PDUFA action date set for August 23, 2026
If approved, DTX401 will be the first treatment to address the underlying cause of GSDIa
Reno, Nevada (UroToday.com) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the Biologics License Application (BLA) seeking approval of DTX401 AAV gene therapy (pariglasgene brecaparvovec) for the treatment of Glycogen Storage Disease Type Ia (GSDIa). The FDA granted the BLA Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026.
“Current dietary approaches to managing GSDIa place an extraordinary burden on individuals and families while still leaving patients with significant medical needs, including the risk of potentially life-threatening episodes of acute hypoglycemia and accumulation of long-term complications over their lifetime,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “If approved, DTX401 would be the first treatment to address the disease at its root cause. We appreciate the FDA’s timely acceptance of the BLA and will continue to work with the Agency throughout its review process.”The BLA 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.
If approved, DTX401 will be manufactured entirely within the U.S. at the new Ultragenyx gene therapy manufacturing facility in Bedford, Massachusetts.
Source: Ultragenyx Pharmaceutical Inc. (2026). 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) [Press release]. https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-us-fda-acceptance-and-priority-review.
This webpage is supported through an unrestricted educational grant from Bayer. Bayer is not involved in content development and the views expressed represent those of the patient and physician contributors.
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