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 | BLA accepted |
| 2.23.2026 | Pegzilarginase | Arginase 1 deficiency (ARG1-D) | Immedica Pharma AB | BLA accepted |
| 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 | NDA accepted |
| 2.28.2026 | Palynziq (pegvaliase) | Phenylketonuria (PKU) | BioMarin | sBLA accepted |
| 2.28.2026 | TransCon CNP (navepegritide) | Achondroplasia | Ascendis Pharma | NDA accepted |
| 2.28.2026 | Idebenone | Leber hereditary optic neuropathy (LHON) | Chiesi | NDA accepted |
| 3.24.2026 | Linerixibat | Primary biliary cholangitis | GSK | NDA accepted |
| 3.28.2026 | Kresladi (marnetegragene autotemcel) | Leukocyte adhesion deficiency-I (LAD-I) | Rocket Pharmaceuticals | BLA accepted |
| 3.29.2026 | LNTH-2501 (Gallium-68 edotreotide) | Somatostatin receptor-positive (SSTR+) neuroendocrine tumors (NETs) | Lantheus Holdings | PDUFA date set |
| 4.5.2026 | Tividenofusp alfa | Hunter syndrome (MPS II) | Denali Therapeutics | BLA accepted |
| 4.6.2026 | Orca-T | Acute myeloid leukemia, acute lymphoblastic leukemia, and myelodysplastic syndromes | Orca Bio | BLA accepted |
| 4.13.2026 | FILSPARI (sparsentan) | Focal segmental glomerulosclerosis (FSGS) | Travere Therapeutics | sNDA accepted |
| 4.23.2026 | Sarclisa | Multiple Myeloma | Sanofi | sBLA accepted |
| 4.8.2026 | Opdivo (nivolumab) | Stage III or IV classical Hodgkin Lymphoma (cHL) | Bristol Myers Squib | sBLA accepted |
| 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 |
| 6.30.2026 | Veligrotug | Thyroid eye disase | Viridian Therapeutics | BLA accepted |
| 7.7.2026 | Atacicept | IgA Nephropathy | Vera Therapeutics | BLA 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 |
| 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 |
| Q4 2026 | Ivonescimab | EGFR-mutated NSCLC | Summit Therapeutics | BLA submitted |