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

Verified Clinical Benefit | 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 with postmarketing trials that have verified clinical benefit and for which traditional approval has been subsequently granted for the specific indication.

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Visit Accelerated Approvals for listings of ongoing, verified clinical benefit, and withdrawn infectious diseases and non-malignant hematological, neurological, and other disorder indications accelerated approvals.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

Total approval time was adjusted based on management decision. This is a legacy practice and is no longer exercised.

Drug Name Accelerated Approval Indication Accelerated Approval Date Traditional Approval Date
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 6/20/2024
Provayblue (methylene blue) For the treatment of pediatric and adult patients with acquired methemoglobinemia 4/8/2016 1/8/2024
Tarpeyo (budesonide) 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 12/15/2021 12/20/2023
Leqembi (lecanemab-irmb) For the treatment of Alzheimer's Disease 1/6/2023 7/6/2023
Carticel (autologous cultured chondrocytes) Articular cartilage defects in the knee 8/22/1997 6/21/2007
RiaSTAP (human; fibrinogen concentrate) Treatment of acute bleeding episodes in patients with congenital fibrinogen deficiency (afibrinogenemia and hypofibrinogenemia) 1/16/2009 6/4/2021
Corifact (human; human factor XIII concentrate) Routine prophylactic treatment of congenital factor XIII deficiency 2/17/2011 1/24/2013
Ferriprox (deferiprone) For the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate 5/19/2020 4/30/2021
Jadenu Sprinkle (deferasirox) For the treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age and older 5/18/2017 5/11/2018
Jadenu Sprinkle (deferasirox) For the treatment of chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia (ntdt) syndromes and with a liver iron concentration (lic) of at least 5 milligrams of iron per gram of liver dry weight (mg fe/g dw) and a serum ferritin greater than 300 mcg/l 5/18/2017  7/23/2020
Praxbind (idarucizumab) For the treatment of patients treated with pradaxa® when reversal of the anticoagulant effects of dabigatran is needed for emergency surgery/urgent procedures and in life-threatening or uncontrolled bleeding 10/16/2015 4/12/2018
Ferriprox (deferiprone) For the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate 9/9/2015 4/30/2021
Jadenu (deferasirox) For the treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age and older 3/30/2015 5/11/2018
Jadenu (deferasirox) For the treatment of chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia (ntdt) syndromes and with a liver iron concentration (lic) of at least 5 milligrams of iron per gram of liver dry weight (mg fe/g dw) and a serum ferritin greater than 300 mcg/l 3/30/2015 7/23/2020
Exjade (deferasirox) For the treatment of chronic iron overload in patients 10 years of age and older with non-transfusion dependent thalassemia (ntdt) syndromes and with a liver iron concentration (lic) of at least5 milligrams of iron per gram of liver dry weight (mg fe/g dw) and a serum ferritin greater than 300 mcg/l 1/23/2013 7/23/2020
Ferriprox (deferiprone) For the treatment of transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate 10/14/2011 4/30/2021
Promacta (eltrombopag) For the treatment thrombocytopneia in patients with chronic immune (idiopathic) thrombocytopenia (itp) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy  11/20/2008 2/25/2011
Exjade (deferasirox) For the treatment of chronic iron overload due to blood transfusions in patients 2 years of age and older 11/2/2005 5/11/2018
Remodulin (treprostinil sodium) Provides for adding the infusion of remodulin (treprostinil sodium) 1, 2.5, 5 & 10 mg/ml injection via an indwelling central venous catheter to the labeling for the treatment of pulmonary arterial hypertension (pah) 11/24/2004 3/20/2006
Tysabri (natalizumab) For the treatment of patients with relapsing forms of multiple sclerosis (ms) to reduce the frequency of clinical exacerbations 11/23/2004 6/5/2006
Fabrazyme (agalsidase beta) For the treatment of fabry disease  4/24/2003 3/11/2021
Remodulin (treprostinil sodium) Treatment of pulmonary arterial hypertension 5/21/2002 3/20/2006
Remicade (infliximab) For the treatment of moderately to severely active crohn's disease for the reduction of the signs and symptoms, in patients who have an inadequate response to conventional therapies and treatment of patients with fistulizing crohn's disease for the reduction in the number of draining enterocutaneous fistulas 8/24/1998 4/1/2003
Betaseron (interferon beta-1b) Use in ambulatory patients with relapsing-remitting multiple sclerosis to reduce the frequency of clinical exacerbations 7/23/1993 3/14/2003
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