Ultragenyx Secures First Gene Therapy Approval for Glycogen Storage Disease Type Ia
Ultragenyx has received accelerated approval from the U.S. Food and Drug Administration (FDA) for its first gene therapy, pariglasgene brecaparvovec-opnr, now branded as Genglycos. This therapy is designed to treat glycogen storage disease type Ia (GSDIa) in adults and children over the age of eight. GSDIa is a rare genetic disorder caused by a deficiency of the G6PC enzyme, which is crucial for releasing glucose from the liver. Genglycos, a one-time AAV8 gene therapy, aims to deliver a functional G6PC gene to the liver, restoring the enzyme and helping to stabilize blood sugar levels. The approval follows a successful late-stage study where Genglycos significantly reduced the daily cornstarch intake required by patients, a common treatment for GSDIa. 
