Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx announced the FDA’s acceptance of the resubmitted Biologics License Application for UX111, an AAV9 gene therapy targeting Sanfilippo syndrome Type A (MPS IIIA), a rare and fatal neurodegenerative disease in children. The FDA granted Priority Review with an expected decision date of September 19, 2026. Clinical data show durable treatment…