Favrille drug fails secondary goal
An interim analysis of the secondary endpoint indicated that FavId did not demonstrate a statistically significant difference between treatment and control groups in response improvement. The primary endpoint
Ultragenyx Pharmaceutical has received accelerated approval from the US Food and Drug Administration (FDA) for its gene therapy, Genglycos (pariglasgene brecaparvovec-opnr), to treat glycogen storage disease type Ia (GSDIa).