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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 serotype 8 vector encoding human glucose-6-phosphatase-alpha (G6Pase or G6PC)
Date Designated: 09/28/2016
Orphan Designation: Treatment of glycogen storage disease type Ia (von Gierke Disease)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Ultragenyx Pharmaceutical, Inc.
60 Leveroni Court
Novato, California 94949
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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