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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: golodirsen
Trade Name: VYONDYS 53
Date Designated: 05/22/2018
Orphan Designation: Treatment of Duchenne muscular dystrophy (DMD)
Orphan Designation Status: Designated/Approved
Sarepta Therapeutics, Inc.
215 First Street
Cambridge, Massachusetts 02142
United States

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

Marketing approved:

1 Generic Name: golodirsen
Trade Name: VYONDYS 53
Marketing Approval Date: 12/12/2019
Approved Labeled Indication: VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Exclusivity End Date: 12/12/2026 
Exclusivity Protected Indication* :  Indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping
*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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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