FDA Greenlights First Gene Therapy for Sanfilippo Syndrome Type A
The FDA has approved Fayuvi, the first gene therapy for children with Sanfilippo syndrome type A, a rare inherited disorder that progressively damages the brain and nervous system. The condition results from changes in the SGSH gene, which reduce the enzyme sulfamidase and cause heparan sulphate to accumulate in cells. Developed by Ultragenyx, the treatment is given as a single intravenous infusion using an adeno-associated virus 9 vector to deliver a working SGSH gene.
The FDA’s decision makes Fayuvi the first approved gene therapy for Sanfilippo syndrome type A. This rare inherited disorder harms the brain and nervous system over time. It stems from alterations in SGSH, lowering sulfamidase and allowing heparan sulphate to collect inside cells, which contributes to declining cognitive and developmental skills.
Ultragenyx developed the therapy. It is administered once through an intravenous infusion. A modified adeno-associated virus 9 vector carries a functional SGSH gene into cells. The approval was reported in BioNews 1358.
For families affected by Sanfilippo syndrome type A, this approval may represent a new treatment option. Because it is given as a single intravenous infusion, it could simplify administration compared with recurring therapies, though long-term outcomes will depend on follow-up. The broader gene therapy field may see increased attention, and patients with other rare inherited disorders could benefit if similar approaches prove safe and effective.