Ultragenyx secures FDA approval for Glenglycos, first gene therapy for GSD Ia

Ultragenyx announced FDA approval of Glenglycos, a one-time gene therapy for glycogen storage disease type Ia, a rare genetic disorder.

The therapy delivers a functional gene to the liver to restore the missing enzyme that regulates blood sugar between meals, addressing life-threatening metabolic imbalance.

The drug carries a wholesale acquisition cost of $2.7 million per patient, and the company expects to launch within 30-60 days at specialized centers. The approval also grants Ultragenyx a priority review voucher.

This marks Ultragenyx’s fifth approved product and its first marketable gene therapy, providing momentum after recent setbacks, though analysts do not expect it to become a blockbuster.