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Ultragenyx Begins FDA Application for DTX401 Gene Therapy

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Ultragenyx Begins FDA Application for DTX401 Gene Therapy

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Ultragenyx Pharmaceutical ( (RARE) ) just unveiled an announcement.

On August 18, 2025, Ultragenyx Pharmaceutical announced the start of a rolling submission of a Biologics License Application to the FDA for DTX401, a gene therapy for Glycogen Storage Disease Type Ia. The submission includes promising 96-week data from a Phase 3 study showing significant reductions in daily cornstarch intake among patients. The company plans to complete the submission by the fourth quarter of 2025, addressing FDA observations related to manufacturing and controls.

The most recent analyst rating on (RARE) stock is a Buy with a $81.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

Spark’s Take on RARE Stock

According to Spark, TipRanks’ AI Analyst, RARE is a Neutral.

Ultragenyx’s overall stock score is primarily impacted by its financial performance challenges, including ongoing losses and cash burn. Technical indicators suggest a bearish trend, and valuation metrics are weak due to a negative P/E ratio. Despite these challenges, the earnings call provided some optimism with strong revenue growth and clinical progress, slightly boosting the overall score.

To see Spark’s full report on RARE stock, click here.

More about Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Inc. operates in the biotechnology industry, focusing on developing novel products for the treatment of rare and ultra-rare genetic diseases. The company specializes in gene therapies and other biologics aimed at addressing unmet medical needs.

Average Trading Volume: 1,904,542

Technical Sentiment Signal: Sell

Current Market Cap: $2.86B

For a thorough assessment of RARE stock, go to TipRanks’ Stock Analysis page.

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