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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: adeno-associated vector-based gene therapy expressing codon-optimized human SLC6A8
Date Designated: 11/25/2025
Orphan Designation: treatment of creatine transporter deficiency
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Ronald Reagan UCLA Medical Center
Room 8501
Ronald Reagan UCLA Medical Center 757 Westwood Plaza
Los Angeles, California 90095
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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