Ultragenyx stock price increases due to FDA priority review acceptance

Investing.com – Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) stock rose 1.6% on Monday after the company announced that the U.S. Food and Drug Administration has accepted its biologics license application for DTX401, an AAV gene therapy for Type Ia glycogen storage disease.

The FDA granted priority review status to the application, with an action date set for August 23, 2026, under the Prescription Drug User Fee Act. If approved, DTX401 will become the first treatment targeting the root cause of GSDIa.

The biologics license application is based on data from a clinical development program involving 52 treated patients and up to six years of follow-up. Data from the Phase 3 GlucoGene study, which was randomized, double-blind, and placebo-controlled, showed that patients treated with DTX401 had reduced daily corn starch intake and frequency, while maintaining lower blood sugar levels, with improved normal blood glucose levels and increased fasting tolerance.

These clinical benefits translated into reported improvements in patients’ quality of life, measured by the Patient Global Impression of Change scale. According to the company, DTX401 was well tolerated, with an acceptable safety profile.

If approved, DTX401 will be produced at Ultragenyx’s gene therapy manufacturing facility in Bedford, Massachusetts.

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