Japan records surprise trade deficit in July as exports weaken further
NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a $2.86 billion market cap biotech company whose shares have surged 8.4% in the past week, has begun a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration for its DTX401 AAV gene therapy, the company announced Monday.
The therapy is being developed as a treatment for Glycogen Storage Disease Type Ia (GSDIa), a rare inherited condition that affects approximately 6,000 people in commercially accessible regions. Ultragenyx, which has demonstrated strong revenue growth of 26.77% over the last twelve months and maintains a healthy current ratio of 2.45, has submitted the non-clinical and clinical modules to the FDA and plans to complete the full BLA with the chemistry, manufacturing and controls module in the fourth quarter of 2025. For detailed financial analysis of biotech companies like Ultragenyx, InvestingPro offers comprehensive research reports covering over 1,400 US stocks.
"Initiating the BLA for DTX401 is an important milestone for this much needed treatment option for individuals and families affected by this disorder," said Eric Crombez, chief medical officer at Ultragenyx, in a press release statement. Wall Street appears optimistic about the company’s prospects, with analysts maintaining a bullish consensus recommendation of 1.38 out of 5 (where 1 is Strong Buy).
The BLA includes 96-week data from a randomized, placebo-controlled Phase 3 study showing patients had reductions in total daily cornstarch at their last visit compared to baseline in both the ongoing DTX401 group (-60%) and the Crossover Placebo to DTX401 group (-64%).
Currently, GSDIa patients must take frequent doses of cornstarch to prevent potentially life-threatening hypoglycemia, as the condition prevents normal blood sugar regulation due to a defective gene coding for the enzyme G6Pase-α.
DTX401 is administered as a single intravenous infusion and has received orphan drug designation, regenerative medicine advanced therapy designation and Fast Track designation from the FDA, as well as PRIority MEdicines and orphan drug designation from the European Medicines Agency.
The company indicated it is working to address FDA observations identified in a previous complete response letter related to its manufacturing facilities. Investors seeking deeper insights into biotech companies’ regulatory milestones and their potential market impact can access advanced analysis tools and expert research through InvestingPro, which offers real-time updates and comprehensive financial metrics for informed decision-making.
In other recent news, Ultragenyx Pharmaceutical Inc. reported better-than-expected financial results for the second quarter of 2025. The company posted an earnings per share (EPS) of -$1.17, which was an improvement over the forecasted -$1.30. Additionally, Ultragenyx’s revenue reached $166 million, surpassing the expected $161.97 million. These financial results represent a positive development for the company. While stock price movements were noted, they will not be discussed further here.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.