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CBER 2024 Orphan Approvals (new BLAs)

This list reflects information regarding the applications as of the approval date. It is not updated with regard to applicant or application status changes. The applications are listed by date of approval in reverse chronological order.

Trade Name/ Proper NameIndicationManufacturer/License NumberApproval Date
RYONCILTreatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older.Mesoblast, Inc12/18/2024
KEBILIDITreatment of adult and pediatric patients with aromatic L amino acid decarboxylase (AADC) deficiencyPTC Therapeutics, Inc11/13/2024
AUCATZYLTreatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)Autolus Inc11/8/2024
TECELRATreatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and whose tumor expresses the MAGE-A4 antigen as determined by FDA-approved or cleared companion diagnostic devices.Adaptimmune LLC8/1/2024
BEQVEZTreatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who: currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and, do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.Pfizer, Inc4/25/2024
LENMELDYTreatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).Orchard Therapeutics Limited3/18/2024
AMTAGVITreatment of adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitorIovance Biotherapeutics, Inc2/16/2024
 
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