Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all orphan drugs for 2021.  

Prescription Drug User Fee Act (PDUFA) dates refer to deadlines for the FDA to review new drugs. The PDUFA date is 10 months after the drug application has been accepted by the FDA or 6 months, if the drug is given a priority review designation.  As each PDFUA date approaches, CheckRare will be covering the FDA meetings and outcomes. 


PDUFA Date Orphan Drug Indication Company Status
N/A Tepotinib Subset of non-small cell lung cancer Merck APPROVED 2.3.2021
2.11.2021 Evinacumab Homozygous familial hypercholesterolemia  Regeneron APPROVED 2.11.2021
2.15.2021 Umbralisib  Marginal zone lymphoma TG Therapeutics APPROVED 2.5.2021
2.25.2021 Casimersen Duchenne muscular dystrophy Sarepta Therapeutics APPROVED 2.25.2021
N/A Fosdenopterin Reduce the risk of mortality in molybdenum cofactor deficiency Type A BridgeBio/Origin Biosciences APPROVED 2.26.2021
2.28.2021 Melphalan flufenamide Relapsed/refractory multiple myeloma Oncopeptides APPROVED 2.26.2021
N/A Tivozanib Renal cell carcinoma Aveo Pharmaceuticals APPROVED 3.10.2021
5.14.2021 Pegcetacoplan Paroxysmal nocturnal hemoglobinuria Apellis APPROVED 5.14.2021
5.21.2021 Loncastuximab tesirine Certain types of relapsed or refractory large B-cell lymphoma ADC Therapeutics APPROVED 4.23.2021
6.1.2021 Infigratinib Cholangiocarcinoma whose disease meets certain criteria QED Therapeutics APPROVED 5.28.2021
N/A Plasminogen Acute lymphoblastic leukemia and lymphoblastic lymphoma in patients who are allergic to E. coli-derived asparaginase products, as a component of a chemotherapy regimen Jazz Pharmaceuticals APPROVED 6.30.2021
N/A Fexinidazole Human African trypanosomiasis caused by the parasite Trypanosoma brucei gambiense Sanofi Genzyme APPROVED 7.19.2021
7.20.2021 Odevixibat Pruritus (common in progressive familial intrahepatic cholestatis) Albireo APPROVED 7.20.2021
8.16.2021 Sotorasib Certain types of non-small cell lung cancer  Amgen APPROVED 5.28.2021
8.18.2021 Avalglucosidase alfa Late-onset Pompe disease Sanofi Genzyme APPROVED 8.6.2021
8.23.2021 Difelikefalin Moderate-to-severe pruritus associated with chronic kidney disease in certain populations Cara Therapeutics APPROVED 8.23.2021
N/A Skytrofa Short stature due to inadequate secretion of endogenous growth hormone Ascendis Pharma APPROVED 8.25.2021
8.30.2021 Belumosudil Chronic graft-versus-host disease Kadmon Pharmaceuticals APPROVED 7.16.2021
9.15.2021 Belzutifan von Hippel-Lindau disease under certain conditions  Merck APPROVED 8.13.2021
9.19.2021 Zanubrutinib Marginal zone lymphoma BeiGene APPROVED 9.15.2021
9.22.2021 Ruxolitinib Steroid-refractory chronic graft versus host disease Incyte sNDA accepted
9.29.2021 Maralixibat Alagille syndrome Mirum Pharmaceuticals NDA accepted
10.1.2021 Brexucabtagene autoleucel Acute lymphoblastic lymphoma Gilead sBLA accepted
10.4.2021 hGH-CTP Grown hormone deficiency in children Opko Health / Pfizer BLA accepted
10.7.2021 CCX168 Associated vasculitis  ChemoCentryx NDA accepted
10.15.2021 FT-218 Excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy Avadel Pharmaceuticals NDA accepted
10.15.2021 Treprostinil Pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease Liquidia Corporation NDA accepted
10.18.2021 Zanubrutinib  Waldenström’s macroglobulinemia BeiGene sNDA accepted
11.19.2021 Vosoritide Achondroplasia Biomarin NDA accepted
11.29.2021 Ciltacabtagene autoleucel Relapsed multiple myeloma Legend biotech/Janssen BLA accepted
11.30.2021 Oleogel-S10 Epidermolysis bullosa Amryt NDA accepted
11.30.2021 Pactitinib Myelofibrosis CTI Biopharm NDA accepted
12.15.2021 Nefecon IgA nephropathy Calliditas Therapeutics NDA accepted