Ultragenyx wins FDA accelerated approval for GENGLYCOS (DTX401) in GSDIa

Ultragenyx Pharmaceutical Inc.

FDA granted accelerated approval for GENGLYCOS (pariglasgene brecaparvovec-opnr) in GSDIa patients aged 8+.
Approval based on Phase 3 GlucoGene: 46 participants, cornstarch requirement reduction at Week 48 (p<0.001).
Efficacy data from 44 mITT (DTX401 n=20, placebo n=24); crossover followed to Week 96/144.
Post-marketing DMP: 2-year data from 50 treated and 20 control patients; 10-year total follow-up.
Control group consists of patients with anti-AAV8 antibodies ineligible for treatment.

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