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Ultragenyx Gains FDA Priority Review for DTX401 Therapy

Tipranks - Tue Feb 24, 8:20AM CST

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The latest announcement is out from Ultragenyx Pharmaceutical ( (RARE) ).

On February 23, 2026, Ultragenyx Pharmaceutical announced that the U.S. Food and Drug Administration accepted for review its Biologics License Application for DTX401, an AAV gene therapy, for the treatment of Glycogen Storage Disease Type Ia. The FDA granted Priority Review status and set a Prescription Drug User Fee Act action date of August 23, 2026, potentially accelerating time to market and reinforcing Ultragenyx’s role as a leading player in rare disease gene therapies.

The most recent analyst rating on (RARE) stock is a Sell with a $19.50 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.

The score is primarily constrained by weak financial performance—large ongoing losses, substantial cash burn, and sharply reduced equity—despite strong revenue growth and gross margins. Technicals add pressure with a clear downtrend versus all major moving averages, partially tempered by near-oversold momentum. The earnings call improves the outlook with credible cost actions and growth guidance, but regulatory and execution risks remain significant.

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

More about Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing novel therapies for rare and ultra-rare genetic diseases. The company’s pipeline includes advanced gene therapies targeting serious metabolic and inherited disorders with limited or no existing treatment options, positioning it within the high-growth rare disease therapeutics market.

Average Trading Volume: 2,717,706

Technical Sentiment Signal: Sell

Current Market Cap: $1.97B

For detailed information about RARE stock, go to TipRanks’ Stock Analysis page.

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