Ultragenyx Pharmaceutical Inc. has finished the rolling submission of its Biologics License Application (BLA) for the AAV8‑based gene therapy DTX401 (pariglasgene brecaparvovec) to the U.S. Food and Drug Administration. The submission incorporates data from a 52‑patient program with up to six years of follow‑up, the chemistry, manufacturing, and controls (CMC) module, and all regulatory designations that the company has earned for the product, including Rare Pediatric Disease, Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT).
DTX401 delivers a functional copy of the glucose‑6‑phosphatase (G6Pase) enzyme to liver cells, correcting the metabolic defect that causes life‑threatening hypoglycemia in Glycogen Storage Disease Type Ia (GSDIa). Clinical data show that treated patients have markedly reduced daily cornstarch intake and improved glycemic control, with a safety profile that aligns with expectations for an AAV‑based vector. The therapy represents the first curative approach for a disease that currently requires lifelong dietary management for an estimated 6,000 patients worldwide.
The regulatory milestone is a key step toward market approval and could unlock a substantial revenue opportunity for Ultragenyx. The company’s 2025 financial results show revenue of $630.6 million and a diluted earnings‑per‑share of –$5.94, indicating that the business remains unprofitable. A market capitalization of $2.18 billion reflects the high valuation that investors place on the company’s rare‑disease pipeline, which also includes GTX‑102 for Angelman syndrome and UX111 for Sanfilippo syndrome type A. Recent setbacks, such as the Phase 3 failure of setrusumab for osteogenesis imperfecta, have weighed on investor sentiment, but the DTX401 filing signals renewed progress in the pipeline.
Chief Medical Officer Eric Crombez said the BLA submission “marks a pivotal moment in our journey to bring a first‑in‑class therapy to patients who have no approved pharmacologic options.” He added that the data supporting the therapy’s efficacy and safety are robust and that the company is prepared to address any remaining questions from the FDA. The company’s leadership remains focused on advancing the DTX401 program while continuing to invest in other rare‑disease assets.
Investors have responded positively to the filing, reflecting confidence in the company’s ability to navigate the regulatory process and bring a transformative therapy to market. The event also reinforces Ultragenyx’s strategy of building a diversified portfolio of gene‑therapy products, positioning the company to capture a share of the growing rare‑disease therapeutics market.
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