Ultragenyx's Genglycos receives FDA accelerated approval for GSDIa

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Ultragenyx Pharmaceutical’s gene therapy, Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401. The treatment is indicated for adult and pediatric patients aged eight years and older with glycogen storage disease type Ia (GSDIa).

GSDIa is a rare genetic metabolic disorder caused by an enzyme deficiency that prevents the liver from releasing glucose into the bloodstream. This condition requires patients to undergo continuous nutritional monitoring, often involving frequent intake of raw cornstarch to manage glucose levels. Genglycos is the first FDA-approved therapy designed to address the underlying cause of the disease, and it has been shown to significantly reduce the daily cornstarch requirements of patients.

The approval is based on results from the 48-week, Phase III GlucoGene study, a randomized, double-blind, placebo-controlled trial involving 46 participants. To maintain approval, Ultragenyx must provide two years of efficacy and safety data from a commercial treatment group and continue long-term monitoring through the GSDIa Disease Monitoring Program for up to ten years.