Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all orphan drugs for 2024.
Prescription Drug User Fee Act (PDUFA) dates refer to deadlines for the FDA to review new drugs. The PDUFA date is 10 months after the drug application has been accepted by the FDA or 6 months, if the drug is given a priority review designation. As each PDFUA date approaches, CheckRare will be covering the FDA meetings and outcomes.
| PDUFA Date | Orphan Drug | Indication | Company | Status |
| First Quarter of 2024 | Brukinsa (zanubrutinib) | Relapsed or refractory follicular lymphoma | BeiGene | APPROVED |
| 1.12.2024 | Olbetuximab | Gastroesophageal junction (GEJ) adenocarcinoma | Astellas | Complete response letter |
| 1.16.2024 | Hyqvia [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] | Chronic inflammatory demyelinating polyneuropathy (CIDP) | Takeda | APPROVED |
| 1.31.2024 | Dupixent (dupilumab) | Eosinophilic esophagitis (EoE) | Sanofi | APPROVED |
| 2.13.2024 | Onivyde (irinotecan liposome injection) | Metastatic pancreatic ductal adenocarcinoma | Ipsen | APPROVED |
| 3.13.2024 | Livmarli (maralixibat) | Cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) | Mirum Pharmaceuticals | APPROVED |
| 3.13.2024 | Tevimbra (tislelizumab-jsgr) | Unresectable or metastatic esophageal squamous cell carcinoma | BeiGene | APPROVED |
| 3.14.2024 | Resmetirom | Nonalcoholic steatohepatitis (NASH) | Madrigal Pharmaceuticals | APPROVED |
| 3.14.2024 | Breyanzi (lisocabtagene maraleucel) | Relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) | Bristol Myers Squibb | APPROVED |
| 3.15.2024 | Abecma | Triple-class exposed relapsed or refractory multiple myeloma | Bristol Myers Squibb and and 2seventy bio | Awaiting new action date |
| 3.18.2024 | Lenmeldy (atidarsagene autotemcel) | Metachromatic leukodystrophy (MLD) | Orchard Therapeutics | APPROVED |
| 3.21.2024 | Givinostat | Duchenne muscular dystrophy (DMD) | ITF Therapeutics | APPROVED |
| 3.22.2024 | OPSYNVI (macitentan and tadalafil) | Pulmonary Arterial Hypertension (PAH) | Johnson & Johnson | APPROVED |
| 3.25.2024 | Ultomiris (ravulizumab-cwvz) | Neuromyelitis optica spectrum disorder (NMOSD) | AstraZeneca | APPROVED |
| 3.26.2024 | Sotatercept | Pulmonary arterial hypertension (PAH) | Merck | APPROVED |
| 3.27.2024 | Vadadustat | Anemia caused by chronic kidney disease | Akebia Therapeutics | APPROVED |
| 3.30.2024 | Exagamglogene autotemcel (exa-cel) | Transfusion-dependent beta thalassemia | Vertex Pharmaceuticals and CRISPR Therapeutics | APPROVED |
| 3.31.2024 | Odronextamab (REGN1979) | Relapsed/refractory follicular lymphoma or relapsed/refractory diffuse large B-cell lymphoma (DLBCL) | Regeneron Pharmaceuticals | Complete response letter |
| 4.1.2024 | Voydeya (danicopan) | Extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH) | AstraZeneca | APPROVED |
| 4.5.2024 | Carvykti (ciltacabtagene autoleucel; cilta-cel) | Relapsed or refractory multiple myeloma | Janssen | APPROVED |
| 4.5.2024 | Opdivo (nivolumab) | Unresectable or metastatic urothelial carcinoma | Bristol Myers Squibb | APPROVED |
| 4.5.2024 | Abecma (idecabtagene vicleucel; ide-cel) |
Triple-Class Exposed Relapsed or Refractory Multiple Myeloma After Two Prior Lines of Therapy |
Bristol Myers Squibb & 2seventy Bio | APPROVED |
| 4.18.2024 | Alecensa (alectinib) |
Anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) |
Genentech, Inc. | APPROVED |
| 4.26.2024 | Beqvez (Fidanacogene elaparvovec) |
Hemophilia B |
Pfizer | APPROVED |
| 4.30.2024 | Tovorafenib | Relapsed or progressive pediatric low-grade glioma (pLGG) | Day One Biopharmaceuticals | APPROVED |
| 4.30.2024 | Mavorixafor | WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome | X4 Pharmaceuticals | APPROVED |
| 5.23.2024 | Breyanzi (lisocabtagene maraleucel) |
Relapsed or refractory follicular lymphoma (FL) |
Bristol Myers Squibb | APPROVED |
| 5.25.2024 | Pz-cel | Recessive dystrophic epidermolysis bullosa | Abeona Therapeutics | Complete Response Letter |
| 5.28.2024 | Bkemv (eculizumab-aeeb) | Paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis and atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy | Amgen | APPROVED |
| 5.31.2024 | Breyanzi (lisocabtagene maraleucel) | Relapsed or refractory mantle cell lymphoma (MCL) | Bristol Myers Squibb | APPROVED |
| 6.10.2024 | Elafibranor | Primary biliary cholangitis (PBC) | Ipsen and GENFIT | APPROVED |
| 6.12.2024 | Tarlatamab | Advanced small cell lung cancer | Amgen | APPROVED |
| 6.14.2024 | Blinatumomab | CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia | Amgen | APPROVED |
| 6.16.2024 | Imetelstat | Lower risk myelodysplastic syndromes (MDS) | Geron Corporation | APPROVED |
| 6.20.2024 | Piasky (crovalimab-akkz) | Paroxysmal nocturnal hemoglobinuria | Genentech | APPROVED |
| 6.21.2024 | ELEVIDYS (delandistrogene moxeparvovec-rokl) | Duchenne muscular dystrophy (DMD) | Sarepta Therapeutics | APPROVED |
| 6.21.2024 | VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) | Chronic inflammatory demyelinating polyneuropathy (CIDP) | Argenx | APPROVED |
| 6.26.2024 | Epkinly (epcoritamab-bysp) | Relapsed or refractory follicular lymphoma | Genmab US | APPROVED |
| 6.30.2024 | RP-L201 (marnetegragene autotemcel) | Leukocyte adhesion deficiency-I (LAD-I) | Rocket Pharmaceuticals | Complete response letter |
| 7.24.2024 | Brineura (cerliponase alfa) | Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) | BioMarin Pharmaceutical | APPROVED |
| 7.31.2024 | Dasynoc (dasatinib amorphous) | Chronic myeloid leukemia (CML) | Xspray Pharma | Complete Response Letter |
| 8.1.2024 | Tecelra (afamitresgene autoleucel) | Unresectable or metastatic synovial sarcoma | Adaptimmune Therapeutics | APPROVED |
| 8.4.2024 | Afami-cel | Advanced synovial sarcoma | Adaptimmune Therapeutics | APPROVED |
| 8.6.2024 | Vorasidenib | Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation | Servier Pharmaceuticals | APPROVED |
| 8.7.2024 | Fabhalta (iptacopan) | IgA nephropathy (IgAN) | Novartis | APPROVED |
| 8.13.2024 | LYMPHIR (denileukin diftitox) | Relapsed or refractory cutaneous T-cell lymphoma (CTCL) | Citius Pharmaceuticals | APPROVED |
| 8.14.2024 | Seladelpar | Primary biliary cholangitis (PBC) | CymaBay Therapeutics | APPROVED |
| 8.14.2024 | Palopegteriparatide | Hypoparathyroidism | Ascendis Pharma | APPROVED |
| 8.14.2024 | Niktimvo (axatilimab) | Chronic graft versus host disease | Incyte and Syndax Pharmaceuticals | APPROVED |
| 8.20.2024 | Lazertinib in combination with Rybrevant (amivantamab-vmjw) | EGFR+ advanced or metastatic non–small cell lung cancer | Janssen Biotech | APPROVED |
| 8.20.2024 | Equecabtagene autoleucel |
Lupus Nephritis and Non-Renal Systemic Lupus Erythematosus |
IASO Bio | APPROVED |
| 8.22.2024 | Linvoseltamab | Relapsed/refractory multiple myeloma | Regeneron Pharmaceuticals | Complete Response Letter |
| 9.5.24 | Filspari (sparsentan) | IgA Nephropathy | Travere Therapeutics | APPROVED |
| 9.21.2024 | Arimoclomol | Niemann-Pick disease type C | Zevra Therapeutics | NDA accepted |
| 9.24.2024 | IB1001 (N-acetyl-L-leucine) | Niemann-Pick disease type C | IntraBio | NDA accepted |
| 9.25.2024 | Keytruda (pembrolizumab) | Unresectable advanced or metastatic malignant pleural mesothelioma | Merck | sBLA accepted |
| 9.27.2024 | Sarclisa (isatuximab-irfc) in combination with bortezomib, lenalidomide, and dexamethasone | Transplant-ineligible multiple myeloma | Sanofi | sBLA accepted |
| 10.15.2024 | Ocaliva (obeticholic acid) | Primary biliary cholangitis (PBC) | Intercept Pharmaceuticals | sNDA accepted |
| 10.21.2024 | Octreotide (CAM2029) | Acromegaly | Camurus | NDA accepted |
| 11.13.2024 | Upstaza (eladocagene exuparvovec) | AADC deficiency | PTC Therapeutics | BLA accepted |
| 11.16.2024 | Obecabtagene autoleucel (obe-cel) | Relapsed/refractory adult B-cell acute lymphoblastic leukemia (ALL) | Autolus Therapeutics | BLA accepted |
| 11.28.2024 | Govorestat | Galactosemia type 1 | Applied Therapeutics | NDA accepted |
| 11.29.2024 | Acoramidis |
Transthyretin amyloid cardiomyopathy (ATTR-CM) |
BridgeBio Pharma | NDA accepted |
| 11.30.2024 | SH-201 | Certain forms of leukemia and other cancers | Shorla Oncology | NDA accepted |
| 12.19.2024 | Olezarsen | Familial chylomicronemia syndrome (FCS) | Ionis Pharmaceuticals | NDA accepted |
| 12.26.2024 | Revumenib | Relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute leukemia | Syndax Pharmaceuticals | NDA accepted |
| 12.26.2024 | Imcivree (setmelanotide) | Bardet-Biedl syndrome or POMC/LEPR deficiency | Rhythm Pharmaceuticals | sNDA accepted |
| 12.27.2024 |
DCCR (Diazoxide Choline) |
Prader-Willi Syndrome | Soleno Therapeutics | NDA accepted |
| 12.28.2024 | Ensartinib | Metastatic ALK-positive non-small cell lung cancer (NSCLC) | Xcovery Holdings | NDA accepted |
| 12.28.2024 | Cosibelimab | Advanced cutaneous squamous cell carcinoma | Checkpoint Therapeutics | BLA accepted |
| 12.30.2024 | Crinecerfont | Congenital adrenal hyperplasia (CAH) | Neurocrine Biosciences | NDA accepted |
| 1.15.2025 | Tabelecleucel (Tab-cel) | Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease | Atara Biotherapeutics | BLA accepted |
| 2.17.2025 | Vimseltinib | Tenosynovial giant cell tumor (TGCT) | Deciphera Pharmaceuticals | NDA accepted |
| 6.17.2025 | Sebetralstat | Hereditary Angioedema | KalVista Pharmaceuticals | NDA accepted |
2023 Orphan Drugs: PDUFA Dates and FDA Approvals
2022 Orphan Drugs: PDUFA Dates and FDA Approvals
2021 Orphan Drugs: PDUFA Dates and FDA Approvals
