StoryRegulatory
FDA grants accelerated approval to Genglycos, the first gene therapy for glycogen storage disease type Ia
The FDA granted accelerated approval to Genglycos (DTX401) for people aged 8 and older with GSDIa, Ultragenyx's first gene therapy approval. In a 46-person placebo-controlled Phase 3 trial, treated patients needed significantly less cornstarch to keep blood sugar up. Full approval depends on a two-year confirmatory study.
- Phase 3 GlucoGene: 48-week randomised, double-blind trial, 46 participants (20 on the therapy, 24 on placebo).
- Cornstarch use fell 41% on average at week 48, versus 10% on placebo (p<0.001).
- Confirmatory study: 50 treated patients and 20 controls who have anti-AAV8 antibodies.
- Earned a rare pediatric disease priority review voucher.