CAMBRIDGE, Mass. - Alnylam Pharmaceuticals , Inc. (NASDAQ:ALNY) has announced the U.S. Food and Drug Administration's (FDA) acceptance of its supplemental New Drug Application (sNDA) for vutrisiran. The FDA will review the application for vutrisiran's potential use in treating ATTR amyloidosis with cardiomyopathy (ATTR-CM), setting a Prescription Drug User Fee Act (PDUFA) action date of March 23, 2025.
Vutrisiran, currently marketed under the brand name AMVUTTRA® for a different indication, aims to treat both the polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis. Alnylam's Chief Medical (TASE:PMCN) Officer, Dr. Pushkal Garg, expressed optimism about the FDA's acceptance, citing the drug's improved cardiovascular outcomes and quality of life in patients from the HELIOS-B study.
The HELIOS-B study, a Phase 3 clinical trial, showed positive effects of vutrisiran on patient survival, disease progression, and quality of life. These findings were presented at the European Society of Cardiology Congress and published in The New England Journal of Medicine on August 30, 2024.
ATTR amyloidosis is a fatal disease characterized by the accumulation of misfolded transthyretin proteins in the body, leading to nerve, heart, and gastrointestinal tract damage. Vutrisiran works by silencing the messenger RNA responsible for the disease-causing proteins.
Alnylam, a pioneer in RNA interference (RNAi) therapeutics, has been at the forefront of translating RNAi into new treatments for diseases with unmet needs. The company's portfolio includes a range of RNAi therapeutics, with vutrisiran being part of its late-stage development pipeline.
The information in this article is based on a press release statement from Alnylam Pharmaceuticals, Inc.
In other recent news, Alnylam Pharmaceuticals reported a substantial 34% year-over-year increase in global net product revenue, totaling $420 million, primarily driven by its transthyretin amyloidosis (ATTR) treatments. The company also revealed promising data from a Phase 1 study of nucresiran, a potential treatment for ATTR, showing a significant and sustained reduction in serum transthyretin levels. This led H.C. Wainwright and TD Cowen to maintain their Buy ratings on Alnylam, expressing confidence in the drug's development trajectory.
However, Wolfe Research downgraded Alnylam's stock from Peer Perform to Underperform due to long-term value concerns, particularly around the company's product Amvuttra. Despite this, Alnylam continues to advance its pipeline, notably in treatments for Alzheimer's and Huntington's diseases. The company aims to double its clinical development pipeline by the end of 2025.
In a shift of resources, Alnylam has ceased the ALN-KHK program for type 2 diabetes but remains open to business development opportunities. These are among the recent developments at Alnylam Pharmaceuticals.
InvestingPro Insights
Alnylam Pharmaceuticals' (NASDAQ:ALNY) recent FDA acceptance for vutrisiran's sNDA aligns with the company's strong market position and growth trajectory. According to InvestingPro data, Alnylam boasts a substantial market capitalization of $31.59 billion, reflecting investor confidence in its innovative RNA interference therapeutics pipeline.
The company's revenue growth of 21.54% over the last twelve months underscores its ability to commercialize its products effectively. This growth is particularly impressive given the competitive landscape of the pharmaceutical industry and supports the potential success of vutrisiran if approved for ATTR-CM.
InvestingPro Tips highlight that Alnylam has seen a strong return over the last year, with a one-year price total return of 50.31%. This performance suggests that investors are optimistic about the company's future prospects, including the potential approval of vutrisiran for its expanded indication.
It's worth noting that while Alnylam shows promise, it currently operates at a loss, with a negative P/E ratio of -95.44. However, this is not uncommon for biotech companies investing heavily in research and development. The company's liquid assets exceeding short-term obligations indicate a stable financial position to support ongoing clinical trials and potential product launches.
For investors seeking a more comprehensive analysis, InvestingPro offers 10 additional tips for Alnylam Pharmaceuticals, providing deeper insights into the company's financial health and market position.
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