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CAMBRIDGE, Mass. - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), a biotechnology company with a market capitalization of $32.57 billion and impressive gross margins of 85.62%, has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for vutrisiran, a treatment for adult patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This recommendation is a step towards potential European Commission approval expected in June 2025. According to InvestingPro data, the company’s strong financial health and robust pipeline position it well for future growth.
ATTR-CM, a condition characterized by the buildup of misfolded proteins in the heart, can lead to heart failure and death. Vutrisiran, which operates by reducing the production of transthyretin (TTR) proteins, is already approved in the EU for hereditary transthyretin-mediated amyloidosis with polyneuropathy. The treatment is administered subcutaneously every three months and could become the first RNAi therapeutic approved in Europe for ATTR-CM. With revenue growth of 22.97% in the last twelve months, Alnylam continues to demonstrate strong commercial execution.
The CHMP’s positive opinion is based on results from the HELIOS-B Phase 3 study, which met all primary and secondary endpoints, demonstrating survival benefits, fewer hospitalizations, and reduced disease progression in patients. The study’s findings were published in The New England Journal of Medicine.
According to Alnylam’s Chief Medical Officer, Dr. Pushkal Garg, the study results showed rapid TTR knockdown, improved survival, and less disease progression, with a safety profile characterized by injection site reactions and increases in certain liver enzymes. Vutrisiran has also been approved by the FDA and ANVISA in Brazil for ATTR-CM, with additional global regulatory submissions planned for 2025. The company’s stock has reflected this positive momentum, delivering a remarkable 75.54% return over the past year. For detailed analysis and more insights, visit InvestingPro, which offers comprehensive research reports on over 1,400 US stocks, including Alnylam Pharmaceuticals.
In Europe, vutrisiran is indicated for hereditary ATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy, with a recommendation for vitamin A supplementation due to treatment-related decreases in serum vitamin A levels. Adverse reactions observed in clinical trials included pain in extremity, arthralgia, dyspnea, and injection site reactions.
Alnylam, a leader in RNA interference (RNAi) therapeutics, continues to pursue global approvals for vutrisiran as part of its strategy to provide innovative treatments for diseases with unmet needs. The information in this article is based on a press release statement from Alnylam Pharmaceuticals.
In other recent news, Alnylam Pharmaceuticals received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for its drug Amvuttra, expanding its use to treat wild-type and hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adults. This development could enhance Alnylam’s revenue streams and market presence in the European Union. Meanwhile, Canaccord Genuity maintained its Buy rating for Alnylam with a $390 price target, expressing confidence in Amvuttra’s revenue potential, particularly in treating ATTR-CM. H.C. Wainwright also reaffirmed a Buy rating with a $500 price target, highlighting the U.S. approval of AMVUTTRA as a financial turning point for the company. Conversely, Stifel lowered its price target for Alnylam to $300, though it kept a Buy rating, citing discussions about vutrisiran’s launch for ATTR-cardiomyopathy. Additionally, Scotiabank raised its price target to $342, maintaining a Sector Outperform rating, following U.S. approvals for Amvuttra and Qfitlia, the latter of which is expected to generate significant revenue by 2034. These recent developments indicate a dynamic period for Alnylam, driven by regulatory advancements and strategic analyst evaluations.
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