for neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function
BLA· Standard Review · Orphan
FDA granted standard full approval to FAYUVI (rebisufligene etisparvovec-hopf, previously UX111) on September 17, 2026 — two days ahead of the September 19 PDUFA target action date — for the treatment of neurologic manifestations of MPS IIIA (Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function. FAYUVI is the first-ever FDA-approved treatment for Sanfilippo syndrome type A and Ultragenyx's second approved gene therapy.
About
Sanfilippo syndrome type A, also called mucopolysaccharidosis type IIIA, is a rare inherited disease that mainly affects the brain and spinal cord: children cannot properly break down certain large sugar molecules, which build up and gradually damage the nervous system. FAYUVI is a one-time intravenous gene therapy that delivers a working copy of the SGSH gene, giving cells the instructions they need to break those sugars down. Because it restores the missing gene at its source, this treatment may help preserve thinking and development in young children
