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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 serotype 9 vector containing the human lysosome-associated membrane glycoprotein 2 isoform B transgene (AAV9.LAMP2B)
Date Designated: 02/01/2019
Orphan Designation: Treatment of Danon Disease
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 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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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