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FDA approves Ultragenyx gene therapy for glycogen storage disease type Ia

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The U.S. FDA granted accelerated approval on August 19, 2026, to Ultragenyx’s GENGLYCOS (pariglasgene brecaparvovec-opnr), formerly known as DTX401. The therapy is approved for adults and children aged 8 and older with glycogen storage disease type Ia (GSDIa), also called Von Gierke disease. GENGLYCOS is designed to address the underlying genetic cause of GSDIa and is indicated to reduce daily cornstarch intake alongside nutritional management—the first FDA-approved treatment for the disorder. Approval was supported by the 48-week Phase 3 GlucoGene trial, in which 46 participants receiving the therapy had a statistically significant reduction in cornstarch requirements compared with placebo. Because the approval is accelerated, continued approval depends on confirmatory evidence of clinical benefit. The therapy is contraindicated in patients with severe hepatic fibrosis or cirrhosis. The approval is Ultragenyx’s first gene-therapy approval and its fifth FDA approval overall; ...

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