FDA grants accelerated approval to Genglycos, first therapy for glycogen storage disease type Ia

The U.S. Food and Drug Administration issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), marking the first therapy for patients aged eight years and older with glycogen storage disease type Ia.

GSDIa is a rare inherited disorder caused by a deficiency of the enzyme glucose-6-phosphatase, which prevents the body from converting stored glycogen into glucose. Patients typically rely on strict, lifelong dietary management, including frequent cornstarch meals, to maintain blood sugar levels.

The approval was based on trial data showing that Genglycos reduces the daily amount of cornstarch needed when used alongside an appropriate diet, a surrogate endpoint the FDA deemed likely to improve clinical outcomes.