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On August 19, 2026, Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration granted…
RAREUltragenyx Pharmaceutical Inc.
On August 19, 2026, Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration granted accelerated approval for GENGLYCOS (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia. The approval is based on positive data from the Phase 3 GlucoGene study, which demonstrated a significant reduction in cornstarch requirements for treated participants compared to placebo. As part of the accelerated approval conditions, Ultragenyx must provide two years of safety and efficacy clinical data from open-label commercial treatment involving 50 patients and 20 control patients through its existing Disease Monitoring Program. The company operates in the biotechnology industry, focusing on gene therapies for rare genetic diseases.