FDA approves Genglycos for glycogen storage disease type Ia

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Genglycos (pariglasgene brecaparvovec-opnr), the first authorized gene therapy for adults and children aged 8 and older with glycogen storage disease type Ia (GSDIa).

GSDIa is a rare genetic disorder caused by a deficiency in the glucose-6-phosphatase enzyme, which prevents the liver from releasing glucose into the bloodstream during fasting. This deficiency can lead to dangerous drops in blood sugar and other metabolic complications.

Genglycos is a one-time administration therapy using an adeno-associated virus serotype 8 (AAV8) vector to deliver a functional copy of the G6PC gene to liver cells. The therapy is intended to reduce the daily requirement for cornstarch supplementation as part of nutritional management, rather than replacing the diet entirely. In a phase 3 trial, the treatment reduced daily cornstarch consumption by 31%.