Yahoo · Sep 5, 2026
Why Ultragenyx Pharmaceutical (RARE) Is Down 40.3% After Angelman Trial Failure And Cost-Cut Plan
In early September 2026, Ultragenyx Pharmaceutical announced that its Phase 3 Aspire trial of apazunersen (GTX-102) in Angelman syndrome failed to meet its primary and key secondary cognitive and functional endpoints, prompting a review of the program and planned significant expense reductions. On the same day, the FDA confirmed that Ultragenyx’s newly approved GENGLYCOS gene therapy for glycogen storage disease type Ia qualifies for a rare pediatric disease priority review voucher,...
