US FDA approves Ultragenyx's gene therapy for rare disorder

The US FDA approved Ultragenyx Pharmaceutical's gene therapy Fayuvi for pediatric patients with Sanfilippo syndrome Type A, the first treatment for this rare and fatal childhood disease. The therapy delivers a functional SGSH gene to produce the enzyme sulfamidase, improving or maintaining cognitive function in treated children.

This Wire brief sits within Fusion42's coverage of Biotech, and 9 sources have reported it between 17 Sep 2026 and 28 Sep 2026.