Ultragenyx starts rolling BLA submission for DTX401 gene therapy in GSDIa

Ultragenyx Pharmaceutical Inc.

Initiated rolling BLA submission to FDA for DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia.
Submitted non-clinical and clinical modules; plans to complete full BLA including CMC module in Q4 2025.
BLA includes 96-week Phase 3 data showing ~60-64% reduction in daily cornstarch use from baseline.
Updates address FDA observations from prior UX111 complete response letter related to CMC and manufacturing.

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