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.
"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%).
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