newsfilter
← Latest News
GlobeNewswire·

Ultragenyx Announces U.S. FDA Approval of GENGLYCOS™ Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)

RARE
  • FDA granted accelerated approval for GENGLYCOS (pariglasgene brecaparvovec-opnr), also known as DTX401, as the first gene therapy & fifth overall FDA approval for Ultragenyx Pharmaceutical Inc.
  • GENGLYCOS is indicated to reduce daily cornstarch intake as an adjunct to nutritional management in adult & pediatric patients aged 8 years and older with glycogen storage disease type Ia (GSDIa).
  • Approval was based on positive data from the Phase 3 GlucoGene study, which showed a statistically significant reduction in cornstarch requirements in the treated group compared to placebo.
  • Ultragenyx received a Priority Review Voucher upon approval of GENGLYCOS.
  • The approval addresses the burden of GSDIa, a disorder affecting 1,500-2,500 patients in the U.S. that currently requires a rigorous, around-the-clock regimen of raw cornstarch to prevent life-threatening hypoglycemia.
  • Ultragenyx agreed to provide two years of safety & efficacy data from a post-marketing Disease Monitoring Program involving 50 commercial patients & 20 controls to confirm clinical benefit for continued approval.
  • GENGLYCOS is contraindicated in patients with known severe hepatic fibrosis or cirrhosis due to risks of hepatotoxicity & adrenal insufficiency.
  • Warnings include potential hypersensitivity reactions, infusion reactions, elevated liver enzymes, adrenal insufficiency, & a theoretical risk of tumorigenicity from AAV vector integration.
  • Common adverse reactions observed in clinical trials included elevated ALT/AST enzymes (71%), nausea (38%), hypertriglyceridemia (29%), headache (24%), & adrenal insufficiency (24%).
  • Patients must use effective contraception for 12 months after administration, & men must not donate semen for 6 months post-treatment.
  • Vector shedding in urine, stool, & saliva can occur for 3 months after infusion, requiring specific hygiene precautions.
  • GENGLYCOS will be administered through a national network of Qualified Treatment Centers & supported by the UltraCare program with dedicated Gene Therapy Guides.
  • Ultragenyx will host a conference call on August 19, 2026, at 6:00 p.m. Eastern Time to discuss the approval.