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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: Recombinant adeno-associated virus retinal pigment epithelium gene vector AAV2-hRPE65v2
Date Designated: 06/24/2008
Orphan Designation: Treatment of Leber congenital amaurosis due to RPE65 mutations.
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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