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Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)

RARE
  • The FDA has accepted the Biologics License Application (BLA) for DTX401 (pariglasgene brecaparvovec) to treat Glycogen Storage Disease Type Ia (GSDIa).
  • The FDA granted Priority Review status & assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026.
  • If approved, DTX401 will be the first treatment to address the underlying root cause of GSDIa.
  • Clinical data from the Phase 3 GlucoGene study involved 52 patients with up to six years of follow-up.
  • Results showed significant reductions in daily cornstarch intake, improved euglycemia, enhanced fasting tolerance, & improved patient-reported quality of life.
  • DTX401 demonstrated a well-tolerated safety profile in the clinical trials.
  • The therapy is an investigational AAV8 gene therapy administered via a single intravenous infusion.
  • DTX401 holds multiple designations including Rare Pediatric Disease, orphan drug, Fast Track, RMAT, & PRIME.
  • If approved, manufacturing will occur entirely within the U.S. at a new facility in Bedford, Massachusetts.
  • GSDIa is a rare genetic disorder affecting approximately 6,000 people with no currently approved pharmacologic therapies.