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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: a recombinant adeno-associated virus (AAV) vector that contains a bioengineered capsid (AAV-Spark100) and a codonoptimized expression cassette to drive expression of a secretable form of alpha-galactosidase A (alpha-Gal A)
Date Designated: 08/11/2023
Orphan Designation: Treatment of Fabry disease
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Spark Therapeutics, Inc.
3737 Market Street, Suite 1300
Philadelphia, Pennsylvania 19104
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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