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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: replication deficient adeno-associated virus gene therapy vector expressing a FRRS1L gene
Date Designated: 05/07/2026
Orphan Designation: treatment of developmental and epileptic encephalopathy type 37 (DEE 37)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Finding Hope for FRRS1L
301 Boardwalk Drive, P.O. Box 270405
Fort Collins, Colorado 80525
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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