FDA grants approval to Ultragenyx's Genglycos gene therapy for ultra-rare glycogen storage disorder

Ultragenyx announced that the U.S. Food and Drug Administration has approved its gene therapy called Genglycos.

The therapy targets an ultra-rare glycogen storage disorder that can lead to severe drops in blood sugar.

This marks the company's first gene-therapy product to receive regulatory clearance, expanding its pipeline beyond its existing treatments.

The approval was granted under the FDA's accelerated pathway, reflecting the unmet medical need for this condition.