The FDA has officially accepted the resubmitted Biologics License Application (BLA) for UX111 (rebisufligene etisparvovec). UX111 is an AAV9 gene therapy candidate for the treatment of Sanfilippo syndrome Type A (MPS IIIA). The FDA has established a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026, for the regulatory decision.