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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: non-replicating AAV-based gene therapy vector with a single stranded DNA genome engineered to carry an expression cassette encoding for human cardiac myosin binding protein-C (cMyBP-C; hMYBPC3 transgene)
Date Designated: 09/26/2023
Orphan Designation: Treatment of patients with hypertrophic cardiomyopathy (HCM) with a mutation in the MYBPC3 gene
Orphan Designation Status: Designated/Designation Withdrawn or Revoked
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
BioMarin Pharmaceutical Inc.
105 Digital Drive
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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