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 2023.
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 |
| 1.20.2023 | Zanubritinib | CLL/SLL | Beigene | APPROVED |
| 1.27.2023 | Jaypirca | Mantle cell lymphoma | Lilly | APPROVED |
| 2.4.2023 |
TAKHZYRO (lanadelumab-flyo) |
Prevent attacks of hereditary angioedema (HAE) | Takeda | APPROVED |
| 2.11.2023 | Aflibercept | Retinopathy of Prematurity (ROP) in preterm infants | Regeneron | APPROVED |
| 2.16.2023 | Lamzede | Alpha-mannosidosis | Chiesi | APPROVED |
| 2.17.2023 | Filspari | Primary immunoglobulin A nephropathy |
Travere Therapeutics |
APPROVED |
| 02.28.2023 | Omaveloxolone | Friedreich’s ataxia | Reata | APPROVED |
| 2.28.2023 | Efanesoctocog alfa | Hemophilia A | Sanofi | APPROVED |
| 3.10.2023 | Trofinetide | Rett syndrome | Acadia Pharmaceuticals | APPROVED |
| 3.22.2023 | Retifanlimab-dlwr | Merkel cell carcinoma | Incyte | APPROVED |
| 3.24.2023 | Leniolisib | Primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) | Pharming | APPROVED |
| 3.30.2023 | Evinacumab | Homozygous familial hypercholesterolemia | Regeneron | APPROVED |
| 4.25.2023 | Tofersen | Superoxide dismutase 1 amyotrophic lateral sclerosis | Biogen / Ionis | APPROVED |
| 5.1.2023 | Omidubicel | Blood Cancers | Gamida Cell | APPROVED |
| 5.9.2023 | Elfabrio | Fabry disease | Chiesi Global Rare Diseases and Protalix BioTherapeutics | APPROVED |
| 5.19.2023 | B-Vec | Epidermolysis bullosa | Krystal | APPROVED |
| 5.19.2023 | Epcoritamab | Elapsed/refractory diffuse large B-cell lymphoma | Epkinly, AbbVie and GenMab | APPROVED |
| 5.23.2023 | Ayvakit | Indolent systemic mastocytosis | Blueprint Medicines | APPROVED |
| 6.15.2023 | Bylvay (odevixibat) | Alagille syndrome | Ipsen | APPROVED |
| 6.15.2023 | Glofitamab (Columvi) | Large B-cell lymphoma | Roche Holding AG | APPROVED |
| 6.20.2023 | Efgartigimod | Myasthenia gravis | Argenx | APPROVED |
| 6.21.2023 | ADX-2191 | Primary vitreoretinal lymphoma | Aldeyra Therapeutics | Complete Response Letter |
| 6.22.2023 | Obeticholic acid | Pre-cirrhotic liver fibrosis due to nonalcoholic steatohepatitis | Intercept Pharmaceuticals | Complete Response Letter |
| 6.22.2023 | SRP-9001 (delandistrogene moxeparvovec) | Duchenne muscular dystrophy | Sarepta Therapeutics | APPROVED |
| 6.27.2023 | Rystiggo (rozanolixizumab-noli) | Myasthenia gravis | UCB | APPROVED |
| 6.30.23 | Valoctocogene Roxaparvovec | Hemophilia A | Biomarin | APPROVED |
| 7.24.2023 | Quizartinib | Acute myeloid leukemia (AML) that is FLT3-ITD positive | Daiichi Sankyo | APPROVED |
| 7.28.2023 | I/ONTAK | Cutaneous T-cell lymphoma (CTCL) | Citius Pharmaceuticals | Complete Response Letter |
| 8.14.2023 | HEPZATO Kit | Hepatic-dominant metastatic ocular melanoma | Delcath Systems | APPROVED |
| 8.16.2023 | Palovarotene (Resubmitted NDA | Fibrodysplasia ossificans progressiva (FOP) | Ipsen | APPROVED |
| 8.20.2023 | Ingrezza | Chorea associated with Huntington’s disease | Neurocrine Biosciences | APPROVED |
| 8.20.2023 | Pozelimab | CHAPLE disease | Regeneron Pharmaceuticals | APPROVED |
| 8.28.2023 | Reblozyl | Lower-Risk Myelodysplastic Syndromes (MDS) | Bristol-Myers Squibb | APPROVED |
| 9.9.2023 | APHEXDA (motixafortide) | Autologous transplantation in multiple myeloma | BioLineRx | APPROVED |
| 9.16.2023 | Momelotinib | Myelofibrosis | GSK | APPROVED |
| 9.27.2023 | NurOwn | Amyotrophic lateral sclerosis (ALS) | Brainstorm Cell Therapeutics | NDA Withdrawn |
| 9.28.2023 | Denileukin Diftitox | Cutaneous T-Cell Lymphoma | Citius Pharmaceutical | Complete Response Letter |
| 10.8.2023 | Onpattro | Cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis | Alnylam Pharmaceuticals | NDA Withdrawn |
| 10.17.2023 | Zilbrysq (zilucoplan) | Myasthenia gravis | UCB | APPROVED |
| 10.25.2023 | Tibsovo (ivosidenib tablets) | Isocitrate dehydrogenase 1 (IDH1)-mutated relapsed or refractory (R/R) myelodysplastic syndromes (MDS) | Servier | APPROVED |
| 10.26.2023 | Vamorolone | Duchenne Muscular Dystrophy | Catalyst Pharmaceutical Partners | APPROVED |
| 11.16.2023 | Adzynma | Congenital thrombotic thrombocytopenic purpura | Takeda Pharmaceutical | APPROVED |
| 11.23.2023 |
Reproxalap |
Dry eye disease | Lord Therapeutics | Complete Response Letter |
| 11.27.2023 |
Nirogacestat |
Desmoid tumors | SpringWorks Therapeutics | APPROVED |
| 12.8.2023 |
Exagamglogene autotemcel |
Sickle cell disease | Vertex Pharmaceuticals | APPROVED |
| 12.8.2023 |
Lovotibeglogene autotemcel |
Sickle cell disease | bluebird bio | APPROVED |
| 12.19.2023 |
Filsuvez (birch triterpenes) |
Junctional Epidermolysis Bullosa (JEB) and Dystrophic Epidermolysis Bullosa (DEB) | Chiesi Global Rare Diseases | APPROVED |
| 12.22.2023 |
Eplontersen |
Hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) | Ionis Pharmaceutical | APPROVED |
| Fourth Quarter of 2023 | Ritlecitinib | Alopecia areata | Pfizer | APPROVED |
2024 Orphan Drugs: PDUFA Dates and FDA Approvals
2022 Orphan Drugs: PDUFA Dates and FDA Approvals
2021 Orphan Drugs: PDUFA Dates and FDA Approvals
