Posted by Michael Wonder on 02 Apr 2026
Ultragenyx announces US FDA acceptance of BLA resubmission for UX111 AAV gene therapy to treat Sanfilippo syndrome type A
2 April 2026 - Ultragenyx Pharmaceutical today announced the US FDA has accepted for review the resubmitted biologics license application seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A .
The FDA set a PDUFA action date of 19 September 2026.
