FDA ApprovedUnited States
First FDA-Approved Gene Therapy for Sanfilippo Syndrome Type A
Fayuvi (rebisufligene etisparvovec-hopf) — one-time IV gene therapy for children with Sanfilippo syndrome type A (MPS IIIA)
FDA approval on 17 September 2026 for Fayuvi (rebisufligene etisparvovec-hopf) suspension — a one-time AAV9 gene therapy given by intravenous infusion — for…
This is for families of children with Sanfilippo syndrome type A (MPS IIIA) who still have preserved neurodevelopmental function and may be candidates for…