Ultragenyx Receives FDA Response on UX111 Application

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

On July 11, 2025, Ultragenyx Pharmaceutical announced that the FDA issued a Complete Response Letter for its Biologics License Application for UX111 gene therapy, intended for treating Sanfilippo syndrome type A. The FDA requested additional information and improvements related to chemistry, manufacturing, and controls, as well as observations from facility inspections. Ultragenyx plans to address these issues and resubmit the application, expecting a six-month review period post-resubmission. The clinical data was acknowledged as robust, with no issues noted, and updated clinical data will be included in the resubmission.

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 reflects strong revenue growth and positive corporate events, tempered by significant profitability and cash flow challenges. The company’s valuation metrics are unattractive due to negative earnings, and technical indicators suggest bearish momentum. Nonetheless, the positive outlook from earnings calls and recent corporate advancements offer a degree of optimism for the future.

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

More about Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Inc. operates in the biopharmaceutical industry, focusing on developing novel products for the treatment of rare and ultra-rare genetic diseases. The company’s primary products include gene therapies and other biologics aimed at addressing unmet medical needs in niche markets.

Average Trading Volume: 1,269,241

Technical Sentiment Signal: Sell

Current Market Cap: $3.92B

Learn more about RARE stock on TipRanks’ Stock Analysis page.