Ultragenyx Pharmaceutical Inc. has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for UX111 (rebisufligene etisparvovec), an AAV9 gene therapy intended to treat patients with Sanfilippo syndrome type A (MPS IIIA). The FDA granted Priority Review to the application in February 2025. A Prescription Drug User Fee Act (PDUFA) action date is expected to be assigned within a month of resubmission, with a review period of up to six months anticipated, and a PDUFA date projected for the third quarter of 2026. If approved, UX111 would become the first therapy available for Sanfilippo syndrome type A.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Ultragenyx Pharmaceutical Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9645894-en) on January 30, 2026, and is solely responsible for the information contained therein.