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  8. Ongoing | Non-malignant Hematological, Neurological, and Other Disorder Indications Accelerated Approvals
  1. Accelerated Approval Program

Ongoing | Non-malignant Hematological, Neurological, and Other Disorder Indications Accelerated Approvals

This listing includes accelerated approvals (AAs) for non-malignant hematological, neurological, and other disorder 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. See Postmarket Requirements and Commitments for the status of specific requirements. 

Visit Accelerated Approvals for listings of ongoing, verified clinical benefit, and withdrawn infectious diseases and non-malignant hematological, neurological, and other disorder indications accelerated approvals.

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

Ongoing 1 Heme/Neuro/Other Accelerated Approvals

Drug Name  Accelerated Approval Indication Accelerated Approval Date AA PMR Original Projected Completion Date 2
Qalsody (tofersen) For the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. 4/25/2023 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. 6/30/2028
Elevidys (delandistrogene moxeparvovec-rokl) Treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. 6/22/2023 PMR 1: Complete Study SRP-9001-301 Part 1, an ongoing, randomized, double-blinded clinical trial intended to describe and verify clinical benefit of delandistrogene moxeparvovec-rokl in ambulatory patients with Duchenne muscular dystrophy (DMD). The trial evaluates the primary endpoint of North Star Ambulatory Assessment (NSAA) and compares delandistrogene moxeparvovec-rokl to placebo in 125 ambulatory patients with DMD with confirmed mutation in the DMD gene. 1/31/2024
Filspari (sparsentan) To reduce proteinuria in adults with primary immunoglobulin a nephropathy (IGAN) at risk of rapid disease progression, generally a urine protein to creatinine ratio (UPCR) ≥ 1.5 G/G. 2/17/2023 PMR 4330-1: Conduct a randomized, double-blind, placebo-controlled trial to describe and verify the clinical benefit of sparsentan for the treatment of 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. 2/28/2024
Vonjo (pacritinib) For the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 x 109/L 2/28/2022 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. 6/30/2026
Skysona (elivaldogene autotemcel) Indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (cald) 9/16/2022 PMR 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.   7/31/2032
Skysona (elivaldogene autotemcel) Indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (cald) 9/16/2022 PMR 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) 12/31/2038
Oxbryta (tablets; voxelotor) For the treatment of sickle cell disease in adults and pediatric patients 4 years of age and older 12/17/2021 PMR 3746-1: 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. 9/30/2026
Oxbryta (tablets for oral suspension; voxelotor) For the treatment of sickle cell disease in adults and pediatric patients 4 years of age and older 12/17/2021 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. 9/30/2026
Voxzogo (vosoritide)

Injection for the treatment of all pediatric patients to increase linear growth in patients with achondroplasia with open epiphyses

 10/20/2023 PMR 4134-2 (This PMR superseded PMR 4134-1): 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.  8/31/2026
Aduhelm (aducanumab-avwa) For the treatment of alzheimer’s disease 6/7/2021 PMR 3971-1: In order to verify the clinical benefit of aducanumab, conduct a randomized, controlled trial to evaluate the efficacy of aducanumab-avwa compared to an appropriate control for the treatment of Alzheimer’s disease. The trial should be of sufficient duration to observe changes on an acceptable endpoint in the patient population enrolled in the trial. 2/28/2030
Amondys 45 (casimersen) For the treatment of duchenne muscular dystrophy (dmd) in patients who have a confirmed mutation of the dmd gene that is amenable to exon 45 skipping 2/25/2021 PMR 4005-1: In order to verify the clinical benefit of casimersen, complete Study 4045­301 (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. The study includes a randomized, double-blind, placebo-controlled period of 96-weeks and concludes after an open label extension period to 144 weeks. The primary endpoint will be the 6-minute walk test.  10/31/2024
Viltepso (viltolarsen) For the treatment of duchenne muscular dystrophy (dmd) in patients who have a confirmed mutation of the dmd gene that is amenable to exon 53 skipping 8/12/2020 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. 12/31/2024
Vyondys 53 (golodirsen) For the treatment of duchenne muscular dystrophy (dmd) in patients who have a confirmed mutation of the dmd gene that is amenable to exon 53 skipping  12/12/2019 PMR 3690-1: In order to verify the clinical benefit of golodirsen, 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. The study includes a randomized, double-blind, placebo-controlled period of 96 weeks, and concludes after an open-label extension period to 144 weeks. The primary endpoint will be the 6-minute walk test. 10/30/2024
Oxbryta (tablets; voxelotor) For the treatment of sickle cell disease in adults and pediatric patients 12 years of age and older 11/25/2019 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. 9/30/2026
Galafold (migalastat) For the treatment of adults with a confirmed diagnosis of fabry disease and an amenable galactosidase alpha gene (gla) variant based on in vitro assay data  8/10/2018 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. 8/31/2026
Galafold (migalastat) For the treatment of adults with a confirmed diagnosis of fabry disease and an amenable galactosidase alpha gene (gla) variant based on in vitro assay data 8/10/2018 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. 4/30/2031
Andexxa (coagulation factor xa (recombinant), inactivated-zhzo) For patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding 5/3/2018 PMR 1: Study-18-513: "A Phase 4 randomized trial of ANDEXXA in acute intracranial hemorrhage in patients receiving oral factor Xa inhibitors": This open-label, randomized trial will include at least 440 adult patients who developed acute intracranial hemorrhage following the treatment with rivaroxaban, apixaban, or edoxaban 15 hours or less prior to randomization. The enrolled patients will be administered ANDEXXA (high or low dose) or standard of care other than ANDEXXA according to 1:1 randomization scheme. To describe and verify the hemostatic effect of ANDEXXA, patients will be assessed with the National Institute of Health Stroke Scale and computed tomography or magnetic resonance imaging at 12-hours post- randomization. The trial assessments will also include evaluation of occurrence of the safety events of special interest, including but not limited to: stroke, transient ischemic event, acute myocardial infarction, deep vein thrombosis, pulmonary embolism, arterial systemic embolism, sudden death, and events suspicious for thrombosis, embolism, and ischemia, all to be observed at least 3 days for immediate occurrence and at least 30 days with weekly intervals for delayed occurrence. The assessments of the hemostatic effect will be made by an adjudication committee blinded to the treatment allocation. 4/30/2023
Exondys 51 (eteplirsen) For the treatment of duchenne muscular dystrophy (dmd) in patients who have a confirmed mutation of the dmd gene that is amenable to exon 51 skipping 9/19/2016 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. 5/31/2021
Ocaliva (obeticholic acid) For the treatment of primary biliary cholangitis (pbc) in combination withursodeoxycholic acid (udca) in adults with an inadequate response to udca, or as monotherapy in adults unable to tolerate udca 5/27/2016 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). 4/30/2023
Ocaliva (obeticholic acid) For the treatment of primary biliary cholangitis (pbc) in combination withursodeoxycholic acid (udca) in adults with an inadequate response to udca, or as monotherapy in adults unable to tolerate udca 5/27/2016 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 standalone trial or in a subset of patients in your confirmatory trial (PMR # 3057-3). 4/30/2023
Ocaliva (obeticholic acid) For the treatment of primary biliary cholangitis (pbc) in combination withursodeoxycholic acid (udca) in adults with an inadequate response to udca, or as monotherapy in adults unable to tolerate udca 5/27/2016 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. 4/30/2023
Provayblue (methylene blue) For the treatment of pediatric and adult patients with acquired methemoglobinemia 4/8/2016 PMR 3065-1: Design and conduct a study and provide the full final report and data sets to evaluate the safety and efficacy of ProvayBlue (methylene blue) for the treatment of methemoglobinemia. The minimal efficacy endpoints should include achieving a 50% reduction in methemoglobin within one hour of  the first dose of ProvayBlue (methylene blue) in addition to normalization of the respiratory rate, heart rate and blood pressure within two hours of the first dose of ProvayBlue (methylene blue). 9/30/2020
Northera (droxidopa) For the treatment of orthostatic dizziness, lightheadedness, or the “feeling that you are about to black out” in adult patients with symptomatic neurogenic orthostatic hypotension caused by primary autonomic failure (parkinson's disease, multiple system atrophy,and pure autonomic failure), dopamine beta-hydroxylase deficiency, and non-diabetic autonomic neuropathy 2/18/2014 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, placebo controlled, withdrawal period. The primary endpoint will be time to treatment intervention. 8/31/2021
Elaprase (idursulfase) Provides for additional safety and efficacy information for the treatment of patients with hunter syndrome 5 years of age and younger  6/24/2013 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 z-scores) 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.  9/30/2022
Elaprase (idursulfase) Provides for additional safety and efficacy information for the treatment of patients with hunter syndrome 5 years of age and younger  6/24/2013 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. 9/30/2022
Proamatine (midodrine hydrochloride) For the treatment of symptomatic orthostatic hypotension (oh) 9/6/1996 PMR 825-1: Applicant to conduct a Phase 4 study to confirm the clinical benefit of midodrine. Protocol drafts submitted at 7/18/1996 meeting. Protocols are 401, 402, and 403. 3/31/2015
  • 1Ongoing" refers to accelerated approval (AA) applications that have not yet converted to full approval, and not to the status of the AA postmarketing requirement (PMR).
  • 2If 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.
 
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