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Ultragenyx Receives FDA Breakthrough Therapy Designation

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Ultragenyx Receives FDA Breakthrough Therapy Designation

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An update from Ultragenyx Pharmaceutical ( (RARE) ) is now available.

On June 27, 2025, Ultragenyx Pharmaceutical announced that it received Breakthrough Therapy Designation from the FDA for GTX-102 as a treatment for Angelman syndrome. This designation is based on positive data from a Phase 1/2 study showing significant developmental improvements in patients, potentially expediting the drug’s development and review process.

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 Pharmaceutical’s stock score is primarily impacted by its financial performance challenges, with persistent profitability issues and cash flow concerns. While there is positive momentum in revenue growth and clinical progress, these factors are overshadowed by ongoing losses and financial instability. Technical indicators and valuation further highlight the mixed outlook, while the earnings call provides some optimism for long-term profitability.

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 the development of novel products for the treatment of rare and ultra-rare genetic diseases.

Average Trading Volume: 1,018,021

Technical Sentiment Signal: Sell

Current Market Cap: $3.54B

See more insights into RARE stock on TipRanks’ Stock Analysis page.

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