct a safety and immunogenicity study (V72_79) to assess concomitant use of BEXSERO with a second dose of Meningococcal (Groups A, C, Y and W-135) Oligosaccharide Diphtheria CRM197 Conjugate Vaccine in persons 16 years through 18 years of age.
"~"Delayed"~"The study completion date has passed and the study has not yet been completed."~10/7/2022 0:00:00~3/20/2026 0:00:00
383286~"CBER"~"BLA"~125546.00~"GlaxoSmithKline Biologicals"~"BEXSERO/Meningococcal Group B Vaccine"~1/23/2015 0:00:00~"Original"~0~"125546/0-4"~"PMR"~"Pediatric Research Equity Act"~~4~"A deferred pediatric study (V72_28) under PREA to evaluate the safety and immunogenicity of BEXSERO in infants 2.5 months through 11 months of age and in children 2 years through 10 years of age for the prevention of invasive group B meningococcal disease.
"~"Submitted"~"The final report was submitted to FDA on February 12, 2016."~12/31/2015 0:00:00~3/20/2026 0:00:00
383287~"CBER"~"BLA"~125546.00~"GlaxoSmithKline Biologicals"~"BEXSERO/Meningococcal Group B Vaccine"~1/23/2015 0:00:00~"Supplement"~1058~"125546/1058-1"~"PMR"~"Pediatric Research Equity Act"~~1~"A deferred pediatric study (V72_28E1) under PREA to evaluate the safety and immunogenicity of BEXSERO in children >1 year through 9 years of age."~"Submitted"~"The final report was submitted to FDA on August 28, 2024."~8/31/2024 0:00:00~3/20/2026 0:00:00
383288~"CBER"~"BLA"~125546.00~"GlaxoSmithKline Biologicals"~"BEXSERO/Meningococcal Group B Vaccine"~1/23/2015 0:00:00~"Supplement"~1058~"125546/1058-2"~"PMR"~"Pediatric Research Equity Act"~~2~"A deferred pediatric study (V72P12E1) under PREA to evaluate the safety and immunogenicity of BEXSERO in children >1 year through 9 years of age."~"Submitted"~"The final report was submitted to FDA on August 28, 2024."~8/31/2024 0:00:00~3/20/2026 0:00:00
383289~"CBER"~"BLA"~125546.00~"GlaxoSmithKline Biologicals"~"BEXSERO/Meningococcal Group B Vaccine"~1/23/2015 0:00:00~"Supplement"~1058~"125546/1058-3"~"PMR"~"Pediatric Research Equity Act"~~3~"A deferred pediatric study (V72P12E2) under PREA to evaluate the safety and immunogenicity of BEXSERO in children >1 year through 9 years of age."~"Submitted"~"The final report was submitted to FDA on August 28, 2024."~8/31/2024 0:00:00~3/20/2026 0:00:00
383290~"CBER"~"BLA"~125546.00~"GlaxoSmithKline Biologicals"~"BEXSERO/Meningococcal Group B Vaccine"~1/23/2015 0:00:00~"Supplement"~1058~"125546/1058-4"~"PMR"~"Pediatric Research Equity Act"~~4~"A deferred pediatric study (V72P13E2) under PREA to evaluate the safety and immunogenicity of BEXSERO in children >1 year through 9 years of age."~"Submitted"~"The final report was submitted to FDA on August 28, 2024."~8/31/2024 0:00:00~3/20/2026 0:00:00
383291~"CBER"~"BLA"~125614.00~"GlaxoSmithKline Biologicals"~"SHINGRIX/Zoster Vaccine Recombinant, Adjuvanted"~10/20/2017 0:00:00~"Original"~0~"125614/0-1"~"PMC"~"506B PMC"~~1~"Study Zoster-062 to assess the safety, reactogenicity and immunogenicity of SHINGRIX in adults >=50 years of age with a prior episode of Herpes Zoster."~"Fulfilled"~~6/30/2025 0:00:00~12/18/2025 0:00:00
383292~"CBER"~"BLA"~125614.00~"GlaxoSmithKline Biologicals"~"SHINGRIX/Zoster Vaccine Recombinant, Adjuvanted"~10/20/2017 0:00:00~"Other"~344~"125614/344-1"~"PMC"~"506B PMC"~~1~"A targeted safety study, EPI-ZOSTER-030 VS (209452), to evaluate the 
safety of Shingrix in adults =50 years of age in the United States."~"Ongoing"~~3/31/2027 0:00:00~12/18/2025 0:00:00
383293~"CBER"~"BLA"~125614.00~"GlaxoSmithKline Biologicals"~"SHINGRIX/Zoster Vaccine Recombinant, Adjuvanted"~10/20/2017 0:00:00~"Other"~344~"125614/344-2"~"PMC"~"506B PMC"~~2~"A targeted safety study, EPI-ZOSTER-032 VS (209696), to evaluate the safety of Shingrix in adults =65 years of age in the United States."~"Ongoing"~~6/30/2027 0:00:00~12/18/2025 0:00:00
383294~"CBER"~"BLA"~125614.00~"GlaxoSmithKline Biologicals"~"SHINGRIX/Zoster Vaccine Recombinant, Adjuvanted"~10/20/2017 0:00:00~"Supplement"~398~"125614/398-1"~"PMC"~"506B PMC"~~1~"A cohort study EPI-ZOSTER-039 VS US DB to evaluate pregnancy and birth outcomes following vaccination with SHINGRIX in immunodeficient or immunosuppressed women between 18 and 49 years of age."~"Ongoing"~~12/31/2029 0:00:00~12/18/2025 0:00:00
383295~"CBER"~"BLA"~125819.00~"GlaxoSmithKline Biologicals"~"PENMENVY/Meningococcal Groups A, B, C, W, and Y Vaccine"~2/14/2025 0:00:00~"Original"~0~"125819/0-3"~"PMR"~"Pediatric Research Equity Act"~~3~"Deferred pediatric study under PREA (Study MENABCWY-027) to evaluate the safety and effectiveness of PENMENVY in children 2 years through 9 years of age."~"Pending"~"The clinical trial has not been initiated but does not meet the criterion for delayed."~9/30/2031 0:00:00~4/10/2026 0:00:00
383296~"CBER"~"BLA"~125819.00~"GlaxoSmithKline Biologicals"~"PENMENVY/Meningococcal Groups A, B, C, W, and Y Vaccine"~2/14/2025 0:00:00~"Original"~0~"125819/0-4"~"PMR"~"Pediatric Research Equity Act"~~4~"Deferred pediatric study under PREA (Study MENABCWY-026) to evaluate the safety and effectiveness of PENMENVY in infants and toddlers 6 weeks through 23 months of age."~"Pending"~"The clinical trial has not been initiated but does not meet the criterion for delayed."~11/30/2032 0:00:00~4/10/2026 0:00:00
383297~"CBER"~"BLA"~125819.00~"GlaxoSmithKline Biologicals"~"PENMENVY/Meningococcal Groups A, B, C, W, and Y Vaccine"~2/14/2025 0:00:00~"Original"~0~"125819/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric study under PREA (Study MENABCWY-022) to evaluate the safety and immunogenicity of PENMENVY in children 12 months through 9 years of age.
"~"Pending"~"The clinical trial has not been initiated but does not meet the criterion for delayed."~7/31/2027 0:00:00~4/10/2026 0:00:00
383298~"CBER"~"BLA"~125819.00~"GlaxoSmithKline Biologicals"~"PENMENVY/Meningococcal Groups A, B, C, W, and Y Vaccine"~2/14/2025 0:00:00~"Original"~0~"125819/0-5"~"PMC"~"506B PMC"~~5~"A Study titled  Assessment of Pregnancy and Birth Outcomes after Exposure to PENMENVY Vaccine in the U.S: A Cohort Study.  This postmarketing pregnancy safety study will use electronic health records to assess the incidence and risk of pregnancy outcomes in at least 50 women exposed to PENMENVY. The study design is a population-based cohort of publicly and commercially insured pregnant women nested within US electronic healthcare claims databases.
"~"Pending"~~2/28/2033 0:00:00~4/10/2026 0:00:00
383299~"CBER"~"BLA"~125819.00~"GlaxoSmithKline Biologicals"~"PENMENVY/Meningococcal Groups A, B, C, W, and Y Vaccine"~2/14/2025 0:00:00~"Original"~0~"125819/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA (Study MENACWY-MEN7B-003) to evaluate the safety and immunogenicity of PENMENVY in infants and toddlers approximately 2 months through 12 months of age (3-dose series administered at 2, 4, and 12 months of age).
"~"Submitted"~"The final report was submitted to FDA on April 28, 2026."~5/31/2026 0:00:00~4/10/2026 0:00:00
383300~"CBER"~"BLA"~125251.00~"Octapharma Pharmazeutika Produktionsges.m.b.H."~"Wilate/von Willebrand Factor/Coagulation Factor VIII Complex (Human)"~12/4/2009 0:00:00~"Supplement"~382~"125251/382-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA for the treatment of routine prophylaxis in pediatric patients with VWD ages 2 to less than 6 years of age.
"~"Fulfilled"~"This PMR was fulfilled on September 2, 2025"~12/31/2024 0:00:00~12/30/2024 0:00:00
383301~"CBER"~"BLA"~125416.00~"Octapharma Pharmazeutika Produktionsges.m.b.H."~"OCTAPLAS/Pooled Plasma (Human), Solvent/Detergent Treated"~1/17/2013 0:00:00~"Supplement"~216~"125416/216-1"~"PMC"~"506B PMC"~~1~~"Pending"~~~3/11/2022 0:00:00
383302~"CBER"~"BLA"~125416.00~"Octapharma Pharmazeutika Produktionsges.m.b.H."~"OCTAPLAS/Pooled Plasma (Human), Solvent/Detergent Treated"~1/17/2013 0:00:00~"Supplement"~216~"125416/216-2"~"PMC"~"506B PMC"~~2~~"Pending"~~~3/11/2022 0:00:00
383303~"CBER"~"BLA"~125416.00~"Octapharma Pharmazeutika Produktionsges.m.b.H."~"OCTAPLAS/Pooled Plasma (Human), Solvent/Detergent Treated"~1/17/2013 0:00:00~"Supplement"~216~"125416/216-3"~"PMC"~"506B PMC"~~3~~"Pending"~~~3/11/2022 0:00:00
383304~"CBER"~"BLA"~125416.00~"Octapharma Pharmazeutika Produktionsges.m.b.H."~"OCTAPLAS/Pooled Plasma (Human), Solvent/Detergent Treated"~1/17/2013 0:00:00~"Supplement"~216~"125416/216-4"~"PMC"~"506B PMC"~~4~~"Pending"~~~3/11/2022 0:00:00
383305~"CBER"~"BLA"~125587.00~"Octapharma Pharmazeutika Produktionsges.m.b.H."~"PANZYGA/Immune Globulin Intravenous (Human)-ifas"~8/2/2018 0:00:00~"Supplement"~70~"125587/70-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study studies under PREA for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) in pediatric patients ages 2 to 17 years of age. "~"Ongoing"~"Ongoing "~6/30/2025 0:00:00~9/10/2025 0:00:00
383306~"CBER"~"BLA"~125776.00~"Octapharma Pharmazeutika Produktionsges.m.b.H."~"Balfaxar/Prothrombin complex concentrate, human-lans"~7/21/2023 0:00:00~"Original"~0~"125776/0-1"~"PMR"~"505 (o)(3)"~~1~"A prospective, observational, cohort study to be conducted using electronic medical records (EMR) linked with administrative claims data, to assess occurrence of thromboembolic events after administration of BALFAXAR. The study will enroll minimum of 3,574 patients (with 1,787 patients exposed to BALFAXAR and 1,787 patients exposed to a comparator treatment)."~"Ongoing"~~6/30/2032 0:00:00~8/11/2025 0:00:00
383307~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Supplement"~6~"125817/6-1"~"PMC"~"506B PMC"~~1~"A Phase 3 Open-Label, Single-Arm Study (Study 2019nCoV-318) to Evaluate the Safety and Immunogenicity of an Omicron JN.1 Subvariant SARS-CoV-2 rS Vaccine (Version 3.0, dated June 9, 2025)."~"Pending"~~9/30/2026 0:00:00~6/30/2026 0:00:00
383308~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Supplement"~6~"125817/6-2"~"PMC"~"506B PMC"~~2~"A Phase IV, Observer-Blind, Placebo-controlled, Randomized Clinical Trial to Evaluate the Circulating Vaccine-derived Spike Protein in Adults 50   64 years of age with no history of PCVS or Long COVID, as Based on a Comprehensive Symptom Survey"~"Pending"~~5/31/2028 0:00:00~6/30/2026 0:00:00
383309~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Original"~0~"125817/0-10"~"PMC"~"506B PMC"~~10~"A study entitled  A Phase 4, Randomized, Double-Blind, Placebo-Controlled, Post-Marketing Study to Evaluate the Efficacy and Safety of a Subvariant SARS-CoV-2 rS Vaccine Adjuvanted with Matrix-M in Adults 50 to < 65 years of Age Without High-Risk Conditions for Severe COVID-19  to evaluate the clinical efficacy and safety and update the benefit-risk assessment of the intended marketed formulation of NUVAXOVID within the current epidemiological environment in a lower risk population aged 50 through 64 years.
"~"Pending"~~5/31/2027 0:00:00~6/30/2026 0:00:00
383310~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Original"~0~"125817/0-3"~"PMR"~"Pediatric Research Equity Act"~~3~"Deferred pediatric study under PREA (Study 2019nCoV-506) to evaluate the safety and immunogenicity of Nuvaxovid in COVID-19 vaccine-na ve individuals 0 to <6 months of age.
"~"Pending"~"The study has not been initiated but does not meet the criterion for delayed. "~10/31/2031 0:00:00~6/30/2026 0:00:00
383311~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Original"~0~"125817/0-6"~"PMR"~"505 (o)(3)"~~6~"Study 2019nCoV-418, entitled  Post-Authorization Safety Study to Evaluate Long-Term Sequalae of Myocarditis and Pericarditis Following Vaccination  to evaluate long-term sequelae of myocarditis and pericarditis following administration of NUVAXOVID with at least 5 years of follow-up."~"Pending"~~9/30/2032 0:00:00~6/30/2026 0:00:00
383312~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Original"~0~"125817/0-9"~"PMC"~"506B PMC"~~9~"Study 2019nCoV-404, entitled ""Safety Profile of the Novavax COVID-19 Vaccine, Adjuvanted in Individuals = 12 Years of Age in the United States"" to evaluate the occurrence of atrial fibrillation and cerebrovascular accident following administration of Novavax COVID-19 Vaccine, Adjuvanted or NUVAXOVID.
"~"Ongoing"~~9/30/2028 0:00:00~6/30/2026 0:00:00
383313~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Original"~0~"125817/0-4"~"PMR"~"505 (o)(3)"~~4~"Study 2019nCoV-402, entitled  Safety of the Novavax COVID-19 vaccine in England using a self-controlled case series design: A post-authorization safety study using data from the Clinical Practice Research Datalink (CPRD) Aurum and linked databases  to evaluate the occurrence of myocarditis and pericarditis following administration of Novavax COVID-19 Vaccine, Adjuvanted or NUVAXOVID.
We acknowledge the timetable you submitted on March 26, 2025, which states that you will conduct this study according to the following schedule:
"~"Ongoing"~~6/30/2028 0:00:00~6/30/2026 0:00:00
383314~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Original"~0~"125817/0-7"~"PMC"~"506B PMC"~~7~"Study 2019nCoV-405, entitled  Global Pregnancy and Infant Outcomes Study Using the COVID-19 Vaccines International Pregnancy Exposure Registry (C-VIPER)  to evaluate obstetric, neonatal, and infant outcomes among women vaccinated during pregnancy with Novavax COVID-19 Vaccine, Adjuvanted or NUVAXOVID.
"~"Ongoing"~~6/30/2027 0:00:00~6/30/2026 0:00:00
383315~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Original"~0~"125817/0-8"~"PMC"~"506B PMC"~~8~"Study 2019nCoV-402, entitled  Safety of the Novavax COVID-19 vaccine in England using a self-controlled case series design: A post-authorization safety study using data from the Clinical Practice Research Datalink (CPRD) Aurum and linked databases  to evaluate the occurrence of atrial fibrillation and cerebrovascular accident following administration of Novavax COVID-19 Vaccine, Adjuvanted or NUVAXOVID.
"~"Ongoing"~~6/30/2028 0:00:00~6/30/2026 0:00:00
383316~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Original"~0~"125817/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA (Study 2019nCoV-503) to evaluate the safety and immunogenicity of Nuvaxovid in COVID-19 vaccine-na ve individuals 6 months to <12 years of age."~"Submitted"~"The final report was submitted to FDA on May 24,2026."~5/22/2026 0:00:00~6/30/2026 0:00:00
383317~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Original"~0~"125817/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric study under PREA (Study 2019nCoV-317) to evaluate the immunogenicity of Nuvaxovid in COVID-19 vaccine-na ve seropositive individuals 2 years to <12 years of age and to evaluate the safety and immunogenicity of Nuvaxovid in individuals 6 months to <2 years of age, using a contemporaneously vaccinated comparator group."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed. "~7/31/2028 0:00:00~6/30/2026 0:00:00
383318~"CBER"~"BLA"~125817.00~"Sanofi Vaccines US Inc."~"NUVAXOVID/COVID-19 Vaccine, Adjuvanted"~5/16/2025 0:00:00~"Original"~0~"125817/0-5"~"PMR"~"505 (o)(3)"~~5~"Study 2019nCoV-404, entitled ""Safety Profile of the Novavax COVID-19 Vaccine, Adjuvanted in Individuals >= 12 Years of Age in the United States"" to evaluate the occurrence of myocarditis and pericarditis following administration of Novavax COVID-19 Vaccine, Adjuvanted or NUVAXOVID.
We acknowledge the timetable you submitted on March 26, 2025, which states that you will conduct this study according to the following schedule:
"~"Ongoing"~~9/30/2028 0:00:00~6/30/2026 0:00:00
383319~"CBER"~"BLA"~125682.00~"Sanofi Vaccines US Inc."~"DENGVAXIA/Dengue Tetravalent Vaccine, Live"~5/1/2019 0:00:00~"Original"~0~"125682/0-5"~"PMC"~"506B PMC"~~5~"To establish a pregnancy registry for DENGVAXIA in the United States to prospectively collect data on spontaneously reported exposures to DENGVAXIA at any time during pregnancy. You will submit annual reports as well as a 5-year summary report, after which you will continue enrolling patients in the registry and submitting annual reports pending CBER review of the reports and determination that the registry can be discontinued."~"Ongoing"~~12/31/2026 0:00:00~6/25/2026 0:00:00
383320~"CBER"~"BLA"~125701.00~"Sanofi Vaccines US Inc."~"MenQuadfi/Meningococcal (Groups A, C, Y, W) Conjugate Vaccine"~4/23/2020 0:00:00~"Original"~0~"125701/0-4"~"PMC"~"506B PMC"~~4~"To establish a pregnancy registry (MEQ00070) for MenQuadfi in the United States to collect and analyze the outcome of exposure to MenQuadfi during pregnancy and monitor for any potential safety signals that may arise in this population in routine public health settings."~"Ongoing"~~6/30/2029 0:00:00~6/17/2026 0:00:00
383321~"CBER"~"BLA"~125111.00~"Sanofi Vaccines Canada Ltd."~"ADACEL/Tetanus Toxoid, Reduced Diphtheria Toxoid and Acellular Pertussis Vaccine, Adsorbed"~6/10/2005 0:00:00~"Supplement"~904~"125111/904-1"~"PMC"~"506B PMC"~~1~"To conduct an observational safety cohort study using electronic health care data with linkage to offspring and access to clinical records in the US, to evaluate pregnancy outcomes in individuals exposed to Adacel. The population will be comprised of eligible pregnant individuals exposed to Adacel as of the 1st day of the 27th week of gestation or later and an active comparator of pregnant individuals not vaccinated with any Tdap vaccines during pregnancy. The study will include pre-defined pregnancy, birth and neonatal outcomes."~"Ongoing"~~12/31/2026 0:00:00~3/6/2026 0:00:00
383322~"CBER"~"BLA"~125419.00~"ID Biomedical Corporation of Quebec"~"AREPANRIX/Influenza A [H5] Monovalent Vaccine, Adjuvanted"~11/22/2013 0:00:00~"Other"~175~"125419/175-1"~"PMC"~"506B PMC"~~1~"Study 213268 An observational pregnancy safety study among pregnant women who use GlaxoSmithKline Biologicals SA s (GSK s) pandemic influenza vaccine AREPANRIX in the United States. Using a retrospective cohort design, the study will evaluate prespecified adverse pregnancy and birth/infant outcomes among pregnant women who receive AREPANRIX during pregnancy or within 28 days preceding conception."~"Pending"~~~1/16/2026 0:00:00
383323~"CBER"~"BLA"~125419.00~"ID Biomedical Corporation of Quebec"~"AREPANRIX/Influenza A [H5] Monovalent Vaccine, Adjuvanted"~11/22/2013 0:00:00~"Original"~0~"125419/0-4"~"PMR"~"Pediatric Research Equity Act"~~4~"Conduct study Q-Pan-025 under PREA to evaluate the safety and immunogenicity of Influenza A (H5N1) Virus Monovalent Vaccine, Adjuvanted when administered to healthy infants < 6 months of age. 
 

   
 
"~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~12/31/2022 0:00:00~1/16/2026 0:00:00
383324~"CBER"~"BLA"~125419.00~"ID Biomedical Corporation of Quebec"~"AREPANRIX/Influenza A [H5] Monovalent Vaccine, Adjuvanted"~11/22/2013 0:00:00~"Supplement"~166~"125419/166-1"~"PMR"~"Accelerated Approval"~~1~"Study 300790, entitled  A Phase 2, partially-blind, randomized, placebo-controlled, dose-confirmation study to evaluate the safety, reactogenicity and immunogenicity of AREPANRIX (Influenza A [H5] Virus Monovalent Vaccine, Adjuvanted) in healthy participants 6 months through 17 years of age. "~"Pending"~"Study is pending but does not meet the criterion for delayed."~~1/16/2026 0:00:00
383325~"CBER"~"BLA"~125419.00~"ID Biomedical Corporation of Quebec"~"AREPANRIX/Influenza A [H5] Monovalent Vaccine, Adjuvanted"~11/22/2013 0:00:00~"Original"~0~"125419/0-5"~"PMC"~"506B PMC"~~5~"Establish a pregnancy registry in the U.S. that is able to prospectively collect data on an actively recruited cohort to study the safety of Influenza A (H5N1) Virus Monovalent Vaccine, Adjuvanted during pregnancy.  A concept protocol for this pregnancy registry will be prepared under the assumption that the vaccine would be distributed to the general population in the U.S. in an officially-declared H5N1 influenza virus pandemic. Once the circumstances of the vaccine use in an officially-declared H5N1 influenza virus pandemic are determined by the U.S. Government, you will work with the FDA to finalize the protocol and you will initiate the registry."~"Released"~~~1/16/2026 0:00:00
383326~"CBER"~"BLA"~125874.00~"Regeneron Pharmaceuticals, Inc."~"OTARMENI/lunsotogene parvec-cwha"~4/23/2026 0:00:00~"Original"~0~"125874/0-1"~"PMR"~"Accelerated Approval"~~1~"Submit analyses of clinical outcomes in: 1) at least 30 lunsotogene parvec-cwha-treated pediatric patients (unilateral or bilateral treatment) enrolled in Study DB-OTO-001 with OTOF gene-associated sensorineural hearing loss after 104 weeks post product administration; including 2) at least 5 lunsotogene parvec-cwha newly treated patients 16 years of age or older, with OTOF associated sensorineural hearing loss (enrolled either within the same trial or in a separate trial). The analyses should evaluate at a minimum the following clinical outcomes: hearing sensitivity 
threshold by PTA, age-appropriate speech and language outcome measures, auditory brainstem response (ABR), global impression of improvement (GII) clinical scales, and quality of life (QoL) scales. 
These analyses should compare data from treated patients to comparable, untreated patients with the same diagnosis followed over a similar duration of follow up for purposes of verifying and describing the clinical benefit of lunsotogene parvec-cwha in pediatric and adult patients with OTOF gene-associated sensorineural hearing loss.
"~"Pending"~"Pending"~8/30/2030 0:00:00~
383327~"CBER"~"BLA"~125874.00~"Regeneron Pharmaceuticals, Inc."~"OTARMENI/lunsotogene parvec-cwha"~4/23/2026 0:00:00~"Original"~0~"125874/0-2"~"PMR"~"505 (o)(3)"~~2~"Conduct a prospective, longitudinal, observational study to characterize the long-term safety of lunsotogene parvec-cwha in a minimum of 50 patients, including patients from the ongoing Study DB-OTO-001. Propose and justify the number of adult patients to be enrolled in the study. All patients will be followed for a minimum of 10 years from product administration. The study will assess the incidence of serious adverse events including the risk of malignancy at pre-specified time intervals and assessments. In addition to annual PMC reports, interim study reports will be submitted to the BLA at 3 years and 6 years and 
should include, at least: cumulative counts of patients enrolled and their demographics, SAEs, deaths, all AEs, and malignancies; and for each SAE and malignancy, patient age and other demographics, descriptions of the event, time to onset after DB-OTO administration, diagnostic 
testing, diagnosis, treatment,and laboratory analysis (including insertional site testing).
"~"Pending"~~6/30/2040 0:00:00~
383328~"CBER"~"BLA"~103821.00~"Emergent BioDefense Operations Lansing LLC"~"BioThrax/Anthrax Vaccine Adsorbed"~11/12/1998 0:00:00~"Supplement"~5344~"103821/5344-1"~"PMR"~"Animal Efficacy"~~1~"Final Protocol Submission: November 30, 2016
Study/Trial Completion: To be determined should an event occur.
Final Report Submission: To be determined should an event occur."~"Pending"~~~
383329~"CBER"~"BLA"~125201.00~"CSL Behring AG"~"Privigen/Immune Globulin Intravenous (Human), 10% Liquid"~7/26/2007 0:00:00~"Supplement"~728~"125201/728-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) to improve neuromuscular disability and impairment and for maintenance therapy to prevent relapse in pediatric patients ages 2 to less than 17 years. The study will be a randomized study of single versus multiple Privigen dosing."~"Ongoing"~"Ongoing "~7/31/2023 0:00:00~9/19/2025 0:00:00
383330~"CBER"~"BLA"~125078.00~"CSL Behring LLC"~"Zemaira/Alpha-1-Proteinase Inhibitor (Human)"~7/8/2003 0:00:00~"Original"~0~"125078/0-5"~"PMC"~"506B PMC"~~5~"Based on the results of the pilot study and the available scientific data at the time that this study is being designed, ZLB Behring will work with entities maintaining registries of alpha1-proteinase inhibitor deficient patients and with the National Institutes of Health (NIH) to design and conduct an adequately-powered study of a clinically meaningful endpoint(s)."~"Delayed"~"The final protocol due date has passed with no final protocol received"~~9/5/2025 0:00:00
383331~"CBER"~"BLA"~125772.00~"CSL Behring LLC"~"HEMGENIX/etranacogene dezaparvovec-drlb"~11/22/2022 0:00:00~"Original"~0~"125772/0-2"~"PMR"~"505 (o)(3)"~~2~"A postmarketing study to assess the association between the serious risk of bleeding related to the failure of expected pharmacological action of HEMGENIX and pre-existing anti-AAV5 NAb to the capsid of HEMGENIX with a validated assay (required in PMR 1). The study will evaluate at least 35 Hemophilia B patients treated with HEMGENIX, to include at least 10 patients with high (1:1400 or higher) pre-treatment anti-AAV5 NAb titers. The assessment will compare pre- and post-treatment annualized bleeding rates (ABRs), with a lead-in period, to establish the patients baseline ABR on routine treatment, and 18-month follow-up after HEMGENIX administration"~"Ongoing"~~5/31/2029 0:00:00~1/16/2026 0:00:00
383332~"CBER"~"BLA"~125020.00~"MedImmune, LLC"~"FluMist; FluMist Quad/Influenza Vaccine Live, Intranasal"~6/17/2003 0:00:00~"Supplement"~2982~"125020/2982-1"~"PMC"~"506B PMC"~~1~"Real-world effectiveness of the live-attenuated influenza vaccine (LAIV) via home administration in individuals aged 2-49 years in the United States."~"Pending"~~10/31/2031 0:00:00~11/28/2025 0:00:00
383333~"CBER"~"BLA"~125506.00~"Bio Products Laboratory Limited"~"REPLAFACTEN; COAGADEX/Coagulation Factor X (Human)"~10/20/2015 0:00:00~"Original"~0~"125506/0-1"~"PMC"~"506B PMC"~~1~"A post-marketing registry study of perioperative management of moderate to severe hereditary factor X deficient patients receiving Coagadex (human factor X concentrate) for major surgical procedures."~"Ongoing"~~12/31/2021 0:00:00~12/20/2021 0:00:00
383334~"CBER"~"BLA"~125348.00~"Fibrocell Technologies, Inc."~"LaViv/Azficel-T"~6/21/2011 0:00:00~"Original"~0~"125348/0-1"~"PMR"~"505 (o)(3)"~~1~"Conduct a study, based on a registry design, to assess the risk of skin cancer in the area of azficel-T injections and the risk of immune-mediated hypersensitivity reactions in 2700 patients who receive azficel-T."~"Delayed"~" Azficel-T is no longer marketed and was added  to the CBER Discontinued Products List on May 19, 2017"~12/31/2016 0:00:00~7/10/2025 0:00:00
383335~"CBER"~"BLA"~125736.00~"Celgene Corporation, a Bristol-Myers Squibb Company"~"ABECMA/idecabtagene vicleucel"~3/26/2021 0:00:00~"Original"~0~"125736/0-1"~"PMR"~"505 (o)(3)"~~1~"A post-marketing, prospective, multi-center, observational study to assess the long-term safety of idecabtagene vicleucel and the risk of secondary malignancies occurring after treatment with idecabtagene vicleucel. The study will include at least 1500 adult patients with relapsed or refractory multiple myeloma after four or more prior lines of systemic therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody; the enrolled patients will be followed for 15 years after product administration"~"Ongoing"~~6/30/2042 0:00:00~5/21/2026 0:00:00
383336~"CBER"~"BLA"~125736.00~"Celgene Corporation, a Bristol-Myers Squibb Company"~"ABECMA/idecabtagene vicleucel"~3/26/2021 0:00:00~"Supplement"~357~"125736/357-1"~"PMR"~"505 (o)(3)"~~1~"Required to conduct an adequate leachables safety assessment for the ABECMA DP through its manufacturing process, storage, and in-use conditions. This assessment must include the following:

1. Assessment of leachables in the CryoStor CS10 and Plasma-Lyte A excipients in their original containers; and assessment of cumulative impact of the detected leachables in the reconstructed ABECMA DP leachables profile. 

2. Analytical assessment of leachables from a simulated leachables study by simulating the DP manufacturing process (without active ingredient, cells) from Unit Operation 8 through Unit Operation 11 and in-use conditions prior to administration to patients, taking into account maximum hold times and temperatures. The simulated DP manufactured material should be analyzed for leachables.

3. A full toxicological risk assessment for the identified leachables from the simulated leachables study.

"~"Pending"~~6/30/2025 0:00:00~5/21/2026 0:00:00
383337~"CBER"~"BLA"~125752.00~"ModernaTX, Inc. "~"SPIKEVAX/COVID-19 Vaccine, mRNA"~1/31/2022 0:00:00~"Supplement"~305~"125752/305-1"~"PMC"~"506B PMC"~~1~"Study mRNA-1273-P403 Subprotocol 03: A Phase 3b/4, Open-label, Single Arm Study to Evaluate the Immunogenicity and Safety of mRNA-1273.251 in Individuals =65 Years and =12 to <65 Years with Risk Factors for Severe COVID-19."~"Pending"~~10/31/2026 0:00:00~3/27/2026 0:00:00
383338~"CBER"~"BLA"~125752.00~"ModernaTX, Inc. "~"SPIKEVAX/COVID-19 Vaccine, mRNA"~1/31/2022 0:00:00~"Supplement"~305~"125752/305-2"~"PMC"~"506B PMC"~~2~"A Phase 4 Randomized, Observer Blind, Placebo-controlled Study to Evaluate the Safety of mRNA-1273 and mRNA-1283 Variant-containing Formulations in Adults 50 to 64 Years of Age without High-Risk Conditions for Severe COVID-19."~"Pending"~~1/31/2028 0:00:00~3/27/2026 0:00:00
383339~"CBER"~"BLA"~125752.00~"ModernaTX, Inc. "~"SPIKEVAX/COVID-19 Vaccine, mRNA"~1/31/2022 0:00:00~"Original"~0~"125752/0-3"~"PMR"~"Pediatric Research Equity Act"~~3~"Deferred pediatric study under PREA (Study mRNA-1273-P206) to evaluate the safety and effectiveness of SPIKEVAX in infants < 6 months of age."~"Ongoing"~"Deferral Extension Requested 6/5/2024. Deferral Extension Granted per FDA letter dated 9/13/2024."~6/30/2030 0:00:00~3/27/2026 0:00:00
383340~"CBER"~"BLA"~125752.00~"ModernaTX, Inc. "~"SPIKEVAX/COVID-19 Vaccine, mRNA"~1/31/2022 0:00:00~"Original"~0~"125752/0-6"~"PMR"~"505 (o)(3)"~~6~"Required safety study under Section 505(o) (Study mRNA-1273-P911), entitled ""Long-term outcomes of myocarditis following administration of SPIKEVAX (Moderna COVID-19, mRNA-1273),"" to evaluate long-term sequelae of myocarditis after vaccination with at least 5 years of follow-up."~"Ongoing"~~10/31/2028 0:00:00~3/27/2026 0:00:00
383341~"CBER"~"BLA"~125752.00~"ModernaTX, Inc. "~"SPIKEVAX/COVID-19 Vaccine, mRNA"~1/31/2022 0:00:00~"Original"~0~"125752/0-12"~"PMC"~"506B PMC"~~12~"Study mRNA-1273-P905, entitled ""Monitoring safety of Spikevax in pregnancy: an observational study using routinely collected health data in five European countries""."~"Submitted"~~12/31/2023 0:00:00~3/27/2026 0:00:00
383342~"CBER"~"BLA"~125752.00~"ModernaTX, Inc. "~"SPIKEVAX/COVID-19 Vaccine, mRNA"~1/31/2022 0:00:00~"Original"~0~"125752/0-5"~"PMR"~"505 (o)(3)"~~5~"Required safety study under Section 505(o) (Study mRNA-1273-P904), entitled ""Post-Authorization Active Surveillance Safety Study Using Secondary Data to Monitor Real-World Safety of Spikevax in Europe,"" to evaluate the occurrence of myocarditis and pericarditis following administration of SPIKEVAX."~"Fulfilled"~~12/31/2023 0:00:00~3/27/2026 0:00:00
383343~"CBER"~"BLA"~125752.00~"ModernaTX, Inc. "~"SPIKEVAX/COVID-19 Vaccine, mRNA"~1/31/2022 0:00:00~"Original"~0~"125752/0-10"~"PMC"~"506B PMC"~~10~"Study mRNA-1273-P901, entitled ""Real-World Study of the Effectiveness of Moderna COVID-19 Vaccine""."~"Fulfilled"~~4/14/2025 0:00:00~3/27/2026 0:00:00
383344~"CBER"~"BLA"~125752.00~"ModernaTX, Inc. "~"SPIKEVAX/COVID-19 Vaccine, mRNA"~1/31/2022 0:00:00~"Supplement"~276~"125752/276-1"~"PMC"~"506B PMC"~~1~"A Phase 4, Randomized, Observer-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of mRNA-1273 Variant-containing Formulation in Adults 50 to 64 Years of Age Without Risk Factors for Severe COVID-19."~"Pending"~~1/31/2027 0:00:00~3/27/2026 0:00:00
383345~"CBER"~"BLA"~125752.00~"ModernaTX, Inc. "~"SPIKEVAX/COVID-19 Vaccine, mRNA"~1/31/2022 0:00:00~"Other"~106~"125752/106-1"~"PMR"~"505 (o)(3)"~~1~"A Phase 4, Randomized, Observer-blind, Placebo-controlled,
Crossover Study to Assess Cardiac Troponin Levels in Participants
12 through 30 years of Age After mRNA-1273.712 Vaccine
(Study mRNA-1273-P404)."~"Submitted"~~6/30/2025 0:00:00~3/27/2026 0:00:00
383346~"CBER"~"BLA"~125752.00~"ModernaTX, Inc. "~"SPIKEVAX/COVID-19 Vaccine, mRNA"~1/31/2022 0:00:00~"Supplement"~220~"125752/220-1"~"PMC"~"506B PMC"~~1~"Study mRNA-1273-P403, Substudy 02, to evaluate immune responses following a single dose of SPIKEVAX (2024-2025 Formula) in previously vaccinated individuals 18 years of age and older.
"~"Fulfilled"~~5/31/2025 0:00:00~3/27/2026 0:00:00
383347~"CBER"~"BLA"~125796.00~"ModernaTX, Inc. "~"MRESVIA/Respiratory Syncytial Virus Vaccine"~5/31/2024 0:00:00~"Original"~0~"125796/0-3"~"PMR"~"Pediatric Research Equity Act"~~3~"Deferred pediatric study under PREA (mRNA-1345-P202) to evaluate the safety and immunogenicity of MRESVIA in healthy children 2 years to < 5 years of age, and children and adolescents at high risk of severe RSV disease 2 years to < 18 years of age."~"Submitted"~"The final study report was submitted to FDA on February 20, 2026."~12/31/2026 0:00:00~7/25/2025 0:00:00
383348~"CBER"~"BLA"~125796.00~"ModernaTX, Inc. "~"MRESVIA/Respiratory Syncytial Virus Vaccine"~5/31/2024 0:00:00~"Original"~0~"125796/0-4"~"PMR"~"Pediatric Research Equity Act"~~4~"Deferred pediatric study under PREA to evaluate the safety and immunogenicity of MRESVIA in infants and children 2 months to 24 months of age."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~3/31/2029 0:00:00~7/25/2025 0:00:00
383349~"CBER"~"BLA"~125796.00~"ModernaTX, Inc. "~"MRESVIA/Respiratory Syncytial Virus Vaccine"~5/31/2024 0:00:00~"Original"~0~"125796/0-5"~"PMR"~"Pediatric Research Equity Act"~~5~"Deferred pediatric study under PREA to evaluate the safety and efficacy of MRESVIA in infants and children 2 months to < 24 months of age."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~12/31/2031 0:00:00~7/25/2025 0:00:00
383350~"CBER"~"BLA"~125796.00~"ModernaTX, Inc. "~"MRESVIA/Respiratory Syncytial Virus Vaccine"~5/31/2024 0:00:00~"Original"~0~"125796/0-6"~"PMR"~"Pediatric Research Equity Act"~~6~"Deferred pediatric study under PREA to evaluate the safety and efficacy of MRESVIA in children 2 years to < 5 years of age who are healthy or at risk of severe RSV disease."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~12/31/2027 0:00:00~7/25/2025 0:00:00
383351~"CBER"~"BLA"~125796.00~"ModernaTX, Inc. "~"MRESVIA/Respiratory Syncytial Virus Vaccine"~5/31/2024 0:00:00~"Original"~0~"125796/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric study under PREA (mRNA-1365-P101) to evaluate the safety and immunogenicity of MRESVIA in infants and children 5 months to < 24 months of age."~"Ongoing"~"Study is Ongoing."~2/28/2028 0:00:00~7/25/2025 0:00:00
383352~"CBER"~"BLA"~125796.00~"ModernaTX, Inc. "~"MRESVIA/Respiratory Syncytial Virus Vaccine"~5/31/2024 0:00:00~"Original"~0~"125796/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA (mRNA-1345-P101) to evaluate the safety and immunogenicity of MRESVIA in RSV-seropositive children 12 months to < 60 months of age."~"Submitted"~"The final report was submitted to FDA on October 22, 2024."~12/31/2025 0:00:00~7/25/2025 0:00:00
383353~"CBER"~"BLA"~125835.00~"ModernaTX, Inc. "~"mNEXSPIKE/COVID-19 Vaccine, mRNA"~5/30/2025 0:00:00~"Supplement"~6~"125835/6-2"~"PMC"~"506B PMC"~~2~"A Phase 4 Randomized, Observer Blind, Placebo-controlled Study to Evaluate the Safety of mRNA-1273 and mRNA-1283 Variant-containing Formulations in Adults 50 to 64 Years of Age without High-Risk Conditions for Severe COVID-19."~"Pending"~~1/31/2028 0:00:00~
383354~"CBER"~"BLA"~125835.00~"ModernaTX, Inc. "~"mNEXSPIKE/COVID-19 Vaccine, mRNA"~5/30/2025 0:00:00~"Original"~0~"125835/0-4"~"PMR"~"505 (o)(3)"~~4~"A postmarketing retrospective cohort study using administrative claims and health system medical records to evaluate long-term sequelae of myocarditis following administration of MNEXSPIKE compared to myocarditis in patients who have not received a COVID-19 vaccine (Study mRNA-1283-P904). Participants will have at least five years of follow-up for long-term outcomes of myocarditis."~"Pending"~~3/31/2034 0:00:00~
383355~"CBER"~"BLA"~125835.00~"ModernaTX, Inc. "~"mNEXSPIKE/COVID-19 Vaccine, mRNA"~5/30/2025 0:00:00~"Other"~105~"125835/105-2"~"PMR"~"Pediatric Research Equity Act"~~2~"A Phase 3, Randomized, Observer-Blind, Active-Controlled Study to Evaluate the Safety, and Vaccine Effectiveness of mRNA-1283 in Children =6 months to <12 Years of Age, for the Prevention of COVID-19 (Study mRNA-1283-P30X)."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~2/28/2036 0:00:00~
383356~"CBER"~"BLA"~125835.00~"ModernaTX, Inc. "~"mNEXSPIKE/COVID-19 Vaccine, mRNA"~5/30/2025 0:00:00~"Original"~0~"125835/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA (mRNA-1283-P302) to evaluate the safety and effectiveness of MNEXSPIKE in infants and children 6 months to <12 years of age for the prevention of COVID-19."~"Released"~"Per FDA letter dated April 8, 2026, this PMR has been released"~6/30/2030 0:00:00~
383357~"CBER"~"BLA"~125835.00~"ModernaTX, Inc. "~"mNEXSPIKE/COVID-19 Vaccine, mRNA"~5/30/2025 0:00:00~"Original"~0~"125835/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric study under PREA (mRNA-1283-PXXX) to evaluate the safety and effectiveness of MNEXSPIKE in neonates and infants < 6 months of age for the prevention of COVID-19."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2035 0:00:00~
383358~"CBER"~"BLA"~125835.00~"ModernaTX, Inc. "~"mNEXSPIKE/COVID-19 Vaccine, mRNA"~5/30/2025 0:00:00~"Supplement"~6~"125835/6-1"~"PMC"~"506B PMC"~~1~"A Phase 3b/4, Open-label, Single Arm Study to Evaluate the Immunogenicity and Safety of mRNA-1283.251 in Individuals =65 Years and =12 to <65 Years with Risk Factors for Severe COVID-19 (mRNA-1283-P401 Subprotocol 01)."~"Pending"~~10/31/2026 0:00:00~
383359~"CBER"~"BLA"~125835.00~"ModernaTX, Inc. "~"mNEXSPIKE/COVID-19 Vaccine, mRNA"~5/30/2025 0:00:00~"Original"~0~"125835/0-3"~"PMR"~"505 (o)(3)"~~3~"A postmarketing retrospective cohort study utilizing commercial and Medicare claims databases to evaluate the occurrence of myocarditis and pericarditis following administration of MNEXSPIKE in the United States (Study mRNA-1283-P901)."~"Pending"~~9/30/2029 0:00:00~
383360~"CBER"~"BLA"~125835.00~"ModernaTX, Inc. "~"mNEXSPIKE/COVID-19 Vaccine, mRNA"~5/30/2025 0:00:00~"Original"~0~"125835/0-5"~"PMC"~"506B PMC"~~5~"An observational cohort study using administrative claims data to assess maternal and infant outcomes following exposure to MNEXSPIKE during pregnancy (Study mRNA-1283-P902)."~"Pending"~~5/31/2027 0:00:00~
383361~"CBER"~"BLA"~125835.00~"ModernaTX, Inc. "~"mNEXSPIKE/COVID-19 Vaccine, mRNA"~5/30/2025 0:00:00~"Original"~0~"125835/0-6"~"PMC"~"506B PMC"~~6~"A Phase 4, Randomized, Observer Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of mRNA-1283 Variant-containing Formulation in Adults 50 to 64 Years of Age Without High-Risk Conditions for Severe COVID-19. (Study mRNA-1283-XXXX)."~"Pending"~~5/31/2027 0:00:00~
383362~"CBER"~"BLA"~125835.00~"ModernaTX, Inc. "~"mNEXSPIKE/COVID-19 Vaccine, mRNA"~5/30/2025 0:00:00~"Other"~105~"125835/105-1"~"PMR"~"Pediatric Research Equity Act"~~1~"A Phase 2, Randomized, Observer-Blind, Dose Ranging Study to Evaluate the Safety and Immunogenicity of mRNA-1283 in Children >=6 Months to <12 Years of Age (Study mRNA-1283-P202)."~"Ongoing"~"Study is ongoing."~6/30/2029 0:00:00~
383363~"CBER"~"BLA"~125389.00~"ADMA Biologics, Inc."~"Bivigam/Immune Globulin Intravenous (Human)"~12/19/2012 0:00:00~"Original"~0~"125389/0-2"~"PMC"~"506B PMC"~~2~"Conduct a Prospective, non-interventional, active-control, observational safety study to further assess the potential risk of hypotension, hepatic and renal impairment in Biotest-IGIV-treated patients with primary humoral immunodeficiency (PI)."~"Ongoing"~~6/30/2021 0:00:00~2/15/2023 0:00:00
383364~"CBER"~"BLA"~125590.00~"ADMA Biologics, Inc."~"ASCENIV/Immune Globulin Intravenous, Human-slra"~4/1/2019 0:00:00~"Original"~0~"125590/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA for the treatment of primary immunodeficiency in pediatric patients ages 2 to 12 years."~"Ongoing"~"Deferral Extension Requested 03/29/2023. Deferral Extension Granted per FDA Letter dated 05/11/2023"~6/30/2023 0:00:00~5/30/2025 0:00:00
383365~"CBER"~"BLA"~125109.00~"Emergent BioSolutions Canada Inc."~"CNJ-016/Vaccinia Immune Globulin Intravenous (Human)"~5/2/2005 0:00:00~"Original"~0~"125109/0-1"~"PMR"~"Accelerated Approval"~~1~"Work with the FDA, and other public agencies as appropriate, to design and implement a clinical study protocol to include VIGIV dose ranging, and conduct a study to describe the treatment and clinical course of patients receiving VIGIV for complications of vaccinia infection when such a study is feasible, due to the actual or impending widespread use of VIGIV."~"Ongoing"~"As per the 180-day AA PMR Progress Report submitted on December 9, 2025, the study/trial is progressing as per the original schedule of milestones. As of November 1, 2025, 3 of 100 planned participants have been enrolled and completed the VA-005 clinical study. This report was found to be complete"~~6/24/2026 0:00:00
383366~"CBER"~"BLA"~125462.00~"Emergent BioSolutions Canada Inc."~"BAT/Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) - (Equine)"~3/22/2013 0:00:00~"Original"~0~"125462/0-1"~"PMR"~"Animal Efficacy"~~1~"You agree to re-initiate the BAT patient registry in the event of a mass botulism exposure scenario. The registry will evaluate the safety and clinical benefit of Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) - (Equine) used to treat adult, pediatric and infant patients with a confirmed or suspected exposure to botulism. "~"Pending"~~~5/5/2026 0:00:00
383367~"CBER"~"BLA"~125462.00~"Emergent BioSolutions Canada Inc."~"BAT/Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) - (Equine)"~3/22/2013 0:00:00~"Original"~0~"125462/0-3"~"PMC"~"506B PMC"~~3~"Conduct a pharmacokinetic (PK) study in pediatric patients to verify the pediatric dosing recommendations for BAT. "~"Ongoing"~~12/31/2017 0:00:00~5/5/2026 0:00:00
383368~"CBER"~"BLA"~125562.00~"Emergent BioSolutions Canada Inc."~"Anthrasil/Anthrax Immune Globulin Intravenous (Human)"~3/24/2015 0:00:00~"Original"~0~"125562/0-2"~"PMR"~"Animal Efficacy"~~2~"To submit annual progress reports as well as interim clinical summary reports
including available cumulative clinical and pharmacokinetic data every three
years from use of Anthrax Immune Globulin (Human) in sporadic systemic
anthrax cases (protocol AX-003B)."~"Ongoing"~~~5/1/2026 0:00:00
383369~"CBER"~"BLA"~125562.00~"Emergent BioSolutions Canada Inc."~"Anthrasil/Anthrax Immune Globulin Intravenous (Human)"~3/24/2015 0:00:00~"Original"~0~"125562/0-1"~"PMR"~"Animal Efficacy"~~1~"Conduct a field study (protocol AX-003A) to evaluate the efficacy,
pharmacokinetics, and safety of Anthrax Immune Globulin (Human) for the
treatment of toxemia associated with inhalational anthrax in adult and pediatric patients in combination with appropriate antibacterial drugs. The primary endpoint of this study will be all-cause mortality.
"~"Ongoing"~~~5/1/2026 0:00:00
383370~"CBER"~"BLA"~125761.00~"Emergent Product Development Gaithersburg, Inc."~"CYFENDUS/Anthrax Vaccine Adsorbed, Adjuvanted"~7/20/2023 0:00:00~"Original"~0~"125761/0-1"~"PMR"~"Animal Efficacy"~~1~"To conduct a field study to evaluate the clinical benefit and safety of CYFENDUS when administered in conjunction with recommended antibacterial drugs for post-exposure prophylaxis following a Bacillus anthracis mass exposure event. The study will be conducted as a Postmarketing Requirement (PMR) under regulations for products approved under the Animal Rule, 21 CFR 601.91(b)(1).
"~"Pending"~~12/31/2033 0:00:00~
383371~"CBER"~"BLA"~125820.00~"Bavarian Nordic A/S"~"VIMKUNYA/Chikungunya Vaccine, Recombinant"~2/14/2025 0:00:00~"Other"~50~"125820/50-2"~"PMR"~"Pediatric Research Equity Act"~~2~"PMR #2 Deferred pediatric study under PREA (EBSI-CV-317-009) to evaluate the safety and immunogenicity of VIMKUNYA in infants 0 to <1 years of age."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~11/30/2032 0:00:00~
383372~"CBER"~"BLA"~125820.00~"Bavarian Nordic A/S"~"VIMKUNYA/Chikungunya Vaccine, Recombinant"~2/14/2025 0:00:00~"Original"~0~"125820/0-4"~"PMC"~"506B PMC"~~4~"Study titled ""VIMKUNYA Pregnancy Registry: An observational prospective study of the safety of VIMKUNYA vaccine exposure in pregnant individuals and their offspring."" This prospective, observational registry study of pregnant individuals residing in the United States and European Union will evaluate maternal and infant outcomes (until one year of age) in at least 50 individuals exposed to VIMKUNYA up to 28 days prior to or at any time during pregnancy.
"~"Pending"~~2/28/2031 0:00:00~
383373~"CBER"~"BLA"~125820.00~"Bavarian Nordic A/S"~"VIMKUNYA/Chikungunya Vaccine, Recombinant"~2/14/2025 0:00:00~"Original"~0~"125820/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric study under PREA (EBSI-CV-317-006) to evaluate the safety and immunogenicity of VIMKUNYA in children 2 to <12 years of age.
"~"Released"~"Per FDA letter dated January 8, 2026, this PMR has been released."~1/31/2029 0:00:00~
383374~"CBER"~"BLA"~125820.00~"Bavarian Nordic A/S"~"VIMKUNYA/Chikungunya Vaccine, Recombinant"~2/14/2025 0:00:00~"Original"~0~"125820/0-3"~"PMR"~"Pediatric Research Equity Act"~~3~"Deferred pediatric study under PREA (EBSI-CV-317-009) to evaluate the safety and immunogenicity of VIMKUNYA in infants 0 to <2 years of age.
"~"Released"~"Per FDA letter dated January 8, 2026, this PMR has been released."~11/30/2032 0:00:00~
383375~"CBER"~"BLA"~125820.00~"Bavarian Nordic A/S"~"VIMKUNYA/Chikungunya Vaccine, Recombinant"~2/14/2025 0:00:00~"Other"~50~"125820/50-1"~"PMR"~"Pediatric Research Equity Act"~~1~"PMR #1 Deferred pediatric study under PREA (EBSI-CV-317-006) to evaluate the safety and immunogenicity of VIMKUNYA in children 1 to <12 years of age."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~1/31/2029 0:00:00~
383376~"CBER"~"BLA"~125820.00~"Bavarian Nordic A/S"~"VIMKUNYA/Chikungunya Vaccine, Recombinant"~2/14/2025 0:00:00~"Original"~0~"125820/0-1"~"PMR"~"Accelerated Approval"~~1~"To conduct a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and immunogenicity of VIMKUNYA, an adjuvanted chikungunya virus virus-like particle (CHIKV VLP) vaccine for the prevention of chikungunya disease in adolescents (12 to <18 years) and adults (>=18 years).
"~"Ongoing"~"The study is ongoing."~8/31/2030 0:00:00~
383377~"CBER"~"BLA"~125714.00~"Juno Therapeutics, Inc. a Bristol-Myers Squibb Company"~"BREYANZI/lisocabtagene maraleucel"~2/5/2021 0:00:00~"Supplement"~644~"125714/644-1"~"PMR"~"505 (o)(3)"~~1~"A postmarketing, multicenter, prospective, observational study to assess the long-term safety and the risk of secondary malignancies occurring after treatment with lisocabtagene maraleucel. The study will include at least 300 adult patients with relapsed or refractory marginal zone lymphoma (MZL); the enrolled patients will be followed for 15 years after the product administration.
"~"Pending"~~3/31/2049 0:00:00~4/3/2026 0:00:00
383378~"CBER"~"BLA"~125714.00~"Juno Therapeutics, Inc. a Bristol-Myers Squibb Company"~"BREYANZI/lisocabtagene maraleucel"~2/5/2021 0:00:00~"Supplement"~205~"125714/205-2"~"PMR"~"505 (o)(3)"~~2~"A post-marketing, multicenter, prospective, observational study to assess the long-term safety of lisocabtagene maraleucel and the risk of secondary malignancies occurring after treatment with lisocabtagene maraleucel. The study will include at least 300 subjects with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL); the enrolled patients will be followed for 15 years after the product administration"~"Ongoing"~~6/30/2045 0:00:00~4/3/2026 0:00:00
383379~"CBER"~"BLA"~125714.00~"Juno Therapeutics, Inc. a Bristol-Myers Squibb Company"~"BREYANZI/lisocabtagene maraleucel"~2/5/2021 0:00:00~"Supplement"~227~"125714/227-1"~"PMR"~"505 (o)(3)"~~1~"A postmarketing, multicenter, prospective, observational study to assess the long-term safety and risk of secondary malignancies occurring after treatment with lisocabtagene maraleucel. The study will include at least 300 patients with relapsed or refractory mantle cell lymphoma (MCL); the enrolled patients will be followed for 15 years after the product administration."~"Ongoing"~~9/30/2045 0:00:00~4/3/2026 0:00:00
383380~"CBER"~"BLA"~125714.00~"Juno Therapeutics, Inc. a Bristol-Myers Squibb Company"~"BREYANZI/lisocabtagene maraleucel"~2/5/2021 0:00:00~"Supplement"~225~"125714/225-2"~"PMR"~"505 (o)(3)"~~2~"A postmarketing, multicenter, prospective, observational study to assess the long-term safety and risk of secondary malignancies occurring after treatment with lisocabtagene maraleucel. The study will include at least 300 patients with relapsed or refractory follicular lymphoma; the enrolled patients will be followed for 15 years after the product administration."~"Pending"~~8/31/2045 0:00:00~4/3/2026 0:00:00
383381~"CBER"~"BLA"~125714.00~"Juno Therapeutics, Inc. a Bristol-Myers Squibb Company"~"BREYANZI/lisocabtagene maraleucel"~2/5/2021 0:00:00~"Original"~0~"125714/0-1"~"PMR"~"505 (o)(3)"~~1~"A post-marketing multicenter, prospective, observational study to assess the long-term safety of lisocabtagene maraleucel and the risk of secondary malignancies occurring after treatment with lisocabtagene maraleucel. The study will include at least 1500 adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy; the enrolled patients will be followed for 15 years after the product administration. 

"~"Ongoing"~~4/30/2041 0:00:00~4/3/2026 0:00:00
383382~"CBER"~"BLA"~125714.00~"Juno Therapeutics, Inc. a Bristol-Myers Squibb Company"~"BREYANZI/lisocabtagene maraleucel"~2/5/2021 0:00:00~"Supplement"~205~"125714/205-1"~"PMR"~"Accelerated Approval"~~1~"Conduct a single arm study of lisocabtagene maraleucel in patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least 2 prior lines of therapy, including a prior Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor, to evaluate overall response rate and durability. The study must include a total of 50 treated patients
and durable response should be based on a minimum follow-up of 15 months after first objective disease response.
"~"Ongoing"~"As per the 180-day AA PMR Progress Report submitted on October 1, 2025, the study/trial is ongoing and according to schedule."~5/31/2027 0:00:00~4/3/2026 0:00:00
383383~"CBER"~"BLA"~125714.00~"Juno Therapeutics, Inc. a Bristol-Myers Squibb Company"~"BREYANZI/lisocabtagene maraleucel"~2/5/2021 0:00:00~"Supplement"~225~"125714/225-1"~"PMR"~"Accelerated Approval"~~1~"Collect and submit the final report, including datasets from the TRANSCEND FL clinical trial (NCT04245839) to verify and describe the clinical benefit of lisocabtagene maraleucel (BREYANZI) in adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy (including an anti-CD20 antibody and an alkylating agent). All partial and complete responders should have completed at least 24 months of follow up starting from the initial objective response."~"Fulfilled"~"This AA PMR was fulfilled as per efficacy supplement approved Feb 20, 2026."~8/31/2025 0:00:00~4/3/2026 0:00:00
383384~"CBER"~"BLA"~125758.00~"Orchard Therapeutics (Europe) Ltd."~"LENMELDY/atidarsagene autotemcel"~3/18/2024 0:00:00~"Original"~0~"125758/0-1"~"PMR"~"505 (o)(3)"~~1~"A postmarketing, prospective, observational, study to assess and characterize the risk of secondary malignancies, and long-term safety following treatment with atidarsagene autotemcel (OTL-200-12). This study will enroll a minimum of 17 subjects. The enrolled patients will be followed for 15 years after product administration."~"Pending"~~12/31/2044 0:00:00~5/14/2026 0:00:00
383385~"CBER"~"BLA"~125758.00~"Orchard Therapeutics (Europe) Ltd."~"LENMELDY/atidarsagene autotemcel"~3/18/2024 0:00:00~"Original"~0~"125758/0-2"~"PMR"~"505 (o)(3)"~~2~"An adequate leachables safety assessment for the OTL-200 drug product (DP) through its manufacturing process, storage, and in-use conditions. This assessment must include the following:
Assessment of elemental extractables from relevant DP manufacturing/ storage components, and both elemental and organic leachables (i.e., cumulative) in the final DP.
b. The leachables study can be conducted by simulating the DP manufacturing process from the step with high-risk for leachables components, should be conducted with all operations performed using maximal hold times and temperatures at respective steps, and continue through the product freezing, shelf-life storage, thawing, and in-use processing.
c. This evaluation will also include a full toxicological risk assessment for the identified leachables."~"Pending"~~9/30/2025 0:00:00~5/14/2026 0:00:00
383386~"CBER"~"BLA"~125787.00~"Vertex Pharmaceuticals Inc"~"Casgevy/exagamglogene autotemcel"~12/8/2023 0:00:00~"Supplement"~57~"125787/57-5"~"PMR"~"505 (o)(3)"~~5~"A postmarketing, prospective, multi-center, observational study, to assess and characterize the risks of secondary malignancies and off-target effects following genome editing occurring after treatment with exagamglogene autotemcel, and to assess the long-term safety of exagamglogene autotemcel. The study will include 150 patients with transfusion dependent beta thalassemia (TDT) who received/will receive exagamglogene autotemcel, and each enrolled patient will be followed for 15 years after product administration. The study design will include monitoring (at pre-specified intervals) with adequate testing strategies (Study Protocol VX22-290-101)."~"Ongoing"~~12/31/2043 0:00:00~2/6/2026 0:00:00
383387~"CBER"~"BLA"~125787.00~"Vertex Pharmaceuticals Inc"~"Casgevy/exagamglogene autotemcel"~12/8/2023 0:00:00~"Original"~0~"125787/0-1"~"PMR"~"505 (o)(3)"~~1~" postmarketing, prospective, multicenter observational study to assess and characterize the risks of secondary malignancies and off-target effects following genome editing occurring after treatment with exagamglogeneautotemcel, and to assess the long-term safety of exagamglogene autotemcel. The study will include 250 subjects with SCD who received/will receive exagamglogene autotemcel, and each enrolled subject will be followed for 15 years after product administration. The study design will include monitoring (at prespecified intervals) with adequate testing strategies (Study Protocol VX22-290-101).
"~"Ongoing"~~12/31/2043 0:00:00~2/6/2026 0:00:00
383388~"CBER"~"BLA"~125787.00~"Vertex Pharmaceuticals Inc"~"Casgevy/exagamglogene autotemcel"~12/8/2023 0:00:00~"Original"~0~"125787/0-2"~"PMR"~"505 (o)(3)"~~2~"Conduct studies to comprehensively assess and screen for the impact of 
sequence heterogeneity on the risk of off-target editing in the patient population for exagamglogene autotemcel. Specifically, 
i. Perform a new in silico off-target analysis using publicly available 
databases/datasets to allow for inclusion of more variants. 

Specifically, perform the analysis using all variants with at least 0.5% 
allele frequency in at least one of the five continental groups (Africa, 
Europe, East Asia, South Asia, and the Americas). 
ii. Perform confirmatory testing, as appropriate and feasible, of all the offtarget loci nominated from the new in silico analysis from (i) as well as 
those that were not accounted for in the previous study using 
appropriate samples harboring variants. 
a. Screen for the presence of all previously identified variants 
(e.g., CPS1) as well as any variants identified in study (i) and 
(ii) in the patients treated in Studies 121, 111, 141, 151, 161, 
and 171. 
b. For patients with a confirmed variant(s), assess for indels and 
chromosomal changes at each respective locus in appropriate 
samples. 
"~"Ongoing"~~6/30/2032 0:00:00~2/6/2026 0:00:00
383389~"CBER"~"BLA"~102475.00~"Grifols Biologicals LLC"~"Alphanate/Antihemophilic Factor/von Willebrand Factor Complex (Human)"~8/15/1978 0:00:00~"Supplement"~1033~"102475/1033-1"~"PMC"~"506B PMC"~~1~"Conduct a study to evaluate the immunogenic potential of the terminal heat-treated product."~"Fulfilled"~~2/15/2011 0:00:00~10/30/2024 0:00:00
383390~"CBER"~"BLA"~125640.00~"Instituto Grifols, S.A."~"VISTASEAL/Fibrin Sealant (Human)"~11/1/2017 0:00:00~"Original"~0~"125640/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Instituto Grifols, S.A. commits to evaluating the safety and efficacy of FIBRIN SEALANT (Human) as an adjunct to hemostasis during surgery in pediatric patients < 18 years of age in the deferred pediatric clinical trial under protocol IG1405 entitled ""A Prospective, Randomized, Active-Controlled, Single-blind, Parallel Group Clinical Trial to Evaluate the Safety and Efficacy of Fibrin Sealant Grifols (FS Grifols) as an Adjunct to Haemostasis during Surgery in Paediatric Subjects.""  Instituto Grifols, S.A. also commits to conducting a study of the Human Factors assessment as part of the pediatric trial. "~"Ongoing"~"Study is ongoing."~6/30/2024 0:00:00~12/20/2023 0:00:00
383391~"CBER"~"BLA"~125611.00~"Novo Nordisk Inc."~"REBINYN/Coagulation Factor IX (Recombinant), GlycoPEGylated"~5/31/2017 0:00:00~"Supplement"~223~"125611/223-1"~"PMC"~"506B PMC"~~1~"A postmarketing, prospective, observational cohort study of at least 30 patients of all ages with Hemophilia B treated with Rebinyn for routine prophylaxis to assess the long-term safety, including central nervous system (CNS) adverse events with detailed assessments of neurologic and neurocognitive function, and pediatric growth and development. Each subject will be followed for at least 5 years after initiating Rebinyn for routine prophylaxis."~"Released"~~12/31/2031 0:00:00~6/26/2025 0:00:00
383392~"CBER"~"BLA"~125646.00~"Novartis Pharmaceuticals Corporation"~"KYMRIAH/tisagenlecleucel"~8/30/2017 0:00:00~"Supplement"~663~"125646/663-2"~"PMR"~"505 (o)(3)"~~2~"A post marketing, prospective, multi-center, observational study to assess the long-term safety of tisagenlecleucel and the risk of secondary malignancies occurring after treatment with tisagenlecleucel. The study will include at least 300 adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy; the enrolled patients will be followed for 15 years after product administration."~"Ongoing"~~9/30/2043 0:00:00~10/23/2025 0:00:00
383393~"CBER"~"BLA"~125646.00~"Novartis Pharmaceuticals Corporation"~"KYMRIAH/tisagenlecleucel"~8/30/2017 0:00:00~"Supplement"~76~"125646/76-1"~"PMR"~"505 (o)(3)"~~1~"A post-marketing, prospective, multi-center, observational study to assess the long-term safety of tisagenlecleucel and the risk of all secondary malignancies occurring after treatment with tisagenlecleucel. The study will include at least 1500 patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma. The enrolled patients will be followed for 15 years after the product administration."~"Ongoing"~~12/31/2038 0:00:00~10/23/2025 0:00:00
383394~"CBER"~"BLA"~125646.00~"Novartis Pharmaceuticals Corporation"~"KYMRIAH/tisagenlecleucel"~8/30/2017 0:00:00~"Original"~0~"125646/0-1"~"PMR"~"505 (o)(3)"~~1~"A post-marketing, prospective, multi-center, observational study to assess the long-term safety of tisagenlecleucel and the risk of all secondary malignancies occurring after treatment with tisagenlecleucel. The study will include at least 1000 pediatric and young adult patients with relapsed / refractory B cell acute lymphoblastic leukemia; the enrolled patients will be followed for 15 years after the product administration."~"Ongoing"~~12/31/2038 0:00:00~10/23/2025 0:00:00
383395~"CBER"~"BLA"~125646.00~"Novartis Pharmaceuticals Corporation"~"KYMRIAH/tisagenlecleucel"~8/30/2017 0:00:00~"Supplement"~663~"125646/663-1"~"PMR"~"Accelerated Approval"~~1~"Conduct a randomized phase 3 trial in adult patients with relapsed or refractory follicular lymphoma. Patients will be randomized to tisagenlecleucel or an investigator choice of regimens consistent with the standard of care. The primary endpoint will be progression-free survival with secondary endpoints that include overall survival and objective response rate.
"~"Pending"~"Pending"~9/30/2028 0:00:00~10/23/2025 0:00:00
383396~"CBER"~"BLA"~125508.00~"Merck Sharp & Dohme LLC"~"Gardasil 9/Human Papillomavirus 9-valent Vaccine, Recombinant"~12/10/2014 0:00:00~"Original"~0~"125508/0-2"~"PMC"~"506B PMC"~~2~"To conduct a 10-year study extension of Protocol V503-001 to evaluate the long-term safety, immunogenicity and effectiveness of Gardasil 9 in women who were 16 to 26 years of age at enrollment."~"Submitted"~~12/31/2026 0:00:00~1/27/2025 0:00:00
383397~"CBER"~"BLA"~125508.00~"Merck Sharp & Dohme LLC"~"Gardasil 9/Human Papillomavirus 9-valent Vaccine, Recombinant"~12/10/2014 0:00:00~"Supplement"~868~"125508/868-1"~"PMR"~"Accelerated Approval"~~1~"To conduct Study V503-049 to evaluate the efficacy of a three-dose regimen of GARDASIL9 in the prevention of oral persistent infection with HPV types 16, 18, 31, 33, 45, 52 or 58 in men 20 through 45 years of age."~"Delayed"~"As per the 180-day AA PMR Progress Report submitted on February 5, 2026, the trial is behind the original schedule because notification of Study Completion has not been submitted to the BLA. There are currently 6033 participants enrolled of the planned number of 6000. This report was found to be complete."~9/30/2026 0:00:00~1/27/2025 0:00:00
383398~"CBER"~"BLA"~125814.00~"Merck Sharp & Dohme LLC"~"CAPVAXIVE/Pneumococcal 21-valent Conjugate Vaccine"~6/17/2024 0:00:00~"Original"~0~"125814/0-1"~"PMR"~"Accelerated Approval"~~1~"To conduct an observational hybrid study using both primary and secondary data collection with a test-negative case-control design with the objective of assessing the effectiveness of CAPVAXIVE in preventing hospitalized, confirmed community acquired pneumonia (CAP, invasive and non-invasive) caused by S. pneumoniae serotypes 3, 6A, 7F, 8, 9N, 10A, 11A, 12F, 15A, 15C, 16F, 17F, 19A, 20A, 22F, 23A, 23B, 24F, 31, 33F, and 35B in individuals =65 years of age. The clinical protocol (V116-011-00) is entitled ""A Test-Negative Case-Control Study to Evaluate the Effectiveness of V116 Against Pneumococcal Pneumonia in Older Adults"""~"Ongoing"~"As per the 180-day AA PMR Progress Report submitted on 2/12/2026, the study is progressing as per the original schedule of milestones. There are currently 9/22,000 planned participants enrolled. This report was found to be complete."~12/31/2029 0:00:00~
383399~"CBER"~"BLA"~125814.00~"Merck Sharp & Dohme LLC"~"CAPVAXIVE/Pneumococcal 21-valent Conjugate Vaccine"~6/17/2024 0:00:00~"Original"~0~"125814/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Deferred pediatric study under PREA to evaluate the safety and immunogenicity of CAPVAXIVE in pediatric patients ages 2 to <18 years."~"Fulfilled"~"Per FDA letter dated June 17, 2026, this PMR has been fulfilled."~12/31/2026 0:00:00~
383400~"CBER"~"BLA"~125549.00~"Wyeth Pharmaceuticals LLC"~"TRUMENBA/Meningococcal Group B Vaccine"~10/29/2014 0:00:00~"Other"~897~"125549/897-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study (B1971067) under PREA to evaluate the safety and immunogenicity of a 3-dose (0, 1-2, and 6 months) regimen of TRUMENBA in children 1 year to less than 10 years of age for the prevention of invasive group B meningococcal disease."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~5/31/2027 0:00:00~12/23/2025 0:00:00
383401~"CBER"~"BLA"~125549.00~"Wyeth Pharmaceuticals LLC"~"TRUMENBA/Meningococcal Group B Vaccine"~10/29/2014 0:00:00~"Original"~0~"125549/0-4"~"PMR"~"Pediatric Research Equity Act"~~4~"A deferred pediatric study (B1971035) under PREA to evaluate the safety and immunogenicity of Trumenba in children 12 months to less than 24 months of age for the prevention of invasive group B meningococcal disease."~"Submitted"~"The final report was submitted to FDA on March 15, 2018."~8/30/2017 0:00:00~12/23/2025 0:00:00
383402~"CBER"~"BLA"~125549.00~"Wyeth Pharmaceuticals LLC"~"TRUMENBA/Meningococcal Group B Vaccine"~10/29/2014 0:00:00~"Original"~0~"125549/0-3"~"PMR"~"Pediatric Research Equity Act"~~3~"A deferred pediatric study (B1971017) under PREA to evaluate the safety and immunogenicity of Trumenba in children 2 years to less than 10 years of age for the prevention of invasive group B meningococcal disease."~"Submitted"~"The final report was submitted to FDA on 11 Oct 2017."~2/28/2017 0:00:00~12/23/2025 0:00:00
383403~"CBER"~"BLA"~125731.00~"Wyeth Pharmaceuticals LLC"~"PREVNAR 20/Pneumococcal 20-valent Conjugate Vaccine"~6/8/2021 0:00:00~"Original"~0~"125731/0-1"~"PMR"~"Accelerated Approval"~~1~"To conduct a study to assess the effectiveness of Prevnar 20 in adults = 65 years of age in preventing community acquired pneumococcal pneumonia caused by pneumococcal serotypes 8, 10A, 11A, 12F, 15B 22F and 33F in the vaccine. The original protocol for this study entitled ""A Phase 4 Study Using a Test-Negative Design to Evaluate the Effectiveness of a 20-valent Pneumococcal Conjugate Vaccine Against Vaccine-Type Radiologically-Confirmed Community Acquired Pneumonia in Adults = 65 years of age"" (Study B7471015) was submitted on January 15, 2021."~"Ongoing"~"As per the 180-day AA PMR Progress Report submitted on February 4, 2026, the study/trial is progressing as per the original schedule of milestones. There are currently 11,018 of 17,500 planned participants enrolled. This report was found to be complete."~11/30/2027 0:00:00~8/2/2023 0:00:00
383404~"CBER"~"BLA"~125696.00~"Greer Laboratories, Inc."~"PALFORZIA/Peanut (Arachis hypogaea) Allergen Powder"~1/31/2020 0:00:00~"Original"~0~"125696/0-2"~"PMC"~"506B PMC"~~2~"To establish a pregnancy registry for PALFORZIA to collect, analyze, and report data on pregnancy outcomes and infant outcomes after exposure of PALFORZIA during pregnancy. You will continue enrolling patients until 72 qualifying patients are enrolled or until 5 years after PALFORZIA is commercially available, whichever is first. You will submit annual reports as well as a summary report at the end of the study, after which you will continue enrolling patients in the registry pending CBER review of the report and determination of whether the registry can be discontinued."~"Ongoing"~~1/30/2026 0:00:00~3/17/2025 0:00:00
383405~"CBER"~"BLA"~125822.00~"Kedrion, S.p.A."~"QIVIGY/Immune Globulin Intravenous, Human-kthm, 10% Solution"~9/26/2025 0:00:00~"Original"~0~"125822/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA for the treatment of primary immune deficiency in pediatric patients ages 2 years to less than 17 years."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~7/30/2026 0:00:00~
383406~"CBER"~"BLA"~125400.00~"Organogenesis, Inc."~"GINTUIT/Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen"~3/9/2012 0:00:00~"Original"~0~"125400/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Assess the safety and effectiveness of Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen for topical (non-submerged) application to a surgically created vascular wound bed in the treatment of mucogingival disorders in pediatric patients ages 12 to 18 years."~"Delayed"~"Deferral Extension Requested 08/17/2017, and received 08/21/2017. Denied per FDA letter dated October 5, 2017. "~3/31/2017 0:00:00~5/15/2014 0:00:00
383407~"CBER"~"BLA"~125746.00~"Janssen Biotech, Inc."~"CARVYKTI/ciltacabtagene autoleucel"~2/28/2022 0:00:00~"Supplement"~74~"125746/74-1"~"PMC"~"506B PMC"~~1~"Submit the final overall survival report and datasets for the CARTITUDE-NCT04181827 clinical trial, titled ""A Phase 3 Study Comparing JNJ-68284528, a Chimeric Antigen Receptor T (CAR T) Cell Therapy Directed Against B-cell Maturation Antigen (BCMA), versus Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd) in Participants With Relapsed and Lenalidomide-Refractory Multiple Myeloma."""~"Ongoing"~~2/29/2032 0:00:00~4/22/2026 0:00:00
383408~"CBER"~"BLA"~125746.00~"Janssen Biotech, Inc."~"CARVYKTI/ciltacabtagene autoleucel"~2/28/2022 0:00:00~"Original"~0~"125746/0-1"~"PMR"~"505 (o)(3)"~~1~"A post-marketing, prospective, multi-center, observational study to assess the long-term safety of ciltacabtagene autoleucel and the risk of secondary malignancies occurring after treatment with ciltacabtagene autoleucel. The study will include at least 1500 adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody; the enrolled patients will be followed for 15 years after product administration"~"Ongoing"~~6/30/2042 0:00:00~4/22/2026 0:00:00
383409~"CBER"~"BLA"~103174.00~"Grifols Therapeutics LLC"~"Prolastin; Prolastin-C; Prolastin-C Liquid/Alpha-1-Proteinase Inhibitor (Human)"~12/2/1987 0:00:00~"Supplement"~5520~"103174/5520-1"~"PMC"~"506B PMC"~~1~"Conduct two clinical trials:  Trial 1:  A pilot study of the safety and pharmacokinetics of Prolastin-C administered weekly at a dose of 120 mg/kg.  Trial 2:  An adequately-powered study of clinically meaningful endpoints -Prolastin-C efficacy study using CT scans as the primary endpoint."~"Delayed"~"The  study completion date has passed, and the study has not yet been completed. 
"~5/18/2028 0:00:00~2/27/2025 0:00:00
383410~"CBER"~"BLA"~125392.00~"Ethicon, Inc."~"EVARREST/Fibrin Sealant Patch"~12/5/2012 0:00:00~"Supplement"~163~"125392/163-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA for use with manual compression as an adjunct to hemostasis in pediatric patients aged 1 month to less than 18 years undergoing surgery, when control of bleeding by conventional surgical techniques (such as suture, ligature, and cautery) is ineffective or impractical. "~"Submitted"~"Ongoing "~12/31/2025 0:00:00~2/3/2026 0:00:00
383411~"CBER"~"BLA"~125566.00~"Takeda Pharmaceuticals U.S.A., Inc."~"ADYNOVATE/Antihemophilic Factor (Recombinant), PEGylated"~11/13/2015 0:00:00~"Original"~0~"125566/0-7"~"PMC"~"506B PMC"~~7~"Conduct ""A phase 3, multi-center, open label study to investigate safety and immunogenicity of ADYNOVATE in previously untreated patients(PUPs)"" [clinical study 261203]. This study will evaluate on-demand treatment andcontrol of bleeding episodes in the setting of routine prophylaxis to reduce the frequencyof bleeding episodes, as well as the perioperative management of bleeding.

"~"Fulfilled"~~9/30/2023 0:00:00~10/9/2025 0:00:00
383412~"CBER"~"BLA"~125512.00~"Takeda Pharmaceuticals U.S.A., Inc."~"OBIZUR/Antihemophilic Factor (Recombinant), Porcine Sequence"~10/23/2014 0:00:00~"Original"~0~"125512/0-1"~"PMC"~"506B PMC"~~1~"To collect additional efficacy and safety data for OBIZUR in adults with acquired hemophilia A in the Treatment Registry study under Protocol 241302, ""A Non-Interventional Study of Safety and Effectiveness of Recombinant Porcine Sequence FVIII (OBIZUR) in the Treatment of Bleeding Episodes for Patients with Acquired Hemophilia A."""~"Delayed"~"
The final report due date has passed with no final report received"~1/31/2020 0:00:00~12/9/2021 0:00:00
383413~"CBER"~"BLA"~125105.00~"Takeda Pharmaceuticals U.S.A., Inc."~"GAMMAGARD LIQUID/Immune Globulin Infusion (Human)"~4/27/2005 0:00:00~"Supplement"~2023~"125105/2023-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Deferred pediatric study under PREA for the treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in pediatric patients ages 2 to less than 17 years. "~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~12/31/2029 0:00:00~5/22/2026 0:00:00
383414~"CBER"~"BLA"~125813.00~"Autolus, Inc."~"Aucatzyl/Obecabtagene autoleucel"~11/8/2024 0:00:00~"Original"~0~"125813/0-2"~"PMR"~"505 (o)(3)"~~2~"A post-marketing, prospective, multi-center, observational study to assess and characterize the risk of secondary malignancies, and the long-term safety following treatment with obecabtagene autoleucel (Study AUTO1-LT2). The study will include at least 500 adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia; each enrolled patient will be followed for 15 years after product administration."~"Pending"~~6/30/2045 0:00:00~1/7/2026 0:00:00
383415~"CBER"~"BLA"~125813.00~"Autolus, Inc."~"Aucatzyl/Obecabtagene autoleucel"~11/8/2024 0:00:00~"Original"~0~"125813/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Conduct a molecularly targeted pediatric cancer investigation to evaluate dosing, pharmacokinetics, safety and antitumor activity of obecabtagene autoleucel following lymphodepletion with fludarabine and cyclophosphamide in patients 1 year to less than 17 years of age who have relapsed refractory (r/r) B-cell acute lymphoblastic leukemia and r/r aggressive mature B-cell non-Hodgkin lymphoma."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed"~3/31/2028 0:00:00~1/7/2026 0:00:00
383416~"CBER"~"BLA"~125812.00~"Humacyte Global Inc."~"SYMVESS/acellular tissue engineered vessel-tyod"~12/19/2024 0:00:00~"Original"~0~"125812/0-1"~"PMR"~"Pediatric Research Equity Act"~~1~"Conduct a prospective, multi-center, open-label study, to assess the safety and efficacy of SYMVESS in patients 17 years of age or younger who have reached Tanner Sexual Maturity Rating Stage 5, in the approved indication. The study will enroll a minimum of 10 patients who will be followed for a minimum of 1 year and will evaluate primary and secondary patency rate, and characterize the incidence of graft thrombosis, rupture, anastomotic failure, infection, limb amputation, and safety and tolerability.
"~"Submitted"~"The study has not been initiated but does not meet the criterion for delayed"~6/30/2029 0:00:00~2/25/2026 0:00:00
383417~"CBER"~"BLA"~125812.00~"Humacyte Global Inc."~"SYMVESS/acellular tissue engineered vessel-tyod"~12/19/2024 0:00:00~"Original"~0~"125812/0-2"~"PMC"~"506B PMC"~~2~"Conduct long-term observational study to further characterize the risk of
graft failure and infection in patients with extremity vascular injury who have
received SYMVESS for the approved indication. The study should evaluate a
minimum of 100 patients for a minimum follow up period of 1 year and should evaluate the incidence of graft rupture, anastomotic failure, and thrombosis, and describe the incidence of limb amputation and death.
"~"Submitted"~~4/30/2031 0:00:00~2/25/2026 0:00:00
383418~"CBER"~"BLA"~125812.00~"Humacyte Global Inc."~"SYMVESS/acellular tissue engineered vessel-tyod"~12/19/2024 0:00:00~"Original"~0~"125812/0-3"~"PMC"~"506B PMC"~~3~"Complete and submit the study report and dataset for Study CLN-PROV005, an open-label, single-arm study conducted in patients treated with SYMVESS as a vascular replacement or reconstruction in life or limb threatening vascular trauma.
"~"Submitted"~~12/31/2026 0:00:00~2/25/2026 0:00:00
383419~"CBER"~"BLA"~125773.00~"Iovance Biotherapeutics, Inc."~"AMTAGVI/Lifileucel"~2/16/2024 0:00:00~"Original"~0~"125773/0-1"~"PMR"~"Accelerated Approval"~~1~"Complete the Phase 3, multiregional, multicenter, randomized, open-label controlled trial (IOV-MEL-301) in patients with previously untreated unresectable or metastatic melanoma. Patients will be randomized to lifileucel (LN-144) regimen in combination with pembrolizumab or to pembrolizumab monotherapy. The dual primary endpoints will be objective response rate (ORR) and progression-free survival (PFS), with overall survival (OS) as the key secondary endpoint.
"~"Ongoing"~"As per the 180-day AA PMR Progress Report submitted on 10/10/2025, the study/trial is progressing as per the original schedule of milestones.  This report was found to be complete."~3/31/2031 0:00:00~4/15/2025 0:00:00
383420~"CBER"~"BLA"~125816.00~"Axogen Corporation "~"acellular nerve allograft; Avance Nerve Graft/acellular nerve allograft-arwx"~12/3/2025 0:00:00~"Original"~0~"125816/0-1"~"PMR"~"Accelerated Approval"~~1~"Conduct a prospective, randomized, assessor-blinded, multicenter study (Protocol ANG-CP-013) to evaluate the clinical benefit of AVANCE (acellular nerve allograft-arwx) compared to sural nerve autograft for functional recovery following mixed and motor peripheral nerve injury. The study targets adults =18 years of age with mixed or motor nerve repairs in nerve gaps greater than 25 mm. The participants will be followed for 24 months post-surgical repair. The final study report will be submitted within 6 months of study completion, anticipated by 2031, including primary efficacy endpoints of Medical Research Council Classification (MRCC) motor and sensory recovery scores at Month 24. 
"~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~6/5/2031 0:00:00~
383421~"CBER"~"BLA"~125789.00~"USWM CT, LLC"~"TECELRA/afamitresgene autoleucel"~8/1/2024 0:00:00~"Original"~0~"125789/0-3"~"PMR"~"505 (o)(3)"~~3~"A postmarketing, prospective, multi-center, observational study to assess and characterize the risk of secondary malignancies, and long-term safety following treatment with afamitresgene autoleucel (Study CM21-177). The study will include patients with synovial sarcoma who received afamitresgene autoleucel, and each enrolled patient will be followed for 15 years after product administration."~"Pending"~~10/31/2045 0:00:00~
383422~"CBER"~"BLA"~125789.00~"USWM CT, LLC"~"TECELRA/afamitresgene autoleucel"~8/1/2024 0:00:00~"Original"~0~"125789/0-2"~"PMR"~"Pediatric Research Equity Act"~~2~"Conduct a molecularly targeted pediatric cancer investigation in a sufficient number of patients with solid tumors expressing MAGE-A4 to evaluate dosing, pharmacokinetics, safety, and antitumor activity of afamitresgene autoleucel following lymphodepletion with fludarabine and cyclophosphamide. The study should enroll patients aged = 2 years <17 years with synovial sarcoma, malignant peripheral nerve sheath tumor, neuroblastoma, or osteosarcoma, who have received prior systemic therapy for advanced disease, and are positive for HLA-A*02:01, HLA-A*02:02, HLA-A*02:03, or HLA-A*02:06 allele."~"Pending"~"The study has not been initiated but does not meet the criterion for delayed."~9/30/2027 0:00:00~
383423~"CBER"~"BLA"~125789.00~"USWM CT, LLC"~"TECELRA/afamitresgene autoleucel"~8/1/2024 0:00:00~"Original"~0~"125789/0-1"~"PMR"~"Accelerated Approval"~~1~"Submit the Final Clinical Study Report, including datasets from ADP-0044-002 Cohorts 2 and 3, to verify and describe the clinical benefit of afamitresgene autoleucel, through more precise estimation of the overall response rate and mature response duration per independent review assessment, in adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive, and whose tumor expresses the MAGE-A4 tumor antigen as determined by FDA-approved or cleared companion diagnostic devices. Overall response rate and duration of response will be assessed by independent review and all patients will be followed for at least 15 months to assess duration of response."~"Fulfilled"~"PMR fulfilled on June 17, 2026"~12/31/2025 0:00:00~
383424~"CBER"~"BLA"~125789.00~"USWM CT, LLC"~"TECELRA/afamitresgene autoleucel"~8/1/2024 0:00:00~"Supplement"~136~"125789/136-1"~"PMC"~"506B PMC"~~1~"Conduct an adequate analytical and clinical validation study to
establish and support labeling of an FDA-authorized in vitro diagnostic device that accurately and reliably detects MAGE-A4 antigen expression to guide safe and effective use of afamitresgene autoleucel treatment in adolescent patients (= 12 years) with synovial sarcoma. The results of the validation studies are intended to inform product labeling.
"~"Pending"~~9/30/2026 0:00:00~
383425~"CBER"~"BLA"~125789.00~"USWM CT, LLC"~"TECELRA/afamitresgene autoleucel"~8/1/2024 0:00:00~"Original"~0~"125789/0-4"~"PMC"~"506B PMC"~~4~"An adequate assessment of leachables in the DP including the contribution of major process components utilized in the afamitresgene autoleucel manufacturing process, and an updated toxicological risk assessment once the study is completed."~"Pending"~~12/31/2025 0:00:00~
383426~"CDER"~"NDA"~209500.00~"INTRA-CELLULAR THERAPIES INC"~"Caplyta (lumateperone)"~12/20/2019 0:00:00~"Supplement"~16~"4934-1"~"PMC"~"506B PMC"~4934.00~1~"PMC 4934-1: Conduct a double-blind, placebo-controlled, randomized withdrawal study with a prospective non-randomized response period, a non-randomized stabilization period, and a double-blind, placebo-controlled tapered randomized withdrawal period, to evaluate the balance of risk and benefit of continued treatment with lumateperone 42 mg as an adjunct to antidepressant treatment (ADT) for recurrent major depressive disorder. The study should be powered to detect a clinically meaningful treatment effect of lumateperone 42 mg + ADT versus placebo + ADT on an endpoint of time to relapse of MDD."~"Pending"~~10/31/2033 0:00:00~2/11/2026 0:00:00
383427~"CDER"~"NDA"~209501.00~"UPJOHN US 2 LLC"~"Lyrica CR (Pregabalin)"~10/11/2017 0:00:00~"Original"~1~"3716-1"~"PMR"~"505 (o)(3)"~3716.00~1~"PMR 3716-1: In a healthy non-drug dependent population with drug abuse experience with opioids, evaluate the abuse potential of pregabalin taken concomitantly with an opioid, such as oxycodone, investigating a range of doses of pregabalin combined with the opioid. This clinical trial will evaluate the pregabalin/opioid combinations in a cross-over design with comparison to placebo, pregabalin alone, and a moderate dose of the opioid taken alone as a positive control. The assessments will include abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and pharmacokinetics of both pregabalin and the opioid with the goal of characterizing any additive or synergistic effects on the abuse potential and physiologic effects of co-administered pregabalin and opioid."~"Delayed"~"The Final Report milestone was missed because the Final Report submitted 01/31/2023 did not fulfill the PMR due to uninterpretable data, per FDA letter dated 01/26/2024. The Applicant proposed revised milestones and FDA determined the applicant demonstrated  good cause  for delay per FDA letter dated 07/11/2025. The Final Protocol was acknowledged by FDA on 03/25/2026."~8/31/2021 0:00:00~9/29/2025 0:00:00
383428~"CDER"~"NDA"~209510.00~"LXO IRELAND DESIGNATED ACTIVITY CO"~"Barhemsys (amisulpride)"~2/26/2020 0:00:00~"Original"~1~"3478-2"~"PMR"~"Pediatric Research Equity Act"~3478.00~2~"PMR 3478-2: Randomized, double-blind, placebo-controlled, dose-ranging study of Barhemsys (amisulpride) for I.V. injection as prophylaxis for postoperative nausea and vomiting in children aged 0-17 years."~"Delayed"~"Applicant plans to submit the final report by July 2026."~6/30/2024 0:00:00~4/23/2026 0:00:00
383429~"CDER"~"NDA"~209510.00~"LXO IRELAND DESIGNATED ACTIVITY CO"~"Barhemsys (amisulpride)"~2/26/2020 0:00:00~"Original"~1~"3478-3"~"PMR"~"Pediatric Research Equity Act"~3478.00~3~"PMR 3478-3: Randomized, double-blind, active-controlled, dose-ranging study of Barhemsys (amisulpride) for I.V. injection as treatment of children aged 0-17 years with postoperative nausea and vomiting."~"Delayed"~"Deferral Extension Requested 04/30/2024. Denied per FDA letter dated 06/17/2024."~6/30/2024 0:00:00~4/23/2026 0:00:00
383430~"CDER"~"NDA"~209511.00~"INNOCOLL PHARMACEUTICALS"~"Xaracoll (Bupivacaine Hydrochloride Collagen-Matrix Implants)"~8/28/2020 0:00:00~"Original"~1~"3928-1"~"PMR"~"Pediatric Research Equity Act"~3928.00~1~"PMR 3928-1: Conduct a randomized, controlled study to evaluate the safety, tolerability, pharmacokinetics, and analgesic effect of the bupivacaine HCl collagen implant in children 2 to less than 17 years of age following open inguinal hernia repair."~"Delayed"~"Original Final Report Due Date: 05/31/2024; Deferral Extension granted per FDA letter dated 09/25/2024. The study completion milestone was also revised.
"~5/31/2027 0:00:00~10/27/2023 0:00:00
383431~"CDER"~"NDA"~209511.00~"INNOCOLL PHARMACEUTICALS"~"Xaracoll (Bupivacaine Hydrochloride Collagen-Matrix Implants)"~8/28/2020 0:00:00~"Original"~1~"3928-2"~"PMR"~"Pediatric Research Equity Act"~3928.00~2~"PMR 3928-2: Conduct a randomized, controlled study to evaluate the safety,tolerability, pharmacokinetics, and efficacy of the bupivacaine HCl collagen implant in children from birth to less than 2 years of age following open inguinal hernia repair"~"Delayed"~"Original Final Report Due Date: 08/31/2023; Deferral Extension granted per FDA letter dated 09/25/2024. The Draft protocol submission, Final protocol and Study completion milestones were also revised.
"~12/31/2029 0:00:00~10/27/2023 0:00:00
383432~"CDER"~"NDA"~209531.00~"BIOGEN INC"~"Spinraza (nusinersen)"~12/23/2016 0:00:00~"Original"~1~"3884-1"~"PMR"~"505 (o)(3)"~3884.00~1~"PMR 3884-1: Establish a single-arm pregnancy safety study to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes for exposed pregnancies, including women exposed to Spinraza (nusinersen) during pregnancy and a relevant exposure period prior to the start of pregnancy. Provide a complete protocol that includes details regarding how you plan to encourage patients and providers to report pregnancy exposures, measures to ensure complete data capture regarding pregnancy outcomes and any adverse effects in offspring, and plans for comprehensive data analysis."~"Ongoing"~~10/31/2033 0:00:00~2/20/2026 0:00:00
383433~"CDER"~"NDA"~209570.00~"CHEMO RESEARCH SL"~"Benznidazole"~8/29/2017 0:00:00~"Original"~1~"3247-1"~"PMR"~"Accelerated Approval"~3247.00~1~"PMR 3247-1: Conduct a prospective, single-arm, multicenter trial, with historical controls, to evaluate safety, efficacy and pharmacokinetics of benznidazole tablets for treatment of Chagas disease in children."~"Delayed"~"Acknowledge revised PMR milestones letter was issued on 7-28-2022. As per the 180-day AA PMR Progress Report submitted on 10/28/2025 and amended report on 1/23/2026, there were 178 subjects randomized, 18 prematurely discontinued and 6 lost to follow up with 168 subjects completing treatment phase. The follow up phase is expected to be completed by August 2027 which is the study/trial completion revised date and is on track with the revised milestones.
"~5/31/2026 0:00:00~10/28/2025 0:00:00
383434~"CDER"~"NDA"~209575.00~"OMNIVIUM PHARMACEUTICALS LLC"~"Numbrino (cocaine hydrochloride)"~1/10/2020 0:00:00~"Original"~1~"3768-1"~"PMR"~"Pediatric Research Equity Act"~3768.00~1~"PMR 3768-1: Conduct a juvenile animal study to characterize the impact of cocaine on brain development and male reproductive tissue and development to support pediatric dosing in children 12 years of age to less than 17 years of age."~"Submitted"~"The final report was submitted to FDA on 7/28/2022"~3/31/2023 0:00:00~3/5/2026 0:00:00
383435~"CDER"~"NDA"~209575.00~"OMNIVIUM PHARMACEUTICALS LLC"~"Numbrino (cocaine hydrochloride)"~1/10/2020 0:00:00~"Original"~1~"3768-2"~"PMR"~"Pediatric Research Equity Act"~3768.00~2~"PMR 3768-2: Conduct a multicenter trial to evaluate the pharmacokinetic and safety profiles of a single topical administration of NUMBRINO for the induction of local anesthesia of the mucous membranes when performing diagnostic procedures and surgeries on or through the nasal cavities in pediatric subjects 12 years of age to less than 17 years of age."~"Ongoing"~"The study has been initiated.

"~7/31/2024 0:00:00~3/5/2026 0:00:00
383436~"CDER"~"NDA"~209575.00~"OMNIVIUM PHARMACEUTICALS LLC"~"Numbrino (cocaine hydrochloride)"~1/10/2020 0:00:00~"Original"~1~"3768-3"~"PMR"~"505 (o)(3)"~3768.00~3~"PMR 3768-3: Conduct a female fertility and early embryonic development study in the rat model to adequately characterize the effect of cocaine on female fertility and early embryonic development."~"Submitted"~~12/31/2021 0:00:00~3/5/2026 0:00:00
383437~"CDER"~"NDA"~209575.00~"OMNIVIUM PHARMACEUTICALS LLC"~"Numbrino (cocaine hydrochloride)"~1/10/2020 0:00:00~"Original"~1~"3768-4"~"PMR"~"505 (o)(3)"~3768.00~4~"PMR 3768-4: Conduct an embryo-fetal development study in the rat model to characterize the teratogenic potential of cocaine."~"Submitted"~~10/31/2021 0:00:00~3/5/2026 0:00:00
383438~"CDER"~"NDA"~209575.00~"OMNIVIUM PHARMACEUTICALS LLC"~"Numbrino (cocaine hydrochloride)"~1/10/2020 0:00:00~"Original"~1~"3768-5"~"PMR"~"505 (o)(3)"~3768.00~5~"PMR 3768-5: Conduct an embryo-fetal development study in the rabbit model to characterize the teratogenic potential of cocaine."~"Submitted"~~10/31/2021 0:00:00~3/5/2026 0:00:00
383439~"CDER"~"NDA"~209575.00~"OMNIVIUM PHARMACEUTICALS LLC"~"Numbrino (cocaine hydrochloride)"~1/10/2020 0:00:00~"Original"~1~"3768-6"~"PMR"~"505 (o)(3)"~3768.00~6~"PMR 3768-6: Conduct a pre- and post-natal development study in the rat model to characterize the impact of cocaine on development, including exposure during lactation to weaning, growth and development, functional assessments, and reproductive capacity of the offspring."~"Submitted"~~4/30/2022 0:00:00~3/5/2026 0:00:00
383440~"CDER"~"NDA"~209589.00~"FERRING PHARMACEUTICALS INC"~"Clenpiq (Citric Acid, Magnesium Oxide, and Sodium Picosulfate)"~11/28/2017 0:00:00~"Original"~1~"3252-4"~"PMR"~"Pediatric Research Equity Act"~3252.00~4~"PMR 3252-4: A Randomized, Assessor-Blind, Multicenter, Dose-Ranging Study Comparing the Safety and Efficacy of Sodium Picosulfate, Magnesium Oxide, Citric Acid versus Active Comparator in Children Aged 2 Years to Less Than 9 Years."~"Delayed"~"Original Final Report Due Date: 06/30/2024; Deferral Extension granted per FDA letter dated 05/10/2024. 9/30 subjects currently enrolled.
"~9/30/2026 0:00:00~1/27/2026 0:00:00
383441~"CDER"~"NDA"~209589.00~"FERRING PHARMACEUTICALS INC"~"Clenpiq (Citric Acid, Magnesium Oxide, and Sodium Picosulfate)"~11/28/2017 0:00:00~"Original"~1~"3252-5"~"PMR"~"Pediatric Research Equity Act"~3252.00~5~"PMR 3252-5: A Randomized, Assessor-Blind, Multicenter, Dose-Ranging Study Comparing the Safety and Efficacy of Sodium Picosulfate, Magnesium Oxide, Citric Acid versus Active Comparator in Children Aged 12 Months to Less Than 2 Years."~"Delayed"~"Original Final Report Due Date: 06/30/2024; Deferral Extension
granted per FDA letter dated 05/10/2024."~6/30/2029 0:00:00~1/27/2026 0:00:00
383442~"CDER"~"NDA"~209607.00~"PROGENICS PHARMACEUTICALS INC A LANTHEUS CO"~"Azedra ([131I]-Iobenguane) "~7/30/2018 0:00:00~"Original"~1~"3360-1"~"PMR"~"505 (o)(3)"~3360.00~1~"PMR 3360-1: Conduct cumulative, integrated safety analyses after 5 and after 10 years of follow-up of patients from an adequate number of clinical trials to identify and characterize the risks of myelodysplastic syndrome, acute leukemia and other secondary malignancies with AZEDRA; include incidence rates, time to onset, predisposing factors, and outcomes. These safety evaluations should be adequate to inform labeling of patient populations at highest risk and to provide evidence-based monitoring recommendations. 
"~"Ongoing"~~4/30/2029 0:00:00~9/28/2023 0:00:00
383443~"CDER"~"NDA"~209627.00~"MAYNE PHARMA LLC"~"Annovera Vaginal System (segesterone acetate/ethinyl estradiol 150/13)"~8/10/2018 0:00:00~"Original"~1~"3454-1"~"PMR"~"505 (o)(3)"~3454.00~1~"PMR 3454-1: A controlled, non-interventional, long-term cohort study that follows a series of cohorts comprising new users of your product (segesterone acetate and ethinylestradiol vaginal system), new users of other vaginal ring contraceptives, new users of any intrauterine system, and new users of combined oral contraceptives containing other progestins. The primary objective of the study is to assess the risk for fatal and non-fatal venous thromboembolism and arterial thromboembolism associated with short-term and long-term use of your product in a study population representative of actual users of the product in the US and other countries where your vaginal system is prescribed. The study should be sufficiently powered and have adequate confounding control to rule out a 1.5 to 2- fold risk for venous thromboembolism."~"Delayed"~"The final protocol submission milestone was missed due to
continued negotiations with the FDA to finalize the study protocol. Second Good Cause letter issued on 5/20/2024 with the acknowledged proposed revised final protocol submission milestone."~8/31/2030 0:00:00~10/8/2025 0:00:00
383444~"CDER"~"NDA"~209637.00~"NOVO NORDISK INC"~"Ozempic (Semaglutide)"~12/5/2017 0:00:00~"Original"~1~"4745-1"~"PMR"~"505 (o)(3)"~4745.00~1~"PMR 4745-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of semaglutide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Ongoing"~~11/30/2026 0:00:00~11/17/2025 0:00:00
383445~"CDER"~"NDA"~209637.00~"NOVO NORDISK INC"~"Ozempic (Semaglutide)"~12/5/2017 0:00:00~"Original"~1~"3294-2"~"PMR"~"505 (o)(3)"~3294.00~2~"PMR 3294-2: Conduct a medullary thyroid carcinoma registry-based case series of at least 15 years duration to systematically monitor the annual incidence of medullary  thyroid carcinoma in the United States and to identify any increase related to the introduction of Ozempic (semaglutide) into the marketplace. This study will also  establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to diabetes and use of Ozempic (semaglutide)."~"Ongoing"~~5/31/2035 0:00:00~11/17/2025 0:00:00
383446~"CDER"~"NDA"~209637.00~"NOVO NORDISK INC"~"Ozempic (Semaglutide)"~12/5/2017 0:00:00~"Original"~1~"3294-5"~"PMR"~"Pediatric Research Equity Act"~3294.00~5~"PMR 3294-5: Conduct a 52-week, randomized, double-blind, placebo-controlled parallel group study of the safety and efficacy of semaglutide for the treatment of type 2 diabetes mellitus in pediatric subjects ages 10 to 17 years (inclusive). Background therapy will consist of either metformin, insulin, or metformin plus insulin."~"Ongoing"~"91 of 132 subjects enrolled.
"~6/30/2027 0:00:00~11/17/2025 0:00:00
383447~"CDER"~"NDA"~209637.00~"NOVO NORDISK INC"~"Ozempic (Semaglutide)"~12/5/2017 0:00:00~"Supplement"~9~"4246-1"~"PMR"~"Pediatric Research Equity Act"~4246.00~1~"PMR 4246-1: Conduct a 52-week, randomized, double-blind, placebo-controlled parallel group study of the safety and efficacy of semaglutide for the treatment of type 2 diabetes mellitus in pediatric subjects ages 10 to 17 years (inclusive). Background therapy will consist of either metformin, insulin, or metformin plus insulin."~"Ongoing"~"91 of 132 subjects enrolled."~6/30/2027 0:00:00~11/17/2025 0:00:00
383448~"CDER"~"NDA"~209661.00~"DUCHESNAY INC"~"Bonjesta (Doxylamine Succinate and Pyridoxine Hydrochloride)"~11/7/2016 0:00:00~"Original"~1~"3812-1"~"PMR"~"Pediatric Research Equity Act"~3812.00~1~"PMR 3812-1: An adequately powered safety and efficacy study in pregnant adolescent girls, 12 to 17 years of age, with nausea and vomiting of pregnancy who are appropriate candidates for pharmacologic therapy."~"Delayed"~"The Study Completion milestone was missed, because of exceptionally low enrollment rate. Original Final Report Due Date 12/2022, then 12/2025. Most recent Deferral
Extension granted per FDA letter dated 04/14/2023."~6/30/2028 0:00:00~12/23/2025 0:00:00
383449~"CDER"~"NDA"~209776.00~"REMPEX PHARMACEUTICALS INC A WHOLLY OWNED SUB OF MELINTA THERAPEUTICS LLC"~"Vabomere (Meropenem and Vaborbactam)"~8/29/2017 0:00:00~"Original"~1~"3248-1"~"PMR"~"Pediatric Research Equity Act"~3248.00~1~"PMR 3248-1: Conduct an open-label, sequential study to assess the pharmacokinetics (PK), safety, and tolerability of VABOMERE and the PK of meropenem and vaborbactam in children from birth to < 18 years of age with selected serious bacterial infections."~"Delayed"~"FDA Issued a Deferral Extension granted /Acknowledge revised PMR letter on 11/15/2024."~4/30/2026 0:00:00~10/24/2025 0:00:00
383450~"CDER"~"NDA"~209776.00~"REMPEX PHARMACEUTICALS INC A WHOLLY OWNED SUB OF MELINTA THERAPEUTICS LLC"~"Vabomere (Meropenem and Vaborbactam)"~8/29/2017 0:00:00~"Original"~1~"3248-2"~"PMR"~"Pediatric Research Equity Act"~3248.00~2~"PMR 3248-2: Conduct a randomized, single-blind, active comparator study to evaluate the safety, tolerability, and PK of VABOMERE versus piperacillin-tazobactam for the treatment of pediatric subjects from 3 months to <18 years of age with complicated Urinary Tract Infections (cUTI) including acute pyelonephritis."~"Delayed"~"Deferral extension was granted per FDA letter dated 1-28-2022."~12/31/2026 0:00:00~10/24/2025 0:00:00
383451~"CDER"~"NDA"~210745.00~"AMGEN INC"~"Otezla XR (apremilast) extended-release tablets"~8/29/2025 0:00:00~"Original"~2~"4885-2"~"PMR"~"Pediatric Research Equity Act"~4885.00~2~"PMR 4885-2: Conduct a relative bioavailability study in healthy adult subjects to determine an appropriate dose and dosing regimen for the use of extended-release apremilast formulation in pediatric patients aged 6 to 17 years weighing 20 kg to 50 kg for the treatment of moderate to severe plaque psoriasis and active psoriatic arthritis."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~2/28/2028 0:00:00~
383452~"CDER"~"NDA"~210795.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY DEVELOPMENT LTD ENGLAND"~"Krintafel (tafenoquine)"~7/20/2018 0:00:00~"Original"~1~"3436-2"~"PMR"~"505 (o)(3)"~3436.00~2~"PMR 3436-2: Conduct a study to evaluate safety, including hypersensitivity, neuropsychiatric, and hematologic adverse reactions, in United States patients taking KRINTAFEL (tafenoquine) for the radical cure of P. vivax malaria."~"Fulfilled"~~9/30/2025 0:00:00~9/16/2025 0:00:00
383453~"CDER"~"NDA"~210797.00~"CLINUVEL INC"~"Scenesse (Afamelanotide) "~10/8/2019 0:00:00~"Original"~1~"3726-1"~"PMR"~"505 (o)(3)"~3726.00~1~"PMR 3726-1: Conduct a thorough QT clinical study to adequately characterize the effect of afamelanotide on cardiac repolarization."~"Released"~~2/28/2022 0:00:00~12/4/2025 0:00:00
383454~"CDER"~"NDA"~210797.00~"CLINUVEL INC"~"Scenesse (Afamelanotide) "~10/8/2019 0:00:00~"Original"~1~"3726-2"~"PMR"~"505 (o)(3)"~3726.00~2~"PMR 3726-2: Conduct a prospective, longitudinal, registry based observational exposure cohort study to collect information on long-term safety of afamelanotide in patients with erythropoietic protoporphyria (EPP) in the United States. Patients will be followed for a minimum of eight years from initiation of treatment with afamelanotide. The primary adverse events of interest are: skin cancer (melanomas and non-melanomas) - administration/injection/implant site reactions. Secondary adverse events of interest are: changes in pigmentary expressions - pregnancy outcomes (including major birth defects and other adverse pregnancy outcomes such as spontaneous abortions, stillbirths, preterm deliveries, and small for gestational age) - exposure during lactation and adverse reactions in breastfed infants - implantation device malfunction or failure."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 05/08/2026."~3/31/2031 0:00:00~12/4/2025 0:00:00
383455~"CDER"~"NDA"~210806.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"PIFELTRO (doravirine)"~8/30/2018 0:00:00~"Original"~1~"3415-2"~"PMR"~"Pediatric Research Equity Act"~3415.00~2~"PMR 3415-2: Conduct a study to evaluate the pharmacokinetics, safety and antiviral activity (efficacy) of doravirine in HIV-1 infected pediatric subjects at least 2 years of age and weighing less than 35 kg. The study participants must be followed for a minimum of 24 weeks to assess the safety and antiviral activity of doravirine."~"Delayed"~"Original Final Report Due Date: 05/31/2024; Deferral Extension granted per FDA letter dated 03/31/2023. The study completion milestone was also revised. Per the applicant's annual status report, reporting enrollment of 74 total subjects, including 68 subjects of all ages weighing less than 35 kg. Of these, 63 subjects are 2 years of age or older as of 08/30/2025."~12/31/2026 0:00:00~10/17/2025 0:00:00
383456~"CDER"~"NDA"~210806.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"PIFELTRO (doravirine)"~8/30/2018 0:00:00~"Original"~1~"3415-3"~"PMR"~"Pediatric Research Equity Act"~3415.00~3~"PMR 3415-3: Conduct a study to evaluate the pharmacokinetics, safety and antiviral activity (efficacy) of doravirine in HIV-1 infected pediatric subjects 4 weeks of age to 23 months of age. The study participants must be followed for a minimum of 24 weeks to assess the safety and antiviral activity of doravirine."~"Ongoing"~"Reporting enrollment of 74 total subjects, including 68 subjects of all ages weighing less than 35kg. Of these, 5 subjects are less than 2 years of age as of 08/30/2025."~2/28/2029 0:00:00~10/17/2025 0:00:00
383457~"CDER"~"NDA"~210807.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"DELSTRIGO (doravirine, lamivudine, and tenofovir disoproxil fumarate) "~8/30/2018 0:00:00~"Original"~1~"3416-2"~"PMR"~"Pediatric Research Equity Act"~3416.00~2~"PMR 3416-2: Conduct a study to evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of doravirine/lamivudine/tenofovir disoproxil fumarate fixed dose combination (FDC) product in HIV-1 infected pediatric subjects age 2 years and older, and weighing less than 35 kg. The study participants must be followed for a minimum of 24 weeks to assess the safety and antiviral activity of the FDC product, doravirine/lamivudine/tenofovir disoproxil fumarate. A clinical trial in pediatric subjects 2 years and older and weighing less than 35 kg may not be required if dosing recommendation for the FDC tablets can be supported by pediatric trials conducted with the individual drug products."~"Delayed"~"Original Final Report Due Date: 05/31/2024; Deferral Extension granted per FDA letter dated 03/31/2023. The study completion milestone was also revised. Per the applicant's annual status report, Rreporting enrollment of 74 total subjects, including 68 subjects of all ages weighing less than 35kg. Of these, 63 subjects are 2 years of age or older as of 08/30/2025. 
"~12/31/2026 0:00:00~10/17/2025 0:00:00
383458~"CDER"~"NDA"~210854.00~"GENENTECH INC"~"Xofluza (baloxavir marboxil)"~10/24/2018 0:00:00~"Supplement"~5~"4317-2"~"PMC"~"506B PMC"~4317.00~2~"PMC 4317-2: Conduct a prospective, multicenter, observational study in baloxavir marboxil-treated patients over at least five influenza seasons that will capture the susceptibility and genotype of influenza viruses in baseline and on-treatment respiratory samples to determine the frequency of baseline and treatment-emergent baloxavir resistance and the impact on outcomes."~"Ongoing"~~10/31/2027 0:00:00~12/18/2025 0:00:00
383459~"CDER"~"NDA"~210854.00~"GENENTECH INC"~"Xofluza (baloxavir marboxil)"~10/24/2018 0:00:00~"Supplement"~5~"4317-3"~"PMC"~"506B PMC"~4317.00~3~"PMC 4317-3: Provide a semi-annual (twice-yearly) update on global baloxavir usage and emergence of resistance to baloxavir as an integrated review of information from national and international influenza drug resistance databases and sequence databases, including but not limited to World Health Organization and US Centers for Disease Control and Prevention surveillance, data collected by the sponsor, and information in the published literature. Each update will include information on the methodologies (e.g., viral gene sequencing and phenotypic assay descriptions) used in studies during that reporting period. Substitutions of particular interest include all those listed as resistance associated in the USPI, as well as substitutions currently identified or identified in the future that reduce susceptibility to baloxavir marboxil."~"Ongoing"~~5/31/2026 0:00:00~12/18/2025 0:00:00
383460~"CDER"~"NDA"~210854.00~"GENENTECH INC"~"Xofluza (baloxavir marboxil)"~10/24/2018 0:00:00~"Supplement"~9~"4317-2"~"PMC"~"506B PMC"~4317.00~2~"PMC 4317-2: Conduct a prospective, multicenter, observational study in baloxavir marboxil-treated patients over at least five influenza seasons that will capture the susceptibility and genotype of influenza viruses in baseline and on-treatment respiratory samples to determine the frequency of baseline and treatment-emergent baloxavir resistance and the impact on outcomes."~"Ongoing"~~10/31/2027 0:00:00~12/18/2025 0:00:00
383461~"CDER"~"NDA"~210854.00~"GENENTECH INC"~"Xofluza (baloxavir marboxil)"~10/24/2018 0:00:00~"Supplement"~9~"4317-3"~"PMC"~"506B PMC"~4317.00~3~"PMC 4317-3: Provide a semi-annual (twice-yearly) update on global baloxavir usage and emergence of resistance to baloxavir as an integrated review of information from national and international influenza drug resistance databases and sequence databases, including but not limited to World Health Organization and US Centers for Disease Control and Prevention surveillance, data collected by the sponsor, and information in the published literature. Each update will include information on the methodologies (e.g., viral gene sequencing and phenotypic assay descriptions) used in studies during that reporting period. Substitutions of particular interest include all those listed as resistance associated in the USPI, as well as substitutions currently identified or identified in the future that reduce susceptibility to baloxavir marboxil."~"Ongoing"~~5/31/2026 0:00:00~12/18/2025 0:00:00
383462~"CDER"~"NDA"~210861.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Vitrakvi (larotrectinib)"~11/26/2018 0:00:00~"Original"~1~"3540-3"~"PMR"~"505 (o)(3)"~3540.00~3~"PMR 3540-3: Conduct a study of larotrectinib in a sufficient number of pediatric patients with NTRK-fusion solid tumors to evaluate the potential serious risk of adverse longterm effects of larotrectinib on the growth and development of pediatric patients. Patients will be evaluated for growth and developmental milestones using age appropriate screening tools and undergo neurological examination at appropriate intervals (for example, every six months) until larotrectinib is discontinued or for minimum of five years, whichever occurs first. Evaluations should include a neurologic exam, developmental milestone assessment, Karnofsky/Lansky score, growth as measured by weight and height, height velocity, height standard deviation scores (SDS), age at adrenarche if applicable (males), age at menarche if applicable (females), and Tanner Stage."~"Delayed"~"The final protocol was amended after recommendations from the Agency and submitted on October 4, 2019, which was after the original milestone.
"~8/31/2028 0:00:00~1/23/2026 0:00:00
383463~"CDER"~"NDA"~210861.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Vitrakvi (larotrectinib)"~11/26/2018 0:00:00~"Original"~1~"3540-4"~"PMR"~"505 (o)(3)"~3540.00~4~"PMR 3540-4: Conduct a study of larotrectinib 100 mg orally once daily in a sufficient number of adult or pediatric patients with a body surface area of at least 1.0 m2 who experienced an adverse reaction requiring a third dosage modification of larotrectinib to better characterize the tolerability of this approved dosage modification for larotrectinib. The following information will be provided for each patient: patient age and body surface area (if pediatric), adverse reactions leading to each prior dose reduction of larotrectinib, duration of treatment on prior dose levels, duration of treatment at the 100 mg orally once daily regimen, best overall response and duration of response, and tumor information collected while receiving the 100 mg orally once daily dosage regimen."~"Delayed"~"The final protocol was amended after recommendations from the Agency and submitted on October 4, 2019, which was after the original milestone."~8/31/2028 0:00:00~1/23/2026 0:00:00
383464~"CDER"~"NDA"~210861.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Vitrakvi (larotrectinib)"~11/26/2018 0:00:00~"Supplement"~4~"3974-1"~"PMR"~"505 (o)(3)"~3974.00~1~"PMR 3974-1: Submit an integrated safety analyses and supporting data from an adequate number of patients enrolled in clinical trial(s) designed to further characterize the risk of fractures and its sequelae; and to identify the risk factors for development of fractures and their sequelae in patients exposed to larotrectinib. The design of the trial should include assessment and collection of sufficient height measurements and measures of bone monitoring, including but not limited to initial and serial assessment of bone mineral density (BMD) with dual x-ray absorptiometry (DXA) scans, and markers of bone formation, bone resorption, and calcium metabolism."~"Fulfilled"~~8/31/2025 0:00:00~1/23/2026 0:00:00
383465~"CDER"~"NDA"~210864.00~"LUPIN INC"~"Sesquient (Fosphenytoin sodium)"~11/5/2020 0:00:00~"Original"~1~"4087-1"~"PMR"~"505 (o)(3)"~4087.00~1~"PMR 4087-1: Conduct in vitro patch clamp studies to assess Sesquient s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Sesquient s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~12/31/2025 0:00:00
383466~"CDER"~"NDA"~210867.00~"MEDICINES DEVELOPMENT FOR GLOBAL HEALTH LTD"~"Moxidectin"~6/13/2018 0:00:00~"Original"~1~"3367-2"~"PMC"~"506B PMC"~3367.00~2~"PMC 3367-2: Conduct a prospective, randomized, ivermectin-controlled trial of repeated doses of 8 mg moxidectin for control of onchocerciasis."~"Delayed"~"The trial completion and final report submission milestones were missed. The applicant requested revised milestones because of ongoing delays with the availability of final data. These revised milestones were acknowledged in a letter dated 04/15/2026."~3/31/2024 0:00:00~8/11/2025 0:00:00
383467~"CDER"~"NDA"~210868.00~"PFIZER INC"~"LORBRENA (Lorlatinib)"~11/2/2018 0:00:00~"Original"~1~"3500-4"~"PMR"~"505 (o)(3)"~3500.00~4~"PMR 3500-4: Complete a pharmacokinetic trial to determine an appropriate dose of lorlatinib to minimize toxicity in patients with moderate and severe hepatic impairment in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling  found at
https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugsgen/documents/document/ucm072123.pdf"~"Fulfilled"~~2/28/2024 0:00:00~12/30/2025 0:00:00
383468~"CDER"~"NDA"~210868.00~"PFIZER INC"~"LORBRENA (Lorlatinib)"~11/2/2018 0:00:00~"Supplement"~4~"4031-1"~"PMC"~"506B PMC"~4031.00~1~"PMC 4031-1: Submit the clinical study report and datasets for the final analysis of overall survival, planned to occur after a total of 198 events, for Study B7461006,  A Phase 3, Randomized, Open-Label Study of Lorlatinib Monotherapy versus Crizotinib Monotherapy in the First-line Treatment of Patients with Advanced ALK-Positive Non-Small Cell Lung Cancer.  The final results of these analyses may inform product labeling."~"Ongoing"~~6/30/2029 0:00:00~12/30/2025 0:00:00
383469~"CDER"~"NDA"~210910.00~"COSMO TECHNOLOGIES LTD"~"AEMCOLO (rifamycin) "~11/16/2018 0:00:00~"Original"~1~"3505-1"~"PMR"~"Pediatric Research Equity Act"~3505.00~1~"PMR 3505-1: Conduct a randomized, placebo-controlled study to evaluate the safety, tolerability, and efficacy of AEMCOLO (rifamycin) for the treatment of travelers  diarrhea in children from 6 to 11 years of age."~"Delayed"~"A notification of non-compliance with PREA letter was issued on 1-23-2026  for missing the final report submission milestone.
"~12/31/2025 0:00:00~1/14/2026 0:00:00
383470~"CDER"~"NDA"~210910.00~"COSMO TECHNOLOGIES LTD"~"AEMCOLO (rifamycin) "~11/16/2018 0:00:00~"Original"~1~"3505-2"~"PMR"~"Pediatric Research Equity Act"~3505.00~2~"PMR 3505-2: Conduct a randomized, placebo-controlled study to evaluate the safety, tolerability, and efficacy of AEMCOLO (rifamycin) for the treatment of travelers  diarrhea in children from 12 to 17 years of age."~"Delayed"~"A notification of non-compliance with PREA letter was issued on 1-23-2026 for missing the final report submission milestone."~12/31/2025 0:00:00~1/14/2026 0:00:00
383471~"CDER"~"NDA"~210922.00~"ALNYLAM PHARMACEUTICALS INC"~"Onpattro (patisiran)"~8/10/2018 0:00:00~"Original"~1~"3425-1"~"PMR"~"505 (o)(3)"~3425.00~1~"PMR 3425-1: Establish a worldwide Pregnancy Surveillance Program to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to Onpattro (patisiran) during pregnancy. Provide a complete protocol which includes details regarding how you plan to encourage patients and providers to report pregnancy exposures (e.g., telephone contact number and/or website in prescribing information), measures to ensure complete data capture regarding pregnancy outcomes and any adverse effects in offspring, and plans for comprehensive data analysis and yearly reporting."~"Ongoing"~~10/31/2031 0:00:00~10/8/2025 0:00:00
383472~"CDER"~"NDA"~211109.00~"TETRAPHASE PHARMACEUTICALS INC"~"XERAVA (eravacycline) "~8/27/2018 0:00:00~"Original"~1~"4458-2"~"PMR"~"Pediatric Research Equity Act"~4458.00~2~"PMR 4458-2: Conduct a multicenter, open-label trial to evaluate the safety and tolerability of intravenous Xerava (eravacycline) in pediatric patients from 8 years to less than 18 years of age with complicated intra-abdominal infections."~"Ongoing"~"There are currently 8 patients enrolled."~10/31/2029 0:00:00~10/20/2025 0:00:00
383473~"CDER"~"NDA"~211109.00~"TETRAPHASE PHARMACEUTICALS INC"~"XERAVA (eravacycline) "~8/27/2018 0:00:00~"Original"~1~"3472-3"~"PMR"~"505 (o)(3)"~3472.00~3~"PMR 3472-3: A United States surveillance study for 5 years from the date of marketing to determine if resistance to XERAVA (eravacycline) has developed in those organisms specific to the indication in the label."~"Fulfilled"~~2/28/2025 0:00:00~10/20/2025 0:00:00
383474~"CDER"~"NDA"~211150.00~"HARMONY BIOSCIENCES MANAGEMENT INC"~"Wakix (pitolisant)"~8/14/2019 0:00:00~"Original"~1~"3675-1"~"PMR"~"505 (o)(3)"~3675.00~1~"PMR 3675-1: Conduct a prospective, registry-based, observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to pitolisant during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~6/30/2030 0:00:00~10/14/2025 0:00:00
383475~"CDER"~"NDA"~211150.00~"HARMONY BIOSCIENCES MANAGEMENT INC"~"Wakix (pitolisant)"~8/14/2019 0:00:00~"Original"~1~"3675-2"~"PMR"~"505 (o)(3)"~3675.00~2~"PMR 3675-2: Conduct a pregnancy study that uses a different design from the Pregnancy Registry (e.g., a case control study or a retrospective cohort study using claims or electronic medical record data with outcome validation) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to pitolisant during pregnancy compared to an unexposed control population."~"Ongoing"~~6/30/2030 0:00:00~10/14/2025 0:00:00
383476~"CDER"~"NDA"~211172.00~"AKCEA THERAPEUTICS INC"~"Tegsedi (Inotersen)"~10/5/2018 0:00:00~"Original"~1~"3491-1"~"PMR"~"505 (o)(3)"~3491.00~1~"PMR 3491-1: A clinical study to characterize adverse events occurring within one day of inotersen administration to adult patients with the polyneuropathy of hereditary transthyretin-mediated amyloidosis. Characterize the events in individual patients and overall with respect to time course of adverse event onset, vital sign changes, preventive measures, treatment required, risk factors, and subsequent adverse outcomes. An adequate number of patients should be enrolled and followed throughout their treatment with inotersen to allow for the characterization of adverse events occurring within one day of inotersen administration (e.g., hypersensitivity, cytokine release syndrome)."~"Released"~~5/31/2025 0:00:00~9/1/2023 0:00:00
383477~"CDER"~"NDA"~211172.00~"AKCEA THERAPEUTICS INC"~"Tegsedi (Inotersen)"~10/5/2018 0:00:00~"Original"~1~"3491-2"~"PMR"~"505 (o)(3)"~3491.00~2~"PMR 3491-2: A prospective observational registry study in adult patients with the polyneuropathy of hereditary transthyretin-mediated amyloidosis recruited from the REMS registry. The primary objectives are to characterize the risks of serious thrombocytopenia, glomerulonephritis, stroke and cervicocephalic arterial dissection, and CNS vasculitis with respect to time course of onset, preventive laboratory monitoring, and identification of risk factors. An adequate number of patients should be enrolled and followed throughout their participation in the REMS registry to allow for the characterization of serious thrombocytopenia, glomerulonephritis, stroke and cervicocephalic arterial dissection, and CNS vasculitis. The protocol should specify an appropriate comparator population(s) to which observed incidence rates will be compared."~"Released"~~5/31/2025 0:00:00~9/1/2023 0:00:00
383478~"CDER"~"NDA"~211172.00~"AKCEA THERAPEUTICS INC"~"Tegsedi (Inotersen)"~10/5/2018 0:00:00~"Original"~1~"3491-3"~"PMR"~"505 (o)(3)"~3491.00~3~"PMR 3491-3: Establish a worldwide Pregnancy Surveillance Program to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to Tegsedi (inotersen) during pregnancy. Provide a complete protocol which includes details regarding how you plan to encourage patients and providers to report pregnancy exposures (e.g., telephone contact number and/or website in prescribing information), measures to ensure complete data capture regarding pregnancy outcomes and any adverse effects in offspring, and plans for comprehensive data analysis and yearly reporting."~"Released"~~11/30/2031 0:00:00~9/1/2023 0:00:00
383479~"CDER"~"NDA"~211192.00~"SERVIER PHARMACEUTICALS LLC"~"Tibsovo (ivosidenib)"~7/20/2018 0:00:00~"Supplement"~1~"3596-1"~"PMR"~"505 (o)(3)"~3596.00~1~"PMR 3596-1: Conduct a clinical pharmacokinetic trial to determine an appropriate safe dose of ivosidenib in patients with hematologic malignancies who have a susceptible IDH1 mutation with moderate (total bilirubin >1.5 to 3 x ULN and any AST) and severe (total bilirubin >3 x ULN and any AST) hepatic impairment dosed with ivosidenib to steady-state versus patients with normal hepatic function dosed with ivosidenib to steady-state. This may be performed as a substudy in the ongoing Phase 1 Study AG120-C-001, A Phase 1, Multicenter, Open-Label, DoseEscalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects with Advanced Hematologic Malignancies with an IDH1 Mutation. This trial should be designed and conducted in accordance with the FDA Guidance for Industry entitled Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling."~"Delayed"~"The applicant requested revised milestones because of challenges with enrollment. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 10/08/2025."~9/30/2025 0:00:00~7/9/2026 0:00:00
383480~"CDER"~"NDA"~211192.00~"SERVIER PHARMACEUTICALS LLC"~"Tibsovo (ivosidenib)"~7/20/2018 0:00:00~"Supplement"~1~"3596-2"~"PMR"~"505 (o)(3)"~3596.00~2~"PMR 3596-2: Conduct a clinical pharmacokinetic trial to determine an appropriate safe dose of ivosidenib in patients with hematologic malignancies who have a susceptible IDH1 mutation with severe renal impairment (creatinine clearance 15-29 mL/min) dosed with ivosidenib to steady-state versus patients with normal renal function dosed with ivosidenib to steady-state. This may be performed as a substudy in the ongoing Phase 1 Study AG120-C-001, A Phase 1, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects with Advanced Hematologic Malignancies with an IDH1 Mutation. This trial should be designed and conducted in accordance with the FDA Guidance for Industry entitled Pharmacokinetics in Patients with Impaired Renal Function: Study Design, Data Analysis, and Impact on Dosing and Labeling."~"Delayed"~"The applicant requested revised milestones because of challenges with enrollment. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 10/08/2025."~9/30/2025 0:00:00~7/9/2026 0:00:00
383481~"CDER"~"NDA"~211230.00~"AXSOME MALTA LTD"~"Sunosi (solriamfetol)"~3/20/2019 0:00:00~"Original"~1~"3475-1"~"PMR"~"505 (o)(3)"~3475.00~1~"PMR 3475-1: A prospective, registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to solriamfetol during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Pending"~~9/30/2030 0:00:00~5/14/2026 0:00:00
383482~"CDER"~"NDA"~211230.00~"AXSOME MALTA LTD"~"Sunosi (solriamfetol)"~3/20/2019 0:00:00~"Original"~1~"3475-2"~"PMR"~"505 (o)(3)"~3475.00~2~"PMR 3475-2: An additional pregnancy study that uses a different design from the Pregnancy Registry (for example a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to solriamfetol during pregnancy compared to an unexposed control population."~"Delayed"~"The Study Completion and Final Report milestones were missed due to insufficient sample size accrual within the
original timeline. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 05/12/2026."~9/30/2025 0:00:00~5/14/2026 0:00:00
383483~"CDER"~"NDA"~211230.00~"AXSOME MALTA LTD"~"Sunosi (solriamfetol)"~3/20/2019 0:00:00~"Original"~2~"3475-1"~"PMR"~"505 (o)(3)"~3475.00~1~"PMR 3475-1: A prospective, registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to solriamfetol during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Pending"~~9/30/2030 0:00:00~5/14/2026 0:00:00
383484~"CDER"~"NDA"~211230.00~"AXSOME MALTA LTD"~"Sunosi (solriamfetol)"~3/20/2019 0:00:00~"Original"~2~"3475-2"~"PMR"~"505 (o)(3)"~3475.00~2~"PMR 3475-2: An additional pregnancy study that uses a different design from the Pregnancy Registry (for example a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to solriamfetol during pregnancy compared to an unexposed control population."~"Delayed"~"The Study Completion and Final Report milestones were missed due to insufficient sample size accrual within the
original timeline. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 05/12/2026."~9/30/2025 0:00:00~5/14/2026 0:00:00
383485~"CDER"~"NDA"~211241.00~"SENTYNL THERAPEUTICS INC"~"Zycubo (copper histidinate) for injection"~1/12/2026 0:00:00~"Original"~1~"4866-1"~"PMC"~"506B PMC"~4866.00~1~"PMC 4866-1: Conduct a clinical trial to assess serum copper and serum ceruloplasmin concentrations at the recommended dosages of copper histidinate in pediatric patients with Menkes disease."~"Pending"~~4/30/2031 0:00:00~
383486~"CDER"~"NDA"~211243.00~"JANSSEN PHARMACEUTICALS INC"~"Spravato (esketamine)"~3/5/2019 0:00:00~"Supplement"~4~"3915-4"~"PMR"~"Pediatric Research Equity Act"~3915.00~4~"PMR 3915-4: Conduct a double-blind, double-dummy, randomized, active-controlled study with open-label safety monitoring to week 29 in pediatric subjects with major depressive disorder ages 12 to <18."~"Delayed"~"final protocol submitted on 21 May 2025, after the milestone date of 3/2025."~3/31/2032 0:00:00~4/29/2026 0:00:00
383487~"CDER"~"NDA"~211280.00~"ELI LILLY AND CO"~"Reyvow (lasmiditan) "~10/11/2019 0:00:00~"Original"~1~"3728-1"~"PMR"~"Pediatric Research Equity Act"~3728.00~1~"PMR 3728-1: An open-label pharmacokinetics, safety, and tolerability study in pediatric migraine patients with body weight less than or equal to 40 kg. This study should identify doses to be used in the efficacy and long-term extension study for patients less than or equal to 40 kg."~"Fulfilled"~"Per FDA letter dated 09/11/2025, this PMR/PMC has been fulfilled."~10/31/2023 0:00:00~12/4/2025 0:00:00
383488~"CDER"~"NDA"~211280.00~"ELI LILLY AND CO"~"Reyvow (lasmiditan) "~10/11/2019 0:00:00~"Original"~1~"3728-2"~"PMR"~"Pediatric Research Equity Act"~3728.00~2~"PMR 3728-2: A randomized, double-blind, placebo-controlled efficacy and safety study under PREA to evaluate two doses of Reyvow (high-dose and low-dose) compared to placebo in the acute treatment of migraine in pediatric patients ages 6 to less than 18 years. This study is to be submitted as a special protocol assessment (SPA)."~"Terminated"~"The trial was terminated on 9/30/2025 because the applicant is permanently discontinuing Reyvow as they continue to prioritize
investment in new medicines and alternative treatments are generally available. The applicant reported that 8 patients remained in trial LAHV at the time of trial termination. Per the Applicant s ASR, the clinical study report will be submitted in a future submission."~5/31/2028 0:00:00~12/4/2025 0:00:00
383489~"CDER"~"NDA"~211280.00~"ELI LILLY AND CO"~"Reyvow (lasmiditan) "~10/11/2019 0:00:00~"Original"~1~"3728-3"~"PMR"~"Pediatric Research Equity Act"~3728.00~3~"PMR 3728-3: An open-label, long-term safety study under PREA in pediatric patients ages 6 to 18 years, for up to one year."~"Terminated"~"The trial was terminated on 9/30/2025 because the applicant is
permanently discontinuing Reyvow as they continue to prioritize
investment in new medicines and alternative treatments are generally available. The applicant reported that 40 patients remained in trial LAHW at the time of trial termination. Per the
Applicant s ASR, the clinical study report will be submitted in a future submission."~5/31/2028 0:00:00~12/4/2025 0:00:00
383490~"CDER"~"NDA"~209776.00~"REMPEX PHARMACEUTICALS INC A WHOLLY OWNED SUB OF MELINTA THERAPEUTICS LLC"~"Vabomere (Meropenem and Vaborbactam)"~8/29/2017 0:00:00~"Original"~1~"3248-3"~"PMR"~"Pediatric Research Equity Act"~3248.00~3~"PMR 3248-3: Conduct an open-label, active comparator study to evaluate the PK, safety, and tolerability of multiple doses of VABOMERE vs. comparator in neonates (less than or equal to 90 days of age) with late onset sepsis."~"Delayed"~"FDA Issued a Deferral Extension granted /Acknowledge revised PMR letter on 11/15/2024."~1/31/2027 0:00:00~10/24/2025 0:00:00
383491~"CDER"~"NDA"~209777.00~"PROTEGA PHARMACEUTICALS INC"~"RoxyBond (Oxycodone)"~4/20/2017 0:00:00~"Original"~1~"3204-4"~"PMR"~"505 (o)(3)"~3204.00~4~"PMR 3204-4: In order to provide meaningful baseline data to support the hypothesis-testing studies which will be required under a separate PMR in the future, conduct a  descriptive study that analyzes data on the following: 1) Utilization of ROXYBOND and selected comparators: Reports should  include nationally-projected quarterly dispensing data, overall and by age group and census region; AND 2) Abuse of ROXYBOND and related clinical outcomes. These studies should  utilize multiple data sources in different populations to establish the scope and patterns of abuse for ROXYBOND as well as mutually agreed-upon, selected  comparators to provide context.    Data should include route-specific abuse outcomes, be nationally representative or from multiple large geographic areas, and use meaningful measures of abuse.   Additional information, either qualitative or quantitative, from sources such as internet forums, spontaneous adverse event reporting, or small cohort studies may also be included to help better understand abuse of this drug, including routes and patterns of abuse in various populations.   Formal hypothesis testing is not necessary during this phase, but provide information on the precision of abuse-related outcome estimates (e.g., 95% confidence intervals for quarterly estimates) and calculate utilization adjusted outcome estimates where possible."~"Delayed"~"The final report submission milestone was missed due to low prescription volume not allowing for data collection and the FDA
revised the milestone to 12/2028."~6/30/2021 0:00:00~6/26/2026 0:00:00
383492~"CDER"~"NDA"~209803.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"Steglatro (ertugliflozin)"~12/19/2017 0:00:00~"Original"~1~"3311-1"~"PMR"~"Pediatric Research Equity Act"~3311.00~1~"PMR 3311-1: Conduct a 24-week, randomized, double-blind, placebo-controlled, parallel group study of the safety, efficacy, and pharmacokinetics (PK) of ertugliflozin as add-on to metformin background therapy for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 17 years (inclusive), followed by a 30-week doubleblind, controlled extension. Patients will be randomized to receive one of two doses of ertugliflozin or placebo once daily. The ertugliflozin doses will be determined using a population PK model derived from the Phase 3 program (in adult subjects) for ertugliflozin. As part of the pediatric study, sparse blood  samples for population PK and exposures-response analysis will be collected. An interim analysis of the PK data will be performed during this study to confirm  acceptable exposure to ertugliflozin with the selected doses."~"Submitted"~"The final report was submitted to FDA on 03/05/2026."~9/30/2026 0:00:00~2/17/2026 0:00:00
383493~"CDER"~"NDA"~209806.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"Segluromet (ertugliflozin and metformin hydrochloride)"~12/19/2017 0:00:00~"Original"~1~"3763-1"~"PMR"~"Pediatric Research Equity Act"~3763.00~1~"PMR 3763-1: Conduct a 24-week, randomized, double-blind, placebo-controlled, parallel group study of the safety, efficacy, and pharmacokinetics (PK) of ertugliflozin as add-on to metformin background therapy for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 17 years (inclusive), followed by a 30-week double-blind, controlled extension. Patients will be randomized to receive one of two doses of ertugliflozin or placebo once daily. The ertugliflozin doses will be determined using a population PK model derived from the Phase 3 program (in adult subjects) for ertugliflozin. As part of the pediatric study, sparse blood samples for population PK and exposures-response analysis will be collected. An interim analysis of the PK data will be performed during this study to confirm acceptable exposure to ertugliflozin with the selected doses."~"Submitted"~"The final report was submitted to FDA on 03/05/2026."~9/30/2026 0:00:00~2/17/2026 0:00:00
383494~"CDER"~"NDA"~209816.00~"PARATEK PHARMACEUTICALS INC"~"Nuzyra (omadacycline)"~10/2/2018 0:00:00~"Original"~1~"3487-1"~"PMR"~"Pediatric Research Equity Act"~3487.00~1~"PMR 3487-1: Conduct a single dose pharmacokinetic and safety study in children ages 8 to 17 years who are receiving antibacterial drug therapy for an infectious disease."~"Submitted"~"The final report was submitted to FDA on 01/12/2026."~5/31/2024 0:00:00~11/21/2025 0:00:00
383495~"CDER"~"NDA"~209816.00~"PARATEK PHARMACEUTICALS INC"~"Nuzyra (omadacycline)"~10/2/2018 0:00:00~"Original"~1~"3487-2"~"PMR"~"Pediatric Research Equity Act"~3487.00~2~"PMR 3487-2: Conduct an active-controlled safety study in children 8-17 years who have acute bacterial skin and skin structure infections."~"Delayed"~"Original draft protocol and final protocol dates were missed. A deferral extension granted letter was issued on 5/5/2022. The draft protocol, final protocol, and study completion milestone dates were also revised. A deferral extension denial letter was issued on 12/20/2023 because it was premature to revise the date."~11/30/2026 0:00:00~11/21/2025 0:00:00
383496~"CDER"~"NDA"~209816.00~"PARATEK PHARMACEUTICALS INC"~"Nuzyra (omadacycline)"~10/2/2018 0:00:00~"Original"~1~"3487-3"~"PMR"~"Pediatric Research Equity Act"~3487.00~3~"PMR 3487-3: Conduct an active-controlled safety study in children 8-17 years who have community-acquired bacterial pneumonia."~"Delayed"~"Original Final Report Due Date: 03/31/2026; Deferral Extension granted per FDA letter dated 04/14/2023. The draft protocol, final protocol and completion milestones were also revised."~3/31/2027 0:00:00~11/21/2025 0:00:00
383497~"CDER"~"NDA"~209816.00~"PARATEK PHARMACEUTICALS INC"~"Nuzyra (omadacycline)"~10/2/2018 0:00:00~"Original"~1~"3487-4"~"PMR"~"505 (o)(3)"~3487.00~4~"PMR 3487-4: Conduct an active-controlled safety and efficacy study in adults with community-acquired bacterial pneumonia"~"Fulfilled"~~4/30/2023 0:00:00~11/21/2025 0:00:00
383498~"CDER"~"NDA"~209817.00~"PARATEK PHARMACEUTICALS INC"~"Nuzyra (omadacycline)"~10/2/2018 0:00:00~"Original"~1~"3487-1"~"PMR"~"Pediatric Research Equity Act"~3487.00~1~"PMR 3487-1: Conduct a single dose pharmacokinetic and safety study in children ages 8 to 17 years who are receiving antibacterial drug therapy for an infectious disease."~"Submitted"~"The final report was submitted to FDA on 01/12/2026."~5/31/2024 0:00:00~11/21/2025 0:00:00
383499~"CDER"~"NDA"~209817.00~"PARATEK PHARMACEUTICALS INC"~"Nuzyra (omadacycline)"~10/2/2018 0:00:00~"Original"~1~"3487-2"~"PMR"~"Pediatric Research Equity Act"~3487.00~2~"PMR 3487-2: Conduct an active-controlled safety study in children 8-17 years who have acute bacterial skin and skin structure infections."~"Delayed"~"Original draft protocol and final protocol dates were missed. A deferral extension granted letter was issued on 5/5/2022. The draft protocol, final protocol, and study completion milestone dates were also revised. A deferral extension denial letter was issued on 12/20/2023 because it was premature to revise the date."~11/30/2026 0:00:00~11/21/2025 0:00:00
383500~"CDER"~"NDA"~209817.00~"PARATEK PHARMACEUTICALS INC"~"Nuzyra (omadacycline)"~10/2/2018 0:00:00~"Original"~1~"3487-3"~"PMR"~"Pediatric Research Equity Act"~3487.00~3~"PMR 3487-3: Conduct an active-controlled safety study in children 8-17 years who have community-acquired bacterial pneumonia."~"Delayed"~"Original Final Report Due Date: 03/31/2026; Deferral Extension granted per FDA letter dated 04/14/2023. The draft protocol, final protocol and completion milestones were also revised."~3/31/2027 0:00:00~11/21/2025 0:00:00
383501~"CDER"~"NDA"~209817.00~"PARATEK PHARMACEUTICALS INC"~"Nuzyra (omadacycline)"~10/2/2018 0:00:00~"Original"~1~"3487-4"~"PMR"~"505 (o)(3)"~3487.00~4~"PMR 3487-4: Conduct an active-controlled safety and efficacy study in adults with community-acquired bacterial pneumonia"~"Fulfilled"~~4/30/2023 0:00:00~11/21/2025 0:00:00
383502~"CDER"~"NDA"~209844.00~"CHARTWELL PHARMA NDA B2 HOLDINGS"~"LymePak (doxycycline hyclate) "~6/15/2018 0:00:00~"Original"~1~"3406-1"~"PMR"~"Pediatric Research Equity Act"~3406.00~1~"PMR 3406-1: An evaluation of LYMEPAK tablets to validate dosing and formulation for use in pediatric patients 8 years of age and older who weigh less than 45 kg."~"Delayed"~"A notification of noncompliance with PREA letter was issued on 11-17-2021."~10/31/2021 0:00:00~5/15/2025 0:00:00
383503~"CDER"~"NDA"~209863.00~"ANTARES PHARMA INC"~"Xyosted (Testosterone Enanthate)"~9/28/2018 0:00:00~"Original"~1~"3493-3"~"PMR"~"Pediatric Research Equity Act"~3493.00~3~"PMR 3493-3: A trial of testosterone replacement therapy in pediatric males ages 12 years to less than 18 years of age for conditions associated with a deficiency or absence of endogenous testosterone due to primary hypogonadism or hypogonadotropic hypogonadism."~"Delayed"~"The Final Protocol was submitted after the original final protocol submission milestone due to continued discussions with the FDA. The final protocol was acknowledged on 10/8/2025. Per the applicant s Annual Status Report, five subjects are enrolled in the trial."~10/31/2028 0:00:00~11/25/2025 0:00:00
383504~"CDER"~"NDA"~209884.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mayzent (siponimod)"~3/26/2019 0:00:00~"Original"~1~"4758-1"~"PMR"~"505 (o)(3)"~4758.00~1~"PMR 4758-1: Conduct an observational study to characterize patients exposed to Mayzent who develop PML in terms of age, duration of exposure to Mayzent, prior immunosuppressant use, lymphopenia, and anti-JCV antibody status. This study should be of at least 15 years duration to assess risk factors for the development of PML among patients with multiple sclerosis exposed to Mayzent. Potential risk factors to be assessed should include age, duration of exposure to Mayzent, prior immunosuppressant use, lymphopenia, and anti-JCV antibody status. The study should also determine the incidence of PML among patients exposed to Mayzent, both overall and by each potential risk factor."~"Pending"~~7/31/2042 0:00:00~5/20/2026 0:00:00
383505~"CDER"~"NDA"~209884.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mayzent (siponimod)"~3/26/2019 0:00:00~"Original"~1~"3591-6"~"PMC"~"506B PMC"~3591.00~6~"PMC 3591-6: Establish an in-vitro diagnostic device to guide the use of siponimod in patients with relapsing forms of multiple sclerosis. The device should detect, at a minimum, the presence of the *2 and *3 alleles in cytochrome P450 2C9 (CYP2C9). The device should detect patients homozygous for the CYP2C9 *3/*3 genotype with statistical confidence."~"Delayed"~"The applicant requested revised milestones because submission of the draft validation protocols by the in-vitro diagnostic (IVD) device partner per the current PMC milestone schedule is not feasible. Revised milestones were acknowledged in a letter dated 03/25/2024."~12/31/2023 0:00:00~5/20/2026 0:00:00
383506~"CDER"~"NDA"~209884.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mayzent (siponimod)"~3/26/2019 0:00:00~"Original"~1~"3591-8"~"PMR"~"Pediatric Research Equity Act"~3591.00~8~"PMR 3591-8: Conduct a two-part study of Mayzent (siponimod) in pediatric patients with relapsing forms of multiple sclerosis (RMS) at least 10 years and less than 18 years of age. Part A is an open-label study of the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Mayzent (siponimod) in pediatric patients weighing 40 kg or less. The objective of Part A is to determine titration and maintenance doses of Mayzent (siponimod) that will result in PK and PD effects that are comparable to those of the 5-day titration and the 1- or 2-mg genotype-based maintenance doses administered to adult patients. Part B is a randomized, blinded, non-inferiority trial with Gilenya (fingolimod) as a comparator."~"Ongoing"~"Original Final Report Due Date: 03/31/2026; Deferral Extension granted per FDA letter dated 06/06/2025. The study completion milestone was also revised. Per the applicant, they have enrolled patients in their study."~5/31/2027 0:00:00~5/20/2026 0:00:00
383507~"CDER"~"NDA"~209884.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mayzent (siponimod)"~3/26/2019 0:00:00~"Original"~1~"3591-9"~"PMR"~"505 (o)(3)"~3591.00~9~"PMR 3591-9: Conduct a worldwide, descriptive study that collects prospective and retrospective data in women exposed to siponimod during
pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse
effects on the infant. Infant outcomes will be assessed through at least the first year of life. The maximum study duration will be specified in the protocol."~"Pending"~~6/30/2031 0:00:00~5/20/2026 0:00:00
383508~"CDER"~"NDA"~209899.00~"BRISTOL MYERS SQUIBB CO"~"Zeposia (Ozanimod)"~3/25/2020 0:00:00~"Original"~1~"4757-1"~"PMR"~"505 (o)(3)"~4757.00~1~"PMR 4757-1: Conduct an observational study to characterize patients exposed to Zeposia who develop PML in terms of age, duration of exposure to
Zeposia, prior immunosuppressant use, lymphopenia, and anti-JCV antibody status. This study should be of at least 15 years duration to assess risk factors for the development of PML among patients with multiple sclerosis exposed to Zeposia. Potential risk factors to be assessed should include age, duration of exposure to Zeposia, prior immunosuppressant use, lymphopenia, and anti-JCV antibody status. The study should also determine the incidence of PML among patients exposed to Zeposia, both overall and by each potential risk factor."~"Pending"~~7/31/2042 0:00:00~5/22/2026 0:00:00
383509~"CDER"~"NDA"~209899.00~"BRISTOL MYERS SQUIBB CO"~"Zeposia (Ozanimod)"~3/25/2020 0:00:00~"Original"~1~"3809-3"~"PMR"~"505 (o)(3)"~3809.00~3~"PMR 3809-3: Prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with multiple sclerosis exposed to Zeposia (ozanimod) during pregnancy with two unexposed control populations: one consisting of women with multiple sclerosis who have not been exposed to Zeposia (ozanimod) before or during pregnancy and the other consisting of women without multiple sclerosis. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~6/30/2033 0:00:00~5/22/2026 0:00:00
383510~"CDER"~"NDA"~209899.00~"BRISTOL MYERS SQUIBB CO"~"Zeposia (Ozanimod)"~3/25/2020 0:00:00~"Original"~1~"3809-4"~"PMR"~"505 (o)(3)"~3809.00~4~"PMR 3809-4: A pregnancy outcomes study using a different study design than provided for in PMR 3809-3 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case-control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Zeposia (ozanimod) during pregnancy compared to an unexposed control population."~"Ongoing"~~6/30/2033 0:00:00~5/22/2026 0:00:00
383511~"CDER"~"NDA"~209899.00~"BRISTOL MYERS SQUIBB CO"~"Zeposia (Ozanimod)"~3/25/2020 0:00:00~"Original"~1~"3809-7"~"PMR"~"Pediatric Research Equity Act"~3809.00~7~"PMR 3809-7: A two-part study of Zeposia (ozanimod) in pediatric patients with relapsing forms of multiple sclerosis (RMS) at least 10 years and less than 18 years of age. Part A is an open-label study of the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Zeposia (ozanimod) in pediatric patients weighing 40 kg or less. The objective of Part A is to determine titration and maintenance doses of Zeposia (ozanimod) that will result in PK and PD effects that are comparable to those of the 8-day titration administered to adult patients. Part B is a randomized, double-blind, parallel-group study to evaluate the efficacy and safety of Zeposia (ozanimod) compared to an appropriate comparator."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 1/3/2024. Per the applicant s Annual Status Report, 194 pediatric participants are planned, and no subjects have enrolled as of the end of the current reporting period."~3/31/2034 0:00:00~5/22/2026 0:00:00
383512~"CDER"~"NDA"~209899.00~"BRISTOL MYERS SQUIBB CO"~"Zeposia (Ozanimod)"~3/25/2020 0:00:00~"Supplement"~1~"4066-1"~"PMR"~"505 (o)(3)"~4066.00~1~"PMR 4066-1: An international, prospective, registry-based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of females exposed to ZEPOSIA (ozanimod) during pregnancy with women exposed to any other ulcerative colitis therapy during pregnancy and an unexposed comparator population. External disease matched comparators and use of existing disease registries can be considered. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life. This study can be conducted as part of the ongoing study under NDA 209899 PMR 3809-3."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 11/6/2025."~6/30/2033 0:00:00~5/22/2026 0:00:00
383513~"CDER"~"NDA"~209899.00~"BRISTOL MYERS SQUIBB CO"~"Zeposia (Ozanimod)"~3/25/2020 0:00:00~"Supplement"~1~"4066-2"~"PMR"~"505 (o)(3)"~4066.00~2~"PMR 4066-2: A pregnancy outcomes study using a different study design than provided for in PMR 4066-1 (for example, a retrospective cohort study using claims or electronic medical record data) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in females exposed to ZEPOSIA (ozanimod) during pregnancy compared to an unexposed control population. This study can be conducted as part of the ongoing study under NDA 209899 PMR 3809-4.
"~"Ongoing"~~6/30/2033 0:00:00~5/22/2026 0:00:00
383514~"CDER"~"NDA"~209899.00~"BRISTOL MYERS SQUIBB CO"~"Zeposia (Ozanimod)"~3/25/2020 0:00:00~"Supplement"~1~"4066-3"~"PMR"~"505 (o)(3)"~4066.00~3~"PMR 4066-3: A lactation study (milk only) in lactating women who have received therapeutic doses of ZEPOSIA (ozanimod) using a validated assay to assess concentrations of ZEPOSIA (ozanimod) and its major metabolites in breast milk, and effects on the breastfed infant.
"~"Released"~~9/30/2025 0:00:00~5/22/2026 0:00:00
383515~"CDER"~"NDA"~209899.00~"BRISTOL MYERS SQUIBB CO"~"Zeposia (Ozanimod)"~3/25/2020 0:00:00~"Supplement"~1~"4066-4"~"PMC"~"506B PMC"~4066.00~4~"PMC 4066-4: A one year, randomized, blinded trial to evaluate the safety, efficacy, and pharmacokinetics of ZEPOSIA (ozanimod) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis."~"Ongoing"~~6/30/2026 0:00:00~5/22/2026 0:00:00
383516~"CDER"~"NDA"~209899.00~"BRISTOL MYERS SQUIBB CO"~"Zeposia (Ozanimod)"~3/25/2020 0:00:00~"Supplement"~1~"4066-5"~"PMC"~"506B PMC"~4066.00~5~"PMC 4066-5: A long term extension study to evaluate the long-term safety of ZEPOSIA (ozanimod) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis who participated in postmarketing commitment Study 3. This study can be conducted as part of postmarketing commitment study 4066-4."~"Ongoing"~~4/30/2031 0:00:00~5/22/2026 0:00:00
383517~"CDER"~"NDA"~209899.00~"BRISTOL MYERS SQUIBB CO"~"Zeposia (Ozanimod)"~3/25/2020 0:00:00~"Supplement"~1~"4066-6"~"PMR"~"505 (o)(3)"~4066.00~6~"PMR 4066-6: Perform a lactation study (milk and plasma) in lactating women who have received Zeposia (ozanimod) to assess concentrations of Zeposia (ozanimod) and its major metabolites in breast milk and plasma using a validated assay."~"Pending"~~3/31/2029 0:00:00~5/22/2026 0:00:00
383518~"CDER"~"NDA"~209935.00~"NOVARTIS PHARMACEUTICALS CORP"~"Kisqali Femara Co-Pack (Letrozole and Ribociclib)"~5/4/2017 0:00:00~"Supplement"~27~"4705-1"~"PMR"~"505 (o)(3)"~4705.00~1~"PMR 4705-1: Conduct a carcinogenicity study in mice to evaluate the potential serious risk of carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Pending"~~8/31/2029 0:00:00~5/7/2026 0:00:00
383519~"CDER"~"NDA"~209935.00~"NOVARTIS PHARMACEUTICALS CORP"~"Kisqali Femara Co-Pack (Letrozole and Ribociclib)"~5/4/2017 0:00:00~"Supplement"~27~"4705-2"~"PMC"~"506B PMC"~4705.00~2~"PMC 4705-2: Conduct an integrated analysis from ongoing, completed, or planned (e.g., CLEE011O12301C/NATALEE/NCT03701334 and adjuvant WIDER/NCT05827081) clinical trials and other potential data sources as appropriate, enrolling sufficient representation of racial and ethnic minority patients to reflect the U.S. population of patients with hormone receptor (HR)-positive, human epidermal growth factor receptor-2 (HER2)-negative early breast cancer, and further characterize the efficacy and safety of the addition of ribociclib to an aromatase inhibitor in the adjuvant setting in these patient subgroups. This analysis should include, but is not limited to, patients who identify as Black or African-American, and should be reflective of the incidence of early breast cancer in the U.S. in these patient populations. The analyses should support comparable efficacy and safety between the aforementioned populations and White or Caucasian patients"~"Pending"~~3/31/2030 0:00:00~5/7/2026 0:00:00
383520~"CDER"~"NDA"~209935.00~"NOVARTIS PHARMACEUTICALS CORP"~"Kisqali Femara Co-Pack (Letrozole and Ribociclib)"~5/4/2017 0:00:00~"Supplement"~27~"4705-3"~"PMC"~"506B PMC"~4705.00~3~"PMC 4705-3: Complete the ongoing clinical trial CLEE011O12301C (NATALEE/NCT03701334) to provide all additional overall survival (OS) analyses as prespecified in the protocol and the statistical analysis plan, including OS at the time of trial completion."~"Ongoing"~~12/31/2026 0:00:00~5/7/2026 0:00:00
383521~"CDER"~"NDA"~209939.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"Prevymis (letermovir)"~11/8/2017 0:00:00~"Supplement"~13~"4692-1"~"PMR"~"505 (o)(3)"~4692.00~1~"PMR 4692-1: Conduct phenotypic analysis of letermovir against human cytomegalovirus (HCMV) mutants carrying substitutions (1) pUL51 P91H and (2) pUL56 S229Y + pUL56 M329I with and without pUL89 D344Y. The analysis should include previously identified substitutions with a range of susceptibilities from low fold change (e.g. pUL56 L257I) to high fold change (e.g. pUL56 C325Y) as references."~"Fulfilled"~~4/30/2026 0:00:00~12/17/2025 0:00:00
383522~"CDER"~"NDA"~209939.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"Prevymis (letermovir)"~11/8/2017 0:00:00~"Supplement"~13~"4693-1"~"PMC"~"506B PMC"~4693.00~1~"PMC 4693-1: Conduct a study to determine the pharmacokinetics and safety of letermovir in kidney transplant recipients less than 18 years of age and weighing less than 40 kg."~"Pending"~~1/31/2029 0:00:00~12/17/2025 0:00:00
383523~"CDER"~"NDA"~209939.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"Prevymis (letermovir)"~11/8/2017 0:00:00~"Supplement"~16~"4692-1"~"PMR"~"505 (o)(3)"~4692.00~1~"PMR 4692-1: Conduct phenotypic analysis of letermovir against human cytomegalovirus (HCMV) mutants carrying substitutions (1) pUL51 P91H and (2) pUL56 S229Y + pUL56 M329I with and without pUL89 D344Y. The analysis should include previously identified substitutions with a range of susceptibilities from low fold change (e.g. pUL56 L257I) to high fold change (e.g. pUL56 C325Y) as references."~"Fulfilled"~~4/30/2026 0:00:00~12/17/2025 0:00:00
383524~"CDER"~"NDA"~209939.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"Prevymis (letermovir)"~11/8/2017 0:00:00~"Supplement"~16~"4693-1"~"PMC"~"506B PMC"~4693.00~1~"PMC 4693-1: Conduct a study to determine the pharmacokinetics and safety of letermovir in kidney transplant recipients less than 18 years of age and weighing less than 40 kg."~"Pending"~~1/31/2029 0:00:00~12/17/2025 0:00:00
383525~"CDER"~"NDA"~209940.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"Prevymis (letermovir)"~11/8/2017 0:00:00~"Supplement"~12~"4693-1"~"PMC"~"506B PMC"~4693.00~1~"PMC 4693-1: Conduct a study to determine the pharmacokinetics and safety of letermovir in kidney transplant recipients less than 18 years of age and weighing less than 40 kg."~"Pending"~~1/31/2029 0:00:00~12/17/2025 0:00:00
383526~"CDER"~"NDA"~209940.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"Prevymis (letermovir)"~11/8/2017 0:00:00~"Supplement"~12~"4692-1"~"PMR"~"505 (o)(3)"~4692.00~1~"PMR 4692-1: Conduct phenotypic analysis of letermovir against human cytomegalovirus (HCMV) mutants carrying substitutions (1) pUL51 P91H and (2) pUL56 S229Y + pUL56 M329I with and without pUL89 D344Y. The analysis should include previously identified substitutions with a range of susceptibilities from low fold change (e.g. pUL56 L257I) to high fold change (e.g. pUL56 C325Y) as references."~"Fulfilled"~~4/30/2026 0:00:00~12/17/2025 0:00:00
383527~"CDER"~"NDA"~209940.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"Prevymis (letermovir)"~11/8/2017 0:00:00~"Supplement"~16~"4692-1"~"PMR"~"505 (o)(3)"~4692.00~1~"PMR 4692-1: Conduct phenotypic analysis of letermovir against human cytomegalovirus (HCMV) mutants carrying substitutions (1) pUL51 P91H and (2) pUL56 S229Y + pUL56 M329I with and without pUL89 D344Y. The analysis should include previously identified substitutions with a range of susceptibilities from low fold change (e.g. pUL56 L257I) to high fold change (e.g. pUL56 C325Y) as references."~"Fulfilled"~~4/30/2026 0:00:00~12/17/2025 0:00:00
383528~"CDER"~"NDA"~209940.00~"MERCK SHARP AND DOHME LLC A SUB OF MERCK AND CO INC"~"Prevymis (letermovir)"~11/8/2017 0:00:00~"Supplement"~16~"4693-1"~"PMC"~"506B PMC"~4693.00~1~"PMC 4693-1: Conduct a study to determine the pharmacokinetics and safety of letermovir in kidney transplant recipients less than 18 years of age and weighing less than 40 kg."~"Pending"~~1/31/2029 0:00:00~12/17/2025 0:00:00
383529~"CDER"~"NDA"~209963.00~"LXO IRELAND DESIGNATED ACTIVITY CO"~"GOPRELTO (cocaine hydrochloride)"~12/14/2017 0:00:00~"Original"~1~"3241-10"~"PMR"~"Pediatric Research Equity Act"~3241.00~10~"PMR 3241-10: Conduct a multicenter trial to evaluate the pharmacokinetic and safety profiles of a single topical administration of GOPRELTO for the induction of local anesthesia of the mucous membranes when performing diagnostic procedures and surgeries on or through the nasal cavities in pediatric subjects 12 years of age to less than 17 years of age."~"Delayed"~"Created to replace PMR 3241-3 and -4. The study has been initiated. The study completion milestone had been missed."~12/31/2026 0:00:00~2/9/2026 0:00:00
383530~"CDER"~"NDA"~209988.00~"SCPHARMACEUTICALS INC A WHOLLY OWNED SUB OF MANNKIND CORP"~"Furoscix Infusor (furosemide) and Furoscix Readyflow (furosemide) injection"~10/7/2022 0:00:00~"Original"~1~"4350-1"~"PMR"~"Pediatric Research Equity Act"~4350.00~1~"PMR 4350-1: Perform an extrapolation analysis based on allometric scaling from adults with heart failure to support the efficacy for children with heart failure 12 to 17 years of age who weigh at least 42.5 kg."~"Fulfilled"~"Per FDA letter dated 12/22/2025, this PMR/PMC has been fulfilled."~3/31/2023 0:00:00~12/5/2025 0:00:00
383531~"CDER"~"NDA"~209988.00~"SCPHARMACEUTICALS INC A WHOLLY OWNED SUB OF MANNKIND CORP"~"Furoscix Infusor (furosemide) and Furoscix Readyflow (furosemide) injection"~10/7/2022 0:00:00~"Supplement"~1~"4675-1"~"PMR"~"Pediatric Research Equity Act"~4675.00~1~"PMR 4675-1: Submit an efficacy supplement that includes an extrapolation analysis based on allometric scaling from adults with heart failure to support the efficacy for children with heart failure 12 to 17 years of age who weigh at least 42.5 kg."~"Fulfilled"~"Per FDA letter dated 12/22/2025, this PMR/PMC has been fulfilled."~2/28/2025 0:00:00~12/5/2025 0:00:00
383532~"CDER"~"NDA"~210136.00~"BRAEBURN INC"~"Brixadi (Buprenorphine Hydrochloride)"~5/23/2023 0:00:00~"Original"~1~"4439-1"~"PMR"~"505 (o)(3)"~4439.00~1~"PMR 4439-1: Conduct a study to quantitate the level of elemental impurities that could be leached from the container closure system over the course of the shelf life and provide a toxicological risk assessment to justify the safety of the levels detected."~"Submitted"~~3/31/2025 0:00:00~7/23/2025 0:00:00
383533~"CDER"~"NDA"~210136.00~"BRAEBURN INC"~"Brixadi (Buprenorphine Hydrochloride)"~5/23/2023 0:00:00~"Original"~1~"4439-2"~"PMR"~"505 (o)(3)"~4439.00~2~"PMR 4439-2: Conduct a study using validated methods to confirm the identity of the unspecified alcohol, the unidentified compound with relative retention time (RRT) of 13.67 minutes, the unknown compound containing silicon with RRT of 17.63 minutes, and the unknown compound with fragment ion of C9H15O with RRT of 3.14 minutes that were detected in your leachable studies above the safety concern threshold of 5 mcg/day and the levels of the identified leachables 4-hydroxy-4-methyl-2-pentanone and hexadecamethyl cyclooctasiloxane, and provide a toxicological risk assessment for each of these compounds and any other compounds detected at =5 mcg/day."~"Delayed"~"The Study/Trial Completion milestone was missed due to continued
discussions with the Agency."~3/31/2026 0:00:00~7/23/2025 0:00:00
383534~"CDER"~"NDA"~210136.00~"BRAEBURN INC"~"Brixadi (Buprenorphine Hydrochloride)"~5/23/2023 0:00:00~"Original"~1~"4439-4"~"PMR"~"505 (o)(3)"~4439.00~4~"PMR 4439-4: Conduct a clinical trial to evaluate precipitated withdrawal when BRIXADI is initiated at full blocking weekly doses of 24 mg and 32 mg. Prespecify case definitions of precipitated withdrawal, lack of tolerability, and dose inadequacy for the purpose of quantifying the risks of a rapid initiation of BRIXADI."~"Delayed"~"The Final Protocol milestone was missed due to continued discussions with the Agency. Current enrollment is 60 participants (30 in each arm)."~10/31/2027 0:00:00~7/23/2025 0:00:00
383535~"CDER"~"NDA"~210136.00~"BRAEBURN INC"~"Brixadi (Buprenorphine Hydrochloride)"~5/23/2023 0:00:00~"Original"~1~"4439-5"~"PMR"~"505 (o)(3)"~4439.00~5~"PMR 4439-5: Conduct a clinical trial to evaluate precipitated withdrawal when BRIXADI is initiated at full blocking monthly doses of 96 mg and 128 mg. Prespecify case definitions of precipitated withdrawal, lack of tolerability, and dose
inadequacy for the purpose of quantifying the risks of a rapid initiation of BRIXADI."~"Delayed"~"The Final Protocol milestone was missed due to continued discussions with the Agency. Current enrollment is 200 participants (100 in each arm)."~10/31/2027 0:00:00~7/23/2025 0:00:00
383536~"CDER"~"NDA"~210166.00~"TAKEDA PHARMACEUTICALS USA INC"~"Motegrity (Prucalopride Succinate)"~12/14/2018 0:00:00~"Original"~1~"3529-3"~"PMR"~"505 (o)(3)"~3529.00~3~"PMR 3529-3: A prospective, registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to Motegrity (prucalopride) during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~6/30/2026 0:00:00~2/6/2026 0:00:00
383537~"CDER"~"NDA"~210166.00~"TAKEDA PHARMACEUTICALS USA INC"~"Motegrity (Prucalopride Succinate)"~12/14/2018 0:00:00~"Original"~1~"3529-4"~"PMR"~"505 (o)(3)"~3529.00~4~"PMR 3529-4: An additional pregnancy study that uses a different design from the Pregnancy Registry (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to Motegrity (prucalopride) during pregnancy compared to an unexposed control population."~"Ongoing"~~6/30/2026 0:00:00~2/6/2026 0:00:00
383538~"CDER"~"NDA"~210166.00~"TAKEDA PHARMACEUTICALS USA INC"~"Motegrity (Prucalopride Succinate)"~12/14/2018 0:00:00~"Original"~1~"3529-5"~"PMR"~"505 (o)(3)"~3529.00~5~"PMR 3529-5: Perform a milk only lactation trial in lactating women who have received therapeutic doses of Motegrity (prucalopride) using a validated assay to assess concentrations of prucalopride in breast milk and the effects on the breastfed infant."~"Delayed"~"The study has enrolled 12 subjects. The final report submission milestone was missed. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 11/26/2024."~8/31/2024 0:00:00~2/6/2026 0:00:00
383539~"CDER"~"NDA"~210251.00~"GILEAD SCIENCES INC"~"Biktarvy (bictegravir, emtricitabine, and tenofovir alafenamide)"~2/7/2018 0:00:00~"Original"~1~"3910-2"~"PMC"~"506B PMC"~3910.00~2~"PMC 3910-2: Conduct a trial in HIV-1 infected pediatric patients who are at least 4 weeks of age and weighing 3 to < 14 kg to assess the pharmacokinetics, safety and tolerability, and antiviral activity of bictegravir/emtricitabine/tenofovir alafenamide. Trial participants must be monitored for a minimum of 24 weeks to assess safety and durability of antiviral response."~"Submitted"~~1/31/2026 0:00:00~4/1/2026 0:00:00
383540~"CDER"~"NDA"~210251.00~"GILEAD SCIENCES INC"~"Biktarvy (bictegravir, emtricitabine, and tenofovir alafenamide)"~2/7/2018 0:00:00~"Original"~1~"3322-4"~"PMC"~"506B PMC"~3322.00~4~"PMC 3322-4: Conduct a trial to evaluate the pharmacokinetics and safety of bictegravir/emtricitabine/tenofovir alafenamide (B/F/TAF) in neonates (birth to less than 4 weeks of age) who are at risk for perinatal HIV-1 infection."~"Ongoing"~~9/30/2028 0:00:00~4/1/2026 0:00:00
383541~"CDER"~"NDA"~210296.00~"BANNER LIFE SCIENCES LLC"~"Bafiertam (monomethyl fumarate)"~4/28/2020 0:00:00~"Original"~1~"3840-3"~"PMR"~"505 (o)(3)"~3840.00~3~"PMR 3840-3: Prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with multiple sclerosis exposed to Bafiertam (monomethyl fumarate) during pregnancy with two unexposed control populations: one consisting of women with multiple sclerosis who have not been exposed to Bafiertam (monomethyl fumarate) before or during pregnancy and the other consisting of women without multiple sclerosis. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small for gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Released"~~6/30/2033 0:00:00~6/8/2026 0:00:00
383542~"CDER"~"NDA"~210296.00~"BANNER LIFE SCIENCES LLC"~"Bafiertam (monomethyl fumarate)"~4/28/2020 0:00:00~"Original"~1~"3840-4"~"PMR"~"505 (o)(3)"~3840.00~4~"PMR 3840-4: A pregnancy outcomes study using a different study design than provided for in PMR 3840-3 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case-control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Bafiertam (monomethyl fumarate) during pregnancy compared to an unexposed control population."~"Released"~~6/30/2033 0:00:00~6/8/2026 0:00:00
383543~"CDER"~"NDA"~210296.00~"BANNER LIFE SCIENCES LLC"~"Bafiertam (monomethyl fumarate)"~4/28/2020 0:00:00~"Original"~1~"3840-5"~"PMR"~"Pediatric Research Equity Act"~3840.00~5~"PMR 3840-5: A two-part study of Bafiertam (monomethyl fumarate) in pediatric patients with relapsing forms of multiple sclerosis (RMS) at least 10 years and less than 18 years of age. Part A is an open-label study of the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Bafiertam (monomethyl fumarate) in pediatric patients weighing 40 kg or less. The objective of Part A is to determine titration and maintenance doses of Bafiertam (monomethyl fumarate) that will result in PK and PD effects that are comparable to those of the 8-day titration administered to adult patients. Part B is a randomized, double-blind, parallel-group study in pediatric patients weighing greater than 40 kg to evaluate the efficacy and safety of Bafiertam (monomethyl fumarate) compared to an appropriate comparator."~"Delayed"~"Deferral Extension Requested 04/10/2026. Denied per FDA letter dated 05/22/2026."~4/30/2033 0:00:00~6/8/2026 0:00:00
383544~"CDER"~"NDA"~210303.00~"CIPLA USA INC"~"Zemdri (Plazomicin)"~6/25/2018 0:00:00~"Original"~1~"3393-6"~"PMR"~"Pediatric Research Equity Act"~3393.00~6~"PMR 3393-6: Conduct a randomized active-controlled pharmacokinetic and safety trial of plazomicin in children from birth to less than 18 years of age with complicated urinary tract infection including acute pyelonephritis; the dose used in the trial will be based on a lead-in single dose pharmacokinetic and safety evaluation."~"Delayed"~"Original study completion due date was 12-2023. A deferral extension granted letter was issued on 10-23-2023."~6/30/2027 0:00:00~8/22/2025 0:00:00
383545~"CDER"~"NDA"~210365.00~"JAZZ PHARMACEUTICALS RESEARCH UK LTD"~"Epidiolex (cannabidiol)"~6/25/2018 0:00:00~"Original"~1~"3429-1"~"PMR"~"505 (o)(3)"~3429.00~1~"PMR 3429-1: An embryofetal development study of 7-COOH-cannabidiol in rat."~"Fulfilled"~~4/30/2020 0:00:00~8/20/2025 0:00:00
383546~"CDER"~"NDA"~210365.00~"JAZZ PHARMACEUTICALS RESEARCH UK LTD"~"Epidiolex (cannabidiol)"~6/25/2018 0:00:00~"Original"~1~"3429-2"~"PMR"~"505 (o)(3)"~3429.00~2~"PMR 3429-2: A pre- and postnatal development study of 7-COOH-cannabidiol in rat."~"Fulfilled"~~4/30/2020 0:00:00~8/20/2025 0:00:00
383547~"CDER"~"NDA"~210365.00~"JAZZ PHARMACEUTICALS RESEARCH UK LTD"~"Epidiolex (cannabidiol)"~6/25/2018 0:00:00~"Original"~1~"3429-3"~"PMR"~"505 (o)(3)"~3429.00~3~"PMR 3429-3: A juvenile animal toxicology study of 7-COOH-cannabidiol in rat."~"Fulfilled"~~4/30/2020 0:00:00~8/20/2025 0:00:00
383548~"CDER"~"NDA"~210365.00~"JAZZ PHARMACEUTICALS RESEARCH UK LTD"~"Epidiolex (cannabidiol)"~6/25/2018 0:00:00~"Original"~1~"3429-5"~"PMR"~"505 (o)(3)"~3429.00~5~"PMR 3429-5: A 2-year carcinogenicity study of cannabidiol and 7-COOH-cannabidiol, both directly administered, in rat."~"Submitted"~~2/28/2023 0:00:00~8/20/2025 0:00:00
383549~"CDER"~"NDA"~210365.00~"JAZZ PHARMACEUTICALS RESEARCH UK LTD"~"Epidiolex (cannabidiol)"~6/25/2018 0:00:00~"Original"~1~"3429-6"~"PMR"~"505 (o)(3)"~3429.00~6~"PMR 3429-6: Assess whether the effect of Epidiolex on serum creatinine reflects an effect on glomerular filtration rate."~"Fulfilled"~~3/31/2020 0:00:00~8/20/2025 0:00:00
383550~"CDER"~"NDA"~210365.00~"JAZZ PHARMACEUTICALS RESEARCH UK LTD"~"Epidiolex (cannabidiol)"~6/25/2018 0:00:00~"Original"~1~"3429-7"~"PMR"~"505 (o)(3)"~3429.00~7~"PMR 3429-7: Assess the potential for chronic liver injury with Epidiolex, with evaluation including physical exam, serum/blood biomarkers, and other noninvasive measures of liver fibrosis, such as MRI or ultrasound-based elastography. Patients should be evaluated yearly for five years."~"Delayed"~"The Final Protocol milestone was missed due to continued discussion with the Agency on the protocol. FDA determined that there was sufficient justification for the anticipated delay of the original Study Completion and Final Report Submission milestones. FDA acknowledged proposed revised milestones with sufficient justification in a letter dated 02/20/2024."~11/30/2027 0:00:00~8/20/2025 0:00:00
383551~"CDER"~"NDA"~210365.00~"JAZZ PHARMACEUTICALS RESEARCH UK LTD"~"Epidiolex (cannabidiol)"~6/25/2018 0:00:00~"Original"~1~"3429-8"~"PMR"~"505 (o)(3)"~3429.00~8~"PMR 3429-8: Conduct a pregnancy outcomes study using a different study design than provided for in the North American Antiepileptic Drug (NAAED) Pregnancy Registry (for example, a retrospective cohort study using claims or electronic medical record data or a case-control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Epidiolex (cannabidiol) during pregnancy compared to an unexposed control population."~"Delayed"~"The Final Protocol milestone was missed due to continued
discussion with the Agency on the protocol. FDA determined that there was sufficient justification for the anticipated delay of the original Study Completion and Final Report Submission milestones. FDA acknowledged proposed revised milestones with sufficient justification in a letter dated 02/20/2024."~3/31/2028 0:00:00~8/20/2025 0:00:00
383552~"CDER"~"NDA"~210428.00~"SUN PHARMA INDUSTRIES LTD"~"KAPSPARGO Sprinkle (metoprolol succinate)"~1/26/2018 0:00:00~"Original"~1~"3638-1"~"PMR"~"Pediatric Research Equity Act"~3638.00~1~"PMR 3638-1: Conduct a randomized, dose-ranging, double-blind, placebo-controlled, parallel group, multi-center clinical study with an open-label 52-week safety extension to evaluate efficacy, safety, tolerability and pharmacokinetics of metoprolol succinate extended release (ER) oral dosage form in hypertensive pediatric subjects from birth to less than 6 years of age"~"Delayed"~"Deferral Extension Requested 07/09/2021. Denied per FDA letter dated 07/23/2021. Final protocol submission missed. Draft Final Protocol Submitted March 7, 2020."~4/30/2027 0:00:00~3/23/2026 0:00:00
383971~"CDER"~"NDA"~214679.00~"AZURITY PHARMACEUTICALS INC"~"Eprontia (topiramate)"~11/5/2021 0:00:00~"Original"~1~"4169-3"~"PMR"~"Pediatric Research Equity Act"~4169.00~3~"PMR 4169-3: A long-term open-label safety study in pediatric patients 1 month up to 2 years of age maintained at topiramate concentrations demonstrated to be therapeutic for partial-onset seizures for a minimum of 6 months of exposure."~"Delayed"~"Original Final Report Due Date: 10/31/2025; Deferral Extension granted per FDA letter dated 02/18/2026. The study completion milestone was also revised."~10/31/2027 0:00:00~12/29/2025 0:00:00
383972~"CDER"~"NDA"~214697.00~"ARS PHARMACEUTICALS OPERATIONS INC"~"Neffy (epinephrine)"~8/9/2024 0:00:00~"Original"~1~"4671-2"~"PMC"~"506B PMC"~4671.00~2~"PMC 4671-2: Conduct a registry-based study in high volume oral food challenge and allergen immunotherapy clinics in which subjects would use either neffy (ARS-1) or an epinephrine injection product for treatment of anaphylaxis. Collect initial symptoms along with clinical outcome data comparing neffy (ARS-1) to epinephrine injection, including time to symptom resolution, adverse events, and whether a repeat dose is needed."~"Delayed"~"The study completion milestone has been missed. The applicant requested revised milestones because due to a slower than anticipated enrollment given longer site initiation. Revised milestones were acknowledged in a letter dated 03/12/2026."~6/30/2026 0:00:00~12/11/2025 0:00:00
383973~"CDER"~"NDA"~214701.00~"BLUEPRINT MEDICINES CORP"~"GAVRETO (pralsetinib)"~12/1/2020 0:00:00~"Original"~1~"3959-2"~"PMR"~"Accelerated Approval"~3959.00~2~"PMR 3959-2: Submit the final report of integrated studies and datasets, to verify and further characterize the clinical benefit of pralsetinib for the treatment of patients with RET fusion-positive thyroid cancer who have received radioactive iodine (if appropriate for their tumor histology) to provide a more precise estimation of the BICR-assessed overall response rate and duration of response in at least 50 patients in a variety of histologies after all responding patients have been followed for 12 months following onset of response or until disease progression, whichever comes first."~"Delayed"~"The Final Report Submission milestone was missed due to awaiting data from the study, which was expected in September 2025."~6/30/2025 0:00:00~
383974~"CDER"~"NDA"~214759.00~"MEDEXUS PHARMA INC"~"Grafapex (treosulfan) for Injection"~1/21/2025 0:00:00~"Original"~1~"4099-1"~"PMR"~"505 (o)(3)"~4099.00~1~"PMR 4099-1: Conduct a clinical pharmacokinetic trial to evaluate a serious potential risk of increased drug toxicity and determine an appropriate, safe dose of treosulfan in patients with moderate renal impairment (estimated glomerular filtration rate or creatinine clearance 30-59 mL/min). This trial should be designed and conducted in accordance with the FDA Guidance for Industry titled  Pharmacokinetics in Patients with Impaired Renal Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Delayed"~"The trial completion milestone was missed because of enrollment difficulties."~2/28/2026 0:00:00~3/20/2026 0:00:00
383975~"CDER"~"NDA"~214759.00~"MEDEXUS PHARMA INC"~"Grafapex (treosulfan) for Injection"~1/21/2025 0:00:00~"Original"~1~"4099-2"~"PMR"~"505 (o)(3)"~4099.00~2~"PMR 4099-2: Conduct a clinical trial to characterize the serious potential risk of QTc prolongation (i.e., exclude large mean increases of >20 msec in the QTc interval) with treosulfan and its active metabolite. Design and conduct the study in accordance with the ICH E14 Guidances for Industry titled, E14 Clinical Evaluation of QT/QTc and E14 Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non-Antiarrhythmic Drugs Questions and Answers (R3) Guidance for Industry."~"Delayed"~"The final report submission was missed due todevelopment of the population PK model."~5/31/2026 0:00:00~3/20/2026 0:00:00
383976~"CDER"~"NDA"~214759.00~"MEDEXUS PHARMA INC"~"Grafapex (treosulfan) for Injection"~1/21/2025 0:00:00~"Original"~1~"4099-3"~"PMR"~"505 (o)(3)"~4099.00~3~"PMR 4099-3: Conduct a clinical pharmacokinetic trial to characterize the serious potential risk of increased drug toxicity and further characterize the safety of treosulfan by evaluating the pharmacokinetics of the active monoepoxide intermediate [(2S,3S)-1,2-epoxybutane-3,4-diol-4-methanesulfonate] in adult patients after administration of treosulfan."~"Delayed"~"The trial completion milestone was missed because of
enrollment difficulties."~2/28/2026 0:00:00~3/20/2026 0:00:00
383977~"CDER"~"NDA"~214759.00~"MEDEXUS PHARMA INC"~"Grafapex (treosulfan) for Injection"~1/21/2025 0:00:00~"Original"~1~"4099-4"~"PMC"~"506B PMC"~4099.00~4~"PMC 4099-4: Conduct a clinical trial to assess the pharmacokinetics of treosulfan among U.S. racial and ethnic groups. This study should characterize the exposure (including pharmacokinetics data) of treosulfan."~"Delayed"~"The trial completion milestone was missed because of enrollment difficulties."~2/28/2026 0:00:00~3/20/2026 0:00:00
383978~"CDER"~"NDA"~214759.00~"MEDEXUS PHARMA INC"~"Grafapex (treosulfan) for Injection"~1/21/2025 0:00:00~"Original"~1~"4099-5"~"PMC"~"506B PMC"~4099.00~5~"PMC 4099-5: Conduct a registry-based observational study to assess outcomes by race and ethnicity for patients undergoing allogeneic hematopoietic stem cell transplantation using treosulfan-fludarabine as the preparative regimen. Include subgroup analyses by race and ethnicity for overall survival, nonrelapse mortality and relapse-free survival."~"Delayed"~"The 10/2025, final protocol submission was missed."~7/31/2029 0:00:00~3/20/2026 0:00:00
383979~"CDER"~"NDA"~215830.00~"PFIZER INC"~"Litfulo (ritlecitinib)"~6/23/2023 0:00:00~"Original"~1~"4463-4"~"PMR"~"505 (o)(3)"~4463.00~4~"PMR 4463-4: Submit the final study report for an ongoing open-label long-term study to investigate the safety of ritlecitinib in adults and adolescents with AA (12 years of age and older)."~"Ongoing"~~8/31/2026 0:00:00~8/20/2025 0:00:00
383980~"CDER"~"NDA"~215830.00~"PFIZER INC"~"Litfulo (ritlecitinib)"~6/23/2023 0:00:00~"Original"~1~"4463-5"~"PMR"~"505 (o)(3)"~4463.00~5~"PMR 4463-5: Collect data from a prospective pregnancy exposure registry, preferably a disease-based multiproduct pregnancy registry, using a cohort analysis that compares the maternal, fetal, and infant outcomes of women with alopecia areata exposed to ritlecitinib during pregnancy with unexposed comparator population(s). Align the study protocol with protocol(s) outside the US to reach the target sample size. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortion, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes described in the protocol will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The Final Protocol Submission Due Date was missed due to continued discussion with the Agency."~6/30/2035 0:00:00~8/20/2025 0:00:00
383981~"CDER"~"NDA"~215830.00~"PFIZER INC"~"Litfulo (ritlecitinib)"~6/23/2023 0:00:00~"Original"~1~"4463-6"~"PMR"~"505 (o)(3)"~4463.00~6~"PMR 4463-6: Conduct an additional pregnancy study that uses a different design from the pregnancy exposure registry (for example a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to ritlecitinib during pregnancy compared to an unexposed control population."~"Pending"~~6/30/2035 0:00:00~8/20/2025 0:00:00
383982~"CDER"~"NDA"~215833.00~"NOVARTIS PHARMACEUTICALS CORP"~"Pluvicto (lutetium (177Lu) vipivotide tetraxetan)"~3/23/2022 0:00:00~"Original"~1~"4241-1"~"PMR"~"505 (o)(3)"~4241.00~1~"PMR 4241-1: Conduct an integrated safety analysis to further characterize the long term outcome of the known serious risk of myelosuppression, renal failure, xerostomia and xerophthalmia and their complications; the potential serious signals of secondary malignancies including myelodysplastic syndrome and acute myeloid leukemia (MDS/AML); and other serious adverse reactions in patients receiving lutetium (177Lu) vipivotide tetraxetan in the VISION study and its sub-study, Trial CAAA617C12301 (NCT04720157), Trial CAAA617B12302 (NCT04689828) and other clinical trials as appropriate. Capture data prospectively in amended case report forms to include incidence, grade, date of onset and resolution of the adverse reaction, predisposing factors and outcomes, date and quantity of red cell and platelet transfusion, use of growth factors for myelosuppression, subsequent antineoplastic therapies, radiation therapy, and hospital admissions. Follow all patients until death, loss to follow-up, or for up to 10 years, whichever occurs first."~"Ongoing"~~3/31/2034 0:00:00~5/19/2026 0:00:00
383983~"CDER"~"NDA"~215833.00~"NOVARTIS PHARMACEUTICALS CORP"~"Pluvicto (lutetium (177Lu) vipivotide tetraxetan)"~3/23/2022 0:00:00~"Original"~1~"4241-2"~"PMR"~"505 (o)(3)"~4241.00~2~"PMR 4241-2: Conduct a clinical trial to determine the kidney biodistribution, dosimetry, pharmacokinetics, and safety of lutetium (177Lu) vipivotide tetraxetan and assess the potential for higher drug exposure and the resultant risk of increased serious toxicities in patients with moderate and severe renal impairment. Assess long-term toxicities in these patients. Follow all patients until death, loss to follow-up, or for up to 10 years, whichever occurs first. Include risk mitigation strategies to reduce potential toxicities in the final report. Design and conduct the trial in accordance with FDA Guidance for Industry titled, Pharmacokinetics in Patients with Impaired Renal Function: Study Design, Data Analysis, and Impact on Dosing."~"Ongoing"~~12/31/2026 0:00:00~5/19/2026 0:00:00
384067~"CDER"~"NDA"~214846.00~"SUMITOMO PHARMA AMERICA INC"~"MYFEMBREE (relugolix, estradiol, norethindrone acetate)"~5/26/2021 0:00:00~"Supplement"~2~"4312-1"~"PMR"~"505 (o)(3)"~4312.00~1~"PMR 4312-1: Conduct a prospective pregnancy exposure registry that compares the maternal, fetal, and neonatal outcomes of women exposed to relugolix containing products during pregnancy to those in an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the infant s first year of life."~"Ongoing"~~5/31/2032 0:00:00~7/24/2025 0:00:00
384068~"CDER"~"NDA"~215973.00~"GILEAD SCIENCES INC"~"Sunlenca (lenacapavir)"~12/22/2022 0:00:00~"Original"~1~"4351-1"~"PMR"~"Pediatric Research Equity Act"~4351.00~1~"PMR 4351-1: Conduct a study to evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of lenacapavir in treatment-experienced children living with HIV-1 infection who are less than 18 years of age and weighing at least 35 kg. The safety and antiviral activity of lenacapavir in pediatric subjects must be evaluated for a minimum of 24 weeks."~"Ongoing"~"Reporting that Week 26 Last Participant Last Visit (LPLV) is expected in Q4 2026; Week 52 LPLV is expected in Q2 2027."~9/30/2027 0:00:00~2/18/2026 0:00:00
384069~"CDER"~"NDA"~215973.00~"GILEAD SCIENCES INC"~"Sunlenca (lenacapavir)"~12/22/2022 0:00:00~"Supplement"~6~"4728-1"~"PMR"~"Pediatric Research Equity Act"~4728.00~1~"PMR 4728-1: Conduct a study to evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of lenacapavir, including oral bridging, in treatmentexperienced children living with HIV-1 infection who are less than 18 years of age and weighing at least 35 kg. The safety and antiretroviral activity of lenacapavir in pediatric subjects must be evaluated for a minimum of 24 weeks. Pharmacokinetic and safety data for oral bridging may be obtained from other ongoing studies, [...]."~"Ongoing"~"Reporting that Week 26 Last Participant Last Visit (LPLV) is expected in Q4 2026; Week 52 LPLV is expected in Q2 2027."~9/30/2027 0:00:00~2/18/2026 0:00:00
384070~"CDER"~"NDA"~215974.00~"GILEAD SCIENCES INC"~"Sunlenca (lenacapavir)"~12/22/2022 0:00:00~"Original"~1~"4365-1"~"PMR"~"Pediatric Research Equity Act"~4365.00~1~"PMR 4365-1: Conduct a study to evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of lenacapavir in treatment-experienced children living with HIV-1 infection who are less than 18 years of age and weighing at least 35 kg. The safety and antiviral activity of lenacapavir in pediatric subjects must be evaluated for a minimum of 24 weeks."~"Ongoing"~"Reporting the first participant first visit is expected in Q1 2025."~9/30/2027 0:00:00~2/18/2025 0:00:00
384071~"CDER"~"NDA"~215974.00~"GILEAD SCIENCES INC"~"Sunlenca (lenacapavir)"~12/22/2022 0:00:00~"Supplement"~8~"4728-1"~"PMR"~"Pediatric Research Equity Act"~4728.00~1~"PMR 4728-1: Conduct a study to evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of lenacapavir, including oral bridging, in treatmentexperienced children living with HIV-1 infection who are less than 18 years of age and weighing at least 35 kg. The safety and antiretroviral activity of lenacapavir in pediatric subjects must be evaluated for a minimum of 24 weeks. Pharmacokinetic and safety data for oral bridging may be obtained from other ongoing studies, [...]."~"Ongoing"~"Reporting that Week 26 Last Participant Last Visit (LPLV) is expected in Q4 2026; Week 52 LPLV is expected in Q2 2027."~9/30/2027 0:00:00~2/18/2025 0:00:00
384072~"CDER"~"NDA"~215985.00~"ARCUTIS BIOTHERAPEUTICS INC"~"Roflumilast "~7/29/2022 0:00:00~"Original"~1~"4314-3"~"PMR"~"Pediatric Research Equity Act"~4314.00~3~"PMR 4314-3: A Phase 3, multicenter, open-label extension study of the long-term safety of ARQ-151 cream 0.3% in subjects ( = 2 years of age) with chronic plaque psoriasis (Study Protocol ARQ-151-306)."~"Submitted"~"The final report was submitted to FDA on 08/29/2025"~6/30/2025 0:00:00~9/26/2025 0:00:00
384073~"CDER"~"NDA"~215985.00~"ARCUTIS BIOTHERAPEUTICS INC"~"Roflumilast "~7/29/2022 0:00:00~"Supplement"~12~"4913-1"~"PMR"~"Pediatric Research Equity Act"~4913.00~1~"PMR 4913-1: Conduct a 4-week, open-label safety trial with Zoryve (roflumilast) cream, 0.05% with 100 evaluable pediatric subjects 3 months to <2 years of age with mild to moderate atopic dermatitis and at least 3% body surface area (BSA) involvement. (Study ARQ-151-218)"~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~2/28/2027 0:00:00~9/26/2025 0:00:00
384074~"CDER"~"NDA"~216059.00~"LOXO ONCOLOGY INC"~"Jaypirca (pirtobrutinib)"~1/27/2023 0:00:00~"Original"~1~"4389-1"~"PMR"~"Accelerated Approval"~4389.00~1~"PMR 4389-1: Complete a randomized clinical trial to obtain data on the clinical efficacy and safety of pirtobrutinib in patients with mantle cell lymphoma. The trial should compare pirtobrutinib monotherapy to an investigator s choice of approved BTK inhibitors in patients with mantle cell lymphoma. The primary endpoint should be progression-free survival as assessed by an independent review committee, with secondary endpoints that include overall survival and objective response rate. The trial should enroll a sufficiently representative study population to reflect the racial and ethnic diversity of the U.S. patient population with mantle cell lymphoma and allow for interpretation of the results in these patient populations."~"Ongoing"~"380 out of the planned 500 patients have been enrolled into the trial."~12/31/2026 0:00:00~3/26/2026 0:00:00
384075~"CDER"~"NDA"~216059.00~"LOXO ONCOLOGY INC"~"Jaypirca (pirtobrutinib)"~1/27/2023 0:00:00~"Original"~1~"4389-2"~"PMR"~"505 (o)(3)"~4389.00~2~"PMR 4389-2: Conduct a study to characterize the longer-term safety of pirtobrutinib monotherapy, at a planned dose of 200 mg daily, in patients with hematologic malignancies treated in Study 18001. Characterize safety and exposure with a minimum of 24 months of follow-up. Include evaluations, supplemented by narratives, of deaths in the absence of treated progressive disease, serious adverse reactions, adverse reactions of special interest (including but not limited to serious infections, cardiac arrhythmias, bleeding, cytopenias, and second primary malignancies), and treatment discontinuations for reasons other than progressive disease."~"Fulfilled"~~9/30/2025 0:00:00~3/26/2026 0:00:00
384076~"CDER"~"NDA"~216059.00~"LOXO ONCOLOGY INC"~"Jaypirca (pirtobrutinib)"~1/27/2023 0:00:00~"Original"~1~"4389-3"~"PMR"~"505 (o)(3)"~4389.00~3~"PMR 4389-3: Conduct an integrated safety analysis of patients with hematologic malignancies treated with pirtobrutinib monotherapy at the 200 mg daily dose in clinical trials and from post-marketing reports to further  characterize the risk of second primary malignancies with extended follow-up. Patients enrolled in clinical trials should have 5 years minimum follow-up for development of second primary malignancies. Include evaluations of incidence rates, types, severity, time to onset, potential predisposing factors, and outcomes."~"Ongoing"~~6/30/2028 0:00:00~3/26/2026 0:00:00
384077~"CDER"~"NDA"~216059.00~"LOXO ONCOLOGY INC"~"Jaypirca (pirtobrutinib)"~1/27/2023 0:00:00~"Supplement"~1~"4557-1"~"PMR"~"Accelerated Approval"~4557.00~1~"PMR 4557-1: Complete a randomized trial in patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Patients should be randomized to receive pirtobrutinib or an investigator s choice arm of idelalisib plus rituximab or bendamustine plus rituximab. The primary endpoint should be progression-free survival, with secondary endpoints that include response rate and overall survival. The trial should include sufficient representation of racial and ethnic minorities to adequately reflect the U.S. patient population with CLL or SLL and allow for interpretation of the results in these patient populations."~"Fulfilled"~"Per FDA letter dated 12/02/2025, this PMR/PMC has been fulfilled."~6/30/2024 0:00:00~3/26/2026 0:00:00
384078~"CDER"~"NDA"~211280.00~"ELI LILLY AND CO"~"Reyvow (lasmiditan) "~10/11/2019 0:00:00~"Original"~1~"3728-5"~"PMR"~"505 (o)(3)"~3728.00~5~"PMR 3728-5: Conduct prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with migraine exposed to Reyvow during pregnancy with two unexposed control populations: one consisting of women with migraine who have not been exposed to Reyvow before or during pregnancy and the other consisting of women without migraine. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~12/31/2034 0:00:00~12/4/2025 0:00:00
384079~"CDER"~"NDA"~211280.00~"ELI LILLY AND CO"~"Reyvow (lasmiditan) "~10/11/2019 0:00:00~"Original"~1~"3728-6"~"PMR"~"505 (o)(3)"~3728.00~6~"PMR 3728-6: Conduct a pregnancy outcomes study using a different study design than provided for in PMR 3728-5 (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Reyvow during pregnancy compared to an unexposed control population."~"Ongoing"~~12/31/2028 0:00:00~12/4/2025 0:00:00
384166~"CDER"~"NDA"~216190.00~"FIDELITY BIOPHARMA CO USA"~"Ontralfy (tizanidine oral solution)"~12/12/2024 0:00:00~"Original"~1~"4749-1"~"PMR"~"Pediatric Research Equity Act"~4749.00~1~"PMR 4749-1: An open-label pharmacokinetic (PK) and safety study of tizanidine in pediatric patients 2-17 years of age with spasticity. Patients will be enrolled from the following age groups: 2 to 5 years, 6 to 11 years, and 12 to 17 years, and each group will be adequately represented."~"Delayed"~"The draft protocol and final protocol, have not been submitted and the milestone dates have been missed and the FDA issued a missed milestone letter on 06/10/2026."~6/30/2028 0:00:00~2/12/2026 0:00:00
384167~"CDER"~"NDA"~216190.00~"FIDELITY BIOPHARMA CO USA"~"Ontralfy (tizanidine oral solution)"~12/12/2024 0:00:00~"Original"~1~"4749-2"~"PMR"~"Pediatric Research Equity Act"~4749.00~2~"PMR 4749-2: An open-label observational safety study in pediatric patients 2 to 17 years of age with spasticity to evaluate for potential short-term and long-term effects of tizanidine in the pediatric population on physical and neurocognitive development. Patients will be enrolled from the following age groups: 2 to 5 years, 6 to 11 years, and 12 to 17 years, and each group will be adequately represented."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed"~12/31/2030 0:00:00~2/12/2026 0:00:00
384168~"CDER"~"NDA"~216196.00~"AGIOS PHARMACEUTICALS INC"~"Pyrukynd (mitapivat) tablet, and Aqvesme (mitapivat) tablets"~2/17/2022 0:00:00~"Original"~1~"4178-1"~"PMR"~"505 (o)(3)"~4178.00~1~"PMR 4178-1: Complete Trial AG-348-C-011,  An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies.  The final study report must include patient level data and a summary of all adverse events including changes in reproductive hormones, changes in blood lipids, bone fractures and other adverse events associated with long-term aromatase inhibition."~"Fulfilled"~~6/30/2025 0:00:00~4/15/2026 0:00:00
384169~"CDER"~"NDA"~216196.00~"AGIOS PHARMACEUTICALS INC"~"Pyrukynd (mitapivat) tablet, and Aqvesme (mitapivat) tablets"~2/17/2022 0:00:00~"Original"~1~"4178-2"~"PMR"~"505 (o)(3)"~4178.00~2~"PMR 4178-2: Complete Trial AG-348-C-003,  A Phase 2, Open-Label, Randomized, Dose-Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency.  The final study report must include patient level data and a summary of all adverse events including changes in reproductive hormones, changes in blood lipids, bone fractures and other adverse events associated with long-term aromatase inhibition."~"Submitted"~~12/31/2025 0:00:00~4/15/2026 0:00:00
384170~"CDER"~"NDA"~216196.00~"AGIOS PHARMACEUTICALS INC"~"Pyrukynd (mitapivat) tablet, and Aqvesme (mitapivat) tablets"~2/17/2022 0:00:00~"Original"~1~"4178-3"~"PMR"~"505 (o)(3)"~4178.00~3~"PMR 4178-3: Conduct a clinical trial to evaluate the impact of hepatic impairment on the pharmacokinetics of mitapivat in subjects with moderate (Child-Pugh B) hepatic impairment relative to adult healthy subjects, in accordance with the reduced study design described in FDA s May 2003 Guidance for Industry    Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling.  Submit subject-level datasets with the final report. Based on the results of this trial, additional subjects with mild (Child-Pugh A) and/or severe (ChildPugh C) hepatic impairment may be enrolled."~"Fulfilled"~~3/31/2024 0:00:00~4/15/2026 0:00:00
384171~"CDER"~"NDA"~216196.00~"AGIOS PHARMACEUTICALS INC"~"Pyrukynd (mitapivat) tablet, and Aqvesme (mitapivat) tablets"~2/17/2022 0:00:00~"Supplement"~3~"4931-1"~"PMR"~"505 (o)(3)"~4931.00~1~"PMR 4931-1: Complete Trial AG348-C-018: A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects with Transfusion-Dependent Alpha- Or Beta-Thalassemia (Energize-T). The final study report will include patient level data and a summary of all adverse events including changes in reproductive hormones, changes in blood lipids, bone fractures and other adverse events associated with long-term aromatase inhibition."~"Ongoing"~~1/31/2027 0:00:00~4/15/2026 0:00:00
384172~"CDER"~"NDA"~216196.00~"AGIOS PHARMACEUTICALS INC"~"Pyrukynd (mitapivat) tablet, and Aqvesme (mitapivat) tablets"~2/17/2022 0:00:00~"Supplement"~3~"4931-2"~"PMR"~"505 (o)(3)"~4931.00~2~"PMR 4931-2: Complete Trial AG348-C-017: A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat In Subjects With Non-Transfusion-Dependent Alpha- Or Beta- Thalassemia (Energize). The final study report will include patient level data and a summary of all adverse events including hepatotoxicity, changes in reproductive hormones, changes in blood lipids, bone fractures and other adverse events associated with long-term aromatase inhibition."~"Ongoing"~~9/30/2026 0:00:00~4/15/2026 0:00:00
384173~"CDER"~"NDA"~216227.00~"HARROW EYE LLC"~"Iheezo (chloroprocaine hydrochloride)"~9/27/2022 0:00:00~"Original"~1~"4352-1"~"PMR"~"Pediatric Research Equity Act"~4352.00~1~"PMR 4352-1: Clinical Study Protocol No: CHL.3-01-2021-M A prospective, investigator-masked, randomized clinical trial to investigate and compare the clinical efficacy of Chloroprocaine 3% gel and Oxybuprocaine 0.4% eye drops anesthesia for clinical practice in pediatric population."~"Delayed"~"The study completion milestone was missed, because of delays due to issues with the study drug and/or comparator drug. Original Final Report Due Date: 3/2023; Deferral Extension granted per FDA letter dated 5/23/2025.
"~12/31/2025 0:00:00~11/27/2024 0:00:00
384174~"CDER"~"NDA"~211675.00~"ABBVIE INC"~"Rinvoq (upadacitinib)"~8/16/2019 0:00:00~"Supplement"~4~"4212-2"~"PMR"~"505 (o)(3)"~4212.00~2~"PMR 4212-2: Conduct a prospective observational study (analyses conducted in patient cohorts enrolled prospectively and followed actively in accordance with a written protocol) to assess the long-term safety of upadacitinib treatment in U.S. patients with moderate-to-severe atopic dermatitis. Fully ascertain and centrally verify serious adverse events, Major Adverse Cardiovascular Events (myocardial infarction, stroke, cardiovascular death, and sudden death), malignancies (including lymphoma, lung cancer, and other malignancies), serious infections, opportunistic infections (including herpes zoster), retinal detachment, thrombosis (including deep venous thrombosis, pulmonary embolism, and arterial thrombosis), hepatoxicity (including drug induced liver injury), and possibly other adverse events of special interest. For each adverse-event outcome separately, compare incidence in upadacitinib-treated patients against reference rates internally derived from analyses conducted in patients treated with dupilumab or other chronic systemic treatments for moderate to-severe atopic dermatitis. Regardless of treatment discontinuation or switch to a different treatment for atopic dermatitis, continue following patients for malignancy outcomes and possibly other adverse events with delayed onset. Enroll a sufficient number of patients to describe the frequency of the adverse events of special interest in representative U.S. patients who start treatment with upadacitinib for atopic dermatitis in the setting of routine clinical practice. Implement a plan that uses rigorous, transparent, and verifiable methods to ascertain and characterize safety events that occur during and after treatment with upadacitinib. Enroll patients over a 4-year period and follow each patient for at least 8 years from time of enrollment."~"Ongoing"~~12/31/2036 0:00:00~10/10/2025 0:00:00
384175~"CDER"~"NDA"~211675.00~"ABBVIE INC"~"Rinvoq (upadacitinib)"~8/16/2019 0:00:00~"Supplement"~4~"4212-3"~"PMR"~"505 (o)(3)"~4212.00~3~"PMR 4212-3: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Rinvoq (upadacitinib), for any indication, during pregnancy and /or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Delayed"~"The final protocol goal date of December 2022 was missed. After discussions and comments from the FDA the final protocol was submitted on March 2023."~6/30/2028 0:00:00~10/10/2025 0:00:00
384176~"CDER"~"NDA"~211675.00~"ABBVIE INC"~"Rinvoq (upadacitinib)"~8/16/2019 0:00:00~"Supplement"~7~"4243-1"~"PMC"~"506B PMC"~4243.00~1~"PMC 4243-1: A one year, randomized, blinded trial to evaluate the safety, efficacy and pharmacokinetics of Rinvoq (upadacitinib) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis."~"Ongoing"~~1/31/2029 0:00:00~10/10/2025 0:00:00
384177~"CDER"~"NDA"~211675.00~"ABBVIE INC"~"Rinvoq (upadacitinib)"~8/16/2019 0:00:00~"Supplement"~7~"4243-2"~"PMC"~"506B PMC"~4243.00~2~"PMC 4243-2: A long term extension study to evaluate the long-term safety of Rinvoq (upadacitinib) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis who participated in postmarketing commitment study 4243-1. This study can be conducted as part of postmarketing commitment study 4243-1."~"Pending"~~6/30/2034 0:00:00~10/10/2025 0:00:00
384499~"CDER"~"NDA"~21751.00~"HAMELN PHARMA PLUS GMBH"~"Pentetate Zinc Trisodium (Zn-DTPA)"~8/11/2004 0:00:00~"Original"~1~"835-4"~"PMC"~"506B PMC"~835.00~4~"PMC 835-4: Human pharmacokinetic study in pediatric subjects to compare and evaluate the absorption, distribution and elimination of Ca and Zn-DTPA via inhalation using a commonly available jet type nebulizer with the intravenous route after an event in which exposure to trans-uranium isotopes occurred. Data/information on dose delivered and the particle size distribution obtained from the specified nebulizer shall be provided."~"Delayed"~"This study is considered delayed because the final protocol was submitted after the milestone due date. This product is for the treatment of contamination resulting from nuclear exposure events and the studies are to start immediately after exposure."~~10/8/2021 0:00:00
384500~"CDER"~"NDA"~21773.00~"ASTRAZENECA AB"~"Byetta (Exenatide)"~4/28/2005 0:00:00~"Original"~1~"4741-1"~"PMR"~"505 (o)(3)"~4741.00~1~"PMR 4741-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of exenatide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Released"~~11/30/2026 0:00:00~11/22/2021 0:00:00
384501~"CDER"~"BLA"~21846.00~"Microbix Biosystems Inc."~"Kinlytic (Urokinase)"~1/16/1978 0:00:00~"Supplement"~76~"187-7"~"PMC"~"506B PMC"~187.00~7~"PMC 187-7: To conduct a study to assess the immunogenicity of Urokinase after primary dosing. Abbott will agree on a study design with CBER prior to initiating the study."~"Delayed"~"The final report milestone was missed. The product is discontinued and the associated IND has been inactivated."~12/31/2004 0:00:00~1/21/2026 0:00:00
384502~"CDER"~"NDA"~21876.00~"DUCHESNAY INC"~"Diclegis (Doxylamine Succinate and Pyridoxine Hydrochloride)"~4/8/2013 0:00:00~"Original"~1~"2033-1"~"PMR"~"Pediatric Research Equity Act"~2033.00~1~"PMR 2033-1: An adequately powered safety and efficacy study in pregnant adolescent girls, 12 to 17 years of age, with nausea and vomiting of pregnancy who are appropriate  candidates for pharmacologic therapy."~"Delayed"~"Original Final Report Due Date: 07/31/2018; Deferral Extension granted per FDA letter dated 04/26/2023. The final protocol submission and study completion milestones were also revised.
"~7/31/2018 0:00:00~6/7/2019 0:00:00
384503~"CDER"~"NDA"~21882.00~"NOVARTIS PHARMACEUTICALS CORP"~"Exjade (Deferasirox)"~11/2/2005 0:00:00~"Original"~1~"750-10"~"PMC"~"506B PMC"~750.00~10~"PMC 750-10: Conduct an ophthalmologic study in patients receiving Exjade. Examinations should include distance visual acuity, applanation tonometry, lens photography, and wide angle fundus photography of retina and optic nerve and should be done at baseline (prior to Exjade initiation) and at six month intervals. At least 60 patients should complete 2 years of follow-up."~"Fulfilled"~~3/31/2010 0:00:00~12/23/2025 0:00:00
384504~"CDER"~"NDA"~21882.00~"NOVARTIS PHARMACEUTICALS CORP"~"Exjade (Deferasirox)"~11/2/2005 0:00:00~"Supplement"~15~"1994-4"~"PMR"~"505 (o)(3)"~1994.00~4~"PMR 1994-4: Establish a registry of children (aged 10 to <18 years old at enrollment) with NTDT and treated with Exjade  (deferasirox) for documented iron overload. Study 2422 will follow at least 40 children for up to 5 years to assess and analyze the long-term safety of treatment with Exjade  (deferasirox), including an assessment of growth, compared to children on a regular transfusion program receiving Exjade  (deferasirox) (based on historical data). Provide annual interim reports on enrollment and outcomes."~"Submitted"~~12/31/2021 0:00:00~12/23/2025 0:00:00
384505~"CDER"~"NDA"~21911.00~"EISAI INC"~"Banzel (Rufinamide)"~11/14/2008 0:00:00~"Original"~1~"4076-1"~"PMR"~"505 (o)(3)"~4076.00~1~"PMR 4076-1: Conduct in vitro patch clamp studies to assess Banzel s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Banzel s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~1/13/2026 0:00:00
384506~"CDER"~"BLA"~125289.00~"Janssen Biotech, Inc."~"Simponi (golimumab) injection"~4/24/2009 0:00:00~"Supplement"~79~"2909-5"~"PMC"~"506B PMC"~2909.00~5~"PMC 2909-5: Conduct a study to evaluate the effectiveness and safety of Simponi  (golimumab) in pediatric patients 5 to 17 years of age with moderately to severely active ulcerative colitis. The study should be designed to  establish that the dose regimen(s) of Simponi (golimumab) identified in  PMC#4 is(are) effective and safe for induction treatment, as well as for  continued treatment after induction. Pharmacokinetic measurements  should be conducted for exposure-response analysis. Collect serum  samples for immunogenicity testing and conduct analyses of the impact of immunogenicity on pharmacokinetics, efficacy and safety."~"Fulfilled"~~5/31/2022 0:00:00~6/17/2026 0:00:00
384507~"CDER"~"BLA"~125319.00~"Novartis Pharmaceuticals Corporation"~"Ilaris (Canakinumab)"~6/17/2009 0:00:00~"Supplement"~62~"2659-2"~"PMR"~"505 (o)(3)"~2659.00~2~"PMR 2659-2: A long-term safety study in 100 pediatric patients 2 to 17 years of age with systemic JIA (SJIA) treated with canakinumab to evaluate for the risks of serious infections, neutropenia, thrombocytopenia, severe injection site reactions, and MAS. The study should include a control group of SJIA patients not receiving canakinumab. Patients should be followed for 5 years."~"Submitted"~~6/30/2023 0:00:00~8/11/2025 0:00:00
384508~"CDER"~"BLA"~125326.00~"Novartis Pharmaceuticals Corporation"~"Arzerra (Ofatumumab)"~10/26/2009 0:00:00~"Supplement"~70~"3901-2"~"PMR"~"505 (o)(3)"~3901.00~2~"PMR 3901-2: Prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with multiple sclerosis exposed to Kesimpta (ofatumumab) during pregnancy with two unexposed control populations: one consisting of women with multiple sclerosis who have not been exposed to Kesimpta (ofatumumab) before or during pregnancy and the other consisting of women without multiple sclerosis. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~2/28/2034 0:00:00~12/22/2025 0:00:00
384509~"CDER"~"BLA"~125326.00~"Novartis Pharmaceuticals Corporation"~"Arzerra (Ofatumumab)"~10/26/2009 0:00:00~"Supplement"~70~"3901-3"~"PMR"~"505 (o)(3)"~3901.00~3~"PMR 3901-3: A pregnancy outcomes study using a different study design than provided for in PMR 3901-2 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case-control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Kesimpta (ofatumumab) during pregnancy compared to an unexposed control population."~"Ongoing"~~2/28/2034 0:00:00~12/22/2025 0:00:00
384510~"CDER"~"BLA"~125326.00~"Novartis Pharmaceuticals Corporation"~"Arzerra (Ofatumumab)"~10/26/2009 0:00:00~"Supplement"~70~"3901-4"~"PMR"~"505 (o)(3)"~3901.00~4~"PMR 3901-4: A safety trial to monitor serum immunoglobulin G and M levels in patients with relapsing forms of multiple sclerosis during treatment with Kesimpta (ofatumumab) to establish the nadir in circulating immunoglobulins during chronic treatment, and to monitor patients after discontinuation of treatment with Kesimpta (ofatumumab) in order to ascertain the time needed to ensure restoration of pre treatment baseline circulating serum levels of immunoglobulins G and M. This trial also should be designed to capture rates of infections, especially opportunistic and recurrent infections associated with immune suppression, and there should be monitoring of B-cell counts throughout treatment and after discontinuation until repletion of immunoglobulin levels."~"Ongoing"~~5/31/2029 0:00:00~12/22/2025 0:00:00
384511~"CDER"~"BLA"~125326.00~"Novartis Pharmaceuticals Corporation"~"Arzerra (Ofatumumab)"~10/26/2009 0:00:00~"Supplement"~70~"3901-6"~"PMR"~"Pediatric Research Equity Act"~3901.00~6~"PMR 3901-6: A two-part study of Kesimpta (ofatumumab) in pediatric patients with relapsing forms of multiple sclerosis (RMS) at least 10 years and less than 18 years of age. Part A is an open-label study of the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Kesimpta (ofatumumab) in pediatric patients weighing 40 kg or less. The objective of Part A is to determine maintenance doses of Kesimpta (ofatumumab) that will result in PK and PD effects that are comparable to those of the dose administered to adult patients. Part B is a randomized, blinded, noninferiority trial with Gilenya (fingolimod) as a comparator.
"~"Delayed"~"Original Final Report Due Date: 03/31/2026; Deferral Extension granted per FDA letter dated 06/06/2025. The study completion milestone was also revised because of delays involving study participant, sites, and/or management."~5/31/2027 0:00:00~12/22/2025 0:00:00
384680~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~139~"4531-2"~"PMC"~"506B PMC"~4531.00~2~"PMC 4531-2: Conduct an integrated analysis from ongoing, completed, or planned clinical trials and other potential data sources as appropriate enrolling a sufficient representation of older adults ages 75 years and older, and United States (U.S.) racial and ethnic minority patients that is reflective of the U.S. population of patients with NSCLC, to further characterize the efficacy and safety of pembrolizumab in combination with platinum-containing chemotherapy and pembrolizumab as a single agent in these patients. In the analysis, include a sufficient number of patients enrolled in the U.S., ages 75 years and older, and a sufficient number of racial and ethnic minorities reflective of the incidence of NSCLC in each subpopulation to allow for interpretation of the results. The analyses should support comparative efficacy and safety outcome analyses between the aforementioned populations and White, and younger patients."~"Pending"~~12/31/2028 0:00:00~10/28/2025 0:00:00
384681~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~151~"4563-1"~"PMC"~"506B PMC"~4563.00~1~"PMC 4563-1: Complete clinical trial KEYNOTE-A39, titled  An open-label, randomized, controlled phase 3 study of enfortumab vedotin in combination with pembrolizumab versus chemotherapy alone in previously untreated locally advanced or metastatic urothelial cancer,  to obtain the final overall survival analysis based on the prespecified final number of events."~"Fulfilled"~~4/30/2025 0:00:00~10/28/2025 0:00:00
384682~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~155~"4654-1"~"PMC"~"506B PMC"~4654.00~1~"PMC 4654-1: Complete the ongoing clinical trial, KEYNOTE-868/ NRG-GY018, titled  A Phase 3, Randomized, Placebo-controlled Study of Pembrolizumab (MK-3475) in Addition to Paclitaxel and Carboplatin for Measurable Stage III or IVA, Stage IVB or Recurrent Endometrial Cancer , to provide the prespecified final overall survival (OS) analyses in both the dMMR and pMMR cohorts."~"Ongoing"~~7/31/2028 0:00:00~10/28/2025 0:00:00
384683~"CDER"~"NDA"~206627.00~"KNOA PHARMA LLC"~"Hysingla ER (Hydrocodone Bitartrate)"~11/20/2014 0:00:00~"Original"~1~"3033-2"~"PMR"~"505 (o)(3)"~3033.00~2~"PMR 3033-2: An observational study designed to measure the incidence and predictors of opioid overdose and death (OOD), as well as opioid abuse/addiction, using patient  health records, insurance claims, and death records. This study must address at a minimum the following specific objectives: a. Estimate the incidence of abuse/addiction, overdose, and death associated with long-term use of opioid analgesics for chronic pain. Stratify overdose by  intentionality wherever possible. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other  clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of psychiatric illness) on the risk of  abuse/addiction, overdose, and death. b. Evaluate and quantify other risk factors for abuse/addiction, overdose, and  death associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors,  psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships. Stratify overdose by intentionality wherever possible."~"Fulfilled"~~9/30/2019 0:00:00~1/13/2026 0:00:00
384684~"CDER"~"NDA"~206627.00~"KNOA PHARMA LLC"~"Hysingla ER (Hydrocodone Bitartrate)"~11/20/2014 0:00:00~"Original"~1~"3033-6"~"PMR"~"505 (o)(3)"~3033.00~6~"PMR 3033-6: An observational study to develop and validate an algorithm using coded medical terminologies and other electronic healthcare data to identify opioid-related overdose and death."~"Fulfilled"~~12/31/2016 0:00:00~1/13/2026 0:00:00
384685~"CDER"~"NDA"~206627.00~"KNOA PHARMA LLC"~"Hysingla ER (Hydrocodone Bitartrate)"~11/20/2014 0:00:00~"Original"~1~"2808-6"~"PMR"~"505 (o)(3)"~2808.00~6~"PMR 2808-6: Conduct a pre- and post-natal development study in the rat model to assess the potential impact of polyethylene oxide (PEO) on development. The study must be designed to adequately qualify the safety of the low molecular weight PEO components impurities/degradants) in the PEO used to manufacture Hysingla ER  when the product is consumed up to the maximum theoretical daily dose of Hysingla ER."~"Submitted"~~8/31/2017 0:00:00~1/13/2026 0:00:00
384686~"CDER"~"NDA"~206627.00~"KNOA PHARMA LLC"~"Hysingla ER (Hydrocodone Bitartrate)"~11/20/2014 0:00:00~"Original"~1~"3033-11"~"PMR"~"505 (o)(3)"~3033.00~11~"PMR 3033-11: Conduct a clinical trial to estimate the serious risk for the development of hyperalgesia following the long-term use of high-dose ER/LA opioid analgesics  for at least one year to treat chronic pain. Include an assessment of risk relative to  efficacy."~"Delayed"~"The Trial Completion and Final Report milestones were missed because of continued discussions needed to reach agreement with the Agency on the Final Protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 8/26/2025."~8/31/2019 0:00:00~1/13/2026 0:00:00
384687~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-1"~"PMR"~"505 (o)(3)"~3462.00~1~"PMR 3462-1: Studies to characterize the in vivo metabolic profile of orally administered stiripentol in the species and strains used in the pivotal developmental toxicity and carcinogenicity studies"~"Submitted"~~4/30/2022 0:00:00~10/17/2025 0:00:00
384688~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-2"~"PMR"~"505 (o)(3)"~3462.00~2~"PMR 3462-2: Bridging toxicokinetic studies to document steady-state plasma exposures to stiripentol and any major human metabolites under the conditions used in the pivotal developmental toxicity and carcinogenicity studies."~"Submitted"~~4/30/2023 0:00:00~10/17/2025 0:00:00
384689~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-3"~"PMR"~"505 (o)(3)"~3462.00~3~"PMR 3462-3: An embryofetal development study of stiripentol in rat."~"Submitted"~~4/30/2023 0:00:00~10/17/2025 0:00:00
384690~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-4"~"PMR"~"505 (o)(3)"~3462.00~4~"PMR 3462-4: An embryofetal development study of stiripentol in rabbit"~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the protocol was submitted on 4/29/2022."~4/30/2023 0:00:00~10/17/2025 0:00:00
384691~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-5"~"PMR"~"505 (o)(3)"~3462.00~5~"PMR 3462-5: A pre- and postnatal development study of stiripentol in rat."~"Submitted"~~5/31/2023 0:00:00~10/17/2025 0:00:00
384692~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-6"~"PMR"~"505 (o)(3)"~3462.00~6~"PMR 3462-6: A juvenile animal toxicology study of stiripentol in one species, with selection of species based on interspecies comparison of in vivo metabolic profiles."~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 12/29/2022."~4/30/2024 0:00:00~10/17/2025 0:00:00
384693~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-7"~"PMR"~"505 (o)(3)"~3462.00~7~"PMR 3462-7: Conduct a pregnancy outcomes study using a different study design than provided for in the North American Antiepileptic Drug (NAAED) Pregnancy Registry (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Diacomit (stiripentol) during pregnancy compared to an unexposed control population"~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 2/24/2021."~8/31/2028 0:00:00~10/17/2025 0:00:00
384769~"CDER"~"NDA"~206940.00~"ABBVIE INC"~"Viberzi (Eluxadoline)"~5/27/2015 0:00:00~"Original"~1~"2901-12"~"PMR"~"Pediatric Research Equity Act"~2901.00~12~"PMR 2901-12: Conduct a randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, and pharmacokinetics (PK) of Viberzi (eluxadoline) in pediatric patients 12 to 17 years of age with diarrhea-predominant irritable bowel syndrome (IBS-D)."~"Delayed"~"The final protocol submission milestone was missed. A revised draft protocol was submitted by the applicant and FDA provided further comment."~6/30/2028 0:00:00~7/22/2025 0:00:00
384770~"CDER"~"NDA"~206940.00~"ABBVIE INC"~"Viberzi (Eluxadoline)"~5/27/2015 0:00:00~"Original"~1~"2901-13"~"PMR"~"Pediatric Research Equity Act"~2901.00~13~"PMR 2901-13: Conduct a randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, and pharmacokinetics (PK) of Viberzi (eluxadoline) in pediatric patients 6 to 11 years of age with diarrhea-predominant irritable bowel syndrome (IBS-D)."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2032 0:00:00~7/22/2025 0:00:00
384771~"CDER"~"NDA"~206940.00~"ABBVIE INC"~"Viberzi (Eluxadoline)"~5/27/2015 0:00:00~"Original"~1~"2901-14"~"PMR"~"Pediatric Research Equity Act"~2901.00~14~"PMR 2901-14: Complete the ongoing 52 week long term extension study of eluxadoline in pediatric participants 6 through 17 years with diarrhea-predominant irritable bowel syndrome (IBS-D) who completed the dose ranging (2901-10 and 2901-11) or efficacy (2901-12 and 2901-13) studies."~"Ongoing"~"26 subjects have been enrolled."~6/30/2033 0:00:00~7/22/2025 0:00:00
384772~"CDER"~"NDA"~206966.00~"LNHC INC"~"Xeglyze (Abametapir) "~7/24/2020 0:00:00~"Original"~1~"3898-4"~"PMC"~"506B PMC"~3898.00~4~"PMC 3898-4: Conduct a study to evaluate the long-term storage stability of abametapir carboxyl in plasma stored at -80  C for duration of at least 1,251 days."~"Submitted"~~2/28/2021 0:00:00~8/22/2024 0:00:00
384773~"CDER"~"NDA"~206976.00~"IBSA INSTITUT BIOCHIMIQUE SA"~"LICART (diclofenac epolamine)"~12/19/2018 0:00:00~"Original"~1~"3537-1"~"PMR"~"Pediatric Research Equity Act"~3537.00~1~"PMR 3537-1: An open-label pharmacokinetics and safety study of diclofenac epolamine topical system in pediatric patients aged 6 to less than 17 years with acute pain due to minor strains, sprains, and contusions."~"Fulfilled"~"Per FDA letter dated 03/27/2026, this PMR/PMC has been fulfilled."~6/30/2025 0:00:00~2/12/2026 0:00:00
384774~"CDER"~"NDA"~207078.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Lokelma (sodium zirconium cyclosilicate)"~5/18/2018 0:00:00~"Original"~1~"3373-1"~"PMR"~"Pediatric Research Equity Act"~3373.00~1~"PMR 3373-1: Conduct a two-part study with an acute and maintenance phase to evaluate the safety, tolerability, and pharmacodynamic effects of Lokelma (sodium zirconium cyclosilicate) in pediatric patients 0 to 17 years of age with hyperkalemia."~"Delayed"~"Original Final Report Due Date: 12/31/2026; Deferral Extension granted per FDA letter dated 04/08/2026. The study completion milestone was also revised."~12/31/2028 0:00:00~7/8/2026 0:00:00
384775~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-1"~"PMR"~"505 (o)(3)"~3462.00~1~"PMR 3462-1: Studies to characterize the in vivo metabolic profile of orally administered stiripentol in the species and strains used in the pivotal developmental toxicity and carcinogenicity studies"~"Submitted"~~4/30/2022 0:00:00~10/17/2025 0:00:00
384776~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-2"~"PMR"~"505 (o)(3)"~3462.00~2~"PMR 3462-2: Bridging toxicokinetic studies to document steady-state plasma exposures to stiripentol and any major human metabolites under the conditions used in the pivotal developmental toxicity and carcinogenicity studies."~"Submitted"~~4/30/2023 0:00:00~10/17/2025 0:00:00
384777~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-3"~"PMR"~"505 (o)(3)"~3462.00~3~"PMR 3462-3: An embryofetal development study of stiripentol in rat."~"Submitted"~~4/30/2023 0:00:00~10/17/2025 0:00:00
384778~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-4"~"PMR"~"505 (o)(3)"~3462.00~4~"PMR 3462-4: An embryofetal development study of stiripentol in rabbit"~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the protocol was submitted on 4/29/2022."~4/30/2023 0:00:00~10/17/2025 0:00:00
384779~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-5"~"PMR"~"505 (o)(3)"~3462.00~5~"PMR 3462-5: A pre- and postnatal development study of stiripentol in rat."~"Submitted"~~5/31/2023 0:00:00~10/17/2025 0:00:00
384780~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-6"~"PMR"~"505 (o)(3)"~3462.00~6~"PMR 3462-6: A juvenile animal toxicology study of stiripentol in one species, with selection of species based on interspecies comparison of in vivo metabolic profiles."~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 12/29/2022."~4/30/2024 0:00:00~10/17/2025 0:00:00
384781~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-7"~"PMR"~"505 (o)(3)"~3462.00~7~"PMR 3462-7: Conduct a pregnancy outcomes study using a different study design than provided for in the North American Antiepileptic Drug (NAAED) Pregnancy Registry (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Diacomit (stiripentol) during pregnancy compared to an unexposed control population"~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 2/24/2021."~8/31/2028 0:00:00~10/17/2025 0:00:00
385383~"CDER"~"BLA"~761310.00~"AbbVie Inc."~"Elahere (mirvetuximab soravtansine-gynx)"~11/14/2022 0:00:00~"Supplement"~5~"4610-2"~"PMC"~"506B PMC"~4610.00~2~"PMC 4610-2: Evaluate the effect of anti-mirvetuximab soravtansine antibodies and the effect of neutralizing antibodies against mirvetuximab soravtansine on the efficacy, safety, and pharmacokinetics of mirvetuximab soravtansine. Submit an amended integrated summary of immunogenicity report with data from a sufficient number of patients in ongoing studies and future studies. Key efficacy endpoints should include overall response rate, progression-free survival, and overall survival."~"Ongoing"~~6/30/2028 0:00:00~1/7/2026 0:00:00
385384~"CDER"~"BLA"~761312.00~"CitiusOncology, Inc."~"Lymphir (denileukin diftitox-cxdl)"~8/7/2024 0:00:00~"Original"~1~"4670-1"~"PMR"~"505 (o)(3)"~4670.00~1~"PMR 4670-1: Conduct a clinical study to further characterize the known serious risk of visual impairment including risk factors, manifestations, and outcomes in patients with cutaneous T-cell lymphoma (CTCL) treated with denileukin diftitox-cxdl. The visual impairment monitoring procedures in this study should include, but should not be limited to, visual acuity, slit lamp examination, dilated funduscopic examination, and optical coherence tomography. The study will ascertain for and closely monitor any potential risk factors associated with visual impairment. Evaluate patients with CTCL treated with denileukin diftitox-cxdl and provide data on at least 50 patients with visual impairment that occurred on denileukin diftitox-cxdl treatment."~"Delayed"~"The draft and final protocol submission milestones were missed."~9/30/2028 0:00:00~9/26/2025 0:00:00
385385~"CDER"~"BLA"~761324.00~"Genmab US, Inc."~"Epkinly (epcoritamab-bysp)"~5/19/2023 0:00:00~"Original"~1~"4435-1"~"PMR"~"Accelerated Approval"~4435.00~1~"PMR 4435-1: Complete a randomized clinical trial in patients with relapsed or refractory large B-cell lymphoma. The trial should compare epcoritamab monotherapy to an investigator s choice of standard therapies for patients with relapsed or refractory large B-cell lymphoma. The primary endpoint should be overall survival with secondary endpoints that include progression-free survival and response rate."~"Delayed"~"The applicant requested revised milestones because of the increased number of targeted overall survival events to mitigate potential impact of confounding factors, including the COVID-19 pandemic. Revised milestones were acknowledged in a letter dated 01/02/2025."~6/30/2025 0:00:00~7/16/2025 0:00:00
385386~"CDER"~"BLA"~761324.00~"Genmab US, Inc."~"Epkinly (epcoritamab-bysp)"~5/19/2023 0:00:00~"Original"~1~"4435-2"~"PMR"~"Pediatric Research Equity Act"~4435.00~2~"PMR 4435-2: Complete a clinical trial to confirm the appropriate dose of epcoritamab and to assess the safety, pharmacokinetics, and preliminary efficacy of epcoritamab monotherapy in pediatric patients with relapsed or refractory aggressive mature B-cell lymphoma."~"Ongoing"~"The trial has completed enrollment."~5/31/2029 0:00:00~7/16/2025 0:00:00
385387~"CDER"~"BLA"~761324.00~"Genmab US, Inc."~"Epkinly (epcoritamab-bysp)"~5/19/2023 0:00:00~"Original"~1~"4435-3"~"PMR"~"505 (o)(3)"~4435.00~3~"PMR 4435-3: Conduct an integrated safety analysis of data from patients with large B-cell lymphoma and other lymphoid malignancies to further characterize the long-term incidence, severity, and outcome of the known serious risks of immune effector cell-associated neurotoxicity syndrome, neurologic toxicity, hematologic adverse events, and infections. Include a comprehensive analysis from all available data sources including but not limited to patient-level and pooled analyses of ongoing and completed clinical trials."~"Pending"~~11/30/2028 0:00:00~7/16/2025 0:00:00
385388~"CDER"~"BLA"~761324.00~"Genmab US, Inc."~"Epkinly (epcoritamab-bysp)"~5/19/2023 0:00:00~"Original"~1~"4435-4"~"PMC"~"506B PMC"~4435.00~4~"PMC 4435-4: Conduct an integrated analysis of data from clinical trials to further characterize the safety, efficacy, pharmacokinetics, and pharmacodynamics of epcoritamab monotherapy among U.S. racial and ethnic minority patients with large B-cell lymphoma. The population should be representative of the U.S. population, including racial and ethnic diversity, of patients with large B-cell lymphoma and allow for interpretation of the results in these populations."~"Pending"~~8/31/2028 0:00:00~7/16/2025 0:00:00
385389~"CDER"~"BLA"~761324.00~"Genmab US, Inc."~"Epkinly (epcoritamab-bysp)"~5/19/2023 0:00:00~"Supplement"~3~"4657-1"~"PMR"~"Accelerated Approval"~4657.00~1~"PMR 4657-1: Complete a randomized clinical trial intended to verify and describe the clinical benefit of epcoritamab in patients with relapsed or refractory follicular lymphoma. Patients should be randomized to receive epcoritamab in combination with rituximab and lenalidomide versus rituximab and lenalidomide. The primary endpoint should be progression-free survival, with overall survival as a secondary endpoint. The trial should enroll a sufficiently representative study population that reflects the racial and ethnic diversity of the U.S. population of patients with follicular lymphoma and allow for interpretation of the results in these populations."~"Fulfilled"~"Per FDA letter dated 11/18/2025, this PMR/PMC has been fulfilled."~11/30/2027 0:00:00~7/16/2025 0:00:00
385390~"CDER"~"BLA"~761324.00~"Genmab US, Inc."~"Epkinly (epcoritamab-bysp)"~5/19/2023 0:00:00~"Supplement"~3~"4657-2"~"PMR"~"505 (o)(3)"~4657.00~2~"PMR 4657-2: Conduct an integrated safety analysis of long-term safety data from patients with relapsed or refractory follicular lymphoma treated in the expansion arm and in the optimization Arm A of study GCT3013-01 to further characterize the long-term incidence of serious and fatal adverse events, as well as the severity and outcomes of the known serious risks of immune effector cell-associated neurotoxicity syndrome (ICANS), neurological adverse events, hematologic adverse events, and infections, including the incidence and severity of viral infections including COVID-19 infections in patients with relapsed/refractory follicular lymphoma. Data should be provided through the last patient treated in each cohort having at least 24 months of follow-up. At a minimum, the information to be submitted should include an updated clinical summary of safety, safety datasets, and patient narratives for any deaths, serious adverse events, adverse events of special interest (Grade 2 or higher cytokine release syndrome; any grade ICANS and clinical tumor lysis syndrome), Grade 3 or higher infections, and any Grade 3 or higher COVID-19 infections."~"Pending"~~8/31/2029 0:00:00~7/16/2025 0:00:00
385391~"CDER"~"NDA"~220152.00~"VANDA PHARMACEUTICALS INC"~"Nereus (tradipitant) capsules"~12/30/2025 0:00:00~"Original"~1~"4946-1"~"PMR"~"Pediatric Research Equity Act"~4946.00~1~"PMR 4946-1: Conduct a clinical trial to evaluate the pharmacokinetics and safety of an age-appropriate formulation of tradipitant in pediatric patients 2 to < 18 years of age with a history of motion sickness."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~3/31/2027 0:00:00~
385392~"CDER"~"NDA"~220152.00~"VANDA PHARMACEUTICALS INC"~"Nereus (tradipitant) capsules"~12/30/2025 0:00:00~"Original"~1~"4946-2"~"PMR"~"Pediatric Research Equity Act"~4946.00~2~"PMR 4946-2: Conduct a randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of tradipitant in pediatric patients 2 to < 18 years of age with a history of motion sickness."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2028 0:00:00~
385393~"CDER"~"NDA"~220152.00~"VANDA PHARMACEUTICALS INC"~"Nereus (tradipitant) capsules"~12/30/2025 0:00:00~"Original"~1~"4946-3"~"PMR"~"505 (o)(3)"~4946.00~3~"PMR 4946-3: Collect data from a prospective pregnancy exposure registry, preferably a disease-based multiproduct pregnancy registry, using a cohort analysis that compares the maternal, fetal, and infant outcomes of women exposed to tradipitant regardless of indication during pregnancy with unexposed comparator population(s) in a timely manner. Align the study protocol with protocol(s) outside the U.S. to reach the target sample size. The registry should identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortion, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes described in the protocol will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Pending"~~6/30/2037 0:00:00~
385394~"CDER"~"NDA"~220152.00~"VANDA PHARMACEUTICALS INC"~"Nereus (tradipitant) capsules"~12/30/2025 0:00:00~"Original"~1~"4946-4"~"PMR"~"505 (o)(3)"~4946.00~4~"PMR 4946-4: Conduct a retrospective pregnancy cohort study using claims or electronic health record data with medical chart validation that is adequately powered to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in individuals exposed to tradipitant during pregnancy compared to appropriate comparator population(s)."~"Pending"~~12/31/2033 0:00:00~
385395~"CDER"~"NDA"~220152.00~"VANDA PHARMACEUTICALS INC"~"Nereus (tradipitant) capsules"~12/30/2025 0:00:00~"Original"~1~"4946-5"~"PMR"~"505 (o)(3)"~4946.00~5~"PMR 4946-5: Perform a lactation study (milk only or mother-infant pair study) in lactating women who have received tradipitant to measure concentrations of tradipitant and its major metabolites in breast milk using a validated assay. Assess the effects on the breastfed infant, if available, based on study population."~"Pending"~~6/30/2029 0:00:00~
383553~"CDER"~"NDA"~210450.00~"ABBVIE INC"~"Orilissa (Elagolix Sodium)"~7/23/2018 0:00:00~"Original"~1~"3856-1"~"PMC"~"506B PMC"~3856.00~1~"PMC 3856-1: ""A Phase 3b Study to Evaluate the Safety and Efficacy of Elagolix in Combination with Combined Oral Contraceptives in Premenopausal Women with Documented Endometriosis and Associated Moderate to Severe Pain."""~"Delayed"~"The Efficacy Results Submission milestone was missed due to enrollment challenges. Per applicant, enrollment is 468 of 800 (17Jul2025) and a meeting will be scheduled to discuss."~6/30/2026 0:00:00~9/17/2025 0:00:00
383554~"CDER"~"NDA"~210450.00~"ABBVIE INC"~"Orilissa (Elagolix Sodium)"~7/23/2018 0:00:00~"Original"~1~"3390-2"~"PMR"~"505 (o)(3)"~3390.00~2~"PMR 3390-2: A pharmacoepidemiology surveillance study to evaluate the effects of Orilissa on pregnancy-related outcomes."~"Delayed"~"The final report submission milestone was missed because of challenges in reaching the target sample size due to lower-than-expected accrual of pregnancies exposed to elagolix. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 01/30/2026."~1/31/2025 0:00:00~9/17/2025 0:00:00
383555~"CDER"~"NDA"~210491.00~"VERTEX PHARMACEUTICALS INC"~"Symdeko (ivacaftor and tezacaftor)"~2/12/2018 0:00:00~"Supplement"~7~"3988-1"~"PMC"~"506B PMC"~3988.00~1~"PMC 3988-1: Conduct a 3-year, single arm, observational study to further understand the clinical response to elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) and tezacaftor/ivacaftor (TEZ/IVA) in cystic fibrosis (CF) patients. The study will include all patients registered in the U.S. Cystic Fibrosis Foundation Patient Registry who do not have an F508del allele but do have a CFTR mutation that has been characterized in vitro as responsive to ELX/TEZ/IVA and/or TEZ/IVA, but not to IVA alone, and who initiate ELX/TEZ/IVA and/or TEZ/IVA therapy following the date of approval of this supplement. Patients will be followed for at least 3 years on ELX/TEZ/IVA and/or TEZ/IVA after ELX/TEZ/IVA and/or TEZ/IVA initiation. The key outcomes of interest will include lung function measurements (FEV1), nutritional parameters (e.g., BMI), pulmonary exacerbations, hospitalizations, select CF complications (e.g., symptomatic sinus disease, CFRD, distal intestinal obstruction), and the presence of select pulmonary microorganisms (e.g., P aeruginosa)."~"Fulfilled"~~12/31/2025 0:00:00~4/3/2026 0:00:00
383556~"CDER"~"NDA"~210493.00~"HELSINN HEALTHCARE SA"~"Akynzeo (fosnetupitant and palonosetron hydrochloride)"~4/19/2018 0:00:00~"Original"~1~"3292-5"~"PMR"~"Pediatric Research Equity Act"~3292.00~5~"PMR 3292-5: A multicenter, multinational, two-part study to assess the pharmacokinetics, safety and efficacy of intravenous fosnetupitant/
palonosetron combination single and repeated dose administration in pediatric cancer patients ages 0 to 17 years for the prevention of nausea and vomiting associated with single and multi-day highly emetogenic chemotherapy. You must conduct this study with an age-appropriate formulation."~"Ongoing"~"The protocol was submitted on 3/31/2024 and the review dated 6/23/2024 finds it acceptable."~7/31/2027 0:00:00~6/17/2026 0:00:00
383557~"CDER"~"NDA"~210496.00~"ARRAY BIOPHARMA INC"~"Braftovi (Encorafenib)"~6/27/2018 0:00:00~"Supplement"~17~"4753-1"~"PMR"~"Accelerated Approval"~4753.00~1~"PMR 4753-1: Conduct a randomized comparative clinical trial intended to verify and describe the clinical benefit of encorafenib and cetuximab in combination with mFOLFOX6 in adult patients with previously untreated BRAF V600E mutation-positive metastatic colorectal cancer by assessing progression free survival. This data may be obtained from the ongoing clinical trial, BREAKWATER (C4221015),  An open-label, multicenter, randomized Phase 3 study of EC alone (EC Arm) or in combination with chemotherapy (mFOLFOX6; EC + mFOLFOX6 Arm) versus standard-of-care chemotherapies (Control Arm: mFOLFOX6, FOLFOXIRI, or CAPOX each with or without bevacizumab) in first-line participants With BRAF V600Emutant mCRC. "~"Fulfilled"~"Per FDA letter dated 02/24/2026, this PMR/PMC has been fulfilled."~5/31/2025 0:00:00~8/21/2025 0:00:00
383558~"CDER"~"NDA"~210526.00~"TRIS PHARMA INC"~"Dyanavel XR (Amphetamine)"~11/4/2021 0:00:00~"Original"~1~"4179-2"~"PMR"~"Pediatric Research Equity Act"~4179.00~2~"PMR 4179-2: A randomized, double-blind, placebo-controlled, flexible-dose study of Dyanavel XR (amphetamine extended-release) oral tablet in children aged 4 to 5 years diagnosed with ADHD."~"Released"~"Per FDA letter dated 11/18/2025, this PMR/PMC has been released."~8/31/2025 0:00:00~1/3/2025 0:00:00
383559~"CDER"~"NDA"~210526.00~"TRIS PHARMA INC"~"Dyanavel XR (Amphetamine)"~11/4/2021 0:00:00~"Original"~1~"4179-3"~"PMR"~"Pediatric Research Equity Act"~4179.00~3~"PMR 4179-3: A one year Pediatric Open-Label Safety Study of patients aged 4 to 5 years diagnosed with ADHD treated with Dyanavel XR (amphetamine extended release) oral tablet."~"Released"~"Per FDA letter dated 11/18/2025, this PMR/PMC has been released."~8/31/2025 0:00:00~1/3/2025 0:00:00
383560~"CDER"~"NDA"~210557.00~"COSETTE PHARMACEUTICALS INC"~"Vyleesi (bremelanotide)"~6/21/2019 0:00:00~"Original"~1~"3635-3"~"PMR"~"505 (o)(3)"~3635.00~3~"PMR 3635-3: A trial in lactating women who have received Vyleesi to assess potential adverse effects in the breastfed infant and measure bremelanotide concentrations in breast milk using a validated assay."~"Fulfilled"~~12/31/2025 0:00:00~8/20/2025 0:00:00
383561~"CDER"~"NDA"~210563.00~"PHARMACYCLICS LLC"~"Imbruvica (Ibrutinib)"~2/16/2018 0:00:00~"Supplement"~12~"4327-1"~"PMR"~"505 (o)(3)"~4327.00~1~"PMR 4327-1: Conduct analyses to characterize long-term safety of ibrutinib in terms of growth and development in pediatric patients. Patients enrolled in Study PCYC-1146-IM should be evaluated for growth and development milestones annually for at least 5 years from the initiation of ibrutinib. Data should include growth parameters as measured by height and weight, sexual maturation by Tanner stage, performance status, immune reconstitution, adverse events, and patient-reported outcomes measures."~"Ongoing"~~7/31/2026 0:00:00~1/7/2026 0:00:00
383562~"CDER"~"NDA"~210589.00~"FRESENIUS KABI USA LLC"~"Omegaven (fish oil triglycerides)"~7/27/2018 0:00:00~"Original"~1~"3445-4"~"PMR"~"505 (o)(3)"~3445.00~4~"PMR 3445-4: Evaluate the occurrence of essential fatty acid deficiency (EFAD) in a prospective, open label, single-arm, multicenter study in pediatric patients with parenteral nutrition-associated cholestasis (PNAC) who require more than eight weeks of Omegaven treatment."~"Ongoing"~~3/31/2027 0:00:00~9/24/2025 0:00:00
383563~"CDER"~"NDA"~210607.00~"60 DEGREES PHARMACEUTICALS INC"~"ARAKODA (tafenoquine)"~8/8/2018 0:00:00~"Original"~1~"3464-1"~"PMR"~"Pediatric Research Equity Act"~3464.00~1~"PMR 3464-1: Conduct a randomized, active comparator study to evaluate the safety, tolerability, and pharmacokinetics of ARAKODA for the prophylaxis of malaria in children from birth to 18 years of age."~"Delayed"~"Original Final Report Due Date: 06/30/2025; Deferral Extension granted per FDA letter dated 09/10/2025. The study completion milestone was also revised.
"~1/31/2030 0:00:00~9/4/2025 0:00:00
383564~"CDER"~"NDA"~210607.00~"60 DEGREES PHARMACEUTICALS INC"~"ARAKODA (tafenoquine)"~8/8/2018 0:00:00~"Original"~1~"3464-3"~"PMR"~"505 (o)(3)"~3464.00~3~"PMR 3464-3: Conduct an observational study to evaluate safety, including neurologic, hypersensitivity, psychiatric and hematologic adverse reactions, in patients taking ARAKODA (tafenoquine) for the prophylaxis of malaria."~"Ongoing"~~12/31/2026 0:00:00~9/4/2025 0:00:00
383565~"CDER"~"NDA"~210730.00~"TREVENA INC"~"Olinvyk (oliceridine)"~8/7/2020 0:00:00~"Original"~1~"3902-1"~"PMR"~"Pediatric Research Equity Act"~3902.00~1~"PMR 3902-1: Conduct a randomized, controlled trial evaluating the pharmacokinetic, safety, tolerability, and efficacy profiles of OLINVYK in pediatric patients aged 6 to less than 17 years for whom parenteral opioid therapy is warranted."~"Delayed"~"The final protocol, study completion, and final report submission milestones were missed. The original final report submission due date was 6/30/2023. The release request, deferral extension request and PREA waiver request submitted on 10/30/2025 have been denied per FDA letter dated 12/15/2025."~6/30/2023 0:00:00~10/3/2025 0:00:00
383566~"CDER"~"NDA"~210730.00~"TREVENA INC"~"Olinvyk (oliceridine)"~8/7/2020 0:00:00~"Original"~1~"3902-2"~"PMR"~"Pediatric Research Equity Act"~3902.00~2~"PMR 3902-2: Conduct a randomized, controlled trial evaluating the pharmacokinetic, safety, tolerability, and efficacy profiles of OLINVYK in pediatric patients aged 3 to less than 6 years for whom parenteral opioid therapy is warranted."~"Delayed"~"The final protocol, study completion, and final report submission milestones were missed. The original final report submission due date was 6/30/2023. The release request, deferral extension request and PREA waiver request submitted on 10/30/2025 have been denied per FDA letter dated 12/15/2025."~6/30/2023 0:00:00~10/3/2025 0:00:00
383567~"CDER"~"NDA"~210563.00~"PHARMACYCLICS LLC"~"Imbruvica (Ibrutinib)"~2/16/2018 0:00:00~"Supplement"~6~"3836-1"~"PMC"~"506B PMC"~3836.00~1~"PMC 3836-1: Submit the overall survival analysis and datasets with the final report for clinical trial E1912 titled,  A Randomized Phase III Study of Ibrutinib- Based Therapy vs Standard Fludarabine, Cyclophosphamide, and Rituximab Chemoimmunotherapy in Untreated Younger Patients with Chronic Lymphocytic Leukemia  to provide additional long term efficacy data."~"Ongoing"~~9/30/2027 0:00:00~1/7/2026 0:00:00
383568~"CDER"~"NDA"~210730.00~"TREVENA INC"~"Olinvyk (oliceridine)"~8/7/2020 0:00:00~"Original"~1~"3902-3"~"PMR"~"Pediatric Research Equity Act"~3902.00~3~"PMR 3902-3: Conduct a randomized, controlled trial evaluating the pharmacokinetic, pharmacodynamic, safety, tolerability, and efficacy profiles of OLINVYK in pediatric patients aged birth to less than 3 years of age for whom parenteral opioid therapy is warranted."~"Delayed"~"The final protocol, study completion, and final report submission milestones were missed. The original final report submission due date was 7/31/2025. The release request, deferral extension request and PREA waiver request submitted on 10/30/2025 have been denied per FDA letter dated 12/15/2025."~7/31/2025 0:00:00~10/3/2025 0:00:00
383569~"CDER"~"NDA"~210730.00~"TREVENA INC"~"Olinvyk (oliceridine)"~8/7/2020 0:00:00~"Original"~1~"3902-4"~"PMR"~"Pediatric Research Equity Act"~3902.00~4~"PMR 3902-4: Conduct a randomized, controlled trial evaluating the safety, tolerability, and efficacy profiles of OLINVYK in pediatric patients aged birth to less than 3 years of age for whom parenteral opioid therapy is warranted."~"Delayed"~"The final protocol submission milestone was missed. The release request, deferral extension request and PREA waiver request submitted on 10/30/2025 have been denied per FDA letter dated 12/15/2025."~2/28/2028 0:00:00~10/3/2025 0:00:00
383570~"CDER"~"NDA"~210730.00~"TREVENA INC"~"Olinvyk (oliceridine)"~8/7/2020 0:00:00~"Original"~1~"3902-5"~"PMR"~"Pediatric Research Equity Act"~3902.00~5~"PMR 3902-5: Conduct a juvenile animal study in the rat model to support pediatric dosing in patients 3 years of age to less than 17 years of age."~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. The original final report submission due date was1/31/2022. The release request, deferral extension request and PREA waiver request submitted on 10/30/2025 have been denied per FDA letter dated 12/15/2025."~1/31/2022 0:00:00~10/3/2025 0:00:00
383571~"CDER"~"NDA"~210730.00~"TREVENA INC"~"Olinvyk (oliceridine)"~8/7/2020 0:00:00~"Original"~1~"3902-6"~"PMR"~"Pediatric Research Equity Act"~3902.00~6~"PMR 3902-6: Conduct a juvenile animal study in the rat model to support pediatric dosing in patients less than 3 years of age."~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. The original final report submission milestone due date was10/31/2022. The release request, deferral extension request and PREA waiver request submitted on 10/30/2025 have been denied per FDA letter dated 12/15/2025."~10/31/2022 0:00:00~10/3/2025 0:00:00
383572~"CDER"~"NDA"~210745.00~"AMGEN INC"~"Otezla XR (apremilast) extended-release tablets"~8/29/2025 0:00:00~"Original"~1~"4885-1"~"PMR"~"Pediatric Research Equity Act"~4885.00~1~"PMR 4885-1: Develop an age appropriate extended-release formulation suitable for use in pediatric patients who weigh 20 kg to 50 kg."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~9/30/2026 0:00:00~
383573~"CDER"~"NDA"~210745.00~"AMGEN INC"~"Otezla XR (apremilast) extended-release tablets"~8/29/2025 0:00:00~"Original"~1~"4885-2"~"PMR"~"Pediatric Research Equity Act"~4885.00~2~"PMR 4885-2: Conduct a relative bioavailability study in healthy adult subjects to determine an appropriate dose and dosing regimen for the use of extended-release apremilast formulation in pediatric patients aged 6 to 17 years weighing 20 kg to 50 kg for the treatment of moderate to severe plaque psoriasis and active psoriatic arthritis."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~2/28/2028 0:00:00~
383574~"CDER"~"NDA"~210745.00~"AMGEN INC"~"Otezla XR (apremilast) extended-release tablets"~8/29/2025 0:00:00~"Original"~2~"4885-1"~"PMR"~"Pediatric Research Equity Act"~4885.00~1~"PMR 4885-1: Develop an age appropriate extended-release formulation suitable for use in pediatric patients who weigh 20 kg to 50 kg."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~9/30/2026 0:00:00~
383575~"CDER"~"NDA"~209296.00~"HERON THERAPEUTICS INC"~"Cinvanti (aprepitant)"~11/9/2017 0:00:00~"Original"~1~"3289-3"~"PMR"~"Pediatric Research Equity Act"~3289.00~3~"PMR 3289-3: A study to evaluate pharmacokinetics, safety, and tolerability of a 3-day regimen of Cinvanti (aprepitant) injectable emulsion (I.V./I.V./I.V.) in pediatric patients 0 to 17 years of age undergoing multi-day highly emetogenic chemotherapy. Conduct this study upon completion of the single-dose regimen PREA PMR."~"Delayed"~"The final protocol milestone was missed."~5/31/2028 0:00:00~1/6/2026 0:00:00
383576~"CDER"~"NDA"~209311.00~"IRONSHORE PHARMACEUTICALS INC A WHOLLY OWNED SUB OF COLLEGIUM PHARMACEUTICAL INC"~"Jornay PM (Methylphenidate Hydrochloride)"~8/8/2018 0:00:00~"Original"~1~"3465-1"~"PMR"~"Pediatric Research Equity Act"~3465.00~1~"PMR 3465-1: A Phase 3, 3-Week, Double-blind, Randomized, Placebo-controlled, Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Evening-dosed Jornay PM (methylphenidate hydrochloride) extended-release in Children Aged 4 to 5 With Attention Deficit Hyperactivity Disorder (ADHD)."~"Released"~"Per FDA letter dated 7/25/2025, this PMR/PMC has been released."~10/31/2026 0:00:00~10/4/2024 0:00:00
383577~"CDER"~"NDA"~209354.00~"BAUSCH HEALTH AMERICAS INC"~"DuobrII (Halobetasol Propionate and Tazarotene)"~4/25/2019 0:00:00~"Original"~1~"3611-1"~"PMR"~"Pediatric Research Equity Act"~3611.00~1~"PMR 3611-1: Pharmacokinetic (PK)/hypothalamic-pituitary-adrenal (HPA) axis suppression/safety open-label study of halobetasol propionate and tazarotene lotion, 0.01%/0.045% in 45 pediatric subjects age 4 to less than 17 years with moderate to severe plaque psoriasis. PK and HPA axis suppression assessments should be done in at least 20 evaluable subjects under maximal use conditions."~"Delayed"~"Original Final Report Due Date: 12/31/2022. Per FDA letter dated 12/07/2022, Final Report due date extended to 12/31/2024. The study completion milestone was also revised. Second Deferral Extension Granted per FDA letter dated 10/24/2025, Final Report due date extended to 12/31/2025. Third Deferral Extension Granted per FDA letter dated 01/13/2026, Final Report due date extended to 04/30/2026."~4/30/2026 0:00:00~6/24/2026 0:00:00
383578~"CDER"~"NDA"~209355.00~"BAUSCH HEALTH AMERICAS INC"~"BRYHALI (halobetasol propionate)"~11/6/2018 0:00:00~"Original"~1~"3470-1"~"PMR"~"Pediatric Research Equity Act"~3470.00~1~"PMR 3470-1: Conduct a maximal use pharmacokinetic (PK) and hypothalamic-pituitary-adrenal (HPA) axis suppression study following topical application of IDP-122 (Halobetasol propionate, 0.01%) Lotion in subjects 6 to less than 17 years of age with moderate to severe plaque psoriasis."~"Delayed"~"Original Final Report Due Date: 12/31/2020. Deferral Extension granted per FDA letter dated 6/22/2020. The final report submission milestone was missed due to recruitment challenges. Per the annual status report, the older age group was fully enrolled and completed, but the younger age group enrolled 3 subjects of the 20 needed. A Second Deferral Extension Request was submitted (sequence 0043) due to COVID-19 study pause."~12/31/2021 0:00:00~12/19/2025 0:00:00
383579~"CDER"~"NDA"~209381.00~"SALIX PHARMACEUTICALS INC"~"Plenvu (PEG 3350, Sodium Ascorbate, Sodium Sulfate, Ascorbic Acid, Sodium Chloride, and Potassium Chloride)"~5/4/2018 0:00:00~"Original"~1~"3371-1"~"PMR"~"Pediatric Research Equity Act"~3371.00~1~"PMR 3371-1: A study to evaluate safety, efficacy, tolerability, palatability and acceptability of PLENVU vs. an approved comparator in patients 12 to < 18 years of age. The study will include dose determination and PK evaluation."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 10/02/2024."~9/30/2026 0:00:00~7/2/2026 0:00:00
383580~"CDER"~"NDA"~209381.00~"SALIX PHARMACEUTICALS INC"~"Plenvu (PEG 3350, Sodium Ascorbate, Sodium Sulfate, Ascorbic Acid, Sodium Chloride, and Potassium Chloride)"~5/4/2018 0:00:00~"Original"~1~"3371-2"~"PMR"~"Pediatric Research Equity Act"~3371.00~2~"PMR 3371-2: A study to evaluate safety, efficacy, tolerability, palatability and acceptability of PLENVU vs. an approved comparator in patients 2 to < 12 years of age. The study will include dose determination and PK evaluation."~"Delayed"~"Original Final Report Due Date: 06/30/2021. Per FDA letter dated 10/30/2020, final report due date extended to 06/30/2023. Second Deferral Extension Granted per FDA letter dated 05/08/2023. The final protocol submission and trial completion milestones were also revised."~9/30/2026 0:00:00~7/2/2026 0:00:00
383581~"CDER"~"NDA"~209381.00~"SALIX PHARMACEUTICALS INC"~"Plenvu (PEG 3350, Sodium Ascorbate, Sodium Sulfate, Ascorbic Acid, Sodium Chloride, and Potassium Chloride)"~5/4/2018 0:00:00~"Original"~1~"3371-3"~"PMR"~"Pediatric Research Equity Act"~3371.00~3~"PMR 3371-3: A study to evaluate safety, efficacy, tolerability of PLENVU vs. an approved comparator in patients 1 to < 2 years of age. The study will include dose determination and PK evaluation."~"Delayed"~"Original Final Report Due Date: 07/31/2022. Per FDA letter dated 10/30/2020, final report due date extended to 02/28/2024. Second Deferral Extension Granted per FDA letter dated 05/08/2023. The final protocol submission and trial completion milestones were also revised."~9/30/2028 0:00:00~7/2/2026 0:00:00
383582~"CDER"~"NDA"~209388.00~"QOL MEDICAL LLC"~"Gimoti (metoclopramide)"~6/19/2020 0:00:00~"Original"~1~"3859-1"~"PMC"~"506B PMC"~3859.00~1~"PMC 3859-1: Conduct a single dose pharmacokinetics trial of Gimoti (metoclopramide) nasal spray in healthy subjects to characterize dose proportionality of the lower strength (e.g.,7.5 mg) and the 15 mg dose strengths. Develop a lower dosage strength (e.g., 7.5-mg) to accommodate various situations requiring further dosage adjustments."~"Delayed"~"The original final report submission milestone was missed. Revised milestones were acknowledged in a letter dated 2/27/2023."~9/30/2022 0:00:00~8/8/2025 0:00:00
383583~"CDER"~"NDA"~209445.00~"SHIONOGI INC"~"FETROJA (cefiderocol)"~11/14/2019 0:00:00~"Original"~1~"3744-3"~"PMR"~"505 (o)(3)"~3744.00~3~"PMR 3744-3: Conduct US surveillance studies for five years from the date of marketing FETROJA to determine if resistance to cefiderocol has
developed in those organisms specific to the cUTI indication in the label."~"Submitted"~~3/31/2026 0:00:00~1/12/2026 0:00:00
383584~"CDER"~"NDA"~209445.00~"SHIONOGI INC"~"FETROJA (cefiderocol)"~11/14/2019 0:00:00~"Original"~1~"3744-4"~"PMR"~"505 (o)(3)"~3744.00~4~"PMR 3744-4: Conduct a study to define the mechanism(s) of resistance to FETROJA (cefiderocol) for isolates identified as being resistant to cefiderocol in the surveillance study (five years from the date of marketing)."~"Ongoing"~~8/31/2026 0:00:00~1/12/2026 0:00:00
383585~"CDER"~"NDA"~209471.00~"AFT PHARMACEUTICALS US INC"~"Combogesic (acetaminophen and ibuprofen)"~3/1/2023 0:00:00~"Original"~1~"4401-1"~"PMR"~"Pediatric Research Equity Act"~4401.00~1~"PMR 4401-1: Conduct a study of the efficacy, safety, and pharmacokinetics of Combogesic in patients aged 12 to less than 17 years of age with mild to moderate acute pain."~"Delayed"~"The final protocol submission was missed because Applicant
needed more time to review with CRO and FDA feedback. Original Final Report Due Date:08/2025; Deferral Extension granted per FDA letter dated 7/31/2025.
"~3/31/2027 0:00:00~4/22/2026 0:00:00
383586~"CDER"~"NDA"~209471.00~"AFT PHARMACEUTICALS US INC"~"Combogesic (acetaminophen and ibuprofen)"~3/1/2023 0:00:00~"Original"~1~"4401-2"~"PMR"~"Pediatric Research Equity Act"~4401.00~2~"PMR 4401-2: Conduct a study of the efficacy, safety, and pharmacokinetics of Combogesic Oral Suspension in patients aged 2 to less than 12 years of age with mild to moderate acute pain."~"Delayed"~"The draft and final protocol submission was missed."~12/31/2027 0:00:00~4/22/2026 0:00:00
383587~"CDER"~"NDA"~209471.00~"AFT PHARMACEUTICALS US INC"~"Combogesic (acetaminophen and ibuprofen)"~3/1/2023 0:00:00~"Original"~1~"4401-3"~"PMR"~"Pediatric Research Equity Act"~4401.00~3~"PMR 4401-3: Conduct a study of the efficacy, safety, and pharmacokinetics of Combogesic Oral Suspension in infants from 6 months to less than 2 years of age with mild to moderate acute pain."~"Pending"~"The study has not begun"~4/30/2030 0:00:00~4/22/2026 0:00:00
383588~"CDER"~"NDA"~209482.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY DEVELOPMENT LTD ENGLAND"~"Trelegy Ellipta (Fluticasone Furoate, Umeclidinium, and Vilanterol)"~9/18/2017 0:00:00~"Supplement"~10~"3935-1"~"PMR"~"Pediatric Research Equity Act"~3935.00~1~"PMR 3935-1: Conduct a 24-week, randomized, double-blind, parallel-group, activecontrolled, efficacy and safety study of fluticasone furoate/umeclidinium/vilanterol inhalation powder via the Ellipta device in children 12-17 years of age with asthma."~"Ongoing"~"The study has been initiated."~4/30/2028 0:00:00~11/14/2025 0:00:00
383589~"CDER"~"NDA"~209482.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY DEVELOPMENT LTD ENGLAND"~"Trelegy Ellipta (Fluticasone Furoate, Umeclidinium, and Vilanterol)"~9/18/2017 0:00:00~"Supplement"~10~"3935-2"~"PMR"~"Pediatric Research Equity Act"~3935.00~2~"PMR 3935-2: Conduct a 4-week randomized, double-blind, parallel-group, activecontrolled dose-ranging trial with at least two doses of umeclidinium inhalation powder via the Ellipta device in children 5 to 11 years of age with asthma."~"Pending"~"The study has not begun but does not meet the criterion for delayed."~6/30/2030 0:00:00~11/14/2025 0:00:00
383590~"CDER"~"NDA"~209482.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY DEVELOPMENT LTD ENGLAND"~"Trelegy Ellipta (Fluticasone Furoate, Umeclidinium, and Vilanterol)"~9/18/2017 0:00:00~"Supplement"~10~"3935-3"~"PMR"~"Pediatric Research Equity Act"~3935.00~3~"PMR 3935-3: Conduct a 24-week, randomized, double-blind, parallel-group, activecontrolled, efficacy and safety study of fluticasone furoate/umeclidinium/vilanterol inhalation powder via the Ellipta device in children 5 to 11 years of age with asthma."~"Pending"~"The study has not begun but does not meet the criterion for delayed."~10/31/2033 0:00:00~11/14/2025 0:00:00
383591~"CDER"~"NDA"~209482.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY DEVELOPMENT LTD ENGLAND"~"Trelegy Ellipta (Fluticasone Furoate, Umeclidinium, and Vilanterol)"~9/18/2017 0:00:00~"Supplement"~11~"3935-1"~"PMR"~"Pediatric Research Equity Act"~3935.00~1~"PMR 3935-1: Conduct a 24-week, randomized, double-blind, parallel-group, activecontrolled, efficacy and safety study of fluticasone furoate/umeclidinium/vilanterol inhalation powder via the Ellipta device in children 12-17 years of age with asthma."~"Ongoing"~"The study has been initiated."~4/30/2028 0:00:00~11/14/2025 0:00:00
383592~"CDER"~"NDA"~209482.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY DEVELOPMENT LTD ENGLAND"~"Trelegy Ellipta (Fluticasone Furoate, Umeclidinium, and Vilanterol)"~9/18/2017 0:00:00~"Supplement"~11~"3935-2"~"PMR"~"Pediatric Research Equity Act"~3935.00~2~"PMR 3935-2: Conduct a 4-week randomized, double-blind, parallel-group, activecontrolled dose-ranging trial with at least two doses of umeclidinium inhalation powder via the Ellipta device in children 5 to 11 years of age with asthma."~"Pending"~"The study has not begun but does not meet the criterion for delayed."~6/30/2030 0:00:00~11/14/2025 0:00:00
383593~"CDER"~"NDA"~209482.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY DEVELOPMENT LTD ENGLAND"~"Trelegy Ellipta (Fluticasone Furoate, Umeclidinium, and Vilanterol)"~9/18/2017 0:00:00~"Supplement"~11~"3935-3"~"PMR"~"Pediatric Research Equity Act"~3935.00~3~"PMR 3935-3: Conduct a 24-week, randomized, double-blind, parallel-group, activecontrolled, efficacy and safety study of fluticasone furoate/umeclidinium/vilanterol inhalation powder via the Ellipta device in children 5 to 11 years of age with asthma."~"Pending"~"The study has not begun but does not meet the criterion for delayed."~10/31/2033 0:00:00~11/14/2025 0:00:00
383594~"CDER"~"NDA"~209483.00~"PRIMUS PHARMACEUTICALS INC"~"Impoyz (clobetasol propionate)"~11/28/2017 0:00:00~"Original"~1~"3310-1"~"PMR"~"Pediatric Research Equity Act"~3310.00~1~"PMR 3310-1: A safety pharmacokinetic / hypothalamic-pituitary-adrenal (HPA) axis suppression study under maximal use conditions in children and adolescents in the age group of 6 years to 16 year and 11 months old."~"Delayed"~"A Not Fulfilled letter was issued on 06/09/2025."~4/30/2024 0:00:00~1/9/2026 0:00:00
383595~"CDER"~"NDA"~209500.00~"INTRA-CELLULAR THERAPIES INC"~"Caplyta (lumateperone)"~12/20/2019 0:00:00~"Original"~1~"3760-3"~"PMR"~"Pediatric Research Equity Act"~3760.00~3~"PMR 3760-3: Conduct an open-label study to assess the long-term safety of lumateperone in patients aged 13 to 17 years diagnosed with
schizophrenia."~"Ongoing"~"First Patient First Visit occurred on January 25, 2024 and the study is ongoing."~6/30/2028 0:00:00~2/11/2026 0:00:00
383596~"CDER"~"NDA"~209500.00~"INTRA-CELLULAR THERAPIES INC"~"Caplyta (lumateperone)"~12/20/2019 0:00:00~"Original"~1~"3760-7"~"PMC"~"506B PMC"~3760.00~7~"PMC 3760-7: Conduct a placebo-controlled, randomized withdrawal maintenance study of lumateperone in patients with schizophrenia."~"Fulfilled"~~6/30/2024 0:00:00~2/11/2026 0:00:00
383597~"CDER"~"NDA"~209500.00~"INTRA-CELLULAR THERAPIES INC"~"Caplyta (lumateperone)"~12/20/2019 0:00:00~"Supplement"~5~"4192-2"~"PMR"~"Pediatric Research Equity Act"~4192.00~2~"PMR 4192-2: Conduct an open-label, multiple oral dose study to demonstrate the safety, tolerability, and pharmacokinetics of lumateperone in patients ages 10 to 17 years with major depressive episode with bipolar I or II disorder (bipolar depression)."~"Fulfilled"~"Per FDA letter dated 06/12/2026, this PMR/PMC has been fulfilled."~7/31/2025 0:00:00~2/11/2026 0:00:00
383598~"CDER"~"NDA"~209500.00~"INTRA-CELLULAR THERAPIES INC"~"Caplyta (lumateperone)"~12/20/2019 0:00:00~"Supplement"~5~"4192-3"~"PMR"~"Pediatric Research Equity Act"~4192.00~3~"PMR 4192-3: Conduct a randomized, double-blind, placebo-controlled study to assess the efficacy and safety of lumateperone for the treatment of major depressive episode associated with bipolar I or II disorder (bipolar depression) in patients aged 10 to 17 years."~"Ongoing"~"First Patient First Visit occurred on May 13, 2024 and the study is ongoing."~11/30/2027 0:00:00~2/11/2026 0:00:00
383599~"CDER"~"NDA"~209478.00~"CMP DEVELOPMENT LLC"~"CaroSpir (Spironolactone)"~8/4/2017 0:00:00~"Original"~1~"3256-4"~"PMR"~"Pediatric Research Equity Act"~3256.00~4~"PMR 3256-4: Conduct a study assessing the pharmacokinetics, safety and pharmacodynamics of single and multiple doses of spironolactone oral suspension in pediatric patients with edema due to heart failure or cirrhosis."~"Ongoing"~"Site recruitment attempts are still ongoing. To date, an insufficient number of sites have shown willingness to participate."~3/30/2027 0:00:00~10/3/2025 0:00:00
383600~"CDER"~"NDA"~209500.00~"INTRA-CELLULAR THERAPIES INC"~"Caplyta (lumateperone)"~12/20/2019 0:00:00~"Supplement"~5~"4192-4"~"PMR"~"Pediatric Research Equity Act"~4192.00~4~"PMR 4192-4: Conduct an open-label study to assess the long-term safety of lumateperone in patients aged 10 to 17 years with major depressive
episode associated with bipolar I or II disorder (bipolar depression)."~"Ongoing"~"First Patient First Visit occurred on January 25, 2024 and the study is ongoing."~5/31/2028 0:00:00~2/11/2026 0:00:00
383601~"CDER"~"NDA"~209500.00~"INTRA-CELLULAR THERAPIES INC"~"Caplyta (lumateperone)"~12/20/2019 0:00:00~"Supplement"~6~"4192-2"~"PMR"~"Pediatric Research Equity Act"~4192.00~2~"PMR 4192-2: Conduct an open-label, multiple oral dose study to demonstrate the safety, tolerability, and pharmacokinetics of lumateperone in patients ages 10 to 17 years with major depressive episode with bipolar I or II disorder (bipolar depression)."~"Fulfilled"~"Per FDA letter dated 06/12/2026, this PMR/PMC has been fulfilled."~7/31/2025 0:00:00~2/11/2026 0:00:00
383602~"CDER"~"NDA"~209500.00~"INTRA-CELLULAR THERAPIES INC"~"Caplyta (lumateperone)"~12/20/2019 0:00:00~"Supplement"~6~"4192-3"~"PMR"~"Pediatric Research Equity Act"~4192.00~3~"PMR 4192-3: Conduct a randomized, double-blind, placebo-controlled study to assess the efficacy and safety of lumateperone for the treatment of major depressive episode associated with bipolar I or II disorder (bipolar depression) in patients aged 10 to 17 years."~"Ongoing"~"First Patient First Visit occurred on May 13, 2024 and the study is ongoing."~11/30/2027 0:00:00~2/11/2026 0:00:00
383603~"CDER"~"NDA"~209500.00~"INTRA-CELLULAR THERAPIES INC"~"Caplyta (lumateperone)"~12/20/2019 0:00:00~"Supplement"~6~"4192-4"~"PMR"~"Pediatric Research Equity Act"~4192.00~4~"PMR 4192-4: Conduct an open-label study to assess the long-term safety of lumateperone in patients aged 10 to 17 years with major depressive
episode associated with bipolar I or II disorder (bipolar depression)."~"Ongoing"~"First Patient First Visit occurred on January 25, 2024 and the study is ongoing."~5/31/2028 0:00:00~2/11/2026 0:00:00
383604~"CDER"~"NDA"~215206.00~"ABBVIE INC"~"Qulipta (atogepant)"~9/28/2021 0:00:00~"Original"~1~"4152-2"~"PMR"~"Pediatric Research Equity Act"~4152.00~2~"PMR 4152-2: A randomized, double-blind, placebo-controlled efficacy and safety study under PREA for the preventive treatment of episodic migraine in children 6 through 17 years of age. This efficacy study must be designed to show superiority of atogepant over placebo and is to be submitted as a special protocol assessment (SPA). A pharmacokinetic evaluation needs to be conducted in children 6 through 11 years of age to determine the appropriate dosing in the efficacy portion of the study in this age group."~"Ongoing"~"The trial has been initiated, and 330 patients are currently enrolled."~2/28/2030 0:00:00~11/20/2025 0:00:00
383605~"CDER"~"NDA"~215206.00~"ABBVIE INC"~"Qulipta (atogepant)"~9/28/2021 0:00:00~"Original"~1~"4152-6"~"PMR"~"505 (o)(3)"~4152.00~6~"PMR 4152-6: Prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with migraine exposed to atogepant during pregnancy with two unexposed control populations: one consisting of women with migraine who have not been exposed to atogepant before and during pregnancy and the other consisting of women without migraine. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~2/28/2037 0:00:00~11/20/2025 0:00:00
383606~"CDER"~"NDA"~215206.00~"ABBVIE INC"~"Qulipta (atogepant)"~9/28/2021 0:00:00~"Original"~1~"4152-7"~"PMR"~"505 (o)(3)"~4152.00~7~"PMR 4152-7: A pregnancy outcomes study using a different study design than provided for in PMR 4152-6 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to atogepant during pregnancy compared to an unexposed control population."~"Ongoing"~~4/30/2031 0:00:00~11/20/2025 0:00:00
383607~"CDER"~"NDA"~215206.00~"ABBVIE INC"~"Qulipta (atogepant)"~9/28/2021 0:00:00~"Supplement"~4~"4431-1"~"PMR"~"Pediatric Research Equity Act"~4431.00~1~"PMR 4431-1: A randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of atogepant for the preventive treatment of chronic migraine in children 12 through 17 years of age. This study must be designed to show superiority of atogepant over placebo. 
"~"Ongoing"~"The trial has been initiated, and 39 patients are currently enrolled."~6/30/2032 0:00:00~11/20/2025 0:00:00
383608~"CDER"~"NDA"~215206.00~"ABBVIE INC"~"Qulipta (atogepant)"~9/28/2021 0:00:00~"Supplement"~4~"4431-2"~"PMR"~"Pediatric Research Equity Act"~4431.00~2~"PMR 4431-2: A study to evaluate the long-term safety of atogepant for the preventive treatment of migraine in patients 6 through 17 years of age. This study should be a minimum of 1 year in length. 
"~"Ongoing"~"The trial has been initiated, and 292 patients are currently enrolled."~6/30/2032 0:00:00~11/20/2025 0:00:00
383609~"CDER"~"NDA"~215211.00~"AMICUS THERAPEUTICS US LLC"~"Opfolda (miglustat)"~9/28/2023 0:00:00~"Original"~1~"4234-1"~"PMR"~"505 (o)(3)"~4234.00~1~"PMR 4234-1: An evaluation of the effects of miglustat on the QTc interval designed according to ICH E14 guidances for industry  E14 Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non- Antiarrhythmic Drugs  (October 2005) and  E14 and S7B Clinical and Nonclinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential - Questions and Answers  (August 2022)."~"Fulfilled"~~9/30/2025 0:00:00~11/21/2025 0:00:00
383610~"CDER"~"NDA"~215211.00~"AMICUS THERAPEUTICS US LLC"~"Opfolda (miglustat)"~9/28/2023 0:00:00~"Original"~1~"4234-2"~"PMR"~"505 (o)(3)"~4234.00~2~"PMR 4234-2: In vitro studies to evaluate whether miglustat is a substrate, inhibitor, or inducer of metabolizing enzymes and transporters as outlined in the FDA Guidance for Industry  In Vitro Drug Interaction Studies - Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions  (January 2020). If in vitro studies suggest a potential for drug interaction, additional in vivo studies may be required."~"Fulfilled"~~6/30/2024 0:00:00~11/21/2025 0:00:00
383611~"CDER"~"NDA"~215239.00~"CATALYST PHARMACEUTICALS INC"~"Agamree (vamorolone)"~10/26/2023 0:00:00~"Original"~1~"4515-1"~"PMR"~"505 (o)(3)"~4515.00~1~"PMR 4515-1: Conduct a 2-year carcinogenicity study of vamorolone in rat."~"Delayed"~"The Draft and Final Protocol milestones were missed because of additional time needed for the Agency to provide a response. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 07/01/2025."~3/31/2028 0:00:00~12/19/2025 0:00:00
383612~"CDER"~"NDA"~215239.00~"CATALYST PHARMACEUTICALS INC"~"Agamree (vamorolone)"~10/26/2023 0:00:00~"Original"~1~"4515-2"~"PMR"~"505 (o)(3)"~4515.00~2~"PMR 4515-2: Conduct a carcinogenicity study of vamorolone in mouse."~"Delayed"~"The final report submission milestone was missed because there was a delay in the final protocol submission leading to a delay in the initiation of the study and a foreseeable delay in the final report submission. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 03/19/2026."~11/30/2025 0:00:00~12/19/2025 0:00:00
383613~"CDER"~"NDA"~215239.00~"CATALYST PHARMACEUTICALS INC"~"Agamree (vamorolone)"~10/26/2023 0:00:00~"Original"~1~"4515-3"~"PMR"~"505 (o)(3)"~4515.00~3~"PMR 4515-3: Conduct a trial to evaluate the CYP3A4 induction potential of vamorolone in a dedicated clinical drug-drug interaction study to address the serious risk of altered pharmacokinetics of CYP3A4 substrates when used with vamorolone. The study will be an open-label, fixed-sequence phase 1 study conducted in healthy volunteers (male and female). A specific CYP3A4 substrate (e.g., midazolam) would be administered on Day 1. After a sufficient washout, vamorolone would be administered at the highest dose (6 mg/kg) for sufficient duration to induce CYP3A4 (usually around 10-14 days), followed by the administration of specific CYP3A4 substrate along with vamorolone. Pharmacokinetics of the CYP3A4 substrate would be evaluated before and after vamorolone administration to assess the magnitude of CYP3A4 induction."~"Fulfilled"~~6/30/2025 0:00:00~12/19/2025 0:00:00
383614~"CDER"~"NDA"~215244.00~"STEALTH BIOTHERAPEUTICS INC"~"Forzinity (elamipretide) injection"~9/19/2025 0:00:00~"Original"~1~"4802-1"~"PMR"~"Accelerated Approval"~4802.00~1~"PMR 4802-1: Conduct a randomized, double-blind, placebo-controlled trial in patients 5 years and older with Barth syndrome to verify and describe the clinical benefit of Forzinity predicted by improvements in knee extensor muscle strength assessed by handheld dynamometry."~"Delayed"~"The applicant requested a revised milestone because you will need to modify protocol SPIBA-401 to address our December 8, 2025 comments under IND 137429, prior to initiating the first study site. A Revised milestone was acknowledged in a letter dated 12/19/2025."~3/31/2030 0:00:00~
383615~"CDER"~"NDA"~215244.00~"STEALTH BIOTHERAPEUTICS INC"~"Forzinity (elamipretide) injection"~9/19/2025 0:00:00~"Original"~1~"4802-2"~"PMR"~"505 (o)(3)"~4802.00~2~"PMR 4802-2: Complete a 26-week GLP carcinogenicity study of elamipretide administered by subcutaneous injection in TgRasH2 mice. Submit the final study report and updated labeling for FDA review."~"Submitted"~~2/28/2026 0:00:00~
383616~"CDER"~"NDA"~215244.00~"STEALTH BIOTHERAPEUTICS INC"~"Forzinity (elamipretide) injection"~9/19/2025 0:00:00~"Original"~1~"4802-3"~"PMR"~"505 (o)(3)"~4802.00~3~"PMR 4802-3: Complete a 2-year GLP carcinogenicity study of elamipretide administered by subcutaneous injection in rats. Submit the final study report and updated labeling for FDA review."~"Pending"~~1/31/2030 0:00:00~
383617~"CDER"~"NDA"~216660.00~"AMYLYX PHARMACEUTICALS INC"~"Relyvrio (sodium phenylbutyrate and taurursodiol)"~9/29/2022 0:00:00~"Original"~1~"4346-5"~"PMR"~"505 (o)(3)"~4346.00~5~"PMR 4346-5: Conduct an in vivo pharmacokinetic drug interaction study to evaluate the effect of Relyvrio as an inhibitor of OAT1, BCRP, and P-gP. We recommend you consider evaluating these drug interactions as a single cocktail DDI study with an appropriate probe substrate of each transporter.Please refer to the Guidance for Industry Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interaction (https://www.fda.gov/media/134581/download)."~"Released"~~7/31/2024 0:00:00~11/26/2024 0:00:00
383618~"CDER"~"NDA"~216660.00~"AMYLYX PHARMACEUTICALS INC"~"Relyvrio (sodium phenylbutyrate and taurursodiol)"~9/29/2022 0:00:00~"Original"~1~"4346-6"~"PMR"~"505 (o)(3)"~4346.00~6~"PMR 4346-6: Conduct a clinical trial to evaluate the effect of hepatic impairment on the exposure of sodium phenylbutyrate and taurursodiol after oral administration of Relyvrio (sodium phenylbutyrate and taurursodiolursodiol) relative to that in subjects with normal hepatic function. Please refer to the Guidance for Industry Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling(https://www.fda.gov/regulatory-information/search-fda-guidance documents/pharmacokinetics-patients- impaired-hepatic-function-study-design-data-analysis-and-impact-dosing-and)"~"Released"~~12/31/2024 0:00:00~11/26/2024 0:00:00
383619~"CDER"~"NDA"~216660.00~"AMYLYX PHARMACEUTICALS INC"~"Relyvrio (sodium phenylbutyrate and taurursodiol)"~9/29/2022 0:00:00~"Original"~1~"4346-7"~"PMR"~"505 (o)(3)"~4346.00~7~"PMR 4346-7: Conduct a clinical trial to evaluate the effect of renal impairment on the exposure of sodium phenylbutyrate and taurursodiol after oral administration of Relyvrio (sodium phenylbutyrate and taurursodiol) relative to that in subjects with normal renal function. Please refer to the Guidance for Industry Pharmacokinetics in Patients with Impaired Renal Function: Study Design, Data Analysis, and Impact on Dosing and Labeling(https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInf ormation/Guidances/UCM204959.pdf)."~"Released"~~12/31/2024 0:00:00~11/26/2024 0:00:00
383620~"CDER"~"NDA"~216665.00~"SOLENO THERAPEUTICS INC"~"Vykat XR (diazoxide choline) extended-release tablets"~3/26/2025 0:00:00~"Original"~1~"4793-1"~"PMR"~"505 (o)(3)"~4793.00~1~"PMR 4793-1: Conduct a 6-month carcinogenicity study of diazoxide choline orally administered to transgenic mice, using an appropriate mouse model."~"Pending"~~2/28/2027 0:00:00~5/22/2026 0:00:00
383621~"CDER"~"NDA"~216665.00~"SOLENO THERAPEUTICS INC"~"Vykat XR (diazoxide choline) extended-release tablets"~3/26/2025 0:00:00~"Original"~1~"4793-2"~"PMR"~"505 (o)(3)"~4793.00~2~"PMR 4793-2: Conduct a dedicated clinical study to assess the effect of hepatic impairment on the pharmacokinetics of diazoxide choline and its major metabolites."~"Pending"~~6/30/2027 0:00:00~5/22/2026 0:00:00
383622~"CDER"~"NDA"~216665.00~"SOLENO THERAPEUTICS INC"~"Vykat XR (diazoxide choline) extended-release tablets"~3/26/2025 0:00:00~"Original"~1~"4793-3"~"PMR"~"505 (o)(3)"~4793.00~3~"PMR 4793-3: Conduct a dedicated clinical study to assess the effect of renal impairment on the pharmacokinetics of diazoxide choline and its major metabolites."~"Pending"~~6/30/2027 0:00:00~5/22/2026 0:00:00
383623~"CDER"~"NDA"~216665.00~"SOLENO THERAPEUTICS INC"~"Vykat XR (diazoxide choline) extended-release tablets"~3/26/2025 0:00:00~"Original"~1~"4793-4"~"PMR"~"505 (o)(3)"~4793.00~4~"PMR 4793-4: Conduct a dedicated drug interaction study to assess the effect of diazoxide on a substrate of CYP1A2"~"Pending"~~6/30/2027 0:00:00~5/22/2026 0:00:00
383624~"CDER"~"NDA"~216675.00~"BAUSCH AND LOMB INC"~"Miebo (perfluorohexyloctane)"~5/18/2023 0:00:00~"Original"~1~"4447-1"~"PMR"~"505 (o)(3)"~4447.00~1~"PMR 4447-1: Conduct a randomized, controlled trial to evaluate the corneal endothelial health of eyes treated with perfluorohexyloctane ophthalmic solution by monitoring the number/density of corneal endothelial cells using specular microscopy at baseline and over a period of at least one year in at least 100 patients receiving perfluorohexyloctane ophthalmic solution."~"Ongoing"~~9/30/2026 0:00:00~7/16/2025 0:00:00
383625~"CDER"~"NDA"~216686.00~"STERISCIENCE PTE LTD"~"Focinvez (fosaprepitant injection)"~8/22/2023 0:00:00~"Original"~1~"4483-1"~"PMR"~"Pediatric Research Equity Act"~4483.00~1~"PMR 4483-1: A study to evaluate pharmacokinetics, safety, and tolerability of a single dose of Fosaprepitant Injection 150 mg/50 mL (3 mg/mL) for prevention of chemotherapy-induced nausea and vomiting in pediatric patients 0 to 6 months of age undergoing highly emetogenic cancer chemotherapy (HEC) or moderately emetogenic cancer chemotherapy (MEC)."~"Delayed"~"The final protocol milestone was missed."~4/30/2030 0:00:00~10/17/2025 0:00:00
383626~"CDER"~"NDA"~216718.00~"BIOGEN US CORPORATION"~"Skyclarys (omaveloxolone)"~2/28/2023 0:00:00~"Original"~1~"4410-1"~"PMR"~"505 (o)(3)"~4410.00~1~"PMR 4410-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to omaveloxolone during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~4/30/2036 0:00:00~4/27/2026 0:00:00
383627~"CDER"~"NDA"~216718.00~"BIOGEN US CORPORATION"~"Skyclarys (omaveloxolone)"~2/28/2023 0:00:00~"Original"~1~"4410-2"~"PMR"~"505 (o)(3)"~4410.00~2~"PMR 4410-2: Perform a lactation study (milk only) in lactating women who have received therapeutic doses of omaveloxolone using a validated assay to assess concentrations of omaveloxolone in breast milk and the effects on the breastfed infant as applicable."~"Ongoing"~~5/31/2026 0:00:00~4/27/2026 0:00:00
383628~"CDER"~"NDA"~216718.00~"BIOGEN US CORPORATION"~"Skyclarys (omaveloxolone)"~2/28/2023 0:00:00~"Original"~1~"4410-4"~"PMR"~"505 (o)(3)"~4410.00~4~"PMR 4410-4: Conduct a 2-year carcinogenicity study of omaveloxolone in rat."~"Fulfilled"~~9/30/2025 0:00:00~4/27/2026 0:00:00
383629~"CDER"~"NDA"~216755.00~"SAPTALIS PHARMACEUTICALS LLC"~"Likmez (metronidazole)"~9/22/2023 0:00:00~"Original"~1~"4502-1"~"PMR"~"Pediatric Research Equity Act"~4502.00~1~"PMR 4502-1: Conduct a phase 2, open-label, single-arm, pharmacokinetic, and safety study in pediatric patients aged 12 months to < 4 years with anaerobic bacterial infections."~"Delayed"~"The final protocol submission milestone had been revised. Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/12/2026."~6/30/2028 0:00:00~11/21/2025 0:00:00
383630~"CDER"~"NDA"~216755.00~"SAPTALIS PHARMACEUTICALS LLC"~"Likmez (metronidazole)"~9/22/2023 0:00:00~"Original"~1~"4502-2"~"PMR"~"Pediatric Research Equity Act"~4502.00~2~"PMR 4502-2: Conduct a search and provide a summary of the published literature assessing dosing, safety, efficacy of metronidazole, and the effect of disease state on metronidazole pharmacokinetics in pediatric patients to support an indication for the treatment of anaerobic bacterial infections in pediatric patients from birth to < 12 months and 4 years to < 18 years of age."~"Delayed"~"Original Final Report Due Date: 06/30/2025. Per FDA letter dated 12/18/2025, final report due date extended to 06/30/2026. Second Deferral Extension Granted Per FDA letter 05/12/2026."~6/30/2028 0:00:00~11/21/2025 0:00:00
383631~"CDER"~"NDA"~216755.00~"SAPTALIS PHARMACEUTICALS LLC"~"Likmez (metronidazole)"~9/22/2023 0:00:00~"Original"~1~"4502-3"~"PMR"~"Pediatric Research Equity Act"~4502.00~3~"PMR 4502-3: Conduct a search and provide a summary of the published literature assessing dosing, safety, efficacy of metronidazole, and the effect of disease state on metronidazole pharmacokinetics in pediatric patients to support an indication for the treatment of trichomoniasis in pediatric patients aged 12 years to < 18 years."~"Delayed"~"Original Final Report Due Date: 06/30/2025. Per FDA letter dated 12/18/2025, final report due date extended to 06/30/2026. Second Deferral Extension Granted Per FDA letter 05/12/2026."~6/30/2026 0:00:00~11/21/2025 0:00:00
383632~"CDER"~"NDA"~211964.00~"SUPERNUS PHARMACEUTICALS INC"~"Qelbree (viloxazine)"~4/2/2021 0:00:00~"Original"~1~"3942-1"~"PMR"~"Pediatric Research Equity Act"~3942.00~1~"PMR 3942-1: Conduct an adequately powered, double-blind, placebo-controlled efficacy and safety study of viloxazine extended-release capsules (viloxazine ER) in male and female patients ages 4 to < 6 years with attention deficit hyperactivity disorder."~"Delayed"~"Original Final Report Due Date: 09/30/2022; Deferral Extension granted per FDA letter dated 10/20/2022. The final protocol submission and study completion milestones were also revised."~9/30/2026 0:00:00~5/29/2026 0:00:00
383633~"CDER"~"NDA"~211964.00~"SUPERNUS PHARMACEUTICALS INC"~"Qelbree (viloxazine)"~4/2/2021 0:00:00~"Original"~1~"3942-2"~"PMR"~"Pediatric Research Equity Act"~3942.00~2~"PMR 3942-2: Conduct a long-term (6-month), open-label safety extension study to evaluate the safety and tolerability of viloxazine extended-release capsules (viloxazine ER) as monotherapy for attention deficit hyperactivity disorder in male and female patients ages 4 to < 6 years."~"Delayed"~"Original Final Report Due Date: 03/31/2023; Deferral Extension granted per FDA letter dated 10/20/2022. The final protocol submission and study completion milestones were also revised."~9/30/2026 0:00:00~5/29/2026 0:00:00
383634~"CDER"~"NDA"~211964.00~"SUPERNUS PHARMACEUTICALS INC"~"Qelbree (viloxazine)"~4/2/2021 0:00:00~"Original"~1~"3942-3"~"PMR"~"505 (o)(3)"~3942.00~3~"PMR 3942-3: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to viloxazine extended-release capsules (viloxazine) during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through the first year of life. Maternal deaths through the first year postpartum will be reported. The study will collect information for a minimum of 10 years. Results will be analyzed and reported descriptively. Data collected retrospectively will be analyzed separately and reported with the interim and final study reports."~"Ongoing"~~5/31/2032 0:00:00~5/29/2026 0:00:00
383635~"CDER"~"NDA"~211970.00~"SAREPTA THERAPEUTICS INC"~"Vyondys 53 (golodirsen)"~12/12/2019 0:00:00~"Original"~1~"3690-7"~"PMR"~"505 (o)(3)"~3690.00~7~"PMR 3690-7: Submit ECG data from Study 4045-301 to support your request to waive a thorough QT study. If these data do not support a TQT study waiver, you will need to evaluate the effect of golodirsen on the QTc interval in a dedicated study as per the ICH E14 guideline."~"Delayed"~"The study completion and final report milestones were missed, because the timelines were dependent on completion of PMR 3690-1 and FDA determined that the applicant had sufficient justification for the anticipated delay and acknowledged revised milestones in 5/18/22 letter."~10/31/2024 0:00:00~2/9/2026 0:00:00
383636~"CDER"~"NDA"~211970.00~"SAREPTA THERAPEUTICS INC"~"Vyondys 53 (golodirsen)"~12/12/2019 0:00:00~"Original"~1~"3690-8"~"PMR"~"Accelerated Approval"~3690.00~8~"PMR 3690-8: Complete Study 4045-301, A Double-Blind, Placebo-Controlled, Multicenter Study with an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy which is intended to verify the clinical benefit of golodirsen."~"Submitted"~"The final report was submitted to FDA on 4/29/2026."~4/30/2026 0:00:00~2/9/2026 0:00:00
383637~"CDER"~"NDA"~211988.00~"HERON THERAPEUTICS INC"~"Zynrelef (bupivacaine and meloxicam)"~5/12/2021 0:00:00~"Original"~1~"4059-3"~"PMR"~"Pediatric Research Equity Act"~4059.00~3~"PMR 4059-3: Conduct a juvenile animal study in an appropriate model to characterize the impact of meloxicam on the developing kidney, liver, lung, and testes to support clinical studies in pediatric patients from birth to less than two years of age."~"Submitted"~"The final report was submitted to FDA on 09/26/2024"~3/31/2024 0:00:00~7/8/2026 0:00:00
383638~"CDER"~"NDA"~211988.00~"HERON THERAPEUTICS INC"~"Zynrelef (bupivacaine and meloxicam)"~5/12/2021 0:00:00~"Original"~1~"4059-4"~"PMR"~"Pediatric Research Equity Act"~4059.00~4~"PMR 4059-4: Conduct a juvenile animal study in the rodent model to characterize the impact of DMSO on the developing brain to support clinical studies in pediatric patients from birth to less than three years of age."~"Submitted"~"The final report was submitted to FDA on 8/31/2022."~2/28/2023 0:00:00~7/8/2026 0:00:00
383639~"CDER"~"NDA"~211988.00~"HERON THERAPEUTICS INC"~"Zynrelef (bupivacaine and meloxicam)"~5/12/2021 0:00:00~"Original"~1~"4059-10"~"PMR"~"Pediatric Research Equity Act"~4059.00~10~"PMR 4059-10: Conduct a multicenter study to assess the pharmacokinetics, safety, and pharmacodynamic response of Zynrelef administered for postoperative analgesia in pediatric patients three to less than 17 years of age undergoing representative soft tissue surgical procedures."~"Pending"~"The Final Protocol was submitted to the FDA on 3/12/2024. The acknowledge protocol letter is being circulated for sign-off."~9/30/2029 0:00:00~7/8/2026 0:00:00
383640~"CDER"~"NDA"~211988.00~"HERON THERAPEUTICS INC"~"Zynrelef (bupivacaine and meloxicam)"~5/12/2021 0:00:00~"Original"~1~"4059-11"~"PMR"~"Pediatric Research Equity Act"~4059.00~11~"PMR 4059-11: Conduct a multicenter study to assess the pharmacokinetics, safety, and pharmacodynamic response of Zynrelef administered for postoperative analgesia in pediatric patients three to less than 17 years of age undergoing representative orthopedic surgical procedures."~"Pending"~"The Final Protocol was submitted to the FDA on 9/12/2024. The acknowledge protocol letter is being circulated for sign-off."~8/31/2030 0:00:00~7/8/2026 0:00:00
383641~"CDER"~"NDA"~211988.00~"HERON THERAPEUTICS INC"~"Zynrelef (bupivacaine and meloxicam)"~5/12/2021 0:00:00~"Original"~1~"4059-12"~"PMR"~"Pediatric Research Equity Act"~4059.00~12~"PMR 4059-12: Conduct a multicenter study to assess the pharmacokinetics, safety, and efficacy of Zynrelef administered for postoperative analgesia in pediatric patients from birth to less than three years of age undergoing representative soft tissue and orthopedic surgical procedures."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~3/31/2031 0:00:00~7/8/2026 0:00:00
383642~"CDER"~"NDA"~211996.00~"FOLDRX PHARMACEUTICALS LLC A WHOLLY OWNED SUB OF PFIZER INC"~"Vyndaqel (tafamidis meglumine)"~5/3/2019 0:00:00~"Original"~1~"3576-1"~"PMR"~"505 (o)(3)"~3576.00~1~"PMR 3576-1: Establish a worldwide pregnancy surveillance study in women exposed to tafamidis meglumine and tafamidis during pregnancy to assess the risks of pregnancy complications and adverse effects on the developing fetus and neonate. The study will collect information for a minimum of 10 years. Provide a complete protocol with details on how pregnancy exposures and outcomes will be captured (e.g., telephone contact number and/or website that will be provided in the product s prescribing information), and measures to ensure complete data capture on pregnancy outcomes and any adverse effects in the offspring 
"~"Ongoing"~~12/31/2030 0:00:00~6/30/2026 0:00:00
383643~"CDER"~"NDA"~212028.00~"EISAI INC"~"Dayvigo (lemborexant)"~12/20/2019 0:00:00~"Original"~1~"3753-4"~"PMR"~"505 (o)(3)"~3753.00~4~"PMR 3753-4: Conduct a prospective, registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to lemborexant during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~2/28/2032 0:00:00~2/13/2026 0:00:00
383644~"CDER"~"NDA"~213411.00~"SEAGEN INC"~"Tukysa (tucatinib)"~4/17/2020 0:00:00~"Supplement"~4~"4388-2"~"PMC"~"506B PMC"~4388.00~2~"PMC 4388-2: Submit adequate analytical and clinical validation results from clinical trial data to support labeling of a test to detect HER2 overexpression or gene amplification, that is essential to the safe and effective use of tucatinib in patients with HER2-positive colorectal cancer."~"Delayed"~"The applicant requested revised milestones because study enrollment has progressed slower than initially anticipated due to low prevalence of HER2+ colorectal cancer. Revised milestones were acknowledged in a letter dated 03/26/2026."~4/30/2026 0:00:00~6/11/2026 0:00:00
383645~"CDER"~"NDA"~213436.00~"IMPEL PHARMACEUTICALS LLC"~"Trudhesa (dihydroergotamine mesylate)"~9/2/2021 0:00:00~"Original"~1~"4136-1"~"PMR"~"Pediatric Research Equity Act"~4136.00~1~"PMR 4136-1: A juvenile animal toxicology study of dihydroergotamine mesylate in rat."~"Delayed"~"The Study Completion and Final Report milestones were missed because the study has not been initiated. Deferral Extension requested on 04/17/2025. Denied per FDA letter dated 05/29/2025."~12/31/2024 0:00:00~11/17/2025 0:00:00
383646~"CDER"~"NDA"~213436.00~"IMPEL PHARMACEUTICALS LLC"~"Trudhesa (dihydroergotamine mesylate)"~9/2/2021 0:00:00~"Original"~1~"4136-2"~"PMR"~"Pediatric Research Equity Act"~4136.00~2~"PMR 4136-2: An open-label, pharmacokinetic study under PREA of Trudhesa in pediatric migraine patients 6 to less than 12 years of age to select dose(s)to be used in the efficacy portion of the study."~"Delayed"~"The Study Completion and Final Report milestones were missed because the juvenile toxicity study has not been initiated. Deferral Extension requested on 04/17/2025. Denied per FDA letter dated 05/29/2025."~12/31/2026 0:00:00~11/17/2025 0:00:00
383647~"CDER"~"NDA"~213436.00~"IMPEL PHARMACEUTICALS LLC"~"Trudhesa (dihydroergotamine mesylate)"~9/2/2021 0:00:00~"Original"~1~"4136-4"~"PMR"~"Pediatric Research Equity Act"~4136.00~4~"PMR 4136-4: A randomized, double blind, placebo controlled efficacy and safety study under PREA to evaluate Trudhesa for the acute treatment of migraine in children 6 to less than 18 years of age. This study should include an initial blinded placebo run-in period to identify placebo non-responders for enrollment into the efficacy portion of the study. The efficacy study must be designed to show superiority of Trudhesa over placebo and is to be submitted as a special protocol assessment (SPA). This study will include an open-label long-term safety study in children 6 to less than 18 years of age for at least 1 year duration."~"Delayed"~"The final protocol submission milestone has been missed.  Deferral Extension requested on 04/17/2025. Denied per FDA letter dated 05/29/2025."~12/31/2026 0:00:00~11/17/2025 0:00:00
383648~"CDER"~"NDA"~213436.00~"IMPEL PHARMACEUTICALS LLC"~"Trudhesa (dihydroergotamine mesylate)"~9/2/2021 0:00:00~"Original"~1~"4136-5"~"PMR"~"505 (o)(3)"~4136.00~5~"PMR 4136-5: A 90-day toxicity study in rat assessing the safety of aci-DHE by intermittent dosing."~"Delayed"~"The Draft Protocol, Final Protocol, Study Completion, and Final Report milestones were missed. Per the applicant s Annual Status Report, the study is currently being discussed with potential contract research organizations."~4/30/2025 0:00:00~11/17/2025 0:00:00
383649~"CDER"~"NDA"~213464.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Lampit (Nifurtimox) "~8/6/2020 0:00:00~"Original"~1~"3868-2"~"PMR"~"505 (o)(3)"~3868.00~2~"PMR 3868-2: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to LAMPIT (nifurtimox) during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant.  Infant outcomes will be assessed through at least the first year of life.  The study will collect information for a minimum of 10 years."~"Ongoing"~~6/30/2032 0:00:00~10/3/2025 0:00:00
383650~"CDER"~"NDA"~213498.00~"VANDA PHARMACEUTICALS INC"~"Ponvory (ponesimod)"~3/18/2021 0:00:00~"Original"~1~"4756-1"~"PMR"~"505 (o)(3)"~4756.00~1~"PMR 4756-1: Conduct an observational study to characterize patients exposed to Ponvory who develop PML in terms of age, duration of exposure to
Ponvory, prior immunosuppressant use, lymphopenia, and anti-JCV antibody status. This study should be of at least 15 years duration to assess risk factors for the development of PML among patients with multiple sclerosis exposed to Ponvory. Potential risk factors to be assessed should include age, duration of exposure to Ponvory, prior immunosuppressant use, lymphopenia, and anti-JCV antibody status. The study should also determine the incidence of PML among patients exposed to Ponvory, both overall and by each potential risk factor."~"Pending"~~7/31/2042 0:00:00~5/13/2026 0:00:00
383651~"CDER"~"NDA"~213498.00~"VANDA PHARMACEUTICALS INC"~"Ponvory (ponesimod)"~3/18/2021 0:00:00~"Original"~1~"4024-2"~"PMR"~"505 (o)(3)"~4024.00~2~"PMR 4024-2: Prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with multiple sclerosis exposed to Ponvory (ponesimod) during pregnancy with two unexposed control populations: one consisting of women with multiple sclerosis who have not been exposed to Ponvory (ponesimod) before or during pregnancy and the other consisting of women without multiple sclerosis. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-forgestational- age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The 2023 Interim Report milestone was missed due to continued negotiations with the Agency pertaining to the protocol. FDA determined there was good cause for the delay and acknowledged the revised milestone in a letter dated 04/11/2024. The 2024 and 2025 interim report milestones have also been missed."~12/31/2034 0:00:00~5/13/2026 0:00:00
383652~"CDER"~"NDA"~213498.00~"VANDA PHARMACEUTICALS INC"~"Ponvory (ponesimod)"~3/18/2021 0:00:00~"Original"~1~"4024-3"~"PMR"~"505 (o)(3)"~4024.00~3~"PMR 4024-3: A pregnancy outcomes study using a different study design than provided for in PMR 4024-2 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case-control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Ponvory (ponesimod) during pregnancy compared to an unexposed control population."~"Delayed"~"The 2023 Interim Report milestone was missed due to continued negotiations with the Agency pertaining to the protocols. FDA determined there was good cause for the delay and acknowledged the revised milestone in a letter dated 04/11/2024. The 2024 and 2025 interim report milestones have also been missed."~12/31/2034 0:00:00~5/13/2026 0:00:00
383653~"CDER"~"NDA"~215244.00~"STEALTH BIOTHERAPEUTICS INC"~"Forzinity (elamipretide) injection"~9/19/2025 0:00:00~"Original"~1~"4802-4"~"PMR"~"505 (o)(3)"~4802.00~4~"PMR 4802-4: Conduct a clinical pharmacokinetic study to evaluate the effect of repeat doses of Forzinity on the single dose pharmacokinetics of metformin, a sensitive multidrug and toxin extrusion protein 1 (MATE1) substrate."~"Pending"~~9/30/2027 0:00:00~
383654~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Original"~1~"4747-1"~"PMR"~"505 (o)(3)"~4747.00~1~"PMR 4747-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of semaglutide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Pending"~~11/30/2026 0:00:00~11/17/2025 0:00:00
383655~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Original"~1~"4081-2"~"PMR"~"Pediatric Research Equity Act"~4081.00~2~"PMR 4081-2: Conduct a randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy after 68 weeks of semaglutide for the treatment of obesity in pediatric patients ages 6 to less than 12. Compare the long-term (at least 2 years) safety and tolerability of semaglutide versus placebo for the treatment of obesity in both children and adolescents (ages 6 to less than 18 years). The trial may not be initiated until results from the semaglutide adolescent trial have been submitted to and reviewed by the Agency."~"Ongoing"~"Recruitment has begun"~6/30/2027 0:00:00~11/17/2025 0:00:00
383656~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Original"~1~"4081-3"~"PMR"~"505 (o)(3)"~4081.00~3~"PMR 4081-3: Conduct a prospective, registry-based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to semaglutide  during pregnancy to an unexposed reference population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~8/31/2033 0:00:00~11/17/2025 0:00:00
383657~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Original"~1~"4081-4"~"PMR"~"505 (o)(3)"~4081.00~4~"PMR 4081-4: Conduct an additional pregnancy study that uses a different observational design from the Pregnancy Exposure Registry, using claims or electronic medical record data, to assess the associations between semaglutide exposure during pregnancy with pregnancy outcomes and infant outcomes, including but not limited to major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age, preterm birth and postnatal growth and development."~"Ongoing"~~8/31/2028 0:00:00~11/17/2025 0:00:00
383658~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Original"~1~"4081-5"~"PMR"~"505 (o)(3)"~4081.00~5~"PMR 4081-5: Conduct a medullary thyroid carcinoma registry-based case series of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of semaglutide for the treatment of obesity into the marketplace. This study will also establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to the use of semaglutide for the treatment of obesity."~"Ongoing"~~2/28/2039 0:00:00~11/17/2025 0:00:00
383659~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Supplement"~7~"4472-2"~"PMR"~"Pediatric Research Equity Act"~4472.00~2~"PMR 4472-2: Conduct exposure and exposure-response analyses to evaluate the efficacy and safety of semaglutide to support the 1.7 mg dose for the treatment of chronic weight management in pediatric patients with obesity ages 6 to less than 12 years. In addition to pharmacokinetic and pharmacodynamic data from STEP 1 and STEP YOUNG, include data from STEP 6 in your analyses."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2027 0:00:00~11/17/2025 0:00:00
383660~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Supplement"~24~"4882-1"~"PMR"~"Accelerated Approval"~4882.00~1~"PMR 4882-1: Complete trial NN9931-4553, a randomized, double-blind, placebocontrolled, parallel-group 240-week trial in patients with metabolic dysfunction-associated steatohepatitis (MASH) with liver fibrosis to demonstrate clinical benefit on the composite endpoint of progression to cirrhosis, hepatic decompensation events, liver transplant, and mortality."~"Ongoing"~"As per the 180-day AA PMR Progress Report submitted on 11/17/2025, the trial is progressing as per the original schedule of milestones. There are currently 1221 of 1231 planned participants enrolled."~12/31/2029 0:00:00~11/17/2025 0:00:00
383661~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Supplement"~24~"4882-2"~"PMR"~"Pediatric Research Equity Act"~4882.00~2~"PMR 4882-2: Conduct a randomized placebo-controlled trial of the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of semaglutide in pediatric patients ages 6 to less than 17 years with non-cirrhotic metabolic dysfunction-associated steatotic liver disease (MASLD) with fibrosis."~"Pending"~"Draft protocol submission 2/2026."~1/31/2030 0:00:00~11/17/2025 0:00:00
383662~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Supplement"~24~"4882-3"~"PMR"~"505 (o)(3)"~4882.00~3~"PMR 4882-3: Modify the ongoing trial NN9931-4553 to consistently evaluate pancreatic safety and function in MASH subjects who present with symptoms and/or signs suggestive of pancreatitis. Assessments should include monitoring of the following at scheduled visits and based on clinical presentation:  symptoms and signs   laboratory tests (i.e., lipase, amylase)   pancreatic imaging   assessment of long-term pancreatic function, including fecal elastase"~"Ongoing"~~12/31/2029 0:00:00~11/17/2025 0:00:00
383663~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Supplement"~24~"4882-4"~"PMR"~"505 (o)(3)"~4882.00~4~"PMR 4882-4: Modify the ongoing trial NN9931-4553 to systematically evaluate bone health in MASH subjects. Assessments should include monitoring of the following: dual-energy X-ray absorptiometry scan   laboratory tests, including 25-hydroxyvitamin D, calcium, phosphorus   biomarkers of bone remodeling   other relevant laboratory or imaging tests, as needed (e.g., PTH) Testing should be conducted at the initiation of this modification, and periodically thereafter."~"Ongoing"~~12/31/2029 0:00:00~11/17/2025 0:00:00
383664~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Supplement"~29~"4975-1"~"PMR"~"Pediatric Research Equity Act"~4975.00~1~"PMR 4975-1: Conduct a randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy after 52 weeks of semaglutide 7.2 mg for the treatment of obesity in pediatric patients ages 12 to less than 18 who still require weight loss after treatment with semaglutide 2.4 mg. Collect information on dysesthesia (altered skin sensation) as an adverse event of special interest, using dedicated case report forms. Provide narrative summaries of dysesthesia adverse events in the clinical study report for this trial."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~1/31/2031 0:00:00~11/17/2025 0:00:00
383665~"CDER"~"NDA"~216774.00~"TEVA PHARMACEUTICALS INC"~"Alvaiz"~11/29/2023 0:00:00~"Original"~1~"4554-1"~"PMR"~"Pediatric Research Equity Act"~4554.00~1~"PMR 4554-1: Develop an appropriate formulation for Alvaiz (eltrombopag choline) that can be used to directly and accurately administer Alvaiz (eltrombopag choline) to pediatric patients less than 6 years of age. Conduct any necessary human factors studies to evaluate the ability of healthcare providers and/or caregivers to measure and administer the appropriate doses."~"Delayed"~"Deferral Extension Requested 03/31/2026. Denied per FDA letter dated 05/27/2026."~12/31/2025 0:00:00~
383666~"CDER"~"NDA"~216793.00~"JANSSEN BIOTECH INC"~"Akeega (niraparib and abiraterone acetate)"~8/11/2023 0:00:00~"Supplement"~1~"4943-1"~"PMC"~"506B PMC"~4943.00~1~"PMC 4943-1: Complete the trial AMPLITUDE (NCT04497844), which is an ongoing phase 3, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of niraparib in combination with abiraterone acetate in patients with homologous recombination repair gene-mutated metastatic castration-sensitive prostate cancer, to obtain the trial s final overall survival (OS) analysis."~"Pending"~~6/30/2028 0:00:00~10/4/2024 0:00:00
383667~"CDER"~"NDA"~216793.00~"JANSSEN BIOTECH INC"~"Akeega (niraparib and abiraterone acetate)"~8/11/2023 0:00:00~"Supplement"~1~"4943-2"~"PMC"~"506B PMC"~4943.00~2~"PMC 4943-2: Conduct an analytical and clinical validation study using clinical trial data, adequate to support the availability of an in vitro diagnostic device using tissue samples that is essential to the safe and effective use of niraparib and abiraterone acetate (AKEEGA) for patients diagnosed with metastatic castration-sensitive prostate cancer (mCSPC), whose tumors harbor BRCA2 mutations."~"Pending"~~3/31/2027 0:00:00~10/4/2024 0:00:00
383668~"CDER"~"NDA"~216834.00~"UCB INC"~"Zilbrysq (zilucoplan)"~10/17/2023 0:00:00~"Original"~1~"4487-1"~"PMR"~"505 (o)(3)"~4487.00~1~"PMR 4487-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to zilucoplan during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~3/31/2036 0:00:00~12/11/2025 0:00:00
383669~"CDER"~"NDA"~216834.00~"UCB INC"~"Zilbrysq (zilucoplan)"~10/17/2023 0:00:00~"Original"~1~"4487-2"~"PMR"~"505 (o)(3)"~4487.00~2~"PMR 4487-2: Perform a lactation trial (milk only) in lactating women who have received therapeutic doses of zilucoplan using a validated assay to assess concentrations of zilucoplan in breast milk and the effects on the breastfed infant as applicable."~"Ongoing"~~12/31/2026 0:00:00~12/11/2025 0:00:00
383670~"CDER"~"NDA"~216951.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY NO 2 LTD ENGLAND"~"Jesduvroq (daprodustat)"~2/1/2023 0:00:00~"Original"~1~"4400-1"~"PMR"~"Pediatric Research Equity Act"~4400.00~1~"PMR 4400-1: Conduct a trial to evaluate the pharmacokinetics, pharmacodynamics and safety of Jesduvroq for the treatment of anemia associated with chronic kidney disease in children and adolescents aged 3 months to under 18 years requiring dialysis. Submit datasets at the time of the final clinical study report submission."~"Released"~"Per FDA letter dated 10/09/2025, this PMR/PMC has been released."~2/28/2030 0:00:00~3/28/2024 0:00:00
383671~"CDER"~"NDA"~216951.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY NO 2 LTD ENGLAND"~"Jesduvroq (daprodustat)"~2/1/2023 0:00:00~"Original"~1~"4400-3"~"PMR"~"505 (o)(3)"~4400.00~3~"PMR 4400-3: Conduct an observational study (at least 5 years follow up) to assess the risk for malignancy (hematological and non-hematological) in adults with dialysis-dependent chronic kidney disease with anemia treated with Jesduvroq versus an ESA comparator arm. The study should include an assessment of primary malignancies among adults with no cancer history (including assessment by type and location), and the impact of Jesduvroq on progression-free survival, and overall survival in adults with prior cancers."~"Released"~~10/31/2032 0:00:00~3/28/2024 0:00:00
383672~"CDER"~"NDA"~216951.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY NO 2 LTD ENGLAND"~"Jesduvroq (daprodustat)"~2/1/2023 0:00:00~"Original"~1~"4400-4"~"PMR"~"505 (o)(3)"~4400.00~4~"PMR 4400-4: Conduct a worldwide descriptive study to collect prospective and retrospective data on women exposed to Jesduvroq during pregnancy to assess the risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Assess infant outcomes through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Released"~~9/30/2030 0:00:00~3/28/2024 0:00:00
383673~"CDER"~"NDA"~216951.00~"GLAXOSMITHKLINE INTELLECTUAL PROPERTY NO 2 LTD ENGLAND"~"Jesduvroq (daprodustat)"~2/1/2023 0:00:00~"Original"~1~"4400-5"~"PMR"~"505 (o)(3)"~4400.00~5~"PMR 4400-5: Conduct an observational study to characterize the long-term safety (at least 5 years follow up) of Jesduvroq in adults with dialysis-dependent chronic kidney disease treated with the approved dosing regimen of Jesduvroq versus an erythropoiesis-stimulating agent (ESA) comparator arm in the United States. Specific safety outcomes of interest include thrombotic vascular events including vascular access thrombosis; hospitalization for heart failure; and serious gastrointestinal bleeds. The study population should include adults previously treated with ESAs and adults na ve to ESAs. The effect of baseline and maximum achieved hemoglobin on the specified safety outcomes should be evaluated."~"Released"~~7/31/2032 0:00:00~3/28/2024 0:00:00
383674~"CDER"~"NDA"~216956.00~"PFIZER INC"~"Velsipity (etrasimod)"~10/12/2023 0:00:00~"Original"~1~"4499-1"~"PMR"~"Pediatric Research Equity Act"~4499.00~1~"PMR 4499-1: Complete the ongoing phase 2 study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of Velsipity (etrasimod) in pediatric patients 12 to less than 18 years of age with moderately to severely active ulcerative colitis."~"Delayed"~"Original Final Report Due Date: 05/31/2026; Deferral Extension granted per FDA letter dated 04/28/2026. The study completion milestone was missed."~10/31/2029 0:00:00~12/9/2025 0:00:00
383675~"CDER"~"NDA"~216956.00~"PFIZER INC"~"Velsipity (etrasimod)"~10/12/2023 0:00:00~"Original"~1~"4499-2"~"PMR"~"Pediatric Research Equity Act"~4499.00~2~"PMR 4499-2: Conduct a one-year study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of Velsipity (etrasimod) in pediatric patients 2 to less than 18 years of age with moderately to severely active ulcerative colitis."~"Delayed"~"The final protocol milestone was missed."~8/31/2028 0:00:00~12/9/2025 0:00:00
383676~"CDER"~"NDA"~216956.00~"PFIZER INC"~"Velsipity (etrasimod)"~10/12/2023 0:00:00~"Original"~1~"4499-3"~"PMR"~"505 (o)(3)"~4499.00~3~"PMR 4499-3: Perform a lactation study (milk only) in lactating women who have received Velsipity (etrasimod), regardless of indication, to assess concentrations of etrasimod in breast milk using a validated assay and to assess the effects on the breastfed infant."~"Delayed"~"The final protocol milestone was missed. The final protocol was acknowledged on 8/25/2025."~11/30/2027 0:00:00~12/9/2025 0:00:00
383677~"CDER"~"NDA"~212028.00~"EISAI INC"~"Dayvigo (lemborexant)"~12/20/2019 0:00:00~"Original"~1~"3753-5"~"PMR"~"505 (o)(3)"~3753.00~5~"PMR 3753-5: Conduct an additional pregnancy study that uses a different design from the Pregnancy Registry (for example a case control study or a retrospective cohort study using claims or electronic medical record data with outcome validation) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to lemborexant during pregnancy compared to an unexposed control population."~"Delayed"~"The study completion milestone date was missed on 4/30/2026."~4/30/2027 0:00:00~2/13/2026 0:00:00
383678~"CDER"~"NDA"~212102.00~"UCB INC"~"Fintepla (Fenfluramine Hydrochloride)"~6/25/2020 0:00:00~"Original"~1~"3887-5"~"PMR"~"505 (o)(3)"~3887.00~5~"PMR 3887-5: A 6-month carcinogenicity study of fenfluramine in transgenic mouse"~"Submitted"~~12/31/2021 0:00:00~8/22/2025 0:00:00
383679~"CDER"~"NDA"~212102.00~"UCB INC"~"Fintepla (Fenfluramine Hydrochloride)"~6/25/2020 0:00:00~"Original"~1~"3887-6"~"PMR"~"505 (o)(3)"~3887.00~6~"PMR 3887-6: A 2-year carcinogenicity study of fenfluramine in rat."~"Submitted"~~12/31/2021 0:00:00~8/22/2025 0:00:00
383680~"CDER"~"NDA"~212102.00~"UCB INC"~"Fintepla (Fenfluramine Hydrochloride)"~6/25/2020 0:00:00~"Original"~1~"3887-7"~"PMR"~"505 (o)(3)"~3887.00~7~"PMR 3887-7: A single-arm pregnancy safety study to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to Fintepla (fenfluramine) during pregnancy. Provide a complete protocol that includes details regarding how you plan to encourage patients and providers to report pregnancy exposures, measures to ensure complete data capture regarding pregnancy outcomes and any adverse effects in offspring, and plans for comprehensive data analysis."~"Ongoing"~~8/31/2033 0:00:00~8/22/2025 0:00:00
383681~"CDER"~"NDA"~212102.00~"UCB INC"~"Fintepla (Fenfluramine Hydrochloride)"~6/25/2020 0:00:00~"Original"~1~"3887-8"~"PMR"~"505 (o)(3)"~3887.00~8~"PMR 3887-8: A prospective observational registry study in epilepsy patients taking Fintepla using data from the REMS Registry and additional data beyond what is collected in the REMS registry. The primary objectives are to characterize the risks of the development of symptomatic or asymptomatic valvular heart disease (VHD) and/or pulmonary arterial hypertension (PAH). This includes recruiting an adequate number of patients to assess the incidence of VHD and PAH, to identify risk factors for VHD and PAH, and to evaluate the impact of duration, dose-exposure, and cumulative exposure on the development of VHD and PAH. Evaluation should include the assessment of echocardiographic data; patients in the study should be evaluated with echocardiograms at baseline and every six months for five years, or until the last echocardiogram following interruption of Fintepla treatment.
"~"Ongoing"~~8/31/2032 0:00:00~8/22/2025 0:00:00
383682~"CDER"~"NDA"~212122.00~"ASTRAZENECA AB"~"BREZTRI AEROSPHERE (budesonide, glycopyrrolate and formoterol fumarate)"~7/23/2020 0:00:00~"Supplement"~9~"4983-1"~"PMR"~"Pediatric Research Equity Act"~4983.00~1~"PMR 4983-1: Conduct a randomized, double-blind, placebo-controlled, 3-way crossover, 3-week treatment period, dose-ranging study of at least two doses of glycopyrrolate in children 4 to 11 years of age with asthma receiving background therapy of budesonide/formoterol fumarate inhalation aerosol (BFF Aerosphere MDI)."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~3/31/2030 0:00:00~
383683~"CDER"~"NDA"~212122.00~"ASTRAZENECA AB"~"BREZTRI AEROSPHERE (budesonide, glycopyrrolate and formoterol fumarate)"~7/23/2020 0:00:00~"Supplement"~9~"4983-2"~"PMR"~"Pediatric Research Equity Act"~4983.00~2~"PMR 4983-2: Conduct an open-label, single dose, single-period pharmacokinetic study of budesonide/ glycopyrrolate/ formoterol fumarate inhalation aerosol (BGF Aerosphere MDI) in children 4 to 11 years of age with asthma."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2033 0:00:00~
383684~"CDER"~"NDA"~212122.00~"ASTRAZENECA AB"~"BREZTRI AEROSPHERE (budesonide, glycopyrrolate and formoterol fumarate)"~7/23/2020 0:00:00~"Supplement"~9~"4983-3"~"PMR"~"Pediatric Research Equity Act"~4983.00~3~"PMR 4983-3: Conduct a 52-week, randomized, double-blind, parallel-group, active-controlled, efficacy and safety study of budesonide/ glycopyrrolate/formoterol fumarate inhalation aerosol (BGF Aerosphere MDI) in children 4 to 11 years of age with asthma."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2035 0:00:00~
383685~"CDER"~"NDA"~212154.00~"NIPPON SHINYAKU CO LTD"~"Viltepso (viltolarsen)"~8/12/2020 0:00:00~"Original"~1~"3895-1"~"PMR"~"Accelerated Approval"~3895.00~1~"PMR 3895-1: In order to verify the clinical benefit of viltolarsen, complete Study NS065/NCNP-01-301,  A Phase 3 Randomized, Double-blind, Placebocontrolled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy.  The study will assess treatment with viltolarsen 80 mg/kg over 48 weeks. The primary endpoint will be Time to Stand."~"Submitted"~"The Final Report was submitted to FDA 12/23/24."~12/31/2024 0:00:00~10/8/2025 0:00:00
383686~"CDER"~"NDA"~212154.00~"NIPPON SHINYAKU CO LTD"~"Viltepso (viltolarsen)"~8/12/2020 0:00:00~"Original"~1~"3895-4"~"PMR"~"505 (o)(3)"~3895.00~4~"PMR 3895-4: Amend the Phase 3 trial protocol (Study # NS-065/NCNP-01-301) identified in PMR 3895-1 to collect additional pharmacokinetic measurements and high quality 12-lead ECG recordings (see ICH E14 and Q&A #1) around Tmax (e.g., at 1 hour and 3 hours from the start of infusion) following the first dose in all patients. Submit data and analysis from your Phase 1/2, and Phase 2 studies, and from the amended Phase 3 study, to characterize the effects of viltolarsen on the QTc interval as per ICH E14 Q&A (R3) 6.1. If these data do not support a TQT study waiver, you will need to evaluate the effect of viltolarsen on the QTc interval in a dedicated study, as per the ICH E14 guideline."~"Submitted"~~12/31/2024 0:00:00~10/8/2025 0:00:00
383687~"CDER"~"NDA"~212154.00~"NIPPON SHINYAKU CO LTD"~"Viltepso (viltolarsen)"~8/12/2020 0:00:00~"Original"~1~"3895-6"~"PMR"~"505 (o)(3)"~3895.00~6~"PMR 3895-6: Use the anti-viltolarsen antibody assay with improved sensitivity provided for in PMR 3895-5 to re-evaluate samples from the Phase 3 confirmatory study."~"Fulfilled"~~12/31/2024 0:00:00~10/8/2025 0:00:00
383688~"CDER"~"NDA"~212154.00~"NIPPON SHINYAKU CO LTD"~"Viltepso (viltolarsen)"~8/12/2020 0:00:00~"Original"~1~"3895-8"~"PMR"~"505 (o)(3)"~3895.00~8~"PMR 3895-8: Use the anti-dystrophin antibody assay with improved sensitivity provided for in PMR 3895-7 to re-evaluate samples from the Phase 3 confirmatory study."~"Fulfilled"~~12/31/2024 0:00:00~10/8/2025 0:00:00
383689~"CDER"~"NDA"~212157.00~"SCILEX PHARMACEUTICALS INC"~"Elyxyb (celecoxib)"~5/5/2020 0:00:00~"Original"~1~"3838-1"~"PMR"~"Pediatric Research Equity Act"~3838.00~1~"PMR 3838-1: A randomized, double-blind, placebo-controlled, efficacy and safety study to evaluate Elyxyb oral solution compared to placebo in the treatment of acute migraine in pediatric patients ages 6 to less than 18 years. The study should include an initial blinded placebo run-in period to identify placebo non-responders for enrollment into the efficacy portion of the trial. The efficacy study must be designed to show superiority of Elyxyb over placebo and should be submitted as a Special Protocol Assessment (SPA)."~"Released"~"Per FDA letter dated 02/26/2026, this PMR/PMC has been released."~2/28/2028 0:00:00~6/30/2026 0:00:00
383690~"CDER"~"NDA"~213498.00~"VANDA PHARMACEUTICALS INC"~"Ponvory (ponesimod)"~3/18/2021 0:00:00~"Original"~1~"4024-5"~"PMR"~"Pediatric Research Equity Act"~4024.00~5~"PMR 4024-5: Conduct a two-part study of Ponvory (ponesimod) in pediatric patients with relapsing forms of multiple sclerosis (RMS) at least 10 years and less than 18 years of age. Part A is an open-label study of the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Ponvory (ponesimod) in pediatric patients weighing 40 kg or less. The objective of Part A is to determine titration and maintenance doses of Ponvory (ponesimod) that will result in PK and PD effects that are comparable to those of the 14-day titration administered to adult patients. Part B is a randomized, blinded, non-inferiority trial with an appropriate active comparator."~"Delayed"~"Per FDA letter dated 04/27/2026, the applicant s request for release was denied."~8/31/2028 0:00:00~5/13/2026 0:00:00
383691~"CDER"~"NDA"~213535.00~"GENENTECH INC"~"Evrysdi (risdiplam)"~8/7/2020 0:00:00~"Original"~1~"3886-2"~"PMR"~"505 (o)(3)"~3886.00~2~"PMR 3886-2: Establish a single-arm pregnancy safety study to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes for exposed pregnancies, including women exposed to Evrysdi (risdiplam) during pregnancy or one month prior to the start of pregnancy. Provide a complete protocol that includes details regarding how you plan to encourage patients and providers to report pregnancy exposures, measures to ensure complete data capture regarding pregnancy outcomes and any adverse effects in offspring, and plans for comprehensive data analysis."~"Ongoing"~~10/31/2032 0:00:00~10/1/2025 0:00:00
383692~"CDER"~"NDA"~213674.00~"ASTELLAS PHARMA US INC"~"Xtandi (Enzalutamide)"~8/4/2020 0:00:00~"Supplement"~10~"4536-1"~"PMC"~"506B PMC"~4536.00~1~"PMC 4536-1: Complete clinical trial, EMBARK,  A Phase 3, Randomized, Efficacy and Safety Study of Enzalutamide Plus Leuprolide, Enzalutamide Monotherapy, and Placebo Plus Leuprolide in Men With High-Risk Nonmetastatic Prostate Cancer Progressing After Definitive Therapy,  to provide the final overall survival analyses."~"Ongoing"~~6/30/2027 0:00:00~10/29/2025 0:00:00
383693~"CDER"~"NDA"~213687.00~"ULTRAGENYX PHARMACEUTICAL INC"~"Dojolvi (triheptanoin) "~6/30/2020 0:00:00~"Original"~1~"3858-1"~"PMR"~"505 (o)(3)"~3858.00~1~"PMR 3858-1: Conduct a worldwide, single-arm, pregnancy safety study in women exposed to Dojolvi during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. Results will be analyzed and reported descriptively."~"Ongoing"~~1/31/2033 0:00:00~8/28/2025 0:00:00
383694~"CDER"~"NDA"~213702.00~"JAZZ PHARMACEUTICALS IRELAND LTD"~"Zepzelca (lurbinectedin)"~6/15/2020 0:00:00~"Original"~1~"3831-7"~"PMR"~"Accelerated Approval"~3831.00~7~"PMR 3831-7: Conduct a clinical trial investigating lurbinectedin in patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy to verify the clinical benefit of lurbinectedin for the treatment of metastatic SCLC. The primary endpoint should be overall survival. Tumor-related secondary endpoints, including progression free survival, should be determined by an Independent Review Committee."~"Delayed"~"As per the 180-day AA PMR Progress Report submitted on 2/20/2025, the confirmatory trial (Study PM1183-C-008-21) is behind the original schedule because of challenges with enrollment and drug shortages. The trial is now fully enrolled. This report was found to be complete."~12/31/2025 0:00:00~8/8/2025 0:00:00
383695~"CDER"~"NDA"~213702.00~"JAZZ PHARMACEUTICALS IRELAND LTD"~"Zepzelca (lurbinectedin)"~6/15/2020 0:00:00~"Original"~1~"3831-8"~"PMR"~"Accelerated Approval"~3831.00~8~"PMR 3831-8: Conduct a clinical trial investigating lurbinectedin in patients with small cell lung cancer (SCLC) receiving first-line maintenance treatment to verify the clinical benefit of lurbinectedin for the treatment of metastatic SCLC. The primary endpoints should be overall survival and progression-free survival as determined by an Independent Review Committee."~"Fulfilled"~"Per FDA letter dated 10/02/2025, this PMR/PMC has been fulfilled."~3/31/2027 0:00:00~8/8/2025 0:00:00
383696~"CDER"~"NDA"~213702.00~"JAZZ PHARMACEUTICALS IRELAND LTD"~"Zepzelca (lurbinectedin)"~6/15/2020 0:00:00~"Supplement"~11~"4911-1"~"PMC"~"506B PMC"~4911.00~1~"PMC 4911-1: Conduct an integrated analysis of data from clinical trials, observational studies (e.g. real world evidence), post-marketing reports and other sources to further characterize the efficacy and safety of lurbinectedin in a pooled population that includes a sufficient representation of racial and ethnic populations to better reflect the U.S. population with extensive-stage small cell lung cancer and allow for interpretation of data in these populations."~"Pending"~~9/30/2028 0:00:00~8/8/2025 0:00:00
383697~"CDER"~"NDA"~213716.00~"AURINIA PHARMACEUTICALS INC"~"Lupkynis (Voclosporin)"~1/22/2021 0:00:00~"Original"~1~"4007-6"~"PMR"~"Pediatric Research Equity Act"~4007.00~6~"PMR 4007-6: An adaptive design, double-blind, placebo-controlled, dose
escalation, PK, efficacy and safety study of voclosporin in lupus nephritis in addition to standard therapy, in pediatric patients from 5 to = 17 years with active lupus nephritis."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2028 0:00:00~3/20/2026 0:00:00
383698~"CDER"~"NDA"~213716.00~"AURINIA PHARMACEUTICALS INC"~"Lupkynis (Voclosporin)"~1/22/2021 0:00:00~"Original"~1~"4007-7"~"PMR"~"Pediatric Research Equity Act"~4007.00~7~"PMR 4007-7: A 12-month, prospective, multi-center, long-term, open-label efficacy and safety extension study of voclosporin in addition to background standard of care with mycophenolate mofetil and oralsteroids in pediatric patients from 5 to =17 years of age with lupus nephritis."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2029 0:00:00~3/20/2026 0:00:00
383699~"CDER"~"NDA"~213721.00~"RIGEL PHARMACEUTICALS INC"~"GAVRETO (pralsetinib)"~9/4/2020 0:00:00~"Original"~1~"3959-2"~"PMR"~"Accelerated Approval"~3959.00~2~"PMR 3959-2: Submit the final report of integrated studies and datasets, to verify and further characterize the clinical benefit of pralsetinib for the treatment of patients with RET fusion-positive thyroid cancer who have received radioactive iodine (if appropriate for their tumor histology) to provide a more precise estimation of the BICR-assessed overall response rate and duration of response in at least 50 patients in a variety of histologies after all responding patients have been followed for 12 months following onset of response or until disease progression, whichever comes first."~"Delayed"~"The Final Report Submission milestone was missed due to awaiting data from the study, which was expected in September 2025."~6/30/2025 0:00:00~10/30/2025 0:00:00
383700~"CDER"~"NDA"~213721.00~"RIGEL PHARMACEUTICALS INC"~"GAVRETO (pralsetinib)"~9/4/2020 0:00:00~"Original"~1~"3916-4"~"PMR"~"505 (o)(3)"~3916.00~4~"PMR 3916-4: Conduct a comprehensive analysis evaluating and characterizing the incidence, clinical presentation, management, and outcome of the potential serious risk of pralsetinib associated gastrointestinal perforations and fistulas. Submit an integrated final report containing data from patientlevel and pooled analyses of on-going and completed clinical trials, postmarketing reports and/or literature reports and a comprehensive pharmacovigilance assessment for this potential serious risk. The results from this report may inform product labelling."~"Ongoing"~~8/31/2026 0:00:00~10/30/2025 0:00:00
383701~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Supplement"~29~"4975-2"~"PMR"~"Pediatric Research Equity Act"~4975.00~2~"PMR 4975-2: Conduct a randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy after 52 weeks of semaglutide 7.2 mg for the treatment of obesity in pediatric patients ages 6 to less than 12 who still require weight loss after treatment with semaglutide 2.4 mg.  Compare the long-term (at least 2 years) safety and tolerability of semaglutide versus placebo for the treatment of obesity in both children and adolescents (ages 6 to less than 18 years). The trial may not be initiated until results from the semaglutide 7.2 mg adolescent trial, and the STEP YOUNG (NN9536-4512) trial in pediatric patients with obesity ages 6 to <12 years treated with semaglutide 2.4 mg, have been submitted to and reviewed by the Agency."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2036 0:00:00~11/17/2025 0:00:00
383702~"CDER"~"NDA"~215256.00~"NOVO NORDISK INC"~"Semaglutide"~6/4/2021 0:00:00~"Supplement"~29~"4975-3"~"PMR"~"505 (o)(3)"~4975.00~3~"PMR 4975-3: Conduct the randomized, double-blind, active-controlled trial NN9838-7910 (comparing subcutaneous semaglutide 7.2 mg to 2.4 mg, both in combination with cagrilintide) with dysesthesia (altered skin sensation) as an adverse event of special interest. Use dedicated case report forms for data collection and provide narrative summaries of dysesthesia adverse events in the clinical study report for this trial."~"Pending"~~7/31/2029 0:00:00~11/17/2025 0:00:00
383703~"CDER"~"NDA"~215272.00~"ORGANON LLC"~"VTAMA (Tapinarof)"~5/23/2022 0:00:00~"Original"~1~"4283-1"~"PMR"~"Pediatric Research Equity Act"~4283.00~1~"PMR 4283-1: Conduct an open-label, long-term safety and pharmacokinetic (PK) study in at least 100 pediatric subjects with plaque psoriasis ages 2 to <18 years. Subjects should be exposed to tapinarof cream, 1%, for a minimum of 52 weeks. For PK evaluation under maximal use conditions, include at least 16 evaluable subjects ages 12 to <18 years with involved Body Surface Area (BSA) = 10%, and at least 8 subjects ages 2 to <12 years with involved BSA = 3% exposed to tapinarof cream, 1%, for a minimum of 12 weeks, with the option of continuing for a total of 52 weeks."~"Released"~"Per FDA letter dated 03/04/2026, this PMR/PMC has been released."~3/31/2027 0:00:00~7/18/2025 0:00:00
383704~"CDER"~"NDA"~215272.00~"ORGANON LLC"~"VTAMA (Tapinarof)"~5/23/2022 0:00:00~"Supplement"~2~"4772-1"~"PMR"~"Pediatric Research Equity Act"~4772.00~1~"PMR 4772-1: Conduct an efficacy, safety and pharmacokinetic (PK) study in pediatric subjects 3 to <24 months with atopic dermatitis."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed"~12/31/2028 0:00:00~7/18/2025 0:00:00
383705~"CDER"~"NDA"~215309.00~"INCYTE CORP"~"Opzelura (ruxolitinib)"~9/21/2021 0:00:00~"Original"~1~"4147-1"~"PMR"~"Pediatric Research Equity Act"~4147.00~1~"PMR 4147-1: Conduct a randomized, double-blind, 8-week trial of ruxolitinib 1.5%, ruxolitinib 0.75%, and vehicle, followed by a 44-week long-term safety extension where vehicle subjects are randomized to either ruxolitinib 1.5% or ruxolitinib 0.75%. The trial should enroll 250 subjects ages = 2 to < 12 years with atopic dermatitis of at least 3 months duration, a baseline Investigator s Global Assessment (IGA) score of 2 to 3, and % body surface area (BSA) involvement (excluding scalp) of 3% to 20% (Study INCB 18424-305)."~"Fulfilled"~"Per FDA letter dated 09/18/2025, this PMR/PMC has been fulfilled."~9/30/2024 0:00:00~11/20/2025 0:00:00
383706~"CDER"~"NDA"~215309.00~"INCYTE CORP"~"Opzelura (ruxolitinib)"~9/21/2021 0:00:00~"Original"~1~"4147-2"~"PMR"~"Pediatric Research Equity Act"~4147.00~2~"PMR 4147-2: Conduct a maximal use pharmacokinetic (PK) study in pediatric subjects with atopic dermatitis ages = 2 years to < 12 and target at least 16 completers."~"Fulfilled"~"Per FDA letter dated 09/18/2025, this PMR/PMC has been fulfilled."~9/30/2024 0:00:00~11/20/2025 0:00:00
383707~"CDER"~"NDA"~215309.00~"INCYTE CORP"~"Opzelura (ruxolitinib)"~9/21/2021 0:00:00~"Original"~1~"4147-3"~"PMR"~"Pediatric Research Equity Act"~4147.00~3~"PMR 4147-3: Conduct an open-label safety study in 100 subjects ages = 3 months to < 24 months with atopic dermatitis with ruxolitinib cream applied twice daily (BID) for 4 weeks with a 48-week extension treatment period and assess PK under maximal use conditions in a subset of at least 16 subjects."~"Pending"~"The study has not begun but does not meet the criterion for
delayed."~1/31/2030 0:00:00~11/20/2025 0:00:00
383708~"CDER"~"NDA"~215309.00~"INCYTE CORP"~"Opzelura (ruxolitinib)"~9/21/2021 0:00:00~"Original"~1~"4147-4"~"PMR"~"505 (o)(3)"~4147.00~4~"PMR 4147-4: Conduct a one-year, open-label safety study in subjects with atopic dermatitis ages = 12 years to < 18 years."~"Submitted"~~6/30/2025 0:00:00~11/20/2025 0:00:00
383709~"CDER"~"NDA"~215309.00~"INCYTE CORP"~"Opzelura (ruxolitinib)"~9/21/2021 0:00:00~"Original"~1~"4147-5"~"PMR"~"505 (o)(3)"~4147.00~5~"PMR 4147-5: Conduct a Pregnancy Exposure Registry, a prospective, registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes in the female population with atopic dermatitis exposed to ruxolitinib cream during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life. For more information, see the May 2019 FDA draft Guidance for Industry Postapproval Pregnancy Safety Studies."~"Delayed"~"The Final Protocol Due Date was missed due to continued discussions with the Agency. The Final Protocol was acknowledged in a letter dated 04/26/2023. Enrollment: 2/479 per the 11/20/2025 submitted annual report."~8/31/2033 0:00:00~11/20/2025 0:00:00
383710~"CDER"~"NDA"~215309.00~"INCYTE CORP"~"Opzelura (ruxolitinib)"~9/21/2021 0:00:00~"Original"~1~"4147-6"~"PMR"~"505 (o)(3)"~4147.00~6~"PMR 4147-6: Conduct an additional pregnancy study that uses a different design from the Pregnancy Registry (for example a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in the female population with atopic dermatitis exposed to ruxolitinib cream during pregnancy compared to an unexposed control population."~"Delayed"~"The Final Protocol Submission date was missed due to continued discussions with the Agency. The Final Protocol was acknowledged in a letter dated 03/27/2023. Enrollment: 7/273 per the 11/20/2025 submitted annual report."~8/31/2028 0:00:00~11/20/2025 0:00:00
383711~"CDER"~"NDA"~215309.00~"INCYTE CORP"~"Opzelura (ruxolitinib)"~9/21/2021 0:00:00~"Supplement"~1~"4304-1"~"PMR"~"Pediatric Research Equity Act"~4304.00~1~"PMR 4304-1: Conduct a randomized, double-blind, vehicle-controlled 24-week trial of ruxolitinib 1.5% cream followed by a 28-week long-term safety extension period. The trial should enroll 150 pediatric subjects = 2 years to < 12 years of age with nonsegmental vitiligo covering up to 10% body surface area (BSA) and minimum depigmentation involvement of at least 0.5% BSA on the face and at least 3% BSA on non-facial areas."~"Delayed"~"The Final Protocol Submission date was missed due to continued discussions with the Agency. The Final Protocol was acknowledged in a letter dated 03/13/2024. Enrollment: 35/180 per the 11/20/2025 submitted annual report."~3/31/2027 0:00:00~11/20/2025 0:00:00
383712~"CDER"~"NDA"~216956.00~"PFIZER INC"~"Velsipity (etrasimod)"~10/12/2023 0:00:00~"Original"~1~"4499-4"~"PMR"~"505 (o)(3)"~4499.00~4~"PMR 4499-4: Collect data from a prospective pregnancy exposure registry, preferably a disease-based multiproduct pregnancy registry, using a cohort analysis that compares the maternal, fetal, and infant outcomes of women exposed to etrasimod regardless of indication during pregnancy with unexposed comparator population(s) in a timely manner. Align the study protocol with protocol(s) outside the US to reach the target sample size. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortion, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes described in the protocol will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The final protocol milestone was missed."~9/30/2033 0:00:00~12/9/2025 0:00:00
383713~"CDER"~"NDA"~216956.00~"PFIZER INC"~"Velsipity (etrasimod)"~10/12/2023 0:00:00~"Original"~1~"4499-5"~"PMR"~"505 (o)(3)"~4499.00~5~"PMR 4499-5: Conduct an additional pregnancy study that uses a different design from the prospective pregnancy registry established to fulfill postmarketing requirement 4499-4 (for example a retrospective cohort study using claims or electronic medical record data or a case-control study) to assess major congenital malformations, spontaneous abortions, stillbirths, small for gestational age births, and preterm births in women exposed to etrasimod regardless of indication during pregnancy compared to an unexposed control population."~"Delayed"~"The final protocol milestone was missed. The final protocol was
acknowledged on 11/5/2025."~3/31/2032 0:00:00~12/9/2025 0:00:00
383714~"CDER"~"NDA"~216964.00~"MERCK SHARP AND DOHME LLC"~"Idvynso (doravirine/islatravir) Tablet"~4/20/2026 0:00:00~"Original"~1~"4978-1"~"PMR"~"Pediatric Research Equity Act"~4978.00~1~"PMR 4978-1: Conduct a study to evaluate the pharmacokinetics, safety and antiviral activity of doravirine/islatravir in pediatric participants with HIV-1 infection who are less than 18 years of age and weighing at least 35kg. Study participants must be evaluated for a minimum of 24 weeks to assess safety and durability of antiviral response."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~9/30/2030 0:00:00~
383715~"CDER"~"NDA"~216964.00~"MERCK SHARP AND DOHME LLC"~"Idvynso (doravirine/islatravir) Tablet"~4/20/2026 0:00:00~"Original"~1~"4978-2"~"PMR"~"Pediatric Research Equity Act"~4978.00~2~"PMR 4978-2: Conduct a study to evaluate the pharmacokinetics, safety and antiviral activity of doravirine/islatravir in pediatric participants with HIV-1 infection who are at least 28 days of age and weighing less than 35kg. Study participants must be evaluated for a minimum of 24 weeks to assess safety and durability of response."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~8/31/2039 0:00:00~
383716~"CDER"~"NDA"~216964.00~"MERCK SHARP AND DOHME LLC"~"Idvynso (doravirine/islatravir) Tablet"~4/20/2026 0:00:00~"Original"~1~"4978-3"~"PMR"~"505 (o)(3)"~4978.00~3~"PMR 4978-3: Conduct a phenotypic study to determine the impact of the following reverse transcriptase amino acid substitutions on doravirine susceptibility and on islatravir susceptibility against HIV-1 in cell culture: A98S, K101A, V179I, M184T, Y188W, K101A+V106M+Y188W+F227L, K101E+K103N, V106I+Y181C, and V106M+Y188W +F227L."~"Pending"~~7/31/2027 0:00:00~
383717~"CDER"~"NDA"~216974.00~"ENTASIS THERAPEUTICS INC"~"Xacduro (sulbactam for injection; durlobactam for injection) co-packaged"~5/23/2023 0:00:00~"Original"~1~"4452-1"~"PMR"~"Pediatric Research Equity Act"~4452.00~1~"PMR 4452-1: Conduct a multi-dose study to assess the pharmacokinetics, safety and tolerability of sulbactam-durlobactam in children from birth to less than 18 years who are receiving systemic antibiotic therapy for suspected or confirmed infection."~"Ongoing"~"Final protocol was submitted 12-20-2023"~11/30/2028 0:00:00~7/17/2025 0:00:00
383718~"CDER"~"NDA"~216974.00~"ENTASIS THERAPEUTICS INC"~"Xacduro (sulbactam for injection; durlobactam for injection) co-packaged"~5/23/2023 0:00:00~"Original"~1~"4452-2"~"PMR"~"505 (o)(3)"~4452.00~2~"PMR 4452-2: Conduct a single-arm, open-label, prospective, observational study to assess the safety of sulbactam-durlobactam, including the risk of hypersensitivity reactions (including anaphylaxis) in patients with Acinetobacter baumannii-calcoaceticus complex infection."~"Ongoing"~~8/31/2029 0:00:00~7/17/2025 0:00:00
383719~"CDER"~"NDA"~216974.00~"ENTASIS THERAPEUTICS INC"~"Xacduro (sulbactam for injection; durlobactam for injection) co-packaged"~5/23/2023 0:00:00~"Original"~1~"4452-4"~"PMR"~"505 (o)(3)"~4452.00~4~"PMR 4452-4: Conduct a US surveillance study for a five-year period after the introduction of sulbactam-durlobactam to the market to monitor changes in susceptibility of Acinetobacter baumannii-calcoaceticus complex organisms to sulbactam-durlobactam."~"Ongoing"~~2/28/2029 0:00:00~7/17/2025 0:00:00
383720~"CDER"~"NDA"~216974.00~"ENTASIS THERAPEUTICS INC"~"Xacduro (sulbactam for injection; durlobactam for injection) co-packaged"~5/23/2023 0:00:00~"Original"~1~"4452-5"~"PMR"~"505 (o)(3)"~4452.00~5~"PMR 4452-5: Conduct studies for durlobactam drug substance and drug product, as identified in the final protocol, to further characterize the safety of their impurity profiles and establish drug substance and drug product impurity controls to ensure that each impurity limit is in accordance with ICH Q3A, Q3B, and M7, as applicable."~"Ongoing"~~12/31/2025 0:00:00~7/17/2025 0:00:00
383721~"CDER"~"NDA"~216986.00~"GUERBET"~"Elucirem (gadopiclenol)"~9/21/2022 0:00:00~"Original"~1~"4341-1"~"PMR"~"Pediatric Research Equity Act"~4341.00~1~"PMR 4341-1: Conduct a study of approximately 40 neonates and infants aged < 2 years that will receive a single dose of 0.05 mmol/kg gadopiclenol intravenously for the evaluation of the plasma pharmacokinetics profile of gadopiclenol (POP-PK analysis). Safety and imaging data will be collected as secondary endpoints."~"Submitted"~"The final report was submitted to FDA on 04/22/2025."~4/30/2025 0:00:00~11/20/2025 0:00:00
383722~"CDER"~"NDA"~216986.00~"GUERBET"~"Elucirem (gadopiclenol)"~9/21/2022 0:00:00~"Original"~1~"4341-2"~"PMR"~"505 (o)(3)"~4341.00~2~"PMR 4341-2: Conduct a developmental and perinatal/postnatal reproduction study of gadopiclenol by intravenous (bolus) injection in mice, including a postnatal behavioral/functional evaluation (Sponsor Reference No. GDX33-073). The study will examine the safety of gadopiclenol following perinatal exposure through repeated dosing in pregnant dams. The study will provide safety data assessing behavioral, neurological, and histopathology findings. The study will examine the pharmacokinetics of gadopiclenol including gadolinium retention in the brain and other organs/tissues."~"Fulfilled"~~5/31/2025 0:00:00~11/20/2025 0:00:00
383723~"CDER"~"NDA"~216986.00~"GUERBET"~"Elucirem (gadopiclenol)"~9/21/2022 0:00:00~"Original"~1~"4341-3"~"PMR"~"505 (o)(3)"~4341.00~3~"PMR 4341-3: Conduct an intravenous (bolus) injection juvenile toxicity study of gadopiclenol in mice (Sponsor Reference No. GDX-33-072). The study will examine the safety of gadopiclenol in juvenile animals, following repeated administration. The study will provide safety data assessing behavioral, neurological, and histopathology findings. The study will also examine the pharmacokinetics of gadopiclenol including gadolinium retention in the brain and other organs/tissues."~"Fulfilled"~~5/31/2025 0:00:00~11/20/2025 0:00:00
383724~"CDER"~"NDA"~212157.00~"SCILEX PHARMACEUTICALS INC"~"Elyxyb (celecoxib)"~5/5/2020 0:00:00~"Original"~1~"3838-2"~"PMR"~"Pediatric Research Equity Act"~3838.00~2~"PMR 3838-2: A randomized, double-blind, placebo-controlled, efficacy and safety study to evaluate Elyxyb oral solution compared to placebo in the treatment of acute migraine in pediatric patients ages 6 to less than 18 years. The study should include an initial blinded placebo run-in period to identify placebo non-responders for enrollment into the efficacy portion of the trial. The efficacy study must be designed to show superiority of Elyxyb over placebo."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2030 0:00:00~6/30/2026 0:00:00
383725~"CDER"~"NDA"~212161.00~"FOLDRX PHARMACEUTICALS LLC A WHOLLY OWNED SUB OF PFIZER INC"~"VYNDAMAX (tafamidis)"~5/3/2019 0:00:00~"Original"~1~"3576-1"~"PMR"~"505 (o)(3)"~3576.00~1~"PMR 3576-1: Establish a worldwide pregnancy surveillance study in women exposed to tafamidis meglumine and tafamidis during pregnancy to assess the risks of pregnancy complications and adverse effects on the developing fetus and neonate. The study will collect information for a minimum of 10 years. Provide a complete protocol with details on how pregnancy exposures and outcomes will be captured (e.g., telephone contact number and/or website that will be provided in the product s prescribing information), and measures to ensure complete data capture on pregnancy outcomes and any adverse effects in the offspring 
"~"Ongoing"~~12/31/2030 0:00:00~6/30/2026 0:00:00
383726~"CDER"~"NDA"~212194.00~"ALNYLAM PHARMACEUTICALS INC"~"GIVLAARI (givosiran)"~11/20/2019 0:00:00~"Original"~1~"3743-1"~"PMC"~"506B PMC"~3743.00~1~"PMC 3743-1: Conduct a controlled trial in pediatric patients to evaluate the dose, clinical outcomes, and safety of givosiran for the treatment of pediatric patients age greater than or equal to 12 years to less than 17 years with acute hepatic porphyria. Appropriate sampling must be incorporated to explore exposure-response relationships for measures of pharmacodynamic biomarkers, safety, and efficacy. The final protocol should be agreed upon with the Agency."~"Ongoing"~~2/28/2027 0:00:00~1/14/2026 0:00:00
383727~"CDER"~"NDA"~212271.00~"MEITHEAL PHARMACEUTICALS INC"~"Contepo (fosfomycin) for injection"~10/22/2025 0:00:00~"Original"~1~"4908-1"~"PMR"~"Pediatric Research Equity Act"~4908.00~1~"PMR 4908-1: Conduct an open-label, single dose trial to evaluate pharmacokinetics, safety and tolerability of Contepo in children from birth (full term) to less than 12 years of age with suspected or confirmed gram-negative bacterial infection, or for peri-operative prophylaxis, and receiving other systemic antibiotic standard of care therapy."~"Ongoing"~"The trial is underway."~4/30/2027 0:00:00~
383728~"CDER"~"NDA"~212271.00~"MEITHEAL PHARMACEUTICALS INC"~"Contepo (fosfomycin) for injection"~10/22/2025 0:00:00~"Original"~1~"4908-2"~"PMR"~"Pediatric Research Equity Act"~4908.00~2~"PMR 4908-2: Conduct an open-label, single arm study to evaluate the pharmacokinetics and safety of Contepo in children from birth (full term 37 weeks) to less than 18 years of age with complicated urinary tract infections (cUTI)."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~9/30/2030 0:00:00~
383729~"CDER"~"NDA"~212271.00~"MEITHEAL PHARMACEUTICALS INC"~"Contepo (fosfomycin) for injection"~10/22/2025 0:00:00~"Original"~1~"4908-3"~"PMR"~"505 (o)(3)"~4908.00~3~"PMR 4908-3: Conduct a clinical lactation study (milk-only) in lactating women administered Contepo to determine the concentrations of fosfomycin and its major metabolites in human milk using validated bioanalytical methods."~"Pending"~~5/31/2029 0:00:00~
383730~"CDER"~"NDA"~212271.00~"MEITHEAL PHARMACEUTICALS INC"~"Contepo (fosfomycin) for injection"~10/22/2025 0:00:00~"Original"~1~"4908-4"~"PMR"~"505 (o)(3)"~4908.00~4~"PMR 4908-4: Conduct a U.S. surveillance study over a five-year period after the introduction of Contepo (fosfomycin) for injection to the market to determine if resistance or decreased susceptibility to fosfomycin is occurring in the target population of bacteria that are in the approved Contepo label."~"Pending"~~11/30/2031 0:00:00~
383731~"CDER"~"NDA"~212273.00~"VERTEX PHARMACEUTICALS INC"~"Trikafta (elexacaftor/tezacaftor/ivacaftor)"~10/21/2019 0:00:00~"Supplement"~2~"3987-1"~"PMC"~"506B PMC"~3987.00~1~"PMC 3987-1: Conduct a 3-year, single arm, observational study to further understand the clinical response to elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) and tezacaftor/ivacaftor (TEZ/IVA) in cystic fibrosis (CF) patients. The study  will include all patients registered in the U.S. Cystic Fibrosis Foundation Patient Registry who do not have an F508del allele but do have a CFTR mutation that has been characterized in vitro as responsive to ELX/TEZ/IVA and/or TEZ/IVA, but not to IVA alone, and who initiate ELX/TEZ/IVA and/or TEZ/IVA therapy following the date of approval of this supplement.  Patients will be followed for at least 3 years on ELX/TEZ/IVA and/or TEZ/IVA after ELX/TEZ/IVA and/or TEZ/IVA initiation.  The key  outcomes of interest will include lung function measurements (FEV1),nutritional parameters (e.g., BMI), pulmonary exacerbations, hospitalizations, select CF complications (e.g., symptomatic sinus disease, CFRD, distal intestinal obstruction), and the presence of select pulmonary microorganisms (e.g., P aeruginosa)."~"Fulfilled"~~12/31/2025 0:00:00~12/18/2025 0:00:00
383732~"CDER"~"NDA"~212295.00~"ACACIA PHARMA LTD"~"BYFAVO (Remimazolam)"~7/2/2020 0:00:00~"Original"~1~"3889-1"~"PMR"~"Pediatric Research Equity Act"~3889.00~1~"PMR 3889-1: Conduct a multicenter, sequential age-group study to evaluate the pharmacokinetics, safety, and efficacy of BYFAVO administered for procedural sedation in pediatric patients three to less than 17 years of age."~"Delayed"~"The study completion and final report submission milestone were missed. A Deferral Extension requested 5/29/2025, denied per FDA letter dated 7/11/2025."~1/31/2025 0:00:00~12/5/2025 0:00:00
383733~"CDER"~"NDA"~212295.00~"ACACIA PHARMA LTD"~"BYFAVO (Remimazolam)"~7/2/2020 0:00:00~"Original"~1~"3889-2"~"PMR"~"Pediatric Research Equity Act"~3889.00~2~"PMR 3889-2: Conduct a multicenter study to evaluate the pharmacokinetics, safety, and efficacy of BYFAVO administered for procedural sedation in pediatric patients from birth to less than three years of age."~"Delayed"~"The draft and final protocol milestones were missed, because the completion of 2 other studies is required before this study can be initiated."~1/31/2029 0:00:00~12/5/2025 0:00:00
383734~"CDER"~"NDA"~212295.00~"ACACIA PHARMA LTD"~"BYFAVO (Remimazolam)"~7/2/2020 0:00:00~"Original"~1~"3889-3"~"PMR"~"Pediatric Research Equity Act"~3889.00~3~"PMR 3889-3: Conduct a juvenile animal toxicology study in a rodent model to characterize the effects of remimazolam on the developing central nervous system to support clinical studies in pediatric patients under 3 years of age."~"Submitted"~"The final report was submitted to FDA on 09/23/2025"~12/31/2022 0:00:00~12/5/2025 0:00:00
383735~"CDER"~"NDA"~212295.00~"ACACIA PHARMA LTD"~"BYFAVO (Remimazolam)"~7/2/2020 0:00:00~"Original"~1~"3889-4"~"PMR"~"Pediatric Research Equity Act"~3889.00~4~"PMR 3889-4: Conduct a juvenile animal toxicology study in a nonrodent model to characterize the effects of remimazolam on the developing central nervous system to support clinical studies in pediatric patients under three years of age."~"Delayed"~"The final protocol, study completion, and final report submission milestones were missed due to the need to conduct pilot work before initiation of study. A Deferral Extension request was denied per FDA letter dated 7/11/2025."~12/31/2024 0:00:00~12/5/2025 0:00:00
383736~"CDER"~"NDA"~213721.00~"RIGEL PHARMACEUTICALS INC"~"GAVRETO (pralsetinib)"~9/4/2020 0:00:00~"Original"~1~"3916-6"~"PMR"~"505 (o)(3)"~3916.00~6~"PMR 3916-6: Conduct a clinical drug interaction study to evaluate the effect of repeat doses of pralsetinib on the pharmacokinetics of transporter substrates of P-gp, BCRP, OATP1B1, OATP1B3, MATE-1, and MATE-2K, assess the magnitude of exposure change, and inform appropriate dosing strategies for coadministration of pralsetinib with these transporter substrates. Design and conduct the study in accordance with the FDA Guidance for Industry titled:  Clinical Drug Interaction Studies - Cytochrome P450 Enzyme and Transporter- Mediated Drug Interactions . Submit the datasets with final report. The results from this study may inform product labeling."~"Delayed"~"The final report milestone was missed due to awaiting data from the study with the final clinical study report expected in June 2026."~3/31/2025 0:00:00~10/30/2025 0:00:00
383737~"CDER"~"NDA"~213721.00~"RIGEL PHARMACEUTICALS INC"~"GAVRETO (pralsetinib)"~9/4/2020 0:00:00~"Original"~1~"3916-7"~"PMR"~"505 (o)(3)"~3916.00~7~"PMR 3916-7: Conduct a clinical drug interaction study to evaluate the effect of repeat doses of pralsetinib on the pharmacokinetics of sensitive substrates of CYP3A4/5, CYP2C8, and CYP2C9, assess the magnitude of exposure change, and inform appropriate dosing strategies for safe coadministration of pralsetinib with sensitive substrates of CYP3A4/5, CYP2C8, and CYP2C9. Design and conduct the study in accordance with the FDA Guidance for Industry titled:  Clinical Drug Interaction Studies - Cytochrome P450 Enzyme and Transporter- Mediated Drug Interactions . Submit the datasets with the final report. The results from this study may inform product labeling."~"Delayed"~"The Study Completion and Final Report Submission Milestones were missed because additional time was needed to select a partner capable of executing a comprehensive and robust study."~3/31/2025 0:00:00~10/30/2025 0:00:00
383738~"CDER"~"NDA"~213721.00~"RIGEL PHARMACEUTICALS INC"~"GAVRETO (pralsetinib)"~9/4/2020 0:00:00~"Original"~1~"3916-9"~"PMC"~"506B PMC"~3916.00~9~"PMC 3916-9: Submit the final report including datasets from a multi-center, randomized trial comparing pralsetinib to physician s choice of platinum-based chemotherapy treatment regimens based on standard of care treatment for the first-line treatment of RET fusion-positive, metastatic non-small cell lung cancer. The results from this trial may inform product labeling."~"Fulfilled"~~6/30/2026 0:00:00~10/30/2025 0:00:00
383739~"CDER"~"NDA"~213721.00~"RIGEL PHARMACEUTICALS INC"~"GAVRETO (pralsetinib)"~9/4/2020 0:00:00~"Supplement"~14~"4475-1"~"PMR"~"505 (o)(3)"~4475.00~1~"PMR 4475-1: Conduct an integrated safety analysis from all available clinical trial data that characterizes the potential serious risk of long-term adverse effects of pralsetinib on growth and development, including an assessment of growth plate abnormalities, in a sufficient number of adolescent patients 12 years of age or older with solid tumors treated with pralsetinib. Patients will be monitored for growth and development using age-appropriate screening tools such as Tanner staging. Evaluations will include growth as measured by height, weight, height velocity and height standard deviation scores, age at adrenarche if applicable (males), age at menarche if applicable (females) and Tanner stage. Patient monitoring will be performed until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first."~"Pending"~~4/30/2033 0:00:00~10/30/2025 0:00:00
383740~"CDER"~"NDA"~213721.00~"RIGEL PHARMACEUTICALS INC"~"GAVRETO (pralsetinib)"~9/4/2020 0:00:00~"Supplement"~14~"4475-2"~"PMC"~"506B PMC"~4475.00~2~"PMC 4475-2: Conduct an analytical and clinical validation study, using clinical trial data, of an in vitro diagnostic device that demonstrates the device is essential to the safe and effective use of pralsetinib for patients with RET gene fusion thyroid cancers."~"Delayed"~"The Final Report Submission Milestone was missed because additional time was needed to select a partner capable of executing a comprehensive and robust study."~10/31/2024 0:00:00~10/30/2025 0:00:00
383741~"CDER"~"NDA"~213736.00~"INCYTE CORP"~"Pemazyre (pemigatinib)"~4/17/2020 0:00:00~"Original"~1~"3801-1"~"PMR"~"Accelerated Approval"~3801.00~1~"PMR 3801-1: Submit the results of a randomized trial demonstrating improvement of progression-free survival or overall survival in patients with unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth receptor 2 (FGFR2) gene fusion or rearrangement, to confirm clinical benefit of pemigatinib."~"Ongoing"~"The trial has completed enrollment."~12/31/2026 0:00:00~6/13/2025 0:00:00
383742~"CDER"~"NDA"~213736.00~"INCYTE CORP"~"Pemazyre (pemigatinib)"~4/17/2020 0:00:00~"Supplement"~2~"4325-1"~"PMC"~"506B PMC"~4325.00~1~"PMC 4325-1: Complete Study FIGHT-203 to obtain data on long-term efficacy and safety of pemigatinib in patients with myeloid/lymphoid neoplasms with FGFR1 gene rearrangement. Provide data after the final patient completes follow-up or withdraws from the study."~"Fulfilled"~~12/31/2023 0:00:00~6/13/2025 0:00:00
383743~"CDER"~"NDA"~213756.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Koselugo (selumetinib)"~4/10/2020 0:00:00~"Original"~1~"3806-1"~"PMR"~"505 (o)(3)"~3806.00~1~"PMR 3806-1: Characterize and evaluate the long-term safety effects and any potential for serious adverse risks of selumetinib on the growth and development of pediatric patients. Submit the complete final report and long-term follow-up data from pediatric patients enrolled on SPRINT and ongoing or completed studies of selumetinib. All patients must be evaluated for growth and development milestones annually for at least 7 years from initiation of selumetinib. Evaluations must include: growth as measured by weight, height, height velocity, height standard deviation scores (SDS), age at thelarche (females), age at adrenarche (males), age at menarche (females), and Tanner Stage progression. Descriptive statistics (including mean and standard deviation values) of onstudy data for growth velocity must be presented. Growth velocity during the trial should be compared with growth velocity at baseline (if pre-baseline data are available). Provide analyses of height and weight data that assess measures of central tendency and outlier analyses using height and weight z-scores."~"Submitted"~~3/31/2026 0:00:00~5/29/2026 0:00:00
383744~"CDER"~"NDA"~213756.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Koselugo (selumetinib)"~4/10/2020 0:00:00~"Original"~1~"3806-2"~"PMR"~"505 (o)(3)"~3806.00~2~"PMR 3806-2: Characterize and evaluate the long-term safety effects and any potential for specific serious adverse risks of selumetinib in pediatric patients. Submit the complete final report and long-term follow-up safety data (minimum of 7 years) from pediatric patients enrolled on SPRINT and all other ongoing or completed studies of selumetinib to include an analysis of the following toxicities in pediatric patients: ocular toxicity (including but not limited to retinal pigment epithelial detachment and retinal vein occlusion), cardiac toxicity (including but not limited to ventricular dysfunction), muscle toxicity (including but not limited to rhabdomyolysis and symptomatic and asymptomatic CPK elevation), serious gastrointestinal toxicity (including but not limited to colitis, ileus, intestinal obstruction, and intestinal perforation), and serious dermatologic toxicity."~"Submitted"~~3/31/2026 0:00:00~5/29/2026 0:00:00
383745~"CDER"~"NDA"~213871.00~"PFIZER INC"~"Cibinqo (Abrocitinib)"~1/14/2022 0:00:00~"Original"~1~"4217-2"~"PMR"~"Pediatric Research Equity Act"~4217.00~2~"PMR 4217-2: Conduct a clinical trial in subjects 2 to 12 years of age with moderate-tosevere atopic dermatitis."~"Ongoing"~"41 patients out of the planned 240 patients have been enrolled
into the study."~12/31/2030 0:00:00~3/13/2026 0:00:00
383746~"CDER"~"NDA"~215320.00~"HIKMA PHARMACEUTICALS USA INC"~"Combogesic IV (acetaminophen and ibuprofen)"~10/17/2023 0:00:00~"Original"~1~"4517-1"~"PMR"~"Pediatric Research Equity Act"~4517.00~1~"PMR 4517-1: Conduct a juvenile animal toxicology study in rats to support clinical dosing of Combogesic IV in pediatric patients from birth to less than 2 years of age. This study will evaluate the effects of acetaminophen and ibuprofen on the development of previously identified target organs of toxicity, which includes the liver, kidney, lung, and testes."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~12/31/2025 0:00:00~
383747~"CDER"~"NDA"~215320.00~"HIKMA PHARMACEUTICALS USA INC"~"Combogesic IV (acetaminophen and ibuprofen)"~10/17/2023 0:00:00~"Original"~1~"4517-2"~"PMR"~"Pediatric Research Equity Act"~4517.00~2~"PMR 4517-2: Conduct a pharmacokinetic and safety study of Combogesic IV in pediatric patients aged 2 to less than 17 years for the short-term management of mild to moderate acute pain."~"Delayed"~"Original Final Report Due Date: 09/30/2025; Deferral Extension granted per FDA letter dated 09/02/2025. The study completion milestone was also revised."~6/30/2027 0:00:00~
383748~"CDER"~"NDA"~215320.00~"HIKMA PHARMACEUTICALS USA INC"~"Combogesic IV (acetaminophen and ibuprofen)"~10/17/2023 0:00:00~"Original"~1~"4517-3"~"PMR"~"Pediatric Research Equity Act"~4517.00~3~"PMR 4517-3: Conduct an efficacy, safety, and pharmacokinetic study of Combogesic IV in infants from birth to less than 2 years of age for the short-term management of mild to moderate acute pain."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~12/31/2027 0:00:00~
383749~"CDER"~"NDA"~215344.00~"AZURITY PHARMACEUTICALS INC"~"SUFLAVE (polyethylene glycol 3350, sodium sulfate, potassium chloride, magnesium sulfate, and sodium chloride)"~6/15/2023 0:00:00~"Original"~1~"4225-1"~"PMR"~"Pediatric Research Equity Act"~4225.00~1~"PMR 4225-1: A study to evaluate safety and efficacy of Suflave (polyethylene glycol 3350, sodium sulfate, potassium chloride, magnesium sulfate, and sodium chloride for oral solution) versus an approved comparator in pediatric patients 12 to <18 years of age. The study will include dose determination and pharmacokinetic evaluation."~"Delayed"~"The final protocol submission milestone was missed."~9/30/2026 0:00:00~8/11/2025 0:00:00
383750~"CDER"~"NDA"~215344.00~"AZURITY PHARMACEUTICALS INC"~"SUFLAVE (polyethylene glycol 3350, sodium sulfate, potassium chloride, magnesium sulfate, and sodium chloride)"~6/15/2023 0:00:00~"Original"~1~"4225-2"~"PMR"~"Pediatric Research Equity Act"~4225.00~2~"PMR 4225-2: A study to evaluate safety and efficacy of Suflave (polyethylene glycol 3350, sodium sulfate, potassium chloride, magnesium sulfate, and sodium chloride for oral solution) versus an approved comparator in pediatric patients 1 year to <12 years of age. The study will include dose determination and pharmacokinetic evaluation."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~4/30/2030 0:00:00~8/11/2025 0:00:00
383751~"CDER"~"NDA"~215358.00~"NOVARTIS PHARMACEUTICALS CORP"~"Scemblix (Asciminib)"~10/29/2021 0:00:00~"Original"~1~"4161-2"~"PMR"~"Pediatric Research Equity Act"~4161.00~2~"PMR 4161-2: Conduct a clinical trial to determine the appropriate dose of asciminib and to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of asciminib in pediatric patients with Ph+ CML-CP previously treated with one or more tyrosine kinase inhibitors, ages =1 to <18 years and provide the core study report with at least 12 months (52 weeks) data for all patients. Include at least 15 patients =1 to <12 years old and 15 patients =12 to <18 years old."~"Ongoing"~"The trial has completed enrollment.
"~7/31/2028 0:00:00~12/12/2025 0:00:00
383752~"CDER"~"NDA"~215358.00~"NOVARTIS PHARMACEUTICALS CORP"~"Scemblix (Asciminib)"~10/29/2021 0:00:00~"Original"~1~"4161-5"~"PMC"~"506B PMC"~4161.00~5~"PMC 4161-5: Conduct a clinical pharmacokinetic study to evaluate the effect of repeat doses of a strong CYP3A and UGT inducer on the single dose (200 mg) pharmacokinetics of asciminib to assess the magnitude of decreased drug exposure and to determine appropriate dosing recommendations. Design and conduct the study in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Cytochrome P450 Enzyme-and Transporter-Mediated Drug Interactions Guidance for Industry "~"Fulfilled"~~7/31/2023 0:00:00~12/12/2025 0:00:00
383753~"CDER"~"NDA"~215358.00~"NOVARTIS PHARMACEUTICALS CORP"~"Scemblix (Asciminib)"~10/29/2021 0:00:00~"Supplement"~1~"4348-1"~"PMC"~"506B PMC"~4348.00~1~"PMC 4348-1: Complete Study ASCEMBL to obtain data on long-term efficacy and safety of asciminib in patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs). Provide data at the end of treatment period on safety and efficacy, and at the end of the 5-year follow up period (overall survival and progression-free survival)."~"Fulfilled"~~7/31/2025 0:00:00~12/12/2025 0:00:00
383754~"CDER"~"NDA"~215358.00~"NOVARTIS PHARMACEUTICALS CORP"~"Scemblix (Asciminib)"~10/29/2021 0:00:00~"Supplement"~8~"4724-1"~"PMR"~"Accelerated Approval"~4724.00~1~"PMR 4724-1: Complete clinical study CABL001J12301 (ASC4FIRST), A Phase 3, Multicenter, Open-label, Randomized Study of Oral Asciminib versus Investigator Selected TKI in Patients with Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase, intended to describe and confirm the clinical benefit of asciminib to include at least 60 months follow-up of all patients."~"Ongoing"~"The trial has completed enrollment."~6/30/2028 0:00:00~12/12/2025 0:00:00
383755~"CDER"~"NDA"~215358.00~"NOVARTIS PHARMACEUTICALS CORP"~"Scemblix (Asciminib)"~10/29/2021 0:00:00~"Supplement"~8~"4724-2"~"PMC"~"506B PMC"~4724.00~2~"PMC 4724-2: Conduct an integrated analysis of data from clinical trials and observational studies (e.g., real world evidence), post-marketing reports, and other sources to further characterize the safety and efficacy/effectiveness of asciminib in patients of underrepresented racial and ethnic minority groups with chronic myelogenous leukemia. The analyses should support an evaluation of comparative efficacy/effectiveness and safety between the population primarily represented in the trial (Study CABL001J12301) and the aforementioned underrepresented population. This should also include pharmacokinetic data if available."~"Pending"~~6/30/2028 0:00:00~12/12/2025 0:00:00
383756~"CDER"~"NDA"~215383.00~"MERCK SHARP AND DOHME CORP A SUB OF MERCK AND CO INC"~"Welireg (belzutifan)"~8/13/2021 0:00:00~"Original"~1~"4132-1"~"PMR"~"505 (o)(3)"~4132.00~1~"PMR 4132-1: Conduct a carcinogenicity study in mice to evaluate the potential for carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Fulfilled"~~4/30/2026 0:00:00~10/7/2025 0:00:00
383757~"CDER"~"NDA"~215383.00~"MERCK SHARP AND DOHME CORP A SUB OF MERCK AND CO INC"~"Welireg (belzutifan)"~8/13/2021 0:00:00~"Original"~1~"4132-2"~"PMR"~"505 (o)(3)"~4132.00~2~"PMR 4132-2: Conduct a carcinogenicity study in rats to evaluate the potential for carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Submitted"~~4/30/2026 0:00:00~10/7/2025 0:00:00
383758~"CDER"~"NDA"~215383.00~"MERCK SHARP AND DOHME CORP A SUB OF MERCK AND CO INC"~"Welireg (belzutifan)"~8/13/2021 0:00:00~"Original"~1~"4132-3"~"PMR"~"505 (o)(3)"~4132.00~3~"PMR 4132-3: Conduct an analysis from Study MK-6482-004 to further characterize and determine the incidence and severity of anemia, hypoxia, second primary malignancies and other serious adverse events in patients receiving belzutifan. Include incidence rates, time to onset, outcomes, red cell transfusion and the use of erythropoiesis stimulating agents for anemia and steps taken to mitigate these risks in the reports. Provide interim reports annually for 3 years."~"Ongoing"~~12/31/2026 0:00:00~10/7/2025 0:00:00
383759~"CDER"~"NDA"~216986.00~"GUERBET"~"Elucirem (gadopiclenol)"~9/21/2022 0:00:00~"Original"~1~"4341-4"~"PMR"~"505 (o)(3)"~4341.00~4~"PMR 4341-4: Conduct a prospective longitudinal cohort study with one or more matched control group(s) to evaluate the effects of repetitive gadopiclenol administration on a comprehensive battery of neurobehavioral testing over the course of at least five administrations. The study should be sufficiently powered to exclude a prespecified magnitude of decline. As a secondary objective, study patients should also have the option of providing blood and urine samples at the time of reimaging, so that normative estimates of gadolinium concentration across an extended range of post-administration timepoints may be documented. We acknowledge that you will participate in the existing ODYSSEY study with the other GBCA sponsors. The Interim Report should contain a summary of patient enrollment listed by:   Study initiation (first patient enrolled at each site)   Number of patients at each site listed by condition under yearly contrast MRI surveillance   Number of patients receiving gadopiclenol   Number of patients providing blood and urine samples for gadolinium levels   Number of dropouts"~"Ongoing"~~4/30/2029 0:00:00~11/20/2025 0:00:00
383760~"CDER"~"NDA"~216993.00~"DAIICHI SANKYO INC"~"Vanflyta (quizartinib) Tablets "~7/20/2023 0:00:00~"Original"~1~"4467-1"~"PMR"~"Pediatric Research Equity Act"~4467.00~1~"PMR 4467-1: Conduct a clinical study to confirm the appropriate dose of quizartinib, and to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of quizartinib in combination with fludarabine and cytarabine, in pediatric patients ages =1 month to <18 years with relapsed/refractory FLT3-ITD positive AML. Patients should be followed for at least 12 months (52 weeks). Include at least 16 patients =1 month to <18 years old, including a minimum of 4 patients < 12 years old."~"Ongoing"~"The trial has completed enrollment."~2/28/2031 0:00:00~9/17/2025 0:00:00
383761~"CDER"~"NDA"~216993.00~"DAIICHI SANKYO INC"~"Vanflyta (quizartinib) Tablets "~7/20/2023 0:00:00~"Original"~1~"4467-2"~"PMR"~"505 (o)(3)"~4467.00~2~"PMR 4467-2: Conduct an observational study using electronic health records (EHR) to further assess the risk of severe (Grades 3-4) and fatal ventricular arrhythmia events in adult patients treated with quizartinib for the indication of acute myeloid leukemia (AML) that is FLT3-ITD positive as detected by an FDA-approved test. The selected EHR data source should contain access to clinical data elements including ECG results, laboratory results, concomitant medications, and clinical data to allow for outcome validation (i.e., via chart review). Evaluate the incidence of severe and fatal arrhythmia events and collect detailed clinical features of the adverse reactions, to investigate associations and temporal relationships between the incidence and severity of arrhythmia events and other potential associated risk factors. Specify case definitions, measurement, validation methods, and procedures for all study outcomes."~"Ongoing"~~5/31/2030 0:00:00~9/17/2025 0:00:00
383762~"CDER"~"NDA"~216993.00~"DAIICHI SANKYO INC"~"Vanflyta (quizartinib) Tablets "~7/20/2023 0:00:00~"Original"~1~"4467-3"~"PMR"~"505 (o)(3)"~4467.00~3~"PMR 4467-3: Conduct an exercise test study in healthy subjects to further evaluate the serious risk of QTc prolongation with quizartinib. Evaluate the impact of rapid acceleration in heart rate on the cardiac safety of quizartinib using a standardized test protocol such as Bruce protocol, modified Bruce protocol, or graded-intensity bicycle exercise test. Identify QT/QTc and RR intervals at rest, peak exercise, and recovery, and capture the incidence of arrhythmias and other adverse reactions."~"Delayed"~"The Final Report milestone was missed because of an unexpected delay in evaluating the required dataset because the study triggered pre-specified additional QTcI modeling and simulation analyses. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 05/05/2026."~4/30/2026 0:00:00~9/17/2025 0:00:00
383763~"CDER"~"NDA"~216993.00~"DAIICHI SANKYO INC"~"Vanflyta (quizartinib) Tablets "~7/20/2023 0:00:00~"Original"~1~"4467-4"~"PMR"~"505 (o)(3)"~4467.00~4~"PMR 4467-4: Conduct a sub-maximal exercise test study in patients with AML to further evaluate the serious risk of QTc prolongation with quizartinib. Evaluate the impact of rapid acceleration in heart rate on the cardiac safety of quizartinib using a 24-Hour Holter study with sub-maximal exercise (e.g.,postural provocation   i.e., supine to standing). Identify QT/QTc and RR intervals at rest and sub-maximal exercise and capture the incidence of arrhythmias and other adverse reactions. Include patients on beta-blockers in order to observe for mitigating effects on the incidence of quizartinib-related adverse reactions."~"Pending"~~10/31/2027 0:00:00~9/17/2025 0:00:00
383764~"CDER"~"NDA"~216993.00~"DAIICHI SANKYO INC"~"Vanflyta (quizartinib) Tablets "~7/20/2023 0:00:00~"Original"~1~"4467-5"~"PMR"~"505 (o)(3)"~4467.00~5~"PMR 4467-5: Conduct a randomized trial of quizartinib maintenance therapy that compares the recommended dosage of 53 mg daily to a lower dosage (e.g., 26.5 mg daily) to further characterize serious adverse reactions including but not limited to the rates of Grade =3 adverse reactions, Grade = 3 neutropenia, QTc interval prolongation, and dose reductions, interruptions, and discontinuations due to adverse reactions and provide an analysis of dose- and exposure-response relationships for safety. Incorporate systematically assessed patient-reported outcome assessments to further characterize safety and tolerability, including patient-reported symptoms, function, and overall side effect impact. Eligible patients will include newly diagnosed, FLT3-ITD positive AML patients in complete remission following consolidation and exclude patients who underwent allogeneic hematopoietic stem cell transplantation. The study should also provide an analysis of dose- and exposure-response relationships for efficacy, including overall survival and relapse-free survival."~"Pending"~~3/31/2033 0:00:00~9/17/2025 0:00:00
383765~"CDER"~"NDA"~216993.00~"DAIICHI SANKYO INC"~"Vanflyta (quizartinib) Tablets "~7/20/2023 0:00:00~"Original"~1~"4467-6"~"PMR"~"505 (o)(3)"~4467.00~6~"PMR 4467-6: Conduct a clinical pharmacokinetic trial to assess the magnitude of change in exposure of quizartinib and its metabolite AC886 and to determine appropriate dosing recommendations of quizartinib in patients with severe hepatic impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry: Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling."~"Delayed"~"The trial completion and final report submission milestones were missed because there is a limited population of severe hepatic impairment patients and a high screening failure rate, so recruitment into the trial has been slower than anticipated. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 04/06/2026."~3/31/2026 0:00:00~9/17/2025 0:00:00
383766~"CDER"~"NDA"~216993.00~"DAIICHI SANKYO INC"~"Vanflyta (quizartinib) Tablets "~7/20/2023 0:00:00~"Original"~1~"4467-7"~"PMC"~"506B PMC"~4467.00~7~"PMC 4467-7: Conduct exploratory analyses aimed at identifying potential mechanisms of primary and acquired resistance to quizartinib using longitudinal samples for cytogenetic and mutational analyses collected at baseline and at the end of treatment or time of relapse from patients treated with quizartinib in QuANTUM-First. Include a discussion of the results in the context of the available published literature."~"Fulfilled"~~12/31/2024 0:00:00~9/17/2025 0:00:00
383767~"CDER"~"NDA"~212295.00~"ACACIA PHARMA LTD"~"BYFAVO (Remimazolam)"~7/2/2020 0:00:00~"Original"~1~"3889-5"~"PMR"~"Pediatric Research Equity Act"~3889.00~5~"PMR 3889-5: Conduct a juvenile animal toxicology study in a rodent model to characterize the effects of remimazolam on the developing central nervous system to support a clinical indication for use in pediatric patients greater than or equal to three years of age and below 18 years of age."~"Delayed"~"The final protocol, study completion, and final report submission milestones were missed because pilot studies need to be conducted prior to finalizing protocol. A
Deferral Extension requested 6/13/2024, denied per FDA letter dated 7/25/2024."~12/31/2022 0:00:00~12/5/2025 0:00:00
383768~"CDER"~"NDA"~212295.00~"ACACIA PHARMA LTD"~"BYFAVO (Remimazolam)"~7/2/2020 0:00:00~"Original"~1~"3889-6"~"PMR"~"505 (o)(3)"~3889.00~6~"PMR 3889-6: Conduct a male fertility study testing the drug product formulation that evaluates reproductive behavior and fertility and obtains pharmacokinetic analysis in a species that supports the maximum clinical exposures (Cmax and AUC) achieved with the proposed clinical indication."~"Delayed"~"The study completion milestone and final report submission milestone were missed because additional time is needed to complete prerequisite exploratory and pilot studies to inform the study designs, and FDA determined that the applicant demonstrated  good cause  for the delay per FDA letter dated 1/17/2025."~6/30/2022 0:00:00~12/5/2025 0:00:00
383769~"CDER"~"NDA"~212295.00~"ACACIA PHARMA LTD"~"BYFAVO (Remimazolam)"~7/2/2020 0:00:00~"Original"~1~"3889-7"~"PMR"~"505 (o)(3)"~3889.00~7~"PMR 3889-7: Conduct an embryo-fetal development study testing the drug product formulation in a species other than the rabbit that supports the maximum clinical exposures (Cmax and AUC) achieved with the proposed clinical indication."~"Delayed"~"The study completion milestone and final report submission milestone were missed because additional time is needed to complete prerequisite exploratory and pilot studies to inform the study designs, and FDA determined that the applicant demonstrated  good cause  for the delay per FDA letter dated 1/17/2025."~12/31/2022 0:00:00~12/5/2025 0:00:00
383770~"CDER"~"NDA"~212295.00~"ACACIA PHARMA LTD"~"BYFAVO (Remimazolam)"~7/2/2020 0:00:00~"Original"~1~"3889-8"~"PMR"~"505 (o)(3)"~3889.00~8~"PMR 3889-8: Conduct a pre-and post-natal development study testing the drug product formulation that evaluates all standard endpoints including learning, memory, and reproductive function of the F1 offspring and obtains adequate toxicokinetic data in a species that supports the maximum clinical exposures (Cmax and AUC) achieved with the proposed clinical indication."~"Delayed"~"The study completion milestone and final report submission milestone were missed because additional time is needed to complete prerequisite exploratory and pilot studies to inform the study designs, and FDA determined that the applicant demonstrated  good cause  for the delay per FDA letter dated 1/17/2025."~6/30/2023 0:00:00~12/5/2025 0:00:00
383771~"CDER"~"NDA"~212306.00~"KARYOPHARM THERAPEUTICS INC"~"XPOVIO (selinexor)"~7/3/2019 0:00:00~"Original"~1~"3657-3"~"PMR"~"505 (o)(3)"~3657.00~3~"PMR 3657-3: Conduct a hepatic impairment trial in patients with NCI classification of moderate, severe hepatic impairment or Child-Pugh classes B, and C compared to patients with normal hepatic function since drug clearance may be reduced with hepatic impairment. 
"~"Fulfilled"~~9/30/2021 0:00:00~8/29/2025 0:00:00
383772~"CDER"~"NDA"~212306.00~"KARYOPHARM THERAPEUTICS INC"~"XPOVIO (selinexor)"~7/3/2019 0:00:00~"Original"~1~"3657-5"~"PMC"~"506B PMC"~3657.00~5~"PMC 3657-5: Conduct a dedicated drug interaction trial in patients to determine the effect of co-administration of a strong CYP3A4 inducer on the
pharmacokinetics of selinexor."~"Fulfilled"~~9/30/2022 0:00:00~8/29/2025 0:00:00
383773~"CDER"~"NDA"~212306.00~"KARYOPHARM THERAPEUTICS INC"~"XPOVIO (selinexor)"~7/3/2019 0:00:00~"Original"~1~"3657-6"~"PMR"~"505 (o)(3)"~3657.00~6~"PMR 3657-6: Conduct a randomized, phase 2 clinical trial evaluating at least two lower doses or alternative dosage regimens of selinexor in combination with dexamethasone in patients with multiple myeloma to characterize and determine the rate of serious adverse reactions including myelosuppression, gastrointestinal toxicity, infection, hyponatremia, and neurological toxicity in all treatment arms. Patients in this trial should have relapsed refractory multiple myeloma, have received at least four prior lines of therapy, and have disease that is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. Include a characterization of all serious adverse events, dose-reductions, interruptions, and discontinuations due to serious adverse events, and efficacy in all treatment arms in the final report. Efficacy should be assessed by overall response rate according to International Myeloma Working Group (IMWG) criteria by investigator assessment."~"Delayed"~"The Trial Completion and Final Report Submission milestones were missed due to delays in enrollment."~1/31/2022 0:00:00~8/29/2025 0:00:00
383774~"CDER"~"NDA"~212306.00~"KARYOPHARM THERAPEUTICS INC"~"XPOVIO (selinexor)"~7/3/2019 0:00:00~"Supplement"~1~"3866-1"~"PMR"~"Accelerated Approval"~3866.00~1~"PMR 3866-1: Submit the final report and datasets from a randomized, double-blind, placebo-controlled phase 3 trial that verifies and describes the clinical benefit of selinexor in patients with relapsed or refractory diffuse large Bcell lymphoma. Patients should be randomized to receive chemoimmunotherapy with or without selinexor. The primary endpoint should be progression-free survival, with secondary endpoints that include overall survival and objective response rate."~"Released"~"Per FDA letter dated 04/30/2026, this PMR/PMC has been released."~4/30/2026 0:00:00~8/29/2025 0:00:00
383775~"CDER"~"NDA"~212306.00~"KARYOPHARM THERAPEUTICS INC"~"XPOVIO (selinexor)"~7/3/2019 0:00:00~"Supplement"~1~"3866-2"~"PMR"~"505 (o)(3)"~3866.00~2~"PMR 3866-2: Provide the interim and final analyses of a randomized phase 2 clinical trial of selinexor to characterize the safety and efficacy of at least two different dosing regimens of selinexor monotherapy in patients with relapsed or refractory diffuse large B-cell lymphoma after at least two prior lines of systemic therapy. Include one regimen with a lower starting dose and one regimen with a prespecified transition to a lower continuation dose, than the dosing regimen of 60 mg orally on Days 1 and 3 of each week. The primary efficacy endpoint should be overall response rate by independent review committee assessment. Include a prospective characterization of the presentation, management, and outcome of treatment-emergent hyponatremia in the final report, including the results of diagnostic evaluations and the supportive care provided. The results of this trial may inform product labeling. Submit datasets with the final report."~"Released"~~10/31/2023 0:00:00~8/29/2025 0:00:00
383776~"CDER"~"NDA"~212320.00~"SHIELD TX UK LTD"~"Accrufer (ferric maltol)"~7/25/2019 0:00:00~"Original"~1~"3667-1"~"PMR"~"Pediatric Research Equity Act"~3667.00~1~"PMR 3667-1: Conduct and submit study report and datasets from a study (PK sub-study of ST10-01-305) in pediatric patients with iron deficiency age 1 month to < 10 years for pharmacokinetics (PK) and pharmacodynamics (PD) to confirm the dosing used in the efficacy and safety study."~"Submitted"~"The final report was submitted to FDA on 06/23/2025."~7/31/2025 0:00:00~9/5/2025 0:00:00
383777~"CDER"~"NDA"~212320.00~"SHIELD TX UK LTD"~"Accrufer (ferric maltol)"~7/25/2019 0:00:00~"Original"~1~"3667-2"~"PMR"~"Pediatric Research Equity Act"~3667.00~2~"PMR 3667-2: Conduct and submit study report and datasets from a study (ST10-01-103) in pediatric patients with iron deficiency age 10 to 17 years for pharmacokinetics and pharmacodynamics to determine the dosing to be used in an efficacy and safety study."~"Fulfilled"~"Per FDA letter dated 12/19/2025, this PMR/PMC has been fulfilled."~8/31/2019 0:00:00~9/5/2025 0:00:00
383778~"CDER"~"NDA"~213871.00~"PFIZER INC"~"Cibinqo (Abrocitinib)"~1/14/2022 0:00:00~"Original"~1~"4217-3"~"PMR"~"505 (o)(3)"~4217.00~3~"PMR 4217-3: Conduct a prospective observational study (analyses conducted in patient cohorts enrolled prospectively and followed actively in accordance with a written protocol) to assess the long-term safety of abrocitinib treatment in U.S. adult patients with moderate-to-severe atopic dermatitis. Fully ascertain and centrally verify serious adverse events, Major Adverse Cardiovascular Events (myocardial infarction, stroke, cardiovascular death, and sudden death), malignancies (including lymphoma, lung cancer, and other malignancies), serious infections, opportunistic infections (including herpes zoster), retinal detachment, thrombosis (including deep venous thrombosis, pulmonary embolism and arterial thrombosis), hepatoxicity (including drug induced liver injury) , and possibly other adverse events of special interest. For each adverse-event outcome separately, compare incidence in abrocitinib-treated patients against reference rates internally derived from analyses conducted in patients treated with dupilumab or other chronic systemic treatments for moderate-to-severe atopic dermatitis. Regardless of treatment discontinuation or switch to a different treatment for atopic dermatitis, continue following patients for malignancy outcomes and possibly other adverse events with delayed onset. Enroll a sufficient number of patients to describe the frequency of the adverse events of special interest in representative U.S. patients who start treatment with abrocitinib for atopic dermatitis in the setting of routine clinical practice. Implement a plan that uses rigorous, transparent, and verifiable methods to ascertain and characterize safety events that occur during and after treatment with abrocitinib. Enroll patients over a 4-year period and follow each patient for at least 8 years from time of enrollment."~"Pending"~~10/31/2034 0:00:00~3/13/2026 0:00:00
383779~"CDER"~"NDA"~213871.00~"PFIZER INC"~"Cibinqo (Abrocitinib)"~1/14/2022 0:00:00~"Original"~1~"4217-4"~"PMR"~"505 (o)(3)"~4217.00~4~"PMR 4217-4: Conduct or participate in a relevant Pregnancy Exposure Registry, a prospective, registry based observational exposure cohort study that compare the maternal, fetal, and infant oucomes of women exposed to abrocitinib during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Pending"~~12/31/2028 0:00:00~3/13/2026 0:00:00
383780~"CDER"~"NDA"~213871.00~"PFIZER INC"~"Cibinqo (Abrocitinib)"~1/14/2022 0:00:00~"Original"~1~"4217-5"~"PMR"~"505 (o)(3)"~4217.00~5~"PMR 4217-5: Conduct an additional pregnancy study that uses a different design from the Pregnancy Exposure Registry (for example a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to abrocitinib during pregnancy compared to an unexposed control population."~"Pending"~~6/30/2028 0:00:00~3/13/2026 0:00:00
383781~"CDER"~"NDA"~213871.00~"PFIZER INC"~"Cibinqo (Abrocitinib)"~1/14/2022 0:00:00~"Original"~1~"4217-6"~"PMC"~"506B PMC"~4217.00~6~"PMC 4217-6: Conduct a drug-drug interaction trial in healthy subjects using clinical dose of oral abrocitinib and gastric acid reducing agents (e.g., esomeprazole)."~"Submitted"~~4/30/2022 0:00:00~3/13/2026 0:00:00
383782~"CDER"~"NDA"~213953.00~"MARIUS PHARMACEUTICALS INC"~"Kyzatrex (testosterone undecanoate)"~7/27/2022 0:00:00~"Original"~1~"4313-1"~"PMR"~"Pediatric Research Equity Act"~4313.00~1~"PMR 4313-1: A trial of testosterone replacement therapy in pediatric males ages 12 years to less than 18 years of age for conditions associated with a deficiency or absence of endogenous testosterone due to primary hypogonadism or hypogonadotropic hypogonadism."~"Delayed"~"The Draft and Final Protocol milestones were missed because of manufacturing issues. FDA communicated that this was not sufficient to justify  good cause  for the delay."~2/28/2030 0:00:00~10/17/2025 0:00:00
383783~"CDER"~"NDA"~213969.00~"SENTYNL THERAPEUTICS INC"~"Zokinvy (lonafarnib)"~11/20/2020 0:00:00~"Original"~1~"3929-1"~"PMR"~"505 (o)(3)"~3929.00~1~"PMR 3929-1: A carcinogenicity study in an appropriate species."~"Fulfilled"~~2/28/2024 0:00:00~1/16/2026 0:00:00
383784~"CDER"~"NDA"~213972.00~"ITERUM THERAPEUTICS US LTD"~"Orlynvah (sulopenem etzadroxil and probenecid) "~10/25/2024 0:00:00~"Original"~1~"4722-1"~"PMR"~"Pediatric Research Equity Act"~4722.00~1~"PMR 4722-1: Conduct an open label, single-dose study to evaluate the pharmacokinetics, safety, and tolerability of sulopenem etzadroxil/probenecid in pediatric patients aged 12 years to less than 18 years with bacterial infection and receiving background antibacterial therapy."~"Delayed"~"The final protocol milestone was missed. The final protocol was submitted 7-21-2025"~12/31/2026 0:00:00~12/5/2025 0:00:00
383785~"CDER"~"NDA"~213972.00~"ITERUM THERAPEUTICS US LTD"~"Orlynvah (sulopenem etzadroxil and probenecid) "~10/25/2024 0:00:00~"Original"~1~"4722-2"~"PMR"~"Pediatric Research Equity Act"~4722.00~2~"PMR 4722-2: Conduct an open label, single-dose study to evaluate the pharmacokinetics, safety, and tolerability of sulopenem etzadroxil/probenecid in pediatric patients aged 2 years to less than 12 years with bacterial infection and receiving background antibacterial therapy."~"Pending"~"Draft protocol is due 3-2027"~12/31/2029 0:00:00~12/5/2025 0:00:00
383786~"CDER"~"NDA"~213972.00~"ITERUM THERAPEUTICS US LTD"~"Orlynvah (sulopenem etzadroxil and probenecid) "~10/25/2024 0:00:00~"Original"~1~"4722-3"~"PMR"~"Pediatric Research Equity Act"~4722.00~3~"PMR 4722-3: Conduct a randomized, controlled study to evaluate the safety, tolerability, and efficacy of sulopenem etzadroxil/probenecid in pediatric patients aged 2 years to less than 18 years with uncomplicated urinary tract infections (uUTI)."~"Pending"~"Draft protocol is due 12-2029."~12/31/2032 0:00:00~12/5/2025 0:00:00
383787~"CDER"~"NDA"~213972.00~"ITERUM THERAPEUTICS US LTD"~"Orlynvah (sulopenem etzadroxil and probenecid) "~10/25/2024 0:00:00~"Original"~1~"4722-4"~"PMR"~"505 (o)(3)"~4722.00~4~"PMR 4722-4: Conduct a U.S. surveillance study over a five-year period after the introduction of sulopenem etzadroxil/probenecid to the market to
determine if resistance or decreased susceptibility to sulopenem etzadroxil/probenecid is occurring in the target population of bacteria identified in the approved sulopenem etzadroxil/probenecid label."~"Pending"~~8/31/2030 0:00:00~12/5/2025 0:00:00
383788~"CDER"~"NDA"~213976.00~"TAKEDA PHARMACEUTICALS USA INC"~"Eohilia (budesonide oral suspension)"~2/9/2024 0:00:00~"Original"~1~"4574-1"~"PMC"~"506B PMC"~4574.00~1~"PMC 4574-1: Conduct a randomized, double blind, placebo-controlled clinical trial to evaluate the safety, efficacy, and pharmacokinetics of Eohilia (budesonide oral suspension) in pediatric patients 1 to less than 11 years of age with active eosinophilic esophagitis."~"Pending"~~12/31/2029 0:00:00~4/8/2025 0:00:00
383789~"CDER"~"NDA"~214012.00~"NOVARTIS PHARMACEUTICALS CORP"~"Leqvio (inclisiran)"~12/22/2021 0:00:00~"Original"~1~"4186-1"~"PMR"~"Pediatric Research Equity Act"~4186.00~1~"PMR 4186-1: Conduct a two-part (double-blind inclisiran versus placebo [Year 1] followed by open-label with placebo-treated subjects switched to inclisiran [Year 2]), multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 12 to <18 years) with heterozygous familial hypercholesterolemia (HeFH)."~"Fulfilled"~"Per FDA letter dated 02/12/2026, this PMR/PMC has been fulfilled."~12/31/2025 0:00:00~2/17/2026 0:00:00
383790~"CDER"~"NDA"~215383.00~"MERCK SHARP AND DOHME CORP A SUB OF MERCK AND CO INC"~"Welireg (belzutifan)"~8/13/2021 0:00:00~"Original"~1~"4132-4"~"PMC"~"506B PMC"~4132.00~4~"PMC 4132-4: Provide an analysis for the overall response rate and duration of response in all patients and in the subgroups of patients with partial or complete VHL gene deletion, CNS hemangioblastoma, and pNET enrolled in Study MK-6482-004 annually for five years. The study reports should include the number of patients who undergo surgical intervention and/or procedures for renal cell carcinoma (RCC) and/or non-RCC tumors and who develop
metastatic RCC."~"Ongoing"~~12/31/2026 0:00:00~10/7/2025 0:00:00
383791~"CDER"~"NDA"~215383.00~"MERCK SHARP AND DOHME CORP A SUB OF MERCK AND CO INC"~"Welireg (belzutifan)"~8/13/2021 0:00:00~"Original"~1~"4132-5"~"PMC"~"506B PMC"~4132.00~5~"PMC 4132-5: Conduct a clinical trial evaluating overall response rate, and duration of response, to further characterize the efficacy and clinical benefit of belzutifan in patients with VHL disease-associated non-RCC tumors, including an adequate number of patients and representation of tumor types, with a minimum of 30 patients in the following subgroups: (1) Pheochromocytoma/paraganglioma and (2) pNET. Characterize response rate and duration of response for at least 24 months from the onset of response."~"Ongoing"~~8/31/2026 0:00:00~10/7/2025 0:00:00
383792~"CDER"~"NDA"~215383.00~"MERCK SHARP AND DOHME CORP A SUB OF MERCK AND CO INC"~"Welireg (belzutifan)"~8/13/2021 0:00:00~"Original"~1~"4132-6"~"PMC"~"506B PMC"~4132.00~6~"PMC 4132-6: Submit the final objective response rate and duration of response to further characterize efficacy in the subgroup of patients with VHL disease from the phase II clinical trial of belzutifan for treatment of advanced and metastatic pheochromocytoma/paraganglioma or pancreatic neuroendocrine tumor (MK-6482-015, NCT04924075).
"~"Ongoing"~~8/31/2026 0:00:00~10/7/2025 0:00:00
383793~"CDER"~"NDA"~215383.00~"MERCK SHARP AND DOHME CORP A SUB OF MERCK AND CO INC"~"Welireg (belzutifan)"~8/13/2021 0:00:00~"Supplement"~12~"4842-1"~"PMR"~"505 (o)(3)"~4842.00~1~"PMR 4842-1: Conduct a comprehensive, integrated safety analysis after 5 years of follow-up from clinical trials in a sufficient number of pediatric patients to adequately characterize and evaluate, in pediatric patients, the known serious risks of anemia and hypoxia following exposure to belzutifan. Include an assessment of clinical responses."~"Pending"~~7/31/2034 0:00:00~10/7/2025 0:00:00
383794~"CDER"~"NDA"~215383.00~"MERCK SHARP AND DOHME CORP A SUB OF MERCK AND CO INC"~"Welireg (belzutifan)"~8/13/2021 0:00:00~"Supplement"~12~"4842-2"~"PMC"~"506B PMC"~4842.00~2~"PMC 4842-2: Complete Cohort A1 of the LITESPARK-015 trial and provide updated duration of response data once all patients have been followed for at least 12 months from the onset of response to further characterize the efficacy and clinical benefit of belzutifan in patients 12 years of age and older with advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). Include biomarker analyses to evaluate genetic correlates of response to belzutifan."~"Ongoing"~~12/31/2026 0:00:00~10/7/2025 0:00:00
383795~"CDER"~"NDA"~215390.00~"BIOXCEL THERAPEUTICS INC"~"Igalmi (dexmedetomidine)"~4/5/2022 0:00:00~"Original"~1~"4254-1"~"PMR"~"Pediatric Research Equity Act"~4254.00~1~"PMR 4254-1: Conduct a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of dexmedetomidine films for the acute treatment of agitation in pediatric patients with schizophrenia (13 to 17 years of age) or bipolar I or II disorder (10 to 17 years of age)."~"Delayed"~"Original Final Report Due Date: 12/31/2024; Deferral Extension granted per FDA letter dated 12/13/2024. The study completion milestone was also revised.
"~6/30/2028 0:00:00~5/27/2026 0:00:00
383796~"CDER"~"NDA"~215390.00~"BIOXCEL THERAPEUTICS INC"~"Igalmi (dexmedetomidine)"~4/5/2022 0:00:00~"Original"~1~"4254-2"~"PMR"~"505 (o)(3)"~4254.00~2~"PMR 4254-2: Conduct a study to determine whether tolerance, tachyphylaxis, or withdrawal occur following repeat dosing of dexmedetomidine oral films."~"Submitted"~~10/31/2023 0:00:00~5/27/2026 0:00:00
383797~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-1"~"PMR"~"Pediatric Research Equity Act"~4233.00~1~"PMR 4233-1: Conduct a juvenile animal study to assess the safety of AXS-05 in animals of an age range and stage of development that are comparable to the population of children aged 7 to 12 years."~"Submitted"~"The final report was submitted to FDA on 10/4/2023."~6/30/2023 0:00:00~9/16/2025 0:00:00
383798~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-2"~"PMR"~"Pediatric Research Equity Act"~4233.00~2~"PMR 4233-2: Evaluate the pharmacokinetics, efficacy, and safety of AXS-05 in pediatric patients aged 7 to 17 years with MDD."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed"~5/31/2026 0:00:00~9/16/2025 0:00:00
383799~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-3"~"PMR"~"Pediatric Research Equity Act"~4233.00~3~"PMR 4233-3: Conduct a 1-year, open-label safety study in pediatric patients aged 7 to 17 years with MDD."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed"~5/31/2027 0:00:00~9/16/2025 0:00:00
383800~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-4"~"PMR"~"505 (o)(3)"~4233.00~4~"PMR 4233-4: Evaluate the effect of severe renal impairment on the pharmacokinetics of AXS-05."~"Submitted"~~10/31/2023 0:00:00~9/16/2025 0:00:00
383801~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-5"~"PMR"~"505 (o)(3)"~4233.00~5~"PMR 4233-5: Evaluate the effect of severe hepatic impairment on the pharmacokinetics of AXS-05."~"Delayed"~"The final protocol submission, study completion, and final report submission milestones have been missed. Protocol Amendments 2 & 3 were submitted on 9/9/24."~10/31/2023 0:00:00~9/16/2025 0:00:00
383802~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-6"~"PMR"~"505 (o)(3)"~4233.00~6~"PMR 4233-6: Perform a lactation study (milk only) in lactating women who have received AXS-05 to assess concentrations of dextromethorphan and its metabolites in breast milk using a validated assay. A mother-infant pair study may be required in the future depending on the results of this milkonly study."~"Submitted"~~3/31/2025 0:00:00~9/16/2025 0:00:00
383803~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-7"~"PMR"~"505 (o)(3)"~4233.00~7~"PMR 4233-7: Conduct an embryofetal toxicity study of AXS-05 in rabbits."~"Submitted"~~6/30/2023 0:00:00~9/16/2025 0:00:00
383804~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-8"~"PMR"~"505 (o)(3)"~4233.00~8~"PMR 4233-8: Conduct a fertility and early embryonic developmental study to address the effect of AXS-05 on fertility."~"Submitted"~~6/30/2023 0:00:00~9/16/2025 0:00:00
383805~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-9"~"PMR"~"505 (o)(3)"~4233.00~9~"PMR 4233-9: Conduct a pre- and postnatal developmental study to address the effect of AXS-05 on embryonic and post-natal development."~"Submitted"~~12/31/2023 0:00:00~9/16/2025 0:00:00
383806~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-11"~"PMR"~"505 (o)(3)"~4233.00~11~"PMR 4233-11: Conduct a study to address the neurotoxic potential of dextromethorphan using animals of an age range and stage of development that are comparable to the third trimester of gestation through the first several months of life but possibly extend to approximately three years of age in humans."~"Submitted"~~2/28/2023 0:00:00~9/16/2025 0:00:00
383807~"CDER"~"NDA"~216993.00~"DAIICHI SANKYO INC"~"Vanflyta (quizartinib) Tablets "~7/20/2023 0:00:00~"Original"~1~"4467-8"~"PMC"~"506B PMC"~4467.00~8~"PMC 4467-8: Conduct a clinical pharmacokinetic trial to assess the magnitude of decreased exposure of quizartinib and its metabolite AC886, and to determine appropriate dosing recommendations for quizartinib, with coadministration of a weak CYP3A inducer. Design and conduct the trial in accordance with the FDA Guidance for Industry: Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions."~"Fulfilled"~~7/31/2025 0:00:00~9/17/2025 0:00:00
383808~"CDER"~"NDA"~217003.00~"PHARMACYCLICS LLC"~"Imbruvica (Ibrutinib)"~8/24/2022 0:00:00~"Original"~1~"4328-1"~"PMR"~"505 (o)(3)"~4328.00~1~"PMR 4328-1: Conduct analyses to characterize long-term safety of ibrutinib in terms of growth and development in pediatric patients. Patients enrolled in Study PCYC-1146-IM should be evaluated for growth and development milestones annually for at least 5 years from the initiation of ibrutinib. Data should include growth parameters as measured by height and weight, sexual maturation by Tanner stage, performance status, immune reconstitution, adverse events, and patient-reported outcomes measures."~"Ongoing"~~7/31/2026 0:00:00~1/7/2026 0:00:00
383809~"CDER"~"NDA"~217026.00~"ACADIA PHARMACEUTICALS INC"~"Daybue (trofinetide)"~3/10/2023 0:00:00~"Original"~1~"4411-1"~"PMR"~"505 (o)(3)"~4411.00~1~"PMR 4411-1: Conduct a carcinogenicity study of trofinetide in mouse."~"Fulfilled"~~8/31/2026 0:00:00~5/7/2026 0:00:00
383810~"CDER"~"NDA"~217026.00~"ACADIA PHARMACEUTICALS INC"~"Daybue (trofinetide)"~3/10/2023 0:00:00~"Original"~1~"4411-2"~"PMR"~"505 (o)(3)"~4411.00~2~"PMR 4411-2: Conduct a 2-year carcinogenicity study of trofinetide in rat."~"Ongoing"~~8/31/2026 0:00:00~5/7/2026 0:00:00
383811~"CDER"~"NDA"~217159.00~"AYRMID PHARMA LTD"~"Aphexda (motixafortide)"~9/8/2023 0:00:00~"Original"~1~"4501-1"~"PMR"~"505 (o)(3)"~4501.00~1~"PMR 4501-1: Complete Study BL-8040.SCM.301:  A Phase III, Randomized, Double-Blinded, Placebo-Controlled, Multi-Centre Study Evaluating the Safety, Tolerability and Efficacy of Combination Treatment of BL-8040 and G-CSF as Compared to Placebo and G-CSF for the Mobilization of Hematopoietic Stem Cells for Autologous Transplantation in Subjects with Multiple Myeloma   the GENESIS Study . Provide at least 5 years of follow-up data for enrolled subjects. Long-term safety outcomes of interest include overall survival, progression free survival and rates of relapse."~"Ongoing"~~3/31/2029 0:00:00~11/6/2025 0:00:00
383812~"CDER"~"NDA"~217188.00~"PFIZER INC"~"Paxlovid (nirmatrelvir and ritonavir)"~5/25/2023 0:00:00~"Original"~1~"4392-1"~"PMR"~"Pediatric Research Equity Act"~4392.00~1~"PMR 4392-1: Conduct a study to evaluate the safety, tolerability, pharmacokinetics, and treatment response of PAXLOVID in pediatric subjects 6 to less than 18 years of age and weighing 20 kg or higher, with mild-to-moderate coronavirus disease 2019 (COVID-19)."~"Ongoing"~"Reporting enrollment of total of 77 participants in various age and weight cohorts as of 07/01/2025."~12/31/2024 0:00:00~7/22/2025 0:00:00
383813~"CDER"~"NDA"~217188.00~"PFIZER INC"~"Paxlovid (nirmatrelvir and ritonavir)"~5/25/2023 0:00:00~"Original"~1~"4392-2"~"PMR"~"Pediatric Research Equity Act"~4392.00~2~"PMR 4392-2: Conduct a study to evaluate the safety, tolerability, pharmacokinetics, and treatment response of PAXLOVID in pediatric subjects 2 to less than 6 years of age, with mild-to-moderate coronavirus disease 2019 (COVID-19)."~"Delayed"~"Original Final Report Due Date: 10/31/2025; Deferral Extension granted per FDA letter dated 06/13/2025. The study completion milestone was also revised. 
"~11/30/2028 0:00:00~7/22/2025 0:00:00
383814~"CDER"~"NDA"~217188.00~"PFIZER INC"~"Paxlovid (nirmatrelvir and ritonavir)"~5/25/2023 0:00:00~"Original"~1~"4392-3"~"PMR"~"Pediatric Research Equity Act"~4392.00~3~"PMR 4392-3: Conduct a study to evaluate the safety, tolerability, pharmacokinetics, and treatment response of PAXLOVID in pediatric subjects from birth to less than 2 years of age, with mild-to-moderate coronavirus disease 2019 (COVID-19)."~"Pending"~"The Deferral Extension Granted and Acknowledge Revised PMR
Milestones letter was issued on 06/13/2025."~1/31/2030 0:00:00~7/22/2025 0:00:00
383815~"CDER"~"NDA"~217188.00~"PFIZER INC"~"Paxlovid (nirmatrelvir and ritonavir)"~5/25/2023 0:00:00~"Original"~1~"4392-8"~"PMC"~"506B PMC"~4392.00~8~"PMC 4392-8: Submit the final study report with datasets for the ongoing trial,  A Phase 1, Open-Label Study To Evaluate The Pharmacokinetics, Safety, And Tolerability Of Orally Administered Nirmatrelvir/Ritonavir In Pregnant Women With Mild-To-Moderate COVID-19  (Study C4671035; NCT05386472)."~"Delayed"~"The trial completion and final report milestones were missed.Acknowledge Revised PMC Milestones letter was issued on 11/20/2024."~12/31/2024 0:00:00~7/22/2025 0:00:00
383816~"CDER"~"NDA"~217188.00~"PFIZER INC"~"Paxlovid (nirmatrelvir and ritonavir)"~5/25/2023 0:00:00~"Original"~1~"4392-10"~"PMC"~"506B PMC"~4392.00~10~"PMC 4392-10: Conduct an observational study to evaluate pregnancy and infant outcomes following exposure to PAXLOVID during pregnancy."~"Ongoing"~~7/31/2028 0:00:00~7/22/2025 0:00:00
383817~"CDER"~"NDA"~217188.00~"PFIZER INC"~"Paxlovid (nirmatrelvir and ritonavir)"~5/25/2023 0:00:00~"Original"~1~"4392-16"~"PMR"~"505 (o)(3)"~4392.00~16~"PMR 4392-16: Conduct a study to monitor genomic database(s) for the emergence of SARS-CoV-2 variants with amino acid polymorphisms in Mpro or Mpro cleavage sites. Conduct surveillance activities on at least a monthly basis. Conduct phenotypic analysis for any Mpro polymorphisms that are detected at a frequency =1% either globally or in the U.S. for any single month. Conduct phenotypic analysis for any Mpro cleavage site polymorphisms that are detected at a frequency =5% either globally or in the U.S. for any single month. These surveillance activities should continue for a period of 3 years post-approval, with re-assessment of the duration, frequency of reporting and additional protocol methods to occur on an annual basis."~"Ongoing"~~7/31/2026 0:00:00~7/22/2025 0:00:00
383818~"CDER"~"NDA"~217202.00~"AOP HEALTH US LLC"~"Rapiblyk (landiolol) for Injection"~11/22/2024 0:00:00~"Original"~1~"4710-1"~"PMR"~"505 (o)(3)"~4710.00~1~"PMR 4710-1: Conduct an in vitro assessment to evaluate the potential of landiolol and its M1 metabolite to inhibit drug transporters including P-glycoprotein (Pgp), breast cancer resistance protein (BCRP), organic anion transporting polypeptide (OATP) 1B1, OATP1B3, organic cation transporter (OCT) 2, multidrug and toxin extrusion (MATE) proteins (MATE1, MATE2-K), organic anion transporter (OAT) 1, and OAT3. Additional in vivo studies may be needed depending on the results of the in vitro assessment."~"Pending"~~8/31/2025 0:00:00~12/11/2025 0:00:00
383819~"CDER"~"NDA"~217202.00~"AOP HEALTH US LLC"~"Rapiblyk (landiolol) for Injection"~11/22/2024 0:00:00~"Original"~1~"4710-2"~"PMR"~"Pediatric Research Equity Act"~4710.00~2~"PMR 4710-2: Multicenter, Open-label Study to Investigate the Effectiveness and Safety of AOP Landiolol in Controlling Supraventricular Tachycardia in Pediatric Patients (LANDIPED)."~"Fulfilled"~"Per FDA letter dated 02/13/2026, this PMR/PMC has been fulfilled."~12/31/2024 0:00:00~12/11/2025 0:00:00
383820~"CDER"~"NDA"~217225.00~"ASTELLAS PHARMA US INC"~"Izervay (avacincaptad pegol)"~8/4/2023 0:00:00~"Original"~1~"4477-2"~"PMC"~"506B PMC"~4477.00~2~"PMC 4477-2: Submit 18 Month long-term safety data from ISEE2009, an open-label extension trial to assess the safety of intravitreal administration of avacincaptad pegol in approximately 230 patients with geographic atrophy who completed ISEE2008 (GATHER2) through Month 24."~"Fulfilled"~~8/31/2025 0:00:00~10/2/2025 0:00:00
383821~"CDER"~"NDA"~217242.00~"ARCUTIS BIOTHERAPEUTICS INC"~"Zoryve (roflumilast)"~12/15/2023 0:00:00~"Supplement"~5~"4838-1"~"PMR"~"Pediatric Research Equity Act"~4838.00~1~"PMR 4838-1: Conduct an open-label, maximal-use PK, safety and tolerability study in pediatric subjects aged 4 to <12 years old with plaque psoriasis of the scalp and body targeting at least 16 evaluable subjects in whom PK assessments will be performed under maximal usage conditions."~"Ongoing"~"The study enrollment was initiated and is ongoing."~10/31/2027 0:00:00~2/13/2026 0:00:00
383822~"CDER"~"NDA"~212320.00~"SHIELD TX UK LTD"~"Accrufer (ferric maltol)"~7/25/2019 0:00:00~"Original"~1~"3667-3"~"PMR"~"Pediatric Research Equity Act"~3667.00~3~"PMR 3667-3: Conduct and submit the study report and datasets from an open-label comparative efficacy and safety study of ST10 and oral ferrous sulfate in infants and children aged 1 month to 17 years with iron deficiency anemia (Study ST10-01-305). Data from PK and PD studies should be utilized to select the doses of ferric maltol for Study ST10-01-305."~"Submitted"~"The final report was submitted to FDA on 06/23/2025."~7/31/2025 0:00:00~9/5/2025 0:00:00
383823~"CDER"~"NDA"~212320.00~"SHIELD TX UK LTD"~"Accrufer (ferric maltol)"~7/25/2019 0:00:00~"Original"~1~"3667-4"~"PMR"~"Pediatric Research Equity Act"~3667.00~4~"PMR 3667-4: Develop a pediatric age appropriate oral formulation and conduct a study to evaluate the bioavailability relative to the capsule formulation in the fasted condition. Subjects should undergo serial blood sampling for serum iron concentration at scheduled intervals during each treatment period. Submit the protocol for review and concurrence prior to commencing."~"Fulfilled"~"Per FDA letter dated 01/13/2026, this PMR/PMC has been fulfilled."~3/31/2021 0:00:00~9/5/2025 0:00:00
383824~"CDER"~"NDA"~212320.00~"SHIELD TX UK LTD"~"Accrufer (ferric maltol)"~7/25/2019 0:00:00~"Original"~1~"3667-5"~"PMR"~"Pediatric Research Equity Act"~3667.00~5~"PMR 3667-5: Evaluate the effect of food on the bioavailability of the pediatric age appropriate oral formulation in healthy adults. Subjects should undergo serial blood sampling for serum iron concentration at scheduled intervals during each treatment period. Submit the protocol for review and concurrence prior to commencing."~"Fulfilled"~"Per FDA letter dated 01/13/2026, this PMR/PMC has been fulfilled."~3/31/2021 0:00:00~9/5/2025 0:00:00
383825~"CDER"~"NDA"~212327.00~"BRISTOL-MYERS SQUIBB CO"~"INREBIC  (Fedratinib Hydrochloride)"~8/16/2019 0:00:00~"Original"~1~"3664-1"~"PMR"~"505 (o)(3)"~3664.00~1~"PMR 3664-1: Conduct a randomized, concurrently controlled clinical trial comparing fedratinib 400 mg once daily to best available therapy in patients with DIPSS-intermediate-2 or high-risk primary myelofibrosis, postpolycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis and previously treated with ruxolitinib. The trial will enroll a sufficient number of patients to ensure that at least 150 subjects are treated with at least 6 cycles of fedratinib and are followed for at least 3 years from first dose, or until death. The protocol should include measures to assess and manage adverse events of nausea, diarrhea, vomiting, thiamine deficiency, and encephalopathy at baseline and during the trial. The final protocol must be agreed upon with the Agency."~"Delayed"~"The Final Report milestone was missed because of the COVID19 pandemic and the ongoing conflict between Russia and Ukraine. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 11/17/2025."~6/30/2025 0:00:00~10/10/2025 0:00:00
383826~"CDER"~"NDA"~212576.00~"TAIHO ONCOLOGY INC"~"INQOVI (decitabine and cedazuridine)"~7/7/2020 0:00:00~"Original"~1~"3848-1"~"PMR"~"505 (o)(3)"~3848.00~1~"PMR 3848-1: Submit the final report with datasets from a thorough QT/QTc study to evaluate the effect of cedazuridine on QT interval prolongation that may further inform labelling. Design and conduct the trial in accordance with the ICH E14 guidances for industry titled; E14 Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for NonAntiarrhythmic Drugs, and E14 Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non-Antiarrhythmic Drugs -Questions and Answers (R3)."~"Fulfilled"~~6/30/2022 0:00:00~9/3/2025 0:00:00
383827~"CDER"~"NDA"~212576.00~"TAIHO ONCOLOGY INC"~"INQOVI (decitabine and cedazuridine)"~7/7/2020 0:00:00~"Original"~1~"3848-2"~"PMR"~"505 (o)(3)"~3848.00~2~"PMR 3848-2: Submit the final report and datasets from a clinical pharmacokinetic trial to determine an appropriate starting dose of INQOVI in patients with moderate and severe hepatic impairment that may inform product labeling. Design and conduct the trial in accordance with the FDA Guidance for Industry titled; Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling.
"~"Delayed"~"The final report milestone was missed because of challenges with enrollment. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 12/15/2025."~6/30/2025 0:00:00~9/3/2025 0:00:00
383828~"CDER"~"NDA"~212576.00~"TAIHO ONCOLOGY INC"~"INQOVI (decitabine and cedazuridine)"~7/7/2020 0:00:00~"Original"~1~"3848-3"~"PMR"~"505 (o)(3)"~3848.00~3~"PMR 3848-3: Conduct a clinical pharmacokinetic trial to determine an appropriate dose of INQOVI that will minimize toxicity in patients with severe renal impairment and/or end-stage renal disease. Design and conduct the trial in accordance with the FDA Guidance for Industry titled, Pharmacokinetics in Patients with Impaired Renal Function: Study Design, Data Analysis, and Impact on Dosing and Labeling.
"~"Delayed"~"The final report milestone was missed because of challenges with enrollment. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 12/15/2025."~6/30/2025 0:00:00~9/3/2025 0:00:00
383829~"CDER"~"NDA"~212576.00~"TAIHO ONCOLOGY INC"~"INQOVI (decitabine and cedazuridine)"~7/7/2020 0:00:00~"Original"~1~"3848-4"~"PMC"~"506B PMC"~3848.00~4~"PMC 3848-4: Submit the final report and datasets from a clinical pharmacokinetic trial to determine the effect of food on decitabine and cedazuridine exposure in patients when administered with INQOVI that may inform product labeling. Design and conduct the trial in accordance with the FDA Guidance for Industry titled; Food-Effect Bioavailability and Fed Bioequivalence Studies. This study can be an add-on to an ongoing or planned clinical study with patients receiving INQOVI with food on one day at steady state in a single cycle."~"Fulfilled"~~12/31/2022 0:00:00~9/3/2025 0:00:00
383830~"CDER"~"NDA"~212608.00~"BLUEPRINT MEDICINES CORP"~"Ayvakit (avapritinib)"~1/9/2020 0:00:00~"Supplement"~6~"4101-3"~"PMC"~"506B PMC"~4101.00~3~"PMC 4101-3: Complete Study BLU-285-2202,  An open-label, single-arm, Phase 2 study to evaluate efficacy and safety of avapritinib (BLU-285), a selective KIT mutation-targeted tyrosine kinase inhibitor, in patients with advanced systemic mastocytosis . Include an updated summary of safety, efficacy analyses, and datasets at the time of final clinical study report submission."~"Ongoing"~~12/31/2026 0:00:00~3/9/2026 0:00:00
383831~"CDER"~"NDA"~214012.00~"NOVARTIS PHARMACEUTICALS CORP"~"Leqvio (inclisiran)"~12/22/2021 0:00:00~"Original"~1~"4186-2"~"PMR"~"Pediatric Research Equity Act"~4186.00~2~"PMR 4186-2: Conduct a two-part (double-blind inclisiran versus placebo [Year 1] followed by open-label with placebo-treated subjects switched to inclisiran [Year 2]), multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in children [aged 6-7 (if on plasma apheresis) or 8 to <12 years] with heterozygous familial hypercholesterolemia (HeFH)."~"Delayed"~"The Draft and Final Protocol Submission milestones were
missed because of a delay in the one-year interim data
from the ORION-16 adolescent study. The Final Protocol
milestone was revised and has been met. 37 of 51 enrolled."~12/31/2027 0:00:00~2/17/2026 0:00:00
383832~"CDER"~"NDA"~214012.00~"NOVARTIS PHARMACEUTICALS CORP"~"Leqvio (inclisiran)"~12/22/2021 0:00:00~"Original"~1~"4186-3"~"PMR"~"505 (o)(3)"~4186.00~3~"PMR 4186-3: Conduct a worldwide, descriptive study that collects prospective and retrospective data in women exposed to Leqvio during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~7/31/2031 0:00:00~2/17/2026 0:00:00
383833~"CDER"~"NDA"~214018.00~"SENTYNL THERAPEUTICS INC"~"Nulibry (fosdenopterin)"~2/26/2021 0:00:00~"Original"~1~"4032-2"~"PMR"~"505 (o)(3)"~4032.00~2~"PMR 4032-2: Embryo-fetal development study in rabbits with fosdenopterin HBr"~"Fulfilled"~~1/31/2027 0:00:00~4/27/2026 0:00:00
383834~"CDER"~"NDA"~214018.00~"SENTYNL THERAPEUTICS INC"~"Nulibry (fosdenopterin)"~2/26/2021 0:00:00~"Original"~1~"4032-4"~"PMR"~"505 (o)(3)"~4032.00~4~"PMR 4032-4: A 104-week carcinogenicity study in CD-1 mice with fosdenopterin HBr."~"Delayed"~"The final protocol and study completion milestones were missed. FDA determined that there was good cause for the delay and acknowledged the revised milestone(s) in a letter dated 8-5-2025. "~7/31/2026 0:00:00~4/27/2026 0:00:00
383835~"CDER"~"NDA"~214044.00~"ROSEMONT PHARMACEUTICALS LLC"~"QDOLO (tramadol HCl)"~9/1/2020 0:00:00~"Original"~1~"3918-1"~"PMR"~"Pediatric Research Equity Act"~3918.00~1~"PMR 3918-1: Conduct a juvenile animal toxicology study to characterize the impact of tramadol on brain development, to support pediatric dosing in adolescent patients 12 to less than 17 years of age."~"Delayed"~"The study completion and final report milestones were missed. A Deferral Extension requested 7/3/2025, denied per FDA letter dated 8/15/2025. 
"~4/30/2025 0:00:00~10/31/2025 0:00:00
383836~"CDER"~"NDA"~214044.00~"ROSEMONT PHARMACEUTICALS LLC"~"QDOLO (tramadol HCl)"~9/1/2020 0:00:00~"Original"~1~"3918-2"~"PMR"~"Pediatric Research Equity Act"~3918.00~2~"PMR 3918-2: Conduct a randomized, double-blind, placebo-controlled study evaluating the clinical effectiveness, safety, and pharmacokinetic profiles of QDOLO (tramadol hydrochloride) oral solution in pediatric patients 12 to less than 17 years of age, outside of the contraindicated setting of tonsillectomy and/or adenoidectomy."~"Delayed"~"The study completion final report milestones were missed. A Deferral Extension requested 6/6/2025, denied per FDA letter dated 7/21/2025."~8/31/2024 0:00:00~10/31/2025 0:00:00
383837~"CDER"~"NDA"~214056.00~"MDD US OPERATIONS LLC A SUB OF SUPERNUS PHARMACEUTICALS INC"~"Onapgo (apomorphine hydrochloride)"~2/3/2025 0:00:00~"Original"~1~"4781-1"~"PMR"~"505 (o)(3)"~4781.00~1~"PMR 4781-1: Conduct a new leachable study using at least three batches of Onapgo (apomorphine hydrochloride) injection. The leachables should be tested at multiple time points on stability storage -from release through the proposed shelf-life."~"Pending"~~5/31/2028 0:00:00~4/1/2026 0:00:00
383838~"CDER"~"NDA"~214096.00~"EMD SERONO INC"~"Tepmetko (tepotinib)"~2/3/2021 0:00:00~"Original"~1~"4013-4"~"PMC"~"506B PMC"~4013.00~4~"PMC 4013-4: Submit a summary of the final report of an analytical and clinical validation study, using clinical trial data, that is adequate to support labeling of an in vitro diagnostic device that demonstrates the device is essential to the safe and effective use of tepotinib for patients diagnosed with non-small cell lung cancer, whose tumors harbor MET exon 14 skipping. The results of the validation study may inform product labeling."~"Delayed"~"The Final Report Submission milestone was missed due to diagnostic partnership changes."~1/31/2022 0:00:00~3/25/2026 0:00:00
383839~"CDER"~"NDA"~214121.00~"ACCORD HEALTHCARE INC"~"Methotrexate "~8/24/2020 0:00:00~"Original"~1~"3927-1"~"PMC"~"506B PMC"~3927.00~1~"PMC 3927-1: Monitor medication errors and submit a summary of all reports associated with Methotrexate Injection 100 mg/mL concentration (e.g., dosing errors, selection errors, etc.) for 3 years. Include a review of medication error reports, (preferable in a PDF file) and a line by line detail of case narratives (preferable in an Excel file) to the Agency every 6 months."~"Submitted"~~12/31/2024 0:00:00~11/7/2025 0:00:00
383840~"CDER"~"NDA"~214154.00~"MAYNE PHARMA LLC"~"Nextstellis (Drospirenone and Estetrol)"~4/15/2021 0:00:00~"Original"~1~"4054-1"~"PMR"~"505 (o)(3)"~4054.00~1~"PMR 4054-1: Conduct an Electronic Health Record (EHR) or primary data collection observational cohort study assessing the risks for fatal and non-fatal venous thromboembolism (VTE) and arterial thromboembolism (ATE) in new users of estetrol monohydrate (E4) and drospirenone (DRSP). Conduct co-primary comparisons of new users of E4/DRSP vs non-DRSP containing combined oral contraceptives (COCs) and E4/DRSP vs other DRSP-containing products among women of reproductive age using COCs primarily for contraceptive reasons. U.S. patients will account for at least 50% of the study population and adequate numbers of obese women should be included for analysis. The study should adequately control for smoking and body mass index and be
sufficiently powered to detect a 1.5 to 2.0-fold increase in risk of VTE for both the E4/DRSP vs. other non-DRSP COCs and other DRSP-containing COCs analyses."~"Delayed"~"The Final Protocol was submitted after the original milestone and acknowledged in a letter issued on 9/13/24. An Acknowledge Revised Milestone letter issued on 11/3/23."~6/30/2027 0:00:00~6/12/2026 0:00:00
383841~"CDER"~"NDA"~214155.00~"REMPEX PHARMACEUTICALS INC A WHOLLY OWNED SUB OF MELINTA THERAPEUTICS LLC"~"Kimyrsa (oritavancin)"~3/12/2021 0:00:00~"Original"~1~"4036-1"~"PMR"~"Pediatric Research Equity Act"~4036.00~1~"PMR 4036-1: Conduct an open-label, dose-finding, pharmacokinetics, safety and tolerability study of oritavancin diphosphate single-dose infusion in pediatric subjects less than 18 years of age with suspected or confirmed bacterial infections."~"Submitted"~"The final report was submitted to FDA on 05/19/2026."~6/30/2025 0:00:00~4/28/2026 0:00:00
383842~"CDER"~"NDA"~214155.00~"REMPEX PHARMACEUTICALS INC A WHOLLY OWNED SUB OF MELINTA THERAPEUTICS LLC"~"Kimyrsa (oritavancin)"~3/12/2021 0:00:00~"Original"~1~"4036-4"~"PMR"~"Pediatric Research Equity Act"~4036.00~4~"PMR 4036-4: Conduct a multicenter, open-label study to evaluate the safety and tolerability of single-dose IV oritavancin diphosphate for the treatment of pediatric subjects less than 18 years of age with acute bacterial skin and skin structure infections."~"Ongoing"~"Final report is due 7-2026."~7/31/2026 0:00:00~4/28/2026 0:00:00
383843~"CDER"~"NDA"~215431.00~"AXSOME THERAPEUTICS INC"~"Symbravo (meloxicam and rizatriptan) tablets"~1/30/2025 0:00:00~"Original"~1~"4782-1"~"PMR"~"Pediatric Research Equity Act"~4782.00~1~"PMR 4782-1: A randomized, double-blind, placebo-controlled efficacy and safety study to evaluate Symbravo for the acute treatment of migraine in pediatric patients ages 6 to less than 18 years. This study should include an initial blinded placebo run-in period to identify placebo nonresponders for enrollment into the efficacy portion of the study. This efficacy study must be designed to show superiority of Symbravo over placebo."~"Delayed"~"Original Final Report Due Date: 08/31/2029; Deferral Extension granted per FDA letter dated 04/13/2026. The draft protocol, final protocol submission and study completion milestones were also revised.
"~7/31/2030 0:00:00~3/18/2026 0:00:00
383844~"CDER"~"NDA"~215431.00~"AXSOME THERAPEUTICS INC"~"Symbravo (meloxicam and rizatriptan) tablets"~1/30/2025 0:00:00~"Original"~1~"4782-2"~"PMR"~"Pediatric Research Equity Act"~4782.00~2~"PMR 4782-2: A long-term open-label safety study in pediatric patients ages 6 years to less than 18 years. The long-term safety study must provide a descriptive analysis of safety data in at least 200 pediatric patients treated with Symbravo for a duration of at least 6 months and 75 pediatric patients treated with Symbravo for 12 months, treating on average at least one migraine attack per month, at doses evaluated in the efficacy study."~"Delayed"~"Original Final Report Due Date: 08/31/2029; Deferral Extension granted per FDA letter dated 04/13/2026. The draft protocol, final protocol submission and study completion milestones were also revised."~7/31/2030 0:00:00~3/18/2026 0:00:00
383845~"CDER"~"NDA"~215431.00~"AXSOME THERAPEUTICS INC"~"Symbravo (meloxicam and rizatriptan) tablets"~1/30/2025 0:00:00~"Original"~1~"4782-3"~"PMR"~"505 (o)(3)"~4782.00~3~"PMR 4782-3: A prospective pregnancy exposure registry cohort study in the United States that compares the maternal, fetal, and infant outcomes of women with migraine exposed to Symbravo during pregnancy with two unexposed control populations: one consisting of women with migraine who have not been exposed to Symbravo before and during pregnancy and the other consisting of women without migraine. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The draft protocol was submitted after the original draft
protocol submission milestone."~9/30/2040 0:00:00~3/18/2026 0:00:00
383846~"CDER"~"NDA"~215431.00~"AXSOME THERAPEUTICS INC"~"Symbravo (meloxicam and rizatriptan) tablets"~1/30/2025 0:00:00~"Original"~1~"4782-4"~"PMR"~"505 (o)(3)"~4782.00~4~"PMR 4782-4: A pregnancy outcomes study using a different study design than provided for in PMR 4782-3 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case-control study) to assess pregnancy complications, major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Symbravo during pregnancy compared to an unexposed control population."~"Delayed"~"The draft protocol was submitted after the original draft protocol submission milestone."~9/30/2034 0:00:00~3/18/2026 0:00:00
383847~"CDER"~"NDA"~215457.00~"KALEO INC"~"Naloxone Hydrochloride Injection"~2/28/2022 0:00:00~"Original"~1~"4228-1"~"PMR"~"Pediatric Research Equity Act"~4228.00~1~"PMR 4228-1: Conduct allometric scaling of PK/PD models of naloxone-opioid interaction to establish the minimal effective dose of naloxone HCl required to reverse respiratory depression induced by fentanyl and carfentanil in pediatric patients from birth to less than 12 years of age."~"Submitted"~"The final report was submitted to FDA on 12/8/2022."~4/30/2023 0:00:00~4/22/2026 0:00:00
383848~"CDER"~"NDA"~215457.00~"KALEO INC"~"Naloxone Hydrochloride Injection"~2/28/2022 0:00:00~"Original"~1~"4228-2"~"PMR"~"Pediatric Research Equity Act"~4228.00~2~"PMR 4228-2: Conduct a juvenile animal toxicology study in rats to support clinical dosing in pediatric patients from birth to less than 12 years of age. This study will evaluate the effects of naloxone on the developing central nervous system, endocrine, and reproductive system."~"Delayed"~"Original Final Report Due Date: 02/28/2024; Per FDA letter dated 05/30/2024, final report due date extended to 07/31/2026. Second Deferral Extension Granted Per FDA letter dated 06/08/2026. The study completion milestone was also revised."~7/31/2027 0:00:00~4/22/2026 0:00:00
383849~"CDER"~"NDA"~215457.00~"KALEO INC"~"Naloxone Hydrochloride Injection"~2/28/2022 0:00:00~"Original"~1~"4228-6"~"PMR"~"505 (o)(3)"~4228.00~6~"PMR 4228-6: Perform structural identification of the specified unknown impurities observed at relative retention time (RRT) of [...]. The chemical structures of these impurities should be confirmed using physical and chemical techniques such as elemental analysis, mass spectrometry (MS), nuclear magnetic resonance (NMR) spectroscopy, ultraviolet (UV) spectroscopy, infrared (IR) spectroscopy, Xray crystallography, and other tests (e.g., functional group analysis, derivatization, complex formation)."~"Submitted"~~9/30/2023 0:00:00~4/22/2026 0:00:00
383850~"CDER"~"NDA"~215457.00~"KALEO INC"~"Naloxone Hydrochloride Injection"~2/28/2022 0:00:00~"Original"~1~"4228-7"~"PMR"~"505 (o)(3)"~4228.00~7~"PMR 4228-7: Conduct a study in order to provide product stability and leachables data through the proposed shelf-life for three batches of to-be-marketed (TBM) drug product stored at both inverted and horizontal orientations. Manufacture these batches at the commercial site and collect stability data including leachables determination at multiple stability time-points per the testing frequency recommended in ICH Q1A(R2)."~"Ongoing"~~9/30/2026 0:00:00~4/22/2026 0:00:00
383851~"CDER"~"NDA"~215487.00~"SCIENTURE LLC"~"Rezenopy (naloxone hydrochloride)"~4/19/2024 0:00:00~"Original"~1~"4440-1"~"PMR"~"505 (o)(3)"~4440.00~1~"PMR 4440-1: Conduct a GLP repeat-dose intranasal toxicology study of at least 14 days duration in a single species to adequately characterize the toxicological potential of the naloxone degradant, [..], to local tissues."~"Pending"~~12/31/2025 0:00:00~6/11/2026 0:00:00
383852~"CDER"~"NDA"~215498.00~"IPSEN BIOPHARMACEUTICALS INC"~"Bylvay (Odevixibat)"~7/20/2021 0:00:00~"Original"~1~"4109-1"~"PMR"~"505 (o)(3)"~4109.00~1~"PMR 4109-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in females of child-bearing potential who are prescribed BYLVAY (odevixibat) during pregnancy and/or lactation to assess risk of maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The study will collect  information for a minimum of 10 years."~"Delayed"~"Acknowledge revised milestone issued 06/13/2022, Acknowledge final protocol 11/23/2022, Interim report received 4/10/2025."~1/31/2033 0:00:00~9/17/2025 0:00:00
383853~"CDER"~"NDA"~215430.00~"AXSOME THERAPEUTICS INC"~"Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride)"~8/18/2022 0:00:00~"Original"~1~"4233-12"~"PMC"~"506B PMC"~4233.00~12~"PMC 4233-12: Conduct a bupropion-controlled, randomized withdrawal study to demonstrate whether the dextromethorphan component of AXS-05 contributes to the long-term efficacy of AXS-05 for the treatment of MDD."~"Delayed"~"The final protocol submission milestone has been missed. The  applicant sent Draft Protocol Amendment 3 on 8/13/25, seeking feedback."~4/30/2024 0:00:00~9/16/2025 0:00:00
383854~"CDER"~"NDA"~217779.00~"GERON CORP"~"Rytelo (imetelstat)"~6/6/2024 0:00:00~"Original"~1~"4631-3"~"PMR"~"505 (o)(3)"~4631.00~3~"PMR 4631-3: Conduct a rodent carcinogenicity study in rats to evaluate the potential for carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Pending"~~8/31/2031 0:00:00~8/5/2025 0:00:00
383855~"CDER"~"NDA"~217779.00~"GERON CORP"~"Rytelo (imetelstat)"~6/6/2024 0:00:00~"Original"~1~"4631-4"~"PMR"~"505 (o)(3)"~4631.00~4~"PMR 4631-4: Complete the randomized part of trial MDS3001 to assess the long-term safety of imetelstat including the incidence of known serious risks of neutropenia, thrombocytopenia, infections, and bleeding in adults with transfusion-dependent lower-risk MDS. Include an integrated report to summarize the safety, overall survival, and efficacy when all patients on the Phase 3 Study MDS3001 have completed at least 5 years of treatment with imetelstat or have 3 years of post-treatment follow-up from the last dose of study treatment, whichever occurs later, or have withdrawn from the study, to demonstrate long term safety of imetelstat."~"Ongoing"~~4/30/2027 0:00:00~8/5/2025 0:00:00
383856~"CDER"~"NDA"~217779.00~"GERON CORP"~"Rytelo (imetelstat)"~6/6/2024 0:00:00~"Original"~1~"4631-5"~"PMR"~"505 (o)(3)"~4631.00~5~"PMR 4631-5: To potentially minimize the serious risks of imetelstat treatment including neutropenia and thrombocytopenia and other serious adverse events and improve tolerability, conduct a randomized trial comparing at least 2 dosages of imetelstat in patients with low or intermediate-1 risk myelodysplastic syndromes (LR-MDS) to evaluate alternative dosing regimens such as different dose levels, dosing frequencies, and duration of treatment. The study should include sufficient clinical pharmacokinetic sampling to analyze the exposure-response (ER) relationships. Update the current population pharmacokinetics (PopPK) and ER analysis with this clinical trial data. The trial should enroll sufficient representation of U.S. racial and ethnic minorities to ensure that the results are reflective of the U.S. population. Additionally, the study should follow subjects for a sufficient time after end of dosing to establish the time required to return to baseline grade for neutrophil and platelet count, in the absence of disease progression or subsequent therapy."~"Delayed"~"Per FDA letter dated 12/17/2025, the applicant s request for release was denied."~6/30/2031 0:00:00~8/5/2025 0:00:00
383857~"CDER"~"NDA"~217779.00~"GERON CORP"~"Rytelo (imetelstat)"~6/6/2024 0:00:00~"Original"~1~"4631-6"~"PMR"~"505 (o)(3)"~4631.00~6~"PMR 4631-6: Conduct a QT assessment and include results from the QT substudy under the Phase 3 portion of Study MDS3001, to further assess and characterize signals of serious risk of QT prolongation with use of imetelstat in patients with low or intermediate-1 risk myelodysplastic syndromes (LR-MDS)."~"Fulfilled"~~10/31/2024 0:00:00~8/5/2025 0:00:00
383858~"CDER"~"NDA"~217779.00~"GERON CORP"~"Rytelo (imetelstat)"~6/6/2024 0:00:00~"Original"~1~"4631-7"~"PMC"~"506B PMC"~4631.00~7~"PMC 4631-7: Conduct exploratory analyses aimed at identifying predictors of response to imetelstat using patient samples for cytogenetic and mutational analyses collected at baseline from patients treated with imetelstat in Study MDS3001. Conduct an analysis to characterize genetic markers, demographics, or other characteristics that predict long term transfusion independence (at least >24 weeks and >1 year) to imetelstat."~"Ongoing"~~4/30/2027 0:00:00~8/5/2025 0:00:00
383859~"CDER"~"NDA"~217785.00~"MADRIGAL PHARMACEUTICALS INC"~"Rezdiffra"~3/14/2024 0:00:00~"Original"~1~"4577-1"~"PMR"~"Accelerated Approval"~4577.00~1~"PMR 4577-1: Complete Trial MGL-3196-11, a randomized, double-blind, placebocontrolled 54-month trial in patients with non-alcoholic steatohepatitis (NASH) and liver fibrosis to demonstrate clinical benefit on the composite endpoint of progression to cirrhosis, hepatic decompensation events, liver transplant, and mortality."~"Ongoing"~"As per the 180-day AA PMR Progress Report
submitted on 05/13/2025, the trial is progressing as
per the original schedule of milestones. There are
currently 1759 subjects enrolled, higher than the
planned 1700; enrollment is complete. This report
was found to be complete."~3/31/2029 0:00:00~5/8/2026 0:00:00
383860~"CDER"~"NDA"~217785.00~"MADRIGAL PHARMACEUTICALS INC"~"Rezdiffra"~3/14/2024 0:00:00~"Original"~1~"4577-2"~"PMR"~"Pediatric Research Equity Act"~4577.00~2~"PMR 4577-2: Conduct a study of the safety, pharmacokinetics, and efficacy of REZDIFFRA in post-pubertal pediatric patients ages 12 to 17 years with non-alcoholic steatohepatitis (NASH) with stage F2 and F3 fibrosis."~"Pending"~"FDA issued a Deferral Extension granted 5/2/2025 to pause conducting pediatric NASH trials until clinical benefit is confirmed in adult NASH. Draft Protocol Submission: 11/2029 (revised date) Final Protocol Submission: 07/2030 (revised date) Trial Completion: 01/2033 (revised date) Final Report Submission: 07/2033 (deferral extension date).
"~7/31/2033 0:00:00~5/8/2026 0:00:00
383861~"CDER"~"NDA"~217785.00~"MADRIGAL PHARMACEUTICALS INC"~"Rezdiffra"~3/14/2024 0:00:00~"Original"~1~"4577-3"~"PMR"~"Pediatric Research Equity Act"~4577.00~3~"PMR 4577-3: Conduct a study of the safety, pharmacokinetics, and efficacy of REZDIFFRA in pre-pubertal pediatric patients ages 6 to 12 years with non-alcoholic steatohepatitis (NASH) with stage F2 and F3 fibrosis."~"Pending"~"FDA issued a Deferral Extension granted 5/2/2025 to pause conducting pediatric NASH trials until clinical benefit is confirmed in adult NASH, and the adolescent trial establishes safety. Draft Protocol Submission: 03/2034 (revised date) Final Protocol Submission: 11/2034 (revised date) Trial Completion: 05/2037 (revised date) Final Report Submission: 11/2037 (deferral extension date)."~11/30/2037 0:00:00~5/8/2026 0:00:00
383862~"CDER"~"NDA"~217785.00~"MADRIGAL PHARMACEUTICALS INC"~"Rezdiffra"~3/14/2024 0:00:00~"Original"~1~"4577-4"~"PMR"~"505 (o)(3)"~4577.00~4~"PMR 4577-4: Conduct a worldwide descriptive study that collects prospective and retrospective data from women exposed to resmetirom during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes should be assessed through the first year of life. The minimum number of patients should be specified in the protocol."~"Ongoing"~~9/30/2030 0:00:00~5/8/2026 0:00:00
383863~"CDER"~"NDA"~217785.00~"MADRIGAL PHARMACEUTICALS INC"~"Rezdiffra"~3/14/2024 0:00:00~"Original"~1~"4577-5"~"PMR"~"505 (o)(3)"~4577.00~5~"PMR 4577-5: Perform a lactation study (milk only) in lactating women who have received resmetirom to measure concentrations of resmetirom in breast milk using a validated assay and to assess any reported adverse effects on the breastfed infant."~"Ongoing"~~9/30/2027 0:00:00~5/8/2026 0:00:00
383864~"CDER"~"NDA"~217785.00~"MADRIGAL PHARMACEUTICALS INC"~"Rezdiffra"~3/14/2024 0:00:00~"Original"~1~"4577-6"~"PMR"~"505 (o)(3)"~4577.00~6~"PMR 4577-6: Conduct a clinical study to evaluate the effects of severe renal impairment on the pharmacokinetics of resmetirom and its major metabolite(s). Design and conduct the trial in accordance with the draft guidance for industry, Pharmacokinetics in Patients with Impaired Renal Function   Study Design, Data Analysis, and Impact on Dosing."~"Fulfilled"~~6/30/2025 0:00:00~5/8/2026 0:00:00
383865~"CDER"~"NDA"~217785.00~"MADRIGAL PHARMACEUTICALS INC"~"Rezdiffra"~3/14/2024 0:00:00~"Original"~1~"4577-7"~"PMC"~"506B PMC"~4577.00~7~"PMC 4577-7: Conduct a clinical drug-drug interaction study to evaluate the effects of resmetirom on the pharmacokinetics of a sensitive substrate of Breast Cancer Resistance Protein (BCRP). Design and conduct the trial in accordance with the Guidance for Industry: Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions."~"Fulfilled"~~7/31/2025 0:00:00~5/8/2026 0:00:00
383866~"CDER"~"NDA"~212725.00~"GENENTECH INC"~"Rozlytrek (Entrectinib)"~8/15/2019 0:00:00~"Original"~1~"3689-2"~"PMR"~"Accelerated Approval"~3689.00~2~"PMR 3689-2: Submit the final report, including datasets, from ongoing and proposed trials conducted to verify and describe the clinical benefit of entrectinib, through more precise estimation of the overall response rate and mature response duration per independent review assessment, in adult and pediatric patients 12 years of age and older with solid tumors with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion and without a known acquired resistance mutation; are metastatic or would require surgical resection that would result in severe morbidity; and have no satisfactory alternative treatment or that have progressed following treatment. A sufficient number of patients will be evaluated to more precisely characterize response and durability of response for each of the following tumor types: pediatric solid tumors, colorectal cancer, central nervous system cancers, gynecological cancers, and melanoma. A minimum of 40 patients with cancers other than pediatric solid tumors, colorectal cancer, central nervous system cancers, gynecological cancers, melanoma, soft tissue sarcoma, non-small cell adenocarcinoma lung cancer, mammary analogue secretory carcinoma, and secretory breast cancer will also be studied. Overall response rate and duration of response will be assessed by independent central review and all responding patients will be followed for at least 12 months from the onset of response."~"Ongoing"~"The trial is underway. The trial is underway. Per the applicant's 180 day status report, the trial continues to enroll patients and is on track to meet its agreed milestone dates."~3/31/2027 0:00:00~10/9/2025 0:00:00
383867~"CDER"~"NDA"~212725.00~"GENENTECH INC"~"Rozlytrek (Entrectinib)"~8/15/2019 0:00:00~"Original"~1~"3686-3"~"PMR"~"505 (o)(3)"~3686.00~3~"PMR 3686-3: Submit integrated safety analyses and supporting data from an adequate number of patients enrolled in clinical trial(s) designed to characterize the risk of fractures and its sequelae in patients exposed to entrectinib with reasonable precision; to identify risk factors for development of these sequelae; and to support labeling recommendations to mitigate the risk of skeletal fractures. The design of the trial should include sufficient bone monitoring to achieve these objectives, including but not limited to initial and serial assessment of bone mineral density (BMD) with dual x-ray absorptiometry (DXA) scans, and markers of bone formation, bone resorption, and calcium metabolism."~"Fulfilled"~~3/31/2025 0:00:00~10/9/2025 0:00:00
383868~"CDER"~"NDA"~212725.00~"GENENTECH INC"~"Rozlytrek (Entrectinib)"~8/15/2019 0:00:00~"Original"~1~"3689-5"~"PMR"~"505 (o)(3)"~3689.00~5~"PMR 3689-5: Conduct clinical trial(s) of entrectinib in a sufficient number of pediatric patients 12 years of age and older with NTRK-fusion solid tumors to evaluate the potential serious risk of adverse long-term effects of entrectinib on growth and development, including neurological outcomes with reasonable precision. Patients will be monitored for growth and developmental milestones using age-appropriate screening tools and undergo neurological examination at appropriate intervals. Evaluations will include neurological exams with neurocognitive assessment, Karnofsky/Lansky score, growth as measured by height, weight, height velocity, and height standard deviation scores (SDS), age at adrenarche if applicable (males), age at menarche if applicable (females) and Tanner Stage. Patient monitoring will be performed until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first."~"Ongoing"~~8/31/2029 0:00:00~10/9/2025 0:00:00
383869~"CDER"~"NDA"~212725.00~"GENENTECH INC"~"Rozlytrek (Entrectinib)"~8/15/2019 0:00:00~"Original"~1~"3689-6"~"PMR"~"505 (o)(3)"~3689.00~6~"PMR 3689-6: Submit integrated safety analyses and supporting data from an adequate number of patients enrolled in clinical trial(s) designed to characterize the risk of fractures and its sequelae in patients exposed to entrectinib with reasonable precision; to identify risk factors for development of these sequelae; and to support labeling recommendations to mitigate the risk of skeletal fractures. The design of the trial should include sufficient bone monitoring to achieve these objectives, including but not limited to initial and serial assessment of bone mineral density (BMD) with dual x-ray absorptiometry (DXA) scans, and markers of bone formation, bone resorption, and calcium metabolism."~"Fulfilled"~~3/31/2025 0:00:00~10/9/2025 0:00:00
383870~"CDER"~"NDA"~212725.00~"GENENTECH INC"~"Rozlytrek (Entrectinib)"~8/15/2019 0:00:00~"Supplement"~9~"4533-1"~"PMR"~"Accelerated Approval"~4533.00~1~"PMR 4533-1: Conduct an integrated analysis from ongoing trials intended to verify and describe the clinical benefit of entrectinib, through more precise estimation of the overall response rate and mature response duration per independent review assessment, in a sufficient number of pediatric patients older than 1 month of age and less than 12 years of age with solid tumors with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion and without a known acquired resistance mutation; that are metastatic or would require surgical resection that would result in severe morbidity; and have no satisfactory alternative treatment or have progressed following treatment. Overall response rate and duration of response will be assessed by independent central review and all responding patients will be followed for at least 12 months from the onset of response."~"Ongoing"~"As per the 180-day AA PMR Progress Report submitted on 03/27/2025, the TAPISTRY Trial is progressing as per the original schedule of milestones. The trial is currently fully enrolled."~3/31/2027 0:00:00~10/9/2025 0:00:00
383871~"CDER"~"NDA"~212726.00~"GENENTECH INC"~"Rozlytrek (Entrectinib)"~8/15/2019 0:00:00~"Original"~1~"3689-2"~"PMR"~"Accelerated Approval"~3689.00~2~"PMR 3689-2: Submit the final report, including datasets, from ongoing and proposed trials conducted to verify and describe the clinical benefit of entrectinib, through more precise estimation of the overall response rate and mature response duration per independent review assessment, in adult and pediatric patients 12 years of age and older with solid tumors with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion and without a known acquired resistance mutation; are metastatic or would require surgical resection that would result in severe morbidity; and have no satisfactory alternative treatment or that have progressed following treatment. A sufficient number of patients will be evaluated to more precisely characterize response and durability of response for each of the following tumor types: pediatric solid tumors, colorectal cancer, central nervous system cancers, gynecological cancers, and melanoma. A minimum of 40 patients with cancers other than pediatric solid tumors, colorectal cancer, central nervous system cancers, gynecological cancers, melanoma, soft tissue sarcoma, non-small cell adenocarcinoma lung cancer, mammary analogue secretory carcinoma, and secretory breast cancer will also be studied. Overall response rate and duration of response will be assessed by independent central review and all responding patients will be followed for at least 12 months from the onset of response."~"Ongoing"~"The trial is underway. The trial is underway. Per the applicant's 180 day status report, the trial continues to enroll patients and is on track to meet its agreed milestone dates."~3/31/2027 0:00:00~
384536~"CDER"~"NDA"~209229.00~"BIOCORRX PHARMACEUTICALS INC"~"Lucemyra (lofexidine)"~5/16/2018 0:00:00~"Original"~1~"3391-6"~"PMR"~"505 (o)(3)"~3391.00~6~"PMR 3391-6: Conduct a pharmacokinetic study in the rat to characterize the plasma levels of lofexidine and the lofexidine metabolites LADP (N-(2-aminoethyl)-2-(2,6- dichlorophenoxy)propenamide), LDPA (2-(2,6-dichlorophenoxy)propionic acid), and 2,6-DCP (2,6-dichlorophenol) using a validated assay."~"Submitted"~~7/31/2018 0:00:00~7/15/2026 0:00:00
384537~"CDER"~"NDA"~209229.00~"BIOCORRX PHARMACEUTICALS INC"~"Lucemyra (lofexidine)"~5/16/2018 0:00:00~"Original"~1~"3391-7"~"PMR"~"505 (o)(3)"~3391.00~7~"PMR 3391-7: Conduct a juvenile animal study in rats from PND 7 to 90 to support pediatric drug development in children and neonates. The study will evaluate the effect of the drug on growth and development, specifically, reproductive capacity, bone development, and the central nervous system histopathology and long-term functional effects (learning and memory, motor function. reflexes, and an assessment of social interaction or other higher-cognitive functioning)."~"Submitted"~~1/31/2021 0:00:00~7/15/2026 0:00:00
384538~"CDER"~"NDA"~209229.00~"BIOCORRX PHARMACEUTICALS INC"~"Lucemyra (lofexidine)"~5/16/2018 0:00:00~"Original"~1~"3391-8"~"PMR"~"505 (o)(3)"~3391.00~8~"PMR 3391-8: Conduct a fertility and early embryonic development study in rats testing lofexidine administration to males and include histopathology of the testes and sperm assessments (count, motility, and morphology)."~"Submitted"~~9/30/2020 0:00:00~7/15/2026 0:00:00
384539~"CDER"~"NDA"~209229.00~"BIOCORRX PHARMACEUTICALS INC"~"Lucemyra (lofexidine)"~5/16/2018 0:00:00~"Original"~1~"3391-9"~"PMR"~"505 (o)(3)"~3391.00~9~"PMR 3391-9: Conduct a 90-day GLP repeat-dose toxicology study in the rat testing lofexidine HCl to establish a NOAEL to support clinical studies that are longer than 14 days in duration. To avoid the potential confounding impact of Cmax-induced fluctuations in physiology, which could impact the NOAEL, dose the animals several times a day, as feasible, to mimic the clinical setting."~"Submitted"~~1/31/2020 0:00:00~7/15/2026 0:00:00
384540~"CDER"~"NDA"~209229.00~"BIOCORRX PHARMACEUTICALS INC"~"Lucemyra (lofexidine)"~5/16/2018 0:00:00~"Original"~1~"3391-16"~"PMR"~"505 (o)(3)"~3391.00~16~"PMR 3391-16: Conduct a randomized, controlled clinical trial to evaluate the occurrenceof hypotension, bradycardia, syncope, rebound hypertension, hepatotoxicity, and unexpected serious risks in patients treated with lofexidine beyond 14 days in the setting of gradual opioid taper."~"Delayed"~"The draft and final protocol milestones were missed. PMR release
requested on 02/22/2024. Denied per FDA letter dated 11/01/2024."~1/31/2027 0:00:00~7/15/2026 0:00:00
384541~"CDER"~"NDA"~209229.00~"BIOCORRX PHARMACEUTICALS INC"~"Lucemyra (lofexidine)"~5/16/2018 0:00:00~"Original"~1~"3391-17"~"PMR"~"Pediatric Research Equity Act"~3391.00~17~"PMR 3391-17: Conduct a dose-ranging pharmacokinetic study of lofexidine in neonates with neonatal opioid withdrawal syndrome."~"Pending"~"Draft protocol is pending by 09/2025."~6/30/2027 0:00:00~7/15/2026 0:00:00
384542~"CDER"~"NDA"~209229.00~"BIOCORRX PHARMACEUTICALS INC"~"Lucemyra (lofexidine)"~5/16/2018 0:00:00~"Original"~1~"3391-18"~"PMR"~"Pediatric Research Equity Act"~3391.00~18~"PMR 3391-18: Conduct a safety and efficacy study of lofexidine in neonates with neonatal opioid withdrawal syndrome."~"Pending"~"Draft protocol is pending by 09/2025."~6/30/2031 0:00:00~7/15/2026 0:00:00
384543~"CDER"~"NDA"~209296.00~"HERON THERAPEUTICS INC"~"Cinvanti (aprepitant)"~11/9/2017 0:00:00~"Original"~1~"3289-2"~"PMR"~"Pediatric Research Equity Act"~3289.00~2~"PMR 3289-2: A study to evaluate pharmacokinetics, safety, and tolerability of a singledose regimen of Cinvanti (aprepitant) injectable emulsion (I.V.) in pediatric patients 0 to 17 years of age undergoing single day highly emetogenic chemotherapy."~"Delayed"~"A Deferral Extension was granted on 12/19/2024 to adjust the timeline with PMR 3289-3. The final protocol and study completion milestones were also revised."~5/31/2028 0:00:00~1/6/2026 0:00:00
384544~"CDER"~"NDA"~21926.00~"AZURITY PHARMACEUTICALS INC"~"Treximet (Naproxen Sodium and Sumatriptan)"~4/15/2008 0:00:00~"Supplement"~11~"2910-1"~"PMR"~"Pediatric Research Equity Act"~2910.00~1~"PMR 2910-1: Conduct a juvenile rat toxicology study to identify the unexpected serious risk of adverse effects of sumatriptan/naproxen on postnatal growth and development. The study should utilize animals of an age range and stage(s) of development that are comparable to the intended pediatric population; the duration of dosing should cover  the intended length of treatment in the pediatric population. In addition to the usual toxicological parameters, this study must evaluate effects of sumatriptan/naproxen on growth, reproductive development, and neurological and neurobehavioral development."~"Delayed"~"Deferral Extension Requested 08/05/2021. Denied per FDA letter dated 09/17/2021. The study completion and final report submission milestones were also missed. Per the applicant s Annual Status Report, a PREA waiver was requested on 9/25/2025 and denied on 11/17/2025."~5/31/2018 0:00:00~5/22/2026 0:00:00
384545~"CDER"~"NDA"~21926.00~"AZURITY PHARMACEUTICALS INC"~"Treximet (Naproxen Sodium and Sumatriptan)"~4/15/2008 0:00:00~"Supplement"~11~"2910-2"~"PMR"~"Pediatric Research Equity Act"~2910.00~2~"PMR 2910-2: Conduct a pharmacokinetics (PK) study in children ages 6 years to 11 years with migraine. Using information from this PK study, conduct a controlled efficacy study in children ages 6 years to 11 years with migraine. Conduct a long-term open-label safety study in pediatric patients with migraine ages 6 years to 11 years. The longterm safety study must provide a descriptive analysis of safety data in at least 50 pediatric patients treated with Treximet for a duration of at least 6 months, treating on average at least one migraine attack per month, at doses evaluated in the efficacy study."~"Delayed"~"Deferral Extension Requested 08/05/2021. Denied per FDA letter dated 09/17/2021.The final protocol, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, a PREA waiver was requested on 9/25/2025 and denied on 11/17/2025."~5/31/2021 0:00:00~5/22/2026 0:00:00
384546~"CDER"~"NDA"~21926.00~"AZURITY PHARMACEUTICALS INC"~"Treximet (Naproxen Sodium and Sumatriptan)"~4/15/2008 0:00:00~"Supplement"~12~"2910-1"~"PMR"~"Pediatric Research Equity Act"~2910.00~1~"PMR 2910-1: Conduct a juvenile rat toxicology study to identify the unexpected serious risk of adverse effects of sumatriptan/naproxen on postnatal growth and development. The study should utilize animals of an age range and stage(s) of development that are comparable to the intended pediatric population; the duration of dosing should cover  the intended length of treatment in the pediatric population. In addition to the usual toxicological parameters, this study must evaluate effects of sumatriptan/naproxen on growth, reproductive development, and neurological and neurobehavioral development."~"Delayed"~"Deferral Extension Requested 08/05/2021. Denied per FDA letter dated 09/17/2021. The study completion and final report submission milestones were also missed. Per the applicant s Annual Status Report, a PREA waiver was requested on 9/25/2025 and denied on 11/17/2025."~5/31/2018 0:00:00~5/22/2026 0:00:00
385402~"CDER"~"NDA"~220295.00~"INTERCEPT PHARMACEUTICALS INC"~"Lynavoy (linerixibat) tablet"~3/17/2026 0:00:00~"Original"~1~"4937-2"~"PMR"~"505 (o)(3)"~4937.00~2~"PMR 4937-2: Complete the ongoing trial 212358 (Linerixibat Long-term Safety and Tolerability study [LLSAT]) to evaluate liver injury, bone health, and bleeding risks of linerixibat. Enroll all eligible and consenting subjects from prior trials."~"Ongoing"~~3/31/2027 0:00:00~
385403~"CDER"~"NDA"~220305.00~"KURA ONCOLOGY INC"~"Komzifti (ziftomenib) capsules, 200 mg"~11/13/2025 0:00:00~"Original"~1~"4929-1"~"PMR"~"Pediatric Research Equity Act"~4929.00~1~"PMR 4929-1: Conduct a molecularly targeted pediatric cancer investigation using age-appropriate pediatric formulations to evaluate dosing, pharmacokinetics, safety, and preliminary efficacy of ziftomenib in combination with fludarabine and cytarabine, in pediatric patients greater than or equal to 1 month to <17 years of age with relapsed/refractory KMT2A-r/NUP98-r/NPM1-m acute leukemia."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~2/28/2030 0:00:00~
385404~"CDER"~"NDA"~220305.00~"KURA ONCOLOGY INC"~"Komzifti (ziftomenib) capsules, 200 mg"~11/13/2025 0:00:00~"Original"~1~"4929-2"~"PMR"~"505 (o)(3)"~4929.00~2~"PMR 4929-2: Conduct a clinical trial to assess and further characterize the known and potential serious risks related to the use of ziftomenib, including whether long-term use of ziftomenib is associated with an increased risk of recurrent differentiation syndrome, serious hepatotoxicity, and increased risk of cardiac deaths. Summarize the safety outcomes when all patients have completed at least three years of treatment with ziftomenib or withdrew earlier."~"Pending"~~5/31/2032 0:00:00~
385405~"CDER"~"NDA"~220305.00~"KURA ONCOLOGY INC"~"Komzifti (ziftomenib) capsules, 200 mg"~11/13/2025 0:00:00~"Original"~1~"4929-3"~"PMR"~"505 (o)(3)"~4929.00~3~"PMR 4929-3: Conduct a clinical pharmacokinetic trial to assess the magnitude of change in exposure of ziftomenib and evaluate a serious potential risk of increased drug toxicity, and to determine an appropriate dosage of ziftomenib to minimize toxicity, in patients with severe hepatic impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~5/31/2030 0:00:00~
385406~"CDER"~"BLA"~761108.00~"Alexion Pharmaceuticals, Inc."~"Ultomiris (ravulizumab-cwvz)"~12/21/2018 0:00:00~"Supplement"~1~"3698-3"~"PMC"~"506B PMC"~3698.00~3~"PMC 3698-3: Provide safety, efficacy, PK, and PD data in pediatric patients with aHUS (Study ALXN1210-aHUS-312) who switch from treatmentwith eculizumab to treatment with ravulizumab."~"Fulfilled"~~6/30/2025 0:00:00~2/20/2026 0:00:00
385407~"CDER"~"BLA"~761108.00~"Alexion Pharmaceuticals, Inc."~"Ultomiris (ravulizumab-cwvz)"~12/21/2018 0:00:00~"Supplement"~23~"4252-1"~"PMR"~"Pediatric Research Equity Act"~4252.00~1~"PMR 4252-1: Conduct a multicenter, open-label, single arm study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ravulizumab in pediatric patients ages 12 through 17 with AChR antibody-positive myasthenia gravis. This study will use comparable C5 level inhibition from adults to pediatrics to establish partial extrapolation using comparative PK/PD between pediatric patients ages 12 through 17 and adults."~"Ongoing"~"The study has been initiated."~1/31/2029 0:00:00~2/20/2026 0:00:00
385408~"CDER"~"BLA"~761108.00~"Alexion Pharmaceuticals, Inc."~"Ultomiris (ravulizumab-cwvz)"~12/21/2018 0:00:00~"Supplement"~23~"4252-2"~"PMR"~"505 (o)(3)"~4252.00~2~"PMR 4252-2: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Ultomiris (ravulizumab-cwvz) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~7/31/2034 0:00:00~2/20/2026 0:00:00
385409~"CDER"~"BLA"~761108.00~"Alexion Pharmaceuticals, Inc."~"Ultomiris (ravulizumab-cwvz)"~12/21/2018 0:00:00~"Supplement"~26~"4449-1"~"PMR"~"505 (o)(3)"~4449.00~1~"PMR 4449-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Ultomiris (ravulizumab-cwvz) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~7/31/2034 0:00:00~2/20/2026 0:00:00
385410~"CDER"~"BLA"~761108.00~"Alexion Pharmaceuticals, Inc."~"Ultomiris (ravulizumab-cwvz)"~12/21/2018 0:00:00~"Supplement"~31~"4449-1"~"PMR"~"505 (o)(3)"~4449.00~1~"PMR 4449-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Ultomiris (ravulizumab-cwvz) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~7/31/2034 0:00:00~2/20/2026 0:00:00
385411~"CDER"~"BLA"~761111.00~"Hospira Inc., a Pfizer Company"~"Nyvepria (pegfilgrastim-apgf)"~6/10/2020 0:00:00~"Original"~1~"3825-1"~"PMR"~"Pediatric Research Equity Act"~3825.00~1~"PMR 3825-1: Submit pediatric assessments for Nyvepria (pegfilgrastim-apgf) as described in section 505B(a)(2)(A) of the FD&C Act, including development of an  appropriate formulation  (presentation) that can be used to directly and accurately administer NYVEPRIA (pegfilgrastim-apgf) to pediatric patients who weigh less than 45 kg and require doses that are less than 0.6 mL (6 mg), and conducting any necessary human factors studies to evaluate the ability of healthcare providers and/or caregivers to measure the appropriate doses."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~4/30/2028 0:00:00~8/7/2025 0:00:00
385412~"CDER"~"BLA"~761113.00~"Sanofi-Aventis U.S. LLC"~"Sarclisa (isatuximab-irfc)"~3/2/2020 0:00:00~"Supplement"~3~"4040-2"~"PMC"~"506B PMC"~4040.00~2~"PMC 4040-2: Submit a final report containing data from clinical trials, post-marketing reports, compassionate use/expanded access programs, real-world evidence, and other sources to further characterize the safety and efficacy of isatuximab in combination with carfilzomib and dexamethasone (Isa-Kd) among U.S. racial and ethnic minority patients with multiple myeloma."~"Delayed"~"The applicant requested revised milestones because a sufficient number of patients with adequate follow-up for OS analysis has not been reached. Revised milestones were acknowledged in a letter dated 02/09/2026."~12/31/2026 0:00:00~4/29/2026 0:00:00
385413~"CDER"~"BLA"~761113.00~"Sanofi-Aventis U.S. LLC"~"Sarclisa (isatuximab-irfc)"~3/2/2020 0:00:00~"Supplement"~14~"4698-1"~"PMR"~"505 (o)(3)"~4698.00~1~"PMR 4698-1: Conduct an integrated analysis of data from clinical trials and other sources such as postmarketing reports or real-world data, in newly
diagnosed, transplant ineligible patients =75 years of age with multiple myeloma receiving isatuximab in combination with bortezomib, lenalidomide, and dexamethasone, to further characterize the risk of serious and fatal adverse reactions including infections and neutropenia, compared to patients <75 years of age. Include an analysis of Grade 3-4 pneumonia and Grade 3-4 neutropenia. Include a sufficient number of patients >80 years of age. The study report should also include overall survival data with longer-term follow up from patients =75 years of age compared to patients <75 years of age in the IMROZ trial to further characterize the safety in older age subgroups."~"Pending"~~8/31/2031 0:00:00~4/29/2026 0:00:00
385500~"CDER"~"BLA"~761032.00~"Bausch Health Ireland, Limited"~"Siliq (Brodalumab)"~2/15/2017 0:00:00~"Original"~1~"3164-1"~"PMR"~"Pediatric Research Equity Act"~3164.00~1~"PMR 3164-1: Open-label study to determine PK of a single dose of brodalumab in 16 children (6 to < 18 years old) with severe plaque psoriasis."~"Delayed"~"The study is underway. 2 of the planned 16 patients have been enrolled into the study. The final report submission milestone was missed."~6/30/2019 0:00:00~4/15/2026 0:00:00
385501~"CDER"~"BLA"~761032.00~"Bausch Health Ireland, Limited"~"Siliq (Brodalumab)"~2/15/2017 0:00:00~"Original"~1~"3164-2"~"PMR"~"Pediatric Research Equity Act"~3164.00~2~"PMR 3164-2: Double-blind, active comparator-controlled, multicenter study with brodalumab to determine the safety and efficacy in adolescent subjects (12 to < 18 years old)  with severe plaque psoriasis."~"Delayed"~"The final protocol, study completion, and final report submission dates have been missed because the applicant intends to submit the protocol near the completion of PMR 3164.1."~6/30/2024 0:00:00~4/15/2026 0:00:00
385502~"CDER"~"NDA"~217865.00~"ITALFARMACO SPA"~"Duvyzat (givinostat)"~3/21/2024 0:00:00~"Original"~1~"4575-3"~"PMR"~"505 (o)(3)"~4575.00~3~"PMR 4575-3: Conduct a prospective observational registry for a minimum of 5 years to characterize the incidence, frequency, and severity of thrombocytopenia and incidence, frequency, and severity of serious events of bleeding in patients with Duchenne muscular dystrophy exposed to givinostat. For each case of thrombocytopenia and severe bleeding identified, provide detailed case narratives that include, but are not limited to, information on concomitant medications, complete blood count results at baseline, and during and after the event, and disposition (e.g., was the givinostat dose reduced in response to the event, discontinued temporarily or permanently, and outcomes if the dose was reduced)."~"Pending"~~3/31/2032 0:00:00~5/19/2026 0:00:00
385503~"CDER"~"NDA"~217865.00~"ITALFARMACO SPA"~"Duvyzat (givinostat)"~3/21/2024 0:00:00~"Original"~1~"4575-4"~"PMR"~"505 (o)(3)"~4575.00~4~"PMR 4575-4: Conduct a clinical trial to evaluate the effect of hepatic impairment on the exposure of givinostat relative to that in subjects with normal hepatic function after oral administration. Please refer to the Guidance for Industry Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling (https://www.fda.gov/media/71311/download)."~"Delayed"~"The final report submission milestone was missed because the clinical trial application (CTA) validation and protocol queries necessitated a protocol amendment, which resulted in delays in
trial initiation and completion. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 01/23/2026."~12/31/2025 0:00:00~5/19/2026 0:00:00
385504~"CDER"~"NDA"~217899.00~"GILEAD SCIENCES INC"~"Livdelzi (seladelpar)"~8/14/2024 0:00:00~"Original"~1~"4645-1"~"PMR"~"Accelerated Approval"~4645.00~1~"PMR 4645-1: Complete Trial CB8025-41837, a randomized, double-blind, placebocontrolled trial to evaluate the effect of seladelpar on clinical outcomes in subjects with primary biliary cholangitis (PBC) and compensated cirrhosis. Efficacy must be demonstrated using a composite endpoint of mortality, liver transplant, MELD =15, ascites requiring treatment, and hospitalization for hepatic decompensation (e.g., variceal bleeding, hepatic encephalopathy, and spontaneous bacterial peritonitis)."~"Ongoing"~"There are currently 90 of 318 planned participants enrolled. Completion of Trial Enrollment: 08/2026."~2/28/2030 0:00:00~10/7/2025 0:00:00
385505~"CDER"~"NDA"~217899.00~"GILEAD SCIENCES INC"~"Livdelzi (seladelpar)"~8/14/2024 0:00:00~"Original"~1~"4645-2"~"PMR"~"505 (o)(3)"~4645.00~2~"PMR 4645-2: Conduct a ten-year worldwide descriptive study that collects prospective and retrospective data from women exposed to seladelpar during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients should be specified in the protocol."~"Pending"~~5/31/2038 0:00:00~10/7/2025 0:00:00
385506~"CDER"~"NDA"~217899.00~"GILEAD SCIENCES INC"~"Livdelzi (seladelpar)"~8/14/2024 0:00:00~"Original"~1~"4645-3"~"PMR"~"505 (o)(3)"~4645.00~3~"PMR 4645-3: Perform a lactation study, milk only, in lactating women who have received seladelpar to measure concentrations of seladelpar in breast milk using a validated assay and to assess the effects on the breastfed infant."~"Pending"~~8/31/2028 0:00:00~10/7/2025 0:00:00
385507~"CDER"~"NDA"~217900.00~"SUN PHARMACEUTICAL INDUSTRIES INC"~"Leqselvi (deuruxolitinib phosphate)"~7/25/2024 0:00:00~"Original"~1~"4655-1"~"PMR"~"Pediatric Research Equity Act"~4655.00~1~"PMR 4655-1: Conduct a juvenile toxicity study to evaluate the potential toxicity of deuruxolitinib, including the potential effects on growth, development, and reproduction, when administered orally to juvenile rats."~"Ongoing"~"The study is underway. The in-life portion of the study is complete."~2/28/2026 0:00:00~9/22/2025 0:00:00
385508~"CDER"~"NDA"~217900.00~"SUN PHARMACEUTICAL INDUSTRIES INC"~"Leqselvi (deuruxolitinib phosphate)"~7/25/2024 0:00:00~"Original"~1~"4655-2"~"PMR"~"Pediatric Research Equity Act"~4655.00~2~"PMR 4655-2: Conduct a randomized, controlled trial to evaluate the safety, efficacy, and pharmacokinetics of deuruxolitinib in the adolescent population (12 years to less than 18 years) with severe alopecia areata. Evaluate at least 300 subjects exposed to deuruxolitinib for a minimum of 52 weeks."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone date. The final protocol was acknowledged in a letter dated 05/05/2025. The trial has begun enrollment with 1 patient enrolled as of 09/2025."~10/31/2029 0:00:00~9/22/2025 0:00:00
385509~"CDER"~"NDA"~217900.00~"SUN PHARMACEUTICAL INDUSTRIES INC"~"Leqselvi (deuruxolitinib phosphate)"~7/25/2024 0:00:00~"Original"~1~"4655-3"~"PMR"~"Pediatric Research Equity Act"~4655.00~3~"PMR 4655-3: Conduct a randomized, controlled trial to evaluate the safety, efficacy, and pharmacokinetics of deuruxolitinib in the pediatric population (6 years to less than 12 years) with severe alopecia areata. Evaluate at least 100 subjects exposed to deuruxolitinib for a minimum of 52 weeks."~"Pending"~"The trial has not been initiated, but it does not meet the criterion for delayed."~7/31/2035 0:00:00~9/22/2025 0:00:00
385510~"CDER"~"NDA"~217900.00~"SUN PHARMACEUTICAL INDUSTRIES INC"~"Leqselvi (deuruxolitinib phosphate)"~7/25/2024 0:00:00~"Original"~1~"4655-4"~"PMR"~"505 (o)(3)"~4655.00~4~"PMR 4655-4: Collect global data from prospective pregnancy exposure registry/registries, preferably disease-based multiproduct pregnancy registry/registries, using a registry-based cohort study design to compare the maternal, fetal, and infant outcomes of women with Alopecia Areata exposed to deuruxolitinib during pregnancy with unexposed comparator population(s).The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortion, stillbirths, pregnancy terminations, preterm births, small forgestational-age births, and any other adverse outcomes, including postnatal growth and development. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Pending"~~1/31/2037 0:00:00~9/22/2025 0:00:00
385511~"CDER"~"NDA"~217900.00~"SUN PHARMACEUTICAL INDUSTRIES INC"~"Leqselvi (deuruxolitinib phosphate)"~7/25/2024 0:00:00~"Original"~1~"4655-5"~"PMR"~"505 (o)(3)"~4655.00~5~"PMR 4655-5: Conduct an additional pregnancy study that uses a different design from the pregnancy exposure registry (for example a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, pregnancy terminations,  preterm births, and small for gestational age infants in women exposed to deuruxolitinib during pregnancy compared to an unexposed comparator population(s)."~"Pending"~~1/31/2037 0:00:00~9/22/2025 0:00:00
383872~"CDER"~"NDA"~214200.00~"PHARMACOSMOS AS"~"COSELA (Trilaciclib)"~2/12/2021 0:00:00~"Original"~1~"4018-1"~"PMR"~"505 (o)(3)"~4018.00~1~"PMR 4018-1: Conduct a study in a sufficient number of adult patients with extensive stage-small cell lung cancer undergoing chemotherapy to evaluate the impact of trilaciclib on disease progression or survival in patients with chemotherapy-induced myelosuppression treated with a platinum/etoposide-containing regimen or topotecan-containing regimen with at least 2 years of follow-up."~"Delayed"~"The study completion and final report submission milestones were missed because of delays in initiating study enrollment due to continued discussions with FDA regarding the final protocol and logistical challenges. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 12/01/2025. 281 patients out of the planned 302 patients have been enrolled into the trial."~11/30/2025 0:00:00~4/10/2026 0:00:00
383873~"CDER"~"NDA"~214231.00~"ZEALAND PHARMA US INC"~"Zegalogue (dasiglucagon)"~3/22/2021 0:00:00~"Original"~1~"4034-1"~"PMR"~"Pediatric Research Equity Act"~4034.00~1~"PMR 4034-1: Conduct an open-label pediatric study to evaluate the safety, efficacy, and pharmacokinetics/pharmacodynamics of dasiglucagon in pediatric patients ages 1 year to <6 years with type 1 diabetes mellitus."~"Submitted"~"The final report was submitted to FDA on 12/19/2025."~12/31/2025 0:00:00~5/18/2026 0:00:00
383874~"CDER"~"NDA"~214273.00~"AZURITY PHARMACEUTICALS INC"~"Zonisade (zonisamide)"~7/15/2022 0:00:00~"Original"~1~"4302-1"~"PMR"~"Pediatric Research Equity Act"~4302.00~1~"PMR 4302-1: A study to evaluate the pharmacokinetics, safety, and tolerability of zonisamide oral suspension to determine a dosing regimen as therapy for partial-onset seizures in children 1 month to 17 years of age that provides drug exposures that are similar to the exposures that are effective in adult patients with partial-onset seizures. The efficacy of zonisamide in children 1 month to 17 years of age for treatment of partial-onset seizures will be addressed by a pharmacokinetic analysis to determine pediatric dosing that will match exposures in children to those in adults. This analysis will require pharmacokinetic data from studies of both adult and pediatric patients (1 month to 17 years of age)."~"Delayed"~"Original Final Report Due Date: 04/30/2024. Per FDA letter dated 03/05/2024, Final Report due date extended to 06/30/2025. Second Deferral Extension Granted per FDA letter dated 04/18/2025, Final Report due date extended to 06/30/2026. Third Deferral Extension Granted per FDA letter dated 04/24/2026 Final Report due date extended to 01/31/2028. The Trial Completion milestone was also revised."~1/31/2028 0:00:00~9/15/2025 0:00:00
383875~"CDER"~"NDA"~214273.00~"AZURITY PHARMACEUTICALS INC"~"Zonisade (zonisamide)"~7/15/2022 0:00:00~"Original"~1~"4302-2"~"PMR"~"Pediatric Research Equity Act"~4302.00~2~"PMR 4302-2: Long-term open-label safety study of zonisamide oral suspension in children 1 month to 17 years of age (at dosing levels found to be therapeutic) in the treatment of partial-onset seizures. Routine safety measures should be monitored and the study population must include an appropriate representation of age distributions as shown below. Behavioral and cognitive endpoints should be included. At least 15% of the study population should be in each of the following age brackets: 1. 1 month to less than 2 years 2. 2 years to less than 4 years 3. 4 years to less than 8 years 4. 8 years to less than 12 years 5. 12 years to 17 years."~"Delayed"~"The Final Protocol Submission milestone was missed
because data collection for PMR 4302-1 is ongoing
and dependent upon its completion. Deferral Extension
requested 03/06/2025. Denied per FDA letter dated
04/18/2025. Per applicant s Annual Status Report, no
patients are enrolled in the trial.  
"~6/30/2027 0:00:00~9/15/2025 0:00:00
383876~"CDER"~"NDA"~214373.00~"THERACOSBIO LLC"~"Brenzavvy (bexagliflozin)"~1/20/2023 0:00:00~"Original"~1~"4296-4"~"PMR"~"Pediatric Research Equity Act"~4296.00~4~"PMR 4296-4: Conduct a 24-week, randomized, double-blind, placebo-controlled, parallel group study of the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of bexagliflozin for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 17 years (inclusive), followed by a 28-week controlled extension. The bexagliflozin dose will be determined based on the initial PK/PD lead-in portion of the study."~"Delayed"~"The original Final Protocol Submission milestone was missed and revised due to ongoing discussions with the Agency. The revised protocol submission milestone has been met."~10/31/2031 0:00:00~3/12/2026 0:00:00
383877~"CDER"~"NDA"~214377.00~"MERCK SHARP AND DOHME LLC"~"Verquvo (vericiguat)"~1/19/2021 0:00:00~"Original"~1~"4001-3"~"PMR"~"505 (o)(3)"~4001.00~3~"PMR 4001-3: A worldwide descriptive study that collects prospective and retrospective data in women exposed to vericiguat during pregnancy to assess risk to the pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The study will collect information for a minimum of 10 years. Results will be analyzed and reported descriptively. Data collected retrospectively will be analyzed separately and reported with the interim and final study reports."~"Ongoing"~~3/31/2034 0:00:00~3/11/2026 0:00:00
383878~"CDER"~"NDA"~214410.00~"GENENTECH INC"~"Xofluza (baloxavir marboxil)"~11/23/2020 0:00:00~"Original"~2~"4316-2"~"PMC"~"506B PMC"~4316.00~2~"PMC 4316-2: Conduct a prospective, multicenter, observational study in baloxavir marboxil-treated patients over at least five influenza seasons that will capture the susceptibility and genotype of influenza viruses in baseline and on-treatment respiratory samples to determine the frequency of baseline and treatment-emergent baloxavir resistance and the impact on outcomes."~"Ongoing"~~10/31/2027 0:00:00~1/16/2026 0:00:00
383879~"CDER"~"NDA"~214410.00~"GENENTECH INC"~"Xofluza (baloxavir marboxil)"~11/23/2020 0:00:00~"Original"~2~"4316-3"~"PMC"~"506B PMC"~4316.00~3~"PMC 4316-3: Provide a semi-annual (twice-yearly) update on global baloxavir usage and emergence of resistance to baloxavir as an integrated review of information from national and international influenza drug resistance databases and sequence databases, including but not limited to World Health Organization and US Centers for Disease Control and Prevention surveillance, data collected by the sponsor, and information in the published literature. Each update will include information on the methodologies (e.g., viral gene sequencing and phenotypic assay descriptions) used in studies during that reporting period. Substitutions of particular interest include all those listed as resistance associated in the USPI, as well as substitutions currently identified or identified in the future that reduce susceptibility to baloxavir marboxil."~"Ongoing"~~5/31/2026 0:00:00~1/16/2026 0:00:00
383880~"CDER"~"NDA"~214429.00~"SANOFI AVENTIS US LLC"~"fexinidazole"~7/16/2021 0:00:00~"Original"~1~"3952-1"~"PMR"~"505 (o)(3)"~3952.00~1~"PMR 3952-1: Submit the results of your current worldwide descriptive study that is collecting prospective and retrospective data in women exposed to fexinidazole during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant."~"Delayed"~"The trial completion and final report submission milestones were missed. FDA acknowledged the revised milestones on 05/17/2024."~4/30/2024 0:00:00~9/11/2025 0:00:00
383881~"CDER"~"NDA"~215498.00~"IPSEN BIOPHARMACEUTICALS INC"~"Bylvay (Odevixibat)"~7/20/2021 0:00:00~"Original"~1~"4109-2"~"PMC"~"506B PMC"~4109.00~2~"PMC 4109-2: Conduct a prospective, long-term, observational study of patients aged 3 months or older with progressive familial intrahepatic cholestasis (PFIC) in order to assess the long-term safety of treatment with BYLVAY (odevixibat) over a 72-week treatment period."~"Fulfilled"~~6/30/2024 0:00:00~9/17/2025 0:00:00
383882~"CDER"~"NDA"~215498.00~"IPSEN BIOPHARMACEUTICALS INC"~"Bylvay (Odevixibat)"~7/20/2021 0:00:00~"Original"~1~"4109-3"~"PMC"~"506B PMC"~4109.00~3~"PMC 4109-3: Conduct a 5-year registry-based study to collect data on the course of liver disease in patients chronically treated with BYLVAY (odevixibat). Report yearly on the incidence of biliary diversion surgery, liver transplantation, and all-cause mortality in patients chronically treated with BYLVAY. Compare the incidence of these safety outcomes to a control group that did not receive BYLVAY."~"Ongoing"~~12/31/2027 0:00:00~9/17/2025 0:00:00
383883~"CDER"~"NDA"~215498.00~"IPSEN BIOPHARMACEUTICALS INC"~"Bylvay (Odevixibat)"~7/20/2021 0:00:00~"Supplement"~3~"4444-1"~"PMC"~"506B PMC"~4444.00~1~"PMC 4444-1: Conduct a prospective, long-term, observational study of patients aged 12 months or older with Alagille syndrome in order to assess the long-term safety of treatment with BYLVAY (odevixibat) over a 72-week treatment period."~"Fulfilled"~~2/28/2025 0:00:00~9/17/2025 0:00:00
383884~"CDER"~"NDA"~215498.00~"IPSEN BIOPHARMACEUTICALS INC"~"Bylvay (Odevixibat)"~7/20/2021 0:00:00~"Supplement"~3~"4444-2"~"PMC"~"506B PMC"~4444.00~2~"PMC 4444-2: Conduct a 5-year registry-based study to collect data on the health of Alagille syndrome patients chronically treated with Bylvay (odevixibat). Report yearly on the following safety endpoints: Incidence of biliary diversion surgery, liver transplantation, and all-cause mortality. Assessment of growth and development. Incidence of fat-soluble vitamin deficiencies and their long-term sequelae"~"Ongoing"~~5/31/2030 0:00:00~9/17/2025 0:00:00
383885~"CDER"~"NDA"~215499.00~"VIIV HEALTHCARE CO"~"APRETUDE (cabotegravir)"~12/20/2021 0:00:00~"Original"~1~"4191-2"~"PMR"~"505 (o)(3)"~4191.00~2~"PMR 4191-2: Conduct a study to collect and analyze data from adults and adolescents who take cabotegravir extended-release injectable suspension (CAB LA) for pre-exposure prophylaxis (PrEP) of sexually acquired HIV-1 infection and who become infected during PrEP use or within one year of discontinuing PrEP. As part of this study, collect and provide information on adherence, risk factors for non-adherence and HIV-1 acquisition. To compare tolerability and rates of HIV-1 acquisition among individuals who initiate CAB LA with an oral lead-in to those who directly initiate CAB LA injections, the following should be included in the study report: a) Frequency of testing and methods used for HIV-1 diagnosis. b) Frequency of prevalent HIV-1 infections that were detected, including those captured during screening. c) Resistance analyses of viral isolates from those who acquire HIV-1, including a description of the methodologies used to evaluate resistance. d) Description of subsequent antiretroviral therapy, including the time required to achieve virologic suppression and the durability of the response (to cover 48-weeks from the time therapy is initiated, at minimum). e) Information on adherence, select adverse events and treatment discontinuations."~"Ongoing"~~3/31/2028 0:00:00~2/17/2026 0:00:00
383886~"CDER"~"NDA"~215500.00~"USWM LLC"~"Iwilfin (eflornithine)"~12/13/2023 0:00:00~"Original"~1~"4555-1"~"PMR"~"505 (o)(3)"~4555.00~1~"PMR 4555-1: Conduct an integrated safety analysis of clinical trial data to assess the serious risk of ototoxicity and to further characterize the serious risks of severe adverse reactions including myelosuppression and hepatotoxicity and their sequelae in a sufficient number of patients exposed to IWILFIN; and to identify risk factors for development of these adverse reactions. Safety assessments will include audiograms prior to and at regular intervals during treatment. The study should include appropriate monitoring and risk mitigation strategies."~"Ongoing"~~12/31/2027 0:00:00~2/11/2026 0:00:00
383887~"CDER"~"NDA"~215515.00~"ALNYLAM PHARMACEUTICALS INC"~"AMVUTTRA (vutrisiran)"~6/13/2022 0:00:00~"Original"~1~"4288-3"~"PMR"~"505 (o)(3)"~4288.00~3~"PMR 4288-3: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Amvuttra (vutrisiran) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol. 
"~"Ongoing"~~12/31/2035 0:00:00~8/8/2025 0:00:00
383888~"CDER"~"NDA"~215559.00~"IPSEN BIOPHARMACEUTICALS INC"~"Sohonos (Palovarotene)"~8/16/2023 0:00:00~"Original"~1~"4474-1"~"PMR"~"505 (o)(3)"~4474.00~1~"PMR 4474-1: Conduct a carcinogenicity study in rats to assess the carcinogenic potential from long-term oral exposure to palovarotene."~"Ongoing"~~10/31/2027 0:00:00~10/10/2025 0:00:00
383889~"CDER"~"NDA"~215559.00~"IPSEN BIOPHARMACEUTICALS INC"~"Sohonos (Palovarotene)"~8/16/2023 0:00:00~"Original"~1~"4474-2"~"PMR"~"505 (o)(3)"~4474.00~2~"PMR 4474-2: Conduct a carcinogenicity study in mice to assess the carcinogenic potential from long-term oral exposure to palovarotene."~"Ongoing"~~3/31/2027 0:00:00~10/10/2025 0:00:00
383890~"CDER"~"NDA"~215559.00~"IPSEN BIOPHARMACEUTICALS INC"~"Sohonos (Palovarotene)"~8/16/2023 0:00:00~"Original"~1~"4474-3"~"PMR"~"505 (o)(3)"~4474.00~3~"PMR 4474-3: Conduct a prospective observational registry study with safety objectives of comparing palovarotene exposed and unexposed patients with fibrodysplasia ossificans progressiva (FOP). Evaluate risks of increased flare-up episodes, alterations in growth, and bone fractures. The registry should also collect information on women exposed to palovarotene during pregnancy to assess for adverse events related to pregnancy through the first year postpartum, and birth and developmental outcomes through the infant s first year of life. Begin safety data collection within 90 days of protocol agreement. After protocol finalization, the PMR progress report should be submitted annually as part of the NDA annual report that also includes an evaluation of the effectiveness of meeting the registry study s safety objectives. Collect 10-year safety data from a minimum of 100 subjects, approximately half of whom will be pediatric patients (8 years to less than 18 years of age for girls and 10 years to less than 18 years of age for boys), and approximately two-thirds of whom will be exposed to palovarotene."~"Delayed"~"The Final Protocol milestone was missed because the design of the study has to be revised. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 04/02/2025."~1/31/2037 0:00:00~10/10/2025 0:00:00
383891~"CDER"~"NDA"~215559.00~"IPSEN BIOPHARMACEUTICALS INC"~"Sohonos (Palovarotene)"~8/16/2023 0:00:00~"Original"~1~"4474-4"~"PMR"~"505 (o)(3)"~4474.00~4~"PMR 4474-4: Conduct a clinical trial to evaluate the impact of hepatic impairment on the pharmacokinetics of palovarotene in subjects with moderate (Child-Pugh B) and severe (Child-Pugh C) hepatic impairment relative to healthy adult subjects with normal hepatic function, in accordance with the study design described in guidance for industry Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. Submit the subject-level datasets with the final report."~"Ongoing"~~6/30/2027 0:00:00~10/10/2025 0:00:00
383892~"CDER"~"NDA"~217513.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mekinist (trametinib)"~3/16/2023 0:00:00~"Original"~1~"4420-1"~"PMR"~"505 (o)(3)"~4420.00~1~"PMR 4420-1: Conduct comprehensive safety analyses from ongoing trials to further assess the serious risks of trametinib in combination with dabrafenib, including but not limited to new primary malignancies (cutaneous and noncutaneous), cardiomyopathy, and ocular toxicities, in pediatric patients with BRAFV600E mutant low grade glioma over a sufficient period of follow-up time to further characterize these risks. The report should include appropriate monitoring and risk mitigation strategies."~"Ongoing"~~5/31/2027 0:00:00~5/12/2026 0:00:00
383893~"CDER"~"NDA"~217513.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mekinist (trametinib)"~3/16/2023 0:00:00~"Original"~1~"4420-2"~"PMR"~"505 (o)(3)"~4420.00~2~"PMR 4420-2: Conduct an integrated safety analyses from clinical studies that further characterize the potential serious risk of long-term adverse effects including but not limited to growth plate abnormalities of trametinib in combination with dabrafenib on growth and development in a sufficient number of pediatric patients. Monitor patients for growth and development using age-appropriate screening tools. Include evaluations of growth as measured by height, weight, height velocity and height standard deviation scores, age at menarche if applicable (females) and Tanner stage. Monitor patients until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first."~"Ongoing"~~5/31/2027 0:00:00~5/12/2026 0:00:00
383894~"CDER"~"NDA"~217513.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mekinist (trametinib)"~3/16/2023 0:00:00~"Original"~1~"4420-5"~"PMC"~"506B PMC"~4420.00~5~"PMC 4420-5: Commitment to support the availability of an in vitro diagnostic device, through an appropriate analytical and clinical validation study using clinical trial data that demonstrates the device is essential to the safe and effective use of dabrafenib in combination with trametinib (D+T) for the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy."~"Delayed"~"The final report milestone was missed because of challenges with sourcing."~2/28/2025 0:00:00~5/12/2026 0:00:00
383895~"CDER"~"NDA"~217514.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tafinlar (dabrafenib) "~3/16/2023 0:00:00~"Original"~1~"4421-1"~"PMR"~"505 (o)(3)"~4421.00~1~"PMR 4421-1: Conduct comprehensive safety analyses from ongoing trials to further assess the serious risks of dabrafenib in combination with trametinib, including but not limited to new primary malignancies (cutaneous and noncutaneous), cardiomyopathy, and ocular toxicities, in pediatric patients with BRAFV600E mutant low grade glioma over a sufficient period of follow-up time to further characterize these risks. The report should include appropriate monitoring and risk mitigation strategies."~"Ongoing"~~5/31/2027 0:00:00~5/12/2026 0:00:00
383896~"CDER"~"NDA"~217514.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tafinlar (dabrafenib) "~3/16/2023 0:00:00~"Original"~1~"4421-2"~"PMR"~"505 (o)(3)"~4421.00~2~"PMR 4421-2: Conduct an integrated safety analyses from clinical studies that further characterize the potential serious risk of long-term adverse effects including but not limited to growth plate abnormalities of dabrafenib in combination with trametinib on growth and development in a sufficient number of pediatric patients. Monitor patients for growth and development using age-appropriate screening tools. Include evaluations of growth as measured by height, weight, height velocity and height standard deviation scores, age at menarche if applicable (females) and Tanner stage. Monitor patients until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first."~"Ongoing"~~5/31/2027 0:00:00~5/12/2026 0:00:00
383897~"CDER"~"NDA"~217514.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tafinlar (dabrafenib) "~3/16/2023 0:00:00~"Original"~1~"4421-5"~"PMC"~"506B PMC"~4421.00~5~"PMC 4421-5: Commitment to support the availability of an in vitro diagnostic device, through an appropriate analytical and clinical validation study using clinical trial data that demonstrates the device is essential to the safe and effective use of dabrafenib in combination with trametinib (D+T) for the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy."~"Delayed"~"The final report milestone was missed because of challenges with sourcing."~2/28/2025 0:00:00~5/12/2026 0:00:00
383898~"CDER"~"NDA"~217564.00~"TAKEDA PHARMACEUTICALS USA INC"~"Fruzaqla (fruquintinib)"~11/8/2023 0:00:00~"Original"~1~"4544-1"~"PMC"~"506B PMC"~4544.00~1~"PMC 4544-1: Conduct a clinical study to further characterize the clinical effects of fruquintinib, including pharmacokinetics (PK), activity, blood pressure assessments, and safety events of palmar-plantar erythrodysesthesia in an underrepresented minority population."~"Pending"~~12/31/2028 0:00:00~
383899~"CDER"~"NDA"~217604.00~"AMNEAL PHARMACEUTICALS LLC"~"pyridostigmine bromide extended release tablet 105 mg"~10/4/2024 0:00:00~"Original"~1~"4713-1"~"PMR"~"Animal Efficacy"~4713.00~1~"PMR 4713-1: Conduct postmarketing studies, such as field studies, intended to verify and describe pyridostigmine's clinical benefit and to assess its safety when used as indicated when such studies are feasible and ethical."~"Delayed"~"The applicant requested revised milestones because of time needed to coordinate with the Department of Defense (DoD) and the Surgeon General s team to develop the protocol. Revised milestones were acknowledged in a letter dated 04/23/2026.

"~~11/25/2025 0:00:00
383900~"CDER"~"NDA"~217639.00~"STEMLINE THERAPEUTICS INC"~"Orserdu (elacestrant)"~1/27/2023 0:00:00~"Original"~1~"4394-1"~"PMR"~"505 (o)(3)"~4394.00~1~"PMR 4394-1: Complete a pharmacokinetic trial to determine an appropriate dose of elacestrant in patients with severe hepatic impairment in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling found at: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM072123.pdf."~"Submitted"~~6/30/2026 0:00:00~3/25/2026 0:00:00
383901~"CDER"~"NDA"~217639.00~"STEMLINE THERAPEUTICS INC"~"Orserdu (elacestrant)"~1/27/2023 0:00:00~"Original"~1~"4394-2"~"PMC"~"506B PMC"~4394.00~2~"PMC 4394-2: Conduct an integrated analysis containing data from clinical trials and other data sources such as post-marketing reports, real-world evidence and other sources to further characterize the safety and efficacy of elacestrant in patients from racial and ethnic minority groups. The analyses should support comparative safety and efficacy outcome analyses between the aforementioned populations and White patients."~"Ongoing"~~6/30/2028 0:00:00~3/25/2026 0:00:00
383902~"CDER"~"NDA"~217645.00~"TRIS PHARMA INC"~"Onyda XR (clonidine hydrochloride)"~5/24/2024 0:00:00~"Original"~1~"4636-1"~"PMR"~"Pediatric Research Equity Act"~4636.00~1~"PMR 4636-1: Conduct an adequate and well-controlled study to evaluate the safety and effectiveness of clonidine hydrochloride extended release
oral suspension in pediatric patients 4 years to <6 years of age with attention-deficit/hyperactivity disorder."~"Delayed"~"The final protocol submission milestone was missed because Tris received information requests from the Agency on September 27, 2024, and October 25, 2024, for the PREA protocols for PMR 4636-1 and 4636-2. Tris submitted a response to both the Information Requests on December 24, 2024 and received additional comments on the protocol on February 20, 2025. Additional information was requested by the agency on January 20, 2026 regarding PMR 4636-1."~1/31/2026 0:00:00~7/23/2025 0:00:00
383903~"CDER"~"NDA"~212726.00~"GENENTECH INC"~"Rozlytrek (Entrectinib)"~8/15/2019 0:00:00~"Original"~1~"3689-5"~"PMR"~"505 (o)(3)"~3689.00~5~"PMR 3689-5: Conduct clinical trial(s) of entrectinib in a sufficient number of pediatric patients 12 years of age and older with NTRK-fusion solid tumors to evaluate the potential serious risk of adverse long-term effects of entrectinib on growth and development, including neurological outcomes with reasonable precision. Patients will be monitored for growth and developmental milestones using age-appropriate screening tools and undergo neurological examination at appropriate intervals. Evaluations will include neurological exams with neurocognitive assessment, Karnofsky/Lansky score, growth as measured by height, weight, height velocity, and height standard deviation scores (SDS), age at adrenarche if applicable (males), age at menarche if applicable (females) and Tanner Stage. Patient monitoring will be performed until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first."~"Ongoing"~~8/31/2029 0:00:00~
383904~"CDER"~"NDA"~212726.00~"GENENTECH INC"~"Rozlytrek (Entrectinib)"~8/15/2019 0:00:00~"Original"~1~"3689-6"~"PMR"~"505 (o)(3)"~3689.00~6~"PMR 3689-6: Submit integrated safety analyses and supporting data from an adequate number of patients enrolled in clinical trial(s) designed to characterize the risk of fractures and its sequelae in patients exposed to entrectinib with reasonable precision; to identify risk factors for development of these sequelae; and to support labeling recommendations to mitigate the risk of skeletal fractures. The design of the trial should include sufficient bone monitoring to achieve these objectives, including but not limited to initial and serial assessment of bone mineral density (BMD) with dual x-ray absorptiometry (DXA) scans, and markers of bone formation, bone resorption, and calcium metabolism."~"Fulfilled"~~3/31/2025 0:00:00~
383905~"CDER"~"NDA"~212728.00~"PFIZER INC"~"Nurtec ODT (rimegepant) "~2/27/2020 0:00:00~"Original"~1~"3799-3"~"PMR"~"Pediatric Research Equity Act"~3799.00~3~"PMR 3799-3: A randomized, double-blind, placebo-controlled efficacy and safety study under PREA for the treatment of acute migraine with or without aura in patients ages 6 through 17 years. This study includes an initial single-blind placebo lead-in to identify patients
who respond to placebo. This efficacy study must be designed to show superiority of rimegepant over placebo and is to be submitted as a special protocol assessment (SPA)."~"Ongoing"~"The trial is underway. Per the applicant s Annual Status Report, 1440 patients have been enrolled into the trial of 2100 planned."~4/30/2028 0:00:00~4/23/2026 0:00:00
383906~"CDER"~"NDA"~212728.00~"PFIZER INC"~"Nurtec ODT (rimegepant) "~2/27/2020 0:00:00~"Original"~1~"3799-4"~"PMR"~"Pediatric Research Equity Act"~3799.00~4~"PMR 3799-4: A pediatric open-label safety study under PREA to evaluate the long-term safety of intermittent treatment with rimegepant in patients ages 6 through 17 years, for up to one year."~"Ongoing"~"The trial is underway. Per the applicant s Annual Status Report, 361 patients have been enrolled into the trial."~4/30/2028 0:00:00~4/23/2026 0:00:00
383907~"CDER"~"NDA"~212728.00~"PFIZER INC"~"Nurtec ODT (rimegepant) "~2/27/2020 0:00:00~"Original"~1~"3799-5"~"PMR"~"505 (o)(3)"~3799.00~5~"PMR 3799-5: A pre- and postnatal development study of rimegepant in rat."~"Submitted"~~9/30/2021 0:00:00~4/23/2026 0:00:00
383908~"CDER"~"NDA"~212728.00~"PFIZER INC"~"Nurtec ODT (rimegepant) "~2/27/2020 0:00:00~"Original"~1~"3799-6"~"PMR"~"505 (o)(3)"~3799.00~6~"PMR 3799-6: Prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with migraine exposed to rimegepant during pregnancy with two unexposed control populations: one consisting of women with migraine who have not been exposed to rimegepant before or during pregnancy, and the other consisting of women without migraine. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~4/30/2035 0:00:00~4/23/2026 0:00:00
383909~"CDER"~"NDA"~212728.00~"PFIZER INC"~"Nurtec ODT (rimegepant) "~2/27/2020 0:00:00~"Original"~1~"3799-7"~"PMR"~"505 (o)(3)"~3799.00~7~"PMR 3799-7: A pregnancy outcomes study using a different study design than provided for in PMR 3799-6 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case-control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small-forgestational-age births in women exposed to rimegepant during pregnancy compared to an unexposed control population."~"Ongoing"~~4/30/2029 0:00:00~4/23/2026 0:00:00
383910~"CDER"~"NDA"~212728.00~"PFIZER INC"~"Nurtec ODT (rimegepant) "~2/27/2020 0:00:00~"Original"~1~"3799-8"~"PMR"~"505 (o)(3)"~3799.00~8~"PMR 3799-8: Clinical pharmacokinetic trial to evaluate the effect of a known P-gp inhibitor and BCRP inhibitor on the pharmacokinetics of rimegepant to address the potential for excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Submitted"~~9/30/2021 0:00:00~4/23/2026 0:00:00
383911~"CDER"~"NDA"~212728.00~"PFIZER INC"~"Nurtec ODT (rimegepant) "~2/27/2020 0:00:00~"Supplement"~6~"4075-2"~"PMR"~"Pediatric Research Equity Act"~4075.00~2~"PMR 4075-2: A randomized, double-blind, placebo-controlled efficacy and safety study under PREA for the preventive treatment of episodic migraine in children ages 6 through 17 years. This efficacy study must be designed to show superiority of rimegepant over placebo and is to be submitted as a special protocol assessment (SPA). 
"~"Ongoing"~"The trial is underway. Per the applicant s Annual Status Report, 119 patients have been enrolled into the trial."~4/30/2028 0:00:00~4/23/2026 0:00:00
383912~"CDER"~"NDA"~212728.00~"PFIZER INC"~"Nurtec ODT (rimegepant) "~2/27/2020 0:00:00~"Supplement"~6~"4075-3"~"PMR"~"Pediatric Research Equity Act"~4075.00~3~"PMR 4075-3: A pediatric open-label safety study under PREA to evaluate the long-term safety of the preventive treatment of episodic migraine in patients ages 6 through 17 years. This study should be a minimum of 1-year in length. 
"~"Ongoing"~"The trial is underway. Per the applicant s Annual Status Report, 79 patients have been enrolled into the trial."~4/30/2028 0:00:00~4/23/2026 0:00:00
383913~"CDER"~"NDA"~212728.00~"PFIZER INC"~"Nurtec ODT (rimegepant) "~2/27/2020 0:00:00~"Supplement"~6~"4075-4"~"PMC"~"506B PMC"~4075.00~4~"PMC 4075-4: Conduct a randomized, double-blind, placebo-controlled trial of Nurtec ODT to evaluate the efficacy of additional dosing regimens for the preventive treatment of episodic migraine. Patients should be randomized 1:1:1 to the approved dosing regimen (Nurtec ODT 75 mg every other day), more frequent dosing of Nurtec ODT 75 mg (based on the available pharmacokinetic data), or placebo. The primary efficacy endpoint will be the change from baseline in mean number of monthly migraine days during weeks 9 through 12. The study duration will be at least 3 months. 
"~"Submitted"~~3/31/2024 0:00:00~4/23/2026 0:00:00
383914~"CDER"~"NDA"~212728.00~"PFIZER INC"~"Nurtec ODT (rimegepant) "~2/27/2020 0:00:00~"Supplement"~6~"4075-5"~"PMC"~"506B PMC"~4075.00~5~"PMC 4075-5: Conduct an open-label long term safety trial lasting an additional 3 months after the controlled trial (PMC 4075-4) in at least 300 patients exposed to the more frequent dosing regimen."~"Submitted"~~3/31/2024 0:00:00~4/23/2026 0:00:00
383915~"CDER"~"NDA"~214439.00~"CMP DEVELOPMENT LLC"~"NORLIQVA (Amlodipine)"~2/24/2022 0:00:00~"Original"~1~"4239-2"~"PMR"~"Pediatric Research Equity Act"~4239.00~2~"PMR 4239-2: Conduct an open-label, randomized, single oral dose, two-treatment, twoperiod, two-sequence crossover bioequivalence and bioavailability study of amlodipine (ethanol-containing) oral solution versus amlodipine (ethanol-free) oral solution in healthy adults under fasting conditions."~"Fulfilled"~"Per FDA letter dated 02/02/2026, this PMR/PMC has been fulfilled."~11/30/2023 0:00:00~4/24/2026 0:00:00
383916~"CDER"~"NDA"~214439.00~"CMP DEVELOPMENT LLC"~"NORLIQVA (Amlodipine)"~2/24/2022 0:00:00~"Original"~1~"4239-3"~"PMR"~"Pediatric Research Equity Act"~4239.00~3~"PMR 4239-3: Conduct a dose-ranging, safety, tolerability, and efficacy study of amlodipine besylate oral solution for the treatment of hypertension in pediatric patients birth to <6 years of age. The study protocol should be agreed with the FDA prior to initiation of the study."~"Delayed"~"Currently drafting a protocol."~2/28/2029 0:00:00~4/24/2026 0:00:00
383917~"CDER"~"NDA"~214460.00~"EMERGENT BIODEFENSE OPERATIONS LANSING LLC"~"Tembexa (brincidofovir)"~6/4/2021 0:00:00~"Original"~1~"4050-3"~"PMR"~"Animal Efficacy"~4050.00~3~"PMR 4050-3: Collaborate with US public health agencies to conduct a field study to evaluate the clinical response, drug concentrations, and safety profile of brincidofovir when used for the treatment of human smallpox disease due to variola virus infection. This trial should evaluate brincidofovir vs. tecovirimat vs. brincidofovir and tecovirimat combination therapy."~"Pending"~~~7/30/2025 0:00:00
383918~"CDER"~"NDA"~214461.00~"EMERGENT BIODEFENSE OPERATIONS LANSING INC"~"Tembexa (brincidofovir)"~6/4/2021 0:00:00~"Original"~1~"4050-3"~"PMR"~"Animal Efficacy"~4050.00~3~"PMR 4050-3: Collaborate with US public health agencies to conduct a field study to evaluate the clinical response, drug concentrations, and safety profile of brincidofovir when used for the treatment of human smallpox disease due to variola virus infection. This trial should evaluate brincidofovir vs. tecovirimat vs. brincidofovir and tecovirimat combination therapy."~"Pending"~~~7/30/2025 0:00:00
383919~"CDER"~"NDA"~214487.00~"CHEMOCENTRYX INC A WHOLLY OWNED SUB OF AMGEN INC"~"Tavneos (Avacopan)"~10/7/2021 0:00:00~"Original"~1~"4155-1"~"PMR"~"505 (o)(3)"~4155.00~1~"PMR 4155-1: Conduct a randomized controlled clinical trial of at least five years duration in patients with anti-neutrophil cytoplasmic autoantibody (ANCA) associated vasculitis to evaluate safety outcomes, including hepatotoxicity and drug-induced liver injury, and serious hypersensitivity reactions, including angioedema and anaphylaxis."~"Delayed"~"The final protocol milestone date was missed because of continued discussions with the Agency. Per the applicant s annual status report, as of 9/30/2025, 21 participants have been randomized into the study."~9/30/2031 0:00:00~11/25/2025 0:00:00
383920~"CDER"~"NDA"~214487.00~"CHEMOCENTRYX INC A WHOLLY OWNED SUB OF AMGEN INC"~"Tavneos (Avacopan)"~10/7/2021 0:00:00~"Original"~1~"4155-3"~"PMC"~"506B PMC"~4155.00~3~"PMC 4155-3: Conduct a randomized controlled clinical trial of at least five years duration in patients with anti-neutrophil cytoplasmic autoantibody (ANCA) associated vasculitis to evaluate efficacy outcomes with long-term avacopan treatment."~"Delayed"~"The final protocol milestone date was missed because of continued discussions with the Agency. Per the applicant s annual status report, as of 9/30/2025, 21 participants have been randomized into the study."~9/30/2031 0:00:00~11/25/2025 0:00:00
383921~"CDER"~"NDA"~214511.00~"LUMICELL INC"~"LUMISIGHT (Pegulicianine)"~4/17/2024 0:00:00~"Original"~1~"4614-1"~"PMR"~"505 (o)(3)"~4614.00~1~"PMR 4614-1: Conduct a prospective observational study of sufficient sample size to evaluate the incidence of and characterize anaphylactic reactions to Lumisight in adults with breast cancer. A secondary study objective will be to evaluate the incidence of and characterize other serious hypersensitivity reactions. Adverse events of interest should be predefined. Study assessments should include assessment of adverse events of interest (including laboratory data as needed), physical examination, and assessment of blood pressure, heart rate, and oxygen saturation before and at multiple timepoints after Lumisight administration. Adjudicate (using an external expert panel) suspected cases of anaphylaxis and serious hypersensitivity reactions."~"Ongoing"~~5/31/2031 0:00:00~6/15/2026 0:00:00
383922~"CDER"~"NDA"~214518.00~"SIGA TECHNOLOGIES INC"~"TPOXX (Tecovirimat)"~5/18/2022 0:00:00~"Original"~1~"4278-1"~"PMR"~"Animal Efficacy"~4278.00~1~"PMR 4278-1: Collaborate with US public health agencies to conduct a field study to evaluate the clinical response, drug concentrations, and safety profile of tecovirimat when used for the treatment of human smallpox disease due to variola virus infection. This trial should evaluate tecovirimat vs. brincidofovir vs. tecovirimat and brincidofovir combination therapy."~"Pending"~~~7/16/2025 0:00:00
383923~"CDER"~"NDA"~214520.00~"CORMEDIX INC"~"Defencath (taurolidine and heparin) catheter lock solution"~11/15/2023 0:00:00~"Original"~1~"4028-1"~"PMR"~"Pediatric Research Equity Act"~4028.00~1~"PMR 4028-1: An open-label, two-arm (Defencath vs. standard of care) study to assess safety and time to CRBSI in subjects from birth to less than 18 years of age with kidney failure receiving hemodialysis via a central venous catheter."~"Delayed"~"Final Protocol was submitted after final protocol due date."~6/30/2028 0:00:00~1/9/2026 0:00:00
383924~"CDER"~"NDA"~214662.00~"MIRUM PHARMACEUTICALS INC"~"Livmarli (maralixibat)"~9/29/2021 0:00:00~"Original"~1~"4105-2"~"PMC"~"506B PMC"~4105.00~2~"PMC 4105-2: Provide a final study report capturing the comprehensive safety experience of ALGS patients treated in trial MRX-800."~"Fulfilled"~~1/31/2024 0:00:00~11/26/2025 0:00:00
383925~"CDER"~"NDA"~214662.00~"MIRUM PHARMACEUTICALS INC"~"Livmarli (maralixibat)"~9/29/2021 0:00:00~"Original"~1~"4105-3"~"PMC"~"506B PMC"~4105.00~3~"PMC 4105-3: Conduct a 5-year registry-based study to collect data on the health of patients chronically treated with Livmarli (maralixibat). Report yearly on the following safety endpoints:   Incidence of biliary diversion surgery, liver transplantation, and all-cause mortality   Assessment of growth and development   Incidence of fat-soluble vitamin deficiencies and their long-term sequelae."~"Ongoing"~~3/31/2028 0:00:00~11/26/2025 0:00:00
383926~"CDER"~"NDA"~214662.00~"MIRUM PHARMACEUTICALS INC"~"Livmarli (maralixibat)"~9/29/2021 0:00:00~"Supplement"~5~"4571-1"~"PMC"~"506B PMC"~4571.00~1~"PMC 4571-1: Provide a final study report capturing the comprehensive safety experience of progressive familial intrahepatic cholestasis (PFIC) patients treated in trial MRX-503."~"Submitted"~~6/30/2025 0:00:00~11/26/2025 0:00:00
383927~"CDER"~"NDA"~214662.00~"MIRUM PHARMACEUTICALS INC"~"Livmarli (maralixibat)"~9/29/2021 0:00:00~"Supplement"~5~"4571-2"~"PMC"~"506B PMC"~4571.00~2~"PMC 4571-2: Provide a final study report capturing the comprehensive safety experience of progressive familial intrahepatic cholestasis (PFIC) patients treated in trial MRX-801."~"Submitted"~~6/30/2025 0:00:00~11/26/2025 0:00:00
383928~"CDER"~"NDA"~214662.00~"MIRUM PHARMACEUTICALS INC"~"Livmarli (maralixibat)"~9/29/2021 0:00:00~"Supplement"~5~"4571-3"~"PMC"~"506B PMC"~4571.00~3~"PMC 4571-3: Conduct a five-year registry-based study to collect data on the health of progressive familial intrahepatic cholestasis (PFIC) patients chronically treated with Livmarli (maralixibat). Report yearly on the following endpoints: Incidence of biliary diversion surgery, liver transplantation, and allcause mortality"~"Ongoing"~~12/31/2030 0:00:00~11/26/2025 0:00:00
383929~"CDER"~"NDA"~214662.00~"MIRUM PHARMACEUTICALS INC"~"Livmarli (maralixibat)"~9/29/2021 0:00:00~"Supplement"~8~"4571-1"~"PMC"~"506B PMC"~4571.00~1~"PMC 4571-1: Provide a final study report capturing the comprehensive safety experience of progressive familial intrahepatic cholestasis (PFIC) patients treated in trial MRX-503."~"Submitted"~~6/30/2025 0:00:00~11/26/2025 0:00:00
383930~"CDER"~"NDA"~214662.00~"MIRUM PHARMACEUTICALS INC"~"Livmarli (maralixibat)"~9/29/2021 0:00:00~"Supplement"~8~"4571-2"~"PMC"~"506B PMC"~4571.00~2~"PMC 4571-2: Provide a final study report capturing the comprehensive safety experience of progressive familial intrahepatic cholestasis (PFIC) patients treated in trial MRX-801."~"Submitted"~~6/30/2025 0:00:00~11/26/2025 0:00:00
383931~"CDER"~"NDA"~215596.00~"TAKEDA PHARMACEUTICALS USA INC"~"LIVTENCITY (maribavir)"~11/23/2021 0:00:00~"Original"~1~"4182-4"~"PMR"~"505 (o)(3)"~4182.00~4~"PMR 4182-4: Conduct phenotypic analysis of maribavir against human cytomegalovirus carrying the following substitutions:. High priority: pUL97 M460I/T, A594E/P/T/V, L595F/W, C603R/W/Y. Medium priority: pUL97 A440V, V466M, A591V, E596G, K599E, C607F/Y . Low priority: o pUL97 P132L, L405P, C518Y, I610T, A613V o pUL27 P10L, N289D, H297Y, D298G, N300G, P307L, V310A, D351N, I367V o pUL54 S290R, K475S"~"Submitted"~~3/31/2025 0:00:00~1/16/2026 0:00:00
383932~"CDER"~"NDA"~215760.00~"ORPHALAN SA"~"Cuvrior (trientine tetrahydrochloride)"~4/28/2022 0:00:00~"Original"~1~"4253-1"~"PMC"~"506B PMC"~4253.00~1~"PMC 4253-1: Conduct a trial that will study the safety and efficacy of trientine tetrahydrochloride in the pediatric population to support pediatric dosing for the labeled indication supported by your study in adults (CHELATE). The trial should evaluate dosing changes using half-tablet administration to allow for dosing in young children."~"Delayed"~"The original final protocol milestone was missed (12/2022). An Acknowledge Final Protocol letter was sent on 4/2/2024."~6/30/2026 0:00:00~6/26/2026 0:00:00
383933~"CDER"~"NDA"~215793.00~"UROGEN PHARMA LTD"~"Zusduri (mitomycin)"~6/12/2025 0:00:00~"Original"~1~"4864-1"~"PMC"~"506B PMC"~4864.00~1~"PMC 4864-1: Complete the ongoing clinical trial BL011 (NCT05243550) to further characterize the clinical benefit of Zusduri (mitomycin) for the treatment of patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (NMIBC). Provide, in interim trial reports, annual updates for the duration of response (DOR) data for all patients with ongoing complete responses as of the Month 21 data cutoff date (October 2024). Annual updates should continue until all patients have either experienced recurrence of low-grade NMIBC, progression, death, been lost to followup, or reached 63 months after the first instillation as planned in the protocol, whichever occurs first."~"Pending"~~7/31/2028 0:00:00~
383934~"CDER"~"NDA"~215814.00~"RIGEL PHARMACEUTICALS INC"~"Rezlidhia (olutasidenib)"~12/1/2022 0:00:00~"Original"~1~"4371-1"~"PMR"~"Pediatric Research Equity Act"~4371.00~1~"PMR 4371-1: Conduct a clinical trial to confirm the appropriate dose of olutasidenib, and to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of  olutasidenib, in pediatric patients ages =12 to <18 years with IDH1- mutated gliomas. Patients should be followed for at least 12 months (52 weeks). Include at least 6 patients =12 to <18 years old."~"Delayed"~"The final protocol submission milestone was missed because of continued discussions with FDA."~1/31/2029 0:00:00~1/28/2026 0:00:00
383935~"CDER"~"NDA"~215814.00~"RIGEL PHARMACEUTICALS INC"~"Rezlidhia (olutasidenib)"~12/1/2022 0:00:00~"Original"~1~"4371-2"~"PMR"~"505 (o)(3)"~4371.00~2~"PMR 4371-2: Conduct a clinical drug interaction study to evaluate the effect of repeated doses of olutasidenib on the pharmacokinetics of substrates of OATP1B1. Assess the magnitude of increased drug exposure and determine appropriate dosage recommendations when olutasidenib is administered concomitantly with OATP1B1 substrates. Design and conduct the study in accordance with the FDA Guidance for Industry titled,  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Delayed"~"The final protocol submission milestone was missed because of continued discussions with FDA."~12/31/2027 0:00:00~1/28/2026 0:00:00
383936~"CDER"~"NDA"~215814.00~"RIGEL PHARMACEUTICALS INC"~"Rezlidhia (olutasidenib)"~12/1/2022 0:00:00~"Original"~1~"4371-4"~"PMC"~"506B PMC"~4371.00~4~"PMC 4371-4: Conduct a clinical drug interaction study to evaluate the effect of repeated doses of a moderate CYP3A inducer on the pharmacokinetics of olutasidenib to assess the magnitude of decreased drug exposure and determine appropriate dosage recommendations when olutasidenib is administered concomitantly with moderate CYP3A inducers. Design and conduct the study in accordance with the FDA Guidance for Industry titled,  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Fulfilled"~~6/30/2025 0:00:00~1/28/2026 0:00:00
383937~"CDER"~"NDA"~215814.00~"RIGEL PHARMACEUTICALS INC"~"Rezlidhia (olutasidenib)"~12/1/2022 0:00:00~"Original"~1~"4371-5"~"PMC"~"506B PMC"~4371.00~5~"PMC 4371-5: Conduct a clinical drug interaction study to evaluate the effect of repeated doses of olutasidenib on the pharmacokinetics of substrates of CYP1A2, 2B6, 2C8, 2C9, 2C19, and 3A. Assess the magnitude of decreased drug exposures to determine appropriate dosage recommendations when olutasidenib is administered concomitantly with CYP substrates. Design and conduct the study in accordance with the FDA Guidance for Industry titled,  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions."~"Delayed"~"The final protocol submission milestone was missed because of continued discussions with FDA."~12/31/2027 0:00:00~1/28/2026 0:00:00
383938~"CDER"~"NDA"~215830.00~"PFIZER INC"~"Litfulo (ritlecitinib)"~6/23/2023 0:00:00~"Original"~1~"4463-1"~"PMR"~"Pediatric Research Equity Act"~4463.00~1~"PMR 4463-1: Conduct the open-label long-term extension (LTE) study to evaluate the safety of ritlecitinib in pediatric subjects 6 to <12 years of age with moderate to severe alopecia areata who have completed previous ritlecitinib studies B7981031 or B7981027 and are eligible to receive ritlecitinib. Study subjects from B7981031 and placebo subjects from B7981027 will be randomly assigned to one of the two dose levels to be administered in this study and study subjects who received active drug in B7981027 will remain on the same dosage level they were assigned. All subjects will receive ritlecitinib for up to an additional 3 years"~"Delayed"~"The final protocol was submitted after the original final protocol
submission milestone."~12/31/2030 0:00:00~8/20/2025 0:00:00
383939~"CDER"~"NDA"~215830.00~"PFIZER INC"~"Litfulo (ritlecitinib)"~6/23/2023 0:00:00~"Original"~1~"4463-2"~"PMR"~"Pediatric Research Equity Act"~4463.00~2~"PMR 4463-2: Conduct a randomized, double-blind, placebo-controlled study to investigate the safety of ritlecitinib in pediatric subjects (6 to <12 years of age) with moderate to severe AA (defined by =50% scalp hair loss, measured by a SALT score of 50 or greater)."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~11/30/2027 0:00:00~8/20/2025 0:00:00
383940~"CDER"~"NDA"~215830.00~"PFIZER INC"~"Litfulo (ritlecitinib)"~6/23/2023 0:00:00~"Original"~1~"4463-3"~"PMR"~"Pediatric Research Equity Act"~4463.00~3~"PMR 4463-3: Submit the final study report for a PK study which aims to compare the systemic exposures of the dose/regimen in adults and adolescents to that of pediatric subjects ages 6 to <12 years with moderate to severe alopecia areata. Provide data on at least 12 evaluable subjects."~"Ongoing"~"The applicant did not submit an efficacy supplement including updated labeling with the final report. Original Final Report Due Date: 08/31/2024; Deferral Extension granted per FDA letter dated 4/19/2024.
"~11/30/2027 0:00:00~8/20/2025 0:00:00
383941~"CDER"~"NDA"~215596.00~"TAKEDA PHARMACEUTICALS USA INC"~"LIVTENCITY (maribavir)"~11/23/2021 0:00:00~"Original"~1~"4648-1"~"PMR"~"505 (o)(3)"~4648.00~1~"PMR 4648-1: Conduct phenotypic analysis of maribavir against human cytomegalovirus (HCMV) mutants expressing the following substitutions:   pUL97 L608P   pUL54 F2V Include evaluation of cross-resistance to ganciclovir (GCV) if not previously known as well as previously identified substitutions with a range of susceptibilities from low fold change (e.g., pUL97: H411Y) to high fold change (e.g., pUL97: C480R) as references."~"Submitted"~~5/30/2025 0:00:00~1/16/2026 0:00:00
383942~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-5"~"PMR"~"505 (o)(3)"~4608.00~5~"PMR 4608-5: Conduct comprehensive safety analyses from clinical studies that further characterize the potential serious risk of gonadal toxicity in a sufficient number of adolescent and young adult patients treated with tovorafenib. Include evaluations of pubertal development and hormonal evaluation in appropriate patients. Monitor patients for a minimum of 5 years from start of treatment. Post-treatment follow-up assessments including reproductive hormone measurements should be obtained at regularly scheduled intervals and after treatment cessation to assess time to gonadal function recovery. Include, in the interim report, a comprehensive safety analysis characterizing gonadal toxicity after all patients have been monitored for a minimum of 12 months from the start of study treatment."~"Ongoing"~~4/30/2032 0:00:00~6/12/2026 0:00:00
383943~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-6"~"PMR"~"505 (o)(3)"~4608.00~6~"PMR 4608-6: Conduct a clinical pharmacokinetic trial of tovorafenib with a strong CYP2C8 inhibitor to evaluate the potential increased drug toxicity and provide dosage recommendations for tovorafenib when used concomitantly with strong and moderate CYP2C8 inhibitors. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme-and Transporter-Mediated Drug Interactions. "~"Pending"~~4/30/2026 0:00:00~6/12/2026 0:00:00
383944~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-7"~"PMR"~"505 (o)(3)"~4608.00~7~"PMR 4608-7: Conduct a clinical pharmacokinetic trial of multi-dose tovorafenib on sensitive substrates of CYP3A4, CYP2C8, CYP1A2, CYP2B6, CYP2C9, and CYP2C19 to evaluate the potential for increased toxicity or reduced efficacy of these substrate drugs and provide appropriate drug interaction management strategies for tovorafenib when used concomitantly with these substrates. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  Clinical Drug Interaction Studies   Cytochrome P450 Enzyme-and Transporter-Mediated Drug Interactions. "~"Pending"~~12/31/2026 0:00:00~6/12/2026 0:00:00
383945~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-8"~"PMR"~"505 (o)(3)"~4608.00~8~"PMR 4608-8: Conduct a clinical pharmacokinetic trial of multi-dose tovorafenib on substrates of BCRP to evaluate the potential for increased toxicity of BCRP substrates and provide appropriate drug interaction management strategies for tovorafenib when used concomitantly with BCRP substrates. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme-and Transporter-Mediated Drug Interactions. "~"Pending"~~12/31/2026 0:00:00~6/12/2026 0:00:00
383946~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-9"~"PMC"~"506B PMC"~4608.00~9~"PMC 4608-9: Complete the FIREFLY-1 trial, including your planned additional follow-up for the 137 patients with relapsed or refractory low-grade glioma harboring a BRAF alteration enrolled to Arms 1 and 2 of FIREFLY-1 and treated with tovorafenib, to further characterize the overall response rate (ORR) and duration of response (DoR) as per Response Assessment in Pediatric Neuro-oncology (RAPNO) criteria including assessment by an independent review committee (IRC) for patients enrolled to Arm 1. Include, in the interim report, an analysis of ORR and DoR in Arm 1 as per RAPNO criteria including assessment by an IRC after all Arm 1 patients have completed a minimum of 26 cycles of treatment in the FIREFLY-1 trial."~"Ongoing"~~12/31/2027 0:00:00~6/12/2026 0:00:00
383947~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-10"~"PMC"~"506B PMC"~4608.00~10~"PMC 4608-10: Conduct a clinical pharmacokinetic trial with a strong or moderate CYP2C8 inducer with tovorafenib to evaluate the effect of a strong or moderate CYP2C8 inducer on decreasing the systemic exposure of tovorafenib and provide dosage recommendations for tovorafenib when used concomitantly with CYP2C8 inducers. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme-and TransporterMediated Drug Interactions. "~"Pending"~~4/30/2026 0:00:00~6/12/2026 0:00:00
383948~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-11"~"PMC"~"506B PMC"~4608.00~11~"PMC 4608-11: Conduct an appropriate analytical and clinical validation study to support the development of an in vitro diagnostic device using clinical trial data that demonstrates that the device is essential to the safe and effective use of tovorafenib for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation."~"Fulfilled"~~7/31/2024 0:00:00~6/12/2026 0:00:00
383949~"CDER"~"NDA"~217759.00~"PHARMING TECHNOLOGIES BV"~"Joenja (leniolisib phosphate)"~3/24/2023 0:00:00~"Original"~1~"4407-2"~"PMR"~"505 (o)(3)"~4407.00~2~"PMR 4407-2: Conduct a 2-year carcinogenicity study in rats."~"Ongoing"~~3/31/2026 0:00:00~5/21/2026 0:00:00
383950~"CDER"~"NDA"~217759.00~"PHARMING TECHNOLOGIES BV"~"Joenja (leniolisib phosphate)"~3/24/2023 0:00:00~"Original"~1~"4407-5"~"PMR"~"505 (o)(3)"~4407.00~5~"PMR 4407-5: Conduct a hepatic impairment clinical study to assess the impact of moderate and severe hepatic impairment on the pharmacokinetics of leniolisib."~"Delayed"~"The trial completion milestone was missed due to low enrollment. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 12/11/2024."~10/31/2024 0:00:00~5/21/2026 0:00:00
383951~"CDER"~"NDA"~217759.00~"PHARMING TECHNOLOGIES BV"~"Joenja (leniolisib phosphate)"~3/24/2023 0:00:00~"Original"~1~"4407-6"~"PMC"~"506B PMC"~4407.00~6~"PMC 4407-6: Submit the final clinical study report (CSR) from the ongoing open-label extension study CCDZ732201E1 with leniolisib in patients 12 years and older with Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency (APDS/PASLI) to provide long-term safety and clinical outcomes including all clinical laboratory and imaging test results, frequency of infections and antibiotic use, occurrence of adverse events, hospitalizations, and deaths."~"Submitted"~~5/31/2028 0:00:00~5/21/2026 0:00:00
383952~"CDER"~"NDA"~217779.00~"GERON CORP"~"Rytelo (imetelstat)"~6/6/2024 0:00:00~"Original"~1~"4631-1"~"PMR"~"505 (o)(3)"~4631.00~1~"PMR 4631-1: Evaluate a minimum of 25 pediatric patients, including a minimum of 6 patients to <12 years of age, enrolled in one or more molecularly targeted pediatric cancer investigations to evaluate dosing, pharmacokinetics, safety, and preliminary efficacy of imetelstat, in combination with fludarabine and cytarabine, in pediatric patients 1 year to <17 years of age with relapsed/refractory acute myeloid leukemia, myelodysplastic syndromes, or juvenile myelomonocytic leukemia."~"Pending"~~12/31/2032 0:00:00~8/5/2025 0:00:00
383953~"CDER"~"NDA"~217779.00~"GERON CORP"~"Rytelo (imetelstat)"~6/6/2024 0:00:00~"Original"~1~"4631-2"~"PMR"~"505 (o)(3)"~4631.00~2~"PMR 4631-2: Conduct a rodent carcinogenicity study in mice to evaluate the potential for carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Pending"~~9/30/2027 0:00:00~8/5/2025 0:00:00
383954~"CDER"~"NDA"~212819.00~"MERCK SHARP AND DOHME CORP A SUB OF MERCK AND CO INC"~"Recarbrio (imipenem/cilastatin/relebactam)"~7/16/2019 0:00:00~"Supplement"~2~"3865-1"~"PMR"~"Pediatric Research Equity Act"~3865.00~1~"PMR 3865-1: Conduct a randomized, open-label, active controlled trial to evaluate the safety and tolerability of imipenem, cilastatin and relebactam in children from birth to less than 18 years of age with complicated urinary tract infections, complicated intra-abdominal infections and hospital- acquired bacterial pneumonia or ventilator-associated bacterial pneumonia."~"Fulfilled"~"Per FDA letter dated 12/09/2025, this PMR/PMC has been fulfilled. 
"~2/28/2025 0:00:00~9/8/2025 0:00:00
383955~"CDER"~"NDA"~212819.00~"MERCK SHARP AND DOHME CORP A SUB OF MERCK AND CO INC"~"Recarbrio (imipenem/cilastatin/relebactam)"~7/16/2019 0:00:00~"Supplement"~2~"3865-2"~"PMR"~"505 (o)(3)"~3865.00~2~"PMR 3865-2: Conduct a United States surveillance study for 5 years from the date of marketing to determine if resistance to imipenem, cilastatin and relebactam has developed in organisms specific to the indications in the label."~"Fulfilled"~~12/31/2024 0:00:00~9/8/2025 0:00:00
383956~"CDER"~"NDA"~212839.00~"SK LIFE SCIENCE INC"~"Xcopri (cenobamate) "~11/21/2019 0:00:00~"Original"~1~"3712-4"~"PMR"~"Pediatric Research Equity Act"~3712.00~4~"PMR 3712-4: A study to evaluate the pharmacokinetics, safety, and tolerability of an age-appropriate formulation of cenobamate in children ages 1 month to less than 2 years with partial-onset seizures. This study should identify doses to be used in the efficacy and long-term extension studies for children 1 month to less than 2 years of age."~"Delayed"~"Deferral Extension Requested 12/15/2025. Denied per FDA letter dated 04/24/2026."~12/31/2030 0:00:00~5/8/2026 0:00:00
383957~"CDER"~"NDA"~212839.00~"SK LIFE SCIENCE INC"~"Xcopri (cenobamate) "~11/21/2019 0:00:00~"Original"~1~"3712-5"~"PMR"~"Pediatric Research Equity Act"~3712.00~5~"PMR 3712-5: A prospective, randomized, controlled, double-blinded, efficacy and safety study of cenobamate for the treatment of partial-onset seizures in children from 1 month to less than 2 years of age. The primary efficacy endpoint during the controlled phase will examine seizure frequency based upon video/electroencephalographic data. The placebo and drug treatment groups will be compared by inferential statistical methods to identify a treatment effect."~"Delayed"~"Deferral Extension Requested 12/15/2025. Denied per FDA letter dated 04/24/2026."~12/31/2030 0:00:00~5/8/2026 0:00:00
383958~"CDER"~"NDA"~212839.00~"SK LIFE SCIENCE INC"~"Xcopri (cenobamate) "~11/21/2019 0:00:00~"Original"~1~"3712-6"~"PMR"~"Pediatric Research Equity Act"~3712.00~6~"PMR 3712-6: Long-term safety study of cenobamate in the treatment of partial-onset seizures in children from 1 month to 2 years of age. Routine safety measures should be monitored. Behavioral and cognitive endpoints should be included."~"Pending"~"Deferral Extension Requested 12/15/2026. Denied per FDA letter dated 04/24/2026."~12/31/2030 0:00:00~5/8/2026 0:00:00
383959~"CDER"~"NDA"~212839.00~"SK LIFE SCIENCE INC"~"Xcopri (cenobamate) "~11/21/2019 0:00:00~"Original"~1~"3712-7"~"PMR"~"Pediatric Research Equity Act"~3712.00~7~"PMR 3712-7: A study to evaluate the pharmacokinetics, safety, and tolerability of an age appropriate formulation of cenobamate to determine a dosing regimen as therapy for partial-onset seizures in children ages 2 years to 17 years of age that provides drug exposure that is similar to the exposure that is effective in adult patients with partial-onset seizures. This analysis will require pharmacokinetic data from studies of both the adult and pediatric patients."~"Delayed"~"The Trial Completion and Interim Report Submission milestone dates were missed. Original Final Report Due Date: 11/30/2025; Deferral Extension granted per FDA letter dated 05/17/2023 due to significant challenges in recruitment and enrollment of pediatric patients."~11/30/2026 0:00:00~5/8/2026 0:00:00
383960~"CDER"~"NDA"~212839.00~"SK LIFE SCIENCE INC"~"Xcopri (cenobamate) "~11/21/2019 0:00:00~"Original"~1~"3712-8"~"PMR"~"Pediatric Research Equity Act"~3712.00~8~"PMR 3712-8: The efficacy of cenobamate in children ages 2 years to 17 years of age for the treatment of partial-onset seizures will be addressed by a report demonstrating matched exposure to that in adults supporting pediatric extrapolation."~"Delayed"~"The Trial Completion milestone date was missed. Original Final Report Due Date: 11/30/2025; Deferral Extension granted per FDA letter dated 05/17/2023 due to significant challenges in recruitment and enrollment of pediatric patients."~11/30/2026 0:00:00~5/8/2026 0:00:00
383961~"CDER"~"NDA"~212839.00~"SK LIFE SCIENCE INC"~"Xcopri (cenobamate) "~11/21/2019 0:00:00~"Original"~1~"3712-9"~"PMR"~"Pediatric Research Equity Act"~3712.00~9~"PMR 3712-9: An open-label long term safety and tolerability study of cenobamate in children ages 2 years to 17 years of age."~"Delayed"~"The Trial Completion milestone date was missed. Original Final Report Due Date: 11/30/2025; Deferral Extension granted per FDA letter dated 05/17/2023 due to significant challenges in recruitment and enrollment of pediatric patients."~11/30/2026 0:00:00~5/8/2026 0:00:00
383962~"CDER"~"NDA"~212839.00~"SK LIFE SCIENCE INC"~"Xcopri (cenobamate) "~11/21/2019 0:00:00~"Original"~1~"3712-10"~"PMR"~"505 (o)(3)"~3712.00~10~"PMR 3712-10: Conduct a pregnancy outcomes study using a different study design than provided for in the North American Antiepileptic Drug (NAAED) Pregnancy Registry (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case-control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Xcopri (cenobamate) during pregnancy compared to an unexposed control population."~"Ongoing"~~7/31/2032 0:00:00~5/8/2026 0:00:00
383963~"CDER"~"NDA"~212854.00~"ZMI PHARMA INC"~"Zimhi (naloxone hydrochloride)"~10/15/2021 0:00:00~"Original"~1~"4153-1"~"PMR"~"505 (o)(3)"~4153.00~1~"PMR 4153-1: Conduct a study to complete testing which evaluates the combination product reliability of successful injection of Zimhi."~"Delayed"~"The final protocols and final reports have not been submitted and the study completion date milestones have been missed. FDA issued a missed milestone letter on 06/11/2026."~10/31/2023 0:00:00~12/15/2025 0:00:00
383964~"CDER"~"NDA"~212854.00~"ZMI PHARMA INC"~"Zimhi (naloxone hydrochloride)"~10/15/2021 0:00:00~"Original"~1~"4153-2"~"PMR"~"505 (o)(3)"~4153.00~2~"PMR 4153-2: Conduct a study of needlestick injuries associated with the use of Zimhi. Provide a detailed analysis of incidents (including reported incidents that did, as well as did not, result in patient and/or provider harm), full event narratives of the incidents and any subsequent adverse events, and the results of root cause analysis performed for the reported event."~"Delayed"~"The final protocols and final reports have not been submitted and the study completion date milestones have been missed. FDA issued a missed milestone letter on 06/11/2026."~10/31/2025 0:00:00~12/15/2025 0:00:00
383965~"CDER"~"NDA"~212860.00~"ROCKWELL MEDICAL INC"~"TRIFERIC AVNU (ferric pyrophosphate citrate)"~3/27/2020 0:00:00~"Original"~1~"3830-1"~"PMR"~"Pediatric Research Equity Act"~3830.00~1~"PMR 3830-1: Efficacy and safety trial of Triferic via hemodialysate and Triferic AVNU intravenously via pre-dialyzer infusion Line or via post-dialyzer infusion line in pediatric patients aged less than 18 years with hemodialysisdependent chronic kidney disease."~"Ongoing"~"16 patients have been enrolled and 1 patient is ongoing in the trial."~12/31/2022 0:00:00~5/26/2022 0:00:00
383966~"CDER"~"NDA"~212862.00~"MYLAN IRELAND LTD"~"Pretomanid Tablets"~8/14/2019 0:00:00~"Original"~1~"3682-2"~"PMR"~"505 (o)(3)"~3682.00~2~"PMR 3682-2: Conduct a global surveillance study for a five-year period after the introduction of Pretomanid Tablets to the market to   monitor changes in M. tuberculosis susceptibility to pretomanid."~"Fulfilled"~~12/31/2025 0:00:00~10/10/2025 0:00:00
383967~"CDER"~"NDA"~214662.00~"MIRUM PHARMACEUTICALS INC"~"Livmarli (maralixibat)"~9/29/2021 0:00:00~"Supplement"~8~"4571-3"~"PMC"~"506B PMC"~4571.00~3~"PMC 4571-3: Conduct a five-year registry-based study to collect data on the health of progressive familial intrahepatic cholestasis (PFIC) patients chronically treated with Livmarli (maralixibat). Report yearly on the following endpoints: Incidence of biliary diversion surgery, liver transplantation, and allcause mortality"~"Ongoing"~~12/31/2030 0:00:00~11/26/2025 0:00:00
383968~"CDER"~"NDA"~214665.00~"AMGEN INC"~"LUMAKRAS (sotorasib)"~5/28/2021 0:00:00~"Original"~1~"4071-6"~"PMR"~"Accelerated Approval"~4071.00~6~"PMR 4071-6: Complete a multicenter, randomized clinical trial intended to verify and describe the clinical benefit of sotorasib in patients with locally advanced or metastatic non-small cell lung cancer and whose tumors harbor Kirsten rat sarcoma (KRAS) G12C mutation. The primary endpoint(s) will be progression free survival as assessed by a Blinded Independent Review Committee and/or overall survival.
"~"Ongoing"~"335 patients out of the planned 750 patients have been enrolled into the trial."~6/30/2028 0:00:00~7/22/2025 0:00:00
383969~"CDER"~"NDA"~214679.00~"AZURITY PHARMACEUTICALS INC"~"Eprontia (topiramate)"~11/5/2021 0:00:00~"Original"~1~"4169-1"~"PMR"~"Pediatric Research Equity Act"~4169.00~1~"PMR 4169-1: A randomized, double-blind, placebo-controlled efficacy and safety study under PREA to evaluate topiramate oral solution for the preventive treatment of migraine in children 6 through 11 years of age. This efficacy study must be designed to demonstrate superiority of topiramate oral solution over placebo."~"Delayed"~"The trial completion and final report submission milestones were missed because CRO identification activities are ongoing."~12/31/2025 0:00:00~12/29/2025 0:00:00
383970~"CDER"~"NDA"~214679.00~"AZURITY PHARMACEUTICALS INC"~"Eprontia (topiramate)"~11/5/2021 0:00:00~"Original"~1~"4169-2"~"PMR"~"Pediatric Research Equity Act"~4169.00~2~"PMR 4169-2: A pharmacokinetic analysis to determine the dosing regimen that provides similar drug exposures (at levels demonstrated to be effective in adults with partial-onset seizures) in pediatric patients 1 month to less than 2 years of age and in adult patients. This analysis will require pharmacokinetic data from both the adult and pediatric (1 month to less than 2 years of age) populations."~"Delayed"~"Original Final Report Due Date: 10/31/2025; Deferral Extension granted per FDA letter dated 02/18/2026. The study completion milestone was also revised.
"~10/31/2027 0:00:00~12/29/2025 0:00:00
383984~"CDER"~"NDA"~215833.00~"NOVARTIS PHARMACEUTICALS CORP"~"Pluvicto (lutetium (177Lu) vipivotide tetraxetan)"~3/23/2022 0:00:00~"Original"~1~"4241-3"~"PMC"~"506B PMC"~4241.00~3~"PMC 4241-3: Conduct a clinical trial to evaluate the efficacy and safety of lutetium (177Lu) vipivotide tetraxetan in patients with advanced/metastatic prostate cancer who have at least one lesion with PSMA expression higher than that in normal liver parenchyma on PSMA-11 PET scan and at least one lesion with PSMA expression less than or equal to uptake in normal liver, with the following size criteria in short axis: size criteria in short axis: organs >1 cm, lymph nodes >2.5 cm, bones (soft tissue component) >1 cm. Alternatively, add cohorts of these patients to ongoing trials. Include an analysis of these safety and efficacy data."~"Ongoing"~~8/31/2026 0:00:00~5/19/2026 0:00:00
383985~"CDER"~"NDA"~215833.00~"NOVARTIS PHARMACEUTICALS CORP"~"Pluvicto (lutetium (177Lu) vipivotide tetraxetan)"~3/23/2022 0:00:00~"Supplement"~21~"4824-1"~"PMC"~"506B PMC"~4824.00~1~"PMC 4824-1: Conduct an image-based dosimetry study calculating the radiation absorbed dose to the bone marrow resulting from treatment with 177Lu-PSMA-617, using the scans collected from the VISION sub-study; considering all the activity in the organs, tissues, and lesions; and following the methodology discussed with the FDA. Provide the average value for all patients and for subgroups of patients with high and low disease burden in bone including categories of unaffected and affected red marrow based on vertebra with no isolated lesions and extensive lesion activity. Also calculate the absorbed dose to the osteogenic cells."~"Fulfilled"~~9/30/2025 0:00:00~5/19/2026 0:00:00
383986~"CDER"~"NDA"~215833.00~"NOVARTIS PHARMACEUTICALS CORP"~"Pluvicto (lutetium (177Lu) vipivotide tetraxetan)"~3/23/2022 0:00:00~"Supplement"~24~"4824-1"~"PMC"~"506B PMC"~4824.00~1~"PMC 4824-1: Conduct an image-based dosimetry study calculating the radiation absorbed dose to the bone marrow resulting from treatment with 177Lu-PSMA-617, using the scans collected from the VISION sub-study; considering all the activity in the organs, tissues, and lesions; and following the methodology discussed with the FDA. Provide the average value for all patients and for subgroups of patients with high and low disease burden in bone including categories of unaffected and affected red marrow based on vertebra with no isolated lesions and extensive lesion activity. Also calculate the absorbed dose to the osteogenic cells."~"Fulfilled"~~9/30/2025 0:00:00~5/19/2026 0:00:00
383987~"CDER"~"NDA"~215842.00~"NOVO NORDISK INC"~"Rivfloza (nedosiran)"~9/29/2023 0:00:00~"Original"~1~"4522-1"~"PMR"~"505 (o)(3)"~4522.00~1~"PMR 4522-1: Complete a 26-week GLP carcinogenicity study of nedosiran and murine surrogate (DCR-m355) by subcutaneous injection in TgRasH2 mice."~"Fulfilled"~~1/31/2025 0:00:00~11/24/2025 0:00:00
383988~"CDER"~"NDA"~215842.00~"NOVO NORDISK INC"~"Rivfloza (nedosiran)"~9/29/2023 0:00:00~"Original"~1~"4522-2"~"PMR"~"505 (o)(3)"~4522.00~2~"PMR 4522-2: Complete a long-term (2-year) GLP carcinogenicity study of nedosiran by subcutaneous injection in one rodent species. (To be conducted after results are available from a dose-range finding study.)"~"Ongoing"~~1/31/2029 0:00:00~11/24/2025 0:00:00
383989~"CDER"~"NDA"~217294.00~"SQ INNOVATION INC"~"Lasix ONYU (furosemide injection)"~10/7/2025 0:00:00~"Original"~1~"4701-1"~"PMC"~"506B PMC"~4701.00~1~"PMC 4701-1: To evaluate the post-market occurrence rate for [...], provide the following: a. The occurrence rate for [...] for the combination product. The occurrence rate should be provided as raw data per quarter (i.e., the number of occurrences in Q1, Q2, etc.) and cumulatively (i.e., total occurrence rate and number over all quarters) in addition to the total number of devices distributed at the time of the report (segmented into quarters). These data should include [...] observed both internally throughout the manufacturing process (e.g., release testing) and from devices distributed in the field presented separately from one another. b. Updates on the investigation including root causes and corrective actions implemented to reduce the occurrence rate for [...]. The post-market reports as outlined above should be submitted quarterly starting from start of distribution of the final finished combination product through 5 years."~"Pending"~~1/31/2031 0:00:00~
383990~"CDER"~"NDA"~217369.00~"BIOGEN INC"~"Zurzuvae (zuranolone)"~8/4/2023 0:00:00~"Original"~2~"4484-1"~"PMR"~"Pediatric Research Equity Act"~4484.00~1~"PMR 4484-1: Conduct a pharmacokinetic and safety assessment for zuranolone in postpubertal females 15 to 17 years of age."~"Submitted"~"The final report was submitted to FDA on 6/11/2025"~6/30/2025 0:00:00~10/3/2025 0:00:00
383991~"CDER"~"NDA"~217369.00~"BIOGEN INC"~"Zurzuvae (zuranolone)"~8/4/2023 0:00:00~"Original"~2~"4484-2"~"PMR"~"505 (o)(3)"~4484.00~2~"PMR 4484-2: Conduct an embryofetal development study in a second species."~"Fulfilled"~~2/28/2025 0:00:00~10/3/2025 0:00:00
383992~"CDER"~"NDA"~217388.00~"ASTRAZENECA AB"~"Wainua (eplontersen)"~12/21/2023 0:00:00~"Original"~1~"4564-1"~"PMR"~"505 (o)(3)"~4564.00~1~"PMR 4564-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to eplontersen during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~12/31/2036 0:00:00~2/10/2026 0:00:00
383993~"CDER"~"NDA"~217388.00~"ASTRAZENECA AB"~"Wainua (eplontersen)"~12/21/2023 0:00:00~"Original"~1~"4564-4"~"PMR"~"505 (o)(3)"~4564.00~4~"PMR 4564-4: Evaluate the incidence and provide analyses of glomerulonephritis, thrombocytopenia, and atrioventricular block observed in the placebo-controlled study, ION-682884-CST, of eplontersen in adult hereditary and wild-type ATTR cardiomyopathy patients."~"Delayed"~"The trial completion milestone was missed, because the original trial timeline was established when the strategy was to stop the trial based on potential early closure timepoints. After receiving feedback from the FDA, the trial will now be completed when the last patient completes the final visit, which is the protocol specified end of full treatment period. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 12/8/2025."~10/31/2026 0:00:00~2/10/2026 0:00:00
383994~"CDER"~"NDA"~217417.00~"MUNDIPHARMA GMBH"~"Rezzayo (rezafungin)"~3/22/2023 0:00:00~"Original"~1~"4416-1"~"PMR"~"505 (o)(3)"~4416.00~1~"PMR 4416-1: Conduct a United States surveillance study for a five-year period after the introduction of rezafungin to the market to monitor changes in susceptibility of Candida species to rezafungin."~"Delayed"~"The Second interim report submission was missed and the FDA issued a missed milestone letter on 08/05/2025."~9/30/2028 0:00:00~5/20/2026 0:00:00
383995~"CDER"~"NDA"~217469.00~"HARROW EYE LLC"~"Vevye (cyclosporine)"~5/30/2023 0:00:00~"Original"~1~"4454-1"~"PMR"~"505 (o)(3)"~4454.00~1~"PMR 4454-1: Conduct a randomized, controlled trial to evaluate the corneal endothelial health of eyes treated with cyclosporine ophthalmic solution, 0.1% by monitoring the number/density of corneal endothelial cells using specular microscopy at baseline and over a period of at least one year in at least 100 patients receiving cyclosporine ophthalmic solution, 0.1%."~"Ongoing"~~12/31/2026 0:00:00~7/29/2024 0:00:00
383996~"CDER"~"NDA"~217470.00~"INDIVIOR INC"~"Opvee (nalmefene hydrochloride)"~5/22/2023 0:00:00~"Original"~1~"4451-1"~"PMR"~"Pediatric Research Equity Act"~4451.00~1~"PMR 4451-1: Conduct a clinical pharmacokinetic, pharmacodynamic, and safety study of Opvee in pediatric patients aged 3 to less than 12 years of age."~"Delayed"~"The final protocol submission milestone was missed. The applicant is in discussion with the agency."~9/30/2027 0:00:00~7/18/2025 0:00:00
383997~"CDER"~"NDA"~217470.00~"INDIVIOR INC"~"Opvee (nalmefene hydrochloride)"~5/22/2023 0:00:00~"Original"~1~"4451-2"~"PMR"~"Pediatric Research Equity Act"~4451.00~2~"PMR 4451-2: Conduct a clinical pharmacokinetic, pharmacodynamic, and safety study of Opvee in pediatric patients from birth to less than 3 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~3/31/2029 0:00:00~7/18/2025 0:00:00
383998~"CDER"~"NDA"~217470.00~"INDIVIOR INC"~"Opvee (nalmefene hydrochloride)"~5/22/2023 0:00:00~"Original"~1~"4451-3"~"PMR"~"Pediatric Research Equity Act"~4451.00~3~"PMR 4451-3: Conduct a juvenile animal study in rats to support the initiation of clinical studies in pediatric patients from 3 to less than 12 years of age. This study will evaluate the effect of the drug on growth and development, specifically reproductive performance/sexual maturation, local tissues including the nasal and respiratory tract, immune capacity, and central nervous system histopathology and long-term behavioral effects."~"Submitted"~"The final report was submitted to FDA on 3/27/2025"~2/28/2025 0:00:00~7/18/2025 0:00:00
383999~"CDER"~"NDA"~217470.00~"INDIVIOR INC"~"Opvee (nalmefene hydrochloride)"~5/22/2023 0:00:00~"Original"~1~"4451-4"~"PMR"~"Pediatric Research Equity Act"~4451.00~4~"PMR 4451-4: Conduct a juvenile animal study in rats to support the initiation of clinical studies in pediatric patients from birth to less than 3 years of age. This study will evaluate the effect of the drug on growth and development, specifically reproductive performance/sexual maturation, local tissues including the nasal and respiratory tract, immune capacity, and central nervous system histopathology and long-term behavioral effects."~"Submitted"~"The final report was submitted to FDA on 07/10/2025."~3/31/2025 0:00:00~7/18/2025 0:00:00
384000~"CDER"~"NDA"~217470.00~"INDIVIOR INC"~"Opvee (nalmefene hydrochloride)"~5/22/2023 0:00:00~"Original"~1~"4451-5"~"PMR"~"505 (o)(3)"~4451.00~5~"PMR 4451-5: Conduct a fertility and early embryonic development study in rats with dodecylmaltoside (DDM)."~"Submitted"~~4/30/2025 0:00:00~7/18/2025 0:00:00
384001~"CDER"~"NDA"~217470.00~"INDIVIOR INC"~"Opvee (nalmefene hydrochloride)"~5/22/2023 0:00:00~"Original"~1~"4451-6"~"PMR"~"505 (o)(3)"~4451.00~6~"PMR 4451-6: Conduct an embryo-fetal development study in rats with dodecylmaltoside (DDM)."~"Submitted"~~8/31/2024 0:00:00~7/18/2025 0:00:00
384002~"CDER"~"NDA"~217470.00~"INDIVIOR INC"~"Opvee (nalmefene hydrochloride)"~5/22/2023 0:00:00~"Original"~1~"4451-7"~"PMR"~"505 (o)(3)"~4451.00~7~"PMR 4451-7: Conduct an embryo-fetal development study in rabbits with dodecylmaltoside (DDM)."~"Submitted"~~3/31/2025 0:00:00~7/18/2025 0:00:00
384003~"CDER"~"NDA"~217470.00~"INDIVIOR INC"~"Opvee (nalmefene hydrochloride)"~5/22/2023 0:00:00~"Original"~1~"4451-8"~"PMR"~"505 (o)(3)"~4451.00~8~"PMR 4451-8: Conduct a pre- and postnatal development study in rats with dodecylmaltoside (DDM)."~"Submitted"~~7/31/2025 0:00:00~7/18/2025 0:00:00
384004~"CDER"~"NDA"~217512.00~"BAXTER HEALTHCARE CORP"~"Pantoprazole Sodium in 0.9% Sodium Chloride Injection"~2/14/2024 0:00:00~"Original"~1~"4578-1"~"PMR"~"Pediatric Research Equity Act"~4578.00~1~"PMR 4578-1: Conduct a study to characterize the pharmacokinetics and safety of shortterm (at least 4 days) Pantoprazole Sodium in 0.9% Sodium Chloride Injection in pediatric patients aged 1 month to less than 18 years of age requiring treatment for gastroesophageal reflux disease, including patients with a history of erosive esophagitis who require acid suppression therapy and are unable to tolerate oral therapy."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2034 0:00:00~4/6/2026 0:00:00
384005~"CDER"~"NDA"~212862.00~"MYLAN IRELAND LTD"~"Pretomanid Tablets"~8/14/2019 0:00:00~"Original"~1~"3682-3"~"PMR"~"505 (o)(3)"~3682.00~3~"PMR 3682-3: Conduct a study to evaluate pharmacokinetics and safety of Pretomanid Tablets in subjects with renal impairment."~"Delayed"~"The study completion and final report submission milestones were missed. On July 08, 2024, FDA issued an Ack revised PMR and communicate good cause letter."~12/31/2021 0:00:00~10/10/2025 0:00:00
384006~"CDER"~"NDA"~212862.00~"MYLAN IRELAND LTD"~"Pretomanid Tablets"~8/14/2019 0:00:00~"Original"~1~"3682-4"~"PMR"~"505 (o)(3)"~3682.00~4~"PMR 3682-4: Conduct a study to evaluate pharmacokinetics and safety of Pretomanid Tablets in subjects with mild, moderate, and severe hepatic impairment."~"Delayed"~"The study completion and final report milestones were missed because of additional time needed to address significant challenges with patient recruitment. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 01/14/2026."~6/30/2021 0:00:00~10/10/2025 0:00:00
384007~"CDER"~"NDA"~212887.00~"VIIV HEALTHCARE CO"~"Vocabria (cabotegravir)"~1/21/2021 0:00:00~"Original"~1~"3997-2"~"PMR"~"Pediatric Research Equity Act"~3997.00~2~"PMR 3997-2: Conduct a study in subjects weighing 25 kg to less than 35 kg (approximately 6 to less than 12 years of age) who are HIV-1 infected, virologically suppressed (HIV-1 RNA <50 copies/mL) and on a stable antiretroviral regimen at the time of enrollment, to assess the pharmacokinetics, tolerability, and short-term safety of VOCABRIA after 4-week administration in combination with other antiretroviral drug(s)."~"Delayed"~"Original Final Report Due Date: 12/31/2023. Per FDA letter dated 6/27/2022, final report due date extended to 06/30/2026. Second Deferral Extension Granted Per FDA letter dated 05/05/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~2/17/2026 0:00:00
384008~"CDER"~"NDA"~212887.00~"VIIV HEALTHCARE CO"~"Vocabria (cabotegravir)"~1/21/2021 0:00:00~"Original"~1~"3997-3"~"PMR"~"Pediatric Research Equity Act"~3997.00~3~"PMR 3997-3: Conduct a study in subjects weighing 10 kg to less than 25 kg (approximately 2 to less than 6 years of age) who are HIV-1 infected, virologically suppressed (HIV-1 RNA <50 copies/mL) and on a stable antiretroviral regimen at the time of enrollment, to assess the pharmacokinetics, tolerability, and short-term safety of VOCABRIA after 4-week administration in combination with other antiretroviral drug(s)."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/05/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~2/17/2026 0:00:00
384009~"CDER"~"NDA"~212887.00~"VIIV HEALTHCARE CO"~"Vocabria (cabotegravir)"~1/21/2021 0:00:00~"Supplement"~1~"4223-6"~"PMR"~"Pediatric Research Equity Act"~4223.00~6~"PMR 4223-6: Conduct a study in subjects weighing 25 kg to less than 35 kg (approximately 6 to less than 12 years of age) who are HIV-1 infected, virologically suppressed (HIV-1 RNA <50 copies/mL) and on a stable antiretroviral regimen at the time of enrollment, to assess the pharmacokinetics, tolerability, and short-term safety of VOCABRIA after 4-week administration in combination with other antiretroviral drug(s)."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/05/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~2/17/2026 0:00:00
384010~"CDER"~"NDA"~212887.00~"VIIV HEALTHCARE CO"~"Vocabria (cabotegravir)"~1/21/2021 0:00:00~"Supplement"~1~"4223-7"~"PMR"~"Pediatric Research Equity Act"~4223.00~7~"PMR 4223-7: Conduct a study in subjects weighing 10 kg to less than 25 kg (approximately 2 to less than 6 years of age) who are HIV-1 infected, virologically suppressed (HIV-1 RNA <50 copies/mL) and on a stable antiretroviral regimen at the time of enrollment, to assess the pharmacokinetics, tolerability, and short-term safety of VOCABRIA after 4- week administration in combination with other antiretroviral drug(s)."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/05/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~2/17/2026 0:00:00
384011~"CDER"~"NDA"~212887.00~"VIIV HEALTHCARE CO"~"Vocabria (cabotegravir)"~1/21/2021 0:00:00~"Supplement"~4~"4196-1"~"PMR"~"505 (o)(3)"~4196.00~1~"PMR 4196-1: Conduct a study to collect and analyze data from adults and adolescents who take cabotegravir extended-release injectable suspension (CAB LA) for pre-exposure prophylaxis (PrEP) of sexually acquired HIV -1 infection and who become infected during PrEP use or within one year of discontinuing PrEP. As part of this study, collect and provide information on adherence, risk factors for non-adherence and HIV-1 acquisition. To compare tolerability and rates of HIV-1 acquisition among individuals who initiate CAB LA with an oral lead-in to those who directly initiate CAB LA injections, the following should be included in the study report: a) Frequency of testing and methods used for HIV -1 diagnosis. b) Frequency of prevalent HIV-1 infections that were detected, including those captured during screening. c) Resistance analyses of viral isolates from those who acquire HIV-1, including a description of the methodologies used to evaluate resistance. d) Description of subsequent antiretroviral therapy, including the time required to achieve virologic suppression and the durability of the response (to cover 48-weeks from the time therapy is initiated, at minimum). e) Information on adherence, select adverse events and treatment discontinuations."~"Ongoing"~~3/31/2028 0:00:00~2/17/2026 0:00:00
384012~"CDER"~"NDA"~212888.00~"VIIV HEALTHCARE CO"~"CABENUVA (cabotegravir extended release injectable suspension; rilpivirine extended release injectable suspension)"~1/21/2021 0:00:00~"Original"~1~"3998-2"~"PMR"~"Pediatric Research Equity Act"~3998.00~2~"PMR 3998-2: Conduct a study in subjects weighing 25 kg to less than 35 kg (approximately 6 to less than 12 years of age) who are HIV-1 infected, virologically suppressed (HIV-1 RNA <50 copies/mL) and on a stable antiretroviral regimen at the time of enrollment, to assess the pharmacokinetics, safety and tolerability, and antiviral activity of CABENUVA. Study participants must be monitored for a minimum of 24 weeks to assess safety and durability of antiviral response."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/05/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~2/17/2026 0:00:00
384013~"CDER"~"NDA"~212888.00~"VIIV HEALTHCARE CO"~"CABENUVA (cabotegravir extended release injectable suspension; rilpivirine extended release injectable suspension)"~1/21/2021 0:00:00~"Original"~1~"3998-3"~"PMR"~"Pediatric Research Equity Act"~3998.00~3~"PMR 3998-3: Conduct a study in subjects weighing 10 kg to less than 25 kg (approximately 2 to less than 6 years of age) who are HIV-1 infected, virologically suppressed (HIV-1 RNA <50 copies/mL) and on a stable antiretroviral regimen at the time of enrollment, to assess the pharmacokinetics, safety and tolerability, and antiviral activity of CABENUVA. Study participants must be monitored for a minimum of 24 weeks to assess safety and durability of antiviral response."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/05/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~2/17/2026 0:00:00
384014~"CDER"~"NDA"~214783.00~"KADMON PHARMACEUTICALS LLC"~"REZUROCK (Belumosudil)"~7/16/2021 0:00:00~"Original"~1~"4106-3"~"PMR"~"505 (o)(3)"~4106.00~3~"PMR 4106-3: Conduct an integrated safety analysis using data obtained from clinical trials to further characterize the safety of long-term treatment with belumosudil and determine the rate of infections, hypertension and other adverse events. The integrated safety analysis should include all adverse events, major safety events, dose-reductions, dose interruptions, withdrawals, and efficacy when all patients have completed at least three years of treatment with belumosudil or withdrew earlier."~"Fulfilled"~~2/28/2023 0:00:00~9/8/2025 0:00:00
384015~"CDER"~"NDA"~214783.00~"KADMON PHARMACEUTICALS LLC"~"REZUROCK (Belumosudil)"~7/16/2021 0:00:00~"Original"~1~"4106-4"~"PMR"~"505 (o)(3)"~4106.00~4~"PMR 4106-4: Conduct an in vitro mechanism-based inhibition study (such as the two-step dilution method) estimating the inactivation parameters (kinact and KI) of CYP1A2, CYP2C19 and CYP2D6 enzymes and measuring nonspecific binding of belumosudil to assess the potential of drug interaction with belumosudil on these enzymes in accordance with the FDA Guidance for Industry titled  In Vitro Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Fulfilled"~~6/30/2022 0:00:00~9/8/2025 0:00:00
384016~"CDER"~"NDA"~214783.00~"KADMON PHARMACEUTICALS LLC"~"REZUROCK (Belumosudil)"~7/16/2021 0:00:00~"Original"~1~"4106-6"~"PMR"~"505 (o)(3)"~4106.00~6~"PMR 4106-6: Conduct a rodent carcinogenicity study in rats to evaluate the potential for carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Delayed"~"The applicant requested revised milestones because additional time is needed to complete the study according to the final protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 07/24/2025."~6/30/2025 0:00:00~9/8/2025 0:00:00
384017~"CDER"~"NDA"~214783.00~"KADMON PHARMACEUTICALS LLC"~"REZUROCK (Belumosudil)"~7/16/2021 0:00:00~"Original"~1~"4106-10"~"PMR"~"505 (o)(3)"~4106.00~10~"PMR 4106-10: Conduct a thorough QT/QTc trial to evaluate the effect of repeat doses of belumosudil on the QT/QTc interval to address the potential for excessive drug toxicity. This trial should be designed and conducted in accordance with the FDA Guidance for Industry titled, E14 Clinical Evaluation of QT/QTc and E14 Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non-Antiarrhythmic Drugs Questions and Answers (R3)."~"Fulfilled"~~12/31/2021 0:00:00~9/8/2025 0:00:00
384018~"CDER"~"NDA"~214783.00~"KADMON PHARMACEUTICALS LLC"~"REZUROCK (Belumosudil)"~7/16/2021 0:00:00~"Original"~1~"4106-11"~"PMR"~"505 (o)(3)"~4106.00~11~"PMR 4106-11: Conduct a clinical trial in a sufficient number of Black patients with chronic graft versus host disease to assess the risk of cardiac toxicities and further characterize Grade 3 toxicities including gastrointestinal and vascular disorders associated with the use of belumosudil. This study should characterize the exposure (including PK data), safety, and efficacy of belumosudil."~"Delayed"~"The trial completion and final report submission milestones were missed because of enrollment challenges and continued discussion with the division. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 01/28/2026."~12/31/2025 0:00:00~9/8/2025 0:00:00
384019~"CDER"~"NDA"~214783.00~"KADMON PHARMACEUTICALS LLC"~"REZUROCK (Belumosudil)"~7/16/2021 0:00:00~"Original"~1~"4106-12"~"PMC"~"506B PMC"~4106.00~12~"PMC 4106-12: Conduct a clinical trial to assess the pharmacokinetics of belumosudil among U.S. racial and ethnic groups. This study should characterize the exposure (including PK data), efficacy, and safety of belumosudil."~"Delayed"~"The applicant requested revised milestones because of enrollment challenges and continued discussion with the division. Revised milestones were acknowledged in a letter dated 01/28/2026.

"~12/31/2022 0:00:00~9/8/2025 0:00:00
384020~"CDER"~"NDA"~214801.00~"TAIHO ONCOLOGY INC"~"Lytgobi (Futibatinib)"~9/30/2022 0:00:00~"Original"~1~"4345-1"~"PMR"~"Accelerated Approval"~4345.00~1~"PMR 4345-1: Conduct a randomized clinical trial comparing dosages of futibatinib 16 mg and 20 mg once daily to verify and describe the clinical benefit of futibatinib in patients with advanced or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or other rearrangement. The overall response rate and duration of response should be assessed by a blinded independent review. The study should also evaluate other clinical outcomes that denote clinical benefit, such as patient reported outcomes. This study should enroll a minimum of 120 patients and all responders should have a minimum of 6 months from the date of initial response (or until disease progression, whichever comes first). Ensure that racial and ethnic minorities are adequately represented in the trial population, at a minimum, proportional to the prevalence of FGFR2 alterations in these subgroups in the US population."~"Ongoing"~"91 patients out of the planned 120 patients have been enrolled into the trial."~10/31/2027 0:00:00~12/3/2025 0:00:00
384021~"CDER"~"NDA"~214801.00~"TAIHO ONCOLOGY INC"~"Lytgobi (Futibatinib)"~9/30/2022 0:00:00~"Original"~1~"4345-3"~"PMR"~"505 (o)(3)"~4345.00~3~"PMR 4345-3: Conduct a clinical trial of futibatinib 20 mg once daily in a sufficient number of patients with cholangiocarcinoma harboring a FGFR2 fusion or other rearrangement, that incorporates prospectively specified, scheduled ophthalmologic assessments that include optic coherence tomography (OCT), for all patients (symptomatic or asymptomatic), at baseline and during treatment with futibatinib, to further characterize the incidence and severity of futibatinib-related ocular adverse events."~"Ongoing"~~10/31/2027 0:00:00~12/3/2025 0:00:00
384022~"CDER"~"NDA"~214801.00~"TAIHO ONCOLOGY INC"~"Lytgobi (Futibatinib)"~9/30/2022 0:00:00~"Original"~1~"4345-4"~"PMR"~"505 (o)(3)"~4345.00~4~"PMR 4345-4: Conduct a randomized study that compares the recommended dosage of 20 mg daily to a lower dosage (e.g., 16 mg) to provide a comparative analysis of dose- and exposure-response relationships for safety including further characterization of the rates of Grade =3 adverse reactions, Grade =3 hyperphosphatemia, serious adverse reactions, and dose reductions, interruptions, and discontinuations due to adverse reactions. Incorporate systematically assessed patient-reported outcome assessments to evaluate tolerability. Core outcomes should include patient-reported symptomatic adverse event data, overall side effect bother, physical function, and role function. The study should also provide a comparative analysis of dose- and exposure-response relationships for efficacy, including overall response rate and duration of response."~"Ongoing"~~10/31/2027 0:00:00~12/3/2025 0:00:00
384023~"CDER"~"NDA"~214801.00~"TAIHO ONCOLOGY INC"~"Lytgobi (Futibatinib)"~9/30/2022 0:00:00~"Original"~1~"4345-7"~"PMC"~"506B PMC"~4345.00~7~"PMC 4345-7: Establish, through the use of clinical trial data, an in-vitro diagnostic device that is essential to the safe and effective use of futibatinib for patients with advanced unresectable or metastatic cholangiocarcinoma harboring an FGFR2 gene fusions or other alterations"~"Ongoing"~~10/31/2027 0:00:00~12/3/2025 0:00:00
384024~"CDER"~"NDA"~215866.00~"ELI LILLY AND CO"~"Mounjaro (tirzepatide) injection, and Mounjaro KwikPen (tirzepatide) injection"~5/13/2022 0:00:00~"Original"~1~"4271-1"~"PMR"~"Pediatric Research Equity Act"~4271.00~1~"PMR 4271-1: Conduct a 30-week, randomized, double-blind, placebo-controlled, multicenter, parallel- arm study of the safety and efficacy of Mounjaro (tirzepatide) for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 17 years (inclusive), followed by a 22- week openlabel extension."~"Fulfilled"~"Per FDA letter dated 12/19/2025, this PMR/PMC has been fulfilled."~9/30/2028 0:00:00~7/9/2026 0:00:00
384025~"CDER"~"NDA"~215866.00~"ELI LILLY AND CO"~"Mounjaro (tirzepatide) injection, and Mounjaro KwikPen (tirzepatide) injection"~5/13/2022 0:00:00~"Original"~1~"4742-1"~"PMR"~"505 (o)(3)"~4742.00~1~"PMR 4742-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of tirzepatide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Pending"~~11/30/2026 0:00:00~7/9/2026 0:00:00
384026~"CDER"~"NDA"~215866.00~"ELI LILLY AND CO"~"Mounjaro (tirzepatide) injection, and Mounjaro KwikPen (tirzepatide) injection"~5/13/2022 0:00:00~"Original"~1~"4272-1"~"PMR"~"505 (o)(3)"~4272.00~1~"PMR 4272-1: Conduct a medullary thyroid carcinoma registry-based case series study of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of tirzepatide into the marketplace. This study will also establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to diabetes and use of tirzepatide."~"Ongoing"~~6/30/2040 0:00:00~7/9/2026 0:00:00
384027~"CDER"~"NDA"~215866.00~"ELI LILLY AND CO"~"Mounjaro (tirzepatide) injection, and Mounjaro KwikPen (tirzepatide) injection"~5/13/2022 0:00:00~"Original"~1~"4272-2"~"PMR"~"505 (o)(3)"~4272.00~2~"PMR 4272-2: Conduct a milk-only lactation study in lactating women who have received a dose of tirzepatide to assess concentrations of tirzepatide in breast milk using a validated assay."~"Fulfilled"~~7/31/2025 0:00:00~7/9/2026 0:00:00
384028~"CDER"~"NDA"~215887.00~"BIOGEN MA INC"~"Qalsody (tofersen)"~4/25/2023 0:00:00~"Original"~1~"4436-1"~"PMR"~"Accelerated Approval"~4436.00~1~"PMR 4436-1: In order to verify the clinical benefit of tofersen, complete Study 233AS303 (ATLAS),  A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation . The study will enroll presymptomatic adults who have a confirmed superoxide dismutase 1 mutation into a natural history run-in period, followed by a randomized, double-blind, placebo-controlled period. Subjects will remain in the double-blind, placebo-controlled study until they develop clinically manifest ALS or the end of the study. The primary endpoint is the proportion of subjects with emergence of clinically manifested ALS. The study should be of sufficient duration to observe changes on the endpoint in the patient population enrolled in the study."~"Ongoing"~"The study has been initiated."~6/30/2028 0:00:00~6/24/2026 0:00:00
384029~"CDER"~"NDA"~215887.00~"BIOGEN MA INC"~"Qalsody (tofersen)"~4/25/2023 0:00:00~"Original"~1~"4436-2"~"PMR"~"505 (o)(3)"~4436.00~2~"PMR 4436-2: Conduct a 2-year carcinogenicity study of tofersen in rat."~"Delayed"~"The draft and final protocol milestones were missed because of the additional time needed for the Agency to review and provide a response to your previous request for a Weight of Evidence Assessment (WoE). FDA determined that there was good cause for the delay and acknowledged the revised milestone(s) in a letter dated 06/24/2025."~2/28/2028 0:00:00~6/24/2026 0:00:00
384030~"CDER"~"NDA"~215887.00~"BIOGEN MA INC"~"Qalsody (tofersen)"~4/25/2023 0:00:00~"Original"~1~"4436-3"~"PMR"~"505 (o)(3)"~4436.00~3~"PMR 4436-3: Conduct a carcinogenicity study of tofersen in mouse. 
"~"Ongoing"~~6/30/2027 0:00:00~6/24/2026 0:00:00
384031~"CDER"~"NDA"~215887.00~"BIOGEN MA INC"~"Qalsody (tofersen)"~4/25/2023 0:00:00~"Original"~1~"4436-4"~"PMC"~"506B PMC"~4436.00~4~"PMC 4436-4: Conduct population pharmacokinetics analysis to evaluate the relationship between tofersen systemic exposure and renal function characteristics using existing data. In addition, collect urine samples following intrathecal administration of tofersen 100 mg from an ongoing clinical study to evaluate the recovery of tofersen in the urine."~"Ongoing"~~6/30/2028 0:00:00~6/24/2026 0:00:00
384032~"CDER"~"NDA"~215904.00~"IMMEDICA PHARMA AB"~"Ztalmy (ganaxolone)"~3/18/2022 0:00:00~"Original"~1~"4218-1"~"PMR"~"505 (o)(3)"~4218.00~1~"PMR 4218-1: A 26-week carcinogenicity study of ganaxolone in the CB6F1-Tg rasH2 transgenic mouse."~"Fulfilled"~~2/28/2024 0:00:00~5/6/2026 0:00:00
384033~"CDER"~"NDA"~215904.00~"IMMEDICA PHARMA AB"~"Ztalmy (ganaxolone)"~3/18/2022 0:00:00~"Original"~1~"4218-2"~"PMR"~"505 (o)(3)"~4218.00~2~"PMR 4218-2: A 104-week carcinogenicity study of ganaxolone in rat."~"Delayed"~"The original final report milestone was missed because of the additional time needed for development and validation of methods for release. FDA acknowledged proposed revised milestones with sufficient justification in a letter dated 7/6/2023."~10/31/2025 0:00:00~5/6/2026 0:00:00
384034~"CDER"~"NDA"~215904.00~"IMMEDICA PHARMA AB"~"Ztalmy (ganaxolone)"~3/18/2022 0:00:00~"Original"~1~"4218-3"~"PMR"~"505 (o)(3)"~4218.00~3~"PMR 4218-3: A 2-year carcinogenicity study of the major human unconjugated plasma metabolite, M2, in rat."~"Delayed"~"The draft protocol submission, final protocol submission, study completion, and final report milestones were missed because of continued discussions with the FDA regarding the study design."~10/31/2025 0:00:00~5/6/2026 0:00:00
384035~"CDER"~"NDA"~215904.00~"IMMEDICA PHARMA AB"~"Ztalmy (ganaxolone)"~3/18/2022 0:00:00~"Original"~1~"4218-4"~"PMR"~"505 (o)(3)"~4218.00~4~"PMR 4218-4: A juvenile animal toxicology study of the major human unconjugated plasma metabolite, M2, in rat."~"Fulfilled"~~2/28/2024 0:00:00~5/6/2026 0:00:00
384036~"CDER"~"NDA"~215910.00~"SUN PHARMACEUTICAL INDUSTRIES INC"~"Sezaby (phenobarbital)"~11/17/2022 0:00:00~"Original"~1~"4353-1"~"PMR"~"505 (o)(3)"~4353.00~1~"PMR 4353-1: Conduct a prospective study with appropriate comparator(s) to assess long-term neurodevelopmental effects of Sezaby in patients with neonatal seizures. Ensure capture of and adjustment for potential confounders. Assess neurodevelopmental effects using validated, age-appropriate developmental assessments of motor skills, cognition, language, and behavior. Follow patients for a minimum of 5 years."~"Delayed"~"The final protocol submission milestone was missed because additional time is needed to address FDA s recommendations. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 3/11/2024."~2/28/2035 0:00:00~1/15/2026 0:00:00
384037~"CDER"~"NDA"~215910.00~"SUN PHARMACEUTICAL INDUSTRIES INC"~"Sezaby (phenobarbital)"~11/17/2022 0:00:00~"Original"~1~"4353-3"~"PMR"~"505 (o)(3)"~4353.00~3~"PMR 4353-3: An electrocardiographic safety study, including assessment of QTc interval, in neonates at therapeutic exposures of Sezaby. The trial should enroll an adequate number of neonates to account for normal physiological changes and inter-individual variability. Electrocardiograms (ECGs) should be performed at multiple timepoints to account for intraindividual variability and for ECG-pharmacokinetic (PK) analysis."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone due to continued discussions with the FDA. The final protocol was acknowledged in a letter dated 2/13/2025."~5/30/2028 0:00:00~1/15/2026 0:00:00
384038~"CDER"~"NDA"~217645.00~"TRIS PHARMA INC"~"Onyda XR (clonidine hydrochloride)"~5/24/2024 0:00:00~"Original"~1~"4636-2"~"PMR"~"Pediatric Research Equity Act"~4636.00~2~"PMR 4636-2: Conduct a long-term, open-label study to evaluate the safety and tolerability of clonidine hydrochloride extended-release oral suspension in pediatric patients 4 years to <6 years of age with attention-deficit/hyperactivity disorder."~"Delayed"~"The final protocol submission milestone was missed, because Tris received information requests from the Agency on September 27, 2024, and October 25, 2024, for the PREA protocols for PMR 4636-1 and 4636-2. Tris submitted a complete response to both the Information Requests on December 24, 2024 and received additional comments on the protocol on February 20, 2025."~1/31/2027 0:00:00~7/23/2025 0:00:00
384039~"CDER"~"NDA"~217673.00~"INSMED INC"~"Brinsupri (brensocatib) tablet"~8/12/2025 0:00:00~"Original"~1~"4840-1"~"PMR"~"Pediatric Research Equity Act"~4840.00~1~"PMR 4840-1: Conduct a 52-week randomized, double-blind, placebo-controlled, parallel group study in children 6 to 11 years of age (inclusive) with non-cystic fibrosis bronchiectasis to assess efficacy, safety, and pharmacokinetic responses to brensocatib."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2033 0:00:00~
384040~"CDER"~"NDA"~217677.00~"SPRINGWORKS THERAPEUTICS INC"~"Ogsiveo (nirogacestat)"~11/27/2023 0:00:00~"Original"~1~"4550-2"~"PMR"~"505 (o)(3)"~4550.00~2~"PMR 4550-2: Conduct a prospective, open-label, single-arm clinical trial in adult premenopausal females with desmoid tumor to further characterize the incidence and severity of ovarian toxicity after a minimum of 12 months of nirogacestat treatment. Ensure that ovarian safety assessments after 12 months of continuous treatment are available from a minimum of 40 subjects. Post-treatment follow-up assessments including reproductive hormone measurements should be obtained at regularly scheduled intervals and after treatment cessation to assess time to ovarian function recovery."~"Ongoing"~~6/30/2029 0:00:00~1/21/2026 0:00:00
384041~"CDER"~"NDA"~217677.00~"SPRINGWORKS THERAPEUTICS INC"~"Ogsiveo (nirogacestat)"~11/27/2023 0:00:00~"Original"~1~"4550-3"~"PMC"~"506B PMC"~4550.00~3~"PMC 4550-3: Conduct physiologic-based pharmacokinetic (PBPK) modeling study and subsequently a clinical pharmacokinetic trial if needed, to evaluate the effect of proton pump inhibitors (PPIs) on the bioavailability of nirogacestat. Design and conduct the trials and modeling analyses in accordance with the FDA guidance for industry,  Evaluation of Gastric pHDependent Drug Interactions with Acid-Reducing Agents: Study Design, Data Analysis, and Clinical Implications  and  Physiologically Based Pharmacokinetic Analyses   Format and Content Guidance for Industry."~"Ongoing"~~8/31/2026 0:00:00~1/21/2026 0:00:00
384042~"CDER"~"NDA"~217677.00~"SPRINGWORKS THERAPEUTICS INC"~"Ogsiveo (nirogacestat)"~11/27/2023 0:00:00~"Original"~1~"4550-4"~"PMC"~"506B PMC"~4550.00~4~"PMC 4550-4: Complete the clinical trial, Study ARST1921, entitled,  A Safety, Pharmacokinetic and Efficacy Study of a gamma-Secretase Inhibitor, Nirogacestat (PF-03084014), in Children and Adolescents with Progressive, Surgically Unresectable Desmoid Tumors  to characterize the pharmacokinetics and preliminary efficacy of nirogacestat in pediatric patients aged 2 to 17 years with desmoid tumors. The trial should also characterize the safety of nirogacestat, including long-term monitoring of epiphyseal disorders and ovarian toxicity, in pediatric patients."~"Delayed"~"The study completion milestone was missed. The applicant requested revised milestones because the data cut-off date to support the primary endpoint of the 2-year progression-free survival rate is now projected to occur March 2026. Revised milestones were acknowledged in a letter dated 04/23/2026."~9/30/2026 0:00:00~1/21/2026 0:00:00
384043~"CDER"~"NDA"~217686.00~"IDORSIA PHARMACEUTICALS LTD"~"Tryvio (aprocitentan)"~3/19/2024 0:00:00~"Original"~1~"4600-1"~"PMR"~"505 (o)(3)"~4600.00~1~"PMR 4600-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Tryvio (aprocitentan) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Pending"~~11/30/2036 0:00:00~5/12/2025 0:00:00
384044~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-1"~"PMR"~"Accelerated Approval"~4608.00~1~"PMR 4608-1: Conduct a multiregional, randomized clinical trial comparing tovorafenib to physician s choice of chemotherapy in pediatric patients with low grade glioma with RAF fusions or rearrangements, or V600 mutations, intended to verify and describe the clinical benefit of tovorafenib through assessment of overall response rate (ORR) as a primary endpoint and progression-free survival (PFS) and duration of response, as determined by blinded independent central review, as key secondary endpoints. Include the protocol-specified ORR and interim PFS analyses in the interim report."~"Ongoing"~"218 patients out of the planned 400 patients have been
enrolled into the trial.."~4/30/2032 0:00:00~6/12/2026 0:00:00
384045~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-2"~"PMR"~"505 (o)(3)"~4608.00~2~"PMR 4608-2: Conduct a carcinogenicity study in mice to evaluate the potential serious risk of carcinogenicity."~"Fulfilled"~~8/31/2025 0:00:00~6/12/2026 0:00:00
384046~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-3"~"PMR"~"505 (o)(3)"~4608.00~3~"PMR 4608-3: Conduct a carcinogenicity study in rats to evaluate the potential serious risk of carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Ongoing"~~5/31/2028 0:00:00~6/12/2026 0:00:00
384047~"CDER"~"NDA"~217700.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4608-4"~"PMR"~"505 (o)(3)"~4608.00~4~"PMR 4608-4: Conduct comprehensive safety analyses from clinical studies that further characterize the known serious risk of long-term adverse effects of tovorafenib on growth and development, including but not limited to growth plate abnormalities, in a sufficient number of pediatric patients. Monitor patients for growth and development using age-appropriate screening tools. Include evaluations of growth as measured by height, weight, height velocity and height standard deviation scores, age at adrenarche if applicable, age at menarche if applicable (females) and Tanner stage. Monitor patients until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first. Include, in the interim report, a comprehensive safety analysis after all patients have been monitored for a minimum of 12 months from the start of study treatment."~"Ongoing"~~4/30/2032 0:00:00~6/12/2026 0:00:00
384048~"CDER"~"NDA"~212888.00~"VIIV HEALTHCARE CO"~"CABENUVA (cabotegravir extended release injectable suspension; rilpivirine extended release injectable suspension)"~1/21/2021 0:00:00~"Supplement"~1~"4221-2"~"PMR"~"Pediatric Research Equity Act"~4221.00~2~"PMR 4221-2: Conduct a study in subjects weighing 25 kg to less than 35 kg (approximately 6 to less than 12 years of age) who are HIV-1 infected, virologically suppressed (HIV-1 RNA <50 copies/mL) and on a stable antiretroviral regimen at the time of enrollment, to assess the pharmacokinetics, safety and tolerability, and antiviral activity of CABENUVA. Study participants must be monitored for a minimum of 24 weeks to assess safety and durability of antiviral response."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/05/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~2/17/2026 0:00:00
384049~"CDER"~"NDA"~212888.00~"VIIV HEALTHCARE CO"~"CABENUVA (cabotegravir extended release injectable suspension; rilpivirine extended release injectable suspension)"~1/21/2021 0:00:00~"Supplement"~1~"4221-3"~"PMR"~"Pediatric Research Equity Act"~4221.00~3~"PMR 4221-3: Conduct a study in subjects weighing 10 kg to less than 25 kg (approximately 2 to less than 6 years of age) who are HIV-1 infected, virologically suppressed (HIV-1 RNA <50 copies/mL) and on a stable antiretroviral regimen at the time of enrollment, to assess the pharmacokinetics, safety and tolerability, and antiviral activity of CABENUVA. Study participants must be monitored for a minimum of 24 weeks to assess safety and durability of antiviral response."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/05/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~2/17/2026 0:00:00
384050~"CDER"~"NDA"~212888.00~"VIIV HEALTHCARE CO"~"CABENUVA (cabotegravir extended release injectable suspension; rilpivirine extended release injectable suspension)"~1/21/2021 0:00:00~"Supplement"~3~"4232-2"~"PMR"~"Pediatric Research Equity Act"~4232.00~2~"PMR 4232-2: Conduct a study in subjects weighing 25 kg to less than 35 kg (approximately 6 to less than 12 years of age) who are HIV-1 infected, virologically suppressed (HIV-1 RNA <50 copies/mL) and on a stable antiretroviral regimen at the time of enrollment, to assess the pharmacokinetics, safety and tolerability, and antiviral activity of Cabenuva. Study participants must be monitored for a minimum of 24 weeks to assess safety and durability of antiviral response."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/05/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~2/17/2026 0:00:00
384051~"CDER"~"NDA"~212888.00~"VIIV HEALTHCARE CO"~"CABENUVA (cabotegravir extended release injectable suspension; rilpivirine extended release injectable suspension)"~1/21/2021 0:00:00~"Supplement"~3~"4232-3"~"PMR"~"Pediatric Research Equity Act"~4232.00~3~"PMR 4232-3: Conduct a study in subjects weighing 10 kg to less than 25 kg (approximately 2 to less than 6 years of age) who are HIV-1 infected, virologically suppressed (HIV-1 RNA <50 copies/mL) and on a stable antiretroviral regimen at the time of enrollment, to assess the pharmacokinetics, safety and tolerability, and antiviral activity of Cabenuva. Study participants must be monitored for a minimum of 24 weeks to assess safety and durability of antiviral response."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/05/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~2/17/2026 0:00:00
384052~"CDER"~"NDA"~212895.00~"CSPC OUYI PHARMACEUTICAL CO LTD"~"Conjupri (Levamlodipine Maleate)"~12/19/2019 0:00:00~"Original"~1~"3737-2"~"PMR"~"Pediatric Research Equity Act"~3737.00~2~"PMR 3737-2: Conduct a pharmacokinetic, pharmacodynamic, and safety study in patients with hypertension <6 years of age."~"Released"~"Per FDA letter dated 12/08/2025, this PMR/PMC has been released."~12/31/2025 0:00:00~1/23/2025 0:00:00
384053~"CDER"~"NDA"~212950.00~"VIIV HEALTHCARE CO"~"RUKOBIA (fostemsavir)"~7/2/2020 0:00:00~"Original"~1~"3862-1"~"PMR"~"Pediatric Research Equity Act"~3862.00~1~"PMR 3862-1: Conduct a study to evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of fostemsavir in treatment-experienced children living with HIV-1 infection who are less than 18 years of age and weighing at least 40 kg. The safety and antiviral activity of fostemsavir in pediatric subjects must be evaluated for a minimum of 24 weeks."~"Delayed"~"Original Final Report Due Date: 09/30/2024; Deferral Extension granted per FDA letter dated 12/21/2023. The study completion milestone was also revised."~9/30/2028 0:00:00~8/28/2025 0:00:00
384054~"CDER"~"NDA"~212994.00~"COMMAVE SUB LLC A WHOLLY OWNED SUB OF COLLEGIUM PHARMACEUTICAL INC"~"Azstarys (Serdexmethylphenidate chloride and Dexmethylphenidate hydrochloride)"~3/2/2021 0:00:00~"Original"~1~"3980-3"~"PMR"~"Pediatric Research Equity Act"~3980.00~3~"PMR 3980-3: Conduct a randomized, double-blind, placebo-controlled, parallel-group, safety and efficacy study of Azstarys in male and female children 4 to 12 years of age diagnosed with ADHD. Randomization should be stratified by two age groups (i.e., 4 to less than 6 years and 6 to 12 years of age)."~"Submitted"~"The final report was submitted to FDA on 11/27/2024."~11/29/2024 0:00:00~7/2/2026 0:00:00
384055~"CDER"~"NDA"~212994.00~"COMMAVE SUB LLC A WHOLLY OWNED SUB OF COLLEGIUM PHARMACEUTICAL INC"~"Azstarys (Serdexmethylphenidate chloride and Dexmethylphenidate hydrochloride)"~3/2/2021 0:00:00~"Original"~1~"3980-4"~"PMR"~"Pediatric Research Equity Act"~3980.00~4~"PMR 3980-4: Conduct a 12-month, open-label study to obtain information on safety and tolerability of Azstarys in male and female children 4 to less than 6 years of age diagnosed with ADHD. Also, this study must include sparse pharmacokinetic (PK) sampling in children ages 4 to less than 6 years to characterize the shape of the PK curve in this population."~"Ongoing"~"Original Final Report Due Date: 12/18/2024; Deferral Extension granted per FDA letter dated 01/31/2025."~6/30/2025 0:00:00~7/2/2026 0:00:00
384056~"CDER"~"NDA"~213005.00~"LIQUIDIA TECHNOLOGIES INC"~"Yutrepia (treprostinil)"~5/23/2025 0:00:00~"Original"~1~"4165-1"~"PMR"~"Pediatric Research Equity Act"~4165.00~1~"PMR 4165-1: Phase 3 randomized, double-blind, placebo-controlled trial to evaluate the efficacy, safety, and pharmacokinetics of LIQ861 (treprostinil) in children with WHO Group 1 Pulmonary Arterial Hypertension, aged 7 to 17 years; 16-week trial"~"Released"~"Per FDA letter dated 06/03/2026, this PMR/PMC has been released."~5/31/2029 0:00:00~
384057~"CDER"~"NDA"~213005.00~"LIQUIDIA TECHNOLOGIES INC"~"Yutrepia (treprostinil)"~5/23/2025 0:00:00~"Original"~1~"4165-2"~"PMR"~"Pediatric Research Equity Act"~4165.00~2~"PMR 4165-2: Phase 3 open-label, dose escalation trial to assess safety, tolerability, and pharmacokinetics of LIQ861 (treprostinil) in children with WHO Group 1 Pulmonary Arterial Hypertension, aged 3 to 6 years; 12-week trial."~"Released"~"Per FDA letter dated 06/03/2026, this PMR/PMC has been released."~1/31/2030 0:00:00~
384058~"CDER"~"NDA"~213006.00~"SUMITOMO PHARMA AMERICA INC"~"Gemtesa (vibegron)"~12/23/2020 0:00:00~"Original"~1~"3991-1"~"PMR"~"Pediatric Research Equity Act"~3991.00~1~"PMR 3991-1: A sequential cohort, vibegron dose-modification trial to determine the multiple-dose pharmacokinetics, safety and efficacy of vibegron in the treatment of pediatric patients with neurogenic detrusor overactivity aged 3 to less than 17 years."~"Delayed"~"Original final protocol milestone was missed. Original Final Report Due Date: 03/31/2028; Deferral Extension granted per FDA letter dated 02/18/2026 due to continued discussions with FDA. The study completion milestone was also revised. Per the applicant s Annual Status Report, the study is underway with 9 patients enrolled. No additional patients were enrolled during this reporting period."~9/30/2030 0:00:00~2/20/2026 0:00:00
384059~"CDER"~"NDA"~214801.00~"TAIHO ONCOLOGY INC"~"Lytgobi (Futibatinib)"~9/30/2022 0:00:00~"Original"~1~"4345-8"~"PMC"~"506B PMC"~4345.00~8~"PMC 4345-8: Conduct exploratory analyses aimed at identifying potential mechanisms of primary and acquired resistance to futibatinib using longitudinal ctDNA samples collected at baseline and at the end of treatment or time of progression from patients treated with futibatinib in TAS-120-101 and in the randomized clinical trial to verify and describe the clinical benefit of futibatinib in patients with advanced or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or other rearrangement. Include a discussion of the results in the context of the available published literature."~"Ongoing"~~10/31/2027 0:00:00~12/3/2025 0:00:00
384060~"CDER"~"NDA"~214801.00~"TAIHO ONCOLOGY INC"~"Lytgobi (Futibatinib)"~9/30/2022 0:00:00~"Original"~1~"4345-10"~"PMR"~"Pediatric Research Equity Act"~4345.00~10~"PMR 4345-10: Conduct a clinical trial of futibatinib in pediatric patients to further characterize the safety, pharmacokinetics, and anti-tumor activity of futibatinib for pediatric patients 1 year of age or older with advanced or metastatic solid tumors harboring FGFR gene alterations. Submit results, including patient-level data, from Arm O of the eSMART trial, to support decision making for the pediatric investigation."~"Pending"~"The trial has not begun but does not meet the criterion for delayed."~6/30/2033 0:00:00~12/3/2025 0:00:00
384061~"CDER"~"NDA"~214835.00~"LABORATORIOS FARMACEUTICOS ROVI SA"~"Risvan (Risperidone)"~3/29/2024 0:00:00~"Original"~1~"4617-1"~"PMC"~"506B PMC"~4617.00~1~"PMC 4617-1: Conduct a pharmacokinetic study that will evaluate exposure of RISVAN approximate to daily administration of 6 mg oral risperidone."~"Released"~~7/31/2026 0:00:00~5/29/2025 0:00:00
384062~"CDER"~"NDA"~214846.00~"SUMITOMO PHARMA AMERICA INC"~"MYFEMBREE (relugolix, estradiol, norethindrone acetate)"~5/26/2021 0:00:00~"Original"~1~"4038-1"~"PMR"~"505 (o)(3)"~4038.00~1~"PMR 4038-1: A prospective observational study in premenopausal women to assess the incidence rate, time to onset, pattern, severity, and reversibility of alopecia using appropriate tools (physician/observer-reported outcomes and/or patient surveys) to capture timing, pattern, severity, and reversibility of alopecia cases. Evaluate a minimum of 50 alopecia cases."~"Delayed"~"The Interim Report milestone was missed because of ongoing discussions with the Agency. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 01/22/2026."~8/31/2028 0:00:00~7/24/2025 0:00:00
384063~"CDER"~"NDA"~214846.00~"SUMITOMO PHARMA AMERICA INC"~"MYFEMBREE (relugolix, estradiol, norethindrone acetate)"~5/26/2021 0:00:00~"Original"~1~"4038-2"~"PMR"~"505 (o)(3)"~4038.00~2~"PMR 4038-2: A cohort study to compare the incidence rate of alopecia in premenopausal women who experience heavy menstrual bleeding from uterine fibroids on relugolix-containing products with the incidence in an appropriate comparator population not treated with relugolix-containing products. If an electronic healthcare database is selected, conduct a validation study to validate an algorithm with an adequate positive predictive value (PPV) to identify alopecia, prior to initiating the comparative safety study. If an adequate PPV cannot be obtained, conduct a prospective cohort study with primary data collection together with independent case adjudication."~"Delayed"~"The Validation/Feasibility Report Submission milestone was missed
because of continued discussions with the Agency. FDA issued an
Acknowledge Revised Milestone with Good Cause letter on 02/19/2025."~8/31/2028 0:00:00~7/24/2025 0:00:00
384064~"CDER"~"NDA"~214846.00~"SUMITOMO PHARMA AMERICA INC"~"MYFEMBREE (relugolix, estradiol, norethindrone acetate)"~5/26/2021 0:00:00~"Original"~1~"4038-3"~"PMR"~"505 (o)(3)"~4038.00~3~"PMR 4038-3: A prospective cohort study in premenopausal women with heavy menstrual bleeding from uterine fibroids to characterize changes in bone mineral density (BMD) as assessed by dual-energy X-ray absorptiometry (DXA) with long-term use of relugolix-containing products. Obtain BMD measurements at initiation of Myfembree and every 6 months over a 48-month treatment period. Determine mean percent change from baseline in BMD (with 95% confidence intervals) at the lumbar spine, total hip, and femoral neck for each timepoint. Include a categorical analysis of change from baseline in BMD, reporting the percentage of patients with various degrees of bone loss/gains. Additionally, measure BMD every 6 months for at least 12 months posttreatment to characterize recovery of bone loss. For post-treatment BMD report: 1) mean change from baseline in BMD, and 2) percent recovery of bone loss for each anatomic site. Include power calculations with your statistical analysis plan. Capture fractures as an adverse event of special interest with information on how the fracture occurred and adjudicate these cases."~"Delayed"~"The Final Protocol Submission milestone was missed because of continued discussions with the Agency. FDA issued an Acknowledge Revised Milestone letter on 08/03/2023 and an Acknowledge Final Protocol letter on 10/11/2023."~8/31/2029 0:00:00~7/24/2025 0:00:00
384065~"CDER"~"NDA"~214846.00~"SUMITOMO PHARMA AMERICA INC"~"MYFEMBREE (relugolix, estradiol, norethindrone acetate)"~5/26/2021 0:00:00~"Original"~1~"4038-4"~"PMR"~"505 (o)(3)"~4038.00~4~"PMR 4038-4: Conduct a prospective pregnancy exposure registry that compares the maternal, fetal, and neonatal outcomes of women exposed to relugolixcontaining products during pregnancy to those in an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the infant s first year of life."~"Ongoing"~~5/31/2032 0:00:00~7/24/2025 0:00:00
384066~"CDER"~"NDA"~214846.00~"SUMITOMO PHARMA AMERICA INC"~"MYFEMBREE (relugolix, estradiol, norethindrone acetate)"~5/26/2021 0:00:00~"Original"~1~"4038-5"~"PMR"~"505 (o)(3)"~4038.00~5~"PMR 4038-5: Conduct an additional pregnancy study that uses a different design from the Pregnancy Exposure Registry (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in premenopausal women exposed to relugolix-containing products during pregnancy compared to an unexposed pregnancy control population."~"Delayed"~"The Final Protocol Submission milestone was missed because of continued discussions with the Agency."~5/31/2028 0:00:00~7/24/2025 0:00:00
384080~"CDER"~"NDA"~211340.00~"AZURITY PHARMACEUTICALS INC"~"Katerzia (Amlodipine Besylate)"~7/8/2019 0:00:00~"Original"~1~"3640-2"~"PMR"~"Pediatric Research Equity Act"~3640.00~2~"PMR 3640-2: Conduct a dose-ranging, safety, tolerability, and efficacy study with amlodipine benzoate oral suspension in hypertensive pediatric patients age birth to less than 6 years of age."~"Delayed"~"Deferral extension granted (for final protocol submission date only) in June 2021; Applicant still working on a clinical design.

"~4/30/2026 0:00:00~9/5/2025 0:00:00
384081~"CDER"~"NDA"~211349.00~"ASTELLAS PHARMA US INC"~"XOSPATA (gilteritinib)"~11/28/2018 0:00:00~"Original"~1~"3542-1"~"PMR"~"505 (o)(3)"~3542.00~1~"PMR 3542-1: Demonstrate safety of long-term treatment with gilteritinib. Submit an integrated report and the supporting data files to summarize the safety outcomes when all patients on 2215-CL-0101, 2215-CL-0102 and 2215-CL-0301 have completed at least three years of treatment with gilteritinib or withdrew earlier."~"Fulfilled"~~2/28/2022 0:00:00~1/24/2025 0:00:00
384082~"CDER"~"NDA"~211367.00~"EXELTIS USA INC"~"Slynd (Drospirenone)"~5/23/2019 0:00:00~"Original"~1~"3631-1"~"PMR"~"505 (o)(3)"~3631.00~1~"PMR 3631-1: A prospective, controlled, long-term trial to assess bone mineral density change in adults and adolescents taking Slynd (drosperinone) compared to users of non-hormonal contraceptive methods."~"Delayed"~"The Final Protocol Submission, Trial Completion, and Final Report Submission milestones were missed because of ongoing negotiations with the Agency on the protocol. FDA determined there was good cause for the delay and acknowledged the revised milestones in a letter dated 08/31/2023. Enrollment: 1029."~4/30/2023 0:00:00~7/21/2025 0:00:00
384083~"CDER"~"NDA"~211448.00~"CMG PHARMACEUTICAL CO LTD"~"Mezofy (aripiprazole) oral films"~4/15/2025 0:00:00~"Original"~1~"4828-1"~"PMR"~"Pediatric Research Equity Act"~4828.00~1~"PMR 4828-1: Develop a 2-mg strength of Mezofy to meet the need for initial dose titration in pediatric patients ages 13 to 17 years with schizophrenia."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~~6/11/2026 0:00:00
384084~"CDER"~"NDA"~211616.00~"ESPERION THERAPEUTICS INC"~"Nexletol (bempedoic acid)"~2/21/2020 0:00:00~"Original"~1~"3797-1"~"PMR"~"Pediatric Research Equity Act"~3797.00~1~"PMR 3797-1: Conduct a pharmacokinetic/pharmacodynamic study evaluating bempedoic acid in patients with heterozygous familial hypercholesterolemia (HeFH) aged 10 years to less than 18 years. The Phase 2 study will be a randomized, open-label, 6-week, dose-finding study of bempedoic acid in 36 patients aged 10 years to less than 18 years with HeFH on stable background lipid-modifying therapy with LDL-C =130 mg/dL."~"Submitted"~"The final report was submitted to FDA on 09/29/2025."~11/30/2025 0:00:00~4/15/2026 0:00:00
384085~"CDER"~"NDA"~211616.00~"ESPERION THERAPEUTICS INC"~"Nexletol (bempedoic acid)"~2/21/2020 0:00:00~"Original"~1~"3797-3"~"PMR"~"505 (o)(3)"~3797.00~3~"PMR 3797-3: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Nexletol (bempedoic acid) during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The study will collect information for a minimum of 10 years. Results will be analyzed and reported descriptively. Data collected retrospectively will be analyzed separately and reported with the interim and final study reports."~"Ongoing"~~1/31/2033 0:00:00~4/15/2026 0:00:00
384086~"CDER"~"NDA"~211616.00~"ESPERION THERAPEUTICS INC"~"Nexletol (bempedoic acid)"~2/21/2020 0:00:00~"Original"~1~"3797-4"~"PMR"~"505 (o)(3)"~3797.00~4~"PMR 3797-4: Perform a lactation study (milk only) in lactating women who have received therapeutic doses of Nexletol (bempedoic acid) using a validated assay to assess concentrations of bempedoic acid in breast milk and the effects on the breastfed infant."~"Fulfilled"~~12/31/2024 0:00:00~4/15/2026 0:00:00
384087~"CDER"~"NDA"~211616.00~"ESPERION THERAPEUTICS INC"~"Nexletol (bempedoic acid)"~2/21/2020 0:00:00~"Original"~1~"3797-6"~"PMR"~"Pediatric Research Equity Act"~3797.00~6~"PMR 3797-6: Conduct an efficacy and safety study evaluating bempedoic acid in patients with heterozygous familial hypercholesterolemia (HeFH) aged 10 years to less than 18 years. The Phase 3 study will be a randomized, double-blind, placebo controlled, parallel group, 6-month, multicenter efficacy and safety study, followed by a 6-month open-label extension."~"Delayed"~"This is delayed due to the delay in PREA PMR 1. A deferral extension was granted on 4/11/25."~3/31/2029 0:00:00~4/15/2026 0:00:00
384088~"CDER"~"NDA"~211617.00~"ESPERION THERAPEUTICS INC"~"Nexlizet (bempedoic acid and ezetimibe)"~2/26/2020 0:00:00~"Original"~1~"3798-1"~"PMR"~"Pediatric Research Equity Act"~3798.00~1~"PMR 3798-1: Conduct a pharmacokinetic/pharmacodynamic study evaluating bempedoic acid in patients with heterozygous familial hypercholesterolemia (HeFH) aged 10 years to less than 18 years. The Phase 2 study will be a randomized, open-label, 6-week, dose-finding study of bempedoic acid in 36 patients aged 10 years to less than  18 years with HeFH on stable background lipid-modifying therapy with LDL-C greater than or equal to 130 mg/dL."~"Submitted"~"The final report was submitted to FDA on 09/29/2025."~11/30/2025 0:00:00~4/15/2026 0:00:00
384089~"CDER"~"NDA"~211617.00~"ESPERION THERAPEUTICS INC"~"Nexlizet (bempedoic acid and ezetimibe)"~2/26/2020 0:00:00~"Original"~1~"3798-3"~"PMR"~"505 (o)(3)"~3798.00~3~"PMR 3798-3: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Nexlizet (bempedoic acid and ezetimibe) during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The study will collect information for a minimum of 10 years. Results will be analyzed and reported descriptively. Data collected retrospectively will be analyzed separately and reported with the interim and final study reports."~"Ongoing"~~1/31/2033 0:00:00~4/15/2026 0:00:00
384090~"CDER"~"NDA"~213026.00~"SAREPTA THERAPEUTICS INC"~"AMONDYS 45 (casimersen)"~2/25/2021 0:00:00~"Original"~1~"4005-2"~"PMR"~"505 (o)(3)"~4005.00~2~"PMR 4005-2: Submit ECG data from Study 4045-301 to support a request to waive a thorough QT study. If these data do not support a thorough QT study waiver, you will need to evaluate the effect of casimersen on the QTc interval in a dedicated study, as per the ICH E14 guideline."~"Delayed"~"This PMR depends on completion of PMR 4005-12 (previously 4005-1) which is delayed, but underway."~10/31/2024 0:00:00~4/22/2026 0:00:00
384091~"CDER"~"NDA"~213026.00~"SAREPTA THERAPEUTICS INC"~"AMONDYS 45 (casimersen)"~2/25/2021 0:00:00~"Original"~1~"4005-3"~"PMR"~"505 (o)(3)"~4005.00~3~"PMR 4005-3: A two-year carcinogenicity study of intravenously administered casimersen in rat."~"Fulfilled"~~10/31/2024 0:00:00~4/22/2026 0:00:00
384092~"CDER"~"NDA"~213026.00~"SAREPTA THERAPEUTICS INC"~"AMONDYS 45 (casimersen)"~2/25/2021 0:00:00~"Original"~1~"4005-6"~"PMR"~"505 (o)(3)"~4005.00~6~"PMR 4005-6: Evaluate patient immune responses to dystrophin in patients from Study 4045-301. Test collected samples using fully validated antidystrophin assays that detect IgM, IgG and IgE antibodies. Provide antibody titers for samples that are positive for antibodies to dystrophin. Assess the impact of immune responses on product pharmacokinetics and clinical efficacy and safety. The final report submission should include the final clinical study report and the 96-week immunogenicity evaluation. The Final Immunogenicity Report should include the 144-week data.
"~"Delayed"~"This PMR depends on completion of PMR 4005-12 (previously 4005-1) which is delayed, but underway."~4/30/2025 0:00:00~4/22/2026 0:00:00
384093~"CDER"~"NDA"~213026.00~"SAREPTA THERAPEUTICS INC"~"AMONDYS 45 (casimersen)"~2/25/2021 0:00:00~"Original"~1~"4005-7"~"PMR"~"505 (o)(3)"~4005.00~7~"PMR 4005-7: Develop and validate assays to measure antibodies to casimersen. The assays should measure IgM, IgG and IgE antibody isotypes. Evaluate the samples from patients in Study 4045-101 and Study 4045-301 for antibodies to casimersen. Test samples that are positive for antibodies to casimersen for titer and neutralizing activity using fully validated assays. Until these assays have been fully validated and reviewed by FDA, sufficient samples should be banked and stored under appropriate conditions to allow for retesting as needed. Determine the impact of immune responses on product pharmacokinetics and clinical efficacy and safety. The final report submission should include the final clinical study report and the 96-week immunogenicity evaluation. The Final Immunogenicity Report should include the 144-week data.
"~"Delayed"~"This PMR depends on completion of PMR 4005-12 (previously 4005-1) which is delayed, but underway. The applicant requested revised milestones for this reason and FDA determined that there was sufficient justification for the anticipated delay and acknowledged the revised milestones in a letter dated 05/18/2022."~4/30/2025 0:00:00~4/22/2026 0:00:00
384094~"CDER"~"NDA"~213026.00~"SAREPTA THERAPEUTICS INC"~"AMONDYS 45 (casimersen)"~2/25/2021 0:00:00~"Original"~1~"4005-10"~"PMR"~"505 (o)(3)"~4005.00~10~"PMR 4005-10: Per the document entitled  qual-info.pdf  in the amendment submitted on September 14, 2020, [...]. Independently perform these [...] studies using casimersen drug product as well."~"Fulfilled"~~3/31/2024 0:00:00~4/22/2026 0:00:00
384095~"CDER"~"NDA"~213026.00~"SAREPTA THERAPEUTICS INC"~"AMONDYS 45 (casimersen)"~2/25/2021 0:00:00~"Original"~1~"4005-11"~"PMR"~"505 (o)(3)"~4005.00~11~"PMR 4005-11: Per the submission, the leachable study was performed using Lot 94EYDT01 after 54 months of storage in the inverted position at 5   3  C. Repeat the leachable study using one batch of to-be-marketed (TBM) drug product manufactured at the commercial site during stability, where the data is collected at multiple stability time-points per the testing frequency recommended in ICH Q1A(R2)."~"Delayed"~"The study completion and final report milestones were anticipated to be missed because of unexpected disruption in the supply of the to-be-marketed (TBM) drug product as aresult of COVID-19. The FDA acknowledged the revised milestones in a letter dated 01/20/23."~3/31/2025 0:00:00~4/22/2026 0:00:00
384096~"CDER"~"NDA"~213026.00~"SAREPTA THERAPEUTICS INC"~"AMONDYS 45 (casimersen)"~2/25/2021 0:00:00~"Original"~1~"4005-12"~"PMR"~"Accelerated Approval"~4005.00~12~"PMR 4005-12: Complete Study 4045-301 (Essence), A Double-Blind, Placebo-Controlled, Multicenter Study with an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients with Duchenne Muscular Dystrophy which is intended to verify the clinical benefit of casimersen."~"Delayed"~"The Interim Report Submission milestone was missed, because of dosing delay in the last subject. FDA Acknowledged revised Interim Report Submission milestone in letter dated 02/04/25."~4/30/2026 0:00:00~4/22/2026 0:00:00
384097~"CDER"~"NDA"~213036.00~"AMIVAS INC"~"Artesunate"~5/26/2020 0:00:00~"Original"~1~"3851-1"~"PMR"~"505 (o)(3)"~3851.00~1~"PMR 3851-1: Conduct a single-arm descriptive international study collecting data in women exposed to IV artesunate during pregnancy to assess risk of pregnancy and maternal complications and adverse effects on the fetus, neonate, and infant. Infant outcomes will be assessed through at least the first year of life. The study will collect information for a minimum of 7 years."~"Ongoing"~~11/30/2028 0:00:00~7/16/2025 0:00:00
384098~"CDER"~"NDA"~213036.00~"AMIVAS INC"~"Artesunate"~5/26/2020 0:00:00~"Original"~1~"3851-2"~"PMR"~"505 (o)(3)"~3851.00~2~"PMR 3851-2: Conduct a 5-year surveillance study to evaluate the potential development of resistance to artesunate and DHA as obtained from ongoing resistance monitoring programs on antimalarials. 
"~"Ongoing"~~5/31/2027 0:00:00~7/16/2025 0:00:00
384099~"CDER"~"NDA"~213036.00~"AMIVAS INC"~"Artesunate"~5/26/2020 0:00:00~"Original"~1~"3851-5"~"PMC"~"506B PMC"~3851.00~5~"PMC 3851-5: Conduct a descriptive 5-year study on the use of Artesunate for Injection in the U.S."~"Ongoing"~~10/31/2026 0:00:00~7/16/2025 0:00:00
384100~"CDER"~"NDA"~213036.00~"AMIVAS INC"~"Artesunate"~5/26/2020 0:00:00~"Original"~1~"3851-6"~"PMC"~"506B PMC"~3851.00~6~"PMC 3851-6: Conduct a multiple-dose safety, tolerability, pharmacokinetics (PK) study in pediatric patients with severe malaria from 0 to 12 months of age receiving 2.4 mg/kg Artesunate for Injection. The majority of pediatric patients in this age group is to be 6 months of age and younger."~"Delayed"~"Draft protocol was submitted 4-20- 2023. Missed submission of final protocol. Revised milestones on 9-9-2022. Missed submission of final protocol based on revised date of 11-2023. Final protocol was submitted 9-2024."~5/31/2027 0:00:00~7/16/2025 0:00:00
384101~"CDER"~"NDA"~213051.00~"NOVO NORDISK INC"~"Rybelsus (semaglutide) Tablets and Ozempic Tablets "~9/20/2019 0:00:00~"Original"~1~"4746-1"~"PMR"~"505 (o)(3)"~4746.00~1~"PMR 4746-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of oral semaglutide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Ongoing"~~11/30/2026 0:00:00~11/17/2025 0:00:00
384102~"CDER"~"NDA"~213051.00~"NOVO NORDISK INC"~"Rybelsus (semaglutide) Tablets and Ozempic Tablets "~9/20/2019 0:00:00~"Original"~1~"3692-1"~"PMR"~"Pediatric Research Equity Act"~3692.00~1~"PMR 3692-1: Conduct a 52-week, randomized, double-blind, placebo-controlled parallel group study of the safety and efficacy of Rybelsus (semaglutide) tablets for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 17 years (inclusive). Background therapy will consist of either metformin, insulin, or metformin plus insulin."~"Ongoing"~"245 screened; 132 randomized"~6/30/2027 0:00:00~11/17/2025 0:00:00
384103~"CDER"~"NDA"~214846.00~"SUMITOMO PHARMA AMERICA INC"~"MYFEMBREE (relugolix, estradiol, norethindrone acetate)"~5/26/2021 0:00:00~"Supplement"~2~"4312-2"~"PMR"~"505 (o)(3)"~4312.00~2~"PMR 4312-2: Conduct an additional pregnancy study that uses a different design from the Pregnancy Exposure Registry (for example, a retrospective cohort study in a claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in premenopausal women exposed to relugolix-containing products during pregnancy compared to an unexposed pregnancy control population."~"Pending"~~5/31/2028 0:00:00~7/24/2025 0:00:00
384104~"CDER"~"NDA"~214846.00~"SUMITOMO PHARMA AMERICA INC"~"MYFEMBREE (relugolix, estradiol, norethindrone acetate)"~5/26/2021 0:00:00~"Supplement"~2~"4312-4"~"PMR"~"505 (o)(3)"~4312.00~4~"PMR 4312-4: Conduct a prospective, open-label, single-arm clinical trial in premenopausal women with moderate to severe EAP to characterize changes in bone mineral density (BMD) after 48-months of Myfembree treatment. Ensure that post-treatment follow-up assessments at 12 months after treatment discontinuation are available from a minimum of 100 subjects."~"Ongoing"~~8/31/2029 0:00:00~7/24/2025 0:00:00
384105~"CDER"~"NDA"~214860.00~"ACER THERAPEUTICS INC"~"OLPRUVA (sodium phenylbutyrate)"~12/22/2022 0:00:00~"Original"~1~"4380-1"~"PMR"~"Pediatric Research Equity Act"~4380.00~1~"PMR 4380-1: Develop dosage strength(s) to accommodate the recommended dosing for pediatric patients who weigh <20 kg and patients who weigh greater than or =20 kg with a body surface area <1.2 m."~"Released"~"Per FDA letter dated 10/10/2025, this PMR/PMC has been released."~10/31/2023 0:00:00~2/19/2025 0:00:00
384106~"CDER"~"NDA"~214860.00~"ACER THERAPEUTICS INC"~"OLPRUVA (sodium phenylbutyrate)"~12/22/2022 0:00:00~"Original"~1~"4380-2"~"PMR"~"505 (o)(3)"~4380.00~2~"PMR 4380-2: Conduct in vitro studies to evaluate whether sodium phenylbutyrate and phenylacetate are substrates, inhibitors, or inducers of metabolizing enzymes and transporters as outlined in the FDA guidance for industry In Vitro Drug Interaction Studies -Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions (January 2020). If in vitro studies suggest a potential for drug interaction, additional in vivo studies may be required."~"Fulfilled"~~1/31/2025 0:00:00~2/19/2025 0:00:00
384107~"CDER"~"NDA"~214860.00~"ACER THERAPEUTICS INC"~"OLPRUVA (sodium phenylbutyrate)"~12/22/2022 0:00:00~"Original"~1~"4380-3"~"PMR"~"505 (o)(3)"~4380.00~3~"PMR 4380-3: Conduct in vitro studies to determine the feasibility of administering Olpruva (sodium phenylbutyrate) for oral suspension through enteral feeding tubes."~"Fulfilled"~~10/31/2023 0:00:00~2/19/2025 0:00:00
384108~"CDER"~"NDA"~214860.00~"ACER THERAPEUTICS INC"~"OLPRUVA (sodium phenylbutyrate)"~12/22/2022 0:00:00~"Original"~1~"4380-4"~"PMR"~"Pediatric Research Equity Act"~4380.00~4~"PMR 4380-4: Develop dosage strength(s) to accommodate pediatric patients 12 months of age and older who weigh between 7 kg to less than 20 kg."~"Fulfilled"~"Per FDA letter dated 10/10/2025, this PMR/PMC has been fulfilled."~12/31/2024 0:00:00~2/19/2025 0:00:00
384109~"CDER"~"NDA"~214876.00~"GLAXOSMITHKLINE LLC"~"ZEJULA (niraparib)"~4/26/2023 0:00:00~"Supplement"~3~"4865-1"~"PMC"~"506B PMC"~4865.00~1~"PMC 4865-1: Conduct an appropriate clinical validation study using clinical trial data to support the availability of an in vitro diagnostic device that is essential for the safe and effective use of niraparib for adult patients with advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer and whose cancer is associated with homologous recombination deficiency (HRD)-positive status defined by either:   a deleterious or suspected deleterious BRCA mutation, and/or   genomic instability."~"Fulfilled"~~3/31/2026 0:00:00~
384110~"CDER"~"NDA"~214876.00~"GLAXOSMITHKLINE LLC"~"ZEJULA (niraparib)"~4/26/2023 0:00:00~"Supplement"~4~"4865-1"~"PMC"~"506B PMC"~4865.00~1~"PMC 4865-1: Conduct an appropriate clinical validation study using clinical trial data to support the availability of an in vitro diagnostic device that is essential for the safe and effective use of niraparib for adult patients with advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer and whose cancer is associated with homologous recombination deficiency (HRD)-positive status defined by either:   a deleterious or suspected deleterious BRCA mutation, and/or   genomic instability."~"Fulfilled"~~3/31/2026 0:00:00~
384111~"CDER"~"NDA"~214900.00~"GLAXOSMITHKLINE LLC"~"BREXAFEMME (ibrexafungerp)"~6/1/2021 0:00:00~"Original"~1~"4069-1"~"PMR"~"505 (o)(3)"~4069.00~1~"PMR 4069-1: Conduct a worldwide single-arm descriptive study that collects prospective and retrospective data in women exposed to BREXAFEMME (ibrexafungerp) during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life."~"Ongoing"~~12/31/2029 0:00:00~7/11/2025 0:00:00
384112~"CDER"~"NDA"~214907.00~"ON TARGET LABORATORIES INC"~"Cytalux (pafolacianine sodium)"~11/29/2021 0:00:00~"Original"~1~"4383-3"~"PMC"~"506B PMC"~4383.00~3~"PMC 4383-3: Conduct a randomized, single dose, multicenter trial designed to demonstrate the intraoperative utility of Cytalux to achieve adequate surgical resection margins for lung lesions."~"Delayed"~"Draft Protocol Milestone missed, FDA Ack Revised Milestone letter."~5/31/2028 0:00:00~2/10/2026 0:00:00
384113~"CDER"~"NDA"~214907.00~"ON TARGET LABORATORIES INC"~"Cytalux (pafolacianine sodium)"~11/29/2021 0:00:00~"Original"~1~"4383-4"~"PMR"~"Pediatric Research Equity Act"~4383.00~4~"PMR 4383-4: Conduct a clinical trial to determine the appropriate dose of Cytalux through assessment of safety, activity, and pharmacokinetics in patients 2 to 17 years old undergoing surgical resection for known or suspected primary or metastatic tumors in the lung."~"Ongoing"~"The study has been initiated"~12/31/2027 0:00:00~2/10/2026 0:00:00
384114~"CDER"~"NDA"~214927.00~"ZEVRA DENMARK AS"~"Miplyffa (arimoclomol)"~9/20/2024 0:00:00~"Original"~1~"4699-1"~"PMC"~"506B PMC"~4699.00~1~"PMC 4699-1: Conduct a clinical drug interaction trial to assess the effect of concomitant administration of arimoclomol on the pharmacokinetics (PK) of miglustat and the effect of concomitant administration of miglustat on the PK of arimoclomol. Design and conduct the trial in accordance with the FDA guidance for industry Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter- Mediated Drug Interactions (January 2020)."~"Pending"~~9/30/2027 0:00:00~11/18/2025 0:00:00
384115~"CDER"~"NDA"~214938.00~"BIOMARIN PHARMACEUTICAL INC"~"Voxzogo (vosoritide)"~11/19/2021 0:00:00~"Original"~1~"4134-2"~"PMR"~"Accelerated Approval"~4134.00~2~"PMR 4134-2: Conduct an open-label, external-controlled trial in pediatric subjects with achondroplasia (ACH), whose epiphyses are not closed, to measure the effect of vosoritide on final adult height. The trial should also evaluate disproportionality and bone age as secondary endpoints. The safety endpoints related to the drug (e.g., blood pressure) or to the disease itself that may improve or worsen with long-term treatment (e.g., neurological complications, bone deformities, sleep apnea) should also be included."~"Ongoing"~"The study has been initiated"~8/31/2026 0:00:00~10/24/2025 0:00:00
384116~"CDER"~"NDA"~214958.00~"BRISTOL MYERS SQUIBB CO"~"Sotyktu (deucravacitinib)"~9/9/2022 0:00:00~"Original"~1~"4336-1"~"PMR"~"Pediatric Research Equity Act"~4336.00~1~"PMR 4336-1: Conduct a randomized, controlled trial to evaluate the safety, efficacy, and pharmacokinetics of deucravacitinib in the adolescent population (12 years to less than 18 years) with moderate to severe psoriasis who are candidates for systemic therapy or phototherapy. Evaluate at least 300 subjects exposed to deucravacitinib at the highest proposed dosage for a minimum of 52 weeks."~"Delayed"~"The Final Protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 03/21/2025."~12/31/2029 0:00:00~11/5/2025 0:00:00
384117~"CDER"~"NDA"~216059.00~"LOXO ONCOLOGY INC"~"Jaypirca (pirtobrutinib)"~1/27/2023 0:00:00~"Supplement"~1~"4557-2"~"PMR"~"505 (o)(3)"~4557.00~2~"PMR 4557-2: Conduct a randomized trial to characterize the safety and further assess the serious risks including infections, cytopenias, and hemorrhage, of at least two different dosages of pirtobrutinib not to exceed 200 mg once daily in patients with CLL or SLL. Include a comparative analysis of safety including dose-and exposure response relationships for safety and further characterization of the rates of Grade =3 adverse reactions, serious adverse reactions, adverse reactions of special interest, and dose reductions, interruptions, and discontinuations due to adverse reactions. Include a comparative analysis of efficacy based on response rate and duration of response along with dose- and exposure-response relationships for efficacy, for the two dosages."~"Ongoing"~~10/31/2028 0:00:00~3/26/2026 0:00:00
384118~"CDER"~"NDA"~216157.00~"GLOBAL BLOOD THERAPEUTICS INC"~"Oxbryta (voxelotor)"~12/17/2021 0:00:00~"Original"~1~"4190-1"~"PMR"~"Accelerated Approval"~4190.00~1~"PMR 4190-1: Complete Study GBT440-032: the ongoing Phase 3, randomized, double-blind, placebo-controlled trial in pediatric patients (age 2 years to <15 years) with Sickle Cell Disease (HOPE Kids 2). Expected enrollment of approximately 224 patients (age 2 years to <15 years) with at least 15 patients from age 2 years to < 4 years of age. Include patients with baseline hemoglobin of less than 6 g/dL. The primary endpoint is change from baseline at 24 weeks in time averaged maximum of mean velocity (TAMMV) arterial cerebral blood flow as measured by transcranial doppler (TCD). The secondary endpoint is change from baseline in TCD flow velocity at Week 48 and Week 96."~"Submitted"~"The Final Report was submitted to FDA on 07/02/2025."~9/30/2026 0:00:00~2/12/2026 0:00:00
384119~"CDER"~"NDA"~216157.00~"GLOBAL BLOOD THERAPEUTICS INC"~"Oxbryta (voxelotor)"~12/17/2021 0:00:00~"Original"~1~"4190-2"~"PMC"~"506B PMC"~4190.00~2~"PMC 4190-2: Complete at least 5 years of follow-up for all patients (on treatment) enrolled in Study GBT440-034: An Open-Label Extension Study of voxelotor Administered Orally to Patients with Sickle Cell Disease who have Participated in GBT440 Clinical trials. Include updated safety and efficacy analysis in yearly reports and submit datasets at the time of final clinical study report submission."~"Submitted"~~6/30/2025 0:00:00~2/12/2026 0:00:00
384120~"CDER"~"NDA"~216158.00~"BRISTOL-MYERS SQUIBB CO"~"Cobenfy (xanomeline and trospium chloride)"~9/26/2024 0:00:00~"Original"~1~"4640-1"~"PMR"~"505 (o)(3)"~4640.00~1~"PMR 4640-1: Perform a lactation study (milk only or mother-infant pair study) in lactating women (healthy subjects or patients) who have received therapeutic doses of COBENFY (xanomeline and trospium chloride) using a validated assay to assess concentrations of xanomeline and trospium chloride in breast milk and the effects on the breastfed infant if available, based on study population."~"Pending"~~6/30/2028 0:00:00~11/21/2025 0:00:00
384121~"CDER"~"NDA"~216158.00~"BRISTOL-MYERS SQUIBB CO"~"Cobenfy (xanomeline and trospium chloride)"~9/26/2024 0:00:00~"Original"~1~"4640-2"~"PMR"~"505 (o)(3)"~4640.00~2~"PMR 4640-2: Collect data from a prospective pregnancy exposure registry, preferably a disease-based multiproduct pregnancy registry, using a cohort analysis that compares the maternal, fetal, and infant outcomes of women with schizophrenia exposed to Cobenfy (xanomeline and trospium chloride) during pregnancy with unexposed comparator population of women with schizophrenia in a timely manner. Align the study protocol with protocol(s) outside the US to reach the target sample size. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortion, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes described in the protocol will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Pending"~~3/31/2037 0:00:00~11/21/2025 0:00:00
384122~"CDER"~"NDA"~216158.00~"BRISTOL-MYERS SQUIBB CO"~"Cobenfy (xanomeline and trospium chloride)"~9/26/2024 0:00:00~"Original"~1~"4640-3"~"PMR"~"505 (o)(3)"~4640.00~3~"PMR 4640-3: Conduct a retrospective pregnancy cohort study using claims or electronic health record data with medical chart validation that is adequately powered to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women with schizophrenia exposed to Cobenfy (xanomeline and trospium chloride) during pregnancy compared to appropriate comparator population(s)."~"Pending"~~3/31/2037 0:00:00~11/21/2025 0:00:00
384123~"CDER"~"NDA"~216158.00~"BRISTOL-MYERS SQUIBB CO"~"Cobenfy (xanomeline and trospium chloride)"~9/26/2024 0:00:00~"Original"~1~"4640-4"~"PMR"~"505 (o)(3)"~4640.00~4~"PMR 4640-4: To evaluate the potential adverse effects of COBENFY on voiding dynamics, especially urinary retention, conduct a non-invasive, 1-year, urological safety study. Assess specific urological safety parameters, such as maximum urinary flow rate (Qmax), post-void residual urine volume (PVR), validated symptom questionnaires, and urinalysis, at periodic intervals."~"Pending"~~6/30/2028 0:00:00~11/21/2025 0:00:00
384124~"CDER"~"NDA"~216158.00~"BRISTOL-MYERS SQUIBB CO"~"Cobenfy (xanomeline and trospium chloride)"~9/26/2024 0:00:00~"Original"~1~"4640-5"~"PMR"~"Pediatric Research Equity Act"~4640.00~5~"PMR 4640-5: Conduct a randomized, double-blind, placebo-controlled study to assess the efficacy and safety (with sparse PK) of Cobenfy for the treatment of schizophrenia in pediatric patients aged 13 to 17 years. We recommend that you use population pharmacokinetic modeling and simulation approach to select appropriate dose levels of Cobenfy in adolescents (13 to 17 years). As part of an efficacy and safety study in adolescents, please plan to collect PK blood samples at steady-state to characterize the PK of Cobenfy in pediatric patients with schizophrenia aged 13 to 17 years."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2030 0:00:00~11/21/2025 0:00:00
384125~"CDER"~"NDA"~216158.00~"BRISTOL-MYERS SQUIBB CO"~"Cobenfy (xanomeline and trospium chloride)"~9/26/2024 0:00:00~"Original"~1~"4640-6"~"PMR"~"Pediatric Research Equity Act"~4640.00~6~"PMR 4640-6: Conduct an open-label, long-term safety study (minimum of 52 weeks) of Cobenfy in pediatric subjects aged 13 to 17 years with schizophrenia."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2031 0:00:00~11/21/2025 0:00:00
384126~"CDER"~"NDA"~216158.00~"BRISTOL-MYERS SQUIBB CO"~"Cobenfy (xanomeline and trospium chloride)"~9/26/2024 0:00:00~"Original"~1~"4640-7"~"PMR"~"505 (o)(3)"~4640.00~7~"PMR 4640-7: Conduct a dedicated drug interaction study to assess the effect of Cobenfy on the PK of a sensitive substrate of CYP3A4."~"Pending"~~3/31/2027 0:00:00~11/21/2025 0:00:00
384127~"CDER"~"NDA"~216158.00~"BRISTOL-MYERS SQUIBB CO"~"Cobenfy (xanomeline and trospium chloride)"~9/26/2024 0:00:00~"Original"~1~"4640-8"~"PMR"~"505 (o)(3)"~4640.00~8~"PMR 4640-8: Conduct a dedicated drug interaction study to assess the effect of Cobenfy on the PK of a substrate of P-gp."~"Pending"~~3/31/2027 0:00:00~11/21/2025 0:00:00
384128~"CDER"~"NDA"~216158.00~"BRISTOL-MYERS SQUIBB CO"~"Cobenfy (xanomeline and trospium chloride)"~9/26/2024 0:00:00~"Original"~1~"4640-9"~"PMR"~"505 (o)(3)"~4640.00~9~"PMR 4640-9: Perform a dedicated pharmacogenetic study to assess the effects of CYP2D6 phenotypes (i.e., normal, intermediate, poor, and ultrarapid metabolizers) on xanomeline pharmacokinetics."~"Pending"~~9/30/2027 0:00:00~11/21/2025 0:00:00
384129~"CDER"~"NDA"~211617.00~"ESPERION THERAPEUTICS INC"~"Nexlizet (bempedoic acid and ezetimibe)"~2/26/2020 0:00:00~"Original"~1~"3798-4"~"PMR"~"505 (o)(3)"~3798.00~4~"PMR 3798-4: Perform a lactation study (milk only) in lactating women who have received therapeutic doses of Nexlizet (bempedoic acid and ezetimibe) using a validated assay to assess concentrations of bempedoic acid and ezetimibe in breast milk and the effects on the breastfed infant."~"Fulfilled"~~12/31/2024 0:00:00~4/15/2026 0:00:00
384130~"CDER"~"NDA"~211617.00~"ESPERION THERAPEUTICS INC"~"Nexlizet (bempedoic acid and ezetimibe)"~2/26/2020 0:00:00~"Original"~1~"3798-6"~"PMR"~"Pediatric Research Equity Act"~3798.00~6~"PMR 3798-6: Conduct an efficacy and safety study evaluating bempedoic acid in patients with heterozygous familial hypercholesterolemia (HeFH) aged 10 years to less than 18 years. The Phase 3 study will be a randomized, double-blind, placebo controlled, parallel group, 6-month, multicenter efficacy and safety study, followed by a 6-month open-label extension."~"Delayed"~"This is delayed due to the delay in PREA PMR 1. A deferral extension was granted on 4/11/25."~3/31/2029 0:00:00~4/15/2026 0:00:00
384131~"CDER"~"NDA"~211651.00~"PFIZER INC"~"Talzenna (talazoparib)"~10/16/2018 0:00:00~"Supplement"~10~"4460-2"~"PMC"~"506B PMC"~4460.00~2~"PMC 4460-2: Conduct an analytical and clinical validation study using clinical trial data, adequate to support the availability of an in vitro diagnostic device using tissue samples that is essential to the safe and effective use of talazoparib for patients diagnosed with metastatic castration-resistant prostate cancer (mCRPC), whose tumors harbor Homologous Recombination Repair (HRR) gene alterations, with HRR genes defined as: ATM, ATR, BRCA1, BRCA2, CDK12, CHEK2, FANCA, MLH1, MRE11A, NBN, PALB2 and RAD51C."~"Submitted"~~10/31/2024 0:00:00~12/11/2025 0:00:00
384132~"CDER"~"NDA"~211651.00~"PFIZER INC"~"Talzenna (talazoparib)"~10/16/2018 0:00:00~"Supplement"~10~"4460-3"~"PMC"~"506B PMC"~4460.00~3~"PMC 4460-3: Conduct an analytical and clinical validation study using clinical trial data, adequate to support the availability of an in vitro diagnostic device using circulating tumor deoxyribonucleic acid (ctDNA) samples from plasma that is essential to the safe and effective use of talazoparib for patients diagnosed with mCRPC, whose ctDNA samples harbor HRR gene alterations, with HRR genes defined as: ATM, ATR, BRCA1, BRCA2, CDK12, CHEK2, FANCA, MLH1, MRE11A, NBN, PALB2 and RAD51C."~"Submitted"~~10/31/2024 0:00:00~12/11/2025 0:00:00
384133~"CDER"~"NDA"~211672.00~"HONG KONG KING-FRIEND INDUSTRIAL CO LTD"~"XENLETA (lefamulin) "~8/19/2019 0:00:00~"Original"~1~"3672-5"~"PMR"~"505 (o)(3)"~3672.00~5~"PMR 3672-5: Conduct a pregnancy surveillance program to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to XENLETA (lefamulin) during pregnancy."~"Ongoing"~~8/31/2030 0:00:00~10/15/2025 0:00:00
384134~"CDER"~"NDA"~211672.00~"HONG KONG KING-FRIEND INDUSTRIAL CO LTD"~"XENLETA (lefamulin) "~8/19/2019 0:00:00~"Original"~1~"3672-8"~"PMR"~"Pediatric Research Equity Act"~3672.00~8~"PMR 3672-8: Conduct a single-dose study to evaluate the pharmacokinetics and safety of intravenous XENLETA (lefamulin) in children from 2 months to less than 18 years of age with suspected or confirmed bacterial infections receiving standard of care."~"Delayed"~"Deferral extension request was granted on 4-3-2025. The study completion milestone was missed.
"~10/31/2026 0:00:00~10/15/2025 0:00:00
384135~"CDER"~"NDA"~211672.00~"HONG KONG KING-FRIEND INDUSTRIAL CO LTD"~"XENLETA (lefamulin) "~8/19/2019 0:00:00~"Original"~1~"3672-9"~"PMR"~"Pediatric Research Equity Act"~3672.00~9~"PMR 3672-9: Conduct a single-dose study to evaluate the pharmacokinetics and safety of oral XENLETA (lefamulin) in children from 2 months to less than 12 years of age with suspected or confirmed bacterial infections receiving standard of care."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/04/2026. The study completion milestone was also revised.
"~1/31/2028 0:00:00~10/15/2025 0:00:00
384136~"CDER"~"NDA"~211672.00~"HONG KONG KING-FRIEND INDUSTRIAL CO LTD"~"XENLETA (lefamulin) "~8/19/2019 0:00:00~"Original"~1~"3672-10"~"PMR"~"Pediatric Research Equity Act"~3672.00~10~"PMR 3672-10: Conduct a randomized active-controlled study to assess the safety and pharmacokinetics of XENLETA (lefamulin) in children from 2 months to less than 12 years of age with CABP."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed. Protocol was submitted by 1-2023."~6/30/2027 0:00:00~10/15/2025 0:00:00
384137~"CDER"~"NDA"~211673.00~"HONG KONG KING-FRIEND INDUSTRIAL CO LTD"~"XENLETA (lefamulin) "~8/19/2019 0:00:00~"Original"~1~"3672-5"~"PMR"~"505 (o)(3)"~3672.00~5~"PMR 3672-5: Conduct a pregnancy surveillance program to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to XENLETA (lefamulin) during pregnancy."~"Ongoing"~~8/31/2030 0:00:00~10/15/2025 0:00:00
384138~"CDER"~"NDA"~211673.00~"HONG KONG KING-FRIEND INDUSTRIAL CO LTD"~"XENLETA (lefamulin) "~8/19/2019 0:00:00~"Original"~1~"3672-8"~"PMR"~"Pediatric Research Equity Act"~3672.00~8~"PMR 3672-8: Conduct a single-dose study to evaluate the pharmacokinetics and safety of intravenous XENLETA (lefamulin) in children from 2 months to less than 18 years of age with suspected or confirmed bacterial infections receiving standard of care."~"Delayed"~"Deferral extension request was granted on 4-3-2025. The study completion milestone was missed.
"~10/31/2026 0:00:00~10/15/2025 0:00:00
384139~"CDER"~"NDA"~211673.00~"HONG KONG KING-FRIEND INDUSTRIAL CO LTD"~"XENLETA (lefamulin) "~8/19/2019 0:00:00~"Original"~1~"3672-9"~"PMR"~"Pediatric Research Equity Act"~3672.00~9~"PMR 3672-9: Conduct a single-dose study to evaluate the pharmacokinetics and safety of oral XENLETA (lefamulin) in children from 2 months to less than 12 years of age with suspected or confirmed bacterial infections receiving standard of care."~"Delayed"~"Original Final Report Due Date: 06/30/2026; Deferral Extension granted per FDA letter dated 05/04/2026. The study completion milestone was also revised.
"~1/31/2028 0:00:00~10/15/2025 0:00:00
384140~"CDER"~"NDA"~211673.00~"HONG KONG KING-FRIEND INDUSTRIAL CO LTD"~"XENLETA (lefamulin) "~8/19/2019 0:00:00~"Original"~1~"3672-10"~"PMR"~"Pediatric Research Equity Act"~3672.00~10~"PMR 3672-10: Conduct a randomized active-controlled study to assess the safety and pharmacokinetics of XENLETA (lefamulin) in children from 2 months to less than 12 years of age with CABP."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed. Protocol was submitted by 1-2023."~6/30/2027 0:00:00~10/15/2025 0:00:00
384141~"CDER"~"NDA"~211675.00~"ABBVIE INC"~"Rinvoq (upadacitinib)"~8/16/2019 0:00:00~"Supplement"~4~"4212-1"~"PMR"~"Pediatric Research Equity Act"~4212.00~1~"PMR 4212-1: Conduct an active controlled efficacy and safety study (with sparse PK assessment) in patients 6 years to 11 years of age with refractory, moderate to severe atopic dermatitis whose disease is not adequately controlled with other systemic therapies, or when use of these therapies is not advisable. Subjects should initiate treatment with low dose upadacitinib or active control. The study should evaluate the treatment benefit of higher upadacitinib dosage in subjects who had inadequate response to the initial upadacitinib lower dosage. The study should include at least 300 subjects treated with the upadacitinib and exposed for at least 52 weeks. Provide the results of PK Study (Study M16-049) with the protocol for Study A."~"Delayed"~"The final protocol goal date of September 2022 was missed. The protocol was received March 28, 2024 on which FDA provided comment. The first subject was dosed in September 2024. Protocol design in discussion with FDA."~6/30/2026 0:00:00~10/10/2025 0:00:00
384142~"CDER"~"NDA"~213051.00~"NOVO NORDISK INC"~"Rybelsus (semaglutide) Tablets and Ozempic Tablets "~9/20/2019 0:00:00~"Original"~1~"3692-2"~"PMR"~"505 (o)(3)"~3692.00~2~"PMR 3692-2: Conduct a medullary thyroid carcinoma registry-based case series of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of Rybelsus (semaglutide) tablets into the marketplace. This study will also establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to diabetes and use of Rybelsus (semaglutide) tablets."~"Ongoing"~~2/28/2037 0:00:00~11/17/2025 0:00:00
384143~"CDER"~"NDA"~213051.00~"NOVO NORDISK INC"~"Rybelsus (semaglutide) Tablets and Ozempic Tablets "~9/20/2019 0:00:00~"Supplement"~20~"4766-1"~"PMR"~"Pediatric Research Equity Act"~4766.00~1~"PMR 4766-1: Conduct a 52-week, randomized, double-blind, placebo-controlled parallel group study of the safety and efficacy of Rybelsus (semaglutide) tablets for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 17 years (inclusive). Background therapy will consist of either metformin, insulin, or metformin plus insulin."~"Ongoing"~"245 screened; 132 randomized."~6/30/2027 0:00:00~11/17/2025 0:00:00
384144~"CDER"~"NDA"~213051.00~"NOVO NORDISK INC"~"Rybelsus (semaglutide) Tablets and Ozempic Tablets "~9/20/2019 0:00:00~"Supplement"~21~"4766-1"~"PMR"~"Pediatric Research Equity Act"~4766.00~1~"PMR 4766-1: Conduct a 52-week, randomized, double-blind, placebo-controlled parallel group study of the safety and efficacy of Rybelsus (semaglutide) tablets for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 17 years (inclusive). Background therapy will consist of either metformin, insulin, or metformin plus insulin."~"Ongoing"~"245 screened; 132 randomized."~6/30/2027 0:00:00~11/17/2025 0:00:00
384145~"CDER"~"NDA"~213082.00~"PFIZER INC"~"Tofacitinib "~9/25/2020 0:00:00~"Original"~1~"3945-1"~"PMR"~"505 (o)(3)"~3945.00~1~"PMR 3945-1: Conduct a long-term observational safety study in pediatric patients 2-17 years of age with polyarticular-course JIA (pcJIA) treated with tofacitinib to evaluate for the risk of malignancies, serious infections (including opportunistic infections), thrombosis, and effects on growth. The study should include a control group of pediatric pcJIA patients treated with other pcJIA medications as standard of care. Patients should be followed for 5 years."~"Delayed"~"The final protocol submission date was missed. Study is ongoing with study completion date of 2030."~9/30/2030 0:00:00~11/20/2025 0:00:00
384146~"CDER"~"NDA"~213137.00~"GLOBAL BLOOD THERAPEUTICS INC"~"Oxbryta (voxelotor)"~11/25/2019 0:00:00~"Original"~1~"3746-1"~"PMR"~"Accelerated Approval"~3746.00~1~"PMR 3746-1: Complete Study GBT440-032: the ongoing Phase 3, randomized, doubleblind, placebo-controlled trial in pediatric patients (age 2 years to < 15 years) with Sickle Cell Disease (HOPE Kids 2). Expected enrollment of approximately 224 patients (age 2 years to < 15 years) with at least 15 patients from age 2 years to < 4 years of age. Include patients with baseline hemoglobin of less than 6 g/dL. The primary endpoint is change from baseline at 24 weeks in time averaged maximum of mean velocity (TAMMV) arterial cerebral blood flow as measured by transcranial Doppler (TCD). The secondary endpoint is change from baseline in TCD flow velocity at Week 48 and Week 96."~"Submitted"~"The final report was submitted to FDA on 07/02/2025."~9/30/2026 0:00:00~1/22/2026 0:00:00
384147~"CDER"~"NDA"~213137.00~"GLOBAL BLOOD THERAPEUTICS INC"~"Oxbryta (voxelotor)"~11/25/2019 0:00:00~"Original"~1~"3746-3"~"PMC"~"506B PMC"~3746.00~3~"PMC 3746-3: Complete at least 5 years of follow-up for all patients (on treatment) enrolled in Study GBT440-034: An Open-Label Extension Study of voxelotor Administered Orally to Patients with Sickle Cell Disease who have Participated in GBT440 Clinical trials. Include updated safety and efficacy analysis in yearly reports and submit datasets at the time of final clinical study report submission."~"Submitted"~~6/30/2025 0:00:00~1/22/2026 0:00:00
384148~"CDER"~"NDA"~213137.00~"GLOBAL BLOOD THERAPEUTICS INC"~"Oxbryta (voxelotor)"~11/25/2019 0:00:00~"Supplement"~6~"3746-1"~"PMR"~"Accelerated Approval"~3746.00~1~"PMR 3746-1: Complete Study GBT440-032: the ongoing Phase 3, randomized, doubleblind, placebo-controlled trial in pediatric patients (age 2 years to < 15 years) with Sickle Cell Disease (HOPE Kids 2). Expected enrollment of approximately 224 patients (age 2 years to < 15 years) with at least 15 patients from age 2 years to < 4 years of age. Include patients with baseline hemoglobin of less than 6 g/dL. The primary endpoint is change from baseline at 24 weeks in time averaged maximum of mean velocity (TAMMV) arterial cerebral blood flow as measured by transcranial Doppler (TCD). The secondary endpoint is change from baseline in TCD flow velocity at Week 48 and Week 96."~"Submitted"~"The final report was submitted to FDA on 07/02/2025."~9/30/2026 0:00:00~1/22/2026 0:00:00
384149~"CDER"~"NDA"~213137.00~"GLOBAL BLOOD THERAPEUTICS INC"~"Oxbryta (voxelotor)"~11/25/2019 0:00:00~"Supplement"~6~"3746-3"~"PMC"~"506B PMC"~3746.00~3~"PMC 3746-3: Complete at least 5 years of follow-up for all patients (on treatment) enrolled in Study GBT440-034: An Open-Label Extension Study of voxelotor Administered Orally to Patients with Sickle Cell Disease who have Participated in GBT440 Clinical trials. Include updated safety and efficacy analysis in yearly reports and submit datasets at the time of final clinical study report submission."~"Submitted"~~6/30/2025 0:00:00~1/22/2026 0:00:00
384150~"CDER"~"NDA"~213217.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~11/14/2019 0:00:00~"Original"~1~"3735-1"~"PMR"~"Accelerated Approval"~3735.00~1~"PMR 3735-1: Complete and submit the final results of Trial BGB-3111-306 - the ongoing randomized, Phase 3 clinical trial of BRUKINSA in combination with rituximab versus bendamustine and rituximab in patients with previously untreated mantle cell lymphoma. The primary endpoint is progression free survival (PFS) as assessed by Independent Review Committee (IRC). Overall survival (OS) is a key secondary endpoint. PFS and OS would be analyzed based on superiority testing. Enrollment of approximately 500 patients is expected.
"~"Ongoing"~"The trial has completed enrollment."~2/28/2027 0:00:00~1/12/2026 0:00:00
384151~"CDER"~"NDA"~213217.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~11/14/2019 0:00:00~"Supplement"~4~"4130-1"~"PMR"~"505 (o)(3)"~4130.00~1~"PMR 4130-1: Conduct an integrated safety analysis of patients with lymphoid malignancies and Waldenstr m s Macroglobulinemia enrolled in clinical trials and from post-marketing reports to further characterize the risk of second primary malignancies with extended follow-up in patients receiving zanubrutinib. Submit interim reports after 2 years and 3 years containing cumulative safety updates. Include incidence rates, time to onset, predisposing factors including coexisting mutations and outcomes in the interim and final reports."~"Ongoing"~~6/30/2027 0:00:00~1/12/2026 0:00:00
384152~"CDER"~"NDA"~213217.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~11/14/2019 0:00:00~"Supplement"~4~"4130-2"~"PMC"~"506B PMC"~4130.00~2~"PMC 4130-2: Conduct a study to further characterize the clinical benefit and safety of zanubrutinib for the treatment of patients with newly diagnosed Waldenstr m s Macroglobulinemia with MYD88 mutation. This should include an assessment of the CXCR4 mutation status. In addition, the study should include a sufficient number of patients enrolled in the United States and sufficient numbers of racial and ethnic minority patients to allow for the interpretation of the results in these patient populations."~"Delayed"~"The study completion and final report submission milestones were missed, because of enrollment challenges."~3/31/2026 0:00:00~1/12/2026 0:00:00
384153~"CDER"~"NDA"~214958.00~"BRISTOL MYERS SQUIBB CO"~"Sotyktu (deucravacitinib)"~9/9/2022 0:00:00~"Original"~1~"4336-2"~"PMR"~"Pediatric Research Equity Act"~4336.00~2~"PMR 4336-2: Conduct a trial to evaluate the safety, efficacy, and pharmacokinetics of deucravacitinib in pediatric subjects 4 years to less than 12 years of age with moderate to severe psoriasis who are candidates for systemic therapy or phototherapy. Evaluate at least 150 subjects exposed to deucravacitinib at the highest proposed dosage for a minimum of 52 weeks."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~3/31/2036 0:00:00~11/5/2025 0:00:00
384154~"CDER"~"NDA"~214958.00~"BRISTOL MYERS SQUIBB CO"~"Sotyktu (deucravacitinib)"~9/9/2022 0:00:00~"Original"~1~"4336-3"~"PMR"~"505 (o)(3)"~4336.00~3~"PMR 4336-3: Conduct a prospective, registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to deucravacitinib during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life. For more information, see the May 2019 FDA draft guidance for Industry Post approval Pregnancy Safety Studies available at https://www.fda.gov/regulatory information/search-fda guidancedocuments/postapprovalpregnancy-safetystudies guidance-industry"~"Delayed"~"The Final Protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 03/21/2025. Enrollment: 0/200 DEUC, 121/600 C2, and 47/100 C3."~2/28/2029 0:00:00~11/5/2025 0:00:00
384155~"CDER"~"NDA"~214958.00~"BRISTOL MYERS SQUIBB CO"~"Sotyktu (deucravacitinib)"~9/9/2022 0:00:00~"Original"~1~"4336-4"~"PMR"~"505 (o)(3)"~4336.00~4~"PMR 4336-4: Conduct a pregnancy study that uses a different design from the pregnancy registry (for example a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to deucravacitinib during pregnancy compared to an unexposed control population."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 08/02/2024. Enrollment: 0 DEUC, 144 Cohort 2, and 792 Cohort 3."~2/28/2029 0:00:00~11/5/2025 0:00:00
384156~"CDER"~"NDA"~214958.00~"BRISTOL MYERS SQUIBB CO"~"Sotyktu (deucravacitinib)"~9/9/2022 0:00:00~"Original"~1~"4336-5"~"PMR"~"505 (o)(3)"~4336.00~5~"PMR 4336-5: Perform a lactation study (milk only) in lactating women who have received deucravacitinib to assess concentrations of deucravacitinib in breastmilk using a validated assay."~"Submitted"~~9/30/2025 0:00:00~11/5/2025 0:00:00
384157~"CDER"~"NDA"~214958.00~"BRISTOL MYERS SQUIBB CO"~"Sotyktu (deucravacitinib)"~9/9/2022 0:00:00~"Original"~1~"4336-6"~"PMR"~"505 (o)(3)"~4336.00~6~"PMR 4336-6: Conduct a randomized, active-controlled clinical trial to evaluate the long-term safety of deucravacitinib in patients with moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy. The trial should be of sufficient size and duration to characterize safety events of interest, including cardiovascular adverse events, opportunistic infections, and malignancy."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 07/16/2025. Enrollment: 0/3040."~12/31/2028 0:00:00~11/5/2025 0:00:00
384158~"CDER"~"NDA"~214958.00~"BRISTOL MYERS SQUIBB CO"~"Sotyktu (deucravacitinib)"~9/9/2022 0:00:00~"Supplement"~2~"4960-1"~"PMR"~"Pediatric Research Equity Act"~4960.00~1~"PMR 4960-1: Provide pharmacokinetic, efficacy, and safety information to support the pediatric assessment of deucravacitinib for the treatment of pediatric psoriatic arthritis in children 5 to less than 18 years of age."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2031 0:00:00~11/5/2025 0:00:00
384159~"CDER"~"NDA"~214962.00~"CYCLE PHARMACEUTICALS LTD"~"TASCENSO ODT (fingolimod)"~12/23/2021 0:00:00~"Original"~1~"4769-1"~"PMR"~"505 (o)(3)"~4769.00~1~"PMR 4769-1: Conduct an observational study to characterize patients exposed to Tascenso ODT who develop PML in terms of age, duration of exposure to Tascenso ODT, prior immunosuppressant use, lymphopenia, and anti- JCV antibody status. This study should be of at least 15 years duration to assess risk factors for the development of PML among patients with multiple sclerosis exposed to Tascenso ODT. Potential risk factors to be assessed should include age, duration of exposure to Tascenso ODT, prior immunosuppressant use, lymphopenia, and anti-JCV antibody status. The study should also determine the incidence of PML among patients exposed to Tascenso ODT, both overall and by each potential risk factor."~"Pending"~~7/31/2042 0:00:00~2/13/2026 0:00:00
384160~"CDER"~"NDA"~214962.00~"CYCLE PHARMACEUTICALS LTD"~"TASCENSO ODT (fingolimod)"~12/23/2021 0:00:00~"Original"~2~"4769-1"~"PMR"~"505 (o)(3)"~4769.00~1~"PMR 4769-1: Conduct an observational study to characterize patients exposed to Tascenso ODT who develop PML in terms of age, duration of exposure to Tascenso ODT, prior immunosuppressant use, lymphopenia, and anti- JCV antibody status. This study should be of at least 15 years duration to assess risk factors for the development of PML among patients with multiple sclerosis exposed to Tascenso ODT. Potential risk factors to be assessed should include age, duration of exposure to Tascenso ODT, prior immunosuppressant use, lymphopenia, and anti-JCV antibody status. The study should also determine the incidence of PML among patients exposed to Tascenso ODT, both overall and by each potential risk factor."~"Pending"~~7/31/2042 0:00:00~2/13/2026 0:00:00
384161~"CDER"~"NDA"~214985.00~"IDORSIA PHARMACEUTICALS LTD"~"QUVIVIQ (daridorexant)"~1/7/2022 0:00:00~"Original"~1~"4150-2"~"PMR"~"505 (o)(3)"~4150.00~2~"PMR 4150-2: Conduct a prospective, registry based cohort study that compares the maternal, fetal, and infant outcomes of women exposed to daridorexant during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The final protocol submission milestone was missed because of continued discussions between FDA and Idorsia regarding the protocol design. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 05/25/2023. Per the applicant's 3/6/2025 annual status report, no patients have been enrolled but, in the United States, all start-up activities have been performed and study sites are ready to enroll."~8/31/2033 0:00:00~3/5/2026 0:00:00
384162~"CDER"~"NDA"~216158.00~"BRISTOL-MYERS SQUIBB CO"~"Cobenfy (xanomeline and trospium chloride)"~9/26/2024 0:00:00~"Original"~1~"4640-10"~"PMC"~"506B PMC"~4640.00~10~"PMC 4640-10: Conduct a placebo-controlled, randomized withdrawal maintenance study of xanomeline and trospium chloride in patients with schizophrenia."~"Delayed"~"The applicant requested revised milestones to allow for adequate deliberation on how best to address the Agency s concerns regarding the protocol. Revised milestones were acknowledged in a letter dated 10/31/2025. A protocol was submitted after the final protocol milestone date had been missed."~10/30/2029 0:00:00~11/21/2025 0:00:00
384163~"CDER"~"NDA"~216165.00~"ORCHID PHARMA LTD"~"Exblifep (cefepime and enmetazobactam)"~2/22/2024 0:00:00~"Original"~1~"4582-1"~"PMR"~"Pediatric Research Equity Act"~4582.00~1~"PMR 4582-1: Conduct an open-label, single arm, non-comparative study to evaluate the pharmacokinetics, safety and tolerability of multiple doses of cefepime/enmetazobactam in pediatric patients from birth to less than 18 years of age with complicated urinary tract infections, including pyelonephritis."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2026 0:00:00~
384164~"CDER"~"NDA"~216165.00~"ORCHID PHARMA LTD"~"Exblifep (cefepime and enmetazobactam)"~2/22/2024 0:00:00~"Original"~1~"4582-2"~"PMR"~"505 (o)(3)"~4582.00~2~"PMR 4582-2: Conduct a US surveillance study over a five-year period after the introduction of cefepime/enmetazobactam to the market to determine if bacterial resistance or decreased susceptibility to cefepime/enmetazobactam is occurring in the target population of bacteria that are in the approved cefepime/enmetazobactam label."~"Delayed"~"The first interim study report was missed and the FDA issued a missed milestone letter on 04/06/2026."~12/31/2029 0:00:00~
384165~"CDER"~"NDA"~216185.00~"AUCTA PHARMACEUTICALS INC"~"Motpoly XR (lacosamide)"~5/4/2023 0:00:00~"Original"~1~"4437-1"~"PMR"~"Pediatric Research Equity Act"~4437.00~1~"PMR 4437-1: Development and validation by adult bioavailability/bioequivalence study(ies) of appropriate pediatric formulation(s) of Motpoly XR (lacosamide extended-release) to be used in pediatric patients weighing less than 50 kg."~"Delayed"~"The Study Completion and Final Report Submission milestones were missed. Deferral Extension requested 12/26/2025. Denied per FDA letter dated 02/09/2026."~3/31/2026 0:00:00~7/1/2026 0:00:00
384178~"CDER"~"NDA"~211675.00~"ABBVIE INC"~"Rinvoq (upadacitinib)"~8/16/2019 0:00:00~"Supplement"~15~"4426-1"~"PMC"~"506B PMC"~4426.00~1~"PMC 4426-1: A one year, randomized, blinded trial to evaluate the safety, efficacy, and pharmacokinetics of RINVOQ (upadacitinib) in pediatric patients 2 to less than 18 years of age with moderately to severely active Crohn's disease."~"Ongoing"~~10/31/2029 0:00:00~10/10/2025 0:00:00
384179~"CDER"~"NDA"~211675.00~"ABBVIE INC"~"Rinvoq (upadacitinib)"~8/16/2019 0:00:00~"Supplement"~15~"4426-2"~"PMC"~"506B PMC"~4426.00~2~"PMC 4426-2: A long-term extension trial to evaluate the long-term safety of RINVOQ (upadacitinib) in pediatric patients 2 to less than 18 years of age with moderately to severely active Crohn's disease who participated in postmarketing commitment 4426-1. This trial can be conducted as part of postmarketing commitment 4426-1."~"Pending"~~10/31/2032 0:00:00~10/10/2025 0:00:00
384180~"CDER"~"NDA"~211675.00~"ABBVIE INC"~"Rinvoq (upadacitinib)"~8/16/2019 0:00:00~"Supplement"~21~"4615-1"~"PMR"~"505 (o)(3)"~4615.00~1~"PMR 4615-1: Conduct a long-term registry study in pediatric patients 2 to less than 18 years of age with polyarticular Juvenile Idiopathic Arthritis (pJIA) or Psoriatic Arthritis (PsA) treated with upadacitinib to characterize the risk of malignancies, serious infections (including opportunistic infections), thrombosis, and effects on growth. The registry should enroll upadacitinibexposed patients and control groups of pediatric pJIA and PsA patients treated with standard-of-care. The applicant should adjudicate the study outcomes. Patients should be followed for 5 years."~"Delayed"~"The final protocol submission milestone was missed because additional time is needed to sufficiently address FDA s comments, dated August 20, 2025. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 09/29/2025."~2/28/2036 0:00:00~10/10/2025 0:00:00
384181~"CDER"~"NDA"~211675.00~"ABBVIE INC"~"Rinvoq (upadacitinib)"~8/16/2019 0:00:00~"Supplement"~22~"4615-1"~"PMR"~"505 (o)(3)"~4615.00~1~"PMR 4615-1: Conduct a long-term registry study in pediatric patients 2 to less than 18 years of age with polyarticular Juvenile Idiopathic Arthritis (pJIA) or Psoriatic Arthritis (PsA) treated with upadacitinib to characterize the risk of malignancies, serious infections (including opportunistic infections), thrombosis, and effects on growth. The registry should enroll upadacitinibexposed patients and control groups of pediatric pJIA and PsA patients treated with standard-of-care. The applicant should adjudicate the study outcomes. Patients should be followed for 5 years."~"Delayed"~"The final protocol submission milestone was missed because additional time is needed to sufficiently address FDA s comments, dated August 20, 2025. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 09/29/2025."~2/28/2036 0:00:00~10/10/2025 0:00:00
384182~"CDER"~"NDA"~211710.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Vitrakvi (larotrectinib)"~11/26/2018 0:00:00~"Original"~1~"3541-3"~"PMR"~"505 (o)(3)"~3541.00~3~"PMR 3541-3: Conduct a study of larotrectinib in a sufficient number of pediatric patients with NTRK-fusion solid tumors to evaluate the potential serious risk of adverse longterm effects of larotrectinib on the growth and development of pediatric patients. Patients will be evaluated for growth and developmental milestones using age appropriate screening tools and undergo neurological examination at appropriate intervals (for example, every six months) until larotrectinib is discontinued or for minimum of five years, whichever occurs first. Evaluations should include a neurologic exam, developmental milestone assessment, Karnofsky/Lansky score, growth as measured by weight and height, height velocity, height standard deviation scores (SDS), age at adrenarche if applicable (males), age at menarche if applicable (females), and Tanner Stage."~"Delayed"~"The final protocol was amended after recommendations from the Agency and submitted on October 4, 2019, which was after the original milestone."~8/31/2028 0:00:00~1/23/2026 0:00:00
384183~"CDER"~"NDA"~213217.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~11/14/2019 0:00:00~"Supplement"~4~"4130-3"~"PMC"~"506B PMC"~4130.00~3~"PMC 4130-3: Conduct a study to further characterize the clinical benefit and safety of zanubrutinib in patients with newly diagnosed and relapsed/refractory Waldenstr m s Macroglobulinemia with MYD88wt. This study should include a sufficient number of patients enrolled in the United States and sufficient numbers of racial and ethnic minority patients to allow for the interpretation of the results in these patient populations."~"Delayed"~"The study completion and final report submission milestones were missed, because of enrollment challenges."~3/31/2026 0:00:00~1/12/2026 0:00:00
384184~"CDER"~"NDA"~213217.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~11/14/2019 0:00:00~"Supplement"~4~"4130-4"~"PMC"~"506B PMC"~4130.00~4~"PMC 4130-4: Conduct an integrated analysis containing data from clinical trials and other data sources such as post-marketing reports, real-world evidence and other sources to further characterize the safety and efficacy of zanubrutinib in racial and ethnic minorities with Waldenstr m s Macroglobulinemia.
"~"Ongoing"~~6/30/2028 0:00:00~1/12/2026 0:00:00
384185~"CDER"~"NDA"~213217.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~11/14/2019 0:00:00~"Supplement"~5~"4128-1"~"PMR"~"Accelerated Approval"~4128.00~1~"PMR 4128-1: Conduct a randomized clinical trial that verifies and describes the clinical benefit of zanubrutinib in patients with relapsed or refractory marginal zone lymphoma. The trial should include sufficient representation of racial and ethnic minorities to better reflect the U.S. patient population and allow for interpretation of the results in these patient populations. The primary endpoint should be progression-free survival, with secondary endpoints that include objective response rate and overall survival."~"Ongoing"~"The trial has completed enrollment."~10/31/2028 0:00:00~1/12/2026 0:00:00
384186~"CDER"~"NDA"~213217.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~11/14/2019 0:00:00~"Supplement"~7~"4390-1"~"PMR"~"505 (o)(3)"~4390.00~1~"PMR 4390-1: Conduct an integrated safety analysis of patients enrolled in Study BGB-3111-304 to further characterize the risk of second primary malignancies, including incidence and types, and other serious risks with extended follow-up in patients receiving zanubrutinib as first-line treatment for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Include incidence rates of second primary malignancies, including after receipt of subsequent anticancer therapy, types, time to onset, potential predisposing factors, and outcomes in patients who received zanubrutinib or bendamustine plus rituximab in Study BGB-3111-304, and an analysis of postmarketing reports of second primary malignancies in recipients of zanubrutinib as first-line treatment for CLL/SLL. Evaluate overall survival n each treatment arm in Study BGB-3111-304, and include causes of death and narrative for death in the absence of treated disease progression, at least 8 years after randomization of the first subject in Cohort 1."~"Submitted"~~5/31/2026 0:00:00~1/12/2026 0:00:00
384187~"CDER"~"NDA"~213217.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~11/14/2019 0:00:00~"Supplement"~11~"4583-1"~"PMR"~"Accelerated Approval"~4583.00~1~"PMR 4583-1: Complete a randomized clinical trial that evaluates the clinical benefit of zanubrutinib plus obinutuzumab versus lenalidomide plus rituximab in patients with relapsed or refractory follicular lymphoma. The primary endpoint should be progression-free survival, with secondary endpoints that include objective response rate and overall survival. The trial should enroll a sufficiently representative study population that reflects the racial and ethnic diversity of the U.S. population of patients with follicular lymphoma and that allows for interpretation of the results in these populations."~"Ongoing"~"The trial has completed enrollment."~1/31/2029 0:00:00~1/12/2026 0:00:00
384188~"CDER"~"NDA"~213246.00~"ELI LILLY AND CO"~"Retevmo (selpercatinib) "~5/8/2020 0:00:00~"Original"~1~"3829-4"~"PMR"~"505 (o)(3)"~3829.00~4~"PMR 3829-4: Submit the final report, of an integrated safety analysis from clinical studies that further characterize the potential serious risk of long-term adverse effects of selpercatinib on growth and development, including an assessment of growth plate abnormalities in a sufficient number of adolescent patients 12 years of age and older with RET mutant MTC and RET fusion-positive thyroid cancer. Patients will be monitored for growth and development using age-appropriate screening tools. Evaluations will include growth as measured by height, weight, height velocity and height standard deviation scores, age at adrenarche if applicable (males), age at menarche if applicable (females) and Tanner stage. Patient monitoring will be performed until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first. Include the datasets with the final report. The results from this report may inform labeling."~"Delayed"~"The trial completion milestone was missed, because of additional time needed for follow up based on the last patient enrollment date. FDA determined that the applicant demonstrated  good cause  for the delay in a letter dated 7/7/2025. The final report submission milestone was also revised."~12/31/2025 0:00:00~6/26/2026 0:00:00
384189~"CDER"~"NDA"~213246.00~"ELI LILLY AND CO"~"Retevmo (selpercatinib) "~5/8/2020 0:00:00~"Supplement"~8~"4342-1"~"PMR"~"Accelerated Approval"~4342.00~1~"PMR 4342-1: Complete clinical trial(s) to obtain data on the clinical efficacy of selpercatinib through more precise estimation of the overall response rate and mature response duration per independent review assessment, in at least 60 patients with locally advanced or metastatic RET-fusion positive solid tumors other than non-small cell lung cancer and thyroid cancer, who have progressed on prior systemic treatment or have no satisfactory alternative treatment options. A sufficient number of patients with tumor types for which responses require additional characterization (e.g., colorectal cancer, esophagogastric cancer, and glioma) will be evaluated. Overall response rate and duration of response will be assessed by independent central review and all responding patients will be followed for at least 12 months following the onset of response or until disease progression or death or early treatment discontinuation, whichever comes first. Include available data regarding RET fusion partners and cooccurring genetic alterations for all patients."~"Submitted"~"The final report was submitted to FDA on 11/12/2025."~12/31/2025 0:00:00~6/26/2026 0:00:00
384190~"CDER"~"NDA"~213246.00~"ELI LILLY AND CO"~"Retevmo (selpercatinib) "~5/8/2020 0:00:00~"Supplement"~11~"4714-1"~"PMC"~"506B PMC"~4714.00~1~"PMC 4714-1: Complete survival follow-up of patients in the LIBRETTO-431 trial at 175 deaths to further characterize the efficacy and clinical benefit of selpercatinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with a RET gene fusion."~"Ongoing"~~3/31/2034 0:00:00~6/26/2026 0:00:00
384191~"CDER"~"NDA"~214985.00~"IDORSIA PHARMACEUTICALS LTD"~"QUVIVIQ (daridorexant)"~1/7/2022 0:00:00~"Original"~1~"4150-3"~"PMR"~"505 (o)(3)"~4150.00~3~"PMR 4150-3: Conduct an additional pregnancy study that uses a different observational design from the pregnancy registry (for example a case control study or a retrospective cohort study using claims or electronic medical record data) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to daridorexant during pregnancy compared to an unexposed control population."~"Delayed"~"The Final Protocol Submission milestone was missed because of continued discussions between FDA and Idorsia regarding the protocol design. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 5/25/2023. Per the applicant's 3/6/2025 annual status report, 141/2095 mother-infant pairs have been enrolled."~4/30/2029 0:00:00~3/5/2026 0:00:00
384192~"CDER"~"NDA"~214985.00~"IDORSIA PHARMACEUTICALS LTD"~"QUVIVIQ (daridorexant)"~1/7/2022 0:00:00~"Original"~1~"4150-5"~"PMR"~"Pediatric Research Equity Act"~4150.00~5~"PMR 4150-5: Conduct a randomized, double-blind, dose-finding study to identify the optimal dose for a confirmatory efficacy and safety study in patients with insomnia disorder aged 10 to <18 years."~"Delayed"~"Although the study has made good progress, challenges with enrollment continue. FDA granted the deferral extension request in a letter dated 25 February 2025."~10/31/2026 0:00:00~3/5/2026 0:00:00
384193~"CDER"~"NDA"~214985.00~"IDORSIA PHARMACEUTICALS LTD"~"QUVIVIQ (daridorexant)"~1/7/2022 0:00:00~"Original"~1~"4150-6"~"PMR"~"Pediatric Research Equity Act"~4150.00~6~"PMR 4150-6: Conduct a randomized, double-blind, placebo-controlled efficacy and safety study of daridorexant for the treatment of insomnia disorder in children 10 to <18 years of age."~"Delayed"~"The study for PMR 4150-6 has not been initiated, Idorsia anticipates delays to this study as it depends on the completion of PMR 4150-5 (Study ID-078A205)
"~4/30/2028 0:00:00~3/5/2026 0:00:00
384194~"CDER"~"NDA"~214985.00~"IDORSIA PHARMACEUTICALS LTD"~"QUVIVIQ (daridorexant)"~1/7/2022 0:00:00~"Original"~1~"4150-7"~"PMR"~"Pediatric Research Equity Act"~4150.00~7~"PMR 4150-7: Conduct a study to evaluate the longer-term safety of continued treatment of insomnia disorder with daridorexant for at least one year in patients 10 to <18 years of age at study entry."~"Delayed"~"The study for PMR 4150-7 has not been initiated, Idorsia anticipates delays to this study as it depends on the
completion of PMR 4150-5 (Study ID-078A205)."~1/31/2029 0:00:00~3/5/2026 0:00:00
384195~"CDER"~"NDA"~214998.00~"BRISTOL MYERS SQUIBB CO"~"Camzyos (Mavacamten)"~4/28/2022 0:00:00~"Original"~1~"4257-1"~"PMR"~"505 (o)(3)"~4257.00~1~"PMR 4257-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Camzyos (mavacamten) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Assess infant outcomes through at least the first year of life. Specify the minimum number of patients in the protocol."~"Ongoing"~~10/31/2027 0:00:00~6/25/2026 0:00:00
384196~"CDER"~"NDA"~215014.00~"APELLIS PHARMACEUTICALS INC"~"EMPAVELI (pegcetacoplan)"~5/14/2021 0:00:00~"Original"~1~"4047-1"~"PMR"~"505 (o)(3)"~4047.00~1~"PMR 4047-1: Establish a registry to characterize the long-term safety of pegcetacoplan in adult patients with paroxysmal nocturnal hemoglobinuria (PNH), including patients who are treatment na ve, with at least 5 years of follow-up. Submit yearly safety follow-up data and a summary of the major safety findings for all patients, including the development or worsening of autoimmune diseases such as systemic lupus erythematosus (SLE), and all serious infections with encapsulated bacteria. The final study report should include an integrated safety dataset and patient level data, including data on pegcetacoplan dosing, meningococcal and pneumococcal vaccination status, and concomitant medications."~"Ongoing"~~7/31/2029 0:00:00~7/10/2026 0:00:00
384197~"CDER"~"NDA"~215014.00~"APELLIS PHARMACEUTICALS INC"~"EMPAVELI (pegcetacoplan)"~5/14/2021 0:00:00~"Original"~1~"4047-3"~"PMR"~"505 (o)(3)"~4047.00~3~"PMR 4047-3: Complete Study APL2-307,  An Open-label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)."" Include an updated summary of safety, efficacy analyses, and datasets at the time of final clinical study report submission."~"Ongoing"~~2/28/2026 0:00:00~7/10/2026 0:00:00
384198~"CDER"~"NDA"~215014.00~"APELLIS PHARMACEUTICALS INC"~"EMPAVELI (pegcetacoplan)"~5/14/2021 0:00:00~"Original"~1~"4047-5"~"PMC"~"506B PMC"~4047.00~5~"PMC 4047-5: Develop a sensitive assay to detect and monitor the presence and titer of antibodies that bind the active moiety of pegcetacoplan. The assay should be capable of detecting neutralizing anti-pegcetacoplan antibodies (ADA) in the presence of pegcetacoplan levels that are expected to be present in serum at the time of patient sampling. The final report should include development and validation data to support use of the assay."~"Fulfilled"~~5/31/2023 0:00:00~7/10/2026 0:00:00
384199~"CDER"~"NDA"~215014.00~"APELLIS PHARMACEUTICALS INC"~"EMPAVELI (pegcetacoplan)"~5/14/2021 0:00:00~"Original"~1~"4047-6"~"PMC"~"506B PMC"~4047.00~6~"PMC 4047-6: Develop and validate a sensitive assay to evaluate the neutralizing activity of anti-pegcetacoplan antibodies (ADA) detected in patient samples. The assay should be capable of detecting neutralizing ADA in the presence of pegcetacoplan levels that are expected to be present in serum at the time of patient sampling. The final report should include development and validation data to support use of the assay."~"Fulfilled"~~5/31/2023 0:00:00~7/10/2026 0:00:00
384200~"CDER"~"NDA"~215014.00~"APELLIS PHARMACEUTICALS INC"~"EMPAVELI (pegcetacoplan)"~5/14/2021 0:00:00~"Original"~1~"4047-7"~"PMC"~"506B PMC"~4047.00~7~"PMC 4047-7: Use the sensitive assays developed under PMCs 4047-5 and 4047-6 to establish the incidence, titer and neutralizing activity of antibodies to pegcetacoplan in patient samples from studies APL2-302, APL2-307, and APL2-308. Establish whether there is an impact of antibodies on safety and efficacy of pegcetacoplan. Submit datasets at the time of final report submission."~"Pending"~~8/31/2026 0:00:00~7/10/2026 0:00:00
384201~"CDER"~"NDA"~215039.00~"NOVARTIS PHARMACEUTICALS CORP"~"Vijoice (Alpelisib)"~4/5/2022 0:00:00~"Original"~1~"4260-1"~"PMR"~"Accelerated Approval"~4260.00~1~"PMR 4260-1: Conduct a multiregional clinical trial to verify and describe the clinical benefit of alpelisib film-coated tablets, through more precise estimation of confirmed objective response rate and mature response duration per blinded independent review, in adult and pediatric patients 2 years of age and older with PIK3CA-Related Overgrowth Spectrum (PROS), including those with severe manifestations of PROS. Responding patients will be followed for at least 36 months from the onset of response, or until disease progression, whichever comes first. Evaluate a sufficient number of patients to characterize response rate and durability of response by PIK3CA mutation type (frequent hotspot mutations vs. other less frequent mutations), PROS subtype, and age (2   5 years, 6   11 years, 12   17 years, = 18 years). Include patient narratives and additional outcomes measures (such as clinical outcomes assessments) to support the assessment of clinical benefit in the study report. The distribution of race and ethnicity in the patient population studied should be sufficiently reflective of the U.S. patient population to support generalizability of results to U.S. patients with PROS."~"Ongoing"~"The study has been initiated."~8/31/2027 0:00:00~6/1/2026 0:00:00
384202~"CDER"~"NDA"~216264.00~"PROVEPHARM SAS"~"Bludigo (indigotindisulfonate sodium)"~7/8/2022 0:00:00~"Original"~1~"4300-1"~"PMR"~"Pediatric Research Equity Act"~4300.00~1~"PMR 4300-1: Deferred pediatric study under PREA -An open-label, multicenter study to evaluate the safety, pharmacokinetics, and conspicuity of Indigo Carmine Injection 0.8% solution when used as an aid in the determination of ureteral patency in pediatric patients from 2 years to less than 18 years of age."~"Delayed"~"Final Protocol milestone was missed, FDA comments/edits received after milestone date.
"~7/31/2026 0:00:00~8/28/2025 0:00:00
384203~"CDER"~"NDA"~216264.00~"PROVEPHARM SAS"~"Bludigo (indigotindisulfonate sodium)"~7/8/2022 0:00:00~"Original"~1~"4300-2"~"PMC"~"506B PMC"~4300.00~2~"PMC 4300-2: Conduct an Open-Label, Randomized, Multicenter Trial to Evaluate the Efficacy and Safety of Two Different Doses of BludigoTM (Indigotindisulfonate Sodium Injection, USP) 0.8% When Used as an Aid in the Determination of Ureteral Patency."~"Delayed"~"Final Protocol milestone was missed, FDA comments/edits received after milestone date.
"~6/30/2025 0:00:00~8/28/2025 0:00:00
384204~"CDER"~"NDA"~216264.00~"PROVEPHARM SAS"~"Bludigo (indigotindisulfonate sodium)"~7/8/2022 0:00:00~"Original"~1~"4300-3"~"PMC"~"506B PMC"~4300.00~3~"PMC 4300-3: Conduct an Open-label, Parallel group, Single-Dose Trial to Investigate the Influence of Renal Impairment on the Efficacy and Safety of Two Doses of BludigoTM (Indigotindisulfonate Sodium Injection, USP) 0.8% When Used as an Aid in the Determination of Ureteral Patency."~"Delayed"~"The applicant requested revised milestones for the Trial completion and Final Report milestones. Revised milestones were acknowledged in a letter dated 01/16/2026."~12/31/2025 0:00:00~8/28/2025 0:00:00
384205~"CDER"~"NDA"~216285.00~"EXELTIS USA INC"~"Drospirenone"~6/29/2022 0:00:00~"Original"~1~"4287-1"~"PMR"~"505 (o)(3)"~4287.00~1~"PMR 4287-1: A prospective, controlled, long-term trial to assess bone mineral density change in adults and adolescents using non-hormonal contraceptive methods compared to Slynd and Drospirenone Chewable Tablets."~"Ongoing"~~10/31/2026 0:00:00~8/26/2025 0:00:00
384206~"CDER"~"NDA"~216318.00~"AZURITY PHARMACEUTICALS INC"~"Azmiro (testosterone cypionate)"~6/2/2022 0:00:00~"Original"~1~"4286-1"~"PMR"~"505 (o)(3)"~4286.00~1~"PMR 4286-1: 24-hour Ambulatory Blood Pressure Monitoring (ABPM) trial to assess whether your product increases blood pressure (BP) in hypogonadal men."~"Delayed"~"The Final Report Submission milestone was missed because
the applicant did not market the product until Dec 2024, due to business reasons. FDA determined that the applicant
failed to demonstrate ""good cause"" for the delay per FDA letter dated 08/01/2025.
"~7/31/2025 0:00:00~8/1/2025 0:00:00
384207~"CDER"~"NDA"~216340.00~"BRISTOL MYERS SQUIBB CO"~"Krazati (adagrasib)"~12/12/2022 0:00:00~"Original"~1~"4378-1"~"PMR"~"Accelerated Approval"~4378.00~1~"PMR 4378-1: Conduct a randomized comparative clinical trial of adagrasib in adult patients with KRAS G12C mutated, locally advanced or metastatic NSCLC who have received at least one prior systemic therapy, to obtain overall survival, progression free survival, overall response rate, and duration of response. This data may be obtained from the ongoing clinical trial entitled,  A Randomized Phase 3 Study of MRTX849 versus Docetaxel in Patients with Previously Treated Non-Small Cell Lung Cancer with KRAS G12C Mutation."~"Delayed"~"The final report milestone was missed, because the final OS
analysis for the trial have not yet been reached. Per the applicant's 2/6/2026 annual status report, the trial is fully enrolled."~12/31/2025 0:00:00~2/6/2026 0:00:00
384208~"CDER"~"NDA"~216340.00~"BRISTOL MYERS SQUIBB CO"~"Krazati (adagrasib)"~12/12/2022 0:00:00~"Original"~1~"4378-2"~"PMR"~"505 (o)(3)"~4378.00~2~"PMR 4378-2: Conduct a multicenter, randomized clinical trial to further characterize serious adverse events, including gastrointestinal toxicity, and compare the safety of adagrasib 600 mg twice daily versus an alternative daily dosage in patients with locally advanced or metastatic, KRAS G12C mutated, non-small cell lung cancer who have received at least one prior systemic therapy. Include a comparative analysis of dose- and exposure-response relationships for safety including further characterization of the rates of Grade =3 adverse reactions, serious adverse reactions, and dose reductions, interruptions, and discontinuations due to adverse reactions. Additionally, conduct efficacy analyses including a comparative analysis of dose- and exposure-response relationships for efficacy for the two dosing regimens. Incorporate systematically assessed patient-reported outcome assessments to evaluate tolerability and conduct exploratory exposure response analyses."~"Delayed"~"The final report milestone was missed, because the addition of sites extended the overall study timeline."~10/31/2025 0:00:00~2/6/2026 0:00:00
384209~"CDER"~"NDA"~216340.00~"BRISTOL MYERS SQUIBB CO"~"Krazati (adagrasib)"~12/12/2022 0:00:00~"Original"~1~"4378-4"~"PMR"~"505 (o)(3)"~4378.00~4~"PMR 4378-4: Conduct a clinical pharmacokinetic trial to evaluate the effect of a BCRP inhibitor on the single-dose pharmacokinetics of adagrasib. Refer to the following FDA Guidance for Industry for additional details:  Clinical Drug Interaction Studies-Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Fulfilled"~~4/30/2024 0:00:00~2/6/2026 0:00:00
384210~"CDER"~"NDA"~216340.00~"BRISTOL MYERS SQUIBB CO"~"Krazati (adagrasib)"~12/12/2022 0:00:00~"Original"~1~"4378-5"~"PMR"~"505 (o)(3)"~4378.00~5~"PMR 4378-5: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeated doses of adagrasib (at steady-state) on the single dose pharmacokinetics of a sensitive CYP2B6 substrate. Refer to the following FDA Guidance for Industry for additional details:  Clinical Drug Interaction Studies- Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions."""~"Delayed"~"The final report milestone was missed, because the study was postponed pending results from a related ongoing study."~5/31/2024 0:00:00~2/6/2026 0:00:00
384211~"CDER"~"NDA"~216340.00~"BRISTOL MYERS SQUIBB CO"~"Krazati (adagrasib)"~12/12/2022 0:00:00~"Original"~1~"4378-6"~"PMR"~"505 (o)(3)"~4378.00~6~"PMR 4378-6: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeated doses of adagrasib (at steady-state) on the single dose pharmacokinetics of a probe MATE-1/-2K substrate. Refer to the following FDA Guidance for Industry for additional details:  Clinical Drug Interaction Studies- Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions."""~"Delayed"~"The final report milestone was missed, because the study was postponed pending results from a related ongoing study."~6/30/2024 0:00:00~2/6/2026 0:00:00
384212~"CDER"~"NDA"~216340.00~"BRISTOL MYERS SQUIBB CO"~"Krazati (adagrasib)"~12/12/2022 0:00:00~"Supplement"~5~"4650-1"~"PMR"~"Accelerated Approval"~4650.00~1~"PMR 4650-1: Complete clinical trial, Study 849-010,  A Randomized Phase 3 Study of adagrasib 600 mg twice daily in combination with cetu ximab versus chemotherapy in patients with advanced colorectal cancer with KRASG12C mutation with disease progression on or after first-line therapy."~"Ongoing"~"461 patients out of the planned 420 patients have been enrolled into the trial."~9/30/2027 0:00:00~2/6/2026 0:00:00
384213~"CDER"~"NDA"~216386.00~"PFIZER INC"~"Zavzpret (zavegepant)"~3/9/2023 0:00:00~"Original"~1~"4408-2"~"PMR"~"Pediatric Research Equity Act"~4408.00~2~"PMR 4408-2: An open-label, single-dose study to evaluate the safety, tolerability, and single-dose pharmacokinetics (PK) of zavegepant in patients with migraine ages 6 through 11 years."~"Delayed"~"The Final Protocol Submission milestone was missed due to
continued discussion with the FDA regarding the trial design."~11/30/2032 0:00:00~5/6/2026 0:00:00
384214~"CDER"~"NDA"~211710.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Vitrakvi (larotrectinib)"~11/26/2018 0:00:00~"Original"~1~"3541-4"~"PMR"~"505 (o)(3)"~3541.00~4~"PMR 3541-4: Conduct a study of larotrectinib 100 mg orally once daily in a sufficient number of adult or pediatric patients with a body surface area of at least 1.0 m2 who experienced an adverse reaction requiring a third dosage modification of larotrectinib to better characterize the tolerability of this approved dosage modification for larotrectinib. The following information will be provided for each patient: patient age and body surface area (if pediatric), adverse reactions leading to each prior dose reduction of larotrectinib, duration of treatment on prior dose levels, duration of treatment at the 100 mg orally once daily regimen, best overall response and duration of response. and tumor information collected while receiving the 100 mg orally once daily dosage regimen."~"Delayed"~"The final protocol was amended after recommendations from the Agency and submitted on October 4, 2019, which was after the original milestone."~8/31/2028 0:00:00~1/23/2026 0:00:00
384215~"CDER"~"NDA"~211710.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Vitrakvi (larotrectinib)"~11/26/2018 0:00:00~"Supplement"~4~"3974-1"~"PMR"~"505 (o)(3)"~3974.00~1~"PMR 3974-1: Submit an integrated safety analyses and supporting data from an adequate number of patients enrolled in clinical trial(s) designed to further characterize the risk of fractures and its sequelae; and to identify the risk factors for development of fractures and their sequelae in patients exposed to larotrectinib. The design of the trial should include assessment and collection of sufficient height measurements and measures of bone monitoring, including but not limited to initial and serial assessment of bone mineral density (BMD) with dual x-ray absorptiometry (DXA) scans, and markers of bone formation, bone resorption, and calcium metabolism."~"Fulfilled"~~8/31/2025 0:00:00~1/23/2026 0:00:00
384216~"CDER"~"NDA"~211723.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~1/23/2020 0:00:00~"Original"~1~"3787-1"~"PMR"~"Accelerated Approval"~3787.00~1~"PMR 3787-1: Submit the final results from a confirmatory randomized trial in patients with epithelioid sarcoma to confirm clinical benefit and provide additional efficacy data that may inform product labeling for tazemetostat."~"Ongoing"~"25 patients have been enrolled in phase 1b of the trial and no patients enrolled in phase 3, out of approximately 172 patients total."~9/30/2029 0:00:00~3/20/2026 0:00:00
384217~"CDER"~"NDA"~211723.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~1/23/2020 0:00:00~"Original"~1~"3872-1"~"PMR"~"Accelerated Approval"~3872.00~1~"PMR 3872-1: Submit the final report and datasets from a randomized, phase 3 clinical trial that verifies and describes the clinical benefit of tazemetostat in patients with relapsed or refractory follicular lymphoma whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test. The trial should include sufficient numbers of racial and ethnic minority patients to better reflect the U.S. patient population and allow for the interpretation of the results in these patient populations. Patients should be randomized to receive immunotherapy with or without tazemetostat. The primary endpoint should be progression-free survival, with secondary endpoints that include overall survival and objective response rate."~"Ongoing"~"187 patients were enrolled in phase 3 out of approximately 568
patients total."~7/31/2025 0:00:00~3/20/2026 0:00:00
384218~"CDER"~"NDA"~211723.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~1/23/2020 0:00:00~"Original"~1~"3872-2"~"PMR"~"Accelerated Approval"~3872.00~2~"PMR 3872-2: Submit the final report and datasets from a randomized, phase 3 clinical trial that verifies and describes the clinical benefit of tazemetostat in patients with relapsed or refractory follicular lymphoma whose tumors do not have an EZH2 mutation as detected by an FDA-approved test. The trial should include sufficient numbers of racial and ethnic minority patients to better reflect the U.S. patient population and allow for the interpretation of the results in these patient populations. Patients should be randomized to receive immunotherapy with or without tazemetostat. The primary endpoint should be progression-free survival, with secondary endpoints that include overall survival and objective response rate."~"Ongoing"~"187 patients were enrolled in phase 3 out of approximately 568
patients total."~7/31/2025 0:00:00~3/20/2026 0:00:00
384219~"CDER"~"NDA"~211723.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~1/23/2020 0:00:00~"Original"~1~"3787-2"~"PMR"~"Accelerated Approval"~3787.00~2~"PMR 3787-2: Submit the final report and datasets for the final analysis of overall response rate and duration of response for clinical trial EZH-202 titled,  A Phase II, Multicenter Study of the EZH2 Inhibitor Tazemetostat in Adult Subjects With INI1-Negative Tumors or Relapsed/Refractory Synovial Sarcoma  to verify and confirm clinical benefit of tazemetostat, that may inform product labeling. An additional 25 patients from Cohort 6 beyond those included in the original NDA submission will be evaluated and all responding patients will be followed for at least 12 months from the onset of response."~"Fulfilled"~"Per FDA letter dated 02/03/2026, this PMR/PMC has been fulfilled."~6/30/2023 0:00:00~3/20/2026 0:00:00
384220~"CDER"~"NDA"~211723.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~1/23/2020 0:00:00~"Original"~1~"3872-3"~"PMR"~"505 (o)(3)"~3872.00~3~"PMR 3872-3: Submit annual safety updates for 10 years from a cumulative, integrated safety analyses of patients with lymphoid malignancies enrolled in clinical trials and from post-marketing reports to characterize the risk of acute myeloid leukemia, myelodysplastic syndrome, T-lymphoblastic lymphoma, and other secondary malignancies in patients receiving TAZVERIK. Include incidence rates, time to onset, predisposing factors, and outcomes in the interim and final reports. These safety evaluations may inform labeling of patient populations at highest risk and to provide evidencebased monitoring recommendations."~"Ongoing"~~4/30/2030 0:00:00~3/20/2026 0:00:00
384221~"CDER"~"NDA"~211723.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~1/23/2020 0:00:00~"Original"~1~"3787-3"~"PMR"~"505 (o)(3)"~3787.00~3~"PMR 3787-3: Conduct cumulative, integrated safety analyses after 5 and 10 years of follow-up from an adequate number of patients enrolled in clinical trials to characterize the risk of acute myeloid leukemia, myelodysplastic syndrome, T-lymphoblastic lymphoma, and other secondary malignancies in patients receiving TAZVERIK; include incidence rates, time to onset, predisposing factors, and outcomes. These safety evaluations will be adequate to inform labeling of patient populations at highest risk and to provide evidence-based monitoring recommendations."~"Ongoing"~~3/31/2030 0:00:00~3/20/2026 0:00:00
384222~"CDER"~"NDA"~211723.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~1/23/2020 0:00:00~"Original"~1~"3787-4"~"PMR"~"505 (o)(3)"~3787.00~4~"PMR 3787-4: Conduct a pharmacokinetic and safety study in cancer patients with moderate or severe hepatic impairment investigating the effects of hepatic impairment (based on the NCI Organ Dysfunction Working Group (ODWG) criteria) on the repeat dose pharmacokinetics of tazemetostat compared to cancer patients with normal hepatic function. This study will assess the magnitude of increased tazemetostat exposure and determine appropriate dosing recommendations of tazemetostat for patients with moderate or severe hepatic impairment."~"Submitted"~~1/31/2023 0:00:00~3/20/2026 0:00:00
384223~"CDER"~"NDA"~213246.00~"ELI LILLY AND CO"~"Retevmo (selpercatinib) "~5/8/2020 0:00:00~"Supplement"~12~"4639-3"~"PMR"~"Accelerated Approval"~4639.00~3~"PMR 4639-3: Complete clinical trial(s) to obtain data on the clinical efficacy of selpercatinib through more precise estimation of the overall response rate and mature response duration per independent review assessment, in at least 60 patients with locally advanced or metastatic RET-fusion positive solid tumors other than non-small cell lung cancer and thyroid cancer, who have progressed on prior systemic treatment or have no satisfactory alternative treatment options. A sufficient number of patients with tumor types for which responses require additional characterization (e.g., colorectal cancer, esophagogastric cancer, and glioma) will be evaluated. Overall response rate and duration of response will be assessed by independent central review and all responding patients will be followed for at least 12 months following the onset of response or until disease progression or death or early treatment discontinuation, whichever comes first. Include available data regarding RET fusion partners and cooccurring genetic alterations for all patients."~"Submitted"~"The final report was submitted to FDA on 11/12/2025."~12/31/2025 0:00:00~6/26/2026 0:00:00
384224~"CDER"~"NDA"~213246.00~"ELI LILLY AND CO"~"Retevmo (selpercatinib) "~5/8/2020 0:00:00~"Supplement"~12~"4639-4"~"PMR"~"505 (o)(3)"~4639.00~4~"PMR 4639-4: Conduct comprehensive safety analyses from clinical studies that further characterize the known serious risk of long-term adverse effects of selpercatinib on growth and development, including but not limited to growth plate abnormalities, in a sufficient number of pediatric patients with RET-altered solid tumors ages 2 to < 12 years of age. Monitor patients for growth and development using age-appropriate screening tools. Include evaluations of growth as measured by height, weight, height velocity and height standard deviation scores, age at menarche if applicable (females) and Tanner stage. Monitor patients until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first."~"Ongoing"~~12/31/2029 0:00:00~6/26/2026 0:00:00
384225~"CDER"~"NDA"~213246.00~"ELI LILLY AND CO"~"Retevmo (selpercatinib) "~5/8/2020 0:00:00~"Supplement"~13~"4697-1"~"PMC"~"506B PMC"~4697.00~1~"PMC 4697-1: Complete survival follow-up of patients in the LIBRETTO-531 trial at 125 deaths or 6 years from randomization of the first patient, whichever occurs first, to further characterize the efficacy and clinical benefit of selpercatinib in patients with advanced or metastatic RET mutant medullary thyroid cancer who are na ve to tyrosine kinase inhibitors."~"Ongoing"~~9/30/2026 0:00:00~6/26/2026 0:00:00
384226~"CDER"~"NDA"~213312.00~"AADI BIOSCIENCES INC"~"Fyarro (sirolimus protein-bound particles for injectable suspension (albumin-bound))"~11/22/2021 0:00:00~"Original"~1~"4177-1"~"PMR"~"505 (o)(3)"~4177.00~1~"PMR 4177-1: Conduct an integrated safety analysis to assess the serious risk of infusion related reactions and to further characterize the risks of severe adverse reactions including myelosuppression, hypokalemia, hyperglycemia, non-infectious pneumonitis, hypersensitivity, and their sequelae in a sufficient number of patients exposed to Fyarro; and to identify risk factors for development of these adverse reactions. The study should include appropriate monitoring and risk mitigation strategies."~"Submitted"~~6/30/2025 0:00:00~10/14/2025 0:00:00
384227~"CDER"~"NDA"~213312.00~"AADI BIOSCIENCES INC"~"Fyarro (sirolimus protein-bound particles for injectable suspension (albumin-bound))"~11/22/2021 0:00:00~"Original"~1~"4177-2"~"PMR"~"505 (o)(3)"~4177.00~2~"PMR 4177-2: Conduct a hepatic impairment clinical trial to evaluate the pharmacokinetics and safety of Fyarro in subjects with mild, moderate, and severe hepatic impairment (based on NCI criteria) and determine the magnitude of increase in exposure and appropriate dosage. Design and conduct the trial in accordance with the FDA Guidance for Industry titled:  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Submitted"~~3/31/2024 0:00:00~10/14/2025 0:00:00
384228~"CDER"~"NDA"~213312.00~"AADI BIOSCIENCES INC"~"Fyarro (sirolimus protein-bound particles for injectable suspension (albumin-bound))"~11/22/2021 0:00:00~"Original"~1~"4177-3"~"PMR"~"505 (o)(3)"~4177.00~3~"PMR 4177-3: Conduct a clinical trial to assess the potential risk of QT interval prolongation and the effect of Fyarro on the QT interval. Design and conduct the trial in accordance with the ICH E14 Guidance for Industry titled:  E14 Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non-Antiarrhythmic Drugs. "~"Submitted"~~6/30/2025 0:00:00~10/14/2025 0:00:00
384229~"CDER"~"NDA"~213378.00~"ALKERMES INC"~"LYBALVI (olanzapine and samidorphan)"~5/28/2021 0:00:00~"Original"~1~"3905-2"~"PMR"~"Pediatric Research Equity Act"~3905.00~2~"PMR 3905-2: Conduct a long-term (at least 12-month), open-label study to evaluate the safety and tolerability of LYBALVI (olanzapine/samidorphan) in patients 13 to 17 years with schizophrenia and patients 10 to 17 years with bipolar I disorder."~"Ongoing"~"Original Final Report Due Date: 03/31/2028; Deferral Extension granted per FDA letter dated 6/08/2026. The study completion milestone was also revised."~3/31/2028 0:00:00~7/25/2025 0:00:00
384230~"CDER"~"NDA"~213378.00~"ALKERMES INC"~"LYBALVI (olanzapine and samidorphan)"~5/28/2021 0:00:00~"Original"~1~"3905-4"~"PMR"~"Pediatric Research Equity Act"~3905.00~4~"PMR 3905-4: Conduct a 52-week, double-blind, active-controlled study enrolling both patients 13 to 17 years with schizophrenia and 10 to 17 years with bipolar I disorder. The study must be adequately powered to evaluate change in weight at 12 weeks, with continuation through 52 weeks to evaluate the safety of LYBALVI (olanzapine/samidorphan)."~"Ongoing"~"Original Final Report Due Date: 03/31/2027; Deferral Extension granted per FDA letter dated 6/08/2026. The study completion milestone was also revised. No milestone has been missed at this time."~3/31/2029 0:00:00~7/25/2025 0:00:00
384231~"CDER"~"NDA"~213378.00~"ALKERMES INC"~"LYBALVI (olanzapine and samidorphan)"~5/28/2021 0:00:00~"Original"~2~"3905-2"~"PMR"~"Pediatric Research Equity Act"~3905.00~2~"PMR 3905-2: Conduct a long-term (at least 12-month), open-label study to evaluate the safety and tolerability of LYBALVI (olanzapine/samidorphan) in patients 13 to 17 years with schizophrenia and patients 10 to 17 years with bipolar I disorder."~"Ongoing"~"Original Final Report Due Date: 03/31/2028; Deferral Extension granted per FDA letter dated 6/08/2026. The study completion milestone was also revised."~3/31/2028 0:00:00~7/25/2025 0:00:00
384232~"CDER"~"NDA"~213378.00~"ALKERMES INC"~"LYBALVI (olanzapine and samidorphan)"~5/28/2021 0:00:00~"Original"~2~"3905-4"~"PMR"~"Pediatric Research Equity Act"~3905.00~4~"PMR 3905-4: Conduct a 52-week, double-blind, active-controlled study enrolling both patients 13 to 17 years with schizophrenia and 10 to 17 years with bipolar I disorder. The study must be adequately powered to evaluate change in weight at 12 weeks, with continuation through 52 weeks to evaluate the safety of LYBALVI (olanzapine/samidorphan)."~"Ongoing"~"Original Final Report Due Date: 03/31/2027; Deferral Extension granted per FDA letter dated 6/08/2026. The study completion milestone was also revised. No milestone has been missed at this time."~3/31/2029 0:00:00~7/25/2025 0:00:00
384233~"CDER"~"NDA"~215039.00~"NOVARTIS PHARMACEUTICALS CORP"~"Vijoice (Alpelisib)"~4/5/2022 0:00:00~"Original"~1~"4260-2"~"PMR"~"505 (o)(3)"~4260.00~2~"PMR 4260-2: Conduct comprehensive safety analyses from clinical studies that further characterize the potential serious risk of long-term adverse effects of alpelisib on growth and development, including an assessment of growth plate abnormalities and development of teeth in a sufficient number of pediatric patients. Monitor patients for growth and development using age- appropriate screening tools. Include evaluations of growth as measured by height, weight, height velocity and height standard deviation scores, age at adrenarche if applicable, age at menarche if applicable (females) and Tanner stage. Monitor patients until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first."~"Ongoing"~~9/30/2030 0:00:00~6/1/2026 0:00:00
384234~"CDER"~"NDA"~215039.00~"NOVARTIS PHARMACEUTICALS CORP"~"Vijoice (Alpelisib)"~4/5/2022 0:00:00~"Original"~1~"4260-3"~"PMR"~"505 (o)(3)"~4260.00~3~"PMR 4260-3: Conduct comprehensive safety analyses from ongoing trials to further assess the serious risks of alpelisib, including severe hypersensitivity, severe cutaneous adverse reactions and pneumonitis, in patients with PROS over a sufficient period of follow-up time to characterize these risks. The analysis should include appropriate monitoring and risk mitigation strategies."~"Ongoing"~~9/30/2030 0:00:00~6/1/2026 0:00:00
384235~"CDER"~"NDA"~215039.00~"NOVARTIS PHARMACEUTICALS CORP"~"Vijoice (Alpelisib)"~4/5/2022 0:00:00~"Original"~1~"4260-5"~"PMR"~"505 (o)(3)"~4260.00~5~"PMR 4260-5: Conduct a carcinogenicity study in mice to evaluate the potential for carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Delayed"~"The final protocol submission milestone was missed because of additional time needed to conduct an additional dose range finding study. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 12/01/2025. In the same letter, FDA also acknowledged that there was sufficient justification for the anticipated delay of the study completion, and final report submission proposed revised milestones."~7/31/2027 0:00:00~6/1/2026 0:00:00
384236~"CDER"~"NDA"~215039.00~"NOVARTIS PHARMACEUTICALS CORP"~"Vijoice (Alpelisib)"~4/5/2022 0:00:00~"Original"~1~"4260-6"~"PMC"~"506B PMC"~4260.00~6~"PMC 4260-6: Conduct a study using a higher starting dose of alpelisib (i.e., 125 mg) in addition to the 50 mg once daily starting dose in pediatric patients 6 to 17 years of age to evaluate the pharmacokinetics, safety, and clinical outcomes (including confirmed objective response rate and duration of response) for dose optimization in this patient population."~"Ongoing"~~8/31/2027 0:00:00~6/1/2026 0:00:00
384237~"CDER"~"NDA"~215039.00~"NOVARTIS PHARMACEUTICALS CORP"~"Vijoice (Alpelisib)"~4/5/2022 0:00:00~"Original"~1~"4260-7"~"PMC"~"506B PMC"~4260.00~7~"PMC 4260-7: Conduct an analysis from EPIK-P3, an ongoing single-arm study, to further describe the long-term outcomes of patients receiving alpelisib for the treatment of severe manifestations of PROS. Evaluate a sufficient number of patients over a sufficient period of time to better characterize clinical response over time, and include case narratives describing additional patient outcome measures (such as clinical outcomes assessments) to support assessment of benefit of alpelisib in this patient population."~"Ongoing"~~9/30/2028 0:00:00~6/1/2026 0:00:00
384238~"CDER"~"NDA"~215064.00~"CHIESI FARMACEUTICI SPA"~"Filsuvez (birch triterpenes)"~12/18/2023 0:00:00~"Original"~1~"4568-1"~"PMR"~"505 (o)(3)"~4568.00~1~"PMR 4568-1: Conduct a dermal carcinogenicity study."~"Delayed"~"The study was initiated. The Final Report submission date 03/2025 was missed. The Applicant submitted a request for a revised milestones on 4/24/25 with the Agency response pending."~3/31/2025 0:00:00~2/13/2026 0:00:00
384239~"CDER"~"NDA"~215092.00~"OCUVEX THERAPEUTICS INC"~"Omlonti (Omidenepag Isoprpopyl)"~9/22/2022 0:00:00~"Original"~1~"4339-1"~"PMR"~"505 (o)(3)"~4339.00~1~"PMR 4339-1: Conduct a randomized, controlled trial to evaluate the corneal endothelial health of eyes treated with omidenepag isopropyl ophthalmic solution by monitoring the number/density of corneal endothelial cells using specular microscopy at baseline and over a period of at least one year in at least 100 patients receiving omidenepag isopropyl ophthalmic solution."~"Ongoing"~~12/31/2026 0:00:00~11/25/2025 0:00:00
384240~"CDER"~"NDA"~215151.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna (vonoprazan)"~11/1/2023 0:00:00~"Original"~1~"4367-1"~"PMR"~"505 (o)(3)"~4367.00~1~"PMR 4367-1: Conduct a prospective, registry based, observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to vonoprazan-containing products during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~4/30/2036 0:00:00~7/2/2026 0:00:00
384241~"CDER"~"NDA"~215151.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna (vonoprazan)"~11/1/2023 0:00:00~"Original"~1~"4367-2"~"PMR"~"505 (o)(3)"~4367.00~2~"PMR 4367-2: An additional pregnancy study that uses a different design from the Pregnancy Registry (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to vonoprazan-containing products during pregnancy compared to an unexposed control population."~"Ongoing"~~4/30/2032 0:00:00~7/2/2026 0:00:00
384242~"CDER"~"NDA"~215151.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna (vonoprazan)"~11/1/2023 0:00:00~"Original"~1~"4367-5"~"PMR"~"Pediatric Research Equity Act"~4367.00~5~"PMR 4367-5: Conduct a randomized, parallel-group, multiple-dose, 14-day, dose ranging study to evaluate the pharmacokinetics and safety of Voquezna (vonoprazan) in pediatric patients =1 month to <1 year of age with erosive esophagitis and pediatric patients =1 year to <6 years of age with gastroesophageal reflux disease with or without erosive esophagitis."~"Delayed"~"The final protocol milestone was missed."~6/30/2027 0:00:00~7/2/2026 0:00:00
384243~"CDER"~"NDA"~215151.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna (vonoprazan)"~11/1/2023 0:00:00~"Original"~1~"4367-6"~"PMR"~"Pediatric Research Equity Act"~4367.00~6~"PMR 4367-6: Conduct a trial to evaluate the efficacy and safety of Voquezna (vonoprazan) for the healing of endoscopically confirmed erosive esophagitis and relief of heartburn symptoms and the maintenance of healed erosive esophagitis and relief of heartburn symptoms in pediatric patients =1 month to <18 years of age with endoscopically confirmed erosive esophagitis. The trial will include two study periods: an 8-week healing phase and a randomized, blinded, placebo-controlled 24-week maintenance phase."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~2/28/2031 0:00:00~7/2/2026 0:00:00
384244~"CDER"~"NDA"~215151.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna (vonoprazan)"~11/1/2023 0:00:00~"Original"~1~"4367-7"~"PMR"~"Pediatric Research Equity Act"~4367.00~7~"PMR 4367-7: Develop an age-appropriate formulation for pediatric patients 1 month to <6 months of age."~"Delayed"~"The final report milestone was missed."~1/31/2025 0:00:00~7/2/2026 0:00:00
384245~"CDER"~"NDA"~216386.00~"PFIZER INC"~"Zavzpret (zavegepant)"~3/9/2023 0:00:00~"Original"~1~"4408-3"~"PMR"~"Pediatric Research Equity Act"~4408.00~3~"PMR 4408-3: A randomized, double-blind, placebo-controlled efficacy and safety study under PREA for the acute treatment of migraine with or without aura in patients ages 6 through 17 years. This study includes an initial single-blind-placebo lead-in phase to identify placebo non-responders to enroll in the efficacy portion of the trial. This efficacy study must be designed to show superiority of zavegepant over placebo."~"Delayed"~"The Final Protocol Submission milestone was missed due to
continued discussion with the FDA regarding the trial design."~11/30/2032 0:00:00~5/6/2026 0:00:00
384246~"CDER"~"NDA"~216386.00~"PFIZER INC"~"Zavzpret (zavegepant)"~3/9/2023 0:00:00~"Original"~1~"4408-4"~"PMR"~"Pediatric Research Equity Act"~4408.00~4~"PMR 4408-4: pediatric open-label safety study under PREA to evaluate the long-term safety of zavegepant in migraine patients ages 6 through 17 years. This study should be a minimum of one year in length. This study should include a minimum of 200 patients treating, on average, one migraine attack per month for 6 months; and 75 patients treating, on average, at least one migraine attack per month for one year."~"Ongoing"~"The trial is underway."~11/30/2032 0:00:00~5/6/2026 0:00:00
384247~"CDER"~"NDA"~216386.00~"PFIZER INC"~"Zavzpret (zavegepant)"~3/9/2023 0:00:00~"Original"~1~"4408-5"~"PMR"~"505 (o)(3)"~4408.00~5~"PMR 4408-5: Prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with migraine exposed to zavegepant during pregnancy with two unexposed control populations: one consisting of women with migraine who have not been exposed to zavegepant before or during pregnancy, and the other consisting of women without migraine. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~6/30/2038 0:00:00~5/6/2026 0:00:00
384248~"CDER"~"NDA"~216386.00~"PFIZER INC"~"Zavzpret (zavegepant)"~3/9/2023 0:00:00~"Original"~1~"4408-6"~"PMR"~"505 (o)(3)"~4408.00~6~"PMR 4408-6: A pregnancy outcomes study using a different study design than provided for in PMR 4408-5 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case-control study) to assess pregnancy complications, major congenital malformations, spontaneous abortions, stillbirths, and small-for-gestational-age births in women exposed to zavegepant during pregnancy compared to an unexposed control population."~"Ongoing"~~6/30/2032 0:00:00~5/6/2026 0:00:00
384249~"CDER"~"NDA"~216403.00~"TRAVERE THERAPEUTICS INC"~"Filspari (sparsentan)"~2/17/2023 0:00:00~"Original"~1~"4330-5"~"PMR"~"505 (o)(3)"~4330.00~5~"PMR 4330-5: Conduct a prospective, single-arm safety study of patients exposed to sparsentan, with two years of follow-up to assess and characterize the risk of drug-induced liver injury (DILI). This study should analyze the clinical features of DILI cases with sparsentan, such as the injury s severity, type, latency, and specifically evaluate the incidence of Hy s law cases. Information for liver injury cases should be captured with structured follow up (e.g., monthly monitoring of serum liver tests) including dechallenge and rechallenge results. A hepatic adjudication committee (HAC) should assess both the severity of the liver injury and sparsentan s role in its development (i.e., causality). This study should aim to enroll enough patients such that if 0 events of Hy s law are observed, then the upper bound of the 95% confidence interval for the rate of Hy s law will be 1/1000."~"Delayed"~"The final protocol was submitted after the original milestone."~4/30/2028 0:00:00~4/8/2026 0:00:00
384250~"CDER"~"NDA"~216457.00~"HERON THERAPEUTICS INC"~"Aponvie (aprepitant)"~9/16/2022 0:00:00~"Original"~1~"4292-1"~"PMR"~"Pediatric Research Equity Act"~4292.00~1~"PMR 4292-1: A randomized, double-blind, active-controlled, dose-ranging trial to evaluate the efficacy, safety, and pharmacokinetics of Aponvie
(aprepitant) injectable emulsion as prophylaxis for postoperative nausea and vomiting in children =12 years to =17 years of age."~"Delayed"~"Deferral Extension Requested 11/24/2025. Denied per FDA letter dated 12/17/2025."~2/28/2026 0:00:00~10/31/2025 0:00:00
384251~"CDER"~"NDA"~216457.00~"HERON THERAPEUTICS INC"~"Aponvie (aprepitant)"~9/16/2022 0:00:00~"Original"~1~"4292-2"~"PMR"~"Pediatric Research Equity Act"~4292.00~2~"PMR 4292-2: A randomized, double-blind, active-controlled, dose-ranging trial to evaluate the efficacy, safety, and pharmacokinetics of Aponvie
(aprepitant) injectable emulsion as prophylaxis for postoperative nausea and vomiting in children =2 years to <12 years."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~10/31/2029 0:00:00~10/31/2025 0:00:00
384252~"CDER"~"NDA"~216457.00~"HERON THERAPEUTICS INC"~"Aponvie (aprepitant)"~9/16/2022 0:00:00~"Original"~1~"4292-3"~"PMR"~"Pediatric Research Equity Act"~4292.00~3~"PMR 4292-3: A randomized, double-blind, active-controlled, dose-ranging trial to evaluate the efficacy, safety, and pharmacokinetics of Aponvie
(aprepitant) injectable emulsion as prophylaxis for postoperative nausea and vomiting in children birth to <2 years."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~10/31/2029 0:00:00~10/31/2025 0:00:00
384253~"CDER"~"NDA"~216483.00~"ALEMBIC THERAPEUTICS LLC"~"Pivya (pivmecillinam)"~4/24/2024 0:00:00~"Original"~1~"4603-2"~"PMR"~"Pediatric Research Equity Act"~4603.00~2~"PMR 4603-2: Conduct a multicenter, randomized, single-blind study to evaluate the safety and tolerability of pivmecillinam hydrochloride versus nitrofurantoin for the treatment of pediatric subjects, 12 years to less than 18 years of age, with uncomplicated urinary tract infections."~"Pending"~"Final protocol was submitted 11-2024."~11/30/2026 0:00:00~6/23/2026 0:00:00
384254~"CDER"~"NDA"~216483.00~"ALEMBIC THERAPEUTICS LLC"~"Pivya (pivmecillinam)"~4/24/2024 0:00:00~"Original"~1~"4603-4"~"PMR"~"505 (o)(3)"~4603.00~4~"PMR 4603-4: Conduct a lactation study (milk only) in lactating women who have received pivmecillinam to assess concentrations of pivmecillinam in breast milk using a validated assay and to assess the effects on the breastfed infant."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone.  The final protocol was acknowledged in a letter dated 03/05/2025."~5/31/2026 0:00:00~6/23/2026 0:00:00
384255~"CDER"~"NDA"~216483.00~"ALEMBIC THERAPEUTICS LLC"~"Pivya (pivmecillinam)"~4/24/2024 0:00:00~"Original"~1~"4603-5"~"PMR"~"505 (o)(3)"~4603.00~5~"PMR 4603-5: Conduct a U.S. surveillance study over a five-year period after the introduction of pivmecillinam to the market to determine if resistance or decreased susceptibility to pivmecillinam is occurring in the target population of bacteria that are in the approved pivmecillinam label."~"Pending"~~8/31/2030 0:00:00~6/23/2026 0:00:00
384256~"CDER"~"NDA"~216483.00~"ALEMBIC THERAPEUTICS LLC"~"Pivya (pivmecillinam)"~4/24/2024 0:00:00~"Original"~1~"4603-7"~"PMR"~"Pediatric Research Equity Act"~4603.00~7~"PMR 4603-7: Conduct an open label single-dose safety, tolerability and pharmacokinetic study in pediatric subjects 2 years to less than 12 years of age receiving systemic antibacterial therapy for the treatment of a suspected or confirmed bacterial infection."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed"~8/31/2028 0:00:00~6/23/2026 0:00:00
384257~"CDER"~"NDA"~211723.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~1/23/2020 0:00:00~"Original"~1~"3872-5"~"PMC"~"506B PMC"~3872.00~5~"PMC 3872-5: Submit the final results and datasets for overall response rate and duration of response from a clinical trial in patients with relapsed or refractory follicular lymphoma whose tumors do not have an EZH2 mutation and have received prior therapies including bendamustine, binutuzumab, rituximab plus lenalidomide, and other therapies consistent with those used in a U.S. population to further characterize the clinical benefit of tazemetostat monotherapy that may inform product labeling."~"Delayed"~"The Study Completion and Final Report Due Date milestones were missed due to a planned new study intended to fulfill this PMC."~12/31/2023 0:00:00~3/20/2026 0:00:00
384258~"CDER"~"NDA"~211723.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~1/23/2020 0:00:00~"Original"~1~"3872-6"~"PMC"~"506B PMC"~3872.00~6~"PMC 3872-6: Submit a final report containing data from clinical trials, post-marketing reports, compassionate use/expanded access program, real-world evidence, and other sources to further characterize the safety and efficacy of tazemetostat monotherapy and tazemetostat in combination with other immunotherapy among U.S. racial and ethnic minority patients with follicular lymphoma."~"Ongoing"~~3/31/2026 0:00:00~3/20/2026 0:00:00
384259~"CDER"~"NDA"~211728.00~"UROGEN PHARMA LTD"~"JELMYTO (mitomycin)"~4/15/2020 0:00:00~"Original"~1~"3832-1"~"PMC"~"506B PMC"~3832.00~1~"PMC 3832-1: Provide annual updates for the duration of response (median and range) for all patients with ongoing complete responses enrolled in clinical trial OLYMPUS. These updates should include information regarding incidence of recurrence of low-grade upper tract urothelial carcinoma (UTUC), incidence of/progression to high-grade UTUC, and incidence of nephroureterectomy. Annual updates should continue for five years or until all patients have either experienced recurrence, death, or been lost to follow up."~"Fulfilled"~~4/30/2025 0:00:00~6/4/2025 0:00:00
384260~"CDER"~"NDA"~211733.00~"HALEON US HOLDINGS LLC"~"Advil Dual Action with Acetaminophen (Ibuprofen and Acetaminophen)"~2/28/2020 0:00:00~"Original"~1~"3808-3"~"PMR"~"Pediatric Research Equity Act"~3808.00~3~"PMR 3808-3: Pediatric targeted Label Comprehension Study for caregivers of children from birth to less than 12 years of age. The objective of the study is to ensure that caregivers of children under the age of 12 adequately understand the labeling."~"Delayed"~"The final protocol submission and study completion milestones were missed. A deferral extension for the final report submission and revised proposed milestones for the final protocol and study completion milestones were granted and acknowledged in a 4-11-2025 letter."~5/31/2028 0:00:00~4/27/2026 0:00:00
384261~"CDER"~"NDA"~211733.00~"HALEON US HOLDINGS LLC"~"Advil Dual Action with Acetaminophen (Ibuprofen and Acetaminophen)"~2/28/2020 0:00:00~"Original"~1~"3808-4"~"PMR"~"Pediatric Research Equity Act"~3808.00~4~"PMR 3808-4: Pediatric Self-Selection Study. The design of this trial will be based on the results of the Label Comprehension Study (PMR 3808-3).
"~"Delayed"~"The final protocol submission was missed. A deferral extension for the final report submission and revised proposed milestones for the final protocol and study completion milestones were granted and acknowledged in a 4-11-2025 letter."~10/31/2029 0:00:00~4/27/2026 0:00:00
384262~"CDER"~"NDA"~211733.00~"HALEON US HOLDINGS LLC"~"Advil Dual Action with Acetaminophen (Ibuprofen and Acetaminophen)"~2/28/2020 0:00:00~"Original"~1~"3808-5"~"PMR"~"Pediatric Research Equity Act"~3808.00~5~"PMR 3808-5: Pediatric Actual Use Study. The design of this trial will be based on the results of the Pediatric Label Comprehension Study (PMR 3808-3)."~"Delayed"~"The final protocol submission has passed. A deferral extension for the final report submission and revised proposed milestones for the final protocol and study completion milestones were granted and acknowledged in a 4-11-2025 letter."~10/31/2029 0:00:00~4/27/2026 0:00:00
384263~"CDER"~"NDA"~211733.00~"HALEON US HOLDINGS LLC"~"Advil Dual Action with Acetaminophen (Ibuprofen and Acetaminophen)"~2/28/2020 0:00:00~"Original"~1~"3808-6"~"PMR"~"Pediatric Research Equity Act"~3808.00~6~"PMR 3808-6: Conduct a pharmacokinetic (PK) assessment in children 6 years to less than 12 years of age to evaluate the PK profile of ibuprofen/acetaminophen fixed-dose combination in children 6 years to less than 12 years."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~2/28/2027 0:00:00~4/27/2026 0:00:00
384264~"CDER"~"NDA"~211746.00~"GLENMARK SPECIALTY SA"~"Ryaltris (Olopatadine Hydrochloride and Mometasone Furoate)"~1/13/2022 0:00:00~"Original"~1~"4215-1"~"PMR"~"Pediatric Research Equity Act"~4215.00~1~"PMR 4215-1: Conduct a 14-day, randomized, double-blind, placebo-controlled parallelgroup efficacy and safety study in children 6 to 11 years of age with seasonal allergic rhinitis."~"Submitted"~"The Final Report was submitted to FDA on 9/26/2025."~1/10/2022 0:00:00~3/9/2026 0:00:00
384265~"CDER"~"NDA"~211765.00~"ABBVIE INC"~"UBRELVY (Ubrogepant)"~12/23/2019 0:00:00~"Original"~1~"3769-2"~"PMR"~"Pediatric Research Equity Act"~3769.00~2~"PMR 3769-2: A randomized, double-blind, placebo-controlled efficacy and safety study under PREA to evaluate two doses of Ubrelvy (high-dose and low-dose) compared to placebo during a single migraine attack in pediatric patients ages 6 to less than 18 years. This study will include an open-label pharmacokinetic cohort in order to select appropriate doses for patients 6-11 years old in the efficacy portion of the study. This efficacy study must be designed to show superiority of Ubrelvy over placebo and is to be submitted as a special protocol assessment (SPA)."~"Ongoing"~"The trial is underway. Per the applicant s Annual Status Report, 709 subjects are currently enrolled in the trial."~12/31/2027 0:00:00~2/17/2026 0:00:00
384266~"CDER"~"NDA"~211765.00~"ABBVIE INC"~"UBRELVY (Ubrogepant)"~12/23/2019 0:00:00~"Original"~1~"3769-3"~"PMR"~"Pediatric Research Equity Act"~3769.00~3~"PMR 3769-3: An open-label, long-term safety study under PREA in pediatric patients ages 6 to 17 years, for up to one year."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 9/21/2021. Per the applicant s Annual Status Report, 685 subjects are currently enrolled in the trial."~12/31/2027 0:00:00~2/17/2026 0:00:00
384267~"CDER"~"NDA"~211765.00~"ABBVIE INC"~"UBRELVY (Ubrogepant)"~12/23/2019 0:00:00~"Original"~1~"3769-4"~"PMR"~"505 (o)(3)"~3769.00~4~"PMR 3769-4: Conduct prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with migraine exposed to Ubrelvy during pregnancy with two unexposed comparator populations: an internal comparator consisting of women with migraine who have not been exposed to Ubrelvy before or during pregnancy, and a comparator consisting of women without migraine. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development will be assessed through at least the first year of life."~"Ongoing"~~2/28/2035 0:00:00~2/17/2026 0:00:00
384268~"CDER"~"NDA"~211765.00~"ABBVIE INC"~"UBRELVY (Ubrogepant)"~12/23/2019 0:00:00~"Original"~1~"3769-5"~"PMR"~"505 (o)(3)"~3769.00~5~"PMR 3769-5: Conduct a pregnancy outcomes study using a different study design than provided for in PMR 3769-4 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-forgestational-age births in women exposed to Ubrelvy during pregnancy compared to an unexposed control population."~"Ongoing"~~2/28/2029 0:00:00~2/17/2026 0:00:00
384269~"CDER"~"NDA"~213388.00~"ABBVIE INC"~"Oriahnn (elagolix, estradiol and norethindrone acetate)"~5/29/2020 0:00:00~"Original"~1~"3837-2"~"PMR"~"505 (o)(3)"~3837.00~2~"PMR 3837-2: A retrospective cohort study in a claims-based database to evaluate the adverse effects of elagolix-containing products, including Oriahnn, on pregnancy-related outcomes."~"Delayed"~"The final report submission milestone was missed because of challenges in reaching the target sample size due to lower-than-expected accrual of pregnancies exposed to elagolix. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 01/30/2026. The study completion milestone was also revised."~1/31/2025 0:00:00~7/23/2025 0:00:00
384270~"CDER"~"NDA"~213388.00~"ABBVIE INC"~"Oriahnn (elagolix, estradiol and norethindrone acetate)"~5/29/2020 0:00:00~"Original"~1~"3837-3"~"PMR"~"505 (o)(3)"~3837.00~3~"PMR 3837-3: A prospective observational study in premenopausal women receiving treatment with Oriahnn to assess the incidence rate, time to onset, pattern, extent, and reversibility of alopecia, as well as any racial/ethnic differences in developing alopecia. Physician/observer-reported outcome and/or patient survey should be developed and included in the PMR study to capture timing, pattern, extent, and reversibility of alopecia cases. The study shall evaluate 50 cases of alopecia."~"Released"~~12/31/2027 0:00:00~7/23/2025 0:00:00
384271~"CDER"~"NDA"~213388.00~"ABBVIE INC"~"Oriahnn (elagolix, estradiol and norethindrone acetate)"~5/29/2020 0:00:00~"Original"~1~"3837-4"~"PMR"~"505 (o)(3)"~3837.00~4~"PMR 3837-4: A cohort study to compare the incidence rate of alopecia in premenopausal women who initiate Oriahnn and an appropriate comparator population of women not treated with Oriahnn. The study should be powered to detect a 2-fold increase in the risk for alopecia with Oriahnn use. The study should also be powered for a subgroup analysis among African Americans who are treated with Oriahnn. If an electronic healthcare database is selected for the study, then conduct a validation study in the selected database to develop and validate an algorithm with a sufficient positive predictive value (PPV) to identify alopecia, prior to initiating the comparative safety study. If a sufficient PPV cannot be obtained, conduct a prospective cohort study with primary data collection with case adjudication."~"Ongoing"~~12/31/2027 0:00:00~7/23/2025 0:00:00
384272~"CDER"~"NDA"~213400.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~6/18/2020 0:00:00~"Original"~1~"3872-1"~"PMR"~"Accelerated Approval"~3872.00~1~"PMR 3872-1: Submit the final report and datasets from a randomized, phase 3 clinical trial that verifies and describes the clinical benefit of tazemetostat in patients with relapsed or refractory follicular lymphoma whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test. The trial should include sufficient numbers of racial and ethnic minority patients to better reflect the U.S. patient population and allow for the interpretation of the results in these patient populations. Patients should be randomized to receive immunotherapy with or without tazemetostat. The primary endpoint should be progression-free survival, with secondary endpoints that include overall survival and objective response rate."~"Ongoing"~"187 patients were enrolled in phase 3 out of approximately 568
patients total."~7/31/2025 0:00:00~
384273~"CDER"~"NDA"~213400.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~6/18/2020 0:00:00~"Original"~1~"3872-2"~"PMR"~"Accelerated Approval"~3872.00~2~"PMR 3872-2: Submit the final report and datasets from a randomized, phase 3 clinical trial that verifies and describes the clinical benefit of tazemetostat in patients with relapsed or refractory follicular lymphoma whose tumors do not have an EZH2 mutation as detected by an FDA-approved test. The trial should include sufficient numbers of racial and ethnic minority patients to better reflect the U.S. patient population and allow for the interpretation of the results in these patient populations. Patients should be randomized to receive immunotherapy with or without tazemetostat. The primary endpoint should be progression-free survival, with secondary endpoints that include overall survival and objective response rate."~"Ongoing"~"187 patients were enrolled in phase 3 out of approximately 568
patients total."~7/31/2025 0:00:00~
384274~"CDER"~"NDA"~213400.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~6/18/2020 0:00:00~"Original"~1~"3872-3"~"PMR"~"505 (o)(3)"~3872.00~3~"PMR 3872-3: Submit annual safety updates for 10 years from a cumulative, integrated safety analyses of patients with lymphoid malignancies enrolled in clinical trials and from post-marketing reports to characterize the risk of acute myeloid leukemia, myelodysplastic syndrome, T-lymphoblastic lymphoma, and other secondary malignancies in patients receiving TAZVERIK. Include incidence rates, time to onset, predisposing factors, and outcomes in the interim and final reports. These safety evaluations may inform labeling of patient populations at highest risk and to provide evidencebased monitoring recommendations."~"Ongoing"~~4/30/2030 0:00:00~
384275~"CDER"~"NDA"~213400.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~6/18/2020 0:00:00~"Original"~1~"3872-5"~"PMC"~"506B PMC"~3872.00~5~"PMC 3872-5: Submit the final results and datasets for overall response rate and duration of response from a clinical trial in patients with relapsed or refractory follicular lymphoma whose tumors do not have an EZH2 mutation and have received prior therapies including bendamustine, binutuzumab, rituximab plus lenalidomide, and other therapies consistent with those used in a U.S. population to further characterize the clinical benefit of tazemetostat monotherapy that may inform product labeling."~"Delayed"~"The Study Completion and Final Report Due Date milestones were missed due to a planned new study intended to fulfill this PMC."~12/31/2023 0:00:00~
384276~"CDER"~"NDA"~213400.00~"EPIZYME INC"~"Tazverik (tazemetostat)"~6/18/2020 0:00:00~"Original"~1~"3872-6"~"PMC"~"506B PMC"~3872.00~6~"PMC 3872-6: Submit a final report containing data from clinical trials, post-marketing reports, compassionate use/expanded access program, real-world evidence, and other sources to further characterize the safety and efficacy of tazemetostat monotherapy and tazemetostat in combination with other immunotherapy among U.S. racial and ethnic minority patients with follicular lymphoma."~"Ongoing"~~3/31/2026 0:00:00~
384277~"CDER"~"NDA"~213411.00~"SEAGEN INC"~"Tukysa (tucatinib)"~4/17/2020 0:00:00~"Supplement"~4~"4388-1"~"PMR"~"Accelerated Approval"~4388.00~1~"PMR 4388-1: Conduct a randomized clinical trial to obtain data on the clinical efficacy of tucatinib for patients with RAS wild type, HER2-positive, unresectable or metastatic colorectal carcinoma. The trial should compare tucatinib in combination with trastuzumab with the standard of care in patients with RAS wild type, HER2-positive, unresectable or metastatic colorectal carcinoma. The primary endpoint should be progression-free survival (PFS) per blinded assessment or overall survival. The trial should enroll a sufficiently representative study population to reflect the racial and ethnic diversity of the U.S. patient population with RAS wild type, HER2-positive, unresectable or metastatic colorectal carcinoma and allow for interpretation of the results across this representative study population."~"Delayed"~"The applicant requested revised milestones because study enrollment has progressed slower than initially anticipated due to low prevalence of HER2+ colorectal cancer. Revised milestones were acknowledged in a letter dated 03/26/2026."~4/30/2026 0:00:00~6/11/2026 0:00:00
384278~"CDER"~"NDA"~215151.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna (vonoprazan)"~11/1/2023 0:00:00~"Supplement"~6~"4580-1"~"PMR"~"Pediatric Research Equity Act"~4580.00~1~"PMR 4580-1: Conduct a randomized, blinded, and placebo-controlled study to evaluate the efficacy and safety of Voquezna (vonoprazan) for the relief of heartburn associated with non-erosive gastroesophageal reflux disease (GERD) in pediatric patients 12 months to less than 18 years of age."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~2/28/2031 0:00:00~7/2/2026 0:00:00
384279~"CDER"~"NDA"~215152.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna Triple Pak (vonoprazan, amoxicillin, and clarithromycin)"~5/3/2022 0:00:00~"Original"~1~"4270-1"~"PMR"~"505 (o)(3)"~4270.00~1~"PMR 4270-1: Conduct a prospective, registry based, observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to vonoprazan-containing products during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~4/30/2036 0:00:00~7/2/2026 0:00:00
384280~"CDER"~"NDA"~215152.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna Triple Pak (vonoprazan, amoxicillin, and clarithromycin)"~5/3/2022 0:00:00~"Original"~1~"4270-2"~"PMR"~"505 (o)(3)"~4270.00~2~"PMR 4270-2: An additional pregnancy study that uses a different design from the Pregnancy Registry (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to vonoprazancontaining products during pregnancy compared to an unexposed control population."~"Ongoing"~~4/30/2031 0:00:00~7/2/2026 0:00:00
384281~"CDER"~"NDA"~215152.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna Triple Pak (vonoprazan, amoxicillin, and clarithromycin)"~5/3/2022 0:00:00~"Original"~1~"4270-3"~"PMR"~"505 (o)(3)"~4270.00~3~"PMR 4270-3: Conduct a lactation study (milk only) in lactating women who have received vonoprazan-containing products to assess concentrations of vonoprazan in breast milk using a validated assay. A mother-infant pair study may be required in the future depending on the results of this milk-only study."~"Fulfilled"~~5/31/2025 0:00:00~7/2/2026 0:00:00
384282~"CDER"~"NDA"~215153.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna Dual Pak (vonoprazan and amoxicillin)"~5/3/2022 0:00:00~"Original"~1~"4270-1"~"PMR"~"505 (o)(3)"~4270.00~1~"PMR 4270-1: Conduct a prospective, registry based, observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to vonoprazan-containing products during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~4/30/2036 0:00:00~7/2/2026 0:00:00
384283~"CDER"~"NDA"~215153.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna Dual Pak (vonoprazan and amoxicillin)"~5/3/2022 0:00:00~"Original"~1~"4270-2"~"PMR"~"505 (o)(3)"~4270.00~2~"PMR 4270-2: An additional pregnancy study that uses a different design from the Pregnancy Registry (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to vonoprazancontaining products during pregnancy compared to an unexposed control population."~"Ongoing"~~4/30/2031 0:00:00~7/2/2026 0:00:00
384284~"CDER"~"NDA"~215153.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna Dual Pak (vonoprazan and amoxicillin)"~5/3/2022 0:00:00~"Original"~1~"4270-3"~"PMR"~"505 (o)(3)"~4270.00~3~"PMR 4270-3: Conduct a lactation study (milk only) in lactating women who have received vonoprazan-containing products to assess concentrations of vonoprazan in breast milk using a validated assay. A mother-infant pair study may be required in the future depending on the results of this milk-only study."~"Fulfilled"~~5/31/2025 0:00:00~7/2/2026 0:00:00
384285~"CDER"~"NDA"~215192.00~"AKEBIA THERAPEUTICS INC"~"Vafseo (Vadadustat)"~3/27/2024 0:00:00~"Original"~1~"4613-1"~"PMR"~"Pediatric Research Equity Act"~4613.00~1~"PMR 4613-1: Conduct a trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of Vafseo for the treatment of anemia associated with chronic kidney disease in children and adolescents aged 3 months to under 17 years requiring dialysis. Submit datasets at the time of the final clinical study report submission."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~4/30/2031 0:00:00~5/19/2026 0:00:00
384286~"CDER"~"NDA"~215192.00~"AKEBIA THERAPEUTICS INC"~"Vafseo (Vadadustat)"~3/27/2024 0:00:00~"Original"~1~"4613-2"~"PMR"~"505 (o)(3)"~4613.00~2~"PMR 4613-2: Conduct an observational study to characterize the long-term safety (up to 5 years follow up) of Vafseo in adults with dialysis-dependent chronic kidney disease treated with the approved dosing regimen of Vafseo in the United States. Specific safety outcomes of interest include: thrombotic vascular events including vascular access thrombosis; hospitalization for heart failure; and serious gastrointestinal bleeds. The study population should include adults previously treated with erythropoiesis-stimulating agents (ESAs) and adults na ve to ESAs. The effect of baseline and maximum achieved hemoglobin on the specified safety outcomes should be evaluated."~"Pending"~~9/30/2033 0:00:00~5/19/2026 0:00:00
384287~"CDER"~"NDA"~215192.00~"AKEBIA THERAPEUTICS INC"~"Vafseo (Vadadustat)"~3/27/2024 0:00:00~"Original"~1~"4613-3"~"PMR"~"505 (o)(3)"~4613.00~3~"PMR 4613-3: Conduct an observational study (up to 5 years follow up) to assess the risk for malignancy (hematological and non-hematological) in adults with dialysis-dependent chronic kidney disease with anemia treated with Vafseo versus an erythropoiesis-stimulating agent comparator arm. The study should include an assessment of primary malignancies among adults with no cancer history (including assessment by type and location), and the impact of Vafseo on progression-free survival, and overall survival in adults with prior cancers."~"Pending"~~2/28/2033 0:00:00~5/19/2026 0:00:00
384288~"CDER"~"NDA"~215192.00~"AKEBIA THERAPEUTICS INC"~"Vafseo (Vadadustat)"~3/27/2024 0:00:00~"Original"~1~"4613-4"~"PMR"~"505 (o)(3)"~4613.00~4~"PMR 4613-4: Conduct a worldwide descriptive study to collect prospective and retrospective data on women exposed to Vafseo during pregnancy to assess the risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Assess infant outcomes through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Pending"~~1/31/2032 0:00:00~5/19/2026 0:00:00
384289~"CDER"~"NDA"~216490.00~"ASCENDIS PHARMA BONE DISEASES AS"~"Yorvipath (palopegteriparatide)"~8/9/2024 0:00:00~"Original"~1~"4677-1"~"PMR"~"505 (o)(3)"~4677.00~1~"PMR 4677-1: Perform a lactation study (milk only) in lactating women who have received therapeutic doses of palopegteriparatide using a validated assay to assess concentrations of palopegteriparatide in breast milk and the effects on the breastfed infant."~"Pending"~~8/31/2028 0:00:00~10/8/2025 0:00:00
384290~"CDER"~"NDA"~216490.00~"ASCENDIS PHARMA BONE DISEASES AS"~"Yorvipath (palopegteriparatide)"~8/9/2024 0:00:00~"Original"~1~"4677-2"~"PMR"~"505 (o)(3)"~4677.00~2~"PMR 4677-2: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to palopegteriparatide during pregnancy to assess risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant. Assess infant outcomes through at least the first year of life. The study should also collect adverse event data for lactating women and infants exposed to palopegteriparatide through breastfeeding to assess for any potential risks to the infant from breastfeeding. The minimum number of patients will be specified in the protocol."~"Pending"~~8/31/2036 0:00:00~10/8/2025 0:00:00
384291~"CDER"~"NDA"~216513.00~"MEDUNIK CANADA INC"~"Pheburane (sodium phenylbutyrate)"~6/17/2022 0:00:00~"Original"~1~"4293-1"~"PMR"~"505 (o)(3)"~4293.00~1~"PMR 4293-1: Conduct in vitro studies to evaluate whether sodium phenylbutyrate and phenylacetate are substrates, inhibitors, or inducers of metabolizing enzymes and transporters as outlined in the FDA guidance for industry In Vitro Drug Interaction Studies - Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions (January 2020). If in vitro studies suggest a potential for drug interaction, additional in vivo studies may be required."~"Fulfilled"~~4/30/2024 0:00:00~8/16/2024 0:00:00
384292~"CDER"~"NDA"~216540.00~"BRIDGEBIO PHARMA INC"~"Attruby (acoramidis)"~11/22/2024 0:00:00~"Original"~1~"4726-1"~"PMR"~"505 (o)(3)"~4726.00~1~"PMR 4726-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to acoramidis during pregnancy or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Pending"~~5/31/2035 0:00:00~1/21/2026 0:00:00
384293~"CDER"~"NDA"~216540.00~"BRIDGEBIO PHARMA INC"~"Attruby (acoramidis)"~11/22/2024 0:00:00~"Original"~1~"4726-2"~"PMR"~"505 (o)(3)"~4726.00~2~"PMR 4726-2: Conduct a clinical pharmacokinetic study to evaluate the effect of mild, moderate and severe hepatic impairment on the pharmacokinetics of acoramidis. Transthyretin (TTR) concentration should be measured in all subjects."~"Ongoing"~~8/31/2026 0:00:00~1/21/2026 0:00:00
384294~"CDER"~"NDA"~216540.00~"BRIDGEBIO PHARMA INC"~"Attruby (acoramidis)"~11/22/2024 0:00:00~"Original"~1~"4726-3"~"PMR"~"505 (o)(3)"~4726.00~3~"PMR 4726-3: Conduct a clinical pharmacokinetic study to evaluate the effect of repeated doses of acoramidis on the pharmacokinetics of a CYP2C9 substrate."~"Delayed"~"All subjects have been enrolled. The final report submission milestone was missed. Applicant expects submission in the 2nd quarter of 2026."~1/31/2026 0:00:00~1/21/2026 0:00:00
384295~"CDER"~"NDA"~216540.00~"BRIDGEBIO PHARMA INC"~"Attruby (acoramidis)"~11/22/2024 0:00:00~"Original"~1~"4726-4"~"PMR"~"505 (o)(3)"~4726.00~4~"PMR 4726-4: Conduct a clinical pharmacokinetic study to evaluate the effect of repeated doses of a UGT inducer (e.g., carbamazepine or phenytoin) on the pharmacokinetics of acoramidis"~"Delayed"~"All subjects have been enrolled. The final report submission milestone was missed. Applicant expects submission in the 2nd quarter of 2026."~1/31/2026 0:00:00~1/21/2026 0:00:00
384296~"CDER"~"NDA"~216579.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Symbicort Aerosphere (budesonide and formeterol fumarate)"~4/28/2023 0:00:00~"Supplement"~1~"4981-1"~"PMR"~"Pediatric Research Equity Act"~4981.00~1~"PMR 4981-1: Conduct a randomized, open-label, single-dose, crossover study to compare the pharmacokinetics of budesonide and formoterol delivered with Symbicort Aerosphere and Symbicort pMDI in children 4 to less than 12 years of age with asthma."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~7/31/2027 0:00:00~
384297~"CDER"~"NDA"~216579.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Symbicort Aerosphere (budesonide and formeterol fumarate)"~4/28/2023 0:00:00~"Supplement"~1~"4981-2"~"PMR"~"Pediatric Research Equity Act"~4981.00~2~"PMR 4981-2: Conduct a phase 3, randomized, active-controlled, double-blind, parallel group, 6-week efficacy and safety lung function study of Symbicort Aerosphere compared to budesonide MDI in children 4 to less than 12 years of age with asthma."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~2/28/2030 0:00:00~
384298~"CDER"~"NDA"~216632.00~"BAUSCH HEALTH US LLC"~"Cabtreo (clindamycin phosphate, benzoyl peroxide, and adapalene)"~10/20/2023 0:00:00~"Original"~1~"4500-1"~"PMR"~"Pediatric Research Equity Act"~4500.00~1~"PMR 4500-1: Conduct an open-label study to assess safety, pharmacokinetics, and treatment effect of Cabtreo (clindamycin phosphate, adapalene, and
benzoyl peroxide) topical gel, 1.2%/0.15%/3.1% in 100 pediatric subjects ages 9 to 11 years 11 months with acne vulgaris."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed"~12/31/2027 0:00:00~12/19/2025 0:00:00
384299~"CDER"~"NDA"~216660.00~"AMYLYX PHARMACEUTICALS INC"~"Relyvrio (sodium phenylbutyrate and taurursodiol)"~9/29/2022 0:00:00~"Original"~1~"4346-1"~"PMR"~"505 (o)(3)"~4346.00~1~"PMR 4346-1: A carcinogenicity study of AMX0035 (sodium phenylbutyrate and taurursodiol) in rat."~"Released"~~6/30/2026 0:00:00~11/26/2024 0:00:00
384300~"CDER"~"NDA"~216660.00~"AMYLYX PHARMACEUTICALS INC"~"Relyvrio (sodium phenylbutyrate and taurursodiol)"~9/29/2022 0:00:00~"Original"~1~"4346-2"~"PMR"~"505 (o)(3)"~4346.00~2~"PMR 4346-2: A carcinogenicity study of AMX0035 (sodium phenylbutyrate and taurursodiol) in mouse."~"Released"~~6/30/2026 0:00:00~11/26/2024 0:00:00
384301~"CDER"~"NDA"~216660.00~"AMYLYX PHARMACEUTICALS INC"~"Relyvrio (sodium phenylbutyrate and taurursodiol)"~9/29/2022 0:00:00~"Original"~1~"4346-3"~"PMR"~"505 (o)(3)"~4346.00~3~"PMR 4346-3: Conduct an in vivo pharmacokinetic drug interaction study to evaluate the effect of Relyvrio on inhibiting and/or inducing CYP2C8, CYP1A2, CYP2B6, and CYP3A4 enzymes using an appropriate probe substrate for each enzyme. We recommend you evaluate these drug interactions as a single cocktail Drug Drug Interaction (DDI) study. Please refer to the Guidance for Industry Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interaction (https://www.fda.gov/media/134581/download)."~"Released"~~6/30/2024 0:00:00~11/26/2024 0:00:00
384302~"CDER"~"NDA"~216483.00~"ALEMBIC THERAPEUTICS LLC"~"Pivya (pivmecillinam)"~4/24/2024 0:00:00~"Original"~1~"4603-8"~"PMR"~"Pediatric Research Equity Act"~4603.00~8~"PMR 4603-8: Conduct a multicenter, randomized, single-blind study to evaluate the safety and tolerability of pivmecillinam hydrochloride versus an
appropriate comparator for the treatment of pediatric subjects, 2 years to less than 12 years of age, with uncomplicated urinary tract infections (uUTI)."~"Pending"~"The study has not been initiated."~1/31/2031 0:00:00~6/23/2026 0:00:00
384303~"CDER"~"NDA"~216660.00~"AMYLYX PHARMACEUTICALS INC"~"Relyvrio (sodium phenylbutyrate and taurursodiol)"~9/29/2022 0:00:00~"Original"~1~"4346-4"~"PMR"~"505 (o)(3)"~4346.00~4~"PMR 4346-4: Conduct an in vivo drug interaction study to evaluate the effect of OATP1B3 transporter inhibitor on the pharmacokinetics of Relyvrio. Please refer to the Guidance for Industry Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug
Interactions https://www.fda.gov/media/134581/download)"~"Released"~~6/30/2024 0:00:00~11/26/2024 0:00:00
384304~"CDER"~"NDA"~211801.00~"ARDELYX INC"~"Ibsrela (tenapanor)"~9/12/2019 0:00:00~"Original"~1~"3583-2"~"PMR"~"Pediatric Research Equity Act"~3583.00~2~"PMR 3583-2: A randomized, double-blind, controlled, dose-ranging study to assess the safety and efficacy of tenapanor for the treatment of irritable bowel syndrome with constipation (IBS-C) in pediatric patients 6 to less than 12 years of age. The study will include at least 2 doses and treatment duration will be 4 weeks."~"Delayed"~"Original Final Report Due Date: 02/28/2023; Deferral Extension granted per FDA letter dated 06/21/2023."~9/30/2026 0:00:00~11/4/2025 0:00:00
384305~"CDER"~"NDA"~211801.00~"ARDELYX INC"~"Ibsrela (tenapanor)"~9/12/2019 0:00:00~"Original"~1~"3583-3"~"PMR"~"Pediatric Research Equity Act"~3583.00~3~"PMR 3583-3: A 12-week, randomized, controlled study to assess the safety and efficacy of tenapanor for the treatment of irritable bowel syndrome with constipation (IBS-C) in pediatric patients 12 to less than 18 years of age. The study will evaluate at least 2 doses."~"Delayed"~"Original final report submission milestone was missed. Original Final Report Due Date: 02/28/2023. Per FDA letter dated 06/21/2023, final report due date extended to 06/30/2026. Second Deferral Extension Granted per FDA letter dated 04/09/2026."~11/30/2026 0:00:00~11/4/2025 0:00:00
384306~"CDER"~"NDA"~211801.00~"ARDELYX INC"~"Ibsrela (tenapanor)"~9/12/2019 0:00:00~"Original"~1~"3583-4"~"PMR"~"Pediatric Research Equity Act"~3583.00~4~"PMR 3583-4: A randomized, controlled study to assess the safety and efficacy of tenapanor for the treatment of irritable bowel syndrome with constipation (IBS-C) in pediatric patients 6 to less than 12 years of age."~"Delayed"~"The study completion milestone was missed. Original Final Report Due Date: 02/28/2026; Deferral Extension granted per FDA letter dated 04/09/2026."~3/31/2029 0:00:00~11/4/2025 0:00:00
384307~"CDER"~"NDA"~211801.00~"ARDELYX INC"~"Ibsrela (tenapanor)"~9/12/2019 0:00:00~"Original"~1~"3583-5"~"PMR"~"Pediatric Research Equity Act"~3583.00~5~"PMR 3583-5: An open-label, long-term extension study to assess the safety of ongoing treatment with tenapanor for irritable bowel syndrome with constipation (IBS-C) in pediatric patients 6 to less than 18 years of age, who participated in study 3583-2, 3583-3, or 3583-4."~"Delayed"~"The final protocol submission milestone was missed and Deferral Extension was denied on 3/3/2021."~5/31/2027 0:00:00~11/4/2025 0:00:00
384308~"CDER"~"NDA"~211810.00~"DAIICHI SANKYO INC"~"Turalio (pexidartinib)"~8/2/2019 0:00:00~"Original"~1~"3673-1"~"PMR"~"505 (o)(3)"~3673.00~1~"PMR 3673-1: Conduct a long-term trial to further evaluate the risk of hepatoxicity in adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery, who are receiving pexidartinib. The trial will include laboratory, imaging, and pathologic assessments of patients who experience liver toxicity due to exposure to pexidartinib. The trial should enroll patients with an AST or ALT > 3 x ULN with concomitant bilirubin >2 x ULN, an isolated bilirubin > 2 x ULN (excluding those with Gilbert s syndrome), or an isolated AST or ALT > 10 x ULN. The trial should evaluate the mechanism of action of liver injury based on liver biopsy information, including a detailed assessment of changes in resident macrophage phenotype, based on marker status, as well as detailed characterization of other immune cell infiltrates. Submit cumulative, integrated safety analyses after 5 and 10 years of follow-up from an adequate number of patients to characterize the long-term risk of hepatic failure with pexidartinib. These safety evaluations should be adequate to inform labeling of patient populations at highest risk and to provide evidence-based dose modifications and monitoring recommendations."~"Pending"~~6/30/2036 0:00:00~9/26/2025 0:00:00
384309~"CDER"~"NDA"~211844.00~"INFORLIFE SA"~"Midazolam "~3/22/2021 0:00:00~"Original"~1~"4039-1"~"PMR"~"505 (o)(3)"~4039.00~1~"PMR 4039-1: Conduct a leachable study testing at least three batches of drug product that includes data from early, middle, and late timepoints on stability to fully inform the trend analysis. Identify any leachable present above [...] mcg/day based on your trend analysis. Submit a revised toxicological risk assessment for all leachables greater than [...] mcg/day taking into consideration the maximum daily dose of your drug product."~"Submitted"~~12/31/2023 0:00:00~4/28/2026 0:00:00
384310~"CDER"~"NDA"~211855.00~"BIOGEN INC"~"VUMERITY (diroximel fumarate)"~10/29/2019 0:00:00~"Original"~1~"3742-3"~"PMR"~"505 (o)(3)"~3742.00~3~"PMR 3742-3: Conduct prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with multiple sclerosis exposed to diroximel fumarate during pregnancy with two unexposed comparator populations: an internal comparator consisting of women with multiple sclerosis who have not been exposed to diroximel fumarate before or during pregnancy, and the other consisting of women without multiple sclerosis. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small for gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~2/28/2033 0:00:00~5/22/2026 0:00:00
384311~"CDER"~"NDA"~211855.00~"BIOGEN INC"~"VUMERITY (diroximel fumarate)"~10/29/2019 0:00:00~"Original"~1~"3742-4"~"PMR"~"505 (o)(3)"~3742.00~4~"PMR 3742-4: Conduct a pregnancy outcomes study using a different study design than provided for in PMR 3742-3 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small-for-gestational-age births in women exposed to diroximel fumarate during pregnancy compared to an unexposed control population."~"Ongoing"~~2/28/2033 0:00:00~5/22/2026 0:00:00
384312~"CDER"~"NDA"~211855.00~"BIOGEN INC"~"VUMERITY (diroximel fumarate)"~10/29/2019 0:00:00~"Original"~1~"3742-5"~"PMR"~"Pediatric Research Equity Act"~3742.00~5~"PMR 3742-5: Conduct a two-part study of Vumerity (diroximel fumarate) in pediatric patients with relapsing forms of multiple sclerosis (RMS) at least 10 years and less than 18 years of age. Part A is an open-label study for evaluating safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Vumerity (diroximel fumarate) in pediatric patients weighing 40 kg or less. The objectives of Part A are to determine titration and maintenance doses of Vumerity (diroximel fumarate) that will result in PK and PD effects that are comparable to those of the maintenance doses administered to adult patients, and to assess the safety of Vumerity. Part B is a randomized, double-blind, parallel-group study in pediatric patients weighing greater than 40 kg to evaluate the efficacy and safety of Vumerity (diroximel fumarate) compared to an appropriate control."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 1/21/2025."~2/28/2029 0:00:00~5/22/2026 0:00:00
384313~"CDER"~"NDA"~203094.00~"GILEAD SCIENCES INC"~"Tybost (Cobicistat)  "~9/24/2014 0:00:00~"Original"~1~"3533-1"~"PMR"~"Pediatric Research Equity Act"~3533.00~1~"PMR 3533-1: Conduct a study to evaluate the pharmacokinetics (PK), safety and antiviral activity of DESCOVY (emtricitabine and tenofovir alafenamide) administered in combination with atazanavir and TYBOST (cobicistat), and in combination with darunavir and TYBOST in HIV-1 infected pediatric subjects weighing less than 25 kg. The safety and activity of the treatment regimen must be assessed for a minimum of 24 weeks. The minimum age criteria for the treatment regimen being evaluated are specified below. DESCOVY administered in combination with atazanavir co-administered with TYBOST must be evaluated in pediatric patients 3 months of age and older. DESCOVY administered in combination with darunavir co-administered with TYBOST must be evaluated in pediatric patients 3 years of age and older."~"Released"~"Per FDA letter dated 07/25/2025, this PMR/PMC has been released."~6/30/2026 0:00:00~11/14/2025 0:00:00
384314~"CDER"~"NDA"~203094.00~"GILEAD SCIENCES INC"~"Tybost (Cobicistat)  "~9/24/2014 0:00:00~"Original"~1~"3533-4"~"PMC"~"506B PMC"~3533.00~4~"PMC 3533-4: Conduct a trial to evaluate the pharmacokinetics (PK), safety and antiviral activity of DESCOVY (emtricitabine and tenofovir alafenamide) administered in combination with atazanavir and TYBOST (cobicistat) in pediatric patients with HIV-1 infection who are 3 months of age and older and weighing less than 14 kg. The safety and activity of the treatment regimen must be assessed for a minimum of 24 weeks."~"Released"~~6/30/2026 0:00:00~11/14/2025 0:00:00
384315~"CDER"~"NDA"~203202.00~"LUNDBECK NA LTD"~"Northera (Droxidopa)"~2/18/2014 0:00:00~"Original"~1~"2129-2"~"PMR"~"Accelerated Approval"~2129.00~2~"PMR 2129-2: A clinical trial of patients with symptomatic neurogenic orthostatic hypotension to assess  sustained effects of droxidopa therapy. The trial design consists of a 3-month, open-label droxidopa treatment period, followed by a 3-month, randomized, double-blind, placebocontrolled, withdrawal period. The primary endpoint will be time to treatment intervention."~"Submitted"~"The final report was submitted on 11/16/2023."~8/31/2021 0:00:00~4/15/2026 0:00:00
384316~"CDER"~"NDA"~203214.00~"PF PRISM CV"~"Xeljanz (Tofacitinib)"~11/6/2012 0:00:00~"Original"~1~"1934-3"~"PMR"~"505 (o)(3)"~1934.00~3~"PMR 1934-3: Controlled clinical trial to evaluate the long term safety of tofacitinib in patients with rheumatoid arthritis. The trial should include two doses of tofacitinib and an active comparator. The trial should be of sufficient size and duration to evaluate safety events of interest, including cardiovascular adverse events, opportunistic infections, and malignancy."~"Submitted"~~6/30/2020 0:00:00~12/23/2025 0:00:00
384317~"CDER"~"NDA"~203214.00~"PF PRISM CV"~"Xeljanz (Tofacitinib)"~11/6/2012 0:00:00~"Supplement"~18~"3400-1"~"PMR"~"Pediatric Research Equity Act"~3400.00~1~"PMR 3400-1: A one-year, multi-center, randomized, controlled trial to evaluate the safety, efficacy and pharmacokinetics of XELJANZ (tofacitinib) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis."~"Delayed"~"A deferral extension was granted per FDA letter dated September 5, 2024. The study completion milestone was also revised."~1/31/2027 0:00:00~12/23/2025 0:00:00
384318~"CDER"~"NDA"~203214.00~"PF PRISM CV"~"Xeljanz (Tofacitinib)"~11/6/2012 0:00:00~"Supplement"~18~"3400-2"~"PMR"~"Pediatric Research Equity Act"~3400.00~2~"PMR 3400-2: A multi-center open-label extension study to evaluate the long-term safety of XELJANZ (tofacitinib) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis who participated in PMR 3400-1."~"Delayed"~"The final protocol milestone date was missed. FDA granted a
deferral extension on September 5, 2024."~7/31/2029 0:00:00~12/23/2025 0:00:00
384319~"CDER"~"NDA"~203214.00~"PF PRISM CV"~"Xeljanz (Tofacitinib)"~11/6/2012 0:00:00~"Supplement"~18~"3400-6"~"PMC"~"506B PMC"~3400.00~6~"PMC 3400-6: A noninterventional, observational cohort study to evaluate the effectiveness and safety of the recombinant zoster vaccine (RZV,  Shingrix vaccine ) in adult patients with moderately to severely active ulcerative colitis treated with XELJANZ (tofacitinib). "~"Fulfilled"~~2/28/2024 0:00:00~12/23/2025 0:00:00
384320~"CDER"~"NDA"~203214.00~"PF PRISM CV"~"Xeljanz (Tofacitinib)"~11/6/2012 0:00:00~"Supplement"~26~"3944-1"~"PMR"~"505 (o)(3)"~3944.00~1~"PMR 3944-1: Conduct a long-term observational safety study in pediatric patients 2-17 years of age with polyarticular-course JIA (pcJIA) treated with tofacitinib to evaluate for the risk of malignancies, serious infections (including opportunistic infections), thrombosis, and effects on growth. The study should include a control group of pediatric pcJIA patients treated with other pcJIA medications as standard of care. Patients should be followed for 5 years."~"Delayed"~"The final protocol milestone date was missed. The final protocol was acknowledged by the FDA on 2/1/2023."~9/30/2030 0:00:00~12/23/2025 0:00:00
384321~"CDER"~"NDA"~203284.00~"HORIZON THERAPEUTICS US HOLDING LLC"~"Ravicti (Glycerol Phenylbutyrate)"~2/1/2013 0:00:00~"Original"~1~"2013-4"~"PMR"~"505 (o)(3)"~2013.00~4~"PMR 2013-4: A randomized, controlled clinical trial to assess the safety and efficacy of Ravicti  (glycerol phenylbutyrate) in patients with Urea Cycle Disorders who are treatment na ve to phenylbutyrate."~"Fulfilled"~~3/31/2017 0:00:00~4/1/2025 0:00:00
384322~"CDER"~"NDA"~202811.00~"ABBVIE INC"~"Linzess (linaclotide)"~8/30/2012 0:00:00~"Original"~1~"1915-3"~"PMR"~"Pediatric Research Equity Act"~1915.00~3~"PMR 1915-3: A safety and efficacy study in pediatric patients with irritable bowel syndrome with constipation ages seven years up to 17 years treated with Linzess (linaclotide)."~"Fulfilled"~"Per FDA letter dated 11/04/2025, this PMR/PMC has been fulfilled. 
"~9/30/2026 0:00:00~10/23/2025 0:00:00
384323~"CDER"~"NDA"~202834.00~"CATALYST PHARMACEUTICALS INC"~"Fycompa (Perampanel)"~10/22/2012 0:00:00~"Supplement"~14~"3496-1"~"PMR"~"Pediatric Research Equity Act"~3496.00~1~"PMR 3496-1: Deferred pediatric study under PREA: A prospective, randomized, controlled, double-blind, efficacy and safety study of FYCOMPA (perampanel) for the adjunctive treatment of partial onset seizures in children ages 1 month to < 2 years with a long term safety extension. The primary efficacy endpoint during the controlled phase will examine seizure frequency based upon Video/EEG data. Safety will be evaluated during the controlled phase and long term extension. In the long term extension component, a minimum of 25 patients must be exposed to perampanel for 6 months at or above the dose or doses identified as effective."~"Submitted"~"The final report was submitted to FDA on 01/30/2026."~12/31/2024 0:00:00~12/15/2025 0:00:00
384324~"CDER"~"NDA"~202992.00~"SANOFI AVENTIS US LLC"~"Aubagio (Teriflunomide)"~9/12/2012 0:00:00~"Original"~1~"1924-5"~"PMR"~"505 (o)(3)"~1924.00~5~"PMR 1924-5: Conduct a worldwide, descriptive study that collects prospective and retrospective data in women exposed to teriflunomide during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Delayed"~"Per the applicant s Annual Status Report, enrollment has initiated, and the original interim report milestone was missed to allow additional time to collect data for the reporting period and align with the PBRER submission. The interim report was submitted on 12/11/2023."~9/30/2031 0:00:00~11/7/2025 0:00:00
384325~"CDER"~"NDA"~208088.00~"VERITY PHARMACEUTICALS INC"~"TLANDO (Testosterone Undecanoate)"~3/28/2022 0:00:00~"Original"~1~"4256-3"~"PMR"~"505 (o)(3)"~4256.00~3~"PMR 4256-3: Conduct in vitro studies to assess the potential of Tlando to inhibit cytochrome P450 (CYP) 3A, P-glycoprotein (P-gp), and breast cancer resistance protein (BCRP). If the in vitro studies suggest a potential or a clinically relevant DDI, that cannot be discounted, a clinical drug interaction study(ies) may be required to assess the potential of Tlando to inhibit CYP3A, P-gp, and BCRP."~"Delayed"~"Draft and Final Protocol Submission milestone dates were missed due to continued discussions with the Agency. The applicant requested revised milestones because the proposed highest concentration of 5-7  M was not high enough to evaluate the potential of Tlando to inhibit intestinal enzymes/transporters. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 09/12/2024."~1/31/2024 0:00:00~6/8/2026 0:00:00
384326~"CDER"~"NDA"~208090.00~"COLLEGIUM PHARMACEUTICAL INC"~"Xtampza ER (Oxycodone)"~4/26/2016 0:00:00~"Original"~1~"3033-1"~"PMR"~"505 (o)(3)"~3033.00~1~"PMR 3033-1: A prospective, observational study designed to quantify the serious risks of misuse, abuse, and addiction associated with long-term use of opioid analgesics  for management of chronic pain among patients prescribed ER/LA opioid analgesics. This study must address at a minimum the following specific objectives: a. Estimate the incidence of misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of  psychiatric illness) on the risk of misuse, abuse, and addiction.  b. Evaluate and quantify other risk factors for misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors, psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships."~"Fulfilled"~~3/31/2020 0:00:00~6/24/2026 0:00:00
384327~"CDER"~"NDA"~208090.00~"COLLEGIUM PHARMACEUTICAL INC"~"Xtampza ER (Oxycodone)"~4/26/2016 0:00:00~"Original"~1~"3033-2"~"PMR"~"505 (o)(3)"~3033.00~2~"PMR 3033-2: An observational study designed to measure the incidence and predictors of opioid overdose and death (OOD), as well as opioid abuse/addiction, using patient  health records, insurance claims, and death records. This study must address at a minimum the following specific objectives: a. Estimate the incidence of abuse/addiction, overdose, and death associated with long-term use of opioid analgesics for chronic pain. Stratify overdose by  intentionality wherever possible. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other  clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of psychiatric illness) on the risk of  abuse/addiction, overdose, and death. b. Evaluate and quantify other risk factors for abuse/addiction, overdose, and  death associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors,  psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships. Stratify overdose by intentionality wherever possible."~"Fulfilled"~~9/30/2019 0:00:00~6/24/2026 0:00:00
384328~"CDER"~"NDA"~208090.00~"COLLEGIUM PHARMACEUTICAL INC"~"Xtampza ER (Oxycodone)"~4/26/2016 0:00:00~"Original"~1~"3033-6"~"PMR"~"505 (o)(3)"~3033.00~6~"PMR 3033-6: An observational study to develop and validate an algorithm using coded medical terminologies and other electronic healthcare data to identify opioid-related overdose and death."~"Fulfilled"~~12/31/2016 0:00:00~6/24/2026 0:00:00
384329~"CDER"~"NDA"~208090.00~"COLLEGIUM PHARMACEUTICAL INC"~"Xtampza ER (Oxycodone)"~4/26/2016 0:00:00~"Original"~1~"3033-11"~"PMR"~"505 (o)(3)"~3033.00~11~"PMR 3033-11: Conduct a clinical trial to estimate the serious risk for the development of hyperalgesia following the long-term use of high-dose ER/LA opioid analgesics  for at least one year to treat chronic pain. Include an assessment of risk relative to  efficacy."~"Delayed"~"The Trial Completion and Final Report milestones were missed because of continued discussions needed to reach agreement with the Agency on the Final Protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 8/26/2025."~8/31/2019 0:00:00~6/24/2026 0:00:00
384330~"CDER"~"NDA"~208090.00~"COLLEGIUM PHARMACEUTICAL INC"~"Xtampza ER (Oxycodone)"~4/26/2016 0:00:00~"Original"~1~"2966-11"~"PMR"~"505 (o)(3)"~2966.00~11~"PMR 2966-11: Conduct a carcinogenicity assessment in the rat model testing a mixture of beeswax,  carnauba wax, and myristic acid that is representative of the drug product composition."~"Submitted"~~3/31/2022 0:00:00~6/24/2026 0:00:00
384331~"CDER"~"NDA"~208090.00~"COLLEGIUM PHARMACEUTICAL INC"~"Xtampza ER (Oxycodone)"~4/26/2016 0:00:00~"Original"~1~"2966-12"~"PMR"~"505 (o)(3)"~2966.00~12~"PMR 2966-12: Conduct a carcinogenicity assessment in the mouse model testing the mixture of beeswax, carnauba wax, and myristic acid that is representative of the drug product composition."~"Submitted"~~4/30/2022 0:00:00~6/24/2026 0:00:00
384332~"CDER"~"NDA"~208147.00~"TRIS PHARMA INC"~"Dyanavel XR (Amphetamine)"~10/19/2015 0:00:00~"Original"~1~"2970-2"~"PMR"~"Pediatric Research Equity Act"~2970.00~2~"PMR 2970-2: A randomized, double-blind, placebo-controlled, flexible-dose titration study of Dyanavel XR (amphetamine extended-release) oral suspension in children ages 4  to 5 years diagnosed with ADHD."~"Released"~"Per FDA letter dated 11/18/2025, this PMR/PMC has been released."~2/28/2020 0:00:00~12/18/2024 0:00:00
384333~"CDER"~"NDA"~208147.00~"TRIS PHARMA INC"~"Dyanavel XR (Amphetamine)"~10/19/2015 0:00:00~"Original"~1~"2970-3"~"PMR"~"Pediatric Research Equity Act"~2970.00~3~"PMR 2970-3: A one year Pediatric Open-Label Safety Study of patients age 4 to 5 years (at the time of entry into PMR 2970-1 or PMR 2970-2, or at the time of enrollment if directly enrolled into PMR 2970-3) diagnosed with ADHD treated with Dyanavel XR (amphetamine extended release) oral suspension."~"Released"~"Per FDA letter dated 11/18/2025, this PMR/PMC has been released."~7/31/2020 0:00:00~12/18/2024 0:00:00
384334~"CDER"~"NDA"~208171.00~"PHARMACOSMOS AS"~"Monoferric (Ferric Derisomaltose)"~1/16/2020 0:00:00~"Original"~1~"3759-1"~"PMR"~"Pediatric Research Equity Act"~3759.00~1~"PMR 3759-1: Conduct a study to assess the efficacy and safety study of intravenous ferric derisomaltose in infants and children 0 to less than 18 years of age with iron deficiency anemia due to non-dialysis dependent chronic kidney disease or iron deficiency anemia intolerant to or unresponsive to oral iron (Study P-Monofer-PED-01). Submit datasets at the time of final clinical study report submission."~"Delayed"~"The final report milestone was missed because of delays involving study participants, sites, and/or management. Original Final Report Due Date: 12/31/2022; Deferral Extension granted 6/9/2022 and 4/26/2024. The study completion milestone was also revised. Deferral Extension Requested 01/16/2026. Denied per FDA letter dated 03/03/2026."~10/31/2026 0:00:00~3/16/2026 0:00:00
384335~"CDER"~"NDA"~21164.00~"FABRE KRAMER PHARMACEUTICALS INC"~"Exxua (gepirone)"~9/22/2023 0:00:00~"Original"~1~"4485-5"~"PMC"~"506B PMC"~4485.00~5~"PMC 4485-5: Conduct a controlled trial to evaluate the longer-term (i.e., maintenance) efficacy of gepirone ER in the treatment of adults with major depressive disorder (MDD). The population should include significant U.S. representation and include underrepresented racial and ethnic minorities. This trial must include a placebo group and must utilize a double-blind, randomized-withdrawal design following an adequate period of stabilization with open-label treatment of gepirone ER. This trial design must incorporate long-term safety assessments (including pre-, post-, and on-treatment electrocardiograms). Because the short-term trials used to support efficacy were not fixed-dose, it is important to establish the dose-response for maintenance. Therefore, following open-label stabilization, this trial should randomize subjects to fixed doses of the to-be-marketed doses of gepirone ER (and placebo) during the maintenance phase."~"Delayed"~"The final protocol due date has passed with no final protocol received."~1/31/2030 0:00:00~11/7/2025 0:00:00
384336~"CDER"~"NDA"~21277.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Avelox IV (Moxifloxacin Hydrochloride)"~11/30/2001 0:00:00~"Supplement"~56~"2898-1"~"PMR"~"Animal Efficacy"~2898.00~1~"PMR 2898-1: Conduct a field study to evaluate the efficacy and safety of moxifloxacin hydrochloride in the event of an attack with the intentional release of Y. pestis in  the United States."~"Pending"~~~1/29/2026 0:00:00
384337~"CDER"~"NDA"~21285.00~"NOVARTIS PHARMACEUTICALS CORP"~"Trileptal (Oxcarbazepine)"~5/25/2001 0:00:00~"Original"~1~"4096-1"~"PMR"~"505 (o)(3)"~4096.00~1~"PMR 4096-1: Conduct in vitro patch clamp studies to assess Trileptal s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Trileptal s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~7/18/2025 0:00:00
384338~"CDER"~"NDA"~21302.00~"BAUSCH HEALTH US LLC"~"Elidel (Pimecrolimus)"~12/13/2001 0:00:00~"Original"~1~"141-1"~"PMC"~"506B PMC"~141.00~1~"PMC 141-1: The preclinical rodent studies found an increased risk for lymphoma and follicular cell thyroid adenoma in the studies evaluating an oral formulation of pimecrolimus. We agree to conduct a registry study of pediatric patients (aged 2-17, with emphasis on the younger ages) with atopic dermatitis followed through adulthood for those who have long-term intermittent treatment with Elidel (pimecrolimus) 1% Cream to assess the risk of developing systemic malignancies. Study proposal for review: April 30, 2002. Report: Every 5 years after the division reviews and accepts the proposal and the study is initiated. Data will be submitted with NDA annual report.  b. A preclinical mouse photocarcinogenicity study showed an accelerated rate of development of cutaneous malignancies. We agree to conduct a registry or case-controlled study of sun-exposed adult patients, aged 40 and above, with atopic dermatitis, who have long-term intermittent treatment with Elidel (pimecrolimus) 1%Cream to assess the risk of developing cutaneous malignancies. Study proposal for review: June 30, 2002. Report: Every 5 years after the division reviews and accepts the proposal and the study is initiated. Data will be submitted with NDA annual report."~"Ongoing"~~12/31/2026 0:00:00~2/11/2026 0:00:00
384339~"CDER"~"NDA"~21306.00~"KNOA PHARMA LLC"~"Butrans (Buprenorphine)"~6/30/2010 0:00:00~"Original"~1~"3033-1"~"PMR"~"505 (o)(3)"~3033.00~1~"PMR 3033-1: A prospective, observational study designed to quantify the serious risks of misuse, abuse, and addiction associated with long-term use of opioid analgesics  for management of chronic pain among patients prescribed ER/LA opioid analgesics. This study must address at a minimum the following specific objectives: a. Estimate the incidence of misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of  psychiatric illness) on the risk of misuse, abuse, and addiction.  b. Evaluate and quantify other risk factors for misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors, psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships."~"Fulfilled"~~3/31/2020 0:00:00~8/27/2018 0:00:00
384340~"CDER"~"NDA"~21306.00~"KNOA PHARMA LLC"~"Butrans (Buprenorphine)"~6/30/2010 0:00:00~"Original"~1~"3033-2"~"PMR"~"505 (o)(3)"~3033.00~2~"PMR 3033-2: An observational study designed to measure the incidence and predictors of opioid overdose and death (OOD), as well as opioid abuse/addiction, using patient  health records, insurance claims, and death records. This study must address at a minimum the following specific objectives: a. Estimate the incidence of abuse/addiction, overdose, and death associated with long-term use of opioid analgesics for chronic pain. Stratify overdose by  intentionality wherever possible. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other  clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of psychiatric illness) on the risk of  abuse/addiction, overdose, and death. b. Evaluate and quantify other risk factors for abuse/addiction, overdose, and  death associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors,  psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships. Stratify overdose by intentionality wherever possible."~"Fulfilled"~~9/30/2019 0:00:00~8/27/2018 0:00:00
384341~"CDER"~"NDA"~21306.00~"KNOA PHARMA LLC"~"Butrans (Buprenorphine)"~6/30/2010 0:00:00~"Original"~1~"3033-6"~"PMR"~"505 (o)(3)"~3033.00~6~"PMR 3033-6: An observational study to develop and validate an algorithm using coded medical terminologies and other electronic healthcare data to identify opioid-related overdose and death."~"Fulfilled"~~12/31/2016 0:00:00~8/27/2018 0:00:00
384342~"CDER"~"NDA"~21306.00~"KNOA PHARMA LLC"~"Butrans (Buprenorphine)"~6/30/2010 0:00:00~"Original"~1~"3033-11"~"PMR"~"505 (o)(3)"~3033.00~11~"PMR 3033-11: Conduct a clinical trial to estimate the serious risk for the development of hyperalgesia following the long-term use of high-dose ER/LA opioid analgesics  for at least one year to treat chronic pain. Include an assessment of risk relative to  efficacy."~"Delayed"~"The Trial Completion and Final Report milestones were missed because of continued discussions needed to reach agreement with the Agency on the Final Protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 8/26/2025."~8/31/2019 0:00:00~8/27/2018 0:00:00
384343~"CDER"~"NDA"~21356.00~"GILEAD SCIENCES INC"~"Viread (Tenofovir Disoproxil Fumarate) "~10/26/2001 0:00:00~"Supplement"~16~"212-2"~"PMR"~"Pediatric Research Equity Act"~212.00~2~"PMR 212-2: Deferred pediatric study under PREA for the use of VIREAD treatment, in combination with other antiretroviral agents, of HIV-1 in pediatric patients ages birth to 2 years of age.  Due to safety concerns for this age group, we are waiting for completion and review of studies in the 2 to 18 years age group before determining whether it is appropriate to study tenofovir in the birth to 2 years age group."~"Delayed"~"DE Granted letter issued on 10/27/2022."~1/31/2027 0:00:00~12/23/2025 0:00:00
384344~"CDER"~"BLA"~103909.00~"Genentech, Inc."~"TNKase (Tenecteplase) "~6/2/2000 0:00:00~"Supplement"~5197~"4716-2"~"PMR"~"Pediatric Research Equity Act"~4716.00~2~"PMR 4716-2: Conduct a clinical study, or studies, in patients at least 2 years through less than 12 years of age with acute ischemic stroke. A study, or substudy in a single study, should determine the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of TNKase (tenecteplase) to determine the appropriate dose or doses in patients less than 12 years of age. A study, or substudies within a single study, should determine if an appropriate dose of TNKase (tenecteplase) is safe and effective for the treatment of acute ischemic stroke in patients at least 2 years through less than 12 years of age. The safety and efficacy study, or studies, may be initiated after completion of the study in patients at least 12 years through less than 18 years of age intended to fulfill PMR 4716-1."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~10/31/2043 0:00:00~7/24/2025 0:00:00
384345~"CDER"~"BLA"~103948.00~"Genzyme Corporation"~"Campath (Alemtuzumab) and Lemtrada (Alemtuzumab)"~5/7/2001 0:00:00~"Supplement"~5139~"2832-3"~"PMR"~"505 (o)(3)"~2832.00~3~"PMR 2832-3: A prospective observational registry study in adult patients with relapsing multiple sclerosis, with the primary objective of determining the necessary duration of monitoring following treatment with Lemtrada (alemtuzumab) for  multiple sclerosis and to further inform appropriate monitoring conditions.Events of interest include autoimmune-mediated conditions, malignancies, serious  infections including opportunistic infections, and pneumonitis. A minimum of 5000 multiple sclerosis patients treated with Lemtrada (alemtuzumab) should be  enrolled and followed for a minimum of 10 years following the first exposure to Lemtrada (alemtuzumab). The protocol should specify an appropriate comparator population(s) to which observed incidence rates will be compared."~"Delayed"~"The final protocol milestone was missed due to continued
discussions between the applicant and the Agency to finalize the study design. The final protocol was acknowledged on 4/3/2024."~3/31/2029 0:00:00~7/2/2026 0:00:00
384346~"CDER"~"BLA"~103948.00~"Genzyme Corporation"~"Campath (Alemtuzumab) and Lemtrada (Alemtuzumab)"~5/7/2001 0:00:00~"Supplement"~5139~"2832-5"~"PMR"~"505 (o)(3)"~2832.00~5~"PMR 2832-5: A worldwide single-arm pregnancy safety study to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes (including any adverse effects in offspring) in women exposed to LEMTRADA (alemtuzumab) during pregnancy in patients with multiple sclerosis."~"Ongoing"~~12/31/2028 0:00:00~7/2/2026 0:00:00
384347~"CDER"~"BLA"~125031.00~"Amgen Inc."~"Neulasta (Pegfilgrastim)"~1/31/2002 0:00:00~"Original"~1~"3731-1"~"PMR"~"Pediatric Research Equity Act"~3731.00~1~"PMR 3731-1: Submit pediatric assessments for Neulasta (pegfilgrastim) as described in section 505B(a)(2)(A) of the FD&C Act, including development of an  appropriate formulation  (presentation) that can be used to directly and accurately administer Neulasta (pegfilgrastim) to pediatric patients who weigh less than 45 kg and require doses that are less than 0.6 mL (6 mg), and conducting any necessary human factors studies to evaluate the ability of healthcare providers and/or caregivers to measure the appropriate doses."~"Fulfilled"~"Per FDA letter dated 8/18/2025, this PMR/PMC has been fulfilled."~4/30/2025 0:00:00~3/27/2026 0:00:00
384348~"CDER"~"BLA"~125031.00~"Amgen Inc."~"Neulasta (Pegfilgrastim)"~1/31/2002 0:00:00~"Supplement"~180~"2997-1"~"PMR"~"Animal Efficacy"~2997.00~1~"PMR 2997-1: Conduct a phase 4 observational study evaluating the efficacy and safety of Neulasta (pegfilgrastim) in the setting of Hematopoietic Syndrome (HS) following acute radiation exposure."~"Pending"~~~3/27/2026 0:00:00
384349~"CDER"~"BLA"~125057.00~"AbbVie Inc."~"Humira (Adalimumab) "~12/31/2002 0:00:00~"Supplement"~110~"2657-2"~"PMC"~"506B PMC"~2657.00~2~"PMC 2657-2: Conduct a prospective, multi-center registry including 5000 adult psoriasis patients treated with Humira in the United States.  This registry will characterize and assess the incidence of serious adverse events (including serious infections, tuberculosis, opportunistic infections, malignancies, hypersensitivity reactions, autoimmune reactions and deaths) as well as other adverse events of interest in the study cohort.  All enrolled study patients will be evaluated for a period of at least 10 years with comprehensive annual reports provided to the Agency.  Collect data on the patient characteristics, demographics and drug exposure (including dose, duration and time to onset of adverse event).  The collection of data will be via active surveillance methods and data will be validated by a review of medical records as per the guidance for industry titled Good Pharmacovigilance Practices and Pharmacoepidemiologic Assessment."~"Submitted"~~1/31/2023 0:00:00~2/25/2026 0:00:00
384350~"CDER"~"BLA"~103846.00~"Solstice NeuroSciences, LLC"~"Myobloc (RimabotulinumtoxinB)"~12/8/2000 0:00:00~"Supplement"~5190~"3687-2"~"PMR"~"Pediatric Research Equity Act"~3687.00~2~"PMR 3687-2: A long-term, open-label safety and efficacy study in pediatric subjects 3 to less than 17 years of age with chronic troublesome sialorrhea that will follow the randomized, double-blind, placebo-controlled study. The long-term safety study needs to include information from at least 100 patients treated with Myobloc for chronic sialorrhea for 4 consecutive treatments over approximately 1-year, with at least 50 of these patients treated with the highest dose of Myobloc recommended in the label.  Submit an interim report for the long-term safety study, for safety information from pediatric patients ages 6 to < 17-years-old treated with Myobloc for sialorrhea for 4 consecutive treatments."~"Terminated"~"The study was terminated on 12/20/22 because there was an issue
with product supply which made initiation of these studies not possible."~12/31/2024 0:00:00~2/5/2026 0:00:00
384351~"CDER"~"BLA"~103909.00~"Genentech, Inc."~"TNKase (Tenecteplase) "~6/2/2000 0:00:00~"Supplement"~5197~"4716-1"~"PMR"~"Pediatric Research Equity Act"~4716.00~1~"PMR 4716-1: Conduct a randomized, controlled trial to evaluate the efficacy and safety of TNKase (tenecteplase), compared to an appropriate control, for the treatment of acute ischemic stroke in patients at least 12 years through less than 18 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2037 0:00:00~7/24/2025 0:00:00
384352~"CDER"~"BLA"~125057.00~"AbbVie Inc."~"Humira (Adalimumab) "~12/31/2002 0:00:00~"Supplement"~114~"2630-1"~"PMR"~"Pediatric Research Equity Act"~2630.00~1~"PMR 2630-1: Conduct Study Protocol P10-262, an 800-patient observational study, with inclusion of a reference group, of pediatric patients 4 to 17 years of age with moderately to severely active polyarticular juvenile idiopathic arthritis (JIA)."~"Submitted"~"The final report was submitted to FDA on 07/31/2024"~12/31/2021 0:00:00~2/25/2026 0:00:00
384353~"CDER"~"NDA"~203341.00~"PF PRISM CV"~"Bosulif (bosutinib)"~9/4/2012 0:00:00~"Supplement"~25~"4509-1"~"PMR"~"505 (o)(3)"~4509.00~1~"PMR 4509-1: Conduct Study BCHILD (ITCC-054/COG AAML1921) to identify, characterize, and determine the incidence of the serious potential risks of growth and developmental delay and worsening of serious adverse reactions including but not limited to gastrointestinal disorders, hematologic toxicity, and infectious adverse events with prolonged exposure (>12 months) to bosutinib in pediatric patients with chronic phase chronic myeloid leukemia (CML). All patients enrolled in Study BCHILD (ITCC-054/COG AAML1921) must be evaluated for growth and development milestones and serious adverse events annually for at least 5 years from the initiation of bosutinib. Include assessments of growth parameters, parameters of bone metabolism and growth, Tanner stage, hormones associated with growth and pubertal development, and serious adverse events. Include incidence rates, time to onset, and outcomes."~"Ongoing"~~1/31/2029 0:00:00~10/30/2025 0:00:00
384354~"CDER"~"NDA"~203415.00~"ASTELLAS PHARMA US INC"~"Xtandi (Enzalutamide)"~8/31/2012 0:00:00~"Supplement"~22~"4537-1"~"PMC"~"506B PMC"~4537.00~1~"PMC 4537-1: Complete clinical trial, EMBARK,  A Phase 3, Randomized, Efficacy and Safety Study of Enzalutamide Plus Leuprolide, Enzalutamide Monotherapy, and Placebo Plus Leuprolide in Men With High-Risk Nonmetastatic Prostate Cancer Progressing After Definitive Therapy,  to provide the final overall survival analyses."~"Ongoing"~~6/30/2027 0:00:00~10/29/2025 0:00:00
384355~"CDER"~"NDA"~203441.00~"TAKEDA PHARMACEUTICALS USA INC"~"Gattex (Teduglutide)"~12/21/2012 0:00:00~"Original"~1~"1978-1"~"PMR"~"505 (o)(3)"~1978.00~1~"PMR 1978-1: A prospective, multi-center, long-term, observational, registry study, of short bowel syndrome patients treated with teduglutide in a routine clinical setting, to assess the long-term safety of teduglutide. Design the study around a testable hypothesis to rule out a clinically meaningful increase in colorectal cancer risk above an estimated background risk in a suitable comparator. Select and justify the choice of appropriate comparator population(s) and corresponding background rate(s) relative to teduglutide-exposed patients. Provide sample sizes and effect sizes that can be ruled out under various enrollment target scenarios and loss to follow-up assumptions. The study's primary outcome should be colorectal cancer, and secondary outcomes should include other malignancies, colorectal polyps, bowel obstruction, pancreatic and biliary disease, heart failure, and long-term effectiveness. Patients should be enrolled over an initial 5-year period and then followed for a period of at least 10 years from the time of enrollment. Progress updates of registry patient accrual and a demographic summary should be provided annually. Registry safety data should be provided in periodic safety reports."~"Ongoing"~~6/30/2031 0:00:00~2/13/2026 0:00:00
384356~"CDER"~"NDA"~203469.00~"TAKEDA PHARMACEUTICALS USA INC"~"Iclusig (Ponatinib)"~12/14/2012 0:00:00~"Supplement"~34~"3960-1"~"PMR"~"505 (o)(3)"~3960.00~1~"PMR 3960-1: Submit the final study report and dataset with 5 years of follow-up for the ongoing Study AP24534-14-203 (OPTIC) titled   A Randomized, Open-label, Phase 2 Trial of Ponatinib in Patients with Resistant Chronic Phase Chronic Myeloid Leukemia to Characterize the Efficacy and Safety of a Range of Doses,  to characterize the risk of arterial occlusive events and the long-term safety of ponatinib in patients with chronic myeloid leukemia (CML) who have received at least two prior tyrosine kinase inhibitors or have a T315I mutation. Include data from approximately 282 patients with CML. Include in the final study report the exploratory subgroup analyses and corresponding subject level data related to baseline cardiovascular risk factors, age, and T315I status."~"Fulfilled"~~1/31/2025 0:00:00~2/10/2026 0:00:00
384357~"CDER"~"NDA"~203469.00~"TAKEDA PHARMACEUTICALS USA INC"~"Iclusig (Ponatinib)"~12/14/2012 0:00:00~"Supplement"~37~"4593-1"~"PMR"~"Accelerated Approval"~4593.00~1~"PMR 4593-1: Complete the randomized trial Ponatinib-3001 (PhALLCON) intended to verify and describe the clinical benefit of ponatinib in adults with newly diagnosed Ph+ acute lymphoblastic leukemia."~"Ongoing"~"As per the 180-day AA PMR Progress Report submitted on 02/10/2026, the PhALLCON trial is progressing as per the original schedule of milestones. The trial is completely enrolled."~6/30/2028 0:00:00~2/10/2026 0:00:00
384358~"CDER"~"NDA"~203469.00~"TAKEDA PHARMACEUTICALS USA INC"~"Iclusig (Ponatinib)"~12/14/2012 0:00:00~"Supplement"~37~"4593-2"~"PMR"~"505 (o)(3)"~4593.00~2~"PMR 4593-2: Complete the randomized trial Ponatinib-3001 (PhALLCON) to assess the long-term safety of ponatinib including the incidence of known serious risks of arterial occlusive events, venous thromboembolic events, and pancreatitis in adults with newly diagnosed Ph+ acute lymphoblastic leukemia."~"Ongoing"~~6/30/2028 0:00:00~2/10/2026 0:00:00
384359~"CDER"~"NDA"~203629.00~"FRESENIUS KABI USA LLC"~"Neostigmine Methylsulfate "~1/8/2015 0:00:00~"Original"~1~"2855-1"~"PMR"~"505 (o)(3)"~2855.00~1~"PMR 2855-1: Conduct an adequate extractable/leachable safety assessment for the [...] gray [...] rubber stopper used in your  container closure system. This assessment must include controlled extraction  studies to qualitatively and quantitatively determine the chemical species that may  migrate into the dosage form, using appropriate solvents that adequately represent  the chemical characteristics of the drug product formulation. Additionally,  leachable data from long-term stability studies (taking into consideration the proposed shelf-life) should be used to determine if the identified/specified  extractables also leach into the drug product over time. Using this information, conduct a toxicological risk assessment justifying the safety of the extractables  and leachables, taking into consideration the maximum daily dose of the identified materials for this drug product. For your toxicological risk assessment,  any leachable that contains a structural alert for mutagenicity should not exceed [..] mcg/day total daily exposure, or it must be adequately qualified for safety. A  toxicological risk assessment should be provided for any non-genotoxic leachable that exceeds 5 mcg/day."~"Delayed"~"The final report milestone was missed, because of delays in feedback provided on the previous final report submission and FDA determined that the applicant demonstrated  good cause  for the delay."~1/31/2015 0:00:00~2/26/2026 0:00:00
384360~"CDER"~"NDA"~203634.00~"SALIX PHARMACEUTICALS INC"~"Uceris (Budesonide)"~1/14/2013 0:00:00~"Original"~1~"1997-1"~"PMR"~"Pediatric Research Equity Act"~1997.00~1~"PMR 1997-1: An 8-week randomized, double blind, placebo-controlled trial in children 5 to 17 years of age with active, mild to moderate ulcerative colitis. The trial will evaluate pharmacokinetics (PK), efficacy for induction of remission, and safety of  at least 2 doses of Uceris (budesonide). The effects of 8 weeks of Uceris (budesonide) on the HPA axis will be assessed."~"Delayed"~"The final report submission milestone was missed. Deferral Extension denied on 7/8/2021. The trial was placed on hold in March 2020 due to the COVID-19 pandemic, but the sponsor restarted activities in Q4 of 2022. 2/70 subjects enrolled as of their March 13, 2026 annual status report."~9/30/2016 0:00:00~3/13/2026 0:00:00
384361~"CDER"~"NDA"~208171.00~"PHARMACOSMOS AS"~"Monoferric (Ferric Derisomaltose)"~1/16/2020 0:00:00~"Original"~1~"3759-2"~"PMR"~"Pediatric Research Equity Act"~3759.00~2~"PMR 3759-2: Conduct a study (PK sub-study of P-Monofer-PED-01) in pediatric patients aged <1 to <17 years of age with iron deficiency anemia due to nondialysis dependent chronic kidney disease or iron deficiency anemia intolerant to or unresponsive to oral iron to confirm the dosing of intravenous (IV) ferric derisomaltose in pediatric patients. Include pharmacokinetic and pharmacodynamic analyses and submit datasets at the time of the final report submission."~"Delayed"~"The final report milestone was missed, because of delays involving study participants, sites, and/or management. Original Final Report Due Date: 12/31/2022; Deferral Extension granted 6/9/2022 and 4/26/2024. The study completion milestone was also revised. Deferral Extension Requested 01/16/2026. Denied per FDA letter dated 03/03/2026."~10/31/2026 0:00:00~3/16/2026 0:00:00
384362~"CDER"~"NDA"~208215.00~"GILEAD SCIENCES INC"~"Descovy (Emtricitabine and Tenofovir Alafenamide)"~4/4/2016 0:00:00~"Original"~1~"3531-1"~"PMR"~"Pediatric Research Equity Act"~3531.00~1~"PMR 3531-1: Conduct a study to evaluate the pharmacokinetics (PK), safety and antiviral activity of DESCOVY (emtricitabine and tenofovir alafenamide) administered in combination with atazanavir and TYBOST (cobicistat), and in combination with darunavir and TYBOST in HIV-1 infected pediatric subjects weighing less than 25 kg. The safety and activity of the treatment regimen must be assessed for a minimum of 24 weeks. The minimum age criteria for the treatment regimen being evaluated are specified below.   DESCOVY administered in combination with atazanavir co-administered with TYBOST must be evaluated in pediatric patients 3 months of age and older   DESCOVY administered in combination with darunavir co-administered with TYBOST must be evaluated in pediatric patients 3 years of age and older"~"Released"~"Per FDA letter dated 07/24/2025, this PMR/PMC has been released."~6/30/2026 0:00:00~5/27/2026 0:00:00
384363~"CDER"~"NDA"~208215.00~"GILEAD SCIENCES INC"~"Descovy (Emtricitabine and Tenofovir Alafenamide)"~4/4/2016 0:00:00~"Original"~1~"3531-3"~"PMR"~"Pediatric Research Equity Act"~3531.00~3~"PMR 3531-3: Conduct a study to evaluate the pharmacokinetics (PK), safety and antiviral activity of DESCOVY administered in combination with regimens that do not contain cobicistat or other PI/CYP3A inhibitor in HIV-1 infected pediatric subjects 4 weeks of age and older and weighing less than 25 kg. The safety and activity of DESCOVY must be assessed for a minimum of 24 weeks. Clinical trials evaluating DESCOVY administered in combination with regimens that do not contain cobicistat or a PI/CYP3A inhibitor are not required if data from other ongoing or completed pediatric studies can be leveraged to provide the requested PK, safety, and antiviral activity data for DESCOVY."~"Released"~"Per FDA letter dated 07/24/2025, this PMR/PMC has been released."~6/30/2026 0:00:00~5/27/2026 0:00:00
384364~"CDER"~"NDA"~208215.00~"GILEAD SCIENCES INC"~"Descovy (Emtricitabine and Tenofovir Alafenamide)"~4/4/2016 0:00:00~"Original"~1~"3531-4"~"PMR"~"Pediatric Research Equity Act"~3531.00~4~"PMR 3531-4: Conduct a study to evaluate the PK, safety and antiviral activity of DESCOVY administered in combination with lopinavir/ritonavir in HIV-1 infected pediatric subjects 4 weeks to less than 3 months of age. The safety and activity of DESCOVY must be assessed for a minimum of 24 weeks."~"Released"~"Per FDA letter dated 07/24/2025, this PMR/PMC has been released."~6/30/2026 0:00:00~5/27/2026 0:00:00
384365~"CDER"~"NDA"~208215.00~"GILEAD SCIENCES INC"~"Descovy (Emtricitabine and Tenofovir Alafenamide)"~4/4/2016 0:00:00~"Original"~1~"3531-5"~"PMC"~"506B PMC"~3531.00~5~"PMC 3531-5: Conduct a trial to evaluate the pharmacokinetics (PK), safety and antiviral activity of DESCOVY (emtricitabine and tenofovir alafenamide) administered in combination with atazanavir and TYBOST (cobicistat) in pediatric patients with HIV-1 infection who are 3 months of age and older and weighing less than 14 kg. The safety and activity of the treatment regimen must be assessed for a minimum of 24 weeks."~"Ongoing"~~6/30/2026 0:00:00~5/27/2026 0:00:00
384366~"CDER"~"NDA"~208215.00~"GILEAD SCIENCES INC"~"Descovy (Emtricitabine and Tenofovir Alafenamide)"~4/4/2016 0:00:00~"Original"~1~"3531-6"~"PMC"~"506B PMC"~3531.00~6~"PMC 3531-6: Conduct a trial to evaluate the pharmacokinetics (PK), safety and antiviral activity of DESCOVY administered in combination with regimens that do not contain cobicistat or other PI/CYP3A inhibitor in pediatric patients with HIV-1 infection who are 4 weeks of age and older and weighing less than 25 kg. The safety and activity of DESCOVY must be assessed for a minimum of 24 weeks. Clinical trials evaluating DESCOVY administered in combination with regimens that do not contain cobicistat or a PI/CYP3A inhibitor are not required if data from other ongoing or completed pediatric studies can be leveraged to provide the requested PK, safety, and antiviral activity data for DESCOVY."~"Ongoing"~~6/30/2026 0:00:00~5/27/2026 0:00:00
384367~"CDER"~"NDA"~208215.00~"GILEAD SCIENCES INC"~"Descovy (Emtricitabine and Tenofovir Alafenamide)"~4/4/2016 0:00:00~"Original"~1~"3531-7"~"PMC"~"506B PMC"~3531.00~7~"PMC 3531-7: Conduct a trial to evaluate the PK, safety and antiviral activity of DESCOVY administered in combination with lopinavir/ritonavir in pediatric patients with HIV-1 infection who are 4 weeks to less than 3 months of age. The safety and activity of DESCOVY must be assessed for a minimum of 24 weeks."~"Ongoing"~~6/30/2026 0:00:00~5/27/2026 0:00:00
384368~"CDER"~"NDA"~208215.00~"GILEAD SCIENCES INC"~"Descovy (Emtricitabine and Tenofovir Alafenamide)"~4/4/2016 0:00:00~"Supplement"~12~"3723-1"~"PMC"~"506B PMC"~3723.00~1~"PMC 3723-1: Conduct a randomized, comparative trial to evaluate the safety and efficacy of Descovy for pre-exposure prophylaxis (PrEP) in cisgender women and adolescent girls weighing at least 35 kg, who are at risk of sexually acquired HIV-1 infection. The trial should include a Truvada arm. The trial should employ two distinct methods to estimate the background HIV-1 incidence rate as external controls. HIV-1 incidence estimates should be based on current estimates from sites involved in recent clinical trials, cross-sectional HIV surveillance surveys, and from high quality local epidemiology data."~"Fulfilled"~~6/30/2025 0:00:00~5/27/2026 0:00:00
384369~"CDER"~"NDA"~208246.00~"PFIZER INC"~"Xeljanz XR (Tofacitinib)"~2/23/2016 0:00:00~"Original"~1~"3045-3"~"PMR"~"Pediatric Research Equity Act"~3045.00~3~"PMR 3045-3: Conduct a pharmacokinetic study in adult healthy volunteers to establish the equivalence of AUC and similarity in other pharmacokinetic parameters between the proposed commercial age-appropriate tofacitinib XR formulation and tofacitinib IR formulation."~"Delayed"~"Missed original final report submission due date of May 2020. Four deferral extension granted letters were issued on 7/19/2019, 2/24/2021, 10/25/2022, and 2/20/2024 with the latter letter stating final report submission due date of 3/2027. The final protocol and study completion milestones have also been revised."~11/30/2022 0:00:00~12/23/2025 0:00:00
384370~"CDER"~"NDA"~21357.00~"BRACCO DIAGNOSTICS INC"~"MultiHance (Gadobenate Dimeglumine) and MultiHance Multipack (Gadobenate Dimeglumine)"~11/23/2004 0:00:00~"Supplement"~17~"3626-4"~"PMR"~"505 (o)(3)"~3626.00~4~"PMR 3626-4: A prospective longitudinal cohort trial with one or more matched control group(s) to evaluate the effects of repetitive GBCA administration on a comprehensive battery of neurobehavioral testing over the course of at least five administrations. The trial should be sufficiently powered to exclude a pre-specified magnitude of decline. As a secondary objective, trial patients should also have the option of providing blood and urine samples at the time of reimaging, so that normative estimates of gadolinium concentration across an extended range of post-administration timepoints may be documented."~"Ongoing"~~4/30/2029 0:00:00~2/2/2026 0:00:00
384371~"CDER"~"NDA"~21358.00~"BRACCO DIAGNOSTICS INC"~"MultiHance (Gadobenate Dimeglumine) and MultiHance Multipack (Gadobenate Dimeglumine)"~11/23/2004 0:00:00~"Supplement"~16~"3626-4"~"PMR"~"505 (o)(3)"~3626.00~4~"PMR 3626-4: A prospective longitudinal cohort trial with one or more matched control group(s) to evaluate the effects of repetitive GBCA administration on a comprehensive battery of neurobehavioral testing over the course of at least five administrations. The trial should be sufficiently powered to exclude a pre-specified magnitude of decline. As a secondary objective, trial patients should also have the option of providing blood and urine samples at the time of reimaging, so that normative estimates of gadolinium concentration across an extended range of post-administration timepoints may be documented."~"Ongoing"~~4/30/2029 0:00:00~12/19/2024 0:00:00
384372~"CDER"~"NDA"~21361.00~"SALIX PHARMACEUTICALS INC"~"Xifaxan (Rifaximin)"~5/25/2004 0:00:00~"Original"~1~"858-1"~"PMR"~"Pediatric Research Equity Act"~858.00~1~"PMR 858-1: Deferred pediatric studies under PREA for the treatment of traveler's diarrhea in pediatric patients ages 3 years to 12 years.  Final Report Submission: May 1, 2009."~"Delayed"~"The final report submission milestone was missed and FDA issued a noncompliance letter dated 9-13-2021. The applicant submitted a request for a deferral extension on 10-27-21 that was denied on 12-8-2021. A revised protocol  and statistical analysis plan were  submitted on 9-25-2024. The applicant is working on revising the protocol."~7/31/2018 0:00:00~7/23/2024 0:00:00
384373~"CDER"~"NDA"~21361.00~"SALIX PHARMACEUTICALS INC"~"Xifaxan (Rifaximin)"~5/25/2004 0:00:00~"Supplement"~12~"2900-1"~"PMR"~"Pediatric Research Equity Act"~2900.00~1~"PMR 2900-1: Conduct a pharmacokinetic, pharmacodynamics, tolerability and dose ranging study in subjects 6 years through 17 years, to assess safety and to determine the  doses to be used in the efficacy study. Age-specific endpoints, i.e. a clinical outcome assessment utilizing Patient Reported Outcomes (PROs) or a PRO instrument to evaluate efficacy of rifaximin for the treatment of irritable bowel disease (IBS) in children, should be developed."~"Delayed"~"The final study report was missed. Deferral Extension request denied per letter dated 8/14/2019 and noncompliance letter issued on 8/21/2019."~3/31/2019 0:00:00~7/23/2024 0:00:00
384374~"CDER"~"NDA"~21361.00~"SALIX PHARMACEUTICALS INC"~"Xifaxan (Rifaximin)"~5/25/2004 0:00:00~"Supplement"~12~"2900-2"~"PMR"~"Pediatric Research Equity Act"~2900.00~2~"PMR 2900-2: Conduct a randomized, controlled, double-blind study to determine the  safety and efficacy of rifaximin in pediatric patients 6 years through 17 years with diarrhea-predominant irritable bowel syndrome (IBS-D)."~"Delayed"~"Original final report due date: 12/31/2023; Deferral Extension granted per FDA letter dated 1/31/2024."~12/31/2026 0:00:00~7/23/2024 0:00:00
384375~"CDER"~"NDA"~21388.00~"SCIARRA LABORATORIES INC"~"Talc"~12/15/2003 0:00:00~"Original"~1~"3332-1"~"PMR"~"505 (o)(3)"~3332.00~1~"PMR 3332-1: Perform a risk assessment study on the lead content of sterile talc powder and indicate any necessary controls on elemental impurities as per ICH Q3D Elemental Impurities: Guidance for Industry."~"Delayed"~"FDA determined that the data in the final report submitted by the applicant on 01/06/2021 did not fulfill the terms of the commitment. FDA issued a not fulfilled letter on 12/15/2021.
"~5/31/2018 0:00:00~2/9/2024 0:00:00
384376~"CDER"~"NDA"~21409.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~7/26/2002 0:00:00~"Original"~1~"4875-1"~"PMR"~"505 (o)(3)"~4875.00~1~"PMR 4875-1: Conduct a study to investigate the temporal and spatial distribution of montelukast into the brain following repeated oral dosing in adult and juvenile rodents."~"Pending"~~9/30/2027 0:00:00~9/19/2012 0:00:00
384377~"CDER"~"NDA"~21409.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~7/26/2002 0:00:00~"Original"~1~"4875-2"~"PMR"~"505 (o)(3)"~4875.00~2~"PMR 4875-2: Conduct a study to investigate off-target binding and functional activity of montelukast, with particular focus on potential central nervous system (CNS) effects."~"Pending"~~9/30/2027 0:00:00~9/19/2012 0:00:00
384378~"CDER"~"NDA"~21409.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~7/26/2002 0:00:00~"Original"~1~"4875-3"~"PMR"~"505 (o)(3)"~4875.00~3~"PMR 4875-3: Conduct a study to investigate potential CNS effects of montelukast after repeated oral dosing in a juvenile rodent study with complex neurobehavioral endpoints."~"Released"~~11/30/2027 0:00:00~9/19/2012 0:00:00
384379~"CDER"~"NDA"~21409.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~7/26/2002 0:00:00~"Original"~1~"4875-4"~"PMR"~"505 (o)(3)"~4875.00~4~"PMR 4875-4: Conduct a study to further characterize human-relevant metabolites of montelukast, including identification, primary and secondary pharmacology, kinetics, and tissue distribution."~"Pending"~~2/28/2027 0:00:00~9/19/2012 0:00:00
384380~"CDER"~"NDA"~21446.00~"UPJOHN US 2 LLC"~"Lyrica (pregabalin)"~12/30/2004 0:00:00~"Original"~1~"3716-1"~"PMR"~"505 (o)(3)"~3716.00~1~"PMR 3716-1: In a healthy non-drug dependent population with drug abuse experience with opioids, evaluate the abuse potential of pregabalin taken concomitantly with an opioid, such as oxycodone, investigating a range of doses of pregabalin combined with the opioid. This clinical trial will evaluate the pregabalin/opioid combinations in a cross-over design with comparison to placebo, pregabalin alone, and a moderate dose of the opioid taken alone as a positive control. The assessments will include abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and pharmacokinetics of both pregabalin and the opioid with the goal of characterizing any additive or synergistic effects on the abuse potential and physiologic effects of co-administered pregabalin and opioid."~"Delayed"~"The Final Report milestone was missed because the Final Report submitted 01/31/2023 did not fulfill the PMR due to uninterpretable data, per FDA letter dated 01/26/2024. The Applicant proposed revised milestones and FDA determined the applicant demonstrated  good cause  for delay per FDA letter dated 07/11/2025. The Final Protocol was acknowledged by FDA on 03/25/2026."~8/31/2021 0:00:00~9/29/2025 0:00:00
384381~"CDER"~"NDA"~21447.00~"LEGACY PHARMA INC SEZC"~"Zanaflex (Tizanidine Hydrochloride)"~8/29/2002 0:00:00~"Original"~1~"1739-1"~"PMR"~"Pediatric Research Equity Act"~1739.00~1~"PMR 1739-1: Deferred pediatric study of tizanidine for acute treatment of spasticity in pediatric patients ages 0 to 16."~"Delayed"~"The PMR was not fulfilled per FDA letter dated 3/8/18. Discussions continue between the agency and the applicant to determine how best to proceed with the study."~12/31/2005 0:00:00~10/29/2025 0:00:00
384382~"CDER"~"BLA"~125057.00~"AbbVie Inc."~"Humira (Adalimumab) "~12/31/2002 0:00:00~"Supplement"~232~"2517-2"~"PMR"~"505 (o)(3)"~2517.00~2~"PMR 2517-2: A multi-center observational study of Humira (adalimumab) in adults with  moderately to severely active ulcerative colitis treated in a routine clinical setting, to assess the long-term safety as measured by the incidence of opportunistic infections and malignancies. Long-term effectiveness should be assessed as a secondary goal. The proposed study should follow patients for a period of at least 10 years from time of enrollment in order to ascertain adverse events with longer latency periods such as malignancies. The primary analysis is to summarize safety data for patients on adalimumab and patients on non-biologic immunomodulator therapy. The study should be adequately sized to sufficiently detect a doubling of the risk of lymphoma events in each treatment group. A secondary analysis is to summarize safety data for patients on adalimumab and patients on the combination of adalimumab and non-biologic immunomodulator therapy. In addition, the study is to document and evaluate effects of withdrawal and re-treatment with adalimumab and switching with other tumor necrosis factor (TNF)-blockers or biologics."~"Ongoing"~~12/31/2029 0:00:00~2/25/2026 0:00:00
384383~"CDER"~"BLA"~125058.00~"Biomarin Pharmaceutical Inc."~"Aldurazyme (laronidase)"~4/30/2003 0:00:00~"Supplement"~160~"2460-7"~"PMC"~"506B PMC"~2460.00~7~"PMC 2460-7: BioMarin commits to developing an assay to detect anti-Aldurazyme antibody mediated neutralization of enzyme uptake. Such an assay could be developed based on the current cell uptake assay already used for Aldurazyme lot release. BioMarin commits to developing this neutralization assay and submitting a completed validation report."~"Submitted"~~10/30/2009 0:00:00~6/30/2026 0:00:00
384384~"CDER"~"BLA"~125104.00~"Biogen Inc."~"Tysabri (Natalizumab) "~11/23/2004 0:00:00~"Original"~1~"3039-1"~"PMR"~"505 (o)(3)"~3039.00~1~"PMR 3039-1: Conduct a study to determine if Tysabri (natalizumab) increases the risk of major congenital malformations. In this study, the risk of fetal and infant outcomes (including malformations) among Tysabri-exposed women identified  from the Tysabri Pregnancy Exposure Registry should be compared to the risk among 1) women with multiple sclerosis (MS) exposed to non-Tysabri therapies during pregnancy, 2) women with MS unexposed to any MS therapy during pregnancy, and 3) women without MS. Women with MS exposed to non-Tysabri therapies will be selected from the following: the Avonex Pregnancy  Registry, the Tecfidera Pregnancy Registry, the Plegridy Pregnancy Registry, and the European Union Interferon-beta Pregnancy Registry. Women with MS  unexposed to any MS therapy and women without MS should be identified from the Nordic pharmacoepidemiology study. The same birth defect classification  system must be used across all populations and comparisons. All comparisons are to be summarized in a final study report."~"Submitted"~~12/31/2025 0:00:00~1/23/2026 0:00:00
384385~"CDER"~"BLA"~125118.00~"Bristol-Myers Squibb Company"~"Orencia (Abatacept)"~12/23/2005 0:00:00~"Supplement"~45~"2709-5"~"PMR"~"505 (o)(3)"~2709.00~5~"PMR 2709-5: A JIA patient safety registry comprised of at least 500 patients.  The protocol for this study should include a plan for more intensive scrutiny for the first 3 years, with annual follow ups (which could be telephonic) assessing for occurrence of malignancies, other autoimmune diseases, and serious infections, for a total of 10 years.  Patients turning 18 years of age or older should continue to be followed until they have completed the 10 year follow-up period.  Information on these patients may be obtained via annual questionnaire/telephonic follow-up with attention to key adverse events rather than full clinic visit with examination. Occurrence of these serious events should be formally compared to the expected rate of these events in historical controls."~"Ongoing"~~6/30/2029 0:00:00~2/14/2024 0:00:00
384386~"CDER"~"BLA"~125118.00~"Bristol-Myers Squibb Company"~"Orencia (Abatacept)"~12/23/2005 0:00:00~"Supplement"~211~"3191-1"~"PMR"~"505 (o)(3)"~3191.00~1~"PMR 3191-1: Observational study in pediatric patients 2 to 5 years of age with juvenile idiopathic arthritis (JIA) treated with abatacept to evaluate the long term safety of abatacept, with respect to the risk of malignancies, autoimmune diseases, and serious infections."~"Ongoing"~~6/30/2029 0:00:00~2/14/2024 0:00:00
384387~"CDER"~"BLA"~125118.00~"Bristol-Myers Squibb Company"~"Orencia (Abatacept)"~12/23/2005 0:00:00~"Supplement"~240~"4189-1"~"PMR"~"505 (o)(3)"~4189.00~1~"PMR 4189-1: Conduct a clinical trial to further characterize the known serious risk of infections with abatacept in patients age 2 to less than 6 years and to characterize the pharmacokinetics (PK) of abatacept to verify the population PK predicted optimal dosing regimen of abatacept (15 mg/kg as a 60-minute intravenous infusion on Day -1, followed by 12 mg/kg on Days 5, 14, and 28 after transplantation) in pediatric patients age 2 to less than 6 years for the prophylaxis of acute graft versus host disease, in combination with a calcineurin inhibitor and methotrexate, undergoing hematopoietic stem cell transplantation from a matched or 1 allele-mismatched unrelated donor."~"Ongoing"~~12/31/2026 0:00:00~2/14/2024 0:00:00
384388~"CDER"~"BLA"~125151.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Elaprase (Idursulfase) "~7/24/2006 0:00:00~"Supplement"~184~"2792-1"~"PMR"~"Accelerated Approval"~2792.00~1~"PMR 2792-1: To conduct a verification trial to describe clinical benefit attributable to Elaprase (idursulfase) in a cohort of Hunter syndrome patients 5 years of age and younger.At a minimum, this trial will assess longitudinal changes in anthropometric  measures (i.e., length/height z-scores, annual growth velocity z-scores, weight zscores)  and the progression of skeletal deformities (i.e. joint stiffness, joint  contractures) in children being treated with Elaprase (idursulfase). The growth  parameters will be followed in these children for a minimum of 5 years from  initiation of Elaprase (idursulfase) treatment or until they have reached at least 10  years of age, whichever is longer. The trials will monitor antibody response  (binding, neutralizing, and IgE) at least every 6 months. Additionally, the trial  will evaluate the relationship between development of immune tolerance and  genetic mutations, endogenous enzyme activity level, and anthropometric  measures. The trial may be conducted as a separate trial or as a sub-trial under a special protocol within the Hunter Outcome Survey."~"Delayed"~"The trial completion milestone was missed. The applicant requested a revised milestone because the Agency has requested the inclusion of additional datasets, post hoc analyses, subgroup analyses, and other pertinent information in the upcoming sBLA package. A Revised milestone was acknowledged in a letter dated 01/21/2026."~9/30/2022 0:00:00~9/18/2025 0:00:00
384389~"CDER"~"NDA"~203826.00~"HIKMA PHARMACEUTICALS USA INC"~"Immphentiv (Phenylephrine Hydrochloride)"~12/20/2012 0:00:00~"Original"~1~"1991-1"~"PMR"~"Pediatric Research Equity Act"~1991.00~1~"PMR 1991-1: Conduct a trial in the > or =12 - 16 year old age group to evaluate the dose effect of phenylephrine hydrochloride injection on blood pressure in patients undergoing general  anesthesia and neuroaxial anesthesia. Administration by both the bolus and infusion methods must be studied for the treatment of hypotension. Dosing of phenylephrine  should be weight-based since weight may be quite variable in this population. The study should include 50 subjects in the bolus treatment group and 50 subjects in the infusion  treatment group. The study should capture, at a minimum, the following information:   * Demographic and medical history information that informs about the subjects' cardiovascular status.  * Concomitant intraoperative and post-operative medications, including their doses and adjustments in inhaled gas concentration or intravenous agent infusion rates.  * Interventions used to treat the hypotension, e.g., other pressor agents, intravenous fluid boluses, changes in patient positioning.  * Intraoperative events relevant to subjects' physiological status, such as blood loss and fluids administered.  * Blood pressures and heart rate, time to onset and maximal response and duration of response should be defined and captured before and during the treatment.  * Pharmacokinetics of the proposed product need to be characterized at points relative to the phenylephrine administration."~"Delayed"~"Three sites are currently recruiting study subjects in the
United States. A total of 77 subjects were successfully
enrolled and completed participation."~12/31/2024 0:00:00~2/12/2026 0:00:00
384390~"CDER"~"NDA"~203858.00~"CHIESI FARMACEUTICI SPA"~"Juxtapid (Lomitapide Mesylate) "~12/21/2012 0:00:00~"Original"~1~"1974-3"~"PMR"~"505 (o)(3)"~1974.00~3~"PMR 1974-3: A long-term prospective observational study (product exposure registry) of patients with homozygous familial hypercholesterolemia (HoFH) treated with Juxtapid (lomitapide) to evaluate known and potential serious risks related to the  use of Juxtapid (lomitapide), including hepatic transaminase elevations, hepatic steatosis, small bowel and hepatic malignancies, teratogenicity, death (including  cause of death), and major adverse cardiovascular events (including  cardiovascular death, non-fatal myocardial infarction, non-fatal stroke, unstable angina, and revascularization procedures). The registry will continue for 10 years  from the date of last patient enrollment."~"Released"~~9/1/2028 0:00:00~2/19/2026 0:00:00
384391~"CDER"~"NDA"~203971.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Xofigo (Radium Ra 223 Dichloride)"~5/15/2013 0:00:00~"Original"~1~"2041-1"~"PMR"~"505 (o)(3)"~2041.00~1~"PMR 2041-1: An observational study (N = 1200) to assess the long-term safety of radium Ra 223 dichloride 50 kBq/kg every 4 weeks for 6 doses in patients with castrationresistant  prostate cancer with bone metastases."~"Fulfilled"~~9/30/2024 0:00:00~7/14/2026 0:00:00
384392~"CDER"~"NDA"~203971.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Xofigo (Radium Ra 223 Dichloride)"~5/15/2013 0:00:00~"Original"~1~"2041-2"~"PMR"~"505 (o)(3)"~2041.00~2~"PMR 2041-2: A randomized clinical trial to assess the safety of radium Ra 223 dichloride in patients with castration-resistant prostate cancer, symptomatic bone metastases and no known visceral metastatic disease."~"Fulfilled"~~3/31/2025 0:00:00~7/14/2026 0:00:00
384393~"CDER"~"NDA"~204017.00~"AGILE THERAPEUTICS INC"~"Twirla (Ethinyl Estradiol and Levonorgestrel)"~2/14/2020 0:00:00~"Original"~1~"3785-1"~"PMR"~"505 (o)(3)"~3785.00~1~"PMR 3785-1: A controlled, non-interventional, prospective, observational cohort study comparing the risks for fatal and non-fatal venous thromboembolism (VTE) and arterial thromboembolism (ATE) in new users of Twirla to new users of oral combined hormonal contraceptives (CHCs) (primary comparator) and new users of Xulane (secondary comparator) in U.S. women of reproductive age using CHCs primarily for contraceptive reasons. The study should be designed to detect a 1.5 to 2-fold increased risk for VTE in new users of Twirla and adequately measure and control for possible confounders, especially age, BMI, and smoking status, among others."~"Delayed"~"Per the applicant s annual status report, the revised final protocol submission milestone was missed due to continued discussions with the Agency."~11/30/2032 0:00:00~4/10/2026 0:00:00
384394~"CDER"~"NDA"~204026.00~"BRISTOL MYERS SQUIBB CO"~"Pomalyst (Pomalidomide)"~2/8/2013 0:00:00~"Supplement"~23~"3855-1"~"PMR"~"Accelerated Approval"~3855.00~1~"PMR 3855-1: Submit the final report from a clinical trial evaluating overall response rate, duration of response, and safety to verify and describe the clinical benefit of pomalidomide in patients with Kaposi sarcoma who are human immunodeficiency virus (HIV) positive (after failure of highly active antiretroviral therapy) and HIV negative, that may inform product labeling."~"Ongoing"~"The trial has been initiated."~12/31/2027 0:00:00~4/6/2026 0:00:00
384395~"CDER"~"NDA"~204026.00~"BRISTOL MYERS SQUIBB CO"~"Pomalyst (Pomalidomide)"~2/8/2013 0:00:00~"Supplement"~24~"3855-1"~"PMR"~"Accelerated Approval"~3855.00~1~"PMR 3855-1: Submit the final report from a clinical trial evaluating overall response rate, duration of response, and safety to verify and describe the clinical benefit of pomalidomide in patients with Kaposi sarcoma who are human immunodeficiency virus (HIV) positive (after failure of highly active antiretroviral therapy) and HIV negative, that may inform product labeling."~"Ongoing"~"The trial has been initiated."~12/31/2027 0:00:00~4/6/2026 0:00:00
384396~"CDER"~"NDA"~204063.00~"BIOGEN INC"~"Tecfidera (Dimethyl Fumarate) "~3/27/2013 0:00:00~"Original"~1~"2014-8"~"PMR"~"Pediatric Research Equity Act"~2014.00~8~"PMR 2014-8: Conduct a two-part study of Tecfidera (dimethyl fumarate) in pediatric patients with relapsing forms of multiple sclerosis (RMS) at least 10 years and less than 18 years of age. Part A is an open-label study for evaluating the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Tecfidera (dimethyl fumarate) in pediatric patients weighing 40 kg or less. The objective of Part A is to determine titration and maintenance doses of Tecfidera (dimethyl fumarate) that will result in PK and PD effect that are comparable to those of the maintenance doses administered to adult patients. Part B is a randomized, double-blind, parallel-group study in pediatric patients weighing greater than 40 kg to evaluate the efficacy and safety of Tecfidera (dimethyl fumarate) compared to an appropriate control."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 01/21/2025."~2/28/2029 0:00:00~5/22/2026 0:00:00
384397~"CDER"~"NDA"~204114.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mekinist (trametinib)"~5/29/2013 0:00:00~"Original"~1~"2045-2"~"PMR"~"505 (o)(3)"~2045.00~2~"PMR 2045-2: Submit integrated safety analyses from an adequate number of randomized controlled clinical trial(s) using Mekinist (trametinib) to identify and characterize  the risk of retinal pigmented epithelial detachments (RPED), including safety evaluations adequate to inform labeling of patient populations at highest risk and to provide evidence-based dose modification and monitoring recommendations in labeling of RPED events."~"Fulfilled"~~9/30/2016 0:00:00~7/23/2025 0:00:00
384398~"CDER"~"NDA"~208246.00~"PFIZER INC"~"Xeljanz XR (Tofacitinib)"~2/23/2016 0:00:00~"Supplement"~9~"3766-1"~"PMR"~"Pediatric Research Equity Act"~3766.00~1~"PMR 3766-1: A phase 3, randomized, controlled study to assess the pharmacokinetics, safety and efficacy of the Xeljanz (tofacitinib) XR formulation (to be determined) for the induction and maintenance of remission in pediatric patients 2 to 17 years of age with moderately to severely active UC. The total duration of this study will be approximately 52 weeks."~"Delayed"~"Deferral extension requested 3/29/2024. Denied per FDA letter dated 5/14/2024. However, per 5/14/2024 letter, the final protocol submission, study completion, and final report submission milestones were revised due to technical challenges. The original final protocol submission milestone has been missed. 

"~11/30/2028 0:00:00~12/23/2025 0:00:00
384399~"CDER"~"NDA"~208246.00~"PFIZER INC"~"Xeljanz XR (Tofacitinib)"~2/23/2016 0:00:00~"Supplement"~9~"3766-2"~"PMR"~"Pediatric Research Equity Act"~3766.00~2~"PMR 3766-2: A two-year, open-label, safety extension study would be designed to collect additional safety data, such as AEs of special interest, in pediatric patients with UC treated with tofacitinib XR formulation (to be determined) from the randomized controlled study (PMR 3766-1). This study would also serve to provide continued access to the drug to patients who responded adequately in the 52-week randomized, controlled study and need continued access to tofacitinib until the time of marketing application submission. Because the controlled clinical study would be only 52 weeks and some AEs of interest have demonstrated a longer latency period, there would be a need to collect prospective safety data outside of the 52- week randomized, controlled study."~"Delayed"~"Deferral extension requested 3/29/2024. Denied per FDA letter dated 5/14/2024. However, per 5/14/2024 letter,  the final protocol submission, study completion, and final report submission milestones were revised  due to technical challenges. The original final protocol submission milestone has been missed."~10/31/2030 0:00:00~12/23/2025 0:00:00
384400~"CDER"~"NDA"~208271.00~"SALIX PHARMACEUTICALS INC"~"Relistor (Methylnaltrexone Bromide)"~7/19/2016 0:00:00~"Original"~1~"3046-1"~"PMR"~"505 (o)(3)"~3046.00~1~"PMR 3046-1: A post-marketing, observational epidemiologic study comparing oral Relistor (methylnaltrexone bromide) to other treatments of opioid induced constipation in patients with chronic non-cancer pain. The study s primary outcome is a composite of major adverse cardiovascular events (MACE): cardiovascular (CV) death, nonfatal myocardial infarction, and nonfatal stroke. Secondary outcomes include, but are not limited to, CV  death, nonfatal myocardial infarction, and nonfatal stroke separately. Specify concise  case definitions and validation algorithms for the primary and secondary outcomes. Justify the choice of appropriate comparator population(s) and estimated background rate(s) relative to oral Relistor (methylnaltrexone bromide)-exposed patients; clearly  define the primary comparator population for the primary objective. Design the study  around a testable hypothesis to assess, with sufficient sample size and power, MACE risk among oral Relistor (methylnaltrexone bromide) users relative to comparator(s) considering important potential confounders including lifestyle risk factors and over the counter (OTC) medications with potential for cardiovascular effects, with a pre-specified statistical analysis method. For the oral Relistor (methylnaltrexone bromide)-exposed and comparator(s), clearly define the new user clean period, including any exclusion and  inclusion criteria. Ensure an adequate number of patients with at least 12 months of oral  Relistor (methylnaltrexone bromide) exposure at the end of the study."~"Delayed"~"The final report milestone was missed. Changes were made to the protocol that expanded eligibility criteria in order to enhance accrual. FDA acknowledged the revised milestones in a letter dated 04/18/2023 under NDA 21964."~7/31/2025 0:00:00~9/17/2025 0:00:00
384401~"CDER"~"NDA"~208277.00~"CATALYST PHARMACEUTICALS INC"~"Fycompa (Perampanel)"~4/29/2016 0:00:00~"Supplement"~2~"3496-1"~"PMR"~"Pediatric Research Equity Act"~3496.00~1~"PMR 3496-1: Deferred pediatric study under PREA: A prospective, randomized, controlled, double-blind, efficacy and safety study of FYCOMPA (perampanel) for the adjunctive treatment of partial onset seizures in children ages 1 month to < 2 years with a long term safety extension. The primary efficacy endpoint during the controlled phase will examine seizure frequency based upon Video/EEG data. Safety will be evaluated during the controlled phase and long term extension. In the long term extension component, a minimum of 25 patients must be exposed to perampanel for 6 months at or above the dose or doses identified as effective."~"Submitted"~"The final report was submitted to FDA on 01/30/2026."~12/31/2024 0:00:00~12/15/2025 0:00:00
384402~"CDER"~"NDA"~208289.00~"EXELA PHARMA SCIENCES LLC"~"Akovaz (Ephedrine Sulfate)"~4/29/2016 0:00:00~"Original"~1~"3062-2"~"PMR"~"Pediatric Research Equity Act"~3062.00~2~"PMR 3062-2: Conduct a single open-label study to evaluate the safety and pharmacodynamics/  pharmacokinetics of intravenous ephedrine dosed on a mg/kg basis with provisions for up-titration (dose or frequency) as clinically indicated in patents 12-16 years."~"Delayed"~"Original Final Report Due Date: 05/31/2021; Deferral Extension granted per FDA letter dated 09/16/2021, final report due date extended to 09/30/2025. Second Deferral Extension Granted per FDA letter dated 03/05/2026. The study completion milestone was also revised.
"~11/30/2028 0:00:00~6/25/2026 0:00:00
384403~"CDER"~"NDA"~208325.00~"KAI PHARMACEUTICALS INC A WHOLLY OWNED SUB OF AMGEN INC"~"Parsabiv (Etelcalcetide)"~2/7/2017 0:00:00~"Original"~1~"3108-2"~"PMR"~"Pediatric Research Equity Act"~3108.00~2~"PMR 3108-2: Conduct a 26-week Phase 3, randomized, multiple-dose titration safety and PK study evaluating Parsabiv (etelcalcetide) injection with a comparator control arm  in patients aged 2 to 17 years (inclusive) (Part 1), and subjects aged 1 month to 2 years (Part 2), both with secondary hyperparathyroidism receiving hemodialysis."~"Delayed"~"Original Final Report Due Date: 12/31/2023; Per FDA letter dated 04/12/2022, final report due date extended to 12/31/2026. Second Deferral Extension Granted per FDA letter dated 07/03/2025. The study completion milestone was also revised.
"~6/30/2029 0:00:00~1/20/2026 0:00:00
384404~"CDER"~"NDA"~208325.00~"KAI PHARMACEUTICALS INC A WHOLLY OWNED SUB OF AMGEN INC"~"Parsabiv (Etelcalcetide)"~2/7/2017 0:00:00~"Original"~1~"3108-3"~"PMR"~"Pediatric Research Equity Act"~3108.00~3~"PMR 3108-3: Conduct a comparative pharmacokinetic/pharmacodynamics (PK/PD) modeling study evaluating Parsabiv (etelcalcetide) injection in adult and pediatric subjects with secondary hyperparathyroidism receiving maintenance hemodialysis."~"Delayed"~"Original Final Report Due Date: 12/31/2023; Per FDA letter dated 04/12/2022, final report due date extended to 12/31/2026.  Second Deferral Extension Granted per FDA letter dated 07/03/2025. The study completion milestone was also revised."~12/31/2029 0:00:00~1/20/2026 0:00:00
384405~"CDER"~"NDA"~208401.00~"AZURITY PHARMACEUTICALS INC"~"Qbrelis (Lisinopril) "~7/29/2016 0:00:00~"Original"~1~"3099-1"~"PMR"~"Pediatric Research Equity Act"~3099.00~1~"PMR 3099-1: An efficacy, safety and dose-finding study of Qbrelis in hypertensive pediatric patients two years to less than six years of age"~"Delayed"~"The study has not been initiated and the final report submission has been missed."~12/31/2020 0:00:00~9/24/2025 0:00:00
384406~"CDER"~"NDA"~21450.00~"AMNEAL PHARMACEUTICALS LLC"~"Zolmitriptan"~9/30/2003 0:00:00~"Supplement"~8~"2921-3"~"PMR"~"Pediatric Research Equity Act"~2921.00~3~"PMR 2921-3: Conduct a randomized, adequately controlled safety and efficacy study of Zomig (zolmitriptan) Nasal Spray in pediatric patients with migraine ages 6 years to 11 years old followed by an evaluation of long-term safety in an openlabel extension of that study. The long-term safety evaluation must provide a descriptive analysis of safety data in at least 50 pediatric patients exposed for at least 6 months, treating on average at least one migraine attack per month, at doses evaluated in the efficacy portion of the study."~"Delayed"~"A Not Fulfilled letter was issued on 11/09/2022. Original Final Report Due Date: 01/31/2022. Per FDA letter dated 11/09/2022, final report due date extended to 11/30/2023. Second Deferral Extension Granted per FDA letter dated 10/06/2023. The final protocol submission and study completion milestones were also revised due to continuing interaction between the applicant and the FDA and the need for additional time to complete the required study."~10/31/2028 0:00:00~11/26/2025 0:00:00
384407~"CDER"~"NDA"~21451.00~"DENTSPLY PHARMACEUTICAL"~"Oraqix (Lidocaine and Prilocaine)"~12/19/2003 0:00:00~"Original"~1~"1440-2"~"PMR"~"Pediatric Research Equity Act"~1440.00~2~"PMR 1440-2: We are deferring pediatric studies for ages 6-17 years for this application."~"Delayed"~"A not fulfilled letter was issued on October 8, 2022."~12/19/2008 0:00:00~2/12/2018 0:00:00
384408~"CDER"~"NDA"~21489.00~"BRACCO DIAGNOSTICS INC"~"ProHance Multipack (Gadoteridol)"~10/9/2003 0:00:00~"Supplement"~6~"3626-4"~"PMR"~"505 (o)(3)"~3626.00~4~"PMR 3626-4: A prospective longitudinal cohort trial with one or more matched control group(s) to evaluate the effects of repetitive GBCA administration on a comprehensive battery of neurobehavioral testing over the course of at least five administrations. The trial should be sufficiently powered to exclude a pre-specified magnitude of decline. As a secondary objective, trial patients should also have the option of providing blood and urine samples at the time of reimaging, so that normative estimates of gadolinium concentration across an extended range of post-administration timepoints may be documented."~"Ongoing"~~4/30/2029 0:00:00~12/19/2024 0:00:00
384409~"CDER"~"NDA"~21606.00~"ABBVIE INC"~"Zemplar (Paricalcitol)"~5/26/2005 0:00:00~"Supplement"~4~"2144-1"~"PMR"~"Pediatric Research Equity Act"~2144.00~1~"PMR 2144-1: Deferred pediatric study under PREA to determine the safety of Zemplar  (paricalcitol) for the treatment of secondary hyperparathyroidism associated with chronic kidney disease (CKD) Stage 5 in pediatric patients ages 0 to 9 years receiving peritoneal dialysis or hemodialysis."~"Ongoing"~"Original Final Report Due Date: 05/30/2020. Per FDA letter dated 05/16/2020, Final Report due date extended to 12/31/2023. Second Deferral Extension Granted per FDA letter dated 10/16/2023."~12/31/2025 0:00:00~7/22/2025 0:00:00
384410~"CDER"~"NDA"~21610.00~"ENDO OPERATIONS LTD"~"Opana ER (Oxymorphone Hydrochloride)"~6/22/2006 0:00:00~"Original"~1~"3033-1"~"PMR"~"505 (o)(3)"~3033.00~1~"PMR 3033-1: A prospective, observational study designed to quantify the serious risks of misuse, abuse, and addiction associated with long-term use of opioid analgesics  for management of chronic pain among patients prescribed ER/LA opioid analgesics. This study must address at a minimum the following specific objectives: a. Estimate the incidence of misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of  psychiatric illness) on the risk of misuse, abuse, and addiction.  b. Evaluate and quantify other risk factors for misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors, psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships."~"Fulfilled"~~3/31/2020 0:00:00~2/18/2025 0:00:00
384411~"CDER"~"NDA"~21610.00~"ENDO OPERATIONS LTD"~"Opana ER (Oxymorphone Hydrochloride)"~6/22/2006 0:00:00~"Original"~1~"3033-2"~"PMR"~"505 (o)(3)"~3033.00~2~"PMR 3033-2: An observational study designed to measure the incidence and predictors of opioid overdose and death (OOD), as well as opioid abuse/addiction, using patient  health records, insurance claims, and death records. This study must address at a minimum the following specific objectives: a. Estimate the incidence of abuse/addiction, overdose, and death associated with long-term use of opioid analgesics for chronic pain. Stratify overdose by  intentionality wherever possible. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other  clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of psychiatric illness) on the risk of  abuse/addiction, overdose, and death. b. Evaluate and quantify other risk factors for abuse/addiction, overdose, and  death associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors,  psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships. Stratify overdose by intentionality wherever possible."~"Fulfilled"~~9/30/2019 0:00:00~2/18/2025 0:00:00
384412~"CDER"~"NDA"~21610.00~"ENDO OPERATIONS LTD"~"Opana ER (Oxymorphone Hydrochloride)"~6/22/2006 0:00:00~"Original"~1~"3033-6"~"PMR"~"505 (o)(3)"~3033.00~6~"PMR 3033-6: An observational study to develop and validate an algorithm using coded medical terminologies and other electronic healthcare data to identify opioid-related overdose and death."~"Fulfilled"~~12/31/2016 0:00:00~2/18/2025 0:00:00
384413~"CDER"~"NDA"~21610.00~"ENDO OPERATIONS LTD"~"Opana ER (Oxymorphone Hydrochloride)"~6/22/2006 0:00:00~"Original"~1~"3033-11"~"PMR"~"505 (o)(3)"~3033.00~11~"PMR 3033-11: Conduct a clinical trial to estimate the serious risk for the development of hyperalgesia following the long-term use of high-dose ER/LA opioid analgesics  for at least one year to treat chronic pain. Include an assessment of risk relative to  efficacy."~"Delayed"~"The Trial Completion and Final Report milestones were missed because of continued discussions needed to reach agreement with the Agency on the Final Protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 8/26/2025."~8/31/2019 0:00:00~2/18/2025 0:00:00
384414~"CDER"~"NDA"~21626.00~"HEYL CHEMISCH PHARMAZEUTISHE FABRIK"~"Radiogardase (Prussian Blue)"~10/2/2003 0:00:00~"Original"~1~"1015-1"~"PMC"~"506B PMC"~1015.00~1~"PMC 1015-1: Longitudinal studies involving follow up on case report forms and placement of data into a database for periodic analyses to determine length of treatment, safety profile, and other factors related to drug effectiveness.  a. Protocol submission: Within 6 months of the date of this letter  b. Study start (i.e. the date the database will be ready to accept patient data, should it be necessary): Within 6 months of agreement to the protocol  We also remind you of your agreement to provide annual reports of ongoing studies beginning one year from study initiation."~"Pending"~~~12/5/2025 0:00:00
384415~"CDER"~"BLA"~125151.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Elaprase (Idursulfase) "~7/24/2006 0:00:00~"Supplement"~184~"2792-2"~"PMR"~"Accelerated Approval"~2792.00~2~"PMR 2792-2: To evaluate a prophylactic immune tolerance regimen in a cohort of Hunter syndrome patients treated with Elaprase (idursulfase) who are at high risk of developing persistent neutralizing antibody that could result in diminished clinical  benefit. This immune tolerance regimen will be implemented before or concomitant with onset of therapy. The trial will monitor antibody status (binding,  neutralizing, and IgE), urinary GAG, and hypersensitivity reactions in patients at  regular intervals. Additionally, the trial will evaluate the relationship between development of immune tolerance and genetic mutations, endogenous enzyme  activity level, and clinical outcome. Completion of this PMR is pending the outcome of an Advisory Committee Meeting and completion of PMR 3."~"Released"~"Per FDA letter dated 08/21/2025, this PMR/PMC has been released."~9/30/2022 0:00:00~9/18/2025 0:00:00
384416~"CDER"~"BLA"~125160.00~"UCB, Inc."~"Cimzia (Certolizumab Pegol) "~4/22/2008 0:00:00~"Supplement"~80~"2563-2"~"PMR"~"505 (o)(3)"~2563.00~2~"PMR 2563-2: An observational study registry in adult patients with moderately to severely active RA that would assess the longer term risks of serious infections, malignancies that have been reported with TNF  blocker therapy, as well as the longer term risk for cardiovascular and thromboembolic events, including congestive heart failure, hypertension, TIA, stroke, tachyarrhythmia, atrial fibrillation, venous thrombosis and phlebitis."~"Submitted"~~2/28/2017 0:00:00~6/18/2026 0:00:00
384417~"CDER"~"BLA"~125160.00~"UCB, Inc."~"Cimzia (Certolizumab Pegol) "~4/22/2008 0:00:00~"Supplement"~283~"3408-1"~"PMR"~"Pediatric Research Equity Act"~3408.00~1~"PMR 3408-1: Conduct a Pharmacokinetics (PK), Safety, and Efficacy Study in pediatric subjects 6 to less than 18 years of age with moderate to severe psoriasis (with a duration of exposure to certolizumab pegol of at least one year)."~"Delayed"~"Original Final Report Due Date: 12/31/2025; Deferral Extension granted per FDA letter dated 09/24/2025. The final protocol and study completion milestones were also revised."~12/31/2026 0:00:00~6/18/2026 0:00:00
384418~"CDER"~"BLA"~125160.00~"UCB, Inc."~"Cimzia (Certolizumab Pegol) "~4/22/2008 0:00:00~"Supplement"~283~"3408-2"~"PMR"~"505 (o)(3)"~3408.00~2~"PMR 3408-2: Utilize the validated immunogenicity assays developed under PMC 3408-5 and PMC 3408-6 to analyze the immunogenicity profile of certolizumab pegol using banked patient samples from Phase 3 trials CIMPASI-1, CIMPASI-2, and CIMPACT. Evaluate the impact of immunogenicity on pharmacokinetics, efficacy, and safety in subjects with psoriasis based on the immunogenicity data generated with the newly validated assays."~"Submitted"~~9/30/2019 0:00:00~6/18/2026 0:00:00
384419~"CDER"~"BLA"~125160.00~"UCB, Inc."~"Cimzia (Certolizumab Pegol) "~4/22/2008 0:00:00~"Supplement"~283~"3408-3"~"PMR"~"505 (o)(3)"~3408.00~3~"PMR 3408-3: A prospective, registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to certolizumab pegol during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including neonatal deaths, infections in the first 6 months of life, and effects on postnatal growth and development, will be assessed through at least the first year of life. You may expand a current prospective registry to include women who are exposed to certolizumab pegol for the treatment of plaque psoriasis."~"Submitted"~~1/31/2030 0:00:00~6/18/2026 0:00:00
384420~"CDER"~"BLA"~125160.00~"UCB, Inc."~"Cimzia (Certolizumab Pegol) "~4/22/2008 0:00:00~"Supplement"~283~"3408-4"~"PMR"~"505 (o)(3)"~3408.00~4~"PMR 3408-4: Conduct a retrospective cohort study using claims or electronic medical record data or a case control study to assess major congenital malformations, spontaneous abortions, stillbirths, small for gestational age, neonatal deaths, and infant infections in women exposed to certolizumab pegol during pregnancy compared to an unexposed control population."~"Ongoing"~~9/30/2027 0:00:00~6/18/2026 0:00:00
384421~"CDER"~"BLA"~125166.00~"Alexion Pharmaceuticals, Inc."~"Soliris (Eculizumab) "~3/16/2007 0:00:00~"Supplement"~443~"4449-1"~"PMR"~"505 (o)(3)"~4449.00~1~"PMR 4449-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Ultomiris (ravulizumab-cwvz) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~7/31/2034 0:00:00~5/15/2018 0:00:00
384422~"CDER"~"BLA"~125261.00~"Janssen Biotech, Inc."~"Stelara (Ustekinumab)"~9/25/2009 0:00:00~"Supplement"~152~"3736-1"~"PMR"~"505 (o)(3)"~3736.00~1~"PMR 3736-1: A long-term, postmarketing, observational study to assess the long-term safety of STELARA (ustekinumab) versus other therapies used in the treatment of adults with moderate to severe ulcerative colitis. The study s primary outcome is malignancy. Secondary outcomes include, but are not limited to, opportunistic infections (e.g., tuberculosis [TB]). Specify concise case definitions and provide outcome validation for both primary and secondary outcomes. Describe and justify the choice of appropriate comparator population(s) and estimated background rate(s) relative to ustekinumab-exposed patients; clearly define the primary comparator population for the primary objective. Design the study around a testable hypothesis to assess, with sufficient sample size and power, a clinically meaningful increase in malignancy risk above the comparator background rate, with a pre-specified statistical analysis method. For the ustekinumab-exposed and comparator(s), the study drug initiation period should be clearly defined, including any exclusion and inclusion criteria. Ensure adequate number of patients with at least 18 months of ustekinumab exposure at the end of the study. Follow for a period of at least 7 years. The ongoing observational study in patients with Crohn s disease with the same objectives, may be amended to also enroll patients with ulcerative colitis.
"~"Delayed"~"The original final protocol milestone was missed. The Final protocol was received and acknowledged per FDA letter dated April 21, 2021."~8/31/2030 0:00:00~11/20/2025 0:00:00
384423~"CDER"~"BLA"~125261.00~"Janssen Biotech, Inc."~"Stelara (Ustekinumab)"~9/25/2009 0:00:00~"Supplement"~152~"3736-2"~"PMC"~"506B PMC"~3736.00~2~"PMC 3736-2: A one-year, randomized, controlled, blinded trial to evaluate the safety, efficacy, and pharmacokinetics of Stelara (ustekinumab) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis."~"Submitted"~~9/30/2025 0:00:00~11/20/2025 0:00:00
384424~"CDER"~"BLA"~125261.00~"Janssen Biotech, Inc."~"Stelara (Ustekinumab)"~9/25/2009 0:00:00~"Supplement"~152~"3736-3"~"PMC"~"506B PMC"~3736.00~3~"PMC 3736-3: A multi-center, open-label extension study to evaluate the long-term safety of Stelara (ustekinumab) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis who participated in PMC 3736-2."~"Delayed"~"The applicant requested revised milestones due to delays in meeting the protocol-defined criteria for end-of-study participation. The study completion and final report submission milestons were revised and acknowledged in a letter dated 05/12/2026."~9/30/2026 0:00:00~11/20/2025 0:00:00
384425~"CDER"~"NDA"~204114.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mekinist (trametinib)"~5/29/2013 0:00:00~"Supplement"~24~"4297-1"~"PMR"~"Accelerated Approval"~4297.00~1~"PMR 4297-1: Conduct a clinical trial(s) in at least 80 patients with solid tumors with a BRAF V600E mutation to verify and describe the clinical benefit of trametinib in combination with dabrafenib, through more precise estimation of the overall response rate and mature response duration. Include patients with unresectable or metastatic solid tumors with a BRAF V600E mutation from the ongoing trial and from a prospectively conducted trial (which will exclude patients with melanoma, non-small cell lung cancer, anaplastic thyroid cancer, biliary tract cancer, gliomas and colorectal cancer). Follow all patients for at least 6 months from the onset of response to characterize the response rate and duration. 
"~"Ongoing"~"The study has been initiated."~10/31/2028 0:00:00~7/23/2025 0:00:00
384426~"CDER"~"NDA"~204114.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mekinist (trametinib)"~5/29/2013 0:00:00~"Supplement"~24~"4297-4"~"PMC"~"506B PMC"~4297.00~4~"PMC 4297-4: Commitment to establish, through the use of clinical trial data, an in-vitro diagnostic device that is essential to the safe and effective use of trametinib and dabrafenib for patients with BRAF V600E mutations in solid tumor specimens, excluding colorectal cancer."~"Delayed"~"The final report milestone was missed due to challenges of sourcing the samples because of the low prevalence of the mutations across the required tumor types."~7/31/2024 0:00:00~7/23/2025 0:00:00
384427~"CDER"~"NDA"~204114.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mekinist (trametinib)"~5/29/2013 0:00:00~"Supplement"~25~"4423-1"~"PMR"~"505 (o)(3)"~4423.00~1~"PMR 4423-1: Conduct comprehensive safety analyses from ongoing trials to further assess the serious risks of trametinib in combination with dabrafenib, including but not limited to new primary malignancies (cutaneous and noncutaneous), cardiomyopathy, and ocular toxicities, in pediatric patients with BRAFV600E mutant low grade glioma over a sufficient period of follow-up time to further characterize these risks. The report should include appropriate monitoring and risk mitigation strategies."~"Ongoing"~~5/31/2027 0:00:00~7/23/2025 0:00:00
384428~"CDER"~"NDA"~204114.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mekinist (trametinib)"~5/29/2013 0:00:00~"Supplement"~25~"4423-2"~"PMR"~"505 (o)(3)"~4423.00~2~"PMR 4423-2: Conduct an integrated safety analyses from clinical studies that further characterize the potential serious risk of long-term adverse effects including but not limited to growth plate abnormalities of trametinib in combination with dabrafenib on growth and development in a sufficient number of pediatric patients. Monitor patients for growth and development using age-appropriate screening tools. Include evaluations of growth as measured by height, weight, height velocity and height standard deviation scores, age at menarche if applicable (females) and Tanner stage. Monitor patients until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first."~"Ongoing"~~5/31/2027 0:00:00~7/23/2025 0:00:00
384429~"CDER"~"NDA"~204114.00~"NOVARTIS PHARMACEUTICALS CORP"~"Mekinist (trametinib)"~5/29/2013 0:00:00~"Supplement"~25~"4423-4"~"PMC"~"506B PMC"~4423.00~4~"PMC 4423-4: Commitment to support the availability of an in vitro diagnostic device, through an appropriate analytical and clinical validation study using clinical trial data that demonstrates the device is essential to the safe and effective use of dabrafenib in combination with trametinib (D+T) for the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy."~"Delayed"~"The final report milestone was missed due to challenges with sourcing of samples due to low prevalence. The applicant has proposed a revised final report milestone date.

"~2/28/2025 0:00:00~7/23/2025 0:00:00
384430~"CDER"~"NDA"~204300.00~"EXELA PHARMA SCIENCES LLC"~"Vazculep (Phenylephrine Hydrochloride)"~6/27/2014 0:00:00~"Original"~1~"2168-1"~"PMR"~"Pediatric Research Equity Act"~2168.00~1~"PMR 2168-1: A study in the greater than or equal to 12- to less than 17-year old age group to  evaluate the pharmacokinetics, efficacy, and safety of different doses of phenylephrine hydrochloride injection in patients undergoing general anesthesia and/or neuroaxial anesthesia."~"Ongoing"~"Exela is currently in the process of selecting CROs to conduct the study."~2/28/2019 0:00:00~8/22/2024 0:00:00
384431~"CDER"~"NDA"~204326.00~"NEOS THERAPEUTICS"~"Adzenys XR-ODT (Amphetamine)"~1/27/2016 0:00:00~"Original"~1~"3029-2"~"PMR"~"Pediatric Research Equity Act"~3029.00~2~"PMR 3029-2: A randomized, double-blind, placebo-controlled, flexible-dose titration study of ADZENYS XR-ODT (amphetamine extended-release orally disintegrating tablets), in children ages 4 to 5 years diagnosed with ADHD."~"Released"~"Per FDA letter dated 11/18/2025, this PMR/PMC has been released."~6/30/2020 0:00:00~3/27/2026 0:00:00
384432~"CDER"~"NDA"~204326.00~"NEOS THERAPEUTICS"~"Adzenys XR-ODT (Amphetamine)"~1/27/2016 0:00:00~"Original"~1~"3029-3"~"PMR"~"Pediatric Research Equity Act"~3029.00~3~"PMR 3029-3: A one year Pediatric Open-Label Safety Study of patients age 4 to 5 years (at the time of entry into PMR 3029-1 or PMR 3029-2, or at the time of enrollment if directly enrolled into PMR 3029-3) diagnosed with ADHD treated with ADZENYS XR-ODT (amphetamine extended-release orally disintegrating tablets)."~"Released"~"Per FDA letter dated 11/18/2025, this PMR/PMC has been released."~11/30/2020 0:00:00~3/27/2026 0:00:00
384433~"CDER"~"NDA"~204370.00~"ABBVIE INC"~"Vraylar (Cariprazine)"~9/17/2015 0:00:00~"Original"~1~"2947-6"~"PMR"~"Pediatric Research Equity Act"~2947.00~6~"PMR 2947-6: Deferred long-term, open-label safety study in pediatric patients with schizophrenia (ages 13 to 17) and bipolar I disorder, recent manic episodes (ages 10 to 17)."~"Fulfilled"~"Per FDA letter dated 12/18/2025, this PMR/PMC has been fulfilled."~6/30/2025 0:00:00~11/12/2025 0:00:00
384434~"CDER"~"NDA"~204370.00~"ABBVIE INC"~"Vraylar (Cariprazine)"~9/17/2015 0:00:00~"Original"~2~"2947-6"~"PMR"~"Pediatric Research Equity Act"~2947.00~6~"PMR 2947-6: Deferred long-term, open-label safety study in pediatric patients with schizophrenia (ages 13 to 17) and bipolar I disorder, recent manic episodes (ages 10 to 17)."~"Fulfilled"~"Per FDA letter dated 12/18/2025, this PMR/PMC has been fulfilled."~6/30/2025 0:00:00~11/12/2025 0:00:00
384435~"CDER"~"NDA"~204370.00~"ABBVIE INC"~"Vraylar (Cariprazine)"~9/17/2015 0:00:00~"Supplement"~6~"3619-1"~"PMR"~"Pediatric Research Equity Act"~3619.00~1~"PMR 3619-1: Deferred pediatric study under PREA (ages 10 to 17) with a diagnosis of acute bipolar depression associated with bipolar I disorder. A study of the efficacy and safety of cariprazine in the relevant pediatric population."~"Delayed"~"Study is delayed due to challenges in site selection, site activation and enrollment. Original Final Report Due Date: 07/31/2024. Per FDA letter dated 12/11/2023, final report due date extended to 01/31/2026. Second Deferral Extension Granted per FDA letter dated 05/21/2025. The study completion milestone was also revised."~7/31/2027 0:00:00~11/12/2025 0:00:00
384436~"CDER"~"NDA"~204441.00~"OTSUKA PHARMACEUTICAL CO LTD"~"JYNARQUE (tolvaptan)"~4/23/2018 0:00:00~"Original"~1~"3384-1"~"PMR"~"505 (o)(3)"~3384.00~1~"PMR 3384-1: Conduct a prospective cohort study of patients enrolled in the Jynarque (tolvaptan) Risk Evaluation and Mitigation Strategies (REMS) registry, with the primary objective of determining the incidence rate of severe (fatal and potentially fatal) drug induced liver injury (DILI). The incidence rate should be compared to that observed in the development program (in TEMPO and REPRISE trials). Incidence rates should be stratified by important risk factors for DILI which at a minimum should include: age, gender, race, alcohol use, cumulative dose of tolvaptan and duration of tolvaptan use."~"Fulfilled"~~4/30/2025 0:00:00~7/7/2026 0:00:00
384437~"CDER"~"NDA"~208434.00~"HOFFMANN LA ROCHE INC"~"Alecensa (Alectinib)"~12/11/2015 0:00:00~"Supplement"~3~"3296-1"~"PMC"~"506B PMC"~3296.00~1~"PMC 3296-1: Submit the clinical report and datasets for the final analysis of overall survival and mature data characterizing the duration of response for Study BO28984,  Randomized, Multicenter, Phase III, Open-Label Study of Alectinib versus Crizotinib in Treatment-Na ve Anaplastic Lymphoma Kinase-Positive Advanced Non-Small Cell Lung Cancer  [ALEX] , to update the label with mature duration of response and overall survival data."~"Fulfilled"~~6/30/2020 0:00:00~2/5/2026 0:00:00
384438~"CDER"~"NDA"~208434.00~"HOFFMANN LA ROCHE INC"~"Alecensa (Alectinib)"~12/11/2015 0:00:00~"Supplement"~15~"4630-1"~"PMR"~"505 (o)(3)"~4630.00~1~"PMR 4630-1: Conduct a carcinogenicity study in mice to evaluate the potential serious risk for carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Ongoing"~~4/30/2027 0:00:00~2/5/2026 0:00:00
384439~"CDER"~"NDA"~208434.00~"HOFFMANN LA ROCHE INC"~"Alecensa (Alectinib)"~12/11/2015 0:00:00~"Supplement"~15~"4630-2"~"PMR"~"505 (o)(3)"~4630.00~2~"PMR 4630-2: Conduct a carcinogenicity study in rats to evaluate the potential serious risk of carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Delayed"~"The final protocol milestone was missed because additional
nonclinical work was needed prior to finalizing the study
design."~12/31/2028 0:00:00~2/5/2026 0:00:00
384440~"CDER"~"NDA"~208434.00~"HOFFMANN LA ROCHE INC"~"Alecensa (Alectinib)"~12/11/2015 0:00:00~"Supplement"~15~"4630-3"~"PMR"~"505 (o)(3)"~4630.00~3~"PMR 4630-3: Conduct a GLP fertility study in female rats to evaluate the potential risk of impairment of fertility."~"Ongoing"~~9/30/2026 0:00:00~2/5/2026 0:00:00
384441~"CDER"~"NDA"~208434.00~"HOFFMANN LA ROCHE INC"~"Alecensa (Alectinib)"~12/11/2015 0:00:00~"Supplement"~15~"4630-4"~"PMR"~"505 (o)(3)"~4630.00~4~"PMR 4630-4: Conduct a GLP fertility study in male rats to evaluate the potential risk of impairment of fertility."~"Ongoing"~~9/30/2026 0:00:00~2/5/2026 0:00:00
384442~"CDER"~"NDA"~208434.00~"HOFFMANN LA ROCHE INC"~"Alecensa (Alectinib)"~12/11/2015 0:00:00~"Supplement"~15~"4630-5"~"PMC"~"506B PMC"~4630.00~5~"PMC 4630-5: Complete clinical trial ALINA, titled,  A Phase III, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of Adjuvant
Alectinib Versus Adjuvant Platinum-Based Chemotherapy in Patients with Completely Resected Stage IB (Tumors = 4 cm) to Stage IIIA Anaplastic Lymphoma Kinase-Positive Non-Small Cell Lung Cancer , to provide the final updated descriptive disease-free survival analyses based on the prespecified final number of events or when all patients have been followed for 5 years, and overall survival analyses when all patients have been followed for 5 years."~"Ongoing"~~9/30/2027 0:00:00~2/5/2026 0:00:00
384443~"CDER"~"NDA"~208464.00~"GILEAD SCIENCES INC"~"Vemlidy (Tenofovir Alafenamide)"~11/10/2016 0:00:00~"Original"~1~"3130-2"~"PMR"~"Pediatric Research Equity Act"~3130.00~2~"PMR 3130-2: Conduct the deferred pediatric study to access the pharmacokinetics, safety/tolerability, and antiviral activity of tenofovir alafenamide in HBV infected subjects 2 to less than 12 years of age, followed by a rollover to a long-term, open-label, extension to assess longer-term pediatric safety and antiviral activity."~"Delayed"~"Deferral Extension granted & Acknowledged revised Final Report PMR milestone letter was issued on 01/17/2024."~3/31/2027 0:00:00~12/29/2025 0:00:00
384444~"CDER"~"BLA"~208471.00~"Sanofi-Aventis U.S. LLC"~"Adlyxin (Lixisenatide) "~7/27/2016 0:00:00~"Original"~1~"4736-1"~"PMR"~"505 (o)(3)"~4736.00~1~"PMR 4736-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of lixisenatide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Pending"~~11/30/2026 0:00:00~9/24/2025 0:00:00
384445~"CDER"~"NDA"~208551.00~"ROCKWELL MEDICAL INC"~"Triferic (ferric pyrophosphate citrate)"~4/25/2016 0:00:00~"Original"~1~"3067-2"~"PMR"~"Pediatric Research Equity Act"~3067.00~2~"PMR 3067-2: Efficacy and safety trial of Triferic via hemodialysate in pediatric patients aged less than 18 years with hemodialysis-dependent chronic kidney disease."~"Ongoing"~"10 patients have been enrolled into the trial."~12/31/2022 0:00:00~7/1/2022 0:00:00
384446~"CDER"~"NDA"~208558.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Lynparza (Olaparib)"~8/17/2017 0:00:00~"Supplement"~6~"3525-1"~"PMC"~"506B PMC"~3525.00~1~"PMC 3525-1: Submit the final overall survival (OS) analysis with datasets from clinical trial D0818C00001 (SOLO-1), the ongoing phase 3, randomized, double-blind, placebo-controlled, multicenter trial of olaparib maintenance monotherapy in patients with BRCA mutated advanced (FIGO Stage III-IV) ovarian cancer following first-line platinum-based chemotherapy."~"Ongoing"~~11/30/2027 0:00:00~2/6/2026 0:00:00
384447~"CDER"~"NDA"~208558.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Lynparza (Olaparib)"~8/17/2017 0:00:00~"Supplement"~14~"3826-1"~"PMC"~"506B PMC"~3826.00~1~"PMC 3826-1: Submit the final report from a study evaluating the response (overall response rate in patients with measurable disease, prostate specific antigen response (measurable and non-measurable disease), CTC conversion (measurable and non-measurable disease)) and duration of responses to olaparib in patients with metastatic castration-resistant prostate cancer who have progressed on a new hormonal agent and had somatic or germline mutations in homologous recombination repair (HRR) genes that were present in five or fewer patients among the HRR genes evaluated in Cohort B of the PROfound trial. HRR mutation should be determined based on an FDA-approved assay and the study will evaluate at least five patients per HRR gene. Provide annual updates on patient enrollment and responses in the interim reports. Annual updates and the final report should include information regarding the assay used to identify each HRR mutation."~"Ongoing"~~12/31/2028 0:00:00~2/6/2026 0:00:00
384448~"CDER"~"NDA"~208558.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Lynparza (Olaparib)"~8/17/2017 0:00:00~"Supplement"~23~"4248-1"~"PMC"~"506B PMC"~4248.00~1~"PMC 4248-1: Conduct analyses of clinical trial data to characterize the safety and pharmacokinetics of olaparib across a diverse population. To support a comparative assessment across all U.S. race and ethnic populations, ensure that racial and ethnic minorities are sufficiently represented in the analysis. Include a tabular summary of the available pharmacokinetic data by each racial and ethnic group. Provide the specific racial ethnic group as well as the geographic location of each patient."~"Fulfilled"~~7/31/2024 0:00:00~2/6/2026 0:00:00
384449~"CDER"~"NDA"~208558.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Lynparza (Olaparib)"~8/17/2017 0:00:00~"Supplement"~25~"4459-1"~"PMC"~"506B PMC"~4459.00~1~"PMC 4459-1: Conduct an analytical and clinical validation study using clinical trial data, adequate to support the availability of an in vitro diagnostic device using tissue samples that is essential to the safe and effective use of olaparib plus abiraterone for patients diagnosed with metastatic castration-resistant prostate cancer (mCRPC), whose tumors harbor BRCA1 or BRCA2 mutations."~"Pending"~~6/30/2025 0:00:00~2/6/2026 0:00:00
384450~"CDER"~"NDA"~208558.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Lynparza (Olaparib)"~8/17/2017 0:00:00~"Supplement"~25~"4459-2"~"PMC"~"506B PMC"~4459.00~2~"PMC 4459-2: Conduct an analytical and clinical validation study using clinical trial data, adequate to support the availability of an in vitro diagnostic device using ctDNA samples from plasma that is essential to the safe and effective use of olaparib plus abiraterone for patients diagnosed with mCRPC, whose ctDNA samples harbor BRCA1 or BRCA2 mutations."~"Pending"~~6/30/2025 0:00:00~2/6/2026 0:00:00
384451~"CDER"~"NDA"~21626.00~"HEYL CHEMISCH PHARMAZEUTISHE FABRIK"~"Radiogardase (Prussian Blue)"~10/2/2003 0:00:00~"Original"~1~"1015-3"~"PMC"~"506B PMC"~1015.00~3~"PMC 1015-3: Studies to determine dosing for neonates to 2 years of age (based on human extrapolation and/or animal models).  i. Protocol submission: Within 6 months of the date of this letter ii. Study start: Within 6 months of agreement to the protocol iii. Final study report submission:  Within 12 months of initiation of the study"~"Delayed"~"The PMC protocol submission milestone was missed."~~12/5/2025 0:00:00
384452~"CDER"~"NDA"~21636.00~"SALIX PHARMACEUTICALS INC"~"Zegerid (Omeprazole and Sodium Bicarbonate) "~6/15/2004 0:00:00~"Original"~1~"1584-1"~"PMR"~"Pediatric Research Equity Act"~1584.00~1~"PMR 1584-1: Deferred pediatric study under PREA for the treatment or prevention of upper gastrointestinal bleeding in critically ill pediatric patients ages 2 to 11 years old and 12 to 16 years old."~"Delayed"~"Sponsor has been issued a PREA noncompliance letter on 7/17/13. The study has not been initiated; no subjects are enrolled. The final report milestone was missed."~12/26/2008 0:00:00~8/5/2025 0:00:00
384453~"CDER"~"NDA"~21636.00~"SALIX PHARMACEUTICALS INC"~"Zegerid (Omeprazole and Sodium Bicarbonate) "~6/15/2004 0:00:00~"Original"~1~"810-1"~"PMR"~"Pediatric Research Equity Act"~810.00~1~"PMR 810-1: Single and multiple-dose pharmacokinetics (PK), pharmacodynamics (PD) and safety study in pediatric patients aged 2 to 11 years."~"Delayed"~"Sponsor has been issued a PREA noncompliance letter on 7/17/13. The study has not been initiated; no subjects are enrolled. The final report milestone was missed."~7/15/2007 0:00:00~8/5/2025 0:00:00
384454~"CDER"~"NDA"~21636.00~"SALIX PHARMACEUTICALS INC"~"Zegerid (Omeprazole and Sodium Bicarbonate) "~6/15/2004 0:00:00~"Original"~1~"810-2"~"PMR"~"Pediatric Research Equity Act"~810.00~2~"PMR 810-2: Single and multiple-dose pharmacokinetics (PK), pharmacodynamics (PD) and safety study in pediatric patients aged 12 to 16 years."~"Delayed"~"Sponsor has been issued a PREA noncompliance letter on 7/17/13. The study has not been initiated; no subjects are enrolled. The final report milestone was missed."~7/15/2007 0:00:00~8/5/2025 0:00:00
384455~"CDER"~"NDA"~21704.00~"CHATTEM INC DBA SANOFI CONSUMER HEALTHCARE"~"Allegra D Allergy and Congestion (Fexofenadine Hydrochloride and Pseudoephedrine Hydrochloride)"~10/19/2004 0:00:00~"Supplement"~17~"2916-2"~"PMR"~"Pediatric Research Equity Act"~2916.00~2~"PMR 2916-2: Conduct a study in children 6 to less than 12 years of age to evaluate the efficacy and safety of the pseudoephedrine component, in your combination product, for  temporary relief of nasal congestion due to the common cold."~"Submitted"~"According to the PMR, the final protocol, study completion, and final report submission are due by October 2021, January 2023, and June 2023 respectively. However, on October 29,2021, the applicant submitted a study report for a 2012 study, A Multicenter, Randomized, Placebo-Controlled Study of Pseudoephedrine for the Temporary Relief of Nasal Congestion in Children with the Common Cold (Study PRGO-PSE-09001), to fulfill PMR 2916-2. FDA's assessment is underway."~6/30/2023 0:00:00~12/17/2025 0:00:00
384456~"CDER"~"NDA"~21704.00~"CHATTEM INC DBA SANOFI CONSUMER HEALTHCARE"~"Allegra D Allergy and Congestion (Fexofenadine Hydrochloride and Pseudoephedrine Hydrochloride)"~10/19/2004 0:00:00~"Supplement"~17~"2916-3"~"PMR"~"Pediatric Research Equity Act"~2916.00~3~"PMR 2916-3: If the studies conducted in children 6 to less than 12 years of age demonstrate safety and efficacy for the intended claim, conduct a study to determine an appropriate dose and dosing interval for use in children, 2 to less than 6 years of  age, who may benefit from the pseudoephedrine component of the drug product (i.e., not in otherwise healthy pediatric volunteers) to relieve nasal congestion due to the common cold."~"Submitted"~"The final report was submitted to FDA on 7/28/2023."~6/30/2024 0:00:00~12/17/2025 0:00:00
384457~"CDER"~"NDA"~21704.00~"CHATTEM INC DBA SANOFI CONSUMER HEALTHCARE"~"Allegra D Allergy and Congestion (Fexofenadine Hydrochloride and Pseudoephedrine Hydrochloride)"~10/19/2004 0:00:00~"Supplement"~17~"2916-4"~"PMR"~"Pediatric Research Equity Act"~2916.00~4~"PMR 2916-4: If the studies conducted in children 6 to less than 12 years of age demonstrate safety and efficacy for the intended claim, conduct a study to evaluate the safety of the pseudoephedrine component, in your combination product, for temporary relief of nasal congestion due to the common cold in children 2 to less than 6  years of age. Efficacy of fexofenadine and pseudoephedrine for the age group 2 to less than 6 years may be extrapolated from data in the older age group."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed. Will begin in the future."~6/30/2026 0:00:00~12/17/2025 0:00:00
384458~"CDER"~"NDA"~21706.00~"SANTARUS INC"~"Zegerid (Omeprazole and Sodium Bicarbonate) "~12/21/2004 0:00:00~"Original"~1~"1584-1"~"PMR"~"Pediatric Research Equity Act"~1584.00~1~"PMR 1584-1: Deferred pediatric study under PREA for the treatment or prevention of upper gastrointestinal bleeding in critically ill pediatric patients ages 2 to 11 years old and 12 to 16 years old."~"Delayed"~"Sponsor has been issued a PREA noncompliance letter on 7/17/13. The study has not been initiated; no subjects are enrolled. The final report milestone was missed."~12/26/2008 0:00:00~
384459~"CDER"~"NDA"~21710.00~"VALIDUS PHARMACEUTICALS LLC"~"Equetro (Carbamazepine)"~12/10/2004 0:00:00~"Original"~1~"4100-1"~"PMR"~"505 (o)(3)"~4100.00~1~"PMR 4100-1: Conduct in vitro patch clamp studies to assess Equetro s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Equetro s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~2/9/2026 0:00:00
384460~"CDER"~"NDA"~21749.00~"HAMELN PHARMA GMBH"~"Pentetate Calcium Trisodium (Ca-DTPA)"~8/11/2004 0:00:00~"Original"~1~"835-2"~"PMC"~"506B PMC"~835.00~2~"PMC 835-2: Longitudinal studies involving follow up of Patient Treatment Data Forms and placement of data into a registry for periodic analyses related to post- marketing drug safety and uses.  a. Protocol submission: Within 6 months of the date of final approval of these applications  b. Study start (i.e., the date the database will be ready to accept patient data, should it be  necessary): Within 6 months of agreement to the protocol  c. Agree to submit annual reports of ongoing longitudinal studies beginning one year from study  initiation."~"Delayed"~"This study is considered delayed because the final protocol was submitted after the milestone due date. This product is for the treatment of contamination resulting from nuclear exposure events and the studies are to start immediately after exposure."~~10/8/2021 0:00:00
384461~"CDER"~"NDA"~21749.00~"HAMELN PHARMA GMBH"~"Pentetate Calcium Trisodium (Ca-DTPA)"~8/11/2004 0:00:00~"Original"~1~"835-3"~"PMC"~"506B PMC"~835.00~3~"PMC 835-3: Human pharmacokinetic study in adult subjects to compare and evaluate the absorption, distribution and elimination of Ca and Zn-DTPA via inhalation using a commonly available jet type nebulizer (FDA approved model to be selected by the sponsor) with the intravenous route. Data/information  on dose delivered and the particle size distribution obtained from the specified nebulizer shall be provided.  a. Protocol submission: Within 6 months of the date of final approval of these applications  b. Study start: Within 6 months of agreement to the protocol  c. Final study report submission: Within 12 months of initiation of the study."~"Delayed"~"This study is considered delayed because the final protocol was submitted after the milestone due date. This product is for the treatment of contamination resulting from nuclear exposure events and the studies are to start immediately after exposure."~~10/8/2021 0:00:00
384462~"CDER"~"BLA"~125268.00~"Amgen Inc."~"Nplate (romiplostim)"~8/22/2008 0:00:00~"Supplement"~167~"4008-1"~"PMR"~"Animal Efficacy"~4008.00~1~"PMR 4008-1: A phase 4 observational study to evaluate the efficacy and safety of Nplate (romiplostim) in the setting of Hematopoietic syndrome of Acute Radiation Syndrome (HS-ARS) following acute exposure to myelosuppressive doses of radiation."~"Pending"~~~12/10/2025 0:00:00
384463~"CDER"~"BLA"~125276.00~"Genentech, Inc."~"Actemra (Tocilizumab)"~1/8/2010 0:00:00~"Supplement"~64~"2678-2"~"PMR"~"505 (o)(3)"~2678.00~2~"PMR 2678-2: A long-term safety study in pediatric patients 2-17 years of age with polyarticular JIA (pJIA) treated with tocilizumab to evaluate for the risk of malignancies, serious infections, gastrointestinal perforation, and effects on growth. The study should include a control group of pediatric pJIA patients treated with other biologics as standard of care. Patients should be followed for 5 years."~"Submitted"~~1/31/2026 0:00:00~2/24/2026 0:00:00
384464~"CDER"~"BLA"~125276.00~"Genentech, Inc."~"Actemra (Tocilizumab)"~1/8/2010 0:00:00~"Supplement"~138~"4369-1"~"PMR"~"Pediatric Research Equity Act"~4369.00~1~"PMR 4369-1: Conduct a pharmacokinetic, pharmacodynamic, and safety study in pediatric patients hospitalized with COVID-19."~"Fulfilled"~"Per FDA letter dated 08/08/2025, this PMR/PMC has been fulfilled."~6/30/2025 0:00:00~2/24/2026 0:00:00
384465~"CDER"~"BLA"~125289.00~"Janssen Biotech, Inc."~"Simponi (golimumab) injection"~4/24/2009 0:00:00~"Supplement"~77~"2909-3"~"PMR"~"505 (o)(3)"~2909.00~3~"PMR 2909-3: A prospective, multi-center, long-term, observational study of ulcerative colitis patients treated with Simponi (golimumab) in a routine clinical setting, to assess the long-term safety of Simponi (golimumab). The study s primary outcome should be the incidence of lymphoma. Design  the study around a testable hypothesis to rule out a clinically meaningful increase in lymphoma above an estimated background risk in a suitable comparator. Secondary endpoints should be pre-specified and may include the incidence of other malignancies. Select and justify the choice of appropriate comparator population(s) and corresponding background  rate(s) relative to Simponi-exposed patients. Provide sample sizes and  effect sizes that can be ruled out under various enrollment target scenarios and loss to follow-up assumptions. Patients should be enrolled over an  initial 5-year period and then followed for a period of at least 10 years from the time of enrollment. Progress updates of patient accrual and a demographic summary should be provided in your annual reports. Safety data should be provided in periodic safety reports."~"Delayed"~"The final protocol date was missed. The study is underway and enrollment has ended."~5/31/2030 0:00:00~6/17/2026 0:00:00
384466~"CDER"~"BLA"~125289.00~"Janssen Biotech, Inc."~"Simponi (golimumab) injection"~4/24/2009 0:00:00~"Supplement"~77~"2909-5"~"PMC"~"506B PMC"~2909.00~5~"PMC 2909-5: Conduct a study to evaluate the effectiveness and safety of Simponi  (golimumab) in pediatric patients 5 to 17 years of age with moderately to severely active ulcerative colitis. The study should be designed to  establish that the dose regimen(s) of Simponi (golimumab) identified in  PMC#4 is(are) effective and safe for induction treatment, as well as for  continued treatment after induction. Pharmacokinetic measurements  should be conducted for exposure-response analysis. Collect serum  samples for immunogenicity testing and conduct analyses of the impact of immunogenicity on pharmacokinetics, efficacy and safety."~"Fulfilled"~~5/31/2022 0:00:00~6/17/2026 0:00:00
384467~"CDER"~"BLA"~125289.00~"Janssen Biotech, Inc."~"Simponi (golimumab) injection"~4/24/2009 0:00:00~"Supplement"~78~"2909-3"~"PMR"~"505 (o)(3)"~2909.00~3~"PMR 2909-3: A prospective, multi-center, long-term, observational study of ulcerative colitis patients treated with Simponi (golimumab) in a routine clinical setting, to assess the long-term safety of Simponi (golimumab). The study s primary outcome should be the incidence of lymphoma. Design  the study around a testable hypothesis to rule out a clinically meaningful increase in lymphoma above an estimated background risk in a suitable comparator. Secondary endpoints should be pre-specified and may include the incidence of other malignancies. Select and justify the choice of appropriate comparator population(s) and corresponding background  rate(s) relative to Simponi-exposed patients. Provide sample sizes and  effect sizes that can be ruled out under various enrollment target scenarios and loss to follow-up assumptions. Patients should be enrolled over an  initial 5-year period and then followed for a period of at least 10 years from the time of enrollment. Progress updates of patient accrual and a demographic summary should be provided in your annual reports. Safety data should be provided in periodic safety reports."~"Delayed"~"The final protocol date was missed. The study is underway and enrollment has ended."~5/31/2030 0:00:00~6/17/2026 0:00:00
384468~"CDER"~"BLA"~125289.00~"Janssen Biotech, Inc."~"Simponi (golimumab) injection"~4/24/2009 0:00:00~"Supplement"~78~"2909-5"~"PMC"~"506B PMC"~2909.00~5~"PMC 2909-5: Conduct a study to evaluate the effectiveness and safety of Simponi  (golimumab) in pediatric patients 5 to 17 years of age with moderately to severely active ulcerative colitis. The study should be designed to  establish that the dose regimen(s) of Simponi (golimumab) identified in  PMC#4 is(are) effective and safe for induction treatment, as well as for  continued treatment after induction. Pharmacokinetic measurements  should be conducted for exposure-response analysis. Collect serum  samples for immunogenicity testing and conduct analyses of the impact of immunogenicity on pharmacokinetics, efficacy and safety."~"Fulfilled"~~5/31/2022 0:00:00~6/17/2026 0:00:00
384469~"CDER"~"BLA"~125289.00~"Janssen Biotech, Inc."~"Simponi (golimumab) injection"~4/24/2009 0:00:00~"Supplement"~79~"2909-3"~"PMR"~"505 (o)(3)"~2909.00~3~"PMR 2909-3: A prospective, multi-center, long-term, observational study of ulcerative colitis patients treated with Simponi (golimumab) in a routine clinical setting, to assess the long-term safety of Simponi (golimumab). The study s primary outcome should be the incidence of lymphoma. Design  the study around a testable hypothesis to rule out a clinically meaningful increase in lymphoma above an estimated background risk in a suitable comparator. Secondary endpoints should be pre-specified and may include the incidence of other malignancies. Select and justify the choice of appropriate comparator population(s) and corresponding background  rate(s) relative to Simponi-exposed patients. Provide sample sizes and  effect sizes that can be ruled out under various enrollment target scenarios and loss to follow-up assumptions. Patients should be enrolled over an  initial 5-year period and then followed for a period of at least 10 years from the time of enrollment. Progress updates of patient accrual and a demographic summary should be provided in your annual reports. Safety data should be provided in periodic safety reports."~"Delayed"~"The final protocol date was missed. The study is underway and enrollment has ended."~5/31/2030 0:00:00~6/17/2026 0:00:00
384470~"CDER"~"NDA"~204442.00~"REACX PHARMACEUTICALS INC"~"Probuphine (Buprenorphine)"~5/26/2016 0:00:00~"Original"~1~"3078-1"~"PMR"~"505 (o)(3)"~3078.00~1~"PMR 3078-1: A prospective descriptive observational cohort study of insertion-, localization-, and removal-related serious adverse events and their sequelae associated with PROBUPHINE (buprenorphine hydrochloride) use."~"Delayed"~"A final protocol was submitted on Jan 31, 2019, and annual interim report, study completion and final report submission dates were revised as per 02/08/2019 letter. The Final Report milestone has been missed."~11/30/2021 0:00:00~11/19/2025 0:00:00
384471~"CDER"~"NDA"~204442.00~"REACX PHARMACEUTICALS INC"~"Probuphine (Buprenorphine)"~5/26/2016 0:00:00~"Original"~1~"3078-3"~"PMR"~"505 (o)(3)"~3078.00~3~"PMR 3078-3: Clinical trial to evaluate the effect of scarring or inflammation related to a prior implant on the safety of re-implantation/reinsertion, the potential for migration of implants, and the bioavailability of PROBUPHINE (buprenorphine hydrochloride) when the drug is implanted in a previously used site."~"Delayed"~"The Final Report submission milestone was missed. The FDA issued a Notification of Missed Milestones letter on 06/16/2023. The FDA determined that there was not good cause for the noncompliance and issued a Notification of Failure to Demonstrate Good Cause letter on 10/03/2023. In their 11/19/2025 annual report the applicant advises that they are looking to develop approaches to address their PMRs."~8/31/2019 0:00:00~11/19/2025 0:00:00
384472~"CDER"~"NDA"~204442.00~"REACX PHARMACEUTICALS INC"~"Probuphine (Buprenorphine)"~5/26/2016 0:00:00~"Original"~1~"3078-4"~"PMR"~"505 (o)(3)"~3078.00~4~"PMR 3078-4: Clinical trial to evaluate the safety, feasibility, and pharmacokinetics of  PROBUPHINE (buprenorphine hydrochloride) implantation at alternate body sites. The trial should also evaluate the safety of other methods of inserting PROBUPHINE (buprenorphine hydrochloride) into additional  locations on the arm."~"Delayed"~"The Final Report submission milestone was missed. The FDA issued a Notification of Missed Milestones letter on 06/16/2023. The FDA determined that there was not good cause for the noncompliance and issued a Notification of Failure to Demonstrate Good Cause letter on 10/03/2023."~8/31/2018 0:00:00~11/19/2025 0:00:00
384473~"CDER"~"NDA"~204508.00~"BAXTER HEALTHCARE CORP"~"Clinolipid (Lipid Emulsion)"~10/3/2013 0:00:00~"Supplement"~22~"4607-1"~"PMR"~"505 (o)(3)"~4607.00~1~"PMR 4607-1: A single-arm open-label safety trial of Clinolipid to evaluate the risk of developing one or both of the following adverse reactions of essential fatty acid deficiency (EFAD) and parenteral nutrition-associated cholestasis (PNAC) in the following patient populations who are anticipated to need 8 weeks or longer of parenteral nutrition treatment: birth   2 years, 2   11 years, 12   17 years, and adults (18 years or older). Plasma fatty acids should be measured using an adequately validated bioanalytical assay method."~"Pending"~~11/30/2030 0:00:00~11/26/2025 0:00:00
384474~"CDER"~"NDA"~204592.00~"ZYLA LIFE SCIENCES US INC"~"Zorvolex (Diclofenac)"~10/18/2013 0:00:00~"Original"~1~"2091-1"~"PMR"~"Pediatric Research Equity Act"~2091.00~1~"PMR 2091-1: An open-label pharmacokinetic and safety study or studies of an ageappropriate formulation of Zorvolex in pediatric patients 6 years to less than 17 years of age with acute pain."~"Delayed"~"The final report was submitted on 6/1/17 (S-010), with no User Fee. An unacceptable for filing letter was sent. The Type C meeting held on 11/28/18, the study report should be re-submitted to the NDA."~5/3/2017 0:00:00~11/29/2021 0:00:00
384475~"CDER"~"NDA"~204592.00~"ZYLA LIFE SCIENCES US INC"~"Zorvolex (Diclofenac)"~10/18/2013 0:00:00~"Original"~1~"2091-2"~"PMR"~"Pediatric Research Equity Act"~2091.00~2~"PMR 2091-2: An open-label pharmacokinetic and safety study or studies of an ageappropriate formulation of Zorvolex in pediatric patients 2 years to less than 6  years of age with acute pain."~"Delayed"~"Study DIC2-15-09 placed on Partial Clinical Hold (letter 11/19/15), therefore delayed."~1/6/2018 0:00:00~11/29/2021 0:00:00
384476~"CDER"~"NDA"~204592.00~"ZYLA LIFE SCIENCES US INC"~"Zorvolex (Diclofenac)"~10/18/2013 0:00:00~"Original"~1~"2091-3"~"PMR"~"Pediatric Research Equity Act"~2091.00~3~"PMR 2091-3: A pharmacokinetic, safety, and efficacy study or studies of an age-appropriate formulation of Zorvolex in pediatric patients 1 year to less than 2 years of age with acute pain."~"Delayed"~"Need to complete the studies listed under 2091-1 and 2 to proceed with this one."~7/30/2020 0:00:00~11/29/2021 0:00:00
384477~"CDER"~"NDA"~204629.00~"BOEHRINGER INGELHEIM PHARMACEUTICALS INC"~"Jardiance (Empagliflozin)"~8/1/2014 0:00:00~"Supplement"~40~"4508-1"~"PMR"~"Pediatric Research Equity Act"~4508.00~1~"PMR 4508-1: Conduct pharmacokinetic, efficacy, and safety assessments in patients 2 to 17 years of age with chronic kidney disease (CKD)"~"Delayed"~"The Final protocol Milestone date was missed.
"~6/30/2029 0:00:00~9/24/2025 0:00:00
384478~"CDER"~"NDA"~204684.00~"KNIGHT THERAPEUTICS USA INC"~"Impavido (Miltefosine) "~3/19/2014 0:00:00~"Original"~1~"2127-1"~"PMR"~"505 (o)(3)"~2127.00~1~"PMR 2127-1: Collect and analyze data regarding pregnancy outcomes for 10 years after approval of Impavido (miltefosine) in women who become pregnant while  taking Impavido (miltefosine) or during 5 months after end of Impavido  (miltefosine) therapy"~"Fulfilled"~~3/31/2026 0:00:00~5/5/2025 0:00:00
384479~"CDER"~"NDA"~204767.00~"FRESENIUS KABI USA LLC"~"Acetaminophen"~10/28/2015 0:00:00~"Supplement"~2~"3975-1"~"PMR"~"505 (o)(3)"~3975.00~1~"PMR 3975-1: Conduct a study to monitor leachable levels of 4-(2-hydroxyethoxy) benzaldehyde in long-term stability batches over the course of stability and submit a toxicological risk ssessment that adequately establishes safety of the highest level of 4-(2-hydroxyethoxy)-benzaldehyde associated with the maximum daily dose of your drug product.
"~"Submitted"~~4/30/2023 0:00:00~12/27/2022 0:00:00
384480~"CDER"~"NDA"~204781.00~"GUERBET LLC"~"Dotarem (Gadoterate Meglumine)"~3/20/2013 0:00:00~"Supplement"~8~"3628-4"~"PMR"~"505 (o)(3)"~3628.00~4~"PMR 3628-4: A prospective longitudinal cohort trial with one or more matched control group(s) to evaluate the effects of repetitive GBCA administration on a comprehensive battery of neurobehavioral testing over the course of at least five administrations. The trial should be sufficiently powered to exclude a pre-specified magnitude of decline. As a secondary objective, trial patients should also have the option of providing blood and urine samples at the time of reimaging, so that normative estimates of gadolinium concentration across an extended range of post-administration timepoints may be documented."~"Ongoing"~~4/30/2029 0:00:00~5/19/2026 0:00:00
384481~"CDER"~"NDA"~204803.00~"INNOCOLL PHARMACEUTICALS LTD"~"Posimir (Bupivacaine)"~2/1/2021 0:00:00~"Original"~1~"4014-1"~"PMR"~"505 (o)(3)"~4014.00~1~"PMR 4014-1: Conduct a nonclinical intravenous toxicity study that includes both an acute and delayed timepoint with full histopathology to determine the ultimate impact of the drug product and fate of the vehicle if it is inadvertently administered into the intravascular space. The study should include collection of pharmacokinetic data to determine if intravascular injection results in more rapid release of the bupivacaine dose from the vehicle."~"Delayed"~"The draft protocol submission and the study completion milestones were missed and the FDA issued a Notification of Missed Milestones letter on 11/30/2023."~9/30/2023 0:00:00~4/3/2024 0:00:00
384482~"CDER"~"NDA"~204803.00~"INNOCOLL PHARMACEUTICALS LTD"~"Posimir (Bupivacaine)"~2/1/2021 0:00:00~"Original"~1~"4014-2"~"PMR"~"505 (o)(3)"~4014.00~2~"PMR 4014-2: Conduct a study to assess the potential serious risk of POSIMIR-induced Local Anesthetic Systemic Toxicity (LAST) following inadvertent intravenous route of administration and the ability of lipid infusions to treat the clinical manifestations of LAST."~"Delayed"~"The draft protocol submission and the study completion milestones were missed. The FDA issued a Notification of Missed Milestones letter on 11/30/2023."~9/30/2023 0:00:00~4/3/2024 0:00:00
384483~"CDER"~"NDA"~208564.00~"MAYNE PHARMA LLC"~"IMVEXXY (estradiol)"~5/29/2018 0:00:00~"Original"~1~"3843-1"~"PMR"~"505 (o)(3)"~3843.00~1~"PMR 3843-1: An observational study evaluating the risk of endometrial cancer in postmenopausal women with a uterus who use low-dose vaginal estrogen unopposed by a progestogen."~"Delayed"~"The final report submission milestone was missed because of an Agency request for additional analyses of the data. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 03/19/2026."~2/28/2026 0:00:00~7/25/2025 0:00:00
384484~"CDER"~"BLA"~208583.00~"Novo Nordisk Inc."~"Xultophy 100/3.6 (Insulin Degludec and Liraglutide)"~11/21/2016 0:00:00~"Original"~1~"4751-1"~"PMR"~"505 (o)(3)"~4751.00~1~"PMR 4751-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of liraglutide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Pending"~~11/30/2026 0:00:00~11/12/2025 0:00:00
384485~"CDER"~"NDA"~208609.00~"RENEW PHARMACEUTICALS LTD"~"Ephedrine Sulfate "~3/1/2017 0:00:00~"Supplement"~1~"3235-1"~"PMR"~"505 (o)(3)"~3235.00~1~"PMR 3235-1: Conduct an adequate leachable safety assessment for the 13 mm V-35 4432/50 gray siliconized level 1 WESTAR RS Stopper used in your container closure system. This assessment must include leachable data collected over time from stability studies to determine if the identified extractables leach into the drug product over time. At least three batches stored under long term conditions of 25  C, should be tested per the approved stability protocol of 0, 6, 12, 18 and 24 months and yearly thereafter or to the end of shelf life. Using this information, conduct a toxicological risk assessment justifying the safety of the leachables, taking into consideration the maximum daily dose of the identified materials for  this drug product. For your toxicological risk assessment, any leachable that contains a structural alert for mutagenicity should not exceed 120 mcg/day total daily exposure, or it must be adequately qualified for safety. A toxicological risk assessment should be provided for any non-genotoxic leachable that exceeds 5  mcg/day."~"Submitted"~~9/30/2020 0:00:00~4/24/2020 0:00:00
384486~"CDER"~"NDA"~208610.00~"MELINTA SUBSIDIARY CORP"~"Baxdela (Delafloxacin Meglumine)"~6/19/2017 0:00:00~"Supplement"~7~"3727-1"~"PMR"~"Pediatric Research Equity Act"~3727.00~1~"PMR 3727-1: Conduct a randomized 2-sequence, 2-period, crossover study in fasted healthy adults to determine the relative bioavailability of the delafloxacin tablet and an oral pediatric formulation of delafloxacin."~"Fulfilled"~"Per FDA letter dated 10/16/2025, this PMR/PMC has been fulfilled."~2/28/2025 0:00:00~8/5/2025 0:00:00
384487~"CDER"~"NDA"~208610.00~"MELINTA SUBSIDIARY CORP"~"Baxdela (Delafloxacin Meglumine)"~6/19/2017 0:00:00~"Supplement"~7~"3727-2"~"PMR"~"Pediatric Research Equity Act"~3727.00~2~"PMR 3727-2: Conduct an open-label, multi-center study evaluating the PK and safety of delafloxacin as a single IV dose, an oral tablet or an oral pediatric formulation administered as add-on therapy to standard of care (SOC) antimicrobial agents in patients 2 months to <18 years of age with suspected or confirmed bacterial infections due to delafloxacin susceptible organisms."~"Released"~"Per FDA letter dated 10/16/2025, this PMR/PMC has been released."~10/31/2026 0:00:00~8/5/2025 0:00:00
384488~"CDER"~"NDA"~208610.00~"MELINTA SUBSIDIARY CORP"~"Baxdela (Delafloxacin Meglumine)"~6/19/2017 0:00:00~"Supplement"~7~"3727-3"~"PMR"~"Pediatric Research Equity Act"~3727.00~3~"PMR 3727-3: Conduct a randomized open label, active-controlled, evaluator-blinded safety and PK trial of delafloxacin in patients 2 months to <18 years of age with suspected or confirmed CABP."~"Released"~"Per FDA letter dated 10/16/2025, this PMR/PMC has been released."~9/30/2026 0:00:00~8/5/2025 0:00:00
384489~"CDER"~"NDA"~208610.00~"MELINTA SUBSIDIARY CORP"~"Baxdela (Delafloxacin Meglumine)"~6/19/2017 0:00:00~"Supplement"~7~"3727-5"~"PMR"~"Pediatric Research Equity Act"~3727.00~5~"PMR 3727-5: Conduct an open-label, multi-center study evaluating the PK and safety of delafloxacin as a single IV dose, an oral tablet or an oral pediatric formulation administered as add-on therapy to standard of care (SOC) antimicrobial agents in patients 2 months to less than 12 years of age with suspected or confirmed bacterial infections due to delafloxacin susceptible organisms."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~10/31/2029 0:00:00~8/5/2025 0:00:00
384490~"CDER"~"NDA"~208610.00~"MELINTA SUBSIDIARY CORP"~"Baxdela (Delafloxacin Meglumine)"~6/19/2017 0:00:00~"Supplement"~7~"3727-6"~"PMR"~"Pediatric Research Equity Act"~3727.00~6~"PMR 3727-6: Conduct an open label, non-comparator safety and PK trial of delafloxacin in patients 2 months to less than 18 years of age with suspected or confirmed community-acquired bacterial pneumonia (CABP)."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~10/31/2031 0:00:00~8/5/2025 0:00:00
384491~"CDER"~"NDA"~208611.00~"MELINTA SUBSIDIARY CORP"~"Baxdela (Delafloxacin Meglumine)"~6/19/2017 0:00:00~"Supplement"~6~"3727-1"~"PMR"~"Pediatric Research Equity Act"~3727.00~1~"PMR 3727-1: Conduct a randomized 2-sequence, 2-period, crossover study in fasted healthy adults to determine the relative bioavailability of the delafloxacin tablet and an oral pediatric formulation of delafloxacin."~"Fulfilled"~"Per FDA letter dated 10/16/2025, this PMR/PMC has been fulfilled."~2/28/2025 0:00:00~8/5/2025 0:00:00
384492~"CDER"~"NDA"~208611.00~"MELINTA SUBSIDIARY CORP"~"Baxdela (Delafloxacin Meglumine)"~6/19/2017 0:00:00~"Supplement"~6~"3727-2"~"PMR"~"Pediatric Research Equity Act"~3727.00~2~"PMR 3727-2: Conduct an open-label, multi-center study evaluating the PK and safety of delafloxacin as a single IV dose, an oral tablet or an oral pediatric formulation administered as add-on therapy to standard of care (SOC) antimicrobial agents in patients 2 months to <18 years of age with suspected or confirmed bacterial infections due to delafloxacin susceptible organisms."~"Released"~"Per FDA letter dated 10/16/2025, this PMR/PMC has been released."~10/31/2026 0:00:00~8/5/2025 0:00:00
384493~"CDER"~"NDA"~208611.00~"MELINTA SUBSIDIARY CORP"~"Baxdela (Delafloxacin Meglumine)"~6/19/2017 0:00:00~"Supplement"~6~"3727-3"~"PMR"~"Pediatric Research Equity Act"~3727.00~3~"PMR 3727-3: Conduct a randomized open label, active-controlled, evaluator-blinded safety and PK trial of delafloxacin in patients 2 months to <18 years of age with suspected or confirmed CABP."~"Released"~"Per FDA letter dated 10/16/2025, this PMR/PMC has been released."~9/30/2026 0:00:00~8/5/2025 0:00:00
384494~"CDER"~"NDA"~208611.00~"MELINTA SUBSIDIARY CORP"~"Baxdela (Delafloxacin Meglumine)"~6/19/2017 0:00:00~"Supplement"~6~"3727-5"~"PMR"~"Pediatric Research Equity Act"~3727.00~5~"PMR 3727-5: Conduct an open-label, multi-center study evaluating the PK and safety of delafloxacin as a single IV dose, an oral tablet or an oral pediatric formulation administered as add-on therapy to standard of care (SOC) antimicrobial agents in patients 2 months to less than 12 years of age with suspected or confirmed bacterial infections due to delafloxacin susceptible organisms."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~10/31/2029 0:00:00~8/5/2025 0:00:00
384495~"CDER"~"NDA"~208611.00~"MELINTA SUBSIDIARY CORP"~"Baxdela (Delafloxacin Meglumine)"~6/19/2017 0:00:00~"Supplement"~6~"3727-6"~"PMR"~"Pediatric Research Equity Act"~3727.00~6~"PMR 3727-6: Conduct an open label, non-comparator safety and PK trial of delafloxacin in patients 2 months to less than 18 years of age with suspected or confirmed community-acquired bacterial pneumonia (CABP)."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~10/31/2031 0:00:00~8/5/2025 0:00:00
384496~"CDER"~"NDA"~21749.00~"HAMELN PHARMA GMBH"~"Pentetate Calcium Trisodium (Ca-DTPA)"~8/11/2004 0:00:00~"Original"~1~"835-4"~"PMC"~"506B PMC"~835.00~4~"PMC 835-4: Human pharmacokinetic study in pediatric subjects to compare and evaluate the absorption, distribution and elimination of Ca and Zn-DTPA via inhalation using a commonly available jet type nebulizer with the intravenous route after an event in which exposure to trans-uranium isotopes occurred. Data/information on dose delivered and the particle size distribution obtained from the specified nebulizer shall be provided."~"Delayed"~"This study is considered delayed because the final protocol was submitted after the milestone due date. This product is for the treatment of contamination resulting from nuclear exposure events and the studies are to start immediately after exposure."~~10/8/2021 0:00:00
384497~"CDER"~"NDA"~21751.00~"HAMELN PHARMA PLUS GMBH"~"Pentetate Zinc Trisodium (Zn-DTPA)"~8/11/2004 0:00:00~"Original"~1~"835-2"~"PMC"~"506B PMC"~835.00~2~"PMC 835-2: Longitudinal studies involving follow up of Patient Treatment Data Forms and placement of data into a registry for periodic analyses related to post- marketing drug safety and uses.  a. Protocol submission: Within 6 months of the date of final approval of these applications  b. Study start (i.e., the date the database will be ready to accept patient data, should it be  necessary): Within 6 months of agreement to the protocol  c. Agree to submit annual reports of ongoing longitudinal studies beginning one year from study  initiation."~"Delayed"~"This study is considered delayed because the final protocol was submitted after the milestone due date. This product is for the treatment of contamination resulting from nuclear exposure events and the studies are to start immediately after exposure."~~10/8/2021 0:00:00
384498~"CDER"~"NDA"~21751.00~"HAMELN PHARMA PLUS GMBH"~"Pentetate Zinc Trisodium (Zn-DTPA)"~8/11/2004 0:00:00~"Original"~1~"835-3"~"PMC"~"506B PMC"~835.00~3~"PMC 835-3: Human pharmacokinetic study in adult subjects to compare and evaluate the absorption, distribution and elimination of Ca and Zn-DTPA via inhalation using a commonly available jet type nebulizer (FDA approved model to be selected by the sponsor) with the intravenous route. Data/information  on dose delivered and the particle size distribution obtained from the specified nebulizer shall be provided.  a. Protocol submission: Within 6 months of the date of final approval of these applications  b. Study start: Within 6 months of agreement to the protocol  c. Final study report submission: Within 12 months of initiation of the study."~"Delayed"~"This study is considered delayed because the final protocol was submitted after the milestone due date. This product is for the treatment of contamination resulting from nuclear exposure events and the studies are to start immediately after exposure."~~10/8/2021 0:00:00
384512~"CDER"~"BLA"~125349.00~"Emergent Manufacturing Operations Baltimore LLC"~"Raxibacumab"~12/14/2012 0:00:00~"Original"~1~"2739-1"~"PMR"~"Animal Efficacy"~2739.00~1~"PMR 2739-1: Conduct a field study to evaluate the efficacy, pharmacokinetics, and safety of raxibacumab use for Bacillus anthracis in the United States."~"Pending"~~~2/9/2026 0:00:00
384513~"CDER"~"BLA"~125370.00~"GlaxoSmithKline LLC"~"Benlysta (Belimumab)"~3/9/2011 0:00:00~"Original"~1~"2661-16"~"PMR"~"505 (o)(3)"~2661.00~16~"PMR 2661-16: Conduct a pregnancy exposure registry using a Pregnancy Exposure Registry with the Organization of Teratology Information Specialists (OTIS) to evaluate pregnancy outcomes for women exposed to Benlysta (belimumab) during pregnancy."~"Ongoing"~~3/31/2030 0:00:00~5/6/2026 0:00:00
384514~"CDER"~"BLA"~125377.00~"Bristol-Myers Squibb Company"~"Yervoy (Ipilimumab) "~3/25/2011 0:00:00~"Supplement"~96~"3450-3"~"PMC"~"506B PMC"~3450.00~3~"PMC 3450-3: Commitment to support the availability through an appropriate analytical and clinical validation study using clinical trial data that will support labeling of an immunohistochemistry-based in vitro diagnostic device that is essential to the safe and effective use of ipilimumab for patients with tumors that are mismatch repair deficient."~"Fulfilled"~~7/31/2024 0:00:00~5/21/2026 0:00:00
384515~"CDER"~"BLA"~125377.00~"Bristol-Myers Squibb Company"~"Yervoy (Ipilimumab) "~3/25/2011 0:00:00~"Supplement"~96~"3450-4"~"PMC"~"506B PMC"~3450.00~4~"PMC 3450-4: Commitment to support the availability through an appropriate analytical and clinical validation study using clinical trial data that will support labeling of a nucleic acid-based in vitro diagnostic device that is essential to the safe and effective use of ipilimumab for patients with tumors that are microsatellite instability-high."~"Fulfilled"~~7/31/2024 0:00:00~5/21/2026 0:00:00
384516~"CDER"~"BLA"~125388.00~"Seagen Inc."~"Adcetris (Brentuximab Vedotin)"~8/19/2011 0:00:00~"Supplement"~97~"3356-1"~"PMR"~"505 (o)(3)"~3356.00~1~"PMR 3356-1: Submit long-term safety follow-up for the ECHELON-1 clinical trial including the following events: (a) secondary malignancies, (b) treatmentrelated serious adverse events, (c) treatment-emergent neuropathy adverse events until resolution or study closure, and (d) deaths and cause of deaths. Submit an interim report and datasets with 60 months of safety follow-up. Submit the final report and datasets after minimum of 120 months of safety follow-up."~"Ongoing"~~8/31/2026 0:00:00~10/16/2025 0:00:00
384517~"CDER"~"NDA"~205122.00~"UPSHER-SMITH LABORATORIES LLC"~"Qudexy XR (Topiramate)"~3/11/2014 0:00:00~"Original"~1~"4090-1"~"PMR"~"505 (o)(3)"~4090.00~1~"PMR 4090-1: Conduct in vitro patch clamp studies to assess Qudexy XR s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Qudexy XR s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~5/8/2026 0:00:00
384518~"CDER"~"NDA"~205122.00~"UPSHER-SMITH LABORATORIES LLC"~"Qudexy XR (Topiramate)"~3/11/2014 0:00:00~"Original"~1~"2137-4"~"PMR"~"Pediatric Research Equity Act"~2137.00~4~"PMR 2137-4: A study to evaluate the pharmacokinetics (PK) and tolerability of the age appropriate formulation of Qudexy XR (topiramate) extended-release capsules as adjunctive therapy in children ages 6 months to less than 2 years with partial onset seizures (POS)."~"Released"~"Per FDA letter dated 10/03/2025, this PMR/PMC has been released."~10/31/2022 0:00:00~5/8/2026 0:00:00
384519~"CDER"~"NDA"~205122.00~"UPSHER-SMITH LABORATORIES LLC"~"Qudexy XR (Topiramate)"~3/11/2014 0:00:00~"Original"~1~"2137-5"~"PMR"~"Pediatric Research Equity Act"~2137.00~5~"PMR 2137-5: An adequately controlled study to assess the efficacy and safety of the age appropriate formulation of Qudexy XR (topiramate) extended-release capsules as adjunctive therapy in children ages 6 months to less than 2 years with POS."~"Released"~"Per FDA letter dated 10/03/2025, this PMR/PMC has been released."~10/31/2029 0:00:00~5/8/2026 0:00:00
384520~"CDER"~"NDA"~205122.00~"UPSHER-SMITH LABORATORIES LLC"~"Qudexy XR (Topiramate)"~3/11/2014 0:00:00~"Supplement"~3~"3171-1"~"PMR"~"Pediatric Research Equity Act"~3171.00~1~"PMR 3171-1: Conduct a pediatric study under PREA to evaluate the efficacy and safety of Qudexy XR for the prophylaxis of migraine in pediatric patients 6-11 years of age. This trial must be designed as a randomized, double-blind, placebo-controlled superiority trial."~"Released"~"Per FDA letter dated 10/24/2025, this PMR/PMC has been released."~9/30/2026 0:00:00~5/8/2026 0:00:00
384521~"CDER"~"NDA"~205122.00~"UPSHER-SMITH LABORATORIES LLC"~"Qudexy XR (Topiramate)"~3/11/2014 0:00:00~"Supplement"~5~"3171-1"~"PMR"~"Pediatric Research Equity Act"~3171.00~1~"PMR 3171-1: Conduct a pediatric study under PREA to evaluate the efficacy and safety of Qudexy XR for the prophylaxis of migraine in pediatric patients 6-11 years of age. This trial must be designed as a randomized, double-blind, placebo-controlled superiority trial."~"Released"~"Per FDA letter dated 10/24/2025, this PMR/PMC has been released."~9/30/2026 0:00:00~5/8/2026 0:00:00
384522~"CDER"~"NDA"~205266.00~"SUN PHARMACEUTICAL INDUSTRIES LTD"~"Odomzo (Sonidegib) "~7/24/2015 0:00:00~"Original"~1~"2931-3"~"PMR"~"505 (o)(3)"~2931.00~3~"PMR 2931-3: A Pregnancy Pharmacovigilance Study to evaluate pregnancy outcomes and infant outcomes following exposure to Odomzo (sonidegib). This study will include a mechanism to collect, classify, and analyze data on direct exposures (women exposed to Odomzo (sonidegib) as treatment) and indirect exposures (women exposed to Odomzo (sonidegib) through the seminal fluid of a male  partner). The Pregnancy Pharmacovigilance Study will be initiated and functioning at the time of product launch. There will be interim annual reporting of the data collected from the study. The study, at a minimum, will include the following key elements (see the Guidance for Industry Establishing Pregnancy Exposure Registries for a detailed description of these elements):    Data collection of prospective and retrospective data points, adequate to produce informative, reliable data outcomes.    Data analysis utilizing descriptive statistics for summarizing data that will fully capture outcomes of concern. Data collected prospectively analyzed separate from data collected retrospectively.    Description of procedures including the patient recruitment, along with healthcare provider awareness of potential safety risk and existence of this study, and the monitoring of pregnancy and infant outcomes."~"Ongoing"~~7/31/2026 0:00:00~9/19/2025 0:00:00
384523~"CDER"~"NDA"~205394.00~"GENSCO LABORATORIES LLC DBA GENSCO PHARMA"~"RizaFilm (rizatriptan)"~4/14/2023 0:00:00~"Original"~1~"4434-1"~"PMR"~"Pediatric Research Equity Act"~4434.00~1~"PMR 4434-1: Develop an age-appropriate dose strength of RizaFilm for administration to pediatric patients 6 to 11 years of age."~"Fulfilled"~"Per FDA letter dated 02/26/2026, this PMR/PMC has been fulfilled."~4/30/2025 0:00:00~6/14/2024 0:00:00
384524~"CDER"~"NDA"~205395.00~"JANSSEN PRODUCTS LP"~"Prezcobix (Cobicistat and Darunavir)"~1/29/2015 0:00:00~"Original"~1~"2845-1"~"PMR"~"Pediatric Research Equity Act"~2845.00~1~"PMR 2845-1: Evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of darunavir and cobicistat fixed dose combination (FDC) age-appropriate  formulation in HIV-infected pediatric subjects 3 years to less than 6 years of age  and weighing at least 15 kg. The safety and antiviral activity (efficacy) of darunavir and cobicistat FDC age-appropriate formulation in pediatric subjects  should be evaluated for a minimum of 24 weeks. A clinical trial in children ages 3 years to less than 6 years may not be required if the dosing recommendation for  the FDC age-appropriate formulation can be supported by pediatric trials already  conducted with the individual drug products and if the age-appropriate FDC produces similar exposures as the individual components."~"Fulfilled"~"Per FDA letter dated 02/27/2026, this PMR/PMC has been fulfilled."~5/31/2025 0:00:00~3/26/2025 0:00:00
384525~"CDER"~"NDA"~205395.00~"JANSSEN PRODUCTS LP"~"Prezcobix (Cobicistat and Darunavir)"~1/29/2015 0:00:00~"Original"~1~"2845-2"~"PMR"~"Pediatric Research Equity Act"~2845.00~2~"PMR 2845-2: Evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of darunavir and cobicistat fixed dose combination (FDC) age-appropriate formulation in HIV-infected pediatric subjects 6 years to less than 12 years of age and weighing at least 15 kg. The safety and antiviral activity (efficacy) of  darunavir and cobicistat FDC age-appropriate formulation in pediatric subjects should be evaluated for a minimum of 24 weeks. A clinical trial in children ages 6 years to less than 12 years may not be required if the dosing recommendation for the FDC age-appropriate formulation can be supported by pediatric trials already conducted with the individual drug products and if the age-appropriate FDC produces similar exposures as the individual components."~"Fulfilled"~"Per FDA letter dated 02/27/2026, this PMR/PMC has been fulfilled."~6/30/2022 0:00:00~3/26/2025 0:00:00
384526~"CDER"~"NDA"~205435.00~"CUBIST PHARMACEUTICALS LLC"~"Sivextro (Tedizolid Phosphate)"~6/20/2014 0:00:00~"Supplement"~16~"4827-1"~"PMR"~"Pediatric Research Equity Act"~4827.00~1~"PMR 4827-1: Complete development of the [...] formulation including product stability studies   long-term and accelerated storage, stress studies, and applicable in-use stability and compatibility."~"Delayed"~"The final report was submitted to FDA on 7/15/2025. A Not Fulfilled letter was issued 04/23/2026."~8/31/2025 0:00:00~8/12/2025 0:00:00
384527~"CDER"~"NDA"~205436.00~"CUBIST PHARMACEUTICALS LLC"~"Sivextro (Tedizolid Phosphate)"~6/20/2014 0:00:00~"Supplement"~13~"4827-1"~"PMR"~"Pediatric Research Equity Act"~4827.00~1~"PMR 4827-1: Complete development of the [...] formulation including product stability studies   long-term and accelerated storage, stress studies, and applicable in-use stability and compatibility."~"Delayed"~"The final report was submitted to FDA on 7/15/2025. A Not Fulfilled letter was issued 04/23/2026."~8/31/2025 0:00:00~8/12/2025 0:00:00
384528~"CDER"~"NDA"~205437.00~"AMGEN INC"~"Otezla (Apremilast)"~3/21/2014 0:00:00~"Original"~1~"2135-1"~"PMR"~"505 (o)(3)"~2135.00~1~"PMR 2135-1: A prospective, observational, controlled, pregnancy exposure registry study to  monitor pregnancies exposed to apremilast with the primary objective to evaluate  whether there is any increase in the risk of birth defects."~"Fulfilled"~~6/30/2022 0:00:00~5/15/2026 0:00:00
384529~"CDER"~"NDA"~209128.00~"VERTICAL PHARMACEUTICALS LLC"~"Dsuvia (Sufentanil)"~11/2/2018 0:00:00~"Original"~1~"3523-1"~"PMR"~"Pediatric Research Equity Act"~3523.00~1~"PMR 3523-1: A safety and pharmacokinetic study of DSUVIA (sufentanil sublingual tablets) in pediatric patients ages 6 to less than 17 years with acute pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate."~"Delayed"~"The Final Protocol milestone was missed. Full pediatric waiver/PMR Release requested 08/26/2024. Full waiver and PMR Release was denied per FDA letter dated 06/24/2025, but granted a Deferral Extension and Acknowledge Revised Milestones.
"~12/31/2027 0:00:00~12/18/2025 0:00:00
384530~"CDER"~"NDA"~209139.00~"CARMEL BIOSCIENCES INC"~"Prexxartan (Valsartan)"~12/19/2017 0:00:00~"Original"~1~"3319-3"~"PMR"~"Pediatric Research Equity Act"~3319.00~3~"PMR 3319-3: Conduct a pharmacokinetic bridging study in adults to determine the exposure of valsartan with the current and new formulation of valsartan oral solution containing a lower quantity of propylene glycol for use in pediatric patients 1 to less than 6 years of age with hypertension."~"Delayed"~"Deferral Extension Requested 04/14/2025. Denied per FDA letter dated 05/14/2025.The drug has not yet been marketed or manufactured.
"~6/30/2025 0:00:00~4/17/2026 0:00:00
384531~"CDER"~"NDA"~209210.00~"ASTRAZENECA AB"~"Bydureon Bcise (exenatide)"~10/20/2017 0:00:00~"Original"~1~"4740-1"~"PMR"~"505 (o)(3)"~4740.00~1~"PMR 4740-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of exenatide extended-release injectable suspension and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Released"~~11/30/2026 0:00:00~11/21/2024 0:00:00
384532~"CDER"~"NDA"~209210.00~"ASTRAZENECA AB"~"Bydureon Bcise (exenatide)"~10/20/2017 0:00:00~"Original"~1~"1860-5"~"PMR"~"505 (o)(3)"~1860.00~5~"PMR 1860-5: A medullary thyroid carcinoma case series registry of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of BYDUREON (exenatide for injectable suspension) into the marketplace. This study will also establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to diabetes and use of BYDUREON (exenatide for injectable suspension)."~"Released"~~9/30/2028 0:00:00~11/21/2024 0:00:00
384533~"CDER"~"NDA"~209229.00~"BIOCORRX PHARMACEUTICALS INC"~"Lucemyra (lofexidine)"~5/16/2018 0:00:00~"Original"~1~"3391-1"~"PMR"~"Pediatric Research Equity Act"~3391.00~1~"PMR 3391-1: Conduct a juvenile animal study in rats from post-natal day (PND) 36 to PND 90 to support pediatric drug development in children aged 12 to 17 years. The study will evaluate the effect of the drug on growth and development, specifically, reproductive capacity, bone development, and the central nervous system histopathology and long-term functional effects (learning and memory, motor function. reflexes, and an assessment of social interaction or other highercognitive functioning)."~"Submitted"~"Final report submitted on May 12, 2021."~10/31/2019 0:00:00~7/15/2026 0:00:00
384534~"CDER"~"NDA"~209229.00~"BIOCORRX PHARMACEUTICALS INC"~"Lucemyra (lofexidine)"~5/16/2018 0:00:00~"Original"~1~"3391-2"~"PMR"~"Pediatric Research Equity Act"~3391.00~2~"PMR 3391-2: Conduct a pharmacokinetic and safety study of lofexidine in adolescents age 12 to 17 years with opioid withdrawal syndrome."~"Delayed"~"The final report milestone was missed, because of slow study initiation. Deferral Extension Requested 06/07/2024. Denied per FDA letter dated 09/26/2024.
"~12/31/2023 0:00:00~7/15/2026 0:00:00
384535~"CDER"~"NDA"~209229.00~"BIOCORRX PHARMACEUTICALS INC"~"Lucemyra (lofexidine)"~5/16/2018 0:00:00~"Original"~1~"3391-3"~"PMR"~"Pediatric Research Equity Act"~3391.00~3~"PMR 3391-3: Conduct a juvenile toxicology in rats to evaluate the impact of lofexidine on early neuronal development during peak synaptogenesis to support pediatric studies in neonates and children under the age of 3."~"Submitted"~"The final report was submitted on May 12, 2021."~3/31/2021 0:00:00~7/15/2026 0:00:00
384547~"CDER"~"NDA"~21926.00~"AZURITY PHARMACEUTICALS INC"~"Treximet (Naproxen Sodium and Sumatriptan)"~4/15/2008 0:00:00~"Supplement"~12~"2910-2"~"PMR"~"Pediatric Research Equity Act"~2910.00~2~"PMR 2910-2: Conduct a pharmacokinetics (PK) study in children ages 6 years to 11 years with migraine. Using information from this PK study, conduct a controlled efficacy study in children ages 6 years to 11 years with migraine. Conduct a long-term open-label safety study in pediatric patients with migraine ages 6 years to 11 years. The longterm safety study must provide a descriptive analysis of safety data in at least 50 pediatric patients treated with Treximet for a duration of at least 6 months, treating on average at least one migraine attack per month, at doses evaluated in the efficacy study."~"Delayed"~"Deferral Extension Requested 08/05/2021. Denied per FDA letter dated 09/17/2021.The final protocol, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, a PREA waiver was requested on 9/25/2025 and denied on 11/17/2025."~5/31/2021 0:00:00~5/22/2026 0:00:00
384548~"CDER"~"NDA"~21957.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Nexium (Esomeprazole Magnesium)"~10/20/2006 0:00:00~"Original"~1~"59-1"~"PMR"~"Pediatric Research Equity Act"~59.00~1~"PMR 59-1: Deferred pediatric study under PREA for the treatment of Gastroesophageal Reflux Disease (GERD): Healing of Erosive Esophagitis, Maintenance of Healing of Erosive Esophagitis, Symptomatic Gastroesophageal Reflux Disease in pediatric patients ages birth to 11 years old."~"Delayed"~"Deferral extension denied on 8/15/2019 and a noncompliance letter was issued on 8/21/2019.  The final protocol was acknowledged on 7/1/2021."~6/30/2008 0:00:00~12/10/2024 0:00:00
384549~"CDER"~"NDA"~21964.00~"SALIX PHARMACEUTICALS INC"~"Relistor (Methylnaltrexone Bromide)"~4/24/2008 0:00:00~"Supplement"~10~"2787-1"~"PMR"~"505 (o)(3)"~2787.00~1~"PMR 2787-1: A post-marketing, observational epidemiologic study comparing Relistor (methylnaltrexone bromide) to other treatments of opioid induced constipation in  patients with chronic non-cancer pain. The study s primary outcome is a composite of major adverse cardiovascular events (MACE): cardiovascular (CV) death, nonfatal myocardial infarction, and nonfatal stroke. Secondary outcomes  include, but are not limited to, CV death, nonfatal myocardial infarction, and nonfatal stroke separately. Specify concise case definitions and validation algorithms for the primary and secondary outcomes. Justify the choice of  appropriate comparator population(s) and estimated background rate(s) relative to Relistor (methylnaltrexone bromide)-exposed patients; clearly define the primary comparator population for the primary objective. Design the study around a testable hypothesis to assess, with sufficient sample size and power, MACE risk among Relistor (methylnaltrexone bromide) users relative to comparator(s)  considering important potential confounders including lifestyle risk factors and over the counter (OTC) medications with potential for cardiovascular effects, with  a pre-specified statistical analysis method. For the Relistor (methylnaltrexone bromide) -exposed and comparator(s), clearly define the new user clean period,  including any exclusion and inclusion criteria. Ensure an adequate number of patients with at least 12 months of Relistor (methylnaltrexone bromide) exposure at the end of the study."~"Delayed"~"The study completion and final report submission milestones were missed because of changes to the protocol that expanded eligibility criteria in order to enhance accrual. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 04/18/2023."~3/31/2023 0:00:00~6/23/2026 0:00:00
384550~"CDER"~"NDA"~22020.00~"WYETH PHARMACEUTICALS LLC"~"Protonix (Pantoprazole Sodium)  "~11/14/2007 0:00:00~"Original"~1~"2063-1"~"PMR"~"Pediatric Research Equity Act"~2063.00~1~"PMR 2063-1: Deferred study under PREA to evaluate the pharmacokinetics,  pharmacodynamics, and safety of pantoprazole in patients 1 month to 11 months of age with erosive esophagitis (EE)."~"Terminated"~"Applicant terminated the Study for business reasons."~11/30/2018 0:00:00~4/1/2026 0:00:00
384551~"CDER"~"BLA"~125388.00~"Seagen Inc."~"Adcetris (Brentuximab Vedotin)"~8/19/2011 0:00:00~"Supplement"~99~"3536-1"~"PMC"~"506B PMC"~3536.00~1~"PMC 3536-1: To develop, in consultation with CDRH, a clinically validated in-vitro diagnostic device for the selection of patients with CD30-expressing peripheral T-cell lymphomas (not including systemic anaplastic large cell lymphoma) for treatment with brentuximab vedotin in combination with CHP (cyclophosphamide, doxorubicin, prednisone) chemotherapy."~"Fulfilled"~~11/30/2021 0:00:00~10/16/2025 0:00:00
384552~"CDER"~"BLA"~125390.00~"Chiesi USA, Inc."~"Myalept (metreleptin)"~2/24/2014 0:00:00~"Original"~1~"2456-1"~"PMR"~"505 (o)(3)"~2456.00~1~"PMR 2456-1: A long-term prospective observational study (product exposure registry) of patients treated with Myalept (metreleptin), regardless of indication, to evaluate serious risks related to the use of Myalept (metreleptin), by indication, including: fatal or necrotizing pancreatitis, hepatic adverse events, severe hypoglycemia, serious hypersensitivity reactions, serious infections resulting in hospitalization or death, new diagnoses of autoimmune disorders (for instance, autoimmune hepatitis, glomerulonephritis, lupus erythematosus, antiphospholipid antibody syndrome, rheumatoid arthritis), autoimmune disease exacerbation, lymphoma, all cancers (excluding non-melanoma skin cancer) by cancer type , exposed pregnancies and pregnancy outcomes, and all deaths (including causes of death). After agreement with FDA on a targeted sample size, the registry will include patients prescribed Myalept (metreleptin) and will continue for 10 years from the date of last patient s enrollment, or September 2029, whichever is later."~"Ongoing"~~3/31/2030 0:00:00~4/24/2026 0:00:00
384553~"CDER"~"BLA"~125390.00~"Chiesi USA, Inc."~"Myalept (metreleptin)"~2/24/2014 0:00:00~"Original"~1~"2456-4"~"PMR"~"505 (o)(3)"~2456.00~4~"PMR 2456-4: To conduct a study to assess for the immunogenicity of Myalept (metreleptin) in a relevant number of patients receiving metreleptin. The study should include testing for anti-metreleptin and anti-native human leptin binding antibodies at times when antibody responses peak, using a validated assay. The presence of neutralizing antibodies should be assessed using a validated cell-based assay and a validated ligand-binding assay in samples that are confirmed positive for binding antibodies to leptin. All patients with suspected loss of metreleptin efficacy (e.g., worsening glycemic control, increases in triglycerides) or loss of endogenous leptin activity (e.g., severe infections) should be tested for neutralizing activity and followed at least until antibody levels revert to baseline. Antibody titers, neutralizing activity, and associated clinical events should be characterized over time."~"Fulfilled"~~12/31/2022 0:00:00~4/24/2026 0:00:00
384554~"CDER"~"BLA"~125409.00~"Genentech, Inc."~"Perjeta (Pertuzumab)"~6/8/2012 0:00:00~"Supplement"~118~"3312-1"~"PMC"~"506B PMC"~3312.00~1~"PMC 3312-1: Submit the overall survival (OS) data and analysis with a final report from the clinical trial APHINITY BIG 4 11/BO25126/TOC4939g clinical trial entitled  A randomized  multicenter, double-blind, placebo-controlled comparison of chemotherapy plus trastuzumab plus placebo versus chemotherapy plus trastuzumab plus pertuzumab as adjuvant therapy in patients with operable HER2-positive primary breast cancer. "~"Fulfilled"~~6/30/2024 0:00:00~7/16/2025 0:00:00
384555~"CDER"~"BLA"~125460.00~"Biomarin Pharmaceutical Inc."~"Vimizim (Elosulfase Alfa)"~2/14/2014 0:00:00~"Original"~1~"2337-1"~"PMR"~"505 (o)(3)"~2337.00~1~"PMR 2337-1: Evaluate the long-term safety of Vimizim in adult and pediatric patients enrolled in the Morquio A Registry for a period of ten years, including but not limited to the occurrence of serious hypersensitivity reactions, anaphylaxis, and changes in antibody status (i.e., detection and titers of binding and neutralizing antibodies, and detection of IgE antibodies). Pregnancy exposure data, including maternal, neonatal and infant outcomes, will also be collected and analyzed. Include incidence rate calculations as part of long-term safety evaluation assessments to monitor and characterize risk of exposure to Vimizim. In addition, assessment of clinical outcomes (e.g., anthropometric measures, progression of skeletal deformities, frequency and time to orthopedic surgeries) will be performed. All safety, immunogenicity, and clinical outcome assessments will be conducted every 6 months. Patients previously enrolled in clinical trials MOR-005 and MOR-007 may be rolled over to this study but will be monitored using the MOR005 and MOR-007 protocols, respectively."~"Fulfilled"~~3/31/2025 0:00:00~4/11/2025 0:00:00
384556~"CDER"~"BLA"~125469.00~"Eli Lilly and Company"~"Trulicity (Dulaglutide)"~9/18/2014 0:00:00~"Original"~1~"4744-1"~"PMR"~"505 (o)(3)"~4744.00~1~"PMR 4744-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of dulaglutide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Pending"~~11/30/2026 0:00:00~11/17/2025 0:00:00
384557~"CDER"~"BLA"~125469.00~"Eli Lilly and Company"~"Trulicity (Dulaglutide)"~9/18/2014 0:00:00~"Original"~1~"2781-3"~"PMR"~"505 (o)(3)"~2781.00~3~"PMR 2781-3: A medullary thyroid carcinoma registry-based case series of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid  carcinoma in the United States and to identify any increase related to the  introduction of Trulicity (dulaglutide) into the marketplace. This study will also  establish a registry of incident cases of medullary thyroid carcinoma and  characterize their medical histories related to diabetes and use of Trulicity  (dulaglutide)."~"Delayed"~"The final protocol submission milestone (June 2015) was missed due to ongoing discussions with FDA and the FDA determined there was good cause for the delay. The applicant submitted a final version of the registry protocol in August 2015, which FDA found to be acceptable. The study is currently ongoing and the applicant has been submitting annual status reports for the registry."~3/31/2032 0:00:00~11/17/2025 0:00:00
384558~"CDER"~"BLA"~125469.00~"Eli Lilly and Company"~"Trulicity (Dulaglutide)"~9/18/2014 0:00:00~"Supplement"~36~"3931-1"~"PMR"~"Pediatric Research Equity Act"~3931.00~1~"PMR 3931-1: Conduct a Phase 3, open-label, multicenter, single-arm study to evaluate the safety and PK of dulaglutide (3.0 mg and/or 4.5 mg) in pediatric patients 10 to <18 years of age with T2DM of at least 26 weeks duration."~"Ongoing"~"3 of 55 enrolled."~6/30/2028 0:00:00~11/17/2025 0:00:00
384559~"CDER"~"BLA"~125476.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Entyvio (vedolizumab)"~5/20/2014 0:00:00~"Original"~1~"2719-28"~"PMR"~"Pediatric Research Equity Act"~2719.00~28~"PMR 2719-28: A randomized, controlled, multicenter study to evaluate the efficacy and safety of vedolizumab in pediatric patients 2 to 17 years of age with moderately to severely active Crohn s disease."~"Submitted"~"The final report was submitted to FDA on 03/31/2026."~3/31/2027 0:00:00~7/15/2025 0:00:00
384560~"CDER"~"BLA"~125476.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Entyvio (vedolizumab)"~5/20/2014 0:00:00~"Original"~1~"2719-29"~"PMR"~"Pediatric Research Equity Act"~2719.00~29~"PMR 2719-29: A randomized, controlled, multicenter study to evaluate the efficacy and safety of vedolizumab in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis."~"Submitted"~"The final report was submitted to FDA on 03/31/2026."~5/31/2026 0:00:00~7/15/2025 0:00:00
384561~"CDER"~"NDA"~205488.00~"ACERUS PHARMACEUTICALS CORP"~"Natesto (Testosterone)"~5/28/2014 0:00:00~"Original"~1~"3749-1"~"PMR"~"505 (o)(3)"~3749.00~1~"PMR 3749-1: 24-hour Ambulatory Blood Pressure Monitoring (ABPM) trial to assess whether Natesto increases blood pressure (BP) in hypogonadal men."~"Delayed"~"The revised final report milestone was missed and FDA determined that there was not good cause for the noncompliance in a letter issued on 02/20/2025. Per the Applicant s annual report submitted on 3/21/2025, the Applicant has reached a resolution with the cardiac safety services vendor and is working to submit the final report as quickly as possible."~3/31/2021 0:00:00~7/25/2025 0:00:00
384562~"CDER"~"NDA"~205489.00~"NEOS THERAPEUTICS INC"~"Cotempla XR-ODT (Methylphenidate)"~6/19/2017 0:00:00~"Original"~1~"3222-4"~"PMR"~"Pediatric Research Equity Act"~3222.00~4~"PMR 3222-4: Conduct a randomized, double-blind, placebo-controlled safety and efficacy study of Cotempla XR-ODT (methylphenidate extended-release orally disintegrating tablets) in children ages 4 to less than 6 years diagnosed with ADHD, followed by a 6-month, open-label extension period to obtain additional information regarding safety and tolerability."~"Released"~"Per FDA letter dated 12/15/2025, this PMR has been released."~8/31/2023 0:00:00~8/15/2025 0:00:00
384563~"CDER"~"NDA"~205525.00~"WELLHOUSE PHARMA LLC"~"Syndros (Dronabinol)"~7/1/2016 0:00:00~"Original"~1~"3044-3"~"PMR"~"Pediatric Research Equity Act"~3044.00~3~"PMR 3044-3: Deferred study under PREA to evaluate the pharmacokinetics of Syndros (dronabinol oral solution) for the treatment of chemotherapy induced nausea and vomiting (CINV) in pediatric cancer patients who failed to respond adequately to  conventional antiemetic treatments from birth to 17 years of age."~"Delayed"~"The final report submission milestone was missed and a noncompliance letter was issued on 12/16/2021. The applicant's deferral extension request was denied on 3/14/2022."~11/30/2021 0:00:00~5/13/2026 0:00:00
384564~"CDER"~"NDA"~205525.00~"WELLHOUSE PHARMA LLC"~"Syndros (Dronabinol)"~7/1/2016 0:00:00~"Original"~1~"3044-4"~"PMR"~"Pediatric Research Equity Act"~3044.00~4~"PMR 3044-4: Deferred pediatric study under PREA to evaluate the tolerability and efficacy of Syndros (dronabinol oral solution) for the treatment of chemotherapy induced nausea and vomiting (CINV) in pediatric patients who failed to respond  adequately to conventional antiemetic treatments aged birth to 17 years."~"Delayed"~"The final report submission milestone was missed and a noncompliance letter was issued on 12/16/2021. The applicant's deferral extension request was denied on 3/14/2022."~3/31/2023 0:00:00~5/13/2026 0:00:00
384565~"CDER"~"NDA"~205552.00~"PHARMACYCLICS LLC"~"Imbruvica (Ibrutinib)"~11/13/2013 0:00:00~"Supplement"~30~"3811-1"~"PMC"~"506B PMC"~3811.00~1~"PMC 3811-1: Submit the overall survival analysis and datasets with the final report for clinical trial E1912 titled,  A Randomized Phase III Study of Ibrutinib- Based Therapy vs Standard Fludarabine, Cyclophosphamide, and Rituximab Chemoimmunotherapy in Untreated Younger Patients with Chronic Lymphocytic Leukemia  to provide additional long term efficacy data."~"Ongoing"~~9/30/2027 0:00:00~1/7/2026 0:00:00
384566~"CDER"~"NDA"~205552.00~"PHARMACYCLICS LLC"~"Imbruvica (Ibrutinib)"~11/13/2013 0:00:00~"Supplement"~36~"4327-1"~"PMR"~"505 (o)(3)"~4327.00~1~"PMR 4327-1: Conduct analyses to characterize long-term safety of ibrutinib in terms of growth and development in pediatric patients. Patients enrolled in Study PCYC-1146-IM should be evaluated for growth and development milestones annually for at least 5 years from the initiation of ibrutinib. Data should include growth parameters as measured by height and weight, sexual maturation by Tanner stage, performance status, immune reconstitution, adverse events, and patient-reported outcomes measures."~"Ongoing"~~7/31/2026 0:00:00~1/7/2026 0:00:00
384567~"CDER"~"NDA"~205613.00~"SALIX PHARMACEUTICALS INC"~"Uceris (Budesonide)"~10/7/2014 0:00:00~"Original"~1~"2800-1"~"PMC"~"506B PMC"~2800.00~1~"PMC 2800-1: A 6-week randomized, double blind, placebo-controlled trial in children 5 to 17 years of age with active, mild to moderate distal ulcerative colitis (extending up to 40 cm from the anal verge). The trial will evaluate pharmacokinetics (PK), efficacy for induction of remission, and safety of at least 2 doses of Uceris (budesonide) rectal foam. The effects of 6 weeks of Uceris (budesonide) rectal foam on the HPA axis will be assessed."~"Delayed"~"The final report milestone was missed, and the applicant has
identified a Contract Research Organization to conduct the study.
"~4/30/2018 0:00:00~12/5/2025 0:00:00
384568~"CDER"~"NDA"~205718.00~"HELSINN HEALTHCARE SA"~"Akynzeo (Netupitant and Palonosetron Hydrochloride)"~10/10/2014 0:00:00~"Original"~1~"2769-6"~"PMR"~"Pediatric Research Equity Act"~2769.00~6~"PMR 2769-6: A PK, PD, safety, and tolerability study with single and multi-day administration of oral netupitant/palonosetron fixed combination for the prevention of nausea and vomiting in pediatric patients ages 0 to 17 years undergoing treatment with highly emetogenic chemotherapy. You must conduct this study with an age-appropriate formulation."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~7/31/2030 0:00:00~12/9/2025 0:00:00
384569~"CDER"~"NDA"~205739.00~"VIFOR PHARMA INC"~"Veltassa (Patiromer)"~10/21/2015 0:00:00~"Original"~1~"2980-1"~"PMR"~"Pediatric Research Equity Act"~2980.00~1~"PMR 2980-1: Study 1: A Phase 2, Open-Label, Multiple Dose Study to Evaluate the  Pharmacodynamic Effects, Safety, and Tolerability of Veltassa (Patiromer Sorbitex Calcium) for Oral Suspension in Children and Adolescents 2 to 18 Years of Age with Hyperkalemia"~"Delayed"~"The final report submitted in December 2022 did not meet the terms of the requirement. Per FDA s letter dated 3/12/2024, the applicant s request for release and request for a deferral extension were denied."~9/30/2021 0:00:00~12/17/2025 0:00:00
384570~"CDER"~"NDA"~205437.00~"AMGEN INC"~"Otezla (Apremilast)"~3/21/2014 0:00:00~"Supplement"~11~"4207-1"~"PMR"~"Pediatric Research Equity Act"~4207.00~1~"PMR 4207-1: A Phase 3, multicenter, open-label study to assess the safety of apremilast in approximately 50 pediatric subjects (6 through 17 years of
age, inclusive) with mild-to-moderate plaque psoriasis."~"Ongoing"~"As of 31 March 2025, a total of 40 subjects were enrolled in the study.
"~9/30/2027 0:00:00~5/15/2026 0:00:00
384571~"CDER"~"NDA"~205739.00~"VIFOR PHARMA INC"~"Veltassa (Patiromer)"~10/21/2015 0:00:00~"Original"~1~"2980-2"~"PMR"~"Pediatric Research Equity Act"~2980.00~2~"PMR 2980-2: Study 2: A Phase 2, Open-Label, Multiple Dose Study to Evaluate the  Pharmacodynamic Effects, Safety, and Tolerability of Veltassa (Patiromer Sorbitex Calcium) for Oral Suspension in Infants and Toddlers Under 2 Years of Age with Hyperkalemia"~"Delayed"~"The original final protocol milestone was missed. The final protocol was submitted November 2023. The study is underway.
"~6/30/2031 0:00:00~12/17/2025 0:00:00
384572~"CDER"~"NDA"~208700.00~"ADVANCED ACCELERATOR APPLICATIONS USA INC"~"Lutathera (lutetium Lu 177 dotatate)"~1/26/2018 0:00:00~"Supplement"~31~"4621-2"~"PMR"~"505 (o)(3)"~4621.00~2~"PMR 4621-2: Conduct cumulative, integrated safety analyses after 5 and after 10 years of follow-up from pediatric patients ages 12 to 17 enrolled in the ongoingclinical trial to further characterize the known serious risk of renal failure with Lutathera; include incidence rates, time to onset, predisposing factors and outcomes. These safety evaluations should adequately characterize the serious risk of renal failure in patient populations at highest risk and provide evidence-based dose modifications and monitoring recommendations as appropriate."~"Ongoing"~~1/31/2035 0:00:00~3/24/2026 0:00:00
384573~"CDER"~"NDA"~208711.00~"NOVARTIS PHARMACEUTICALS CORP"~"Egaten (Triclabendazole)"~2/13/2019 0:00:00~"Original"~1~"3556-2"~"PMC"~"506B PMC"~3556.00~2~"PMC 3556-2: Conduct an open-label, single-arm study to assess outcomes of treatment with Egaten 20 mg/kg in patients with fascioliasis."~"Delayed"~"Revised milestones were acknowledged in a letter dated 07/12/2021. Fourth interim report was submitted on 6-2025
"~12/31/2025 0:00:00~4/9/2026 0:00:00
384574~"CDER"~"NDA"~208712.00~"SOBI INC"~"Vonjo (Pacritinib)"~2/28/2022 0:00:00~"Original"~1~"4154-1"~"PMR"~"Accelerated Approval"~4154.00~1~"PMR 4154-1: Conduct a randomized, controlled trial to verify and describe the clinical benefit of Vonjo in adults with intermediate-1, intermediate-2 or high-risk myelofibrosis (MF) [as defined by the Dynamic International Prognostic Scoring System (DIPSS), including primary MF, post-polycythemia vera and post-essential thrombocythemia MF] with platelet counts of less than 50 x 109/L. The co-primary efficacy endpoints are the proportion of patients achieving =35% spleen volume reduction as measured by magnetic resonance imaging or computed tomography imaging; and the proportion of patients achieving a =50% reduction in modified total symptom score (mTSS) [as defined by the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF TSS 2.0) that excludes the  tiredness  component] from baseline through 24 weeks of therapy. Longterm safety outcomes include the risks for bleeding, thrombosis, infections, major adverse cardiac events, secondary malignancies, and survival."~"Delayed"~"The original trial completion milestone was missed. Revised trial completion and final report submission milestones were acknowledged in a letter dated 10/28/2025. Per the applicants 4/6/2026 annual status report, 372 patients out of the planned 399 patients have been enrolled into the trial."~6/30/2026 0:00:00~4/6/2026 0:00:00
384575~"CDER"~"NDA"~208712.00~"SOBI INC"~"Vonjo (Pacritinib)"~2/28/2022 0:00:00~"Original"~1~"4154-4"~"PMR"~"505 (o)(3)"~4154.00~4~"PMR 4154-4: Conduct a dedicated pharmacokinetic trial to evaluate the impact of hepatic impairment on the pharmacokinetics of Vonjo following
administration of Vonjo 200 mg twice daily dosed to steady state in adults with moderate (Child-Pugh B) and severe hepatic impairment (Child-PughC) compared to matched healthy adult subjects."~"Fulfilled"~~12/31/2023 0:00:00~4/6/2026 0:00:00
384576~"CDER"~"NDA"~208716.00~"ELI LILLY AND CO"~"Verzenio (abemaciclib)"~9/28/2017 0:00:00~"Supplement"~6~"4158-1"~"PMC"~"506B PMC"~4158.00~1~"PMC 4158-1: Submit the final overall survival (OS) analysis and datasets with the final report for adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, early breast cancer from the ongoing clinical trial, monarchE, titled  A Randomized, OpenLabel, Phase 3 Study of Abemaciclib Combined With Standard Adjuvant Endocrine Therapy Versus Standard Adjuvant Endocrine Therapy Alone in Patients With High Risk, Node Positive, Early Stage, Hormone Receptor Positive, Human Epidermal Receptor 2 Negative, Breast Cancer "~"Submitted"~~6/30/2025 0:00:00~11/25/2025 0:00:00
384577~"CDER"~"NDA"~208745.00~"SALIX PHARMACEUTICALS INC"~"Trulance (Plecanatide)"~1/19/2017 0:00:00~"Original"~1~"3117-1"~"PMR"~"Pediatric Research Equity Act"~3117.00~1~"PMR 3117-1: Determine the appropriate Trulance (plecanatide) treatment dose for pediatric patients with chronic idiopathic constipation (CIC) who are 12 years to less than 18 years of age by assessing the safety and efficacy of once daily oral plecanatide in an eight (8)  week, proof-of-concept, dose-ranging with sparse pharmacokinetic (PK) sampling study."~"Delayed"~"The original final report submission milestone was missed 2/28/2022. Deferral Extension granted per FDA letter dated 11/29/2022."~3/31/2026 0:00:00~3/20/2026 0:00:00
384578~"CDER"~"NDA"~208745.00~"SALIX PHARMACEUTICALS INC"~"Trulance (Plecanatide)"~1/19/2017 0:00:00~"Original"~1~"3117-2"~"PMR"~"Pediatric Research Equity Act"~3117.00~2~"PMR 3117-2: Determine the appropriate Trulance (plecanatide) treatment dose for pediatric patients with chronic idiopathic constipation (CIC) who are 6 years to less than 12 years of age by assessing the safety and efficacy of once daily oral plecanatide in an eight (8) week,  proof-of-concept, dose-ranging with sparse pharmacokinetic (PK) sampling study."~"Delayed"~"Original Final Report Due Date: 12/31/2021. Per FDA letter dated 11/29/2022, Final Report due date extended to 03/31/2025. Second Deferral Extension Granted per FDA letter dated 04/10/2025.
"~3/31/2026 0:00:00~3/20/2026 0:00:00
384579~"CDER"~"NDA"~208745.00~"SALIX PHARMACEUTICALS INC"~"Trulance (Plecanatide)"~1/19/2017 0:00:00~"Original"~1~"3117-3"~"PMR"~"Pediatric Research Equity Act"~3117.00~3~"PMR 3117-3: Confirm the efficacy and safety of Trulance (plecanatide) in pediatric patients with chronic idiopathic constipation (CIC) who are 6 years to less than 18 years of age by  performing a randomized, double-blind, placebo-controlled, parallel group, 12 week treatment study."~"Delayed"~"The original final report submission milestone was missed 2/28/2022. Deferral Extension granted per FDA letter dated 11/29/2022."~8/31/2027 0:00:00~3/20/2026 0:00:00
384580~"CDER"~"NDA"~208745.00~"SALIX PHARMACEUTICALS INC"~"Trulance (Plecanatide)"~1/19/2017 0:00:00~"Original"~1~"3117-4"~"PMR"~"Pediatric Research Equity Act"~3117.00~4~"PMR 3117-4: Determine the appropriate Trulance (plecanatide) treatment dose for pediatric patients with chronic idiopathic constipation (CIC) who are 2 years to less than 6 years of age by assessing the safety and efficacy of once daily oral plecanatide in an eight (8) week,  proof-of-concept, dose-ranging with sparse pharmacokinetic (PK) sampling study."~"Delayed"~"Original Final Report Due Date: 02/28/2023; Deferral Extension granted per FDA letter dated 06/21/2023. The final protocol submission and study completion milestones were also revised.
"~10/31/2027 0:00:00~3/20/2026 0:00:00
384581~"CDER"~"NDA"~208700.00~"ADVANCED ACCELERATOR APPLICATIONS USA INC"~"Lutathera (lutetium Lu 177 dotatate)"~1/26/2018 0:00:00~"Supplement"~31~"4621-1"~"PMR"~"505 (o)(3)"~4621.00~1~"PMR 4621-1: Conduct cumulative, integrated safety analyses after 5 and after 10 years of follow-up from pediatric patients ages 12 to 17 enrolled in the ongoing clinical trial to further characterize the known serious risks of myelodysplastic syndrome (MDS) and acute leukemia with Lutathera; include incidence rates, time to onset, predisposing factors and outcomes. These safety evaluations should adequately characterize the serious risks of MDS and acute leukemia in patient populations at highest risk and provide evidence-based dose modifications and monitoring recommendations as appropriate."~"Ongoing"~~1/31/2035 0:00:00~3/24/2026 0:00:00
384582~"CDER"~"NDA"~22020.00~"WYETH PHARMACEUTICALS LLC"~"Protonix (Pantoprazole Sodium)  "~11/14/2007 0:00:00~"Original"~1~"2063-3"~"PMR"~"Pediatric Research Equity Act"~2063.00~3~"PMR 2063-3: Deferred study under PREA to evaluate the pharmacokinetics, healing,  maintenance of healing, and symptoms of erosive esophagitis (EE) in patients  12 years to 17 years of age."~"Terminated"~"Applicant terminated the Study for business reasons."~10/31/2018 0:00:00~4/1/2026 0:00:00
384583~"CDER"~"NDA"~22041.00~"BTG INTERNATIONAL INC"~"Cyanokit [Hydroxocabalamin (Vitamin B12a)]"~12/15/2006 0:00:00~"Original"~1~"6-1"~"PMR"~"Animal Efficacy"~6.00~1~"PMR 6-1: To conduct a study in both adult and pediatric patients to verify and describe the clinical benefit of Cyanokit and to assess its safety when used as indicated."~"Submitted"~~2/1/2009 0:00:00~2/12/2026 0:00:00
384584~"CDER"~"NDA"~22066.00~"GE HEALTHCARE INC"~"Omniscan (Gadodiamide)"~9/5/2007 0:00:00~"Supplement"~8~"3627-4"~"PMR"~"505 (o)(3)"~3627.00~4~"PMR 3627-4: A prospective longitudinal cohort trial with one or more matched control group(s) to evaluate the effects of repetitive GBCA administration on a comprehensive battery of neurobehavioral testing over the course of at least five administrations. The trial should be sufficiently powered to exclude a pre-specified magnitude of decline. As a secondary objective, trial patients should also have the option of providing blood and urine samples at the time of reimaging, so that normative estimates of gadolinium concentration across an extended range of post-administration timepoints may be documented."~"Ongoing"~~4/30/2029 0:00:00~3/3/2026 0:00:00
384585~"CDER"~"NDA"~22068.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tasigna (Nilotinib) "~10/29/2007 0:00:00~"Supplement"~26~"3323-2"~"PMR"~"505 (o)(3)"~3323.00~2~"PMR 3323-2: Characterize the potential risk of resistance to treatment after discontinuation of Tasigna by collecting and reporting information on late relapses, loss of response,  and occurrence of mutations (risk of developing resistance), on a yearly basis from trials CAMN107A2408 and CAMN10712201. Provide gene expression profile information for patients that relapse from treatment-free period compared to patients who relapse on treatment in the 5-year trial reports. Submit annual summaries with the annual post-marketing reporting and submit cumulative 3, 5, and 10 year reports."~"Fulfilled"~~2/28/2026 0:00:00~12/23/2025 0:00:00
384586~"CDER"~"NDA"~22068.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tasigna (Nilotinib) "~10/29/2007 0:00:00~"Supplement"~26~"3323-3"~"PMR"~"505 (o)(3)"~3323.00~3~"PMR 3323-3: Characterize safety for patients who are still in remission or experienced loss of major molecular response and restarted Tasigna in trials CAMN107A2408 and CAMN10712201. Assess mortality, late adverse events, incidence of pancreatitis, diabetes, arterial vascular disease, and other comorbid conditions. Submit annual summaries with the annual post-marketing reporting and submit cumulative 3, 5, and 10 year reports."~"Fulfilled"~~2/28/2026 0:00:00~12/23/2025 0:00:00
384587~"CDER"~"NDA"~22090.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Eovist (Gadoxetate Disodium) "~7/3/2008 0:00:00~"Supplement"~14~"3624-4"~"PMR"~"505 (o)(3)"~3624.00~4~"PMR 3624-4: A prospective longitudinal cohort trial with one or more matched control group(s) to evaluate the effects of repetitive GBCA administration on a comprehensive battery of neurobehavioral testing over the course of at least five administrations. The trial should be sufficiently powered to exclude a pre-specified magnitude of decline. As a secondary objective, trial patients should also have the option of providing blood and urine samples at the time of reimaging, so that normative estimates of gadolinium concentration across an extended range of post-administration timepoints may be documented."~"Ongoing"~~4/30/2029 0:00:00~8/29/2025 0:00:00
384588~"CDER"~"NDA"~22108.00~"BAUSCH HEALTH US LLC"~"Aplenzin (Bupropion Hydrobromide)"~4/23/2008 0:00:00~"Original"~1~"133-1"~"PMR"~"Pediatric Research Equity Act"~133.00~1~"PMR 133-1: Deferred pediatric study under PREA for the treatment of Major Depressive Disorder in pediatric patients ages 7 to 17."~"Delayed"~"The final report submission milestone was missed. The Clinical Research Organization selection has been completed. The Sponsor intends to submit the revised protocols and start site initiation activities during the upcoming reporting period."~2/28/2019 0:00:00~6/22/2026 0:00:00
384589~"CDER"~"NDA"~22156.00~"CHIESI USA INC"~"Cleviprex (Clevidipine Butyrate)"~8/1/2008 0:00:00~"Original"~1~"1258-1"~"PMR"~"Pediatric Research Equity Act"~1258.00~1~"PMR 1258-1: Deferred pediatric studies under PREA for the reduction of blood pressure in pediatric patients ages 1 to 16 years old."~"Delayed"~"A Partial Clinical Hold Letter was sent in July 2022 that prevents the applicant from initiating or resuming the study until the required information is submitted."~8/1/2011 0:00:00~9/30/2025 0:00:00
384590~"CDER"~"NDA"~22165.00~"COSETTE PHARMACEUTICALS INC"~"Cambia (Diclofenac Potassium)"~6/17/2009 0:00:00~"Original"~1~"974-7"~"PMR"~"Pediatric Research Equity Act"~974.00~7~"PMR 974-7: Deferred controlled safety and efficacy study under PREA for the acute treatment of migraine with or without aura in pediatric patients age 6-17 years old with a PK run-in  phase and a 12 month open-label long-term safety extension."~"Delayed"~"Deferral Extension Requested 12/07/2022. Denied per FDA letter dated 01/23/2023. The Final Protocol, Study Completion, and Final Report Submission milestones were missed. Per the applicant s Annual Status Report, the study is pending initiation of subject enrollment."~12/31/2021 0:00:00~7/29/2025 0:00:00
384591~"CDER"~"NDA"~22180.00~"AZURITY PHARMACEUTICALS SWITZERLAND GMBH"~"Feraheme (ferumoxytol)"~6/30/2009 0:00:00~"Original"~1~"24-1"~"PMR"~"Pediatric Research Equity Act"~24.00~1~"PMR 24-1: To conduct a clinical trial in pediatric patients aged 2 to < 18 years who have iron deficiency anemia and who are receiving either hemodialysis or peritoneal dialysis. In addition to any other items, the trial will obtain pharmacokinetic (PK), pharmacodynamic (PD) and safety data from at least 50 patients exposed to ferumoxytol. In this trial, patients will be randomized to oral iron (25 patients) or one of two dose ferumoxytol dose regimens (25 patients in each dose cohort). Endpoints will consist of PK, PD, comparisons of hemoglobin changes and safety summaries."~"Delayed"~"The final report was missed due to delays in study enrollment. Original Final Report Due Date: 03/2017; Deferral Extension granted per FDA letter dated 11/26/2024. Subsequent Deferral Extension Requested 10/31/2025. Denied per FDA letter dated 12/15/2025."~9/30/2026 0:00:00~8/29/2025 0:00:00
384592~"CDER"~"NDA"~22180.00~"AZURITY PHARMACEUTICALS SWITZERLAND GMBH"~"Feraheme (ferumoxytol)"~6/30/2009 0:00:00~"Original"~1~"24-2"~"PMR"~"Pediatric Research Equity Act"~24.00~2~"PMR 24-2: To conduct a clinical trial in pediatric patients aged 2 to < 18 years who have iron deficiency anemia and chronic kidney disease that does not require dialysis. In addition to any other items, the trial will obtain pharmacokinetic (PK), pharmacodynamic (PD) and safety data from at least 50 patients exposed to ferumoxytol. In this trial, patients will be randomized to oral iron (25 patients) or one of two dose ferumoxytol dose regimens (25 patients in each dose cohort). Endpoints will consist of PK, PD, comparisons of hemoglobin changes and safety summaries."~"Delayed"~"The final report was missed due to delays in study enrollment. Original Final Report Due Date: 03/2017; Deferral Extension granted per FDA letter dated 11/26/2024. Subsequent Deferral Extension Requested 10/31/2025. Denied per FDA letter dated 12/15/2025."~9/30/2026 0:00:00~8/29/2025 0:00:00
384593~"CDER"~"NDA"~22020.00~"WYETH PHARMACEUTICALS LLC"~"Protonix (Pantoprazole Sodium)  "~11/14/2007 0:00:00~"Original"~1~"2063-2"~"PMR"~"Pediatric Research Equity Act"~2063.00~2~"PMR 2063-2: Deferred study under PREA to evaluate the pharmacokinetics, healing, maintenance of healing, and symptoms of erosive esophagitis (EE) in patients  1 year to 11 years of age."~"Terminated"~"Applicant terminated the Study for business reasons."~10/31/2018 0:00:00~4/1/2026 0:00:00
384594~"CDER"~"BLA"~125476.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Entyvio (vedolizumab)"~5/20/2014 0:00:00~"Supplement"~21~"2719-28"~"PMR"~"Pediatric Research Equity Act"~2719.00~28~"PMR 2719-28: A randomized, controlled, multicenter study to evaluate the efficacy and safety of vedolizumab in pediatric patients 2 to 17 years of age with moderately to severely active Crohn s disease."~"Submitted"~"The final report was submitted to FDA on 03/31/2026."~3/31/2027 0:00:00~7/15/2025 0:00:00
384595~"CDER"~"BLA"~125476.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Entyvio (vedolizumab)"~5/20/2014 0:00:00~"Supplement"~21~"2719-29"~"PMR"~"Pediatric Research Equity Act"~2719.00~29~"PMR 2719-29: A randomized, controlled, multicenter study to evaluate the efficacy and safety of vedolizumab in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis."~"Submitted"~"The final report was submitted to FDA on 03/31/2026."~5/31/2026 0:00:00~7/15/2025 0:00:00
384596~"CDER"~"BLA"~125486.00~"Genentech, Inc."~"Gazyva (obinutuzumab)"~11/1/2013 0:00:00~"Supplement"~37~"4887-1"~"PMR"~"Pediatric Research Equity Act"~4887.00~1~"PMR 4887-1: Conduct a multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of obinutuzumab in pediatric patients ages 12 to <18 years with proliferative lupus nephritis (Class III and IV) receiving standard of care therapy"~"Ongoing"~"The study is underway."~12/31/2027 0:00:00~12/19/2025 0:00:00
384597~"CDER"~"BLA"~125486.00~"Genentech, Inc."~"Gazyva (obinutuzumab)"~11/1/2013 0:00:00~"Supplement"~37~"4887-2"~"PMR"~"Pediatric Research Equity Act"~4887.00~2~"PMR 4887-2: Provide pharmacokinetic and safety information to support the pediatric assessment of obinutuzumab for the treatment of pediatric patients ages 5 to <12 years with proliferative lupus nephritis (Class III and IV) receiving standard of care therapy."~"Ongoing"~"The study is underway."~6/30/2029 0:00:00~12/19/2025 0:00:00
384598~"CDER"~"BLA"~125486.00~"Genentech, Inc."~"Gazyva (obinutuzumab)"~11/1/2013 0:00:00~"Supplement"~38~"4887-1"~"PMR"~"Pediatric Research Equity Act"~4887.00~1~"PMR 4887-1: Conduct a multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of obinutuzumab in pediatric patients ages 12 to <18 years with proliferative lupus nephritis (Class III and IV) receiving standard of care therapy"~"Ongoing"~"The study is underway."~12/31/2027 0:00:00~12/19/2025 0:00:00
384599~"CDER"~"BLA"~125486.00~"Genentech, Inc."~"Gazyva (obinutuzumab)"~11/1/2013 0:00:00~"Supplement"~38~"4887-2"~"PMR"~"Pediatric Research Equity Act"~4887.00~2~"PMR 4887-2: Provide pharmacokinetic and safety information to support the pediatric assessment of obinutuzumab for the treatment of pediatric patients ages 5 to <12 years with proliferative lupus nephritis (Class III and IV) receiving standard of care therapy."~"Ongoing"~"The study is underway."~6/30/2029 0:00:00~12/19/2025 0:00:00
384600~"CDER"~"BLA"~125504.00~"Novartis Pharmaceuticals Corporation"~"Cosentyx Unoready Pen (secukinumab)"~1/21/2015 0:00:00~"Original"~1~"2848-2"~"PMR"~"505 (o)(3)"~2848.00~2~"PMR 2848-2: A postmarketing prospective, long-term, observational study to assess the longterm safety of secukinumab compared to other therapies used in the treatment of  adults with moderate to severe plaque psoriasis who are candidates for systemic  therapy or phototherapy in a real world clinical setting. The study s primary outcome is malignancies. Describe and justify the choice of appropriate comparator population(s). Design the study around a testable hypothesis to assess,  with sufficient sample size and power, a clinically meaningful increase in malignancy risk above the comparator background rate. Specify concise case  definitions and validation algorithms for the primary outcome. Enroll patients over an initial 4-year period and follow for a minimum of 8 years from the time of enrollment. Provide progress updates on registry patient accrual and demographic  summary data in your Annual Report, and provide registry safety data in your Periodic Benefit-Risk Evaluation Reports (PBERs) for the reporting period as well as cumulatively, and a complete final study report."~"Delayed"~"4017 of the planned 3000 have been enrolled into the study. The 03/2015 final protocol submission was missed
due to low enrollment projections. The Applicant requested
revised milestones on 7/27/18. The Agency issued revised
milestones on 9/26/25."~6/30/2030 0:00:00~2/19/2026 0:00:00
384601~"CDER"~"BLA"~125504.00~"Novartis Pharmaceuticals Corporation"~"Cosentyx Unoready Pen (secukinumab)"~1/21/2015 0:00:00~"Supplement"~63~"4543-1"~"PMR"~"Pediatric Research Equity Act"~4543.00~1~"PMR 4543-1: Conduct an open-label study to evaluate the pharmacokinetics (PK) and safety in the adolescent population 12 to less than 17 years of age with moderate to severe hidradenitis suppurativa who are candidates for systemic treatment. Evaluate at least 80 subjects exposed to the highest approved dosage of subcutaneous secukinumab (300 mg every 2 weeks) for a minimum of 52 weeks."~"Released"~"Per FDA letter dated 02/27/2026, this PMR/PMC has been released."~2/28/2030 0:00:00~2/19/2026 0:00:00
384602~"CDER"~"BLA"~125504.00~"Novartis Pharmaceuticals Corporation"~"Cosentyx Unoready Pen (secukinumab)"~1/21/2015 0:00:00~"Supplement"~63~"4543-2"~"PMR"~"Pediatric Research Equity Act"~4543.00~2~"PMR 4543-2: Conduct an assessment of the pharmacokinetics (PK), efficacy, and safety of secukinumab administered subcutaneously in adolescents from 12 to less than 17 years of age with moderate to severe hidradenitis suppurativa who are candidates for systemic treatment based on available date in adult and pediatric patients."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~3/31/2026 0:00:00~2/19/2026 0:00:00
384603~"CDER"~"BLA"~125509.00~"Elusys Therapeutics, Inc."~"Anthim (Obiltoxaximab)"~3/18/2016 0:00:00~"Original"~1~"3050-1"~"PMR"~"Animal Efficacy"~3050.00~1~"PMR 3050-1: Conduct a Field Study to evaluate the clinical response, pharmacokinetics, and safety profile of Anthim (obiltoxaximab) when used in the treatment of suspected  or confirmed cases of inhalational anthrax due to B. anthracis in the United States."~"Pending"~~~5/8/2026 0:00:00
384604~"CDER"~"BLA"~125511.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Natpara (Parathyroid Hormone) "~1/23/2015 0:00:00~"Original"~1~"2856-2"~"PMR"~"505 (o)(3)"~2856.00~2~"PMR 2856-2: An enhanced pharmacovigilance study of osteosarcoma in patients with hypoparathyroidism treated with Natpara (parathyroid hormone). The study will  include reports of osteosarcoma for a period of 15 years from the date of  approval, and will include assessment and analysis of spontaneous reports of  osteosarcoma in patients treated with Natpara (parathyroid hormone), with specialized follow-up to collect additional information on these cases."~"Ongoing"~~9/30/2030 0:00:00~3/18/2025 0:00:00
384605~"CDER"~"BLA"~125511.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Natpara (Parathyroid Hormone) "~1/23/2015 0:00:00~"Original"~1~"2856-4"~"PMR"~"505 (o)(3)"~2856.00~4~"PMR 2856-4: A 26-week randomized, controlled clinical trial to evaluate the longer term safety and effect of an alternative dose(s) and/or dosing regimen(s) of Natpara  (parathyroid hormone), including longer term safety with respect to  hypercalciuria. This trial should not be initiated until the results from the clinical pharmacology trial (PMR 2856-3) and the nonclinical rat study (PMR 2856-1)  have been submitted to and reviewed by the Agency."~"Delayed"~"PMR 2856-4 is delayed because the original milestones were missed. The Acknowledge Final Protocol letter was issued on May 3, 2022. The revised trial completion milestone was acknowledged with good cause in a letter dated March 24,2022."~5/31/2022 0:00:00~3/18/2025 0:00:00
384606~"CDER"~"NDA"~205750.00~"MIRUM PHARMACEUTICALS INC"~"Cholbam (Cholic Acid)"~3/17/2015 0:00:00~"Original"~1~"2882-1"~"PMR"~"505 (o)(3)"~2882.00~1~"PMR 2882-1: A prospective, long-term, observational study in a routine clinical setting of patients aged 3 weeks or older with bile acid synthesis disorders due to single  enzyme defects and patients with peroxisomal disorders, including Zellweger spectrum disorders, who exhibit manifestations of liver disease, steatorrhea, fat  soluble vitamin deficiency, or a neuropathic process related to a vitamin deficiency. The purpose of the study is to assess primarily the long term safety of treatment with Cholbam (cholic acid) capsules with respect to incidence rates of  worsening cholestasis, steatorrhea leading to poor growth, fat soluble vitamin deficiency, or neuropathic process related to a vitamin deficiency, and the  incidence of death and adverse effects on pregnancy, pregnancy outcomes and infant status. Additional evaluations will include dosing regimens and reasons for  any dose modifications, weight gain, length/height and developmental outcomes.  Specify concise case definitions and validation algorithms for all outcomes. Enroll at least 55 patients (25 receiving cholic acid and 30 not receiving cholic  acid) including at least 20 peroxisomal disorder patients with liver dysfunction,  steatorrhea, fat soluble vitamin deficiency or a neuropathic process related to the  latter. Enroll over an initial 3-year period and follow for a minimum of 10 years from the time of enrollment or until death, whichever comes first."~"Delayed"~"79 patients have been enrolled. The final protocol was submitted after the milestone. Enrollment is slow due to rarity of patients with disease for which the drug is indicated."~3/31/2029 0:00:00~5/13/2026 0:00:00
384607~"CDER"~"NDA"~205874.00~"KERYX BIOPHARMACEUTICALS INC"~"Auryxia (Ferric Citrate)"~9/5/2014 0:00:00~"Original"~1~"3153-2"~"PMR"~"Pediatric Research Equity Act"~3153.00~2~"PMR 3153-2: A multicenter clinical trial to evaluate the dosing and safety of ferric citrate for the treatment of hyperphosphatemia in children 2 to <18 years of age with chronic kidney disease."~"Ongoing"~"Sponsor submitted a request release from PMR 3153-2. On 27 August 2025, FDA denied this request and acknowledged the challenges with enrollment and retention. FDA suggested that Sponsor halt further enrollment in the KRX-0502-308 trial and request a meeting with the Division of Cardiology and Nephrology to discuss the topline results of the study, based on the data collected to date."~4/30/2024 0:00:00~10/30/2025 0:00:00
384608~"CDER"~"NDA"~206030.00~"LUNDBECK PHARMACEUTICALS LLC"~"Carnexiv (Carbamazepine)"~10/7/2016 0:00:00~"Original"~1~"4084-1"~"PMR"~"505 (o)(3)"~4084.00~1~"PMR 4084-1: Conduct in vitro patch clamp studies to assess Carnexiv s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Carnexiv s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~2/26/2026 0:00:00
384609~"CDER"~"NDA"~206089.00~"TOLMAR INC"~"Jatenzo (testosterone undecanoate)"~3/27/2019 0:00:00~"Original"~1~"3582-2"~"PMR"~"505 (o)(3)"~3582.00~2~"PMR 3582-2: An appropriately designed one-year trial to evaluate for the development of adrenal insufficiency with chronic Jatenzo therapy. Assess adrenal function with Cosyntropin stimulation testing prior to starting Jatenzo, and again after six months and one year on Jatenzo. Test at earlier timepoints for subjects who demonstrate signs or symptoms consistent with adrenal insufficiency. Assess serum cortisol, adrenocorticotropic hormone, and corticosteroid binding globulin concentrations prior to Cosyntropin 0.25 mg injection and serum cortisol concentrations at 30 minutes and 60 minutes after the injection. Standardize the testing time to 8 AM and the route of Cosyntropin administration (intramuscular or intravenous). Perform hormonal analytical assays in a central laboratory on batched serum samples. Use appropriate serum cortisol criteria to interpret results as normal and provide an algorithm for managing abnormal test results."~"Delayed"~"The original final report submission milestone was missed because the delay was due to the transfer of ownership, and FDA determined that there was not good cause for the delay in a letter dated 12/12/2023. The applicant reports 116 subjects have been enrolled and are anticipating trial completion by the end of 2025."~10/31/2022 0:00:00~5/22/2026 0:00:00
384610~"CDER"~"NDA"~206089.00~"TOLMAR INC"~"Jatenzo (testosterone undecanoate)"~3/27/2019 0:00:00~"Original"~1~"3582-5"~"PMR"~"Pediatric Research Equity Act"~3582.00~5~"PMR 3582-5: A trial of testosterone replacement therapy in pediatric males ages 12 years to less than 18 years of age for conditions associated with a deficiency or absence of endogenous testosterone due to primary hypogonadism or hypogonadotropic hypogonadism."~"Delayed"~"Per FDA letter dated 05/20/2026, the applicant s request for release was denied."~6/30/2029 0:00:00~5/22/2026 0:00:00
384611~"CDER"~"NDA"~206099.00~"AZURITY PHARMACEUTICALS INC"~"Onzetra Xsail (Sumatriptan Succinate)"~1/27/2016 0:00:00~"Original"~1~"3025-1"~"PMR"~"Pediatric Research Equity Act"~3025.00~1~"PMR 3025-1: Conduct a pediatric study under the Pediatric Research Equity Act (PREA) to evaluate the efficacy and safety, including sparse pharmacokinetic (PK) sampling, of Onzetra Xsail (sumatriptan) for the acute treatment of migraine in  pediatric patients of ages 12 to 17 years."~"Delayed"~"The Final Protocol Submission and Study Completion milestones were missed due to early termination of Study 17-AVP-825-301 in Q2 2022. Original Final Report due date 06/30/2020. Per FDA letter dated 11/23/2020, Final Report due date extended to 10/31/2022. Second Deferral Extension granted per FDA letter dated 02/03/2023. The Final Protocol Submission and Study Completion milestones were also revised."~12/31/2027 0:00:00~3/10/2026 0:00:00
384612~"CDER"~"NDA"~206256.00~"ACROTECH BIOPHARMA INC"~"Beleodaq (Belinostat)"~7/3/2014 0:00:00~"Original"~1~"2178-2"~"PMR"~"Accelerated Approval"~2178.00~2~"PMR 2178-2: Characterize the comparative efficacy and safety of belinostat when used in combination with a treatment regimen such as CHOP, versus the combination of pralatrexate plus CHOP, versus CHOP alone for the initial therapy of patients with PTCL. Perform a confirmatory, prospective randomized (1:1:1) trial of previously untreated patients with PTCL, with progression free survival (PFS) as the primary efficacy endpoint. Enroll a sufficient number of patients to characterize the efficacy and safety of each drug added to CHOP, versus CHOP alone. The PFS endpoint should be determined by a blinded independent review committee. PFS analysis should be performed when the trial has experienced the planned number of events necessary for trial completion. Using the same data cutoff date as  the PFS analysis, perform an interim analysis of overall survival. Submit a complete final report with all supporting datasets."~"Delayed"~"The trial is delayed because of delays in site activation which has
impacted patient enrollment."~1/31/2021 0:00:00~8/29/2025 0:00:00
384613~"CDER"~"NDA"~206256.00~"ACROTECH BIOPHARMA INC"~"Beleodaq (Belinostat)"~7/3/2014 0:00:00~"Original"~1~"2178-6"~"PMR"~"505 (o)(3)"~2178.00~6~"PMR 2178-6: Evaluate the safety and pharmacokinetics of belinostat in patients with wild-type, heterozygous, and homozygous UGT1A1*28 genotypes. The evaluations should be conducted for sufficient duration and in a sufficient number of subjects in order to evaluate safety following multiple dose administration. Submit a complete final report with all supporting datasets."~"Delayed"~"FDA determined that the data in the final report submitted by the applicant on 12/22/2021 did not fulfill the terms of the commitment. A Not Fulfilled letter was issued 11/12/2024."~3/31/2016 0:00:00~8/29/2025 0:00:00
384614~"CDER"~"NDA"~208943.00~"PH HEALTH LTD"~"Corphedra (Ephedrine Sulfate)"~1/27/2017 0:00:00~"Original"~1~"3145-6"~"PMR"~"505 (o)(3)"~3145.00~6~"PMR 3145-6: Conduct an adequate leachable safety assessment for the [...] rubber stopper (13 mm) used in your container closure system. This assessment must include leachable data from long-term stability studies testing at least three batches (taking into consideration the proposed shelf-life) to determine if the identified extractables leach into the drug  product over time. Using this information, conduct a toxicological risk assessment justifying the safety of the leachables, taking into consideration the maximum daily dose of the identified materials for this drug product. For your  toxicological risk assessment, any leachable that contains a structural alert for mutagenicity should not exceed 120 mcg/day total daily exposure, or it must be  adequately qualified for safety. A toxicological risk assessment should be provided for any non-genotoxic leachable that exceeds 5 mcg/day."~"Submitted"~~9/30/2017 0:00:00~3/28/2024 0:00:00
384615~"CDER"~"NDA"~208943.00~"PH HEALTH LTD"~"Corphedra (Ephedrine Sulfate)"~1/27/2017 0:00:00~"Original"~1~"3145-7"~"PMR"~"505 (o)(3)"~3145.00~7~"PMR 3145-7: Conduct an extractable study using model solvents, [...]. Using this information, extrapolate the worst-case daily exposure via the drug product and conduct a toxicological risk assessment justifying the safety of the extractables that could be present in the final drug product  ormulation, taking into consideration the maximum daily dose of the identified materials for this drug product. For your toxicological risk assessment, any leachable that contains a  structural alert for mutagenicity should not exceed 120 mcg/day total daily exposure, or it must be adequately qualified for safety. A toxicological risk assessment should be provided for any non-genotoxic leachable that exceeds 5 mcg/day."~"Submitted"~~5/31/2017 0:00:00~3/28/2024 0:00:00
384616~"CDER"~"NDA"~209022.00~"PARATEK PHARMACEUTICALS INC"~"Xhance (Fluticasone Propionate)"~9/18/2017 0:00:00~"Original"~1~"3272-3"~"PMR"~"Pediatric Research Equity Act"~3272.00~3~"PMR 3272-3: Conduct a randomized, double-blind, placebo controlled, parallel group clinical study in children and adolescents 12 to 17 years of age with bilateral nasal polyps associated with nasal congestion to assess the safety, efficacy, pharmacokinetics, and pharmacodynamics of Xhance in improving nasal polyp grade and symptoms (nasal congestion/obstruction, sense of smell, rhinorrhea and facial pain or pressure), using a Bayesian borrowing approach to evaluate efficacy."~"Ongoing"~"37 patients out of the planned 72 patients have been enrolled into the trial."~9/30/2026 0:00:00~11/14/2025 0:00:00
384617~"CDER"~"NDA"~209022.00~"PARATEK PHARMACEUTICALS INC"~"Xhance (Fluticasone Propionate)"~9/18/2017 0:00:00~"Supplement"~19~"4602-1"~"PMR"~"Pediatric Research Equity Act"~4602.00~1~"PMR 4602-1: Conduct a randomized, double-blind, placebo controlled, parallel group clinical study in children and adolescents 12 to 17 years of age with chronic rhinosinusitis without nasal polyps to assess the safety, efficacy, and pharmacokinetics of Xhance using a Bayesian borrowing approach to evaluate efficacy."~"Ongoing"~"7 patients out of the planned 84 patients have been enrolled into the trial."~10/31/2028 0:00:00~11/14/2025 0:00:00
384618~"CDER"~"NDA"~209092.00~"NOVARTIS PHARMACEUTICALS CORP"~"Kisqali (Ribociclib)"~3/13/2017 0:00:00~"Supplement"~18~"4705-1"~"PMR"~"505 (o)(3)"~4705.00~1~"PMR 4705-1: Conduct a carcinogenicity study in mice to evaluate the potential serious risk of carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Pending"~~8/31/2029 0:00:00~5/8/2026 0:00:00
384619~"CDER"~"NDA"~209092.00~"NOVARTIS PHARMACEUTICALS CORP"~"Kisqali (Ribociclib)"~3/13/2017 0:00:00~"Supplement"~18~"4705-2"~"PMC"~"506B PMC"~4705.00~2~"PMC 4705-2: Conduct an integrated analysis from ongoing, completed, or planned (e.g., CLEE011O12301C/NATALEE/NCT03701334 and adjuvant WIDER/NCT05827081) clinical trials and other potential data sources as appropriate, enrolling sufficient representation of racial and ethnic minority patients to reflect the U.S. population of patients with hormone receptor (HR)-positive, human epidermal growth factor receptor-2 (HER2)-negative early breast cancer, and further characterize the efficacy and safety of the addition of ribociclib to an aromatase inhibitor in the adjuvant setting in these patient subgroups. This analysis should include, but is not limited to, patients who identify as Black or African-American, and should be reflective of the incidence of early breast cancer in the U.S. in these patient populations. The analyses should support comparable efficacy and safety between the aforementioned populations and White or Caucasian patients"~"Pending"~~3/31/2030 0:00:00~5/8/2026 0:00:00
384620~"CDER"~"NDA"~209092.00~"NOVARTIS PHARMACEUTICALS CORP"~"Kisqali (Ribociclib)"~3/13/2017 0:00:00~"Supplement"~18~"4705-3"~"PMC"~"506B PMC"~4705.00~3~"PMC 4705-3: Complete the ongoing clinical trial CLEE011O12301C (NATALEE/NCT03701334) to provide all additional overall survival (OS) analyses as prespecified in the protocol and the statistical analysis plan, including OS at the time of trial completion."~"Ongoing"~~12/31/2026 0:00:00~5/8/2026 0:00:00
384621~"CDER"~"NDA"~209115.00~"PHARMAAND GMBH"~"Rubraca (Rucaparib)"~12/19/2016 0:00:00~"Supplement"~4~"3833-1"~"PMR"~"Accelerated Approval"~3833.00~1~"PMR 3833-1: Submit the progression-free survival and overall survival analyses and datasets demonstrating clinical benefit of rucaparib from a phase 3 randomized trial in patients with metastatic castrate-resistant prostate cancer associated with homologous recombination deficiency who have been treated with androgen receptor directed therapy. The results may inform product labeling."~"Fulfilled"~"Per FDA letter dated 12/17/2025, this PMR/PMC has been fulfilled."~10/31/2025 0:00:00~2/25/2026 0:00:00
384622~"CDER"~"NDA"~209115.00~"PHARMAAND GMBH"~"Rubraca (Rucaparib)"~12/19/2016 0:00:00~"Supplement"~4~"3833-3"~"PMC"~"506B PMC"~3833.00~3~"PMC 3833-3: Submit the final report from analytically and clinically validated tumor tissue test, which may be used in conjunction with the plasma-based test, to maximize the identification of BRCA1 and BRCA2 gene mutations in patients with prostate cancer who may benefit from rucaparib. The analytical validation should consist of precision, limit of detection, and accuracy studies using samples, consistent with the clinical trial patient population, to support prostate cancer indication. The clinical validation should be supported by a clinical bridging study comparing intended CDx test(s) and the clinical trial enrollment assays. The results of the validation study may inform product labeling."~"Delayed"~"The final report milestone was missed, because of ongoing discussions with the Agency regarding the proposed development strategy."~6/30/2025 0:00:00~2/25/2026 0:00:00
384623~"CDER"~"NDA"~209115.00~"PHARMAAND GMBH"~"Rubraca (Rucaparib)"~12/19/2016 0:00:00~"Supplement"~13~"4386-1"~"PMC"~"506B PMC"~4386.00~1~"PMC 4386-1: Support the availability, through appropriate analytical and clinical validation studies using the ARIEL3 clinical trial or other data adequate to support labeling, of an in vitro diagnostic device that is essential to the safe and effective use of rucaparib for the maintenance treatment of adult patients with a deleterious BRCA mutation (germline and/or somatic)-associated recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy."~"Delayed"~"The final report milestone was missed, because further alignment between all parties is needed and requires more discussions."~6/30/2024 0:00:00~2/25/2026 0:00:00
384624~"CDER"~"NDA"~22180.00~"AZURITY PHARMACEUTICALS SWITZERLAND GMBH"~"Feraheme (ferumoxytol)"~6/30/2009 0:00:00~"Supplement"~9~"3340-1"~"PMR"~"Pediatric Research Equity Act"~3340.00~1~"PMR 3340-1: To conduct a clinical trial in pediatric patients aged 2 to < 18 years who have iron deficiency anemia and who have intolerance to oral iron or have had unsatisfactory response to oral iron. In addition to any other items, the trial will obtain pharmacokinetic (PK), pharmacodynamic (PD) and safety data from at least 50 patients exposed to ferumoxytol. In this trial, patients will be randomized  to standard of care or ferumoxytol dose regimen. Endpoints will consist of PK, PD, comparisons of hemoglobin changes and safety summaries."~"Delayed"~"The final report and original study completion milestone were missed due to delays in study enrollment. Original Final Report Due Date: 11/2022; Deferral Extension granted per FDA letter dated 11/26/2024. Subsequent Deferral Extension Requested 10/31/2025. Denied per FDA letter dated 12/15/2025.
"~9/30/2026 0:00:00~8/29/2025 0:00:00
384625~"CDER"~"NDA"~22192.00~"VANDA PHARMACEUTICALS INC"~"Fanapt (Iloperidone) "~5/6/2009 0:00:00~"Original"~1~"4-7"~"PMR"~"Pediatric Research Equity Act"~4.00~7~"PMR 4-7: A study of the long-term safety (exposure for at least 1 year) of iloperidone tablets in pediatric patients ages 13 to 17."~"Delayed"~"29/100 subjects have been enrolled. Original Final Report Due Date: 01/31/2023. Per FDA letter dated 11/14/2022, final report due date extended to 02/28/2025. Second Deferral Extension Granted per FDA letter dated 08/15/2024. The study completion milestone was also revised."~11/30/2026 0:00:00~6/30/2026 0:00:00
384626~"CDER"~"NDA"~22192.00~"VANDA PHARMACEUTICALS INC"~"Fanapt (Iloperidone) "~5/6/2009 0:00:00~"Supplement"~23~"4606-1"~"PMR"~"Pediatric Research Equity Act"~4606.00~1~"PMR 4606-1: Conduct a GLP juvenile animal study to assess the toxicology of iloperidone in juvenile rats to support clinical trials of iloperidone in the intended pediatric population ages 10 to <13 years."~"Pending"~"Original Final Report Due Date: 10/31/2025; Deferral Extension granted per FDA letter dated 03/10/2025. The study completion milestones was also revised.
"~7/31/2026 0:00:00~6/30/2026 0:00:00
384627~"CDER"~"NDA"~22192.00~"VANDA PHARMACEUTICALS INC"~"Fanapt (Iloperidone) "~5/6/2009 0:00:00~"Supplement"~23~"4606-2"~"PMR"~"Pediatric Research Equity Act"~4606.00~2~"PMR 4606-2: Conduct an open-label, multiple oral dose study to demonstrate the safety, tolerability, and pharmacokinetics of iloperidone in patients ages 10 to <13 years with manic or mixed episode associated with bipolar I disorder."~"Pending"~"Deferral Extension Requested 05/02/2025. Denied per FDA letter dated 06/16/2025."~1/31/2027 0:00:00~6/30/2026 0:00:00
384628~"CDER"~"NDA"~22192.00~"VANDA PHARMACEUTICALS INC"~"Fanapt (Iloperidone) "~5/6/2009 0:00:00~"Supplement"~23~"4606-3"~"PMR"~"Pediatric Research Equity Act"~4606.00~3~"PMR 4606-3: Conduct an open-label study to assess the long-term safety of iloperidone in patients aged 10 to <13 years with bipolar I disorder."~"Pending"~"Deferral Extension Requested 05/02/2025. Denied per FDA letter dated 06/16/2025."~1/31/2031 0:00:00~6/30/2026 0:00:00
384629~"CDER"~"NDA"~22195.00~"HIKMA PHARMACEUTICALS USA INC"~"Morphine Sulfate "~3/17/2008 0:00:00~"Original"~1~"204-4"~"PMR"~"Pediatric Research Equity Act"~204.00~4~"PMR 204-4: Deferred pediatric study of pharmacokinetics, safety and efficacy under PREA for the  treatment of moderate to severe pain where an opioid analgesic is appropriate in pediatric patients ages birth to 2 years."~"Delayed"~"Final protocol submission milestone was missed because a waiver request was denied due to ongoing Agency discussions on the clinical study design for the birth to <2-year age group PMRs.
"~1/31/2023 0:00:00~5/12/2025 0:00:00
384630~"CDER"~"NDA"~22200.00~"ASTRAZENECA AB"~"Bydureon (Exenatide)"~1/27/2012 0:00:00~"Original"~1~"4739-1"~"PMR"~"505 (o)(3)"~4739.00~1~"PMR 4739-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of exenatide extended-release injectable suspension and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Released"~~11/30/2026 0:00:00~11/21/2024 0:00:00
384631~"CDER"~"NDA"~22200.00~"ASTRAZENECA AB"~"Bydureon (Exenatide)"~1/27/2012 0:00:00~"Original"~1~"1860-5"~"PMR"~"505 (o)(3)"~1860.00~5~"PMR 1860-5: A medullary thyroid carcinoma case series registry of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of BYDUREON (exenatide for injectable suspension) into the marketplace. This study will also establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to diabetes and use of BYDUREON (exenatide for injectable suspension)."~"Released"~~9/30/2028 0:00:00~11/21/2024 0:00:00
384632~"CDER"~"NDA"~22202.00~"COSETTE PHARMACEUTICALS INC"~"Zipsor (Diclofenac Potassium) "~6/16/2009 0:00:00~"Original"~1~"1053-2"~"PMR"~"Pediatric Research Equity Act"~1053.00~2~"PMR 1053-2: Deferred pediatric study under PREA for the treatment of relief of mild to moderate acute pain in pediatric patients ages 2 to 12 years."~"Delayed"~"The final report milestone was missed."~6/30/2016 0:00:00~7/14/2025 0:00:00
384633~"CDER"~"NDA"~22202.00~"COSETTE PHARMACEUTICALS INC"~"Zipsor (Diclofenac Potassium) "~6/16/2009 0:00:00~"Original"~1~"1053-3"~"PMR"~"Pediatric Research Equity Act"~1053.00~3~"PMR 1053-3: Deferred pediatric study under PREA for the treatment of relief of mild to moderate acute pain in pediatric patients ages 1 to 2 years."~"Delayed"~"The final report milestone was missed."~9/30/2019 0:00:00~7/14/2025 0:00:00
384634~"CDER"~"NDA"~22204.00~"ABBVIE INC"~"Gelnique (Oxybutynin Chloride)"~1/27/2009 0:00:00~"Original"~1~"1099-1"~"PMR"~"Pediatric Research Equity Act"~1099.00~1~"PMR 1099-1: Deferred pediatric study(ies) under PREA for the treatment of overactive bladder in the subgroup of pediatric patients with neurologic disease ages 6 to 16 years, 11 months."~"Released"~"Per FDA letter dated 11/04/2025, this PMR/PMC has been released."~12/31/2024 0:00:00~3/20/2024 0:00:00
384635~"CDER"~"NDA"~22207.00~"HIKMA PHARMACEUTICALS USA INC"~"Morphine Sulfate "~3/17/2008 0:00:00~"Original"~1~"204-4"~"PMR"~"Pediatric Research Equity Act"~204.00~4~"PMR 204-4: Deferred pediatric study of pharmacokinetics, safety and efficacy under PREA for the  treatment of moderate to severe pain where an opioid analgesic is appropriate in pediatric patients ages birth to 2 years."~"Delayed"~"Final protocol submission milestone was missed because a waiver request was denied due to ongoing Agency discussions on the clinical study design for the birth to <2-year age group PMRs.
"~1/31/2023 0:00:00~5/14/2026 0:00:00
384636~"CDER"~"NDA"~22253.00~"UCB INC"~"Vimpat (Lacosamide)"~10/28/2008 0:00:00~"Original"~1~"4095-1"~"PMR"~"505 (o)(3)"~4095.00~1~"PMR 4095-1: Conduct in vitro patch clamp studies to assess Vimpat s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Vimpat s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~6/18/2026 0:00:00
384637~"CDER"~"NDA"~22254.00~"UCB INC"~"Vimpat (Lacosamide)"~10/28/2008 0:00:00~"Original"~1~"4095-1"~"PMR"~"505 (o)(3)"~4095.00~1~"PMR 4095-1: Conduct in vitro patch clamp studies to assess Vimpat s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Vimpat s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~6/18/2026 0:00:00
384638~"CDER"~"NDA"~22255.00~"UCB INC"~"Vimpat (Lacosamide)"~4/20/2010 0:00:00~"Original"~1~"4095-1"~"PMR"~"505 (o)(3)"~4095.00~1~"PMR 4095-1: Conduct in vitro patch clamp studies to assess Vimpat s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Vimpat s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~6/18/2026 0:00:00
384639~"CDER"~"BLA"~125513.00~"Alexion Pharmaceuticals, Inc."~"Strensiq (Asfotase Alfa)"~10/23/2015 0:00:00~"Original"~1~"2949-6"~"PMR"~"505 (o)(3)"~2949.00~6~"PMR 2949-6: Observational study to evaluate the potential serious risk of hypophosphatasia (HPP)-associated early death and serious morbidity (e.g. need for ventilatory support, rickets, fractures, failure to thrive) that may result from immune-mediated loss of pharmacological effect, including reduced effectiveness, of Strensiq (asfotase alfa) in patients with HPP (perinatal/infantile-onset and juvenile-onset) enrolled as part of the Global HPP Registry. At least 30 patients (of whom at least 15 will have perinatal/infantile-onset HPP and at least 10 will be treatment-naive at enrollment) will be followed prospectively with pre-specified longitudinal assessments for at least 5 years from study enrollment and at least 200 patients (followed within the Global HPP registry) will be retrospectively evaluated for potential immune-mediated loss of pharmacologic effect (including reduced effectiveness) based on biochemical, imaging, and clinical markers of response to treatment. At baseline, all patients will have CRIM status and genotype assessed. The prospective longitudinal assessments will include at a minimum: (1) plasma asfotase alfa concentrations; (2) antidrug antibody (ADA) and neutralizing antibody (Nab) levels (and titers if appropriate); (3) plasma PPi and PLP; (4) imaging evaluation for HPP-related rickets and fractures; (5) weight and height z-scores; (6) vital status (survival, invasive ventilation-free survival); and (7) occurrence of serious adverse events including serious hypersensitivity reactions and anaphylaxis (according to NIAID/FAAN criteria). The frequency and timecourse of immune-mediated loss of effectiveness of Strensiq will be evaluated and correlated with antibody levels (and titers), CRIM status, Strensiq dosage regimen, patient s age at start of treatment, and HPP subtype."~"Ongoing"~~1/31/2029 0:00:00~12/22/2025 0:00:00
384640~"CDER"~"BLA"~125513.00~"Alexion Pharmaceuticals, Inc."~"Strensiq (Asfotase Alfa)"~10/23/2015 0:00:00~"Original"~1~"2949-7"~"PMR"~"505 (o)(3)"~2949.00~7~"PMR 2949-7: Single-arm, open-label clinical trial to assess different immune tolerance induction (ITI) strategies in patients with hypophosphatasia (HPP) who have demonstrated biochemical and/or clinical evidence of immune-mediated reduced therapeutic response to Strensiq (asfotase alfa). These immune tolerance induction strategies (such as short course(s) of immunosuppressive medications) will be evaluated in patients with evidence of reduced effectiveness (from PMR 2949-6) for their ability to overcome the immunemediated loss of pharmacologic effect and mitigate the serious risk of reduced effectiveness with long-term treatment. All clinical, imaging, and biochemical HPP-associated parameters (as described under PMR 2949-6), pharmacokinetic (PK) parameters, anti-drug antibody and neutralizing antibody levels/titers, and serious adverse events (including anaphylaxis) should be monitored at pre-specified time intervals. In this clinical trial, at least 8 patients with HPP (at least 3 patients with perinatal/infantile-onset HPP) should be evaluated over at least 2 years from their trial enrollment."~"Released"~~2/28/2028 0:00:00~12/22/2025 0:00:00
384641~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~15~"3188-2"~"PMR"~"505 (o)(3)"~3188.00~2~"PMR 3188-2: Characterize complications after allogeneic hematopoietic stem cell transplantation (HSCT) following pembrolizumab in at least 90 patients with hematologic malignancies, of which at least 30% had received pembrolizumab  alone or in combination as the regimen immediately prior to the allogeneic HSCT conditioning regimen. Evaluate toxicities at least through transplant Day 180. Include details of prior pembrolizumab treatment and the transplant regimen. Characterize toxicities including hyperacute graft-versus-host disease (GVHD), severe (grade 3-4) acute GVHD, febrile syndromes treated with steroids, immune mediated adverse events, pulmonary complications, hepatic veno-occlusive disease and/or sinusoidal obstruction syndrome, critical illness, and transplantrelated mortality. Toxicities may be characterized prospectively, or through a combination of prospective and retrospective data analysis."~"Fulfilled"~~12/31/2024 0:00:00~10/28/2025 0:00:00
384642~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~15~"3188-4"~"PMR"~"505 (o)(3)"~3188.00~4~"PMR 3188-4: Characterize the long-term safety of pembrolizumab 2 mg/kg every 3 weeks, in pre-pubertal pediatric patients and those who have not completed pubertal development. Submit a report and datasets that include long-term follow-up of patients enrolled on KN051, a Phase I/II Study of Pembrolizumab (MK-3475) in children with advanced melanoma or a PD-L1 positive advanced, relapsed or refractory solid tumor or lymphoma. Enroll at least 20 patients, including at least 5 patients who are pre-pubertal and 10 who have not yet completed pubertal development. For any pre-pubertal patients and those who have not completed pubertal development, perform the following actions: include in the safety evaluation, immune-mediated, endocrine, and reproductive toxicities for subjects with at least 5 years of follow-up or until pubertal development is complete, whichever is longer."~"Ongoing"~~4/30/2027 0:00:00~10/28/2025 0:00:00
384643~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~40~"3571-1"~"PMC"~"506B PMC"~3571.00~1~"PMC 3571-1: Submit the final clinical report and datasets at the time of the final analysis for overall survival (OS) of Trial KEYNOTE-054, entitled  Adjuvant Immunotherapy with Anti-PD-1 Monoclonal Antibody Pembrolizumab (MK-3475) versus Placebo After Complete Resection of High-Risk Stage III Melanoma: A Randomized, Double-Blind Phase 3 study of the EORTC Melanoma Group , to revise the product label with updated relapse-free survival data and mature OS data."~"Delayed"~"The final report milestone was missed because of slower than expected accumulation of OS events."~12/31/2023 0:00:00~10/28/2025 0:00:00
384644~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~60~"3853-1"~"PMR"~"Accelerated Approval"~3853.00~1~"PMR 3853-1: Submit the final analysis of overall response rate, duration of response, and safety from a trial evaluating pembrolizumab 400 mg every six weeks in participants with classical Hodgkin lymphoma and primary mediastinal B-cell lymphoma to verify and describe the anticipated effects of the alternative dosing regimen of pembrolizumab 400 mg administered every six weeks, that may inform product labeling across indications. All responding patients should be followed for at least 12 months from the onset of response. Provide pharmacokinetic data at first cycle and at steady state and the datasets in the final report."~"Fulfilled"~"Per FDA letter dated 07/24/2025, this PMR/PMC has been fulfilled."~9/30/2025 0:00:00~10/28/2025 0:00:00
384645~"CDER"~"NDA"~206317.00~"ROCKWELL MEDICAL INC"~"Triferic (ferric pyrophosphate citrate)"~1/23/2015 0:00:00~"Original"~1~"2853-2"~"PMR"~"Pediatric Research Equity Act"~2853.00~2~"PMR 2853-2: Efficacy and safety trial of Triferic via hemodialysate in pediatric patients aged less than 18 years with hemodialysis-dependent chronic kidney disease."~"Delayed"~"Original Final Report Due Date: 12/31/2020; Deferral Extension granted per FDA letter dated 07/16/2020. 10 sites active with 16 patients randomized and 1 still ongoing. First Patient First visit was 21 October 2020."~12/31/2022 0:00:00~3/2/2022 0:00:00
384646~"CDER"~"NDA"~206321.00~"NOVO NORDISK INC"~"Saxenda (Liraglutide)"~12/23/2014 0:00:00~"Original"~1~"4750-1"~"PMR"~"505 (o)(3)"~4750.00~1~"PMR 4750-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of liraglutide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Pending"~~11/30/2026 0:00:00~11/12/2025 0:00:00
384647~"CDER"~"NDA"~206321.00~"NOVO NORDISK INC"~"Saxenda (Liraglutide)"~12/23/2014 0:00:00~"Original"~1~"2802-5"~"PMR"~"Pediatric Research Equity Act"~2802.00~5~"PMR 2802-5: A 56-week randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of Saxenda for the treatment of obesity in pediatric patients  ages 7 to 11 (inclusive). The trial may not be initiated until results from the Saxenda adolescent safety and efficacy trial have been submitted to and reviewed by the Agency."~"Submitted"~"The final report was submitted to FDA on 07/22/2024. Original Final Report Due Date: 08/31/2024; Deferral Extension granted per FDA letter dated 06/16/2025."~7/31/2027 0:00:00~11/12/2025 0:00:00
384648~"CDER"~"NDA"~206321.00~"NOVO NORDISK INC"~"Saxenda (Liraglutide)"~12/23/2014 0:00:00~"Original"~1~"2802-6"~"PMR"~"505 (o)(3)"~2802.00~6~"PMR 2802-6: A medullary thyroid carcinoma registry-based case series of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid  carcinoma in the United States and to identify any increase related to the introduction of Saxenda (liraglutide [rDNA origin] injection) into the marketplace. This study will also establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to diabetes and use of Saxenda (liraglutide [rDNA origin] injection)."~"Delayed"~"The final protocol submission milestone (June 2015) was missed due to ongoing discussions with FDA and the FDA determined there was good cause for the delay. The applicant submitted a final version of the registry protocol in August 2015, which FDA found to be acceptable. The study is currently underway and the applicant has been submitting annual status reports for the registry."~9/30/2031 0:00:00~11/12/2025 0:00:00
384649~"CDER"~"NDA"~206334.00~"REMPEX PHARMACEUTICALS INC A WHOLLY OWNED SUB OF MELINTA THERAPEUTICS LLC"~"Orbactiv (Oritavancin Diphosphate)"~8/6/2014 0:00:00~"Original"~1~"2165-1"~"PMR"~"Pediatric Research Equity Act"~2165.00~1~"PMR 2165-1: Conduct an open-label, dose-finding, pharmacokinetics, safety and tolerability study of Orbactiv (oritavancin diphosphate) single dose infusion in pediatric  subjects less than 18 years of age with suspected or confirmed bacterial infections."~"Delayed"~"The study completion milestone was missed and revised. An FDA Deferral Extension granted letter dated 11-15-2024 was issued to revise the final report submission milestone as well."~6/30/2026 0:00:00~9/17/2025 0:00:00
384650~"CDER"~"NDA"~206334.00~"REMPEX PHARMACEUTICALS INC A WHOLLY OWNED SUB OF MELINTA THERAPEUTICS LLC"~"Orbactiv (Oritavancin Diphosphate)"~8/6/2014 0:00:00~"Original"~1~"2165-8"~"PMR"~"Pediatric Research Equity Act"~2165.00~8~"PMR 2165-8: Conduct a multicenter, open-label study to evaluate the safety and tolerability of single-dose IV oritavancin diphosphate for the treatment of pediatric subjects less than 18 years of age with acute bacterial skin and skin structure infections."~"Ongoing"~"Per FDA letter dated 2-28-25, this new PMR was issued to replace PMR 2165-2. (release & reissue)"~7/31/2026 0:00:00~9/17/2025 0:00:00
384651~"CDER"~"NDA"~206426.00~"BIOCRYST PHARMACEUTICALS INC"~"Rapivab (Peramivir)"~12/19/2014 0:00:00~"Original"~1~"2831-1"~"PMR"~"Pediatric Research Equity Act"~2831.00~1~"PMR 2831-1: Conduct a clinical trial to evaluate the pharmacokinetics, safety, and antiviral activity of peramivir administration in pediatric subjects with acute uncomplicated influenza infection from birth to less than 18 years of age. Include characterization of peramivir resistance-associated substitutions in viral isolates  from subjects with prolonged viral shedding."~"Delayed"~"A  deferral extension was granted per FDA letter dated 8/21/2024 so the revised final report submission milestone is 10/2026. The study completion milestone was also revised to 4/2026."~10/31/2026 0:00:00~2/5/2026 0:00:00
384652~"CDER"~"NDA"~206488.00~"SAREPTA THERAPEUTICS INC"~"Exondys 51 (Eteplirsen)"~9/19/2016 0:00:00~"Original"~1~"3095-1"~"PMR"~"Accelerated Approval"~3095.00~1~"PMR 3095-1: In order to verify the clinical benefit of eteplirsen, conduct a 2-year randomized, double-blind, controlled trial of eteplirsen in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Patients should be randomized to the approved dosage of eteplirsen (30 mg/kg weekly) or to a dosage that provides significantly higher exposure, e.g., 30 mg/kg daily. The primary endpoint will be the North Star Ambulatory Assessment."~"Delayed"~"The final report milestone was missed, because the sponsor requested milestone extensions due to study delays. Agency Acknowledged revised milestone in letter dated 12/20/24."~5/31/2021 0:00:00~11/14/2025 0:00:00
384653~"CDER"~"NDA"~206627.00~"KNOA PHARMA LLC"~"Hysingla ER (Hydrocodone Bitartrate)"~11/20/2014 0:00:00~"Original"~1~"3033-1"~"PMR"~"505 (o)(3)"~3033.00~1~"PMR 3033-1: A prospective, observational study designed to quantify the serious risks of misuse, abuse, and addiction associated with long-term use of opioid analgesics  for management of chronic pain among patients prescribed ER/LA opioid analgesics. This study must address at a minimum the following specific objectives: a. Estimate the incidence of misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of  psychiatric illness) on the risk of misuse, abuse, and addiction.  b. Evaluate and quantify other risk factors for misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors, psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships."~"Fulfilled"~~3/31/2020 0:00:00~1/13/2026 0:00:00
384654~"CDER"~"NDA"~208623.00~"AMICUS THERAPEUTICS US LLC"~"Galafold (Migalastat)"~8/10/2018 0:00:00~"Original"~1~"3412-1"~"PMR"~"Accelerated Approval"~3412.00~1~"PMR 3412-1: A randomized, double-blind, placebo-controlled clinical trial to verify and describe the clinical benefit of Galafold (migalastat) in patients with Fabry disease. The trial will evaluate the efficacy and pharmacodynamic effects (e.g., on plasma lyso-Gb3, alpha-Gal A enzyme activity) of Galafold (migalastat) in patients with a confirmed diagnosis of Fabry disease and amenable, diseasecausing GLA variants. The trial should be of sufficient duration to observe clinically meaningful changes in Fabry-related symptoms in the intended patient population."~"Delayed"~"The final protocol was submitted after the original milestone and was acknowledged by FDA in a letter dated 9/19/2023. The trial completion and final report milestones were  revised."~8/31/2026 0:00:00~10/8/2025 0:00:00
384655~"CDER"~"NDA"~208623.00~"AMICUS THERAPEUTICS US LLC"~"Galafold (Migalastat)"~8/10/2018 0:00:00~"Original"~1~"3412-2"~"PMR"~"Accelerated Approval"~3412.00~2~"PMR 3412-2: A prospective, longitudinal, observational study to evaluate the efficacy and pharmacodynamic effects of Galafold (migalastat) in patients with a confirmed diagnosis of Fabry disease and amenable, disease-causing GLA variants. The study will collect, analyze, and compare long-term data on clinical outcomes (major renal, cardiac, and cerebrovascular events) and pharmacodynamic changes (e.g., in WBC alpha-Gal A enzyme activity, plasma lyso-Gb3, serum and urine creatinine, eGFR, urine protein, urine albumin) in treated and untreated patients with Fabry disease and amenable GLA variants. The data will be used for the verification and description of the clinical benefit of Galafold (migalastat) in patients with Fabry disease and amenable GLA variants."~"Delayed"~"The final protocol was submitted after the original milestone. An Acknowledge Final Protocol letter was issued on 9/19/2023. As of 10/8/2025, 0 subjects have been enrolled."~4/30/2031 0:00:00~10/8/2025 0:00:00
384656~"CDER"~"NDA"~208623.00~"AMICUS THERAPEUTICS US LLC"~"Galafold (Migalastat)"~8/10/2018 0:00:00~"Original"~1~"3412-3"~"PMR"~"505 (o)(3)"~3412.00~3~"PMR 3412-3: A worldwide, prospective, single-arm, observational study in women exposed to Galafold (migalastat) during pregnancy and lactation to assess: risks of pregnancy complications, adverse effects on the developing fetus and neonate, and adverse effects on lactation and the breastfed infant. Pregnancy exposures and outcomes will be reported voluntarily by providers and patients (e.g., a telephone contact number and/or website will be provided in the product s prescribing information). Complete data will be captured regarding pregnancy outcomes and any adverse effects in offspring. Results will be analyzed and reported descriptively. The study will collect information for a minimum of 10 years. Interim reports on the cumulative findings and analyses will be submitted annually. Data collected retrospectively from other sources will be analyzed separately and reported with the interim and final study reports."~"Ongoing"~~2/28/2030 0:00:00~10/8/2025 0:00:00
384657~"CDER"~"NDA"~208623.00~"AMICUS THERAPEUTICS US LLC"~"Galafold (Migalastat)"~8/10/2018 0:00:00~"Original"~1~"3412-4"~"PMC"~"506B PMC"~3412.00~4~"PMC 3412-4: A PK/PD trial to evaluate an appropriate dosing regimen of Galafold (migalastat) in patients with Fabry disease and amenable GLA variants who have severe renal impairment (eGFR < 30 mL/min/1.73m2/year) or who are on kidney dialysis."~"Delayed"~"The trial completion and final report submission milestones have been missed.  As of 10/8/2025, 11 subjects have been enrolled."~10/31/2021 0:00:00~10/8/2025 0:00:00
384658~"CDER"~"NDA"~208627.00~"SIGA TECHNOLOGIES INC"~"TPOXX (Tecovirimat)"~7/13/2018 0:00:00~"Original"~1~"3795-1"~"PMC"~"506B PMC"~3795.00~1~"PMC 3795-1: Conduct and submit final study reports for relevant nonclinical and/or clinical studies, including a human factors (HF) validation study to support accurate dosing in pediatric patients requiring less than a 200mg dose, including the development of new oral formulation if needed."~"Delayed"~"The acknowledge revised milestone letter was issued on 08/25/2025."~12/31/2025 0:00:00~8/27/2025 0:00:00
384659~"CDER"~"NDA"~208627.00~"SIGA TECHNOLOGIES INC"~"TPOXX (Tecovirimat)"~7/13/2018 0:00:00~"Original"~1~"3417-7"~"PMR"~"Animal Efficacy"~3417.00~7~"PMR 3417-7: Collaborate with US public health agencies to conduct a field study to evaluate the clinical response, drug concentrations, and safety profile of tecovirimat when used for the treatment of human smallpox disease due to variola virus infection. This trial should evaluate tecovirimat vs. brincidofovir vs. tecovirimat and brincidofovir combination therapy."~"Pending"~~~8/27/2025 0:00:00
384660~"CDER"~"BLA"~208673.00~"Sanofi-Aventis U.S. LLC"~"Soliqua (Insulin Glargine and Lixisenatide)"~11/21/2016 0:00:00~"Original"~1~"4737-1"~"PMR"~"505 (o)(3)"~4737.00~1~"PMR 4737-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of lixisenatide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Released"~~11/30/2026 0:00:00~1/16/2026 0:00:00
384661~"CDER"~"NDA"~208692.00~"EXELIXIS INC"~"Cabometyx (Cabozantinib)"~4/25/2016 0:00:00~"Supplement"~17~"4783-1"~"PMC"~"506B PMC"~4783.00~1~"PMC 4783-1: Complete survival follow-up of patients in the CABINET trial to further characterize the efficacy and clinical benefit of cabozantinib in adult patients with previously treated, unresectable, locally advanced or metastatic, well-or moderately-differentiated pancreatic or extrapancreatic neuroendocrine tumors."~"Ongoing"~~6/30/2027 0:00:00~6/22/2026 0:00:00
384662~"CDER"~"NDA"~208700.00~"ADVANCED ACCELERATOR APPLICATIONS USA INC"~"Lutathera (lutetium Lu 177 dotatate)"~1/26/2018 0:00:00~"Original"~1~"3326-1"~"PMR"~"505 (o)(3)"~3326.00~1~"PMR 3326-1: Submit cumulative, integrated safety analyses after 5 and after 10 years of followup from an adequate number of patients enrolled in clinical trials to identify and characterize the risk of renal failure with Lutathera; include incidence rates, time to onset, predisposing factors and outcomes. These safety evaluations should be adequate to inform labeling of patient populations at highest risk and to provide evidence-based dose modifications and monitoring recommendations."~"Delayed"~"The final report milestone was missed because of additional 5-year interim data collection needed for a comprehensive 10-year safety requirement. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 01/05/2026."~12/31/2025 0:00:00~3/24/2026 0:00:00
384663~"CDER"~"NDA"~208700.00~"ADVANCED ACCELERATOR APPLICATIONS USA INC"~"Lutathera (lutetium Lu 177 dotatate)"~1/26/2018 0:00:00~"Original"~1~"3326-2"~"PMR"~"505 (o)(3)"~3326.00~2~"PMR 3326-2: Submit cumulative, integrated safety analyses after 5 and after 10 years of followup from an adequate number of patients enrolled in clinical trials to identify and characterize the risks of myelodysplastic syndrome and acute leukemia with Lutathera; include incidence rates, time to onset, predisposing factors and  outcomes. These safety evaluations should be adequate to inform labeling of patient populations at highest risk and to provide evidence-based dose  modifications and monitoring recommendations."~"Delayed"~"The final report milestone was missed because of additional 5-year interim data collection needed for a comprehensive 10-year safety requirement. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 01/05/2026."~12/31/2025 0:00:00~3/24/2026 0:00:00
384664~"CDER"~"NDA"~22256.00~"ABBVIE INC"~"Savella (Milnacipran Hydrochloride)"~1/14/2009 0:00:00~"Original"~1~"845-5"~"PMR"~"505 (o)(3)"~845.00~5~"PMR 845-5: Conduct a retrospective pregnancy cohort study using claims or electronic health record data with medical chart validation to describe safety events (major congenital malformations, spontaneous abortions, stillbirths, preterm birth, small-for-gestational age, and low birth weight births) in individuals exposed to milnacipran during pregnancy and in an appropriate comparator population."~"Pending"~~11/30/2028 0:00:00~3/10/2026 0:00:00
384665~"CDER"~"NDA"~22272.00~"KNOA PHARMA LLC"~"OxyContin (Oxycodone Hydrochloride)"~4/5/2010 0:00:00~"Original"~1~"3033-1"~"PMR"~"505 (o)(3)"~3033.00~1~"PMR 3033-1: A prospective, observational study designed to quantify the serious risks of misuse, abuse, and addiction associated with long-term use of opioid analgesics  for management of chronic pain among patients prescribed ER/LA opioid analgesics. This study must address at a minimum the following specific objectives: a. Estimate the incidence of misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of  psychiatric illness) on the risk of misuse, abuse, and addiction.  b. Evaluate and quantify other risk factors for misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors, psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships."~"Fulfilled"~~3/31/2020 0:00:00~6/1/2026 0:00:00
384666~"CDER"~"NDA"~22272.00~"KNOA PHARMA LLC"~"OxyContin (Oxycodone Hydrochloride)"~4/5/2010 0:00:00~"Original"~1~"3033-2"~"PMR"~"505 (o)(3)"~3033.00~2~"PMR 3033-2: An observational study designed to measure the incidence and predictors of opioid overdose and death (OOD), as well as opioid abuse/addiction, using patient  health records, insurance claims, and death records. This study must address at a minimum the following specific objectives: a. Estimate the incidence of abuse/addiction, overdose, and death associated with long-term use of opioid analgesics for chronic pain. Stratify overdose by  intentionality wherever possible. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other  clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of psychiatric illness) on the risk of  abuse/addiction, overdose, and death. b. Evaluate and quantify other risk factors for abuse/addiction, overdose, and  death associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors,  psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships. Stratify overdose by intentionality wherever possible."~"Fulfilled"~~9/30/2019 0:00:00~6/1/2026 0:00:00
384667~"CDER"~"NDA"~22272.00~"KNOA PHARMA LLC"~"OxyContin (Oxycodone Hydrochloride)"~4/5/2010 0:00:00~"Original"~1~"3033-6"~"PMR"~"505 (o)(3)"~3033.00~6~"PMR 3033-6: An observational study to develop and validate an algorithm using coded medical terminologies and other electronic healthcare data to identify opioid-related overdose and death."~"Fulfilled"~~12/31/2016 0:00:00~6/1/2026 0:00:00
384668~"CDER"~"NDA"~22272.00~"KNOA PHARMA LLC"~"OxyContin (Oxycodone Hydrochloride)"~4/5/2010 0:00:00~"Original"~1~"3033-11"~"PMR"~"505 (o)(3)"~3033.00~11~"PMR 3033-11: Conduct a clinical trial to estimate the serious risk for the development of hyperalgesia following the long-term use of high-dose ER/LA opioid analgesics  for at least one year to treat chronic pain. Include an assessment of risk relative to  efficacy."~"Delayed"~"The Trial Completion and Final Report milestones were missed because of continued discussions needed to reach agreement with the Agency on the Final Protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 8/26/2025."~8/31/2019 0:00:00~6/1/2026 0:00:00
384669~"CDER"~"NDA"~22272.00~"KNOA PHARMA LLC"~"OxyContin (Oxycodone Hydrochloride)"~4/5/2010 0:00:00~"Supplement"~27~"2923-3"~"PMR"~"505 (o)(3)"~2923.00~3~"PMR 2923-3: Conduct a retrospective cohort study in pediatric patients aged 17 years and younger (including patients younger than the approved age of 11 years) to examine the association between opioid nontolerance at initiation of OxyContin therapy and serious risks such as respiratory depression as well as non-fatal and fatal overdose. The sample size must be sufficient to detect any clinically relevant difference in risk from that of an appropriate comparator group(s), and information must be sufficiently granular to assess all relevant demographic, clinical, prescription covariates and outcomes (in a multivariate model). Investigators must be able to ascertain all exposures and deaths regardless of location (i.e., in-hospital or outof-hospital)."~"Submitted"~~8/31/2022 0:00:00~6/1/2026 0:00:00
384670~"CDER"~"NDA"~22287.00~"TAKEDA PHARMACEUTICALS USA INC"~"Dexilant (Dexlansoprazole) "~1/30/2009 0:00:00~"Original"~1~"1356-7"~"PMR"~"Pediatric Research Equity Act"~1356.00~7~"PMR 1356-7: Deferred pediatric study under PREA for treating heartburn associated with non-erosive GERD in pediatric patients aged 2 years to 11 years."~"Delayed"~"Original Final Report Due Date: 02/28/2024; Deferral Extension granted per FDA letter dated 01/11/2024. The final report due date was extended to 03/2028. The study completion milestone was also revised. 65 out 70 subjects are currently enrolled.
"~3/31/2028 0:00:00~3/25/2026 0:00:00
384671~"CDER"~"NDA"~22287.00~"TAKEDA PHARMACEUTICALS USA INC"~"Dexilant (Dexlansoprazole) "~1/30/2009 0:00:00~"Supplement"~8~"1788-6"~"PMR"~"Pediatric Research Equity Act"~1788.00~6~"PMR 1788-6: Deferred study under PREA to evaluate the pharmacokinetics, healing, maintenance of healing, and symptoms of endoscopy-proven erosive esophagitis (EE) in patients 2 years to 11 years of age."~"Delayed"~"The final protocol milestone was missed. DE was granted per letter dated January 11, 2024. Final report due date extended to 03/2028. 24/76 subjects enrolled."~3/31/2028 0:00:00~3/25/2026 0:00:00
384672~"CDER"~"NDA"~22287.00~"TAKEDA PHARMACEUTICALS USA INC"~"Dexilant (Dexlansoprazole) "~1/30/2009 0:00:00~"Supplement"~8~"1788-7"~"PMR"~"Pediatric Research Equity Act"~1788.00~7~"PMR 1788-7: Deferred study under PREA to evaluate the long-term safety of dexlansoprazole for the healing and maintenance of healing of erosive esophagitis (EE) in pediatric patients 2 years through 17 years of age, who require chronic treatment with dexlansoprazole due to underlying conditions that predispose to chronic gastroesophageal reflux disease and relapsing EE."~"Delayed"~"The final protocol milestone was missed."~8/31/2030 0:00:00~3/25/2026 0:00:00
384673~"CDER"~"NDA"~22301.00~"SALIX PHARMACEUTICALS INC"~"Apriso (Mesalamine)"~10/31/2008 0:00:00~"Original"~1~"1464-1"~"PMR"~"Pediatric Research Equity Act"~1464.00~1~"PMR 1464-1: Deferred pediatric study under PREA for the maintenance of remission of ulcerative colitis in pediatric patients ages 5 to 17 years to evaluate safety, effectiveness, and pharmacokinetics with Apriso (mesalamine granules) in at least two dosing regimens."~"Delayed"~"The final report submission milestone was missed. Study initiation is currently paused, and reactivation discussions are ongoing."~6/1/2013 0:00:00~12/19/2025 0:00:00
384674~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~61~"3853-1"~"PMR"~"Accelerated Approval"~3853.00~1~"PMR 3853-1: Submit the final analysis of overall response rate, duration of response, and safety from a trial evaluating pembrolizumab 400 mg every six weeks in participants with classical Hodgkin lymphoma and primary mediastinal B-cell lymphoma to verify and describe the anticipated effects of the alternative dosing regimen of pembrolizumab 400 mg administered every six weeks, that may inform product labeling across indications. All responding patients should be followed for at least 12 months from the onset of response. Provide pharmacokinetic data at first cycle and at steady state and the datasets in the final report."~"Fulfilled"~"Per FDA letter dated 07/24/2025, this PMR/PMC has been fulfilled."~9/30/2025 0:00:00~10/28/2025 0:00:00
384675~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~71~"3871-1"~"PMR"~"Accelerated Approval"~3871.00~1~"PMR 3871-1: Submit the final report and datasets from clinical trials evaluating overall response rate and duration of response, to verify and describe the clinical benefit of pembrolizumab in adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [=10 mutations/megabase (mut/Mb)] solid tumors (as determined by an FDA-approved test) that have progressed following prior treatment and who have no satisfactory alternative treatment options. A sufficient number of patients and representation of tumor types (other than lung cancers, MSIH or dMMR cancers, or melanoma; and including CNS tumors that were determined to be TMB-H based on testing of tissue obtained prior to initiation of temozolomide chemotherapy), and with cancers having a TMB of 10 to <13 mut/Mb, will be evaluated to characterize response and duration of response. A minimum of 20 pediatric patients will be studied. Overall response rate and duration of response will be assessed by independent central review for patients with cancers having a TMB of =10 mut/Mb, =10 mut/Mb to <13 mut/Mb, and =13 mut/Mb. All responding patients will be followed for at least 12 months from the onset of response."~"Submitted"~"The final report was submitted to FDA on 12/17/2025."~12/31/2025 0:00:00~10/28/2025 0:00:00
384676~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~85~"3938-1"~"PMR"~"505 (o)(3)"~3938.00~1~"PMR 3938-1: Submit the final results and datasets characterizing the risk of immune-mediated or potentially immune-mediated toxicities, serious adverse events, and long-term safety for pediatric patients with lymphoma enrolled in KEYNOTE-051 who receive pembrolizumab. All patients with Hodgkin lymphoma should be followed for safety for a minimum of 6 months on pembrolizumab."~"Fulfilled"~~9/30/2024 0:00:00~10/28/2025 0:00:00
384677~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~85~"3938-2"~"PMC"~"506B PMC"~3938.00~2~"PMC 3938-2: Submit the final results and datasets for response rate and duration of response as assessed by an independent review committee in all pediatric patients who received pembrolizumab for Hodgkin lymphoma in KEYNOTE-051, to further characterize the clinical benefit of pembrolizumab. All patients who achieve an objective response should be followed for duration of response for a minimum of 6 months. The results from this trial may inform product labeling."~"Fulfilled"~~9/30/2024 0:00:00~10/28/2025 0:00:00
384678~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~128~"4396-1"~"PMC"~"506B PMC"~4396.00~1~"PMC 4396-1: Complete EORTC/ETOP Study 1419 (KEYNOTE 091/PEARLS; NCT02504372) to further characterize the clinical benefit of pembrolizumab as adjuvant treatment following resection with or without platinum-based chemotherapy for adult patients with non-small cell lung cancer. Provide all planned analyses for interim analysis 3, including final disease-free survival for the PD-L1 TPS =50% population and interim overall survival in the intention-to-treat (ITT) population and in the population of patients with PD-L1 TPS =50%. Additionally, provide the final analyses of Overall Survival in the ITT population and in the population of patients with PD-L1 TPS =50%."~"Ongoing"~~7/31/2026 0:00:00~10/28/2025 0:00:00
384679~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~139~"4531-1"~"PMC"~"506B PMC"~4531.00~1~"PMC 4531-1: Complete Study KEYNOTE-671 including all final and planned interim analyses 3 and 4 of overall survival of adult patients with resectable stage II-IIIB NSCLC treated with pembrolizumab in combination with neoadjuvant platinum-containing chemotherapy followed by surgery and adjuvant pembrolizumab."~"Delayed"~"The Interim Report 4 Submission milestone was missed due to slower than expected overall survival event accrual."~7/31/2027 0:00:00~10/28/2025 0:00:00
384694~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-8"~"PMR"~"505 (o)(3)"~3462.00~8~"PMR 3462-8: Conduct a thorough QT trial for Diacomit as per the ICH E14 guidelines."~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 2/24/2021. applicant s Annual Status Report, the revised protocol was submitted on 7/30/2020."~9/30/2020 0:00:00~10/17/2025 0:00:00
384695~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-9"~"PMR"~"505 (o)(3)"~3462.00~9~"PMR 3462-9: Conduct a clinical pharmacokinetic trial to determine an appropriate dose of Diacomit (stiripentol) to minimize toxicity in patients with varying degrees of hepatic impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 3/25/2020."~9/30/2022 0:00:00~10/17/2025 0:00:00
384696~"CDER"~"NDA"~208745.00~"SALIX PHARMACEUTICALS INC"~"Trulance (Plecanatide)"~1/19/2017 0:00:00~"Original"~1~"3117-5"~"PMR"~"Pediatric Research Equity Act"~3117.00~5~"PMR 3117-5: Confirm the efficacy and safety of Trulance (plecanatide) treatment in pediatric patients with chronic idiopathic constipation (CIC) who are 2 years to less than 6 years  of age by performing a randomized, double-blind, placebo-controlled, parallel group, 12 week treatment study."~"Delayed"~"Original Final Report Due Date: 02/28/2026; Deferral Extension granted per FDA letter dated 06/21/2023. The final protocol submission and study completion milestones were also revised."~12/31/2029 0:00:00~3/20/2026 0:00:00
384697~"CDER"~"NDA"~208745.00~"SALIX PHARMACEUTICALS INC"~"Trulance (Plecanatide)"~1/19/2017 0:00:00~"Original"~1~"3117-6"~"PMR"~"Pediatric Research Equity Act"~3117.00~6~"PMR 3117-6: Assess the long-term safety of Trulance (plecanatide) in pediatric patients with chronic idiopathic constipation (CIC) who are 2 years to less than 18 years of age and have completed a confirmatory efficacy and safety study with plecanatide."~"Delayed"~"The final protocol submission milestone was missed."~8/31/2026 0:00:00~3/20/2026 0:00:00
384698~"CDER"~"NDA"~208745.00~"SALIX PHARMACEUTICALS INC"~"Trulance (Plecanatide)"~1/19/2017 0:00:00~"Supplement"~1~"3304-1"~"PMR"~"Pediatric Research Equity Act"~3304.00~1~"PMR 3304-1: Perform a double-blind, dose ranging study in pediatric patients ages 6 years to less than 18 years in order to evaluate the safety and efficacy of once daily oral Trulance (plecanatide) for 4 weeks as treatment of IBS-C. Patients will be stratified by age group (6 years to 11 years and 12 years to less than 18 years of age)."~"Submitted"~"The final report was submitted to FDA on 07/31/2025."~7/31/2025 0:00:00~3/20/2026 0:00:00
384699~"CDER"~"NDA"~208745.00~"SALIX PHARMACEUTICALS INC"~"Trulance (Plecanatide)"~1/19/2017 0:00:00~"Supplement"~1~"3304-2"~"PMR"~"Pediatric Research Equity Act"~3304.00~2~"PMR 3304-2: Confirm the efficacy and safety of Trulance (plecanatide) treatment in pediatric patients with IBS-C who are 6 years to less than 18 years of age by performing a randomized, double-blind, placebo-controlled, parallel group, 12-week treatment study. Patients will be stratified by age (6 years to 11 years and 12 years to less than 18 years of age)."~"Delayed"~"Original Final Report Due Date: 11/30/2022; Deferral Extension granted per FDA letter dated 03/30/2023. The  study completion milestone was also revised."~6/30/2028 0:00:00~3/20/2026 0:00:00
384700~"CDER"~"NDA"~208745.00~"SALIX PHARMACEUTICALS INC"~"Trulance (Plecanatide)"~1/19/2017 0:00:00~"Supplement"~1~"3304-3"~"PMR"~"Pediatric Research Equity Act"~3304.00~3~"PMR 3304-3: Assess the long-term safety of Trulance (plecanatide) once-daily treatment for chronic idiopathic constipation (CIC) or IBS-C in pediatric patients who are 6 years to less than 18 years of age and have completed the confirmatory efficacy and safety study with Trulance (plecanatide) by performing an open-label, active,  comparator-controlled, safety and tolerability study for up to 24 weeks."~"Delayed"~"Original Final Report Due Date: 11/30/2022; Deferral Extension granted per FDA letter dated 03/30/2023. The final protocol submission and study completion milestones were also revised."~6/30/2029 0:00:00~3/20/2026 0:00:00
384701~"CDER"~"NDA"~208791.00~"B BRAUN MEDICAL INC"~"CLOROTEKAL (chloroprocaine HCl)"~9/26/2017 0:00:00~"Original"~1~"3242-2"~"PMR"~"Pediatric Research Equity Act"~3242.00~2~"PMR 3242-2: Conduct an intrathecal juvenile animal toxicology study to characterize the impact of chloroprocaine on neuronal development."~"Submitted"~"The Final Report was submitted to FDA on 02/21/2020."~2/28/2020 0:00:00~11/13/2025 0:00:00
384702~"CDER"~"NDA"~208791.00~"B BRAUN MEDICAL INC"~"CLOROTEKAL (chloroprocaine HCl)"~9/26/2017 0:00:00~"Original"~1~"3242-3"~"PMR"~"505 (o)(3)"~3242.00~3~"PMR 3242-3: Conduct an adequate intrathecal toxicity study in a non-rodent species up to 28-days duration that not only defines a no-adverse-effect level, but also characterizes the toxicological potential of the drug product formulation. Submit  justification for the study design prior to initiation of the study in order to obtain Division concurrence with the study design."~"Submitted"~~10/31/2019 0:00:00~11/13/2025 0:00:00
384703~"CDER"~"NDA"~208854.00~"BIODELIVERY SCIENCES INTERNATIONAL INC"~"Symproic (naldemedine)"~3/23/2017 0:00:00~"Original"~1~"3174-1"~"PMR"~"505 (o)(3)"~3174.00~1~"PMR 3174-1: A post-marketing, observational epidemiologic study comparing Symproic (naldemedine) to other treatments of opioid induced constipation in patients with  chronic non-cancer pain. The study s primary outcome is a composite of major adverse cardiovascular events (MACE): cardiovascular (CV) death, nonfatal  myocardial infarction, and nonfatal stroke. Secondary outcomes include, but are not limited to, CV death, nonfatal myocardial infarction, and nonfatal stroke  separately. Ensure an adequate number of patients with at least 12 months of Symproic (naldemedine) exposure at the end of the study."~"Delayed"~"The study completion and final report submission milestones were missed because the accrual of patients in the naldemedine cohorts of the study is slower than originally anticipated. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 04/17/2026."~11/30/2025 0:00:00~5/21/2026 0:00:00
384704~"CDER"~"NDA"~208912.00~"EYEPOINT PHARMACEUTICALS INC"~"Dexycu (Dexamethasone)"~2/9/2018 0:00:00~"Original"~1~"3334-1"~"PMR"~"Pediatric Research Equity Act"~3334.00~1~"PMR 3334-1: A Phase 3/4, Prospective, Randomized, Active Treatment-controlled, Parallel-design, Multicenter Trial to Evaluate the Safety of IBI-10090 for the Treatment of Inflammation Following Ocular Surgery for Childhood Cataract."~"Delayed"~"The final report milestone was missed because of delays
involving study participants, sites, and/or management.
Original Final Report Due Date: 6/2022. Per FDA letter dated 3/17/2022, Final Report due date extended to 6/2025. Second Deferral Extension Granted per FDA letter dated 6/27/2025.
"~6/30/2027 0:00:00~4/2/2026 0:00:00
384705~"CDER"~"NDA"~208943.00~"PH HEALTH LTD"~"Corphedra (Ephedrine Sulfate)"~1/27/2017 0:00:00~"Original"~1~"3145-1"~"PMR"~"505 (o)(3)"~3145.00~1~"PMR 3145-1: Conduct a fertility and early embryonic development toxicology study in the rat model for ephedrine sulfate."~"Submitted"~~12/31/2018 0:00:00~3/28/2024 0:00:00
384706~"CDER"~"NDA"~208943.00~"PH HEALTH LTD"~"Corphedra (Ephedrine Sulfate)"~1/27/2017 0:00:00~"Original"~1~"3145-2"~"PMR"~"505 (o)(3)"~3145.00~2~"PMR 3145-2: Conduct an embryo-fetal developmental toxicology study using the rat model for ephedrine sulfate."~"Submitted"~~10/31/2018 0:00:00~3/28/2024 0:00:00
384707~"CDER"~"NDA"~208943.00~"PH HEALTH LTD"~"Corphedra (Ephedrine Sulfate)"~1/27/2017 0:00:00~"Original"~1~"3145-3"~"PMR"~"505 (o)(3)"~3145.00~3~"PMR 3145-3: Conduct an embryo-fetal developmental toxicology study using the rabbit model for ephedrine sulfate."~"Submitted"~~1/31/2019 0:00:00~3/28/2024 0:00:00
384708~"CDER"~"NDA"~208943.00~"PH HEALTH LTD"~"Corphedra (Ephedrine Sulfate)"~1/27/2017 0:00:00~"Original"~1~"3145-4"~"PMR"~"505 (o)(3)"~3145.00~4~"PMR 3145-4: Conduct a pre- and post-natal developmental toxicology study in the rat model for ephedrine sulfate."~"Submitted"~~2/28/2020 0:00:00~3/28/2024 0:00:00
384709~"CDER"~"NDA"~208943.00~"PH HEALTH LTD"~"Corphedra (Ephedrine Sulfate)"~1/27/2017 0:00:00~"Original"~1~"3145-5"~"PMR"~"505 (o)(3)"~3145.00~5~"PMR 3145-5: Conduct an in vivo micronucleus assay with ephedrine sulfate."~"Submitted"~~6/30/2018 0:00:00~3/28/2024 0:00:00
384710~"CDER"~"NDA"~22341.00~"NOVO NORDISK INC"~"Victoza  (Liraglutide)"~1/25/2010 0:00:00~"Original"~1~"4748-1"~"PMR"~"505 (o)(3)"~4748.00~1~"PMR 4748-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of liraglutide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Pending"~~11/30/2026 0:00:00~11/12/2025 0:00:00
384711~"CDER"~"NDA"~22341.00~"NOVO NORDISK INC"~"Victoza  (Liraglutide)"~1/25/2010 0:00:00~"Original"~1~"1583-7"~"PMR"~"505 (o)(3)"~1583.00~7~"PMR 1583-7: A medullary thyroid carcinoma case series registry of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of Victoza (liraglutide [rDNA origin]) Injection into the marketplace. This study will also establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to diabetes and use of Victoza (liraglutide [rDNA origin]) Injection."~"Delayed"~"The final protocol submission milestone was missed due to ongoing discussions with FDA and the FDA determined there was good cause for the delay. The study is currently underway and the applicant has been submitting annual status reports."~9/15/2026 0:00:00~11/12/2025 0:00:00
384712~"CDER"~"NDA"~22372.00~"AZURITY PHARMACEUTICALS INC"~"Suprep Bowel Prep Kit (Sodium Sulfate, Potassium Sulfate, and Magnesium Sulfate)"~8/5/2010 0:00:00~"Original"~1~"1580-4"~"PMR"~"Pediatric Research Equity Act"~1580.00~4~"PMR 1580-4: Conduct a randomized, single-blind, multicenter dose ranging study comparing the safety and efficacy of SUPREP to NuLytely in children (3 years to 11 years)."~"Delayed"~"The final report milestone was missed. FDA issued a notification of noncompliance on 7/19/2019."~11/30/2017 0:00:00~10/3/2025 0:00:00
384713~"CDER"~"NDA"~22372.00~"AZURITY PHARMACEUTICALS INC"~"Suprep Bowel Prep Kit (Sodium Sulfate, Potassium Sulfate, and Magnesium Sulfate)"~8/5/2010 0:00:00~"Original"~1~"1580-9"~"PMR"~"Pediatric Research Equity Act"~1580.00~9~"PMR 1580-9: Conduct a randomized, single-blind, multicenter dose ranging study comparing the safety and efficacy of SUPREP to NuLytely in children (6 months to 2 years)."~"Delayed"~"The final report submission milestone was missed. Per the applicant's annual status report, discussions continue with the agency."~5/31/2019 0:00:00~10/3/2025 0:00:00
384714~"CDER"~"NDA"~22382.00~"COSETTE PHARMACEUTICALS INC"~"Sprix (Ketorolac Tromethamine)"~5/14/2010 0:00:00~"Original"~1~"1551-2"~"PMR"~"Pediatric Research Equity Act"~1551.00~2~"PMR 1551-2: An open label pharmacokinetic and safety study of SPRIX in pediatric patients ages 12 to less than 17 years of age"~"Submitted"~"S-010 was submitted to fulfill this PREA PMR. User Fee not paid, the
application was unacceptable for filing."~5/31/2014 0:00:00~6/2/2026 0:00:00
384715~"CDER"~"NDA"~22382.00~"COSETTE PHARMACEUTICALS INC"~"Sprix (Ketorolac Tromethamine)"~5/14/2010 0:00:00~"Original"~1~"1551-3"~"PMR"~"Pediatric Research Equity Act"~1551.00~3~"PMR 1551-3: An open label pharmacokinetic and safety study of SPRIX in pediatric patients ages 2 to less than 12 years of age"~"Delayed"~"The Final Report milestone was missed because the Applicant must
resubmit the Final Report with a complete supplement for review. Original Final Report Due Date: 06/30/2015. There have been three Deferral Extensions granted, the latest per FDA letter dated 11/09/2022, which extended the Final Report due date to 06/30/2023. There have been two Non-Compliance letters issued
09/08/2021 and 04/16/2024. There has been no response submitted by the Applicant to the 04/16/2024 letter."~6/30/2023 0:00:00~6/2/2026 0:00:00
384716~"CDER"~"NDA"~22399.00~"AZURITY PHARMACEUTICALS INC"~"Horizant (Gabapentin Enacarbil) "~4/6/2011 0:00:00~"Original"~1~"3644-1"~"PMR"~"505 (o)(3)"~3644.00~1~"PMR 3644-1: In a healthy non-drug dependent population with drug abuse experience with sedative drugs, evaluate the abuse potential of gabapentin enacarbil. This clinical trial will evaluate gabapentin enacarbil in a cross-over design with comparison to placebo and a positive control (such as diazepam) regarding abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and drug pharmacokinetics."~"Submitted"~~4/30/2021 0:00:00~6/4/2026 0:00:00
384717~"CDER"~"NDA"~22399.00~"AZURITY PHARMACEUTICALS INC"~"Horizant (Gabapentin Enacarbil) "~4/6/2011 0:00:00~"Original"~1~"3644-2"~"PMR"~"505 (o)(3)"~3644.00~2~"PMR 3644-2: In a healthy non-drug dependent population with drug abuse experience with opioids, evaluate the abuse potential of gabapentin enacarbil taken concomitantly with an opioid, such as oxycodone, investigating a range of doses of gabapentin enacarbil combined with the opioid. This clinical trial will evaluate the gabapentin enacarbil/opioid combinations in a cross-over design with comparison to placebo, gabapentin enacarbil alone, and a moderate dose of the opioid taken alone as a positive control. The assessments will include abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and pharmacokinetics of both gabapentin enacarbil and the opioid (oxycodone) with the goal of characterizing any additive or synergistic effects on the abuse potential and physiologic effects of co-administered gabapentin enacarbil and opioid."~"Submitted"~~8/31/2021 0:00:00~6/4/2026 0:00:00
384718~"CDER"~"NDA"~22404.00~"GALT PHARMACEUTICALS LLC"~"Oravig (Miconazole) "~4/16/2010 0:00:00~"Original"~1~"1635-1"~"PMR"~"Pediatric Research Equity Act"~1635.00~1~"PMR 1635-1: Deferred pediatric study under PREA investigating ORAVIG safety, pharmacokinetics, efficacy and compliance with use instructions in patients with oropharyngeal candidiasis ages  > 5 to < or = 17 years."~"Delayed"~"Non Compliance letter was issued 4-4-2014."~3/31/2014 0:00:00~7/23/2025 0:00:00
384719~"CDER"~"NDA"~22408.00~"CIPHER PHARMACEUTICALS INC"~"Natroba (Spinosad)"~1/18/2011 0:00:00~"Supplement"~8~"4062-1"~"PMR"~"Pediatric Research Equity Act"~4062.00~1~"PMR 4062-1: Conduct a pediatric PK and safety study in pediatric subjects 1 month to 3 years 11 months of age with a total of 50 pediatric subjects with scabies infestation being enrolled, with a minimum of 16 completers in the PK evaluation, and with adequate number of subjects within the lowest age range."~"Delayed"~"The Final Report milestone was missed, due to lack of
enrollment. The Original Final Report Due Date:06/2023;
Deferral Extension granted per FDA letter dated 11/21/2023. The Final Report was submitted on 3/31/2026
and is under review.
"~3/31/2026 0:00:00~3/18/2026 0:00:00
384720~"CDER"~"NDA"~22408.00~"CIPHER PHARMACEUTICALS INC"~"Natroba (Spinosad)"~1/18/2011 0:00:00~"Supplement"~10~"4062-1"~"PMR"~"Pediatric Research Equity Act"~4062.00~1~"PMR 4062-1: Conduct a pediatric PK and safety study in pediatric subjects 1 month to 3 years 11 months of age with a total of 50 pediatric subjects with scabies infestation being enrolled, with a minimum of 16 completers in the PK evaluation, and with adequate number of subjects within the lowest age range."~"Delayed"~"The Final Report milestone was missed, due to lack of
enrollment. The Original Final Report Due Date:06/2023;
Deferral Extension granted per FDA letter dated 11/21/2023. The Final Report was submitted on 3/31/2026
and is under review.
"~3/31/2026 0:00:00~3/18/2026 0:00:00
384721~"CDER"~"NDA"~22416.00~"SUMITOMO PHARMA AMERICA INC"~"Aptiom (Eslicarbazepine Acetate)"~11/8/2013 0:00:00~"Original"~1~"4085-1"~"PMR"~"505 (o)(3)"~4085.00~1~"PMR 4085-1: Conduct in vitro patch clamp studies to assess eslicarbazepine s inhibitory profile on cardiac sodium channel (NaV1.5), including
establishing eslicarbazepine s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~6/18/2026 0:00:00
384722~"CDER"~"BLA"~125521.00~"Eli Lilly and Company"~"Taltz (Ixekizumab)"~3/22/2016 0:00:00~"Original"~1~"3049-2"~"PMR"~"505 (o)(3)"~3049.00~2~"PMR 3049-2: Conduct a retrospective cohort study using administrative databases to identify pregnancy outcomes in a cohort of women exposed to ixekizumab and a nonixekizumab systemic medication exposure cohort. The outcomes will include major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age births. This study may use multiple data sources in order to obtain a sufficient sample size as women with psoriasis are counseled to avoid systemic  treatments while trying to conceive and during the course of pregnancy."~"Delayed"~"59 of the targeted 138 patients have been enrolled into this study. Per FDA letter dated 04/04/2025, the applicant s request for release was denied. The final report due date has passed. The applicant is compiling a report of the data from their study for submission to the FDA."~6/30/2022 0:00:00~5/19/2026 0:00:00
384723~"CDER"~"BLA"~125521.00~"Eli Lilly and Company"~"Taltz (Ixekizumab)"~3/22/2016 0:00:00~"Original"~1~"3049-3"~"PMR"~"505 (o)(3)"~3049.00~3~"PMR 3049-3: Conduct a prospective registry-based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to  ixekizumab during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous  abortions, stillbirths, elective terminations, small for gestational age births, and any other adverse pregnancy outcomes. These outcomes will be assessed  throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~5/31/2030 0:00:00~5/19/2026 0:00:00
384724~"CDER"~"BLA"~125521.00~"Eli Lilly and Company"~"Taltz (Ixekizumab)"~3/22/2016 0:00:00~"Original"~1~"3049-4"~"PMR"~"505 (o)(3)"~3049.00~4~"PMR 3049-4: Conduct a prospective, observational study to assess the long-term safety of ixekizumab compared to other therapies used in the treatment of adults with  moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy in the course of actual clinical care. The study s primary outcome is malignancy. Secondary outcomes include, but are not limited to, serious infection,  tuberculosis, opportunistic infections, hypersensitivity reactions, autoimmune  disease, neurologic or demyelinating disease, cardiovascular, gastrointestinal or  hematologic adverse events. Describe and justify the choice of appropriate comparator population(s) for the primary objective. Design the study around a  testable hypothesis to assess, with sufficient sample size and power, a clinically meaningful increase in malignancy risk above the comparator background rate(s),  with a pre-specified statistical analysis method. Specify concise case definitions and validation algorithms for both primary and secondary outcomes. For the  ixekizumab-exposed and comparator(s), clearly define the study drug initiation period and any exclusion and inclusion criteria. Enroll patients over an initial 4-  year period and follow for a minimum of 8 years from the time of enrollment."~"Ongoing"~~5/31/2030 0:00:00~5/19/2026 0:00:00
384725~"CDER"~"BLA"~125522.00~"Amgen Inc."~"Repatha (Evolocumab)"~8/27/2015 0:00:00~"Original"~1~"2946-10"~"PMR"~"505 (o)(3)"~2946.00~10~"PMR 2946-10: Conduct a worldwide, single-arm, descriptive study that actively collects prospective and retrospective data in women exposed to Repatha (evolocumab) during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant in all exposed pregnancies of which you become aware. Infant outcomes should be assessed through at least the first year of life. The single arm pregnancy study will collect information for a minimum of 10 years from the date of market approval for Repatha."~"Ongoing"~~9/30/2026 0:00:00~10/24/2025 0:00:00
384726~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~19~"3089-1"~"PMR"~"Accelerated Approval"~3089.00~1~"PMR 3089-1: Conduct a randomized phase 3 clinical trial in classical Hodgkin lymphoma that verifies and isolates the clinical benefit of nivolumab for patients with classical Hodgkin lymphoma. The primary endpoint would be progression-free survival as determined by an independent review committee. Overall survival would be a key secondary endpoint."~"Fulfilled"~"Per FDA letter dated 03/20/2026, this PMR/PMC has been fulfilled."~12/31/2026 0:00:00~2/19/2026 0:00:00
384727~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~31~"3089-1"~"PMR"~"Accelerated Approval"~3089.00~1~"PMR 3089-1: Conduct a randomized phase 3 clinical trial in classical Hodgkin lymphoma that verifies and isolates the clinical benefit of nivolumab for patients with classical Hodgkin lymphoma. The primary endpoint would be progression-free survival as determined by an independent review committee. Overall survival would be a key secondary endpoint."~"Fulfilled"~"Per FDA letter dated 03/20/2026, this PMR/PMC has been fulfilled."~12/31/2026 0:00:00~2/19/2026 0:00:00
384728~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~34~"3243-2"~"PMC"~"506B PMC"~3243.00~2~"PMC 3243-2: Commitment to support the availability through an appropriate analytical and clinical validation study using clinical trial data that will support labeling of an  immunohistochemistry based in vitro diagnostic device that is essential to the safe and effective use of nivolumab for patients with tumors that are mismatch repair  deficient."~"Submitted"~~9/30/2021 0:00:00~2/19/2026 0:00:00
384729~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~34~"3243-3"~"PMC"~"506B PMC"~3243.00~3~"PMC 3243-3: Commitment to support the availability through an appropriate analytical and clinical validation study using clinical trial data that will support labeling of a nucleic acid-based in vitro diagnostic device that is essential to the safe and effective use of nivolumab for patients with tumors that are microsatellite instability high."~"Submitted"~~9/30/2021 0:00:00~2/19/2026 0:00:00
384730~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~180~"4859-1"~"PMC"~"506B PMC"~4859.00~1~"PMC 4859-1: Complete clinical trial, KEYNOTE-689 (NCT03765918), titled,  Study of Pembrolizumab Given Prior to Surgery and in Combination With Radiotherapy Given Post-surgery for Advanced Head and Neck Squamous Cell Carcinoma (MK-3475-689)  to provide long-term efficacy data. Provide the final overall survival (OS) analysis once the required number of events for OS, as prespecified in the statistical analysis plan, have occurred in the intention-to-treat population."~"Ongoing"~~5/31/2027 0:00:00~10/28/2025 0:00:00
384731~"CDER"~"BLA"~125514.00~"MSD International Business GmbH"~"Keytruda (pembrolizumab)"~9/4/2014 0:00:00~"Supplement"~182~"4845-1"~"PMC"~"506B PMC"~4845.00~1~"PMC 4845-1: Conduct an appropriate analytical and clinical validation study, using clinical trial data from KEYNOTE-590, to support the availability of an in vitro diagnostic device to detect PD-L1 expression that is essential for the safe and effective use of pembrolizumab in patients with esophageal carcinoma."~"Fulfilled"~~12/31/2025 0:00:00~10/28/2025 0:00:00
384732~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~63~"3449-3"~"PMC"~"506B PMC"~3449.00~3~"PMC 3449-3: Commitment to support the availability through an appropriate analytical and clinical validation study using clinical trial data that will support labeling of an immunohistochemistry-based in vitro diagnostic device that is essential to the safe and effective use of nivolumab for patients with tumors that are mismatch repair
deficient."~"Submitted"~~7/31/2024 0:00:00~2/19/2026 0:00:00
384733~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-10"~"PMR"~"505 (o)(3)"~3462.00~10~"PMR 3462-10: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of rifampin, a CYP3A, CYP2C19, and UGT inducer, on the single dose pharmacokinetics of Diacomit (stiripentol) in healthy volunteers, and to assess the magnitude of the decrease in stiripentol exposure. You should also evaluate metabolite concentrations if any of the identified metabolites is less polar than the parent drug (stiripentol) and has an AUC =25% of the AUC of stiripentol. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 9/30/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384734~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-11"~"PMR"~"505 (o)(3)"~3462.00~11~"PMR 3462-11: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of caffeine (a sensitive CYP1A2 substrate) in healthy volunteers to address the potential for excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. 
"~"Delayed"~"The final protocol submission, study completion, and final report
submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 11/6/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384735~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-12"~"PMR"~"505 (o)(3)"~3462.00~12~"PMR 3462-12: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of a CYP2B6 sensitive substrate in healthy volunteers. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s annual report a revised draft protocol was submitted on 9/30/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384736~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-13"~"PMR"~"505 (o)(3)"~3462.00~13~"PMR 3462-13: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of a CYP3A4 sensitive substrate in healthy volunteers. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 11/6/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384737~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-14"~"PMR"~"505 (o)(3)"~3462.00~14~"PMR 3462-14: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of a CYP2C19 sensitive substrate in healthy volunteers to address the potential for excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. 
"~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 11/6/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384738~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-15"~"PMR"~"505 (o)(3)"~3462.00~15~"PMR 3462-15: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of a P-gp sensitive substrate in healthy volunteers to address the potential for excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 9/30/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384739~"CDER"~"NDA"~206709.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-16"~"PMR"~"505 (o)(3)"~3462.00~16~"PMR 3462-16: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of a BCRP sensitive substrate in healthy volunteers to address the potential for excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 9/30/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384740~"CDER"~"NDA"~206829.00~"CUBIST PHARMACEUTICALS LLC"~"Zerbaxa (Ceftolozane Sulfate and Tazobactam Sodium) "~12/19/2014 0:00:00~"Supplement"~8~"3637-1"~"PMR"~"Pediatric Research Equity Act"~3637.00~1~"PMR 3637-1: Conduct a safety and pharmacokinetic study in HABP/VABP in children from birth to less than 18 years of age."~"Fulfilled"~"Per FDA letter dated 05/12/2026, this PMR/PMC has been fulfilled."~6/30/2026 0:00:00~2/10/2026 0:00:00
384741~"CDER"~"NDA"~206910.00~"NOVARTIS PHARMACEUTICALS CORP"~"Jadenu (Deferasirox)"~3/30/2015 0:00:00~"Original"~1~"2888-5"~"PMR"~"505 (o)(3)"~2888.00~5~"PMR 2888-5: Conduct a study, using your established registry, to evaluate the risk of growth inhibition in children (aged 10 to <18 years old at enrollment) with NTDT and treated with deferasirox for documented iron overload. Follow at least 40 children for up to 5 years to assess and analyze the long-term safety of treatment with deferasirox, including an assessment of growth, compared to children on a regular transfusion program receiving deferasirox (based on historical data). Provide annual interim reports on enrollment and outcomes."~"Delayed"~"The Study Completion and Final Report Submission milestones were missed because there were challenges in enrollment in the study. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 09/22/2022."~12/31/2021 0:00:00~5/22/2026 0:00:00
384742~"CDER"~"NDA"~206910.00~"NOVARTIS PHARMACEUTICALS CORP"~"Jadenu (Deferasirox)"~3/30/2015 0:00:00~"Original"~1~"2888-8"~"PMR"~"505 (o)(3)"~2888.00~8~"PMR 2888-8: Conduct a trial to assess ocular toxicity in patients receiving deferasirox. Examinations should include distance visual acuity, applanation tonometry, lens  photography, and wide angle fundus photography of retina and optic nerve and should be done at baseline (prior to deferasirox initiation) and at six month  intervals. At least 60 patients should complete 2 years of follow-up."~"Fulfilled"~~12/31/2019 0:00:00~5/22/2026 0:00:00
384743~"CDER"~"NDA"~6488.00~"FRESENIUS KABI USA LLC"~"Xylocaine-MPF (Epinephrine and Lidocaine Hydrochloride)"~11/19/1948 0:00:00~"Supplement"~90~"3321-1"~"PMR"~"505 (o)(3)"~3321.00~1~"PMR 3321-1: Conduct a simulation study to characterize the potential leachables profile from your drug product container closure system that employs the [...] adhesive that will be used on the [...] labels that are placed on the [...] ampoules [...] Evaluate at least three batches of your to-be-marketed drug product for leachables and include assessments at multiple timepoints over the course of your  accelerated storage conditions in order to identify trends in potential  leachable levels over time. Batches for each of your drug product presentations that employ this new adhesive should be tested unless you can justify that there would be no difference in leachable profile across the drug product presentations or that the presentations chosen represent the  worst-case scenario. The materials tested should include any secondary container closure systems, if present, and be subjected to the same to-bemarketed [...] as appropriate. Establish an AET of [...] mcg/day. Based on these results, provide a revised toxicological risk assessment for any leachable above [...] mcg/day that addresses both systemic and local tissue effects, including the potential for neurotoxicity if the product is inadvertently administered via the intrathecal route."~"Submitted"~~2/28/2019 0:00:00~1/15/2026 0:00:00
384744~"CDER"~"NDA"~6488.00~"FRESENIUS KABI USA LLC"~"Xylocaine-MPF (Epinephrine and Lidocaine Hydrochloride)"~11/19/1948 0:00:00~"Supplement"~90~"3321-2"~"PMR"~"505 (o)(3)"~3321.00~2~"PMR 3321-2: Conduct a long-term leachables study to adequately characterize the leachables profile from your drug product container closure system that employs the [...] adhesive that will be used on the [...] labels that are placed on the [...] ampoules [...] Evaluate at least three batches of your to-be-marketed drug product for leachables and include assessments at multiple timepoints over the course of your stability studies, through the proposed expiry duration, in order to identify trends in leachable levels over time. Batches for each  of your drug product presentations that employ this new adhesive should be tested unless you can justify that there would be no difference in leachable profile across the drug product presentations or that the  presentations chosen represent the worst-case scenario. The materials tested should include any secondary container closure systems, if present, and be subjected to the same to-be-marketed [...], as appropriate. Establish an AET of [...] mcg/day. Based on these results, provide a revised toxicological risk assessment for any leachable above [...] mcg/day that addresses both systemic and local tissue effects, including the potential for neurotoxicity if the product is inadvertently  administered via the intrathecal route. Submit annual reports over the course of this study that discuss trends in the leachable profile to date."~"Submitted"~~8/31/2021 0:00:00~1/15/2026 0:00:00
384745~"CDER"~"NDA"~6488.00~"FRESENIUS KABI USA LLC"~"Xylocaine-MPF (Epinephrine and Lidocaine Hydrochloride)"~11/19/1948 0:00:00~"Supplement"~95~"3321-1"~"PMR"~"505 (o)(3)"~3321.00~1~"PMR 3321-1: Conduct a simulation study to characterize the potential leachables profile from your drug product container closure system that employs the [...] adhesive that will be used on the [...] labels that are placed on the [...] ampoules [...] Evaluate at least three batches of your to-be-marketed drug product for leachables and include assessments at multiple timepoints over the course of your  accelerated storage conditions in order to identify trends in potential  leachable levels over time. Batches for each of your drug product presentations that employ this new adhesive should be tested unless you can justify that there would be no difference in leachable profile across the drug product presentations or that the presentations chosen represent the  worst-case scenario. The materials tested should include any secondary container closure systems, if present, and be subjected to the same to-bemarketed [...] as appropriate. Establish an AET of [...] mcg/day. Based on these results, provide a revised toxicological risk assessment for any leachable above [...] mcg/day that addresses both systemic and local tissue effects, including the potential for neurotoxicity if the product is inadvertently administered via the intrathecal route."~"Submitted"~~2/28/2019 0:00:00~1/15/2026 0:00:00
384746~"CDER"~"NDA"~6488.00~"FRESENIUS KABI USA LLC"~"Xylocaine-MPF (Epinephrine and Lidocaine Hydrochloride)"~11/19/1948 0:00:00~"Supplement"~95~"3321-2"~"PMR"~"505 (o)(3)"~3321.00~2~"PMR 3321-2: Conduct a long-term leachables study to adequately characterize the leachables profile from your drug product container closure system that employs the [...] adhesive that will be used on the [...] labels that are placed on the [...] ampoules [...] Evaluate at least three batches of your to-be-marketed drug product for leachables and include assessments at multiple timepoints over the course of your stability studies, through the proposed expiry duration, in order to identify trends in leachable levels over time. Batches for each  of your drug product presentations that employ this new adhesive should be tested unless you can justify that there would be no difference in leachable profile across the drug product presentations or that the  presentations chosen represent the worst-case scenario. The materials tested should include any secondary container closure systems, if present, and be subjected to the same to-be-marketed [...], as appropriate. Establish an AET of [...] mcg/day. Based on these results, provide a revised toxicological risk assessment for any leachable above [...] mcg/day that addresses both systemic and local tissue effects, including the potential for neurotoxicity if the product is inadvertently  administered via the intrathecal route. Submit annual reports over the course of this study that discuss trends in the leachable profile to date."~"Submitted"~~8/31/2021 0:00:00~1/15/2026 0:00:00
384747~"CDER"~"NDA"~8762.00~"VIATRIS SPECIALTY LLC"~"Dilantin-125 (Phenytoin Sodium)"~1/6/1953 0:00:00~"Original"~1~"4089-1"~"PMR"~"505 (o)(3)"~4089.00~1~"PMR 4089-1: Conduct in vitro patch clamp studies to assess Dilantin s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Dilantin s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~3/27/2026 0:00:00
384748~"CDER"~"NDA"~16608.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tegretol (Carbamazepine) "~3/11/1968 0:00:00~"Original"~1~"4094-1"~"PMR"~"505 (o)(3)"~4094.00~1~"PMR 4094-1: Conduct in vitro patch clamp studies to assess Tegretol s and Tegretol XR s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Tegretol s and Tegretol XR s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~5/6/2026 0:00:00
384749~"CDER"~"NDA"~16822.00~"B BRAUN MEDICAL INC"~"Amino Acids"~9/24/1971 0:00:00~"Original"~1~"3756-1"~"PMR"~"505 (o)(3)"~3756.00~1~"PMR 3756-1: A postmarketing study that will allow you to test for and control the levels of the following [...] impurities in your product: [...] that are present during the manufacturing process and have the potential to contaminate your product. As part of your post marketing study, you will need to 1) develop a validated method for testing the finished product for the [...] impurities as stated above, and 2) analyze at least three batches of your product using the validated method."~"Fulfilled"~~3/31/2016 0:00:00~4/7/2017 0:00:00
384750~"CDER"~"NDA"~22416.00~"SUMITOMO PHARMA AMERICA INC"~"Aptiom (Eslicarbazepine Acetate)"~11/8/2013 0:00:00~"Original"~1~"2099-7"~"PMR"~"Pediatric Research Equity Act"~2099.00~7~"PMR 2099-7: A prospective, randomized, controlled, double-blind, efficacy and safety study of eslicarbazepine acetate for the adjunctive treatment of partial onset seizures in children ages 1 month to < 4 years. The primary efficacy endpoint must examine  seizure frequency based upon Video/EEG data. Safety must be evaluated. Subgroup analyses of the effect of the concomitant use of enzyme-inducing anticonvulsants (i.e., carbamazepine, phenytoin, phenobarbital or primidone) on  the safety and efficacy of eslicarbazepine acetate must be performed. At least 75% of children in the study should be less than or equal to 2 years old."~"Delayed"~"Original Final Report Due Date: 07/31/2024. Per FDA letter dated 03/30/2021, final report due date extended to 05/31/2025. Second Deferral Extension Granted per FDA letter dated 04/09/2025."~6/30/2032 0:00:00~6/18/2026 0:00:00
384751~"CDER"~"NDA"~22416.00~"SUMITOMO PHARMA AMERICA INC"~"Aptiom (Eslicarbazepine Acetate)"~11/8/2013 0:00:00~"Original"~1~"2099-8"~"PMR"~"Pediatric Research Equity Act"~2099.00~8~"PMR 2099-8: Long term extension study for PMR 2099-7 (A prospective, randomized, controlled, double-blind, efficacy and safety study of eslicarbazepine acetate for  the adjunctive treatment of partial onset seizures in children ages 1 month to < 4 years). Safety must be evaluated. Subgroup analyses of the effect of the concomitant use of enzyme-inducing anticonvulsants (i.e., carbamazepine, phenytoin, phenobarbital or primidone) on the safety of eslicarbazepine acetate  must be performed. At least 75% of children in the study should be less than or equal to 2 years old."~"Delayed"~"Original Final Report Due Date: 05/31/2025; Deferral Extension granted per FDA letter dated 04/09/2025. The final protocol submission and study completion milestones were also revised.
"~6/30/2032 0:00:00~6/18/2026 0:00:00
384752~"CDER"~"NDA"~22445.00~"HERON THERAPEUTICS INC"~"Sustol (Granisetron)"~8/9/2016 0:00:00~"Original"~1~"3094-8"~"PMR"~"505 (o)(3)"~3094.00~8~"PMR 3094-8: A prospective, repeat dose, open-label, trial to assess the safety of Sustol (granisetron) extended-release injection, for subcutaneous use, when administered to patients receiving chemotherapy, for 4 cycles."~"Released"~~4/30/2025 0:00:00~9/19/2025 0:00:00
384753~"CDER"~"NDA"~22468.00~"ACROTECH BIOPHARMA INC"~"Folotyn (Pralatrexate)"~9/24/2009 0:00:00~"Original"~1~"3086-1"~"PMR"~"505 (o)(3)"~3086.00~1~"PMR 3086-1: Evaluate the effect of hepatic impairment on the pharmacokinetics and safety of  Folotyn (pralatrexate). Submit a complete final report with all supporting datasets."~"Delayed"~"The Trial Completion and Final Report Submission Milestones were missed because of enrollment difficulties. The FDA issued a Notification of Missed Milestones letter on 11/08/2023."~6/30/2021 0:00:00~11/19/2025 0:00:00
384754~"CDER"~"NDA"~22468.00~"ACROTECH BIOPHARMA INC"~"Folotyn (Pralatrexate)"~9/24/2009 0:00:00~"Original"~1~"2179-2"~"PMR"~"Accelerated Approval"~2179.00~2~"PMR 2179-2: Characterize the comparative efficacy and safety of pralatrexate when used in combination with a treatment regimen such as CHOP versus the combination of Beleodaq plus CHOP, versus CHOP alone for the initial therapy of patients with PTCL. Perform a confirmatory, prospective randomized (1:1:1) trial of previously untreated patients with PTCL, with progression free survival (PFS) as the primary efficacy endpoint. Enroll a sufficient number of patients to characterize the efficacy and safety of each drug added to CHOP, versus CHOP alone. The PFS endpoint should be determined by a blinded independent review committee. PFS analysis should be performed when the trial has experienced the planned number of events necessary for trial completion. Using the same data cutoff date as the PFS analysis, perform an interim analysis of overall survival. Submit a complete final report with all supporting datasets."~"Delayed"~"Accrual of 25% of Subjects, Accrual of 50% of Subjects, Accrual of 75% of Subjects, Trial Completion, and Final Report Submission milestones were missed, because of difficulties in site activation."~1/31/2021 0:00:00~11/19/2025 0:00:00
384755~"CDER"~"NDA"~22468.00~"ACROTECH BIOPHARMA INC"~"Folotyn (Pralatrexate)"~9/24/2009 0:00:00~"Supplement"~12~"3086-1"~"PMR"~"505 (o)(3)"~3086.00~1~"PMR 3086-1: Evaluate the effect of hepatic impairment on the pharmacokinetics and safety of  Folotyn (pralatrexate). Submit a complete final report with all supporting datasets."~"Delayed"~"The Trial Completion and Final Report Submission Milestones were missed because of enrollment difficulties. The FDA issued a Notification of Missed Milestones letter on 11/08/2023."~6/30/2021 0:00:00~11/19/2025 0:00:00
384756~"CDER"~"BLA"~22472.00~"MannKind Corporation"~"Afrezza (insulin human)"~6/27/2014 0:00:00~"Original"~1~"2166-5"~"PMR"~"505 (o)(3)"~2166.00~5~"PMR 2166-5: Conduct a 5-year, randomized, controlled trial in 8,000-10,000 patients with type 2 diabetes to assess the serious potential risk of pulmonary malignancy with Afrezza use. The primary objective of the trial should be to compare the incidence of pulmonary malignancy observed with Afrezza to that observed in the standard of care control group. Secondary endpoints should include mortality due to pulmonary malignancy and allcause mortality. Randomization to Afrezza or standard of care should be 1 to 1. The patient population should be enriched with respect to lung cancer risk (i.e., predicted incidence of no less than 200/100,000 patient-year). The potential for detection bias should be adequately addressed in the trial design. Subjects who discontinue randomized intervention due to lack of efficacy or tolerability issues should continue to be followed for the outcomes of interest and prospective measures to encourage subject retention and capture outcomes in patients who withdraw or are lost to follow-up should be in place. Glucose control and glycemic rescue should be per standard of care. The trial must also include an assessment of cardiovascular risk based on prospectively defined major adverse cardiovascular events or MACE (i.e., cardiovascular death, non-fatal myocardial infarction, and non-fatal stroke). Also include as part of the trial a substudy (also with 1 to 1 randomization to either Afrezza or standard or care) to evaluate the long-term effect of Afrezza on pulmonary function. Patients in the substudy should have pulmonary function tests at baseline and every 6 months until end of treatment.
"~"Released"~~12/31/2023 0:00:00~8/25/2025 0:00:00
384757~"CDER"~"BLA"~22472.00~"MannKind Corporation"~"Afrezza (insulin human)"~6/27/2014 0:00:00~"Original"~1~"2166-6"~"PMR"~"Pediatric Research Equity Act"~2166.00~6~"PMR 2166-6: An open-label pharmacokinetic (PK), and multiple-dose safety and tolerability dose-titration study of Afrezza in pediatric patients ages 8 to 17 years (inclusive) with type 1 diabetes (Part 1), followed by a prospective, multicenter, open-label, randomized, controlled study comparing the efficacy and safety of prandial Afrezza to prandial subcutaneous rapidacting insulin used in combination with subcutaneous basal insulin in pediatric patients 4 to 17 years old (inclusive) with type 1 or type 2 diabetes (Part 2). Part 2 of the study should include a run-in phase, a 26-week randomized intervention phase, and a 26-week extension phase."~"Fulfilled"~"Per FDA letter dated 05/29/2026, this PMR/PMC has been fulfilled."~11/30/2025 0:00:00~8/25/2025 0:00:00
384758~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~63~"3449-4"~"PMC"~"506B PMC"~3449.00~4~"PMC 3449-4: Commitment to support the availability through an appropriate analytical and clinical validation study using clinical trial data that will support labeling of a nucleic acid- based in vitro diagnostic device that is essential to the safe and effective use of nivolumab for patients with tumors that are microsatellite instability-high."~"Submitted"~~7/31/2024 0:00:00~2/19/2026 0:00:00
384759~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~92~"4065-1"~"PMC"~"506B PMC"~4065.00~1~"PMC 4065-1: Submit the final overall survival analyses and datasets for the ongoing clinical trial CA209577  A Randomized, Multicenter, Double Blind, Phase III Study of Adjuvant Nivolumab or Placebo in Subjects With Resected Esophageal, or Gastroesophageal Junction Cancer,  to further characterize the clinical benefit of nivolumab as adjuvant therapy following neoadjuvant concurrent chemotherapy and radiation in patients with residual disease following complete resection of esophageal or gastroesophageal junction cancer."~"Fulfilled"~~12/31/2024 0:00:00~2/19/2026 0:00:00
384760~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~97~"4125-1"~"PMC"~"506B PMC"~4125.00~1~"PMC 4125-1: Submit the second interim and final overall survival analysis and datasets from clinical trial CheckMate 274 titled,  A Phase 3 Randomized,Double-blind, Multi-center Study of Adjuvant Nivolumab Versus Placebo in Subjects with High Risk Invasive Urothelial Carcinoma  to provide additional efficacy data for nivolumab as adjuvant therapy in patients with high-risk urothelial carcinoma that may inform product labeling."~"Ongoing"~~11/30/2026 0:00:00~2/19/2026 0:00:00
384761~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~97~"4125-2"~"PMC"~"506B PMC"~4125.00~2~"PMC 4125-2: Submit annual interim and final reports including meeting minutes and the charter from the Data Monitoring Committee analyzing the overall survival results of the upper tract urothelial carcinoma subgroup from clinical trial CheckMate 274 titled,  A Phase 3 Randomized, Double-blind, Multi-center Study of Adjuvant Nivolumab Versus Placebo in Subjects with High Risk Invasive Urothelial Carcinoma ."~"Ongoing"~~11/30/2026 0:00:00~2/19/2026 0:00:00
384762~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~112~"4245-1"~"PMC"~"506B PMC"~4245.00~1~"PMC 4245-1: Conduct clinical trial CA209816 titled,  Randomized, Open-Label, Phase 3 Trial of Nivolumab plus Ipilimumab or Nivolumab plus Platinum-Doublet Chemotherapy versus Platinum-Doublet Chemotherapy in Early Stage NSCLC (CheckMate 816: CHECKpoint pathway and nivoluMAb clinical Trial Evaluation 816),  to obtain additional long-term efficacy and safety data including the interim and final analysis of overall survival (OS)."~"Submitted"~~6/30/2025 0:00:00~2/19/2026 0:00:00
384763~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~112~"4245-2"~"PMC"~"506B PMC"~4245.00~2~"PMC 4245-2: Conduct an integrated analysis from postmarketing data sources or ongoing/planned clinical trials enrolling a sufficient representation of adults ages 75 years and older, and United States (U.S.) racial and ethnic minority patients that is reflective of the U.S. population of patients with NSCLC to further characterize the safety and efficacy of nivolumab in combination with platinum-doublet chemotherapy in these patients. In the analysis, include a sufficient number of patients enrolled in the U.S. ages 75 years and older, and a sufficient number of racial and ethnic minorities reflecting of the incidence of NSCLC in each subpopulation to allow for interpretation of the results."~"Delayed"~"The final protocol submission milestone was missed because additional time is needed to support a robust final protocol. A revised milestone was acknowledged in a letter dated 05/17/2023."~9/30/2028 0:00:00~2/19/2026 0:00:00
384764~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~127~"4711-1"~"PMC"~"506B PMC"~4711.00~1~"PMC 4711-1: Complete the ongoing clinical trial, CheckMate 77T (CA20977T, NCT04025879), and analyze the final overall survival (OS) once the
required 174 events for the OS endpoint have occurred, to further characterize the clinical benefit of nivolumab in combination with platinumcontaining chemotherapy as neoadjuvant treatment, followed by nivolumab continued as a single agent as adjuvant treatment after surgery for adults with resectable (tumors =4 cm or node positive) non-small cell lung cancer (NSCLC) and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements."~"Ongoing"~~4/30/2027 0:00:00~2/19/2026 0:00:00
384765~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~127~"4711-2"~"PMC"~"506B PMC"~4711.00~2~"PMC 4711-2: Conduct an integrated analysis of completed, ongoing, and planned trials of perioperative nivolumab for patients with resectable non-small cell lung cancer (NSCLC) to evaluate the contribution of phase of nivolumab when given in combination with platinum-containing chemotherapy as neoadjuvant treatment and continued as a single agent as adjuvant treatment after surgery for adults with resectable (tumors =4 cm or node positive) NSCLC and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements. This analysis should also include an evaluation of safety, including immunemediated adverse reactions that occur during each phase of treatment."~"Ongoing"~~4/30/2027 0:00:00~2/19/2026 0:00:00
384766~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~127~"4711-3"~"PMC"~"506B PMC"~4711.00~3~"PMC 4711-3: Conduct an integrated analysis of data from clinical trials and observational studies (e.g., real world evidence), post-marketing reports, and other sources to further characterize the safety and efficacy/effectiveness of the perioperative nivolumab regimen in older adults ages 75 years and older and patients of underrepresented racial and ethnic minority groups with non-small cell lung cancer (NSCLC). The analyses should support an evaluation of comparative efficacy/effectiveness and safety between the population primarily represented in the trial (CheckMate77T trial) and the aforementioned underrepresented racial and ethnic minority population as well as the aforementioned older adult population and younger patients represented in CheckMate77T."~"Ongoing"~~9/30/2028 0:00:00~2/19/2026 0:00:00
384767~"CDER"~"BLA"~125554.00~"Bristol-Myers Squibb Company"~"Opdivo (Nivolumab)"~12/22/2014 0:00:00~"Supplement"~133~"4852-1"~"PMC"~"506B PMC"~4852.00~1~"PMC 4852-1: Conduct an appropriate analytical validation study, based on clinical trial data from nivolumab studies to support development of a test to detect PD-L1 expression that is essential for the safe and effective use of nivolumab in patients with HER2-negative gastric/gastroesophageal junction adenocarcinoma and esophageal cancer."~"Ongoing"~~12/31/2026 0:00:00~2/19/2026 0:00:00
384768~"CDER"~"NDA"~206940.00~"ABBVIE INC"~"Viberzi (Eluxadoline)"~5/27/2015 0:00:00~"Original"~1~"2901-11"~"PMR"~"Pediatric Research Equity Act"~2901.00~11~"PMR 2901-11: Complete the ongoing phase 2 dose-ranging study to evaluate pharmacokinetics (PK), safety, and efficacy of Viberzi (eluxadoline) in pediatric patients 6 to 11 years of age with diarrhea-predominant irritable bowel syndrome (IBS-D)."~"Ongoing"~"8/15 subjects have been enrolled."~6/15/2027 0:00:00~7/22/2025 0:00:00
384782~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-8"~"PMR"~"505 (o)(3)"~3462.00~8~"PMR 3462-8: Conduct a thorough QT trial for Diacomit as per the ICH E14 guidelines."~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 2/24/2021. applicant s Annual Status Report, the revised protocol was submitted on 7/30/2020."~9/30/2020 0:00:00~10/17/2025 0:00:00
384783~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-9"~"PMR"~"505 (o)(3)"~3462.00~9~"PMR 3462-9: Conduct a clinical pharmacokinetic trial to determine an appropriate dose of Diacomit (stiripentol) to minimize toxicity in patients with varying degrees of hepatic impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 3/25/2020."~9/30/2022 0:00:00~10/17/2025 0:00:00
384784~"CDER"~"NDA"~17766.00~"B BRAUN MEDICAL INC"~"Nephramine (Amino Acid)"~2/24/1978 0:00:00~"Original"~1~"3756-1"~"PMR"~"505 (o)(3)"~3756.00~1~"PMR 3756-1: A postmarketing study that will allow you to test for and control the levels of the following [...] impurities in your product: [...] that are present during the manufacturing process and have the potential to contaminate your product. As part of your post marketing study, you will need to 1) develop a validated method for testing the finished product for the [...] impurities as stated above, and 2) analyze at least three batches of your product using the validated method."~"Fulfilled"~~3/31/2016 0:00:00~3/2/2022 0:00:00
384785~"CDER"~"NDA"~18582.00~"B BRAUN MEDICAL INC"~"Procalamine"~5/6/1982 0:00:00~"Original"~1~"3756-1"~"PMR"~"505 (o)(3)"~3756.00~1~"PMR 3756-1: A postmarketing study that will allow you to test for and control the levels of the following [...] impurities in your product: [...] that are present during the manufacturing process and have the potential to contaminate your product. As part of your post marketing study, you will need to 1) develop a validated method for testing the finished product for the [...] impurities as stated above, and 2) analyze at least three batches of your product using the validated method."~"Fulfilled"~~3/31/2016 0:00:00~4/7/2017 0:00:00
384786~"CDER"~"NDA"~18927.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tegretol (Carbamazepine) "~12/18/1987 0:00:00~"Original"~1~"4094-1"~"PMR"~"505 (o)(3)"~4094.00~1~"PMR 4094-1: Conduct in vitro patch clamp studies to assess Tegretol s and Tegretol XR s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Tegretol s and Tegretol XR s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~2/9/2026 0:00:00
384787~"CDER"~"NDA"~19034.00~"FRESENIUS KABI USA LLC"~"Dilaudid and Dilaudid-HP (Hydromorphone Hydrochloride)"~1/11/1984 0:00:00~"Supplement"~18~"347-1"~"PMR"~"Pediatric Research Equity Act"~347.00~1~"PMR 347-1: Deferred pediatric study under PREA for the management of pain in patients where an opioid analgesic is appropriate in pediatric patients ages 0 to 16 years."~"Delayed"~"The final report submission is missed. Sponsor working with contract research organization (CRO)."~12/31/2015 0:00:00~2/25/2026 0:00:00
384788~"CDER"~"NDA"~19430.00~"VIATRIS SPECIALTY LLC"~"EpiPen (Epinephrine) and EpiPen Jr (Epinephrine)"~12/22/1987 0:00:00~"Original"~1~"3301-1"~"PMR"~"505 (o)(3)"~3301.00~1~"PMR 3301-1: Develop reliability requirement(s) and specification(s) to mitigate the failure to fire risk, provide evidence to verify and validate the reliability specification(s), and control the reliability of EpiPen (epinephrine injection, USP) Auto-Injector  through design and manufacturing."~"Fulfilled"~~2/28/2019 0:00:00~2/20/2025 0:00:00
384789~"CDER"~"NDA"~19516.00~"KNOA PHARMA LLC"~"MS Contin (Morphine Sulfate)"~5/29/1987 0:00:00~"Original"~1~"3033-1"~"PMR"~"505 (o)(3)"~3033.00~1~"PMR 3033-1: A prospective, observational study designed to quantify the serious risks of misuse, abuse, and addiction associated with long-term use of opioid analgesics  for management of chronic pain among patients prescribed ER/LA opioid analgesics. This study must address at a minimum the following specific objectives: a. Estimate the incidence of misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of  psychiatric illness) on the risk of misuse, abuse, and addiction.  b. Evaluate and quantify other risk factors for misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors, psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships."~"Fulfilled"~~3/31/2020 0:00:00~2/28/2019 0:00:00
384790~"CDER"~"NDA"~19516.00~"KNOA PHARMA LLC"~"MS Contin (Morphine Sulfate)"~5/29/1987 0:00:00~"Original"~1~"3033-2"~"PMR"~"505 (o)(3)"~3033.00~2~"PMR 3033-2: An observational study designed to measure the incidence and predictors of opioid overdose and death (OOD), as well as opioid abuse/addiction, using patient  health records, insurance claims, and death records. This study must address at a minimum the following specific objectives: a. Estimate the incidence of abuse/addiction, overdose, and death associated with long-term use of opioid analgesics for chronic pain. Stratify overdose by  intentionality wherever possible. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other  clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of psychiatric illness) on the risk of  abuse/addiction, overdose, and death. b. Evaluate and quantify other risk factors for abuse/addiction, overdose, and  death associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors,  psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships. Stratify overdose by intentionality wherever possible."~"Fulfilled"~~9/30/2019 0:00:00~2/28/2019 0:00:00
384791~"CDER"~"NDA"~19516.00~"KNOA PHARMA LLC"~"MS Contin (Morphine Sulfate)"~5/29/1987 0:00:00~"Original"~1~"3033-6"~"PMR"~"505 (o)(3)"~3033.00~6~"PMR 3033-6: An observational study to develop and validate an algorithm using coded medical terminologies and other electronic healthcare data to identify opioid-related overdose and death."~"Fulfilled"~~12/31/2016 0:00:00~2/28/2019 0:00:00
384792~"CDER"~"NDA"~19516.00~"KNOA PHARMA LLC"~"MS Contin (Morphine Sulfate)"~5/29/1987 0:00:00~"Original"~1~"3033-11"~"PMR"~"505 (o)(3)"~3033.00~11~"PMR 3033-11: Conduct a clinical trial to estimate the serious risk for the development of hyperalgesia following the long-term use of high-dose ER/LA opioid analgesics  for at least one year to treat chronic pain. Include an assessment of risk relative to  efficacy."~"Delayed"~"The Trial Completion and Final Report milestones were missed because of continued discussions needed to reach agreement with the Agency on the Final Protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 8/26/2025."~8/31/2019 0:00:00~2/28/2019 0:00:00
384793~"CDER"~"NDA"~19537.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Cipro (Ciprofloxacin Hydrochloride) "~10/22/1987 0:00:00~"Supplement"~83~"2843-1"~"PMR"~"Animal Efficacy"~2843.00~1~"PMR 2843-1: Conduct a field study to evaluate the efficacy and safety of ciprofloxacin in the event of an attack with the intentional release of Y. pestis in the United States."~"Pending"~~~12/19/2025 0:00:00
384794~"CDER"~"NDA"~19627.00~"FRESENIUS KABI USA LLC"~"Diprivan (Propofol)"~10/2/1989 0:00:00~"Original"~1~"720-3"~"PMC"~"506B PMC"~720.00~3~"PMC 720-3: Zeneca is in the process of gathering and assessing clinical information about the use of propofol in ICU Sedation in children. During the past year, Zeneca has conducted two retrospective post-marketing surveys regarding the use of propofol in pediatric ICU sedation, one in the UK and one in Sweden. The firm anticipates the results from these surveys will be analized before the end of the year. Once the analyses are completed, Zeneca intends to have the data reviewed by an independent panel of experts and they will report the outcome of these reviews to the FDA by end 1Q94. As a result of these reviews, it is anticipated that Zeneca and the FDA and/or the ALSAC will jointly agree an appropriate course of action."~"Pending"~~~12/1/2025 0:00:00
384795~"CDER"~"NDA"~22488.00~"UPJOHN US 2 LLC"~"Lyrica (pregabalin)"~1/4/2010 0:00:00~"Original"~1~"3716-1"~"PMR"~"505 (o)(3)"~3716.00~1~"PMR 3716-1: In a healthy non-drug dependent population with drug abuse experience with opioids, evaluate the abuse potential of pregabalin taken concomitantly with an opioid, such as oxycodone, investigating a range of doses of pregabalin combined with the opioid. This clinical trial will evaluate the pregabalin/opioid combinations in a cross-over design with comparison to placebo, pregabalin alone, and a moderate dose of the opioid taken alone as a positive control. The assessments will include abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and pharmacokinetics of both pregabalin and the opioid with the goal of characterizing any additive or synergistic effects on the abuse potential and physiologic effects of co-administered pregabalin and opioid."~"Delayed"~"The Final Report milestone was missed because the Final Report submitted 01/31/2023 did not fulfill the PMR due to uninterpretable data, per FDA letter dated 01/26/2024. The Applicant proposed revised milestones and FDA determined the applicant demonstrated  good cause  for delay per FDA letter dated 07/11/2025. The Final Protocol was acknowledged by FDA on 03/25/2026."~8/31/2021 0:00:00~9/29/2025 0:00:00
384796~"CDER"~"NDA"~22496.00~"PACIRA PHARMACEUTICALS INC"~"Exparel (Bupivacaine)"~10/28/2011 0:00:00~"Original"~1~"1834-8"~"PMR"~"Pediatric Research Equity Act"~1834.00~8~"PMR 1834-8: A multicenter, randomized, open-label, assessor-blinded, parallel-group, standard-of-care-controlled study to evaluate the safety, pharmacokinetic profile, and comparative efficacy of a single intraoperative administration of EXPAREL via infiltration for postoperative analgesia in an invasive and likely painful procedure in which the systemic exposure to bupivacaine is expected to be high and requires prolonged in-patient hospitalization in children 2 to less than 6 years old: where local anesthetic administration is considered the standard of care; or where EXPAREL may provide additional safety and efficacy advantage over other standard-of-care pain regimens."~"Delayed"~"The draft protocol, final protocol and study completion milestones were missed and revised. Original Final Report Due Date: 05/31/2025. Deferral Extension granted per FDA letter dated 03/24/2025."~9/30/2031 0:00:00~12/11/2025 0:00:00
384797~"CDER"~"NDA"~22496.00~"PACIRA PHARMACEUTICALS INC"~"Exparel (Bupivacaine)"~10/28/2011 0:00:00~"Original"~1~"1834-9"~"PMR"~"Pediatric Research Equity Act"~1834.00~9~"PMR 1834-9: A multicenter, randomized, open-label, assessor-blinded, parallel-group, standard-of-care-controlled study to evaluate the safety, pharmacokinetic profile, and comparative efficacy of a single intraoperative administration of EXPAREL via infiltration for postoperative analgesia in an invasive and likely painful procedure in which the systemic exposure to bupivacaine is expected to be high and requires prolonged in-patient hospitalization in children from birth to less than 2 years old: where local anesthetic administration is considered the standard of care; or where EXPAREL may provide additional safety and efficacy advantage over other standard-of-care pain regimens."~"Delayed"~"The draft protocol, final protocol and study completion milestones were misse and revised. Original Final Report Due Date: 05/31/2025. Deferral Extension granted per FDA letter dated 03/24/2025."~9/30/2031 0:00:00~12/11/2025 0:00:00
384798~"CDER"~"NDA"~22499.00~"TRIS PHARMA INC"~"Nexiclon XR (Clonidine) "~12/3/2009 0:00:00~"Original"~1~"1579-1"~"PMR"~"Pediatric Research Equity Act"~1579.00~1~"PMR 1579-1: Deferred pediatric study under PREA for the treatment of hypertension in pediatric patients ages 1 to 18."~"Delayed"~"The drug product is discontinued but not withdrawn."~12/3/2011 0:00:00~1/25/2019 0:00:00
384799~"CDER"~"NDA"~22499.00~"TRIS PHARMA INC"~"Nexiclon XR (Clonidine) "~12/3/2009 0:00:00~"Original"~1~"1579-2"~"PMC"~"506B PMC"~1579.00~2~"PMC 1579-2: You will perform a ""Thorough QT Study"" as described in  ICH E14. This study can utilize your extended release formulation or an immediate release formulation. It can utilize a single 0.6 mg dose group and a single administration."~"Delayed"~"The drug product is discontinued but not withdrawn."~12/3/2011 0:00:00~1/25/2019 0:00:00
384800~"CDER"~"NDA"~22527.00~"NOVARTIS PHARMACEUTICALS CORP"~"Gilenya (Fingolimod)"~9/21/2010 0:00:00~"Original"~1~"4767-1"~"PMR"~"505 (o)(3)"~4767.00~1~"PMR 4767-1: Conduct an observational study to characterize patients exposed to Gilenya who develop PML in terms of age, duration of exposure to Gilenya, prior immunosuppressant use, lymphopenia, and anti-JCV antibody status. This study should be of at least 15 years duration to assess risk factors for the development of PML among patients with multiple sclerosis exposed to Gilenya. Potential risk factors to be assessed should include age, duration of exposure to Gilenya, prior immunosuppressant use, lymphopenia, and anti-JCV antibody status. The study should also determine the incidence of PML among patients exposed to Gilenya, both overall and by each potential risk factor."~"Pending"~~7/31/2042 0:00:00~11/17/2025 0:00:00
384801~"CDER"~"NDA"~22527.00~"NOVARTIS PHARMACEUTICALS CORP"~"Gilenya (Fingolimod)"~9/21/2010 0:00:00~"Original"~1~"1679-3"~"PMR"~"505 (o)(3)"~1679.00~3~"PMR 1679-3: Develop and maintain a prospective, observational pregnancy exposure registry  study conducted in the United States that compares the maternal, fetal, and infant  outcomes of women exposed to fingolimod during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, adverse effects on immune system development, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes will be assessed through at least the first year of life."~"Fulfilled"~~10/31/2017 0:00:00~11/17/2025 0:00:00
384802~"CDER"~"NDA"~22544.00~"ALMATICA PHARMA LLC"~"Gralise (Gabapentin)"~1/28/2011 0:00:00~"Original"~1~"3642-1"~"PMR"~"505 (o)(3)"~3642.00~1~"PMR 3642-1: In a healthy non-drug dependent population with drug abuse experience with sedative drugs, evaluate the abuse potential of gabapentin. This clinical trial will evaluate gabapentin in a cross-over design with comparison to placebo and a positive control (such as diazepam) regarding abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and drug pharmacokinetics."~"Submitted"~~4/30/2021 0:00:00~9/25/2023 0:00:00
384803~"CDER"~"NDA"~22544.00~"ALMATICA PHARMA LLC"~"Gralise (Gabapentin)"~1/28/2011 0:00:00~"Original"~1~"3642-2"~"PMR"~"505 (o)(3)"~3642.00~2~"PMR 3642-2: In a healthy non-drug dependent population with drug abuse experience with opioids, evaluate the abuse potential of gabapentin taken concomitantly with an opioid, such as oxycodone, investigating a range of doses of gabapentin combined with the opioid. This clinical trial will evaluate the gabapentin/opioid combinations in a cross-over design with comparison to placebo, gabapentin alone, and a moderate dose of the opioid taken alone as a positive control. The assessments will include abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and pharmacokinetics of both gabapentin and the opioid (oxycodone) with the goal of characterizing any additive or synergistic effects on the abuse potential and physiologic effects of co-administered gabapentin and opioid."~"Submitted"~~8/31/2021 0:00:00~9/25/2023 0:00:00
384804~"CDER"~"BLA"~125557.00~"Amgen Inc."~"Blincyto (Blinatumomab)"~12/3/2014 0:00:00~"Supplement"~8~"3230-1"~"PMR"~"505 (o)(3)"~3230.00~1~"PMR 3230-1: Characterize the impact, if any, of administration of blinatumomab as salvage therapy prior to allogeneic hematopoietic stem cell transplantation (HSCT) on early safety outcomes after HSCT as compared to standard of care (SOC) chemotherapy. Conduct an analysis of registry data (for example the Center for International Blood and Marrow Transplantation Research registry) to determine whether or not prior treatment with blinatumomab increases the risk of day-100  mortality or acute graft-versus-host disease as compared to SOC chemotherapy."~"Fulfilled"~~4/30/2025 0:00:00~1/16/2026 0:00:00
384805~"CDER"~"BLA"~125559.00~"Regeneron Pharmaceuticals, Inc."~"Praluent (Alirocumab)"~7/24/2015 0:00:00~"Original"~1~"2927-12"~"PMR"~"505 (o)(3)"~2927.00~12~"PMR 2927-12: Conduct a worldwide, single-arm, descriptive study that actively collects prospective and retrospective data in women exposed to Praluent (alirocumab) during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant in all exposed pregnancies of which you become aware. Infant outcomes should be assessed through at least the first year of life. The single arm pregnancy study will collect information for a minimum of 10 years from the date of market approval for Praluent (alirocumab)."~"Ongoing"~~1/31/2026 0:00:00~9/22/2025 0:00:00
384806~"CDER"~"BLA"~125561.00~"Alexion Pharmaceuticals, Inc."~"Kanuma (Sebelipase Alfa)"~12/8/2015 0:00:00~"Original"~1~"2920-1"~"PMC"~"506B PMC"~2920.00~1~"PMC 2920-1: Evaluate the long-term, prospective clinical outcomes of treatment with sebelipase alfa in adult and pediatric patients with LAL deficiency, including but not limited to progression of liver and cardiovascular diseases and changes in anthropometric  assessments (i.e., length/height z-scores, and weight z-scores). At a minimum, liver assessments will include results of liver biopsies and imaging studies, changes in liver synthetic function, evidence for clinical progression to end stage liver disease (e.g., assessed by the Model for End-Stage Liver Disease [MELD] score), receipt of  liver transplantation, and fatal outcomes. Cardiovascular assessments will include  incidence rates of non-fatal stroke, myocardial infarction, and cardiovascular death. Additional evaluations will include dosing regimens administered and reasons for any dose modifications. This trial will also collect data on the occurrence of any serious hypersensitivity reactions, such as anaphylaxis, as well as changes in antibody status  (i.e., detection and titers of binding and neutralizing antibodies, and detection of IgE antibodies). Eligible patients will be enrolled over an initial 3-year period and  followed for a minimum of 10 years from the time of enrollment or until death, whichever comes first. This trial may be conducted as a separate study or as a substudy  within the Lysosomal Acid Lipase registry."~"Ongoing"~~4/30/2030 0:00:00~2/5/2026 0:00:00
384807~"CDER"~"NDA"~200063.00~"AZURITY PHARMACEUTICALS INC"~"Contrave (Bupropion Hydrochloride and Naltrexone Hydrochloride)"~9/10/2014 0:00:00~"Original"~1~"2778-5"~"PMR"~"505 (o)(3)"~2778.00~5~"PMR 2778-5: A randomized, double-blind, placebo-controlled trial to evaluate the effect of long-term treatment with Contrave (naltrexone hydrochloride/bupropion  hydrochloride) extended-release tablets on the incidence of major adverse cardiovascular events (MACE) in obese and overweight subjects with cardiovascular disease or multiple cardiovascular risk factors. The primary objective of this trial should be to demonstrate that the upper bound of the 2-sided confidence interval for the estimated risk ratio comparing the incidence of MACE  (non-fatal myocardial infarction, non-fatal stroke, cardiovascular death) observed with Contrave to that observed in the placebo group is less than 1.4. The trial should be designed to provide sufficient data to reflect the  on-treatment   cardiovascular risk associated with Contrave. Sample size calculation should take into account that  on-study  events would be censored 365 days after treatment  discontinuation. The ongoing LIGHT trial will not be sufficient to meet this requirement; a new trial is required."~"Delayed"~"The January 2022 Final Report Submission milestone was missed and a Notification of Failure to Demonstrate Good Cause letter was issued. The applicant has since submitted a revised protocol to proceed with the trial. As of 9/9/25 5845 patients have been randomized."~1/31/2022 0:00:00~11/7/2025 0:00:00
384808~"CDER"~"NDA"~200533.00~"COLLEGIUM PHARMACEUTICAL INC"~"Nucynta ER (Tapentadol)"~8/25/2011 0:00:00~"Original"~1~"3033-1"~"PMR"~"505 (o)(3)"~3033.00~1~"PMR 3033-1: A prospective, observational study designed to quantify the serious risks of misuse, abuse, and addiction associated with long-term use of opioid analgesics  for management of chronic pain among patients prescribed ER/LA opioid analgesics. This study must address at a minimum the following specific objectives: a. Estimate the incidence of misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of  psychiatric illness) on the risk of misuse, abuse, and addiction.  b. Evaluate and quantify other risk factors for misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors, psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships."~"Fulfilled"~~3/31/2020 0:00:00~10/23/2025 0:00:00
384809~"CDER"~"NDA"~200533.00~"COLLEGIUM PHARMACEUTICAL INC"~"Nucynta ER (Tapentadol)"~8/25/2011 0:00:00~"Original"~1~"3033-2"~"PMR"~"505 (o)(3)"~3033.00~2~"PMR 3033-2: An observational study designed to measure the incidence and predictors of opioid overdose and death (OOD), as well as opioid abuse/addiction, using patient  health records, insurance claims, and death records. This study must address at a minimum the following specific objectives: a. Estimate the incidence of abuse/addiction, overdose, and death associated with long-term use of opioid analgesics for chronic pain. Stratify overdose by  intentionality wherever possible. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other  clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of psychiatric illness) on the risk of  abuse/addiction, overdose, and death. b. Evaluate and quantify other risk factors for abuse/addiction, overdose, and  death associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors,  psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships. Stratify overdose by intentionality wherever possible."~"Fulfilled"~~9/30/2019 0:00:00~10/23/2025 0:00:00
384810~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-10"~"PMR"~"505 (o)(3)"~3462.00~10~"PMR 3462-10: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of rifampin, a CYP3A, CYP2C19, and UGT inducer, on the single dose pharmacokinetics of Diacomit (stiripentol) in healthy volunteers, and to assess the magnitude of the decrease in stiripentol exposure. You should also evaluate metabolite concentrations if any of the identified metabolites is less polar than the parent drug (stiripentol) and has an AUC =25% of the AUC of stiripentol. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 9/30/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384811~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-11"~"PMR"~"505 (o)(3)"~3462.00~11~"PMR 3462-11: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of caffeine (a sensitive CYP1A2 substrate) in healthy volunteers to address the potential for excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. 
"~"Delayed"~"The final protocol submission, study completion, and final report
submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 11/6/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384812~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-12"~"PMR"~"505 (o)(3)"~3462.00~12~"PMR 3462-12: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of a CYP2B6 sensitive substrate in healthy volunteers. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s annual report a revised draft protocol was submitted on 9/30/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384813~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-13"~"PMR"~"505 (o)(3)"~3462.00~13~"PMR 3462-13: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of a CYP3A4 sensitive substrate in healthy volunteers. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 11/6/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384814~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-14"~"PMR"~"505 (o)(3)"~3462.00~14~"PMR 3462-14: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of a CYP2C19 sensitive substrate in healthy volunteers to address the potential for excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. 
"~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 11/6/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384815~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-15"~"PMR"~"505 (o)(3)"~3462.00~15~"PMR 3462-15: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of a P-gp sensitive substrate in healthy volunteers to address the potential for excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 9/30/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384816~"CDER"~"NDA"~207223.00~"BIOCODEX SA"~"Diacomit (Stiripentol) "~8/20/2018 0:00:00~"Original"~1~"3462-16"~"PMR"~"505 (o)(3)"~3462.00~16~"PMR 3462-16: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of Diacomit (stiripentol) on the single dose pharmacokinetics of a BCRP sensitive substrate in healthy volunteers to address the potential for excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Study Design, Data Analysis, and Clinical Implications Guidance for Industry. "~"Delayed"~"The final protocol submission, study completion, and final report submission milestones were missed. Per the applicant s Annual Status Report, the revised protocol was submitted on 9/30/2019."~9/30/2020 0:00:00~10/17/2025 0:00:00
384817~"CDER"~"NDA"~207356.00~"INSMED INC"~"Arikayce (Amikacin)"~9/28/2018 0:00:00~"Original"~1~"3480-1"~"PMR"~"Accelerated Approval"~3480.00~1~"PMR 3480-1: Conduct a randomized, double-blind, placebo-controlled clinical trial to assess and describe the clinical benefit of ARIKAYCE in patients with nontuberculous mycobacterial (NTM) lung disease caused by MAC. The trial will evaluate the effect of ARIKAYCE on a clinically meaningful endpoint, as compared to an appropriate control in the intended patient population of patients with MAC infection."~"Delayed"~"The Trial Completion Milestone date was missed and the FDA issued a missed milestone letter on 07/25/2023. The study sample size was increased. An Acknowledge revised PMR milestones letter was issued on 3-05-2025"~6/30/2024 0:00:00~11/19/2025 0:00:00
384818~"CDER"~"NDA"~207648.00~"FRESENIUS KABI USA LLC"~"SMOFlipid (Fish Oil, Medium Chain Triglycerides, Olive Oil, and Soybean Oil)"~7/13/2016 0:00:00~"Supplement"~5~"4240-1"~"PMR"~"505 (o)(3)"~4240.00~1~"PMR 4240-1: A single-arm open-label safety study of SMOFlipid to evaluate the risk of developing one or both of the following adverse reactions:   essential fatty acid deficiency (EFAD)   parenteral nutrition-associated cholestasis (PNAC) in the following pa tient populations who are anticipated to need 8 weeks or longer of parenteral nutrition treatment:   pediatric patients 1 month of age and older; there shou ld be adequate patient representation in each of these age groups:1 month - 2 years; 2 - 11 years; 12-17 years   adults"~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 5/10/2023. Per the applicant's annual status report one patient was enrolled."~9/30/2026 0:00:00~8/27/2025 0:00:00
384819~"CDER"~"NDA"~19815.00~"TAKEDA PHARMACEUTICALS USA INC"~"ProAmatine (Midodrine Hydrochloride)"~9/6/1996 0:00:00~"Original"~1~"825-1"~"PMR"~"Accelerated Approval"~825.00~1~"PMR 825-1: Applicant to conduct a Phase 4 study to confirm the clinical benefit of midodrine. Protocol drafts submitted at 7/18/96 meeting.  Protocols are 401, 402 and 403."~"Submitted"~"The Agency continues to discuss this application and PMR."~3/31/2015 0:00:00~11/3/2025 0:00:00
384820~"CDER"~"NDA"~19847.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Cipro IV (Ciprofloxacin)"~12/26/1990 0:00:00~"Supplement"~55~"2843-1"~"PMR"~"Animal Efficacy"~2843.00~1~"PMR 2843-1: Conduct a field study to evaluate the efficacy and safety of ciprofloxacin in the event of an attack with the intentional release of Y. pestis in the United States."~"Pending"~~~4/24/2026 0:00:00
384821~"CDER"~"NDA"~19857.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Cipro IV (Ciprofloxacin)"~12/26/1990 0:00:00~"Supplement"~63~"2843-1"~"PMR"~"Animal Efficacy"~2843.00~1~"PMR 2843-1: Conduct a field study to evaluate the efficacy and safety of ciprofloxacin in the event of an attack with the intentional release of Y. pestis in the United States."~"Pending"~~~4/25/2024 0:00:00
384822~"CDER"~"NDA"~20006.00~"B BRAUN MEDICAL INC"~"Mannitol  "~7/26/1993 0:00:00~"Original"~1~"3756-1"~"PMR"~"505 (o)(3)"~3756.00~1~"PMR 3756-1: A postmarketing study that will allow you to test for and control the levels of the following [...] impurities in your product: [...] that are present during the manufacturing process and have the potential to contaminate your product. As part of your post marketing study, you will need to 1) develop a validated method for testing the finished product for the [...] impurities as stated above, and 2) analyze at least three batches of your product using the validated method."~"Fulfilled"~~3/31/2016 0:00:00~12/19/2018 0:00:00
384823~"CDER"~"NDA"~20123.00~"GE HEALTHCARE INC"~"Omniscan (Gadodiamide)"~1/8/1993 0:00:00~"Supplement"~46~"3627-4"~"PMR"~"505 (o)(3)"~3627.00~4~"PMR 3627-4: A prospective longitudinal cohort trial with one or more matched control group(s) to evaluate the effects of repetitive GBCA administration on a comprehensive battery of neurobehavioral testing over the course of at least five administrations. The trial should be sufficiently powered to exclude a pre-specified magnitude of decline. As a secondary objective, trial patients should also have the option of providing blood and urine samples at the time of reimaging, so that normative estimates of gadolinium concentration across an extended range of post-administration timepoints may be documented."~"Ongoing"~~4/30/2029 0:00:00~3/3/2026 0:00:00
384824~"CDER"~"NDA"~20131.00~"BRACCO DIAGNOSTICS INC"~"ProHance (Gadoteridol) and Prohance Multipack (Gadoteridol)"~11/16/1992 0:00:00~"Supplement"~28~"3626-4"~"PMR"~"505 (o)(3)"~3626.00~4~"PMR 3626-4: A prospective longitudinal cohort trial with one or more matched control group(s) to evaluate the effects of repetitive GBCA administration on a comprehensive battery of neurobehavioral testing over the course of at least five administrations. The trial should be sufficiently powered to exclude a pre-specified magnitude of decline. As a secondary objective, trial patients should also have the option of providing blood and urine samples at the time of reimaging, so that normative estimates of gadolinium concentration across an extended range of post-administration timepoints may be documented."~"Ongoing"~~4/30/2029 0:00:00~1/30/2026 0:00:00
384825~"CDER"~"NDA"~20234.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tegretol-XR (Carbamazepine)"~3/25/1996 0:00:00~"Original"~1~"4094-1"~"PMR"~"505 (o)(3)"~4094.00~1~"PMR 4094-1: Conduct in vitro patch clamp studies to assess Tegretol s and Tegretol XR s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Tegretol s and Tegretol XR s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~5/20/2026 0:00:00
384826~"CDER"~"NDA"~20235.00~"VIATRIS SPECIALTY LLC"~"Neurontin (Gabapentin) "~12/30/1993 0:00:00~"Original"~1~"3643-1"~"PMR"~"505 (o)(3)"~3643.00~1~"PMR 3643-1: In a healthy non-drug dependent population with drug abuse experience with sedative drugs, evaluate the abuse potential of gabapentin. This clinical trial will evaluate gabapentin in a cross-over design with comparison to placebo and a positive control (such as diazepam) regarding abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and drug pharmacokinetics."~"Delayed"~"The applicant requested revised milestones because of continued discussions with the Agency to provide necessary guidance for them to conduct a new study for this PMR. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 07/11/2025."~4/30/2021 0:00:00~4/30/2026 0:00:00
384827~"CDER"~"NDA"~20235.00~"VIATRIS SPECIALTY LLC"~"Neurontin (Gabapentin) "~12/30/1993 0:00:00~"Original"~1~"3643-2"~"PMR"~"505 (o)(3)"~3643.00~2~"PMR 3643-2: In a healthy non-drug dependent population with drug abuse experience with opioids, evaluate the abuse potential of gabapentin taken concomitantly with an opioid, such as oxycodone, investigating a range of doses of gabapentin combined with the opioid. This clinical trial will evaluate the gabapentin/opioid combinations in a cross-over design with comparison to placebo, gabapentin alone, and a moderate dose of the opioid taken alone as a positive control. The assessments will include abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and pharmacokinetics of both gabapentin and the opioid (oxycodone) with the goal of characterizing any additive or synergistic effects on the abuse potential and physiologic effects of co-administered gabapentin and opioid."~"Delayed"~"The applicant requested revised milestones because of continued discussions with the Agency to provide necessary guidance for the applicant to conduct a new study for this PMR. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 07/11/2025."~8/31/2021 0:00:00~4/30/2026 0:00:00
384828~"CDER"~"NDA"~20414.00~"UNITED STATES ARMY OFFICE SURGEON GENERAL"~"Pyridostigmine Bromide"~2/5/2003 0:00:00~"Original"~1~"1248-4"~"PMR"~"Animal Efficacy"~1248.00~4~"PMR 1248-4: Subpart I Confirmatory Studies: We note your commitment to provide, within 1 month of the date of this letter, detailed protocols to address the requirement to conduct postmarketing studies, such as field studies, to verify and describe pyridostigmine's clinical  benefit and to assess its safety when used as indicated when such studies are feasible and ethical. We would be happy to meet with you to discuss the design of these protocols prior to their submission."~"Pending"~~~3/5/2026 0:00:00
384829~"CDER"~"NDA"~20430.00~"ASPEN GLOBAL INC"~"Orgaran (Danaparoid)"~12/24/1996 0:00:00~"Supplement"~4~"3634-1"~"PMC"~"506B PMC"~3634.00~1~"PMC 3634-1: Perform a clinical study to assess the immunogenicity risk of danaparoid and its ability to form complexes. The study will provide the evidence as to whether the treatment is associated with:   antibodies binding to danaparoid alone   antibodies binding to PF4/danaparoid complexes   antibodies activating platelets. The above study is to be performed in conjunction with the alternate HIT indication assessment (under IND 138723); data on both HIT and non-HIT patients will be provided. The IND clinical study, coupled with in vitro study results that provide data comparing the formation of complexes with PF4, will serve to assess the immunogenicity risk of danaparoid. We refer you to the Guidance for Industry: ImmunogenicityRelated Considerations for Low Molecular Weight Heparin products for reference."~"Released"~~8/31/2020 0:00:00~2/10/2025 0:00:00
384830~"CDER"~"NDA"~22549.00~"NOVA PNEUMA INC"~"Adasuve (Loxapine)"~12/21/2012 0:00:00~"Original"~1~"1891-4"~"PMR"~"505 (o)(3)"~1891.00~4~"PMR 1891-4: Conduct a large, non-randomized, open-label, postmarketing observational study to assess the risks of bronchospasm and related respiratory adverse events and serious outcomes (e.g., hospitalization, intubation, mechanical ventilation, or rescue medication for the management of respiratory reactions) associated with Adasuve (loxapine) inhalation powder treatment. The study must have a large sample size (approximately 10,000 patients exposed to Adasuve (loxapine) inhalation powder), in order to adequately characterize the frequency, nature, and severity of the risk of bronchospasm. The study must assess the use of Adasuve (loxapine) inhalation powder as used in clinical practice under the requirements of  the REMS for Adasuve (loxapine) inhalation powder and per the product labeling."~"Delayed"~"On August 26, 2015, Teva requested a change in timetables for PMR 1891-4 study completion and final report submission due to the extremely slow patient enrollment into the requirement postmarketing observational study. On November 30, 2015, FDA issued acknowledge revised postmarketing requirement milestones letter with good cause for study completion delay to October 1, 2019 and final report submission to March 31, 2020. Per the 2022 annual status report, the applicant has been in discussions with FDA and intends to reengage with the division regarding an alternative design to PMR 1891-4."~12/1/2015 0:00:00~2/14/2024 0:00:00
384831~"CDER"~"NDA"~22561.00~"EMD SERONO INC"~"Mavenclad (cladribine)"~3/29/2019 0:00:00~"Original"~1~"3592-1"~"PMR"~"505 (o)(3)"~3592.00~1~"PMR 3592-1: Conduct an observational study to assess the long-term risk of malignancy for Mavenclad compared to other therapies used in the treatment of adults with relapsing forms of multiple sclerosis. Describe and justify the choice of appropriate comparator population(s) and estimated background rate(s) relative to cladribine-exposed patients; clearly define the primary comparator population. Design the study around a testable hypothesis to assess, with sufficient sample size and power, a clinically meaningful increase in malignancy risk above the comparator background rate(s), with a pre-specified statistical analysis method. Specify concise case definitions and validation algorithms. For the Mavencladexposed and comparator(s) cohorts, clearly define the study drug initiation period and any exclusion and inclusion criteria. Enroll patients over an initial 4-year period and follow for a minimum of 8 years from the time of enrollment."~"Delayed"~"The final protocol submission milestone was missed due to continued discussions between the applicant and the Agency to finalize the study design. The final protocol was acknowledged in a letter dated 4/15/2021."~2/28/2034 0:00:00~5/27/2026 0:00:00
384832~"CDER"~"NDA"~22561.00~"EMD SERONO INC"~"Mavenclad (cladribine)"~3/29/2019 0:00:00~"Original"~1~"3592-2"~"PMR"~"505 (o)(3)"~3592.00~2~"PMR 3592-2: Establish a worldwide Pregnancy Surveillance Program to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to Mavenclad during pregnancy. Provide a complete protocol which includes details regarding how you plan to encourage patients and providers to report pregnancy exposures (e.g., telephone contact number and/or website in prescribing information), measures to ensure complete data capture regarding pregnancy outcomes, and any adverse effects in offspring and plans for comprehensive data analysis and yearly reporting."~"Ongoing"~~2/28/2032 0:00:00~5/27/2026 0:00:00
384833~"CDER"~"NDA"~22562.00~"RECORDATI RARE DISEASES INC"~"Carbaglu (N-Carbamyl-L-Glutamic Acid)"~3/18/2010 0:00:00~"Original"~1~"4810-1"~"PMR"~"505 (o)(3)"~4810.00~1~"PMR 4810-1: Conduct in vitro studies to determine the required amount of water and mixing procedures to disperse Carbaglu (carglumic acid) tablets to inform safe administration of Carbaglu through enteral feeding tubes."~"Delayed"~"The study completion and final report submission milestones have been missed. The applicant requested revised milestones because Recordati had to source a new facility to conduct the required in vitro study. Revised milestones were acknowledged in a letter dated 11/19/2025."~5/31/2025 0:00:00~5/15/2026 0:00:00
384834~"CDER"~"NDA"~22562.00~"RECORDATI RARE DISEASES INC"~"Carbaglu (N-Carbamyl-L-Glutamic Acid)"~3/18/2010 0:00:00~"Original"~1~"4009-1"~"PMC"~"506B PMC"~4009.00~1~"PMC 4009-1: Collect data on biochemical effects, clinical outcomes, and serious safety events associated with the short-term and long-term use of Carbaglu in pediatric and adult patients with propionic acidemia (PA) or methylmalonic acidemia (MMA), including data on pregnancy and fetal outcomes. This data collection may be performed as part of an existing study or registry. Specifically, assess plasma ammonia and related metabolites, short-term and long-term clinical outcomes, serious adverse events, risks of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant (through the first year of life) with exposure to Carbaglu over short-term and long-term treatment. This collection of data should span at least 10 years."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 02/03/2022. The applicant reports that the study has been initiated and 14 patients have been enrolled."~9/30/2032 0:00:00~5/15/2026 0:00:00
384835~"CDER"~"NDA"~22562.00~"RECORDATI RARE DISEASES INC"~"Carbaglu (N-Carbamyl-L-Glutamic Acid)"~3/18/2010 0:00:00~"Original"~1~"1604-2"~"PMR"~"505 (o)(3)"~1604.00~2~"PMR 1604-2: A registry of patients, including infants, with NAGS deficiency being treated with carglumic acid to obtain long-term clinical safety information. Data to be collected will include patient demographics, details of treatment with carglumic acid, other therapies  for hyperammonemia, dietary protein management, clinical status, neurocognitive and  psychomotor status, growth and development status, and adverse events.  Information from this registry should be submitted annually (in annual reports) with a final report submission at 15 years post-pproval."~"Ongoing"~~1/31/2027 0:00:00~5/15/2026 0:00:00
384836~"CDER"~"NDA"~22562.00~"RECORDATI RARE DISEASES INC"~"Carbaglu (N-Carbamyl-L-Glutamic Acid)"~3/18/2010 0:00:00~"Original"~1~"1604-3"~"PMR"~"505 (o)(3)"~1604.00~3~"PMR 1604-3: A study of the effects of carglumic acid on pregnancy and fetal outcomes. This study can be performed as a sub-study within the registry for all patients with NAGS deficiency. Information on pregnancy and fetal outcomes should be submitted annually  (in annual reports) and included in the final report submission on the registry at 15 years post-approval."~"Ongoing"~~1/31/2027 0:00:00~5/15/2026 0:00:00
384837~"CDER"~"NDA"~22549.00~"NOVA PNEUMA INC"~"Adasuve (Loxapine)"~12/21/2012 0:00:00~"Original"~1~"1891-2"~"PMR"~"Pediatric Research Equity Act"~1891.00~2~"PMR 1891-2: A deferred pediatric study under PREA for the acute treatment of agitation associated with schizophrenia or bipolar I disorder in pediatric patients ages 10 to 17 years. A study of the efficacy and safety of Adasuve (loxapine) inhalation powder in the relevant pediatric population."~"Delayed"~"Deferral Extension Requested 04/01/2021. Denied per FDA letter dated 05/13/2021."~7/31/2021 0:00:00~2/14/2024 0:00:00
384838~"CDER"~"NDA"~200533.00~"COLLEGIUM PHARMACEUTICAL INC"~"Nucynta ER (Tapentadol)"~8/25/2011 0:00:00~"Original"~1~"3033-6"~"PMR"~"505 (o)(3)"~3033.00~6~"PMR 3033-6: An observational study to develop and validate an algorithm using coded medical terminologies and other electronic healthcare data to identify opioid-related overdose and death."~"Fulfilled"~~12/31/2016 0:00:00~10/23/2025 0:00:00
384839~"CDER"~"NDA"~200533.00~"COLLEGIUM PHARMACEUTICAL INC"~"Nucynta ER (Tapentadol)"~8/25/2011 0:00:00~"Original"~1~"3033-11"~"PMR"~"505 (o)(3)"~3033.00~11~"PMR 3033-11: Conduct a clinical trial to estimate the serious risk for the development of hyperalgesia following the long-term use of high-dose ER/LA opioid analgesics  for at least one year to treat chronic pain. Include an assessment of risk relative to  efficacy."~"Delayed"~"The Trial Completion and Final Report milestones were missed because of continued discussions needed to reach agreement with the Agency on the Final Protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 8/26/2025."~8/31/2019 0:00:00~10/23/2025 0:00:00
384840~"CDER"~"NDA"~200534.00~"GENUS LIFE SCIENCES INC"~"Oxycodone Hydrochloride "~10/20/2010 0:00:00~"Original"~1~"1698-1"~"PMR"~"Pediatric Research Equity Act"~1698.00~1~"PMR 1698-1: Pharmacokinetic, safety, and efficacy study in subjects from birth to 2 years of age."~"Delayed"~"Original Final Report Due Date: 11/30/2015; Deferral Extension
granted and revised milestones acknowledged per FDA letter dated
08/02/2024. The original Final Protocol Submission and Study Completion milestones were missed and revised due to ongoing
Agency discussions regarding the study design for analgesic studies in the pediatric population from birth to 2 years of age."~9/30/2027 0:00:00~12/19/2025 0:00:00
384841~"CDER"~"NDA"~200535.00~"GENUS LIFE SCIENCES INC"~"Oxycodone Hydrochloride "~10/20/2010 0:00:00~"Original"~1~"1695-1"~"PMR"~"Pediatric Research Equity Act"~1695.00~1~"PMR 1695-1: Pharmacokinetic, safety, and efficacy study in subjects from birth to 2 years of age."~"Delayed"~"Original Final Report Due Date: 11/30/2015; Deferral Extension granted and revised milestones acknowledged per FDA letter dated 08/02/2024. Final Protocol and Study Completion milestones were revised. This is due to ongoing Agency discussions regarding the study design for analgesic studies in the pediatric population from birth to 2 years of age."~9/30/2027 0:00:00~12/19/2025 0:00:00
384842~"CDER"~"NDA"~200796.00~"AZURITY PHARMACEUTICALS INC"~"Edarbi (Azilsartan Kamedoxomil)"~2/25/2011 0:00:00~"Original"~1~"1733-4"~"PMR"~"Pediatric Research Equity Act"~1733.00~4~"PMR 1733-4: Conduct a comparative single-dose pharmacokinetic and safety trial of azilsartan between infants, children, and adolescents with hypertension and healthy adults.  Using data from the PK and safety trial, conduct an efficacy and safety, dose-finding trial in children 6 years to less than 18 years with hypertension."~"Delayed"~"Enrollment has ended and all subjects have completed the
double-blind,withdrawal and open label phases of the study. Final report submission milestone has been missed."~12/31/2019 0:00:00~4/24/2026 0:00:00
384843~"CDER"~"NDA"~200796.00~"AZURITY PHARMACEUTICALS INC"~"Edarbi (Azilsartan Kamedoxomil)"~2/25/2011 0:00:00~"Original"~1~"1733-5"~"PMR"~"Pediatric Research Equity Act"~1733.00~5~"PMR 1733-5: A safety and pharmacokinetic study to identify a dosing regimen for azilsartan medoxomil in pediatric patients 2 to <6 years of age with hypertension and weighing <25 kg."~"Delayed"~"A final protocol has been agreed to. Azurity has begun start up activities and anticipates the study to be initiated during the next reporting period."~9/30/2028 0:00:00~4/24/2026 0:00:00
384844~"CDER"~"NDA"~201194.00~"VISTAPHARM LLC"~"Oxycodone Hydrochloride "~1/12/2012 0:00:00~"Original"~1~"1863-2"~"PMR"~"Pediatric Research Equity Act"~1863.00~2~"PMR 1863-2: Pharmacokinetic, safety, and efficacy study in subjects from birth to 2 years of age."~"Delayed"~"Original Final Report Due Date: 01/31/2015; Deferral Extension granted per FDA letter dated 07/27/2023. Revised milestones acknowledged per FDA letter dated  08/02/2024. This is due to ongoing internal Agency discussions of the most feasible study design for analgesic studies in the pediatric population from birth to 2 years of age."~9/30/2027 0:00:00~3/11/2025 0:00:00
384845~"CDER"~"NDA"~201277.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Gadavist (gadobutrol)"~3/14/2011 0:00:00~"Supplement"~13~"3624-4"~"PMR"~"505 (o)(3)"~3624.00~4~"PMR 3624-4: A prospective longitudinal cohort trial with one or more matched control group(s) to evaluate the effects of repetitive GBCA administration on a comprehensive battery of neurobehavioral testing over the course of at least five administrations. The trial should be sufficiently powered to exclude a pre-specified magnitude of decline. As a secondary objective, trial patients should also have the option of providing blood and urine samples at the time of reimaging, so that normative estimates of gadolinium concentration across an extended range of post-administration timepoints may be documented."~"Ongoing"~~4/30/2029 0:00:00~5/13/2026 0:00:00
384846~"CDER"~"NDA"~201367.00~"EISAI INC"~"Banzel (Rufinamide)"~3/3/2011 0:00:00~"Original"~1~"4076-1"~"PMR"~"505 (o)(3)"~4076.00~1~"PMR 4076-1: Conduct in vitro patch clamp studies to assess Banzel s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Banzel s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~1/13/2026 0:00:00
384847~"CDER"~"NDA"~201635.00~"SUPERNUS PHARMACEUTICALS INC"~"Trokendi XR (Topiramate)"~8/16/2013 0:00:00~"Original"~1~"4091-1"~"PMR"~"505 (o)(3)"~4091.00~1~"PMR 4091-1: Conduct in vitro patch clamp studies to assess Trokendi XR s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Trokendi XR s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~10/30/2025 0:00:00
384848~"CDER"~"NDA"~201803.00~"HALEON US HOLDINGS LLC"~"Advil (Ibuprofen Sodium)"~6/12/2012 0:00:00~"Original"~1~"1905-1"~"PMR"~"Pediatric Research Equity Act"~1905.00~1~"PMR 1905-1: The applicant currently proposes to develop a liquid pediatric formulation and then perform a pharmacokinetic (PK) study in adults comparing the pediatric formulation to an approved pediatric ibuprofen product. The proposed PK study is  a single-dose, randomized, open-label, two-way crossover PK study. Approximately 30 healthy male and female subjects, 18 to 45 years of age, will be enrolled. The treatment groups will be: * 10 ml of currently marketed Children's Advil Suspension (equivalent to ibuprofen 200 mg)  * 10 ml of sodium ibuprofen solution (equivalent to ibuprofen 200 mg) The statistical hypothesis to be tested is that sodium ibuprofen pediatric solution is bioequivalent to Children's Advil suspension in the fasted state."~"Delayed"~"Pfizer submitted the study report but did not submit the NDA to support the pediatric dosage form. It appears that the Applicant has no plans to market the pediatric product. 6/12/17 FDA issued a PREA noncompliance (NC) letter. 7/21/17 Pfizer acknowledged but did not contest the NC letter. 12/30/21 FDA issued a request to
the Applicant to submit the missing status information. 8/30/24 No new updates to report. 8/7/2025 no new updates reported."~6/30/2014 0:00:00~8/7/2025 0:00:00
384849~"CDER"~"NDA"~207648.00~"FRESENIUS KABI USA LLC"~"SMOFlipid (Fish Oil, Medium Chain Triglycerides, Olive Oil, and Soybean Oil)"~7/13/2016 0:00:00~"Supplement"~5~"4240-3"~"PMR"~"505 (o)(3)"~4240.00~3~"PMR 4240-3: Three-month toxicity study of [ ] (impurity) in rats to support a safety assessment."~"Delayed"~"The final report submission milestone was missed. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 9-3-2024. The draft protocol was received 8/28/2025."~8/31/2024 0:00:00~8/27/2025 0:00:00
384850~"CDER"~"NDA"~207648.00~"FRESENIUS KABI USA LLC"~"SMOFlipid (Fish Oil, Medium Chain Triglycerides, Olive Oil, and Soybean Oil)"~7/13/2016 0:00:00~"Supplement"~5~"4240-4"~"PMR"~"505 (o)(3)"~4240.00~4~"PMR 4240-4: Conduct a twenty-eight day dose-range finding toxicity study of 2- ethylfuran (impurity) in rats to provide a rationale for dose selection and route of administration for the three-month rat toxicity study (PMR 4240-3)."~"Fulfilled"~~4/30/2025 0:00:00~8/27/2025 0:00:00
384851~"CDER"~"NDA"~207924.00~"ELI LILLY AND CO"~"Olumiant (baricitinib)"~5/31/2018 0:00:00~"Original"~1~"3421-1"~"PMR"~"505 (o)(3)"~3421.00~1~"PMR 3421-1: Randomized controlled clinical trial to evaluate the long-term safety of baricitinib in patients with rheumatoid arthritis. The trial will include two doses of baricitinib and an active comparator. The trial should be of sufficient size and duration to evaluate safety events of interest, including arterial and venous thrombosis, cardiovascular adverse events, opportunistic infections, and malignancy."~"Submitted"~~10/31/2026 0:00:00~7/25/2025 0:00:00
384852~"CDER"~"NDA"~207924.00~"ELI LILLY AND CO"~"Olumiant (baricitinib)"~5/31/2018 0:00:00~"Supplement"~6~"4275-1"~"PMR"~"Pediatric Research Equity Act"~4275.00~1~"PMR 4275-1: Provide the final study report including PK and safety assessment of the ongoing pediatric trial in COVID-19. In addition, submit a PK and safety assessment from pediatric subjects treated with baricitinib for other indications from completed or ongoing trials."~"Ongoing"~"Original Final Report Due Date: 01/31/2024. Per FDA letter dated 12/08/2023, Final Report due date extended to 04/30/2025. Second Deferral Extension Granted per FDA letter dated 12/19/2024, Final Report due date extended to 04/30/2026. Third Deferral Extension Granted per FDA letter dated 03/12/2026, Final Report due date extended to 01/31/2027."~1/31/2027 0:00:00~7/25/2025 0:00:00
384853~"CDER"~"NDA"~207924.00~"ELI LILLY AND CO"~"Olumiant (baricitinib)"~5/31/2018 0:00:00~"Supplement"~7~"4289-1"~"PMR"~"Pediatric Research Equity Act"~4289.00~1~"PMR 4289-1: Conduct a randomized, controlled trial to evaluate the safety, efficacy, and pharmacokinetics of baricitinib in the adolescent population (12 years to less than 18 years) with severe alopecia areata. Evaluate at least 300 subjects exposed to baricitinib for a minimum of 52 weeks. 
"~"Ongoing"~"The trial is underway. First patient visit was 27 Feb 2023."~6/30/2027 0:00:00~7/25/2025 0:00:00
384854~"CDER"~"NDA"~207924.00~"ELI LILLY AND CO"~"Olumiant (baricitinib)"~5/31/2018 0:00:00~"Supplement"~7~"4289-2"~"PMR"~"Pediatric Research Equity Act"~4289.00~2~"PMR 4289-2: Conduct a randomized, controlled trial to evaluate the safety, efficacy, and pharmacokinetics of baricitinib in the pediatric population (6 years to less than 12 years) with severe alopecia areata. Evaluate at least 100 subjects exposed to baricitinib for a minimum of 52 weeks."~"Ongoing"~"The trial is underway. First patient screened on 14 May 2025."~11/30/2031 0:00:00~7/25/2025 0:00:00
384855~"CDER"~"NDA"~207924.00~"ELI LILLY AND CO"~"Olumiant (baricitinib)"~5/31/2018 0:00:00~"Supplement"~7~"4289-3"~"PMR"~"505 (o)(3)"~4289.00~3~"PMR 4289-3: Conduct a Pregnancy Exposure Registry, a prospective, registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to baricitinib during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The Final Protocol Submission due date was missed due to ongoing discussions with the Agency."~6/30/2034 0:00:00~7/25/2025 0:00:00
384856~"CDER"~"NDA"~207924.00~"ELI LILLY AND CO"~"Olumiant (baricitinib)"~5/31/2018 0:00:00~"Supplement"~7~"4289-4"~"PMR"~"505 (o)(3)"~4289.00~4~"PMR 4289-4: Conduct a pregnancy study that uses a different design from the Pregnancy Registry (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to baricitinib during pregnancy compared to an unexposed control population."~"Delayed"~"The Final Protocol submission milestone was missed. Per the applicant s annual status report, first annual monitoring data extraction commenced in April 2025. Between 01 June 2018 and 29 May 2024, there have been a total of 71 pregnancies in the database exposed to baricitinib, 7 of which have an alopecia areata diagnosis at baseline."~6/30/2030 0:00:00~7/25/2025 0:00:00
384857~"CDER"~"NDA"~207932.00~"BIODELIVERY SCIENCES INTERNATIONAL INC"~"Belbuca (Buprenorphine Hydrochloride) "~10/23/2015 0:00:00~"Original"~1~"3033-1"~"PMR"~"505 (o)(3)"~3033.00~1~"PMR 3033-1: A prospective, observational study designed to quantify the serious risks of misuse, abuse, and addiction associated with long-term use of opioid analgesics  for management of chronic pain among patients prescribed ER/LA opioid analgesics. This study must address at a minimum the following specific objectives: a. Estimate the incidence of misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of  psychiatric illness) on the risk of misuse, abuse, and addiction.  b. Evaluate and quantify other risk factors for misuse, abuse, and addiction associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors, psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships."~"Fulfilled"~~3/31/2020 0:00:00~2/20/2026 0:00:00
384858~"CDER"~"NDA"~20450.00~"PARKE DAVIS A SUB OF PFZIER INC"~"Cerebyx (Fosphenytoin Sodium)"~8/5/1996 0:00:00~"Original"~1~"4086-1"~"PMR"~"505 (o)(3)"~4086.00~1~"PMR 4086-1: Conduct in vitro patch clamp studies to assess Cerebyx s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Cerebyx s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~9/30/2025 0:00:00
384859~"CDER"~"NDA"~20505.00~"JANSSEN PHARMACEUTICALS INC"~"Topamax (Topiramate)"~12/24/1996 0:00:00~"Original"~1~"4092-1"~"PMR"~"505 (o)(3)"~4092.00~1~"PMR 4092-1: Conduct in vitro patch clamp studies to assess Topamax s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Topamax s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~11/24/2025 0:00:00
384860~"CDER"~"NDA"~20511.00~"ULURU INC"~"Aphthasol (Amlexanox)"~12/17/1996 0:00:00~"Original"~1~"1432-2"~"PMC"~"506B PMC"~1432.00~2~"PMC 1432-2: Commitment to conduct a study to investigate the use of this agent in the pediatric population."~"Delayed"~"PMC not fulfilled letter signed on 8/25/2010."~~8/10/2011 0:00:00
384861~"CDER"~"NDA"~20533.00~"FRESENIUS KABI AUSTRIA GMBH"~"Naropin (Ropivacaine Hydrochloride)"~9/24/1996 0:00:00~"Supplement"~34~"3331-1"~"PMR"~"505 (o)(3)"~3331.00~1~"PMR 3331-1: Conduct a new leachables study to adequately characterize the leachables profile associated with the newly proposed [...] Evaluate at least three batches of your to-be-marketed drug product for leachables and include assessments at multiple timepoints over the course of your stability studies in order to identify trends in leachable levels over time. The materials tested should include any secondary container closure systems, if present, and be subjected to the same  [...] methods, as appropriate. Establish an AET of [...] mcg/day. Based on these results, provide a revised toxicological risk assessment for any leachable above [...] mcg/day."~"Delayed"~"The Final Report and Study Completion milestones were missed."~8/31/2020 0:00:00~11/21/2025 0:00:00
384862~"CDER"~"NDA"~20533.00~"FRESENIUS KABI AUSTRIA GMBH"~"Naropin (Ropivacaine Hydrochloride)"~9/24/1996 0:00:00~"Supplement"~34~"3331-2"~"PMR"~"505 (o)(3)"~3331.00~2~"PMR 3331-2: Conduct a single-dose intrathecal toxicity study with acute and delayed sacrifices in a single species to qualify the local and systemic safety of [...] at the levels identified as leachables in the migration studies."~"Submitted"~~1/31/2019 0:00:00~11/21/2025 0:00:00
384863~"CDER"~"NDA"~20533.00~"FRESENIUS KABI AUSTRIA GMBH"~"Naropin (Ropivacaine Hydrochloride)"~9/24/1996 0:00:00~"Supplement"~36~"3636-1"~"PMR"~"505 (o)(3)"~3636.00~1~"PMR 3636-1: Based on the results of adequate extraction studies, conduct an adequate leachables study with the newly proposed phthalate-free Freeflex bag container closure system. Evaluate at least three batches of your to-bemarketed drug product for leachables and include assessments at multiple timepoints over the course of your stability studies (early, middle, late) in order to identify trends in leachable levels over time. The materials tested should include any secondary container closure systems, if present, and be subjected to the same sterilization methods, as appropriate. These data are essential to determine the appropriate shelf life of your product. For epidural drug products, establish an AET of 0.15 mcg/day, unless adequately justified based on technical feasibility. Submit a toxicological risk assessment to justify the systemic and local safety of any compound detected."~"Delayed"~"The Final Report milestone was missed. The applicant submitted a draft protocol. FDA provided feedback on 11/12/2025."~3/31/2022 0:00:00~11/21/2025 0:00:00
384864~"CDER"~"NDA"~20533.00~"FRESENIUS KABI AUSTRIA GMBH"~"Naropin (Ropivacaine Hydrochloride)"~9/24/1996 0:00:00~"Supplement"~36~"3636-2"~"PMR"~"505 (o)(3)"~3636.00~2~"PMR 3636-2: Conduct a drug product stability study over the course of the entire 24 months of the drug product shelf-life and submit the data to the NDA to confirm the proposed specifications. Via an interim report, provide batch release data and at least 3 months of accelerated and long-term stability data for the Naropin (ropivacaine HCl Injection, USP) drug product packaged in the phthalate catalyst-free polypropylene Freeflex bags.
"~"Submitted"~~3/31/2022 0:00:00~11/21/2025 0:00:00
384865~"CDER"~"NDA"~20551.00~"ABBVIE INC"~"Nimbex (Cisatracurium Besylate) "~12/15/1995 0:00:00~"Original"~1~"3847-1"~"PMR"~"505 (o)(3)"~3847.00~1~"PMR 3847-1: Conduct a leachables study to adequately characterize the leachables profile associated with the newly proposed container closure rubber stoppers. Evaluate at least three batches of your to-be-marketed drug product for leachables and include assessments at multiple timepoints over the course of your stability studies in order to identify trends in leachable levels over time. The materials tested should include any secondary container closure systems, if present, and be subjected to the same sterilization methods, as appropriate. Establish an AET of 5 mcg/day. Based on these results, provide a revised toxicological risk assessment for any leachable above 5 mcg/day."~"Released"~~12/31/2022 0:00:00~2/6/2025 0:00:00
384866~"CDER"~"NDA"~20587.00~"SCIARRA LABORATORIES INC"~"Sclerosol Intrapleural Aerosol (Talc)"~12/24/1997 0:00:00~"Supplement"~8~"3325-1"~"PMR"~"505 (o)(3)"~3325.00~1~"PMR 3325-1: Perform a risk assessment study on the lead content of sterile talc powder and indicate any necessary controls on elemental impurities as per ICH Q3D  Elemental Impurities: Guidance for Industry."~"Delayed"~"FDA determined that the data in the final report submitted by the applicant on 01/06/2021 did not fulfill the terms of the commitment.
"~5/31/2018 0:00:00~2/16/2024 0:00:00
384867~"CDER"~"NDA"~20712.00~"TAKEDA PHARMACEUTICALS USA INC"~"Carbatrol (Carbamazepine)"~9/30/1997 0:00:00~"Original"~1~"4093-1"~"PMR"~"505 (o)(3)"~4093.00~1~"PMR 4093-1: Conduct in vitro patch clamp studies to assess Carbatrol s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Carbatrol s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~11/24/2025 0:00:00
384868~"CDER"~"NDA"~20780.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Cipro (Ciprofloxacin Hydrochloride) "~9/26/1997 0:00:00~"Supplement"~41~"2843-1"~"PMR"~"Animal Efficacy"~2843.00~1~"PMR 2843-1: Conduct a field study to evaluate the efficacy and safety of ciprofloxacin in the event of an attack with the intentional release of Y. pestis in the United States."~"Pending"~~~11/25/2025 0:00:00
384869~"CDER"~"NDA"~20786.00~"CHATTEM INC DBA SANOFI CONSUMER HEALTHCARE"~"Allegra D Allergy and Congestion (Fexofenadine Hydrochloride and Pseudoephedrine Hydrochloride)"~12/24/1997 0:00:00~"Supplement"~33~"2916-2"~"PMR"~"Pediatric Research Equity Act"~2916.00~2~"PMR 2916-2: Conduct a study in children 6 to less than 12 years of age to evaluate the efficacy and safety of the pseudoephedrine component, in your combination product, for  temporary relief of nasal congestion due to the common cold."~"Submitted"~"According to the PMR, the final protocol, study completion, and final report submission are due by October 2021, January 2023, and June 2023 respectively. However, on October 29,2021, the applicant submitted a study report for a 2012 study, A Multicenter, Randomized, Placebo-Controlled Study of Pseudoephedrine for the Temporary Relief of Nasal Congestion in Children with the Common Cold (Study PRGO-PSE-09001), to fulfill PMR 2916-2. FDA's assessment is underway."~6/30/2023 0:00:00~2/19/2026 0:00:00
384870~"CDER"~"NDA"~22562.00~"RECORDATI RARE DISEASES INC"~"Carbaglu (N-Carbamyl-L-Glutamic Acid)"~3/18/2010 0:00:00~"Supplement"~8~"4009-1"~"PMC"~"506B PMC"~4009.00~1~"PMC 4009-1: Collect data on biochemical effects, clinical outcomes, and serious safety events associated with the short-term and long-term use of Carbaglu in pediatric and adult patients with propionic acidemia (PA) or methylmalonic acidemia (MMA), including data on pregnancy and fetal outcomes. This data collection may be performed as part of an existing study or registry. Specifically, assess plasma ammonia and related metabolites, short-term and long-term clinical outcomes, serious adverse events, risks of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant (through the first year of life) with exposure to Carbaglu over short-term and long-term treatment. This collection of data should span at least 10 years."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 02/03/2022. The applicant reports that the study has been initiated and 14 patients have been enrolled."~9/30/2032 0:00:00~5/15/2026 0:00:00
384871~"CDER"~"NDA"~22562.00~"RECORDATI RARE DISEASES INC"~"Carbaglu (N-Carbamyl-L-Glutamic Acid)"~3/18/2010 0:00:00~"Supplement"~9~"4009-1"~"PMC"~"506B PMC"~4009.00~1~"PMC 4009-1: Collect data on biochemical effects, clinical outcomes, and serious safety events associated with the short-term and long-term use of Carbaglu in pediatric and adult patients with propionic acidemia (PA) or methylmalonic acidemia (MMA), including data on pregnancy and fetal outcomes. This data collection may be performed as part of an existing study or registry. Specifically, assess plasma ammonia and related metabolites, short-term and long-term clinical outcomes, serious adverse events, risks of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant (through the first year of life) with exposure to Carbaglu over short-term and long-term treatment. This collection of data should span at least 10 years."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 02/03/2022. The applicant reports that the study has been initiated and 14 patients have been enrolled."~9/30/2032 0:00:00~5/15/2026 0:00:00
384872~"CDER"~"NDA"~22577.00~"GILEAD SCIENCES INC"~"Viread (Tenofovir Disoproxil Fumarate) "~1/18/2012 0:00:00~"Original"~1~"212-2"~"PMR"~"Pediatric Research Equity Act"~212.00~2~"PMR 212-2: Deferred pediatric study under PREA for the use of VIREAD treatment, in combination with other antiretroviral agents, of HIV-1 in pediatric patients ages birth to 2 years of age.  Due to safety concerns for this age group, we are waiting for completion and review of studies in the 2 to 18 years age group before determining whether it is appropriate to study tenofovir in the birth to 2 years age group."~"Delayed"~"DE Granted letter issued on 10/27/2022."~1/31/2027 0:00:00~12/23/2025 0:00:00
384873~"CDER"~"NDA"~22580.00~"VIVUS LLC"~"Qsymia (Phentermine and Topiramate)"~7/17/2012 0:00:00~"Original"~1~"4098-1"~"PMR"~"505 (o)(3)"~4098.00~1~"PMR 4098-1: Conduct in vitro patch clamp studies to assess the topiramate component of Qsymia s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing the topiramate component of Qsymia s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~9/16/2025 0:00:00
384874~"CDER"~"NDA"~22580.00~"VIVUS LLC"~"Qsymia (Phentermine and Topiramate)"~7/17/2012 0:00:00~"Original"~1~"1901-11"~"PMR"~"Pediatric Research Equity Act"~1901.00~11~"PMR 1901-11: A 52-week randomized, double-blind, placebo-controlled pediatric trial to evaluate the safety and efficacy of Qsymia for the treatment of obesity in pediatric patients ages 7 to 11 years (inclusive)."~"Released"~"Per FDA letter dated 05/13/2026, this PMR/PMC has been released. 
"~2/28/2027 0:00:00~9/16/2025 0:00:00
384875~"CDER"~"NDA"~22581.00~"FRESENIUS MEDICAL CARE RENAL THERAPIES GROUP LLC"~"Phoslyra (Calcium Acetate)"~4/18/2011 0:00:00~"Original"~1~"1755-2"~"PMC"~"506B PMC"~1755.00~2~"PMC 1755-2: A multi-phase clinical trial in a hyperphosphatemic pediatric dialysis population, with a placebo-controlled dose-response phase, followed by an open-label titration and maintenance phase, followed by a placebo-controlled randomized withdrawal phase."~"Delayed"~"Study status: FMCNA requested that the Agency release the study commitment on February 22, 2013.  The study is terminated."~3/31/2014 0:00:00~6/18/2012 0:00:00
384876~"CDER"~"NDA"~50777.00~"LEO PHARMA AS"~"Protopic (Tacrolimus)"~12/8/2000 0:00:00~"Original"~1~"869-2"~"PMC"~"506B PMC"~869.00~2~"PMC 869-2: A commitment to conduct a registry study of pediatric patients with atopic dermatitis to address the risk of developing cutaneous or systemic malignancies in patients who have long term intermittent treatment with Protopic Ointment 0.03% or 0.01%. The proposal for this study will be provided to the Division for review by June 30, 2001."~"Submitted"~~12/31/2023 0:00:00~2/4/2020 0:00:00
384877~"CDER"~"BLA"~103353.00~"Amgen Inc."~"Neupogen (Filgrastim)"~2/20/1991 0:00:00~"Supplement"~5183~"2893-1"~"PMR"~"Animal Efficacy"~2893.00~1~"PMR 2893-1: Conduct a phase 4 observational study to evaluate the efficacy and safety of  Neupogen (filgrastim) in the setting of Hematopoietic syndrome (HS) following  acute radiation exposure."~"Pending"~~~3/28/2025 0:00:00
384878~"CDER"~"BLA"~103362.00~"Partner Therapeutics Inc."~"Leukine (Sargramostim)"~3/5/1991 0:00:00~"Supplement"~5240~"3363-1"~"PMR"~"Animal Efficacy"~3363.00~1~"PMR 3363-1: Conduct a Phase 4 observational study to evaluate the efficacy and safety of Leukine (sargramostim) in the setting of Hematopoietic Syndrome (HS) following acute radiation exposure."~"Pending"~~~4/29/2026 0:00:00
384879~"CDER"~"BLA"~103411.00~"Servier Pharmaceuticals  LLC"~"Oncaspar (pegaspargase)"~2/1/1994 0:00:00~"Supplement"~5052~"2656-1"~"PMC"~"506B PMC"~2656.00~1~"PMC 2656-1: To commit to providing complete validation data for the anti-Oncaspar ELISA assay. The validation studies will provide an assessment of the sensitivity (in mass units of antibodies), specificity, and reproducibility of the assay. The cutpoint for the assay (the value that discriminates positive samples from negative samples) will be determined by using samples from unexposed patients and validated positive controls. This cut point value will be used to determine the number and percent of patients who develop antibodies to Oncaspar during clinical trials. The assay validation will be performed with insight obtained from Mire-Sluis et al. J. of Immunol. Methods, 2004, 289: 1-16."~"Submitted"~~1/31/2007 0:00:00~9/12/2025 0:00:00
384880~"CDER"~"BLA"~103411.00~"Servier Pharmaceuticals  LLC"~"Oncaspar (pegaspargase)"~2/1/1994 0:00:00~"Supplement"~5052~"2656-2"~"PMC"~"506B PMC"~2656.00~2~"PMC 2656-2: To commit to development and validation of an assay to detect the presence of neutralizing antibodies to Oncaspar. Validation studies will provide an assessment of the sensitivity (in mass units of antibodies), specificity, and reproducibility of the assay. The cutpoint for the assay (the value that discriminates positive samples from negative samples) will be determined by using samples from unexposed patients and validated positive controls. This value will be used to determine the number and percent of patients who develop neutralizing antibodies to the Oncaspar during clinical trials."~"Submitted"~~1/31/2007 0:00:00~9/12/2025 0:00:00
384881~"CDER"~"NDA"~202022.00~"JANSSEN PRODUCTS LP"~"Edurant (Rilpivirinee)"~5/20/2011 0:00:00~"Supplement"~14~"4004-1"~"PMR"~"Pediatric Research Equity Act"~4004.00~1~"PMR 4004-1: Conduct a study in subjects weighing 10 kg to less than 25 kg (approximately 2 to less than 6 years of age) to assess the pharmacokinetics, tolerability, and short-term safety of rilpivirine after 4-week administration in combination with other antiretroviral drug(s). Data from any ongoing or completed clinical trial(s) in children evaluating the safety, PK and antiviral activity of rilpivirine in this age or weight group could be provided to support this study requirement."~"Delayed"~"Original Final Report Due Date: 03/31/2023. Per FDA letter dated 04/25/2023, final report due date extended to 06/30/2026. Second Deferral Extension Granted Per FDA letter dated 05/07/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~7/13/2026 0:00:00
384882~"CDER"~"NDA"~202022.00~"JANSSEN PRODUCTS LP"~"Edurant (Rilpivirinee)"~5/20/2011 0:00:00~"Supplement"~14~"4004-2"~"PMR"~"Pediatric Research Equity Act"~4004.00~2~"PMR 4004-2: Conduct a study in subjects weighing 25 kg to less than 35 kg (approximately 6 to less than 12 years of age) to assess the pharmacokinetics, tolerability, and short-term safety of rilpivirine after 4-week administration in combination with other antiretroviral drug(s). Data from any ongoing or completed clinical trial(s) in children evaluating the safety, PK and antiviral activity of rilpivirine in this age or weight group could be provided to support this study requirement."~"Delayed"~"Original Final Report Due Date: 03/31/2023. Per FDA letter dated 04/25/2023, final report due date extended to 06/30/2026. Second Deferral Extension Granted Per FDA letter dated 05/07/2026. The study completion milestone was also revised."~8/31/2027 0:00:00~7/13/2026 0:00:00
384883~"CDER"~"NDA"~202080.00~"ZYLA LIFE SCIENCES US INC"~"Oxaydo (Oxycodone Hydrochloride)"~6/17/2011 0:00:00~"Original"~1~"1793-1"~"PMR"~"505 (o)(3)"~1793.00~1~"PMR 1793-1: An epidemiological study to address whether this formulation of Oxycodone HCl results in a decrease in misuse and abuse, and their consequences: overdose, death and addiction."~"Delayed"~"The final protocol submission, study completion, and final report milestones were missed, because the applicant is working with a consulting firm to address the items noted in the Agency s June 6, 2018, letter. FDA issued a failure to respond letter on 12/23/2022."~6/30/2016 0:00:00~8/12/2025 0:00:00
384884~"CDER"~"NDA"~202535.00~"FERRING PHARMACEUTICALS INC"~"Prepopik (Citric Acid, Magnesium Oxide, and Sodium Picosulfate)"~7/16/2012 0:00:00~"Original"~1~"1902-5"~"PMR"~"Pediatric Research Equity Act"~1902.00~5~"PMR 1902-5: A Randomized, Assessor-Blind, Multicenter, Dose-Ranging Study Comparing the Safety and Efficacy of Sodium Picosulfate, Magnesium Oxide, and Citric Acid Versus Active Comparator in Children Aged 2 Years to Less Than 9 Years."~"Delayed"~"Original Final Report Due Date: 06/30/2023;Deferral Extension granted per FDA letter dated 05/08/2023. The study completion milestone was also revised. A total of 6 patients have been randomized."~9/30/2026 0:00:00~9/11/2025 0:00:00
384885~"CDER"~"NDA"~202535.00~"FERRING PHARMACEUTICALS INC"~"Prepopik (Citric Acid, Magnesium Oxide, and Sodium Picosulfate)"~7/16/2012 0:00:00~"Original"~1~"1902-6"~"PMR"~"Pediatric Research Equity Act"~1902.00~6~"PMR 1902-6: A Randomized, Assessor-Blind, Multicenter, Dose-Ranging Study Comparing the Safety and Efficacy of Sodium Picosulfate, Magnesium Oxide, and Citric Acid Versus Active Comparator in Children Aged 12 Months to Less Than 2 Years"~"Delayed"~"Original Final Report Due Date: 06/30/2024; Deferral Extension granted per FDA letter dated 05/10/2024. The study completion milestone was also revised."~6/30/2029 0:00:00~9/11/2025 0:00:00
384886~"CDER"~"NDA"~202570.00~"PF PRISM CV"~"Xalkori (Crizotinib)"~8/26/2011 0:00:00~"Supplement"~30~"4003-1"~"PMR"~"505 (o)(3)"~4003.00~1~"PMR 4003-1: Conduct a prospective study to evaluate the risk factors, manifestations, and outcomes of ocular toxicity associated with crizotinib in pediatric and young adult patients with anaplastic large cell lymphoma (ALCL). The study will include a mechanism to collect, classify, and analyze data on ocular toxicity in patients exposed to crizotinib. Evaluate a minimum of 30 total patients (6 patients at 165 mg/m2 twice daily and 24 patients at 280 mg/m2 twice daily) who receive crizotinib monotherapy for ALCL, inflammatory myofibroblastic tumor (IMT), or other tumor types that are ALK-positive, ROS1-positive, or MET-positive. At least 50% of patients evaluated should be pediatric patients less than 17 years old. Include baseline, scheduled follow-up, and symptom-driven ocular assessments to include visual acuity assessments, ophthalmologic evaluations including slit lamp examination, and elicitation for visual symptoms."~"Ongoing"~~2/28/2027 0:00:00~10/23/2025 0:00:00
384887~"CDER"~"NDA"~202570.00~"PF PRISM CV"~"Xalkori (Crizotinib)"~8/26/2011 0:00:00~"Supplement"~30~"4003-2"~"PMC"~"506B PMC"~4003.00~2~"PMC 4003-2: Submit the final results from a clinical trial(s) of crizotinib monotherapy conducted in pediatric and young adult patients with relapsed or refractory, ALK-positive systemic anaplastic large cell lymphoma (ALCL), with a starting dosage of crizotinib that is lower than 280 mg/m2 twice daily (165 mg/m2 twice daily). At least 50% of the patients should be less than 17 years old. The primary efficacy endpoint should be overall response rate (ORR) using uniform response criteria. Other endpoints should include duration of response, pharmacokinetics from approximately 50% of patients, and safety. Data from more than one prospective clinical trial of crizotinib in the intended population may be used to support the analyses. Evaluate a minimum of 25 patients with ALCL at the 165 mg/m2 twice daily dosage of crizotinib to estimate the ORR. The results of this trial may inform product labeling."~"Ongoing"~~8/31/2027 0:00:00~10/23/2025 0:00:00
384888~"CDER"~"NDA"~202714.00~"ONYX PHARMACEUTICALS INC A WHOLLY OWNED SUB OF AMGEN INC"~"Kyprolis (Carfilzomib)"~7/20/2012 0:00:00~"Supplement"~33~"4183-1"~"PMC"~"506B PMC"~4183.00~1~"PMC 4183-1: Conduct an integrated study analysis containing data from clinical trials, post-marketing reports, compassionate use/expanded access programs, real-world evidence, and other sources to further characterize the safety and efficacy of daratumumab (SC) in combination with carfilzomib and dexamethasone among U.S. racial and ethnic minority patients with multiple myeloma. 
"~"Ongoing"~~8/31/2026 0:00:00~9/5/2025 0:00:00
384889~"CDER"~"NDA"~202714.00~"ONYX PHARMACEUTICALS INC A WHOLLY OWNED SUB OF AMGEN INC"~"Kyprolis (Carfilzomib)"~7/20/2012 0:00:00~"Supplement"~34~"4279-2"~"PMC"~"506B PMC"~4279.00~2~"PMC 4279-2: Conduct an integrated analysis that contains data from clinical trials, postmarketing reports, compassionate use/expanded access programs, realworld evidence, and other sources to further characterize the safety and efficacy of carfilzomib in combination with isatuximab and dexamethasone (Isa-Kd) among U.S. racial and ethnic minority patients with multiple myeloma. 
"~"Delayed"~"The final protocol submission milestone was missed because of continued discussions with FDA."~12/31/2026 0:00:00~9/5/2025 0:00:00
384890~"CDER"~"NDA"~207932.00~"BIODELIVERY SCIENCES INTERNATIONAL INC"~"Belbuca (Buprenorphine Hydrochloride) "~10/23/2015 0:00:00~"Original"~1~"3033-2"~"PMR"~"505 (o)(3)"~3033.00~2~"PMR 3033-2: An observational study designed to measure the incidence and predictors of opioid overdose and death (OOD), as well as opioid abuse/addiction, using patient  health records, insurance claims, and death records. This study must address at a minimum the following specific objectives: a. Estimate the incidence of abuse/addiction, overdose, and death associated with long-term use of opioid analgesics for chronic pain. Stratify overdose by  intentionality wherever possible. Examine the effect of product/formulation, dose and duration of opioid use, prescriber specialty, indication, and other  clinical factors (e.g., concomitant psychotropic medications, personal or family history of substance abuse, history of psychiatric illness) on the risk of  abuse/addiction, overdose, and death. b. Evaluate and quantify other risk factors for abuse/addiction, overdose, and  death associated with long-term use of opioid analgesics for chronic pain, including but not limited to the following: demographic factors,  psychosocial/behavioral factors, medical factors, and genetic factors. Identify confounders and effect modifiers of individual risk factor/outcome relationships. Stratify overdose by intentionality wherever possible."~"Fulfilled"~~9/30/2019 0:00:00~2/20/2026 0:00:00
384891~"CDER"~"NDA"~207932.00~"BIODELIVERY SCIENCES INTERNATIONAL INC"~"Belbuca (Buprenorphine Hydrochloride) "~10/23/2015 0:00:00~"Original"~1~"3033-6"~"PMR"~"505 (o)(3)"~3033.00~6~"PMR 3033-6: An observational study to develop and validate an algorithm using coded medical terminologies and other electronic healthcare data to identify opioid-related overdose and death."~"Fulfilled"~~12/31/2016 0:00:00~2/20/2026 0:00:00
384892~"CDER"~"NDA"~207932.00~"BIODELIVERY SCIENCES INTERNATIONAL INC"~"Belbuca (Buprenorphine Hydrochloride) "~10/23/2015 0:00:00~"Original"~1~"3033-11"~"PMR"~"505 (o)(3)"~3033.00~11~"PMR 3033-11: Conduct a clinical trial to estimate the serious risk for the development of hyperalgesia following the long-term use of high-dose ER/LA opioid analgesics  for at least one year to treat chronic pain. Include an assessment of risk relative to  efficacy."~"Delayed"~"The Trial Completion and Final Report milestones were missed because of continued discussions needed to reach agreement with the Agency on the Final Protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 8/26/2025."~8/31/2019 0:00:00~2/20/2026 0:00:00
384893~"CDER"~"NDA"~207960.00~"NEXTWAVE PHARMACEUTICALS INC"~"QuilliChew ER (Methylphenidate Hydrochloride)"~12/4/2015 0:00:00~"Original"~1~"3009-2"~"PMR"~"Pediatric Research Equity Act"~3009.00~2~"PMR 3009-2: Conduct a 6-week, double-blind, placebo-controlled, randomized, parallel-group safety and efficacy study in children with Attention Deficit Hyperactivity Disorder (ADHD) 4 to 5 years of age using methylphenidate ER formulation  (QuilliChew ER)"~"Released"~"Per FDA letter dated 03/18/2026, this PMR/PMC has been released."~6/30/2028 0:00:00~2/2/2026 0:00:00
384894~"CDER"~"NDA"~207960.00~"NEXTWAVE PHARMACEUTICALS INC"~"QuilliChew ER (Methylphenidate Hydrochloride)"~12/4/2015 0:00:00~"Original"~1~"3009-3"~"PMR"~"Pediatric Research Equity Act"~3009.00~3~"PMR 3009-3: Conduct a 6-month, open-label extension study to obtain additional information on safety and tolerability of methylphenidate ER formulation (QuilliChew ER) in  children 4 to 5 years of age with Attention Deficit Hyperactivity Disorder (ADHD)"~"Released"~"Per FDA letter dated 03/18/2026, this PMR/PMC has been released."~12/31/2028 0:00:00~2/2/2026 0:00:00
384895~"CDER"~"NDA"~207962.00~"SCILEX PHARMACEUTICALS INC"~"ZTLido (lidocaine HCL topical system)"~2/28/2018 0:00:00~"Original"~1~"3348-2"~"PMR"~"505 (o)(3)"~3348.00~2~"PMR 3348-2: Conduct a 2-year dermal carcinogenicity study to evaluate the carcinogenic potential of terpene resin, dipropylene glycol, SIS block copolymer, isostearic  acid, and polyisobutylene."~"Delayed"~"Final report submission milestone has been missed.  Applicant is awaiting FDA feedback on protocol submitted to IND."~10/31/2023 0:00:00~4/27/2026 0:00:00
384896~"CDER"~"NDA"~207962.00~"SCILEX PHARMACEUTICALS INC"~"ZTLido (lidocaine HCL topical system)"~2/28/2018 0:00:00~"Original"~1~"3348-3"~"PMR"~"505 (o)(3)"~3348.00~3~"PMR 3348-3: Conduct a leachables study employing validated analytical methods capable of detecting at least a [..] mcg/day safety concern threshold that demonstrates what  level [...] leaches from the patches at the maximum daily dose (3 patches per day)."~"Submitted"~~9/30/2018 0:00:00~4/27/2026 0:00:00
384897~"CDER"~"NDA"~207997.00~"NOVARTIS PHARMACEUTICALS CORP"~"Rydapt (Midostaurin, PKC412)"~4/28/2017 0:00:00~"Original"~1~"3210-1"~"PMR"~"505 (o)(3)"~3210.00~1~"PMR 3210-1: Establish a worldwide Pregnancy Surveillance Program (enhanced pharmacovigilance) to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to midostaurin during pregnancy. Add notice of the Pregnancy Surveillance Program and telephone contact number (and/or website) to the prescribing information. Provide a complete protocol which includes details regarding how you plan to  encourage patients and providers to report pregnancy exposures (e.g., telephone contact number and/or website in prescribing information), measures to ensure complete data capture regarding pregnancy outcomes and any adverse effects in offspring, and plans for comprehensive data analysis and yearly reporting. Submit yearly reports on the cumulative findings and analyses from the Pregnancy Surveillance Program."~"Ongoing"~~6/30/2027 0:00:00~6/23/2026 0:00:00
384898~"CDER"~"NDA"~207997.00~"NOVARTIS PHARMACEUTICALS CORP"~"Rydapt (Midostaurin, PKC412)"~4/28/2017 0:00:00~"Original"~2~"3210-1"~"PMR"~"505 (o)(3)"~3210.00~1~"PMR 3210-1: Establish a worldwide Pregnancy Surveillance Program (enhanced pharmacovigilance) to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to midostaurin during pregnancy. Add notice of the Pregnancy Surveillance Program and telephone contact number (and/or website) to the prescribing information. Provide a complete protocol which includes details regarding how you plan to  encourage patients and providers to report pregnancy exposures (e.g., telephone contact number and/or website in prescribing information), measures to ensure complete data capture regarding pregnancy outcomes and any adverse effects in offspring, and plans for comprehensive data analysis and yearly reporting. Submit yearly reports on the cumulative findings and analyses from the Pregnancy Surveillance Program."~"Ongoing"~~6/30/2027 0:00:00~6/23/2026 0:00:00
384899~"CDER"~"NDA"~207999.00~"INTERCEPT PHARMACEUTICALS INC"~"Ocaliva (Obeticholic Acid)"~5/27/2016 0:00:00~"Original"~1~"3057-1"~"PMR"~"Accelerated Approval"~3057.00~1~"PMR 3057-1: A randomized, placebo-controlled clinical trial to evaluate the safety, efficacy and steady-state pharmacokinetics of OCALIVA (obeticholic acid) in patients  with primary biliary cholangitis (PBC) with Child-Pugh Classes B and C hepatic impairment, including Child-Pugh Class C patients with varying levels of Model  for End-Stage Liver Disease (MELD) scores. You may conduct this as a standalone trial or in a subset of patients in your confirmatory trial (PMR# 3057-3)."~"Released"~"Per FDA letter dated 11/26/2025, this PMR/PMC has been released."~4/30/2023 0:00:00~7/25/2025 0:00:00
384900~"CDER"~"NDA"~20786.00~"CHATTEM INC DBA SANOFI CONSUMER HEALTHCARE"~"Allegra D Allergy and Congestion (Fexofenadine Hydrochloride and Pseudoephedrine Hydrochloride)"~12/24/1997 0:00:00~"Supplement"~33~"2916-3"~"PMR"~"Pediatric Research Equity Act"~2916.00~3~"PMR 2916-3: If the studies conducted in children 6 to less than 12 years of age demonstrate safety and efficacy for the intended claim, conduct a study to determine an appropriate dose and dosing interval for use in children, 2 to less than 6 years of  age, who may benefit from the pseudoephedrine component of the drug product (i.e., not in otherwise healthy pediatric volunteers) to relieve nasal congestion due to the common cold."~"Submitted"~"The final report was submitted to FDA on 7/28/2023."~6/30/2024 0:00:00~2/19/2026 0:00:00
384901~"CDER"~"NDA"~20786.00~"CHATTEM INC DBA SANOFI CONSUMER HEALTHCARE"~"Allegra D Allergy and Congestion (Fexofenadine Hydrochloride and Pseudoephedrine Hydrochloride)"~12/24/1997 0:00:00~"Supplement"~33~"2916-4"~"PMR"~"Pediatric Research Equity Act"~2916.00~4~"PMR 2916-4: If the studies conducted in children 6 to less than 12 years of age demonstrate safety and efficacy for the intended claim, conduct a study to evaluate the safety of the pseudoephedrine component, in your combination product, for temporary relief of nasal congestion due to the common cold in children 2 to less than 6  years of age. Efficacy of fexofenadine and pseudoephedrine for the age group 2 to less than 6 years may be extrapolated from data in the older age group."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed. Will begin in the future."~6/30/2026 0:00:00~2/19/2026 0:00:00
384902~"CDER"~"NDA"~20789.00~"ADVANZ PHARMA (US) CORP"~"Zonegran (Zonisamide)"~3/27/2000 0:00:00~"Original"~1~"4097-1"~"PMR"~"505 (o)(3)"~4097.00~1~"PMR 4097-1: Conduct in vitro patch clamp studies to assess Zonegran s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Zonegran s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~5/20/2025 0:00:00
384903~"CDER"~"NDA"~20800.00~"IMPAX LABORATORIES LLC"~"Twinject/Adrenaclick (Epinephrine)"~5/30/2003 0:00:00~"Original"~1~"3479-1"~"PMR"~"505 (o)(3)"~3479.00~1~"PMR 3479-1: Develop reliability requirement(s) and specification(s) to mitigate the failure to fire and bent needle risks, provide evidence to verify and validate the reliability specification(s), and control the reliability of Twinject and Adrenaclick (epinephrine injection, USP) Auto-Injectors and the epinephrine authorized generic through design and manufacturing."~"Submitted"~~1/31/2020 0:00:00~7/24/2025 0:00:00
384904~"CDER"~"NDA"~20800.00~"IMPAX LABORATORIES LLC"~"Twinject/Adrenaclick (Epinephrine)"~5/30/2003 0:00:00~"Original"~1~"3479-2"~"PMR"~"505 (o)(3)"~3479.00~2~"PMR 3479-2: Conduct a case study analysis of all reports of failure of Twinject and Adrenaclick (epinephrine injection, USP) Auto-Injectors and the epinephrine authorized generic to activate, or failure of Twinject and Adrenaclick (epinephrine injection, USP) Auto-Injectors and the epinephrine authorized generic to deliver the fulllabeled dose, or other device malfunctions. Perform detailed analyses of reported device failures (including reported malfunctions that did, as well as did not result in patient harm), full event narratives of the failure and any subsequent adverse events, and the results of root cause analysis performed for the reported failure. Interim analysis reports should also include a description of your procedures for monitoring and analyzing the adverse event reports. Additionally, include in your interim reports an analysis of the reporting period dates as well as overall analysis of the cumulative data."~"Submitted"~~11/30/2021 0:00:00~7/24/2025 0:00:00
384905~"CDER"~"NDA"~20829.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~2/20/1998 0:00:00~"Original"~1~"4875-1"~"PMR"~"505 (o)(3)"~4875.00~1~"PMR 4875-1: Conduct a study to investigate the temporal and spatial distribution of montelukast into the brain following repeated oral dosing in adult and juvenile rodents."~"Pending"~~9/30/2027 0:00:00~4/16/2026 0:00:00
384906~"CDER"~"NDA"~20829.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~2/20/1998 0:00:00~"Original"~1~"4875-2"~"PMR"~"505 (o)(3)"~4875.00~2~"PMR 4875-2: Conduct a study to investigate off-target binding and functional activity of montelukast, with particular focus on potential central nervous system (CNS) effects."~"Pending"~~9/30/2027 0:00:00~4/16/2026 0:00:00
384907~"CDER"~"NDA"~20829.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~2/20/1998 0:00:00~"Original"~1~"4875-3"~"PMR"~"505 (o)(3)"~4875.00~3~"PMR 4875-3: Conduct a study to investigate potential CNS effects of montelukast after repeated oral dosing in a juvenile rodent study with complex neurobehavioral endpoints."~"Released"~~11/30/2027 0:00:00~4/16/2026 0:00:00
384908~"CDER"~"NDA"~20829.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~2/20/1998 0:00:00~"Original"~1~"4875-4"~"PMR"~"505 (o)(3)"~4875.00~4~"PMR 4875-4: Conduct a study to further characterize human-relevant metabolites of montelukast, including identification, primary and secondary pharmacology, kinetics, and tissue distribution."~"Pending"~~2/28/2027 0:00:00~4/16/2026 0:00:00
384909~"CDER"~"NDA"~20830.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~2/20/1998 0:00:00~"Original"~1~"4875-1"~"PMR"~"505 (o)(3)"~4875.00~1~"PMR 4875-1: Conduct a study to investigate the temporal and spatial distribution of montelukast into the brain following repeated oral dosing in adult and juvenile rodents."~"Pending"~~9/30/2027 0:00:00~4/16/2026 0:00:00
384910~"CDER"~"NDA"~20830.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~2/20/1998 0:00:00~"Original"~1~"4875-2"~"PMR"~"505 (o)(3)"~4875.00~2~"PMR 4875-2: Conduct a study to investigate off-target binding and functional activity of montelukast, with particular focus on potential central nervous system (CNS) effects."~"Pending"~~9/30/2027 0:00:00~4/16/2026 0:00:00
384911~"CDER"~"NDA"~20830.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~2/20/1998 0:00:00~"Original"~1~"4875-3"~"PMR"~"505 (o)(3)"~4875.00~3~"PMR 4875-3: Conduct a study to investigate potential CNS effects of montelukast after repeated oral dosing in a juvenile rodent study with complex neurobehavioral endpoints."~"Released"~~11/30/2027 0:00:00~4/16/2026 0:00:00
384912~"CDER"~"NDA"~20830.00~"ORGANON LLC A SUB OF ORGANON AND CO"~"Singulair (Montelukast Sodium) "~2/20/1998 0:00:00~"Original"~1~"4875-4"~"PMR"~"505 (o)(3)"~4875.00~4~"PMR 4875-4: Conduct a study to further characterize human-relevant metabolites of montelukast, including identification, primary and secondary pharmacology, kinetics, and tissue distribution."~"Pending"~~2/28/2027 0:00:00~4/16/2026 0:00:00
384913~"CDER"~"NDA"~20844.00~"JANSSEN PHARMACEUTICALS INC"~"Topamax (Topiramate)"~10/26/1998 0:00:00~"Original"~1~"4092-1"~"PMR"~"505 (o)(3)"~4092.00~1~"PMR 4092-1: Conduct in vitro patch clamp studies to assess Topamax s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Topamax s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~11/24/2025 0:00:00
384914~"CDER"~"NDA"~20882.00~"VIATRIS SPECIALTY LLC"~"Neurontin (Gabapentin) "~10/9/1998 0:00:00~"Original"~1~"3643-1"~"PMR"~"505 (o)(3)"~3643.00~1~"PMR 3643-1: In a healthy non-drug dependent population with drug abuse experience with sedative drugs, evaluate the abuse potential of gabapentin. This clinical trial will evaluate gabapentin in a cross-over design with comparison to placebo and a positive control (such as diazepam) regarding abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and drug pharmacokinetics."~"Delayed"~"The applicant requested revised milestones because of continued discussions with the Agency to provide necessary guidance for them to conduct a new study for this PMR. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 07/11/2025."~4/30/2021 0:00:00~4/30/2026 0:00:00
384915~"CDER"~"BLA"~103411.00~"Servier Pharmaceuticals  LLC"~"Oncaspar (pegaspargase)"~2/1/1994 0:00:00~"Supplement"~5052~"2656-3"~"PMC"~"506B PMC"~2656.00~3~"PMC 2656-3: To commit to testing samples from patients treated with Oncaspar for the presence of binding and neutralizing antibodies to Oncaspar using the validated assays discussed in items 1 and 2 in the following study, entitled, ""A Multi-Center, Open Label, Phase 1 Study Evaluating the Safety and Tolerability of Intravenous Pegaspargase in Combination with Intravenous Gemcitabine HCl in Patients with Advanced and/or Metastatic Solid Tumors and Lymphoma""."~"Terminated"~"Study terminated because the initial dose level tested exceeded the MTD. Report submitted September 15, 2009 (STN 5117)"~12/31/2008 0:00:00~9/12/2025 0:00:00
384916~"CDER"~"BLA"~103772.00~"Janssen Biotech, Inc."~"Remicade (infliximab) "~8/24/1998 0:00:00~"Supplement"~5129~"2466-1"~"PMC"~"506B PMC"~2466.00~1~"PMC 2466-1: Centocor agrees to conduct a prospective, multi-center registry including 4000 adult psoriasis patients treated with commercial REMICADE in the United States.  This registry will characterize and assess the incidence of serious adverse events (including serious infection, tuberculosis, opportunistic infections, malignancies, hypersensitivity reactions, autoimmune reactions and deaths) as well as other adverse events of interest in the study cohort.  All enrolled study patients will be evaluated twice yearly for a period of at least 8 years with comprehensive annual reports provided to the agency.  Centocor agrees to collect data on the patient characteristics, demographics and drug exposure (including dose, duration and time to onset of adverse event).  The collection of data will be via active surveillance methods and data will be validated by a review of medical records as per the Guidance for Industry: Good Pharmacovigilance Practices and Pharmacoepidemiologic Assessment."~"Submitted"~~12/1/2018 0:00:00~10/16/2025 0:00:00
384917~"CDER"~"BLA"~103772.00~"Janssen Biotech, Inc."~"Remicade (infliximab) "~8/24/1998 0:00:00~"Supplement"~5138~"2380-1"~"PMC"~"506B PMC"~2380.00~1~"PMC 2380-1: Centocor commits to designing and implementing a registry of patients with pediatric Crohn's disease being treated with REMICADE that will be established to obtain long-term clinical status and safety information.  Information will be collected on patient demographics, disease characteristics, history of concomitant medications, dose and duration and frequency of REMICADE administration, clinical status, adverse events including dysplasias and malignancies of all types, infections, autoimmune disease, assessment of immunogenicity, and potential effects of antibody formation.  The age range should include patients ages 0 to 19 years.  This registry will be designed so that detailed clinical status information is collected at registry entry and on a 6 month basis for at least 20 years.  Centocor commits to expand the currently existing Pediatric IBD Registry, and will actively encourage both patients and physicians to participate in the registry through an advertisement campaign, that includes a plan for proactive communication of associated risk.  Centocor also commits to recruiting at least 2,000 REMICADE treated pediatric Crohn's patients, which will provide an adequate number of patients to participate in the registry so that outcome measures will be collected and adequate risk assessment can be made.  Centocor commits to provide prompt risk communication for serious adverse events that are reported through the registry.  The registry data will be analyzed at yearly intervals and the results will be submitted in annual reports for BB-IND 5389."~"Ongoing"~~6/30/2027 0:00:00~10/16/2025 0:00:00
384918~"CDER"~"BLA"~103772.00~"Janssen Biotech, Inc."~"Remicade (infliximab) "~8/24/1998 0:00:00~"Supplement"~5301~"2714-2"~"PMR"~"505 (o)(3)"~2714.00~2~"PMR 2714-2: Expand the Pediatric IBD Registry (DEVELOP) to include pediatric patients with ulcerative colitis (UC) and indeterminate colitis (IC)."~"Ongoing"~~12/31/2045 0:00:00~10/16/2025 0:00:00
384919~"CDER"~"BLA"~103772.00~"Janssen Biotech, Inc."~"Remicade (infliximab) "~8/24/1998 0:00:00~"Supplement"~5301~"2714-7"~"PMC"~"506B PMC"~2714.00~7~"PMC 2714-7: A study to analyze samples from the Pediatric IBD registry (DEVELOP) and PMR 3 to determine the presence of ADA using the new assay developed in PMC 5."~"Ongoing"~~12/31/2045 0:00:00~10/16/2025 0:00:00
384920~"CDER"~"BLA"~103846.00~"Solstice NeuroSciences, LLC"~"Myobloc (RimabotulinumtoxinB)"~12/8/2000 0:00:00~"Original"~1~"2324-9"~"PMR"~"505 (o)(3)"~2324.00~9~"PMR 2324-9: Submit safety data assessing distant spread of toxin effects after multiple administrations off Myobloc (botulinum toxin Type B), during a minimum period of 12 months, collected in at least 100 pediatric patients (ages 2-17 years) and 100 adult patients (approximately half  upper, and half lower extremity spasticity). In addition, submit data assessing the effects of Myobloc (botulinum toxin Type B) on blood glucose and alkaline phosphatase as a marker of bone metabolism. These safety data could come from open-label extensions of the clinical studies, from separate longer-term open-label safety studies, or from a long-term controlled safety and efficacy study. The doses evaluated must be at least as high as those shown effective in these studies, or those commonly used to treat spasticity."~"Delayed"~"The Final Report was submitted to FDA on 6/14/24. FDA issued a PMR and PMC Not Fulfilled letter on 01/28/25."~3/31/2019 0:00:00~2/5/2026 0:00:00
384921~"CDER"~"BLA"~103846.00~"Solstice NeuroSciences, LLC"~"Myobloc (RimabotulinumtoxinB)"~12/8/2000 0:00:00~"Original"~1~"2324-10"~"PMC"~"506B PMC"~2324.00~10~"PMC 2324-10: Randomized,-double-blind, adequately controlled, multiple fixed doses, parallel group clinical trials of Myobloc (botulinum toxin Type B) in the following populations: botulinum toxin-naive  children age 2-17 years with lower extremity spasticity, botulinum toxin-naive children age 2-17 years with upper extremity spasticity, botulinum toxin-naive adults with lower extremity spasticity, and botulinum toxin-naive adults with upper extremity spasticity. The minimum duration of each trial should be 12 weeks. The protocols for the trials should be submitted to the FDA as special protocol assessments (SPA)."~"Delayed"~"The Final Report was submitted to FDA on 6/14/24. FDA issued a PMR and PMC Not Fulfilled letter on 01/28/25."~5/31/2017 0:00:00~2/5/2026 0:00:00
384922~"CDER"~"BLA"~103846.00~"Solstice NeuroSciences, LLC"~"Myobloc (RimabotulinumtoxinB)"~12/8/2000 0:00:00~"Supplement"~5190~"3687-1"~"PMR"~"Pediatric Research Equity Act"~3687.00~1~"PMR 3687-1: A randomized, double-blind, placebo-controlled, dose-response study in pediatric subjects 3 to less than 17 years of age with chronic troublesome sialorrhea. Subjects will be randomized to receive a single injection session of low-dose Myobloc, high-dose Myobloc, or placebo. Children ages 6 to less than 17 years of age will be enrolled first."~"Terminated"~"The study was terminated on 12/20/22 because there was an issue
with product supply which made initiation of these studies not possible."~12/31/2023 0:00:00~2/5/2026 0:00:00
384923~"CDER"~"NDA"~202806.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tafinlar (dabrafenib) "~5/29/2013 0:00:00~"Supplement"~22~"4298-1"~"PMR"~"Accelerated Approval"~4298.00~1~"PMR 4298-1: Conduct a clinical trial(s) in at least 80 patients with solid tumors with a BRAF V600E mutation to verify and describe the clinical benefit of dabrafenib in combination with trametinib, through more precise estimation of the overall response rate and mature response duration. Include patients with unresectable or metastatic solid tumors with a BRAF V600E mutation from the ongoing trial and from a prospectively conducted trial (which will exclude patients with melanoma, non-small cell lung cancer, anaplastic thyroid cancer, biliary tract cancer, gliomas and colorectal cancer). Follow all patients for at least 6 months from the onset of response to characterize the response rate and duration. 
"~"Ongoing"~"The study has been initiated.."~10/31/2028 0:00:00~7/23/2025 0:00:00
384924~"CDER"~"NDA"~202806.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tafinlar (dabrafenib) "~5/29/2013 0:00:00~"Supplement"~22~"4298-4"~"PMC"~"506B PMC"~4298.00~4~"PMC 4298-4: Commitment to establish, through the use of clinical trial data, an in-vitro diagnostic device that is essential to the safe and effective use of dabrafenib and trametinib for patients with BRAF V600E mutations in solid tumor specimens, excluding colorectal cancer."~"Delayed"~"The final report milestone was missed due to challenges of sourcing the samples because of the low prevalence of the mutations across the required tumor types."~7/31/2024 0:00:00~7/23/2025 0:00:00
384925~"CDER"~"NDA"~202806.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tafinlar (dabrafenib) "~5/29/2013 0:00:00~"Supplement"~25~"4424-1"~"PMR"~"505 (o)(3)"~4424.00~1~"PMR 4424-1: Conduct comprehensive safety analyses from ongoing trials to further assess the serious risks of dabrafenib in combination with trametinib,including but not limited to new primary malignancies (cutaneous and non-cutaneous), cardiomyopathy, and ocular toxicities, in pediatric patients with BRAFV600E mutant low grade glioma over a sufficient period of follow-up time to further characterize these risks. The report should include appropriate monitoring and risk mitigation strategies."~"Ongoing"~~5/31/2027 0:00:00~7/23/2025 0:00:00
384926~"CDER"~"NDA"~202806.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tafinlar (dabrafenib) "~5/29/2013 0:00:00~"Supplement"~25~"4424-2"~"PMR"~"505 (o)(3)"~4424.00~2~"PMR 4424-2: Conduct an integrated safety analyses from clinical studies that further characterize the potential serious risk of long-term adverse effects including but not limited to growth plate abnormalities of dabrafenib in combination with trametinib on growth and development in a sufficient number of pediatric patients. Monitor patients for growth and development using age-appropriate screening tools. Include evaluations of growth as measured by height, weight, height velocity and height standard deviation scores, age at menarche if applicable (females) and Tanner stage. Monitor patients until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first."~"Ongoing"~~5/31/2027 0:00:00~7/23/2025 0:00:00
384927~"CDER"~"NDA"~202806.00~"NOVARTIS PHARMACEUTICALS CORP"~"Tafinlar (dabrafenib) "~5/29/2013 0:00:00~"Supplement"~25~"4424-4"~"PMC"~"506B PMC"~4424.00~4~"PMC 4424-4: Commitment to support the availability of an in vitro diagnostic device, through an appropriate analytical and clinical validation study using clinical trial data that demonstrates the device is essential to the safe and effective use of dabrafenib in combination with trametinib (D+T) for the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy."~"Delayed"~"The final report milestone was missed due to challenges with sourcing of samples due to low prevalence."~2/28/2025 0:00:00~7/23/2025 0:00:00
384928~"CDER"~"NDA"~202810.00~"SUPERNUS PHARMACEUTICALS INC"~"Oxtellar XR (Oxcarbazepine)"~10/19/2012 0:00:00~"Original"~1~"1938-1"~"PMR"~"Pediatric Research Equity Act"~1938.00~1~"PMR 1938-1: Deferred pediatric trial under PREA: A prospective, randomized, controlled, double-blind, efficacy/safety study of oxcarbazepine ER for the adjunctive  treatment of partial onset seizures in children ages one month to < 2 years. The primary efficacy endpoint during the controlled phase will examine seizure  frequency based upon Video/EEG data.treatment of partial onset seizures in children ages one month to < 2 years. The  primary efficacy endpoint during the controlled phase will examine seizure frequency based upon Video/EEG data."~"Delayed"~"The final report due date has passed with no final report received. Deferral Extension Requested 03/10/2017. Denied per FDA letter dated 04/21/2017. Notification of Non-Compliance With PREA letter issued 10/15/21. Per the applicants 12/23/2025 annual report, they continue to evaluate alternative means to formulate an oral solution of oxcarbazepine extended-release."~9/30/2021 0:00:00~12/23/2025 0:00:00
384929~"CDER"~"NDA"~202810.00~"SUPERNUS PHARMACEUTICALS INC"~"Oxtellar XR (Oxcarbazepine)"~10/19/2012 0:00:00~"Original"~1~"4088-1"~"PMR"~"505 (o)(3)"~4088.00~1~"PMR 4088-1: Conduct in vitro patch clamp studies to assess Oxtellar XR s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Oxtellar XR s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~12/23/2025 0:00:00
384930~"CDER"~"NDA"~202810.00~"SUPERNUS PHARMACEUTICALS INC"~"Oxtellar XR (Oxcarbazepine)"~10/19/2012 0:00:00~"Original"~1~"1938-2"~"PMR"~"Pediatric Research Equity Act"~1938.00~2~"PMR 1938-2: Deferred pediatric trial under PREA: A prospective, randomized, controlled, double-blind, efficacy/safety study of oxcarbazepine ER for the adjunctive  treatment of partial onset seizures in children ages 2 to <6 years. The primary efficacy endpoint during the controlled phase will examine seizure frequency based upon diary data."~"Delayed"~"The final report due date has passed with no final report received. Deferral Extension Requested 03/10/2017. Denied per FDA letter dated 04/21/2017. Notification of Non Compliance With PREA letter issued 10/15/21. Per the applicants 12/23/2025 annual report, they continue to evaluate alternative means to formulate an oral solution of oxcarbazepine extended-release."~9/30/2021 0:00:00~12/23/2025 0:00:00
384931~"CDER"~"NDA"~202810.00~"SUPERNUS PHARMACEUTICALS INC"~"Oxtellar XR (Oxcarbazepine)"~10/19/2012 0:00:00~"Original"~1~"1938-3"~"PMR"~"Pediatric Research Equity Act"~1938.00~3~"PMR 1938-3: A clinical trial to examine pharmacokinetics and tolerability in children ages 6 months to 4 years using an age appropriate extended release oxcarbazepine formulation."~"Delayed"~"The final report due date has passed with no final report received. Deferral Extension Requested 03/10/2017. Denied per FDA letter dated 04/21/2017."~12/31/2016 0:00:00~12/23/2025 0:00:00
384932~"CDER"~"NDA"~202810.00~"SUPERNUS PHARMACEUTICALS INC"~"Oxtellar XR (Oxcarbazepine)"~10/19/2012 0:00:00~"Original"~1~"1938-4"~"PMR"~"Pediatric Research Equity Act"~1938.00~4~"PMR 1938-4: A clinical trial to examine pharmacokinetics and tolerability in children, ages 1 month to 6 months, using an age appropriate extended release oxcarbazepine  formulation."~"Delayed"~"The final report due date has passed with no final report received. Deferral Extension Requested 03/10/2017. Denied per FDA letter dated 04/21/2017."~12/31/2016 0:00:00~12/23/2025 0:00:00
384933~"CDER"~"NDA"~202811.00~"ABBVIE INC"~"Linzess (linaclotide)"~8/30/2012 0:00:00~"Original"~1~"2161-2"~"PMR"~"Pediatric Research Equity Act"~2161.00~2~"PMR 2161-2: Conduct a safety and efficacy study in pediatric patients with chronic idiopathic constipation ages 2 to 5 years treated with Linzess  (linaclotide)."~"Fulfilled"~"Per FDA letter dated 05/21/2026, this PMR/PMC has been fulfilled."~3/31/2026 0:00:00~10/23/2025 0:00:00
384934~"CDER"~"NDA"~207999.00~"INTERCEPT PHARMACEUTICALS INC"~"Ocaliva (Obeticholic Acid)"~5/27/2016 0:00:00~"Original"~1~"3057-2"~"PMR"~"Accelerated Approval"~3057.00~2~"PMR 3057-2: A randomized, placebo-controlled trial to evaluate the safety and efficacy of OCALIVA (obeticholic acid) used as monotherapy in patients with primary biliary cholangitis (PBC) who are intolerant of or non-responsive to  ursodeoxycholic acid (UDCA). Enroll patients across all stages of PBC, by the Rotterdam criteria. You may conduct this as a stand-alone trial or in a sub-set of patients in your confirmatory trial (PMR # 3057-3)."~"Released"~"Per FDA letter dated 11/26/2025, this PMR/PMC has been released."~4/30/2023 0:00:00~7/25/2025 0:00:00
384935~"CDER"~"NDA"~207999.00~"INTERCEPT PHARMACEUTICALS INC"~"Ocaliva (Obeticholic Acid)"~5/27/2016 0:00:00~"Original"~1~"3057-3"~"PMR"~"Accelerated Approval"~3057.00~3~"PMR 3057-3: A randomized, double-blind, placebo-controlled trial to verify and describe that OCALIVA (obeticholic acid) induced reductions in alkaline phosphatase and/or total bilirubin are associated with improvements in the composite clinical endpoint of progression to cirrhosis, death, transplant, decompensation events and hepatocellular cancer. Your ongoing trial (747-302) should be revised to include patients across the spectrum of stages of primary biliary cholangitis (PBC), including patients with early, moderately advanced and advanced PBC by the Rotterdam criteria, and should be adequately powered to demonstrate benefit in each stage."~"Released"~"Per FDA letter dated 11/26/2025, this PMR/PMC has been released."~4/30/2023 0:00:00~7/25/2025 0:00:00
384936~"CDER"~"NDA"~208010.00~"EIRGEN PHARMA LTD"~"Rayaldee (Calcifediol) "~6/17/2016 0:00:00~"Original"~1~"3052-7"~"PMR"~"Pediatric Research Equity Act"~3052.00~7~"PMR 3052-7: Conduct a single arm study evaluating PK/PD and safety of Rayaldee (calcifediol) in patients with stage 3 or 4 chronic kidney disease with secondary hyperparathyroidism and 25-OH vitamin D levels < 30 mcg/L and age 1 month to less than 18 years."~"Pending"~"The study/trial has not begun but does not meet the criterion for delayed."~2/28/2029 0:00:00~8/22/2025 0:00:00
384937~"CDER"~"NDA"~208065.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Tagrisso (Osimertinib) "~11/13/2015 0:00:00~"Supplement"~30~"4584-1"~"PMC"~"506B PMC"~4584.00~1~"PMC 4584-1: Complete clinical trial D5169C00001 (FLAURA2), titled  A Phase III, Open-label, Randomized Study of Osimertinib With or Without Platinum Plus Pemetrexed Chemotherapy, as First-line Treatment in Patients with Epidermal Growth Factor Receptor Mutation Positive, Locally Advanced or Metastatic Non-small Cell Lung Cancer , to obtain mature overall survival data in accordance with the prespecified final analysis of FLAURA2."~"Submitted"~~7/31/2026 0:00:00~1/2/2026 0:00:00
384938~"CDER"~"NDA"~208065.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Tagrisso (Osimertinib) "~11/13/2015 0:00:00~"Supplement"~33~"4684-1"~"PMC"~"506B PMC"~4684.00~1~"PMC 4684-1: Complete clinical trial LAURA, titled,  A Phase III randomized, double-blind, placebo controlled, multicenter, global study assessing the efficacy and safety of osimertinib in patients with locally advanced, unresectable
(Stage III) NSCLC whose disease has not progressed during or following platinum-based chemoradiation therapy and has an EGFR exon 19 deletion or L858R mutation (LAURA)  to provide additional long-term efficacy data. Include the overall survival analysis and datasets after 120
overall survival events have occurred."~"Ongoing"~~12/31/2027 0:00:00~1/2/2026 0:00:00
384939~"CDER"~"NDA"~208065.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Tagrisso (Osimertinib) "~11/13/2015 0:00:00~"Supplement"~33~"4684-2"~"PMC"~"506B PMC"~4684.00~2~"PMC 4684-2: Conduct an integrated analysis containing data from clinical trials and observational studies (e.g., real world evidence), post-marketing reports, and other sources to further characterize the safety and efficacy of osimertinib following chemoradiation in Black or African American patients diagnosed with unresectable Stage III non-small cell lung cancer harboring EGFR exon 19 deletion or exon 21 L858R mutations. The analyses should support an evaluation of comparative efficacy/effectiveness and safety between the aforementioned populations and Asian and White patients."~"Pending"~~10/31/2031 0:00:00~1/2/2026 0:00:00
384940~"CDER"~"NDA"~208078.00~"CATALYST PHARMACEUTICALS INC"~"Firdapse (amifampridine phosphate)"~11/28/2018 0:00:00~"Original"~1~"3544-2"~"PMR"~"505 (o)(3)"~3544.00~2~"PMR 3544-2: Establish a Pregnancy Surveillance Program to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to Firdapse (amifampridine) during pregnancy. Provide a complete protocol that includes details regarding how you plan to encourage patients and providers to report pregnancy exposures (e.g., telephone contact number and/or website in prescribing information), measures to ensure complete data capture regarding pregnancy outcomes and any adverse effects in offspring, and plans for comprehensive data analysis and yearly reporting."~"Ongoing"~~7/31/2030 0:00:00~1/26/2026 0:00:00
384941~"CDER"~"NDA"~208078.00~"CATALYST PHARMACEUTICALS INC"~"Firdapse (amifampridine phosphate)"~11/28/2018 0:00:00~"Supplement"~8~"4344-1"~"PMR"~"505 (o)(3)"~4344.00~1~"PMR 4344-1: A juvenile animal toxicology study of amifampridine in rat."~"Submitted"~~9/30/2024 0:00:00~1/26/2026 0:00:00
384942~"CDER"~"NDA"~208088.00~"VERITY PHARMACEUTICALS INC"~"TLANDO (Testosterone Undecanoate)"~3/28/2022 0:00:00~"Original"~1~"4255-1"~"PMR"~"Pediatric Research Equity Act"~4255.00~1~"PMR 4255-1: A trial of testosterone replacement therapy in pediatric males ages 12 years to less than 18 years of age for conditions associated with a
deficiency or absence of endogenous testosterone due to primary hypogonadism or hypogonadotropic hypogonadism."~"Delayed"~"Draft and Final Protocol Submission dates were missed due to continued discussions with the Agency."~10/31/2029 0:00:00~6/8/2026 0:00:00
384943~"CDER"~"NDA"~208088.00~"VERITY PHARMACEUTICALS INC"~"TLANDO (Testosterone Undecanoate)"~3/28/2022 0:00:00~"Original"~1~"4256-2"~"PMR"~"505 (o)(3)"~4256.00~2~"PMR 4256-2: An appropriately designed one-year trial to evaluate for the development of adrenal insufficiency with chronic oral testosterone undecanoate therapy. Assess adrenal function with Cosyntropin stimulation testing prior to starting testosterone undecanoate, and again after six months and one year on testosterone undecanoate. Test at earlier timepoints for subjects who demonstrate signs or symptoms consistent with adrenal insufficiency. Assess serum cortisol, adrenocorticotropic hormone, and corticosteroid binding globulin concentrations prior to Cosyntropin 0.25 mg injection and serum cortisol concentrations at 30 minutes and 60 minutes after the injection. Standardize the testing time to 8 AM and the route of Cosyntropin administration (intramuscular or intravenous). Perform hormonal analytical assays in a central laboratory on batched serum samples."~"Delayed"~"Draft and Final Protocol Submission dates were missed due to
continued discussions with the Agency."~10/31/2025 0:00:00~6/8/2026 0:00:00
384944~"CDER"~"NDA"~20882.00~"VIATRIS SPECIALTY LLC"~"Neurontin (Gabapentin) "~10/9/1998 0:00:00~"Original"~1~"3643-2"~"PMR"~"505 (o)(3)"~3643.00~2~"PMR 3643-2: In a healthy non-drug dependent population with drug abuse experience with opioids, evaluate the abuse potential of gabapentin taken concomitantly with an opioid, such as oxycodone, investigating a range of doses of gabapentin combined with the opioid. This clinical trial will evaluate the gabapentin/opioid combinations in a cross-over design with comparison to placebo, gabapentin alone, and a moderate dose of the opioid taken alone as a positive control. The assessments will include abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and pharmacokinetics of both gabapentin and the opioid (oxycodone) with the goal of characterizing any additive or synergistic effects on the abuse potential and physiologic effects of co-administered gabapentin and opioid."~"Delayed"~"The applicant requested revised milestones because of continued discussions with the Agency to provide necessary guidance for the applicant to conduct a new study for this PMR. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 07/11/2025."~8/31/2021 0:00:00~4/30/2026 0:00:00
384945~"CDER"~"NDA"~21014.00~"NOVARTIS PHARMACEUTICALS CORP"~"Trileptal (Oxcarbazepine)"~1/14/2000 0:00:00~"Original"~1~"4096-1"~"PMR"~"505 (o)(3)"~4096.00~1~"PMR 4096-1: Conduct in vitro patch clamp studies to assess Trileptal s inhibitory profile on cardiac sodium channel (NaV1.5), including establishing Trileptal s potency and Vaughan Williams antiarrhythmic drug (AAD) classification."~"Submitted"~~1/31/2023 0:00:00~3/11/2026 0:00:00
384946~"CDER"~"NDA"~21085.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Avelox (Moxifloxacin Hydrochloride)"~12/10/1999 0:00:00~"Supplement"~60~"2898-1"~"PMR"~"Animal Efficacy"~2898.00~1~"PMR 2898-1: Conduct a field study to evaluate the efficacy and safety of moxifloxacin hydrochloride in the event of an attack with the intentional release of Y. pestis in  the United States."~"Pending"~~~2/6/2026 0:00:00
384947~"CDER"~"NDA"~21129.00~"VIATRIS SPECIALTY LLC"~"Neurontin (Gabapentin) "~3/2/2000 0:00:00~"Original"~1~"3643-1"~"PMR"~"505 (o)(3)"~3643.00~1~"PMR 3643-1: In a healthy non-drug dependent population with drug abuse experience with sedative drugs, evaluate the abuse potential of gabapentin. This clinical trial will evaluate gabapentin in a cross-over design with comparison to placebo and a positive control (such as diazepam) regarding abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and drug pharmacokinetics."~"Delayed"~"The applicant requested revised milestones because of continued discussions with the Agency to provide necessary guidance for them to conduct a new study for this PMR. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 07/11/2025."~4/30/2021 0:00:00~4/30/2026 0:00:00
384948~"CDER"~"NDA"~21129.00~"VIATRIS SPECIALTY LLC"~"Neurontin (Gabapentin) "~3/2/2000 0:00:00~"Original"~1~"3643-2"~"PMR"~"505 (o)(3)"~3643.00~2~"PMR 3643-2: In a healthy non-drug dependent population with drug abuse experience with opioids, evaluate the abuse potential of gabapentin taken concomitantly with an opioid, such as oxycodone, investigating a range of doses of gabapentin combined with the opioid. This clinical trial will evaluate the gabapentin/opioid combinations in a cross-over design with comparison to placebo, gabapentin alone, and a moderate dose of the opioid taken alone as a positive control. The assessments will include abuse-related subjective responses, physiological responses (including an assessment of respiratory depression), and pharmacokinetics of both gabapentin and the opioid (oxycodone) with the goal of characterizing any additive or synergistic effects on the abuse potential and physiologic effects of co-administered gabapentin and opioid."~"Delayed"~"The applicant requested revised milestones because of continued discussions with the Agency to provide necessary guidance for the applicant to conduct a new study for this PMR. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 07/11/2025."~8/31/2021 0:00:00~4/30/2026 0:00:00
384949~"CDER"~"NDA"~21164.00~"FABRE KRAMER PHARMACEUTICALS INC"~"Exxua (gepirone)"~9/22/2023 0:00:00~"Original"~1~"4485-1"~"PMR"~"505 (o)(3)"~4485.00~1~"PMR 4485-1: Collect data from a prospective pregnancy exposure registry, preferably a disease-based multiproduct registry, using a cohort analysis that compares the maternal, fetal, and infant outcomes of women with MDD exposed to Exxua (gepirone) during pregnancy with an unexposed comparator population(s) in a timely manner. Align the study protocol with protocol(s) outside the US to reach the target sample size. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortion, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes described in the protocol will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The final protocol submission was missed and the FDA issued a missed milestone letter on 04/09/2026."~3/31/2036 0:00:00~11/7/2025 0:00:00
384950~"CDER"~"NDA"~21164.00~"FABRE KRAMER PHARMACEUTICALS INC"~"Exxua (gepirone)"~9/22/2023 0:00:00~"Original"~1~"4485-2"~"PMR"~"505 (o)(3)"~4485.00~2~"PMR 4485-2: Conduct an additional pregnancy study that uses a different design from the pregnancy registry (for example a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, small for gestational age births and preterm births in women exposed to Exxua (gepirone) during pregnancy compared to an unexposed control population."~"Delayed"~"The final protocol submission was missed and the FDA issued a missed milestone letter on 04/09/2026."~4/30/2031 0:00:00~11/7/2025 0:00:00
384951~"CDER"~"NDA"~21164.00~"FABRE KRAMER PHARMACEUTICALS INC"~"Exxua (gepirone)"~9/22/2023 0:00:00~"Original"~1~"4485-3"~"PMR"~"505 (o)(3)"~4485.00~3~"PMR 4485-3: Perform a lactation study (milk only) in lactating women who have received Exxua (gepirone) to assess concentrations of gepirone in breastmilk using a validated assay and to assess the effects on the breastfed infant."~"Delayed"~"The final protocol submission was missed and the FDA issued a missed milestone letter on 04/09/2026."~12/31/2027 0:00:00~11/7/2025 0:00:00
384952~"CDER"~"NDA"~21164.00~"FABRE KRAMER PHARMACEUTICALS INC"~"Exxua (gepirone)"~9/22/2023 0:00:00~"Original"~1~"4485-4"~"PMR"~"505 (o)(3)"~4485.00~4~"PMR 4485-4: Conduct a multiple dose thorough QT/QTc study in healthy subjects to assess the effects of gepirone ER and its major metabolites on the QTc interval using 12-lead digital ECG recordings. The study should include a positive control, a placebo control, the highest recommended dose (72.6 mg once daily) and a supratherapeutic dose which covers the high clinical exposure scenario. The primary analysis should be by-time as described in the ICH E14 guideline. The sample size of the study should have at least as much power as one be based on excluding a 10-msec mean increase from placebo in baseline-adjusted QTc interval."~"Delayed"~"The final protocol submission was missed and the FDA issued a missed milestone letter on 04/09/2026."~2/28/2027 0:00:00~11/7/2025 0:00:00
384953~"CDER"~"BLA"~761363.00~"Merck Sharp & Dohme LLC"~"Winrevair (sotatercept-csrk)"~3/26/2024 0:00:00~"Original"~1~"4590-1"~"PMR"~"505 (o)(3)"~4590.00~1~"PMR 4590-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Winerevair (sotatercept-csrk) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~10/31/2035 0:00:00~5/19/2026 0:00:00
384954~"CDER"~"BLA"~761364.00~"Accord BioPharma Inc."~"Imuldosa (ustekinumab-srlf) injection"~10/10/2024 0:00:00~"Original"~1~"4700-1"~"PMR"~"Pediatric Research Equity Act"~4700.00~1~"PMR 4700-1: Develop a presentation that can be used to accurately administer Imuldosa (ustekinumab-srlf) to pediatric patients who weigh less than 60 kg."~"Delayed"~"Original Final Report Due Date: 04/30/2025; Deferral Extension request received after the milestone date had passed but was granted per FDA letter dated 08/05/2025. Final report due date extended to 01/31/2026. Second Deferral Extension Granted per FDA letter dated 02/18/2026."~12/31/2027 0:00:00~
384955~"CDER"~"BLA"~761365.00~"Astellas Pharma US, Inc."~"Vyloy (zolbetuximab-clzb)"~10/18/2024 0:00:00~"Original"~1~"4723-1"~"PMC"~"506B PMC"~4723.00~1~"PMC 4723-1: Conduct a clinical trial to further characterize the clinical effects of zolbetuximab-clzb, including pharmacokinetics (PK), activity, and safety in the underrepresented racial and ethnic minority populations. The analysis should support an evaluation of comparative efficacy and safety between the aforementioned population and the population primarily represented in your trial."~"Ongoing"~~6/30/2031 0:00:00~12/12/2025 0:00:00
384956~"CDER"~"BLA"~761365.00~"Astellas Pharma US, Inc."~"Vyloy (zolbetuximab-clzb)"~10/18/2024 0:00:00~"Original"~1~"4723-3"~"PMC"~"506B PMC"~4723.00~3~"PMC 4723-3: Conduct an analysis of the incidence of neutralizing antibodies against zolbetuximab-clzb, using a validated neutralization assay, in patients enrolled in the pivotal clinical studies of zolbetuximab-clzb (i.e., SPOTLIGHT and GLOW), and evaluate the potential clinical impact of the neutralizing antibodies on the pharmacokinetics, pharmacodynamics, safety, and efficacy of zolbetuximab-clzb."~"Submitted"~~11/30/2025 0:00:00~12/12/2025 0:00:00
384957~"CDER"~"BLA"~761371.00~"Genentech, Inc."~"Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) injection"~9/13/2024 0:00:00~"Original"~1~"4689-1"~"PMR"~"Pediatric Research Equity Act"~4689.00~1~"PMR 4689-1: Conduct an open-label study of the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ocrelizumab and hyaluronidase in pediatric patients with relapsing multiple sclerosis (RMS) at least 10 years and less than 17 years of age, weighing 40 kg or less. The objective of this study is to determine a dose of ocrelizumab and hyaluronidase that will result in PK and PD effects that are comparable to those of the dose administered to adult patients with RMS. Safety assessments will continue for at least 2 years after the last dose of ocrelizumab and hyaluronidase."~"Pending"~"Per FDA letter dated 03/26/2026, the applicant s request for release was denied. The study has not been initiated, but does not meet the criterion for delayed."~4/30/2032 0:00:00~5/22/2026 0:00:00
384958~"CDER"~"BLA"~761371.00~"Genentech, Inc."~"Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) injection"~9/13/2024 0:00:00~"Original"~1~"4689-2"~"PMR"~"Pediatric Research Equity Act"~4689.00~2~"PMR 4689-2: Conduct a pediatric assessment, with extrapolation to ocrelizumab and hyaluronidase, of the findings from Studies WN42086 (a randomized, double-blind, parallel-group study in pediatric patients ages 10 through 17 years to evaluate the safety and efficacy of Ocrevus [intravenous ocrelizumab] compared to an appropriate control for the treatment of RMS) and WA39085 (an open-label study of the safety, tolerability, PK, and PD of ocrelizumab in pediatric patients), which are studies intended to fulfill PMR 3194-14 for Ocrevus (ocrelizumab)."~"Pending"~"The trial has not begun but does not meet the criterion for
delayed."~4/30/2032 0:00:00~5/22/2026 0:00:00
384959~"CDER"~"BLA"~761371.00~"Genentech, Inc."~"Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) injection"~9/13/2024 0:00:00~"Original"~1~"4689-3"~"PMR"~"505 (o)(3)"~4689.00~3~"PMR 4689-3: Conduct an observational, single-arm safety study to assess the risk of injection-related reactions in patients treated with subcutaneouslyadministered ocrelizumab. The study should evaluate the incidence, clinical features, and severity of injection-related reactions to enable a detailed characterization of local and systemic injection-related reactions that can occur with subcutaneously-administered ocrelizumab. The study should involve monitoring subjects for at least 2 years following subcutaneously-administered ocrelizumab treatment initiation."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 03/30/2026."~2/28/2031 0:00:00~5/22/2026 0:00:00
384960~"CDER"~"BLA"~761371.00~"Genentech, Inc."~"Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) injection"~9/13/2024 0:00:00~"Original"~1~"4689-4"~"PMR"~"505 (o)(3)"~4689.00~4~"PMR 4689-4: Conduct a prospective longitudinal observational study in adult patients with relapsing multiple sclerosis and primary progressive multiple sclerosis exposed to ocrelizumab to determine the incidence and mortality rates of breast cancer and all malignancies. All patients enrolled in the study should be followed for a minimum of 5 years or until death following their first exposure to ocrelizumab. The protocol must specify two appropriate populations to which the observed incidence and mortality rates will be compared."~"Ongoing"~~11/30/2030 0:00:00~5/22/2026 0:00:00
384961~"CDER"~"BLA"~761375.00~"Eisai, Incorporated"~"Leqembi IQLIK (lecanemab-irmb) Injection"~8/29/2025 0:00:00~"Original"~1~"4871-1"~"PMR"~"505 (o)(3)"~4871.00~1~"PMR 4871-1: Conduct a registry-based, prospective, observational study to evaluate clinical safety outcomes among Alzheimer s disease patients treated with lecanemab-irmb, using, for example, the Alzheimer s Network for Treatment and Diagnostics (ALZ-NET) registry, including patients who are ApoE e4 homozygotes, and/or exposed to antithrombotics, and/or have a diagnosis of, or imaging findings consistent with a high risk for, cerebral amyloid angiopathy. The primary clinical safety outcomes should include amyloid related imaging abnormalities (ARIA)-edema (ARIA-E), and ARIA hemosiderin deposition (ARIA-H) and any associated clinical symptoms, and intracerebral hemorrhage >1 cm in size. Additional outcomes of interest should also include seizures, anaphylaxis, and death. Baseline characterization of the registry population should include demographic data, diagnosis and stage of disease, ApoE genotype, baseline MRI findings (e.g., microhemorrhages, evidence of cerebral amyloid angiography or other imaging findings consistent with high risk of cerebral amyloid angiography, etc.), other biomarkers that are potential predictors of disease course or adverse outcomes, and prior medications including prior Alzheimer s disease (AD) therapy and antithrombotic therapy. The registry should also collect information on concomitant medications (e.g., antiplatelet and antithrombotic drugs, other AD treatments). When available, the study should provide a comparison of safety outcomes to estimated background rates in an appropriate comparator population."~"Delayed"~"The final protocol submission milestone was missed because of time needed to revise the draft study protocol based on the Agency s recommendations. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 03/17/2026."~1/31/2036 0:00:00~
384962~"CDER"~"BLA"~761375.00~"Eisai, Incorporated"~"Leqembi IQLIK (lecanemab-irmb) Injection"~8/29/2025 0:00:00~"Original"~1~"4871-2"~"PMR"~"505 (o)(3)"~4871.00~2~"PMR 4871-2: Use emerging safety data from ongoing studies and published literature, validate administrative claim codes for intracerebral hemorrhage in patients with Alzheimer s disease. The outcome of intracerebral hemorrhage should distinguish between amyloid related imaging abnormalities-hemosiderin deposition (ARIA-H) and cerebral hemorrhage greater than 1 cm. Secondary outcomes of interest include ARIA-edema (ARIA-E) and ARIA-H, seizures, anaphylaxis, and death. For secondary outcomes not well validated, develop algorithms and/or computable phenotypes using data leveraged from PMR 4871-1 and other sources for the outcomes of interest. Describe an approach to identifying an appropriate comparator group with Alzheimer s disease untreated with lecanemab-irmb. Obtain FDA agreement with the outcome algorithm specifications and comparator population prior to proceeding to conducting the retrospective cohort study. Based upon validated algorithms agreed to by the Sponsor and FDA, conduct a comparative retrospective cohort study using claims data with available medical chart review as needed or electronic health record data to assess clinical safety outcomes in a broad population of Alzheimer s disease patients treated with lecanemab-irmb."~"Delayed"~"The interim report submission milestone was missed because of time needed to revise the draft study protocol based on the Agency s recommendations. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 03/17/2026."~12/31/2030 0:00:00~
384963~"CDER"~"BLA"~761375.00~"Eisai, Incorporated"~"Leqembi IQLIK (lecanemab-irmb) Injection"~8/29/2025 0:00:00~"Original"~1~"4871-3"~"PMR"~"505 (o)(3)"~4871.00~3~"PMR 4871-3: Further characterize the safety of treatment with lecanemab-irmb in patients who are homozygous for ApoE e4. We would accept information on this risk from a randomized, clinical trial in participants with early preclinical Alzheimer s disease and intermediate amyloid (i.e., AHEAD 3-45 Study). Ensure that approximately 15% of the population, distributed equally among lecanemab-irmb and control, is homozygous for ApoE e4."~"Pending"~~2/28/2030 0:00:00~
384964~"CDER"~"BLA"~761380.00~"BeiGene USA, Inc."~"Tevimbra (tislelizumab- jsgr)"~3/3/2025 0:00:00~"Original"~1~"4774-1"~"PMC"~"506B PMC"~4774.00~1~"PMC 4774-1: Conduct an appropriate analytical and clinical validation study, using clinical trial data from RATIONALE-306, to support the availability of an in vitro diagnostic device to detect PD-L1 expression that is essential for the safe and effective use of tislelizumab in patients with esophageal squamous cell carcinoma."~"Pending"~~5/31/2026 0:00:00~
384965~"CDER"~"BLA"~761381.00~"Bristol-Myers Squibb Company"~"Opdivo Qvantig (nivolumab hyaluronidase-nvhy)"~12/27/2024 0:00:00~"Original"~1~"4762-1"~"PMR"~"Accelerated Approval"~4762.00~1~"PMR 4762-1: Submit the final report demonstrating an improvement in overall survival from a multicenter, randomized trial to confirm the clinical benefit of nivolumab in combination with ipilimumab over standard therapy in patients with advanced hepatocellular carcinoma, that may inform product labeling. Submit the datasets with the final report."~"Fulfilled"~"Per FDA letter dated 12/27/2025, this PMR/PMC has been fulfilled."~1/31/2025 0:00:00~2/20/2026 0:00:00
384966~"CDER"~"BLA"~761381.00~"Bristol-Myers Squibb Company"~"Opdivo Qvantig (nivolumab hyaluronidase-nvhy)"~12/27/2024 0:00:00~"Original"~1~"4762-2"~"PMR"~"Accelerated Approval"~4762.00~2~"PMR 4762-2: Submit the final report, including datasets, from trials conducted to verify and describe the clinical benefit of nivolumab 240 mg intravenously every two weeks in patients with microsatellite instability high or mismatch repair deficient metastatic colorectal cancer who have progressed following treatment with fluoropyrimidine, oxaliplatin and irinotecan, including at least 150 patients enrolled in BMS-initiated trials. In order to characterize response rate and duration, patients will be followed for at least 12 months from the onset of response."~"Fulfilled"~"Per FDA letter dated 10/27/2025, this PMR/PMC has been fulfilled."~11/30/2026 0:00:00~2/20/2026 0:00:00
384967~"CDER"~"BLA"~761381.00~"Bristol-Myers Squibb Company"~"Opdivo Qvantig (nivolumab hyaluronidase-nvhy)"~12/27/2024 0:00:00~"Original"~1~"4762-3"~"PMR"~"Accelerated Approval"~4762.00~3~"PMR 4762-3: Submit the final report, including datasets, from a randomized trial conducted to verify and describe the clinical benefit of nivolumab,
administered in combination with ipilimumab, in patients with microsatellite instability-high or mismatch repair deficient metastatic colorectal cancer. The trial will be designed to demonstrate a clinically meaningful improvement in progression-free survival in patients randomized to receive nivolumab and ipilimumab as compared to patients randomized to receive nivolumab alone. In addition, the trial should evaluate for differences in overall survival between arms based on a pre specified analysis. The analysis plan should describe the power for the overall survival analysis, as well as all assumptions made in determining the power."~"Fulfilled"~"Per FDA letter dated 10/27/2025, this PMR/PMC has been fulfilled."~11/30/2026 0:00:00~2/20/2026 0:00:00
384968~"CDER"~"BLA"~761381.00~"Bristol-Myers Squibb Company"~"Opdivo Qvantig (nivolumab hyaluronidase-nvhy)"~12/27/2024 0:00:00~"Original"~1~"4762-4"~"PMR"~"Pediatric Research Equity Act"~4762.00~4~"PMR 4762-4: Conduct a molecularly targeted pediatric cancer investigation using an age-appropriate formulation of nivolumab and hyaluronidase-nvhy in pediatric patients 12 years of age and older."~"Released"~"Per FDA letter dated 11/24/2025, this PMR/PMC has been released."~1/31/2025 0:00:00~2/20/2026 0:00:00
384969~"CDER"~"BLA"~761381.00~"Bristol-Myers Squibb Company"~"Opdivo Qvantig (nivolumab hyaluronidase-nvhy)"~12/27/2024 0:00:00~"Supplement"~2~"4925-1"~"PMR"~"Pediatric Research Equity Act"~4925.00~1~"PMR 4925-1: Conduct a modeling and simulation study to support dosing of nivolumab and hyaluronidase-nvhy as first-line treatment in pediatric patients 12 years of age and older with unresectable or metastatic microsatellite instability-high or mismatch repair deficient colorectal cancer (MSI-H/dMMR mCRC)."~"Submitted"~"The final report was submitted to FDA on 10/27/2025."~1/31/2025 0:00:00~2/20/2026 0:00:00
384970~"CDER"~"BLA"~761381.00~"Bristol-Myers Squibb Company"~"Opdivo Qvantig (nivolumab hyaluronidase-nvhy)"~12/27/2024 0:00:00~"Supplement"~8~"4854-1"~"PMC"~"506B PMC"~4854.00~1~"PMC 4854-1: Conduct an appropriate analytical validation study, based on clinical trial data from nivolumab studies to support development of a test to detect PD-L1 expression that is essential for the safe and effective use of nivolumab in patients with HER2-negative gastric/gastroesophageal junction adenocarcinoma and esophageal cancer."~"Ongoing"~~12/31/2026 0:00:00~2/20/2026 0:00:00
384971~"CDER"~"BLA"~761384.00~"AbbVie Inc."~"Emrelis (telisotuzumab vedotin-tllv) for injection"~5/14/2025 0:00:00~"Original"~1~"4844-1"~"PMR"~"Accelerated Approval"~4844.00~1~"PMR 4844-1: Complete the ongoing randomized trial, Study M18-868, intended to verify and describe the clinical benefit of telisotuzumab vedotin in patients with previously treated c-Met overexpressing, EGFR wildtype, locally advanced/metastatic non-squamous non-small cell lung cancer."~"Ongoing"~"The trial is underway."~6/30/2029 0:00:00~
384972~"CDER"~"BLA"~761384.00~"AbbVie Inc."~"Emrelis (telisotuzumab vedotin-tllv) for injection"~5/14/2025 0:00:00~"Original"~1~"4844-2"~"PMR"~"505 (o)(3)"~4844.00~2~"PMR 4844-2: Complete clinical trial, Study M25-274,  A Phase 2, Open-Label, Randomized, Global Study of Three Telisotuzumab Vedotin Regimens in Subjects with Previously Treated c-Met Overexpressing, EGFR Wildtype, Locally Advanced/Metastatic Non-Squamous Non-Small Cell Lung Cancer  to further assess known serious risks with telisotuzumab vedotin including interstitial lung disease, ocular disorders, and other severe adverse reactions, evaluating alternative dosages (flat dosage 1.9 mg/kg Q2W vs flat dosage 1.6 mg/kg Q2W vs. 1.6 mg/kg -> 1.9 mg/kg Q2W dose up-titration). Assess whether the alternative dosages tested may decrease serious toxicity in patients with high baseline tumor burden based on evaluation of the clinical trial safety and tolerability data and using population pharmacokinetic and exposure-response analyses updated with the clinical trial data."~"Ongoing"~~9/30/2029 0:00:00~
384973~"CDER"~"BLA"~761388.00~"Genentech, Inc."~"Piasky (crovalimab-akkz) injection"~6/20/2024 0:00:00~"Original"~1~"4647-1"~"PMR"~"505 (o)(3)"~4647.00~1~"PMR 4647-1: Establish a registry to characterize the long-term safety of Piasky (crovalimab-akkz) in adult and pediatric patients 13 years and older with paroxysmal nocturnal hemoglobinuria (PNH) with up to 5 years of follow- up. Yearly safety follow-up data should include a summary of major safety findings including meningococcal infections and other infections with encapsulated bacteria, infusion and injection site related reactions, hypersensitivity reactions including type III hypersensitivity reactions, and axonal peripheral neuropathy, including multifocal mononeuropathy."~"Pending"~~12/31/2032 0:00:00~8/14/2025 0:00:00
384974~"CDER"~"BLA"~761388.00~"Genentech, Inc."~"Piasky (crovalimab-akkz) injection"~6/20/2024 0:00:00~"Original"~1~"4647-2"~"PMR"~"505 (o)(3)"~4647.00~2~"PMR 4647-2: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Piasky (crovalimab-akkz) during pregnancy to assess the risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant. Assess infant outcomes through at least the first year of life. The registry should also collect adverse event data for lactating women and infants exposed to crovalimab through breastfeeding to assess for any potential risks to the infant from breastfeeding. The minimum number of patients will be specified in the protocol."~"Pending"~~12/31/2032 0:00:00~8/14/2025 0:00:00
384975~"CDER"~"BLA"~761388.00~"Genentech, Inc."~"Piasky (crovalimab-akkz) injection"~6/20/2024 0:00:00~"Original"~1~"4647-3"~"PMC"~"506B PMC"~4647.00~3~"PMC 4647-3: Complete Study BO42161 (COMMODORE 1),  A Phase 3, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Crovalimab Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated with Complement Inhibitors "~"Ongoing"~~2/28/2028 0:00:00~8/14/2025 0:00:00
384976~"CDER"~"BLA"~761388.00~"Genentech, Inc."~"Piasky (crovalimab-akkz) injection"~6/20/2024 0:00:00~"Original"~1~"4647-4"~"PMC"~"506B PMC"~4647.00~4~"PMC 4647-4: Complete Study BO42162 (COMMODORE 2),  A Phase 3, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated with Complement Inhibitors "~"Ongoing"~~2/28/2028 0:00:00~8/14/2025 0:00:00
384977~"CDER"~"BLA"~761388.00~"Genentech, Inc."~"Piasky (crovalimab-akkz) injection"~6/20/2024 0:00:00~"Original"~1~"4647-5"~"PMC"~"506B PMC"~4647.00~5~"PMC 4647-5: Complete Study BP39144 (COMPOSER),  An Adaptive Phase 1/2 Study to Assess Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) ."~"Ongoing"~~6/30/2027 0:00:00~8/14/2025 0:00:00
384978~"CDER"~"BLA"~761388.00~"Genentech, Inc."~"Piasky (crovalimab-akkz) injection"~6/20/2024 0:00:00~"Original"~1~"4647-6"~"PMC"~"506B PMC"~4647.00~6~"PMC 4647-6: Develop and validate a competitive ligand binding neutralizing antibody (NAb) assay with adequate sensitivity and drug tolerance to test inhibition of crovalimab-akkz. This NAb assay will be used to test available confirmed anti-drug antibody positive samples from banked and ongoing clinical studies. Provide a final validation report detailing the performance of the NAb assay."~"Submitted"~~6/30/2026 0:00:00~8/14/2025 0:00:00
384979~"CDER"~"BLA"~761391.00~"Galderma Laboratories, L.P."~"Nemluvio (nemolizumab-ilto)"~12/13/2024 0:00:00~"Original"~1~"4721-1"~"PMR"~"Pediatric Research Equity Act"~4721.00~1~"PMR 4721-1: Conduct a single-arm, open-label clinical trial to evaluate pharmacokinetics and safety in pediatric subjects from 2 years to less than 12 years of age with moderate-to-severe atopic dermatitis, which is not adequately controlled with topical therapy for a minimum duration of 52 weeks."~"Submitted"~"The final report was submitted to FDA on 10/29/2025 under BLA 761390."~10/31/2025 0:00:00~
384980~"CDER"~"BLA"~761391.00~"Galderma Laboratories, L.P."~"Nemluvio (nemolizumab-ilto)"~12/13/2024 0:00:00~"Original"~1~"4721-2"~"PMR"~"Pediatric Research Equity Act"~4721.00~2~"PMR 4721-2: Conduct a single-arm, open-label clinical trial to evaluate pharmacokinetics and safety in pediatric subjects from 6 months to less than 2 years of age with moderate-to-severe atopic dermatitis, which is not adequately controlled with topical therapy for a minimum duration of 52 weeks."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~5/31/2028 0:00:00~
384981~"CDER"~"BLA"~761392.00~"Samsung Bioepis Co., Ltd."~"Ospomyv (denosumab-dssb) injection"~2/13/2025 0:00:00~"Original"~1~"4799-1"~"PMR"~"Pediatric Research Equity Act"~4799.00~1~"PMR 4799-1: Provide an assessment of Ospomyv (denosumab-dssb) for the treatment of glucocorticoid-induced osteoporosis in pediatric patients 5 to 17 years of age."~"Fulfilled"~"Per FDA letter dated 10/29/2025, this PMR/PMC has been fulfilled."~6/30/2026 0:00:00~
384982~"CDER"~"BLA"~761394.00~"Daiichi Sankyo, Inc."~"Datroway (datopotamab deruxtecan-dlnk)"~1/17/2025 0:00:00~"Original"~1~"4776-1"~"PMC"~"506B PMC"~4776.00~1~"PMC 4776-1: Conduct an integrated analysis of data from clinical trials and observational studies (e.g., real world evidence), post-marketing reports, and other sources to further characterize the safety and efficacy/effectiveness of datopotamab deruxtecan in patients of underrepresented racial and ethnic minority groups with unresectable or metastatic HR+HER2-breast cancer (especially Black patients given their underrepresentation in the TROPION-Breast01 trial). The analyses should support an evaluation of comparative efficacy/effectiveness and safety between the population primarily represented in the trial (TROPIONBreast01) and the aforementioned underrepresented racial and ethnic minority population(s). This should also include pharmacokinetic data, if available."~"Pending"~~7/31/2030 0:00:00~
384983~"CDER"~"BLA"~761394.00~"Daiichi Sankyo, Inc."~"Datroway (datopotamab deruxtecan-dlnk)"~1/17/2025 0:00:00~"Original"~1~"4776-2"~"PMC"~"506B PMC"~4776.00~2~"PMC 4776-2: Complete [..] anti-drug antibody (ADA) assay validation and include report addendums containing high resolution drug tolerance data and justification to support that the ADA assays are fit-forpurpose to characterize the effects of ADA on pharmacokinetics, pharmacodynamics, safety, and effectiveness of datopotamab deruxtecan."~"Submitted"~~6/30/2025 0:00:00~
384984~"CDER"~"BLA"~761398.00~"Fresenius Kabi USA, LLC"~"Conexxence and Bomyntra (denosumab-bnht) injection"~3/25/2025 0:00:00~"Original"~1~"4819-1"~"PMR"~"Pediatric Research Equity Act"~4819.00~1~"PMR 4819-1: Provide an assessment of Conexxence (denosumab-bnht) for the treatment of glucocorticoid-induced osteoporosis in pediatric patients 5 to 17 years of age."~"Fulfilled"~"Per FDA letter dated 10/29/2025, this PMR/PMC has been fulfilled."~6/30/2026 0:00:00~
384985~"CDER"~"BLA"~761400.00~"Regeneron Pharmaceuticals, Inc."~"Lynozyfic (linvoseltamab-gcpt) injection"~7/2/2025 0:00:00~"Original"~1~"4848-1"~"PMR"~"Accelerated Approval"~4848.00~1~"PMR 4848-1: Complete a randomized clinical trial in patients with relapsed or refractory multiple myeloma. Patients should be randomized to receive linvoseltamab compared to standard therapy for relapsed or refractory multiple myeloma. The primary endpoint should be progression-free survival and key secondary endpoints should include overall response rate and overall survival. The trial should enroll a sufficiently representative study population to allow for generalizability of the result to the U.S. patient population with multiple myeloma."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2027 0:00:00~
384986~"CDER"~"BLA"~761404.00~"CELLTRION, Inc."~"Stoboclo (denosumab-bmwo), Osenvelt (denosumab-bmwo) "~2/28/2025 0:00:00~"Original"~1~"4792-1"~"PMR"~"Pediatric Research Equity Act"~4792.00~1~"PMR 4792-1: Provide an assessment of Stoboclo (denosumab-bmwo) for the treatment of glucocorticoid-induced osteoporosis in pediatric patients 5 to 17 years of age."~"Fulfilled"~"Per FDA letter dated 10/29/2025, this PMR/PMC has been fulfilled."~6/30/2026 0:00:00~
384987~"CDER"~"BLA"~761411.00~"Incyte Corporation"~"Niktimvo (axatilimab-csfr) Injection"~8/14/2024 0:00:00~"Original"~1~"4669-1"~"PMR"~"505 (o)(3)"~4669.00~1~"PMR 4669-1: Conduct a study to further characterize the nature, incidence, and severity of the known serious risk of infusion-related reactions (IRR) in at least 50 patients treated with at least 4 doses of axatilimab. The safety evaluation should include vital sign monitoring (temperature, pulse, respiratory rate, blood pressure, and oxygen saturation) at minimum immediately before initiation of the infusion; at 15 minutes, 30 minutes, and at the end of the infusion; and for a prescribed period post-infusion. Characterize changes in vital signs, anaphylaxis, respiratory distress, chills, back pain, flushing, chest-tightness, nausea, vomiting, and any other relevant signs or symptoms of IRR, as well as infusion interruptions and details regarding re-challenge (rate, time after initial interruption, outcome), if applicable. Collect details regarding medications used as pretreatment to prevent infusion-related reactions and medications used to alleviate infusionrelated reactions."~"Ongoing"~~2/28/2028 0:00:00~10/10/2025 0:00:00
384988~"CDER"~"BLA"~761411.00~"Incyte Corporation"~"Niktimvo (axatilimab-csfr) Injection"~8/14/2024 0:00:00~"Original"~1~"4669-2"~"PMR"~"505 (o)(3)"~4669.00~2~"PMR 4669-2: Complete Study SNDX-6352-0504 and follow patients until all have completed 5 years of follow-up or discontinued earlier, to further characterize and determine the long-term effects of axatilimab on liver enzymes, lipase, creatine kinase, bone homeostasis, and infections that may develop or not subside due to prolonged exposure to axatilimab, to assess the potential serious risks of hepatoxicity, pancreatitis, myositis, bone or growth disorders, and infections, in these patients with cGvHD. Include sufficient bone monitoring, including but not limited to serial assessments of bone mineral density, markers of bone formation and bone resorption, and calcium and phosphate levels. Include incidence rates of each adverse event, risk factors, time to onset, and outcomes."~"Ongoing"~~8/31/2028 0:00:00~10/10/2025 0:00:00
384989~"CDER"~"BLA"~761416.00~"Jazz Pharmaceuticals Ireland Limited"~"Ziihera (zanidatamab-hrii) for injection"~11/20/2024 0:00:00~"Original"~1~"4732-1"~"PMR"~"Accelerated Approval"~4732.00~1~"PMR 4732-1: Complete the ongoing randomized clinical trial, Study JZP598-302, entitled,  An Open-Label Randomized Trial of the Efficacy and Safety of Zanidatamab with Standard-of-Care Therapy Against Standard-of-Care Therapy Alone for Advanced HER2-Positive Biliary Tract Cancer , intended to verify and describe the clinical benefit of zanidatamab for patients with HER2-positive (IHC3+), unresectable or metastatic biliary tract cancer. The trial should compare zanidatamab in combination with the standard of care in patients with HER2-positive (IHC3+), unresectable or metastatic biliary tract cancer."~"Ongoing"~"102 patients our of the planned 286 patients have been enrolled."~9/30/2029 0:00:00~1/16/2026 0:00:00
384990~"CDER"~"BLA"~761416.00~"Jazz Pharmaceuticals Ireland Limited"~"Ziihera (zanidatamab-hrii) for injection"~11/20/2024 0:00:00~"Original"~1~"4732-2"~"PMC"~"506B PMC"~4732.00~2~"PMC 4732-2: Conduct an integrated analysis of clinical trial data to allow for further characterization of the clinical effects of zanidatamab, including pharmacokinetics (PK), efficacy and safety in the underrepresented racial and ethnic minority populations. These clinical data, which may come from other ongoing trials with zanidatamab, should report an evaluation of comparative efficacy and safety between the aforementioned population and the population primarily represented in your trial."~"Ongoing"~~9/30/2029 0:00:00~1/16/2026 0:00:00
384991~"CDER"~"BLA"~761416.00~"Jazz Pharmaceuticals Ireland Limited"~"Ziihera (zanidatamab-hrii) for injection"~11/20/2024 0:00:00~"Original"~1~"4732-3"~"PMC"~"506B PMC"~4732.00~3~"PMC 4732-3: Conduct an assessment of binding and neutralizing anti-zanidatamab antibody responses to evaluate the incidence of anti-drug antibodies (ADAs). Reanalyze the biliary tract cancer (BTC) samples from Study ZWI-ZW25-101 and all samples from Study ZWI-ZW25-203 using a validated assay capable of sensitively detecting ADA responses in the presence of zanidatamab levels that are expected to be present in the serum at the time of patient sampling. Reevaluate the immunogenicity of zanidatamab and the effect of ADAs on efficacy, pharmacokinetics (PKs), and safety of zanidatamab. Include the level of zanidatamab in each patient s test sample at each sampling point in the final report."~"Ongoing"~~9/30/2026 0:00:00~1/16/2026 0:00:00
384992~"CDER"~"BLA"~761417.00~"BeiGene USA, Inc."~"Tevimbra (tislelizumab- jsgr)"~12/26/2024 0:00:00~"Original"~1~"4773-1"~"PMC"~"506B PMC"~4773.00~1~"PMC 4773-1: Conduct a clinical trial to further characterize the clinical effects of tislelizumab, including pharmacokinetics, activity, and safety assessments in U.S racial and ethnic minority patients with gastroesophageal cancers. The trial should enroll a sufficiently representative study population to reflect the racial and ethnic diversity of the U.S. patient population with HER2-negative, unresectable or metastatic gastroesophageal cancer and support an evaluation of comparative efficacy and safety between racial and ethnic minorities (including Black/African American and Hispanic/Latino populations) underrepresented in trial RATIONALE 305."~"Pending"~~6/30/2032 0:00:00~
384993~"CDER"~"BLA"~761417.00~"BeiGene USA, Inc."~"Tevimbra (tislelizumab- jsgr)"~12/26/2024 0:00:00~"Original"~1~"4773-2"~"PMC"~"506B PMC"~4773.00~2~"PMC 4773-2: Conduct an appropriate analytical and clinical validation study, using clinical trial data from RATIONALE-305, to support the availability of an in vitro diagnostic device to detect PD-L1 expression that is essential for the safe and effective use of tislelizumab in patients with HER2-negative gastric/gastroesophageal junction adenocarcinoma."~"Submitted"~~5/31/2026 0:00:00~
384994~"CDER"~"BLA"~761420.00~"CELLTRION, Inc."~"Avtozma (tocilizumab-anoh) injection"~1/24/2025 0:00:00~"Original"~1~"4790-1"~"PMR"~"Pediatric Research Equity Act"~4790.00~1~"PMR 4790-1: Assessment of Avtozma (tocilizumab-anoh) for the treatment of
COVID-19 in pediatric patients 1 year to 18 years of age."~"Submitted"~"The final report was submitted to FDA on 3/30/2026."~6/30/2026 0:00:00~
384995~"CDER"~"BLA"~761427.00~"LIB Therapeutics, Inc."~"Lerochol (lerodalcibep-liga) Injection"~12/12/2025 0:00:00~"Original"~1~"4928-1"~"PMR"~"Pediatric Research Equity Act"~4928.00~1~"PMR 4928-1: Conduct a double-blind, placebo-controlled, parallel group design study followed by an open-label extension to assess the efficacy and safety of lerodalcibep in pediatric patients with heterozygous familial hypercholesterolemia (HeFH) aged 6 years to less than 18 years."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~10/31/2028 0:00:00~
384996~"CDER"~"BLA"~761430.00~"Janssen Biotech, Inc."~"Imaavy (nipocalimab-aahu) injection"~4/29/2025 0:00:00~"Original"~1~"4833-1"~"PMR"~"505 (o)(3)"~4833.00~1~"PMR 4833-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Imaavy (nipocalimab)
during pregnancy and lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol. The protocol must be agreed upon with the Agency."~"Pending"~~10/31/2036 0:00:00~6/22/2026 0:00:00
384997~"CDER"~"BLA"~761430.00~"Janssen Biotech, Inc."~"Imaavy (nipocalimab-aahu) injection"~4/29/2025 0:00:00~"Original"~1~"4833-2"~"PMR"~"505 (o)(3)"~4833.00~2~"PMR 4833-2: Perform a lactation study (milk only or mother/infant pair study) in lactating women who have received therapeutic doses of Imaavy
using a validated assay to assess concentrations of nipocalimab in breast milk and to assess the effects on the breastfed infant, if
available, based on study population."~"Pending"~~10/31/2027 0:00:00~6/22/2026 0:00:00
384998~"CDER"~"BLA"~761432.00~"Merck Sharp & Dohme LLC"~"Enflonsia (clesrovimab-cfor) injection"~6/9/2025 0:00:00~"Original"~1~"4841-1"~"PMR"~"Pediatric Research Equity Act"~4841.00~1~"PMR 4841-1: Conduct a study in children up to 24 months of age with underlying conditions who are at increased risk for respiratory syncytial virus (RSV) disease."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~8/31/2026 0:00:00~
384999~"CDER"~"BLA"~761432.00~"Merck Sharp & Dohme LLC"~"Enflonsia (clesrovimab-cfor) injection"~6/9/2025 0:00:00~"Original"~1~"4841-2"~"PMR"~"505 (o)(3)"~4841.00~2~"PMR 4841-2: Conduct a surveillance study of current and emerging respiratory syncytial virus (RSV) variants from global locations, with F protein sequencing and identification of clesrovimab binding site substitutions and their frequency. These surveillance activities should include active collection and characterization of RSV samples from global regions (i.e., North America [US/Canada], Europe, rest of the world) and will target at least 100 samples from each region when fully operational, as well as periodic analysis of sequences from public databases (i.e., GISAID, NCBI, GenBank). The surveillance study should also determine the cell culture neutralization activity of clesrovimab against those RSV clesrovimab binding epitope variants carrying substitutions (VAF >10%) and with unknown impact on clesrovimab susceptibility that are capable of growing in cell culture. Phenotypic characterization will include RSV variants whose prevalence is =5% within an RSV season (or over two consecutive years in public databases) and/or =3-fold increase above 1% from the previous season across all sequenced samples and from all sites within a global region. RSV variants of interest for phenotypic testing will include those carrying substitutions of unknown impact on clesrovimab susceptibility, detected in Site IV, adjacent to the clesrovimab binding epitope (within =5   distance), or outside Site IV at highly conserved positions (=99% in GenBank)."~"Pending"~~7/31/2034 0:00:00~
385000~"CDER"~"BLA"~761432.00~"Merck Sharp & Dohme LLC"~"Enflonsia (clesrovimab-cfor) injection"~6/9/2025 0:00:00~"Original"~1~"4841-3"~"PMR"~"505 (o)(3)"~4841.00~3~"PMR 4841-3: Conduct a study to assess F protein substitutions in cell culture neutralization assays, in the background subtype in which they were identified based on non-clinical, surveillance, and clinical studies of clesrovimab. The list of substitutions is provided below:
RSV A: N380S, N426H, N428D, R429H, S436F, V447M, Y457H, K465R, S466N, K470R Also: test S443T if RSV B S443L shows reduced susceptibility to clesrovimab, and test K445R if RSV B K445N shows reduced susceptibility. RSV B: I402V, K433R, F435S, V442M, S443L, K445N, G446V, V452E Also: test V447I if RSV A V447M shows reduced susceptibility to clesrovimab."~"Pending"~~7/31/2029 0:00:00~
385001~"CDER"~"BLA"~761432.00~"Merck Sharp & Dohme LLC"~"Enflonsia (clesrovimab-cfor) injection"~6/9/2025 0:00:00~"Original"~1~"4841-4"~"PMC"~"506B PMC"~4841.00~4~"PMC 4841-4: Conduct a study to assess the cell culture neutralization of clesrovimab against RSV with substitutions that confer >100-fold reduction in susceptibility to nirsevimab, as reported in the BEYFORTUS  USPI: RSV A: N67I+N208Y RSV B: K68N+N201S, K68N+N208S, L203I, N208D"~"Pending"~~8/31/2027 0:00:00~
385002~"CDER"~"BLA"~761434.00~"Otsuka Pharmaceutical Company, Ltd."~"Voyxact (sibeprenlimab-szsi) Injection"~11/25/2025 0:00:00~"Original"~1~"4907-1"~"PMR"~"Accelerated Approval"~4907.00~1~"PMR 4907-1: Conduct a randomized, double-blind, placebo-controlled trial to describe and verify the clinical benefit of Voyxact for the treatment of primary IgA nephropathy. The trial should be adequately powered and of sufficient duration to detect a treatment effect on the endpoint that will be used to describe and verify the clinical benefit."~"Ongoing"~"The trial is underway."~4/30/2027 0:00:00~
385003~"CDER"~"BLA"~761434.00~"Otsuka Pharmaceutical Company, Ltd."~"Voyxact (sibeprenlimab-szsi) Injection"~11/25/2025 0:00:00~"Original"~1~"4907-2"~"PMR"~"Pediatric Research Equity Act"~4907.00~2~"PMR 4907-2: Conduct a pharmacodynamic, pharmacokinetic, safety and tolerability study of Voyxact in pediatric patients 2 years to 17 years of age with primary immunoglobulin A nephropathy. The pharmacodynamic assessment should be based on effects on proteinuria."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~12/31/2031 0:00:00~
385004~"CDER"~"BLA"~761434.00~"Otsuka Pharmaceutical Company, Ltd."~"Voyxact (sibeprenlimab-szsi) Injection"~11/25/2025 0:00:00~"Original"~1~"4907-3"~"PMR"~"505 (o)(3)"~4907.00~3~"PMR 4907-3: Perform a lactation study (milk only or mother-infant pair study) in lactating women who have received Voyxact to measure concentrations of sibeprenlimab-szsi and its major metabolites in breast milk using a validated assay. Assess the effects on the breastfed infant, if available, based on study population."~"Pending"~~10/31/2030 0:00:00~
385005~"CDER"~"BLA"~761434.00~"Otsuka Pharmaceutical Company, Ltd."~"Voyxact (sibeprenlimab-szsi) Injection"~11/25/2025 0:00:00~"Original"~1~"4907-4"~"PMR"~"505 (o)(3)"~4907.00~4~"PMR 4907-4: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Voyxact during pregnancy to assess risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant. Assess infant outcomes through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Pending"~~12/31/2036 0:00:00~
385006~"CDER"~"BLA"~761440.00~"GlaxoSmithKline LLC"~"Blenrep (belantamab mafodotin-blmf) for injection"~10/23/2025 0:00:00~"Original"~1~"4910-1"~"PMR"~"505 (o)(3)"~4910.00~1~"PMR 4910-1: Conduct a randomized clinical trial to further characterize the serious risk of ocular toxicity using alternative dosage regimens (e.g., lower doses and/or longer dosing intervals) of belantamab mafodotin in combination with bortezomib and dexamethasone in patients with relapsed or refractory multiple myeloma who have received at least 2 prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent. The primary objective of the trial should be to assess the ocular toxicity in all treatment arms, with efficacy measures and PK evaluations as secondary endpoints. The trial should enroll a sufficiently representative study population to allow for generalizability of the results to the U.S. patient population with multiple myeloma."~"Pending"~~6/30/2029 0:00:00~
385007~"CDER"~"BLA"~761440.00~"GlaxoSmithKline LLC"~"Blenrep (belantamab mafodotin-blmf) for injection"~10/23/2025 0:00:00~"Original"~1~"4910-2"~"PMR"~"505 (o)(3)"~4910.00~2~"PMR 4910-2: Conduct a pharmacokinetic trial to evaluate the serious potential risk of increased adverse reactions such as corneal events and severe thrombocytopenia, and to determine the appropriate dose of belantamab mafodotin in patients with moderate hepatic impairment compared to patients with normal hepatic function. This trial should be designed and conducted in accordance with the FDA Guidance for Industry entitled,  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling "~"Pending"~~12/31/2031 0:00:00~
385008~"CDER"~"BLA"~761451.00~"AstraZeneca AB"~"Saphnelo (anifrolumab-fnia) Injection and Saphnelo Pen (anifrolumab-fnia) injection"~4/24/2026 0:00:00~"Original"~1~"4980-1"~"PMR"~"Pediatric Research Equity Act"~4980.00~1~"PMR 4980-1: Conduct a study to evaluate the pharmacokinetics, pharmacodynamics, and safety of subcutaneous (SC) anifrolumab-fnia in children aged 5 to <18 years with moderate to severe systemic lupus erythematosus (SLE) despite standard of care."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~3/31/2031 0:00:00~
385009~"CDER"~"BLA"~761458.00~"GlaxoSmithKline LLC"~"Exdensur (depemokimab-ulaa) injection"~12/16/2025 0:00:00~"Original"~1~"4920-1"~"PMR"~"Pediatric Research Equity Act"~4920.00~1~"PMR 4920-1: Conduct a 52-week, open-label, pharmacokinetic, pharmacodynamic, and safety study of depemokimab in pediatric patients 6 years to 11 years of age with severe asthma characterized by an eosinophilic phenotype."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~12/31/2029 0:00:00~
385010~"CDER"~"BLA"~761458.00~"GlaxoSmithKline LLC"~"Exdensur (depemokimab-ulaa) injection"~12/16/2025 0:00:00~"Original"~1~"4920-2"~"PMR"~"505 (o)(3)"~4920.00~2~"PMR 4920-2: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to depemokimab during
pregnancy to assess risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant.
Assess infant outcomes through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Pending"~~6/30/2037 0:00:00~
385011~"CDER"~"BLA"~761458.00~"GlaxoSmithKline LLC"~"Exdensur (depemokimab-ulaa) injection"~12/16/2025 0:00:00~"Original"~1~"4920-3"~"PMR"~"505 (o)(3)"~4920.00~3~"PMR 4920-3: Perform a lactation study (milk-only or mother-infant pair study) in lactating women who have received depemokimab to measure
concentrations of depemokimab and its major metabolites in breast milk using a validated assay. Assess the effects on the breastfed infant, if available, based on study population."~"Pending"~~6/30/2029 0:00:00~
385012~"CDER"~"BLA"~761464.00~"Daiichi Sankyo, Inc."~"Datroway (datopotamab deruxtecan-dlnk) Injection"~6/23/2025 0:00:00~"Original"~1~"4860-1"~"PMR"~"Accelerated Approval"~4860.00~1~"PMR 4860-1: Conduct a multicenter, randomized clinical trial, TROPION-LUNG15, intended to verify and describe the clinical benefit of datopotamab
deruxtecan in patients with EGFR-mutated non-small cell lung cancer who have had disease progression after osimertinib."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed"~11/30/2029 0:00:00~
385013~"CDER"~"BLA"~761464.00~"Daiichi Sankyo, Inc."~"Datroway (datopotamab deruxtecan-dlnk) Injection"~6/23/2025 0:00:00~"Original"~1~"4860-2"~"PMC"~"506B PMC"~4860.00~2~"PMC 4860-2: Complete ICDIM 521 and ICSH 22-038 anti-drug antibody (ADA) assay validation and include report addendums containing high resolution drug tolerance data and justification to support that the ADA assays are fit-forpurpose to characterize the effects of ADA on pharmacokinetics, pharmacodynamics, safety, and effectiveness of datopotamab deruxtecan."~"Submitted"~~8/31/2025 0:00:00~
385014~"CDER"~"BLA"~761315.00~"Novo Nordisk Inc."~"Alhemo (concizumab-mtci) Injection"~12/20/2024 0:00:00~"Original"~1~"4761-1"~"PMC"~"506B PMC"~4761.00~1~"PMC 4761-1: Conduct adequate analytical and clinical validation testing to establish an FDA cleared or approved in-vitro diagnostic device to accurately and reliably detect concizumab concentration in human 3.2% citrated plasma that is safe and effective for concizumab dose adjustment decisions 4 weeks after initiation of treatment with concizumab in patients with Hemophilia A and B with inhibitors. The final report should include the results of the validation studies to inform product labeling."~"Fulfilled"~~3/31/2025 0:00:00~
385015~"CDER"~"NDA"~213953.00~"MARIUS PHARMACEUTICALS INC"~"Kyzatrex (testosterone undecanoate)"~7/27/2022 0:00:00~"Original"~1~"4313-2"~"PMR"~"505 (o)(3)"~4313.00~2~"PMR 4313-2: An appropriately designed label comprehension study that assesses patients  understanding of key risk messages in the Medication Guide for testosterone replacement therapy. The primary objective of this study is to assess patient comprehension of materials related to increases in blood pressure that can increase the risk of major adverse cardiovascular events with testosterone replacement therapy. Include men representative of those who use prescription testosterone therapy with a range of cardiac risk factors, a range of education levels, and various literacy levels. The study findings may result in revisions to the Medication Guide to optimize patients  understanding of important risks of testosterone replacement therapy."~"Fulfilled"~~3/31/2024 0:00:00~10/17/2025 0:00:00
385016~"CDER"~"NDA"~210166.00~"TAKEDA PHARMACEUTICALS USA INC"~"Motegrity (Prucalopride Succinate)"~12/14/2018 0:00:00~"Original"~1~"3529-1"~"PMR"~"Pediatric Research Equity Act"~3529.00~1~"PMR 3529-1: Evaluate the pharmacokinetics, efficacy, and safety of Motegrity (prucalopride) in pediatric patients with chronic idiopathic constipation (CIC) who are 6 months to less than 18 years of age by performing a randomized, double-blind, placebocontrolled, parallel group, 12-week treatment study."~"Fulfilled"~"Per FDA letter dated 07/10/2025, this PMR/PMC has been fulfilled."~6/30/2025 0:00:00~2/6/2026 0:00:00
385017~"CDER"~"NDA"~210166.00~"TAKEDA PHARMACEUTICALS USA INC"~"Motegrity (Prucalopride Succinate)"~12/14/2018 0:00:00~"Original"~1~"3529-6"~"PMR"~"Pediatric Research Equity Act"~3529.00~6~"PMR 3529-6: Assess the long-term safety of Motegrity (prucalopride) in pediatric patients with chronic idiopathic constipation (CIC) who are 6 months to less than 18 years of age and have completed a confirmatory efficacy and safety study with Motegrity (prucalopride) by performing a safety and tolerability study comparing two doses in a blinded design."~"Fulfilled"~"Per FDA letter dated 07/10/2025, this PMR/PMC has been fulfilled."~6/30/2025 0:00:00~2/6/2026 0:00:00
385018~"CDER"~"NDA"~213388.00~"ABBVIE INC"~"Oriahnn (elagolix, estradiol and norethindrone acetate)"~5/29/2020 0:00:00~"Original"~1~"3837-1"~"PMR"~"505 (o)(3)"~3837.00~1~"PMR 3837-1: A prospective pregnancy registry to evaluate the adverse effects of elagolix-containing products, including Oriahnn, on pregnancy and maternal and fetal/neonatal outcomes."~"Released"~~1/31/2030 0:00:00~7/23/2025 0:00:00
385019~"CDER"~"NDA"~210450.00~"ABBVIE INC"~"Orilissa (Elagolix Sodium)"~7/23/2018 0:00:00~"Original"~1~"3390-1"~"PMR"~"505 (o)(3)"~3390.00~1~"PMR 3390-1: A prospective pregnancy registry to evaluate the effects of Orilissa on pregnancy and maternal and fetal/neonatal outcomes."~"Released"~~1/31/2030 0:00:00~9/17/2025 0:00:00
385020~"CDER"~"NDA"~216718.00~"BIOGEN US CORPORATION"~"Skyclarys (omaveloxolone)"~2/28/2023 0:00:00~"Original"~1~"4410-6"~"PMR"~"505 (o)(3)"~4410.00~6~"PMR 4410-6: Conduct a 26-week toxicity study of metabolite M22 in rat."~"Fulfilled"~~7/31/2024 0:00:00~4/27/2026 0:00:00
385021~"CDER"~"BLA"~761034.00~"Genentech, Inc."~"Tecentriq (Atezolizumab) "~5/18/2016 0:00:00~"Supplement"~42~"4162-1"~"PMC"~"506B PMC"~4162.00~1~"PMC 4162-1: Conduct clinical trial IMpower010 titled,  A Phase 3, Open-Label, Randomized Study to Investigate the Efficacy and Safety of Atezolizumab (Anit-PD-L1 antibody) Compared with Best Supportive Care Following Adjuvant Cisplatin-Based Chemotherapy in Patients with Completely Resected Stage IB-IIIA Non-Small Cell Lung Cancer , and submit results for the final analysis of disease-free survival (DFS) in the intent-to-treat population, as well as updated analyses of DFS in the first two analysis populations in the testing hierarchy, updated analyses of overall survival in all three analysis populations and exploratory analyses of these endpoints in subgroups of clinical interest (e.g., subgroups based on PD-L1 status), to further characterize the clinical benefit of atezolizumab as a single agent as adjuvant treatment following resection and platinum-based chemotherapy for adult patients with non-small cell lung cancer."~"Fulfilled"~~12/31/2022 0:00:00~7/11/2025 0:00:00
385022~"CDER"~"NDA"~206940.00~"ABBVIE INC"~"Viberzi (Eluxadoline)"~5/27/2015 0:00:00~"Original"~1~"2901-10"~"PMR"~"Pediatric Research Equity Act"~2901.00~10~"PMR 2901-10: Complete the ongoing phase 2 dose-ranging study to evaluate pharmacokinetics (PK), safety, and efficacy of Viberzi (eluxadoline) in pediatric patients 12 to 17 years of age with diarrhea-predominant irritable bowel syndrome (IBS-D)."~"Fulfilled"~"Per FDA letter dated 07/07/2025, this PMR/PMC has been fulfilled."~7/31/2024 0:00:00~7/22/2025 0:00:00
385023~"CDER"~"NDA"~208447.00~"GLAXOSMITHKLINE LLC"~"ZEJULA (niraparib)"~3/27/2017 0:00:00~"Supplement"~25~"4379-1"~"PMC"~"506B PMC"~4379.00~1~"PMC 4379-1: Support the availability, through appropriate analytical and clinical validation studies using the NOVA clinical trial or other data adequate to support labeling, of an in vitro diagnostic device that is essential to the safe and effective use of niraparib for the maintenance treatment of adult patients with deleterious or suspected deleterious germline BRCAmutated recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy."~"Fulfilled"~~2/28/2023 0:00:00~5/21/2020 0:00:00
385024~"CDER"~"NDA"~211371.00~"SAGE THERAPEUTICS LLC A WHOLLY OWNED SUB OF SUPERNUS PHARMACEUTICALS INC"~"Zulresso (brexanolone)"~3/19/2019 0:00:00~"Original"~1~"3535-3"~"PMC"~"506B PMC"~3535.00~3~"PMC 3535-3: Conduct a study to evaluate the efficacy of a lower dose of brexanolone."~"Released"~~1/31/2023 0:00:00~5/12/2023 0:00:00
385025~"CDER"~"NDA"~211371.00~"SAGE THERAPEUTICS LLC A WHOLLY OWNED SUB OF SUPERNUS PHARMACEUTICALS INC"~"Zulresso (brexanolone)"~3/19/2019 0:00:00~"Original"~1~"3535-4"~"PMC"~"506B PMC"~3535.00~4~"PMC 3535-4: Evaluate outpatient daytime dosing allowing for administration of brexanolone at a non-24 hour facility."~"Released"~~6/30/2025 0:00:00~5/12/2023 0:00:00
385026~"CDER"~"NDA"~218275.00~"ISTX LLC"~"Zevtera (ceftobiprole medocaril sodium)"~4/3/2024 0:00:00~"Original"~1~"4612-2"~"PMR"~"Pediatric Research Equity Act"~4612.00~2~"PMR 4612-2: Conduct a randomized, controlled study to evaluate the safety, tolerability, and efficacy of ceftobiprole medocaril sodium for injection in pediatric patients from birth to less than 18 years of age with Staphylococcus aureus bacteremia (SAB)."~"Pending"~"Draft protocol submitted 4-2025"~4/30/2029 0:00:00~5/29/2026 0:00:00
385027~"CDER"~"NDA"~218275.00~"ISTX LLC"~"Zevtera (ceftobiprole medocaril sodium)"~4/3/2024 0:00:00~"Original"~1~"4612-3"~"PMR"~"505 (o)(3)"~4612.00~3~"PMR 4612-3: Conduct an in vivo drug interaction study evaluating ceftobiprole medocaril sodium for injection as an inhibitor of OATP1B1 and OATB1B3 transporters in healthy subjects."~"Ongoing"~~12/31/2025 0:00:00~5/29/2026 0:00:00
385028~"CDER"~"NDA"~218275.00~"ISTX LLC"~"Zevtera (ceftobiprole medocaril sodium)"~4/3/2024 0:00:00~"Original"~1~"4612-4"~"PMR"~"505 (o)(3)"~4612.00~4~"PMR 4612-4: Conduct a US surveillance study for a five-year period after the introduction of ceftobiprole medocaril sodium for injection to the market to determine if bacterial resistance to ceftobiprole has developed in those organisms specific to the indications in the labeling."~"Ongoing"~~3/31/2031 0:00:00~5/29/2026 0:00:00
385029~"CDER"~"NDA"~218276.00~"NOVARTIS PHARMACEUTICALS CORP"~"Fabhalta (iptacopan)"~12/5/2023 0:00:00~"Original"~1~"4553-1"~"PMR"~"505 (o)(3)"~4553.00~1~"PMR 4553-1: Provide data from a registry that characterizes the long-term safety of Fabhalta in adults with paroxysmal nocturnal hemoglobinuria (PNH), with up to 5 years of follow-up."~"Ongoing"~~7/31/2030 0:00:00~1/29/2026 0:00:00
385030~"CDER"~"NDA"~218276.00~"NOVARTIS PHARMACEUTICALS CORP"~"Fabhalta (iptacopan)"~12/5/2023 0:00:00~"Original"~1~"4553-4"~"PMR"~"505 (o)(3)"~4553.00~4~"PMR 4553-4: Complete Study CLNP023C12001B PNH REP: An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have completed PNH phase 2 and phase 3 studies with iptacopan."~"Ongoing"~~5/31/2029 0:00:00~1/29/2026 0:00:00
385031~"CDER"~"NDA"~218276.00~"NOVARTIS PHARMACEUTICALS CORP"~"Fabhalta (iptacopan)"~12/5/2023 0:00:00~"Supplement"~1~"4673-1"~"PMR"~"Accelerated Approval"~4673.00~1~"PMR 4673-1: Conduct a randomized, double-blind, placebo-controlled trial to verify and describe the clinical benefit of iptacopan for the treatment of primary IgA nephropathy. The trial should be adequately powered and of sufficient duration to detect a treatment effect on the endpoint that will be used to verify and describe the clinical benefit."~"Ongoing"~"The trial has completed enrollment."~7/31/2026 0:00:00~1/29/2026 0:00:00
385032~"CDER"~"NDA"~218276.00~"NOVARTIS PHARMACEUTICALS CORP"~"Fabhalta (iptacopan)"~12/5/2023 0:00:00~"Supplement"~1~"4673-2"~"PMR"~"Pediatric Research Equity Act"~4673.00~2~"PMR 4673-2: Conduct an efficacy, pharmacokinetic, safety and tolerability study in pediatric patients 2 years to less than 18 years of age with primary IgA nephropathy. The efficacy outcome should be based on effects on proteinuria."~"Released"~"Per FDA letter dated 01/12/2026, this PMR/PMC has been released."~12/31/2030 0:00:00~1/29/2026 0:00:00
385033~"CDER"~"NDA"~218275.00~"ISTX LLC"~"Zevtera (ceftobiprole medocaril sodium)"~4/3/2024 0:00:00~"Original"~1~"4612-1"~"PMR"~"Pediatric Research Equity Act"~4612.00~1~"PMR 4612-1: Conduct a randomized, controlled study to evaluate the safety, tolerability, and efficacy of ceftobiprole medocaril sodium for injection in pediatric patients from birth to less than 18 years of age with acute bacterial skin and skin structure infections (ABSSSI) requiring intravenous antibacterial drugs."~"Pending"~"Final protocol submitted 2-2025."~4/30/2028 0:00:00~5/29/2026 0:00:00
385034~"CDER"~"NDA"~218276.00~"NOVARTIS PHARMACEUTICALS CORP"~"Fabhalta (iptacopan)"~12/5/2023 0:00:00~"Supplement"~1~"4673-3"~"PMR"~"Pediatric Research Equity Act"~4673.00~3~"PMR 4673-3: ."~"Ongoing"~"No subjects have been enrolled; there are subjects being screened."~12/31/2030 0:00:00~1/29/2026 0:00:00
385035~"CDER"~"NDA"~218316.00~"NOVO NORDISK INC"~"Wegovy (semaglutide) tablets"~12/22/2025 0:00:00~"Original"~1~"4954-1"~"PMR"~"Pediatric Research Equity Act"~4954.00~1~"PMR 4954-1: Conduct population pharmacokinetic (PK) and exposure-response analyses to evaluate the efficacy and safety of oral semaglutide (25 mg tablet) for the treatment of long-term weight reduction in pediatric patients with obesity ages 12 to less than 18 years."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~1/31/2027 0:00:00~
385036~"CDER"~"NDA"~218316.00~"NOVO NORDISK INC"~"Wegovy (semaglutide) tablets"~12/22/2025 0:00:00~"Original"~1~"4954-2"~"PMR"~"Pediatric Research Equity Act"~4954.00~2~"PMR 4954-2: Conduct population pharmacokinetic (PK) and exposure-response analyses to evaluate the efficacy and safety of semaglutide to support the 25 mg tablet for the treatment of long-term weight reduction in pediatric patients with obesity ages 6 to less than 12 years."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~1/31/2028 0:00:00~
385037~"CDER"~"NDA"~218316.00~"NOVO NORDISK INC"~"Wegovy (semaglutide) tablets"~12/22/2025 0:00:00~"Original"~1~"4954-3"~"PMR"~"505 (o)(3)"~4954.00~3~"PMR 4954-3: Conduct a prospective, registry-based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to semaglutide during pregnancy to an unexposed reference population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Pending"~~8/31/2035 0:00:00~
385038~"CDER"~"NDA"~218316.00~"NOVO NORDISK INC"~"Wegovy (semaglutide) tablets"~12/22/2025 0:00:00~"Original"~1~"4954-4"~"PMR"~"505 (o)(3)"~4954.00~4~"PMR 4954-4: Conduct an additional pregnancy study that uses a different observational design from the Pregnancy Exposure Registry, using claims or electronic medical record data, to assess the associations between semaglutide exposure during pregnancy with pregnancy outcomes and infant outcomes, including but not limited to major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age, preterm birth and postnatal growth and development."~"Pending"~~8/31/2035 0:00:00~
385039~"CDER"~"NDA"~218316.00~"NOVO NORDISK INC"~"Wegovy (semaglutide) tablets"~12/22/2025 0:00:00~"Original"~1~"4954-5"~"PMR"~"505 (o)(3)"~4954.00~5~"PMR 4954-5: Conduct a medullary thyroid carcinoma registry-based case series of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of semaglutide tablet for the treatment of obesity into the marketplace. This study will also establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to the use of semaglutide for the treatment of obesity/overweight."~"Pending"~~2/28/2042 0:00:00~
385040~"CDER"~"NDA"~218316.00~"NOVO NORDISK INC"~"Wegovy (semaglutide) tablets"~12/22/2025 0:00:00~"Original"~1~"4954-6"~"PMR"~"505 (o)(3)"~4954.00~6~"PMR 4954-6: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of semaglutide 25 mg tablets and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Pending"~~11/30/2026 0:00:00~
385041~"CDER"~"BLA"~761258.00~"Akeso Biopharma Co., Ltd. "~"penpulimab-kcqx"~4/23/2025 0:00:00~"Original"~1~"4815-2"~"PMC"~"506B PMC"~4815.00~2~"PMC 4815-2: Conduct a clinical trial enrolling an adequate number of patients in the United States (U.S.), that includes a sufficient representation of patients in demographic subgroups that are reflective of the U.S. patient population with nasopharyngeal carcinoma (NPC), to further characterize the efficacy and safety of penpulimab in combination with gemcitabine and either cisplatin or carboplatin in these patients. Conduct sparse sampling for supportive population pharmacokinetic and Exposure-Response (E-R) analyses. The E-R analyses may be used as supportive evidence for the efficacy and safety in the intended patient population. In the E-R analyses report, include an analysis of the presence and clinical impact of the neutralizing anti-drug antibodies on pharmacokinetics, efficacy, and safety of penpulimab."~"Pending"~~12/31/2029 0:00:00~6/18/2026 0:00:00
385042~"CDER"~"BLA"~761261.00~"Genzyme Corporation"~"Xenpozyme (olipudase alfa-rpcp)"~8/31/2022 0:00:00~"Original"~1~"4291-1"~"PMR"~"505 (o)(3)"~4291.00~1~"PMR 4291-1: Conduct a five-year observational study to evaluate the long-term safety of olipudase alfa-rpcp, including severe hypersensitivity reactions, infusion-associated reactions, and laboratory abnormalities in pediatric patients less than two years of age with ASMD and patients with ASMD Type A. Assess anti-olipudase alfarpcp antibody responses and evaluate the relationship between anti-olipudase alfa-rpcp antibodies and safety."~"Ongoing"~~4/30/2030 0:00:00~10/29/2025 0:00:00
385043~"CDER"~"BLA"~761262.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~6/16/2022 0:00:00~"Original"~1~"4294-1"~"PMR"~"505 (o)(3)"~4294.00~1~"PMR 4294-1: Conduct an observational study to assess the incidence of severe acute liver injury in adults with moderately to severely active Crohn's disease who are exposed to Skyrizi (risankizumab-rzaa), relative to other therapies used to treat Crohn's disease. Compare rates (per person-time) or risks (proportion of patients with a minimum amount of follow-up). Describe and justify the choice of appropriate comparator population(s). Specify concise case definition for severe liver injury and validation of algorithm(s) to identify severe liver injury in the proposed data source. For the Skyrizi (risankizumab-rzaa)-exposed and comparator(s) cohorts, clearly define the study drug initiation period and any exclusion and inclusion criteria. Ensure that the data source allows for average follow-up for at least 1 year. Specify a minimum sample size and justify the precision of the estimate achievable with the proposed study."~"Delayed"~"The final protocol was submitted on 10/13/2023, which was after the original milestone, and acknowledged on 11/6/2023."~12/31/2033 0:00:00~6/17/2026 0:00:00
385044~"CDER"~"BLA"~761262.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~6/16/2022 0:00:00~"Original"~1~"4294-2"~"PMR"~"505 (o)(3)"~4294.00~2~"PMR 4294-2: A prospective, registry-based, observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to risankizumab-containing products regardless of indication during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age births, preterm births, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, neonatal deaths, and infections, will be assessed through at least the first year of life."~"Ongoing"~~6/30/2034 0:00:00~6/17/2026 0:00:00
385045~"CDER"~"BLA"~761262.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~6/16/2022 0:00:00~"Original"~1~"4294-3"~"PMR"~"505 (o)(3)"~4294.00~3~"PMR 4294-3: Conduct an additional pregnancy study that uses a different design from the prospective pregnancy registry established to fulfil postmarketing requirement study 2 (for example a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm births in women exposed to risankizumab-containing products regardless of indication during pregnancy compared to an unexposed control population.
"~"Delayed"~"The final protocol was submitted after the original milestone and acknowledged in a letter dated 7/11/2024."~6/30/2033 0:00:00~6/17/2026 0:00:00
385046~"CDER"~"BLA"~761262.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~6/16/2022 0:00:00~"Original"~1~"4294-4"~"PMR"~"505 (o)(3)"~4294.00~4~"PMR 4294-4: Perform a lactation trial (milk only) in lactating women who have received Skyrizi (risankizumab-rzaa) to assess concentrations of Skyrizi (risankizumab-rzaa) in breast milk using a validated assay and to assess the effects on the breastfed infant.
"~"Ongoing"~~6/30/2026 0:00:00~6/17/2026 0:00:00
385047~"CDER"~"BLA"~761262.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~6/16/2022 0:00:00~"Original"~1~"4294-5"~"PMC"~"506B PMC"~4294.00~5~"PMC 4294-5: Conduct a one-year, randomized trial to evaluate the safety, efficacy, and pharmacokinetics of Skyrizi (risankizumab-rzaa) in pediatric patients 2 to 17 years of age with moderately to severely active Crohn s disease.
"~"Ongoing"~~12/31/2029 0:00:00~6/17/2026 0:00:00
385048~"CDER"~"BLA"~761262.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~6/16/2022 0:00:00~"Original"~1~"4294-6"~"PMC"~"506B PMC"~4294.00~6~"PMC 4294-6: Conduct a long-term extension study to evaluate the long-term safety of Skyrizi (risankizumab-rzaa) in pediatric patients 2 to 17 years of age with moderately to severely active Crohn s disease who participated in postmarketing commitment study 4294-5. This study can be conducted as part of postmarketing commitment study 4294-5."~"Ongoing"~~9/30/2033 0:00:00~6/17/2026 0:00:00
385049~"CDER"~"BLA"~761262.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~6/16/2022 0:00:00~"Supplement"~7~"4656-1"~"PMR"~"Pediatric Research Equity Act"~4656.00~1~"PMR 4656-1: Conduct a one-year, randomized trial to evaluate the safety, efficacy, and pharmacokinetics of Skyrizi (risankizumab-rzaa) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2031 0:00:00~6/17/2026 0:00:00
385050~"CDER"~"BLA"~761262.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~6/16/2022 0:00:00~"Supplement"~7~"4656-2"~"PMR"~"Pediatric Research Equity Act"~4656.00~2~"PMR 4656-2: Conduct a long-term extension study to evaluate the long-term safety of Skyrizi (risankizumab-rzaa) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis who participated in postmarketing requirement study #1. This study can be conducted as part of postmarketing requirement study #1 or as a basket trial with study PMC 4294-6."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2035 0:00:00~6/17/2026 0:00:00
385051~"CDER"~"NDA"~219209.00~"VERTEX PHARMACEUTICALS INC"~"Journavx (suzetrigine) tablet"~1/30/2025 0:00:00~"Original"~1~"4787-7"~"PMR"~"505 (o)(3)"~4787.00~7~"PMR 4787-7: Collect global data from prospective pregnancy exposure registry/registries, preferably disease-based multiproduct pregnancy registry/registries, using a registry-based cohort study design to compare the maternal, fetal, and infant outcomes of women exposed to Journavx during pregnancy with unexposed comparator population(s). Align the U.S. study protocol with protocol(s) outside the U.S. to reach the target sample size. The registry will identify and record pregnancy complications, spontaneous abortion, stillbirths, neonatal deaths, major and minor congenital malformations, pregnancy terminations, preterm births, smallfor-gestational-age births, and any other adverse outcomes, including postnatal growth and development. These outcomes should be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life"~"Delayed"~"The Final Protocol milestone was missed because of additional time needed to negotiate and finalize the protocols based on FDA s feedback. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 06/04/2026"~7/31/2036 0:00:00~3/27/2026 0:00:00
385052~"CDER"~"NDA"~219209.00~"VERTEX PHARMACEUTICALS INC"~"Journavx (suzetrigine) tablet"~1/30/2025 0:00:00~"Original"~1~"4787-8"~"PMR"~"505 (o)(3)"~4787.00~8~"PMR 4787-8: Conduct a retrospective pregnancy cohort study using claims or electronic health record data with medical chart validation that is adequately powered to assess spontaneous abortions, stillbirths, neonatal deaths, major congenital malformations, preterm births, and small-for-gestationalage births in individuals exposed to Journavx during pregnancy compared to appropriate comparator population(s)."~"Delayed"~"The final protocol milestone was missed because of additional time needed to negotiate with FDA. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 06/04/2026."~7/31/2036 0:00:00~3/27/2026 0:00:00
385053~"CDER"~"NDA"~219209.00~"VERTEX PHARMACEUTICALS INC"~"Journavx (suzetrigine) tablet"~1/30/2025 0:00:00~"Original"~1~"4787-9"~"PMR"~"505 (o)(3)"~4787.00~9~"PMR 4787-9: Perform a milk-only lactation study in lactating women who have received Journavx to measure concentrations of suzetrigine in breast milk using a validated assay. Assess the effects on the breastfed infant, if available, based on study population."~"Pending"~~7/31/2028 0:00:00~3/27/2026 0:00:00
385054~"CDER"~"NDA"~219249.00~"GENENTECH INC"~"Itovebi (inavolisib) tablets"~10/10/2024 0:00:00~"Original"~1~"4718-1"~"PMR"~"505 (o)(3)"~4718.00~1~"PMR 4718-1: Conduct a multicenter, randomized clinical trial to further characterize known serious risks with inavolisib including severe hyperglycemia, stomatitis/mucosal inflammation, and diarrhea, and compare the safety and activity of inavolisib 9 mg daily versus a lower daily dose in patients with PIK3CA-mutant, hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer."~"Pending"~~4/30/2029 0:00:00~12/4/2025 0:00:00
385055~"CDER"~"NDA"~219249.00~"GENENTECH INC"~"Itovebi (inavolisib) tablets"~10/10/2024 0:00:00~"Original"~1~"4718-2"~"PMR"~"505 (o)(3)"~4718.00~2~"PMR 4718-2: Complete the ongoing renal impairment clinical trial and evaluate the pharmacokinetics and safety of inavolisib in participants with normal renal function and patients with severe renal impairment, to evaluate the serious potential risk of increased drug exposure. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Renal Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Fulfilled"~~6/30/2025 0:00:00~12/4/2025 0:00:00
385056~"CDER"~"NDA"~219249.00~"GENENTECH INC"~"Itovebi (inavolisib) tablets"~10/10/2024 0:00:00~"Original"~1~"4718-3"~"PMR"~"505 (o)(3)"~4718.00~3~"PMR 4718-3: Conduct a hepatic impairment clinical trial to evaluate the serious potential risk of increased drug exposure and determine a safe and
appropriate dose of inavolisib in patients with moderate and severe hepatic impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~1/31/2029 0:00:00~12/4/2025 0:00:00
385057~"CDER"~"NDA"~219249.00~"GENENTECH INC"~"Itovebi (inavolisib) tablets"~10/10/2024 0:00:00~"Original"~1~"4718-4"~"PMC"~"506B PMC"~4718.00~4~"PMC 4718-4: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of inavolisib on the single dose pharmacokinetics of a sensitive CYP3A substrate to inform appropriate management strategies for clinically relevant drug interactions. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Pending"~~8/31/2027 0:00:00~12/4/2025 0:00:00
385058~"CDER"~"NDA"~219249.00~"GENENTECH INC"~"Itovebi (inavolisib) tablets"~10/10/2024 0:00:00~"Original"~1~"4718-5"~"PMC"~"506B PMC"~4718.00~5~"PMC 4718-5: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of inavolisib on the single dose pharmacokinetics of a CYP2B6 substrate to inform appropriate management strategies for clinically relevant drug interactions. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Pending"~~8/31/2027 0:00:00~12/4/2025 0:00:00
385059~"CDER"~"NDA"~219249.00~"GENENTECH INC"~"Itovebi (inavolisib) tablets"~10/10/2024 0:00:00~"Original"~1~"4718-6"~"PMC"~"506B PMC"~4718.00~6~"PMC 4718-6: Complete the ongoing clinical trial, INAVO120 (Study WO41554), entitled  A Phase III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of Inavolisib plus Palbociclib and Fulvestrant versus Placebo plus Palbociclib and Fulvestrant in Patients with PIK3CA-Mutant, Hormone Receptor-Positive, HER2- Negative Locally Advanced or Metastatic Breast Cancer , to provide the final overall survival (OS) analysis."~"Submitted"~~1/31/2026 0:00:00~12/4/2025 0:00:00
385060~"CDER"~"NDA"~219249.00~"GENENTECH INC"~"Itovebi (inavolisib) tablets"~10/10/2024 0:00:00~"Original"~1~"4718-7"~"PMC"~"506B PMC"~4718.00~7~"PMC 4718-7: Conduct an integrated analysis containing data from clinical trials and post-marketing reports, observational studies (e.g., real-world evidence), and other sources to further characterize the pharmacokinetics (PK), pharmacodynamics (PD), safety and efficacy of inavolisib in racial and ethnic minority patients and older patients age >65 years with PIK3CA-mutant, hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer. The analyses should support comparative safety and efficacy outcome analyses and characterization of potential differences in PK and PD between racial and ethnic minority
patients and White patients, and older and younger patients."~"Pending"~~6/30/2030 0:00:00~12/4/2025 0:00:00
385061~"CDER"~"NDA"~219249.00~"GENENTECH INC"~"Itovebi (inavolisib) tablets"~10/10/2024 0:00:00~"Original"~1~"4718-8"~"PMC"~"506B PMC"~4718.00~8~"PMC 4718-8: Conduct an appropriate analytical and clinical validation study using the INAVO120 clinical trial or other data adequate to support the development of an in vitro diagnostic device for tumor tissue that is essential to the safe and effective use of inavolisib for treatment of patients with PIK3CA-mutant, hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer."~"Submitted"~~12/31/2024 0:00:00~12/4/2025 0:00:00
385062~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Supplement"~21~"4696-1"~"PMR"~"Pediatric Research Equity Act"~4696.00~1~"PMR 4696-1: Conduct a one-year, randomized, open-label induction, double-blind maintenance, parallel-group, multicenter study to assess the pharmacokinetics, safety, and efficacy of Tremfya (guselkumab) in pediatric patients 2 to <18 years of age with moderately to severely active ulcerative colitis."~"Ongoing"~"The trial is underway.."~1/31/2029 0:00:00~9/5/2025 0:00:00
385063~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Supplement"~21~"4696-2"~"PMR"~"Pediatric Research Equity Act"~4696.00~2~"PMR 4696-2: Conduct a long-term extension study to evaluate the long-term safety of Tremfya (guselkumab) in patients 2 to <18 years of age with moderately to severely active ulcerative colitis who participated in postmarketing requirement 4696-1. This Study can be conducted as a part of postmarketing requirement 4696-1."~"Ongoing"~"The study has been initiated."~8/31/2030 0:00:00~9/5/2025 0:00:00
385064~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Supplement"~21~"4696-3"~"PMR"~"505 (o)(3)"~4696.00~3~"PMR 4696-3: Complete the treatment and evaluation of subjects enrolled in the ongoing CNTO1959UCO3001 (QUASAR), CNTO1959UCO3004 (ASTRO), CNTO1959CRD3001 (GALAXI), and CNTO1959CRD3004 (GRAVITI) studies. Follow subjects in these studies for 5 years unless a safety signal is identified that indicates the potential risks of such continued long-term treatment outweigh the benefits. Evaluation of subjects should continue through the end of the trial when achievable. Adverse events (including, but not limited to, malignancy, serious infection, tuberculosis, opportunistic infections, hypersensitivity reactions, autoimmune disease, neurologic or demyelinating disease, cardiovascular, gastrointestinal [including hepatic] or hematologic adverse events) will continue to be collected throughout the long-term extensions."~"Ongoing"~~3/31/2031 0:00:00~9/5/2025 0:00:00
385065~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Supplement"~21~"4696-4"~"PMR"~"505 (o)(3)"~4696.00~4~"PMR 4696-4: Collect data from prospective pregnancy exposure registry/registries, preferably disease-based multiproduct pregnancy registry/registries, using a registry-based cohort study design to compare the maternal, fetal, and infant outcomes of women with inflammatory bowel disease exposed to guselkumab during pregnancy with unexposed comparator population(s). Align the U.S. protocol with protocols(s) outside the U.S. to reach the target sample size, if applicable. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortion, stillbirths, pregnancy terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The final protocol milestone was missed due to continued
discussions with the Agency."~6/30/2031 0:00:00~9/5/2025 0:00:00
385066~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Supplement"~21~"4696-5"~"PMR"~"505 (o)(3)"~4696.00~5~"PMR 4696-5: Conduct a retrospective cohort study using claims or electronic medical record data or a case control study with confirmation based upon review of data from medical records to assess adverse pregnancy outcomes such as major congenital malformations, spontaneous abortions, stillbirths, small-for-gestational-age births, neonatal deaths, and infant infections in women exposed to guselkumab regardless of indication during pregnancy compared to an unexposed control population."~"Delayed"~"The final protocol milestone was missed due to continued
discussions with the Agency."~6/30/2031 0:00:00~9/5/2025 0:00:00
385067~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Supplement"~21~"4696-6"~"PMR"~"505 (o)(3)"~4696.00~6~"PMR 4696-6: Perform a lactation study (milk only) in lactating women who have received guselkumab, regardless of indication, to measure concentrations of guselkumab in breast milk using a validated assay."~"Pending"~~9/30/2028 0:00:00~9/5/2025 0:00:00
385068~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Supplement"~24~"4798-1"~"PMR"~"Pediatric Research Equity Act"~4798.00~1~"PMR 4798-1: Conduct a one-year, randomized, open-label induction, double-blind maintenance, parallel-group, multicenter study to assess the
pharmacokinetics, safety, and efficacy of Tremfya (guselkumab) in pediatric patients 2 to <18 years of age with moderately to severely active Crohn s disease."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~12/31/2029 0:00:00~9/5/2025 0:00:00
385069~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Supplement"~24~"4798-2"~"PMR"~"Pediatric Research Equity Act"~4798.00~2~"PMR 4798-2: Conduct a long-term extension study to evaluate the long-term safety of Tremfya (guselkumab) in patients 2 to <18 years of age with moderately to severely active Crohn s disease who participated in postmarketing requirement 4798-1. This study can be conducted as a part of postmarketing requirement 4798-1."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~8/31/2031 0:00:00~9/5/2025 0:00:00
385070~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Supplement"~24~"4798-3"~"PMR"~"505 (o)(3)"~4798.00~3~"PMR 4798-3: Conduct an observational study to assess the incidence of severe acute liver injury in in adults with inflammatory bowel disease who are exposed to Tremfya (guselkumab), relative to other therapies used to treat inflammatory bowel disease. Compare rates (per person-time) or risks (proportion of patients with a minimum amount of follow-up). Describe and justify the choice of appropriate comparator population(s). Specify concise case definition for severe liver injury and validation of algorithm(s) to identify severe liver injury in the proposed data source. For the Tremfya (guselkumab)-exposed and comparator(s) cohorts, clearly define the study drug initiation period and any exclusion and inclusion criteria. Ensure that the data source allows for average follow-up for at least 1 year. Specify a minimum sample size and justify the precision of the estimate achievable with the proposed study."~"Pending"~~12/31/2038 0:00:00~9/5/2025 0:00:00
385071~"CDER"~"NDA"~218316.00~"NOVO NORDISK INC"~"Wegovy (semaglutide) tablets"~12/22/2025 0:00:00~"Supplement"~5~"4975-1"~"PMR"~"Pediatric Research Equity Act"~4975.00~1~"PMR 4975-1: Conduct a randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy after 52 weeks of semaglutide 7.2 mg for the treatment of obesity in pediatric patients ages 12 to less than 18 who still require weight loss after treatment with semaglutide 2.4 mg. Collect information on dysesthesia (altered skin sensation) as an adverse event of special interest, using dedicated case report forms. Provide narrative summaries of dysesthesia adverse events in the clinical study report for this trial."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~1/31/2031 0:00:00~
385072~"CDER"~"NDA"~218316.00~"NOVO NORDISK INC"~"Wegovy (semaglutide) tablets"~12/22/2025 0:00:00~"Supplement"~5~"4975-2"~"PMR"~"Pediatric Research Equity Act"~4975.00~2~"PMR 4975-2: Conduct a randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy after 52 weeks of semaglutide 7.2 mg for the treatment of obesity in pediatric patients ages 6 to less than 12 who still require weight loss after treatment with semaglutide 2.4 mg.  Compare the long-term (at least 2 years) safety and tolerability of semaglutide versus placebo for the treatment of obesity in both children and adolescents (ages 6 to less than 18 years). The trial may not be initiated until results from the semaglutide 7.2 mg adolescent trial, and the STEP YOUNG (NN9536-4512) trial in pediatric patients with obesity ages 6 to <12 years treated with semaglutide 2.4 mg, have been submitted to and reviewed by the Agency."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2036 0:00:00~
385073~"CDER"~"NDA"~218316.00~"NOVO NORDISK INC"~"Wegovy (semaglutide) tablets"~12/22/2025 0:00:00~"Supplement"~5~"4975-3"~"PMR"~"505 (o)(3)"~4975.00~3~"PMR 4975-3: Conduct the randomized, double-blind, active-controlled trial NN9838-7910 (comparing subcutaneous semaglutide 7.2 mg to 2.4 mg, both in combination with cagrilintide) with dysesthesia (altered skin sensation) as an adverse event of special interest. Use dedicated case report forms for data collection and provide narrative summaries of dysesthesia adverse events in the clinical study report for this trial."~"Pending"~~7/31/2029 0:00:00~
385074~"CDER"~"NDA"~218436.00~"NOVARTIS PHARMACEUTICALS CORP"~"Rhapsido (remibrutinib) tablet"~9/30/2025 0:00:00~"Original"~1~"4896-1"~"PMR"~"Pediatric Research Equity Act"~4896.00~1~"PMR 4896-1: Conduct a 24-week randomized, double-blind, placebo-controlled, parallel-group study in pediatric patients aged 12 to less than 18 years of age with chronic spontaneous urticaria who remain symptomatic despite H1-antihistamine treatment to assess the efficacy, safety, and pharmacokinetic responses to remibrutinib."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~2/28/2028 0:00:00~
385075~"CDER"~"NDA"~218436.00~"NOVARTIS PHARMACEUTICALS CORP"~"Rhapsido (remibrutinib) tablet"~9/30/2025 0:00:00~"Original"~1~"4896-2"~"PMR"~"Pediatric Research Equity Act"~4896.00~2~"PMR 4896-2: Conduct a 24-week, open-label, pharmacokinetic study in pediatric patients aged 6 to less than 12 years with chronic spontaneous urticaria who remain symptomatic despite H1-antihistamine treatment."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~3/31/2031 0:00:00~
385076~"CDER"~"NDA"~218436.00~"NOVARTIS PHARMACEUTICALS CORP"~"Rhapsido (remibrutinib) tablet"~9/30/2025 0:00:00~"Original"~1~"4896-3"~"PMR"~"Pediatric Research Equity Act"~4896.00~3~"PMR 4896-3: Conduct an additional oral (gavage) juvenile toxicity study in the rat. The age of rats at study start should be equivalent to pediatric patients approximately 6 years old. The study should include ophthalmic examinations performed pre-study, at the end of the dosing period, and at the end of the recovery period. Potential neurobehavioral effects should also be evaluated in animals at the end of the dosing and recovery periods."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~8/31/2027 0:00:00~
385077~"CDER"~"NDA"~218436.00~"NOVARTIS PHARMACEUTICALS CORP"~"Rhapsido (remibrutinib) tablet"~9/30/2025 0:00:00~"Original"~1~"4896-4"~"PMR"~"505 (o)(3)"~4896.00~4~"PMR 4896-4: Collect data from a prospective pregnancy exposure registry, preferably a disease-based multiproduct pregnancy registry, using a cohort analysis that compares the maternal, fetal, and infant outcomes of women exposed
to remibrutinib during pregnancy with an appropriate comparator population(s). Collect data outside the U.S. to reach the target sample size, if feasible. The registry will identify and record major and minor congenital malformations, pregnancy complications, spontaneous
abortion, stillbirths, neonatal deaths, pregnancy terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. These outcomes should be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the
first year of life."~"Pending"~~12/31/2038 0:00:00~
385078~"CDER"~"NDA"~218436.00~"NOVARTIS PHARMACEUTICALS CORP"~"Rhapsido (remibrutinib) tablet"~9/30/2025 0:00:00~"Original"~1~"4896-5"~"PMR"~"505 (o)(3)"~4896.00~5~"PMR 4896-5: Conduct a retrospective pregnancy cohort study using claims or electronic health record data with medical chart validation that is adequately powered to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to remibrutinib during pregnancy compared to appropriate comparator population(s)."~"Pending"~~12/31/2033 0:00:00~
385079~"CDER"~"NDA"~218436.00~"NOVARTIS PHARMACEUTICALS CORP"~"Rhapsido (remibrutinib) tablet"~9/30/2025 0:00:00~"Original"~1~"4896-6"~"PMR"~"505 (o)(3)"~4896.00~6~"PMR 4896-6: Perform a milk-only lactation study in lactating women who have received remibrutinib to measure concentrations of remibrutinib in breast milk using a validated assay. Assess the effects on the breastfed infant, if available, based on study population."~"Pending"~~9/30/2028 0:00:00~
385080~"CDER"~"NDA"~218550.00~"GENENTECH INC"~"Rozlytrek (Entrectinib)"~10/20/2023 0:00:00~"Original"~1~"4532-1"~"PMR"~"Accelerated Approval"~4532.00~1~"PMR 4532-1: Conduct an integrated analysis from completed and ongoing trials intended to verify and describe the clinical benefit of entrectinib, through more precise estimation of the overall response rate and mature response duration per independent review assessment, in adult and pediatric patients 12 years of age and older with solid tumors with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion and without a known acquired resistance mutation; that are metastatic or would require surgical resection that would result in severe morbidity; and that have no satisfactory alternative treatment or have progressed following treatment. A sufficient number of patients will be evaluated to more precisely characterize response and durability of response for each of the following tumor types: pediatric solid tumors, colorectal cancer, central nervous system cancers, gynecological cancers, and melanoma. A minimum of 40 patients with cancers other than pediatric solid tumors, colorectal cancer, central nervous system cancers, gynecological cancers, melanoma, soft tissue sarcoma, non-small cell adenocarcinoma lung cancer, mammary analogue secretory carcinoma, and secretory breast cancer will also be studied. Overall response rate and duration of response will be assessed by independent central review and all responding patients will be followed for at least 12 months from the onset of response."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~3/31/2027 0:00:00~12/11/2025 0:00:00
385081~"CDER"~"BLA"~761262.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~6/16/2022 0:00:00~"Supplement"~7~"4656-3"~"PMR"~"505 (o)(3)"~4656.00~3~"PMR 4656-3: Conduct an observational study to assess the incidence of severe acute liver injury in adults with moderately to severely active ulcerative colitis who are exposed to Skyrizi (risankizumab-rzaa), relative to other therapies used to treat ulcerative colitis. Compare rates (per person-time) or risks (proportion of patients with a minimum amount of follow-up). Describe and justify the choice of appropriate comparator population(s). Specify concise case definition for severe liver injury and validation of algorithm(s) to identify severe liver injury in the proposed data source. For the Skyrizi (risankizumab-rzaa)-exposed and comparator(s) cohorts, clearly define the study drug initiation period and any exclusion and inclusion criteria. Ensure that the data source allows for average follow-up for at least 1 year. Specify a minimum sample size and justify the precision of the estimate achievable with the proposed study. The ongoing observational study in patients with Crohn s disease (PMR 4294-1) with the same objectives may be amended to also enroll patients with ulcerative colitis."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 8/20/2025."~12/31/2033 0:00:00~6/17/2026 0:00:00
385082~"CDER"~"BLA"~761262.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~6/16/2022 0:00:00~"Supplement"~7~"4656-4"~"PMC"~"506B PMC"~4656.00~4~"PMC 4656-4: Submit postmarketing device reports to evaluate the on-body injector device used to deliver Skyrizi (risankizumab-rzaa) in patients with moderately to severely active Crohn's disease or ulcerative colitis. For reported device failure or use error events, collect information to allow root-cause analysis and assessment of associated adverse events. Specific information collected should include essential elements to allow for root-cause analysis such as patient narratives of the events and evaluation of returned devices, dosing information including the start of maintenance therapy, and any adverse events associated with the device failure or use error event (i.e., injection site pain/erythema) including disease activity as reported. Among confirmed device failure or use error events, identify a cohort of patients to prospectively follow for 6 months to assess disease activity (i.e., remission, flare/worsening disease symptoms, escalation of care, hospitalization, procedures)."~"Ongoing"~~2/28/2027 0:00:00~6/17/2026 0:00:00
385083~"CDER"~"BLA"~761263.00~"Genentech, Inc."~"Lunsumio (mosunetuzumab-axgb) Injection and Lunsumio VELO (mosunetuzumab-axgb) Injection"~12/22/2022 0:00:00~"Original"~1~"4375-1"~"PMR"~"Accelerated Approval"~4375.00~1~"PMR 4375-1: Conduct a randomized clinical trial in patients with relapsed or refractory follicular lymphoma, with patients randomized to receive mosunetuzumab in combination with lenalidomide or rituximab in combination with lenalidomide. The primary endpoint should be progression-free survival, with secondary endpoints that include response rate and overall survival. The trial should enroll a sufficiently representative study population to reflect the racial and ethnic diversity of the U.S. patient population with follicular lymphoma and allow for interpretation of the results in these patient populations."~"Delayed"~"Per the submitted 8/21/2025 progress report, the study is fully enrolled.The applicant requested revised milestones because of slower than anticipated event accumulation. Revised milestones were acknowledged in a letter dated 05/13/2026."~12/31/2025 0:00:00~2/14/2025 0:00:00
385084~"CDER"~"BLA"~761263.00~"Genentech, Inc."~"Lunsumio (mosunetuzumab-axgb) Injection and Lunsumio VELO (mosunetuzumab-axgb) Injection"~12/22/2022 0:00:00~"Original"~1~"4375-2"~"PMR"~"505 (o)(3)"~4375.00~2~"PMR 4375-2: Conduct an integrated safety analysis in patients with follicular lymphoma and other lymphoid malignancies to further characterize the incidence and severity of neurologic adverse reactions, hematologic adverse reactions,hemophagocytic lymphohistiocytosis, infections, and pneumonitis/interstitial lung disease, that may develop with longer term use of mosunetuzumab."~"Submitted"~~12/31/2025 0:00:00~2/14/2025 0:00:00
385085~"CDER"~"BLA"~761263.00~"Genentech, Inc."~"Lunsumio (mosunetuzumab-axgb) Injection and Lunsumio VELO (mosunetuzumab-axgb) Injection"~12/22/2022 0:00:00~"Supplement"~6~"4945-1"~"PMR"~"Accelerated Approval"~4945.00~1~"PMR 4945-1: Complete a randomized clinical trial intended to verify and describe the clinical benefit of mosunetuzumab in patients with follicular lymphoma. The trial should randomize patients with relapsed or refractory follicular lymphoma to receive mosunetuzumab in combination with lenalidomide or rituximab in combination with lenalidomide. The primary endpoint should be progression-free survival, with secondary endpoints that include response rate and overall survival. The trial should enroll a sufficiently representative study population to reflect the racial and ethnic populations to better reflect the U.S. patient population with follicular lymphoma and allow for interpretation of the results in these patient populations.  If the trial evaluates mosunetuzumab by intravenous injection, submit the final report of integrated studies and data to verify and further characterize the comparability, efficacy, and safety of mosunetuzumab by subcutaneous injection in combination with lenalidomide, to mosunetuzumab by intravenous injection in combination with lenalidomide. The final protocol should include the integrated comparative assessment plan"~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~3/31/2027 0:00:00~2/14/2025 0:00:00
385086~"CDER"~"BLA"~761269.00~"Eisai, Incorporated"~"Leqembi (lecanemab-irmb)"~1/6/2023 0:00:00~"Original"~1~"4384-3"~"PMR"~"505 (o)(3)"~4384.00~3~"PMR 4384-3: Using the improved and validated assays developed in response to PMR 4384-2, evaluate the impact of ADA and NAb on the pharmacokinetics, pharmacodynamics, safety, and efficacy of lecanemab-irmb in patients enrolled in the confirmatory study."~"Fulfilled"~~6/30/2025 0:00:00~3/3/2026 0:00:00
385087~"CDER"~"NDA"~219304.00~"DECIPHERA PHARMACEUTICALS LLC"~"Romvimza (vimseltinib) capsules"~2/14/2025 0:00:00~"Original"~1~"4791-1"~"PMR"~"505 (o)(3)"~4791.00~1~"PMR 4791-1: Complete the ongoing 104-week rat carcinogenicity study (Study No. DCC-3014-04-0025) to evaluate the potential for carcinogenicity."~"Fulfilled"~~6/30/2025 0:00:00~4/17/2026 0:00:00
385088~"CDER"~"NDA"~219304.00~"DECIPHERA PHARMACEUTICALS LLC"~"Romvimza (vimseltinib) capsules"~2/14/2025 0:00:00~"Original"~1~"4791-2"~"PMR"~"505 (o)(3)"~4791.00~2~"PMR 4791-2: Conduct a prospective clinical registry to evaluate the potential risk of serious hepatotoxicity in patients receiving vimseltinib for the treatment of tenosynovial giant cell tumor. Analyses should include evaluation of alanine aminotransferase, aspartate aminotransferase, bilirubin, symptoms suggestive of hepatotoxicity, and dose reduction and treatment discontinuation related to hepatotoxicity. Include investigations of time to event and severity, and potential risk factors such as age, race, and comorbidities. The registry should aim to enroll at least 100 patients. Monitor patients until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first. Interim safety analyses should be performed yearly beginning one year after the first patient has received vimseltinib to characterize the long-term risk of hepatotoxicity with vimseltinib."~"Delayed"~"The interim report submission #1 milestone was missed. A request to revise the milestone was submitted on 4-3-2026 and the revised milestone was acknowledged with good cause on 6-23-2026."~5/31/2031 0:00:00~4/17/2026 0:00:00
385089~"CDER"~"NDA"~219304.00~"DECIPHERA PHARMACEUTICALS LLC"~"Romvimza (vimseltinib) capsules"~2/14/2025 0:00:00~"Original"~1~"4791-3"~"PMR"~"505 (o)(3)"~4791.00~3~"PMR 4791-3: Conduct a clinical pharmacokinetic trial to evaluate the serious potential risk of increased drug toxicity and determine an appropriate dose of vimseltinib to minimize potential serious toxicity, in patients with moderate hepatic impairment using the NCI-ODWG criteria. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Ongoing"~~2/28/2027 0:00:00~4/17/2026 0:00:00
385090~"CDER"~"NDA"~219304.00~"DECIPHERA PHARMACEUTICALS LLC"~"Romvimza (vimseltinib) capsules"~2/14/2025 0:00:00~"Original"~1~"4791-4"~"PMR"~"505 (o)(3)"~4791.00~4~"PMR 4791-4: Conduct a clinical pharmacokinetic trial to evaluate the effect of multiple doses of vimseltinib on the single dose pharmacokinetics of a sensitive BCRP substrate to evaluate the serious potential risk of increased drug toxicity. Design and conduct the trial in accordance with the ICH Harmonized Guidance entitled  M12 Drug Interaction Studies. "~"Ongoing"~~12/31/2026 0:00:00~4/17/2026 0:00:00
385091~"CDER"~"NDA"~219304.00~"DECIPHERA PHARMACEUTICALS LLC"~"Romvimza (vimseltinib) capsules"~2/14/2025 0:00:00~"Original"~1~"4791-5"~"PMR"~"505 (o)(3)"~4791.00~5~"PMR 4791-5: Conduct a clinical pharmacokinetic trial to evaluate the effect of multiple doses of vimseltinib on the single dose pharmacokinetics of sensitive OATP1B1 and OATP1B3 substrates to evaluate the serious potential risk of increased drug toxicity. Design and conduct the trial in accordance with the ICH Harmonized Guidance entitled  M12 Drug Interaction Studies. "~"Ongoing"~~12/31/2026 0:00:00~4/17/2026 0:00:00
385092~"CDER"~"NDA"~219304.00~"DECIPHERA PHARMACEUTICALS LLC"~"Romvimza (vimseltinib) capsules"~2/14/2025 0:00:00~"Original"~1~"4791-6"~"PMR"~"505 (o)(3)"~4791.00~6~"PMR 4791-6: Conduct a clinical pharmacokinetic trial to evaluate the effect of multiple doses of vimseltinib on the single dose pharmacokinetics of a sensitive OCT2 substrate to evaluate the serious potential risk of increased drug toxicity. Design and conduct the trial in accordance with the ICH Harmonized Guidance entitled  M12 Drug Interaction Studies. "~"Ongoing"~~12/31/2026 0:00:00~4/17/2026 0:00:00
385093~"CDER"~"NDA"~219373.00~"RUBICON RESEARCH LTD"~"Lopressor (metoprolol tartrate) Oral Solution"~4/10/2025 0:00:00~"Original"~1~"4804-1"~"PMR"~"Pediatric Research Equity Act"~4804.00~1~"PMR 4804-1: Conduct a study to evaluate efficacy, safety, pharmacokinetics and pharmacodynamics of metoprolol oral solution in pediatric patients 0 to < 18 years old with hypertension."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~5/31/2029 0:00:00~6/8/2026 0:00:00
385094~"CDER"~"NDA"~219379.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib)"~2/11/2025 0:00:00~"Original"~1~"4784-1"~"PMR"~"505 (o)(3)"~4784.00~1~"PMR 4784-1: Complete a carcinogenicity study in rats to evaluate the potential serious risk of carcinogenicity."~"Pending"~~4/30/2028 0:00:00~4/8/2026 0:00:00
385095~"CDER"~"NDA"~219379.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib)"~2/11/2025 0:00:00~"Original"~1~"4784-2"~"PMR"~"505 (o)(3)"~4784.00~2~"PMR 4784-2: Conduct an integrated safety analysis of data from clinical trials to characterize and evaluate the potential serious risk of long-term adverse effects of mirdametinib on growth and development in pediatric patients. Include long-term follow-up data from pediatric patients enrolled on the MEK-NF-201 (ReNeu) trial (NCT03962543) and from ongoing and completed clinical trials of mirdametinib. Pediatric patients must be evaluated for growth and sexual development milestones annually for at least 7 years from initiation of mirdametinib or until a patient has achieved sexual maturity. Evaluations must include growth as measured by weight, height, height velocity, and height standard deviation scores (SDS); if available, include, age at thelarche (females), age at adrenarche (males), age at menarche (females), and Tanner Stage progression."~"Pending"~~6/30/2029 0:00:00~4/8/2026 0:00:00
385096~"CDER"~"NDA"~219379.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib)"~2/11/2025 0:00:00~"Original"~1~"4784-3"~"PMR"~"505 (o)(3)"~4784.00~3~"PMR 4784-3: Conduct an integrated safety analysis of data from clinical trials to further characterize the long-term incidence, severity, and outcomes of the known serious risks of ocular toxicity, left ventricular dysfunction, and dermatologic adverse reactions. Include the long-term follow-up safety data (minimum of 7 years) from adult and pediatric patients enrolled on the MEK-NF-201 (ReNeu) trial (NCT03962543) and all other ongoing and completed trials of mirdametinib to include an analysis of the following toxicities: ocular toxicity (including but not limited to retinal pigment epithelial detachment and retinal vein occlusion), cardiac toxicity (including but not limited to left ventricular dysfunction), and serious dermatologic toxicity."~"Pending"~~6/30/2029 0:00:00~4/8/2026 0:00:00
385097~"CDER"~"NDA"~219293.00~"AZURITY PHARMACEUTICALS INC"~"Danziten (nilotinib) Tablets"~11/7/2024 0:00:00~"Original"~1~"4717-1"~"PMR"~"Pediatric Research Equity Act"~4717.00~1~"PMR 4717-1: Conduct a molecularly targeted pediatric cancer investigation, which includes developling an age-appropriate formulation."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~12/31/2027 0:00:00~1/6/2026 0:00:00
385098~"CDER"~"NDA"~219301.00~"KALVISTA PHARMACEUTICALS LTD"~"Ekterly (sebetralstat) tablets"~7/3/2025 0:00:00~"Original"~1~"4814-1"~"PMR"~"505 (o)(3)"~4814.00~1~"PMR 4814-1: Conduct a 2-year carcinogenicity study in rats with sebetralstat"~"Submitted"~~10/31/2025 0:00:00~
385099~"CDER"~"NDA"~219379.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib)"~2/11/2025 0:00:00~"Original"~1~"4784-4"~"PMR"~"505 (o)(3)"~4784.00~4~"PMR 4784-4: Conduct a clinical pharmacokinetic trial to evaluate the potential serious risk of increased drug toxicity, and to determine an appropriate, safe dose of mirdametinib, in patients with moderate (total bilirubin > 1.5   3.0 x ULN, any AST) and severe (total bilirubin > 3   10 x ULN, any AST) hepatic impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~1/31/2027 0:00:00~4/8/2026 0:00:00
385100~"CDER"~"BLA"~761062.00~"Amgen Inc."~"Evenity (romosozumab-aqqg)"~4/9/2019 0:00:00~"Original"~1~"3595-1"~"PMR"~"505 (o)(3)"~3595.00~1~"PMR 3595-1: To evaluate the feasibility of a required post-marketing study or trial assessing the cardiovascular safety of Evenity, conduct a study using a sequential analysis design (e.g., repeated analyses within five 1-year blocks of calendar time following marketing approval of Evenity) to assess utilization patterns and channeling bias. Using an appropriate look back period for each characteristic, compare relevant patient characteristics measured at baseline, including patient demographics, history of stroke or myocardial infarction (MI) in the 1 year prior to initiation of therapy, history of fractures or falls, fracture risk scores, pertinent comorbidities (e.g., other history of MI, stroke, other cardiovascular diseases), pertinent medication use (e.g., other osteoporosis medications, glucocorticoids), healthcare utilization, and prescribing provider specialty among new users of Evenity compared to new users of other anti-osteoporosis therapies."~"Fulfilled"~~9/30/2024 0:00:00~6/8/2026 0:00:00
385101~"CDER"~"BLA"~761063.00~"Eli Lilly and Company"~"Emgality (Galcanezumab) "~9/27/2018 0:00:00~"Original"~1~"3498-1"~"PMR"~"Pediatric Research Equity Act"~3498.00~1~"PMR 3498-1: Deferred pediatric randomized, double-blind, placebo-controlled study under PREA to assess the pharmacokinetics (Part A), and efficacy and safety (Part B) for the preventive treatment of episodic migraine in children and adolescents ages 6 through 17 years. Part A includes an option to enroll in an open-label safety extension phase (9 months), followed by a post treatment phase (4 months). Part B includes a double-blind treatment phase (3 months) and an open-label safety extension phase (9 months), followed by a post treatment phase (4 months). This study is to be submitted as a special protocol assessment (SPA)."~"Delayed"~"Original Final Report Due Date: 03/31/2024; Deferral Extension granted per FDA letter dated 09/12/2022. The study completion milestone was also revised due to challenges in recruitment/enrollment of pediatric patients as a result of the COVID-19 pandemic."~5/31/2027 0:00:00~11/24/2025 0:00:00
385102~"CDER"~"BLA"~761063.00~"Eli Lilly and Company"~"Emgality (Galcanezumab) "~9/27/2018 0:00:00~"Original"~1~"3498-2"~"PMR"~"Pediatric Research Equity Act"~3498.00~2~"PMR 3498-2: Deferred pediatric randomized, double-blind, placebo-controlled efficacy and safety study under PREA for the preventive treatment of chronic migraine in adolescents ages 12 through 17 years. This study includes a double-blind treatment phase (3 months) and an open-label safety extension phase (9 months), followed by a post treatment phase (4 months). This study is to be submitted as a special protocol assessment (SPA)."~"Delayed"~"Original Final Report Due Date: 03/31/2024; Deferral Extension granted per FDA letter dated 09/12/2022. The study completion milestone was also revised due to challenges in recruitment/enrollment of pediatric patients as a result of the COVID-19 pandemic."~5/31/2027 0:00:00~11/24/2025 0:00:00
385103~"CDER"~"BLA"~761063.00~"Eli Lilly and Company"~"Emgality (Galcanezumab) "~9/27/2018 0:00:00~"Original"~1~"3498-3"~"PMR"~"505 (o)(3)"~3498.00~3~"PMR 3498-3: Conduct prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with migraine exposed to Emgality during pregnancy with two unexposed control populations: one consisting of women with migraine who have not been exposed to Emgality before or during pregnancy and the other consisting of women without migraine. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~11/30/2033 0:00:00~11/24/2025 0:00:00
385104~"CDER"~"BLA"~761063.00~"Eli Lilly and Company"~"Emgality (Galcanezumab) "~9/27/2018 0:00:00~"Original"~1~"3498-4"~"PMR"~"505 (o)(3)"~3498.00~4~"PMR 3498-4: Conduct a pregnancy outcomes study using a different study design than provided for in PMR 3498-3 (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-forgestational-age births in women exposed to Emgality during pregnancy compared to an unexposed control population."~"Ongoing"~~12/31/2027 0:00:00~11/24/2025 0:00:00
385105~"CDER"~"BLA"~761065.00~"Theratechnologies Inc."~"Trogarzo (ibalizumab-uiyk)"~3/6/2018 0:00:00~"Supplement"~11~"3989-1"~"PMR"~"505 (o)(3)"~3989.00~1~"PMR 3989-1: Conduct a study as a registry to collect prospective data in individuals exposed to Trogarzo (ibalizumab-uiyk) during pregnancy to monitor maternal and pregnancy outcomes, including adverse effects on the developing fetus, neonate, and infant. The study will assess infant outcomes, such as infections, hospitalizations, and death through the first year of life. The study will collect information for 10 years."~"Ongoing"~~4/30/2033 0:00:00~5/5/2026 0:00:00
385106~"CDER"~"BLA"~761066.00~"Samsung Bioepis Co., Ltd."~"Eticovo Auto Injector (etanercept-ykro)"~4/25/2019 0:00:00~"Original"~1~"3608-3"~"PMR"~"Pediatric Research Equity Act"~3608.00~3~"PMR 3608-3: Develop a presentation that can be used to accurately administer Eticovo (etanercept-ykro) to pediatric patients who weigh less than 63 kg.
"~"Delayed"~"The final report was missed because of delays due to issues with the study drug and/or comparator drug. Original Final Report Due Date: 09/2023; Deferral Extension granted per FDA letter dated 08/28/2023."~12/31/2026 0:00:00~6/22/2026 0:00:00
385107~"CDER"~"BLA"~761067.00~"Sun Pharmaceutical Industries Limited "~"Ilumya (tildrakizumab-asmn)"~3/20/2018 0:00:00~"Original"~1~"3357-1"~"PMR"~"Pediatric Research Equity Act"~3357.00~1~"PMR 3357-1: Conduct a pharmacokinetics (PK), safety and efficacy study in pediatric subjects 6 years to 17 years of age with moderate-to-severe plaque psoriasis (with a duration of exposure to tildrakizumab-asmn of at least one year)."~"Delayed"~"Original Final Report Due Date: 10/31/2025; Deferral Extension granted per FDA letter dated 07/01/2025. The study completion milestone was also revised.
"~4/30/2027 0:00:00~5/18/2026 0:00:00
385108~"CDER"~"BLA"~761067.00~"Sun Pharmaceutical Industries Limited "~"Ilumya (tildrakizumab-asmn)"~3/20/2018 0:00:00~"Original"~1~"3357-2"~"PMR"~"505 (o)(3)"~3357.00~2~"PMR 3357-2: A prospective, registry-based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to tildrakizumab-asmn during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including neonatal deaths, infections in the first 6 months of life, and effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~1/31/2030 0:00:00~5/18/2026 0:00:00
385109~"CDER"~"BLA"~761119.00~"Lundbeck Seattle BioPharmaceuticals, Inc."~"Vyepti (eptinezumab-jjmr)"~2/21/2020 0:00:00~"Original"~1~"3796-2"~"PMR"~"Pediatric Research Equity Act"~3796.00~2~"PMR 3796-2: A single-arm, single-dose, pediatric pharmacokinetic study to evaluate eptinezumab in pediatric migraine patients ages 6 through 11 years. The primary objective will be to characterize the pharmacokinetic profile of eptinezumab compared to adult migraine patients, with additional secondary pharmacokinetic and safety endpoints. Immunogenicity will also be assessed through development of specific anti-eptinezumab antibodies."~"Fulfilled"~"Per FDA letter dated 12/08/2025, this PMR/PMC has been fulfilled."~4/30/2028 0:00:00~4/20/2026 0:00:00
385110~"CDER"~"BLA"~761119.00~"Lundbeck Seattle BioPharmaceuticals, Inc."~"Vyepti (eptinezumab-jjmr)"~2/21/2020 0:00:00~"Original"~1~"3796-3"~"PMR"~"Pediatric Research Equity Act"~3796.00~3~"PMR 3796-3: A randomized, double-blind, placebo-controlled, parallel-group, efficacy and safety study of eptinezumab in patients with episodic migraine age 6 through 17 years. The study will include an initial double-blind treatment phase (minimum of 3 months) followed by an open-label extension phase for a minimum of 9 months duration. Of the total number of patients enrolled in this trial, a minimum of 25% of patients will be in the 6-11 year age group. There will be a similar number of patients in the 12-14 year old age range, and the 15-17 year old age range. This study is to be submitted as a Special Protocol Assessment (SPA)."~"Delayed"~"Original Final Report Due Date: 04/30/2025; Deferral Extension granted per FDA letter dated 12/02/2024. The study completion milestone was also revised because of delays involving study participants, site, and/or management (specifically, difficulties with subject recruitment). Current enrollment 86 of 315."~4/30/2028 0:00:00~4/20/2026 0:00:00
385111~"CDER"~"BLA"~761119.00~"Lundbeck Seattle BioPharmaceuticals, Inc."~"Vyepti (eptinezumab-jjmr)"~2/21/2020 0:00:00~"Original"~1~"3796-4"~"PMR"~"Pediatric Research Equity Act"~3796.00~4~"PMR 3796-4: A randomized, double-blind, placebo-controlled, parallel-group, efficacy and safety study of eptinezumab in patients with chronic migraine age 12 through 17 years. The study will include an initial double-blind treatment phase (minimum of 3 months), followed by open-label extension phase for a minimum of 9 months duration. There will be a similar number of patients in the 12-14 year old age range, and the 15-17 year old age range. This study is to be submitted as a Special Protocol Assessment (SPA)."~"Delayed"~"Original Final Report Due Date: 04/30/2025; Deferral Extension granted per FDA letter dated 12/02/2024. The study completion milestone was also revised because of delays involving study participants, site, and/or management (specifically, difficulties with subject recruitment). Current enrollment 177 of 285.
"~4/30/2028 0:00:00~4/20/2026 0:00:00
385112~"CDER"~"BLA"~761119.00~"Lundbeck Seattle BioPharmaceuticals, Inc."~"Vyepti (eptinezumab-jjmr)"~2/21/2020 0:00:00~"Original"~1~"3796-5"~"PMR"~"505 (o)(3)"~3796.00~5~"PMR 3796-5: Prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with migraine exposed to Vyepti during pregnancy with two unexposed control populations: one consisting of women with migraine who have not been exposed to Vyepti before or during pregnancy, and the other consisting of women without migraine. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The study is underway. The interim report was submitted by the Acknowledged revised milestone on 12/17/2025. Enrollment: 7/422."~12/31/2034 0:00:00~4/20/2026 0:00:00
385113~"CDER"~"BLA"~761119.00~"Lundbeck Seattle BioPharmaceuticals, Inc."~"Vyepti (eptinezumab-jjmr)"~2/21/2020 0:00:00~"Original"~1~"3796-6"~"PMR"~"505 (o)(3)"~3796.00~6~"PMR 3796-6: A pregnancy outcomes study using a different study design than provided for in PMR 3795-5 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small-for-gestational-age births in women exposed to Vyepti during pregnancy compared to an unexposed control population."~"Delayed"~"The study is underway. The interim report was submitted by the Acknowledged revised milestone on 12/17/2025. Two women exposed to eptinezumab during 1st trimester."~12/31/2028 0:00:00~4/20/2026 0:00:00
385114~"CDER"~"BLA"~761121.00~"Genentech, Inc."~"Polivy (polatuzumab vedotin (DCDS4501A or RO5541077))"~6/10/2019 0:00:00~"Supplement"~8~"4430-1"~"PMR"~"505 (o)(3)"~4430.00~1~"PMR 4430-1: Conduct an analysis of patients enrolled in Study GO39942 (POLARIX) to further characterize the serious risks, including infections and fatal adverse events, with extended follow-up in patients receiving polatuzumab vedotin in combination with R-CHP (rituximab, cyclophosphamide, doxorubicin, and prednisone) as first-line treatment for large B-cell lymphoma. Include evaluation of overall survival in each treatment arm in POLARIX, with a minimum of 2 additional years of follow-up, and include causes of death and narratives for death in the absence of treated disease progression."~"Fulfilled"~~12/31/2024 0:00:00~7/31/2025 0:00:00
385115~"CDER"~"BLA"~761121.00~"Genentech, Inc."~"Polivy (polatuzumab vedotin (DCDS4501A or RO5541077))"~6/10/2019 0:00:00~"Supplement"~8~"4430-2"~"PMC"~"506B PMC"~4430.00~2~"PMC 4430-2: Complete Study MO40598 (POLARGO), a randomized clinical trial evaluating polatuzumab vedotin in combination with rituximab, gemcitabine, and oxaliplatin (R-GemOx) versus R-GemOx alone in patients with relapsed or refractory large B-cell lymphoma, with a primary endpoint of overall survival."~"Submitted"~~6/30/2025 0:00:00~7/31/2025 0:00:00
385116~"CDER"~"BLA"~761123.00~"AstraZeneca AB"~"Saphnelo (anifrolumab-fnia)"~7/30/2021 0:00:00~"Original"~1~"4116-1"~"PMR"~"Pediatric Research Equity Act"~4116.00~1~"PMR 4116-1: Conduct a study to evaluate the safety, efficacy, and pharmacokinetics of anifrolumab-fnia plus background standard therapy in pediatric subjects ages 5 years to 17 years of age with active systemic lupus erythematosus (SLE)."~"Delayed"~"The final protocol submission milestone was missed due to continued discussion with the Agency. Per applicant, to date, 25 patients have been screened on the trial and 10 randomized (last patient randomized on 18 Sep 2025). As of 19 September 2025, 74 sites have been activated across 13 countries."~5/31/2027 0:00:00~9/25/2025 0:00:00
385117~"CDER"~"BLA"~761123.00~"AstraZeneca AB"~"Saphnelo (anifrolumab-fnia)"~7/30/2021 0:00:00~"Original"~1~"4116-2"~"PMR"~"505 (o)(3)"~4116.00~2~"PMR 4116-2: Conduct a prospective pregnancy registry to evaluate the effects of Saphnelo (anifrolumab-fnia) on pregnancy and maternal and fetal/neonatal outcomes. This pregnancy registry study may be conducted as part of a multiple-product or disease-based pregnancy registry."~"Delayed"~"The final protocol submission milestone was missed because additional time is needed to establish study requirements. The interim report was submitted by the milestone date on 4/30/2025."~4/30/2032 0:00:00~9/25/2025 0:00:00
385118~"CDER"~"BLA"~761269.00~"Eisai, Incorporated"~"Leqembi (lecanemab-irmb)"~1/6/2023 0:00:00~"Supplement"~1~"4497-1"~"PMR"~"505 (o)(3)"~4497.00~1~"PMR 4497-1: Conduct a registry-based, prospective, observational study to evaluate clinical safety outcomes among Alzheimer s disease patients treated with lecanemab-irmb, using, for example, the Alzheimer s Network for Treatment and Diagnostics (ALZ-NET) registry, including patients who are ApoE e4 homozygotes, and/or exposed to antithrombotics, and/or have a diagnosis of, or imaging findings consistent with a high risk for, cerebral amyloid angiopathy. The primary clinical safety outcomes should include amyloid related imaging abnormalities (ARIA)-edema (ARIA-E), and ARIA hemosiderin deposition (ARIA-H) and any associated clinical symptoms, and intracerebral hemorrhage >1 cm in size. Additional outcomes of interest should also include seizures, anaphylaxis, and death. Baseline characterization of the registry population should include demographic data, diagnosis and stage of disease, ApoE genotype, baseline MRI findings (e.g., microhemorrhages, evidence of cerebral amyloid angiography or other imaging findings consistent with high risk of cerebral amyloid angiography, etc.), other biomarkers that are potential predictors of disease course or adverse outcomes, and prior medications including prior Alzheimer s disease (AD) therapy and antithrombotic therapy. The registry should also collect information on concomitant medications (e.g., antiplatelet and antithrombotic drugs, other AD treatments). When available, the study should provide a comparison of safety outcomes to estimated background rates in an appropriate comparator population."~"Delayed"~"The final protocol submission milestone was missed because of time needed to revise the draft study protocol based on the Agency s recommendations. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 03/17/2026."~1/31/2036 0:00:00~3/3/2026 0:00:00
385119~"CDER"~"BLA"~761269.00~"Eisai, Incorporated"~"Leqembi (lecanemab-irmb)"~1/6/2023 0:00:00~"Supplement"~1~"4497-2"~"PMR"~"505 (o)(3)"~4497.00~2~"PMR 4497-2: Use emerging safety data from ongoing studies and published literature, validate administrative claim codes for intracerebral hemorrhage in patients with Alzheimer s disease. The outcome of intracerebral hemorrhage should distinguish between amyloid related imaging abnormalities-hemosiderin deposition (ARIA-H) and cerebral hemorrhage greater than 1 cm. Secondary outcomes of interest include ARIA-edema (ARIA-E) and ARIA-H, seizures, anaphylaxis, and death. For secondary outcomes not well validated, develop algorithms and/or computable phenotypes using data leveraged from PMR 4497-1 and other sources for the outcomes of interest. Describe an approach to identifying an appropriate comparator group with Alzheimer s disease untreated with lecanemab-irmb. Obtain FDA agreement with the outcome algorithm specifications and comparator population prior to proceeding to conducting the retrospective cohort study. Based upon validated algorithms agreed to by the Sponsor and FDA, conduct a comparative retrospective cohort study using claims data with available medical chart review as needed or electronic health record data to assess clinical safety outcomes in a broad population of Alzheimer s disease patients treated with lecanemab-irmb."~"Delayed"~"The interim report submission milestone was missed because of time needed to revise the draft study protocol based on the Agency s recommendations. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 03/17/2026."~12/31/2030 0:00:00~3/3/2026 0:00:00
385120~"CDER"~"BLA"~761269.00~"Eisai, Incorporated"~"Leqembi (lecanemab-irmb)"~1/6/2023 0:00:00~"Supplement"~1~"4497-3"~"PMR"~"505 (o)(3)"~4497.00~3~"PMR 4497-3: Further characterize the safety of treatment with lecanemab-irmb in patients who are homozygous for ApoE e4. We would accept information on this risk from a randomized, clinical trial in participants with early preclinical Alzheimer s disease and intermediate amyloid (i.e., AHEAD 3-45 Study). Ensure that approximately 15% of the population, distributed equally among lecanemab-irmb and control, is homozygous for ApoE e4."~"Ongoing"~~2/28/2030 0:00:00~3/3/2026 0:00:00
385121~"CDER"~"BLA"~761275.00~"Fresenius Kabi USA, LLC"~"Tyenne (tocilizumab-aazg) Injection"~3/5/2024 0:00:00~"Supplement"~8~"4806-1"~"PMR"~"Pediatric Research Equity Act"~4806.00~1~"PMR 4806-1: Assessment of Tyenne (tocilizumab-aazg) for the treatment of Coronavirus Disease 2019 (COVID-19) in pediatric patients 1 to <18 years of age."~"Fulfilled"~"Per FDA letter dated 05/20/2026, this PMR/PMC has been fulfilled."~6/30/2026 0:00:00~4/30/2026 0:00:00
385122~"CDER"~"BLA"~761278.00~"Chiesi Farmaceutici S.p.A."~"Lamzede (velmanase alfa-tycv)"~2/16/2023 0:00:00~"Original"~1~"4397-1"~"PMR"~"505 (o)(3)"~4397.00~1~"PMR 4397-1: Perform an assessment of the potential effects of increased alphamannosidase exposure (and increased MAN2B1 expression) on tumor formation."~"Fulfilled"~~8/31/2024 0:00:00~4/17/2025 0:00:00
385123~"CDER"~"BLA"~761278.00~"Chiesi Farmaceutici S.p.A."~"Lamzede (velmanase alfa-tycv)"~2/16/2023 0:00:00~"Original"~1~"4397-2"~"PMC"~"506B PMC"~4397.00~2~"PMC 4397-2: Perform a bridging pharmacokinetic study in rats to characterize the velmanase alfa-tycv exposure in the reproductive toxicity and pre- and postnatal development studies."~"Fulfilled"~~2/28/2025 0:00:00~4/17/2025 0:00:00
385124~"CDER"~"BLA"~761278.00~"Chiesi Farmaceutici S.p.A."~"Lamzede (velmanase alfa-tycv)"~2/16/2023 0:00:00~"Original"~1~"4397-3"~"PMC"~"506B PMC"~4397.00~3~"PMC 4397-3: Conduct a 26-week repeat-dose pharmacodynamic (PD) study in alpha mannosidase-deficient transgenic-knockout mice to evaluate changes in the M2 biomarker and histopathology in response to treatment with velmanase alfa-tycv."~"Submitted"~~10/31/2025 0:00:00~4/17/2025 0:00:00
385125~"CDER"~"BLA"~761278.00~"Chiesi Farmaceutici S.p.A."~"Lamzede (velmanase alfa-tycv)"~2/16/2023 0:00:00~"Original"~1~"4397-4"~"PMC"~"506B PMC"~4397.00~4~"PMC 4397-4: Evaluate the pharmacodynamics of velmanase alfa-tycv in pediatric patients less than 3 years of age with a confirmed diagnosis of alphamannosidosis. If the results suggest inadequate pharmacodynamic response at the currently recommended dose of 1 mg/kg, additional clinical studies may be needed to explore doses higher than 1 mg/kg for patients who cannot achieve an optimal pharmacodynamic response at 1 mg/kg."~"Ongoing"~~9/30/2030 0:00:00~4/17/2025 0:00:00
385126~"CDER"~"BLA"~761279.00~"Eli Lilly and Company"~"Omvoh (mirikizumab-mrkz)"~10/26/2023 0:00:00~"Original"~1~"4409-1"~"PMR"~"505 (o)(3)"~4409.00~1~"PMR 4409-1: Perform a lactation study (milk only) in lactating women who have received Omvoh (mirikizumab-mrkz), regardless of indication, to assess concentrations of mirikizumab-mrkz in breast milk using a validated assay and to assess the effects on the breastfed infant."~"Delayed"~"The 2025 study completion submission milestone was missed because of continued communications with the agency to an agreed final protocol which was acknowledged on April 1, 2025.  FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 01/05/2026.  In the same letter, FDA also acknowledged that there was sufficient justification for the anticipated delay of the final report  submission 2026 proposed revised milestone."~12/31/2026 0:00:00~10/9/2025 0:00:00
385127~"CDER"~"NDA"~219379.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib)"~2/11/2025 0:00:00~"Original"~1~"4784-5"~"PMC"~"506B PMC"~4784.00~5~"PMC 4784-5: Complete the MEK-NF-201 (ReNeu) trial (NCT03962543) to further characterize the clinical benefit of mirdametinib in adult and pediatric patients with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. To characterize response rate and duration, patients will be followed for at least 24 months from the onset of response."~"Pending"~~6/30/2029 0:00:00~4/8/2026 0:00:00
385128~"CDER"~"NDA"~219379.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib)"~2/11/2025 0:00:00~"Original"~1~"4784-6"~"PMC"~"506B PMC"~4784.00~6~"PMC 4784-6: Conduct a clinical pharmacokinetic trial with a strong CYP3A4 inducer (that also induces UGTs, P-gp, and CES enzymes) with mirdametinib to evaluate the effect of strong CYP3A4 induction on decreasing the systemic exposure of mirdametinib and to provide dosage recommendations for mirdametinib when used concomitantly with strong CYP3A4 inducers. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  M12 Drug Interaction Studies. "~"Pending"~~4/30/2027 0:00:00~4/8/2026 0:00:00
385129~"CDER"~"NDA"~219389.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib) Capsule"~2/11/2025 0:00:00~"Original"~1~"4785-1"~"PMR"~"505 (o)(3)"~4785.00~1~"PMR 4785-1: Complete a carcinogenicity study in rats to evaluate the potential serious risk of carcinogenicity."~"Pending"~~4/30/2028 0:00:00~4/8/2026 0:00:00
385130~"CDER"~"NDA"~219389.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib) Capsule"~2/11/2025 0:00:00~"Original"~1~"4785-2"~"PMR"~"505 (o)(3)"~4785.00~2~"PMR 4785-2: Conduct an integrated safety analysis of data from clinical trials to characterize and evaluate the potential serious risk of long-term adverse effects of mirdametinib on growth and development in pediatric patients. Include long-term follow-up data from pediatric patients enrolled on the MEK-NF-201 (ReNeu) trial (NCT03962543) and from ongoing and completed clinical trials of mirdametinib. Pediatric patients must be evaluated for growth and sexual development milestones annually for at least 7 years from initiation of mirdametinib or until a patient has achieved sexual maturity. Evaluations must include growth as measured by weight, height, height velocity, and height standard deviation scores (SDS); if available, include, age at thelarche (females), age at adrenarche (males), age at menarche (females), and Tanner Stage progression."~"Pending"~~6/30/2029 0:00:00~4/8/2026 0:00:00
385131~"CDER"~"NDA"~219389.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib) Capsule"~2/11/2025 0:00:00~"Original"~1~"4785-3"~"PMR"~"505 (o)(3)"~4785.00~3~"PMR 4785-3: Conduct an integrated safety analysis of data from clinical trials to further characterize the long-term incidence, severity, and outcomes of the known serious risks of ocular toxicity, left ventricular dysfunction, and dermatologic adverse reactions. Include the long-term follow-up safety data (minimum of 7 years) from adult and pediatric patients enrolled on the MEK-NF-201 (ReNeu) trial (NCT03962543) and all other ongoing and completed trials of mirdametinib to include an analysis of the following toxicities: ocular toxicity (including but not limited to retinal pigment epithelial detachment and retinal vein occlusion), cardiac toxicity (including but not limited to left ventricular dysfunction), and serious dermatologic toxicity."~"Pending"~~6/30/2029 0:00:00~4/8/2026 0:00:00
385132~"CDER"~"NDA"~219389.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib) Capsule"~2/11/2025 0:00:00~"Original"~1~"4785-4"~"PMR"~"505 (o)(3)"~4785.00~4~"PMR 4785-4: Conduct a clinical pharmacokinetic trial to evaluate the potential serious risk of increased drug toxicity, and to determine an appropriate, safe dose of mirdametinib, in patients with moderate (total bilirubin > 1.5   3.0 x ULN, any AST) and severe (total bilirubin > 3   10 x ULN, any AST) hepatic impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~1/31/2027 0:00:00~4/8/2026 0:00:00
385133~"CDER"~"NDA"~219389.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib) Capsule"~2/11/2025 0:00:00~"Original"~1~"4785-5"~"PMC"~"506B PMC"~4785.00~5~"PMC 4785-5: Complete the MEK-NF-201 (ReNeu) trial (NCT03962543) to further characterize the clinical benefit of mirdametinib in  adult and pediatric patients with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. To characterize response rate and duration, patients will be followed for at least 24 months from the onset of response."~"Pending"~~6/30/2029 0:00:00~4/8/2026 0:00:00
385134~"CDER"~"NDA"~219389.00~"SPRINGWORKS THERAPEUTICS INC"~"Gomekli (mirdametinib) Capsule"~2/11/2025 0:00:00~"Original"~1~"4785-6"~"PMC"~"506B PMC"~4785.00~6~"PMC 4785-6: Conduct a clinical pharmacokinetic trial with a strong CYP3A4 inducer (that also induces UGTs, P-gp, and CES enzymes) with mirdametinib to evaluate the effect of strong CYP3A4 induction on decreasing the systemic exposure of mirdametinib and to provide dosage recommendations for mirdametinib when used concomitantly with strong CYP3A4 inducers. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  M12 Drug Interaction Studies. "~"Pending"~~4/30/2027 0:00:00~4/8/2026 0:00:00
385135~"CDER"~"NDA"~219407.00~"IONIS PHARMACEUTICALS INC"~"Dawnzera (donidalorsen) Injection"~8/21/2025 0:00:00~"Original"~1~"4862-1"~"PMR"~"505 (o)(3)"~4862.00~1~"PMR 4862-1: Complete the ongoing four-year open-label extension (OLE) trial (ISIS 721744-CS3) and the three-year OLE (ISIS 721744-CS7) to evaluate the potential for drug induced liver injury with donidalorsen."~"Ongoing"~~9/30/2027 0:00:00~
385136~"CDER"~"NDA"~219407.00~"IONIS PHARMACEUTICALS INC"~"Dawnzera (donidalorsen) Injection"~8/21/2025 0:00:00~"Original"~1~"4862-2"~"PMR"~"505 (o)(3)"~4862.00~2~"PMR 4862-2: Complete the ongoing 2-year carcinogenicity study in rats administered Donidalorsen."~"Submitted"~~2/28/2026 0:00:00~
385137~"CDER"~"NDA"~219428.00~"TONIX PHARMACEUTICALS INC"~"Tonmya (cyclobenzaprine) sublingual tablet"~8/15/2025 0:00:00~"Original"~1~"4872-1"~"PMR"~"Pediatric Research Equity Act"~4872.00~1~"PMR 4872-1: Conduct a juvenile animal study in the rat model to characterize the impact of Tonmya (cyclobenzaprine) on the developing brain and reproductive tissues to support clinical studies in pediatric patients from 13 years of age to less than 17 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~10/31/2026 0:00:00~
385138~"CDER"~"NDA"~219428.00~"TONIX PHARMACEUTICALS INC"~"Tonmya (cyclobenzaprine) sublingual tablet"~8/15/2025 0:00:00~"Original"~1~"4872-2"~"PMR"~"Pediatric Research Equity Act"~4872.00~2~"PMR 4872-2: Conduct a randomized controlled study evaluating the pharmacokinetics, safety, and efficacy of Tonmya (cyclobenzaprine) in pediatric patients 13 years of age to less than 17 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~5/31/2030 0:00:00~
385139~"CDER"~"NDA"~219428.00~"TONIX PHARMACEUTICALS INC"~"Tonmya (cyclobenzaprine) sublingual tablet"~8/15/2025 0:00:00~"Original"~1~"4872-3"~"PMR"~"Pediatric Research Equity Act"~4872.00~3~"PMR 4872-3: Conduct a clinical open-label extension safety study of Tonmya (cyclobenzaprine) in pediatric patients 13 years of age to less than 17 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2031 0:00:00~
385140~"CDER"~"NDA"~219428.00~"TONIX PHARMACEUTICALS INC"~"Tonmya (cyclobenzaprine) sublingual tablet"~8/15/2025 0:00:00~"Original"~1~"4872-4"~"PMR"~"505 (o)(3)"~4872.00~4~"PMR 4872-4: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Tonmya (cyclobenzaprine) during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant."~"Pending"~~2/28/2037 0:00:00~
385141~"CDER"~"BLA"~761067.00~"Sun Pharmaceutical Industries Limited "~"Ilumya (tildrakizumab-asmn)"~3/20/2018 0:00:00~"Original"~1~"3357-3"~"PMR"~"505 (o)(3)"~3357.00~3~"PMR 3357-3: Conduct a retrospective cohort study using claims or electronic medical record data or a case control study to assess major congenital malformations, spontaneous abortions, stillbirths, small for gestational age, neonatal deaths, and infant infections in women exposed to tildrakizumab-asmn during pregnancy compared to an unexposed control population."~"Ongoing"~~1/31/2027 0:00:00~5/18/2026 0:00:00
385142~"CDER"~"BLA"~761067.00~"Sun Pharmaceutical Industries Limited "~"Ilumya (tildrakizumab-asmn)"~3/20/2018 0:00:00~"Original"~1~"3357-4"~"PMR"~"505 (o)(3)"~3357.00~4~"PMR 3357-4: Conduct an observational study to assess the long-term safety of tildrakizumabasmn compared to other therapies used in the treatment of adults with moderateto- severe plaque psoriasis who are candidates for systemic therapy or phototherapy in the course of actual clinical care. The study s primary outcome is the long-term risk of malignancy. Secondary outcomes include, but are not limited to, serious infections, tuberculosis, opportunistic infections, hypersensitivity reactions, autoimmune disease, neurologic or demyelinating disease, cardiovascular, gastrointestinal and hematologic adverse events. Describe and justify the choice of appropriate comparator populations(s) and estimated background rate(s) relative to tildrakizumab-exposed patients; clearly define the primary comparator population for the primary objective. Design the study around a testable hypothesis to assess, with sufficient sample size and power, a clinically meaningful increase in malignancy risk above the comparator background rate(s), with a pre-specified statistical analysis method. Specify concise case definitions and validation algorithms for both primary and secondary outcomes. For the tildrakizumab-exposed and comparator(s) cohorts, clearly define the study drug initiation period and any exclusion and inclusion criteria. Enroll patients over an initial 4-year period and follow for a minimum of 8 years from the time of enrollment."~"Ongoing"~~2/28/2034 0:00:00~5/18/2026 0:00:00
385143~"CDER"~"BLA"~761068.00~"Kyowa Kirin, Inc."~"Crysvita (burosumab-twza)"~4/17/2018 0:00:00~"Original"~1~"3370-1"~"PMR"~"505 (o)(3)"~3370.00~1~"PMR 3370-1: A post-approval surveillance program with safety objectives of evaluating the potential association between burosumab treatment and the risks of nephrocalcinosis, renal failure, and spinal stenosis. Pregnancy exposure data, including maternal, neonatal and infant outcomes, will also be collected and analyzed. This program will be incorporated into the X-linked Hypophosphatemia Disease Monitoring Program (study UX023-CL401) that collects information on the disease for up to 10 years. Safety data collection will begin within 90 days of protocol agreement. After marketing starts, submit progress reports to the FDA at six months, one year, and annually thereafter, with an evaluation of the effectiveness of meeting the surveillance program s safety objectives. Collect surveillance data from a minimum of 500 subjects, 200 of whom will be pediatric patients, and approximately two-thirds will be treated with burosumab."~"Ongoing"~~8/31/2029 0:00:00~4/29/2026 0:00:00
385144~"CDER"~"BLA"~761068.00~"Kyowa Kirin, Inc."~"Crysvita (burosumab-twza)"~4/17/2018 0:00:00~"Original"~1~"3370-2"~"PMR"~"505 (o)(3)"~3370.00~2~"PMR 3370-2: In XLH patients enrolled in the prospective, longitudinal, surveillance study, perform a lactation sub-study in lactating women who have received therapeutic doses of burosumab using a validated assay to assess concentrations of burosumab in breast milk, the effects on milk composition (to include calcium and phosphorus levels), and the effects on the breastfed infant"~"Ongoing"~~8/31/2029 0:00:00~4/29/2026 0:00:00
385145~"CDER"~"BLA"~761068.00~"Kyowa Kirin, Inc."~"Crysvita (burosumab-twza)"~4/17/2018 0:00:00~"Supplement"~5~"3881-1"~"PMR"~"505 (o)(3)"~3881.00~1~"PMR 3881-1: A post-approval surveillance program in adult and pediatric patients with tumor-induced osteomalacia, with safety objectives of evaluating the potential association between burosumab treatment and the risks of nephrocalcinosis, renal failure, and spinal stenosis; and monitoring the effect of burosumab treatment on the course of the underlying phosphaturic mesenchymal tumor over time. Pregnancy exposure data, including maternal, neonatal and infant outcomes, will also be collected and analyzed. This program will be incorporated into the Tumor-induced Osteomalacia Disease Monitoring Program (study UX023T-CL403) that collects information on the disease for up to 10 years. Safety data collection will begin within 90 days of protocol agreement. After marketing starts, submit progress reports to the FDA at six months, one year, and annually thereafter, with an evaluation of the effectiveness of meeting the surveillance program s safety objectives. Collect surveillance data from a minimum of 20 subjects, of whom approximately two-thirds will be treated with burosumab."~"Ongoing"~~2/28/2032 0:00:00~4/29/2026 0:00:00
385146~"CDER"~"BLA"~761069.00~"AstraZeneca UK Ltd"~"Imfinzi (Durvalumab)"~5/1/2017 0:00:00~"Supplement"~43~"4679-1"~"PMC"~"506B PMC"~4679.00~1~"PMC 4679-1: Complete the ongoing clinical trial, AEGEAN (NCT04538664), and analyze the final overall survival (OS) once the required 371 events for the primary endpoint (event-free survival) have occurred, to further characterize the clinical benefit of durvalumab in combination with platinum-containing chemotherapy as neoadjuvant treatment, followed by durvalumab continued as a single agent as adjuvant treatment after surgery for adults with resectable (tumors = 4 cm or node positive) nonsmall cell lung cancer (NSCLC) and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements."~"Pending"~~6/30/2029 0:00:00~6/22/2026 0:00:00
385147~"CDER"~"BLA"~761069.00~"AstraZeneca UK Ltd"~"Imfinzi (Durvalumab)"~5/1/2017 0:00:00~"Supplement"~43~"4679-2"~"PMC"~"506B PMC"~4679.00~2~"PMC 4679-2: Conduct an integrated analysis of completed, ongoing, and planned trials of perioperative durvalumab for patients with resectable non-small cell lung cancer (NSCLC) to evaluate the contribution of phase of durvalumab when given in combination with platinum-containing chemotherapy as neoadjuvant treatment and continued as a single agent as adjuvant treatment after surgery for adults with resectable (tumors = 4 cm or node positive) NSCLC and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements. This may include data from the following trials: AEGEAN (NCT04538664),BR.31 (NCT02273375), and ADOPT-lung (NCT06284317). This analysis should also include an evaluation of safety, including immune mediated adverse reactions that occur during each phase of treatment."~"Pending"~~6/30/2031 0:00:00~6/22/2026 0:00:00
385148~"CDER"~"BLA"~761123.00~"AstraZeneca AB"~"Saphnelo (anifrolumab-fnia)"~7/30/2021 0:00:00~"Original"~1~"4116-3"~"PMR"~"505 (o)(3)"~4116.00~3~"PMR 4116-3: Conduct a retrospective cohort study in a claims-based database to evaluate the effects of Saphnelo (anifrolumab-fnia) on pregnancy-related outcomes."~"Delayed"~"The final protocol submission milestone was missed due to continued discussion with the Agency."~4/30/2032 0:00:00~9/25/2025 0:00:00
385149~"CDER"~"BLA"~761123.00~"AstraZeneca AB"~"Saphnelo (anifrolumab-fnia)"~7/30/2021 0:00:00~"Original"~1~"4116-4"~"PMR"~"505 (o)(3)"~4116.00~4~"PMR 4116-4: Perform a lactation study, milk only, in lactating women who have received Saphnelo (anifrolumab-fnia) to assess concentrations of anifrolumab-fnia in breast milk using a validated assay. A mother-infant pair study may be required in the future depending on the results of this milk-only study."~"Delayed"~"The study completion milestone was missed because of delays in reaching an agreement for the Final Protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 12/12/2024."~2/28/2026 0:00:00~9/25/2025 0:00:00
385150~"CDER"~"BLA"~761126.00~"Tanvex BioPharma USA, Inc."~"Nypozi (filgrastim-txid)"~6/28/2024 0:00:00~"Original"~1~"4399-1"~"PMR"~"Pediatric Research Equity Act"~4399.00~1~"PMR 4399-1: To develop a vial presentation that can be used to directly and accurately administer Nypozi (filgrastim-txid) to pediatric patients who weigh less than 36 kg requiring doses that are less than 0.3 mL (180 mcg)."~"Delayed"~"A Not Fulfilled letter was issued 02/25/2026."~2/28/2025 0:00:00~
385151~"CDER"~"BLA"~761128.00~"Novartis Pharmaceuticals Corporation"~"Adakveo (crizanlizumab-tmca)"~11/15/2019 0:00:00~"Original"~1~"3741-2"~"PMR"~"505 (o)(3)"~3741.00~2~"PMR 3741-2: Assess neutralizing anti-drug antibody (NADA) responses with a validated NADA assay. NADA response will be evaluated in all confirmed ADA positive samples from studies CSEG101 A2102, A2202 and the primary analysis of A2301."~"Submitted"~~12/31/2025 0:00:00~1/9/2026 0:00:00
385152~"CDER"~"BLA"~761128.00~"Novartis Pharmaceuticals Corporation"~"Adakveo (crizanlizumab-tmca)"~11/15/2019 0:00:00~"Original"~1~"3741-4"~"PMR"~"505 (o)(3)"~3741.00~4~"PMR 3741-4: Complete Study B2201: Phase II, Multicenter, Open-label study to Access Appropriate Dosing and to Evaluate Safety of Crizanlizumab, with or without Hydroxyurea in Sequential, Descending Age Groups of Pediatric Sickle Cell Disease Patients with Vasoocclusive Crises and evaluate the serious risks of infusion related reactions, bleeding complications, and infections."~"Submitted"~~12/31/2025 0:00:00~1/9/2026 0:00:00
385153~"CDER"~"BLA"~761128.00~"Novartis Pharmaceuticals Corporation"~"Adakveo (crizanlizumab-tmca)"~11/15/2019 0:00:00~"Original"~1~"3741-6"~"PMR"~"505 (o)(3)"~3741.00~6~"PMR 3741-6: Assess immunogenicity of crizanlizumab, including anti-drug antibodies (ADA) and neutralizing anti-drug antibodies (NADA) in all crizanlizumabtreated subjects in Study A2301. Evaluate the effect of immunogenicity on pharmacokinetics, pharmacodynamics, safety and efficacy of crizanlizumab."~"Submitted"~~12/31/2025 0:00:00~1/9/2026 0:00:00
385154~"CDER"~"BLA"~761128.00~"Novartis Pharmaceuticals Corporation"~"Adakveo (crizanlizumab-tmca)"~11/15/2019 0:00:00~"Original"~1~"3741-8"~"PMC"~"506B PMC"~3741.00~8~"PMC 3741-8: Submit an Integrated Summary of Immunogenicity that describes the totality of the immunogenicity program, as recommended in Section VIII Documentation of the 2019 FDA Guidance for Industry: Immunogenicity Testing of Therapeutic Protein Products   Developing and Validating Assays for Anti-Drug Antibody Detection."~"Submitted"~~12/31/2025 0:00:00~1/9/2026 0:00:00
385155~"CDER"~"BLA"~761128.00~"Novartis Pharmaceuticals Corporation"~"Adakveo (crizanlizumab-tmca)"~11/15/2019 0:00:00~"Supplement"~6~"4658-1"~"PMC"~"506B PMC"~4658.00~1~"PMC 4658-1: Conduct a randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo in patients aged 12 years or older with sickle cell disease and who have had at least 4 vasoocclusive crises events (VOCs) in the 12 months prior to enrollment. The primary endpoint is the annualized rate of VOCs requiring management by a health care provider over the 52-week treatment period post randomization. Safety outcomes of interest include infusion related reactions, bleeding complications, and infections (serious, non-serious and opportunistic)."~"Ongoing"~~4/30/2031 0:00:00~1/9/2026 0:00:00
385156~"CDER"~"BLA"~761133.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Entyvio (vedolizumab)"~9/27/2023 0:00:00~"Original"~1~"4493-2"~"PMR"~"Pediatric Research Equity Act"~4493.00~2~"PMR 4493-2: A study to evaluate the pharmacokinetics, immunogenicity, and safety of Entyvio (vedolizumab) as a subcutaneous injection for maintenance treatment in pediatric patients =2 to <18 years of age with ulcerative colitis or Crohn s disease who achieve clinical response following induction treatment with intravenous Entyvio (vedolizumab) for injection."~"Delayed"~"The final protocol was submitted after the original milestone and was acknowledged per FDA letter dated 4/16/2024. 4/70 subjects have been enrolled."~12/31/2028 0:00:00~7/15/2025 0:00:00
385157~"CDER"~"BLA"~761133.00~"Takeda Pharmaceuticals U.S.A., Inc."~"Entyvio (vedolizumab)"~9/27/2023 0:00:00~"Original"~1~"4493-3"~"PMR"~"Pediatric Research Equity Act"~4493.00~3~"PMR 4493-3: A study to evaluate the long-term safety of Entyvio (vedolizumab) as a subcutaneous injection in pediatric subjects =2 to <18 years of age with ulcerative colitis or Crohn s disease."~"Ongoing"~"The final protocol was acknowledged per FDA letter dated 8/13/2024."~12/31/2033 0:00:00~7/15/2025 0:00:00
385158~"CDER"~"BLA"~761134.00~"Evive Biotechnology Singapore PTE. LTD."~"Ryzneuta 
"~11/16/2023 0:00:00~"Original"~1~"4530-1"~"PMR"~"Pediatric Research Equity Act"~4530.00~1~"PMR 4530-1: Conduct a multicenter, open-label, study to assess the safety, pharmacokinetics and pharmacodynamics of efbemalenograstim alfa in at least 40 pediatric patients 1 month to <17 years of age with non-myeloid solid tumors or lymphomas receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia."~"Delayed"~"Deferral Extension Requested 04/30/2026. Denied per FDA letter dated 06/10/2026."~12/31/2028 0:00:00~1/13/2026 0:00:00
385159~"CDER"~"BLA"~761134.00~"Evive Biotechnology Singapore PTE. LTD."~"Ryzneuta 
"~11/16/2023 0:00:00~"Original"~1~"4530-2"~"PMR"~"Pediatric Research Equity Act"~4530.00~2~"PMR 4530-2: Submit pediatric assessments for Ryzneuta (efbemalenograstim alfa) as described in section 505B(a)(2)(A) of the FD&C Act,
including development of an  appropriate formulation  (presentation) that can be used to directly and accurately administer Ryzneuta (efbemalenograstim alpha) to pediatric patients (1 month to <17 years of age). Conduct any necessary human factors studies to evaluate the ability of healthcare providers and caregivers to measure the appropriate doses. The final report should include a report for the pediatric presentation development and the completed human factors study."~"Delayed"~"Original Final Report Due Date: 12/31/2025; Deferral Extension request was received after the milestone was missed. Deferral Extension granted per FDA letter dated 06/10/2026."~7/31/2026 0:00:00~1/13/2026 0:00:00
385160~"CDER"~"BLA"~761134.00~"Evive Biotechnology Singapore PTE. LTD."~"Ryzneuta 
"~11/16/2023 0:00:00~"Original"~1~"4530-3"~"PMC"~"506B PMC"~4530.00~3~"PMC 4530-3: Conduct a study to assess the efficacy and safety of efbemalenograstim alfa in pediatric patients 1 month to <17 years of age with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia. Submit the final clinical study report including datasets as a supplemental BLA."~"Pending"~~2/28/2032 0:00:00~1/13/2026 0:00:00
385161~"CDER"~"BLA"~761279.00~"Eli Lilly and Company"~"Omvoh (mirikizumab-mrkz)"~10/26/2023 0:00:00~"Original"~1~"4409-2"~"PMR"~"505 (o)(3)"~4409.00~2~"PMR 4409-2: Conduct a prospective, registry-based, observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to mirikizumab-containing products regardless of indication during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age births, preterm births, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, neonatal deaths, and infections, will be assessed through at least the first year of life."~"Delayed"~"The final protocol milestone was missed. Per the applicant annual status report, an amended draft protocol was submitted in June 2025 for review."~12/31/2035 0:00:00~10/9/2025 0:00:00
385162~"CDER"~"BLA"~761279.00~"Eli Lilly and Company"~"Omvoh (mirikizumab-mrkz)"~10/26/2023 0:00:00~"Original"~1~"4409-3"~"PMR"~"505 (o)(3)"~4409.00~3~"PMR 4409-3: Conduct an additional pregnancy study that uses a different design from the prospective pregnancy registry established to fulfill postmarketing requirement 4409-2 (for example a retrospective cohort study using claims or electronic medical record data with outcome validation or a case-control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm births in women exposed to mirikizumab-containing products regardless of indication during pregnancy compared to an unexposed control population."~"Delayed"~"The final protocol milestone was missed. Per the applicant annual status report, an amended draft protocol was submitted in June 2025 for review."~12/31/2031 0:00:00~10/9/2025 0:00:00
385163~"CDER"~"BLA"~761279.00~"Eli Lilly and Company"~"Omvoh (mirikizumab-mrkz)"~10/26/2023 0:00:00~"Original"~1~"4409-4"~"PMR"~"505 (o)(3)"~4409.00~4~"PMR 4409-4: Conduct an observational study to assess the incidence of severe acute liver injury in adults with moderately to severely active ulcerative colitis who are exposed to Omvoh (mirikizumab-mrkz), relative to other therapies used to treat ulcerative colitis. Compare rates (per person-time) or risks (proportion of patients with a minimum amount of follow-up). Describe and justify the choice of appropriate comparator population(s). Specify concise case definition for severe liver injury and validation of algorithm(s) to identify severe liver injury in the proposed data source. For the Omvoh (mirikizumab-mrkz)-exposed and comparator(s) cohorts, clearly define the study drug initiation period and any exclusion and inclusion criteria. Ensure that the data source allows for average follow-up for at least 1 year. Specify a minimum sample size and justify the precision of the estimate achievable with the proposed study."~"Delayed"~"The final protocol milestone was missed. Per the applicant annual status report, an amended draft protocol was submitted in August 2025 for review."~6/30/2037 0:00:00~10/9/2025 0:00:00
385164~"CDER"~"BLA"~761279.00~"Eli Lilly and Company"~"Omvoh (mirikizumab-mrkz)"~10/26/2023 0:00:00~"Original"~1~"4409-6"~"PMC"~"506B PMC"~4409.00~6~"PMC 4409-6: Conduct a one-year trial to evaluate the safety, efficacy, and pharmacokinetics of Omvoh (mirikizumab-mrkz) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis."~"Ongoing"~~10/31/2029 0:00:00~10/9/2025 0:00:00
385165~"CDER"~"BLA"~761279.00~"Eli Lilly and Company"~"Omvoh (mirikizumab-mrkz)"~10/26/2023 0:00:00~"Original"~1~"4409-7"~"PMC"~"506B PMC"~4409.00~7~"PMC 4409-7: Conduct a long-term extension trial to evaluate the long-term safety of Omvoh (mirikizumab-mrkz) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis who participated in the postmarketing commitment 4409-6. This study can be conducted as part of the postmarketing commitment 4409-6."~"Ongoing"~~10/31/2034 0:00:00~10/9/2025 0:00:00
385166~"CDER"~"BLA"~761279.00~"Eli Lilly and Company"~"Omvoh (mirikizumab-mrkz)"~10/26/2023 0:00:00~"Supplement"~2~"4770-1"~"PMR"~"505 (o)(3)"~4770.00~1~"PMR 4770-1: Conduct an observational study to assess the incidence of severe acute liver injury in adults with moderately to severely active Crohn s disease who are exposed to Omvoh (mirikizumab-mrkz), relative to other therapies used to treat Crohn s disease. Compare rates (per person-time) or risks (proportion of patients with a minimum amount of follow-up). Describe and justify the choice of appropriate comparator population(s). Specify concise case definition for severe liver injury and validation of algorithm(s) to identify severe liver injury in the proposed data source. For the Omvoh (mirikizumab-mrkz)-exposed and comparator(s) cohorts, clearly define the study drug initiation period and any inclusion and exclusion criteria. Ensure that the data source allows for average follow-up for at least 1 year. Specify a minimum sample size and justify the precision of the estimate achievable with the proposed study. The ongoing observational study in patients with ulcerative colitis (PMR 4409-4) with the same objectives may be amended to also enroll patients with Crohn s disease."~"Pending"~~6/30/2037 0:00:00~10/9/2025 0:00:00
385167~"CDER"~"BLA"~761279.00~"Eli Lilly and Company"~"Omvoh (mirikizumab-mrkz)"~10/26/2023 0:00:00~"Supplement"~2~"4770-2"~"PMC"~"506B PMC"~4770.00~2~"PMC 4770-2: Complete the ongoing one-year phase 3 trial to evaluate the safety, efficacy, and pharmacokinetics of Omvoh (mirikizumab-mrkz) in pediatric patients 2 to 17 years of age with moderately to severely active CD."~"Pending"~~10/31/2029 0:00:00~10/9/2025 0:00:00
385168~"CDER"~"BLA"~761279.00~"Eli Lilly and Company"~"Omvoh (mirikizumab-mrkz)"~10/26/2023 0:00:00~"Supplement"~2~"4770-3"~"PMC"~"506B PMC"~4770.00~3~"PMC 4770-3: Complete the long-term extension trial to evaluate the long-term safety of Omvoh (mirikizumab-mrkz) in pediatric patients 2 to 17 years of age with moderately to severely active CD who participated in the postmarketing commitment 4770-2."~"Pending"~~10/31/2034 0:00:00~10/9/2025 0:00:00
385169~"CDER"~"BLA"~761286.00~"UCB, Inc."~"Rystiggo (rozanolixizumab-noli)"~6/26/2023 0:00:00~"Original"~1~"4427-1"~"PMR"~"505 (o)(3)"~4427.00~1~"PMR 4427-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to rozanolixizumab-noli during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~1/31/2036 0:00:00~8/22/2025 0:00:00
385170~"CDER"~"BLA"~761286.00~"UCB, Inc."~"Rystiggo (rozanolixizumab-noli)"~6/26/2023 0:00:00~"Original"~1~"4427-2"~"PMR"~"505 (o)(3)"~4427.00~2~"PMR 4427-2: Perform a lactation study (milk only) in lactating women who have received therapeutic doses of rozanolixizumab-noli using a validated assay to assess concentrations of rozanolixizumab-noli in breast milk and the effects on the breastfed infant as applicable"~"Ongoing"~~7/31/2026 0:00:00~8/22/2025 0:00:00
385171~"CDER"~"NDA"~219474.00~"ACROTECH BIOPHARMA INC"~"Adquey (difamilast) ointment"~2/12/2026 0:00:00~"Original"~1~"4963-1"~"PMR"~"Pediatric Research Equity Act"~4963.00~1~"PMR 4963-1: Conduct an adequate and well-controlled trial in the United States in subjects ages 3 months to <2 years with mild to moderate atopic dermatitis. Evaluate the safety and pharmacokinetics of difamilast under maximal use conditions in an adequate number of subjects with disease at the upper range of severity."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2029 0:00:00~
385172~"CDER"~"NDA"~219491.00~"ENTASIS THERAPEUTICS INC"~"Nuzolvence (zoliflodacin) "~12/12/2025 0:00:00~"Original"~1~"4942-1"~"PMR"~"505 (o)(3)"~4942.00~1~"PMR 4942-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Nuzolvence (zoliflodacin) during pregnancy to assess risk of maternal complications and adverse effects on the developing fetus, neonate, and infant during the first year of life."~"Pending"~~1/31/2037 0:00:00~
385173~"CDER"~"NDA"~219491.00~"ENTASIS THERAPEUTICS INC"~"Nuzolvence (zoliflodacin) "~12/12/2025 0:00:00~"Original"~1~"4942-2"~"PMR"~"505 (o)(3)"~4942.00~2~"PMR 4942-2: Perform a lactation study (milk only or mother-infant pair study) in lactating women who have received Nuzolvence (zoliflodacin) to measure concentrations of zoliflodacin in breast milk using a validated assay. Assess the effects on the breastfed infant, if available, based on the study population."~"Pending"~~6/30/2028 0:00:00~
385174~"CDER"~"NDA"~219491.00~"ENTASIS THERAPEUTICS INC"~"Nuzolvence (zoliflodacin) "~12/12/2025 0:00:00~"Original"~1~"4942-3"~"PMR"~"505 (o)(3)"~4942.00~3~"PMR 4942-3: Conduct a U.S. surveillance study over a five-year period after the introduction of Nuzolvence (zoliflodacin) to the market to determine if resistance or decreased susceptibility to Nuzolvence is occurring in the target population of bacteria that are in the approved Nuzolvence label."~"Pending"~~6/30/2033 0:00:00~
385175~"CDER"~"NDA"~219491.00~"ENTASIS THERAPEUTICS INC"~"Nuzolvence (zoliflodacin) "~12/12/2025 0:00:00~"Original"~1~"4942-4"~"PMR"~"505 (o)(3)"~4942.00~4~"PMR 4942-4: Conduct a clinical trial to evaluate the effect of Nuzolvence (zoliflodacin) on human testicular function."~"Pending"~~6/30/2028 0:00:00~
385176~"CDER"~"NDA"~219500.00~"CORSTASIS THERAPEUTICS INC"~"Enbumyst (bumetanide) Nasal Spray"~9/12/2025 0:00:00~"Original"~1~"4898-1"~"PMR"~"Pediatric Research Equity Act"~4898.00~1~"PMR 4898-1: Develop a new formulation that can be used to directly administer bumetanide nasal spray to pediatric patients who weigh less than 15 kg. This weight band includes neonates."~"Ongoing"~"The study/trial is underway."~9/30/2026 0:00:00~
385177~"CDER"~"NDA"~219500.00~"CORSTASIS THERAPEUTICS INC"~"Enbumyst (bumetanide) Nasal Spray"~9/12/2025 0:00:00~"Original"~1~"4898-2"~"PMR"~"Pediatric Research Equity Act"~4898.00~2~"PMR 4898-2: Conduct a study to evaluate the pharmacokinetics, pharmacodynamics, and safety of bumetanide nasal spray in pediatric patients who weigh less than 15 kg with edema associated with congestive heart failure, hepatic and renal disease, including the nephrotic syndrome."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~11/30/2029 0:00:00~
385178~"CDER"~"NDA"~219500.00~"CORSTASIS THERAPEUTICS INC"~"Enbumyst (bumetanide) Nasal Spray"~9/12/2025 0:00:00~"Original"~1~"4898-3"~"PMR"~"Pediatric Research Equity Act"~4898.00~3~"PMR 4898-3: Conduct a study to evaluate the pharmacokinetics, pharmacodynamics, and safety of bumetanide nasal spray in pediatric patients who weigh greater than or equal to 15 kg with edema associated with congestive heart failure, hepatic and renal disease, including the nephrotic syndrome."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~9/30/2029 0:00:00~
385179~"CDER"~"NDA"~219500.00~"CORSTASIS THERAPEUTICS INC"~"Enbumyst (bumetanide) Nasal Spray"~9/12/2025 0:00:00~"Original"~1~"4898-4"~"PMR"~"505 (o)(3)"~4898.00~4~"PMR 4898-4: Perform a milk-only lactation study in lactating women who have received bumetanide nasal spray to measure concentrations of bumetanide in breast milk using a validated assay."~"Pending"~~9/30/2028 0:00:00~
385180~"CDER"~"NDA"~219500.00~"CORSTASIS THERAPEUTICS INC"~"Enbumyst (bumetanide) Nasal Spray"~9/12/2025 0:00:00~"Original"~1~"4898-5"~"PMC"~"506B PMC"~4898.00~5~"PMC 4898-5: Conduct a PK comparison study in adult subjects under nasal allergen challenge conditions to evaluate the impact of nasal mucosa condition on the absorption of bumetanide."~"Pending"~~3/31/2028 0:00:00~
385181~"CDER"~"NDA"~219531.00~"BRILLIAN PHARMA INC"~"Sdamlo (amlodipine) powder for oral solution"~7/24/2025 0:00:00~"Original"~1~"4873-1"~"PMR"~"Pediatric Research Equity Act"~4873.00~1~"PMR 4873-1: Conduct a dose-ranging, safety, tolerability, and efficacy study of amlodipine oral solution for the treatment of hypertension in pediatric patients birth to <6 years of age. The study protocol should be agreed with the FDA prior to initiation of the study."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~3/31/2031 0:00:00~
385182~"CDER"~"NDA"~219428.00~"TONIX PHARMACEUTICALS INC"~"Tonmya (cyclobenzaprine) sublingual tablet"~8/15/2025 0:00:00~"Original"~1~"4872-5"~"PMR"~"505 (o)(3)"~4872.00~5~"PMR 4872-5: Perform a milk-only lactation study in lactating women who have received Tonmya (cyclobenzaprine) to measure concentrations of cyclobenzaprine in breast milk using a validated assay. Assess the effects on the breastfed infant based on study population."~"Pending"~~2/28/2029 0:00:00~
385183~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-1"~"PMR"~"Accelerated Approval"~4835.00~1~"PMR 4835-1: Complete the ongoing trial titled,  RAMP 301: A Phase 3, Randomized, Open-Label Study of Combination Therapy with Avutometinib plus Defactinib Versus Investigator s Choice of Treatment in Patients with Recurrent Low-Grade Serous Ovarian Cancer (LGSOC) , and provide the progression-free survival and the final overall survival analyses, intended to describe and verify the clinical benefit of avutometinib and defactinib in combination in adult patients with recurrent, KRAS-mutated low grade serous ovarian cancer. Include central KRAS testing results for all patients."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2028 0:00:00~6/26/2026 0:00:00
385184~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-2"~"PMR"~"Pediatric Research Equity Act"~4835.00~2~"PMR 4835-2: Conduct a molecularly targeted pediatric investigation of avutometinib as a single agent and in combination with defactinib to evaluate preliminary efficacy, and characterize dosing, pharmacokinetics/pharmacodynamics and preliminary safety in a sufficient number of pediatric patients ages 2 to <17 years of age with relapsed or refractory unresectable or metastatic RAS/MAPK pathway-driven pediatric cancers."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~9/30/2034 0:00:00~6/26/2026 0:00:00
385185~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-3"~"PMR"~"505 (o)(3)"~4835.00~3~"PMR 4835-3: Conduct a clinical pharmacokinetic trial or conduct a physiologically-based PK (PBPK) modeling assessing the effect of multiple doses of a moderate CYP3A4 inhibitor on the single dose PK of defactinib and its active metabolite M4 to evaluate the potential serious risk of increased serious adverse reactions and identify a proposed dosage of defactinib in patients taking defactinib concomitant with moderate CYP3A4 inhibitors. Design and conduct the trial or PBPK analysis in accordance with the ICH Harmonized Guidance entitled  M12 Drug Interaction Studies."~"Pending"~~12/31/2026 0:00:00~6/26/2026 0:00:00
385186~"CDER"~"BLA"~761069.00~"AstraZeneca UK Ltd"~"Imfinzi (Durvalumab)"~5/1/2017 0:00:00~"Supplement"~45~"4653-1"~"PMC"~"506B PMC"~4653.00~1~"PMC 4653-1: Complete the ongoing clinical trial, DUO-E, titled  A Randomised, Multicenter, Double-blind, Placebo-controlled, Phase III Study of First-line Carboplatin and Paclitaxel in Combination with Durvalumab, Followed by Maintenance Durvalumab with or without Olaparib in Patients with Newly Diagnosed Advanced or Recurrent Endometrial Cancer (DUO-E) , to provide the pre-specified interim and final overall survival analyses."~"Pending"~~12/31/2026 0:00:00~6/22/2026 0:00:00
385187~"CDER"~"BLA"~761069.00~"AstraZeneca UK Ltd"~"Imfinzi (Durvalumab)"~5/1/2017 0:00:00~"Supplement"~49~"4754-1"~"PMC"~"506B PMC"~4754.00~1~"PMC 4754-1: Conduct an integrated analysis of data from clinical trials and observational studies (e.g., real world evidence), post-marketing reports, and other sources to further characterize the safety, and efficacy/effectiveness of durvalumab in Black/African American patients with small cell lung cancer. The analyses should include an evaluation of the comparative efficacy and safety between the populations primarily represented in the ADRIATIC (NCT03703297) and CASPIAN (NCT03043872) trials and in Black/African American patients."~"Pending"~~9/30/2032 0:00:00~6/22/2026 0:00:00
385188~"CDER"~"BLA"~761069.00~"AstraZeneca UK Ltd"~"Imfinzi (Durvalumab)"~5/1/2017 0:00:00~"Supplement"~50~"4823-1"~"PMC"~"506B PMC"~4823.00~1~"PMC 4823-1: Complete the ongoing clinical trial, NIAGARA (NCT03732677), titled  A Phase 3, Randomized, Open-Label, Multi-Center, Global Study to Determine the Efficacy and Safety of Durvalumab in Combination With Gemcitabine+Cisplatin for Neoadjuvant Treatment Followed by Durvalumab Alone for Adjuvant Treatment in Patients With Muscle-Invasive Bladder Cancer , and provide the pre-planned final overall survival analysis, to further characterize the clinical benefit of durvalumab in combination with gemcitabine and cisplatin as neoadjuvant treatment, followed by durvalumab as a single agent as adjuvant treatment after radical cystectomy for the treatment of adult patients with muscle invasive bladder cancer."~"Pending"~~6/30/2028 0:00:00~6/22/2026 0:00:00
385189~"CDER"~"BLA"~761071.00~"Sandoz Inc."~"Hyrimoz (adalimumab-adaz)"~10/30/2018 0:00:00~"Original"~1~"3506-3"~"PMR"~"Pediatric Research Equity Act"~3506.00~3~"PMR 3506-3: Assessment of Hyrimoz (adalimumab-adaz) for the treatment of pediatric ulcerative colitis (UC) in pediatric patients 5 years to 17 years of age"~"Delayed"~"Original Final Report Due Date: December 2020. Per FDA letter dated August 18, 2020, Final Report due date extended to September 2021. Second FDA Deferral Extension letter dated September 1, 2021, extended to February 2028."~2/28/2028 0:00:00~12/11/2025 0:00:00
385190~"CDER"~"BLA"~761075.00~"Biocon Biologics Inc."~"Fulphila (pegfilgrastim-jmdb)"~6/4/2018 0:00:00~"Original"~1~"3732-1"~"PMR"~"Pediatric Research Equity Act"~3732.00~1~"PMR 3732-1: Submit pediatric assessments for Fulphila (pegfilgrastim-jmdb) as described in section 505B(a)(2)(A) of the FD&C Act, including development of an  appropriate formulation  (presentation) that can be used to directly and accurately administer Fulphila (pegfilgrastim-jmdb) to pediatric patients who weigh less than 45 kg and require doses that are less than 0.6 mL (6 mg), and conducting any necessary human factors studies to evaluate the ability of healthcare providers and/or caregivers to measure the appropriate doses."~"Pending"~"The study has not been initiated, but it does not meet the
criterion for delayed."~4/30/2028 0:00:00~8/1/2025 0:00:00
385191~"CDER"~"BLA"~761077.00~"Amgen Inc."~"Aimovig (erenumab-aooe)"~5/17/2018 0:00:00~"Original"~1~"3392-2"~"PMR"~"Pediatric Research Equity Act"~3392.00~2~"PMR 3392-2: An open-label pharmacokinetic, safety, and tolerability study in pediatric migraine patients ages 6 through 11 years. Dosing will depend on body weight, according to two weight bands: <40 kg and =40 kg. The study should identify doses that provide exposures that match those observed with the 70-mg and 140- mg doses of Aimovig in adults."~"Submitted"~"Original Final Report Due Date: 12/31/2025; Deferral Extension granted per FDA letter dated 06/13/2024. The final study report was submitted to the IND with a cross reference letter to the application on 8/11/2022."~10/31/2027 0:00:00~7/28/2025 0:00:00
385192~"CDER"~"BLA"~761077.00~"Amgen Inc."~"Aimovig (erenumab-aooe)"~5/17/2018 0:00:00~"Original"~1~"3392-3"~"PMR"~"Pediatric Research Equity Act"~3392.00~3~"PMR 3392-3: Deferred pediatric randomized, double-blind, placebo-controlled efficacy and safety study under PREA for the preventive treatment of chronic migraine in adolescents ages 12 through 17 years. This study includes a double-blind treatment phase (of at least 12 weeks duration), with an open-label extension (of at least 40 weeks duration). Two weight bands should be utilized for dosing. In each weight band, two different dosing levels of Aimovig should be tested. Dosing should provide exposures matching those observed with the 70-mg dose and with the 140-mg dose of Aimovig in adults. This study is to be submitted as a special protocol assessment (SPA)."~"Delayed"~"Original Final Report Due Date: 12/31/2025; Deferral Extension granted per FDA letter dated 06/13/2024. The study completion milestone was also revised.
"~10/31/2027 0:00:00~7/28/2025 0:00:00
385193~"CDER"~"BLA"~761077.00~"Amgen Inc."~"Aimovig (erenumab-aooe)"~5/17/2018 0:00:00~"Original"~1~"3392-4"~"PMR"~"Pediatric Research Equity Act"~3392.00~4~"PMR 3392-4: Deferred pediatric randomized, double-blind, placebo-controlled efficacy and safety study under PREA for the preventive treatment of episodic migraine in children and adolescents ages 6 through 17 years. This study includes a doubleblind treatment phase (of at least 12 weeks duration), with an open-label extension (of at least 40 weeks duration). Two weight bands should be utilized for dosing. In each weight band, two different dosing levels of Aimovig should be tested. Dosing should provide exposures matching those observed with the 70-mg dose and with the 140-mg dose of Aimovig in adults. This study is to be submitted as a special protocol assessment (SPA)."~"Delayed"~"Original Final Report Due Date: 12/31/2025; Deferral Extension granted per FDA letter dated 06/13/2024. The study completion milestone was also revised. 
"~10/31/2027 0:00:00~7/28/2025 0:00:00
385194~"CDER"~"BLA"~761069.00~"AstraZeneca UK Ltd"~"Imfinzi (Durvalumab)"~5/1/2017 0:00:00~"Supplement"~43~"4679-3"~"PMC"~"506B PMC"~4679.00~3~"PMC 4679-3: Conduct an integrated analysis from ongoing, completed, or planned clinical trials and other potential data sources as appropriate enrolling a sufficient representation of older adults ages 75 years and older, and racial and ethnic minority patients that is reflective of the U.S. population of patients with non-small cell lung cancer (NSCLC), to further characterize the efficacy and safety of the perioperative durvalumab regimen in these patient subgroups. In the analysis, include a sufficient number of patients ages 75 years and older and Black or African American patients reflective of the incidence of NSCLC in the U.S. for each subpopulation to allow for interpretation of the results. The analyses should support comparative efficacy and safety between the aforementioned populations and White, and younger patients."~"Pending"~~6/30/2031 0:00:00~6/22/2026 0:00:00
385195~"CDER"~"BLA"~761136.00~"Celgene Corporation, a Bristol-Myers Squibb Company"~"Reblozyl (luspatercept-aamt)"~11/8/2019 0:00:00~"Original"~1~"3709-4"~"PMR"~"505 (o)(3)"~3709.00~4~"PMR 3709-4: Conduct an assessment of cases of secondary primary malignancies (and malignancies for B-thalassemia), to include hematological malignancies (AML, de-novo AML, transformation to AML), and solid tumors identified in sponsor-initiated and investigator-initiated clinical trials across the entire luspatercept development program for 5 years post approval."~"Fulfilled"~~12/31/2024 0:00:00~12/16/2025 0:00:00
385196~"CDER"~"BLA"~761136.00~"Celgene Corporation, a Bristol-Myers Squibb Company"~"Reblozyl (luspatercept-aamt)"~11/8/2019 0:00:00~"Supplement"~9~"4486-1"~"PMR"~"505 (o)(3)"~4486.00~1~"PMR 4486-1: Complete Study ACE-536-MDS-002: A phase 3, open-label, randomized study to compare the efficacy and safety of Luspatercept (ACE-536) versus Epoetin Alfa for the treatment of anemia due to IPSS-R very low, low or intermediate risk myelodysplastic syndromes (MDS) in ESA naive subjects who require red blood cell transfusions. Provide at least 5 years of follow-up from the first dose or 3 years after the last dose of study drug (or until death) for enrolled subjects. Long-term safety outcomes of interest include thromboembolic events reported as related to study medication by the investigator, transformation to acute myeloid leukemia, secondary malignancies, and survival. Include an updated safety analysis and submit datasets with the final clinical study report submission."~"Ongoing"~~9/30/2031 0:00:00~12/16/2025 0:00:00
385197~"CDER"~"BLA"~761137.00~"Astellas Pharma US, Inc."~"Padcev (enfortumab vedotin-ejfv)"~12/18/2019 0:00:00~"Supplement"~18~"4428-2"~"PMR"~"505 (o)(3)"~4428.00~2~"PMR 4428-2: Conduct an integrated safety analysis of ongoing trials across the enfortumab vedotin development program to further characterize the known serious risk of peripheral neuropathy in relation to prolonged exposure to enfortumab vedotin in the settings of perioperative and firstline metastatic treatment of urothelial carcinoma. The analyses should include assessments of long-term outcomes and reversibility of existing neuropathy and data on any late-onset neuropathy, including duration and resolution."~"Ongoing"~~6/30/2028 0:00:00~2/13/2026 0:00:00
385198~"CDER"~"BLA"~761137.00~"Astellas Pharma US, Inc."~"Padcev (enfortumab vedotin-ejfv)"~12/18/2019 0:00:00~"Supplement"~24~"4562-1"~"PMC"~"506B PMC"~4562.00~1~"PMC 4562-1: Complete clinical trial EV-302, titled  An open-label, randomized, controlled phase 3 study of enfortumab vedotin in combination with pembrolizumab versus chemotherapy alone in previously untreated locally advanced or metastatic urothelial cancer,  to obtain the final overall survival analysis based on the prespecified final number of events."~"Fulfilled"~~4/30/2025 0:00:00~2/13/2026 0:00:00
385199~"CDER"~"BLA"~761137.00~"Astellas Pharma US, Inc."~"Padcev (enfortumab vedotin-ejfv)"~12/18/2019 0:00:00~"Supplement"~25~"4562-1"~"PMC"~"506B PMC"~4562.00~1~"PMC 4562-1: Complete clinical trial EV-302, titled  An open-label, randomized, controlled phase 3 study of enfortumab vedotin in combination with pembrolizumab versus chemotherapy alone in previously untreated locally advanced or metastatic urothelial cancer,  to obtain the final overall survival analysis based on the prespecified final number of events."~"Fulfilled"~~4/30/2025 0:00:00~2/13/2026 0:00:00
385200~"CDER"~"BLA"~761137.00~"Astellas Pharma US, Inc."~"Padcev (enfortumab vedotin-ejfv)"~12/18/2019 0:00:00~"Supplement"~32~"4935-1"~"PMC"~"506B PMC"~4935.00~1~"PMC 4935-1: Complete the ongoing clinical trial, KEYNOTE-905/EV-303 (NCT03924895), titled  Perioperative Pembrolizumab (MK-3475) Plus Cystectomy or Perioperative Pembrolizumab Plus Enfortumab Vedotin Plus Cystectomy Versus Cystectomy Alone in Participants Who Are Cisplatin-ineligible or Decline Cisplatin With Muscle-invasive Bladder Cancer , and provide the pre-planned final overall survival analysis, to further characterize the clinical benefit of pembrolizumab in combination with enfortumab vedotin as neoadjuvant treatment, and then continued after radical cystectomy as adjuvant treatment for the treatment of adult patients with muscle invasive bladder cancer who are ineligible for or declined cisplatin-containing chemotherapy."~"Ongoing"~~12/31/2028 0:00:00~2/13/2026 0:00:00
385201~"CDER"~"BLA"~761139.00~"Daiichi Sankyo, Inc."~"ENHERTU (fam-trastuzumab deruxtecan-nxki)"~12/20/2019 0:00:00~"Supplement"~21~"4321-2"~"PMR"~"Accelerated Approval"~4321.00~2~"PMR 4321-2: Conduct a multicenter, randomized clinical trial of fam-trastuzumab deruxtecan-nxki in patients with treatment-na ve, unresectable or metastatic non-small cell lung cancer whose tumors have an activating HER2 (ERBB2) mutation. The final analysis should include the final progression-free survival and overall survival results."~"Ongoing"~"The trial has completed enrollment."~9/30/2028 0:00:00~2/13/2026 0:00:00
385202~"CDER"~"BLA"~761139.00~"Daiichi Sankyo, Inc."~"ENHERTU (fam-trastuzumab deruxtecan-nxki)"~12/20/2019 0:00:00~"Supplement"~22~"4318-1"~"PMC"~"506B PMC"~4318.00~1~"PMC 4318-1: Conduct an integrated analysis containing data from clinical trials and other data sources such as post-marketing reports, real-world evidence and other sources to further characterize the safety and efficacy of T-DXd in racial and ethnic minority patients and older patients age >65 years with HER2-low metastatic breast cancer. The analyses should support comparative safety and efficacy outcome analyses between the aforementioned populations and White and younger patients."~"Ongoing"~~9/30/2027 0:00:00~2/13/2026 0:00:00
385203~"CDER"~"BLA"~761139.00~"Daiichi Sankyo, Inc."~"ENHERTU (fam-trastuzumab deruxtecan-nxki)"~12/20/2019 0:00:00~"Supplement"~28~"4624-1"~"PMR"~"Accelerated Approval"~4624.00~1~"PMR 4624-1: Complete Cohort A in Part 2 of the ongoing DESTINY-PanTumor02 trial intended to verify and describe the clinical benefit of Enhertu in adult patients with unresectable or metastatic HER2-positive (IHC3+) solid tumors that have progressed following prior treatment and have no satisfactory alternative treatment options. Include a minimum of 40 patients across all tumor types, other than breast, colorectal, lung, and gastric/GEJ cancer, including a sufficient number of patients with and representation of HER2-positive (IHC3+) tumor types that require additional characterization. In order to characterize response rate and duration of response, patients should be followed for at least 12 months from the onset of response."~"Ongoing"~"The trial has completed enrollment."~6/30/2027 0:00:00~2/13/2026 0:00:00
385204~"CDER"~"BLA"~761139.00~"Daiichi Sankyo, Inc."~"ENHERTU (fam-trastuzumab deruxtecan-nxki)"~12/20/2019 0:00:00~"Supplement"~28~"4624-2"~"PMR"~"Pediatric Research Equity Act"~4624.00~2~"PMR 4624-2: Conduct a clinical study of Enhertu to evaluate dosing, pharmacokinetics, safety, and efficacy of Enhertu in a sufficient number of pediatric patients ages 1 month to <17 years of age with unresectable or metastatic HER2-expressing solid tumors, that have progressed following prior treatment or who have no satisfactory alternative treatment options, deferred until results of completed or planned nonclinical in vitro and in vivo studies or available pediatric clinical data are submitted to and reviewed by FDA, and have been found to support a clinical investigation in the pediatric population."~"Pending"~"The trial has not begun but does not meet the criterion for delayed."~12/31/2032 0:00:00~2/13/2026 0:00:00
385205~"CDER"~"BLA"~761291.00~"Janssen Biotech, Inc."~"Tecvayli (teclistamab-cqyv)"~10/25/2022 0:00:00~"Original"~1~"4334-1"~"PMR"~"Accelerated Approval"~4334.00~1~"PMR 4334-1: Conduct a randomized clinical trial in patients with relapsed or refractory multiple myeloma. The trial should enroll sufficient numbers of racial and ethnic minority patients and older patients (ages 65-74 and 75 and above) to enable an evaluation of teclistamab in a study population that better reflects the U.S. population of patients with multiple myeloma. Patients should be randomized to receive a teclistamab-based regimen compared to standard therapy for relapsed or refractory multiple myeloma. The primary endpoint should be progression-free survival and secondary endpoints should include overall survival, overall response rate, and duration of response."~"Fulfilled"~"Per FDA letter dated 03/05/2026, this PMR/PMC has been fulfilled."~3/31/2026 0:00:00~12/16/2025 0:00:00
385206~"CDER"~"BLA"~761291.00~"Janssen Biotech, Inc."~"Tecvayli (teclistamab-cqyv)"~10/25/2022 0:00:00~"Original"~1~"4334-2"~"PMR"~"505 (o)(3)"~4334.00~2~"PMR 4334-2: Conduct a clinical trial to further characterize and determine the incidence of neurologic toxicities in patients receiving teclistamab, including immune effector cell-associated neurotoxicity syndrome, encephalopathy, peripheral neuropathy including Guillain-Barr  syndrome, and motor dysfunction including Parkinsonism. This data may come from Study 64007957MMY3001 (MajesTEC-3) and other clinical trials across the teclistamab development program including long term follow-up from Study 64007957MMY1001 (MajesTEC-1). Include the incidence rates, time to onset, and outcomes in the final report. Also include investigation of associations and temporal relationships between the incidence and severity of neurologic adverse events and potential associated risk factors, such as age and comorbidities."~"Fulfilled"~~3/31/2026 0:00:00~12/16/2025 0:00:00
385207~"CDER"~"BLA"~761299.00~"Alvotech USA Inc."~"Simlandi (adalimumab-ryvk)"~2/23/2024 0:00:00~"Supplement"~7~"4660-1"~"PMR"~"Pediatric Research Equity Act"~4660.00~1~"PMR 4660-1: Develop a presentation that can be used to accurately administer Simlandi (adalimumab-ryvk) to pediatric patients who weigh less than 15 kg."~"Ongoing"~"The study has been initiated."~6/30/2026 0:00:00~4/16/2026 0:00:00
385208~"CDER"~"BLA"~761299.00~"Alvotech USA Inc."~"Simlandi (adalimumab-ryvk)"~2/23/2024 0:00:00~"Supplement"~16~"4803-1"~"PMR"~"Pediatric Research Equity Act"~4803.00~1~"PMR 4803-1: Develop a presentation that can be used to accurately administer Simlandi (adalimumab-ryvk) to pediatric patients who weigh less than 15 kg"~"Ongoing"~"The study has been initiated."~6/30/2026 0:00:00~4/16/2026 0:00:00
385209~"CDER"~"BLA"~761304.00~"Argenx BV"~"Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc)"~6/20/2023 0:00:00~"Original"~1~"4462-1"~"PMR"~"505 (o)(3)"~4462.00~1~"PMR 4462-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to VYVGART Hytrulo (efgartigimodalfa and hyaluronidase-qvfc) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~12/31/2033 0:00:00~8/15/2025 0:00:00
385210~"CDER"~"BLA"~761304.00~"Argenx BV"~"Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc)"~6/20/2023 0:00:00~"Original"~1~"4462-2"~"PMR"~"505 (o)(3)"~4462.00~2~"PMR 4462-2: Perform a lactation study (milk only) in lactating women who have received therapeutic doses of efgartigimod alfa and hyaluronidase-qvfc using a validated assay to assess concentrations of efgartigimod alfa in breast milk and the effects on the breastfed infant as applicable."~"Delayed"~"The final protocol milestone was missed because of time taken by the agency to provide comments and feedback to the applicant on the draft protocol. On 4/16/25 the Agency issued a Notification of Missed Milestone letter. Final protocol acknowledged by the agency in a letter dated 6/2/25."~9/30/2026 0:00:00~8/15/2025 0:00:00
385211~"CDER"~"BLA"~761304.00~"Argenx BV"~"Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc)"~6/20/2023 0:00:00~"Supplement"~5~"4652-1"~"PMR"~"505 (o)(3)"~4652.00~1~"PMR 4652-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women with chronic inflammatory demyelinating polyneuropathy (CIDP) exposed to Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) during pregnancy and/or
lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~12/31/2033 0:00:00~8/15/2025 0:00:00
385212~"CDER"~"BLA"~761304.00~"Argenx BV"~"Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc)"~6/20/2023 0:00:00~"Supplement"~6~"4652-1"~"PMR"~"505 (o)(3)"~4652.00~1~"PMR 4652-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women with chronic inflammatory demyelinating polyneuropathy (CIDP) exposed to Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) during pregnancy and/or
lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~12/31/2033 0:00:00~8/15/2025 0:00:00
385213~"CDER"~"BLA"~761306.00~"Eli Lilly and Company"~"Ebglyss (lebrikizumab-lbkz)"~9/13/2024 0:00:00~"Original"~1~"4514-1"~"PMR"~"Pediatric Research Equity Act"~4514.00~1~"PMR 4514-1: Conduct a randomized, double-blind, placebo-controlled trial to assess the PK and safety of lebrikizumab in pediatric patients 6 months to <6 years, 6 years to <12 years, and =12 years to <18 years weighing <40 kg with moderate to severe atopic dermatitis."~"Delayed"~"Deferral Extension granted per FDA letter dated 01/13/2026. The study completion milestone was also revised. As of the cutoff date of 09/13/2025, enrollment is ongoing and 365 subjects have been enrolled. The original study completion milestone has been missed.
"~4/30/2027 0:00:00~11/10/2025 0:00:00
385214~"CDER"~"BLA"~761306.00~"Eli Lilly and Company"~"Ebglyss (lebrikizumab-lbkz)"~9/13/2024 0:00:00~"Original"~1~"4514-2"~"PMR"~"Pediatric Research Equity Act"~4514.00~2~"PMR 4514-2: Conduct an open-label, long-term extension study to evaluate the longterm safety of lebrikizumab in pediatric patients 6 months to <12 years, and > 12 years <18 years weighing less than 40 kg with moderate to severe atopic dermatitis."~"Ongoing"~"The study is underway. Per the applicant's annual status report, as of the cutoff date of 9/13/2025, enrollment is ongoing and 307 subjects have been enrolled."~4/30/2028 0:00:00~11/10/2025 0:00:00
385215~"CDER"~"BLA"~761306.00~"Eli Lilly and Company"~"Ebglyss (lebrikizumab-lbkz)"~9/13/2024 0:00:00~"Original"~1~"4514-3"~"PMR"~"505 (o)(3)"~4514.00~3~"PMR 4514-3: Conduct or participate in a relevant Pregnancy Exposure Registry, a prospective registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to lebrikizumab during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, neonatal deaths, and serious infections, will be assessed through at least the first year of life."~"Delayed"~"Final Protocol Submission Due Date 09/30/2025 was missed due to
continued discussions with the Agency."~9/30/2036 0:00:00~11/10/2025 0:00:00
385216~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-4"~"PMR"~"505 (o)(3)"~4835.00~4~"PMR 4835-4: Conduct a clinical trial to characterize and evaluate the potential serious risk of cardiomyopathy related to the use of the combination of avutometinib and defactinib in adult patients with recurrent, low grade serous ovarian cancer. The trial should include serial monitoring of blood pressure, left ventricular ejection fraction (LVEF), high-sensitivity troponin, and N-terminal brain natriuretic peptide. The cardiac assessments must include a sufficient number of patients, over a sufficient time period, to systematically determine the incidence of LVEF reductions =10% from baseline. A randomized trial design is preferred given the unknown population risk. Include an analysis of the risk, and monitoring and mitigation measures."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 11/07/2025."~4/30/2029 0:00:00~6/26/2026 0:00:00
385217~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-5"~"PMR"~"505 (o)(3)"~4835.00~5~"PMR 4835-5: Conduct a clinical trial enrolling an adequate number of patients with moderate hepatic impairment (i.e., at least 15 evaluable patients), using the NCI-ODWG criteria, to evaluate the potential serious risk of increased serious adverse reactions in patients with moderate hepatic impairment who receive the treatment of avutometinib in combination with defactinib. The evaluation should be in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~4/30/2029 0:00:00~6/26/2026 0:00:00
385218~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-6"~"PMR"~"505 (o)(3)"~4835.00~6~"PMR 4835-6: Conduct a clinical pharmacokinetic trial assessing the effect of severe hepatic impairment on the pharmacokinetics of defactinib and its active metabolite M4 to evaluate the potential serious risk of increased serious adverse reactions in patients with severe hepatic impairment who receive treatment with defactinib. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling."~"Pending"~~6/30/2028 0:00:00~6/26/2026 0:00:00
385219~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-7"~"PMR"~"505 (o)(3)"~4835.00~7~"PMR 4835-7: Conduct a clinical pharmacokinetic trial assessing the effect of severe hepatic impairment, using the NCI-ODWG criteria, on the pharmacokinetics of avutometinib to evaluate the potential serious risk of increased serious adverse reactions in patients with severe hepatic impairment who receive treatment with avutometinib. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~7/31/2028 0:00:00~6/26/2026 0:00:00
385220~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-8"~"PMR"~"505 (o)(3)"~4835.00~8~"PMR 4835-8: Conduct a clinical pharmacokinetic trial assessing the effect of severe renal impairment, as estimated by the Cockcroft-Gault equation, on the pharmacokinetics of avutometinib to evaluate the potential serious risk of increased serious adverse reactions in patients with severe renal impairment who receive treatment with avutometinib. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Renal Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~8/31/2028 0:00:00~6/26/2026 0:00:00
385221~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-9"~"PMR"~"505 (o)(3)"~4835.00~9~"PMR 4835-9: Complete the clinical trial, Study VS-6063-108, assessing the effect of multiple doses of a BCRP inhibitor, a P-gp Inhibitor, and a moderate CYP2C9 inhibitor on the pharmacokinetics of defactinib and its active metabolite M4 to evaluate the potential serious risk of increased serious adverse reactions from increased levels of defactinib when defactinib is used concomitantly with a BCRP inhibitor, a P-gp Inhibitor, and a moderate CYP2C9 inhibitor, respectively. Design and conduct the trial in accordance with ICH Harmonized Guidance entitled  M12 Drug Interaction Studies. "~"Submitted"~~6/30/2025 0:00:00~6/26/2026 0:00:00
385222~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-10"~"PMR"~"505 (o)(3)"~4835.00~10~"PMR 4835-10: Conduct a clinical pharmacokinetic trial assessing the effects of multiple doses of defactinib on single dose pharmacokinetics of substrates of CYP3A4, CYP2C9, P-gp, BCPR, OATP1B1, and OATP1B3 to evaluate the potential serious risks of increased serious adverse reactions from elevated levels of CYP3A4, CYP2C9, P-gp, BCPR, OATP1B1, and OATP1B3 substrates, respectively, when they are used concomitantly with defactinib. Design and conduct the trial in accordance with the ICH Harmonized Guidance entitled  M12 Drug Interaction Studies."~"Pending"~~9/30/2026 0:00:00~6/26/2026 0:00:00
385223~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-11"~"PMR"~"505 (o)(3)"~4835.00~11~"PMR 4835-11: Conduct a clinical pharmacokinetic trial assessing the effect of multiple doses of defactinib on single dose pharmacokinetics of a substrate of MATE2-K to evaluate the potential serious risk of increased serious adverse reactions from elevated levels of MATE2-K substrates when used concomitantly with defactinib. Alternatively, evaluate the change in exposure of a well-characterized endogenous substrate of MATE2-K to assess the impact of defactinib on the inhibition of MATE2-K. Design and conduct the trial in accordance with the ICH Harmonized Guidance entitled  M12 Drug Interaction Studies. "~"Pending"~~9/30/2026 0:00:00~6/26/2026 0:00:00
385224~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-12"~"PMC"~"506B PMC"~4835.00~12~"PMC 4835-12: Conduct an in vitro study to assess the potential for CYP1A2, CYP2B6, CYP2C8, CYP2C19, and CYP2D6 inhibitors to impact the pharmacokinetics of defactinib metabolite M4 to determine for the need of additional clinical drug-drug interaction studies between M4 and inhibitors of these CYP enzymes. Design and conduct the in vitro study in accordance with the ICH Harmonized Guidance entitled  M12 Drug Interaction Studies. "~"Pending"~~11/30/2025 0:00:00~6/26/2026 0:00:00
385225~"CDER"~"NDA"~219616.00~"VERASTEM INC"~"Avmapki Fakzynja Co-Pack (avutometinib capsules, 0.8 mg; defactinib tablets, 200 mg)"~5/8/2025 0:00:00~"Original"~1~"4835-13"~"PMC"~"506B PMC"~4835.00~13~"PMC 4835-13: Conduct an appropriate analytical and clinical validation study to support the development of a diagnostic device that is essential to the safe and effective use of the combination of avutometinib and defactinib in KRASmutated, recurrent low grade serous ovarian cancer."~"Pending"~~5/31/2026 0:00:00~6/26/2026 0:00:00
385226~"CDER"~"BLA"~761077.00~"Amgen Inc."~"Aimovig (erenumab-aooe)"~5/17/2018 0:00:00~"Original"~1~"3392-5"~"PMR"~"505 (o)(3)"~3392.00~5~"PMR 3392-5: Conduct prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with migraine exposed to Aimovig during pregnancy with two unexposed control populations: one consisting of women with migraine who have not been exposed to Aimovig before or during pregnancy and the other consisting of women without migraine. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"Per FDA letter dated 01/28/2026, the applicant s request for release was denied."~7/31/2027 0:00:00~7/28/2025 0:00:00
385227~"CDER"~"BLA"~761077.00~"Amgen Inc."~"Aimovig (erenumab-aooe)"~5/17/2018 0:00:00~"Original"~1~"3392-6"~"PMR"~"505 (o)(3)"~3392.00~6~"PMR 3392-6: Conduct a pregnancy outcomes study using a different study design than provided for in PMR 3392-5 (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small-for-gestational-age births in women exposed to Aimovig during pregnancy compared to an unexposed control population."~"Delayed"~"The 5th interim report was submitted on 11/26/2025 by the revised milestone date."~5/31/2027 0:00:00~7/28/2025 0:00:00
385228~"CDER"~"BLA"~761079.00~"Biomarin Pharmaceutical Inc."~"Palynziq (pegvaliase-pqpz)"~5/24/2018 0:00:00~"Original"~1~"3349-1"~"PMR"~"505 (o)(3)"~3349.00~1~"PMR 3349-1: Prospective, longitudinal, observational study to assess long-term risks of severe immune-mediated adverse reactions in adult patients with phenylketonuria (PKU) treated with Palynziq (pegvaliase-pqpz). Each patient will be treated with Palynziq (pegvaliase-pqpz) over a minimum of 10 years. Evaluate the incidence rates of severe immune-mediated adverse reactions (including, but not limited to, hypersensitivity reactions, anaphylaxis, generalized skin reactions, and arthralgias), and collect information, including a full description of clinical features of the adverse reactions, to investigate associations and temporal relationships between the incidence and severity of all immune-mediated adverse reactions and other potential associated risk factors. Evaluate immunologic and inflammatory responses (immunologic testing, inflammatory markers), their effects on major organ function (e.g., kidney function), and immune-mediated effects on blood phenylalanine therapeutic response. Collect and analyze additional information, including, but not limited to, PAH genotype, dietary practices, and prior medical history. Specify concise case definitions, validation methods, and procedures for all study outcomes. An interim report will be submitted every two years during the conduct of the study."~"Delayed"~"The final protocol milestone was missed. FDA issued acknowledge final protocol letter on 5/4/2022."~6/30/2030 0:00:00~7/23/2025 0:00:00
385229~"CDER"~"BLA"~761079.00~"Biomarin Pharmaceutical Inc."~"Palynziq (pegvaliase-pqpz)"~5/24/2018 0:00:00~"Original"~1~"3349-2"~"PMR"~"505 (o)(3)"~3349.00~2~"PMR 3349-2: Prospective, observational study to assess the risks of pregnancy complications and adverse effects on the developing fetus and newborn (including, but not limited to, fetal malformations and pre-natal and post-natal growth restriction) from Palynziq (pegvaliase-pqpz) treatment during pregnancy. The study will collect and analyze data on blood phenylalanine concentrations during pregnancy in treated pregnant women with PKU and examine associations between Palynziq (pegvaliase-pqpz) treatment, blood phenylalanine concentrations, and adverse outcomes in the pregnant women and their offspring (fetus/newborn). The study duration will be a minimum of 10 years. An interim report will be submitted every two years during the conduct of the study."~"Delayed"~"The final protocol submission milestone was missed. FDA issued an acknowledge final protocol letter on 4/14/2022."~6/30/2030 0:00:00~7/23/2025 0:00:00
385230~"CDER"~"BLA"~761079.00~"Biomarin Pharmaceutical Inc."~"Palynziq (pegvaliase-pqpz)"~5/24/2018 0:00:00~"Original"~1~"3349-8"~"PMR"~"505 (o)(3)"~3349.00~8~"PMR 3349-8: Evaluate anti-PEG IgM and IgG ADA in samples from the observational study (according to postmarketing study 3349-1) using the anti-PEG IgM and IgG ADA assays with improved drug tolerance. Assess the impact of anti-PEG IgM and IgG ADA on efficacy and safety. An interim report will be submitted every two years during the conduct of the study."~"Pending"~~6/30/2030 0:00:00~7/23/2025 0:00:00
385231~"CDER"~"BLA"~761079.00~"Biomarin Pharmaceutical Inc."~"Palynziq (pegvaliase-pqpz)"~5/24/2018 0:00:00~"Original"~1~"3349-9"~"PMR"~"505 (o)(3)"~3349.00~9~"PMR 3349-9: Evaluate the sensitivity of the anti-pegvaliase-pqpz IgE ImmunoCAP assay to detect anti-PEG IgE antibodies, and make modifications to the method as needed. Test samples from treated patients with the current or modified assay in the prospective study (according to postmarketing study 3349-1) who experience anaphylaxis episodes in order to more comprehensively examine the underlying mechanism of the anaphylaxis. An interim report will be submitted every two years during the conduct of the study."~"Pending"~~6/30/2030 0:00:00~7/23/2025 0:00:00
385232~"CDER"~"BLA"~761079.00~"Biomarin Pharmaceutical Inc."~"Palynziq (pegvaliase-pqpz)"~5/24/2018 0:00:00~"Original"~1~"3349-10"~"PMR"~"505 (o)(3)"~3349.00~10~"PMR 3349-10: Clinical trial of an appropriate immune tolerance induction (ITI) regimen to evaluate the ability of the ITI regimen (given prior to or concurrently with Palynziq (pegvaliase-pqpz) treatment) to suppress immune responses, to reduce the risks of immune-mediated adverse reactions, and to enable improved therapeutic responses in adult patients with PKU treated with Palynziq (pegvaliase-pqpz)."~"Delayed"~"The final protocol submission was missed and the FDA issued a Notification of Missed Milestone letter on 06/25/2025."~12/31/2025 0:00:00~7/23/2025 0:00:00
385233~"CDER"~"BLA"~761079.00~"Biomarin Pharmaceutical Inc."~"Palynziq (pegvaliase-pqpz)"~5/24/2018 0:00:00~"Supplement"~31~"4958-1"~"PMR"~"505 (o)(3)"~4958.00~1~"PMR 4958-1: Conduct a clinical trial to evaluate the effect of Palynziq (pegvaliase-pqpz) on ammonia levels in patients with phenylketonuria."~"Pending"~~5/31/2029 0:00:00~7/23/2025 0:00:00
385234~"CDER"~"BLA"~761083.00~"Genentech, Inc."~"Hemlibra (emicizumab-kxwh)"~11/16/2017 0:00:00~"Original"~1~"3299-1"~"PMC"~"506B PMC"~3299.00~1~"PMC 3299-1: Conduct an assessment of binding anti-product antibody (APA) responses with a validated assay capable of sensitively detecting APA responses in the presence of  emicizumab levels that are expected to be present in the serum at the time of patient sampling. The APA response will be evaluated in at least 50 emicizumab-treated patients. The final report will include information on the level of emicizumab in each patient s test sample at each sampling point."~"Delayed"~"The final report milestone was missed because the final report was submitted to the FDA on 11/02/2018, but was considered not fulfilled after FDA review."~1/31/2019 0:00:00~12/22/2025 0:00:00
385235~"CDER"~"BLA"~761139.00~"Daiichi Sankyo, Inc."~"ENHERTU (fam-trastuzumab deruxtecan-nxki)"~12/20/2019 0:00:00~"Supplement"~28~"4624-3"~"PMC"~"506B PMC"~4624.00~3~"PMC 4624-3: Conduct a new pragmatic clinical trial with a sufficient number of patients to verify and describe the efficacy of Enhertu in adult patients with unresectable or metastatic HER2-positive (IHC3+) solid tumors with disease that has progressed following prior treatment and have no satisfactory alternative treatment options. The trial should include a sufficient number of tumor types, other than breast, colorectal, lung, and gastric/GEJ cancer. In order to characterize response rate and duration of response, patients should be followed for at least 12 months from the onset of response."~"Ongoing"~~2/28/2029 0:00:00~2/13/2026 0:00:00
385236~"CDER"~"BLA"~761139.00~"Daiichi Sankyo, Inc."~"ENHERTU (fam-trastuzumab deruxtecan-nxki)"~12/20/2019 0:00:00~"Supplement"~28~"4624-4"~"PMC"~"506B PMC"~4624.00~4~"PMC 4624-4: Commitment to submit appropriate analytical and clinical validation study data using the DESTINY-PanTumor02, DESTINY-CRC02, and DESTINYLung01 clinical trials, to support labeling of an immunohistochemistrybased in-vitro diagnostic device that is essential for the safe and effective use of Enhertu for treatment of adults with unresectable or metastatic HER2-positive (IHC3+) solid tumors."~"Ongoing"~~6/30/2026 0:00:00~2/13/2026 0:00:00
385237~"CDER"~"BLA"~761139.00~"Daiichi Sankyo, Inc."~"ENHERTU (fam-trastuzumab deruxtecan-nxki)"~12/20/2019 0:00:00~"Supplement"~32~"4777-1"~"PMC"~"506B PMC"~4777.00~1~"PMC 4777-1: Complete the ongoing clinical trial DESTINY-Breast06, titled  A Phase 3,Randomized, Multi-center, Open-label Study of Trastuzumab Deruxtecan (T-DXd) Versus Investigator s Choice Chemotherapy in HER2-low, Hormone Receptor Positive Breast Cancer Patients whose Disease has Progressed on Endocrine Therapy in the Metastatic Setting  to provide the pre-specified interim and final overall survival analyses."~"Ongoing"~~3/31/2027 0:00:00~2/13/2026 0:00:00
385238~"CDER"~"BLA"~761139.00~"Daiichi Sankyo, Inc."~"ENHERTU (fam-trastuzumab deruxtecan-nxki)"~12/20/2019 0:00:00~"Supplement"~35~"4777-1"~"PMC"~"506B PMC"~4777.00~1~"PMC 4777-1: Complete the ongoing clinical trial DESTINY-Breast06, titled  A Phase 3,Randomized, Multi-center, Open-label Study of Trastuzumab Deruxtecan (T-DXd) Versus Investigator s Choice Chemotherapy in HER2-low, Hormone Receptor Positive Breast Cancer Patients whose Disease has Progressed on Endocrine Therapy in the Metastatic Setting  to provide the pre-specified interim and final overall survival analyses."~"Ongoing"~~3/31/2027 0:00:00~2/13/2026 0:00:00
385239~"CDER"~"BLA"~761139.00~"Daiichi Sankyo, Inc."~"ENHERTU (fam-trastuzumab deruxtecan-nxki)"~12/20/2019 0:00:00~"Supplement"~38~"4951-1"~"PMC"~"506B PMC"~4951.00~1~"PMC 4951-1: Complete the ongoing clinical trial, DESTINY-Breast09, titled  Phase III Study of Trastuzumab Deruxtecan (T-DXd) with or without Pertuzumab versus Taxane, Trastuzumab and Pertuzumab (THP) in HER2-positive, First-line Metastatic Breast Cancer (DESTINY-Breast09),  to provide the pre-specified final progression-free survival (PFS) analysis for the T-DXd monotherapy arm as compared to the THP standard of care (SOC) arm and interim and final overall survival (OS) analyses for all T-DXd investigational arms of the trial as compared to the THP SOC arm."~"Ongoing"~~5/31/2033 0:00:00~2/13/2026 0:00:00
385240~"CDER"~"BLA"~761142.00~"Horizon Therapeutics Ireland DAC"~"Uplizna (inebilizumab)"~6/11/2020 0:00:00~"Original"~1~"3869-1"~"PMR"~"505 (o)(3)"~3869.00~1~"PMR 3869-1: A worldwide single-arm pregnancy safety study to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to UPLIZNA (inebilizumab-cdon) during pregnancy in patients with neuromyelitis optica spectrum disorder (NMOSD). Provide a complete protocol which includes details regarding how you plan to encourage patients and providers to report pregnancy exposures (e.g.,telephone contact number and/or website in prescribing information), measures to ensure complete data capture regarding pregnancy outcomes and any adverse effects in offspring, and plans for comprehensive data analysis and yearly reporting."~"Ongoing"~~8/31/2033 0:00:00~8/8/2025 0:00:00
385241~"CDER"~"BLA"~761142.00~"Horizon Therapeutics Ireland DAC"~"Uplizna (inebilizumab)"~6/11/2020 0:00:00~"Original"~1~"3869-2"~"PMR"~"505 (o)(3)"~3869.00~2~"PMR 3869-2: A safety trial to monitor serum immunoglobulin G and M levels in patients with neuromyelitis optica spectrum disorder (NMOSD) during treatment with UPLIZNA (inebilizumab-cdon) to establish the nadir in circulating  immunoglobulins   during chronic treatment, and to monitor patients after discontinuation of treatment with UPLIZNA (inebilizumab-cdon) in order to ascertain the time needed to ensure restoration of pre-treatment baseline circulating serum levels of  immunoglobulins G and M.This trial also should be designed to capture rates of infections, especially opportunistic and  recurrent infections associated with immune suppression, and there should be monitoring of B-cell counts throughout  treatment and after discontinuation until repletion of immunoglobulin levels."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 1/19/2023."~8/31/2028 0:00:00~8/8/2025 0:00:00
385242~"CDER"~"BLA"~761142.00~"Horizon Therapeutics Ireland DAC"~"Uplizna (inebilizumab)"~6/11/2020 0:00:00~"Supplement"~4~"4930-1"~"PMR"~"505 (o)(3)"~4930.00~1~"PMR 4930-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Uplizna (inebilizumabcdon) during pregnancy to assess risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Pending"~~8/31/2033 0:00:00~8/8/2025 0:00:00
385243~"CDER"~"BLA"~761142.00~"Horizon Therapeutics Ireland DAC"~"Uplizna (inebilizumab)"~6/11/2020 0:00:00~"Supplement"~4~"4930-2"~"PMR"~"505 (o)(3)"~4930.00~2~"PMR 4930-2: Perform a lactation study (milk only or mother-infant pair study) in lactating women who have received Uplizna (inebilizumab-cdon) to measure concentrations of Uplizna (inebilizumab-cdon) and its major metabolites in breastmilk using a validated assay. Assess the effects on the breastfed infant, if available, based on study population."~"Pending"~~9/30/2029 0:00:00~8/8/2025 0:00:00
385244~"CDER"~"BLA"~761142.00~"Horizon Therapeutics Ireland DAC"~"Uplizna (inebilizumab)"~6/11/2020 0:00:00~"Supplement"~4~"4930-3"~"PMR"~"505 (o)(3)"~4930.00~3~"PMR 4930-3: A safety trial to monitor serum immunoglobulin G and M levels in patients with myasthenia gravis during treatment with Uplizna (inebilizumab-cdon) to establish the nadir in circulating immunoglobulins during chronic treatment, and to monitor patients after discontinuation of treatment with Uplizna (inebilizumab-cdon) in order to ascertain the time needed to ensure restoration of pretreatment baseline circulating serum levels of immunoglobulins G and M. This trial also should be designed to capture rates of infections, especially opportunistic and recurrent infections associated with immune suppression, and there should be monitoring of B-cell counts throughout treatment and after discontinuation until repletion of immunoglobulin levels."~"Pending"~~11/30/2029 0:00:00~8/8/2025 0:00:00
385245~"CDER"~"BLA"~761143.00~"Horizon Therapeutics Ireland DAC"~"Tepezza (teprotumumab-trbw)"~1/21/2020 0:00:00~"Original"~1~"3780-8"~"PMR"~"505 (o)(3)"~3780.00~8~"PMR 3780-8: A descriptive clinical trial to evaluate the safety, efficacy and need for retreatment of three different teprotumumab treatment durations for the treatment of Thyroid Eye Disease."~"Ongoing"~~11/30/2026 0:00:00~3/20/2026 0:00:00
385246~"CDER"~"BLA"~761309.00~"Genentech, Inc."~"Columvi (glofitamab-gxbm)"~6/15/2023 0:00:00~"Original"~1~"4464-1"~"PMR"~"Accelerated Approval"~4464.00~1~"PMR 4464-1: Complete a randomized clinical trial that evaluates the clinical benefit of glofitamab in patients with diffuse large B-cell lymphoma. The trial should compare glofitamab in combination with gemcitabine and oxaliplatin (GemOx) to rituximab in combination with GemOx for patients with relapsed or refractory diffuse large B-cell lymphoma. The primary endpoint should be overall survival with secondary endpoints that include progression-free survival and response rate."~"Fulfilled"~"Per FDA letter dated 10/09/2025, this PMR/PMC has been fulfilled."~9/30/2024 0:00:00~8/7/2025 0:00:00
385247~"CDER"~"BLA"~761309.00~"Genentech, Inc."~"Columvi (glofitamab-gxbm)"~6/15/2023 0:00:00~"Original"~1~"4464-2"~"PMR"~"Pediatric Research Equity Act"~4464.00~2~"PMR 4464-2: Conduct a study assessing the efficacy and safety of glofitamab in pediatric patients 6 months or older with relapsed or refractory non-Hodgkin lymphoma. The safety endpoints should evaluate safety and tolerability of glofitamab monotherapy and in combination with standard chemotherapy and identify the recommended dosage. The efficacy endpoints should evaluate activity as assessed by response rate and durability of response."~"Ongoing"~"14 patients out of the planned 65 patients have been enrolled into the study."~6/30/2028 0:00:00~8/7/2025 0:00:00
385248~"CDER"~"BLA"~761309.00~"Genentech, Inc."~"Columvi (glofitamab-gxbm)"~6/15/2023 0:00:00~"Original"~1~"4464-3"~"PMC"~"506B PMC"~4464.00~3~"PMC 4464-3: Conduct an integrated analysis of data from clinical trials to further characterize the safety, efficacy, pharmacokinetics, and pharmacodynamics of glofitamab among U.S. racial and ethnic minority patients with large B-cell lymphoma. The population should be representative of the U.S. population of patients with large B-cell lymphoma, including racial and ethnic diversity, and allow for interpretation of the results in these populations."~"Ongoing"~~12/31/2027 0:00:00~8/7/2025 0:00:00
385249~"CDER"~"BLA"~761309.00~"Genentech, Inc."~"Columvi (glofitamab-gxbm)"~6/15/2023 0:00:00~"Original"~1~"4464-4"~"PMC"~"506B PMC"~4464.00~4~"PMC 4464-4: Conduct an analysis of duration of response with extended follow-up in patients with diffuse large B-cell lymphoma or transformed follicular lymphoma who discontinue glofitamab monotherapy after receiving 12 cycles. Provide this additional duration of response data, as assessed by an IRC, in patients in the primary efficacy population of Study NP30179, with a targeted minimum of one year of extended follow-up after completion of 12 cycles of glofitamab. Include data regarding new anti-lymphoma therapy and efficacy outcomes after glofitamab retreatment."~"Fulfilled"~~11/30/2024 0:00:00~8/7/2025 0:00:00
385250~"CDER"~"BLA"~761309.00~"Genentech, Inc."~"Columvi (glofitamab-gxbm)"~6/15/2023 0:00:00~"Original"~1~"4464-5"~"PMR"~"Accelerated Approval"~4464.00~5~"PMR 4464-5: Complete a randomized clinical trial that is intended to verify and describe the clinical benefit of glofitamab in patients with large B-cell lymphoma. The trial should compare glofitamab in combination with polatuzumab, rituximab, cyclophosphamide, doxorubicin, and prednisone (pola-R-CHP) to pola-R-CHP for patients with previously untreated large B-cell lymphoma. The primary endpoint should be progression-free survival by Independent Review Facility with secondary endpoints that include overall survival and overall response rate by Independent Review Facility."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~3/31/2029 0:00:00~8/7/2025 0:00:00
385251~"CDER"~"BLA"~761310.00~"AbbVie Inc."~"Elahere (mirvetuximab soravtansine-gynx)"~11/14/2022 0:00:00~"Original"~1~"4347-2"~"PMR"~"505 (o)(3)"~4347.00~2~"PMR 4347-2: Conduct a randomized clinical trial of at least 2 dose schedules of mirvetuximab soravtansine to characterize the safety of the recommended dosage of 6 mg/kg AIBW Q3W and alternative dosing schedule(s) (i.e., modified weekly dosing regimen, where lower doses are administered at a weekly frequency, instead of every 3 weeks) for the treatment of adult patients with FRa-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens. The trial should evaluate safety parameters including ocular AEs (all grade, Grade =2), peripheral neuropathy, (all grade, Grade =2), and other adverse events of interest. The trial should evaluate efficacy parameters of the alternate dosing schedules. The trial should also include sufficient clinical pharmacokinetic sampling to analyze the exposure-response relationship for efficacy and safety."~"Ongoing"~~2/28/2027 0:00:00~1/7/2026 0:00:00
385252~"CDER"~"BLA"~761310.00~"AbbVie Inc."~"Elahere (mirvetuximab soravtansine-gynx)"~11/14/2022 0:00:00~"Original"~1~"4347-3"~"PMR"~"505 (o)(3)"~4347.00~3~"PMR 4347-3: Conduct an open-label, non-randomized, dose escalation trial, with expansion in a sufficient number of patients, to determine an appropriate starting dose of mirvetuximab soravtansine in patients with moderate hepatic impairment, according to National Cancer Institute Organ Dysfunction Working Group (NCI-ODWG) criteria, in the target patient population. Collect safety and pharmacokinetic information for mirvetuximab soravtansine, unconjugated DM4, and s-methyl DM4 from the trial and utilize this information to determine the appropriate starting dose of mirvetuximab soravtansine for this population."~"Ongoing"~~2/28/2027 0:00:00~1/7/2026 0:00:00
385253~"CDER"~"BLA"~761310.00~"AbbVie Inc."~"Elahere (mirvetuximab soravtansine-gynx)"~11/14/2022 0:00:00~"Original"~1~"4347-4"~"PMR"~"505 (o)(3)"~4347.00~4~"PMR 4347-4: Conduct a clinical trial, or amend existing clinical trials, of mirvetuximab soravtansine to incorporate prospectively specified, scheduled ophthalmologic assessments in all patients (symptomatic or asymptomatic) to further characterize the incidence and severity of mirvetuximab soravtansine-related ocular adverse events and evaluate the additional risk mitigation strategies for ocular adverse events."~"Delayed"~"The Study Completion and Final Report milestones were missed because the original timeline to conduct the protocol specified analyses would not allow enough time to fully characterize ocular adverse events (OAEs) including resolution, as discussed during the Type D meeting held on
August 28, 2025. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 05/04/2026."~2/28/2026 0:00:00~1/7/2026 0:00:00
385254~"CDER"~"BLA"~761306.00~"Eli Lilly and Company"~"Ebglyss (lebrikizumab-lbkz)"~9/13/2024 0:00:00~"Original"~1~"4514-4"~"PMR"~"505 (o)(3)"~4514.00~4~"PMR 4514-4: Conduct an additional pregnancy study that uses a different design from the Pregnancy Exposure Registry (for example a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to lebrikizumab during pregnancy compared to an unexposed control population."~"Delayed"~"Final Protocol Submission Due Date 09/30/2025 was missed due to continued discussions with the Agency."~9/30/2033 0:00:00~11/10/2025 0:00:00
385255~"CDER"~"NDA"~219683.00~"JANSSEN BIOTECH INC"~"Inlexzo (gemcitabine) intravesical system"~9/9/2025 0:00:00~"Original"~1~"4894-1"~"PMC"~"506B PMC"~4894.00~1~"PMC 4894-1: Complete the ongoing clinical trial SunRISe-1 (NCT05243550) to further characterize the clinical benefit of TAR-200 for the treatment of patients with Bacillus Calmette-Gu rin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. Provide updated duration of response (DOR) data for all patients with current ongoing response in the final trial report after all of the patients who have current ongoing responses have either experienced recurrence of high-grade NMIBC, progression, death, or been lost to followup, for up to 48 months after the first instillation."~"Pending"~~6/30/2028 0:00:00~
385256~"CDER"~"NDA"~219685.00~"GENZYME CORP"~"Wayrilz (rilzabrutinib) Tablet"~8/29/2025 0:00:00~"Original"~1~"4878-1"~"PMR"~"505 (o)(3)"~4878.00~1~"PMR 4878-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Wayrilz (rilzabrutinib) during pregnancy to assess the risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant. Assess infant outcomes through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Pending"~~9/30/2037 0:00:00~
385257~"CDER"~"NDA"~219685.00~"GENZYME CORP"~"Wayrilz (rilzabrutinib) Tablet"~8/29/2025 0:00:00~"Original"~1~"4878-2"~"PMR"~"505 (o)(3)"~4878.00~2~"PMR 4878-2: Perform a milk-only lactation study in lactating women who have received rilzabrutinib to measure concentrations of Wayrilz (rilzabrutinib) in breast milk using a validated assay. Assess the effects on the breastfed infant, if available, based on study population."~"Pending"~~6/30/2030 0:00:00~
385258~"CDER"~"NDA"~219685.00~"GENZYME CORP"~"Wayrilz (rilzabrutinib) Tablet"~8/29/2025 0:00:00~"Original"~1~"4878-3"~"PMR"~"505 (o)(3)"~4878.00~3~"PMR 4878-3: Conduct a clinical drug interaction study to evaluate the effect of Wayrilz (rilzabrutinib) on the pharmacokinetics of P-gp, BCRP, OATP1B1, and OATP1B3 transporter substrates in adult healthy volunteers. The study will assess the magnitude of exposure change of the transporter substrates and inform appropriate drug interaction management strategy for coadministration of rilzabrutinib with these transporter substrates in adult patients with persistent or chronic immune thrombocytopenia. This study should be designed and conducted in accordance with the ICH Guideline on Drug Interaction Studies M12 (August 2024)."~"Pending"~~9/30/2027 0:00:00~
385259~"CDER"~"NDA"~219685.00~"GENZYME CORP"~"Wayrilz (rilzabrutinib) Tablet"~8/29/2025 0:00:00~"Original"~1~"4878-4"~"PMR"~"505 (o)(3)"~4878.00~4~"PMR 4878-4: Conduct a dedicated renal impairment study to evaluate the effect of severe renal impairment (eGFR <30 mL/min) on Wayrilz (rilzabrutinib) pharmacokinetics compared to subjects with normal renal function. The study will assess the magnitude of exposure change of rilzabrutinib and inform appropriate dosing instructions for patients with severe renal impairment. This study should be designed and conducted in accordance with Guidance for Industry: Pharmacokinetics in Patients with Impaired Renal Function   Study Design, Data Analysis, and Impact on Dosing (March 2024)."~"Pending"~~3/31/2028 0:00:00~
385260~"CDER"~"NDA"~219713.00~"NUVATION BIO INC"~"Ibtrozi (taletrectinib) Capsule"~6/11/2025 0:00:00~"Original"~1~"4856-1"~"PMR"~"Pediatric Research Equity Act"~4856.00~1~"PMR 4856-1: Conduct a molecularly targeted pediatric cancer investigation to assess dosing, pharmacokinetics, safety and preliminary efficacy of taletrectinib in pediatric patients with advanced or metastatic solid tumors with NTRK or ROS1 alterations."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~12/31/2033 0:00:00~
385261~"CDER"~"NDA"~219713.00~"NUVATION BIO INC"~"Ibtrozi (taletrectinib) Capsule"~6/11/2025 0:00:00~"Original"~1~"4856-2"~"PMR"~"505 (o)(3)"~4856.00~2~"PMR 4856-2: Conduct a multicenter trial to further characterize known serious risks with taletrectinib including severe hepatotoxicity, interstitial lung disease, other serious adverse reactions, and the risk of gastrointestinal toxicity, by evaluating the safety, activity, and pharmacokinetics of taletrectinib 400 mg daily taken with standard meals, in patients with advanced or metastatic ROS1-positive non-small cell lung cancer who are na ve to prior ROS1 tyrosine kinase inhibitors (TKIs) and in patients who have received one prior ROS1 TKI. Include an adequate number of patients from each subgroup (i.e., ROS1 TKI-na ve and previously treated with ROS1 TKI)."~"Pending"~~6/30/2028 0:00:00~
385262~"CDER"~"NDA"~219713.00~"NUVATION BIO INC"~"Ibtrozi (taletrectinib) Capsule"~6/11/2025 0:00:00~"Original"~1~"4856-3"~"PMR"~"505 (o)(3)"~4856.00~3~"PMR 4856-3: Conduct a hepatic impairment clinical trial to evaluate the serious potential risk of increased drug exposure and determine a safe and appropriate dosage of taletrectinib in patients with moderate and severe hepatic impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~12/31/2027 0:00:00~
385263~"CDER"~"NDA"~219713.00~"NUVATION BIO INC"~"Ibtrozi (taletrectinib) Capsule"~6/11/2025 0:00:00~"Original"~1~"4856-4"~"PMR"~"505 (o)(3)"~4856.00~4~"PMR 4856-4: Conduct a clinical drug interaction study to evaluate the effect of taletrectinib on the pharmacokinetics of sensitive substrates of CYP3A, CYP2D6, BCRP, OATP1B1, and OATP1B3 to evaluate the magnitude of exposure change of these substrates and serious potential risk of increased drug toxicity and inform appropriate drug interaction management strategy for coadministration of taletrectinib with these CYP and transporter substrates. This study should be designed and conducted in accordance with  M12 Drug Interaction Studies. "~"Pending"~~12/31/2029 0:00:00~
385264~"CDER"~"NDA"~219713.00~"NUVATION BIO INC"~"Ibtrozi (taletrectinib) Capsule"~6/11/2025 0:00:00~"Original"~1~"4856-5"~"PMC"~"506B PMC"~4856.00~5~"PMC 4856-5: Conduct a clinical drug interaction study to evaluate the effect of taletrectinib on the pharmacokinetics of sensitive substrates of CYP3A and CYP1A2 in healthy subjects to evaluate the magnitude of decreased drug exposure and inform appropriate drug interaction management strategy for coadministration of taletrectinib with these CYP substrates. This study should be designed and conducted in accordance with  M12 Drug Interaction Studies. "~"Pending"~~12/31/2029 0:00:00~
385265~"CDER"~"NDA"~219713.00~"NUVATION BIO INC"~"Ibtrozi (taletrectinib) Capsule"~6/11/2025 0:00:00~"Original"~1~"4856-6"~"PMC"~"506B PMC"~4856.00~6~"PMC 4856-6: Conduct a clinical trial to evaluate if staggered administration of an H2receptor antagonist decreases the exposure of taletrectinib and to inform instructions for taking taletrectinib with H2-receptor antagonists. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Evaluation of Gastric pH-Dependent Drug Interactions with AcidReducing Agents: Study Design, Data Analysis, and Clinical Implications. "~"Pending"~~12/31/2027 0:00:00~
385266~"CDER"~"BLA"~761088.00~"CELLTRION, Inc."~"Truxima (rituximab-abbs)"~11/28/2018 0:00:00~"Supplement"~6~"3772-1"~"PMR"~"Pediatric Research Equity Act"~3772.00~1~"PMR 3772-1: Assessment of Truxima (rituximab-abbs) for the treatment of Granulomatosis with Polyangiitis and Microscopic Polyangiitis in pediatric patients 2 years of age and older."~"Pending"~"The study has not begun but does not meet the criterion for
delayed."~9/30/2026 0:00:00~1/20/2026 0:00:00
385267~"CDER"~"BLA"~761089.00~"Teva Branded Pharmaceutical Products R&D, Inc."~"Ajovy (fremanezumab-vfrm)"~9/14/2018 0:00:00~"Original"~1~"3485-1"~"PMR"~"Pediatric Research Equity Act"~3485.00~1~"PMR 3485-1: A juvenile animal toxicology study in one species."~"Submitted"~"The final report was submitted on 5/29/2018."~4/30/2018 0:00:00~11/12/2025 0:00:00
385268~"CDER"~"BLA"~761089.00~"Teva Branded Pharmaceutical Products R&D, Inc."~"Ajovy (fremanezumab-vfrm)"~9/14/2018 0:00:00~"Original"~1~"3485-2"~"PMR"~"Pediatric Research Equity Act"~3485.00~2~"PMR 3485-2: An open-label pharmacokinetic, safety, and tolerability study in pediatric migraine patients ages 6 through 11 years, with an optional open-label safety extension phase (40 weeks)."~"Fulfilled"~"Per FDA letter dated 8/05/2025, this PMR/PMC has been fulfilled."~12/31/2022 0:00:00~11/12/2025 0:00:00
385269~"CDER"~"BLA"~761089.00~"Teva Branded Pharmaceutical Products R&D, Inc."~"Ajovy (fremanezumab-vfrm)"~9/14/2018 0:00:00~"Original"~1~"3485-3"~"PMR"~"Pediatric Research Equity Act"~3485.00~3~"PMR 3485-3: Deferred pediatric randomized, double-blind, placebo-controlled efficacy and safety study under PREA for the preventive treatment of episodic migraine in children and adolescents ages 6 through 17 years. This study includes a doubleblind treatment phase (12 weeks) and an open-label safety extension phase (40 weeks). This study is to be submitted as a special protocol assessment (SPA)."~"Delayed"~"Original Final Report Due Date: 12/31/2022; Deferral Extension granted per FDA letter dated 09/22/2023. The final protocol submission and study completion milestones were also revised due to delays involving study participants, sites, and/or management."~12/31/2026 0:00:00~11/12/2025 0:00:00
385270~"CDER"~"BLA"~761089.00~"Teva Branded Pharmaceutical Products R&D, Inc."~"Ajovy (fremanezumab-vfrm)"~9/14/2018 0:00:00~"Original"~1~"3485-4"~"PMR"~"Pediatric Research Equity Act"~3485.00~4~"PMR 3485-4: Deferred pediatric randomized, double-blind, placebo-controlled efficacy and safety study under PREA for the preventive treatment of chronic migraine in adolescents ages 12 through 17 years. This study includes a double-blind treatment phase (12 weeks) and an open-label safety extension phase (40 weeks). This study is to be submitted as a special protocol assessment (SPA)."~"Delayed"~"Original Final Report Due Date: 12/31/2023; Deferral Extension granted per FDA letter dated 09/22/2023. The final protocol submission and study completion milestones were also revised due to delays involving study participants, sites, and/or management."~12/31/2026 0:00:00~11/12/2025 0:00:00
385271~"CDER"~"BLA"~761089.00~"Teva Branded Pharmaceutical Products R&D, Inc."~"Ajovy (fremanezumab-vfrm)"~9/14/2018 0:00:00~"Original"~1~"3485-5"~"PMR"~"505 (o)(3)"~3485.00~5~"PMR 3485-5: Conduct prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with migraine exposed to Ajovy during pregnancy with two unexposed control populations: one consisting of women with migraine who have not been exposed to Ajovy before or during pregnancy and the other consisting of women without migraine. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~12/31/2028 0:00:00~11/12/2025 0:00:00
385272~"CDER"~"BLA"~761089.00~"Teva Branded Pharmaceutical Products R&D, Inc."~"Ajovy (fremanezumab-vfrm)"~9/14/2018 0:00:00~"Original"~1~"3485-6"~"PMR"~"505 (o)(3)"~3485.00~6~"PMR 3485-6: Conduct a pregnancy outcomes study using a different study design than provided for in PMR 3485-5 (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small-for-gestational-age births in women exposed to Ajovy during pregnancy compared to an unexposed control population."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 03/06/2023."~12/31/2028 0:00:00~11/12/2025 0:00:00
385273~"CDER"~"BLA"~761097.00~"Regeneron Pharmaceuticals, Inc."~"LIBTAYO (cemiplimab-rwlc)"~9/28/2018 0:00:00~"Supplement"~8~"4011-1"~"PMC"~"506B PMC"~4011.00~1~"PMC 4011-1: Submit the final analysis of objective response rate and duration of response for the 84 patients with locally advanced basal cell carcinoma (laBCC) whose data was included in BLA supplement-008, in order to further characterize the durability of response in this cohort. For the analysis, all patients will have the opportunity for at least 1.5 years of follow-up following completion of cemiplimab-rwlc treatment. The study results may inform product labeling."~"Fulfilled"~~5/31/2024 0:00:00~11/21/2025 0:00:00
385274~"CDER"~"BLA"~761097.00~"Regeneron Pharmaceuticals, Inc."~"LIBTAYO (cemiplimab-rwlc)"~9/28/2018 0:00:00~"Supplement"~9~"4012-2"~"PMC"~"506B PMC"~4012.00~2~"PMC 4012-2: Submit the final analysis of objective response rate and duration of response for the 53 patients with metastatic basal cell carcinoma (mBCC) from clinical study R2810 ONC 1620, in order to further characterize the durability of response in this cohort. For the analysis, all patients will have  the opportunity for at least 1.5 years of follow-up following completion of cemiplimab-rwlc treatment. The study results may inform product labeling."~"Fulfilled"~~5/31/2024 0:00:00~11/21/2025 0:00:00
385275~"CDER"~"BLA"~761097.00~"Regeneron Pharmaceuticals, Inc."~"LIBTAYO (cemiplimab-rwlc)"~9/28/2018 0:00:00~"Supplement"~14~"4349-1"~"PMC"~"506B PMC"~4349.00~1~"PMC 4349-1: Complete Part 2 of Study 16113 entitled,  A Two-Part Randomized, Phase 3 Study of Combination of Cemiplimab (Anti-PD-1 Antibody) and Platinum-Based Doublet Chemotherapy in First-Line Treatment of Patients with Advanced or Metastatic Non-Small Cell Lung Cancer , to obtain mature overall survival data in accordance with the prespecified final analysis of Part 2 of Study 16113."~"Submitted"~~6/30/2023 0:00:00~11/21/2025 0:00:00
385276~"CDER"~"BLA"~761084.00~"Kashiv BioSciences, LLC"~"Fylnetra (pegfilgrastim-pbbk)"~5/26/2022 0:00:00~"Original"~1~"4277-1"~"PMR"~"Pediatric Research Equity Act"~4277.00~1~"PMR 4277-1: Develop an appropriate formulation (presentation) that can be used to directly and accurately administer Fylnetra (pegfilgrastim-pbbk) to pediatric patients who weigh less than 45 kg and require doses that are less than 0.6 mL (6 mg), and conduct any necessary human factors studies to evaluate the ability of healthcare providers and/or caregivers to measure the appropriate doses."~"Pending"~"Extension for Pediatric Assessment Study received from the Agency on September 18, 2024, up to April 2028. The study/trial has not begun but does not meet the criterion for delayed. Original Final Report Due Date: 10/31/2025; Deferral Extension granted per FDA letter dated 09/18/2024."~4/30/2028 0:00:00~7/8/2026 0:00:00
385277~"CDER"~"BLA"~761145.00~"Janssen Biotech, Inc."~"Darzalex Faspro (daratumumab and hyaluronidase-fihj)"~5/1/2020 0:00:00~"Supplement"~2~"3951-1"~"PMR"~"Accelerated Approval"~3951.00~1~"PMR 3951-1: Submit the final study report and datasets from a randomized clinical trial to verify and further characterize the clinical benefit and safety of daratumumab subcutaneous for the treatment of patients with AL amyloidosis. This submission should include the final analysis and datasets of progression free survival or overall survival results."~"Fulfilled"~"Per FDA letter dated 11/19/2025, this PMR has been fulfilled."~6/30/2025 0:00:00~6/23/2026 0:00:00
385278~"CDER"~"BLA"~761145.00~"Janssen Biotech, Inc."~"Darzalex Faspro (daratumumab and hyaluronidase-fihj)"~5/1/2020 0:00:00~"Supplement"~2~"3951-2"~"PMR"~"505 (o)(3)"~3951.00~2~"PMR 3951-2: Conduct clinical trials in newly diagnosed and relapsed/refractory AL amyloidosis to assess (1) all serious cardiovascular adverse events on study treatment; (2) all deaths on study treatment and; (3) the risk factors for cardiac toxicity and the adequacy of monitoring in at least 100 patients treated with daratumumab subcutaneous for at least 6 months. Data from clinical trials in patients with relapsed / refractory AL amyloidosis will be considered supportive. Characterize the incidence, clinical presentation, management and outcome of these events and identify those that represent major adverse cardiovascular events; namely nonfatal myocardial infarction, cardiac failure and arrhythmia, or fatal cardiovascular adverse events and eventsof sudden death. Also, identify hospitalizations for unstable angina, coronary revascularization procedures, and serious adverse events of heart failure. Include an evaluation of potential mitigation strategies for cardiac toxicity. The interim report should contain results from the first completed clinical trial."~"Submitted"~~2/28/2026 0:00:00~6/23/2026 0:00:00
385279~"CDER"~"BLA"~761145.00~"Janssen Biotech, Inc."~"Darzalex Faspro (daratumumab and hyaluronidase-fihj)"~5/1/2020 0:00:00~"Supplement"~2~"3951-3"~"PMR"~"505 (o)(3)"~3951.00~3~"PMR 3951-3: Conduct a clinical trial to assess the safety of daratumumab subcutaneous (SC) among U.S. racial and ethnic minorities including African American patients with AL amyloidosis given the higher pharmacokinetic (PK) exposure and hematologic toxicity rates (neutropenia, lymphopenia, thrombocytopenia and anemia). This study should characterize the exposure (including PK data), safety, and efficacy of daratumumab SC."~"Submitted"~~6/30/2024 0:00:00~6/23/2026 0:00:00
385280~"CDER"~"BLA"~761145.00~"Janssen Biotech, Inc."~"Darzalex Faspro (daratumumab and hyaluronidase-fihj)"~5/1/2020 0:00:00~"Supplement"~7~"4074-1"~"PMR"~"505 (o)(3)"~4074.00~1~"PMR 4074-1: Conduct a clinical study to further characterize the exposure of daratumumab (D) subcutaneous (SC), the increased risk of severe and serious adverse events, including severe neutropenia, and efficacy among U.S. racial and ethnic minority patients with relapsed or refractory multiple myeloma. Include an assessment of the PK, PD, safety, and efficacy of daratumumab SC in combination with other agents including pomalidomide and dexamethasone (Pd) in U.S. racial and ethnic minority patients including Black and Asian patients with relapsed or refractory multiple myeloma in the final study report. The population pharmacokinetic and exposure-response analyses for both efficacy and safety should be updated."~"Submitted"~~1/31/2026 0:00:00~6/23/2026 0:00:00
385281~"CDER"~"BLA"~761145.00~"Janssen Biotech, Inc."~"Darzalex Faspro (daratumumab and hyaluronidase-fihj)"~5/1/2020 0:00:00~"Supplement"~7~"4074-2"~"PMR"~"505 (o)(3)"~4074.00~2~"PMR 4074-2: Conduct a prospective, observational single-arm study to assess the risk of severe (Grades 3-4) and fatal infusion-related reactions (IRRs) inpatients treated with intravenous (IV) or sub-cutaneous (SC) daratumumab. Evaluate the incidence of severe and fatal IRRs, and collect information, including a full description of clinical features of the adverse reactions, to investigate associations and temporal relationships between the incidence and severity of IRRs and other potential associated risk factors. Specify case definitions, validation methods, and procedures for all study outcomes. Submit interim reports of the data collected from the study annually until the study is completed."~"Ongoing"~~7/31/2026 0:00:00~6/23/2026 0:00:00
385282~"CDER"~"BLA"~761145.00~"Janssen Biotech, Inc."~"Darzalex Faspro (daratumumab and hyaluronidase-fihj)"~5/1/2020 0:00:00~"Supplement"~7~"4074-3"~"PMC"~"506B PMC"~4074.00~3~"PMC 4074-3: Submit a final report containing data from clinical trials, post-marketing reports, compassionate use/expanded access programs, real-world evidence, and other sources to further characterize the safety and efficacy of daratumumab (SC) in combination with pomalidomide and dexamethasone among U.S. racial and ethnic minority patients with multiple myeloma."~"Submitted"~~1/31/2026 0:00:00~6/23/2026 0:00:00
385283~"CDER"~"BLA"~761145.00~"Janssen Biotech, Inc."~"Darzalex Faspro (daratumumab and hyaluronidase-fihj)"~5/1/2020 0:00:00~"Supplement"~9~"4183-1"~"PMC"~"506B PMC"~4183.00~1~"PMC 4183-1: Conduct an integrated study analysis containing data from clinical trials, post-marketing reports, compassionate use/expanded access programs, real-world evidence, and other sources to further characterize the safety and efficacy of daratumumab (SC) in combination with carfilzomib and dexamethasone among U.S. racial and ethnic minority patients with multiple myeloma. 
"~"Ongoing"~~8/31/2026 0:00:00~6/23/2026 0:00:00
385284~"CDER"~"BLA"~761145.00~"Janssen Biotech, Inc."~"Darzalex Faspro (daratumumab and hyaluronidase-fihj)"~5/1/2020 0:00:00~"Supplement"~29~"4868-1"~"PMC"~"506B PMC"~4868.00~1~"PMC 4868-1: Conduct an integrated analysis of data from clinical trials, and other sources such as post-marketing reports, to further characterize the efficacy and safety of daratumumab and hyaluronidase in a pooled population that includes a sufficient representation of racial and ethnic populations that are reflective of the U.S. population with high-risk smoldering multiple myeloma and allow for an interpretation of the data in these subgroups."~"Pending"~~2/28/2030 0:00:00~6/23/2026 0:00:00
385285~"CDER"~"BLA"~761147.00~"Accord BioPharma Inc."~"Ennumo (pegfilgrastim-pccg)"~5/7/2026 0:00:00~"Original"~1~"4320-1"~"PMR"~"Pediatric Research Equity Act"~4320.00~1~"PMR 4320-1: Develop a presentation that can be used to accurately administer Ennumo (pegfilgrastim-pccg) to pediatric patients who weigh less than 45 kg."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~10/31/2027 0:00:00~
385286~"CDER"~"BLA"~761148.00~"Assertio Specialty Pharmaceuticals, LLC"~"Rolvedon (eflapegrastim)"~9/9/2022 0:00:00~"Original"~1~"3891-1"~"PMR"~"Pediatric Research Equity Act"~3891.00~1~"PMR 3891-1: Conduct a study to assess the safety, pharmacokinetics and pharmacodynamics of eflapegrastim-xnst in pediatric patients 1 month to <17 years of age with solid tumors treated with myelosuppressive chemotherapy. Submit the final clinical study report including datasets as a supplemental BLA."~"Ongoing"~"This study is currently ongoing. 13 patients out of the planned 40 patients have been enrolled into the trial.
"~12/31/2027 0:00:00~11/6/2025 0:00:00
385287~"CDER"~"BLA"~761148.00~"Assertio Specialty Pharmaceuticals, LLC"~"Rolvedon (eflapegrastim)"~9/9/2022 0:00:00~"Original"~1~"3891-2"~"PMR"~"Pediatric Research Equity Act"~3891.00~2~"PMR 3891-2: Submit pediatric assessments for Rolvedon (eflapegrastim-xnst) as described in section 505B(a)(2)(A) of the FD&C Act, including development of an  appropriate formulation  (presentation) that can be used to directly and accurately administer Rolvedon (eflapegrastim-xnst) to pediatric patients (1 month to <17 years of age). Conduct any necessary human factors studies to evaluate the ability of healthcare providers and caregivers to measure the appropriate doses."~"Ongoing"~"The study has been initiated."~6/30/2026 0:00:00~11/6/2025 0:00:00
385288~"CDER"~"BLA"~761326.00~"Novo Nordisk Inc."~"Awiqli FlexTouch (insulin icodec-abae)"~3/26/2026 0:00:00~"Original"~2~"4974-1"~"PMR"~"Pediatric Research Equity Act"~4974.00~1~"PMR 4974-1: Complete the multicenter, open-label, one-period, single-dose trial to evaluate the pharmacokinetic properties of insulin icodec-abae
(NNC0148-0287) in children and adolescent patients ages 10 to <18 years with type 2 diabetes mellitus."~"Ongoing"~"The study is underway"~6/30/2026 0:00:00~
385289~"CDER"~"BLA"~761328.00~"Sanofi Vaccines US Inc."~"Beyfortus (nirsevimab-alip)"~7/17/2023 0:00:00~"Original"~1~"4470-1"~"PMR"~"505 (o)(3)"~4470.00~1~"PMR 4470-1: Provide reports on the prevalence of current and emerging RSV variants, including the frequency of known nirsevimab resistance-associated substitutions, on an annual (AZ-sponsored surveillance studies) or 6-monthly (clinical trials, public sequence databases) basis with the periodic safety report. These study reports should include genotypic data
from public sequence databases (i.e., GISAID, NCBI GenBank), and both genotypic and phenotypic data from ongoing clinical studies and surveillance activities, including all new variants and substitutions showing
=5-fold reduction in susceptibility: OUTSMART-RSV and INFORM-RSV studies Clinical trials of nirsevimab"~"Ongoing"~~1/31/2030 0:00:00~9/12/2025 0:00:00
385290~"CDER"~"BLA"~761328.00~"Sanofi Vaccines US Inc."~"Beyfortus (nirsevimab-alip)"~7/17/2023 0:00:00~"Original"~1~"4470-2"~"PMR"~"505 (o)(3)"~4470.00~2~"PMR 4470-2: Based on surveillance studies and a pooled analysis of RSV A and RSV B isolates from nirsevimab-treated subjects (inclusive of all subjects meeting primary, secondary, or exploratory case definitions), assess phenotypically the individual and concurrent substitutions shown below. RSV A Individual substitutions S62G, K65Q, K65R, E110G, L111I, L119I, K123Q, D200N, V247L, W341R, K419E, N515H RSV B Individual substitutions R42K, K68R, S190N, N200Y, L204S, N208I, K272R, S389P Concurrent substitutions  K68N+I206M+Q209R  I206M+Q209R+K272R  F15L+A103V+L172Q+S173L  F15L+A103V+L172Q+S173L+K191R+I206M+Q209R  F15L+A103V+L172Q+S173L+S190N+K191R+I206M+Q209R+S211N+S389P  F15L+R42K+A103V+L172Q+S173L+S190N+K191R+I206M+Q209R+ S211N+S389P   L204S+I206M+Q209R+S211N"~"Fulfilled"~~12/31/2025 0:00:00~9/12/2025 0:00:00
385291~"CDER"~"BLA"~761328.00~"Sanofi Vaccines US Inc."~"Beyfortus (nirsevimab-alip)"~7/17/2023 0:00:00~"Original"~1~"4470-3"~"PMC"~"506B PMC"~4470.00~3~"PMC 4470-3: Conduct a long-term follow-up study (Study D5290N00001: HARMONIE study extension for the UK cohort) to evaluate antibody dependent enhancement of RSV disease after nirsevimab administration to neonates and infants prior to or during their first RSV season. The assessment for
antibody dependent enhancement of RSV disease should include RSV lower respiratory tract infection (LRTI) hospitalization events. The follow- up period should continue through Day 511 post-dosing."~"Ongoing"~~10/31/2025 0:00:00~9/12/2025 0:00:00
385292~"CDER"~"BLA"~761328.00~"Sanofi Vaccines US Inc."~"Beyfortus (nirsevimab-alip)"~7/17/2023 0:00:00~"Original"~1~"4470-4"~"PMC"~"506B PMC"~4470.00~4~"PMC 4470-4: Conduct an observational, U.S.-based long-term follow-up study of infants eligible to receive nirsevimab in their first year of life to assess the impact of RSV disease through Day 511 post dosing. This study should include assessment of MA RSV LRTI and RSV hospitalization. The study may be conducted using existing databases for the 2023 and 2024 RSV seasons."~"Pending"~~3/31/2029 0:00:00~9/12/2025 0:00:00
385293~"CDER"~"BLA"~761328.00~"Sanofi Vaccines US Inc."~"Beyfortus (nirsevimab-alip)"~7/17/2023 0:00:00~"Original"~1~"4470-5"~"PMC"~"506B PMC"~4470.00~5~"PMC 4470-5: Submit the interim and the final study reports and datasets for the ongoing HARMONIE trial,  A Phase IIIb Randomized Open-label Study of Nirsevimab (Versus no Intervention) in Preventing Hospitalizations Due to Respiratory Syncytial Virus in Infants. "~"Fulfilled"~~7/31/2024 0:00:00~9/12/2025 0:00:00
385294~"CDER"~"BLA"~761328.00~"Sanofi Vaccines US Inc."~"Beyfortus (nirsevimab-alip)"~7/17/2023 0:00:00~"Original"~1~"4470-9"~"PMC"~"506B PMC"~4470.00~9~"PMC 4470-9: Submit the final study report and datasets for the CHIMES trial (D5290C00006; NCT05110261),  A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Nirsevimab, a Monoclonal Antibody With Extended Half-life Against Respiratory Syncytial Virus, in Healthy Preterm and Term Infants in China. "~"Ongoing"~~8/31/2026 0:00:00~9/12/2025 0:00:00
385295~"CDER"~"BLA"~761334.00~"Incyte Corporation"~"Zynyz (retifanlimab-dlwr)"~3/22/2023 0:00:00~"Original"~1~"4412-1"~"PMR"~"Accelerated Approval"~4412.00~1~"PMR 4412-1: Conduct a multicenter clinical trial intended to confirm the clinical benefit of retifanlimab-dlwr in patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC) who have not received prior systemic therapies for metastatic or recurrent locally advanced MCC. The trial will enroll at least 100 patients to be followed for a minimum of 12 months to establish the objective response rate and characterize the durability of response. Include an analysis of overall survival, when 70% of patients have died, or all patients have been followed for at least three years."~"Fulfilled"~"Per FDA letter dated 12/17/2025, this PMR/PMC has been fulfilled."~3/31/2025 0:00:00~5/18/2026 0:00:00
385296~"CDER"~"BLA"~761334.00~"Incyte Corporation"~"Zynyz (retifanlimab-dlwr)"~3/22/2023 0:00:00~"Supplement"~4~"4839-1"~"PMC"~"506B PMC"~4839.00~1~"PMC 4839-1: Complete the ongoing randomized clinical trial, INCMGA 0012-303 (NCT04472429), entitled  A Phase 3 Global, Multicenter, Double-Blind Randomized Study of Carboplatin-Paclitaxel With INCMGA00012 or Placebo in Participants With Inoperable Locally Recurrent or Metastatic Squamous Cell Carcinoma of the Anal Canal Not Previously Treated With Systemic Chemotherapy (POD1UM-303/InterAACT 2)  and provide the final overall survival analysis to further characterize the efficacy of retifanlimab in combination with systemic chemotherapy for the treatment of inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal."~"Fulfilled"~~3/31/2027 0:00:00~5/18/2026 0:00:00
385297~"CDER"~"BLA"~761336.00~"Altor BioScience, LLC, an indirect wholly-owned subsidiary of ImmunityBio, Inc."~"Anktiva (nogapendekin alfa inbakicept-pmln)"~4/22/2024 0:00:00~"Original"~1~"4417-1"~"PMC"~"506B PMC"~4417.00~1~"PMC 4417-1: Complete clinical trial  QUILT-3.032: A Multicenter Clinical Trial of Intravesical Bacillus Calmette-Guerin (BCG) in Combination With ALT-803 (N-803) in Patients With BCG Unresponsive High Grade Non-Muscle Invasive Bladder Cancer,  and provide annual updates on enrollment, complete response rate, and duration of response for all patients enrolled in Cohort 1 (non-muscle invasive bladder cancer [NMIBC] carcinoma in situ [CIS]). Annual reports should continue until all patients have either experienced recurrence of high-grade non-muscle invasive bladder cancer, progression, death, or been lost to follow-up, for up to 4 years."~"Ongoing"~~12/31/2029 0:00:00~6/16/2026 0:00:00
385298~"CDER"~"BLA"~761339.00~"Regeneron Pharmaceuticals, Inc."~"Veopoz (pozelimab-bbfg)"~8/18/2023 0:00:00~"Original"~1~"4490-1"~"PMC"~"506B PMC"~4490.00~1~"PMC 4490-1: Re-evaluate the current anti-drug antibody (ADA) assay with the goal of improving sensitivity in the presence of the trough level of drug expected to be present during sampling. The assay validation assessments will include selectivity, system suitability specifications for negative and positive controls, effects of hemolysis, and a statistical evaluation of distribution and outlier exclusion for cutpoint samples."~"Submitted"~~11/30/2025 0:00:00~10/17/2025 0:00:00
385299~"CDER"~"NDA"~219713.00~"NUVATION BIO INC"~"Ibtrozi (taletrectinib) Capsule"~6/11/2025 0:00:00~"Original"~1~"4856-7"~"PMR"~"505 (o)(3)"~4856.00~7~"PMR 4856-7: Complete a clinical trial to further characterize the clinical benefit of taletrectinib for the treatment of adult patients with ROS1 fusion-positive metastatic NSCLC by providing a more precise estimation of the BICRassessed overall response rate (ORR) and duration of response (DOR) in the ROS1 TKI-naive patients with ROS1-positive NSCLC and ROS1 TKIpretreated patients enrolled in TRUST-I and TRUST-II studies. Include updated DOR results for the 139 responders in the response evaluable population of 157 ROS1 TKI-na ve patients and for the 63 responders in the response evaluable population of 113 ROS1 TKI-pretreated patients,after all responders have been followed for at least 18 months from the date of initial response."~"Submitted"~~6/30/2028 0:00:00~
385300~"CDER"~"NDA"~219713.00~"NUVATION BIO INC"~"Ibtrozi (taletrectinib) Capsule"~6/11/2025 0:00:00~"Original"~1~"4856-8"~"PMC"~"506B PMC"~4856.00~8~"PMC 4856-8: Conduct an appropriate analytical and clinical validation study to support the development of an in vitro diagnostic device using clinical trial data that demonstrates that the device is essential to the safe and effective use of taletrectinib for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer."~"Pending"~~6/30/2027 0:00:00~
385301~"CDER"~"NDA"~219757.00~"ROSEMONT PHARMACEUTICALS HOLDINGS INC"~"Vostally (ramipril) oral solution"~7/23/2025 0:00:00~"Original"~1~"4870-1"~"PMR"~"Pediatric Research Equity Act"~4870.00~1~"PMR 4870-1: Conduct an efficacy, safety, and pharmacokinetic study to identify a dosing regimen of ramipril oral solution for the treatment of hypertension in pediatric patients aged 2 years to less than 17 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~1/31/2029 0:00:00~
385302~"CDER"~"NDA"~219792.00~"UCB INC"~"Kygevvi (doxecitine and doxribtimine)"~11/3/2025 0:00:00~"Original"~1~"4880-1"~"PMR"~"505 (o)(3)"~4880.00~1~"PMR 4880-1: Conduct a 26-week carcinogenicity study in Tg-rasH2 mice to identify and evaluate the carcinogenic risk of doxecitine and doxribtimine."~"Pending"~~11/30/2028 0:00:00~
385303~"CDER"~"NDA"~219792.00~"UCB INC"~"Kygevvi (doxecitine and doxribtimine)"~11/3/2025 0:00:00~"Original"~1~"4880-2"~"PMR"~"505 (o)(3)"~4880.00~2~"PMR 4880-2: Conduct an in vitro study(ies) with quantitative data to describe the effects of doxecitine and doxribtimine on the mutagenic and clastogenic potential of [...]."~"Pending"~~6/30/2028 0:00:00~
385304~"CDER"~"NDA"~219792.00~"UCB INC"~"Kygevvi (doxecitine and doxribtimine)"~11/3/2025 0:00:00~"Original"~1~"4880-3"~"PMR"~"Pediatric Research Equity Act"~4880.00~3~"PMR 4880-3: Conduct a nonclinical Pig-A assay study to evaluate the mutagenic potential of [...] in the presence and absence of doxecitine and doxribtimine. The design and conduct of this study will be informed by the study results from PMR 4880-2."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~7/31/2030 0:00:00~
385305~"CDER"~"NDA"~219792.00~"UCB INC"~"Kygevvi (doxecitine and doxribtimine)"~11/3/2025 0:00:00~"Original"~1~"4880-4"~"PMC"~"506B PMC"~4880.00~4~"PMC 4880-4: Conduct a 39-week toxicology study in dogs to identify and evaluate the potential toxic effects of doxecitine and doxribtimine administered chronically."~"Pending"~~12/31/2029 0:00:00~
385306~"CDER"~"NDA"~219835.00~"RIGEL PHARMACEUTICALS INC"~"Veppanu (vepdegestrant) tablets"~5/1/2026 0:00:00~"Original"~1~"4993-1"~"PMR"~"505 (o)(3)"~4993.00~1~"PMR 4993-1: Conduct a clinical trial that evaluates the effect of moderate and severe hepatic impairment on the pharmacokinetics of vepdegestrant, to assess the potential serious risk of increased drug toxicity and to inform appropriate dose recommendations in patients with hepatic impairment. Design and conduct the trial in accordance with the FDA guidance for industry titled,  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling ."~"Pending"~~6/30/2027 0:00:00~
385307~"CDER"~"NDA"~219835.00~"RIGEL PHARMACEUTICALS INC"~"Veppanu (vepdegestrant) tablets"~5/1/2026 0:00:00~"Original"~1~"4993-2"~"PMC"~"506B PMC"~4993.00~2~"PMC 4993-2: Complete the ongoing clinical trial, VERITAC-2 (NCT05654623), titled ""A Phase 3, Randomized, Open-Label, Multicenter Trial of ARV-471 (PF-07850327) vs Fulvestrant in Participants With Estrogen Receptor-Positive, HER2-Negative Advanced Breast Cancer Whose Disease Progressed After Prior Endocrine Based Treatment for Advanced Disease,"" and provide the subsequent pre-planned interim and final overall survival (OS) results, to further characterize the clinical benefit of vepdegestrant as monotherapy treatment for adult patients with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy."~"Pending"~~3/31/2030 0:00:00~
385308~"CDER"~"NDA"~219839.00~"DIZAL PHARMACEUTICAL CO LTD"~"Zegfrovy (sunvozertinib) Tablet, 150mg and 200mg"~7/2/2025 0:00:00~"Original"~1~"4867-1"~"PMR"~"Accelerated Approval"~4867.00~1~"PMR 4867-1: Conduct a multicenter, randomized clinical trial intended to verify and describe the clinical benefit of sunvozertinib in patients with locally advanced, unresectable or metastatic non-small cell lung cancer whose tumors have EGFR exon 20 insertion mutations. The final analysis should include the final progression-free survival and overall survival results. This data may be obtained from the ongoing clinical trial, entitled, DZ2022E0005 (WU-KONG28),  A Phase 3, Open-Label, Randomized, Multi-Center Study of DZD9008 versus Platinum-Based Doublet Chemotherapy as First-Line Treatment for Patients with Locally Advanced or Metastatic Non-Small Cell Lung Cancer Harboring Epidermal Growth Factor Receptor Exon 20 Insertion Mutation. "~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2026 0:00:00~
385309~"CDER"~"NDA"~219843.00~"ROSEMONT PHARMACEUTICALS HOLDINGS INC"~"Atmeksi (methocarbamol) oral suspension"~7/30/2025 0:00:00~"Original"~1~"4858-1"~"PMC"~"506B PMC"~4858.00~1~"PMC 4858-1: Perform a leachable study for the first three commercial batches placed on stability per ICH Q1A with samples stored in the inverted position and tested at the 0, 3, 9, 12-month time points through to the end of shelf-life. Submit a toxicological risk assessment for all individual leachable compounds that exceed the 5 mcg/day qualification threshold. The risk assessment should be based on the maximum level of each leachable detected in long-term stability samples. Include copies of all referenced studies upon which a safety assessment is based."~"Delayed"~"The applicant requested revised milestones because of manufacturing delays. Revised milestones were acknowledged in a letter dated 12/03/2025."~7/31/2027 0:00:00~
385310~"CDER"~"NDA"~219876.00~"CHIMERIX INC"~"Modeyso (dordaviprone) Capsules"~8/6/2025 0:00:00~"Original"~1~"4861-1"~"PMR"~"Accelerated Approval"~4861.00~1~"PMR 4861-1: Complete a multiregional, randomized clinical trial in patients with H3 K27M-mutant diffuse midline glioma, intended to verify and describe the clinical benefit of dordaviprone through assessment of overall survival (OS) as a primary endpoint."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2031 0:00:00~
385311~"CDER"~"BLA"~761097.00~"Regeneron Pharmaceuticals, Inc."~"LIBTAYO (cemiplimab-rwlc)"~9/28/2018 0:00:00~"Supplement"~14~"4349-2"~"PMC"~"506B PMC"~4349.00~2~"PMC 4349-2: Conduct an integrated analysis containing data from ongoing or planned clinical trials and other data sources such as post-marketing reports, real-world evidence and other sources to further characterize the safety and efficacy of cemiplimab-rwlc in combination with platinum-doublet chemotherapy in older adults (e.g., ages 75 years and older), females, and racial and ethnic minority patients that is reflective of the U.S. population of patients with NSCLC. In the analysis, include a sufficient number of patients enrolled in the U.S., ages 75 years and older, females, and a sufficient number of racial and ethnic minorities reflective of the incidence of NSCLC in each subpopulation to allow for interpretation of the results. The analyses should support comparative safety and efficacy outcome analyses between the aforementioned populations and White, male, and younger patients."~"Ongoing"~~9/30/2027 0:00:00~11/21/2025 0:00:00
385312~"CDER"~"BLA"~761097.00~"Regeneron Pharmaceuticals, Inc."~"LIBTAYO (cemiplimab-rwlc)"~9/28/2018 0:00:00~"Supplement"~30~"4899-1"~"PMC"~"506B PMC"~4899.00~1~"PMC 4899-1: Complete the ongoing clinical trial R2810-ONC-1788 and provide subsequent analyses of disease free survival (DFS) and overall survival (OS) to further characterize the clinical benefit of cemiplimab in patients with cutaneous squamous cell carcinoma at high risk of recurrence following treatment with surgery and radiation."~"Ongoing"~~4/30/2027 0:00:00~11/21/2025 0:00:00
385313~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Original"~1~"3594-1"~"PMR"~"Pediatric Research Equity Act"~3594.00~1~"PMR 3594-1: Conduct a pharmacokinetics (PK), safety and efficacy study in pediatric subjects 6 to <18 years of age with moderate to severe plaque psoriasis (with a duration of exposure to risankizumab of at least one year)."~"Fulfilled"~"Per FDA letter dated 06/12/2026, this PMR/PMC has been fulfilled."~3/31/2026 0:00:00~6/17/2026 0:00:00
385314~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Original"~1~"3594-2"~"PMR"~"505 (o)(3)"~3594.00~2~"PMR 3594-2: A prospective, registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to risankizumab during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including neonatal deaths, infections in the first 6 months of life, and effects on postnatal growth and development, will be assessed through at least the first year of life.
"~"Ongoing"~~6/30/2033 0:00:00~6/17/2026 0:00:00
385315~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Original"~1~"3594-3"~"PMR"~"505 (o)(3)"~3594.00~3~"PMR 3594-3: Conduct a retrospective cohort study using claims or electronic medical record data or a case control study to assess adverse pregnancy outcomes such as major congenital malformations, spontaneous abortions, stillbirths, small for gestational age, neonatal deaths, and infant infections in women exposed to risankizumab during pregnancy compared to an unexposed control population.
"~"Ongoing"~~10/31/2030 0:00:00~6/17/2026 0:00:00
385316~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Original"~1~"3594-4"~"PMR"~"505 (o)(3)"~3594.00~4~"PMR 3594-4: Conduct an observational study to assess the long-term safety of risankizumab compared to other therapies used in the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy in the course of actual clinical care. The study s primary outcome is long-term malignancy. Secondary outcomes include, but are not limited to, serious infections, tuberculosis, opportunistic infections, hypersensitivity reactions, autoimmune disease, neurologic or demyelinating disease, cardiovascular, gastrointestinal and hematologic adverse events. Describe and justify the choice of appropriate comparator population(s) and estimated background rate(s) relative to risankizumab-exposed patients; clearly define the primary comparator population for the primary objective. Design the study around a testable hypothesis to assess, with sufficient sample size and power, a clinically meaningful increase in malignancy risk above the comparator background rate(s), with a prespecified statistical analysis method. Specify concise case definitions and validation algorithms for both primary and secondary outcomes. For the risankizumab-exposed and comparator(s) cohorts, clearly define the study drug initiation period and any exclusion and inclusion criteria. Enroll patients over an initial 4-year period and follow for a minimum of 8 years from the time of enrollment."~"Ongoing"~~1/31/2034 0:00:00~6/17/2026 0:00:00
385317~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~14~"4206-1"~"PMR"~"Pediatric Research Equity Act"~4206.00~1~"PMR 4206-1: Provide pharmacokinetic (PK) and safety information to support the pediatric assessment of risankizumab for the treatment of juvenile psoriatic arthritis in children 5 to 17 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~3/31/2026 0:00:00~6/17/2026 0:00:00
385318~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~16~"4294-1"~"PMR"~"505 (o)(3)"~4294.00~1~"PMR 4294-1: Conduct an observational study to assess the incidence of severe acute liver injury in adults with moderately to severely active Crohn's disease who are exposed to Skyrizi (risankizumab-rzaa), relative to other therapies used to treat Crohn's disease. Compare rates (per person-time) or risks (proportion of patients with a minimum amount of follow-up). Describe and justify the choice of appropriate comparator population(s). Specify concise case definition for severe liver injury and validation of algorithm(s) to identify severe liver injury in the proposed data source. For the Skyrizi (risankizumab-rzaa)-exposed and comparator(s) cohorts, clearly define the study drug initiation period and any exclusion and inclusion criteria. Ensure that the data source allows for average follow-up for at least 1 year. Specify a minimum sample size and justify the precision of the estimate achievable with the proposed study."~"Delayed"~"The final protocol was submitted on 10/13/2023, which was after the original milestone, and acknowledged on 11/6/2023."~12/31/2033 0:00:00~6/17/2026 0:00:00
385319~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~16~"4294-2"~"PMR"~"505 (o)(3)"~4294.00~2~"PMR 4294-2: A prospective, registry-based, observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to risankizumab-containing products regardless of indication during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age births, preterm births, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, neonatal deaths, and infections, will be assessed through at least the first year of life."~"Ongoing"~~6/30/2034 0:00:00~6/17/2026 0:00:00
385320~"CDER"~"BLA"~761149.00~"Genentech, Inc."~"Enspryng (satralizumab-mwge)"~8/14/2020 0:00:00~"Original"~1~"3920-1"~"PMR"~"505 (o)(3)"~3920.00~1~"PMR 3920-1: A worldwide single-arm pregnancy safety study to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes in women exposed to Enspryng (satralizumab-mwge) during pregnancy in patients with neuromyelitis optica spectrum disorder (NMOSD). Provide a complete protocol that includes details regarding how you plan to encourage patients and providers to report pregnancy exposures, measures to ensure complete data capture regarding pregnancy outcomes and any adverse effects in offspring, and plans for comprehensive data analysis."~"Ongoing"~~12/31/2033 0:00:00~10/8/2025 0:00:00
385321~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Original"~1~"4268-1"~"PMR"~"Pediatric Research Equity Act"~4268.00~1~"PMR 4268-1: Conduct a multicenter, open-label trial to assess the pharmacokinetics and safety of bimekizumab in adolescents 12 to <18 years of age with moderate to severe plaque psoriasis."~"Submitted"~"The final report was submitted to FDA on 12/03/2025."~12/31/2025 0:00:00~12/12/2025 0:00:00
385322~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Original"~1~"4268-2"~"PMR"~"Pediatric Research Equity Act"~4268.00~2~"PMR 4268-2: Conduct a multicenter, randomized, parallel-group, blinded, activecontrolled trial to assess the safety and pharmacokinetics of bimekizumab in pediatric subjects 6 to <18 years old with moderate to severe plaque psoriasis."~"Ongoing"~"142 patients out of the planned 168 patients have been enrolled into the trial."~8/31/2031 0:00:00~12/12/2025 0:00:00
385323~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Original"~1~"4268-3"~"PMR"~"505 (o)(3)"~4268.00~3~"PMR 4268-3: Conduct a prospective, registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to bimekizumab during pregnancy to an unexposed control population. The registry should be designed to detect and record major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age, preterm birth, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, neonatal deaths, and infections, will be assessed through at least the first year of life."~"Ongoing"~~6/30/2035 0:00:00~12/12/2025 0:00:00
385324~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Original"~1~"4268-4"~"PMR"~"505 (o)(3)"~4268.00~4~"PMR 4268-4: Conduct an additional pregnancy study that uses a different design from the pregnancy registry (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, small for gestational age, and preterm birth in women exposed to bimekizumab during pregnancy compared to an unexposed control population."~"Ongoing"~~12/31/2035 0:00:00~12/12/2025 0:00:00
385325~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Original"~1~"4268-5"~"PMR"~"505 (o)(3)"~4268.00~5~"PMR 4268-5: Perform a lactation study (milk only) in lactating women who have received therapeutic doses of bimekizumab to assess concentrations of bimekizumab in breastmilk using a validated assay and to assess the effects on the breastfed infant. A mother-infant pair study may be required in the future depending on the results of this milk-only study."~"Ongoing"~~11/30/2028 0:00:00~12/12/2025 0:00:00
385326~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Original"~1~"4268-6"~"PMR"~"505 (o)(3)"~4268.00~6~"PMR 4268-6: Conduct a prospective observational study to assess the long-term safety of bimekizumab treatment in US adult patients with moderate to severe plaque psoriasis. Fully ascertain and centrally verify malignancy (including lymphoma), opportunistic infections, reactivation of Hepatitis B, tuberculosis, and serious infections. Other outcomes include hypersensitivity, gastrointestinal events (including inflammatory bowel disease, elevated liver enzymes/drug-induced liver injury), Major Adverse Cardiovascular Events (myocardial infarction, stroke, cardiovascular death, and sudden death) and hematologic events. For each adverse event outcome separately, compare incidence in bimekizumab-treated patients against reference rates internally derived from analyses conducted in patients treated with other chronic systemic treatments for moderate-to-severe plaque psoriasis. Regardless of treatment discontinuation or switch to a different treatment for plaque psoriasis, continue following patients for malignancy outcomes and possibly other adverse events with delayed onset. Enroll a sufficient number of patients to describe the frequency of the adverse events in representative U.S. patients who start treatment with bimekizumab for plaque psoriasis in the setting of routine clinical practice. Implement a plan that uses rigorous, transparent, and verifiable methods to ascertain and characterize safety events that occur during and after treatment with bimekizumab. Enroll patients over a 4-year period and plan to follow for a minimum of 8 years from time of enrollment."~"Delayed"~"Original Final Report Due Date: 03/31/2038; Deferral Extension granted per FDA letter dated 09/11/2025. The final protocol submission was also revised."~3/31/2038 0:00:00~12/12/2025 0:00:00
385327~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Supplement"~5~"4703-1"~"PMR"~"Pediatric Research Equity Act"~4703.00~1~"PMR 4703-1: Provide pharmacokinetic and safety information to support the pediatric assessment of bimekizumab for the treatment of pediatric psoriatic arthritis in children 6 to less than 18 years of age."~"Pending"~"The study has not begun but does not meet the criterion for delayed."~12/31/2030 0:00:00~12/12/2025 0:00:00
385328~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Supplement"~6~"4703-1"~"PMR"~"Pediatric Research Equity Act"~4703.00~1~"PMR 4703-1: Provide pharmacokinetic and safety information to support the pediatric assessment of bimekizumab for the treatment of pediatric psoriatic arthritis in children 6 to less than 18 years of age."~"Pending"~"The study has not begun but does not meet the criterion for delayed."~12/31/2030 0:00:00~12/12/2025 0:00:00
385329~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Supplement"~7~"4703-1"~"PMR"~"Pediatric Research Equity Act"~4703.00~1~"PMR 4703-1: Provide pharmacokinetic and safety information to support the pediatric assessment of bimekizumab for the treatment of pediatric psoriatic arthritis in children 6 to less than 18 years of age."~"Pending"~"The study has not begun but does not meet the criterion for delayed."~12/31/2030 0:00:00~12/12/2025 0:00:00
385330~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Supplement"~10~"4704-1"~"PMR"~"Pediatric Research Equity Act"~4704.00~1~"PMR 4704-1: Conduct a multicenter, open-label clinical trial to evaluate the pharmacokinetic and safety of bimekizumab in pediatric patients including those 12 years of age and older with moderate to severe hidradenitis suppurativa."~"Ongoing"~"20 of the targeted 40 patients have been enrolled into the study."~11/30/2031 0:00:00~12/12/2025 0:00:00
385331~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Supplement"~10~"4704-2"~"PMR"~"505 (o)(3)"~4704.00~2~"PMR 4704-2: Conduct a prospective pregnancy exposure registry/registries in pregnant patients with hidradenitis suppurativa. You can use the registry required under PMR 4268-3 issued in the BLA 761151 Approval letter dated October 17, 2023."~"Ongoing"~~6/30/2035 0:00:00~12/12/2025 0:00:00
385332~"CDER"~"BLA"~761345.00~"Pfizer Inc."~"Elrexfio (elranatamab-bcmm)"~8/14/2023 0:00:00~"Original"~1~"4476-2"~"PMC"~"506B PMC"~4476.00~2~"PMC 4476-2: Conduct an integrated analysis of data from clinical trials to further characterize the efficacy, pharmacokinetics, pharmacodynamics, and safety of elranatamab among U.S. racial and ethnic minority patients with multiple myeloma. The population should be representative of the U.S. population of patients with multiple myeloma, including racial and ethnic diversity, and allow for interpretation of the results in these populations."~"Ongoing"~~12/31/2026 0:00:00~10/9/2025 0:00:00
385333~"CDER"~"BLA"~761347.00~"Genentech, Inc."~"Tecentriq Hybreza (atezolizumab and hyaluronidase-tqjs)"~9/12/2024 0:00:00~"Original"~1~"4637-1"~"PMR"~"Pediatric Research Equity Act"~4637.00~1~"PMR 4637-1: Conduct a molecularly targeted pediatric cancer investigation using an age-appropriate formulation of atezolizumab and hyaluronidase-tqjs in pediatric patients 12 years of age and older."~"Fulfilled"~"Per FDA letter dated 11/24/2025, this PMR/PMC has been fulfilled."~6/30/2025 0:00:00~7/10/2026 0:00:00
385334~"CDER"~"BLA"~761349.00~"Novartis Pharmaceuticals Corporation"~"Cosentyx (secukinumab)"~10/6/2023 0:00:00~"Original"~1~"4523-1"~"PMR"~"Pediatric Research Equity Act"~4523.00~1~"PMR 4523-1: Conduct an open-label study to evaluate the pharmacokinetics and safety of IV secukinumab plus background standard therapy in pediatric subjects ages 2 years to 17 years of age with psoriatic arthritis"~"Ongoing"~"2 out of 20 planned patients have been enrolled."~4/30/2030 0:00:00~2/19/2026 0:00:00
385335~"CDER"~"BLA"~761352.00~"Partner Therapeutics Inc."~"Bizengri (zenocutuzumab-zbco) injection"~12/4/2024 0:00:00~"Original"~1~"4727-1"~"PMR"~"Accelerated Approval"~4727.00~1~"PMR 4727-1: Complete a clinical trial intended to verify and describe the clinical benefit of zenocutuzumab 750 mg intravenously every two weeks in at least 100 evaluable adult patients with advanced unresectable or metastatic nonsmall cell lung cancer harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy. To characterize response rate and duration, patients will be followed for at least 12 months from the onset of response."~"Ongoing"~"The trial has completed enrollment."~8/31/2026 0:00:00~1/29/2026 0:00:00
385336~"CDER"~"BLA"~761352.00~"Partner Therapeutics Inc."~"Bizengri (zenocutuzumab-zbco) injection"~12/4/2024 0:00:00~"Original"~1~"4727-2"~"PMR"~"Accelerated Approval"~4727.00~2~"PMR 4727-2: Complete a clinical trial intended to verify and describe the clinical benefit of zenocutuzumab 750 mg intravenously every two weeks in at least 50 evaluable adult patients with advanced unresectable or metastatic pancreatic adenocarcinoma harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy (or who are not eligible for standard of care therapy). To characterize response rate and duration, patients will be followed for at least 12 months from the onset of response."~"Ongoing"~"The trial has completed enrollment."~8/31/2026 0:00:00~1/29/2026 0:00:00
385337~"CDER"~"BLA"~761352.00~"Partner Therapeutics Inc."~"Bizengri (zenocutuzumab-zbco) injection"~12/4/2024 0:00:00~"Original"~1~"4727-3"~"PMR"~"505 (o)(3)"~4727.00~3~"PMR 4727-3: Conduct a comprehensive integrated safety analysis in a sufficient number of adult patients from clinical trials to adequately characterize the known serious risks of infusion-related reactions/hypersensitivity/anaphylaxis, interstitial lung disease/pneumonitis, and left ventricular dysfunction following exposure to zenocutuzumab. The integrated safety analysis should include all adverse events, major safety events, dose-reductions, dose interruptions, and withdrawals, when all patients have completed at least two years of treatment with zenocutuzumab or withdrew earlier."~"Ongoing"~~6/30/2027 0:00:00~1/29/2026 0:00:00
385338~"CDER"~"BLA"~761352.00~"Partner Therapeutics Inc."~"Bizengri (zenocutuzumab-zbco) injection"~12/4/2024 0:00:00~"Original"~1~"4727-4"~"PMC"~"506B PMC"~4727.00~4~"PMC 4727-4: Develop and validate a neutralizing antibody (NAb) assay and submit a full validation report of the developed NAb assay. The assay format should be adequately justified to be suitable for the detection of NAbs. This NAb assay will be used to test available confirmed anti-drug antibody positive samples from banked and ongoing clinical studies. Include the updated NAb results analyzed using the validated NAb assay to address the effects of neutralizing antibody on the pharmacokinetics, pharmacodynamics, safety, and effectiveness of zenocutuzumab."~"Submitted"~~8/31/2025 0:00:00~1/29/2026 0:00:00
385339~"CDER"~"BLA"~761352.00~"Partner Therapeutics Inc."~"Bizengri (zenocutuzumab-zbco) injection"~12/4/2024 0:00:00~"Original"~1~"4727-5"~"PMC"~"506B PMC"~4727.00~5~"PMC 4727-5: Conduct an appropriate analytical and clinical validation study to establish and support the availability of an in vitro diagnostic device using clinical trial data that demonstrates the device is essential to the safe and effective use of zenocutuzumab for the treatment of adult patients with advanced unresectable or metastatic non-small cell lung cancer harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy."~"Ongoing"~~3/31/2028 0:00:00~1/29/2026 0:00:00
385340~"CDER"~"BLA"~761352.00~"Partner Therapeutics Inc."~"Bizengri (zenocutuzumab-zbco) injection"~12/4/2024 0:00:00~"Original"~1~"4727-6"~"PMC"~"506B PMC"~4727.00~6~"PMC 4727-6: Conduct an appropriate analytical and clinical validation study to establish and support the availability of an in vitro diagnostic device using clinical trial data that demonstrates the device is essential to the safe and effective use of zenocutuzumab for the treatment of adult patients with advanced unresectable or metastatic pancreatic adenocarcinoma harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy."~"Ongoing"~~3/31/2028 0:00:00~1/29/2026 0:00:00
385341~"CDER"~"BLA"~761352.00~"Partner Therapeutics Inc."~"Bizengri (zenocutuzumab-zbco) injection"~12/4/2024 0:00:00~"Supplement"~10~"4995-1"~"PMC"~"506B PMC"~4995.00~1~"PMC 4995-1: Conduct an appropriate analytical and clinical validation study, using clinical trial data, to establish an in vitro diagnostic device capable of accurately and reliably identifying adult patients with advanced unresectable or metastatic cholangiocarcinoma harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy for whom the use of zenocutuzumab therapy is indicated."~"Pending"~~3/31/2028 0:00:00~1/29/2026 0:00:00
385342~"CDER"~"BLA"~761354.00~"Biogen MA Inc. "~"Tofidence (tocilizumab-bavi)"~9/29/2023 0:00:00~"Supplement"~2~"4664-1"~"PMR"~"Pediatric Research Equity Act"~4664.00~1~"PMR 4664-1: Assessment of Tofidence (tocilizumab-bavi) for the treatment of COVID-19 in pediatric patients 1 year to <18 years of age."~"Fulfilled"~"Per FDA letter dated 05/22/2026, this PMR/PMC has been fulfilled."~6/30/2026 0:00:00~11/26/2025 0:00:00
385343~"CDER"~"BLA"~761358.00~"CELLTRION, Inc."~"Zymfentra (infliximab)"~10/20/2023 0:00:00~"Original"~1~"4492-1"~"PMR"~"Pediatric Research Equity Act"~4492.00~1~"PMR 4492-1: Conduct a one-year, randomized trial to evaluate the safety, efficacy, and pharmacokinetics of Zymfentra (infliximab-dyyb) as maintenance treatment in pediatric patients 6 to < 18 years of age with moderately to severely active Crohn s disease."~"Ongoing"~"The study has been initiated."~12/31/2027 0:00:00~12/18/2025 0:00:00
385344~"CDER"~"BLA"~761358.00~"CELLTRION, Inc."~"Zymfentra (infliximab)"~10/20/2023 0:00:00~"Original"~1~"4492-2"~"PMR"~"Pediatric Research Equity Act"~4492.00~2~"PMR 4492-2: Conduct a one-year, randomized trial to evaluate the safety, efficacy, and pharmacokinetics of Zymfentra (infliximab-dyyb) as maintenance treatment in pediatric patients 6 to < 18 years of age with moderately to severely active ulcerative colitis."~"Ongoing"~"The study has been initiated."~12/31/2027 0:00:00~12/18/2025 0:00:00
385345~"CDER"~"NDA"~219876.00~"CHIMERIX INC"~"Modeyso (dordaviprone) Capsules"~8/6/2025 0:00:00~"Original"~1~"4861-2"~"PMR"~"Accelerated Approval"~4861.00~2~"PMR 4861-2: Conduct a clinical trial in pediatric patients (<17 years of age) with H3 K27M-mutant diffuse midline glioma, inclusive of a sufficient number of patients with Diffuse Intrinsic Pontine Glioma (DIPG), intended to verify and describe the clinical benefit of dordaviprone in the pediatric population. Endpoints evaluated should include overall response rate (ORR), duration of response (DOR), and overall survival (OS)."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2031 0:00:00~
385346~"CDER"~"NDA"~219876.00~"CHIMERIX INC"~"Modeyso (dordaviprone) Capsules"~8/6/2025 0:00:00~"Original"~1~"4861-3"~"PMC"~"506B PMC"~4861.00~3~"PMC 4861-3: Conduct an appropriate analytical and clinical validation study to support the development of an in vitro diagnostic device using clinical trial data that demonstrates that the device is essential to the effective and safe use of dordaviprone for the treatment of adult and pediatric patients with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy."~"Pending"~~7/31/2030 0:00:00~
385347~"CDER"~"NDA"~219884.00~"ACADIA PHARMACEUTICALS INC"~"Daybue (trofinetide) powder for oral solution, and Daybue Stix (trofinetide) powder for oral solution"~12/11/2025 0:00:00~"Original"~1~"4944-1"~"PMR"~"505 (o)(3)"~4944.00~1~"PMR 4944-1: Conduct a carcinogenicity study of trofinetide in mouse."~"Fulfilled"~~8/31/2026 0:00:00~
385348~"CDER"~"NDA"~219884.00~"ACADIA PHARMACEUTICALS INC"~"Daybue (trofinetide) powder for oral solution, and Daybue Stix (trofinetide) powder for oral solution"~12/11/2025 0:00:00~"Original"~1~"4944-2"~"PMR"~"505 (o)(3)"~4944.00~2~"PMR 4944-2: Conduct a 2-year carcinogenicity study of trofinetide in rat."~"Pending"~~8/31/2026 0:00:00~
385349~"CDER"~"NDA"~219947.00~"ARROWHEAD PHARMACEUTICALS INC"~"Redemplo (plozasiran) Injection"~11/18/2025 0:00:00~"Original"~1~"4909-1"~"PMR"~"505 (o)(3)"~4909.00~1~"PMR 4909-1: Complete the ongoing 2-year rat carcinogenicity study to assess the carcinogenic potential from long-term subcutaneous exposure to ARO-APOC3 (plozasiran)."~"Submitted"~~1/31/2026 0:00:00~
385350~"CDER"~"NDA"~219947.00~"ARROWHEAD PHARMACEUTICALS INC"~"Redemplo (plozasiran) Injection"~11/18/2025 0:00:00~"Original"~1~"4909-2"~"PMR"~"505 (o)(3)"~4909.00~2~"PMR 4909-2: Complete the ongoing randomized, double-blind, placebo-controlled trials evaluating plozasiran in patients with severe hypertriglyceridemia (AROAPOC3-3003 [SHASTA-3], including the magnetic resonance imaging proton density fat fraction substudy, and AROAPOC3-3004 [SHASTA-4]) and evaluate the potential for drug-induced liver injury and drug-induced dysglycemia (e.g., new-onset diabetes mellitus, worsening glycemic control in those with existing diabetes mellitus)."~"Ongoing"~~6/30/2027 0:00:00~
385351~"CDER"~"NDA"~219972.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Hyrnuo (sevabertinib) tablets"~11/19/2025 0:00:00~"Original"~1~"4933-1"~"PMR"~"Accelerated Approval"~4933.00~1~"PMR 4933-1: Complete the ongoing multicenter, randomized clinical trial, Study SOHO-02, intended to verify and describe the clinical benefit of sevabertinib in adult patients with locally advanced or metastatic non-squamous nonsmall cell lung cancer whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations and who have not received prior therapy for advanced disease."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~10/31/2029 0:00:00~
385352~"CDER"~"NDA"~219972.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Hyrnuo (sevabertinib) tablets"~11/19/2025 0:00:00~"Original"~1~"4933-2"~"PMR"~"505 (o)(3)"~4933.00~2~"PMR 4933-2: Conduct a hepatic impairment clinical trial to evaluate the serious potential risk of increased drug exposure and determine a safe and appropriate dosage of sevabertinib in patients with moderate hepatic impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~7/31/2026 0:00:00~
385353~"CDER"~"NDA"~219972.00~"BAYER HEALTHCARE PHARMACEUTICALS INC"~"Hyrnuo (sevabertinib) tablets"~11/19/2025 0:00:00~"Original"~1~"4933-3"~"PMR"~"505 (o)(3)"~4933.00~3~"PMR 4933-3: Conduct a clinical drug interaction study to evaluate the effect of sevabertinib on the pharmacokinetics of sensitive substrates of MATE1 and MATE2-K in healthy subjects to assess the magnitude of exposure change of these substrates and serious potential risk of increased drug toxicity and inform an appropriate drug interaction management strategy for coadministration of sevabertinib with these transporter substrates. This study should be designed and conducted in accordance with  M12 Drug Interaction Studies ."~"Pending"~~9/30/2027 0:00:00~
385354~"CDER"~"NDA"~220018.00~"GILEAD SCIENCES INC"~"Yeztugo (lenacapavir) Injection"~6/18/2025 0:00:00~"Original"~1~"4836-1"~"PMC"~"506B PMC"~4836.00~1~"PMC 4836-1: Conduct a study to determine the phenotype of lenacapavir against the following HIV-1 capsids: 1) subtype A1 containing the P111T substitution; 2) subtype C containing the T54A, Q67R, I73V, and A105V substitutions, 3) subtype C containing the T54S and T107A substitutions, and 4) subtype D containing the L56M and L69V substitutions, assessed individually and in the context in which these were identified. In your study, use a wildtype control and known resistance-associated substitutions, such as a subtype B capsid with the K70N substitution (24-fold change in EC50 value) representing the range of reductions in susceptibility for comparison."~"Pending"~~6/30/2026 0:00:00~
385355~"CDER"~"NDA"~220020.00~"GILEAD SCIENCES INC"~"Yeztugo (lenacapavir) Tablet"~6/18/2025 0:00:00~"Original"~1~"4837-1"~"PMC"~"506B PMC"~4837.00~1~"PMC 4837-1: Conduct a study to determine the phenotype of lenacapavir against the following HIV-1 capsids: 1) subtype A1 containing the P111T substitution; 2) subtype C containing the T54A, Q67R, I73V, and A105V substitutions, 3) subtype C containing the T54S and T107A substitutions, and 4) subtype D containing the L56M and L69V substitutions, assessed individually and in the context in which these were identified. In your study, use a wildtype control and known resistance-associated substitutions, such as a subtype B capsid with the K70N substitution (24-fold change in EC50 value) representing the range of reductions in susceptibility for comparison."~"Pending"~~6/30/2026 0:00:00~
385356~"CDER"~"NDA"~220149.00~"JANSSEN BIOTECH INC"~"Icotyde (icotrokinra) tablet"~3/17/2026 0:00:00~"Original"~1~"4964-1"~"PMR"~"Pediatric Research Equity Act"~4964.00~1~"PMR 4964-1: Conduct a trial to evaluate the safety and pharmacokinetics of icotrokinra in pediatric subjects 4 years to less than 12 years of age with moderate to severe psoriasis who are candidates for systemic therapy or phototherapy. Evaluate a sufficient number of subjects exposed to icotrokinra at the highest proposed dosage for a minimum of 26 weeks to evaluate for safety and pharmacokinetics of icotrokinra in pediatric subjects 4 years to less than 12 years of age with moderate to severe psoriasis who are candidates for systemic therapy or phototherapy."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~9/30/2030 0:00:00~
385357~"CDER"~"NDA"~220149.00~"JANSSEN BIOTECH INC"~"Icotyde (icotrokinra) tablet"~3/17/2026 0:00:00~"Original"~1~"4964-2"~"PMR"~"505 (o)(3)"~4964.00~2~"PMR 4964-2: Conduct a worldwide, as applicable, descriptive study that collects prospective and retrospective data in women exposed to Icotyde (icotrokinra) during pregnancy to assess risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant. Assess infant outcomes up to the first year of life. The minimum number of patients will be specified in the protocol."~"Pending"~~9/30/2037 0:00:00~
385358~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~16~"4294-3"~"PMR"~"505 (o)(3)"~4294.00~3~"PMR 4294-3: Conduct an additional pregnancy study that uses a different design from the prospective pregnancy registry established to fulfil postmarketing requirement study 2 (for example a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm births in women exposed to risankizumab-containing products regardless of indication during pregnancy compared to an unexposed control population.
"~"Delayed"~"The final protocol was submitted after the original milestone and acknowledged in a letter dated 7/11/2024."~6/30/2033 0:00:00~6/17/2026 0:00:00
385359~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~16~"4294-4"~"PMR"~"505 (o)(3)"~4294.00~4~"PMR 4294-4: Perform a lactation trial (milk only) in lactating women who have received Skyrizi (risankizumab-rzaa) to assess concentrations of Skyrizi (risankizumab-rzaa) in breast milk using a validated assay and to assess the effects on the breastfed infant.
"~"Ongoing"~~6/30/2026 0:00:00~6/17/2026 0:00:00
385360~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~16~"4294-5"~"PMC"~"506B PMC"~4294.00~5~"PMC 4294-5: Conduct a one-year, randomized trial to evaluate the safety, efficacy, and pharmacokinetics of Skyrizi (risankizumab-rzaa) in pediatric patients 2 to 17 years of age with moderately to severely active Crohn s disease.
"~"Ongoing"~~12/31/2029 0:00:00~6/17/2026 0:00:00
385361~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~16~"4294-6"~"PMC"~"506B PMC"~4294.00~6~"PMC 4294-6: Conduct a long-term extension study to evaluate the long-term safety of Skyrizi (risankizumab-rzaa) in pediatric patients 2 to 17 years of age with moderately to severely active Crohn s disease who participated in postmarketing commitment study 4294-5. This study can be conducted as part of postmarketing commitment study 4294-5."~"Ongoing"~~9/30/2033 0:00:00~6/17/2026 0:00:00
385362~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~29~"4656-1"~"PMR"~"Pediatric Research Equity Act"~4656.00~1~"PMR 4656-1: Conduct a one-year, randomized trial to evaluate the safety, efficacy, and pharmacokinetics of Skyrizi (risankizumab-rzaa) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2031 0:00:00~6/17/2026 0:00:00
385363~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~29~"4656-2"~"PMR"~"Pediatric Research Equity Act"~4656.00~2~"PMR 4656-2: Conduct a long-term extension study to evaluate the long-term safety of Skyrizi (risankizumab-rzaa) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis who participated in postmarketing requirement study #1. This study can be conducted as part of postmarketing requirement study #1 or as a basket trial with study PMC 4294-6."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2035 0:00:00~6/17/2026 0:00:00
385364~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~29~"4656-3"~"PMR"~"505 (o)(3)"~4656.00~3~"PMR 4656-3: Conduct an observational study to assess the incidence of severe acute liver injury in adults with moderately to severely active ulcerative colitis who are exposed to Skyrizi (risankizumab-rzaa), relative to other therapies used to treat ulcerative colitis. Compare rates (per person-time) or risks (proportion of patients with a minimum amount of follow-up). Describe and justify the choice of appropriate comparator population(s). Specify concise case definition for severe liver injury and validation of algorithm(s) to identify severe liver injury in the proposed data source. For the Skyrizi (risankizumab-rzaa)-exposed and comparator(s) cohorts, clearly define the study drug initiation period and any exclusion and inclusion criteria. Ensure that the data source allows for average follow-up for at least 1 year. Specify a minimum sample size and justify the precision of the estimate achievable with the proposed study. The ongoing observational study in patients with Crohn s disease (PMR 4294-1) with the same objectives may be amended to also enroll patients with ulcerative colitis."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 8/20/2025."~12/31/2033 0:00:00~6/17/2026 0:00:00
385365~"CDER"~"BLA"~761105.00~"AbbVie Inc."~"Skyrizi (risankizumab-rzaa)"~4/23/2019 0:00:00~"Supplement"~29~"4656-4"~"PMC"~"506B PMC"~4656.00~4~"PMC 4656-4: Submit postmarketing device reports to evaluate the on-body injector device used to deliver Skyrizi (risankizumab-rzaa) in patients with moderately to severely active Crohn's disease or ulcerative colitis. For reported device failure or use error events, collect information to allow root-cause analysis and assessment of associated adverse events. Specific information collected should include essential elements to allow for root-cause analysis such as patient narratives of the events and evaluation of returned devices, dosing information including the start of maintenance therapy, and any adverse events associated with the device failure or use error event (i.e., injection site pain/erythema) including disease activity as reported. Among confirmed device failure or use error events, identify a cohort of patients to prospectively follow for 6 months to assess disease activity (i.e., remission, flare/worsening disease symptoms, escalation of care, hospitalization, procedures)."~"Ongoing"~~2/28/2027 0:00:00~6/17/2026 0:00:00
385366~"CDER"~"BLA"~761107.00~"Swedish Orphan Biovitrum AB (publ)"~"Gamifant (emapalumab-lzsg)"~11/20/2018 0:00:00~"Supplement"~18~"4863-1"~"PMR"~"505 (o)(3)"~4863.00~1~"PMR 4863-1: Establish a registry to characterize the long-term safety of emapalumab-lzsg in at least 50 adult and pediatric patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still s disease, including systemic juvenile idiopathic arthritis (sJIA) with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS, with at least 1 year of follow-up. Provide clinical narratives and tabular summaries of all pertinent safety data in the yearly interim reports. The final study report should include a summary of the major safety findings for all patients including patient level data on emapalumab-lzsg dosing, all serious infections in particular, mycobacterium and cytomegalovirus infections and reactivations, laboratory results (i.e., CBC with differential and liver function tests), and concomitant medications."~"Pending"~~3/31/2031 0:00:00~1/16/2026 0:00:00
385367~"CDER"~"BLA"~761108.00~"Alexion Pharmaceuticals, Inc."~"Ultomiris (ravulizumab-cwvz)"~12/21/2018 0:00:00~"Supplement"~1~"3698-1"~"PMR"~"505 (o)(3)"~3698.00~1~"PMR 3698-1: Provide safety, efficacy, PK, and PD data in pediatric patients with aHUS (Study ALXN 1210-aHUS-312) and a weight of greater than or equal to 5 kg to less than 10 kg treated with the recommended dosing regimen of ravulizumab (600 mg loading dose followed two weeks later by 300 mg maintenance doses every 4 weeks)."~"Fulfilled"~~6/30/2025 0:00:00~2/20/2026 0:00:00
385368~"CDER"~"BLA"~761108.00~"Alexion Pharmaceuticals, Inc."~"Ultomiris (ravulizumab-cwvz)"~12/21/2018 0:00:00~"Supplement"~1~"3698-2"~"PMC"~"506B PMC"~3698.00~2~"PMC 3698-2: Submit the final clinical study report and datasets for the single arm trial of ULTOMIRIS in pediatric patients with aHUS (ALXN1210- aHUS-312) to include the extension period of up to 4.5 years of follow-up on safety and efficacy."~"Fulfilled"~~6/30/2025 0:00:00~2/20/2026 0:00:00
385369~"CDER"~"BLA"~761151.00~"UCB, Inc."~"Bimzelx (bimekizumab)"~10/17/2023 0:00:00~"Supplement"~10~"4704-3"~"PMR"~"505 (o)(3)"~4704.00~3~"PMR 4704-3: Conduct a retrospective pregnancy cohort study in patients with hidradenitis suppurativa using claims or electronic health record data with medical chart validation. You can use the registry required under PMR 4268-4 issued in the BLA 761151 Approval letter dated October 17, 2023."~"Ongoing"~~12/31/2035 0:00:00~12/12/2025 0:00:00
385370~"CDER"~"BLA"~761152.00~"Omeros Corporation"~"Yartemla (narsoplimab-wuug) injection"~12/23/2025 0:00:00~"Original"~1~"4938-1"~"PMR"~"505 (o)(3)"~4938.00~1~"PMR 4938-1: Establish a registry to characterize the long-term safety of narsoplimab-wuug in at least 50 adult and pediatric patients with transplant-associated thrombotic microangiopathy (TA-TMA), with at least 1 year of follow-up. The final study report should include a summary of the major safety findings for all patients including patient level data on narsoplimab-wuug dosing, serious infections, serious treatment emergent adverse events and laboratory data (i.e., CBC with differential and renal function tests), autoimmune disorders, concomitant medications, vital status, and cause of death for any fatalities."~"Pending"~~12/31/2034 0:00:00~
385371~"CDER"~"BLA"~761152.00~"Omeros Corporation"~"Yartemla (narsoplimab-wuug) injection"~12/23/2025 0:00:00~"Original"~1~"4938-2"~"PMC"~"506B PMC"~4938.00~2~"PMC 4938-2: Provide safety, pharmacokinetic (PK) and pharmacodynamic (PD) data in pediatric patients <17 years of age treated with the recommended dosing regimen of narsoplimab for transplant-associated thrombotic microangiopathy (TA-TMA)."~"Pending"~~12/31/2033 0:00:00~
385372~"CDER"~"BLA"~761154.00~"Biocon Biologics Inc."~"Hulio (adalimumab-fkjp)"~7/6/2020 0:00:00~"Original"~1~"3894-4"~"PMR"~"Pediatric Research Equity Act"~3894.00~4~"PMR 3894-4: Develop a presentation that can be used to accurately administer Hulio (adalimumab-fkjp) to pediatric patients who weigh less than 15 kg."~"Ongoing"~"Original Final Report Due Date: 12/31/2023. Per FDA letter dated 11/16/2023, final report due date extended to 06/30/2025. Second Deferral Extension Granted per FDA letter dated 05/15/2025 because of delays due to issues with the study drug."~6/30/2026 0:00:00~9/5/2025 0:00:00
385373~"CDER"~"BLA"~761156.00~"Novo Nordisk Inc."~"Sogroya (somapacitan-beco)"~8/28/2020 0:00:00~"Original"~1~"3890-1"~"PMR"~"Pediatric Research Equity Act"~3890.00~1~"PMR 3890-1: Complete ongoing study NN8640-4172, a 1-year phase 2, randomized, open-label, active-control, dose-finding trial, to investigate efficacy and safety of once-weekly somapacitan versus daily Norditropin (somatropin) in pre-pubertal children with Growth Hormone Deficiency (GHD) (boys = 2.5 and = 10 years of age, and girls = 2.5 and = 9 years of age) followed by a 2-year single-arm period to evaluate safety in this cohort, and a 4 year single-arm period that also enrolls cohorts of younger children with GHD (boys and girls < 2.5 years of age) and older children with GHD (boys > 10 and = 17 years of age, and girls > 9 and = 17 years of age) to evaluate safety in all three cohorts."~"Submitted"~"The final report was submitted to FDA on 02/26/2026."~2/28/2026 0:00:00~5/28/2026 0:00:00
385374~"CDER"~"BLA"~761156.00~"Novo Nordisk Inc."~"Sogroya (somapacitan-beco)"~8/28/2020 0:00:00~"Original"~1~"3890-2"~"PMR"~"Pediatric Research Equity Act"~3890.00~2~"PMR 3890-2: Complete ongoing study NN8640-4263, a 1-year phase 3 randomized, parallel group, open-label trial, to evaluate the efficacy and safety of once weekly somapacitan versus daily Norditropin (somatropin) in pre-pubertal pediatric patients (boys = 2.5 and = 10 years of age, and girls = 2.5 and = 9 years of age) with GHD, followed by a 3-year single-arm extension period to evaluate long-term safety."~"Submitted"~"The final report was submitted to FDA on 02/26/2026."~2/28/2026 0:00:00~5/28/2026 0:00:00
385375~"CDER"~"BLA"~761161.00~"Chiesi Farmaceutici S.p.A."~"Elfabrio (pegunigalsidase alfa-iwxj)"~5/9/2023 0:00:00~"Original"~1~"3972-1"~"PMR"~"Pediatric Research Equity Act"~3972.00~1~"PMR 3972-1: Clinical trial to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamic effects of pegunigalsidase alfa-iwxj in pediatric patients aged 2 to <18 years with confirmed Fabry disease. The trial will evaluate patients over at least 1 year from the time of enrollment and will include assessments of immunogenicity and correlative analyses between antibody formation (and titers if appropriate) and safety, efficacy, pharmacokinetics, and pharmacodynamics in treated patients."~"Ongoing"~"No patients have enrolled as of May 9, 2025."~1/31/2027 0:00:00~7/9/2026 0:00:00
385376~"CDER"~"BLA"~761161.00~"Chiesi Farmaceutici S.p.A."~"Elfabrio (pegunigalsidase alfa-iwxj)"~5/9/2023 0:00:00~"Original"~1~"3972-2"~"PMR"~"505 (o)(3)"~3972.00~2~"PMR 3972-2: Conduct a worldwide descriptive study that collects prospective and retrospective data in women and their offspring exposed to ELFABRIO (pegunigalsidase alfa-iwxj) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 12-13-2024. The applicant requested revised milestones because the Agency s agreement on the final protocol was necessary before starting the study. Revised milestones were acknowledged in a letter dated 09/06/2025."~12/31/2035 0:00:00~7/9/2026 0:00:00
385377~"CDER"~"BLA"~761161.00~"Chiesi Farmaceutici S.p.A."~"Elfabrio (pegunigalsidase alfa-iwxj)"~5/9/2023 0:00:00~"Original"~1~"3972-4"~"PMR"~"505 (o)(3)"~3972.00~4~"PMR 3972-4: Develop and validate an anti-PEG IgE antibody assay."~"Submitted"~~5/31/2024 0:00:00~7/9/2026 0:00:00
385378~"CDER"~"BLA"~761161.00~"Chiesi Farmaceutici S.p.A."~"Elfabrio (pegunigalsidase alfa-iwxj)"~5/9/2023 0:00:00~"Original"~1~"3972-7"~"PMR"~"505 (o)(3)"~3972.00~7~"PMR 3972-7: Evaluate neutralizing antibodies that inhibit the cellular uptake of pegunigalsidase alfa-iwxj in clinical samples from studies PB-102-F01/02, PB-102-F03, and PB-102-F20 using the assay developed and validated under PMR 3972-3. Assess the impact of cellular uptake neutralizing antibodies on the pharmacokinetics, pharmacodynamics, efficacy, and safety of pegunigalsidase alfa-iwxj."~"Submitted"~~9/30/2025 0:00:00~7/9/2026 0:00:00
385379~"CDER"~"BLA"~761161.00~"Chiesi Farmaceutici S.p.A."~"Elfabrio (pegunigalsidase alfa-iwxj)"~5/9/2023 0:00:00~"Original"~1~"3972-8"~"PMR"~"505 (o)(3)"~3972.00~8~"PMR 3972-8: A pre- and postnatal development study in rats treated with pegunigalsidase alfa-iwxj."~"Submitted"~~12/31/2025 0:00:00~7/9/2026 0:00:00
385380~"CDER"~"BLA"~761161.00~"Chiesi Farmaceutici S.p.A."~"Elfabrio (pegunigalsidase alfa-iwxj)"~5/9/2023 0:00:00~"Original"~1~"3972-9"~"PMC"~"506B PMC"~3972.00~9~"PMC 3972-9: Conduct a 13-week repeat-dose pharmacokinetic and pharmacodynamic (PK/PD) study in a-galactosidase deficient (aGAL KO) mice to evaluate changes in the GL3 biomarker in plasma and in the kidney, skin, heart, brain, spleen, and liver in relation to treatment with pegunigalsidase alfa. Correlate reductions in GL3 with pharmacokinetic exposures to pegunigalsidase alfa in this study."~"Pending"~~12/31/2025 0:00:00~7/9/2026 0:00:00
385381~"CDER"~"BLA"~761310.00~"AbbVie Inc."~"Elahere (mirvetuximab soravtansine-gynx)"~11/14/2022 0:00:00~"Original"~1~"4347-5"~"PMC"~"506B PMC"~4347.00~5~"PMC 4347-5: Conduct an integrated analysis containing data from clinical trials and other data sources such as post-marketing reports, real-world evidence and other sources to further characterize the safety and efficacy of mirvetuximab soravtansine in patients from racial and ethnic minority groups. The analyses should support comparative safety and efficacy outcome analyses between the aforementioned populations and White patients."~"Ongoing"~~11/30/2028 0:00:00~1/7/2026 0:00:00
385382~"CDER"~"BLA"~761310.00~"AbbVie Inc."~"Elahere (mirvetuximab soravtansine-gynx)"~11/14/2022 0:00:00~"Supplement"~5~"4610-1"~"PMC"~"506B PMC"~4610.00~1~"PMC 4610-1: Complete the ongoing clinical trial, Study 0416, entitled,  A Study of Mirvetuximab Soravtansine vs. Investigator s Choice of Chemotherapy in Platinum-Resistant, Advanced High-Grade Epithelial Ovarian, Primary Peritoneal, or Fallopian Tube Cancers with High Folate Receptor-Alpha Expression (MIRASOL),  including the pre-specified final overall survival analysis."~"Fulfilled"~~12/31/2024 0:00:00~1/7/2026 0:00:00
385396~"CDER"~"NDA"~220152.00~"VANDA PHARMACEUTICALS INC"~"Nereus (tradipitant) capsules"~12/30/2025 0:00:00~"Original"~1~"4946-6"~"PMC"~"506B PMC"~4946.00~6~"PMC 4946-6: Conduct a clinical pharmacology study to evaluate the effects of hepatic impairment on the pharmacokinetics of tradipitant and its major metabolites compared to subjects with normal hepatic function."~"Pending"~~6/30/2028 0:00:00~
385397~"CDER"~"NDA"~220152.00~"VANDA PHARMACEUTICALS INC"~"Nereus (tradipitant) capsules"~12/30/2025 0:00:00~"Original"~1~"4946-7"~"PMC"~"506B PMC"~4946.00~7~"PMC 4946-7: Conduct a clinical pharmacology study to evaluate the effects of severe renal impairment on the pharmacokinetics of tradipitant and its major metabolites compared to subjects with normal renal function."~"Pending"~~6/30/2028 0:00:00~
385398~"CDER"~"NDA"~220152.00~"VANDA PHARMACEUTICALS INC"~"Nereus (tradipitant) capsules"~12/30/2025 0:00:00~"Original"~1~"4946-8"~"PMC"~"506B PMC"~4946.00~8~"PMC 4946-8: Conduct in vitro assessment(s) to evaluate the contribution of all major CYP enzymes to the overall metabolism of tradipitant and its active metabolites, and to assess the relative contribution of specific CYP enzymes to tradipitant metabolism. The in vitro study results will determine the need for subsequent clinical drug-drug interaction study(ies)."~"Pending"~~10/31/2027 0:00:00~
385399~"CDER"~"NDA"~220152.00~"VANDA PHARMACEUTICALS INC"~"Nereus (tradipitant) capsules"~12/30/2025 0:00:00~"Original"~1~"4946-9"~"PMC"~"506B PMC"~4946.00~9~"PMC 4946-9: Conduct a study to evaluate the effects of tradipitant on QT intervals in the fed state."~"Pending"~~8/31/2027 0:00:00~
385400~"CDER"~"NDA"~220149.00~"JANSSEN BIOTECH INC"~"Icotyde (icotrokinra) tablet"~3/17/2026 0:00:00~"Original"~1~"4964-3"~"PMR"~"505 (o)(3)"~4964.00~3~"PMR 4964-3: Perform a lactation study in lactating women who have received icotrokinra to measure concentrations of icotrokinra in breast milk using a validated assay. Assess the effects on the breastfed infant, if available, based on study population."~"Pending"~~9/30/2029 0:00:00~
385401~"CDER"~"NDA"~220295.00~"INTERCEPT PHARMACEUTICALS INC"~"Lynavoy (linerixibat) tablet"~3/17/2026 0:00:00~"Original"~1~"4937-1"~"PMR"~"505 (o)(3)"~4937.00~1~"PMR 4937-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to linerixibat during pregnancy to assess risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant. Assess infant outcomes through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Pending"~~9/30/2037 0:00:00~
385414~"CDER"~"BLA"~761113.00~"Sanofi-Aventis U.S. LLC"~"Sarclisa (isatuximab-irfc)"~3/2/2020 0:00:00~"Supplement"~14~"4698-2"~"PMC"~"506B PMC"~4698.00~2~"PMC 4698-2: Conduct an integrated analysis containing data from clinical trials, postmarketing reports, compassionate use/expanded access programs, real-world evidence, and other sources, to further characterize the safety and efficacy of isatuximab in combination with bortezomib, lenalidomide, and dexamethasone (Isa-VRd) among U.S. racial and ethnic minority patients with multiple myeloma."~"Pending"~~8/31/2031 0:00:00~4/29/2026 0:00:00
385415~"CDER"~"BLA"~761115.00~"Gilead Sciences, Inc. "~"Trodelvy (sacituzumab govitecan-hziy)"~4/22/2020 0:00:00~"Original"~1~"3504-3"~"PMR"~"505 (o)(3)"~3504.00~3~"PMR 3504-3: Conduct an open-label, non-randomized, dose-escalation trial to determine an appropriate starting dose of sacituzumab govitecan in patients with moderate hepatic impairment, according to the National Cancer Institute Organ Dysfunction Working Group criteria in the target patient population. Safety and pharmacokinetic information of IMMU-132 and SN-38 will be collected to determine the appropriate starting dose of sacituzumab govitecan for this population. Submit the datasets with the final report."~"Delayed"~"The revised Trial Completion and Final Report Submission milestones were missed and the FDA issued a Notification of Missed Milestones letter on 03/11/2024."~9/30/2021 0:00:00~6/18/2026 0:00:00
385416~"CDER"~"BLA"~761115.00~"Gilead Sciences, Inc. "~"Trodelvy (sacituzumab govitecan-hziy)"~4/22/2020 0:00:00~"Supplement"~5~"4043-1"~"PMC"~"506B PMC"~4043.00~1~"PMC 4043-1: Submit the final results from the ongoing study  Testing Sacituzumab Govitecan Therapy in Patients with HER2-Negative Breast Cancer and Brain Metastases  (NCT04647916) or another trial that includes triple negative breast cancer (TNBC) patients with brain metastases that may further inform the efficacy and safety of sacituzumab govitecan in a brain metastases population in TNBC."~"Delayed"~"The applicant requested revised milestones because of challenges enrolling patients into the study. Revised milestones were acknowledged in a letter dated 04/07/2025.

"~2/28/2025 0:00:00~6/18/2026 0:00:00
385417~"CDER"~"BLA"~761163.00~"Incyte Corporation"~"Monjuvi (tafasitamab-cxix)"~7/31/2020 0:00:00~"Original"~1~"3904-1"~"PMR"~"Accelerated Approval"~3904.00~1~"PMR 3904-1: Submit the final report and datasets from a randomized, Phase 3 clinical trial to verify the clinical benefit of tafasitamab in patients with diffuse large B-cell lymphoma. The trial should include sufficient numbers of racial and ethnic minority patients to better reflect the U.S. patient population and allow for the interpretation of the results in these patient populations. Patients should be randomized to receive immunotherapy and/or chemotherapy with or without tafasitamab and lenalidomide. The primary endpoint should be progression-free survival, with secondary endpoints that include overall survival and objective response rate."~"Submitted"~"The final report was submitted to FDA on 3/30/2026."~12/31/2025 0:00:00~9/29/2025 0:00:00
385418~"CDER"~"BLA"~761163.00~"Incyte Corporation"~"Monjuvi (tafasitamab-cxix)"~7/31/2020 0:00:00~"Original"~1~"3904-2"~"PMC"~"506B PMC"~3904.00~2~"PMC 3904-2: Submit an integrated final report containing data from clinical trials (including data from the ongoing clinical development program), post-marketing reports, compassionate use/expanded access program, real-world data and other sources to further characterize the safety and efficacy of tafasitamab in combination with lenalidomide among U.S. racial and ethnic minority patients with DLBCL."~"Delayed"~"The final protocol was submitted after the original final protocol submission milestone."~9/30/2026 0:00:00~9/29/2025 0:00:00
385419~"CDER"~"BLA"~761164.00~"Recordati Rare Diseases, Inc."~"Enjaymo (sutimlimab-jome)"~2/4/2022 0:00:00~"Original"~1~"3922-1"~"PMR"~"505 (o)(3)"~3922.00~1~"PMR 3922-1: Conduct a 5-year registry study to characterize the safety of sutimlimab in patients with primary Cold Agglutinin Disease. Yearly interim reports and the final study report should include a summary of the major safety findings and outcome of events. The summaries must discuss available information on autoimmune diseases, serious bacterial infections, sutimlimab dosing, vaccination status, serotype/serogroup of Streptococcus pneumoniae and Neisseria meningitidis isolates, and concomitant medications."~"Ongoing"~~10/31/2031 0:00:00~4/3/2026 0:00:00
385420~"CDER"~"BLA"~761166.00~"PharmaEssentia Corporation"~"Besremi (ropeginterferon alfa-2b-njft) injection, Besremi Pen (ropeginterferon alfa-2b-njft) injection"~11/12/2021 0:00:00~"Original"~1~"4171-1"~"PMR"~"505 (o)(3)"~4171.00~1~"PMR 4171-1: Complete Study EUPAS29462  A Prospective, Multicenter, Non-interventional, Observational, Post-authorization Safety Study of Ropeginterferon alfa-2b in Polycythemia Vera Patients  to evaluate safety and tolerability during the titration and maintenance phase of Besremi administration in patients with Polycythemia Vera. Specific safety outcomes of interest include hepatotoxicity, cardiovascular and thromboembolic events in treated patients."~"Delayed"~"The final report date milestone has been missed and the FDA issued a Notification of Missed Milestone letter on 03/03/2026."~9/30/2023 0:00:00~1/9/2026 0:00:00
385421~"CDER"~"BLA"~761169.00~"Regeneron Pharmaceuticals, Inc."~"Inmazeb (atoltivimab, maftivimab, and odesivimab-ebgn)"~10/14/2020 0:00:00~"Original"~1~"3936-5"~"PMC"~"506B PMC"~3936.00~5~"PMC 3936-5: Perform complete resistance analysis of sequences derived from subjects treated with REGN-EB3 in the PALM trial, if access to the data becomes available."~"Released"~~9/30/2022 0:00:00~12/12/2025 0:00:00
385422~"CDER"~"BLA"~761169.00~"Regeneron Pharmaceuticals, Inc."~"Inmazeb (atoltivimab, maftivimab, and odesivimab-ebgn)"~10/14/2020 0:00:00~"Original"~1~"3936-6"~"PMC"~"506B PMC"~3936.00~6~"PMC 3936-6: Collaborate with US public health agencies, other public health agencies, and local health authorities, as appropriate to design and conduct a trial to evaluate the efficacy, safety and pharmacokinetics of a higher dose of INMAZEB (atoltivimab, maftivimab, and odesivimab-ebgn) vs. INMAZEB 150 mg/kg in Zaire ebolavirusinfected adult and pediatric patients with cycle threshold (CT) values for nucleoprotein targets of less than or equal to 22 to determine if a change in dosing regimen is needed in these
patients."~"Ongoing"~~~12/12/2025 0:00:00
385423~"CDER"~"BLA"~761171.00~"BTG International Inc."~"DANYELZA (naxitamab-gqgk)"~11/25/2020 0:00:00~"Original"~1~"3950-1"~"PMR"~"Accelerated Approval"~3950.00~1~"PMR 3950-1: Submit the final report, including datasets, from an ongoing multicenter clinical trial to verify and further characterize the clinical benefit of naxitamab for the treatment of patients with relapsed or refractory neuroblastoma in combination with granulocyte macrophage colonystimulating factor (GM-CSF) in bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy; and to provide a more precise estimation of overall response rate and duration of response (according to the revised International Neuroblastoma Response Criteria by blinded independent review). Enroll a minimum of 80 patients with evaluable disease and systematically follow all responding patients for at least 12 months from the onset of response, or until disease progression, whichever comes first. Include subgroup analyses of overall response rate and duration of response by prior antiGD2 exposure, disease status (refractory vs. relapsed disease), anti-drug antibody (ADA) status (neutralizing antibody [nAb] positive versus nAb negative, ADA positive versus ADA negative), and area of disease involvement (bone marrow vs. bone) in the final report reflecting a sufficient number of patients in relevant subgroups. Derive the clinical data from a minimum of six study sites, with at least five patients enrolled per site in at least five sites."~"Ongoing"~"101 patients out of the planned 122 patients have been enrolled into the trial."~9/30/2027 0:00:00~1/23/2025 0:00:00
385424~"CDER"~"BLA"~761171.00~"BTG International Inc."~"DANYELZA (naxitamab-gqgk)"~11/25/2020 0:00:00~"Original"~1~"3950-2"~"PMR"~"505 (o)(3)"~3950.00~2~"PMR 3950-2: Conduct a comprehensive analysis evaluating and characterizing the incidence, clinical presentation, management, and outcome of the potential serious risk of naxitamab-associated infusion-related reactions in a sufficient number of patients to adequately characterize the risk. Submit an integrated final report containing data using the preferred terms included in product labelling from patient-level and pooled analyses of ongoing and completed clinical trials, post-marketing reports and/or literature reports and a comprehensive pharmacovigilance assessment for this potential serious risk. The report should also include an analysis of premedication administered. The results from this report may inform product labelling."~"Ongoing"~~9/30/2027 0:00:00~1/23/2025 0:00:00
385425~"CDER"~"BLA"~761171.00~"BTG International Inc."~"DANYELZA (naxitamab-gqgk)"~11/25/2020 0:00:00~"Original"~1~"3950-4"~"PMC"~"506B PMC"~3950.00~4~"PMC 3950-4: Conduct immunogenicity testing in all patients enrolled in the ongoing multicenter clinical trial (Trial 201) of naxitamab using the validated assays to adequately characterize the incidence of anti-drug antibody (ADA) and neutralizing antibodies against naxitamab and assess the impact of ADA on the pharmacokinetics (PK), safety and efficacy of naxitamab in accordance with FDA Guidance for Industry: Immunogenicity Assessment for Therapeutic Protein Products available at https://www.fda.gov/downloads/drugs/guidances/ucm338856.pdf. Submit the integrated immunogenicity assessment report in the final report submission. The results from this study may inform product labeling."~"Ongoing"~~9/30/2027 0:00:00~1/23/2025 0:00:00
385426~"CDER"~"BLA"~761339.00~"Regeneron Pharmaceuticals, Inc."~"Veopoz (pozelimab-bbfg)"~8/18/2023 0:00:00~"Original"~1~"4490-2"~"PMC"~"506B PMC"~4490.00~2~"PMC 4490-2: Re-evaluate clinical samples for anti-pozelimab antibodies (ADA) from clinical Trial R3918-PLE-1878 using the assay described in 4490-1. Evaluate neutralizing capacity of ADA using the assay described in 4490-3 from clinical Trial R3918-PLE-1878 in all confirmed ADA positive samples."~"Pending"~~8/31/2028 0:00:00~10/17/2025 0:00:00
385427~"CDER"~"BLA"~761339.00~"Regeneron Pharmaceuticals, Inc."~"Veopoz (pozelimab-bbfg)"~8/18/2023 0:00:00~"Original"~1~"4490-3"~"PMC"~"506B PMC"~4490.00~3~"PMC 4490-3: Develop and validate a sensitive assay to evaluate the neutralizing capacity of anti-pozelimab antibodies (ADA) in confirmed ADA positive patient samples."~"Pending"~~8/31/2028 0:00:00~10/17/2025 0:00:00
385428~"CDER"~"BLA"~761339.00~"Regeneron Pharmaceuticals, Inc."~"Veopoz (pozelimab-bbfg)"~8/18/2023 0:00:00~"Original"~1~"4490-4"~"PMC"~"506B PMC"~4490.00~4~"PMC 4490-4: Conduct a study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), immunogenicity, and safety of pozelimab in pediatric subjects with CHAPLE disease 1 to 5 years of age. The study will evaluate a minimum of 5 subjects, including at least 3 subjects less than 3 years of age. Samples will be analyzed for anti-pozelimab antibodies (ADA) using the assays described in PMC 4490-1 and 4490-3. Subjects will be evaluated for a minimum of 12 months."~"Delayed"~"The final protocol milestone was missed. The final protocol was
acknowledged in a letter dated 1/10/2025."~2/28/2031 0:00:00~10/17/2025 0:00:00
385429~"CDER"~"BLA"~761342.00~"Janssen Biotech, Inc."~"Talvey (talquetamab-tgvs)"~8/9/2023 0:00:00~"Original"~1~"4473-1"~"PMR"~"Accelerated Approval"~4473.00~1~"PMR 4473-1: Conduct a randomized clinical trial that evaluates the clinical efficacy and safety of talquetamab in patients with relapsed or refractory multiple myeloma. Patients should be randomized to receive a talquetamab-based regimen compared to standard therapy for relapsed or refractory multiple myeloma. The primary endpoint should be progression-free survival and secondary endpoints should include overall survival and overall response rate. The trial should enroll sufficient numbers of racial and ethnic minority patients and older patients (ages 65-74 years and 75 years and above) to enable an evaluation of talquetamab in a study population that reflects the U.S. population of patients with multiple myeloma."~"Submitted"~"The final report was submitted to FDA on 03/30/2026."~12/31/2026 0:00:00~10/3/2024 0:00:00
385430~"CDER"~"BLA"~761342.00~"Janssen Biotech, Inc."~"Talvey (talquetamab-tgvs)"~8/9/2023 0:00:00~"Original"~1~"4473-2"~"PMR"~"505 (o)(3)"~4473.00~2~"PMR 4473-2: Conduct a clinical trial to further characterize the serious risk of oral toxicity including severe stomatitis, mucositis, and weight loss, and potential mitigation measures, in patients receiving talquetamab 0.4 mg/kg weekly and talquetamab 0.8 mg/kg every two weeks. Evaluate incidence rates, time to onset, and outcomes including patient reported outcomes. Include investigation of associations and temporal relationships between incidence and severity of oral toxicity adverse events and other potential risk factors such as age, race and comorbidities, and impact of the mitigation measures evaluated. The study should enroll sufficient numbers of Black/African American patients to enable an evaluation of the serious risk of oral toxicity with talquetamab in this population."~"Ongoing"~~9/30/2027 0:00:00~10/3/2024 0:00:00
385431~"CDER"~"BLA"~761342.00~"Janssen Biotech, Inc."~"Talvey (talquetamab-tgvs)"~8/9/2023 0:00:00~"Original"~1~"4473-3"~"PMC"~"506B PMC"~4473.00~3~"PMC 4473-3: Evaluate overall response rate and duration of response with talquetamab 0.4 mg/kg weekly and talquetamab 0.8 mg/kg every two weeks, administered as monotherapy in patients with relapsed or refractory multiple myeloma who have received at least 4 prior lines of therapies including a proteasome inhibitor, immunomodulatory agent, and anti-CD38 monoclonal antibody and have been exposed to T cell redirection therapy, from trial 64407564MMY1001 (MonumenTAL-1)."~"Fulfilled"~~3/31/2025 0:00:00~10/3/2024 0:00:00
385432~"CDER"~"BLA"~761344.00~"Amgen Inc."~"IMDELLTRA (tarlatamab-dlle)"~5/16/2024 0:00:00~"Original"~1~"4635-1"~"PMR"~"Accelerated Approval"~4635.00~1~"PMR 4635-1: Conduct a multicenter randomized clinical trial intended to verify anddescribe the clinical benefit of tarlatamab in patients with extensive stage small cell lung cancer (ES-SCLC) who have had disease progression on or after platinum-based chemotherapy."~"Fulfilled"~"Per FDA letter dated 11/19/2025, this PMR/PMC has been fulfilled."~8/31/2026 0:00:00~5/30/2025 0:00:00
385433~"CDER"~"BLA"~761344.00~"Amgen Inc."~"IMDELLTRA (tarlatamab-dlle)"~5/16/2024 0:00:00~"Original"~1~"4635-2"~"PMR"~"505 (o)(3)"~4635.00~2~"PMR 4635-2: Conduct an integrated safety analysis of data from patients with extensive stage small cell lung cancer to further characterize the long-term incidence, severity, and outcome of the known serious risks of cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, and neurologic toxicity. Include a comprehensive analysis from all available data sources including but not limited to patient-level and pooled analyses of ongoing and completed clinical trials. These data could also be obtained from the confirmatory trial titled  A Randomized, Open-label, Phase 3 Study of Tarlatamab Compared with Standard of Care in Subjects with Relapsed Small Cell Lung Cancer After Platinum-based First-line Chemotherapy (DeLLphi-304) ."~"Fulfilled"~~8/31/2026 0:00:00~5/30/2025 0:00:00
385434~"CDER"~"BLA"~761344.00~"Amgen Inc."~"IMDELLTRA (tarlatamab-dlle)"~5/16/2024 0:00:00~"Original"~1~"4635-3"~"PMC"~"506B PMC"~4635.00~3~"PMC 4635-3: Conduct an integrated analysis from ongoing, completed, or planned clinical trials and other potential data sources as appropriate enrolling a sufficient representation of United States (U.S.) racial and ethnic minority patients that is reflective of the U.S. population of patients with SCLC, to further characterize the efficacy, safety and pharmacokinetics of tarlatamab in these patients. In the analysis, include a sufficient number of patients enrolled in the U.S. and a sufficient number of racial and ethnic minority patients reflective of the incidence of SCLC in each subpopulation to allow for interpretation of the results. The analyses should support comparative efficacy and safety outcome analyses between the aforementioned populations and White patients."~"Ongoing"~~4/30/2027 0:00:00~5/30/2025 0:00:00
385435~"CDER"~"BLA"~761345.00~"Pfizer Inc."~"Elrexfio (elranatamab-bcmm)"~8/14/2023 0:00:00~"Original"~1~"4476-1"~"PMR"~"Accelerated Approval"~4476.00~1~"PMR 4476-1: Complete a randomized clinical trial in patients with relapsed or refractory multiple myeloma. Patients should be randomized to receive an elranatamab-based regimen compared to standard therapy for relapsed or refractory multiple myeloma. The primary endpoint should be progression-free survival and secondary endpoints should include overall survival and overall response rate. The trial should enroll sufficient numbers of older patients (ages 65-74 years and 75 years and above) to enable an evaluation of elranatamab in a study population that reflects the age of the U.S. population of patients with multiple myeloma."~"Ongoing"~"The trial has completed enrollment."~9/30/2026 0:00:00~10/9/2025 0:00:00
385436~"CDER"~"NDA"~220305.00~"KURA ONCOLOGY INC"~"Komzifti (ziftomenib) capsules, 200 mg"~11/13/2025 0:00:00~"Original"~1~"4929-4"~"PMR"~"505 (o)(3)"~4929.00~4~"PMR 4929-4: Conduct a clinical pharmacokinetic trial to assess the magnitude of change in exposure of ziftomenib and evaluate a serious potential risk of increased drug toxicity, and to determine an appropriate dosage of ziftomenib to minimize toxicity, in patients with severe renal impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Renal Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~5/31/2030 0:00:00~
385437~"CDER"~"NDA"~220305.00~"KURA ONCOLOGY INC"~"Komzifti (ziftomenib) capsules, 200 mg"~11/13/2025 0:00:00~"Original"~1~"4929-5"~"PMR"~"505 (o)(3)"~4929.00~5~"PMR 4929-5: Complete KO-MEN-001 Sub-study 2, a clinical pharmacokinetic trial to evaluate the effect of repeat doses of itraconazole on the single dose pharmacokinetics of ziftomenib to assess the serious potential risk of excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled M12 Drug Interaction Studies."~"Ongoing"~~12/31/2027 0:00:00~
385438~"CDER"~"NDA"~220305.00~"KURA ONCOLOGY INC"~"Komzifti (ziftomenib) capsules, 200 mg"~11/13/2025 0:00:00~"Original"~1~"4929-6"~"PMR"~"505 (o)(3)"~4929.00~6~"PMR 4929-6: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of BCRP inhibitors on the single dose pharmacokinetics of ziftomenib to assess the serious potential risk of excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled M12 Drug Interaction Studies."~"Pending"~~5/31/2030 0:00:00~
385439~"CDER"~"NDA"~220305.00~"KURA ONCOLOGY INC"~"Komzifti (ziftomenib) capsules, 200 mg"~11/13/2025 0:00:00~"Original"~1~"4929-7"~"PMR"~"505 (o)(3)"~4929.00~7~"PMR 4929-7: Complete KO-MEN-001 Sub-study 1, a clinical pharmacokinetic trial to evaluate the effect of repeat doses of ziftomenib on the single dose pharmacokinetics of midazolam to assess the serious potential risk of excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled M12 Drug Interaction Studies."~"Ongoing"~~6/30/2027 0:00:00~
385440~"CDER"~"NDA"~220305.00~"KURA ONCOLOGY INC"~"Komzifti (ziftomenib) capsules, 200 mg"~11/13/2025 0:00:00~"Original"~1~"4929-8"~"PMC"~"506B PMC"~4929.00~8~"PMC 4929-8: Conduct a study to support the availability of an in vitro diagnostic device (companion diagnostic) for identifying susceptible NPM1 mutations in patients with acute myeloid leukemia for the safe and effective use of ziftomenib."~"Pending"~~3/31/2027 0:00:00~
385441~"CDER"~"NDA"~220305.00~"KURA ONCOLOGY INC"~"Komzifti (ziftomenib) capsules, 200 mg"~11/13/2025 0:00:00~"Original"~1~"4929-9"~"PMC"~"506B PMC"~4929.00~9~"PMC 4929-9: Conduct an in vitro study to clarify the activity of ziftomenib in acute myeloid leukemia cells with NPM1 mutations other than the A, B, and D subtypes."~"Pending"~~12/31/2027 0:00:00~
385442~"CDER"~"NDA"~220358.00~"VANDA PHARMACEUTICALS INC"~"Bysanti (milsaperidone) tablets"~2/20/2026 0:00:00~"Original"~1~"4968-1"~"PMR"~"Pediatric Research Equity Act"~4968.00~1~"PMR 4968-1: Conduct an open-label study to assess the long-term safety in patients aged 10 to less than 13 years with bipolar I disorder."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~1/31/2031 0:00:00~
385443~"CDER"~"NDA"~220358.00~"VANDA PHARMACEUTICALS INC"~"Bysanti (milsaperidone) tablets"~2/20/2026 0:00:00~"Original"~1~"4968-2"~"PMR"~"Pediatric Research Equity Act"~4968.00~2~"PMR 4968-2: A long-term safety study in pediatric patients 13 to 17 years of age."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~11/30/2026 0:00:00~
385444~"CDER"~"NDA"~220358.00~"VANDA PHARMACEUTICALS INC"~"Bysanti (milsaperidone) tablets"~2/20/2026 0:00:00~"Original"~1~"4968-3"~"PMR"~"Pediatric Research Equity Act"~4968.00~3~"PMR 4968-3: Conduct a GLP juvenile animal study to assess the toxicology in juvenile rats to support clinical trials in the intended pediatric population ages 10 to less than 13 years."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~7/31/2026 0:00:00~
385445~"CDER"~"NDA"~220358.00~"VANDA PHARMACEUTICALS INC"~"Bysanti (milsaperidone) tablets"~2/20/2026 0:00:00~"Original"~1~"4968-4"~"PMR"~"Pediatric Research Equity Act"~4968.00~4~"PMR 4968-4: Conduct an open-label, multiple oral dose study to demonstrate the safety, tolerability, and pharmacokinetics in patients ages 10 to less than 13 years with manic or mixed episode associated with bipolar I disorder."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~1/31/2027 0:00:00~
385446~"CDER"~"NDA"~220358.00~"VANDA PHARMACEUTICALS INC"~"Bysanti (milsaperidone) tablets"~2/20/2026 0:00:00~"Original"~1~"4968-5"~"PMR"~"505 (o)(3)"~4968.00~5~"PMR 4968-5: Conduct a lactation study in lactating women who have received therapeutic doses of milsaperidone to assess concentrations of
milsaperidone in breast milk using a validated assay. Assess the effects on the breastfed infant if available, based on study population."~"Pending"~~8/31/2029 0:00:00~
385447~"CDER"~"NDA"~220449.00~"BOEHRINGER INGELHEIM PHARMACEUTICALS INC"~"Jascayd (nerandomilast) Film-Coated Tablets, 9 mg and 18 mg"~12/19/2025 0:00:00~"Original"~1~"4950-1"~"PMR"~"Pediatric Research Equity Act"~4950.00~1~"PMR 4950-1: Conduct a study of at least 24-weeks duration to evaluate the doseexposure, safety, and exploratory efficacy of nerandomilast in children from 2 to < 18 years of age with fibrosing interstitial lung disease using a randomized, double-blind, placebo-controlled design for children from 6 to < 18 years of age."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~10/31/2029 0:00:00~
385448~"CDER"~"NDA"~220641.00~"CORCEPT THERAPEUTICS INC"~"Lifyorli (relacorilant) capsules"~3/25/2026 0:00:00~"Original"~1~"4972-1"~"PMR"~"505 (o)(3)"~4972.00~1~"PMR 4972-1: Conduct a clinical trial enrolling an adequate number of patients with moderate hepatic impairment, using the NCI-ODWG criteria, to evaluate the potential serious risk of increased serious adverse reactions and the overall safety profile in patients with moderate hepatic impairment, to support dose selection for relacorilant in combination with nab-paclitaxel and inform labeling. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~8/31/2030 0:00:00~
385449~"CDER"~"NDA"~220641.00~"CORCEPT THERAPEUTICS INC"~"Lifyorli (relacorilant) capsules"~3/25/2026 0:00:00~"Original"~1~"4972-2"~"PMC"~"506B PMC"~4972.00~2~"PMC 4972-2: Conduct an exploratory analysis to assess the relationship between baseline expression of tumor glucocorticoid receptor (GR) and response to relacorilant in combination with nab-paclitaxel for patients with advanced, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer enrolled to ROSELLA (NCT05257408). Conduct an assessment of the impact of GR expression level cutoff on the analyses, including at a cutoff H-score of 100."~"Pending"~~8/31/2027 0:00:00~
385450~"CDER"~"NDA"~220934.00~"ELI LILLY AND CO"~"Foundayo"~4/1/2026 0:00:00~"Original"~1~"4977-1"~"PMR"~"Pediatric Research Equity Act"~4977.00~1~"PMR 4977-1: Complete the ongoing 72-week, randomized, double-blind, placebo-controlled, multicenter, parallel-arm study J4M-MC-PW01 (ADVANCE-ATTAIN-ADOLESCENTS) to evaluate the safety and efficacy of orforglipron as an adjunct to lifestyle intervention to reduce excess body weight and maintain weight reduction long term in a cohort of pediatric patients aged 12 to less than 18 years with obesity."~"Ongoing"~"The study is underway."~12/31/2028 0:00:00~
385451~"CDER"~"NDA"~217785.00~"MADRIGAL PHARMACEUTICALS INC"~"Rezdiffra"~3/14/2024 0:00:00~"Original"~1~"4577-8"~"PMC"~"506B PMC"~4577.00~8~"PMC 4577-8: Conduct a clinical drug-drug interaction study to evaluate the effects of an inhibitor of organic anion transporting polypeptide (OATP) 1B1 and OATP1B3 on the pharmacokinetics of resmetirom. Design and conduct the trial in accordance with the Guidance for Industry: Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter- Mediated Drug Interactions."~"Fulfilled"~~4/30/2025 0:00:00~5/8/2026 0:00:00
385452~"CDER"~"NDA"~217806.00~"ELI LILLY AND CO"~"Zepbound (tirzepatide) injection, and Zepbound KwikPen (tirzepatide) injection"~11/8/2023 0:00:00~"Original"~1~"4743-1"~"PMR"~"505 (o)(3)"~4743.00~1~"PMR 4743-1: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of tirzepatide and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Pending"~~11/30/2026 0:00:00~7/9/2026 0:00:00
385453~"CDER"~"NDA"~217806.00~"ELI LILLY AND CO"~"Zepbound (tirzepatide) injection, and Zepbound KwikPen (tirzepatide) injection"~11/8/2023 0:00:00~"Original"~1~"4534-1"~"PMR"~"Pediatric Research Equity Act"~4534.00~1~"PMR 4534-1: Conduct a 72-week, randomized, double-blind, placebo-controlled, multicenter, parallel-arm study to evaluate the safety and efficacy of Zepbound (tirzepatide) as an adjunct to lifestyle intervention for chronic weight management in pediatric patients aged 12 to 17 years (inclusive) with obesity."~"Ongoing"~"160 participants have been enrolled into the trial."~8/31/2027 0:00:00~7/9/2026 0:00:00
385454~"CDER"~"NDA"~217806.00~"ELI LILLY AND CO"~"Zepbound (tirzepatide) injection, and Zepbound KwikPen (tirzepatide) injection"~11/8/2023 0:00:00~"Original"~1~"4534-2"~"PMR"~"Pediatric Research Equity Act"~4534.00~2~"PMR 4534-2: Conduct a safety, tolerability, pharmacokinetic, and pharmacodynamic study of Zepbound (tirzepatide) administered subcutaneously in pediatric patients aged 6 to 11 years (inclusive) with obesity. Data from this study should be considered when choosing dose(s) for the safety and efficacy trial in this pediatric population."~"Fulfilled"~"Per FDA letter dated 05/12/2026, this PMR/PMC has been fulfilled."~10/31/2025 0:00:00~7/9/2026 0:00:00
385455~"CDER"~"NDA"~217806.00~"ELI LILLY AND CO"~"Zepbound (tirzepatide) injection, and Zepbound KwikPen (tirzepatide) injection"~11/8/2023 0:00:00~"Original"~1~"4534-3"~"PMR"~"Pediatric Research Equity Act"~4534.00~3~"PMR 4534-3: Conduct a 72-week, randomized, double-blind, placebo-controlled, multicenter, parallel-arm study to evaluate the safety and efficacy of Zepbound (tirzepatide) as an adjunct to lifestyle intervention for chronic weight management in pediatric patients aged 6 to 11 years (inclusive) with obesity."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2030 0:00:00~7/9/2026 0:00:00
385456~"CDER"~"NDA"~217806.00~"ELI LILLY AND CO"~"Zepbound (tirzepatide) injection, and Zepbound KwikPen (tirzepatide) injection"~11/8/2023 0:00:00~"Original"~1~"4534-4"~"PMR"~"505 (o)(3)"~4534.00~4~"PMR 4534-4: Collect global data from a prospective pregnancy exposure registry, preferably a disease-based multi-product pregnancy registry, using a registry-based cohort study to compare the maternal, fetal, and infant outcomes of women exposed to Zepbound (tirzepatide) for chronic weight management during pregnancy with unexposed comparator population(s). Align the study protocol with protocol(s) outside the United States to reach the target sample size. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-forgestational-age births, and any other adverse outcomes, including postnatal growth and development. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of
life."~"Pending"~~6/30/2036 0:00:00~7/9/2026 0:00:00
385457~"CDER"~"NDA"~217806.00~"ELI LILLY AND CO"~"Zepbound (tirzepatide) injection, and Zepbound KwikPen (tirzepatide) injection"~11/8/2023 0:00:00~"Original"~1~"4534-5"~"PMR"~"505 (o)(3)"~4534.00~5~"PMR 4534-5: Conduct an additional pregnancy study that uses a different design from the pregnancy exposure registry (for example a cohort study using claims or electronic medical record data) to compare the risks and prevalence of pregnancy and infant outcomes (including but not limited to major congenital malformations, spontaneous abortions, stillbirths, small-forgestational-age births, preterm births, and postnatal growth and development) between women exposed to Zepbound (tirzepatide) for chronic weight management during pregnancy and unexposed comparator population(s)."~"Pending"~~9/30/2032 0:00:00~7/9/2026 0:00:00
385458~"CDER"~"NDA"~217806.00~"ELI LILLY AND CO"~"Zepbound (tirzepatide) injection, and Zepbound KwikPen (tirzepatide) injection"~11/8/2023 0:00:00~"Original"~1~"4534-6"~"PMR"~"505 (o)(3)"~4534.00~6~"PMR 4534-6: Conduct a medullary thyroid carcinoma registry-based case series of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of Zepbound (tirzepatide) for the treatment of overweight and obesity into the marketplace. This study will also establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to the use of Zepbound (tirzepatide) for the treatment of obesity/overweight."~"Ongoing"~~8/31/2041 0:00:00~7/9/2026 0:00:00
385459~"CDER"~"NDA"~217806.00~"ELI LILLY AND CO"~"Zepbound (tirzepatide) injection, and Zepbound KwikPen (tirzepatide) injection"~11/8/2023 0:00:00~"Supplement"~13~"4759-1"~"PMR"~"Pediatric Research Equity Act"~4759.00~1~"PMR 4759-1: Conduct a 72-week randomized, double-blind, placebo-controlled, parallelgroup clinical study in adolescents 12 to 17 years of age (inclusive) with obesity-related obstructive sleep apnea (OSA) to assess the safety, efficacy, and pharmacokinetics of tirzepatide."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~10/31/2036 0:00:00~7/9/2026 0:00:00
385460~"CDER"~"NDA"~217806.00~"ELI LILLY AND CO"~"Zepbound (tirzepatide) injection, and Zepbound KwikPen (tirzepatide) injection"~11/8/2023 0:00:00~"Supplement"~13~"4759-2"~"PMR"~"Pediatric Research Equity Act"~4759.00~2~"PMR 4759-2: Conduct a 72-week randomized, double-blind, placebo controlled, parallel group clinical study in children 6 to 11 years of age (inclusive) with obesity-related obstructive sleep apnea to assess the safety, efficacy, and pharmacokinetics of tirzepatide."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~5/30/2040 0:00:00~7/9/2026 0:00:00
385461~"CDER"~"NDA"~217865.00~"ITALFARMACO SPA"~"Duvyzat (givinostat)"~3/21/2024 0:00:00~"Original"~1~"4575-1"~"PMR"~"505 (o)(3)"~4575.00~1~"PMR 4575-1: Conduct a carcinogenicity study of givinostat in mouse."~"Ongoing"~~12/31/2025 0:00:00~5/19/2026 0:00:00
385462~"CDER"~"NDA"~217865.00~"ITALFARMACO SPA"~"Duvyzat (givinostat)"~3/21/2024 0:00:00~"Original"~1~"4575-2"~"PMR"~"505 (o)(3)"~4575.00~2~"PMR 4575-2: Conduct a 2-year carcinogenicity study of givinostat in rat."~"Ongoing"~~12/31/2025 0:00:00~5/19/2026 0:00:00
385463~"CDER"~"BLA"~761172.00~"Emergent Manufacturing Operations Baltimore LLC"~"EBANGA (ansuvimab-zykl)"~12/21/2020 0:00:00~"Original"~1~"3965-4"~"PMC"~"506B PMC"~3965.00~4~"PMC 3965-4: Collaborate with US public health agencies, other public health agencies and local health authorities, as appropriate to design and conduct a trial to evaluate the efficacy, safety and pharmacokinetics of a higher dose of Ebanga (ansuvimab-zykl) vs. Ebanga (ansuvimab-zykl) 50 mg/kg in Zaire ebolavirus infected adult and pediatric patients with cycle-threshold (CT) values for nucleoprotein gene targets of less than or equal to 22 to determine if a change in dosing regimen is needed in these patients."~"Ongoing"~~~2/18/2026 0:00:00
385464~"CDER"~"BLA"~761173.00~"Fresenius Kabi USA, LLC"~"Stimufend (pegfilgrastim-fpgk)"~9/1/2022 0:00:00~"Original"~1~"3977-1"~"PMR"~"Pediatric Research Equity Act"~3977.00~1~"PMR 3977-1: Submit pediatric assessments for Stimufend (pegfilgrastim-fpgk) as described in section 505B(a)(2)(A) of the FD&C Act, including development of an  appropriate formulation  (presentation) that can be used to directly and accurately administer Stimufend (pegfilgrastim-fpgk) to pediatric patients who weigh less than 45 kg and require doses that are less than 0.6 mL (6 mg), and conducting any necessary human factors studies to evaluate the ability of healthcare providers and/or caregivers to measure the appropriate doses."~"Delayed"~"Study completion milestone has been missed. Deferral Extension Requested 03/04/2026. Denied per FDA letter dated 04/17/2026."~4/30/2028 0:00:00~10/30/2025 0:00:00
385465~"CDER"~"BLA"~761174.00~"GlaxoSmithKline LLC"~"Jemperli (dostarlimab-gxly)"~4/22/2021 0:00:00~"Original"~1~"4124-1"~"PMR"~"Accelerated Approval"~4124.00~1~"PMR 4124-1: Conduct a clinical trial evaluating overall response rate, and duration of response, to verify and describe the clinical benefit of Jemperli in patients with mismatch repair deficient (dMMR), recurrent or advanced solid tumors, including at least 300 patients across all tumor types, and  including a sufficient number of patients and representation of tumor types (other than endometrial and gastrointestinal tumors). In order to characterize response rate and duration of response, patients should be followed for at least 12 months from the onset of response. Submit the datasets with final report."~"Delayed"~"Delayed due to enrollment issues.  Revised milestones were acknowledged in a letter dated 10/28/2022."~10/31/2022 0:00:00~6/18/2026 0:00:00
385466~"CDER"~"BLA"~761174.00~"GlaxoSmithKline LLC"~"Jemperli (dostarlimab-gxly)"~4/22/2021 0:00:00~"Original"~1~"4124-2"~"PMC"~"506B PMC"~4124.00~2~"PMC 4124-2: Commitment to establish and support the availability of a nucleic acid based in vitro diagnostic device that is essential to support the safe and effective use of Jemperli for patients with tumors that are microsatellite instability high (MSI-H) through an appropriate analytical and clinical validation study using clinical trial data. Summarize the study results in the final report submission."~"Released"~~12/31/2025 0:00:00~6/18/2026 0:00:00
385467~"CDER"~"BLA"~761174.00~"GlaxoSmithKline LLC"~"Jemperli (dostarlimab-gxly)"~4/22/2021 0:00:00~"Supplement"~4~"4404-1"~"PMC"~"506B PMC"~4404.00~1~"PMC 4404-1: Conduct an integrated analysis containing data from clinical trials and other data sources such as post-marketing reports, real-world evidence and other sources to further characterize the efficacy and safety of dostarlimab in patients with dMMR endometrial cancer from racial and ethnic minority groups. The analyses should support comparative efficacy and safety analyses between the aforementioned populations and White patients."~"Ongoing"~~12/31/2026 0:00:00~6/18/2026 0:00:00
385468~"CDER"~"BLA"~761174.00~"GlaxoSmithKline LLC"~"Jemperli (dostarlimab-gxly)"~4/22/2021 0:00:00~"Supplement"~6~"4481-1"~"PMC"~"506B PMC"~4481.00~1~"PMC 4481-1: Complete the ongoing clinical trial, RUBY Part 1, titled  A Phase 3, Randomized, Double-blind, Multicenter Study of Dostarlimab (TSR-042) plus Carboplatin-paclitaxel versus Placebo plus Carboplatin-paclitaxel in Patients with Recurrent or Primary Advanced Endometrial Cancer (RUBY) , to provide the pre-specified interim and final overall survival (OS) analyses."~"Ongoing"~~6/30/2029 0:00:00~6/18/2026 0:00:00
385469~"CDER"~"BLA"~761179.00~"Jazz Pharmaceuticals Ireland Limited"~"JZP-458"~6/30/2021 0:00:00~"Original"~1~"4112-2"~"PMC"~"506B PMC"~4112.00~2~"PMC 4112-2: Conduct an assessment of the binding and neutralizing anti-drug antibody (ADA) responses in all JZP-458 treated patients from Study JZP458-201 with a validated assay (requested in PMC 4112-3 and PMC 4112-4) capable of sensitively detecting ADA responses in the presence of JZP458 levels that are expected to be present in the serum at the time of patient sampling. Include information on the level of JZP-458 in each patient s test sample at each sampling point and an assessment of the effects of binding and neutralizing ADA on clinical hypersensitivity reactions in the final report. In addition, assess the effects of binding and neutralizing ADA on JZP-458 exposure (serum asparaginase activity and serum asparaginase content) in the final report."~"Submitted"~~12/31/2022 0:00:00~10/30/2025 0:00:00
385470~"CDER"~"BLA"~761179.00~"Jazz Pharmaceuticals Ireland Limited"~"JZP-458"~6/30/2021 0:00:00~"Original"~1~"4112-5"~"PMC"~"506B PMC"~4112.00~5~"PMC 4112-5: Conduct a study examining quantitative recovery of anti-JZP458 antibodies from serum samples pretreated with Melon Gel Spin Plate Kit if immunogenicity data from an assay that uses the Melon Gel Spin Plate kit are used to support the license."~"Submitted"~~2/28/2022 0:00:00~10/30/2025 0:00:00
385471~"CDER"~"BLA"~761180.00~"LEO Pharma A/S"~"Adbry (Tralokinumab-ldrm)"~12/27/2021 0:00:00~"Original"~1~"4015-2"~"PMR"~"Pediatric Research Equity Act"~4015.00~2~"PMR 4015-2: Study LP0162-1335: A pharmacokinetic (PK) and safety [randomized, single (observer) blinded, parallel-group, monotherapy] dose-ranging study in pediatric subjects 2 to <12 years of age with moderate-to-severe atopic dermatitis (AD) who are candidates for systemic AD treatment (studied sequentially in 2 cohorts: 6 to <12 years and 2 to <6 years)."~"Ongoing"~"Original Final Report Due Date: 03/31/2026; Deferral Extension granted per FDA letter dated 03/03/2025. The study completion milestone was also revised.
"~11/30/2026 0:00:00~2/24/2025 0:00:00
385472~"CDER"~"BLA"~761180.00~"LEO Pharma A/S"~"Adbry (Tralokinumab-ldrm)"~12/27/2021 0:00:00~"Original"~1~"4015-3"~"PMR"~"Pediatric Research Equity Act"~4015.00~3~"PMR 4015-3: Study LP0162-1336: An efficacy and safety (phase 3, randomized, double-blind, placebo-controlled, parallel-group) study with
tralokinumab and placebo in combination with topical corticosteroid (TCS) therapy in pediatric subjects 2 to <12 years of age with moderate-to-severe atopic dermatitis (AD) who are candidates for systemic AD treatment (studied simultaneously in 2 cohorts: 6 to <12 years and 2 to <6 years)."~"Delayed"~"The final protocol date was missed because there was a delay in
FDA comments going to the applicant. Original Final Report Due Date: 09/30/2027; Deferral Extension denied per FDA letter dated 03/03/2025.
"~9/30/2027 0:00:00~2/24/2025 0:00:00
385473~"CDER"~"BLA"~761180.00~"LEO Pharma A/S"~"Adbry (Tralokinumab-ldrm)"~12/27/2021 0:00:00~"Original"~1~"4015-4"~"PMR"~"Pediatric Research Equity Act"~4015.00~4~"PMR 4015-4: Study LP0162-1381: An efficacy, safety, and pharmacokinetic (PK) (phase 2, single-arm, open-label, monotherapy) study in infants and pediatric subjects 6 months to <2 years of age with moderate-to severe atopic dermatitis (AD) who are candidates for systemic AD treatment."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2029 0:00:00~2/24/2025 0:00:00
385474~"CDER"~"BLA"~761180.00~"LEO Pharma A/S"~"Adbry (Tralokinumab-ldrm)"~12/27/2021 0:00:00~"Original"~1~"4015-5"~"PMR"~"505 (o)(3)"~4015.00~5~"PMR 4015-5: A prospective, pregnancy exposure registry based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to tralokinumab during pregnancy to an unexposed control population."~"Ongoing"~~9/30/2035 0:00:00~2/24/2025 0:00:00
385475~"CDER"~"NDA"~218550.00~"GENENTECH INC"~"Rozlytrek (Entrectinib)"~10/20/2023 0:00:00~"Original"~1~"4532-2"~"PMR"~"Accelerated Approval"~4532.00~2~"PMR 4532-2: Conduct an integrated analysis from ongoing trials intended to verify and describe the clinical benefit of entrectinib, through more precise estimation of the overall response rate and mature response duration per independent review assessment, in a sufficient number of pediatric patients older than 1 month of age and less than 12 years of age with solid tumors with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion and without a known acquired resistance mutation; that are metastatic or would require surgical resection that would result in severe morbidity; and have no satisfactory alternative treatment or have progressed following treatment. Overall response rate and duration of response will be assessed by independent central review and all responding patients will be followed for at least 12 months from the onset of response."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~3/31/2027 0:00:00~12/11/2025 0:00:00
385476~"CDER"~"NDA"~218571.00~"MILESTONE PHARMACEUTICALS USA INC"~"Cardamyst (etripamil) nasal spray"~12/12/2025 0:00:00~"Original"~1~"4914-1"~"PMR"~"Pediatric Research Equity Act"~4914.00~1~"PMR 4914-1: Conduct a study to evaluate safety, pharmacokinetics and pharmacodynamics of etripamil nasal spray in pediatric patients 1 to < 18 years old with paroxysmal supraventricular tachycardia (PSVT)."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2031 0:00:00~
385477~"CDER"~"NDA"~218590.00~"KNOA PHARMA LLC"~"Zurnai (nalmefene hydrochloride)"~8/7/2024 0:00:00~"Original"~1~"4665-1"~"PMR"~"Pediatric Research Equity Act"~4665.00~1~"PMR 4665-1: Conduct a clinical pharmacokinetic, pharmacodynamic, and safety study of Zurnai in pediatric patients aged birth to less than 12 years of age."~"Pending"~"The study has not begun but does not meet criteria for delayed."~4/30/2029 0:00:00~10/3/2025 0:00:00
385478~"CDER"~"NDA"~218590.00~"KNOA PHARMA LLC"~"Zurnai (nalmefene hydrochloride)"~8/7/2024 0:00:00~"Original"~1~"4665-2"~"PMR"~"Pediatric Research Equity Act"~4665.00~2~"PMR 4665-2: Conduct a juvenile animal study in rats to support the initiation of clinical studies in pediatric patients from 3 years to less than 12 years of age. This study will evaluate the effect of the drug on growth and development, specifically reproductive performance/sexual maturation and central nervous system histopathology and long-term behavioral effects."~"Submitted"~"The final report was submitted to FDA on 09/26/2025."~9/30/2025 0:00:00~10/3/2025 0:00:00
385479~"CDER"~"NDA"~218590.00~"KNOA PHARMA LLC"~"Zurnai (nalmefene hydrochloride)"~8/7/2024 0:00:00~"Original"~1~"4665-3"~"PMR"~"Pediatric Research Equity Act"~4665.00~3~"PMR 4665-3: Conduct a juvenile animal study in rats to support the initiation of clinical studies in pediatric patients from birth to less than 3 years of age. This study will evaluate the effect of the drug on development, specifically neuroapoptosis and central nervous system histopathology."~"Submitted"~"The final report was submitted to FDA on 09/26/2025."~9/30/2025 0:00:00~10/3/2025 0:00:00
385480~"CDER"~"NDA"~218614.00~"IONIS PHARMACEUTICALS INC"~"Tryngolza (olezarsen) injection"~12/19/2024 0:00:00~"Original"~1~"4760-1"~"PMR"~"505 (o)(3)"~4760.00~1~"PMR 4760-1: Complete the ongoing randomized, double-blind, placebo-controlled trials (ISIS 678354-CS5 [CORE] and ISIS 678354-CS6 [CORE 2]) and the two year open-label extension (OLE) trial (ISIS 678354-CS15) to evaluate the potential for drug induced liver injury with Tryngolza."~"Ongoing"~~6/30/2029 0:00:00~2/17/2026 0:00:00
385481~"CDER"~"NDA"~218637.00~"KAMAT PHARMATECH LLC"~"Raldesy (trazodone hydrochloride) oral solution"~11/26/2024 0:00:00~"Original"~1~"4725-1"~"PMR"~"Pediatric Research Equity Act"~4725.00~1~"PMR 4725-1: Conduct a juvenile animal study to assess the safety of Raldesy in animals of an age range and stage of development that are comparable to the population of children 7 to 12 years."~"Delayed"~"Final Protocol submission milestone was missed on 2/28/2025."~1/31/2027 0:00:00~1/23/2026 0:00:00
385482~"CDER"~"NDA"~218637.00~"KAMAT PHARMATECH LLC"~"Raldesy (trazodone hydrochloride) oral solution"~11/26/2024 0:00:00~"Original"~1~"4725-2"~"PMR"~"Pediatric Research Equity Act"~4725.00~2~"PMR 4725-2: Conduct a randomized, double-blind, placebo-controlled study to assess the safety, efficacy, and pharmacokinetics of Raldesy for the treatment of major depressive disorder in adolescents 13 through 17 years of age."~"Delayed"~"Final Protocol submission milestone was missed on 2/28/2025."~3/31/2028 0:00:00~1/23/2026 0:00:00
385483~"CDER"~"NDA"~218637.00~"KAMAT PHARMATECH LLC"~"Raldesy (trazodone hydrochloride) oral solution"~11/26/2024 0:00:00~"Original"~1~"4725-3"~"PMR"~"Pediatric Research Equity Act"~4725.00~3~"PMR 4725-3: Conduct a randomized, double-blind, placebo-controlled study to assess the safety, efficacy and pharmacokinetics of Raldesy for the treatment of major depressive disorder in a mixed child and adolescent population 7 through 17 years of age."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2031 0:00:00~1/23/2026 0:00:00
385484~"CDER"~"NDA"~218643.00~"THEA PHARMA INC"~"Cyklx (articaine hydrochloride) ophthalmic solution, 8%"~8/15/2025 0:00:00~"Original"~1~"4886-1"~"PMR"~"505 (o)(3)"~4886.00~1~"PMR 4886-1: Conduct a randomized, controlled trial to evaluate the corneal endothelial health of eyes treated with articaine ophthalmic solution, 8% by monitoring the number/density of corneal endothelial cells using specular microscopy at baseline and at 3 months in at least 100 patients receiving articaine ophthalmic solution, 8%."~"Pending"~~11/30/2028 0:00:00~
385485~"CDER"~"NDA"~218709.00~"X4 PHARMACEUTICALS INC"~"Xolremdi (mavorixafor)"~4/26/2024 0:00:00~"Original"~1~"4625-1"~"PMR"~"505 (o)(3)"~4625.00~1~"PMR 4625-1: Conduct a dedicated hepatic impairment clinical study to evaluate the effect of hepatic impairment on the pharmacokinetics of mavorixafor."~"Ongoing"~~9/30/2026 0:00:00~6/18/2026 0:00:00
385486~"CDER"~"NDA"~218709.00~"X4 PHARMACEUTICALS INC"~"Xolremdi (mavorixafor)"~4/26/2024 0:00:00~"Original"~1~"4625-2"~"PMR"~"505 (o)(3)"~4625.00~2~"PMR 4625-2: Conduct a long-term (2-year) GLP-compliant carcinogenicity study by oral gavage administration in rats or provide equivalent risk information to assess the carcinogenic potential from chronic exposure to mavorixafor. Consult the expanded evaluation process outlined in FDA guidance for industry S1B(R1) Addendum to S1B Testing for Carcinogenicity of Pharmaceuticals for recommendations on assessment strategies."~"Delayed"~"The Draft Protocol and Final Protocols were missed because your weight-of-evidence (WoE) assessment submitted on 02/28/2025 was deemed incomplete and the Agency requested a 6-month
toxicity study in rats with free base X4P-001 is to be conducted before a revised WoE with a comprehensive assessment can be submitted. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 11/05/2025."~12/31/2028 0:00:00~6/18/2026 0:00:00
385487~"CDER"~"NDA"~218709.00~"X4 PHARMACEUTICALS INC"~"Xolremdi (mavorixafor)"~4/26/2024 0:00:00~"Original"~1~"4625-3"~"PMR"~"505 (o)(3)"~4625.00~3~"PMR 4625-3: Conduct a 26-week GLP carcinogenicity study by oral gavage administration in Tg.RasH2 mice to assess the carcinogenic potential from chronic exposure to mavorixafor."~"Ongoing"~~11/30/2026 0:00:00~6/18/2026 0:00:00
385488~"CDER"~"NDA"~218709.00~"X4 PHARMACEUTICALS INC"~"Xolremdi (mavorixafor)"~4/26/2024 0:00:00~"Original"~1~"4625-4"~"PMC"~"506B PMC"~4625.00~4~"PMC 4625-4: Conduct a clinical drug interaction study to evaluate the effect of a moderate CYP3A4 inducer on the pharmacokinetics of mavorixafor."~"Ongoing"~~4/30/2026 0:00:00~6/18/2026 0:00:00
385489~"CDER"~"NDA"~218710.00~"PHATHOM PHARMACEUTICALS INC"~"Voquezna (vonoprazan)"~7/17/2024 0:00:00~"Original"~1~"4580-1"~"PMR"~"Pediatric Research Equity Act"~4580.00~1~"PMR 4580-1: Conduct a randomized, blinded, and placebo-controlled study to evaluate the efficacy and safety of Voquezna (vonoprazan) for the relief of heartburn associated with non-erosive gastroesophageal reflux disease (GERD) in pediatric patients 12 months to less than 18 years of age."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~2/28/2031 0:00:00~
385490~"CDER"~"NDA"~220934.00~"ELI LILLY AND CO"~"Foundayo"~4/1/2026 0:00:00~"Original"~1~"4977-2"~"PMR"~"Pediatric Research Equity Act"~4977.00~2~"PMR 4977-2: Conduct an 84-week, randomized, double-blind, placebo-controlled, multicenter, parallel-arm study J4M-MC-PW03 to evaluate the pharmacokinetics, safety, and efficacy of orforglipron as an adjunct to lifestyle intervention to reduce excess body weight and maintain weight reduction long term in a cohort of pediatric patients aged 6 to less than 12 years with obesity."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~2/29/2032 0:00:00~
385491~"CDER"~"NDA"~220934.00~"ELI LILLY AND CO"~"Foundayo"~4/1/2026 0:00:00~"Original"~1~"4977-3"~"PMR"~"505 (o)(3)"~4977.00~3~"PMR 4977-3: Establish a disease-based registry for pediatric obesity focused on safety in pediatric patients who receive pharmacological treatment for weight reduction beginning at ages less than 12 years old. The registry will enroll pediatric patients aged 6 years and older receiving non-pharmacological or pharmacological treatment for weight reduction during childhood and adolescence. Conduct a registry-based cohort study of at least 10 years duration to describe growth and development (height, weight, and pubertal development by Tanner staging), adverse events (including fractures and malnutrition), and development of obesity-related comorbid conditions. The registry must provide an option to enroll some patients who have participated in clinical trials of weight reduction medications for obesity, including orforglipron."~"Pending"~~4/30/2039 0:00:00~
385492~"CDER"~"NDA"~220934.00~"ELI LILLY AND CO"~"Foundayo"~4/1/2026 0:00:00~"Original"~1~"4977-4"~"PMR"~"505 (o)(3)"~4977.00~4~"PMR 4977-4: Collect global data from a prospective pregnancy exposure registry, preferably a disease-based multi-product pregnancy registry, using a registry-based cohort study to compare the maternal, fetal, and infant outcomes of women exposed to orforglipron for weight reduction during pregnancy with unexposed comparator population(s). Align the study protocol with protocol(s) outside the United States to reach the target sample size. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Pending"~~6/30/2038 0:00:00~
385493~"CDER"~"NDA"~220934.00~"ELI LILLY AND CO"~"Foundayo"~4/1/2026 0:00:00~"Original"~1~"4977-5"~"PMR"~"505 (o)(3)"~4977.00~5~"PMR 4977-5: Conduct an additional pregnancy study that uses a different design from the pregnancy exposure registry (for example a cohort study using claims or electronic medical record data) to compare the risks and prevalence of pregnancy and infant outcomes (including but not limited to major congenital malformations, spontaneous abortions, stillbirths, small-for-gestational-age births, preterm births, and postnatal growth and development) between women exposed to orforglipron for weight reduction during pregnancy and unexposed comparator population(s)."~"Pending"~~9/30/2035 0:00:00~
385494~"CDER"~"NDA"~220934.00~"ELI LILLY AND CO"~"Foundayo"~4/1/2026 0:00:00~"Original"~1~"4977-6"~"PMR"~"505 (o)(3)"~4977.00~6~"PMR 4977-6: Conduct a medullary thyroid carcinoma registry-based case series of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of orforglipron for the treatment of overweight and obesity into the marketplace. This study will also establish a registry of incident cases of medullary thyroid carcinoma and characterize their medical histories related to the use of orforglipron for the treatment of obesity/overweight."~"Pending"~~8/31/2043 0:00:00~
385495~"CDER"~"NDA"~220934.00~"ELI LILLY AND CO"~"Foundayo"~4/1/2026 0:00:00~"Original"~1~"4977-7"~"PMR"~"505 (o)(3)"~4977.00~7~"PMR 4977-7: Complete the ongoing randomized, open-label, active-controlled trial J2A-MC-GZGS (ACHIEVE-4) and provide additional safety data on orforglipron related to major adverse cardiovascular events (MACE) and the potential for drug-induced liver injury (DILI). Submit a clinical study report with adjudicated MACE (cardiovascular death, nonfatal myocardial infarction, hospitalization for unstable angina, and nonfatal stroke) and an assessment of potential DILI, including Hy s Law plots, shift tables for liver enzymes and bilirubin, and case reports in FDA-preferred DILI format for all cases meeting potential Hy s Law, having transaminase elevations >10x upper limit of normal (ULN), or having study drug discontinued due to DILI concerns."~"Ongoing"~~7/31/2026 0:00:00~
385496~"CDER"~"NDA"~220934.00~"ELI LILLY AND CO"~"Foundayo"~4/1/2026 0:00:00~"Original"~1~"4977-8"~"PMR"~"505 (o)(3)"~4977.00~8~"PMR 4977-8: Conduct a clinical pharmacology trial that uses ultrasound to measure the effect of both temporary withholding of orforglipron and fasting duration on retained gastric contents to evaluate delayed gastric emptying associated with glucagon-like peptide-1 receptor agonist (GLP-1 RA) use and inform potential recommendations to mitigate the serious risk of pulmonary aspiration."~"Pending"~~3/31/2029 0:00:00~
385497~"CDER"~"NDA"~220934.00~"ELI LILLY AND CO"~"Foundayo"~4/1/2026 0:00:00~"Original"~1~"4977-9"~"PMR"~"505 (o)(3)"~4977.00~9~"PMR 4977-9: Conduct a milk-only lactation study in lactating women who have received a dose of orforglipron to assess concentrations of orforglipron in breast milk using a validated assay."~"Pending"~~6/30/2029 0:00:00~
385498~"CDER"~"BLA"~761024.00~"Amgen Inc."~"Amjevita (adalimumab-atto)"~9/23/2016 0:00:00~"Original"~1~"3125-3"~"PMR"~"Pediatric Research Equity Act"~3125.00~3~"PMR 3125-3: Assessment of Amjevita (adalimumab-atto) for the treatment of pediatric ulcerative colitis in pediatric patients 5 years to 17 years of age."~"Delayed"~"The study/trial has been delayed. The original Final Report Due Date was September 2020. A Deferral Extension was granted until 2/24/2028 per 9/8/2021 FDA letter."~2/28/2028 0:00:00~11/13/2025 0:00:00
385499~"CDER"~"BLA"~761027.00~"Accord BioPharma Inc."~"Filkri (filgrastim-laha) injection"~1/15/2026 0:00:00~"Original"~1~"4956-1"~"PMR"~"Pediatric Research Equity Act"~4956.00~1~"PMR 4956-1: Develop a presentation that can be used to accurately administer Filkri (filgrastim-laha) to pediatric patients who weigh less than 36 kg."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2026 0:00:00~
385512~"CDER"~"NDA"~217900.00~"SUN PHARMACEUTICAL INDUSTRIES INC"~"Leqselvi (deuruxolitinib phosphate)"~7/25/2024 0:00:00~"Original"~1~"4655-6"~"PMR"~"505 (o)(3)"~4655.00~6~"PMR 4655-6: Conduct a PK study in subjects who are CYP2C9 Normal Metabolizers (NM), Intermediate Metabolizers (IM), and Poor Metabolizers (PM) in order to characterize the systemic exposure of deuruxolitinib in these subgroups."~"Ongoing"~~10/31/2026 0:00:00~9/22/2025 0:00:00
385513~"CDER"~"NDA"~217900.00~"SUN PHARMACEUTICAL INDUSTRIES INC"~"Leqselvi (deuruxolitinib phosphate)"~7/25/2024 0:00:00~"Original"~1~"4655-7"~"PMC"~"506B PMC"~4655.00~7~"PMC 4655-7: Establish an in-vitro diagnostic device to guide the use of deuruxolitinib in patients with severe alopecia areata. The device should detect, at a minimum, the presence of the nonfunctional alleles in cytochrome P450 2C9 (CYP2C9) relevant to the US population."~"Pending"~~3/31/2031 0:00:00~9/22/2025 0:00:00
385514~"CDER"~"BLA"~761180.00~"LEO Pharma A/S"~"Adbry (Tralokinumab-ldrm)"~12/27/2021 0:00:00~"Original"~1~"4015-6"~"PMR"~"505 (o)(3)"~4015.00~6~"PMR 4015-6: An additional pregnancy study that uses a different design from the Pregnancy Registry (for example a retrospective cohort study using claims or electronic medical record data with outcome validation or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age and preterm birth in women exposed to tralokinumab during pregnancy compared to an unexposed control population."~"Ongoing"~~12/30/2030 0:00:00~2/24/2025 0:00:00
385515~"CDER"~"BLA"~761181.00~"Regeneron Pharmaceuticals, Inc."~"Evkeeza (evinacumab-dgnb)"~2/11/2021 0:00:00~"Original"~1~"4019-1"~"PMR"~"505 (o)(3)"~4019.00~1~"PMR 4019-1: Conduct a worldwide study that collects prospective and retrospective data in women exposed to Evkeeza (evinacumab) during pregnancy to assess the risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The study will collect information for a minimum of 10 years. Results will be analyzed and reported descriptively. Data collected retrospectively will be analyzed separately and reported with the interim and final study reports."~"Ongoing"~~8/31/2033 0:00:00~4/10/2026 0:00:00
385516~"CDER"~"BLA"~761183.00~"Provention Bio, Inc."~"Tzield (teplizumab-mzwv)"~11/17/2022 0:00:00~"Original"~1~"4359-1"~"PMR"~"Pediatric Research Equity Act"~4359.00~1~"PMR 4359-1: Conduct a 12-month single-arm, open-label study to assess the safety and pharmacokinetics (PK) of teplizumab-mzwv in pediatric patients 0 to less than 8 years of age with two type-1 diabetes (T1D)-related autoantibodies and dysglycemia (Stage 2 T1D) [Part A], followed by a 12-month open-label extension [Part B]."~"Released"~"Per FDA letter dated 04/20/2026, this PMR/PMC has been released."~4/30/2026 0:00:00~1/15/2026 0:00:00
385517~"CDER"~"BLA"~761183.00~"Provention Bio, Inc."~"Tzield (teplizumab-mzwv)"~11/17/2022 0:00:00~"Original"~1~"4359-2"~"PMR"~"505 (o)(3)"~4359.00~2~"PMR 4359-2: Conduct an observational registry study to assess the long-term safety of teplizumab-mzwv in patients with Stage 2 type 1 diabetes. The study should evaluate cytokine release syndrome, serious infections, hypersensitivity reactions, lymphoproliferative disorders and malignancy. The registry should also collect information on women exposed during pregnancy to assess for adverse events related to pregnancy through the first year postpartum, and birth and developmental outcomes through the infant s first year of life. The study design should include a comparator group and monitor patients for at least 10 years after their first course of treatment. The study should enroll at least 150 subjects exposed to teplizumab-mzwv and collect sufficient clinical information to assess for sources of confounding for the target outcomes."~"Delayed"~"The Final Protocol Submission milestone was missed because of continued protocol negotiations. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 01/11/2024. The study has not been initiated."~9/30/2037 0:00:00~1/15/2026 0:00:00
385518~"CDER"~"BLA"~761183.00~"Provention Bio, Inc."~"Tzield (teplizumab-mzwv)"~11/17/2022 0:00:00~"Original"~1~"4359-3"~"PMR"~"505 (o)(3)"~4359.00~3~"PMR 4359-3: Provide comparative safety data on the commercial formulation manufactured by [ ] versus the TN-10 clinical trial product by submitting the clinical study reports for study PRV-031-001 (PROTECT), and study PRV-031-003 (PROTECT Extension) that seeks to collect an additional 42 months of long-term safety data in participants who complete the PROTECT study."~"Ongoing"~~5/31/2027 0:00:00~1/15/2026 0:00:00
385519~"CDER"~"BLA"~761183.00~"Provention Bio, Inc."~"Tzield (teplizumab-mzwv)"~11/17/2022 0:00:00~"Original"~1~"4359-9"~"PMR"~"505 (o)(3)"~4359.00~9~"PMR 4359-9: Complete the 12-month single-arm, open-label extension study PRV-031-005/SFY18116 to assess the long-term safety of teplizumab-mzwv in pediatric patients 0 to less than 8 years of age with at least two type-1 diabetes (T1D)-related autoantibodies and dysglycemia (Stage 2 T1D)."~"Ongoing"~~4/30/2027 0:00:00~1/15/2026 0:00:00
385520~"CDER"~"BLA"~761194.00~"Genzyme Corporation"~"Nexviazyme (avalglucosidase alfa-ngpt)"~8/6/2021 0:00:00~"Original"~1~"4026-1"~"PMR"~"505 (o)(3)"~4026.00~1~"PMR 4026-1: Conduct a worldwide,  descriptive safety study that collects data in women and their  offspring who are exposed  to Nexviazyme (avalglucosidase  alfa-ngpt) during  pregnancy and/or  lactation  to assess  risk of pregnancy and maternal  complications,  adverse effects on the developing  fetus and neonate,  and adverse effects on the  infant. Outcomes of exposed  infants, including  growth  and development,  will be assessed  through  at least  the first year of life. The study will collect information for 10 years."~"Ongoing"~~4/30/2033 0:00:00~9/30/2025 0:00:00
385521~"CDER"~"BLA"~761195.00~"Argenx BV"~"Vyvgart (Efgartigimod alfa)"~12/17/2021 0:00:00~"Original"~1~"4202-1"~"PMR"~"505 (o)(3)"~4202.00~1~"PMR 4202-1: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Vyvgart (efgartigimod) during pregnancy and/or lactation to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Ongoing"~~12/31/2033 0:00:00~2/13/2026 0:00:00
385522~"CDER"~"BLA"~761196.00~"ADC Therapeutics SA"~"Loncastuximab Tesirine"~4/23/2021 0:00:00~"Original"~1~"4029-1"~"PMR"~"Accelerated Approval"~4029.00~1~"PMR 4029-1: Conduct a randomized, phase 3 clinical trial to verify and describe the clinical benefit of loncastuximab tesirine-lpyl in patients with relapsed or refractory large B-cell lymphoma. The trial should include sufficient numbers of racial and ethnic minority patients to better reflect the U.S. patient population and allow for interpretation of the results in these patient populations. Patients should be randomized to receive loncastuximab tesirine-lpyl plus immunotherapy or immunochemotherapy. The primary endpoint should be progression-free survival, with secondary endpoints that include overall survival and objective response rate. 
"~"Delayed"~"The applicant requested revised milestones because of the delay in achievement of the required number of PFS events due to increased enrollment. Revised milestones were acknowledged in a letter dated 08/01/2025."~12/31/2025 0:00:00~6/20/2025 0:00:00
385523~"CDER"~"BLA"~761196.00~"ADC Therapeutics SA"~"Loncastuximab Tesirine"~4/23/2021 0:00:00~"Original"~1~"4029-2"~"PMR"~"Pediatric Research Equity Act"~4029.00~2~"PMR 4029-2: Conduct a study assessing the efficacy and safety of loncastuximab tesirine-lpyl in pediatric patients 1 year or older with relapsed or refractory non-Hodgkin lymphoma. The safety endpoints should determine safety and tolerability of loncastuximab tesirine-lpyl and identify the recommended dose and schedule. The efficacy endpoints should determine activity as assessed by response rate and durability of response."~"Delayed"~"The final protocol submission milestone was missed, because of delays due to the necessary change in study design. Original Final Report Due Date: 07/2028; Deferral Extension granted per FDA letter dated 09/09/2024.
"~8/31/2030 0:00:00~6/20/2025 0:00:00
385524~"CDER"~"NDA"~218730.00~"VERTEX PHARMACEUTICALS INC"~"Alyftrek (vanzacaftor, tezacaftor, and deutivacaftor) Tablets"~12/20/2024 0:00:00~"Original"~1~"4734-1"~"PMR"~"505 (o)(3)"~4734.00~1~"PMR 4734-1: Conduct a 2 year carcinogenicity study in rats administered vanzacaftor."~"Submitted"~~4/30/2025 0:00:00~2/13/2026 0:00:00
385525~"CDER"~"NDA"~218730.00~"VERTEX PHARMACEUTICALS INC"~"Alyftrek (vanzacaftor, tezacaftor, and deutivacaftor) Tablets"~12/20/2024 0:00:00~"Original"~1~"4734-2"~"PMR"~"505 (o)(3)"~4734.00~2~"PMR 4734-2: Conduct a drug-drug interaction (DDI) clinical trial to assess the effect of vanzacaftor/tezacaftor/deutivacaftor on the pharmacokinetics of a sensitive breast cancer resistance protein (BCRP) substrate."~"Ongoing"~~11/30/2026 0:00:00~2/13/2026 0:00:00
385526~"CDER"~"NDA"~218764.00~"BOEHRINGER INGELHEIM PHARMACEUTICALS INC"~"Jascayd (nerandomilast) Film-Coated Tablets"~10/7/2025 0:00:00~"Original"~1~"4912-1"~"PMC"~"506B PMC"~4912.00~1~"PMC 4912-1: Conduct a drug-drug interaction (DDI) clinical trial to assess the effect of pirfenidone on the pharmacokinetics of nerandomilast."~"Pending"~~6/30/2027 0:00:00~
385527~"CDER"~"NDA"~218764.00~"BOEHRINGER INGELHEIM PHARMACEUTICALS INC"~"Jascayd (nerandomilast) Film-Coated Tablets"~10/7/2025 0:00:00~"Original"~1~"4912-2"~"PMC"~"506B PMC"~4912.00~2~"PMC 4912-2: Conduct DDI clinical trials to assess the magnitude of change in nerandolimast exposure with repeat doses of a moderate CYP3A inducer
and a strong CYP3A inducer to determine appropriate dosing recommendations with CYP3A inducers. Conduct the trials in accordance
with the FDA Guidance for Industry entitled  M12 Drug Interaction Studies. "~"Submitted"~~4/30/2026 0:00:00~
385528~"CDER"~"NDA"~218784.00~"SERVIER PHARMACEUTICALS LLC"~"Voranigo (vorasidenib)"~8/6/2024 0:00:00~"Original"~1~"4659-1"~"PMR"~"505 (o)(3)"~4659.00~1~"PMR 4659-1: Conduct a carcinogenicity study in mice to evaluate the potential serious risk of carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Pending"~~4/30/2027 0:00:00~10/1/2025 0:00:00
385529~"CDER"~"NDA"~218784.00~"SERVIER PHARMACEUTICALS LLC"~"Voranigo (vorasidenib)"~8/6/2024 0:00:00~"Original"~1~"4659-2"~"PMR"~"505 (o)(3)"~4659.00~2~"PMR 4659-2: Conduct a carcinogenicity study in rats to evaluate the potential serious risk of carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Pending"~~12/31/2028 0:00:00~10/1/2025 0:00:00
385530~"CDER"~"NDA"~218784.00~"SERVIER PHARMACEUTICALS LLC"~"Voranigo (vorasidenib)"~8/6/2024 0:00:00~"Original"~1~"4659-3"~"PMR"~"505 (o)(3)"~4659.00~3~"PMR 4659-3: Conduct a comprehensive safety analysis from clinical trials in a sufficient number of pediatric patients to adequately characterize baseline risk factors and safety outcomes, including hepatotoxicity and effects on growth and development, following exposure to vorasidenib. Include assessment of clinical responses."~"Pending"~~12/31/2033 0:00:00~10/1/2025 0:00:00
385531~"CDER"~"NDA"~218784.00~"SERVIER PHARMACEUTICALS LLC"~"Voranigo (vorasidenib)"~8/6/2024 0:00:00~"Original"~1~"4659-4"~"PMR"~"505 (o)(3)"~4659.00~4~"PMR 4659-4: Conduct a comprehensive safety analysis from clinical trials in a sufficient number of adult patients to adequately characterize hepatotoxicity following exposure to vorasidenib. The integrated safety analysis should include all adverse events, major safety events, dose-reductions, dose interruptions, and withdrawals, when all patients have completed at least two years of treatment with vorasidenib or withdrew earlier."~"Pending"~~7/31/2026 0:00:00~10/1/2025 0:00:00
385532~"CDER"~"NDA"~218784.00~"SERVIER PHARMACEUTICALS LLC"~"Voranigo (vorasidenib)"~8/6/2024 0:00:00~"Original"~1~"4659-5"~"PMR"~"505 (o)(3)"~4659.00~5~"PMR 4659-5: Conduct a clinical pharmacokinetic trial to determine an appropriate dosage of vorasidenib to minimize toxicity in patients with severe hepatic impairment. Design and conduct the trial in accordance with the FDA Draft Guidance for Industry titled:  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~4/30/2027 0:00:00~10/1/2025 0:00:00
385533~"CDER"~"NDA"~218784.00~"SERVIER PHARMACEUTICALS LLC"~"Voranigo (vorasidenib)"~8/6/2024 0:00:00~"Original"~1~"4659-6"~"PMC"~"506B PMC"~4659.00~6~"PMC 4659-6: Conduct a clinical study to assess the effect of vorasidenib on the pharmacokinetics of a CYP3A substrate to determine recommendations on appropriate dose adjustments when vorasidenib is administered concomitantly with CYP3A substrates. Design and conduct the assessment in accordance with the FDA Guidance for Industry titled  Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions ."~"Pending"~~12/31/2026 0:00:00~10/1/2025 0:00:00
385534~"CDER"~"NDA"~218784.00~"SERVIER PHARMACEUTICALS LLC"~"Voranigo (vorasidenib)"~8/6/2024 0:00:00~"Original"~1~"4659-7"~"PMC"~"506B PMC"~4659.00~7~"PMC 4659-7: Complete survival follow-up of patients in the INDIGO trial to further characterize the efficacy and clinical benefit of vorasidenib in patients with Grade 2 astrocytoma or oligodendroglioma with susceptible IDH1 or IDH2 mutations."~"Ongoing"~~11/30/2028 0:00:00~10/1/2025 0:00:00
385535~"CDER"~"NDA"~218785.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~6/10/2025 0:00:00~"Original"~2~"4855-1"~"PMR"~"Accelerated Approval"~4855.00~1~"PMR 4855-1: Complete and submit the final results of Trial BGB-3111-306, the ongoing randomized clinical trial of zanubrutinib in combination with rituximab versus bendamustine and rituximab, intended to verify and describe the clinical benefit of zanubrutinib in patients with previously untreated mantle cell lymphoma. The primary endpoint is progression-free survival (PFS) as assessed by Independent Review Committee (IRC). Overall Survival (OS) is a key secondary endpoint. PFS and OS would be analyzed based on superiority testing. Enrollment of approximately 500 patients is expected."~"Ongoing"~"The trial has completed enrollment."~2/28/2027 0:00:00~1/12/2026 0:00:00
385536~"CDER"~"NDA"~218785.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~6/10/2025 0:00:00~"Original"~2~"4855-2"~"PMR"~"Accelerated Approval"~4855.00~2~"PMR 4855-2: Complete a randomized clinical trial intended to verify and describe the clinical benefit of zanubrutinib in patients with relapsed or refractory marginal zone lymphoma. The trial should include sufficient representation of racial and ethnic populations to better reflect the U.S. patient population and allow for interpretation of the results in these patient populations. The primary endpoint should be progression-free survival, with secondary endpoints that include objective response rate and overall survival."~"Ongoing"~"The trial has completed enrollment."~10/31/2028 0:00:00~1/12/2026 0:00:00
385537~"CDER"~"NDA"~218785.00~"BEONE MEDICINES USA INC"~"Brukinsa (zanubrutinib)"~6/10/2025 0:00:00~"Original"~2~"4855-3"~"PMR"~"Accelerated Approval"~4855.00~3~"PMR 4855-3: Complete a randomized clinical trial of zanubrutinib plus obinutuzumab versus lenalidomide plus rituximab, intended to verify and describe the clinical benefit of zanubrutinib in patients with relapsed or refractory follicular lymphoma. The primary endpoint should be progression-free survival, with secondary endpoints that include objective response rate and overall survival. The trial should enroll a sufficiently representative study population that reflects the racial and ethnic populations of the U.S. population of patients with follicular lymphoma and that allows for interpretation of the results in these populations."~"Ongoing"~"The trial has completed enrollment."~1/31/2029 0:00:00~1/12/2026 0:00:00
385538~"CDER"~"NDA"~218808.00~"NEUROCRINE BIOSCIENCES INC"~"Crenessity (crinecerfont) Oral Capsules"~12/13/2024 0:00:00~"Original"~1~"4738-1"~"PMR"~"505 (o)(3)"~4738.00~1~"PMR 4738-1: Perform a lactation study (milk only or mother-infant pair study) in lactating women who have received Crenessity to measure concentrations of crinecerfont and its major metabolites in breast milk using a validated assay and to assess the effects on the breastfed infant, if a mother-infant pair study is conducted."~"Pending"~~6/30/2028 0:00:00~2/11/2026 0:00:00
385539~"CDER"~"BLA"~761032.00~"Bausch Health Ireland, Limited"~"Siliq (Brodalumab)"~2/15/2017 0:00:00~"Original"~1~"3164-3"~"PMR"~"Pediatric Research Equity Act"~3164.00~3~"PMR 3164-3: Open label, single arm study with brodalumab to determine safety and efficacy in children (6 to <12) with severe plaque psoriasis."~"Delayed"~"The final protocol submission date was missed and will be submitted based upon revised timelines."~6/30/2029 0:00:00~4/15/2026 0:00:00
385540~"CDER"~"BLA"~761032.00~"Bausch Health Ireland, Limited"~"Siliq (Brodalumab)"~2/15/2017 0:00:00~"Original"~1~"3164-4"~"PMR"~"505 (o)(3)"~3164.00~4~"PMR 3164-4: Conduct a retrospective cohort study using administrative databases to identify pregnancy outcomes in a cohort of women with a diagnosis of psoriasis exposed to brodalumab versus a non-brodalumab systemic medication exposure cohort.  The outcomes will include major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age births. This study may use multiple data sources in order to obtain a sufficient sample size as women with psoriasis are counseled to avoid systemic treatments while trying to conceive and during the course of pregnancy."~"Delayed"~"The final protocol submission, study completion, and final report submission dates have been missed because the applicant is awaiting feedback on their protocol."~6/30/2023 0:00:00~4/15/2026 0:00:00
385541~"CDER"~"BLA"~761032.00~"Bausch Health Ireland, Limited"~"Siliq (Brodalumab)"~2/15/2017 0:00:00~"Original"~1~"3164-5"~"PMR"~"505 (o)(3)"~3164.00~5~"PMR 3164-5: Conduct a prospective, registry-based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women with the diagnosis of psoriasis exposed to brodalumab during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital  malformations, spontaneous abortions, stillbirths, elective terminations, small for gestational age births, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The final protocol submission date was missed because the applicant is still awaiting FDA feedback on their protocol."~6/30/2031 0:00:00~4/15/2026 0:00:00
385542~"CDER"~"BLA"~761032.00~"Bausch Health Ireland, Limited"~"Siliq (Brodalumab)"~2/15/2017 0:00:00~"Original"~1~"3164-6"~"PMR"~"505 (o)(3)"~3164.00~6~"PMR 3164-6: Conduct a prospective, observational study to assess the long-term safety of Siliq (brodalumab) compared to other therapies used in the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy in the course of actual clinical care. The study s primary outcome is malignancy. Secondary outcomes include, but are not limited to, opportunistic infections (e.g., tuberculosis [TB], opportunistic mycoses) and neutropenia. Describe and justify the choice of appropriate comparator population(s) and estimated background rate(s) relative to brodalumab-exposed patients; clearly define the primary comparator population for the primary objective. Design the study around a testable hypothesis to assess, with sufficient sample size and  power, a clinically meaningful increase in malignancy risk above the comparator background rate(s), with a prespecified statistical analysis method. Specify concise case definitions and validation algorithms for both primary and secondary outcomes. For the brodalumab-exposed and comparator(s) cohorts, clearly define the study drug initiation period, including any exclusion and inclusion criteria. Enroll patients over an initial 4 year period and follow for a minimum of 8 years from the time of enrollment."~"Delayed"~"The final protocol submission date was missed because the applicant is still awaiting FDA feedback on their protocol."~11/30/2031 0:00:00~4/15/2026 0:00:00
385543~"CDER"~"BLA"~761034.00~"Genentech, Inc."~"Tecentriq (Atezolizumab) "~5/18/2016 0:00:00~"Supplement"~28~"3908-1"~"PMC"~"506B PMC"~3908.00~1~"PMC 3908-1: Submit the final overall survival analysis and datasets with the final report from Trial CO39262 (IMspire150):  A phase III, double-blinded, randomized, placebo-controlled study of atezolizumab plus cobimetinib and vemurafenib versus placebo plus cobimetinib and vemurafenib in previously untreated BRAF V600 mutation-positive patients with unresectable locally advanced or metastatic melanoma  to further characterize the clinical benefit of atezolizumab in combination with cobimetinib and vemurafenib. The results from this report may inform product labeling."~"Submitted"~~7/31/2027 0:00:00~7/10/2026 0:00:00
385544~"CDER"~"BLA"~761036.00~"Janssen Biotech, Inc."~"Darzalex (daratumumab)"~11/16/2015 0:00:00~"Supplement"~35~"4127-1"~"PMR"~"505 (o)(3)"~4127.00~1~"PMR 4127-1: Conduct a prospective, observational single-arm study to assess the risk of severe (Grades 3-4) and fatal infusion-related reactions (IRRs) in patients treated with intravenous (IV) or sub-cutaneous (SC) daratumumab. Evaluate the incidence of severe and fatal IRRs, and collect information, including a full description of clinical features of the adverse reactions, to investigate associations and temporal relationships between the incidence and severity of IRRs and other potential associated risk factors. Specify case definitions, validation methods, and procedures for all study outcomes. Submit interim reports of the data collected from the study annually until the study is completed."~"Ongoing"~~7/31/2026 0:00:00~1/9/2026 0:00:00
385545~"CDER"~"BLA"~761037.00~"Sanofi-Aventis U.S. LLC"~"Kevzara (Sarilumab)"~5/22/2017 0:00:00~"Original"~1~"3218-3"~"PMR"~"Pediatric Research Equity Act"~3218.00~3~"PMR 3218-3: A study to assess the pharmacokinetics and pharmacodynamics (PK/PD), immunogenicity, dosing, safety, and clinical outcomes of sarilumab in children ages =2 years to 17 years with polyarticular juvenile idiopathic arthritis (pJIA) (study DRI13925)."~"Delayed"~"Applicant submitted final report with supplement 17. A complete response letter was sent to the applicant on June 10, 2024."~8/31/2023 0:00:00~7/18/2025 0:00:00
385546~"CDER"~"BLA"~761037.00~"Sanofi-Aventis U.S. LLC"~"Kevzara (Sarilumab)"~5/22/2017 0:00:00~"Supplement"~13~"4413-1"~"PMR"~"505 (o)(3)"~4413.00~1~"PMR 4413-1: Conduct a randomized controlled clinical trial of at least one year duration in patients with polymyalgia rheumatica to evaluate safety outcomes, including infections, hepatotoxicity, elevated liver enzymes, neutropenia, thrombocytopenia, and elevated lipids, of the sarilumab 200 mg once every two weeks dosing regimen."~"Delayed"~"The final study protocol was not submitted by the original milestone date. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 02/24/2025."~3/31/2029 0:00:00~7/18/2025 0:00:00
385547~"CDER"~"BLA"~761037.00~"Sanofi-Aventis U.S. LLC"~"Kevzara (Sarilumab)"~5/22/2017 0:00:00~"Supplement"~13~"4413-2"~"PMC"~"506B PMC"~4413.00~2~"PMC 4413-2: Conduct a randomized controlled clinical trial of at least one year duration in patients with polymyalgia rheumatica to evaluate efficacy and safety of the sarilumab 150 mg once every two weeks dosing regimen."~"Delayed"~"The final study protocol was not submitted by the original milestone date. The applicant requested a revised milestone. A revised milestone was acknowledged in a letter dated 02-24-2025. 
"~3/31/2029 0:00:00~7/18/2025 0:00:00
385548~"CDER"~"NDA"~217901.00~"SATSUMA PHARMACEUTICALS INC"~"Atzumi (dihydroergotamine) nasal powder"~4/30/2025 0:00:00~"Original"~1~"4834-1"~"PMR"~"Pediatric Research Equity Act"~4834.00~1~"PMR 4834-1: An open-label, single-dose study to evaluate the safety, tolerability, and single-dose pharmacokinetics (PK) of dihydroergotamine in patients with migraine 6 through 11 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~10/31/2028 0:00:00~6/25/2026 0:00:00
385549~"CDER"~"NDA"~217901.00~"SATSUMA PHARMACEUTICALS INC"~"Atzumi (dihydroergotamine) nasal powder"~4/30/2025 0:00:00~"Original"~1~"4834-2"~"PMR"~"Pediatric Research Equity Act"~4834.00~2~"PMR 4834-2: A juvenile animal toxicology study of dihydroergotamine in one species."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~4/30/2028 0:00:00~6/25/2026 0:00:00
385550~"CDER"~"NDA"~217901.00~"SATSUMA PHARMACEUTICALS INC"~"Atzumi (dihydroergotamine) nasal powder"~4/30/2025 0:00:00~"Original"~1~"4834-3"~"PMR"~"Pediatric Research Equity Act"~4834.00~3~"PMR 4834-3: A randomized, double-blind, placebo-controlled efficacy and safety study for the treatment of acute migraine with or without aura in patients 6 through 17 years of age. Part A should be for patients 12 through 17 years of age and Part B for patients 6 through 11 years of age. This study includes an initial single-blind placebo lead-in to identify patients who are non-responders to placebo for enrollment into the efficacy portion of the trial. The efficacy study must be designed to show superiority of dihydroergotamine over placebo."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~10/31/2031 0:00:00~6/25/2026 0:00:00
385551~"CDER"~"NDA"~217901.00~"SATSUMA PHARMACEUTICALS INC"~"Atzumi (dihydroergotamine) nasal powder"~4/30/2025 0:00:00~"Original"~1~"4834-4"~"PMR"~"Pediatric Research Equity Act"~4834.00~4~"PMR 4834-4: A pediatric open-label safety study to evaluate the long-term safety of intermittent treatment with dihydroergotamine in patients 6 through 17 years of age, for up to one year. Part A should be for patients 12 through 17 years of age and Part B for patients 6 through 11 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~1/31/2033 0:00:00~6/25/2026 0:00:00
385552~"CDER"~"NDA"~217906.00~"ABBVIE INC"~"Emblaveo (aztreonam and avibactam) for Injection"~2/7/2025 0:00:00~"Original"~1~"4778-1"~"PMR"~"Pediatric Research Equity Act"~4778.00~1~"PMR 4778-1: Conduct a multiple-dose, open-label (with a blinded observer), comparator controlled study to evaluate the pharmacokinetics, safety, and tolerability of aztreonam-avibactam for injection in patients aged 9 months and older to less than 18 years who are hospitalized due to complicated intraabdominal infections (cIAI), complicated urinary tract infections (cUTI), hospital-acquired bacterial pneumonia (HABP), ventilator-associated bacterial pneumonia (VABP), or bloodstream infections (BSI) caused by gram-negative organisms (confirmed or suspected)."~"Delayed"~"The study completion date of 3-2026 was missed. Applicant
submitted deferral extension request on 12-22-2025. The deferral extension was granted on 1/23/2026. The study completion milestone was also revised. 45 subjects enrolled.
"~11/30/2027 0:00:00~4/1/2026 0:00:00
385553~"CDER"~"NDA"~217906.00~"ABBVIE INC"~"Emblaveo (aztreonam and avibactam) for Injection"~2/7/2025 0:00:00~"Original"~1~"4778-2"~"PMR"~"Pediatric Research Equity Act"~4778.00~2~"PMR 4778-2: Conduct a two-part, open-label study enrolling neonates and infants from birth to less than 9 months of age to evaluate the pharmacokinetics, safety, and tolerability of aztreonam-avibactam for injection."~"Ongoing"~"Current enrollment 24."~2/28/2028 0:00:00~4/1/2026 0:00:00
385554~"CDER"~"NDA"~217906.00~"ABBVIE INC"~"Emblaveo (aztreonam and avibactam) for Injection"~2/7/2025 0:00:00~"Original"~1~"4778-3"~"PMR"~"505 (o)(3)"~4778.00~3~"PMR 4778-3: Conduct a U.S. surveillance study over a five-year period after the introduction of aztreonam-avibactam to the market to determine if resistance or decreased susceptibility to aztreonam-avibactam is occurring in the target population of bacteria identified in the approved aztreonam-avibactam label."~"Ongoing"~~12/31/2030 0:00:00~4/1/2026 0:00:00
385555~"CDER"~"NDA"~217927.00~"SOLUBIOMIX LLC"~"Coxanto (oxaprozin)"~10/20/2023 0:00:00~"Original"~1~"4503-1"~"PMR"~"505 (o)(3)"~4503.00~1~"PMR 4503-1: Perform a milk-only lactation study in lactating women who have received oxaprozin to assess concentrations of oxaprozin and its active metabolites in breast milk using a validated assay, and to assess the effects on the breastfed infant."~"Delayed"~"The final protocol was missed because of continued discussion with the Agency. The final protocol was acknowledged per FDA letter dated September 18, 2025."~4/30/2027 0:00:00~12/17/2025 0:00:00
385556~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-1"~"PMR"~"Accelerated Approval"~4626.00~1~"PMR 4626-1: Conduct a multiregional, randomized clinical trial comparing tovorafenib to physician s choice of chemotherapy in pediatric patients with low grade glioma with RAF fusions or rearrangements, or V600 mutations, intended to verify and describe the clinical benefit of tovorafenib through assessment of overall response rate (ORR) as a primary endpoint and progression-free survival (PFS) and duration of response, as determined by blinded independent central review, as key secondary endpoints. Include the protocol-specified ORR and interim PFS analyses in the interim report."~"Ongoing"~"218 patients out of the planned 400 patients have been
enrolled into the trial."~4/30/2032 0:00:00~6/12/2026 0:00:00
385557~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-2"~"PMR"~"505 (o)(3)"~4626.00~2~"PMR 4626-2: Conduct a carcinogenicity study in mice to evaluate the potential serious risk of carcinogenicity."~"Fulfilled"~~8/31/2025 0:00:00~6/12/2026 0:00:00
385558~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-3"~"PMR"~"505 (o)(3)"~4626.00~3~"PMR 4626-3: Conduct a carcinogenicity study in rats to evaluate the potential serious risk of carcinogenicity. Submit a carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study."~"Ongoing"~~5/31/2028 0:00:00~6/12/2026 0:00:00
385559~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-4"~"PMR"~"505 (o)(3)"~4626.00~4~"PMR 4626-4: Conduct comprehensive safety analyses from clinical studies that further characterize the known serious risk of long-term adverse effects of tovorafenib on growth and development, including but not limited to growth plate abnormalities, in a sufficient number of pediatric patients. Monitor patients for growth and development using age-appropriate screening tools. Include evaluations of growth as measured by height, weight, height velocity and height standard deviation scores, age at adrenarche if applicable, age at menarche if applicable (females) and Tanner stage. Monitor patients until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first. Include, in the interim report, a comprehensive safety analysis after all patients have been monitored for a minimum of 12 months from the start of study treatment."~"Ongoing"~~4/30/2032 0:00:00~6/12/2026 0:00:00
385560~"CDER"~"BLA"~761196.00~"ADC Therapeutics SA"~"Loncastuximab Tesirine"~4/23/2021 0:00:00~"Original"~1~"4029-3"~"PMR"~"505 (o)(3)"~4029.00~3~"PMR 4029-3: Conduct a comprehensive analysis evaluating and characterizing the incidence, clinical presentation, management, and outcomes of the potential serious risks of loncastuximab tesirine-associated toxicities of edema, effusion, cutaneous reactions, and inflammatory-related conditions. Submit an integrated final report from patient-level and pooled analyses of ongoing and completed clinical trials, postmarketing reports and/or literature reports and a comprehensive pharmacovigilance assessment for these potential serious risks."~"Delayed"~"The Final Report milestone was missed because of the delay in reaching the required progression free survival (PFS) events of the study. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 05/15/2026."~3/31/2026 0:00:00~6/20/2025 0:00:00
385561~"CDER"~"BLA"~761196.00~"ADC Therapeutics SA"~"Loncastuximab Tesirine"~4/23/2021 0:00:00~"Original"~1~"4029-4"~"PMR"~"505 (o)(3)"~4029.00~4~"PMR 4029-4: Submit an integrated final report containing data from clinical trials to further characterize the exposure of loncastuximab tesirine-lpyl monotherapy and in combination with immunochemotherapy, the increased risk of severe and serious adverse events, including severe neutropenia, and efficacy among U.S. racial and ethnic minority patients with large B-cell lymphoma. Provide the population pharmacokinetic and exposure-response analyses for both efficacy and safety in the interim report."~"Delayed"~"The Final Report milestone were missed because of the delay in reaching the required PFS events of the study. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 05/15/2026."~3/31/2026 0:00:00~6/20/2025 0:00:00
385562~"CDER"~"BLA"~761196.00~"ADC Therapeutics SA"~"Loncastuximab Tesirine"~4/23/2021 0:00:00~"Original"~1~"4029-5"~"PMR"~"505 (o)(3)"~4029.00~5~"PMR 4029-5: Conduct an open-label, non-randomized, dose-escalation trial to determine a safe and appropriate dosing regimen of loncastuximab tesirine-lpyl in patients with moderate and severe hepatic impairmen according to the National Cancer Institute Organ Dysfunction Working Group criteria in the target patient population. Collect safety and pharmacokinetic information for loncastuximab tesirine-lpyl and SG3199 to determine the appropriate starting dose and dosing regimen of loncastuximab tesirine-lpyl for this population."~"Released"~~6/30/2027 0:00:00~6/20/2025 0:00:00
385563~"CDER"~"BLA"~761197.00~"Genentech, Inc."~"Susvimo (ranibizumab)"~10/22/2021 0:00:00~"Original"~1~"4157-2"~"PMR"~"505 (o)(3)"~4157.00~2~"PMR 4157-2: Conduct a controlled trial in which the health of the corneal endothelial cells are evaluated by monitoring the number/density of corneal endothelial cells over a period of at least one year in at least 100 patients receiving the 100 mg/mL ranibizumab administered through the Port Delivery System."~"Delayed"~"The study completion and final report milestones were missed because patient enrollment was paused due to a voluntary recall and FDA determined that the applicant demonstrated good cause for the delay and acknowledged the revised milestones in a letter dated 04/07/2025."~5/31/2025 0:00:00~12/16/2024 0:00:00
385564~"CDER"~"BLA"~761208.00~"Seagen Inc."~"Tivdak (tisotumab vedotin-tftv)"~9/20/2021 0:00:00~"Original"~1~"4131-2"~"PMR"~"Pediatric Research Equity Act"~4131.00~2~"PMR 4131-2: Conduct a pre-clinical study in pediatric-specific pre-clinical models with input from recognized key opinion leaders in pediatric oncology to support the conduct of clinical investigations of tisotumab vedotin in pediatric tumors."~"Fulfilled"~"Per FDA letter dated 11/12/2025, this PMR/PMC has been fulfilled."~6/30/2023 0:00:00~5/18/2026 0:00:00
385565~"CDER"~"BLA"~761208.00~"Seagen Inc."~"Tivdak (tisotumab vedotin-tftv)"~9/20/2021 0:00:00~"Original"~1~"4131-3"~"PMR"~"Pediatric Research Equity Act"~4131.00~3~"PMR 4131-3: Conduct a clinical investigation of the dose, tolerability, and preliminary evidence of activity of tisotumab vedotin in pediatric patients with cancer(s) in which tissue factor is a relevant therapeutic target."~"Released"~"Per FDA letter dated 11/12/2025, this PMR/PMC has been released."~12/31/2029 0:00:00~5/18/2026 0:00:00
385566~"CDER"~"BLA"~761208.00~"Seagen Inc."~"Tivdak (tisotumab vedotin-tftv)"~9/20/2021 0:00:00~"Supplement"~7~"4632-1"~"PMR"~"505 (o)(3)"~4632.00~1~"PMR 4632-1: Conduct a study of tisotumab vedotin, to include prospectively specified, scheduled ophthalmologic assessments in approximately 100 enrolled patients to further characterize the incidence and severity of tisotumab vedotin-related ocular toxicity. The study will include scheduled comprehensive ophthalmologic exams and a mechanism to collect, classify, and analyze data on ocular toxicity and exam findings. The scheduled ophthalmologic exams must be conducted prior to each cycle for the first 9 cycles of treatment, monthly for 90 days (+/- 7) following last treatment, and as clinically indicated, in order to assess and further characterize ocular adverse events and evaluate risk mitigation strategies. The ophthalmologic exam must include, at a minimum, slit lamp exam of the anterior segment of the eye, visual acuity, intraocular pressure, and elicitation for visual symptoms."~"Ongoing"~~2/28/2028 0:00:00~5/18/2026 0:00:00
385567~"CDER"~"BLA"~761208.00~"Seagen Inc."~"Tivdak (tisotumab vedotin-tftv)"~9/20/2021 0:00:00~"Supplement"~7~"4632-2"~"PMC"~"506B PMC"~4632.00~2~"PMC 4632-2: Conduct an integrated analysis of data from clinical trials and other data sources such as post-marketing reports, real-world evidence, and other sources, to further characterize the safety and efficacy of tisotumab vedotin in racial and ethnic minority patients with recurrent or metastatic cervical cancer (r/mCC). The population should be representative of the US population of patients with r/mCC, including racial and ethnic minorities, and allow for characterization of the results in these populations."~"Ongoing"~~3/31/2027 0:00:00~5/18/2026 0:00:00
385568~"CDER"~"BLA"~761210.00~"Janssen Biotech, Inc."~"RYBREVANT (amivantamab-vmjw)"~5/21/2021 0:00:00~"Supplement"~3~"4597-1"~"PMC"~"506B PMC"~4597.00~1~"PMC 4597-1: Complete clinical trial PAPILLON (NCT04538664) and include the results of the final overall survival analysis, once the anticipated 210 death events have occurred, to further characterize the clinical benefit of amivantamab in combination with carboplatin and pemetrexed, for the first line treatment of adult patients with metastatic non-small cell lung cancer harboring epidermal growth factor receptor exon 20 insertion mutations."~"Delayed"~"The trial completion milestone was missed due to slowing accumulation of overall survival events."~3/31/2026 0:00:00~7/14/2026 0:00:00
385569~"CDER"~"BLA"~761210.00~"Janssen Biotech, Inc."~"RYBREVANT (amivantamab-vmjw)"~5/21/2021 0:00:00~"Supplement"~3~"4597-2"~"PMC"~"506B PMC"~4597.00~2~"PMC 4597-2: Conduct an integrated analysis containing data from clinical trials and other data sources, such as real-world evidence and post-marketing clinical trial reports, to further characterize the efficacy and safety of amivantamab in combination with carboplatin and pemetrexed in patients ages 75 years and older, Black or African American patients, and patients of Latino ethnicity, diagnosed with metastatic NSCLC harboring EGFR exon 20 insertion mutations."~"Pending"~~12/31/2028 0:00:00~7/14/2026 0:00:00
385570~"CDER"~"NDA"~218808.00~"NEUROCRINE BIOSCIENCES INC"~"Crenessity (crinecerfont) Oral Capsules"~12/13/2024 0:00:00~"Original"~1~"4738-2"~"PMR"~"505 (o)(3)"~4738.00~2~"PMR 4738-2: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Crenessity during pregnancy to assess risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant. Assess infant outcomes through at least the first year of life. The study should also collect adverse event data for lactating women and infants exposed to Crenessity through breastfeeding to assess for any potential risks to the infant from breastfeeding. The minimum number of patients will be specified in the protocol."~"Pending"~~6/30/2036 0:00:00~2/11/2026 0:00:00
385571~"CDER"~"NDA"~218820.00~"NEUROCRINE BIOSCIENCES INC"~"Crenessity (crinecerfont)"~12/13/2024 0:00:00~"Original"~1~"4738-1"~"PMR"~"505 (o)(3)"~4738.00~1~"PMR 4738-1: Perform a lactation study (milk only or mother-infant pair study) in lactating women who have received Crenessity to measure concentrations of crinecerfont and its major metabolites in breast milk using a validated assay and to assess the effects on the breastfed infant, if a mother-infant pair study is conducted."~"Pending"~~6/30/2028 0:00:00~2/11/2026 0:00:00
385572~"CDER"~"NDA"~218820.00~"NEUROCRINE BIOSCIENCES INC"~"Crenessity (crinecerfont)"~12/13/2024 0:00:00~"Original"~1~"4738-2"~"PMR"~"505 (o)(3)"~4738.00~2~"PMR 4738-2: Conduct a worldwide descriptive study that collects prospective and retrospective data in women exposed to Crenessity during pregnancy to assess risk of pregnancy and maternal complications, and adverse effects on the developing fetus, neonate, and infant. Assess infant outcomes through at least the first year of life. The study should also collect adverse event data for lactating women and infants exposed to Crenessity through breastfeeding to assess for any potential risks to the infant from breastfeeding. The minimum number of patients will be specified in the protocol."~"Pending"~~6/30/2036 0:00:00~2/11/2026 0:00:00
385573~"CDER"~"NDA"~218860.00~"IPSEN BIOPHARMACEUTICALS INC"~"Iqirvo (elafibranor)"~6/10/2024 0:00:00~"Original"~1~"4629-2"~"PMR"~"505 (o)(3)"~4629.00~2~"PMR 4629-2: Conduct a ten-year worldwide descriptive study that collects prospective and retrospective data in women exposed to elafibranor during pregnancy to assess risk of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant. Infant outcomes will be assessed through at least the first year of life. The minimum number of patients will be specified in the protocol."~"Pending"~~3/31/2038 0:00:00~7/31/2025 0:00:00
385574~"CDER"~"NDA"~218860.00~"IPSEN BIOPHARMACEUTICALS INC"~"Iqirvo (elafibranor)"~6/10/2024 0:00:00~"Original"~1~"4629-3"~"PMR"~"505 (o)(3)"~4629.00~3~"PMR 4629-3: Perform a lactation study (milk and plasma) in lactating women who have received elafibranor to measure concentrations of elafibranor and its major metabolites in breast milk and maternal plasma using a validated assay."~"Pending"~~6/30/2028 0:00:00~7/31/2025 0:00:00
385575~"CDER"~"NDA"~218860.00~"IPSEN BIOPHARMACEUTICALS INC"~"Iqirvo (elafibranor)"~6/10/2024 0:00:00~"Original"~1~"4629-4"~"PMR"~"505 (o)(3)"~4629.00~4~"PMR 4629-4: Conduct a clinical drug-drug interaction study to evaluate the effect of elafibranor on the pharmacokinetics of combined oral contraceptives."~"Ongoing"~~12/31/2027 0:00:00~7/31/2025 0:00:00
385576~"CDER"~"NDA"~218860.00~"IPSEN BIOPHARMACEUTICALS INC"~"Iqirvo (elafibranor)"~6/10/2024 0:00:00~"Original"~1~"4629-5"~"PMR"~"505 (o)(3)"~4629.00~5~"PMR 4629-5: Conduct a clinical drug-drug interaction study to evaluate the effect of elafibranor on the pharmacokinetics of rosuvastatin"~"Pending"~~12/31/2027 0:00:00~7/31/2025 0:00:00
385577~"CDER"~"NDA"~218860.00~"IPSEN BIOPHARMACEUTICALS INC"~"Iqirvo (elafibranor)"~6/10/2024 0:00:00~"Original"~1~"4629-6"~"PMC"~"506B PMC"~4629.00~6~"PMC 4629-6: Conduct an in vitro phenotyping study (or studies) to assess the contribution of cytochrome P450 (CYP) and UDP glucuronosyltransferase (UGT) enzymes to the metabolism of GFT1007. The findings from in vitro studies should be followed up with in vivo drug-drug interaction studies if warranted."~"Submitted"~~3/31/2026 0:00:00~7/31/2025 0:00:00
385578~"CDER"~"NDA"~218860.00~"IPSEN BIOPHARMACEUTICALS INC"~"Iqirvo (elafibranor)"~6/10/2024 0:00:00~"Original"~1~"4629-7"~"PMC"~"506B PMC"~4629.00~7~"PMC 4629-7: Conduct a clinical drug-drug interaction study to evaluate the effect of a breast cancer resistance protein (BCRP) inhibitor on the pharmacokinetics of elafibranor and its major metabolites"~"Pending"~~12/31/2027 0:00:00~7/31/2025 0:00:00
385579~"CDER"~"NDA"~218860.00~"IPSEN BIOPHARMACEUTICALS INC"~"Iqirvo (elafibranor)"~6/10/2024 0:00:00~"Original"~1~"4629-8"~"PMR"~"Accelerated Approval"~4629.00~8~"PMR 4629-8: Complete Trial CLIN-60190-454, a randomized, double-blind, placebocontrolled study to evaluate the efficacy and safety of elafibranor in adults with primary biliary cholangitis (PBC). Efficacy must be demonstrated using a composite endpoint of all-cause mortality, liver transplant, hepatic decompensation, and change in MELD 3.0 to =15 in subjects with baseline MELD =12."~"Delayed"~"The applicant requested a revised milestone due to slower than anticipated patient enrollment from unforeseen challenges. A Revised milestone was acknowledged in a letter dated 06/01/2026 for the completion of trial enrollment milestone. There were 124 of the 276 planned participants enrolled per the 180-day AA PMR Progress Report submitted on 2/6/2026."~5/31/2030 0:00:00~7/31/2025 0:00:00
385580~"CDER"~"NDA"~218879.00~"OWP PHARMACEUTICALS INC"~"Subvenite (lamotrigine) Oral Suspension"~9/16/2025 0:00:00~"Original"~1~"4884-1"~"PMR"~"Pediatric Research Equity Act"~4884.00~1~"PMR 4884-1: Conduct a study to evaluate the pharmacokinetics, safety, and tolerability of an age-appropriate formulation of lamotrigine oral suspension (Subvenite) to determine a dosing regimen for partial-onset seizures in pediatric patients 1 month to less than 2 years of age. The study should identify the drug exposure that is similar to the exposure that is effective in patients 2 years and older with partial-onset seizures"~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~1/31/2029 0:00:00~
385581~"CDER"~"NDA"~218879.00~"OWP PHARMACEUTICALS INC"~"Subvenite (lamotrigine) Oral Suspension"~9/16/2025 0:00:00~"Original"~1~"4884-2"~"PMR"~"Pediatric Research Equity Act"~4884.00~2~"PMR 4884-2: Conduct a long-term open-label safety study of lamotrigine oral suspension (Subvenite) in pediatric patients 1 month to 2 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~7/31/2031 0:00:00~
385582~"CDER"~"NDA"~218881.00~"ELI LILLY AND CO"~"Inluriyo (imlunestrant) tablets"~9/25/2025 0:00:00~"Original"~1~"4902-1"~"PMC"~"506B PMC"~4902.00~1~"PMC 4902-1: Complete the pre-specified, event-driven overall survival (OS) analyses comparing the imlunestrant monotherapy and standard-of-care endocrine therapy arms from the ongoing clinical trial, EMBER-3 (Study J2J-OXJZLC),
titled  A Phase 3, Randomized, Open-Label Study of Imlunestrant, Investigator s Choice of Endocrine Therapy, and Imlunestrant plus Abemaciclib in Patients with Estrogen-Receptor Positive, HER2 Negative Locally Advanced or Metastatic Breast Cancer Previously Treated with Endocrine Therapy , to further characterize the clinical benefit of imlunestrant in patients with estrogen-receptor positive, HER2 negative ESR1-mutated breast cancer."~"Pending"~~5/31/2027 0:00:00~
385583~"CDER"~"NDA"~218922.00~"CIPLA LTD"~"Nilceya (nilotinib) capsules"~2/19/2025 0:00:00~"Original"~1~"4794-1"~"PMR"~"Pediatric Research Equity Act"~4794.00~1~"PMR 4794-1: Conduct a molecularly targeted pediatric cancer investigation, which includes developing an age-appropriate formulation."~"Pending"~"The study has not begun but does not meet the criterion for
delayed."~11/30/2032 0:00:00~4/15/2026 0:00:00
385584~"CDER"~"BLA"~761040.00~"Wyeth Pharmaceuticals LLC"~"Besponsa (Inotuzumab Ozogamicin)"~8/17/2017 0:00:00~"Supplement"~3~"4585-1"~"PMR"~"505 (o)(3)"~4585.00~1~"PMR 4585-1: Conduct an integrated analysis of data from available sources, including data from Study B1931030, to support optimization of pediatric dosage(s), to assess whether lower doses of inotuzumab ozogamicin can minimize the serious risk of severe liver veno-occlusive disease (VOD) in pediatric patients and the serious potential risk of increased adverse reactions due to higher exposure in pediatric patients with lower body size. Evaluate for alternative dosing regimen(s) based on body weight to potentially minimize exposure difference between pediatric and adult patients with ALL, particularly for pediatric patients with low body surface area or weight."~"Submitted"~~5/31/2025 0:00:00~10/21/2025 0:00:00
385585~"CDER"~"BLA"~761044.00~"Janssen Biotech, Inc."~"Stelara (Ustekinumab)"~9/23/2016 0:00:00~"Original"~1~"3112-1"~"PMR"~"505 (o)(3)"~3112.00~1~"PMR 3112-1: Conduct a long-term, postmarketing, observational study to assess the long-term safety of STELARA (ustekinumab) versus other therapies used in the treatment of adults with moderate to severe Crohn s disease. The study s primary outcome is  malignancy. Secondary outcomes include, but are not limited to, opportunistic infections (e.g., tuberculosis [TB]). Specify concise case definitions, and provide outcome validation for both primary and secondary outcomes. Describe and justify the choice of appropriate comparator population(s) and estimated background rate(s) relative to ustekinumab-exposed patients; clearly define the  primary comparator population for the primary objective. Design the study around a testable hypothesis to assess, with sufficient sample size and power, a clinically meaningful increase in malignancy risk above the comparator background rate, with a pre-specified statistical analysis method. For the ustekinumab-exposed and comparator(s), the study drug initiation period should be clearly defined, including any exclusion and inclusion criteria. Ensure adequate number of patients with at least 18 months of ustekinumab exposure at the end of the study. Follow for a period of at least 7 years."~"Delayed"~"The interim report submission milestone was missed because of challenges related to access to the Department of Defense (DOD) database. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 01/28/2026."~8/31/2030 0:00:00~11/17/2025 0:00:00
385586~"CDER"~"BLA"~761044.00~"Janssen Biotech, Inc."~"Stelara (Ustekinumab)"~9/23/2016 0:00:00~"Original"~1~"3112-3"~"PMC"~"506B PMC"~3112.00~3~"PMC 3112-3: Conduct a randomized, controlled, blinded, multicenter trial to evaluate the safety and efficacy of STELARA (ustekinumab) in pediatric patients 2 to 17 years of age with moderately to severely active Crohn s disease despite conventional therapy."~"Fulfilled"~~9/30/2024 0:00:00~11/17/2025 0:00:00
385587~"CDER"~"BLA"~761044.00~"Janssen Biotech, Inc."~"Stelara (Ustekinumab)"~9/23/2016 0:00:00~"Supplement"~3~"3736-1"~"PMR"~"505 (o)(3)"~3736.00~1~"PMR 3736-1: A long-term, postmarketing, observational study to assess the long-term safety of STELARA (ustekinumab) versus other therapies used in the treatment of adults with moderate to severe ulcerative colitis. The study s primary outcome is malignancy. Secondary outcomes include, but are not limited to, opportunistic infections (e.g., tuberculosis [TB]). Specify concise case definitions and provide outcome validation for both primary and secondary outcomes. Describe and justify the choice of appropriate comparator population(s) and estimated background rate(s) relative to ustekinumab-exposed patients; clearly define the primary comparator population for the primary objective. Design the study around a testable hypothesis to assess, with sufficient sample size and power, a clinically meaningful increase in malignancy risk above the comparator background rate, with a pre-specified statistical analysis method. For the ustekinumab-exposed and comparator(s), the study drug initiation period should be clearly defined, including any exclusion and inclusion criteria. Ensure adequate number of patients with at least 18 months of ustekinumab exposure at the end of the study. Follow for a period of at least 7 years. The ongoing observational study in patients with Crohn s disease with the same objectives, may be amended to also enroll patients with ulcerative colitis.
"~"Delayed"~"The original final protocol milestone was missed. The Final protocol was received and acknowledged per FDA letter dated April 21, 2021."~8/31/2030 0:00:00~11/17/2025 0:00:00
385588~"CDER"~"BLA"~761044.00~"Janssen Biotech, Inc."~"Stelara (Ustekinumab)"~9/23/2016 0:00:00~"Supplement"~3~"3736-2"~"PMC"~"506B PMC"~3736.00~2~"PMC 3736-2: A one-year, randomized, controlled, blinded trial to evaluate the safety, efficacy, and pharmacokinetics of Stelara (ustekinumab) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis."~"Submitted"~~9/30/2025 0:00:00~11/17/2025 0:00:00
385589~"CDER"~"BLA"~761044.00~"Janssen Biotech, Inc."~"Stelara (Ustekinumab)"~9/23/2016 0:00:00~"Supplement"~3~"3736-3"~"PMC"~"506B PMC"~3736.00~3~"PMC 3736-3: A multi-center, open-label extension study to evaluate the long-term safety of Stelara (ustekinumab) in pediatric patients 2 to 17 years of age with moderately to severely active ulcerative colitis who participated in PMC 3736-2."~"Delayed"~"The applicant requested revised milestones due to delays in meeting the protocol-defined criteria for end-of-study participation. The study completion and final report submission milestons were revised and acknowledged in a letter dated 05/12/2026."~9/30/2026 0:00:00~11/17/2025 0:00:00
385590~"CDER"~"BLA"~761045.00~"Sandoz Inc."~"Ziextenzo (pegfilgrastim-bmez)"~11/4/2019 0:00:00~"Original"~1~"3734-1"~"PMR"~"Pediatric Research Equity Act"~3734.00~1~"PMR 3734-1: Submit pediatric assessments for Ziextenzo (pegfilgrastim-bmez) as described in section 505B(a)(2)(A) of the FD&C Act, including
development of an  appropriate formulation  (presentation) that can be used to directly and accurately administer Ziextenzo (pegfilgrastim-bmez) to pediatric patients who weigh less than 45 kg and require doses that are less than 0.6 mL (6 mg), and conducting any necessary human factors studies to evaluate the ability of healthcare providers and/or caregivers to measure the appropriate doses.
"~"Pending"~"Per FDA letter dated 01/13/2026, the applicant s request for release was denied"~4/30/2028 0:00:00~12/16/2025 0:00:00
385591~"CDER"~"BLA"~761039.00~"Accord BioPharma Inc."~"Udenyca Onbody (pegfilgrastim-cbqv)"~11/2/2018 0:00:00~"Original"~1~"3733-1"~"PMR"~"Pediatric Research Equity Act"~3733.00~1~"PMR 3733-1: Submit pediatric assessments for Udenyca (pegfilgrastim-cbqv) as described in section 505B(a)(2)(A) of the FD&C Act, including development of an  appropriate formulation  (presentation) that can be used to directly and accurately administer Udenyca (pegfilgrastim-cbqv) to pediatric patients who weigh less than 45 kg and require doses that are less than 0.6 mL (6 mg), and conducting any necessary human factors studies to evaluate the ability of healthcare providers and/or caregivers to measure the appropriate doses."~"Pending"~"Original Final Report Due Date: 10/31/2025; Deferral Extension granted per FDA letter dated 09/18/2024."~4/30/2028 0:00:00~12/29/2025 0:00:00
385592~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-5"~"PMR"~"505 (o)(3)"~4626.00~5~"PMR 4626-5: Conduct comprehensive safety analyses from clinical studies that further characterize the potential serious risk of gonadal toxicity in a sufficient number of adolescent and young adult patients treated with tovorafenib. Include evaluations of pubertal development and hormonal evaluation in appropriate patients. Monitor patients for a minimum of 5 years from start of treatment. Post-treatment follow-up assessments including reproductive hormone measurements should be obtained at regularly scheduled intervals and after treatment cessation to assess time to gonadal function recovery. Include, in the interim report, a comprehensive safety analysis characterizing gonadal toxicity after all patients have been monitored for a minimum of 12 months from the start of study treatment."~"Ongoing"~~4/30/2032 0:00:00~6/12/2026 0:00:00
385593~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-6"~"PMR"~"505 (o)(3)"~4626.00~6~"PMR 4626-6: Conduct a clinical pharmacokinetic trial of tovorafenib with a strong CYP2C8 inhibitor to evaluate the potential increased drug toxicity and provide dosage recommendations for tovorafenib when used concomitantly with strong and moderate CYP2C8 inhibitors. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Pending"~~4/30/2026 0:00:00~6/12/2026 0:00:00
385594~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-7"~"PMR"~"505 (o)(3)"~4626.00~7~"PMR 4626-7: Conduct a clinical pharmacokinetic trial of multi-dose tovorafenib on sensitive substrates of CYP3A4, CYP2C8, CYP1A2, CYP2B6, CYP2C9, and CYP2C19 to evaluate the potential for increased toxicity or reduced efficacy of these substrate drugs and provide appropriate drug interaction management strategies for tovorafenib when used concomitantly with these substrates. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Pending"~~12/31/2026 0:00:00~6/12/2026 0:00:00
385595~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-8"~"PMR"~"505 (o)(3)"~4626.00~8~"PMR 4626-8: Conduct a clinical pharmacokinetic trial of multi-dose tovorafenib on substrates of BCRP to evaluate the potential for increased toxicity of BCRP substrates and provide appropriate drug interaction management strategies for tovorafenib when used concomitantly with BCRP substrates. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Pending"~~12/31/2026 0:00:00~6/12/2026 0:00:00
385596~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-9"~"PMC"~"506B PMC"~4626.00~9~"PMC 4626-9: Complete the FIREFLY-1 trial, including your planned additional follow-up for the 137 patients with relapsed or refractory low-grade glioma harboring a BRAF alteration enrolled to Arms 1 and 2 of FIREFLY-1 and treated with tovorafenib, to further characterize the overall response rate (ORR) and duration of response (DoR) as per Response Assessment in Pediatric Neuro-oncology (RAPNO) criteria including assessment by an independent review committee (IRC) for patients enrolled to Arm 1. Include, in the interim report, an analysis of ORR and DoR in Arm 1 as per RAPNO criteria including assessment by an IRC after all Arm 1 patients have completed a minimum of 26 cycles of treatment in the FIREFLY-1trial."~"Ongoing"~~12/31/2027 0:00:00~6/12/2026 0:00:00
385597~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-10"~"PMC"~"506B PMC"~4626.00~10~"PMC 4626-10: Conduct a clinical pharmacokinetic trial with a strong or moderate CYP2C8 inducer with tovorafenib to evaluate the effect of a strong or moderate CYP2C8 inducer on decreasing the systemic exposure of tovorafenib and provide dosage recommendations for tovorafenib when used concomitantly with CYP2C8 inducers. Design and conduct the trial in accordance with the FDA Guidance for Industry titled  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter- Mediated Drug Interactions. "~"Pending"~~4/30/2026 0:00:00~6/12/2026 0:00:00
385598~"CDER"~"NDA"~218033.00~"DAY ONE BIOPHARMACEUTICALS INC"~"Ojemda (tovorafenib)"~4/23/2024 0:00:00~"Original"~1~"4626-11"~"PMC"~"506B PMC"~4626.00~11~"PMC 4626-11: Conduct an appropriate analytical and clinical validation study to support the development of an in vitro diagnostic device using clinical trial data that demonstrates that the device is essential to the safe and effective use of tovorafenib for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation."~"Fulfilled"~~7/31/2024 0:00:00~6/12/2026 0:00:00
385599~"CDER"~"NDA"~218037.00~"ALEXION PHARMACEUTICALS INC"~"Voydeya (danicopan)"~3/29/2024 0:00:00~"Original"~1~"4601-1"~"PMR"~"505 (o)(3)"~4601.00~1~"PMR 4601-1: Establish a registry to characterize the long-term safety of danicopan in adults with paroxysmal nocturnal hemoglobinuria (PNH), with at least 5 years of follow-up. Yearly safety follow-up data should include a summary of the major safety findings for all patients and all serious infections with encapsulated bacteria. The final study report should include an integrated safety dataset and patient level data, including data on danicopan dosing, meningococcal and pneumococcal vaccination status, and concomitant medications."~"Ongoing"~~12/31/2029 0:00:00~5/28/2026 0:00:00
385600~"CDER"~"NDA"~218037.00~"ALEXION PHARMACEUTICALS INC"~"Voydeya (danicopan)"~3/29/2024 0:00:00~"Original"~1~"4601-2"~"PMC"~"506B PMC"~4601.00~2~"PMC 4601-2: Complete Study ALXN2040-PNH-301: A phase 3 study of danicopan (ALXN2040) as add-on therapy to a C5 inhibitor (eculizumab or ravulizumab) in patients with paroxysmal nocturnal hemoglobinuria who have clinically evident extravascular hemolysis (EVH). Include an updated summary of efficacy and safety analyses and datasets at the time of final clinical study report submission."~"Fulfilled"~~3/31/2026 0:00:00~5/28/2026 0:00:00
385601~"CDER"~"NDA"~218160.00~"ELI LILLY AND CO"~"Retevmo (selpercatinib) "~4/10/2024 0:00:00~"Supplement"~2~"4706-1"~"PMC"~"506B PMC"~4706.00~1~"PMC 4706-1: Complete survival follow-up of patients in the LIBRETTO-531 trial at 125 deaths or 6 years from randomization of the first patient, whichever occurs first, to further characterize the efficacy and clinical benefit of selpercatinib in patients with advanced or metastatic RET mutant medullary thyroid cancer who are na ve to tyrosine kinase inhibitors."~"Ongoing"~~9/30/2026 0:00:00~6/26/2026 0:00:00
385602~"CDER"~"NDA"~218160.00~"ELI LILLY AND CO"~"Retevmo (selpercatinib) "~4/10/2024 0:00:00~"Supplement"~3~"4715-1"~"PMC"~"506B PMC"~4715.00~1~"PMC 4715-1: Complete survival follow-up of patients in the LIBRETTO-431 trial at 175 deaths to further characterize the efficacy and clinical benefit of selpercatinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with a RET gene fusion."~"Ongoing"~~3/31/2034 0:00:00~6/26/2026 0:00:00
385603~"CDER"~"NDA"~218171.00~"XCOVERY HOLDINGS INC"~"Ensacove (ensartinib) Capsules"~12/18/2024 0:00:00~"Original"~1~"4752-1"~"PMR"~"Pediatric Research Equity Act"~4752.00~1~"PMR 4752-1: Conduct a molecularly targeted pediatric cancer investigation (Pediatric MATCH sub-study F) to assess the safety and preliminary efficacy of ensartinib in pediatric patients with advanced or metastatic solid tumors with ALK or ROS1 alterations."~"Ongoing"~"The study has been initiated."~6/30/2026 0:00:00~2/13/2026 0:00:00
385604~"CDER"~"BLA"~761210.00~"Janssen Biotech, Inc."~"RYBREVANT (amivantamab-vmjw)"~5/21/2021 0:00:00~"Supplement"~4~"4685-1"~"PMC"~"506B PMC"~4685.00~1~"PMC 4685-1: Complete clinical trial MARIPOSA-2 (NCT04988295) and include the results of the final overall survival analysis, once the anticipated 400 death events have occurred in all three arms of the trial, to further characterize the clinical benefit of amivantamab in combination with carboplatin and pemetrexed for the treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R substitution mutations, whose disease has progressed on or after treatment with an EGFR tyrosine kinase inhibitor."~"Delayed"~"The trial completion milestone was missed to ensure sufficient follow-up to complete the final overall survival analysis."~1/31/2026 0:00:00~7/14/2026 0:00:00
385605~"CDER"~"BLA"~761210.00~"Janssen Biotech, Inc."~"RYBREVANT (amivantamab-vmjw)"~5/21/2021 0:00:00~"Supplement"~4~"4685-2"~"PMC"~"506B PMC"~4685.00~2~"PMC 4685-2: Conduct an integrated analysis containing data from clinical trials and other data sources such as post-marketing reports, real-world evidence and other sources to further characterize the safety and efficacy of amivantamab in combination with carboplatin and pemetrexed in Black or African American patients diagnosed with metastatic NSCLC harboring EGFR exon 19 deletions or exon 21 L858R substitution mutations."~"Pending"~~6/30/2029 0:00:00~7/14/2026 0:00:00
385606~"CDER"~"BLA"~761210.00~"Janssen Biotech, Inc."~"RYBREVANT (amivantamab-vmjw)"~5/21/2021 0:00:00~"Supplement"~5~"4680-1"~"PMR"~"505 (o)(3)"~4680.00~1~"PMR 4680-1: Conduct comprehensive safety analyses from clinical studies that further characterize the known serious risk of venous thromboembolic events (VTEs) in patients treated with amivantamab intravenously (IV) and patients treated with amivantamab subcutaneously (SC), in combination with lazertinib. Provide the incidence, timing, and outcome of VTEs by grade in patients who did not receive prophylactic anticoagulation, who received prophylactic anticoagulation for the initial 4 months of study therapy only, who continued prophylactic anticoagulation beyond 4 months of study therapy, and who required initiation of treatment-level anticoagulation. Monitor patients for VTEs throughout study therapy until treatment discontinuation."~"Fulfilled"~~12/31/2024 0:00:00~7/14/2026 0:00:00
385607~"CDER"~"BLA"~761210.00~"Janssen Biotech, Inc."~"RYBREVANT (amivantamab-vmjw)"~5/21/2021 0:00:00~"Supplement"~5~"4680-2"~"PMC"~"506B PMC"~4680.00~2~"PMC 4680-2: Complete the MARIPOSA trial and include the results of the final overall survival analysis once the anticipated 390 death events have occurred in the amivantamab in combination with lazertinib and osimertinib arms to further characterize the clinical benefit of amivantamab in combination with lazertinib for the first line treatment of adult patients with metastatic NSCLC harboring EGFR exon 19 deletion or L858R mutations."~"Fulfilled"~~11/30/2025 0:00:00~7/14/2026 0:00:00
385608~"CDER"~"BLA"~761210.00~"Janssen Biotech, Inc."~"RYBREVANT (amivantamab-vmjw)"~5/21/2021 0:00:00~"Supplement"~5~"4680-3"~"PMC"~"506B PMC"~4680.00~3~"PMC 4680-3: Conduct an integrated analysis containing data from clinical trials and other data sources such as post-marketing reports, real-world evidence and other sources to further characterize the safety and efficacy of amivantamab in combination with lazertinib in patients who are Black or African American diagnosed with metastatic NSCLC harboring EGFR exon 19 deletions or exon 21 L858R substitution mutations."~"Pending"~~6/30/2029 0:00:00~7/14/2026 0:00:00
385609~"CDER"~"BLA"~761211.00~"Immedica Pharma AB"~"Loargys (pegzilarginase-nbln)"~2/23/2026 0:00:00~"Original"~1~"4966-1"~"PMR"~"Accelerated Approval"~4966.00~1~"PMR 4966-1: Conduct a trial to verify and describe the clinical benefit of pegzilarginase in patients with Arginase-1 Deficiency."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2035 0:00:00~
385610~"CDER"~"BLA"~761211.00~"Immedica Pharma AB"~"Loargys (pegzilarginase-nbln)"~2/23/2026 0:00:00~"Original"~1~"4966-2"~"PMC"~"506B PMC"~4966.00~2~"PMC 4966-2: Conduct adequate analytical and clinical validation to establish and support labeling of an FDA-authorized in vitro diagnostic device that accurately and reliably measures arginine to guide safe and effective use of pegzilarginase in patients with arginase 1 deficiency. The results of the validation studies are intended to inform product labeling."~"Pending"~~8/31/2027 0:00:00~
385611~"CDER"~"BLA"~761211.00~"Immedica Pharma AB"~"Loargys (pegzilarginase-nbln)"~2/23/2026 0:00:00~"Original"~1~"4966-3"~"PMC"~"506B PMC"~4966.00~3~"PMC 4966-3: Conduct adequate analytical and clinical validation to establish and support labeling of an FDA-authorized in vitro diagnostic blood collection device, containing nor-NOHA, for collection and storage of specimens containing pegzilarginase, in the treatment of patients with arginase 1 deficiency. The results of the validation studies are intended to inform product labeling."~"Pending"~~8/31/2027 0:00:00~
385612~"CDER"~"BLA"~761212.00~"Lupin Limited (Biotech Division)"~"Armlupeg (pegfilgrastim-unne) Injection"~11/28/2025 0:00:00~"Original"~1~"4932-1"~"PMR"~"Pediatric Research Equity Act"~4932.00~1~"PMR 4932-1: Develop a presentation that can be used to accurately administer Armlupeg (pegfilgrastim-unne) to pediatric patients who weigh less than 45 kg."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~4/30/2028 0:00:00~
385613~"CDER"~"BLA"~761216.00~"Emerge Bioscience Pte. Ltd."~"Yusimry (adalimumab-aqvh)"~12/17/2021 0:00:00~"Original"~1~"4184-1"~"PMR"~"Pediatric Research Equity Act"~4184.00~1~"PMR 4184-1: Develop presentations that can be used to accurately administer Yusimry (adalimumab-aqvh) to pediatric patients weighing 10 kg to less than 40 kg."~"Ongoing"~"Original Final Report Due Date was 12/2023; Per Deferral
Extension Granted letter issued on 6/22/2023, new final report submission date is 9/2024. 2nd Deferral Extension Granted letter issued on 6/26/2024, new final report submission date is 9/2027.
"~9/30/2027 0:00:00~2/13/2026 0:00:00
385614~"CDER"~"BLA"~761219.00~"CELLTRION, Inc."~"Yuflyma (adalimumab-aaty)"~5/23/2023 0:00:00~"Original"~1~"4433-1"~"PMR"~"Pediatric Research Equity Act"~4433.00~1~"PMR 4433-1: Develop presentation(s) that can be used to accurately administer Yuflyma (adalimumab-aaty) to pediatric patients weighing 10 kg to less than 40 kg."~"Fulfilled"~"Per FDA letter dated 10/28/2025, this PMR/PMC has been fulfilled."~6/30/2025 0:00:00~7/23/2025 0:00:00
385615~"CDER"~"BLA"~761223.00~"GlaxoSmithKline LLC"~"Jemperli (dostarlimab-gxly)"~8/17/2021 0:00:00~"Original"~1~"4124-1"~"PMR"~"Accelerated Approval"~4124.00~1~"PMR 4124-1: Conduct a clinical trial evaluating overall response rate, and duration of response, to verify and describe the clinical benefit of Jemperli in patients with mismatch repair deficient (dMMR), recurrent or advanced solid tumors, including at least 300 patients across all tumor types, and  including a sufficient number of patients and representation of tumor types (other than endometrial and gastrointestinal tumors). In order to characterize response rate and duration of response, patients should be followed for at least 12 months from the onset of response. Submit the datasets with final report."~"Delayed"~"Delayed due to enrollment issues.  Revised milestones were acknowledged in a letter dated 10/28/2022."~10/31/2022 0:00:00~
385616~"CDER"~"BLA"~761223.00~"GlaxoSmithKline LLC"~"Jemperli (dostarlimab-gxly)"~8/17/2021 0:00:00~"Original"~1~"4124-2"~"PMC"~"506B PMC"~4124.00~2~"PMC 4124-2: Commitment to establish and support the availability of a nucleic acid based in vitro diagnostic device that is essential to support the safe and effective use of Jemperli for patients with tumors that are microsatellite instability high (MSI-H) through an appropriate analytical and clinical validation study using clinical trial data. Summarize the study results in the final report submission."~"Released"~~12/31/2025 0:00:00~
385617~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-1"~"PMR"~"505 (o)(3)"~4730.00~1~"PMR 4730-1: Conduct a clinical trial to assess and further characterize known serious risks related to the use of revumenib of sudden death, QTc prolongation, differentiation syndrome and elevated transaminases, and assess serious potential risks such as elevated parathyroid hormone. The trial will determine whether long-term use of revumenib is associated with increased risk of cardiac deaths, recurrent differentiation syndrome, serious hepatotoxicity, and serious potential risk of endocrine-related disorders due to chronic menin inhibition. All patients should have at least 3 years of treatment or discontinued earlier."~"Ongoing"~~5/31/2028 0:00:00~1/13/2026 0:00:00
385618~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-2"~"PMR"~"505 (o)(3)"~4730.00~2~"PMR 4730-2: Conduct a clinical pharmacokinetic trial to assess the magnitude of change in exposure of revumenib and its metabolite M1 and serious potential risk of increased drug toxicities to determine an appropriate dosage of revumenib in patients with severe hepatic impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~7/31/2032 0:00:00~1/13/2026 0:00:00
385619~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-3"~"PMR"~"505 (o)(3)"~4730.00~3~"PMR 4730-3: Conduct a clinical pharmacokinetic trial to assess the magnitude of change in exposure of revumenib and its metabolite M1 and serious potential risk of increased drug toxicities to determine an appropriate dosage of revumenib in patients with severe renal impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Renal Function: Study Design, Data Analysis, and Impact on Dosing. "~"Pending"~~8/31/2032 0:00:00~1/13/2026 0:00:00
385620~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-4"~"PMR"~"505 (o)(3)"~4730.00~4~"PMR 4730-4: Complete the clinical pharmacokinetic trial to characterize the absorption, distribution, metabolism, and excretion of revumenib and its metabolite M1 and assess the serious potential risk of increased drug toxicities to determine an appropriate dosage of revumenib in patients with both hepatic and renal impairment. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Pharmacology Considerations for Human Radiolabeled Mass Balance Studies. "~"Fulfilled"~~7/31/2025 0:00:00~1/13/2026 0:00:00
385621~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-5"~"PMR"~"505 (o)(3)"~4730.00~5~"PMR 4730-5: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of OATP1B1 inhibitors on the pharmacokinetics of revumenib and M1 to assess the serious potential risk of excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Cytochrome P450 Enzymeand Transporter-Mediated Drug Interactions. "~"Pending"~~8/31/2031 0:00:00~1/13/2026 0:00:00
385622~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-6"~"PMR"~"505 (o)(3)"~4730.00~6~"PMR 4730-6: Conduct a clinical pharmacokinetic trial to evaluate the effect of repeat doses of revumenib on the single dose pharmacokinetics of a substrate of the multidrug and toxin extrusion protein 1 (MATE1) transporter to assess the serious potential risk of excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter- Mediated Drug Interactions. "~"Pending"~~9/30/2031 0:00:00~1/13/2026 0:00:00
385623~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-7"~"PMR"~"505 (o)(3)"~4730.00~7~"PMR 4730-7: Conduct a physiologically-based pharmacokinetic (PBPK) modeling study and a clinical pharmacokinetic trial if the FDA determines that the PBPK modeling study results are insufficient, to evaluate the effect of repeat doses of revumenib on the single dose pharmacokinetics of a sensitive substrate of CYP3A4 to assess the serious potential risk of excessive drug toxicity. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Pending"~~11/30/2033 0:00:00~1/13/2026 0:00:00
385624~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-8"~"PMC"~"506B PMC"~4730.00~8~"PMC 4730-8: Complete the clinical pharmacokinetic trial to evaluate the effect of a high fat meal on revumenib exposure and to evaluate appropriate administration of revumenib with regard to food. Design and conduct the trial in accordance with the FDA Guidance for Industry:  Assessing the Effects of Food on Drugs in INDs and NDAs   Clinical Pharmacology Considerations. "~"Pending"~~10/31/2031 0:00:00~1/13/2026 0:00:00
385625~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-9"~"PMC"~"506B PMC"~4730.00~9~"PMC 4730-9: Conduct an in vitro study to clarify the activity of revumenib in acute leukemias with various KMT2A translocations. Include fusion partners that are rare and those that are not involved in the process of transcriptional elongation."~"Pending"~~6/30/2026 0:00:00~1/13/2026 0:00:00
385626~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-10"~"PMC"~"506B PMC"~4730.00~10~"PMC 4730-10: Conduct a clinical trial to support the availability of an in vitro diagnostic device for identifying KMT2A translocations in patients with acute leukemias for the safe and effective use of revumenib."~"Fulfilled"~~1/31/2025 0:00:00~1/13/2026 0:00:00
385627~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Original"~1~"4730-11"~"PMC"~"506B PMC"~4730.00~11~"PMC 4730-11: Develop an oral solution formulation and conduct a study to assess relative bioavailability between the oral solution and capsule formulations. Design and conduct the trial in accordance with the FDA Guidance for Industry:  Bioavailability Studies Submitted in NDAs or INDs   General Considerations. "~"Pending"~~11/30/2027 0:00:00~1/13/2026 0:00:00
385628~"CDER"~"NDA"~218944.00~"SYNDAX PHARMACEUTICALS INC"~"Revuforj (revumenib)"~11/15/2024 0:00:00~"Supplement"~3~"4919-1"~"PMC"~"506B PMC"~4919.00~1~"PMC 4919-1: Conduct a clinical trial to support the availability of an in vitro diagnostic device for identification of susceptible NPM1 mutations in patients with acute myeloid leukemia that is essential for the safe and effective use of revumenib."~"Pending"~~6/30/2027 0:00:00~1/13/2026 0:00:00
385629~"CDER"~"NDA"~219008.00~"JANSSEN BIOTECH INC"~"Lazcluze (lazertinib)"~8/19/2024 0:00:00~"Original"~1~"4681-1"~"PMR"~"505 (o)(3)"~4681.00~1~"PMR 4681-1: Conduct comprehensive safety analyses from clinical studies that further characterize the known serious risk of venous thromboembolic events (VTEs) in patients treated with amivantamab intravenously (IV) and patients treated with amivantamab subcutaneously (SC), in combination with lazertinib. Provide the incidence, timing, and outcome of VTEs by grade in patients who did not receive prophylactic anticoagulation, who received prophylactic anticoagulation for the initial 4 months of study therapy only, who continued prophylactic anticoagulation beyond 4 months of study therapy, and who required initiation of treatment-level anticoagulation. Monitor patients for VTEs throughout study therapy until treatment discontinuation."~"Fulfilled"~~12/31/2024 0:00:00~10/14/2025 0:00:00
385630~"CDER"~"BLA"~761047.00~"Ultragenyx Pharmaceutical Inc."~"Mepsevii (vestronidase alfa-vjbk)"~11/15/2017 0:00:00~"Original"~1~"3271-1"~"PMR"~"505 (o)(3)"~3271.00~1~"PMR 3271-1: A prospective, longitudinal study (Study UX003-CL401) to assess the long-term risk of immunogenicity and the risk of serious hypersensitivity reactions, including anaphylaxis, in patients with mucopolysaccharidosis type VII (MPS VII) followed for three years on Mepsevii (vestronidase alfa-vjbk). The following information will be collected and analyzed: (1) incidence rates for serious hypersensitivity reactions, (2) incidence rates for the appearance of anti-drug antibodies (ADA) and neutralizing antibodies (Nab) against Mepsevii (vestronidase alfa-vjbk), (3) temporal associations between ADA or Nab formation and serious hypersensitivity reactions, (4) associations between betaglucuronidase (GUSB) genotype and serious hypersensitivity reaction risk, (5)  associations between intrinsic GUSB enzymatic activity and serious hypersensitivity reaction risk, and (6) assessments of the risk of immunogenicity on clinical safety outcomes. To complete these analyses, protocol UX003-CL401 will require collection of molecular genotype and intrinsic GUSB enzymatic activity (apart from any concurrent enzyme replacement). Submit annual study reports that contain results from analyses of interim data. The final study report will be based on a study population that contains at least 12 new patients (including at least six patients less than one year old) treated with Mepsevii (vestronidase alfa-vjbk) and enrolled in Study UX003-CL401."~"Released"~~5/31/2026 0:00:00~7/25/2025 0:00:00
385631~"CDER"~"BLA"~761047.00~"Ultragenyx Pharmaceutical Inc."~"Mepsevii (vestronidase alfa-vjbk)"~11/15/2017 0:00:00~"Original"~1~"3271-3"~"PMC"~"506B PMC"~3271.00~3~"PMC 3271-3: In MPS VII patients enrolled in the prospective, longitudinal Study UX003-CL401 (PMR-1), to collect and analyze: (1) beta glucuronidase (GUSB) genotype, (2) in patients without a history of Mepsevii (vestronidase alfa-vjbk) treatment, baseline intrinsic GUSB enzymatic activity apart from any concurrent enzyme replacement treatment, (3) a complete record of treatments with Mepsevii (vestronidase alfa-vjbk) pre- and post-UX003-CL401 enrollment, and (4) results from baseline and periodic tests for MPS VII clinical outcomes to include liver and spleen size measurement, pulmonary function, motor function, and neurocognitive function."~"Ongoing"~~5/31/2033 0:00:00~7/25/2025 0:00:00
385632~"CDER"~"BLA"~761047.00~"Ultragenyx Pharmaceutical Inc."~"Mepsevii (vestronidase alfa-vjbk)"~11/15/2017 0:00:00~"Original"~1~"3271-13"~"PMR"~"505 (o)(3)"~3271.00~13~"PMR 3271-13: A prospective, longitudinal study (Study UX003-CL401) to assess the long-term risk of immunogenicity and the risk of serious hypersensitivity reactions, including anaphylaxis, in patients with mucopolysaccharidosis type VII (MPS VII) followed for three years on Mepsevii (vestronidase alfa-vjbk).
The following information will be collected and analyzed: (1)
incidence rates for serious hypersensitivity reactions, (2) incidence rates for the appearance of anti-drug antibodies (ADA) and neutralizing antibodies (Nab) against Mepsevii (vestronidase alfa-vjbk), (3) temporal associations between ADA or Nab formation and serious hypersensitivity
reactions, (4) associations between betaglucuronidase (GUSB) genotype and serious hypersensitivity reaction risk, (5) associations between intrinsic GUSB enzymatic activity and serious hypersensitivity reaction risk, and (6) assessments of the risk of immunogenicity on clinical safety
outcomes. To complete these analyses, protocol UX003-CL401 will require collection of molecular genotype and intrinsic GUSB enzymatic activity (apart from any concurrent enzyme replacement). The final study
report will be based on a study population that contains at least 12 new patients treated with Mepsevii (vestronidase alfa-vjbk) and enrolled in Study UX003-CL401."~"Submitted"~~5/31/2026 0:00:00~7/25/2025 0:00:00
385633~"CDER"~"BLA"~761051.00~"Kyowa Kirin, Inc."~"Poteligeo (mogamulizumab-kpkc)"~8/8/2018 0:00:00~"Original"~1~"3459-1"~"PMR"~"505 (o)(3)"~3459.00~1~"PMR 3459-1: Characterize complications after allogeneic hematopoietic stem cell transplantation (HSCT) following mogamulizumab-kpkc in at least 50 patients with hematologic malignancies (at least 40 with cutaneous T-cell lymphoma), of whom at least two-thirds received mogamulizumab-kpkc as the last regimen prior to HSCT. Evaluate toxicities at least through transplant Day 180, and include details of prior mogamulizumab-kpkc treatment and the transplant regimen. Characterize toxicities including grade II-IV acute graft-versus-host disease (GVHD), severe (grade III-IV) acute GVHD, steroid-refractory acute GVHD, grade 4, potentially immune-mediated adverse events, graft failure, hepatic veno-occlusive disease, critical illness, and non-relapse mortality and the cause. The study may characterize toxicities prospectively, or through a combination of prospective and retrospective data analysis. Utilize registry data (for example the Center for International Blood and Marrow Transplantation Research registry) to inform the analysis."~"Delayed"~"The Study Completion and Final Report Submission milestones were missed because of enrollment difficulties. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter 10/14/2025."~8/31/2024 0:00:00~10/6/2025 0:00:00
385634~"CDER"~"BLA"~761052.00~"Biomarin Pharmaceutical Inc."~"Brineura (cerliponase alfa)"~4/27/2017 0:00:00~"Original"~1~"3207-1"~"PMR"~"505 (o)(3)"~3207.00~1~"PMR 3207-1: Conduct an observational post approval safety study (Study 190-501) to evaluate the long-term safety of Brineura (cerliponase alfa) in patients with neuronal  ceroid lipofuscinosis Type 2 (CLN2 disease), and further assess the occurrence of serious hypersensitivity reactions (including anaphylaxis), serious cardiovascular adverse events, and serious device related complications in patients followed for a minimum of ten years. In addition, this study will evaluate the effects of serious adverse events on patient performance on the CLN2 motor and language clinical scales."~"Ongoing"~~6/30/2037 0:00:00~6/26/2026 0:00:00
385635~"CDER"~"BLA"~761052.00~"Biomarin Pharmaceutical Inc."~"Brineura (cerliponase alfa)"~4/27/2017 0:00:00~"Original"~1~"3207-6"~"PMC"~"506B PMC"~3207.00~6~"PMC 3207-6: For patients in Studies 190-501 and 190-203, obtain a blood sample prior to cerliponase alfa treatment to determine TPP1 enzyme activity at baseline and  collect TPP1 genotype information. Evaluate the association of enzyme activity with efficacy and safety data from PMRs 3207-1 and 3207-5. Derive the predicted protein function from the TPP1 genotype for each patient, and compare efficacy and safety in patients with different TPP1 genotypes based on their  predicted protein function. In addition, perform similar analyses using a combined dataset from 4 clinical studies, including Studies 190-203, 190-501, 190-201 and 190-202."~"Ongoing"~~12/31/2037 0:00:00~6/26/2026 0:00:00
385636~"CDER"~"NDA"~218171.00~"XCOVERY HOLDINGS INC"~"Ensacove (ensartinib) Capsules"~12/18/2024 0:00:00~"Original"~1~"4752-2"~"PMR"~"505 (o)(3)"~4752.00~2~"PMR 4752-2: Conduct a dedicated drug-drug interaction study to assess the effect of a strong CYP3A inhibitor and a P-gp inhibitor on the pharmacokinetics of ensartinib, to assess the serious potential risk of increased drug toxicity. For greater detail regarding the study design, data analysis and study report, refer to the FDA guidance for industry,  M12 Drug Interaction Studies ."~"Delayed"~"The final protocol milestone was missed due to a change in the study approach."~6/30/2026 0:00:00~2/13/2026 0:00:00
385637~"CDER"~"NDA"~218171.00~"XCOVERY HOLDINGS INC"~"Ensacove (ensartinib) Capsules"~12/18/2024 0:00:00~"Original"~1~"4752-3"~"PMR"~"505 (o)(3)"~4752.00~3~"PMR 4752-3: Conduct a clinical study to assess the effect of moderate and severe hepatic impairment on the pharmacokinetics of ensartinib, to assess the serious potential risk of increased drug toxicity, to inform appropriate dose recommendations in patients with hepatic impairment. For details regarding the study design, data analysis and study report, refer to the FDA guidance for industry,  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling ."~"Delayed"~"The trial completion milestone was missed, because recruitment was slower than anticipated."~6/30/2026 0:00:00~2/13/2026 0:00:00
385638~"CDER"~"NDA"~218171.00~"XCOVERY HOLDINGS INC"~"Ensacove (ensartinib) Capsules"~12/18/2024 0:00:00~"Original"~1~"4752-4"~"PMC"~"506B PMC"~4752.00~4~"PMC 4752-4: Conduct a dedicated drug-drug interaction study to assess the effect of a strong CYP3A inducer on the pharmacokinetics of ensartinib. For greater details regarding the study design, data analysis and study report, refer to the FDA guidance for industry,  M12 Drug Interaction Studies ."~"Delayed"~"The final protocol milestone was missed due to a change in the study approach."~6/30/2026 0:00:00~2/13/2026 0:00:00
385639~"CDER"~"NDA"~218171.00~"XCOVERY HOLDINGS INC"~"Ensacove (ensartinib) Capsules"~12/18/2024 0:00:00~"Original"~1~"4752-5"~"PMC"~"506B PMC"~4752.00~5~"PMC 4752-5: Conduct an integrated analysis of data from clinical trials and observational studies (e.g., real world evidence), post-marketing reports, and other sources to further characterize the safety and efficacy/effectiveness of ensartinib in older adults ages 65 years and older, and in patients of underrepresented racial and ethnic minority groups, with locally advanced or metastatic ALK positive non-small cell lung cancer. The analyses should support an evaluation of comparative efficacy/effectiveness and safety between the population primarily represented in the trial (eXALT3) and the aforementioned underrepresented racial and ethnic minority population as well as between younger patients represented in eXALT3 and the aforementioned older adult population."~"Ongoing"~~6/30/2031 0:00:00~2/13/2026 0:00:00
385640~"CDER"~"NDA"~218171.00~"XCOVERY HOLDINGS INC"~"Ensacove (ensartinib) Capsules"~12/18/2024 0:00:00~"Original"~1~"4752-6"~"PMC"~"506B PMC"~4752.00~6~"PMC 4752-6: Conduct an appropriate analytical and clinical validation study to support the development of an in vitro diagnostic device using clinical trial data that demonstrates that the device is essential to the safe and effective use of ensartinib for the treatment of patients with ALK-positive locally advanced or metastatic non-small cell lung cancer."~"Fulfilled"~~7/31/2025 0:00:00~2/13/2026 0:00:00
385641~"CDER"~"NDA"~218197.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Truqap (capivasertib)"~11/16/2023 0:00:00~"Original"~1~"4548-1"~"PMR"~"Pediatric Research Equity Act"~4548.00~1~"PMR 4548-1: Conduct a clinical study of capivasertib in combination with one or more active drugs to evaluate dosing, pharmacokinetics, safety, and preliminary efficacy of capivasertib in combination with other active agents in a sufficient number of pediatric patients 2 years of age and older with relapsed or refractory cancer harboring AKT pathway gene alterations."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~7/31/2028 0:00:00~1/13/2026 0:00:00
385642~"CDER"~"NDA"~218197.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Truqap (capivasertib)"~11/16/2023 0:00:00~"Original"~1~"4548-2"~"PMR"~"Pediatric Research Equity Act"~4548.00~2~"PMR 4548-2: Develop an age appropriate pediatric formulation of capivasertib and conduct a clinical study of this pediatric formulation in combination with one or more active drugs to evaluate dosing, pharmacokinetics, safety, and preliminary efficacy in a sufficient number of pediatric patients 6 months of age and older with relapsed or refractory cancer harboring AKT pathway gene alterations."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2029 0:00:00~1/13/2026 0:00:00
385643~"CDER"~"NDA"~218197.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Truqap (capivasertib)"~11/16/2023 0:00:00~"Original"~1~"4548-3"~"PMR"~"505 (o)(3)"~4548.00~3~"PMR 4548-3: Conduct a hepatic impairment clinical trial to evaluate the pharmacokinetics and safety of capivasertib in patients with moderate hepatic impairment to assess for potential increase in capivasertib exposure and serious risk of increased adverse reactions. Design and conduct the trial in accordance with the FDA Guidance for Industry titled:  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. "~"Pending"~~6/30/2027 0:00:00~1/13/2026 0:00:00
385644~"CDER"~"NDA"~218197.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Truqap (capivasertib)"~11/16/2023 0:00:00~"Original"~1~"4548-4"~"PMR"~"505 (o)(3)"~4548.00~4~"PMR 4548-4: Conduct a clinical drug interaction study to evaluate the effect of repeat doses of capivasertib on the pharmacokinetics and safety of transporter substrates of BCRP, OATP1B1 and OATP1B3, to assess for potential serious risk associated with the magnitude of exposure change and inform appropriate drug interaction management strategy for coadministration of capivasertib with these transporter substrates. This trial should be designed and conducted in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. "~"Delayed"~"The final protocol milestone was missed because of ongoing discussions."~6/30/2027 0:00:00~1/13/2026 0:00:00
385645~"CDER"~"NDA"~218197.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Truqap (capivasertib)"~11/16/2023 0:00:00~"Original"~1~"4548-5"~"PMC"~"506B PMC"~4548.00~5~"PMC 4548-5: Conduct an integrated analysis of data from clinical trials and other data sources such as post-marketing reports, real-world evidence and other sources to further characterize the safety and efficacy of capivasertib in combination with fulvestrant in patients of underrepresented racial and ethnic minority groups with hormone receptor positive, HER2 negative metastatic breast cancer with one or more PIK3CA/AKT1/PTENalterations. The analyses should include safety and efficacy outcome analyses by race and ethnicity."~"Pending"~~7/31/2029 0:00:00~1/13/2026 0:00:00
385646~"CDER"~"NDA"~218197.00~"ASTRAZENECA PHARMACEUTICALS LP"~"Truqap (capivasertib)"~11/16/2023 0:00:00~"Original"~1~"4548-6"~"PMC"~"506B PMC"~4548.00~6~"PMC 4548-6: Complete the ongoing CAPItello-291 study entitled  A Phase III Doubleblind Randomised Study Assessing the Efficacy and Safety of Capivasertib + Fulvestrant Versus Placebo + Fulvestrant as Treatment for Locally Advanced (Inoperable) or Metastatic Hormone Receptor Positive, Human Epidermal Growth Factor Receptor 2 Negative (HR+/HER2-) Breast Cancer Following Recurrence or Progression On or After Treatment with an Aromatase Inhibitor , to obtain the final overall survival (OS) analysis, to further describe the clinical benefit of capivasertib."~"Submitted"~~10/31/2025 0:00:00~1/13/2026 0:00:00
385647~"CDER"~"BLA"~761224.00~"AstraZeneca AB"~"Tezspire (tezepelumab-ekko)"~12/17/2021 0:00:00~"Original"~1~"4188-2"~"PMR"~"Pediatric Research Equity Act"~4188.00~2~"PMR 4188-2: Conduct a 52-week efficacy and safety study in children 5 to < 12 years of age with severe asthma."~"Ongoing"~"The study has been initiated."~9/30/2028 0:00:00~2/12/2026 0:00:00
385648~"CDER"~"BLA"~761224.00~"AstraZeneca AB"~"Tezspire (tezepelumab-ekko)"~12/17/2021 0:00:00~"Original"~1~"4188-3"~"PMR"~"Pediatric Research Equity Act"~4188.00~3~"PMR 4188-3: Conduct an efficacy, safety, and PK study in children 2 years to < 5 years of age with severe asthma with a continued safety evaluation out to a minimum of 52 weeks."~"Pending"~"The study has not begun but does not meet the criterion for delayed."~9/30/2036 0:00:00~2/12/2026 0:00:00
385649~"CDER"~"BLA"~761234.00~"Bristol-Myers Squibb Company"~"Opdualag (nivolumab and relatlimab-rmbw)"~3/18/2022 0:00:00~"Original"~1~"4222-1"~"PMR"~"Pediatric Research Equity Act"~4222.00~1~"PMR 4222-1: Conduct Study CA224069 (A Phase 1/2 Study of the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Relatlimab Plus Nivolumab in Pediatric and Young Adult Participants with Recurrent or Refractory Classical Hodgkin Lymphoma and Non-Hodgkin Lymphoma) to further characterize the safety, pharmacokinetics, pharmacodynamics and efficacy of relatlimab in combination with nivolumab in participants 0 to <30 years of age with relapsed or refractory Hodgkin lymphoma and an exploratory assessment in Non-Hodgkin lymphoma. Include at least 6 patients 0-11 years old and 6 patients 12-17 years old."~"Ongoing"~"5 patients out of the planned 84 patients have been enrolled
into the study."~9/30/2028 0:00:00~5/8/2026 0:00:00
385650~"CDER"~"BLA"~761234.00~"Bristol-Myers Squibb Company"~"Opdualag (nivolumab and relatlimab-rmbw)"~3/18/2022 0:00:00~"Original"~1~"4222-2"~"PMR"~"Pediatric Research Equity Act"~4222.00~2~"PMR 4222-2: Conduct a study, Study 2 (Modeling and Simulation/Extrapolation Study), to further characterize the pharmacokinetics and evaluate the dose regimen of nivolumab and relatlimab combination therapy in pediatric lymphoma."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~2/28/2029 0:00:00~5/8/2026 0:00:00
385651~"CDER"~"BLA"~761235.00~"Genentech, Inc."~"Vabysmo (faricimab-svoa)"~1/28/2022 0:00:00~"Original"~1~"4214-1"~"PMR"~"505 (o)(3)"~4214.00~1~"PMR 4214-1: Conduct a controlled trial to evaluate the corneal endothelial health of eyes treated with faricimab by monitoring the number/density of corneal endothelial cells using specular microscopy at baseline and over a period of at least one year in at least 100 patients receiving faricimab."~"Fulfilled"~~4/30/2025 0:00:00~3/24/2026 0:00:00
385652~"CDER"~"BLA"~761238.00~"TG Therapeutics, Inc."~"Briumvi (ublituximab-xiiy)"~12/28/2022 0:00:00~"Original"~1~"4337-3"~"PMR"~"505 (o)(3)"~4337.00~3~"PMR 4337-3: Prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with multiple sclerosis exposed to Briumvi (ublituximab-xiiy) during pregnancy with two unexposed control populations: one consisting of women with multiple sclerosis who have not been exposed to Briumvi (ublituximab-xiiy) before or during pregnancy and the other consisting of women without multiple sclerosis. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-for-gestational-age births, and any other adverse outcomes, including postnatal growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~3/31/2036 0:00:00~2/25/2026 0:00:00
385653~"CDER"~"BLA"~761238.00~"TG Therapeutics, Inc."~"Briumvi (ublituximab-xiiy)"~12/28/2022 0:00:00~"Original"~1~"4337-4"~"PMR"~"505 (o)(3)"~4337.00~4~"PMR 4337-4: A pregnancy outcomes study using a different study design than provided for in PMR 4337-2 (for example, a retrospective cohort study using claims or electronic medical record data with outcome validation or a case-control study) to assess pregnancy complications, major congenital malformations, spontaneous abortions, stillbirths, preterm births, and small-for-gestational-age births in women exposed to Briumvi (ublituximab-xiiy) during pregnancy compared to an unexposed control population."~"Ongoing"~~3/31/2036 0:00:00~2/25/2026 0:00:00
385654~"CDER"~"BLA"~761238.00~"TG Therapeutics, Inc."~"Briumvi (ublituximab-xiiy)"~12/28/2022 0:00:00~"Original"~1~"4337-5"~"PMR"~"505 (o)(3)"~4337.00~5~"PMR 4337-5: Perform a lactation study (milk only) in lactating women who have received therapeutic doses of Briumvi (ublituximab-xiiy) using a validated assay to assess concentrations of Briumvi (ublituximab-xiiy) in breast milk and the effects on the breastfed infant."~"Delayed"~"The Study Completion milestone was missed and  FDA determined that there was good cause in a letter dated 07/29/2025.  The anticipated delay of the Final Report Submission milestone was also acknowledged in that letter. Per the applicant s Annual Status Report, 4 out of the planned 10 patients have been enrolled."~1/31/2026 0:00:00~2/25/2026 0:00:00
385655~"CDER"~"BLA"~761238.00~"TG Therapeutics, Inc."~"Briumvi (ublituximab-xiiy)"~12/28/2022 0:00:00~"Original"~1~"4337-6"~"PMR"~"505 (o)(3)"~4337.00~6~"PMR 4337-6: A safety trial to monitor serum immunoglobulin G and M levels in patients with relapsing forms of multiple sclerosis during treatment with Briumvi (ublituximab-xiiy) to establish the nadir in circulating immunoglobulins during chronic treatment, and to monitor patients after discontinuation of treatment with Briumvi (ublituximab-xiiy) in order to ascertain the time needed to ensure restoration of pretreatment baseline circulating serum levels of immunoglobulins G and M. This trial also should be designed to capture rates of infections, especially opportunistic and recurrent infections associated with immune suppression, and there should be monitoring of B-cell counts throughout treatment and after discontinuation until repletion of immunoglobulin levels."~"Ongoing"~~6/30/2031 0:00:00~2/25/2026 0:00:00
385656~"CDER"~"BLA"~761238.00~"TG Therapeutics, Inc."~"Briumvi (ublituximab-xiiy)"~12/28/2022 0:00:00~"Original"~1~"4337-11"~"PMR"~"Pediatric Research Equity Act"~4337.00~11~"PMR 4337-11: Conduct a two-part study of Briumvi (ublituximab-xiiy) in pediatric patients with relapsing forms of multiple sclerosis (RMS) at least 10 years and less than 18 years of age. Part A is an open-label study of the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Briumvi (ublituximab-xiiy) in pediatric patients weighing 40 kg or less. The objective of Part A is to determine maintenance doses of Briumvi (ublituximab-xiiy) that will result in PK and PD effects that are comparable to those of the dose administered to adult patients. Part B is a randomized, blinded, non-inferiority trial with Gilenya (fingolimod) as a comparator.
"~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~9/30/2030 0:00:00~2/25/2026 0:00:00
385657~"CDER"~"NDA"~219008.00~"JANSSEN BIOTECH INC"~"Lazcluze (lazertinib)"~8/19/2024 0:00:00~"Original"~1~"4681-2"~"PMC"~"506B PMC"~4681.00~2~"PMC 4681-2: Complete the MARIPOSA trial and include the results of the final overall survival analysis once the anticipated 390 death events have occurred in the amivantamab in combination with lazertinib and osimertinib arms to further characterize the clinical benefit of amivantamab in combination with lazertinib for the first line treatment of adult patients with metastatic NSCLC harboring EGFR exon 19 deletion or L858R mutations."~"Fulfilled"~~11/30/2025 0:00:00~10/14/2025 0:00:00
385658~"CDER"~"NDA"~219008.00~"JANSSEN BIOTECH INC"~"Lazcluze (lazertinib)"~8/19/2024 0:00:00~"Original"~1~"4681-3"~"PMC"~"506B PMC"~4681.00~3~"PMC 4681-3: Conduct an integrated analysis containing data from clinical trials and other data sources such as post-marketing reports, real-world evidence and other sources to further characterize the safety and efficacy of amivantamab in combination with lazertinib in patients who-are Black or African American diagnosed with metastatic NSCLC harboring EGFR exon 19 deletions or exon 21 L858R substitution mutations."~"Pending"~~6/30/2029 0:00:00~10/14/2025 0:00:00
385659~"CDER"~"NDA"~219042.00~"BOEHRINGER INGELHEIM PHARMACEUTICALS INC"~"Hernexeos (zongertinib) tablets"~8/8/2025 0:00:00~"Original"~1~"4881-1"~"PMR"~"Accelerated Approval"~4881.00~1~"PMR 4881-1: Complete the ongoing multicenter, randomized clinical trial, Study Beamion LUNG-2, intended to verify and describe the clinical benefit of zongertinib in adult patients with advanced, unresectable or metastatic non-squamous non-small cell lung cancer whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~11/30/2029 0:00:00~
385660~"CDER"~"NDA"~219042.00~"BOEHRINGER INGELHEIM PHARMACEUTICALS INC"~"Hernexeos (zongertinib) tablets"~8/8/2025 0:00:00~"Original"~1~"4881-2"~"PMC"~"506B PMC"~4881.00~2~"PMC 4881-2: Conduct a clinical drug-drug interaction study to evaluate the effects of a moderate CYP3A inducer on the pharmacokinetics of zongertinib. Collect pharmacokinetic information for zongertinib and utilize this information to determine the appropriate dose of zongertinib when co-administered with CYP3A inducers."~"Submitted"~~2/28/2027 0:00:00~
385661~"CDER"~"NDA"~219042.00~"BOEHRINGER INGELHEIM PHARMACEUTICALS INC"~"Hernexeos (zongertinib) tablets"~8/8/2025 0:00:00~"Supplement"~1~"4881-1"~"PMR"~"Accelerated Approval"~4881.00~1~"PMR 4881-1: Complete the ongoing multicenter, randomized clinical trial, Study Beamion LUNG-2, intended to verify and describe the clinical benefit of zongertinib in adult patients with advanced, unresectable or metastatic non-squamous non-small cell lung cancer whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~11/30/2029 0:00:00~
385662~"CDER"~"NDA"~219042.00~"BOEHRINGER INGELHEIM PHARMACEUTICALS INC"~"Hernexeos (zongertinib) tablets"~8/8/2025 0:00:00~"Supplement"~1~"4881-2"~"PMC"~"506B PMC"~4881.00~2~"PMC 4881-2: Conduct a clinical drug-drug interaction study to evaluate the effects of a moderate CYP3A inducer on the pharmacokinetics of zongertinib. Collect pharmacokinetic information for zongertinib and utilize this information to determine the appropriate dose of zongertinib when co-administered with CYP3A inducers."~"Submitted"~~2/28/2027 0:00:00~
385663~"CDER"~"NDA"~219104.00~"MERCK SHARP AND DOHME LLC"~"Prevymis (letermovir)"~8/30/2024 0:00:00~"Original"~1~"4692-1"~"PMR"~"505 (o)(3)"~4692.00~1~"PMR 4692-1: Conduct phenotypic analysis of letermovir against human cytomegalovirus (HCMV) mutants carrying substitutions (1) pUL51 P91H and (2) pUL56 S229Y + pUL56 M329I with and without pUL89 D344Y. The analysis should include previously identified substitutions with a range of susceptibilities from low fold change (e.g. pUL56 L257I) to high fold change (e.g. pUL56 C325Y) as references."~"Fulfilled"~~4/30/2026 0:00:00~12/17/2025 0:00:00
385664~"CDER"~"NDA"~219104.00~"MERCK SHARP AND DOHME LLC"~"Prevymis (letermovir)"~8/30/2024 0:00:00~"Original"~1~"4693-1"~"PMC"~"506B PMC"~4693.00~1~"PMC 4693-1: Conduct a study to determine the pharmacokinetics and safety of letermovir in kidney transplant recipients less than 18 years of age and weighing less than 40 kg."~"Pending"~~1/31/2029 0:00:00~12/17/2025 0:00:00
385665~"CDER"~"NDA"~219132.00~"INTRABIO INC"~"Aqneursa (levacetylleucine)"~9/24/2024 0:00:00~"Original"~1~"4683-1"~"PMR"~"505 (o)(3)"~4683.00~1~"PMR 4683-1: Conduct an embryo-fetal development (EFD) toxicity and toxicokinetic study with levacetylleucine in the rodent."~"Fulfilled"~~12/31/2024 0:00:00~11/21/2025 0:00:00
385666~"CDER"~"NDA"~219132.00~"INTRABIO INC"~"Aqneursa (levacetylleucine)"~9/24/2024 0:00:00~"Original"~1~"4683-2"~"PMR"~"505 (o)(3)"~4683.00~2~"PMR 4683-2: Conduct an embryo-fetal development toxicity and toxicokinetic study with levacetylleucine in the non-rodent"~"Fulfilled"~~12/31/2024 0:00:00~11/21/2025 0:00:00
385667~"CDER"~"NDA"~219132.00~"INTRABIO INC"~"Aqneursa (levacetylleucine)"~9/24/2024 0:00:00~"Original"~1~"4683-3"~"PMR"~"505 (o)(3)"~4683.00~3~"PMR 4683-3: Conduct a juvenile toxicity and toxicokinetic study with levacetylleucine."~"Submitted"~~5/31/2025 0:00:00~11/21/2025 0:00:00
385668~"CDER"~"NDA"~219132.00~"INTRABIO INC"~"Aqneursa (levacetylleucine)"~9/24/2024 0:00:00~"Original"~1~"4683-4"~"PMR"~"505 (o)(3)"~4683.00~4~"PMR 4683-4: Conduct a fertility and early embryonic development (FEED) toxicity study with levacetylleucine."~"Fulfilled"~~12/31/2024 0:00:00~11/21/2025 0:00:00
385669~"CDER"~"NDA"~219132.00~"INTRABIO INC"~"Aqneursa (levacetylleucine)"~9/24/2024 0:00:00~"Original"~1~"4683-5"~"PMR"~"505 (o)(3)"~4683.00~5~"PMR 4683-5: Conduct a pre- and postnatal development (PPND) toxicity study with levacetylleucine."~"Fulfilled"~~1/31/2025 0:00:00~11/21/2025 0:00:00
385670~"CDER"~"NDA"~219132.00~"INTRABIO INC"~"Aqneursa (levacetylleucine)"~9/24/2024 0:00:00~"Original"~1~"4683-6"~"PMR"~"505 (o)(3)"~4683.00~6~"PMR 4683-6: Conduct a 26-week carcinogenicity study with levacetylleucine, administered by daily oral gavage, in an appropriate transgenic mouse model."~"Ongoing"~~3/31/2027 0:00:00~11/21/2025 0:00:00
385671~"CDER"~"NDA"~219132.00~"INTRABIO INC"~"Aqneursa (levacetylleucine)"~9/24/2024 0:00:00~"Original"~1~"4683-7"~"PMR"~"505 (o)(3)"~4683.00~7~"PMR 4683-7: Conduct a long-term (2-year) GLP-complaint carcinogenicity study by oral gavage administration in rats."~"Released"~~6/30/2029 0:00:00~11/21/2025 0:00:00
385672~"CDER"~"NDA"~219132.00~"INTRABIO INC"~"Aqneursa (levacetylleucine)"~9/24/2024 0:00:00~"Original"~1~"4683-8"~"PMR"~"505 (o)(3)"~4683.00~8~"PMR 4683-8: Conduct a clinical drug-drug interaction (DDI) trial in healthy volunteers to evaluate the effect of levacetylleucine on a probe
substrate of P-gp to thoroughly assess its inhibitory potential and its impact on patient safety. Design and conduct the trial in
accordance with FDA guidance for industry Clinical Drug Interaction Studies   Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions (January 2020)."~"Ongoing"~~9/30/2027 0:00:00~11/21/2025 0:00:00
385673~"CDER"~"NDA"~219132.00~"INTRABIO INC"~"Aqneursa (levacetylleucine)"~9/24/2024 0:00:00~"Original"~1~"4683-9"~"PMC"~"506B PMC"~4683.00~9~"PMC 4683-9: Conduct a dedicated food effect trial to evaluate the impact of food on exposure of levacetylleucine granules for oral suspension in
accordance with FDA guidance for industry Assessing the Effects of Food on Drugs in INDs and NDAs   Clinical Pharmacology Considerations Guidance for Industry (June 2022)"~"Ongoing"~~3/31/2026 0:00:00~11/21/2025 0:00:00
385674~"CDER"~"NDA"~219155.00~"LEO PHARMA AS"~"Anzupgo (delgocitinib) cream"~7/23/2025 0:00:00~"Original"~1~"4877-1"~"PMR"~"Pediatric Research Equity Act"~4877.00~1~"PMR 4877-1: Conduct an assessment of the efficacy, safety and pharmacokinetics of delgocitinib cream in adolescent subjects ages 12 to 17 years with moderate to severe chronic hand eczema."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~10/31/2025 0:00:00~
385675~"CDER"~"NDA"~219155.00~"LEO PHARMA AS"~"Anzupgo (delgocitinib) cream"~7/23/2025 0:00:00~"Original"~1~"4877-2"~"PMR"~"505 (o)(3)"~4877.00~2~"PMR 4877-2: Conduct a milk-only lactation study in lactating women who have received delgocitinib to measure concentrations of delgocitinib in breast milk using a validated assay. Assess the effects on the breastfed infant, if available, based on the study population."~"Pending"~~1/31/2029 0:00:00~
385676~"CDER"~"BLA"~761053.00~"Genentech, Inc."~"Ocrevus (Ocrelizumab)"~3/28/2017 0:00:00~"Original"~1~"3194-2"~"PMR"~"505 (o)(3)"~3194.00~2~"PMR 3194-2: Conduct a prospective longitudinal observational study in adult patients with relapsing multiple sclerosis and primary progressive multiple sclerosis exposed to  Ocrevus (ocrelizumab) to determine the incidence and mortality rates of breast cancer and all malignancies. All patients enrolled in the study should be followed for a minimum of 5 years or until death following their first exposure to Ocrevus. The protocol must specify two appropriate populations to which the observed  incidence and mortality rates will be compared."~"Delayed"~"The Final Protocol was submitted after the original protocol
submission milestone. The final protocol was acknowledged in a letter dated 04/04/2019. Enrollment for the study closed on 03/31/2022; the study population was 4,092 ocrelizumab-exposed patients and 1,270 patients in the comparator cohort."~11/30/2030 0:00:00~5/22/2026 0:00:00
385677~"CDER"~"BLA"~761053.00~"Genentech, Inc."~"Ocrevus (Ocrelizumab)"~3/28/2017 0:00:00~"Original"~1~"3194-3"~"PMR"~"505 (o)(3)"~3194.00~3~"PMR 3194-3: Conduct prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with  multiple sclerosis exposed to ocrelizumab during pregnancy with two unexposed control populations: one consisting of women with multiple sclerosis who have not been exposed to ocrelizumab before or during pregnancy and the other consisting of women without multiple sclerosis. The registry will identify and record pregnancy complications, major and minor congenital malformations, spontaneous abortions, stillbirths, elective terminations, preterm births, small-forgestational-  age births, and any other adverse outcomes, including postnatal  growth and development. Outcomes will be assessed throughout pregnancy. Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Submitted"~~10/31/2029 0:00:00~5/22/2026 0:00:00
385678~"CDER"~"BLA"~761053.00~"Genentech, Inc."~"Ocrevus (Ocrelizumab)"~3/28/2017 0:00:00~"Original"~1~"3194-4"~"PMR"~"505 (o)(3)"~3194.00~4~"PMR 3194-4: Conduct a pregnancy outcomes study using a different study design than provided for in PMR 3194-3 (for example, a retrospective cohort study using claims or electronic medical record data or a case control study) to assess major congenital malformations, spontaneous abortions, stillbirths, and small-for-gestational-age births in women exposed to ocrelizumab during pregnancy compared to an unexposed control population."~"Delayed"~"The Study Completion and the Final Report milestones were missed because of ongoing negotiations with the FDA pertaining to the protocol. FDA determined that there was good cause for the delay and acknowledged the revised milestone(s) in a letter dated 1/15/2020."~3/31/2024 0:00:00~5/22/2026 0:00:00
385679~"CDER"~"BLA"~761053.00~"Genentech, Inc."~"Ocrevus (Ocrelizumab)"~3/28/2017 0:00:00~"Original"~1~"3194-14"~"PMR"~"Pediatric Research Equity Act"~3194.00~14~"PMR 3194-14: Conduct a two-part study of ocrelizumab in pediatric patients with relapsing multiple sclerosis (RMS) at least 10 years and less than 17 years of age. Part A is an open-label study of the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ocrelizumab in pediatric patients weighing 40 kg or less. The objective of Part A is to determine a dose of ocrelizumab that will result in PK and PD effects that are comparable to those of a 600 mg dose (300 mg given twice 14 days apart) in adult patients with RMS. Safety assessments will continue for at least 2 years after the last dose of ocrelizumab. Part B is a randomized, double-blind, parallel-group study to evaluate the efficacy and safety of ocrelizumab compared to an appropriate comparator."~"Fulfilled"~"Per FDA letter dated 05/08/2026, this PMR/PMC has been fulfilled."~11/30/2025 0:00:00~5/22/2026 0:00:00
385680~"CDER"~"BLA"~761055.00~"Regeneron Pharmaceuticals, Inc."~"Dupixent (dupilumab)"~3/28/2017 0:00:00~"Original"~1~"3183-3"~"PMR"~"Pediatric Research Equity Act"~3183.00~3~"PMR 3183-3: Conduct an open-label study to characterize the long-term safety (at least 1 year) of dupilumab in pediatric subjects 6 months to less than 18 years with moderate and/or severe atopic dermatitis."~"Delayed"~"The original study completion and the final report submission milestones were missed. Original Final Report Due Date: 03/31/2023; Deferral Extension granted per FDA letter dated 09/24/2025. The study completion milestone was also revised.
"~4/30/2027 0:00:00~5/23/2025 0:00:00
385681~"CDER"~"BLA"~761055.00~"Regeneron Pharmaceuticals, Inc."~"Dupixent (dupilumab)"~3/28/2017 0:00:00~"Original"~1~"3183-5"~"PMR"~"505 (o)(3)"~3183.00~5~"PMR 3183-5: A prospective, registry-based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to dupilumab during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths,  elective terminations, small for gestational age births, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy.  Infant outcomes, including effects on postnatal growth and development, will be assessed through at least the first year of life."~"Delayed"~"The original study completion milestone was missed. The Study Completion and Final Report milestones were revised to allow follow-up and complete record capture for the last infant enrolled who was born in September 2024 (PMR 3183-5) and for the procurement and adjudication of medical charts (PMR 3183-6).  FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 09/23/2025."~7/31/2026 0:00:00~5/23/2025 0:00:00
385682~"CDER"~"BLA"~761055.00~"Regeneron Pharmaceuticals, Inc."~"Dupixent (dupilumab)"~3/28/2017 0:00:00~"Original"~1~"3183-6"~"PMR"~"505 (o)(3)"~3183.00~6~"PMR 3183-6: Conduct a retrospective cohort study using administrative databases to identify pregnancy outcomes in a cohort of women exposed to dupilumab and a nondupilumab systemic medication or phototherapy exposure cohort. The outcomes  will include major congenital malformations, spontaneous abortions, stillbirths, and small for gestational age births. This study may use multiple data sources in  order to obtain a sufficient sample size as women with atopic dermatitis are counseled to avoid systemic treatments while trying to conceive and during the course of pregnancy."~"Delayed"~"The Study Completion and Final Report milestones were missed. FDA determined that there was good cause for the delay and acknowledged the revised milestones in a letter dated 09/23/2025. The final protocol was acknowledged in a letter dated 03/23/2026."~10/31/2026 0:00:00~5/23/2025 0:00:00
385683~"CDER"~"BLA"~761055.00~"Regeneron Pharmaceuticals, Inc."~"Dupixent (dupilumab)"~3/28/2017 0:00:00~"Supplement"~7~"3508-2"~"PMR"~"Pediatric Research Equity Act"~3508.00~2~"PMR 3508-2: Conduct a safety and efficacy study with dupilumab in children 2 years to less than 6 years of age with moderate to severe asthma with a continued safety evaluation out to a minimum of 52 weeks (Study EFC14771)."~"Ongoing"~"Original Final Report Due Date: 06/30/2027; Deferral Extension granted per FDA letter dated 04/10/2026."~10/31/2027 0:00:00~5/23/2025 0:00:00
385684~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Original"~1~"4547-1"~"PMR"~"Pediatric Research Equity Act"~4547.00~1~"PMR 4547-1: Conduct a clinical study (ongoing CARE study) to assess the appropriate dose of repotrectinib and to assess safety, tolerability, pharmacokinetics (PK), and efficacy of repotrectinib, in pediatric and young adult patients with advanced or metastatic solid tumors, primary central nervous system (CNS) tumors, or anaplastic large cell lymphoma (ALCL), with ALK, ROS1, or NTRK alterations. At least 3 patients 6 years of age or younger will be evaluated in the dose-finding phase."~"Ongoing"~"47 patients out of the planned 63 patients have been enrolled into
the trial."~3/31/2027 0:00:00~1/9/2026 0:00:00
385685~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Original"~1~"4547-2"~"PMR"~"505 (o)(3)"~4547.00~2~"PMR 4547-2: Conduct a prospective study to evaluate risk factors, manifestations, and outcomes associated with the signal of serious ocular toxicity with repotrectinib in patients with ROS1 positive NSCLC or other solid tumors. The study will include collection, grading, classification, and analysis of data on ocular toxicity in patients exposed to repotrectinib. Evaluate a sufficient number of patients treated at the recommended phase 2 dose (160 mg daily for the first 14 days, then increase to 160 mg twice daily) who receive repotrectinib as a single agent. Include baseline ophthalmologic exam with vision test, scheduled follow-up, and symptomdriven ocular assessments to include visual acuity assessments, ophthalmologic evaluations including slit lamp examination, and elicitation for visual symptoms."~"Ongoing"~~8/31/2028 0:00:00~1/9/2026 0:00:00
385686~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Original"~1~"4547-3"~"PMR"~"505 (o)(3)"~4547.00~3~"PMR 4547-3: Conduct a clinical pharmacokinetic trial in non-cancer hepatically impaired subjects to evaluate the effect of moderate and severe hepatic impairment on the single dose pharmacokinetics and safety of repotrectinib. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling.  In addition, conduct a physiologically based pharmacokinetic (PBPK) modeling study, using data from the clinical trial, to evaluate the effect of hepatic impairment on multiple dose pharmacokinetics of repotrectinib, to determine an appropriate dosage of repotrectinib, and to identify and assess the potential serious risk of increased drug toxicity, in patients with moderate and severe hepatic impairment. Design and conduct the modeling study in accordance with FDA Guidance for industry entitled  Physiologically Based Pharmacokinetic Analyses - Format and Content. "~"Ongoing"~~6/30/2026 0:00:00~1/9/2026 0:00:00
385687~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Original"~1~"4547-4"~"PMR"~"505 (o)(3)"~4547.00~4~"PMR 4547-4: Conduct a clinical pharmacokinetic trial to evaluate the effects of multiple doses of a specific strong CYP3A inhibitor and a specific P-gp inhibitor, respectively on the single-dose pharmacokinetics and safety of repotrectinib. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions. In addition, conduct a physiologically based pharmacokinetic (PBPK) modeling study, using data from the clinical trial, to evaluate the effects of a specific strong CYP3A inhibitor, a specific moderate CYP3A inhibitor, a specific P-gp inhibitor, and a dual P-gp and moderate CYP3A inhibitor, respectively on the multiple-dose pharmacokinetics of repotrectinib to identify and assess the potential serious risk of increased drug toxicity and to identify appropriate dosage recommendations for repotrectinib when used concomitantly with these inhibitors. Design and conduct the modeling study in accordance with FDA Guidance for industry entitled,  In Vitro Metabolism- and Transporter-Mediated Drug-Drug Interaction Studies  and  Physiologically Based Pharmacokinetic Analyses - Format and Content. "~"Ongoing"~~6/30/2026 0:00:00~1/9/2026 0:00:00
385688~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Original"~1~"4547-5"~"PMR"~"505 (o)(3)"~4547.00~5~"PMR 4547-5: Conduct a clinical pharmacokinetic trial to evaluate the effect of multiple doses of repotrectinib on the single dose pharmacokinetics of a substrate of MATE2-K, P-gp, OATP1B1, and BCRP, respectively to identify and assess the potential serious risk of increased drug toxicity with repotrectinib. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions ."~"Ongoing"~~7/31/2029 0:00:00~1/9/2026 0:00:00
385689~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Original"~1~"4547-7"~"PMC"~"506B PMC"~4547.00~7~"PMC 4547-7: Conduct a physiologically based pharmacokinetic (PBPK) modeling study, using data from the clinical trials with specific CYP3A and P-gp inhibitors, to evaluate the effect of multiple doses of a moderate CYP3A inducer on the multiple-dose pharmacokinetics of repotrectinib to assess the magnitude of decreased drug exposure with appropriate dosage recommendations of repotrectinib when concomitantly used with moderate CYP3A inducers. Design and conduct the modeling study in accordance with FDA Guidance for industry entitled,  In Vitro Metabolism- and Transporter-Mediated Drug-Drug Interaction Studies  and  Physiologically Based Pharmacokinetic Analyses - Format and Content. "~"Ongoing"~~6/30/2026 0:00:00~1/9/2026 0:00:00
385690~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Original"~1~"4547-8"~"PMC"~"506B PMC"~4547.00~8~"PMC 4547-8: Conduct a clinical pharmacokinetic trial with repeat doses of repotrectinib on the single dose pharmacokinetics of a substrate of CYP2B6, CYP2C9, and CYP2C19, respectively to assess the magnitude of decreased drug exposure. Design and conduct the trial in accordance with the FDA Guidance for Industry entitled  Clinical Drug Interaction Studies  Cytochrome P450 Enzyme- and Transporter-Mediated Drug Interactions . In addition, conduct a physiologically based pharmacokinetic (PBPK) modeling study to predict impact of repotrectinib on the magnitude of decreased drug exposure of CYP2C8 substrates. Design and conduct the modeling study in accordance with FDA Guidance for industry entitled,  In Vitro Metabolism- and Transporter-Mediated Drug-Drug Interaction Studies  and  Physiologically Based Pharmacokinetic Analyses - Format and Content. "~"Ongoing"~~7/31/2029 0:00:00~1/9/2026 0:00:00
385691~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Original"~1~"4547-9"~"PMC"~"506B PMC"~4547.00~9~"PMC 4547-9: Commitment to support the availability of an in vitro diagnostic device, through an appropriate analytical and clinical validation study using clinical trial data that demonstrates the device is essential to the safe and effective use of repotrectinib for the treatment of adult patients with locally advanced or metastatic ROS1 positive non-small cell lung cancer."~"Submitted"~~12/31/2024 0:00:00~1/9/2026 0:00:00
385692~"CDER"~"BLA"~761240.00~"Coherus BioSciences, Inc."~"Loqtorzi (toripalimab-tpzi)"~10/27/2023 0:00:00~"Original"~1~"4471-1"~"PMC"~"506B PMC"~4471.00~1~"PMC 4471-1: Conduct a clinical trial enrolling a total sample size of 100 patients in the United States (U.S.) and Canada, that includes a sufficient representation of patients in racial and ethnic minority subgroups and is reflective of the U.S. population of patients with nasopharyngeal carcinoma (NPC), to further characterize the safety and efficacy of toripalimab in combination with cisplatin and gemcitabine in these patients. Include a sufficient number of patients with the keratinizing subtype reflecting the incidence of
keratinizing NPC in the U.S. population. Conduct sparse sampling for supportive population pharmacokinetic and Exposure-Response (E-R) analyses. The E-R analyses may be used as supportive evidence for the efficacy and safety in the intended patient population. In the E-R analyses report, include an analysis of the presence and clinical impact of the neutralizing anti-drug antibodies on pharmacokinetics, efficacy and safety of toripalimab."~"Ongoing"~~6/30/2028 0:00:00~11/24/2025 0:00:00
385693~"CDER"~"BLA"~761244.00~"LEO Pharma A/S"~"Spevigo (spesolimab-sbzo)"~9/1/2022 0:00:00~"Original"~1~"4332-1"~"PMR"~"505 (o)(3)"~4332.00~1~"PMR 4332-1: Conduct an open label safety trial to evaluate the ability of antidrug antibodies (ADA) to induce adverse reactions and the immune-mediated loss of pharmacological effect, including reduced effectiveness, of spesolimab when used in the re-treatment of GPP flares that occur after the first flare incidence has been treated and resolved. This trial should also assess the effect of immunogenicity on pharmacokinetics of spesolimab-sbzo."~"Ongoing"~~4/30/2028 0:00:00~10/29/2025 0:00:00
385694~"CDER"~"BLA"~761244.00~"LEO Pharma A/S"~"Spevigo (spesolimab-sbzo)"~9/1/2022 0:00:00~"Original"~1~"4332-3"~"PMR"~"505 (o)(3)"~4332.00~3~"PMR 4332-3: Complete the ongoing trial to obtain data about the safety of re-treatment of Generalized Pustular Psoriasis (GPP) flares: Effisayil-ON): An Openlabel, Long Term Extension Study to Assess the Safety and Efficacy of BI 655130 Treatment in Patients With Generalized Pustular Psoriasis (GPP) (clinicaltrials.gov identifier: NCT03886246, study ID number: 1368-0025."~"Ongoing"~~9/30/2028 0:00:00~10/29/2025 0:00:00
385695~"CDER"~"BLA"~761244.00~"LEO Pharma A/S"~"Spevigo (spesolimab-sbzo)"~9/1/2022 0:00:00~"Original"~1~"4332-4"~"PMC"~"506B PMC"~4332.00~4~"PMC 4332-4: Complete the planned voluntary European Post-Authorization Safety Study (PASS)."~"Ongoing"~~9/30/2029 0:00:00~10/29/2025 0:00:00
385696~"CDER"~"BLA"~761248.00~"Eli Lilly and Company"~"Kisunla (donanemab-azbt)"~7/2/2024 0:00:00~"Original"~1~"4605-1"~"PMR"~"505 (o)(3)"~4605.00~1~"PMR 4605-1: Conduct a registry-based, prospective, observational study to evaluate clinical safety outcomes among Alzheimer s disease patients treated with donanemab, using, for example, the Alzheimer s Network for Treatment and Diagnostics (ALZ-NET) registry, including patients who are ApoE e4 homozygotes, and/or exposed to antithrombotics or thrombolytics, and/or have a diagnosis of, or imaging findings consistent with a high risk for, cerebral amyloid angiopathy. The primary clinical safety outcomes should include amyloid related imaging abnormalities (ARIA)-edema (ARIA-E), and ARIA-hemosiderin deposition (ARIA-H) and any associated clinical symptoms, and intracerebral hemorrhage >1 cm in size. Additional outcomes of interest should also include seizures, anaphylaxis, and death. Baseline characterization of the registry population should include demographic data, diagnosis and stage of disease, ApoE genotype, baseline MRI findings (e.g., microhemorrhages, evidence of cerebral amyloid angiography or other imaging findings consistent with high risk of cerebral amyloid angiography, etc.), other biomarkers that are potential predictors of disease course or adverse outcomes, and prior medications including prior Alzheimer s disease (AD) therapy and antithrombotic therapy. The registry should also collect information on concomitant medications (e.g., antiplatelet and antithrombotic drugs, other AD treatments). When available, the study should provide a comparison of safety outcomes to estimated background rates in an appropriate comparator population."~"Pending"~~2/28/2037 0:00:00~8/29/2025 0:00:00
385697~"CDER"~"BLA"~761248.00~"Eli Lilly and Company"~"Kisunla (donanemab-azbt)"~7/2/2024 0:00:00~"Original"~1~"4605-2"~"PMR"~"505 (o)(3)"~4605.00~2~"PMR 4605-2: Use emerging safety data from ongoing studies and published literature, validate administrative claim codes for intracerebral hemorrhage in patients with Alzheimer s disease. The outcome of intracerebral hemorrhage should distinguish between amyloid related imaging abnormalities-hemosiderin deposition (ARIA-H) and cerebral hemorrhage > 1 cm. Secondary outcomes of interest include ARIA-edema (ARIA-E) and ARIA-H, seizures, anaphylaxis, and death. For secondary outcomes not well validated, develop algorithms and/or computable phenotypes using data leveraged from PMR 4605-1 and other sources for the outcomes of interest. Describe an approach to identifying an appropriate comparator group with Alzheimer disease untreated with donanemab. Obtain FDA agreement with the outcome algorithm specifications and comparator population prior to proceeding to conducting the retrospective cohort study. Based upon validated algorithms agreed to by the Sponsor and FDA, conduct a comparative retrospective cohort study using claims data with available medical chart review as needed or electronic health record data to assess clinical safety outcomes in a broad population of Alzheimer s disease patients treated with donanemab."~"Pending"~~12/31/2031 0:00:00~8/29/2025 0:00:00
385698~"CDER"~"BLA"~761248.00~"Eli Lilly and Company"~"Kisunla (donanemab-azbt)"~7/2/2024 0:00:00~"Original"~1~"4605-3"~"PMC"~"506B PMC"~4605.00~3~"PMC 4605-3: Conduct a randomized, double-blind, controlled trial of donanemab in participants with early-stage Alzheimer s disease. All participants should receive an initial dosing regimen in which dosing is stopped based on reduction of amyloid, followed by randomization to either a maintenance dose(s) of donanemab or placebo for a period of at least 2 years. The primary efficacy endpoint will be the Clinical Dementia Rating scale. The study should also collect biomarkers of Alzheimer s disease pathology. This trial may be conducted through cross over of participants in TRAILBLAZER-ALZ 3 upon completion of that trial."~"Pending"~~10/31/2031 0:00:00~8/29/2025 0:00:00
385699~"CDER"~"BLA"~761258.00~"Akeso Biopharma Co., Ltd. "~"penpulimab-kcqx"~4/23/2025 0:00:00~"Original"~1~"4815-1"~"PMC"~"506B PMC"~4815.00~1~"PMC 4815-1: Complete the ongoing clinical trial AK105-304 (NCT04974398) titled  A Randomized, Double-blind, Multi-center Phase III Study of Penpulimab (AK105) combined with Chemotherapy versus Placebo Combined with Chemotherapy as First-Line Treatment for Recurrent or Metastatic Nasopharyngeal Carcinoma,  to obtain mature overall survival (OS) data in accordance with the pre-specified statistical analysis plan to formally test OS at 80% information fraction and at the final OS analysis when 172 OS events are observed."~"Pending"~~10/31/2026 0:00:00~6/18/2026 0:00:00
385700~"CDER"~"NDA"~219155.00~"LEO PHARMA AS"~"Anzupgo (delgocitinib) cream"~7/23/2025 0:00:00~"Original"~1~"4877-3"~"PMR"~"505 (o)(3)"~4877.00~3~"PMR 4877-3: Conduct a randomized, active-controlled clinical trial to evaluate the longterm safety of delgocitinib in patients with moderate to severe chronic hand eczema. The trial should be of sufficient size and duration to characterize safety events of interest including cytopenias and viral reactivation."~"Pending"~~3/31/2030 0:00:00~
385701~"CDER"~"NDA"~219164.00~"ASCENDIS PHARMA GROWTH DISORDERS AS"~"Yuviwel (navepegritide) Injection"~2/27/2026 0:00:00~"Original"~1~"4969-1"~"PMR"~"Accelerated Approval"~4969.00~1~"PMR 4969-1: Conduct an open-label, external-controlled trial in subjects with achondroplasia 2 years of age and older with open epiphyses to measure the effect of Yuviwel on final adult height. Include a secondary endpoint that evaluates disproportionality. Include safety endpoints related to the drug or to the disease that may improve or worsen with long-term treatment (e.g., neurological complications, bone deformities, bone age, sleep apnea)."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~6/30/2035 0:00:00~
385702~"CDER"~"NDA"~219164.00~"ASCENDIS PHARMA GROWTH DISORDERS AS"~"Yuviwel (navepegritide) Injection"~2/27/2026 0:00:00~"Original"~1~"4969-2"~"PMR"~"505 (o)(3)"~4969.00~2~"PMR 4969-2: Conduct a study in subjects who received the 100 mcg/kg/week navepegritide dose in trials ASND0036 and TCC-201 and were confirmed to be anti-drug antibody positive, to assess the incidence of neutralizing antibodies to navepegritide and their ability to cross-neutralize native C-type natriuretic peptide (CNP). For this study use a sensitive assay that is able to detect the neutralizing activity of anti-navepegritide antibodies and their cross-neutralizing effect on native CNP. Perform this analysis after FDA has confirmed the adequacy of your new assay. Include in the final study report your development and validation data to support the use of the neutralizing antibody assay, as well as analyses of the effects of neutralizing antibodies on the pharmacokinetics, efficacy and safety of navepegritide."~"Pending"~~3/31/2028 0:00:00~
385703~"CDER"~"NDA"~219208.00~"NOVARTIS PHARMACEUTICALS CORP"~"Vanrafia (atrasentan) tablet"~4/2/2025 0:00:00~"Original"~1~"4829-1"~"PMR"~"Accelerated Approval"~4829.00~1~"PMR 4829-1: Conduct a randomized, double-blind, placebo-controlled trial to describe and verify the clinical benefit of atrasentan for the treatment of primary IgA nephropathy. The trial should be adequately powered and of sufficient duration to detect a treatment effect on the endpoint that will be used to describe and verify the clinical benefit."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~9/30/2026 0:00:00~5/28/2026 0:00:00
385704~"CDER"~"NDA"~219208.00~"NOVARTIS PHARMACEUTICALS CORP"~"Vanrafia (atrasentan) tablet"~4/2/2025 0:00:00~"Original"~1~"4829-2"~"PMR"~"Pediatric Research Equity Act"~4829.00~2~"PMR 4829-2: Develop an age-appropriate formulation of atrasentan for use in pediatric patients down to 2 years of age."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~3/31/2026 0:00:00~5/28/2026 0:00:00
385705~"CDER"~"NDA"~219208.00~"NOVARTIS PHARMACEUTICALS CORP"~"Vanrafia (atrasentan) tablet"~4/2/2025 0:00:00~"Original"~1~"4829-3"~"PMR"~"Pediatric Research Equity Act"~4829.00~3~"PMR 4829-3: Conduct an efficacy, pharmacokinetic, safety and tolerability study in pediatric patients 2 years to less than 18 years of age with primary immunoglobulin A nephropathy. The efficacy outcome should be based on effects on proteinuria."~"Released"~"Per FDA letter dated 01/12/2026, this PMR/PMC has been released."~8/31/2031 0:00:00~5/28/2026 0:00:00
385706~"CDER"~"NDA"~219208.00~"NOVARTIS PHARMACEUTICALS CORP"~"Vanrafia (atrasentan) tablet"~4/2/2025 0:00:00~"Original"~1~"4829-4"~"PMR"~"Pediatric Research Equity Act"~4829.00~4~"PMR 4829-4: Conduct a pharmacodynamic, pharmacokinetic, safety and tolerability study of Vanrafia in pediatric patients 2 years to less than 18 years of age with primary immunoglobulin A nephropathy. The pharmacodynamic outcome should be based on effects on proteinuria."~"Pending"~"The study has not been initiated, but does not meet the criterion for delayed."~8/31/2031 0:00:00~5/28/2026 0:00:00
385707~"CDER"~"NDA"~219209.00~"VERTEX PHARMACEUTICALS INC"~"Journavx (suzetrigine) tablet"~1/30/2025 0:00:00~"Original"~1~"4787-1"~"PMR"~"Pediatric Research Equity Act"~4787.00~1~"PMR 4787-1: Conduct a juvenile animal study in the rodent model to characterize the impact of suzetrigine on the developing brain and reproductive tissues to support clinical studies in pediatric patients from 3 to less than 12 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~3/31/2027 0:00:00~3/27/2026 0:00:00
385708~"CDER"~"NDA"~219209.00~"VERTEX PHARMACEUTICALS INC"~"Journavx (suzetrigine) tablet"~1/30/2025 0:00:00~"Original"~1~"4787-2"~"PMR"~"Pediatric Research Equity Act"~4787.00~2~"PMR 4787-2: Conduct a juvenile animal study in the rodent model to characterize the impact of suzetrigine on the developing brain to support clinical studies in pediatric patients from birth to less than 3 years of age."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~12/31/2028 0:00:00~3/27/2026 0:00:00
385709~"CDER"~"NDA"~219209.00~"VERTEX PHARMACEUTICALS INC"~"Journavx (suzetrigine) tablet"~1/30/2025 0:00:00~"Original"~1~"4787-3"~"PMR"~"Pediatric Research Equity Act"~4787.00~3~"PMR 4787-3: Conduct a randomized, controlled study evaluating the pharmacokinetics, safety, and efficacy of Journavx in pediatric patients aged 12 to less than 17 years with moderate to severe acute pain."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~12/31/2027 0:00:00~3/27/2026 0:00:00
385710~"CDER"~"NDA"~219209.00~"VERTEX PHARMACEUTICALS INC"~"Journavx (suzetrigine) tablet"~1/30/2025 0:00:00~"Original"~1~"4787-4"~"PMR"~"Pediatric Research Equity Act"~4787.00~4~"PMR 4787-4: Conduct a randomized, controlled study evaluating the pharmacokinetics, safety, and efficacy of Journavx in pediatric patients aged 6 to less than 12 years with moderate to severe acute pain."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~9/30/2030 0:00:00~3/27/2026 0:00:00
385711~"CDER"~"NDA"~219209.00~"VERTEX PHARMACEUTICALS INC"~"Journavx (suzetrigine) tablet"~1/30/2025 0:00:00~"Original"~1~"4787-5"~"PMR"~"Pediatric Research Equity Act"~4787.00~5~"PMR 4787-5: Conduct a randomized, controlled study evaluating the pharmacokinetics, safety, and efficacy of Journavx in pediatric patients aged 1 to less than 6 years with moderate to severe acute pain."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2032 0:00:00~3/27/2026 0:00:00
385712~"CDER"~"NDA"~219209.00~"VERTEX PHARMACEUTICALS INC"~"Journavx (suzetrigine) tablet"~1/30/2025 0:00:00~"Original"~1~"4787-6"~"PMR"~"Pediatric Research Equity Act"~4787.00~6~"PMR 4787-6: Conduct a randomized, controlled study evaluating the pharmacokinetics, safety, and efficacy of Journavx in pediatric patients from birth to less than 1 year of age with moderate to severe acute pain."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~3/31/2034 0:00:00~3/27/2026 0:00:00
385713~"CDER"~"BLA"~761055.00~"Regeneron Pharmaceuticals, Inc."~"Dupixent (dupilumab)"~3/28/2017 0:00:00~"Supplement"~40~"4276-3"~"PMC"~"506B PMC"~4276.00~3~"PMC 4276-3: A long-term extension study to evaluate the long-term safety of Dupixent (dupilumab) in pediatric patients 1 to less than 12 years of age with active eosinophilic esophagitis, who participated in postmarketing commitment study 4276-2. This study may be conducted as part of postmarketing commitment study 4276-2."~"Fulfilled"~~2/28/2026 0:00:00~5/23/2025 0:00:00
385714~"CDER"~"BLA"~761055.00~"Regeneron Pharmaceuticals, Inc."~"Dupixent (dupilumab)"~3/28/2017 0:00:00~"Supplement"~44~"4329-1"~"PMR"~"Pediatric Research Equity Act"~4329.00~1~"PMR 4329-1: Conduct an open-label pharmacokinetic and safety trial in subjects 6 months to <18 years of age with prurigo nodularis."~"Delayed"~"The Final Protocol Submission milestone date was missed because the applicant was awaiting feedback from the FDA. The final report submission milestone date is has not yet occurred.  As of 3/27/2025, 4 patients have been enrolled."~3/31/2028 0:00:00~5/23/2025 0:00:00
385715~"CDER"~"BLA"~761055.00~"Regeneron Pharmaceuticals, Inc."~"Dupixent (dupilumab)"~3/28/2017 0:00:00~"Supplement"~57~"4573-1"~"PMC"~"506B PMC"~4573.00~1~"PMC 4573-1: Conduct a 24-week trial to evaluate the safety, efficacy, and pharmacokinetics of Dupixent (dupilumab) in pediatric patients weighing less than 15 kilograms with active eosinophilic esophagitis."~"Delayed"~"The applicant requested revised milestones due to continuous communications with the agency. Revised milestones were acknowledged in a letter dated 06/27/2025.

"~7/31/2028 0:00:00~5/23/2025 0:00:00
385716~"CDER"~"BLA"~761060.00~"Wyeth Pharmaceuticals LLC"~"Mylotarg (Gemtuzumab Ozogamicin) "~9/1/2017 0:00:00~"Supplement"~4~"3880-1"~"PMR"~"505 (o)(3)"~3880.00~1~"PMR 3880-1: Provide pharmacokinetic, immunogenicity, safety, and preliminary efficacy (response and minimal residual disease) data to confirm safe and appropriate dosing of gemtuzumab ozogamicin for up to 3 doses in combination with induction chemotherapy in infants 12 weeks to < 12 months of age and in pediatric patients = 12 months of age with newly-diagnosed AML. Submit a study report, including datasets, for the nonrandomized embedded major and minor dose-finding study for gemtuzumab ozogamicin in combination with induction chemotherapy from Study MyeChild01 (NCT02724163), including approximately 55 pediatric patients = 12 months of age and approximately 10 pediatric patients 12 weeks to < 12 months of age."~"Delayed"~"The final report milestone was missed because enrollment took longer than expected."~9/30/2022 0:00:00~10/29/2025 0:00:00
385717~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Original"~1~"3225-1"~"PMR"~"Pediatric Research Equity Act"~3225.00~1~"PMR 3225-1: Conduct a Pharmacokinetics (PK), Safety and Efficacy Study in pediatric subjects 6 years to less than18 years of age with moderate to severe plaque psoriasis (with  a duration of exposure to guselkumab of at least one year)."~"Delayed"~"The Trial Completion and Final Report Submission dates were missed due to enrollment issues. Original Final Report Due Date:04/30/2024. Per FDA letter dated 08/11/2023, final report due date extended to 04/30/2024. Second Deferral Extension Granted per FDA letter dated 09/29/2025."~7/31/2027 0:00:00~9/5/2025 0:00:00
385718~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Original"~1~"3225-2"~"PMR"~"505 (o)(3)"~3225.00~2~"PMR 3225-2: A prospective, registry-based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to guselkumab during pregnancy to an unexposed control population. The registry will detect and record major and minor congenital malformations, spontaneous abortions, stillbirths,  elective terminations, small for gestational age, and any other adverse pregnancy outcomes. These outcomes will be assessed throughout pregnancy. Infant  outcomes, including neonatal deaths, infections in the first 6 months of life, and effects on postnatal growth and development, will be assessed through at least the first year of life."~"Ongoing"~~12/31/2026 0:00:00~9/5/2025 0:00:00
385719~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Original"~1~"3225-3"~"PMR"~"505 (o)(3)"~3225.00~3~"PMR 3225-3: Conduct a retrospective cohort study using claims or electronic medical record data or a case control study to assess adverse pregnancy outcomes such as major congenital malformations, spontaneous abortions, stillbirths, small for gestational age, neonatal deaths, and infant infections in women exposed to guselkumab  during pregnancy compared to an unexposed control population."~"Delayed"~"The 2024 study completion submission milestone was missed because a larger number of exposure cases would provide more precise and meaningful analysis of any effect of guselkumab on pregnancy outcomes. FDA determined that there was good cause for the delay and acknowledged the revised milestone in a letter dated 01/30/2026.  In the same letter, FDA also acknowledged that there was sufficient justification for the anticipated delay of the final report submission proposed revised milestone.
"~12/31/2025 0:00:00~9/5/2025 0:00:00
385720~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Original"~1~"3225-4"~"PMR"~"505 (o)(3)"~3225.00~4~"PMR 3225-4: Conduct an observational study to assess the long-term safety of guselkumab compared to other therapies used in the treatment of adults with moderate-tosevere  plaque psoriasis who are candidates for systemic therapy or phototherapy in the course of actual clinical care. The study s primary outcome is the long-term  risk of malignancy. Secondary outcomes include, but are not limited to, serious infections, tuberculosis, opportunistic infections, hypersensitivity reactions, autoimmune disease, neurologic or demyelinating disease, cardiovascular,  gastrointestinal and hematologic adverse events. Describe and justify the choice of appropriate comparator population(s) and estimated background rate(s) relative to guselkumab-exposed patients; clearly define the primary comparator population for the primary objective. Design the study around a testable hypothesis to assess, with sufficient sample size and power, a clinically meaningful increase in malignancy risk above the comparator background rate(s),  with a pre-specified statistical analysis method. Specify concise case definitions and validation algorithms for both primary and secondary outcomes. For the  guselkumab-exposed and comparator(s) cohorts, clearly define the study drug initiation period and any exclusion and inclusion criteria. Enroll patients over an initial 6 year period and follow for a minimum of 8 years from the time of enrollment."~"Delayed"~"The Final Protocol was submitted after the original final protocol submission milestone. The final protocol was acknowledged in a letter dated 1/25/2021."~12/31/2031 0:00:00~9/5/2025 0:00:00
385721~"CDER"~"BLA"~761061.00~"Janssen Biotech, Inc."~"Tremfya (guselkumab) injection and Tremfya Pen (guselkumab) injection"~7/13/2017 0:00:00~"Supplement"~7~"3899-1"~"PMR"~"Pediatric Research Equity Act"~3899.00~1~"PMR 3899-1: Provide PK and safety information to support the pediatric assessment of guselkumab for the treatment of juvenile psoriatic arthritis (jPsA) in children 5 to 17 years of age."~"Delayed"~"Original Final Report Due Date: 04/30/2024; Per FDA letter dated 08/11/2023, final report due date extended to 12/31/2024. Second Deferral Extension Granted per FDA letter dated 09/26/2025.
"~7/31/2027 0:00:00~9/5/2025 0:00:00
385722~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Supplement"~1~"4649-1"~"PMR"~"Accelerated Approval"~4649.00~1~"PMR 4649-1: Conduct an analysis of patients from ongoing or planned trials intended to verify and describe the clinical benefit of repotrectinib through more precise estimation of the overall response rate and mature response duration per independent review assessment, in adult and pediatric patients 12 years of age and older with solid tumors with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion who have locally advanced or metastatic disease or would require surgical resection that would result in severe morbidity; and have no satisfactory alternative treatment or that have progressed following treatment. A sufficient number of patients will be evaluated to more precisely characterize response and durability of response for a spectrum of patients with different TKI-na ve and TKI-pretreated tumor types. All responding patients will be followed for at least 12 months from the onset of response or until disease progression whichever comes first."~"Ongoing"~"The study has been initiated."~5/31/2030 0:00:00~1/9/2026 0:00:00
385723~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Supplement"~1~"4649-2"~"PMR"~"Accelerated Approval"~4649.00~2~"PMR 4649-2: Complete the analysis of the 23 responding patients in the efficacy evaluable population of 40 TKI-na ve patients and the 24 responding patients in the efficacy evaluable population of 48 TKI-pretreated patients in the TRIDENT-1 trial intended to verify and describe the clinical benefit and further characterize the duration of response in patients who achieved a complete or partial response to repotrectinib. All responding patients will be followed for at least 2 years from the onset of response or until disease progression, whichever comes first. Duration of response will be assessed by independent central review."~"Fulfilled"~"Per FDA letter dated 04/22/2026, this PMR/PMC has been fulfilled."~3/31/2025 0:00:00~1/9/2026 0:00:00
385724~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Supplement"~1~"4649-3"~"PMR"~"505 (o)(3)"~4649.00~3~"PMR 4649-3: Conduct integrated safety analyses from an adequate number of patients enrolled in clinical trial(s) designed to characterize the known serious risk of fractures and sequelae of fractures in patients exposed to repotrectinib with reasonable precision; to identify risk factors for development of these sequelae; and to identify mitigating measures for the risk of skeletal fractures. Include sufficient bone monitoring to achieve these objectives including but not limited to initial and serial assessment of bone mineral density (BMD) with dual x-ray absorptiometry (DXA) scans, and markers of bone formation, bone resorption, and calcium metabolism."~"Ongoing"~~11/30/2029 0:00:00~1/9/2026 0:00:00
385725~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Supplement"~1~"4649-4"~"PMR"~"505 (o)(3)"~4649.00~4~"PMR 4649-4: Conduct a clinical trial or analysis of clinical trials of repotrectinib in a sufficient number of pediatric patients 12 years of age and older with NTRK-fusion positive solid tumors to evaluate the potential serious risk of adverse long-term effects of repotrectinib on growth and development and neurological outcomes, with reasonable precision. Patients will be monitored for growth and developmental milestones using age-appropriate screening tools and undergo neurological examination at appropriate intervals. Evaluations will include neurological exams with neurocognitive assessment, Karnofsky/Lansky score, growth as measured by height, weight, height velocity, and height standard deviation scores (SDS), age at adrenarche if applicable (males), age at menarche if applicable (females) and Tanner Stage. Patient monitoring will be performed until discontinuation of study treatment or a minimum of 5 years from start of treatment, whichever occurs first."~"Ongoing"~~5/31/2030 0:00:00~1/9/2026 0:00:00
385726~"CDER"~"NDA"~218213.00~"BRISTOL MYERS SQUIBB CO"~"Augtyro (repotrectinib)"~11/15/2023 0:00:00~"Supplement"~1~"4649-5"~"PMC"~"506B PMC"~4649.00~5~"PMC 4649-5: Complete an appropriate clinical validation study using clinical trial data to establish and support the availability of an in vitro diagnostic device that is essential to the safe and effective use of repotrectinib for patients with solid tumors that carry NTRK1/2/3 gene fusions."~"Submitted"~~7/30/2025 0:00:00~1/9/2026 0:00:00
385727~"CDER"~"NDA"~218230.00~"GLAXOSMITHKLINE LLC"~"Blujepa (gepotidacin)"~3/25/2025 0:00:00~"Original"~1~"4822-1"~"PMR"~"Pediatric Research Equity Act"~4822.00~1~"PMR 4822-1: Conduct an open-label, add-on, non-comparator study to evaluate the pharmacokinetics and safety of a single dose of oral gepotidacin administered in addition to antibacterial standard of care, in pediatric patients aged 2 years to less than 12 years with suspected or confirmed bacterial infection or who are receiving antibacterial prophylaxis."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~6/30/2028 0:00:00~5/20/2026 0:00:00
385728~"CDER"~"NDA"~218230.00~"GLAXOSMITHKLINE LLC"~"Blujepa (gepotidacin)"~3/25/2025 0:00:00~"Original"~1~"4822-2"~"PMR"~"Pediatric Research Equity Act"~4822.00~2~"PMR 4822-2: Conduct an open-label, active-controlled comparator study to evaluate the safety and tolerability of oral gepotidacin in pediatric patients aged 2 years to less than 12 years with a confirmed or suspected uncomplicated UTI."~"Pending"~"The study has not been initiated, but it does not meet the criterion for delayed."~3/31/2033 0:00:00~5/20/2026 0:00:00
385729~"CDER"~"NDA"~218230.00~"GLAXOSMITHKLINE LLC"~"Blujepa (gepotidacin)"~3/25/2025 0:00:00~"Original"~1~"4822-3"~"PMR"~"505 (o)(3)"~4822.00~3~"PMR 4822-3: Collect data from a prospective pregnancy exposure registry, preferably a disease-based multiproduct pregnancy registry, using a registry-based cohort study design that compares the maternal, fetal, and infant outcomes of women exposed to gepotidacin during pregnancy with comparator population(s) unexposed to gepotidacin. Align the study protocol with protocol(s) outside the U.S. to reach a target sample size."~"Pending"~~10/31/2032 0:00:00~5/20/2026 0:00:00
385730~"CDER"~"NDA"~218230.00~"GLAXOSMITHKLINE LLC"~"Blujepa (gepotidacin)"~3/25/2025 0:00:00~"Original"~1~"4822-4"~"PMR"~"505 (o)(3)"~4822.00~4~"PMR 4822-4: Conduct a retrospective pregnancy cohort study using claims or electronic health record data with medical chart validation that is adequately powered to assess small-for-gestational-age births, spontaneous abortions, major congenital malformations, stillbirths, and preterm births in individuals exposed to gepotidacin during pregnancy compared to appropriate comparator population(s)."~"Pending"~~10/31/2032 0:00:00~5/20/2026 0:00:00
385731~"CDER"~"NDA"~218230.00~"GLAXOSMITHKLINE LLC"~"Blujepa (gepotidacin)"~3/25/2025 0:00:00~"Original"~1~"4822-5"~"PMR"~"505 (o)(3)"~4822.00~5~"PMR 4822-5: Perform a clinical lactation study (milk only or mother-infant pair study) in lactating women who have received gepotidacin to measure concentrations of gepotidacin in breast milk using a validated assay. Assess the effects on the breastfed infant, if available, based on the study population."~"Pending"~~10/31/2028 0:00:00~5/20/2026 0:00:00
385732~"CDER"~"NDA"~218230.00~"GLAXOSMITHKLINE LLC"~"Blujepa (gepotidacin)"~3/25/2025 0:00:00~"Original"~1~"4822-6"~"PMR"~"505 (o)(3)"~4822.00~6~"PMR 4822-6: Conduct a United States surveillance study, or studies as appropriate, for 5 years from the date of marketing to determine if resistance to gepotidacin has developed in those organisms specific to the indication in the label."~"Pending"~~9/30/2031 0:00:00~5/20/2026 0:00:00
385733~"CDER"~"NDA"~218230.00~"GLAXOSMITHKLINE LLC"~"Blujepa (gepotidacin)"~3/25/2025 0:00:00~"Supplement"~1~"4939-1"~"PMR"~"505 (o)(3)"~4939.00~1~"PMR 4939-1: Conduct an open-label, single-arm, prospective trial for the treatment of uncomplicated gonorrhea to evaluate the safety of Blujepa (gepotidacin)."~"Pending"~~4/30/2029 0:00:00~5/20/2026 0:00:00
