Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval for GENGLYCOS™ (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa).

"The approval of GENGLYCOS fulfills our commitment to provide the first therapy that directly targets the root cause of GSDIa. The reduced reliance on cornstarch, experienced by patients in our clinical studies, demonstrates this gene therapy’s ability to establish the normal breakdown of glycogen to produce glucose during fasting or episodes of metabolic stress. This ability to regulate glucose has alleviated the disease burden and has the potential to mitigate the risk of severe or life-threatening hypoglycemia for these patients," said Eric Crombez, M.D., chief medical officer at Ultragenyx. "As our first gene therapy approval, GENGLYCOS represents an important achievement for our company and the realization of the promise of a powerful new tool to deliver transformative medicines for people living with rare diseases."