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: non-replicating recombinant adeno-associated virus serotype 9 containing the solute carrier family 6 member 1 transgene (SLC6A1)
Date Designated: 08/21/2026
Orphan Designation: treatment of SLC6A1-related disease
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Nationwide Children’s Hospital
700 Children’s Drive
Columbus, Ohio 43205
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
-
-