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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 Name Indication Manufacturer/License Number Approval Date
BEQVEZ Treatment 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, Inc 4/25/2024
LENMELDY Treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD). Orchard Therapeutics Limited 3/18/2024
AMTAGVI Treatment 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 inhibitor Iovance Biotherapeutics, Inc 2/16/2024
CASGEVY Indicated for the treatment of patients 12 years of age and older with transfusion-dependent beta thalassemia (TDT). Vertex Pharmaceuticals Inc 1/16/2024
 
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