Posted by Michael Wonder on 13 Jul 2025
Ultragenyx receives complete response letter from FDA for UX111 AAV gene therapy to treat Sanfilippo syndrome type A
11 July 2025 - complete response letter cited specific chemistry, manufacturing and controls related observations that are resolvable.
Ultragenyx today announced that the US FDA has issued a complete response letter for its biologics license application for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A.
