Milestone Sanfilippo nod helps Ultragenyx regroup after tough Angelman defeat

Ultragenyx secured FDA approval for Fayuvi to treat Sanfilippo syndrome type A and for Genglycos, its first gene therapy for glycogen storage disease type Ia, both accompanied by valuable priority review vouchers. These regulatory successes follow a costly Phase 3 failure in Angelman syndrome, prompting the company to reduce expenses and reassess its operational strategy.

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