The U.S. FDA has approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment specifically approved for pediatric patients with Sanfilippo syndrome type A (MPS IIIA), a rare and progressive genetic disorder affecting the brain and nervous system.
Fayuvi is a one-time intravenous gene therapy designed to deliver a functional copy of the SGSH gene, enabling cells to produce the enzyme needed to break down harmful heparan sulfate buildup. Clinical data in children aged 2 to 5 showed maintenance or improvement of cognitive function compared with an untreated historical control group.
The approval represents a new treatment option for children and families affected by MPS IIIA, for which care had previously focused on managing symptoms. Fayuvi carries important safety considerations, including liver enzyme elevations and the risk tof hrombotic microangiopathy, and requires corticosteroid treatment around the infusion.
Read the full press release: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-pediatric-patients-sanfilippo-syndrome-type