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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: recombinant adeno-associated virus (AAV) vector encoding the human frataxin (hFXN) gene
Date Designated: 05/07/2026
Orphan Designation: treatment of Friedreich ataxia
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Neurocrine Biosciences, Inc.
6027 Edgewood Bend Ct
San Diego, California 92130
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
*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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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