FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A

The U.S. Food and Drug Administration (FDA) has approved Fayuvi (rebisufligene etisparvovec-hopf), marking the first gene therapy authorized for Sanfilippo syndrome type A. This one-time AAV9 gene therapy is indicated for pediatric patients who have preserved neurodevelopmental function, aiming to address the neurological manifestations of this ultrarare lysosomal storage disorder.