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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: Self-complimentary adeno-associated virus 9 gene therapy containing codon optimized human AP4M1 (melpida)
Date Designated: 12/01/2022
Orphan Designation: Treatment of patients with Spastic Paraplegia Type 50 (SPG50) caused by the AP4M1 gene mutation
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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