Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares are up during Thursday’s premarket session on the recent U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy, Genglycos (pariglasgene brecaparvovec-opnr, or DTX401).
Genglycos Approved For Ultra-Rare Genetic Disorder
This marks a significant milestone as it is the first-ever FDA-approved treatment for Glycogen Storage Disease Type Ia (GSDIa).
The approval of Genglycos represents Ultragenyx’s first gene therapy and fifth FDA approval.
GSDIa is an ultra-rare genetic metabolic disorder caused by a deficiency of the enzyme needed to release glucose from the liver to the bloodstream.
