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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
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
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