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2026 Orphan Drugs: PDUFA Dates and FDA Approvals

by and | Updated on: Oct 20, 2026

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 DateOrphan DrugIndicationCompanyStatus
1.5.2026Cablivi (caplacizumab)Acquired thrombotic thrombocytopenic purpura (aTTP)SanofiAPPROVED
1.10.2026TabelecleucelEpstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD)Atara BiotherapeuticsComplete Response Letter
1.13.2026Cerezyme (imiglucerase)Gaucher disease type 3SanofiAPPROVED
1.14.2026Copper histidinate (CUTX-101)Menkes diseaseSentynl TherapeuticsAPPROVED
1.27.2026Darzalex Faspro with D-VRdMultiple myelomaJohnson & JohnsonAPPROVED
1.31.2026LeniolisibActivated phosphoinositide 3-kinase delta syndrome (APDS)Pharming GroupComplete Response Letter
2.8.2026Clemidsogene lanparvovec (RGX-121)Mucopolysaccharidosis II (MPS II)REGENXBIOComplete Response Letter
2.13.2026BitopertinErythropoietic protoporphyria (EPP)Disc MedicineComplete Response Letter
2.20.2026Venetoclax plus acalabrutinibChronic lymphocytic leukemiaAbbVieAPPROVED
2.23.2026PegzilarginaseArginase 1 deficiency (ARG1-D)Immedica Pharma ABAPPROVED
2.25.2026Inqovi (decitabine and cedazuridine) plus Venclexta (venetoclax)Acute myeloid leukemiaTaiho OncologyAPPROVED
2.25.2026ET-600Arginine vasopressin deficiency (AVP-D)Eton PharmaceuticalsAPPROVED
2.28.2026Palynziq (pegvaliase)Phenylketonuria (PKU)BioMarin APPROVED
2.28.2026TransCon CNP (navepegritide)AchondroplasiaAscendis PharmaAPPROVED
2.28.2026IdebenoneLeber hereditary optic neuropathy (LHON)ChiesiComplete Response Letter
3.5.2026Tec-Dara [Tecvayli(teclistamab) plus Darzalex Faspro (daratumumab and hyaluronidase)]Multiple myelomaJohnson & JohnsonAPPROVED
3.3.2026Juxtapid (lomitapide)Homozygous familial hypercholesterolemia (HoFH)ChiesiAPPROVED
3.10.2026Wellcovorin (leucovorin calcium)Cerebral folate deficiencyGSKAPPROVED
3.24.2026LinerixibatPrimary biliary cholangitis AlfasigmaAPPROVED
3.28.2026Kresladi (marnetegragene autotemcel)Leukocyte adhesion deficiency-I (LAD-I)Rocket PharmaceuticalsAPPROVED
4.2.2026Tecartus (brexucabtagene autoleucel)Mantle cell lymphomaGilead/Kite OncologyAPPROVED
4.3.2026Nusinersen high-dose regimenSpinal muscular atrophyBiogenAPPROVED
4.5.2026Tividenofusp alfaHunter syndrome (MPS II)Denali TherapeuticsAPPROVED
4.8.2026Opdivo (nivolumab)Stage III or IV classical Hodgkin Lymphoma (cHL)Bristol Myers SquibAPPROVED
4.13.2026FILSPARI (sparsentan)Focal segmental glomerulosclerosis (FSGS)Travere TherapeuticsAPPROVED
4.23.2026Otarmeni (lunsotogene parvec-cwha)OTOF-related hearing lossRegeneron PharmaceuticalsAPPROVED
5.1.2026Jakafi XR (ruxolitinib)Myelofibrosis, polycythemia vera, and graft-versus-host diseaseIncyteAPPROVED
5.10.2026Vyvgart (efgartigimod alfa-fcab)AChR-Ab seronegative generalized myasthenia gravis (gMG)ArgenxAPPROVED
5.27.2026Pivekimab sunirineBlastic plasmacytoid dendritic cell neoplasm (BPDCN)AbbVieAPPROVED
6.2.2026Cavhanza (nilotinib)Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML)
Cycle PharmaAPPROVED
6.10.2026Oclaiz (CAM2029)AcromegalyCamurusComplete Response Letter
6.18.2026XS003 (nilotinib)Chronic myeloid leukemia (CML)Xspray PharmaComplete Response Letter
6.29.2026LNTH-2501 (Gallium-68 edotreotide)Somatostatin receptor-positive (SSTR+) neuroendocrine tumors (NETs)Lantheus HoldingsComplete Response Letter
6.30.2026VeligrotugThyroid eye disaseViridian TherapeuticsAPPROVED
Q3 2026RusfertidePolycythemia vera (PV)Takeda and Protagonist TherapeuticsAPPROVED
7.6.2026Tregzi (Orca-T)Acute myeloid leukemia, acute lymphoblastic leukemia, and myelodysplastic syndromesOrca BioAPPROVED
7.7.2026AtaciceptIgA NephropathyVera TherapeuticsAPPROVED
7.23.2026SarclisaMultiple MyelomaSanofiAPPROVED
8.17.2026IberdomideMultiple MyelomaBristol Myers SquibAPPROVED
8.19.2026Pasatru (garetosmab)Fibrodysplasia ossificans progressiva (FOP)RegeneronAPPROVED
8.23.2026DTX401 (pariglasgene brecaparvovec)Glycogen storage disease type 1a (GSD1a)UltragenyxAPPROVED
8.26.2026Rasonque (daraxonrasib)Metastatic pancreatic adenocarcinomaRevolution MedicinesAPPROVED
8.25.2026DasynocChronic myeloid leukemia (CML) and acute lymphoblastic leukemia (ALL)Xspray PharmaComplete Response Letter
8.28.2026177Lu-edotreotideGastroenteropancreatic neuroendocrine tumorsITMComplete Response Letter
8.30.2026Besremi (ropeginterferon alfa-2b-njft)Essential thrombocythemiaPharmaEssentia USA CorporationAPPROVED
9.2026Orzeyful (oveporexton)Narcolepsy type 1TakedaAPPROVED
9.14.2026Bexlutry (lutetium Lu 177 dotatate)Gastroenteropancreatic neuroendocrine tumorsCuriumAPPROVED
9.18.2026ZidesamtinibROS1-positive non-small cell lung cancerNuvalentAPPROVED
9.19.2026UX111 (rebisufligene etisparvovec)Sanfilippo syndrome Type A (MPS IIIA)UltragenyxAPPROVED
9.19.2026Aqneursa (levacetylleucine)Ataxia-telangiectasiaIntraBioAPPROVED
9.22.2026ZilganersenAlexander diseaseIonis PharmaceuticalsAPPROVED
9.26.2026ZilurgisertibFibrodysplasia ossificans progressivaMirumAPPROVED
9.27.2026LirafugratinibCholangiocarcinomaElevar TherapeuticsAPPROVED
New PDUFA Date: 12/27/2026RelutrigineSCN2A and SCN8A developmental and epileptic encephalopathies (DEEs)PraxisNDA accepted; PDUFA moved to 12.27.2026
9.28.2026Emcitate (tiratricol)MCT8 deficiencyEgetis TherapeuticsAPPROVED
9.25.2026Gazyva (obinutuzumab)Idiopathic nephrotic syndrome (INS)GenentechAPPROVED
9.30.2026ApitegromabSpinal muscular atrophy (SMA)ScholarRockAPPROVED
9.30.2026Camzyos (mavacamten)Obstructive hypertrophic cardiomyopathy (oHCM)Bristol Myers SquibbAPPROVED
September 2026BrepocitinibDermatomyositisPriovant TherapeuticsAPPROVED
10.2.2026Jaypirca (pirtobrutinib)Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)Eli LillyAPPROVED
10.15.2026Enspryng (satralizumab)Thyroid eye diseaseRocheAPPROVED
10.20.2026Evoxac (cevimeline HCI)Sjögren’s diseaseCosette PharmaceuticalsAPPROVED
10.21.2026Orladeyo (berotralstat)Hereditary angioedema (HAE)BioCryst PharmaceuticalsAPPROVED
10.30.2026INO-3107Recurrent respiratory papillomatosis (RRP)INOVIOBLA accepted
11.14.2026IvonescimabEGFR-mutated NSCLCSummit TherapeuticsBLA submitted
11.1.2026MitapivatSickle cell diseaseAgios PharmaceuticalssNDA accepted
11.17.2026AdrabetadexNiemann-Pick type CBeren TherapeuticsNDA accepted
11.22.2026MolbreeviAutoimmune pulmonary alveolar proteinosis (autoimmune PAP)Savara BLA accepted
11.22.2026DeramiocelDuchenne muscular dystrophyCapricor TherapeuticsExtended
11.2026Zeleciment rostudirsenDuchenne muscular dystrophyDyne TherapeuticsBLA accepted
11.25.2026VenglustatType 3 Gaucher diseaseSanofiNDA accepted
11.27.2026BBP-418Limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)BridgeBioNDA accepted
11.27.2026SaroglitazarPrimary biliary cholangitisZydus TherapeuticsNDA accepted
11.27.2026NeladalkibALK-positive non-small cell lung cancerNuvalentNDA accepted
11.30.2026PovetaciceptIgA nephropathyVertex PharmaceuticalsBLA accepted
11.30.2026BezuclastinibGastrointestinal Stromal Tumors (GIST)Cogent BiosciencesNDA accepted
12.12.2026ImsidolimabGeneralized pustular psoriasis (GPP)Vanda PharmaceuticalsBLA accepted
12.18.2026TirabrutinibR/R primary central nervous system lymphoma (PCNSL)Deciphera PharmaceuticalsNDA accepted
12.23.2026Anito-celR/R multiple myelomaGileadBLA accepted
12.27.2026RelutrigineSCN2A and SCN8A DEEsPraxisNDA accepted
12.30.26BezuclastinibNonadvanced systemic mastocytosisCogent BiosciencesNDA accepted
12.30.2026SL1009, sodium dichloroacetate (DCA)Pyruvate dehydrogenase complex deficiency (PDCD)Saol TherapeuticsNDA accepted
Q4 2026EncaleretAutosomal dominant hypocalcemia type 1 (ADH1)BridgeBioNDA accepted
Q4 2026Ultomiris (ravulizumab)Immunoglobulin A nephropathy (IgAN)AstraZenecaACCEPTED
1.20.2027Enspryng (satralizumab)Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD)GenentechsBLAC accepted
1.21.2027Zeleciment rostudirsenDuchenne muscular dystrophyDyne TherapeuticsBLA accepted
1.28.2027Arikayce(amikacin liposome inhalation suspension)Mycobacterium avium complex (MAC) lung diseaseInsmed IncorporatedsNDA accepted
2.4.2027InfigratinibAchondroplasiaBridgeBioNDA accepted
2.12.2027TinlarebantStargardt disease type 1Belite BioNDA accepted
2.27.2027ZipalertinibEGFR non-small cell lung cancerTaiho OncologyNDA accepted
2.28.2027Amondys 45 (casimersen) and Vyondys 53 (golodirsen)Duchenne muscular dystrophySarepta TherapeuticssNDA accepted
2.28.2027Voxzogo (vosoritide)AchondroplasiaBioMarinsNDA accepted
February 2027DersimelagonErythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP)Leo PharmaNDA accepted
3.2027Mogenry (sonpiretigene isteparvovec, MCO-010)Retinitis pigmentosaNanoscope TherapeuticsBLA accepted
3.10.2027Lonvo-z (ziclumeran)Hereditary angioedemaIntellia TherapeuticsBLA accepted
3.31.2027TabelecleucelEpstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD)Pierre Fabre PharmaceuticalsBLA accepted
4.1.2027Pitolisant GRNarcolepsyHarmony BiosciencesNDA accepted
4.23.2027DeucrictibantHereditary angioedemaPharvaris NDA accepted
4.28.2027VaregacestatDesmoid tumorsImmunomeNDA accepted
4.2027Tyvaso (treprostinil)Idiopathic pulmonary fibrosisUnited TherapeuticssNDA accepted
5.1.2027AXS-12 (reboxetine)NarcolepsyAxsome TherapeuticsNDA accepted
5.8.2027EncaleretAutosomal dominant hypocalcemia type 1 (ADH1)BridgeBio PharmaNDA accepted
5.13.2027MezigdomideMultiple myelomaBristol Myers Squibb NDA accepted
5.27.2027ObexelimabImmunoglobulin G4-Related Disease (IgG4-RD)Zenas BioPharmaBLA accepted
6.24.2027RalinepagPulmonary arterial hypertension (PAH)United TherapeuticsNDA accepted
6.29.2027BezuclastinibAdvanced systemic mastocytosisCogent BiosciencesNDA accepted
2025 Orphan Drugs: PDUFA Dates and FDA Approvals 
2024 Orphan Drugs: PDUFA Dates and FDA Approvals
2023 Orphan Drugs: PDUFA Dates and FDA Approvals
2022 Orphan Drugs: PDUFA Dates and FDA Approvals
2021 Orphan Drugs: PDUFA Dates and FDA Approvals

 

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