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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 recombinant adeno-associated virus serotype 9 viral vector containing the transgene encoding Transcription Factor 4
Date Designated: 03/08/2026
Orphan Designation: treatment of Pitt Hopkins syndrome
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Mahzi Therapeutics
470 Noor Ave, Ste B #1036
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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