FDA grants Ultragenyx accelerated approval for GENGLYCOS in GSDIa patients 8 and older

RARE•2026-08-19

Post-marketing follow-up commitment

Ultragenyx said the approval includes a post-marketing commitment for a 2-year open-label follow-up in 50 treated patients, with 20 antibody-positive controls, tracked via its monitoring program.

FDA accelerates approval for GENGLYCOS

The FDA granted Ultragenyx accelerated approval for GENGLYCOS (DTX401) in patients with glycogen storage disease type Ia (GSDIa) aged 8 and older.

Clearance rests on 48-week Phase 3 data showing reduced cornstarch requirements versus placebo, with p<0.001.

Sources

FDA grants Ultragenyx accelerated approval for GENGLYCOS in GSDIa patients 8 and older

Sec

FDA grants Ultragenyx accelerated approval for GENGLYCOS in GSDIa patients 8 and older

Post-marketing follow-up commitment

FDA accelerates approval for GENGLYCOS

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Sources

FDA grants Ultragenyx accelerated approval for GENGLYCOS in GSDIa patients 8 and older

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