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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 serotype 9 (AAV9) gene transfer vector carrying the pantothenate kinase 2 (PANK2) cDNA
Date Designated: 07/31/2026
Orphan Designation: treatment of pantothenate kinase-associated neurodegeneration
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Susan J. Hayflick, MD PhD
Molecular & Medical Genetics, Oregon Health & Science University
3181 SW Sam Jackson Park Road
Portland, Oregon 97239
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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