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  1. Resources for Information | Approved Drugs

Ongoing | Cancer Accelerated Approvals

This listing includes accelerated approvals (AAs) for malignant hematology and oncology indications that have postmarketing requirement(s) for ongoing clinical trial(s) to verify clinical benefit. Please refer to Drugs@FDA for the latest approvals and prescribing information for specific products. Visit the verified and withdrawn AA indication pages for more information and the Postmarket Requirements and Commitments page for the status of specific requirements.

Indications may remain on this page until FDA updates product labeling or publishes a Federal Register notice regarding a change in status.

Drug Name Accelerated Approval (AA) Indication AA Date Original Projected Completion AA Post-Marketing Requirement 1
Augtyro (repotrectinib) Treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic where surgical resection is likely results in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy 6/13/2024 4649-1: Conduct an analysis of patients from ongoing or planned trials 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-naïve 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. 5/31/2030
Imdelltra (tarlatamab-dlle) Adult patients with extensive stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy 5/16/2024 4635-1: Conduct a multicenter randomized clinical trial intended to verify and describe the clinical benefit of tarlatamab in patients with ES-SCLC who have had disease progression on or after platinum-based chemotherapy 8/31/2026
Breyanzi (lisocabtagene maraleucel) Adults patients with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy 5/15/2024 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. 8/31/2025
Ojemda (tovorafenib) Patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. 4/23/2024 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. 4/30/2032
Enhertu (fam-trastuzumab deruxtecan-nxki) Adult patients with unresectable or metastatic HER2-positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options. 4/5/2024 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. 6/30/2027
Iclusig (ponatinib) Adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy. 3/19/2024 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. 06/30/2028
Brukinsa (zanubrutinib) Adult patients with relapsed or refractory follicular lymphoma (FL), in combination with obinutuzumab, after two or more lines of systemic therapy 3/7/2024 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 FL. The primary endpoint should be PFS, with secondary endpoints that include ORR and OS. The trial should enroll a sufficiently representative study population that reflects the racial and ethnic diversity of the U.S. population of patients with FL and that allows for interpretation of the results in these populations. 1/31/2029
Amtagvi (lifileucel) Adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. 2/16/2024 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 ORR and PFS, with OS as the key secondary endpoint. 03/31/2031
Jaypirca (pirtobrutinib) Adults with CLL or small lymphocytic lymphoma (SLL) who have received at least two prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor 12/1/2023

4557-1: Complete a randomized trial in patients with relapsed or refractory CLL or 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.

6/30/2024
Elrexfio (elranatamab-bcmm) Adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. 8/14/2023

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.

9/30/2026
Talvey (talquetamab-tgvs) Adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody 8/9/2023

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.

12/31/2026
Columvi (glofitamab-gxbm) Relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified or large B-cell lymphoma arising from follicular lymphoma, after two or more lines of systemic therapy 6/15/2023 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. 9/30/2024
Epkinly (epcoritamab-bysp) Adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from indolent lymphoma, and high-grade B-cell lymphoma after two or more lines of systemic therapy. 5/19/2023 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 OS with secondary endpoints that include PFS and response rate. 6/30/2025
Zynyz (retifanlimab-dlwr) Adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC). 3/22/2023 4412-1: Conduct a multicenter clinical trial intended to confirm the clinical benefit of retifanlimab-dlwr in patients with metastatic or recurrent locally advanced 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 ORR and characterize the DOR. Include an analysis of OS, when 70% of patients have died, or all patients have been followed for at least three years. 3/31/2025
Jaypirca (pirtobrutinib) Adult patients with relapsed or refractory mantle cel lymphoma (MCL) after at least two lines of systemic therapy, including a BTK inhibitor. 1/27/2023 4389-1: Complete a randomized clinical trial to obtain data on the clinical efficacy and safety of pirtobrutinib in patients with MCL. The trial should compare pirtobrutinib monotherapy to an investigator’s choice of approved BTK inhibitors in patients with MCL. The primary endpoint should be PFS as assessed by an independent review committee, with secondary endpoints that include OS 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 MCL and allow for interpretation of the results in these patient populations. 12/31/2026
Tukysa (tucatinib) In combination with trastuzumab for the treatment of adult patients with RAS wild-type, HER2-positive, unresectable or metastatic colorectal cancer that has progressed following treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy 1/19/2023 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 PFS per blinded assessment or OS. 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. 04/30/2026
Lunsumio (mosunetuzumab-axgb) Adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy. 12/22/2022 4375-1: Conduct a randomized clinical trial in patients with relapsed or refractory follicular lymphoma (FL), with patients randomized to receive mosunetuzumab in combination with lenalidomide or rituximab in combination with lenalidomide. The primary endpoint should be PFS, with secondary endpoints that include RR and OS. The trial should enroll a sufficiently representative study population to reflect the racial and ethnic diversity of the U.S. patient population with FL and allow for interpretation of the results in these patient populations. 12/31/2025
Krazati (adagrasib) Adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy. 12/12/2022 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 OS, PFS, ORR, and DOR. 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.” 12/31/2025
Tecvayli (teclistamab-cqyv) Adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody 10/25/2022 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 PFS and secondary endpoints should include OS, ORR, and DOR. 3/31/2026
Lytgobi (futibatinib) Adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements 9/30/2022 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 ORR and DOR should be assessed by a blinded independent review. The study should also evaluate other clinical outcomes that denote clinical benefit, such as PROs. 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. 10/31/2027
Retevmo (selpercatinib) Adult patients with locally advanced or metastatic solid tumors with a RET gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options * 9/21/2022 4342-1: Complete clinical trial(s) to obtain data on the clinical efficacy of selpercatinib through more precise estimation of the ORR 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 NSCLC 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. ORR and DOR 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 co-occurring genetic alterations for all patients. 12/31/2025
Enhertu (Fam-trastuzumab deruxtecan-nxki) Adult patients with unresectable or metastatic NSCLC whose tumors have an activating HER2 (ERBB2) mutation, as detected by an FDA-approved test, and who have received a prior systemic therapy 8/11/2022

4321-1: Complete a clinical trial to obtain data on the clinical efficacy of fam-trastuzumab deruxtecan-nxki for the treatment of patients with unresectable or metastatic NSCLC whose tumors have an activating HER2 (ERBB2) mutation and have previously received systemic therapy, to provide a more precise estimation of the BICR-assessed ORR and DOR. This report will contain data from patients with NSCLC harboring HER2 mutations and data from at least 102 patients who have received prior systemic therapy, after all responders have been followed for at least 6 months from the date of initial response (or until disease progression, whichever comes first).

4321-2: Conduct a multicenter, randomized clinical trial of fam-trastuzumab deruxtecan-nxki in patients with treatment-naïve, unresectable or metastatic NSCLC whose tumors have an activating HER2 (ERBB2) mutation. The final analysis should include the final PFS and OS results.

3/31/2024
Tafinlar (dabrafenib) In combination with trametinib for adult and pediatric patients 6 years of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. * 6/22/2022 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 ORR 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. 10/31/2028
Mekinist (trametinib) In combination with dabrafenib for adult and pediatric patients 6 years of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options * 6/22/2022 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 ORR 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. 10/31/2028
Kymriah (tisagenlecleucel) Treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy 5/27/2022 Conduct a randomized phase 3 trial in adult patients with r/r FL. Patients will be randomized to tisagenlecleucel or an investigator’s choice of regimens consistent with the standard of care. The primary endpoint will be PFS with secondary endpoints that include OS and ORR. 9/30/2028
Vijoice (alpelisib) Treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy 4/5/2022 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 ORR and mature response duration per blinded independent review, in adult and pediatric patients 2 years of age and older with PROS, including those with severe manifestations. 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, PROS subtype, and age. 8/31/2027
Gavreto (pralsetinib) Treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) 12/1/2020 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 ORR and DOR 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. 6/30/2025
Exkivity (mobocertinib) Treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy 9/15/2021 4148-1: Conduct a multicenter, randomized clinical trial and submit the final PFS results that verify and describe the clinical benefit of mobocertinib in patients with locally advanced or metastatic NSCLC whose tumors harbor EGFR exon 20 insertion mutations 3/31/2024
Brukinsa (zanubrutinib) Treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen 9/14/2021 4128-1: Conduct a randomized clinical trial that verifies and describes the clinical benefit of zanubrutinib in patients with relapsed or refractory MZL. 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 PFS, with secondary endpoints that include ORR and OS. 10/31/2028
Jemperli (dostarlimab-gxly) Treatment for adult patients with mismatch repair deficient (dMMR) recurrent or advanced solid tumors, as determined by an FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options 8/17/2021 4124-1: Conduct a clinical trial evaluating ORR, and DOR, to verify and describe the clinical benefit of Jemperli in patients with 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 GI tumors). In order to characterize response rate and DOR, patients should be followed for at least 12 months from the onset of response. Submit the datasets with final report. 10/31/2022
Lumakras (sotorasib) Treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy. 5/28/2021 4071-1: Conduct a multicenter, randomized clinical trial and submit the final PFS results that verify and describe the clinical benefit of sotorasib in patients with locally advanced or metastatic NSCLC with a history of prior systemic therapy for advanced disease and whose tumors harbor KRAS G12C mutation. 7/30/2022
Keytruda (pembrolizumab) In combination with trastuzumab and fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of patients with locally advanced unresectable or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. 3 5/5/2021 4033-1: Submit the final PFS and final OS analyses and datasets for the ongoing clinical trial KEYNOTE-811, “A Phase III, Randomized, Double-blind Trial Comparing Trastuzumab Plus Chemotherapy and Pembrolizumab With Trastuzumab Plus Chemotherapy and Placebo as First-line Treatment in Participants With HER2 Positive Advanced Gastric or Gastroesophageal Junction Adenocarcinoma” to verify and describe the clinical benefit of pembrolizumab with trastuzumab plus chemotherapy for patients with HER2-positive advanced or metastatic gastric or gastroesophageal adenocarcinoma. 9/30/2024
Zynlonta (loncastuximab tesirine-lpyl) Treatment of adult 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, DLBCL arising from low grade lymphoma, and high-grade B-cell lymphoma. 4/23/2021 4029-1: Conduct a randomized, phase 3 clinical trial to verify and describe the clinical benefit of loncastuximab tesirine-lpyl in patients with R/R 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 PFS , with secondary endpoints that include OS and ORR. 12/31/2025
Trodelvy (sacituzumab govitecan-hziy) Treatment of adult patients with locally advanced or metastatic urothelial cancer (mUC) who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor 4/13/2021 4044-1: Submit the final study report and datasets for OS and PFS from trial IMMU-132-13 titled “A Randomized Open-Label Phase III Study of Sacituzumab Govitecan Versus Treatment of Physician's Choice in Subjects With Metastatic or Locally Advanced Unresectable Urothelial Cancer (TROPiCS-04)”, to verify and confirm the clinical benefit of sacituzumab in the urothelial cancer patient population. 4/30/2025
Yescarta (axicabtagene ciloleucel) Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy 3/5/2021 1: A randomized phase 3 trial of axicabtagene ciloleucel in patients with relapsed or refractory FL. 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 PFS, with secondary endpoints that include ORR and OS. 9/30/2027
Darzalex Faspro (daratumumab and hyaluronidase-fihj) Treatment of patients with light chain (AL) amyloidosis in combination with bortezomib, cyclophosphamide and dexamethasone in newly diagnosed patients. 1/15/2021 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 PFS or OS results. 6/30/2025
Danyelza (naxitamab-gqgk) In combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. 11/25/2020 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 R/R neuroblastoma in combination with 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 ORR and DOR (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 ORR and DOR by prior anti-GD2 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 6 study sites, with at least 5 patients enrolled per site in at least 5 sites. 9/30/2027
Monjuvi (tafasitamab-cxix) In combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). 7/31/2020 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 DLBCL. 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 PFS, with secondary endpoints that include OS and ORR. 12/31/2025
Tecartus (brexucabtagene autoleucel) Treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL). 7/24/2020 1: Complete additional follow-up of all 68 subjects treated with brexucabtagene autoleucel in ZUMA-2 Cohort 1 to a minimum of 18 months from the time of first response. Data will continue to be collected according to the ZUMA-2 protocol’s established schedule of assessments.
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 naïve 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.
10/31/2025
Xpovio (selinexor) Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma (FL), after at least 2 lines of systemic therapy. 6/22/2020 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 DLBCL. Patients should be randomized to receive chemoimmunotherapy with or without selinexor. The primary endpoint should be PFS , with secondary endpoints that include OS and ORR. 4/30/2026
Tazverik (tazemetostat) Treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least 2 prior systemic therapies. Treatment of adult patients with R/R FL who have no satisfactory alternative treatment options. 6/18/2020 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 R/R FL 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 PFS, with secondary endpoints that include OS and ORR.
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 R/R FL 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 PFS, with secondary endpoints that include OS and ORR.
7/31/2025
Keytruda (pembrolizumab) Treatment of 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. 6/16/2020 3871-1: Submit the final report and datasets from clinical trials evaluating ORR and DOR, to verify and describe the clinical benefit of pembrolizumab in adult and pediatric patients with unresectable or metastatic 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, MSI-H 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 DOR. A minimum of 20 pediatric patients will be studied. ORR and DOR 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. 12/31/2025
Zepzelca (lurbinectedin) Treatment of adult patients with metastatic small cell lung cancer (SCLC) with disease progression on or after prior platinum-based chemotherapy. 6/15/2020 3831-1: Submit the final report and datasets for the OS and PFS analysis as determined by an Independent Review Committee from a clinical trial to confirm the clinical benefit of lurbinectedin in SCLC that may inform product labeling. This could be from the Study titled, “Phase III Randomized Clinical Trial of Lurbinectedin (PM01183)/Doxorubicin Versus Cyclophosphamide, Doxorubicin and Vincristine (CAV) or Topotecan as Treatment in Patients With SCLC Who Failed One Prior Platinumcontaining Line (ATLANTIS)”. 2/28/2021
Rubraca (rucaparib) Treatment of adult patients with a deleterious BRCA mutation (germline and/or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor-directed therapy and a taxane-based chemotherapy 5/15/2020 3833-1: Submit the PFS and OS analyses and datasets demonstrating clinical benefit of rucaparib from a phase 3 randomized trial in patients with mCRPC associated with homologous recombination deficiency who have been treated with androgen receptor directed therapy. The results may inform product labeling. 10/31/2025
Pomalyst (pomalidomide) Treatment of patients with AIDS-related Kaposi’s Sarcoma after failure of highly active antiretroviral therapy (HAART). 5/14/2020 3855-1: Submit the final report from a clinical trial evaluating ORR , DOR, and safety to verify and describe the clinical benefit of pomalidomide in patients with Kaposi sarcoma who are HIV positive (after failure of HAART) and HIV negative, that may inform product labeling. 12/31/2027
Pomalyst (pomalidomide) Treatment of Kaposi’s Sarcoma in patients who are HIV-negative 5/14/2020 3855-1: Submit the final report from a clinical trial evaluating ORR, DOR, and safety to verify and describe the clinical benefit of pomalidomide in patients with Kaposi sarcoma who are HIV positive (after failure of HAART) and HIV negative, that may inform product labeling. 12/31/2027
Retevmo (selpercatinib) Adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy * 5/8/2020 3829-1: Submit the final report including datasets from a multi-center, randomized trial comparing selpercatinib to physician’s choice of approved therapies in patients with kinase inhibitor-naïve, progressive, advanced or metastatic RET-mutant MTC to confirm clinical benefit of selpercatinib with PFS as a key secondary end point as assessed by blinded independent central review. 10/31/2026
Pemazyre (pemigatinib) Treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. 4/17/2020 3801-1: Submit the results of a randomized trial demonstrating improvement of PFS or OS in patients with unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 gene fusion or rearrangement, to confirm clinical benefit of pemigatinib. 12/31/2026
Yervoy (ipilimumab) In combination with nivolumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib 3/10/2020 3823-1: Submit the final report demonstrating an improvement in OS from a multicenter, randomized trial to confirm the clinical benefit of nivolumab in combination with ipilimumab over standard therapy in patients with advanced HCC, that may inform product labeling. Submit the datasets with the final report. 7/31/2024
Opdivo (nivolumab) In combination with ipilimumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib 3/10/2020 3822-1: Submit the final report demonstrating an improvement in OS from a multicenter, randomized trial to confirm the clinical benefit of nivolumab in combination with ipilimumab over standard therapy in patients with advanced HCC, that may inform product labeling. Submit the datasets with the final report. 7/31/2024
Tazverik (tazemetostat) Treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection. 1/23/2020 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.
3787-2: Submit the final report and datasets for the final analysis of ORR and DOR 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.
9/30/2029
Brukinsa (zanubrutinib) Treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. 11/14/2019 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 MCL. The primary endpoint is PFS as assessed by Independent Review Committee (IRC). OS is a key secondary endpoint. PFS and OS would be analyzed based on superiority testing. Enrollment of approximately 500 patients is expected 2/28/2027
Rozlytrek (entrectinib) Treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy* 8/15/2019

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 ORR and mature response duration per independent review assessment, in adult and pediatric patients 12 years of age and older with solid tumors with a 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, CRC, CNS cancers, GYN cancers, and melanoma. A minimum of 40 patients with cancers other than pediatric solid tumors, CRC, CNS cancers, GYN cancers, melanoma, STS, non-small cell adenocarcinoma lung cancer, mammary analogue secretory carcinoma, and secretory breast cancer will also be studied. ORR and DOR will be assessed by independent central review and all responding patients will be followed for at least 12 months from the onset of response.

3689-1: Submit the final report, including datasets, from the first 54 patients with NTRK-fusion solid tumors enrolled across the ALKA, STARTRK-1 [NCT02097810], and STARTRK-2 [NCT02568267] studies to verify and describe the clinical benefit and further characterize the DOR in patients who achieved a complete or partial response to entrectinib. All responding patients will be followed for at least 2 years from the onset of response or until disease progression, whichever comes first. DOR will be assessed by independent central review.*

3/31/2027
Vitrakvi (Capsules) (larotrectinib) Treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation; are metastatic or where surgical resection is likely to result in severe morbidity; and have no satisfactory alternative treatments or that have progressed following treatment 11/26/2018 3540-1: Submit the final report, including datasets, from ongoing and proposed trials conducted to verify and describe the clinical benefit of larotrectinib, through more precise estimation of the ORR and mature response duration per independent review assessment, in adult and pediatric patients with solid tumors with a NTRK gene fusion and without a known acquired resistance mutation; are metastatic or where surgical resection is likely to 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 characterize response and durability of response for each of the following tumor types: CRC, NSCLC, CNS tumors, and melanoma. A minimum of 40 patients with cancers other than CRC, NSCLC, CNS tumors, melanoma, STS, thyroid cancer, infantile fibrosarcoma, and salivary cancers (e.g., BC, GI stromal tumors, cholangiocarcinoma, biliary tract cancers) will also be studied. ORR and DOR will be assessed by independent central review and all responding patients will be followed for at least 12 months from the onset of response. 8/31/2025
Opdivo (nivolumab) In combination with ipilimumab, is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or DNA mismatch repair deficient (dMMR), metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan 7/10/2018 3449-1: 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 MSI-H or dMMR metastatic colorectal cancer. The trial will be designed to demonstrate a clinically meaningful improvement in PFS 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 OS between arms based on a pre-specified analysis. The analysis plan should describe the power for the OS analysis, as well as all assumptions made in determining the power. 7/31/2024
Yervoy (ipilimumab) In combination with nivolumab, is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or DNA mismatch repair deficient (dMMR), metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan 7/10/2018 3450-1: Submit the final report, including datasets, from a randomized trial conducted to verify and describe the clinical benefit of ipilimumab, administered in combination with nivolumab, in patients with MSI-H or dMMR metastatic colorectal cancer. The trial will be designed to demonstrate a clinically meaningful improvement in PFS in patients randomized to receive ipilimumab and nivolumab as compared to patients randomized to receive nivolumab alone. In addition, the trial should evaluate for differences in OS between arms based on a pre-specified analysis. The analysis plan should describe the power for the OS analysis, as well as all assumptions made in determining the power. 7/31/2024
Calquence (acalabrutinib) Treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. 10/31/2017 3291-1: Submit the complete final report and datasets demonstrating clinical efficacy and safety from a randomized, double-blind, placebo-controlled, clinical trial of Calquence in combination with standard immunochemotherapy versus immunochemotherapy alone in patients with MCL. 11/30/2024
Opdivo (nivolumab) For the treatment of adult and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. 7/31/2017 3243-1: 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 MSI-H or dMMR CRC 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. 9/30/2021
Opdivo (nivolumab) Treatment of adult patients with classical Hodgkin lymphoma that has relapsed or progressed after: 4/25/2017 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 PFS as determined by an independent review committee. OS would be a key secondary endpoint. 12/31/2026
Opdivo (nivolumab) For the treatment of classical Hodgkin Lymphoma that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and post-transplantation brentuximab vedotin. 5/17/2016 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 PFS as determined by an independent review committee. OS would be a key secondary endpoint. 12/31/2026
Beleodaq (belinostat) Treatment of relapsed or refractory peripheral T-Cell Lymphoma (PTCL) 7/3/2014 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 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 OS. Submit a complete final report with all supporting datasets.
2178-1: Establish the optimal and safe dose of belinostat in combination with the CHOP regimen. Perform a Phase 1 dose finding trial of belinostat plus CHOP for the treatment of patients with PTCL. Enroll a sufficient number of patients to characterize the safety of belinostat in combination with the CHOP regimen. Submit a complete final report with all supporting datasets
1/31/2021
Folotyn (pralatrexate) Treatment of relapsed or refractory peripheral T-Cell Lymphoma (PTCL) 9/24/2009 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 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 OS. Submit a complete final report with all supporting datasets.
2179-1 : Establish the optimal and safe dose of pralatrexate in combination with the CHOP regimen. Perform a Phase 1 dose-finding trial of pralatrexate plus CHOP for the treatment of patients with PTCL. Enroll a sufficient number of patients to characterize the safety of pralatrexate in combination with the CHOP regimen. Submit a complete final report with all supporting datasets.
6/30/2017

1. If multiple PMRs were required, the latest date is reported here. This date represents the original agreed-upon Final Study Report date and does not include any updated deadlines submitted by the Applicant.

2. a b The original AA indication was modified to include an expanded population.

3. The original AA indication was modified to include a restricted population

 

 
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