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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Generic Name: lentiviral vector containing the human liver and erythroid pyruvate kinase gene
Date Designated: 03/23/2016
Orphan Designation: Treatment of pyruvate kinase deficiency.
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Rocket Pharmaceuticals, Inc.
The Empire State Building
350 Fifth Avenue, Suite 7530
New York, New York 10118
United States

The sponsor address listed is the last reported by the sponsor to OOPD.

*Exclusivity Protected Indications are shown for approvals from Jan. 1, 2013, to the present.