Ultragenyx Submits BLA for DTX401 Gene Therapy Targeting GSDIa
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 1h ago
0mins
Source: Newsfilter
- Significant Milestone: Ultragenyx has completed the rolling submission of its Biologics License Application (BLA) for DTX401 gene therapy, targeting approximately 6,000 GSDIa patients, marking a crucial step toward delivering the first therapy that addresses the underlying cause of the disease to the FDA.
- Clinical Data Support: The application is based on rigorous clinical data from 52 treated patients, demonstrating that DTX401 significantly reduces daily cornstarch intake while maintaining low hypoglycemia levels and improving patient-reported quality of life, indicating strong market demand and therapeutic efficacy.
- Regulatory Progress: Ultragenyx received rolling review from the FDA in August 2025 and has submitted all necessary modules, reflecting the company's strategic commitment to accelerating drug development and approval processes, which is expected to provide patients with faster access to treatment options.
- Market Potential: GSDIa is a serious metabolic disease with no approved pharmacologic therapies; the successful launch of DTX401 will fill a significant market gap, addressing the urgent need for effective treatments and further solidifying Ultragenyx's leadership in the rare disease sector.
RARE
$22.78+Infinity%1D
Analyst Views on RARE
Wall Street analysts forecast RARE stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for RARE is 77.08 USD with a low forecast of 35.00 USD and a high forecast of 125.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
14 Analyst Rating
13 Buy
1 Hold
0 Sell
Strong Buy
Current: 19.720
Low
35.00
Averages
77.08
High
125.00
Current: 19.720
Low
35.00
Averages
77.08
High
125.00
About RARE
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. Its therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. Its four approved product candidates include Crysvita (burosumab) for the treatment of X-linked hypophosphatemia (XLH), and tumor-induced osteomalacia (TIO), Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII (MPSVII) or Sly Syndrome, Dojolvi (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its clinical product candidates include DTX401, DTX301, UX701, UX143, UX111, and GTX-102. UX143 for the treatment of Osteogenesis Imperfecta.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
