Ultragenyx Completes Rolling BLA Submission to FDA for DTX401 Gene Therapy

Ultragenyx Pharmaceutical Inc. has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for DTX401 AAV gene therapy (pariglasgene brecaparvovec) as a treatment for Glycogen Storage Disease Type Ia (GSDIa). The submission is supported by clinical data from 52 treated patients with up to six years of follow-up, showing significant reductions in daily cornstarch intake and improvements in glycemic control, fasting tolerance, and patient quality of life. The FDA previously granted rolling review, allowing Ultragenyx to submit modules of the BLA on an ongoing basis, and the company has now completed the process with the submission of the final chemistry, manufacturing, and controls module.

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