Ultragenyx Says FDA Accepts Resubmitted BLA For Review Seeking Accelerated Approval For UX111 AAV9 Gene Therapy As Treatment For Patients With Sanfilippo Syndrome Type A; FDA Sets PDUFA Action Date Of Sept. 19

Ultragenyx Pharmaceutical Inc. announced that the FDA has accepted its resubmitted Biologics License Application (BLA) for UX111, a gene therapy aimed at treating Sanfilippo syndrome Type A. If approved, UX111 would be the first therapy for this rare disease, which affects young children. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date for September 19, 2026.