Bank of America Securities Reiterated a Buy Rating on Ultragenyx Pharmaceutical (RARE)
The firm held its positive outlook even after the recent challenge from the FDA's complete response letter for UX111 in Sanfilippo syndrome. The CRL was focused on specific chemistry, manufacturing, and controls issues. Importantly, the clinical data for UX111 was not questioned; however, the approval is still achievable once the CMC issues are resolved.
A research team in a laboratory peering through microscopes at a biologic product.
The analyst noted that the company is responding quickly and plans to address the CMC concerns and resubmit its Biologics License Application in the coming months. The analyst is also optimistic due to Ultragenyx Pharmaceutical Inc.'s (NASDAQ:RARE) robust pipeline. The company has promising programs for conditions like osteogenesis imperfecta and Angelman syndrome.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops treatments for rare and ultra-rare genetic diseases. They focus on biologics, small molecules, gene therapy, and nucleic acid products.
While we acknowledge the potential of RARE as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock.
READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now.
Disclosure: None. This article is originally published at Insider Monkey.
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