Yes. On September 17, 2026, the U.S. Food and Drug Administration approved Fayuvi (rebisufligene etisparvovec-hopf), the first FDA-approved treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also called Sanfilippo syndrome type A.
Fayuvi is a one-time intravenous AAV9 gene therapy that delivers a working copy of the SGSH gene so cells can make the missing sulfamidase enzyme and break down heparan sulfate. Until this approval, care for the progressive neurodevelopmental disease was limited to managing symptoms. The FDA granted the approval to Ultragenyx Pharmaceutical, Inc.