FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Ia

The U.S. Food and Drug Administration today issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr) to reduce daily cornstarch intake as an adjunct to nutritional management in adults and pediatric patients 8 years of age and older with glycogen storage disease type Ia (GSDIa), a rare, inherited genetic disorder caused by a deficiency of the enzyme glucose-6-phosphatase, which prevents the body from properly breaking down stored glycogen into glucose. Genglycos is the first approved treatment for this condition.The accelerated approval pathway allows the FDA to approve certain drugs or biologics intended to treat a serious or life-threatening disease or condition if th...