\nVutrisiran is the generic name for AMVUTTRA\u00ae<\/sup>, which is currently approved by the FDA for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. By rapidly knocking down both mutant and wild\u2011type transthyretin (TTR), vutrisiran addresses the underlying cause of transthyretin amyloidosis (ATTR). If approved, vutrisiran would become the first therapeutic approved in the U.S. to treat both the polyneuropathy manifestations of hATTR and cardiomyopathy manifestations of ATTR amyloidosis.<\/p>\n \n\u201cWe are pleased that the FDA has accepted our sNDA for vutrisiran for the treatment of ATTR with cardiomyopathy \u2013 a steadily progressing, debilitating and ultimately fatal disease,\u201d said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. \u201cIn HELIOS-B, treatment with vutrisiran improved cardiovascular outcomes, survival, disease progression and quality of life, as compared to placebo, in a population reflective of today\u2019s patients on substantial background treatment. We look forward to working with the FDA to support their review of the application and bring vutrisiran to patients with ATTR-CM in the U.S. early next year.\u201d<\/p>\n \nThe supplemental application to the FDA was based on positive results from HELIOS-B, a randomized, double-blind, placebo-controlled multicenter global Phase 3 study in patients with ATTR-CM. The study demonstrated favorable effects of vutrisiran on outcomes of death and cardiovascular events, functional capacity and quality of life in patients with ATTR-CM. These treatment effects were seen on top of substantial background standard of care treatments. Vutrisiran demonstrated encouraging safety and tolerability consistent with the established profile of the drug. Detailed results from the study were presented<\/a> at the European Society of Cardiology Congress and simultaneously published in The New England Journal of Medicine<\/i><\/a> on August 30, 2024.<\/p>\n AMVUTTRA\u00ae <\/sup>(vutrisiran) U.S. Indication and Important Safety Information<\/b><\/p>\n Indication Important Safety Information<\/b><\/p>\n Reduced Serum Vitamin A Levels and Recommended Supplementation \nPatients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).<\/p>\n Adverse Reactions For additional information about AMVUTTRA, please see the full U.S. <\/b>Prescribing Information<\/b><\/a>.<\/b><\/p>\n About AMVUTTRA\u00ae<\/sup> (vutrisiran) About ATTR About RNAi About Alnylam Pharmaceuticals Alnylam Forward-Looking Statements View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20241125980610\/en\/<\/a><\/span><\/p>\n Alnylam Pharmaceuticals, Inc.<\/b><\/p>\n \nChristine Regan Lindenboom \nJosh Brodsky KEYWORDS:<\/b> United States North America Massachusetts<\/p>\n INDUSTRY KEYWORDS:<\/b> Science Cardiology Biotechnology Research Pharmaceutical Health FDA Clinical Trials<\/p>\n
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<\/b>AMVUTTRA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.<\/p>\n
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<\/b>AMVUTTRA treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body.<\/p>\n
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<\/b>The most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%).<\/p>\n
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<\/b>AMVUTTRA\u00ae<\/sup> (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of mutant and wild-type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, AMVUTTRA is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Vutrisiran is also in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), which encompasses both wild-type and hereditary forms of the disease. For more information about AMVUTTRA, including the full U.S. Prescribing Information<\/a>, visit AMVUTTRA.com<\/a>.<\/p>\n
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<\/b>Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR \u2013 hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 \u2013 300,000 people worldwide.<\/p>\n
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<\/b>RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as \u201ca major scientific breakthrough that happens once every decade or so,\u201d and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam\u2019s RNAi therapeutic platform, function upstream of today\u2019s medicines by potently silencing messenger RNA (mRNA) \u2013 the genetic precursors that encode for disease-causing or disease pathway proteins \u2013 thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.<\/p>\n
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<\/b>Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam\u2019s commercial RNAi therapeutic products are ONPATTRO\u00ae (patisiran), AMVUTTRA\u00ae (vutrisiran), GIVLAARI\u00ae (givosiran), OXLUMO\u00ae (lumasiran), and Leqvio\u00ae (inclisiran), which is being developed and commercialized by Alnylam\u2019s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its \u201cAlnylam P5<\/sup>x25<\/i>\u201d strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com<\/a> and engage with us on X (formerly Twitter) at @Alnylam<\/a>, or on LinkedIn<\/a>, Facebook<\/a>, or Instagram<\/a>.<\/p>\n
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<\/b>This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam\u2019s expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam\u2019s expectations regarding the safety and efficacy of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy, including the safety, tolerability and favorable effects of vutrisiran on outcomes of death and cardiovascular events, functional capacity and quality of life in patients with ATTR-CM; whether the FDA will convene an advisory committee in connection with the FDA\u2019s review of Alnylam\u2019s supplemental application for approval of vutrisiran for the treatment of patients with ATTR-CM; the potential for vutrisiran to obtain FDA approval for the treatment of patients with ATTR-CM, and the anticipated timing of such FDA approval; and the potential for Alnylam to achieve its Alnylam<\/i>P5<\/sup>x25 <\/i>vision of becoming a leading biopharma company should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam\u2019s ability to successfully execute on its \u201cAlnylam P5<\/sup>x25<\/i>\u201d strategy; Alnylam\u2019s ability to successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam\u2019s product candidates, including vutrisiran; actions or advice of regulatory agencies and Alnylam\u2019s ability to obtain regulatory approval for its product candidates, including vutrisiran, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam\u2019s approved products globally; and any delays, interruptions or failures in the manufacture and supply of Alnylam\u2019s product candidates or its marketed products; as well as those risks more fully discussed in the \u201cRisk Factors\u201d filed with Alnylam\u2019s 2023 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam\u2019s subsequent Quarterly Reports on Form 10-Q and in its other SEC filings. In addition, any forward-looking statements represent Alnylam\u2019s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.<\/p>\n<\/p>\n
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