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CAMBRIDGE, Mass. - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), a biotechnology company with a market capitalization of $34.89 billion and impressive revenue growth of 23% over the last twelve months, has announced the U.S. Food and Drug Administration’s approval of Qfitlia™ (fitusiran), marking it as the first and only treatment designed to lower antithrombin for hemophilia A or B patients, regardless of inhibitors presence. This regulatory milestone represents the sixth RNAi therapeutic from Alnylam to receive FDA approval. According to InvestingPro analysis, the company maintains a strong financial health score and has delivered an exceptional 81.34% return over the past year.
Qfitlia is approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 and older. The therapeutic is based on RNA interference (RNAi), a natural cellular process of gene silencing, which has been a significant scientific advancement in biology and drug development. With an industry-leading gross profit margin of 85.62%, Alnylam’s business model demonstrates strong operational efficiency in bringing innovative treatments to market.
Clinical studies have indicated that Qfitlia can reduce annualized bleeding rates by 90% through its unique mechanism of action. The treatment, administered via subcutaneous injection once every two months, has shown efficacy in hemophilia A or B patients with or without inhibitors.
The approval of Qfitlia is a key achievement in Alnylam’s P5x25 strategy, aimed at developing and achieving regulatory milestones for its pipeline of RNAi therapeutics by 2025. Alnylam’s CEO, Yvonne Greenstreet, MBChB, expressed optimism about Qfitlia’s potential to transform the treatment landscape for the estimated one million people worldwide living with hemophilia.
Alnylam’s RNAi therapeutic platform operates by silencing messenger RNA that encodes disease-causing proteins, thus preventing their production. This innovative approach has led to the development of several commercial RNAi therapeutic products, with Qfitlia being the latest addition.
Regulatory submissions for Qfitlia have also been completed in other markets, including China and Brazil, expanding its potential global reach. Under a license and collaboration agreement with Sanofi, amended in 2018, Alnylam is set to receive tiered royalties of 15 to 30 percent on global net sales of Qfitlia.
This announcement is based on a press release statement from Alnylam Pharmaceuticals, Inc. and does not include any speculative information or endorsement of the product’s claims. The FDA’s approval of Qfitlia is a factual development in the field of RNAi therapeutics and hemophilia treatment.
In other recent news, Alnylam Pharmaceuticals has been the focus of several analyst reports following the FDA approval of its drug, Amvuttra. RBC Capital Markets maintained an Outperform rating with a $330 price target, citing confidence in the drug’s rapid market uptake and its potential to become the dominant treatment in the second-line setting. H.C. Wainwright also upheld a Buy rating with a $500 target, emphasizing the FDA’s approval as a pivotal moment for Alnylam and highlighting the drug’s promising results from the HELIOS-B study. JPMorgan upgraded Alnylam from Neutral to Overweight, raising its price target to $328, reflecting optimism about Amvuttra’s market potential and Alnylam’s broad platform.
Bernstein maintained an Outperform rating with a $305 target, expressing confidence in Alnylam’s future performance despite skepticism from some experts about the drug’s superiority over existing treatments. Stifel also kept a Buy rating with a $300 target, supporting the company’s pricing strategy for Amvuttra and noting the inclusion of mortality data in the drug’s label as a positive differentiator. The analysts from these firms suggest a strong outlook for Alnylam’s market presence and its potential to capture a significant share of the transthyretin-mediated cardiomyopathy market. These developments come as Alnylam guides towards profitability, with analysts highlighting the company’s strategic approach and robust clinical evidence backing its products.
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