Quick Links: Skip to main page content Skip to Search Skip to Topics Menu Skip to Common Links
  • Decrease font size
  • Return font size to normal
  • Increase font size
U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
  • Print
  • Share
  • E-mail
-
Generic Name: exagamglogene autotemcel
Trade Name: Casgevy
Date Designated: 05/11/2020
Orphan Designation: Treatment of sickle cell disease
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: 12/08/2023
Approved Labeled Indication: treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs)
Exclusivity End Date: 12/08/2030 
Exclusivity Protected Indication* :  treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs)
*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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
-
-