EU Medicines Regulator Validates Ultragenyx's Investigational Sanfilippo Syndrome Treatment

03:58 AM EDT, 10/02/2026 (MT Newswires) -- Biopharmaceutical group Ultragenyx Pharmaceutical (UP0.F) received the European Medicines Agency's validation for its marketing authorization application for investigational gene therapy rebisufligene etisparvovec. The therapy is being developed to treat mucopolysaccharidosis type IIIA, or Sanfilippo syndrome Type A, and has previously received PRIority MEdicines and Orphan Drug designations from the regulator, according to a Friday release. The application is expected to be evaluated under the Joint Clinical Assessment framework established by the EU Health Technology Assessment Regulation. Ultragenyx has also initiated regulatory engagement with the UK's Medicines and Healthcare products Regulatory Agency and the Saudi Food and Drug Authority as it explores potential expansion into additional markets.