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 PharmaceuticalsNDA accepted
2.28.2025MirdametinibNeurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN)Springworks TherapeuticsNDA accepted
3.20.2025Rivoceranib/Camrelizumab Unresectable or metastatic hepatocellular carcinomaElevar TherapeuticsNDA accepted
3.23.2025VutrisiranATTR-CMAlnylam PharmaceuticalssNDA accepted
3.27.2025Diazoxide choline (DCCR)Prader-Willi syndromeSoleno TherapeuticsNDA accepted
3.28.2025FitusiranHemophilia A or BSanofiNDA accepted
4.21.2025Nivolumab plus ipilimumabHepatocellular carcinoma (HCC)Bristol Myers SquibbsBLA accepted
4.29.2025Prademagene zamikeracel (pz-cel)Epidermolysis bullosaAbeona TherapeuticsBLA accepted
4.29.2025ElamipretideBarth syndromeStealth BioTherapeuticsNDA accepted
6.17.2025SebetralstatHereditary AngioedemaKalVista PharmaceuticalsNDA accepted
6.23.2025Taletrectinib Advanced ROS1-positive non-small cell lung cancerNuvation BioNDA accepted
7.23.2025Belantamab mafodotinMultiple myelomaGSKBLA accepted
7.24.2025AvatrombopagImmune thrombocytopeniaSobi North AmericasNDA accepted
7.27.2025TransCon hGHGrowth hormone deficiencyAscendis PharmasBLA accepted
8.21.2025DonidalorsenHereditary AngioedemaIonis PharmaceuticalsNDA accepted
8.28.2025FilspariIgA nephropathyTravere TherapeuticssNDA accepted
8.29.2025RilzabrutinibImmune thrombocytopeniaSanofiUnder review
9.7.2025Pyrukynd (mitapivat)Beta-thalassemiaAgios PharmaceuticalssNDA accepted
9.25.2025PaltusotineAcromegalyCrinetics PharmaceuticalsNDA accepted
11.18.2025PlozasiranFamilial chylomicronemia syndromeArrowhead PharmaceuticalsNDA accepted