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 | APPROVED 9.22.2001 |
9.29.2021 | Maralixibat | Alagille syndrome | Mirum Pharmaceuticals | APPROVED 9.29.2021 |
10.1.2021 | Brexucabtagene autoleucel | Acute lymphoblastic lymphoma | Gilead | APPROVED 10.1.2021 |
10.7.2021 | CCX168 | Associated vasculitis | ChemoCentryx | APPROVED 10.7.2021 |
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 | COMPLETE RESPONSE DECLINING TO APPROVE 10.18.2021 |
10.18.2021 | Zanubrutinib | Waldenström’s macroglobulinemia | BeiGene | APPROVED 8.31.2021 |
11.19.2021 | Vosoritide | Achondroplasia | Biomarin | APPROVED 11.19.21 |
11.29.2021 | Ciltacabtagene autoleucel | Relapsed multiple myeloma | Legend biotech/Janssen | DELAYED DECISION |
2.28.2022 | Oleogel-S10 | Epidermolysis bullosa | Amryt | NDA accepted |
2.28.2021 | Pactitinib | Myelofibrosis | CTI Biopharm | NDA accepted |
12.15.2021 | Nefecon | IgA nephropathy | Calliditas Therapeutics | APPROVED 12.15.21 |
2024 Orphan Drugs: PDUFA Dates and FDA Approvals
2023 Orphan Drugs: PDUFA Dates and FDA Approvals
2022 Orphan Drugs: PDUFA Dates and FDA Approvals