CBER 2024 Orphan Approvals
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 |
|---|---|---|---|
| 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 |
| CASGEVY | Indicated for the treatment of patients 12 years of age and older with transfusion-dependent beta thalassemia (TDT). | Vertex Pharmaceuticals Inc | 1/16/24 |
| 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 | 2/16/2924 |