Ultragenyx gets FDA standard full approval for FAYUVI, first Sanfilippo type A treatment

Ultragenyx Pharmaceutical Inc.

• FDA granted standard full approval of FAYUVI (UX111) for pediatric MPS IIIA (Sanfilippo syndrome type A) on Sept 17, 2026.
• FAYUVI is the first-ever FDA-approved treatment for Sanfilippo syndrome type A, a progressive fatal neurodegenerative disease.
• This is Ultragenyx's second gene therapy approval; company received a Priority Review Voucher upon approval.
• Approval covers pediatric patients with mucopolysaccharidosis type IIIA; trade name rebisufligene etisparvovec-hopf.