Ultragenyx Initiates BLA Submission for DTX401 Gene Therapy in GSDIa Treatment.

Ultragenyx has initiated a rolling submission of a Biologics License Application (BLA) to the FDA for its gene therapy, DTX401, targeting Glycogen Storage Disease Type Ia (GSDIa). The Phase 3 trial data showed a significant 60-64% reduction in daily cornstarch requirements. The complete BLA is expected in Q4 2025.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has taken a significant step forward in its efforts to develop a treatment for Glycogen Storage Disease Type Ia (GSDIa) by initiating a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA). The company expects to complete the full BLA, including the chemistry, manufacturing and controls (CMC) module, by the fourth quarter of 2025 [1].

The rolling submission allows the FDA to begin reviewing the non-clinical and clinical modules of the BLA while Ultragenyx completes the CMC module. The BLA includes data from a Phase 3 clinical trial that demonstrated a substantial reduction in daily cornstarch requirements. Specifically, patients in the ongoing DTX401 group and the crossover placebo to DTX401 group showed reductions of 60% and 64%, respectively, compared to baseline [2].

The submission also addresses FDA observations related to the CMC and manufacturing facilities, which were previously identified in the UX111 complete response letter. By proactively addressing these observations, Ultragenyx aims to expedite the regulatory review process and increase the likelihood of a first-cycle approval [1].

GSDIa is a serious inherited glycogen storage disease caused by a defective gene coding for the enzyme G6Pase-α. It leads to the inability to regulate blood sugar, potentially resulting in life-threatening hypoglycemia and other complications. Currently, there are no approved pharmacologic therapies for GSDIa, making DTX401 a promising treatment option [2].

Ultragenyx's strategy of rolling submission and proactive resolution of regulatory hurdles demonstrates its commitment to delivering safe and effective therapies to patients with high unmet medical needs. The company's forward-looking statements, however, should be considered with the understanding that they involve substantial risks and uncertainties, including the unpredictability of clinical drug development and regulatory approval processes [1].

References:
[1] https://finance.yahoo.com/news/ultragenyx-initiates-rolling-submission-biologics-120000140.html
[2] https://www.stocktitan.net/news/RARE/ultragenyx-initiates-rolling-submission-of-biologics-license-l8g61yk6xz0u.html