Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has received accelerated approval from the U.S. Food and Drug Administration (FDA) for GENGLYCOS™ (pariglasgene brecaparvovec-opnr), also known as DTX401, for adults and children aged eight years and older with glycogen storage disease type Ia (GSDIa).

GENGLYCOS is the first FDA-approved treatment designed to address the underlying cause of GSDIa. The approval marks Ultragenyx’s first gene therapy approval and expands treatment options for patients with this rare metabolic disorder.

GSDIa is caused by a deficiency of an enzyme required to release glucose from the liver into the bloodstream. This can lead to severe hypoglycaemia and other complications. Patients typically rely on strict dietary management and frequent intake of uncooked cornstarch to help maintain blood glucose levels, creating a demanding daily treatment routine.

The disease affects an estimated 1,500 to 2,500 people in the US and 6,000 to 8,000 people worldwide across commercially accessible markets.

The FDA approval is supported by results from the 48-week Phase 3 GlucoGene study, a randomised, double-blind, placebo-controlled trial involving 46 participants aged eight years and older. The study found that patients treated with DTX401 required significantly less cornstarch than those receiving placebo. At Week 48, eligible participants crossed over to the alternative treatment and continued to be monitored through Weeks 96 and 144.

As part of the accelerated approval, Ultragenyx will provide two years of additional safety and efficacy data through an expanded GSDIa Disease Monitoring Program. The programme will include 50 patients receiving commercial treatment and 20 control patients who cannot receive GENGLYCOS because of anti-AAV8 antibodies.

The programme will assess measures including cornstarch requirements and fasting tolerance in a post-marketing setting. Previously treated clinical trial participants and newly treated patients will also be followed for up to 10 years to provide longer-term safety and efficacy data.

 

Source: ultragenyx.com

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