Federal regulators have approved the first treatment designed to change the course of Sanfilippo syndrome type A, a rare inherited disease that slowly strips young children of speech, thinking, and movement. The U.S. Food and Drug Administration approved Fayuvi (rebisufligene etisparvovec-hopf) on Sept. 17, a one-time intravenous gene therapy for pediatric patients with mucopolysaccharidosis type IIIA. Until that decision, care was limited to managing symptoms.
For the small number of American families living with this diagnosis, the approval turns an abstract hope into a series of concrete, urgent decisions. Parents now have to ask which hospitals will administer the infusion, whether their insurer will cover it, and whether their child still meets the criteria on the label, which limits treatment to children with preserved neurodevelopmental function.
