Ultragenyx Pharmaceutical announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for its gene therapy, GENGLYCOS™ (pariglasgene brecaparvovec-opnr), for treating glycogen storage disease type Ia (GSDIa) in adult and pediatric patients aged eight and older. This approval marks a significant regulatory milestone, providing a new treatment option for this rare metabolic disorder.

Key Details

  • Product: GENGLYCOS™, also known as DTX401, is a gene therapy for Glycogen Storage Disease Type Ia (GSDIa).
  • Approval Basis: The decision was based on positive data from a 48-week, placebo-controlled Phase 3 study of 46 participants, which showed a statistically significant reduction in cornstarch requirements (p<0.001) for treated patients.
  • Post-Marketing Commitment: As a condition of accelerated approval, Ultragenyx must provide two years of safety and efficacy data from a study of 50 commercial patients and 20 control patients to confirm the drug's clinical benefit.