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 2022.
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 |
| 1.29.2022 | Zonisamide oral suspension | Epilepsy | Eton Pharmaceuticals / Azurity Pharmaceuticals | APPROVED |
| 1.31.2022 | Somatrogon (hGH-CTP) | Children – Growth Hormone Deficiency | Opko Health / Pfizer | |
| 2.17.2022 | Mitapivat (AG-348) | Pyruvate Kinase Deficiency | Immunocore | APPROVED |
| 2.25.2022 | Bardoxolone methyl | Alport Syndrome | Reata Pharmaceuticals | Received CRL |
| 2.28.2022 | Filsuvez (Oleogel-S10) | Epidermolysis Bullosa | Amryt Pharma | Received CRL |
| 2.28.2022 | Ciltacabtagene autoleucel (cilta-cel) | Multiple Myeloma | Legend Biotech | APPROVED |
| 2.28.2021 | Pactitinib | Myelofibrosis | CTI Biopharm | APPROVED |
| 3.16.2022 | Imcivree (setmelanotide) | Alstrom Syndrome and Bardet-Biedl Syndrome | Rhythm Pharmaceuticals | PDUFA moved to June 16 |
| 3.20.2022 | Ganaxolone | CDKL5 Deficiency Disorder (CDD) | Marinus Pharmaceuticals | APPROVED |
| 3.25.2022 | FINTEPLA (fenfluramine) | Lennox-Gastaut syndrome | Zogenix Pharmaceuticals | APPROVED |
| 3.29.2022 | Vadadustat | Anemia due to chronic kidney disease (CKD) in patients on dialysis and not on dialysis. | Akebia Therapeutics | Received CRL |
| 4.2.2022 | Yescarta (axicabtagene ciloleucel) |
Initial Treatment of Relapsed or Refractory Large B-cell Lymphoma (LBCL) |
Kite (a Gilead Company) | APPROVED |
| 4.14.2022 | Vutrisiran | ATTR amyloidosis | Alnylam Pharmaceuticals | PDUFA moved to July 14 |
| 4.28.2022 | Mavacamten (MYK-461) | Hypertrophic cardiomyopathy (HCM) | BMS / LianBio | APPROVED |
| 4.30.2022 | Parsaclisib | Marginal Zone Lymphoma
Follicular lymphoma |
Incyte | NDA withdrawn |
| 4.30.2022 | Surufatinib | Neuroendocrine Tumors | Hutchmed | Received CRL |
| 5.20.2022 | Dupilumab (Dupixent) | Eosinophilic Esophagitis (EoE) | Regeneron / Sanofi | APPROVED |
| 6.5.2022 | ACER-101 | Urea cycle disorder (UCD) | Acer Therapeutics / Relief Therapeutics | WAITING |
| 6.14.2022 | Vutrisiran (Amvuttra) | Polyneuropathy observed in patients with hereditary ATTR (hATTR) amyloidosis | Alnylam | APPROVED |
| 6.17.2022 | Lenti-D (Elivaldogene Autotemcel / Eli-Cel) | Cerebral Adrenoleukodystrophy (CALD) | Bluebird bio | APPROVED |
| 6.24.2022 | Breyanzi (lisocabtagene maraleucel) | Relapsed or Refractory Large B-cell Lymphoma | Bristol Myers Squibb | APPROVED |
| 6.25.2022 | Ukoniq (umbralisib) and Ublituximab | Chronic Lymphocytic Leukemia (CLL) and Small Lymphocytic Lymphoma (SLL) | TG Therapeutics | BLA withdrawn |
| 6.27.2022 | Reblozyl (Luspatercept) | Non-transfusion-dependent beta-thalassemia | Bristol Myers Squibb | BLA withdrawn |
| 8.19.2022 | Betibeglogene autotemcel (beti-cel) (LentiGlobin) | β-Thalassemia | Bluebird bio | APPROVED |
| 8.26.2022 | Pemigatinib (pemazyre) | Relapsed or Refractory Myeloid/Lymphoid Neoplasms (MLNs) | Incyte | APPROVED |
| 9.29.2022 | AMX0035 | Amyotrophic Lateral Sclerosis (ALS) | Amylyx Pharmaceuticals | APPROVED |
| 9.30.2022 | Dupilumab | Prurigo Noduaris | sanofi | APPROVED |
| 10.6.2022 | Lumasiran | Primary Hyperoxaluria Type 1 | Alnylam Pharmaceuticals | APPROVED |
| 10.25.22 | Teclistamab-cqyv (Tecvayli) | Relapsed or Refractory Multiple Myeloma | Janssen | APPROVED |
| 10.29.2022 | AT-GAA – Propel | Pompe Disease | Amicus | FDA defers action |
| 11.30.2022 | Omburtamab | Neuroblastoma | Y-mAbs | Received CRL |
| 12.23.2022 | Toripalimab | Nasopharyngeal Carcinoma | Coherus | Decision Delayed |
| 1.15.2023 | Sodium phenylbutyrate | Urea Cycle Disorders | Acer | NDA resubmission |
| 1.20.2023 | Zanubritinib | CLL/SLL | Beigene | sNDA accepted |
| 1.30.2023 | Omidubicel | Blood Cancers | Gamida Cell | BLA accepted |
| 02.15.2023 | Olutasidenib | AML | Rigel | NDA accepted |
| 02.17.2023 | B-Vec | Epidermolysis bullosa | Krystal | BLA accepted |
| 02.28.2023 | Omaveloxolone | Friedreich’s ataxia | Reata | NDA accepted |
| 03.23.2023 | Trofinetide | Rett syndrome | Acadia Pharmaceuticals | NDA accepted |
| 05.29.2023 | SRP-9001 (delandistrogene moxeparvovec) | Duchenne muscular dystrophy | Sarepta Therapeutics | BLA accepted |
2021 Orphan Drugs: PDUFA Dates and FDA Approvals
2023 Orphan Drugs: PDUFA Dates and FDA Approvals
