Below is the list of important regulatory dates for all orphan drugs for 2026.
Prescription Drug User Fee Act (PDUFA) dates refer to deadlines for the FDA to review new drugs.
| PDUFA Date | Orphan Drug | Indication | Company | Status |
|---|---|---|---|---|
| 1.5.2026 | Cablivi (caplacizumab) | Acquired thrombotic thrombocytopenic purpura (aTTP) | Sanofi | APPROVED |
| 1.10.2026 | Tabelecleucel | Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) | Atara Biotherapeutics | Complete Response Letter |
| 1.13.2026 | Cerezyme (imiglucerase) | Gaucher disease type 3 | Sanofi | APPROVED |
| 1.14.2026 | Copper histidinate (CUTX-101) | Menkes disease | Sentynl Therapeutics | APPROVED |
| 1.27.2026 | Darzalex Faspro with D-VRd | Multiple myeloma | Johnson & Johnson | APPROVED |
| 1.31.2026 | Leniolisib | Activated phosphoinositide 3-kinase delta syndrome (APDS) | Pharming Group | Complete Response Letter |
| 2.8.2026 | Clemidsogene lanparvovec (RGX-121) | Mucopolysaccharidosis II (MPS II) | REGENXBIO | Complete Response Letter |
| 2.13.2026 | Bitopertin | Erythropoietic protoporphyria (EPP) | Disc Medicine | Complete Response Letter |
| 2.20.2026 | Venetoclax plus acalabrutinib | Chronic lymphocytic leukemia | AbbVie | APPROVED |
| 2.23.2026 | Pegzilarginase | Arginase 1 deficiency (ARG1-D) | Immedica Pharma AB | APPROVED |
| 2.25.2026 | Inqovi (decitabine and cedazuridine) plus Venclexta (venetoclax) | Acute myeloid leukemia | Taiho Oncology | sNDA accepted |
| 2.25.2026 | ET-600 | Arginine vasopressin deficiency (AVP-D) | Eton Pharmaceuticals | APPROVED |
| 2.28.2026 | Palynziq (pegvaliase) | Phenylketonuria (PKU) | BioMarin | APPROVED |
| 2.28.2026 | TransCon CNP (navepegritide) | Achondroplasia | Ascendis Pharma | APPROVED |
| 2.28.2026 | Idebenone | Leber hereditary optic neuropathy (LHON) | Chiesi | Complete Response Letter |
| 3.5.2026 | Tec-Dara [Tecvayli(teclistamab) plus Darzalex Faspro (daratumumab and hyaluronidase)] | Multiple myeloma | Johnson & Johnson | APPROVED |
| 3.3.2026 | Juxtapid (lomitapide) | Homozygous familial hypercholesterolemia (HoFH) | Chiesi | APPROVED |
| 3.10.2026 | Wellcovorin (leucovorin calcium) | Cerebral folate deficiency | GSK | APPROVED |
| 3.24.2026 | Linerixibat | Primary biliary cholangitis | Alfasigma | APPROVED |
| 3.28.2026 | Kresladi (marnetegragene autotemcel) | Leukocyte adhesion deficiency-I (LAD-I) | Rocket Pharmaceuticals | APPROVED |
| 4.2.2026 | Tecartus (brexucabtagene autoleucel) | Mantle cell lymphoma | Gilead/Kite Oncology | APPROVED |
| 4.3.2026 | Nusinersen high-dose regimen | Spinal muscular atrophy | Biogen | APPROVED |
| 4.5.2026 | Tividenofusp alfa | Hunter syndrome (MPS II) | Denali Therapeutics | APPROVED |
| 4.8.2026 | Opdivo (nivolumab) | Stage III or IV classical Hodgkin Lymphoma (cHL) | Bristol Myers Squib | APPROVED |
| 4.13.2026 | FILSPARI (sparsentan) | Focal segmental glomerulosclerosis (FSGS) | Travere Therapeutics | APPROVED |
| 4.23.2026 | Otarmeni (lunsotogene parvec-cwha) | OTOF-related hearing loss | Regeneron Pharmaceuticals | APPROVED |
| 5.10.2026 | Vyvgart (efgartigimod alfa-fcab) | AChR-Ab seronegative generalized myasthenia gravis (gMG) | Argenx | sBLA accepted |
| 6.10.2026 | Oclaiz (CAM2029) | Acromegaly | Camurus | NDA accepted |
| 6.18.2026 | XS003 (nilotinib) | Chronic myeloid leukemia (CML) | Xspray Pharma | NDA accepted |
| 6.29.2026 | LNTH-2501 (Gallium-68 edotreotide) | Somatostatin receptor-positive (SSTR+) neuroendocrine tumors (NETs) | Lantheus Holdings | Delayed |
| 6.30.2026 | Veligrotug | Thyroid eye disase | Viridian Therapeutics | BLA accepted |
| Q3 2026 | Rusfertide | Polycythemia vera (PV) | Takeda and Protagonist Therapeutics | NDA accepeted |
| 7.6.2026 | Orca-T | Acute myeloid leukemia, acute lymphoblastic leukemia, and myelodysplastic syndromes | Orca Bio | BLA accepted |
| 7.7.2026 | Atacicept | IgA Nephropathy | Vera Therapeutics | BLA accepted |
| 8.17.2026 | Iberdomide | Multiple Myeloma | Bristol Myers Squib | NDA accepted |
| 7.23.2026 | Sarclisa | Multiple Myeloma | Sanofi | sBLA accepted |
| 8.17.2026 | Adrabetadex | Niemann-Pick type C | Beren Therapeutics | NDA accepted |
| 8.22.2026 | Deramiocel | Duchenne muscular dystrophy | Capricor Therapeutics | CRL lifted; BLA accepted |
| 8.23.2026 | DTX401 (pariglasgene brecaparvovec) | Glycogen storage disease type 1a (GSD1a) | Ultragenyx | BLA accepted |
| 8.25.2026 | Dasynoc | Chronic myeloid leukemia (CML) and acute lymphoblastic leukemia (ALL) | Xspray Pharma | NDA accepted |
| 8.28.2026 | 177Lu-edotreotide | Gastroenteropancreatic neuroendocrine tumors | ITM | NDA accepted |
| 8.30.2026 | Besremi (ropeginterferon alfa-2b-njft) | Essential thrombocythemia | PharmaEssentia USA Corporation | sBLA accepted |
| 9.18.2026 | Zidesamtinib | ROS1-positive non-small cell lung cancer | Nuvalent | NDA accepted |
| 9.19.2026 | UX111 (rebisufligene etisparvovec) | Sanfilippo syndrome Type A (MPS IIIA) | Ultragenyx | BLA accepted |
| 8.22.2026 | Zilganersen | Alexander disease | Ionis Pharmaceuticals | NDA accepted |
| 9.27.2026 | Relutrigine | SCN2A and SCN8A DEEs | Praxis | NDA accepted |
| 9.27.2026 | Lirafugratinib | Cholangiocarcinoma | Elevar Therapeutics | NDA accepted |
| 9.28.2026 | Emcitate (tiratricol) | MCT8 deficiency | Egetis Therapeutics | NDA accepted |
| September 2026 | Brepocitinib | Dermatomyositis | Priovant Therapeutics | NDA accepted |
| Q4 2026 | Ivonescimab | EGFR-mutated NSCLC | Summit Therapeutics | BLA submitted |
| 10.10.2026 | Tabelecleucel | Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) | Pierre Fabre Pharmaceuticals | BLA accepted |
| 10.30.2026 | INO-3107 | Recurrent respiratory papillomatosis (RRP) | INOVIO | BLA accepted |
| 11.22.2026 | Molbreevi | Autoimmune pulmonary alveolar proteinosis (autoimmune PAP) | Savara | BLA accepted |
| 12.12.2026 | Imsidolimab | Generalized pustular psoriasis (GPP) | Vanda Pharmaceuticals | BLA accepted |
| 12.18.2026 | Tirabrutinib | R/R primary central nervous system lymphoma (PCNSL) | Deciphera Pharmaceuticals | NDA accepted |
| 12.23.2026 | Anito-cel | R/R multiple myeloma | Gilead | BLA accepted |
| 12.30.26 | Bezuclastinib | Nonadvanced systemic mastocytosis | Cogent Biosciences | NDA accepted |