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 |
| 1.16.2025 | Acalabrutinib | Mantle cell lymphoma | Calquence, AstraZeneca | APPROVED |
| 1.21.2025 | Treosulfan | Allogeneic hematopoietic stem cell transplantation (alloHSCT) | Medexus | APPROVED |
| 2.17.2025 | Vimseltinib | Tenosynovial giant cell tumor (TGCT) | Deciphera Pharmaceuticals | APPROVED |
| 2.21.2025 | Ctexli (chenodiol) | Cerebrotendinous xanthomatosis | Mirum Therapeutics | APPROVED |
| 2.28.2025 | Mirdametinib | Neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) | Springworks Therapeutics | APPROVED |
| 3.6.2025 | Encelto (revakinagene taroretcel-lwey) | Macular telangiectasia type 2 (MacTel) | Neurotech Pharmaceuticals | APPROVED |
| 3.20.2025 | Fabhalta (iptacopan) | C3 glomerulopathy (C3G) | Novartis | APPROVED |
| 3.20.2025 | Rivoceranib/Camrelizumab | Unresectable or metastatic hepatocellular carcinoma | Elevar Therapeutics | Complete Response Letter |
| 3.23.2025 | Vutrisiran | ATTR-CM | Alnylam Pharmaceuticals | APPROVED |
| 3.26.2025 | Cabozantinib | Pancreatic neuroendocrine tumors | Exelixis | APPROVED |
| 3.27.2025 | Diazoxide choline (DCCR) | Prader-Willi syndrome | Soleno Therapeutics | APPROVED |
| 3.28.2025 | Fitusiran | Hemophilia A or B | Sanofi | APPROVED |
| 4.2.2025 | Vanrafia (atrasentan) | IgA nephropathy | Novartis | APPROVED |
| 4.3.2025 | Uplizna (inebilizumab) | Immunoglobulin G4-related disease (IgG4-RD) | Amgen | APPROVED |
| 4.21.2025 | Nivolumab plus ipilimumab | Hepatocellular carcinoma (HCC) | Bristol Myers Squibb | APPROVED |
| 4.23.2025 | Penpulimab | Non-keratinizing nasopharyngeal carcinoma (NPC) | Akeso | APPROVED |
| 4.29.2025 | Prademagene zamikeracel (pz-cel) | Epidermolysis bullosa | Abeona Therapeutics | APPROVED |
| 4.29.2025 | Elamipretide | Barth syndrome | Stealth BioTherapeutics | APPROVED |
| 4.29.2025 | Rinvoq (upadacitinib) | Giant cell arteritis (GCA) | AbbVie | APPROVED |
| 4.30.2025 | Nipocalimab | Generalized myasthenia gravis | Johnson & Johnson | APPROVED |
| 5.24.2025 | Yutrepia (treprostinil) | Pulmonary arterial hypertension | Liquidia Corporation | APPROVED |
| 5.26.2025 | Welireg (belzutifan) | Advanced pheochromocytoma and paraganglioma (PPGL) | Merck | APPROVED |
| 5.28.2025 | Khindivi (hydrocortisone) | Pediatric adrenal insufficiency | Eton Pharmaceuticals | APPROVED |
| 6.17.2025 | Sebetralstat | Hereditary angioedema | KalVista Pharmaceuticals | PDUFA Delayed |
| 6.18.2025 | Andembry (garadacimab) | Hereditary angioedema | CSL | APPROVED |
| 6.19.2025 | Nitisinone | Alkaptonuria | Cycle Pharmaceuticals | APPROVED |
| 6.20.2025 | Dupixent | Bullous Pemphigoid | Sanofi | APPROVED |
| 6.23.2025 | Taletrectinib | Advanced ROS1-positive non-small cell lung cancer | Nuvation Bio | APPROVED |
| 6.28.2025 | Gamifant (emapalumab) | Still's disease | Sobi | APPROVED |
| 6.30.2025 | Gammagard Liquid ERC (immune globulin infusion) | Primary immunodeficiencies | Takeda | APPROVED |
| 7.2.2025 | Sunvozertinib | NSCLC with EGFR exon 20 insertion mutations | Dizal | APPROVED |
| 7.10.2025 | Linvoseltamab | Multiple myeloma | Regeneron Pharmaceuticals | APPROVED |
| 7.24.2025 | Avatrombopag | Immune thrombocytopenia | Sobi North America | APPROVED |
| 7.27.2025 | TransCon hGH | Growth hormone deficiency | Ascendis Pharma | APPROVED |
| 7.28.2025 | Empaveli (pegcetacoplan) | C3G and primary IC-MPGN | Apellis Pharmaceuticals | APPROVED |
| 7.29.2025 | Sepiapterin | Phenylketonuria (PKU) | PTC Therapeutics | APPROVED |
| 7.30.2025 | Odronextamab | Follicular lymphoma | Regeneron Pharmaceuticals | Complete Response Letter |
| 7.31.2025 | Alhemo (concizumab) | Hemophilia A or B without inhibitors | Novo Nordisk | APPROVED |
| 8.8.2025 | Zongertinib | NSCLC with HER2 (ERBB2) TKD activating mutations | Boehringer Ingelheim Pharmaceuticals | APPROVED |
| 8.18.2025 | UX111 | Sanfilippo syndrome (MPS IIIA) | Ultragenyx | Complete Response Letter |
| 8.18.2025 | Dordaviprone | H3 K27M-mutant diffuse glioma | Jazz Pharmaceuticals | APPROVED |
| 8.19.2025 | Vatiquinone | Friedreich's ataxia | PTC Therapeutics | Complete Response Letter |
| 8.21.2025 | Donidalorsen | Hereditary Angioedema | Ionis Pharmaceuticals | APPROVED |
| 8.27.2025 | PRGN-2012 (zopapogene imadenovec) | Recurrent respiratory papillomatosis (RRP) | Precigen | APPROVED |
| 8.28.2025 | Filspari | IgA nephropathy | Travere Therapeutics | APPROVED |
| 8.29.2025 | Rilzabrutinib | Immune thrombocytopenia | Sanofi | APPROVED |
| 8.31.2025 | Deramiocel | Duchenne muscular dystrophy | Capricor Therapeutics | Complete Response Letter |
| 9.22.2025 | Apitegromab | Spinal muscular atrophy | Scholar Rock | Complete Response Letter |
| 9.25.2025 | Paltusotine | Acromegaly | Crinetics Pharmaceuticals | APPROVED |
| 9.30.2025 | CUTX-101 (Copper Histidinate) | Menkes disease | Fortress Biotech | Complete Response Letter |
| 10.9.2025 | Jascayd (nerandomilast) | Idiopathic pulmonary fibrosis | Boehringer Ingelheim | APPROVED |
| 10.19.2025 | Gazyva/Gazyvaro (obinutuzumab) | Lupus nephritis | Roche | APPROVED |
| 10.20.2025 | Epioxa (Epi-on) | Keratoconus | Glaukos Corporation | APPROVED |
| 10.23.2025 | Belantamab mafodotin | Multiple myeloma | GSK | APPROVED |
| 10.25.2025 | Revuforj (revumenib) | mNPM1 acute myeloid leukemia | Syndax Pharmaceuticals | APPROVED |
| 11.3.2025 | Kygevvi (doxecitine and doxribtimine) | Thymidine kinase 2 deficiency | UCB | APPROVED |
| 11.4.2025 | Vyglxia (troriluzole) | Spinocerebellar ataxia | Biohaven | Complete Response Letter |
| 11.6.2025 | Darzalex Faspro (daratumumab and hyaluronidase-fihj) | Smoldering multiple myeloma (SMM) | Johnson & Johnson | APPROVED |
| 11.7.2025 | Dasatinib | CML, AML | Alembic Pharmaceuticals | APPROVED |
| 11.18.2025 | Plozasiran | Familial chylomicronemia syndrome | Arrowhead Pharmaceuticals | APPROVED |
| 11.19.2025 | Koselugo (selumetinib) | Neurofibromatosis type 1 | AstraZeneca Pharmaceuticals | APPROVED |
| 11.19.2025 | Sevabertinib | Non-squamous non-small cell lung cancer | Bayer | APPROVED |
| 11.24.2025 | Itvisma (onasemnogene abeparvovec) | Spinal muscular atrophy (SMA) | Novartis | APPROVED |
| 11.28.2025 | Sibeprenlimab | Immunoglobulin A nephropathy (IgAN) | Otsuka Pharmaceutical Co. | APPROVED |
| 11.30.2025 | Ziftomenib | Relapsed or refractory acute myeloid leukemia (AML) with an NPM1 mutation | Kura Oncology and Kyowa Kirin | APPROVED |
| 12.4.2025 | Breyanzi (lisocabtagene maraleucel) | Marginal zone lymphoma | Juno Therapeutics | APPROVED |
| 12.7.2025 | Pyrukynd (mitapivat) | Alpha- and beta-thalassemia | Agios Pharmaceuticals | APPROVED |
| 12.9.2025 | Waskyra (etuvetidigene autotemcel) | Wiskott-Aldrich syndrome | Fondazione Telethon | APPROVED |
| 12.12.2025 | Orladeyo (berotralstat) | Hereditary angioedema (HAE) | BioCryst Pharmaceuticals | APPROVED |
| 12.14.2025 | Uplizna | Myasthenia gravis | Amgen | APPROVED |
| 12.26.2025 | Aficamten | Obstructive hypertrophic cardiomyopathy (oHCM) | Cytokinetics | APPROVED |
| 12.30.2025 | Relacorilant | Cushing's syndrome | Corcept Therapeutics | Complete Response Letter |
| Q4 2025 | Troriluzole | Spinocerebellar ataxia (SCA) | Biohaven | Complete Response Letter |