2025 Orphan Drugs: PDUFA Dates and FDA Approvals

By: | Updated on: May 15, 2025

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 DateOrphan DrugIndicationCompanyStatus
1.7.2025. Ryoncil (remestemcel)Steroid-refractory acute graft versus host diseaseMesoblast LimitedAPPROVED
1.15.2025Tabelecleucel (Tab-cel)Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative DiseaseAtara BiotherapeuticsComplete Response Letter
2.17.2025VimseltinibTenosynovial giant cell tumor (TGCT)Deciphera PharmaceuticalsAPPROVED
2.21.2025Ctexli (chenodiol)Cerebrotendinous xanthomatosisMirum TherapeuticsAPPROVED
2.28.2025MirdametinibNeurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN)Springworks TherapeuticsAPPROVED
3.6.2025Encelto (revakinagene taroretcel-lwey)Macular telangiectasia type 2 (MacTel)Neurotech PharmaceuticalsAPPROVED
3.20.2025Fabhalta (iptacopan)C3 glomerulopathy (C3G)NovartisAPPROVED
3.20.2025Rivoceranib/Camrelizumab Unresectable or metastatic hepatocellular carcinomaElevar TherapeuticsComplete Response Letter
3.23.2025VutrisiranATTR-CMAlnylam PharmaceuticalsAPPROVED
3.27.2025Diazoxide choline (DCCR)Prader-Willi syndromeSoleno TherapeuticsAPPROVED
3.28.2025FitusiranHemophilia A or BSanofiAPPROVED
4.2.2025Vanrafia (atrasentan)IgA nephropathyNovartisAPPROVED
4.3.2025Uplizna (inebilizumab)Immunoglobulin G4-related disease (IgG4-RD)AmgenAPPROVED
4.21.2025Nivolumab plus ipilimumabHepatocellular carcinoma (HCC)Bristol Myers SquibbAPPROVED
4.29.2025Prademagene zamikeracel (pz-cel)Epidermolysis bullosaAbeona TherapeuticsAPPROVED
4.29.2025ElamipretideBarth syndromeStealth BioTherapeuticsAPPROVED
4.30.2025NipocalimabGeneralized myasthenia gravisJohnson & JohnsonAPPROVED
5.24.2025Yutrepia (treprostinil)Pulmonary arterial hypertensionLiquidia CorporationAPPROVED
5.26.2025Welireg (belzutifan)Advanced pheochromocytoma and paraganglioma (PPGL)MerckAPPROVED
5.28.2025Khindivi (hydrocortisone) Pediatric adrenal insufficiencyEton PharmaceuticalsAPPROVED
6.17.2025SebetralstatHereditary angioedemaKalVista PharmaceuticalsPDUFA Delayed
6.18.2025Andembry (garadacimab)Hereditary angioedemaCSLAPPROVED
6.19.2025Nitisinone AlkaptonuriaCycle PharmaceuticalsAPPROVED
6.20.2025DupixentBullous PemphigoidSanofiAPPROVED
6.23.2025Taletrectinib Advanced ROS1-positive non-small cell lung cancerNuvation BioAPPROVED
6.28.2025Gamifant (emapalumab)Still's disease SobiAPPROVED
6.30.2025Gammagard Liquid ERC (immune globulin infusion)Primary immunodeficienciesTakedaAPPROVED
7.10.2025LinvoseltamabMultiple myelomaRegeneron PharmaceuticalsAPPROVED
7.24.2025AvatrombopagImmune thrombocytopeniaSobi North AmericaAPPROVED
7.27.2025TransCon hGHGrowth hormone deficiencyAscendis PharmaAPPROVED
7.28.2025Empaveli (pegcetacoplan)C3G and primary IC-MPGNApellis PharmaceuticalsAPPROVED
7.29.2025SepiapterinPhenylketonuria (PKU)PTC TherapeuticsAPPROVED
7.30.2025OdronextamabFollicular lymphoma Regeneron PharmaceuticalsComplete Response Letter
7.31.2025Alhemo (concizumab)Hemophilia A or B without inhibitorsNovo Nordisk APPROVED
8.18.2025UX111Sanfilippo syndrome (MPS IIIA)UltragenyxComplete Response Letter
8.18.2025DordaviproneH3 K27M-mutant diffuse gliomaJazz PharmaceuticalsAPPROVED
8.19.2025VatiquinoneFriedreich's ataxiaPTC TherapeuticsComplete Response Letter
8.21.2025DonidalorsenHereditary AngioedemaIonis PharmaceuticalsAPPROVED
8.27.2025PRGN-2012 (zopapogene imadenovec)Recurrent respiratory papillomatosis (RRP)PrecigenAPPROVED
8.28.2025FilspariIgA nephropathyTravere TherapeuticsAPPROVED
8.29.2025RilzabrutinibImmune thrombocytopeniaSanofiAPPROVED
8.31.2025DeramiocelDuchenne muscular dystrophyCapricor TherapeuticsComplete Response Letter
9.22.2025ApitegromabSpinal muscular atrophyScholar RockComplete Response Letter
9.25.2025PaltusotineAcromegalyCrinetics PharmaceuticalsAPPROVED
9.30.2025CUTX-101 (Copper Histidinate)Menkes diseaseFortress BiotechComplete Response Letter
10.9.2025Jascayd (nerandomilast)Idiopathic pulmonary fibrosisBoehringer IngelheimAPPROVED
10.19.2025Gazyva/Gazyvaro (obinutuzumab)Lupus nephritisRocheAPPROVED
10.20.2025Epioxa (Epi-on)KeratoconusGlaukos CorporationAPPROVED
10.23.2025Belantamab mafodotinMultiple myelomaGSKBLA accepted
10.25.2025Revuforj (revumenib)mNPM1 acute myeloid leukemiaSyndax PharmaceuticalssNDA accepted
11.9.2025Clemidsogene lanparvovec (RGX-121)Mucopolysaccharidosis II (MPS II); Hunter syndromeREGENXBIOBLA accepted
11.18.2025PlozasiranFamilial chylomicronemia syndromeArrowhead PharmaceuticalsNDA accepted
11.28.2025SibeprenlimabImmunoglobulin A nephropathy (IgAN)Otsuka Pharmaceutical Co.BLA accepted
11.30.2025ZiftomenibRelapsed or refractory acute myeloid leukemia (AML) with an NPM1 mutationKura Oncology and Kyowa KirinNDA accepted
11.30.2025TransCon CNP (navepegritide)AchondroplasiaAscendis PharmaNDA accepted
12.7.2025Pyrukynd (mitapivat)Alpha- and beta-thalassemiaAgios PharmaceuticalssNDA accepted
12.12.2025Orladeyo (berotralstat)Hereditary angioedema (HAE)BioCryst PharmaceuticalsNDA accepted
12.14.2025UpliznaMyasthenia gravisAmgenRegulatory submission accepted
12.26.2025AficamtenObstructive hypertrophic cardiomyopathy (oHCM)CytokineticsNDA accepted
12.30.2025RelacorilantCushing's syndromeCorcept TherapeuticsNDA accepted
Q4 2025TroriluzoleSpinocerebellar ataxia (SCA) BiohavenNDA accepted
1.10.2026TabelecleucelEpstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD)Atara BiotherapeuticsBLA accepted
1.13.2026FILSPARI (sparsentan)Focal segmental glomerulosclerosis (FSGS)Travere TherapeuticssNDA accepted
1.13.2026Cerezyme (imiglucerase)Gaucher disease type 3SanofisBLA accepted
1.31.2026LeniolisibActivated phosphoinositide 3-kinase delta syndrome (APDS)Pharming GroupsNDA accepted
2.8.2026Clemidsogene lanparvovec (RGX-121)Mucopolysaccharidosis II (MPS II)REGENXBIOBLA accepted
2.25.2026Inqovi (decitabine and cedazuridine) plus Venclexta (venetoclax)Acute myeloid leukemiaTaiho OncologysNDA accepted
3.24.2026LinerixibatPrimary biliary cholangitis GSK NDA accepted
3.28.2026Kresladi (marnetegragene autotemcel)Leukocyte adhesion deficiency-I (LAD-I)Rocket PharmaceuticalsBLA accepted
4.5.2026Tividenofusp alfaHunter syndrome (MPS II)Denali TherapeuticsBLA accepted
4.6.2026Orca-TAcute myeloid leukemia, acute lymphoblastic leukemia, and myelodysplastic syndromesOrca BioBLA accepted
6.18.2026XS003 (nilotinib)Chronic myeloid leukemia (CML)Xspray PharmaNDA accepted
10.10.2026TabelecleucelEpstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD)Pierre Fabre PharmaceuticalsBLA accepted

 

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