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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 nonreplicating recombinant adeno-associated virus gene therapy vector expressing human MTM1 gene
Date Designated: 10/22/2024
Orphan Designation: treatment of X-linked myotubular myopathy
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Astellas Gene Therapies, Inc.
480 Forbes Boulevard
South San Francisco, California 94080
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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