Quick Links: Skip to main page content Skip to Search Skip to Topics Menu Skip to Common Links
  • Decrease font size
  • Return font size to normal
  • Increase font size
U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
  • Print
  • Share
  • E-mail
-
Generic Name: a recombinant adeno-associated virus serotype 9-based vector containing an expression cassette of the human AADC and human GDNF genes
Date Designated: 01/23/2026
Orphan Designation: treatment of aromatic L-amino acid decarboxylase (AADC) deficiency
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Shanghai Vitalgen BioPharma Co., Ltd.
Building 2, No.11 Jia Lilue Road
Pudong New District
Shanghai, Shanghai 201210
China

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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
-
-