The Food and Drug Administration approved Fayuvi, the first gene therapy for pediatric patients with Sanfilippo syndrome type A, also known as MPS IIIA.
The inherited disorder causes progressive damage to the brain and nervous system and can lead to loss of cognitive, language and developmental abilities. Until now, FDA-approved care was limited to managing symptoms rather than targeting the disease itself.
The approval gives families a new treatment option for an exceptionally rare condition while adding to the growing number of gene therapies reaching the U.S. market.