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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 Virus 9 human Propionyl-CoA Carboxylase, alpha subunit (AAV9-hPCCA)
Date Designated: 09/27/2021
Orphan Designation: Treatment of propionic acidemia
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
National Institutes of Health, National Center for Advancing Translational Sciences
9800 Medical Center Dr.
Rockville, Maryland 20850
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.