Below is the list of important regulatory dates for all orphan drugs for 2025.
Prescription Drug User Fee Act (PDUFA) dates refer to deadlines for the FDA to review new drugs.
| PDUFA Date | Orphan Drug | Indication | Company | Status |
|---|---|---|---|---|
| 1.7.2025. | Ryoncil (remestemcel) | Steroid-refractory acute graft versus host disease | Mesoblast Limited | APPROVED |
| 1.15.2025 | Tabelecleucel (Tab-cel) | Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease | Atara Biotherapeutics | Complete Response Letter |
| 2.17.2025 | Vimseltinib | Tenosynovial giant cell tumor (TGCT) | Deciphera Pharmaceuticals | NDA accepted |
| 2.28.2025 | Mirdametinib | Neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) | Springworks Therapeutics | NDA accepted |
| 3.20.2025 | Rivoceranib/Camrelizumab | Unresectable or metastatic hepatocellular carcinoma | Elevar Therapeutics | NDA accepted |
| 3.23.2025 | Vutrisiran | ATTR-CM | Alnylam Pharmaceuticals | sNDA accepted |
| 3.27.2025 | Diazoxide choline (DCCR) | Prader-Willi syndrome | Soleno Therapeutics | NDA accepted |
| 3.28.2025 | Fitusiran | Hemophilia A or B | Sanofi | NDA accepted |
| 4.21.2025 | Nivolumab plus ipilimumab | Hepatocellular carcinoma (HCC) | Bristol Myers Squibb | sBLA accepted |
| 4.29.2025 | Prademagene zamikeracel (pz-cel) | Epidermolysis bullosa | Abeona Therapeutics | BLA accepted |
| 4.29.2025 | Elamipretide | Barth syndrome | Stealth BioTherapeutics | NDA accepted |
| 6.17.2025 | Sebetralstat | Hereditary Angioedema | KalVista Pharmaceuticals | NDA accepted |
| 6.23.2025 | Taletrectinib | Advanced ROS1-positive non-small cell lung cancer | Nuvation Bio | NDA accepted |
| 7.23.2025 | Belantamab mafodotin | Multiple myeloma | GSK | BLA accepted |
| 7.24.2025 | Avatrombopag | Immune thrombocytopenia | Sobi North America | sNDA accepted |
| 7.27.2025 | TransCon hGH | Growth hormone deficiency | Ascendis Pharma | sBLA accepted |
| 8.21.2025 | Donidalorsen | Hereditary Angioedema | Ionis Pharmaceuticals | NDA accepted |
| 8.28.2025 | Filspari | IgA nephropathy | Travere Therapeutics | sNDA accepted |
| 8.29.2025 | Rilzabrutinib | Immune thrombocytopenia | Sanofi | Under review |
| 9.7.2025 | Pyrukynd (mitapivat) | Beta-thalassemia | Agios Pharmaceuticals | sNDA accepted |
| 9.25.2025 | Paltusotine | Acromegaly | Crinetics Pharmaceuticals | NDA accepted |
| 11.18.2025 | Plozasiran | Familial chylomicronemia syndrome | Arrowhead Pharmaceuticals | NDA accepted |
