Ultragenyx completes rolling BLA submission for DTX401 gene therapy to FDA for GSDIa

Ultragenyx Pharmaceutical Inc.

BLA submission for DTX401 (pariglasgene brecaparvovec) completed for glycogen storage disease type Ia; based on 52 treated patients with up to 6 years follow-up.
Phase 3 GlucoGene study showed significant reductions in daily cornstarch intake while maintaining euglycemia and improved quality of life.
Rolling review began with non-clinical/clinical modules in August 2025; CMC module now submitted to complete the package.
DTX401 was well tolerated with an acceptable safety profile per the press release.

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