Ultragenyx receives accelerated approval for GSDIa gene therapy

Ultragenyx has received accelerated FDA approval for DTX401, marketed as Genglycos, for glycogen storage disease type Ia. The therapy is indicated to reduce daily cornstarch intake alongside nutritional management in adults and children aged eight and older. It uses an adeno-associated virus vector to deliver the G6Pase gene. The approval requires two years of safety and efficacy data from open-label commercial treatment involving 50 patients and 20 controls.