Ultragenyx Positive Phase 3 Data on Rare Metabolic Disorder Treatment

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced positive 96-week results from its Phase 3 study of DTX401, a gene therapy for glycogen storage disease type Ia (GSDIa). The therapy demonstrated significant improvements in reducing cornstarch dependency while maintaining glycemic control.

Key findings at Week 96 include a 61% mean reduction in daily cornstarch intake for both treatment and crossover groups, with 70-75% reduction in nighttime cornstarch usage. Two-thirds of participants eliminated at least one nighttime dose. The therapy's efficacy was further validated by data from a Japanese cohort, where all three pediatric participants achieved 95% reduction in cornstarch intake by Week 24, with complete elimination in all cases.

Patient quality of life improved significantly, with 83-95% of participants reporting reduced disease burden. The treatment demonstrated an acceptable safety profile with manageable hepatic reactions.

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