Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biotechnology company with over $590 million in trailing twelve-month revenue, announced Friday that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for UX111 (ABO-102), an AAV gene therapy intended for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). According to InvestingPro data, the company's stock has fallen over 22% in the past week following this development.
According to a press release statement included in the company's SEC filing, the FDA has requested additional information and improvements related to certain chemistry, manufacturing, and controls (CMC) aspects, as well as observations from recently completed manufacturing facility inspections. Ultragenyx stated that it believes these observations are related to facilities and processes and not directly to the quality of the product.
The company plans to work with the FDA over the coming months to address the observations. Once resolved, Ultragenyx expects to resubmit the BLA and anticipates a review period of up to six months following the resubmission. While the stock is currently trading near its 52-week low, InvestingPro analysis suggests the company is undervalued, with analysts maintaining a bullish consensus.
The filing also noted that the FDA's clinical review is ongoing. The agency acknowledged that the neurodevelopmental outcome data provided are robust and that biomarker data offer additional supportive evidence. The CRL did not cite any issues related to the clinical data package or clinical inspections but requested that updated clinical data from current patients be included in the resubmission.
Ultragenyx is incorporated in Delaware and its common stock is listed on the Nasdaq Global Select Market under the symbol RARE.
This article is based on a statement from the company's SEC filing on Friday.
In other recent news, Ultragenyx Pharmaceutical Inc. has faced a regulatory setback as the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter for its gene therapy UX111, intended for treating Sanfilippo syndrome type A. The FDA requested additional information related to chemistry, manufacturing, and controls, which the company believes are addressable. Ultragenyx plans to work with the FDA to resolve these issues and resubmit the Biologics License Application, with a potential approval delay until 2026. On the analyst front, TD Cowen reiterated its Buy rating on Ultragenyx despite disappointing interim results for its setrusumab treatment, emphasizing a strong chance of success in the final analysis. Morgan Stanley also maintained an Overweight rating, highlighting the positive safety profile of setrusumab in ongoing trials. However, Wells Fargo lowered its price target for Ultragenyx to $65, citing delays in the Phase 3 Orbit trial but remained optimistic about the drug's potential. These developments reflect ongoing challenges and opportunities for Ultragenyx in its pursuit of advancing its treatment pipeline.