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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: AAV9 gene therapy containing codon optimized human FIG4 cDNA coding sequence
Date Designated: 10/29/2024
Orphan Designation: treatment of Charcot-Marie-Tooth disease type 4J (CMT4J)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Elpida Therapeutics
16501 Ventura Blvd, Suite 400
Encino, California 91436
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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