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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Generic Name: exagamglogene autotemcel
Trade Name: Casgevy
Date Designated: 04/28/2020
Orphan Designation: Treatment of beta-thalassemia
Orphan Designation Status: Designated/Approved
Vertex Pharmaceuticals Inc.
50 Northern Avenue
Boston, Massachusetts 02210
United States

The sponsor address listed is the last reported by the sponsor to OOPD.

Marketing approved:

1 Generic Name: exagamglogene autotemcel
Trade Name: Casgevy
Marketing Approval Date: 01/16/2024
Approved Labeled Indication: treatment of patients aged 12 years and older with transfusion-dependent beta-thalassemia (TDT)
Exclusivity End Date: 01/16/2031 
Exclusivity Protected Indication* :  treatment of patients aged 12 years and older with transfusion-dependent beta-thalassemia (TDT)

*Exclusivity Protected Indications are shown for approvals from 01/01/2013 to the present.
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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