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Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)
If approved, DTX401 will be the first therapy approved to treat the underlying cause of GSDIa NOVATO, Calif., Dec. 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"If approved, DTX401 will be the first therapy approved to treat the underlying cause of GSDIa\nNOVATO, Calif., Dec. 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA), seeking approval for DTX401 AAV gene therapy (pariglasgene brecaparvovec) as a treatment for Glycogen Storage Disease Type Ia (GSDIa). \"The completion of our rolling submission of the BLA for DTX401 is a significant step toward our commitment to deliver the first therapy that directly targets the underlying cause of GSDIa,\" said Eric Crombez, M.D., Chief Medical Officer at Ultragenyx. \"Despite burdensome daily dietary and cornstarch management, patients continue to face life-threatening risks from acute hypoglycemia and chronic complications impacting the liver, kidneys, gastrointestinal system, bones, and growth. We look forward to continuing our collaboration with the FDA throughout the review process to be able to provide this potentially life-changing therapy to as many people living with GSDIa as possible.” The BLA for DTX401 is based on data from a rigorous clinical development program that includes 52 treated patients and up to six years of follow-up. Previously announced data from the randomized, double-blind, placebo-controlled Phase 3 GlucoGene study demonstrate that patients treated with DTX401 experienced significant and clinically meaningful reductions in both the quantity and frequency of daily cornstarch intake while maintaining low levels of hypoglycemia, improved levels of euglycemia, and improved fasting tolerance. These clinical benefits translated to meaningful improvements in patient-reported quality of life, as measured by the Patient Global Impression of Change (PGIC) scale. DTX401 was well tolerated, with an acceptable safety profile. The company was previously granted rolling review and submitted the non-clinical and clinical modules to the FDA in August; and has now completed the package through submission of the chemistry, manufacturing, and controls (CMC) module. About DTX401 (pariglasgene brecaparvovec)DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase under control of the native promoter to allow the tre...