2024 Orphan Drugs: PDUFA Dates and FDA Approvals (2024)

PDUFA DateOrphan DrugIndicationCompanyStatusFirst Quarter of 2024Brukinsa (zanubrutinib)Relapsed or refractory follicular lymphomaBeiGeneAPPROVED1.12.2024OlbetuximabGastroesophageal junction (GEJ) adenocarcinomaAstellasComplete response letter1.16.2024Hyqvia [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase]Chronic inflammatory demyelinating polyneuropathy (CIDP)TakedaAPPROVED1.31.2024Dupixent (dupilumab)Eosinophilic esophagitis (EoE)SanofiAPPROVED2.13.2024Onivyde (irinotecan liposome injection)Metastatic pancreatic ductal adenocarcinomaIpsenAPPROVED3.13.2024Livmarli(maralixibat)Cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC)Mirum PharmaceuticalsAPPROVED3.13.2024Tevimbra (tislelizumab-jsgr)Unresectable or metastatic esophageal squamous cell carcinomaBeiGeneAPPROVED3.14.2024ResmetiromNonalcoholic steatohepatitis (NASH)Madrigal PharmaceuticalsAPPROVED3.14.2024Breyanzi (lisocabtagene maraleucel)Relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)Bristol Myers SquibbAPPROVED3.15.2024AbecmaTriple-class exposed relapsed or refractory multiple myelomaBristol Myers Squibb and and 2seventy bioAwaiting new action date3.18.2024Lenmeldy (atidarsagene autotemcel)Metachromatic leukodystrophy (MLD)Orchard TherapeuticsAPPROVED3.21.2024Givinostatduch*enne muscular dystrophy (DMD)ITF TherapeuticsAPPROVED3.22.2024OPSYNVI (macitentan and tadalafil)Pulmonary Arterial Hypertension (PAH)Johnson & JohnsonAPPROVED3.25.2024Ultomiris (ravulizumab-cwvz)Neuromyelitis optica spectrum disorder (NMOSD)AstraZenecaAPPROVED3.26.2024SotaterceptPulmonary arterial hypertension (PAH)MerckAPPROVED3.27.2024VadadustatAnemia caused by chronic kidney diseaseAkebia TherapeuticsAPPROVED3.30.2024Exagamglogene autotemcel (exa-cel)Transfusion-dependent beta thalassemiaVertex Pharmaceuticals and CRISPR TherapeuticsAPPROVED3.31.2024Odronextamab (REGN1979)Relapsed/refractory follicular lymphoma or relapsed/refractory diffuse large B-cell lymphoma (DLBCL)Regeneron PharmaceuticalsComplete response letter4.1.2024Voydeya (danicopan)Extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH)AstraZenecaAPPROVED4.5.2024Carvykti(ciltacabtagene autoleucel; cilta-cel)Relapsed or refractory multiple myelomaJanssenAPPROVED4.5.2024Opdivo (nivolumab)Unresectable or metastatic urothelial carcinomaBristol Myers SquibbAPPROVED4.5.2024Abecma (idecabtagene vicleucel; ide-cel)

Triple-Class Exposed Relapsed or Refractory Multiple Myeloma After Two Prior Lines of Therapy

Bristol Myers Squibb & 2seventy BioAPPROVED4.18.2024Alecensa (alectinib)

Anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC)

Genentech, Inc.APPROVED4.26.2024Beqvez (Fidanacogene elaparvovec)

Hemophilia B

PfizerAPPROVED4.30.2024TovorafenibRelapsed or progressive pediatric low-grade glioma (pLGG)Day One BiopharmaceuticalsAPPROVED4.30.2024MavorixaforWHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndromeX4 PharmaceuticalsAPPROVED5.23.2024Breyanzi (lisocabtagene maraleucel)

Relapsed or refractory follicular lymphoma (FL)

Bristol Myers SquibbAPPROVED5.25.2024Pz-celRecessive dystrophic epidermolysis bullosaAbeona TherapeuticsComplete Response Letter5.28.2024Bkemv (eculizumab-aeeb)Paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis and atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathyAmgenAPPROVED5.31.2024Breyanzi (lisocabtagene maraleucel)Relapsed or refractory mantle cell lymphoma (MCL)Bristol Myers SquibbAPPROVED6.10.2024ElafibranorPrimary biliary cholangitis (PBC)Ipsen and GENFITAPPROVED6.12.2024TarlatamabAdvanced small cell lung cancerAmgenAPPROVED6.14.2024BlinatumomabCD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemiaAmgenAPPROVED6.16.2024ImetelstatLower risk myelodysplastic syndromes (MDS)Geron CorporationAPPROVED6.20.2024Piasky (crovalimab-akkz)Paroxysmal nocturnal hemoglobinuriaGenentechAPPROVED6.21.2024ELEVIDYS (delandistrogene moxeparvovec-rokl)duch*enne muscular dystrophy (DMD)Sarepta TherapeuticsAPPROVED6.21.2024VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc)Chronic inflammatory demyelinating polyneuropathy (CIDP)ArgenxAPPROVED6.26.2024Epkinly (epcoritamab-bysp)Relapsed or refractory follicular lymphomaGenmab USAPPROVED6.30.2024RP-L201 (marnetegragene autotemcel)Leukocyte adhesion deficiency-I (LAD-I)Rocket PharmaceuticalsComplete response letter7.24.2024Brineura (cerliponase alfa)Neuronal ceroid lipofuscinosis type 2 (CLN2 disease)BioMarin PharmaceuticalAPPROVED7.31.2024Dasynoc (dasatinib amorphous)Chronic myeloid leukemia (CML)Xspray PharmaComplete Response Letter8.1.2024Tecelra (afamitresgene autoleucel)Unresectable or metastatic synovial sarcomaAdaptimmune TherapeuticsAPPROVED8.4.2024Afami-celAdvanced synovial sarcomaAdaptimmune TherapeuticsAPPROVED8.6.2024VorasidenibGrade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutationServier PharmaceuticalsAPPROVED8.7.2024Fabhalta (iptacopan)IgA nephropathy (IgAN)NovartisAPPROVED8.13.2024LYMPHIR (denileukin diftitox)Relapsed or refractory cutaneous T-cell lymphoma (CTCL)Citius PharmaceuticalsAPPROVED8.14.2024SeladelparPrimary biliary cholangitis (PBC)CymaBay TherapeuticsAPPROVED8.14.2024PalopegteriparatideHypoparathyroidismAscendis PharmaAPPROVED8.14.2024Niktimvo (axatilimab)Chronic graft versus host diseaseIncyte and Syndax PharmaceuticalsAPPROVED8.20.2024Lazertinib in combination with Rybrevant (amivantamab-vmjw)EGFR+ advanced or metastatic non–small cell lung cancerJanssen BiotechAPPROVED8.20.2024Equecabtagene autoleucel

Lupus Nephritis and Non-Renal Systemic Lupus Erythematosus

IASO BioAPPROVED8.22.2024LinvoseltamabRelapsed/refractory multiple myelomaRegeneron PharmaceuticalsComplete Response Letter9.5.24Filspari (sparsentan)IgA NephropathyTravere TherapeuticsAPPROVED9.21.2024ArimoclomolNiemann-Pick disease type CZevra TherapeuticsNDA accepted9.24.2024IB1001 (N-acetyl-L-leucine)Niemann-Pick disease type CIntraBioNDA accepted9.25.2024Keytruda (pembrolizumab)Unresectable advanced or metastatic malignant pleural mesotheliomaMercksBLA accepted9.27.2024Sarclisa (isatuximab-irfc) in combination with bortezomib, lenalidomide, and dexamethasoneTransplant-ineligible multiple myelomaSanofisBLA accepted10.15.2024Ocaliva (obeticholic acid)Primary biliary cholangitis (PBC)Intercept PharmaceuticalssNDA accepted10.21.2024Octreotide (CAM2029)AcromegalyCamurusNDA accepted11.13.2024Upstaza (eladocagene exuparvovec)AADC deficiencyPTC TherapeuticsBLA accepted11.16.2024Obecabtagene autoleucel (obe-cel)Relapsed/refractory adult B-cell acute lymphoblastic leukemia (ALL)Autolus TherapeuticsBLA accepted11.28.2024GovorestatGalactosemia type 1Applied TherapeuticsNDA accepted11.29.2024Acoramidis

Transthyretin amyloid cardiomyopathy (ATTR-CM)

BridgeBio PharmaNDA accepted11.30.2024SH-201Certain forms of leukemia and other cancersShorla OncologyNDA accepted12.19.2024OlezarsenFamilial chylomicronemia syndrome (FCS)Ionis PharmaceuticalsNDA accepted12.26.2024RevumenibRelapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute leukemiaSyndax PharmaceuticalsNDA accepted12.26.2024Imcivree (setmelanotide)Bardet-Biedl syndrome or POMC/LEPR deficiencyRhythm PharmaceuticalssNDA accepted12.27.2024

DCCR (Diazoxide Choline)

Prader-Willi SyndromeSoleno TherapeuticsNDA accepted12.28.2024EnsartinibMetastatic ALK-positive non-small cell lung cancer (NSCLC)Xcovery HoldingsNDA accepted12.28.2024CosibelimabAdvanced cutaneous squamous cell carcinomaCheckpoint TherapeuticsBLA accepted12.30.2024CrinecerfontCongenital adrenal hyperplasia (CAH)Neurocrine BiosciencesNDA accepted1.15.2025Tabelecleucel (Tab-cel)Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative DiseaseAtara BiotherapeuticsBLA accepted2.17.2025VimseltinibTenosynovial giant cell tumor (TGCT)Deciphera PharmaceuticalsNDA accepted6.17.2025SebetralstatHereditary AngioedemaKalVista PharmaceuticalsNDA accepted