On September 17, FDA approved Fayuvi, a one-time intravenous gene therapy for pediatric patients with Sanfilippo syndrome type A, a progressive inherited condition affecting the brain and nervous system. The agency describes it as the first approved treatment for the condition.
FDA's account says treated children maintained or improved cognitive function relative to an untreated historical cohort. That comparison came from an open-label, single-arm study; it was not a randomized comparison with a concurrent control group. Cognitive changes were measured in children aged two to five.
The announcement also identifies safety concerns, including liver-enzyme increases, blood-cell changes and thrombotic microangiopathy, plus a potential long-term tumor risk from genetic integration. Treatment takes place in an equipped healthcare setting, with corticosteroids starting before infusion.
Families considering treatment need the prescribing information and a specialist's assessment. The announcement does not establish price, local availability or an individual child's likely benefit.