Latest news with #ATTR
Yahoo
12-04-2025
- Business
- Yahoo
Intellia Focuses on Pipeline Development Amid Stiff Competition
Intellia Therapeutics, Inc. NTLA is developing its lead CRISPR-based, in vivo genome-editing candidate, nexiguran ziclumeran (nex-z, formerly known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis. Intellia has collaborated with Regeneron Pharmaceuticals REGN for the development of nex-z. Nex-z is part of the company's co-development and co-promotion agreement with Regeneron. While NTLA is the lead party in the deal for nex-z, REGN shares 25% of the development costs and commercial profits. Two separate phase III studies are evaluating nex-z for treating transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM) and hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). The phase III MAGNITUDE study is evaluating the safety and efficacy of nex-z in patients with ATTR-CM. If the data from the MAGNITUDE study is found to be positive, it will enable global regulatory filings for nex-z. The phase III MAGNITUDE 2 study is actively screening patients with ATTRv-PN, with the first patient expected to be dosed shortly. The collaboration with Regeneron for nex-z is a boost for Intellia as it provides the latter with resources to support the development of the candidate. Intellia is developing its wholly owned CRISPR-based pipeline candidate, NTLA-2002, for the treatment of hereditary angioedema ('HAE'). The pivotal phase III HAELO study is actively enrolling patients evaluating NTLA-2002 for treating HAE. Intellia dosed the first patient in the HAELO study in January 2025. Enrollment in the same is likely to be completed in the second half of 2025. The company plans to submit a potential biologics license application for NTLA-2002 in HAE in the second half of 2026. In January 2025, the company announced a strategic reorganization to prioritize its portfolio of late-stage pipeline candidates. As part of the portfolio reorganization, NTLA is planning to prioritize the development of nex-z and NTLA-2002. The company will also reduce its workforce by 27% in 2025. Intellia is gearing up to transition from a late-stage development company to a commercial-ready organization by the end of 2026. The encouraging pipeline progress with nex-z and NTLA-2002 should maintain the momentum for Intellia in 2025. Intellia's pipeline candidates are still in early-to-mid-stage development and some years away from commercialization. Upon successful development and potential approval, the candidates are likely to face stiff competition from companies that are also using the CRISPR/Cas9 gene editing technology to address various diseases in specific areas. The FDA approval of Vertex Pharmaceuticals VRTX and CRISPR Therapeutics' CRSP CRISPR/Cas9 genome-edited cell therapy, Casgevy, for the treatment of sickle cell disease has put the limelight on the gene editing space. The approval for Vertex and CRSP's Casgevy was a breakthrough for medical science, as it was the first for a CRISPR-based gene-editing therapy in the world. VRTX leads the global development and commercialization of Casgevy under the terms of the 2021 agreement, with support from CRISPR Therapeutics. Meanwhile, owing to the latest portfolio reorganization, Intellia has decided to stop the development of its in vivo gene insertion candidate, NTLA-3001, for the treatment of alpha-1 antitrypsin deficiency-associated lung disease. In the absence of a marketed product, the successful development of its pipeline candidates remains a key focus area for Intellia. Failure in the ongoing studies will be a big setback for the company. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Regeneron Pharmaceuticals, Inc. (REGN) : Free Stock Analysis Report Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report Intellia Therapeutics, Inc. (NTLA) : Free Stock Analysis Report CRISPR Therapeutics AG (CRSP) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Sign in to access your portfolio
Yahoo
03-04-2025
- Business
- Yahoo
Intellia Therapeutics Announces First Patient Dosed in the MAGNITUDE-2 Phase 3 Study of Nexiguran Ziclumeran (nex-z), a One-Time Gene Editing-Based Treatment for Transthyretin (ATTR) Amyloidosis with Polyneuropathy
CAMBRIDGE, Mass., April 03, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the first patient has been dosed in MAGNITUDE-2, a global, pivotal Phase 3 trial of nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). 'We are pleased to have dosed the first patient with a treatment that has such strong potential to redefine the treatment paradigm for those living with ATTR with polyneuropathy. This is a debilitating, progressive disease that leaves people feeling increasingly helpless,' said Dr. Paulo Sgobbi, Medical Director, PSEG Clinical Research Center. 'Through nex-z's potential to favorably impact disease progression, patients living with ATTR polyneuropathy could experience life-changing benefit while being freed from the existing chronic treatment regimen of pills, injections and infusions.' 'This milestone marks important progress toward our goal of completing the MAGNITUDE-2 clinical program and we are optimistic the study will enable us to demonstrate nex-z's potential to be the first to halt or reverse disease progression with a single dose in hereditary ATTR with polyneuropathy,' said Intellia President and Chief Executive Officer John Leonard, M.D. The Phase 3 MAGNITUDE-2 study is informed by Intellia's Phase 1 data, showing that a single dose of nex-z led to consistently rapid, deep and durable reduction in serum TTR. Intellia expects to present longer-term data from the Phase 1 studies of nex-z for both polyneuropathy and cardiomyopathy later this year. The company plans to submit a biologics licensing application (BLA) for ATTRv-PN by 2028. About the MAGNITUDE-2 StudyThe pivotal Phase 3 MAGNITUDE-2 clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of nexiguran ziclumeran (nex-z) in approximately 50 patients with transthyretin amyloidosis with polyneuropathy (ATTRv-PN). The primary endpoint of the study includes a modified neuropathy impairment score and change in serum TTR levels. Adult patients with ATTRv-PN will be randomized 1:1 to receive a single 55 mg infusion of nex-z or placebo. For more information on MAGNITUDE-2 (NCT06672237), please visit About nexiguran ziclumeran (nex-z, formerly known as NTLA-2001)Based on Nobel Prize-winning CRISPR/Cas9 technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene that encodes for the mutated transthyretin (TTR) protein causing the polyneuropathy. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Nex-z has been granted Regenerative Medicine Advanced Therapy designations by the U.S. FDA for both cardiomyopathy and polyneuropathy. Nex-z has also been granted Orphan Drug Designation by the U.S. FDA and European Commission. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron. About Transthyretin (ATTR) AmyloidosisTransthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without the genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis. There is no known cure for ATTR amyloidosis and currently available medications are limited to slowing accumulation of misfolded TTR protein. About Intellia TherapeuticsIntellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. Intellia's deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at and follow us @intelliatx. Forward-Looking Statements This press release contains 'forward-looking statements' of Intellia Therapeutics, Inc. ('Intellia' or the 'Company') within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia's beliefs and expectations concerning: its ability to successfully develop and commercialize nexiguran ziclumeran ('nex-z'), formerly known as NTLA-2001, for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN); the planned release of longer-term data from the Phase 1 studies of nex-z for both polyneuropathy and cardiomyopathy in 2025; its planned submission of a biologics license application ('BLA') for ATTRv-PN by 2028; the potential of nex-z to be a single-dose treatment and the first treatment to halt or reverse disease progression in ATTRv-PN and for patients to experience life-changing benefit while being freed from chronic treatment; its ability to optimize the impact of its collaborations on its development programs, including its collaboration with Regeneron Pharmaceuticals, Inc. and their co-development program for ATTR amyloidosis. Any forward-looking statements in this press release are based on management's current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to Intellia's relationship with third parties, including its contract manufacturers, licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of clinical studies and other development requirements for its product candidates, including nex-z, including uncertainties related to regulatory approvals to conduct clinical trials; risks related to the ability to develop and commercialize nex-z successfully; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; risks related to Intellia's future financial condition and its ability to fund its operations; and risks related to Intellia's collaboration with Regeneron Pharmaceuticals, Inc. not continuing or not being successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia's actual results to differ from those contained in the forward-looking statements, see the section entitled 'Risk Factors' in Intellia's most recent annual report on Form 10-K and quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in Intellia's other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by ChavesSenior Manager, Investor Media:Matt CrensonTen Bridge Communicationsmedia@ mcrenson@ in to access your portfolio
Yahoo
24-03-2025
- Health
- Yahoo
FDA approves new drug to treat protein buildup in the heart
The U.S. Food and Drug Administration has approved a new drug for a serious heart condition that affects thousands of people. The drug, called Amvuttra (vutrisiran), is made by Alnylam Pharmaceuticals Inc. and is used to treat transthyretin amyloid cardiomyopathy. ATTR-CM is a disease in which harmful proteins build up in the heart, making it harder to pump blood, according to the American Heart Association. In one study, Amvuttra helped lower the risk of death and heart problems by 28% over three years compared to a placebo. The drug is given as a shot every three months. "We hope this could become our flagship franchise. We're going to help thousands more patients," Alnylam CEO Yvonne Greenstreet said in an interview with STAT News. "It's also a transformational moment for the company." But the treatment comes at a steep price. Each dose costs about $119,000, or $476,000 per year. That's more than double the cost of a similar drug from BridgeBio, and more expensive than Pfizer's pills for the same condition. "There are many, many, many undiagnosed patients and substantial unmet need in this space. This market is very large, underdiagnosed, undertreated," said Dr. Mani Foroohar, an analyst with the health care investment company Leerink. Still, the high cost may make it harder for patients -- especially those on Medicare Advantage plans -- to get the drug. Private insurers may prefer cheaper options like pills from Pfizer or BridgeBio. Amvuttra works differently from those drugs. It uses RNA technology to stop the body from making the bad protein, while the other treatments work to stabilize the protein before it causes harm. In trials, BridgeBio's treatment showed a 42% drop in deaths and heart hospital visits over 30 months. Amvuttra's study showed a 28% drop over three years. But the trials were different, so it's hard to compare the results directly. Amvuttra is already approved to treat a nerve-related form of ATTR. With this new approval, Alnylam hopes the drug will reach many more people. More information Learn more about vutrisiran. Copyright © 2025 HealthDay. All rights reserved.
Yahoo
20-03-2025
- Business
- Yahoo
Alnylam Announces FDA Approval of AMVUTTRA® (vutrisiran), the First RNAi Therapeutic to Reduce Cardiovascular Death, Hospitalizations and Urgent Heart Failure Visits in Adults with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)
− Novel Mechanism of Action Delivers Rapid Knockdown of Transthyretin, Addressing the Disease at its Source – − Proven Consistency of Effect on Cardiovascular Outcomes, Function, and Quality of Life in ATTR-CM Population Representative of Today's Patients – – Only Therapeutic Approved in the U.S. to Address Both Cardiomyopathy and Polyneuropathy Manifestations of ATTR Amyloidosis − – Alnylam Offers Multiple Programs to Support Broad and Seamless Patient Access; Majority of Patients Expected to Pay $0 in Out-of-Pocket Costs for AMVUTTRA – – Alnylam to Host Conference Call Today at 6:00 pm ET – CAMBRIDGE, Mass., March 20, 2025--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the U.S. Food and Drug Administration (FDA) approval of the supplemental New Drug Application (sNDA) for its RNAi therapeutic, AMVUTTRA® (vutrisiran), for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. The approval expands the indication for AMVUTTRA, which now becomes the first and only therapeutic approved by the FDA for the treatment of ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. ATTR-CM is a devastating, rapidly progressive, and ultimately fatal disease estimated to affect approximately 150,000 people in the U.S. and over 300,000 people worldwide. There is no cure for ATTR-CM, and the deposition of misfolded transthyretin (TTR) fibrils causes irreversible damage over time which can lead to premature death. Today, most patients remain undiagnosed and untreated, and a significant portion of those who are treated with commonly used therapies experience disease progression. "The FDA approval of AMVUTTRA for ATTR-CM marks a pivotal advancement for patients, providing a new and clinically differentiated treatment option that has been shown to improve outcomes, including cardiovascular mortality, and reduce progression for those living with this devastating disease," said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. "I would like to extend my deepest gratitude to the patients who participated in our clinical trials, their families and caregivers, the clinical researchers, regulators, and my colleagues at Alnylam who made this approval possible. Today represents a significant milestone in our nearly twenty years of partnership with the ATTR amyloidosis community, but we are not stopping here. We will continue to innovate for patients with ATTR amyloidosis so they can live longer, better, healthier lives." AMVUTTRA is an RNAi therapeutic that works upstream to deliver rapid knockdown of TTR, addressing the disease at its source, with only four convenient subcutaneous doses per year. By rapidly knocking down TTR production, AMVUTTRA substantially decreases deposition of TTR fibrils, which form amyloid and cause irreversible cardiovascular damage and premature death in patients with ATTR-CM. "This FDA approval provides an opportunity to further transform ATTR-CM treatment with a new mechanism of action. The HELIOS-B clinical trial found that vutrisiran allowed patients to live longer, experience fewer hospitalizations, and improve how they function and feel," said Ronald Witteles, M.D., HELIOS-B Investigator, Professor of Medicine at Stanford University School of Medicine and Co-Director of the Stanford Amyloid Center. "The trial enrolled patients who mirror the real-world population with this disease, and I am very encouraged by vutrisiran's ability to demonstrate meaningful clinical benefits across both cardiovascular outcomes and multiple measures of disease progression. This is a very exciting day for patients with this challenging disease." This approval is based on the HELIOS-B Phase 3 clinical trial which evaluated AMVUTTRA for the treatment of ATTR-CM. The trial achieved statistical significance compared to placebo on all 10 pre-specified primary and secondary endpoints. The results were presented at the European Society of Cardiology Congress and simultaneously published in The New England Journal of Medicine. In the overall population, AMVUTTRA reduced the risk of all-cause mortality (ACM) and recurrent cardiovascular (CV) events by 28% during the double-blind treatment period of up to 36 months. Mortality in this population was significantly reduced by 36% through 42 months in a pre-specified secondary endpoint analysis which included up to 36 months of the double-blind period plus six months of open-label extension. In the monotherapy population, AMVUTTRA significantly reduced the risk of ACM and recurrent CV events by 33% in the double-blind period and significantly reduced the risk of mortality by 35% through 42 months. As compared to patients treated with placebo, patients treated with AMVUTTRA also experienced preservation of functional capacity and quality of life, as well as early improvements in biomarkers NT-proBNP and troponin I, which are predictive of cardiovascular outcomes. The safety and tolerability of AMVUTTRA are well-established, as demonstrated in the positive HELIOS-A clinical trial for AMVUTTRA in hATTR-PN which resulted in FDA approval in 2022 and over 5,000 patient-years exposure to-date, globally. In that study, the most common adverse reactions in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%). No new safety concerns were identified in the HELIOS-B clinical trial of patients with ATTR-CM. "Despite recent advances, there remains a significant need for patients living with ATTR-CM and I've witnessed, firsthand, the impact that ATTR amyloidosis can have on families, including diminished quality of life and the loss of loved ones," said Muriel Finkel, President of the Amyloidosis Support Groups. "The availability of this groundbreaking treatment option is a significant moment for patients living with ATTR amyloidosis. It represents a beacon of hope for our community." AMVUTTRA in hATTR-PN is covered by insurers for ~99% of patients with the majority paying $0 out-of-pocket. Similar broad coverage and out-of-pocket costs are expected in ATTR-CM given comparable payer dynamics and the clinical value demonstrated in the HELIOS-B clinical trial. Alnylam has a proven track record of working closely with payers to ensure broad patient access to our medicines, including pioneering innovative value-based agreements linked to clinical outcomes that ensure value and predictability for payers. Alnylam offers multiple support services and resources for patients prescribed its products through Alnylam Assist®, the only in-house patient support program in ATTR-CM. Alnylam Assist® is designed to provide one-on-one support for patients and their healthcare teams to help navigate the treatment journey, and includes a Quick Start program that provides eligible patients who encounter coverage delays or delays in treatment with an initial dose at no cost. In addition, Alnylam Assist® offers support with insurance coverage, financial assistance, and disease and treatment education for a seamless start to the AMVUTTRA treatment experience. Physicians and patients can learn more about Alnylam's patient support services by visiting or calling 1-833-256-2748. Marketing authorization applications based on HELIOS-B data are currently under review by several global health agencies including the European Medicines Agency (EMA), the Brazilian Health Regulatory Agency (ANVISA), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025. Visit for more information. Investor Webcast Information Alnylam management will discuss the FDA approval of AMVUTTRA for ATTR-CM via webcast today at 6:00 pm ET. A live audio webcast of the call will be available on the Investors section of the Company's website at An archived webcast will be available on the Company's website approximately two hours after the event. INDICATIONS AND IMPORTANT SAFETY INFORMATION Indications AMVUTTRA® (vutrisiran) is indicated for the treatment of the: cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation 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. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse Reactions In a study of patients with hATTR-PN, 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%). In a study of patients with ATTR-CM, no new safety issues were identified. For additional information about AMVUTTRA, please see the full Prescribing Information. About AMVUTTRA® (vutrisiran) AMVUTTRA® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection by a healthcare professional, 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 and is approved in the U.S. for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit About ATTR 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 – 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 – 300,000 people worldwide.1-4 About RNAi 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 "a major scientific breakthrough that happens once every decade or so," 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's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, 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. About Alnylam Pharmaceuticals Alnylam Pharmaceuticals (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's commercial RNAi therapeutic products are ONPATTRO® (patisiran), AMVUTTRA® (vutrisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), and Leqvio® (inclisiran), which is being developed and commercialized by Alnylam's 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 "Alnylam P5x25" 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 and engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn, Facebook, or Instagram. Alnylam Forward-Looking Statements 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's expectations, beliefs, goals, plans or prospects, including, without limitation, statements regarding Alnylam's expectations regarding the safety and efficacy of AMVUTTRA for the treatment of ATTR-CM and hATTR-PN; that FDA approval of AMVUTTRA for ATTR-CM represents a pivotal advancement for patients, providing a new and clinically differentiated treatment option for ATTR-CM; the ability of AMVUTTRA to improve outcomes and reduce progression for patients with ATTR-CM; Alnylam's ability to continue to innovate for patients with ATTR amyloidosis; the ability of AMVUTTRA or any of Alnylam's other products or product candidates to enable patients with ATTR amyloidosis to live longer, better and/or healthier lives; the potential for AMVUTRA to transform the treatment of ATTR-CM; the ability of AMVUTTRA to provide ATTR-CM patients with benefits comparable to the clinical benefits observed in the HELIOS-B clinical trial; Alnylam's expectations that AMVUTTRA in ATTR-CM will have coverage and out-of-pocket cost dynamics similar to AMVUTTRA's coverage and out-of-pocket cost dynamics in hATTR-PN; and the potential receipt and timing of any future marketing authorizations or the timing of any future regulatory submissions for vutrisiran, 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's ability to successfully execute on its "Alnylam P5x25" goals; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam's product candidates; actions or advice of regulatory agencies and Alnylam's ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam's product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam's subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing Alnylam's views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. 1 Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638.2 Gertz MA. Am J Manag Care. 2017;23(7):S107-S112.3 Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9.4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31. View source version on Contacts Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom(Investors and Media)+1-617-682-4340Josh Brodsky(Investors)+1-617-551-8276 Sign in to access your portfolio
Associated Press
20-03-2025
- Business
- Associated Press
Alnylam Announces FDA Approval of AMVUTTRA® (vutrisiran), the First RNAi Therapeutic to Reduce Cardiovascular Death, Hospitalizations and Urgent Heart Failure Visits in Adults with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the U.S. Food and Drug Administration (FDA) approval of the supplemental New Drug Application (sNDA) for its RNAi therapeutic, AMVUTTRA ® (vutrisiran), for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. The approval expands the indication for AMVUTTRA, which now becomes the first and only therapeutic approved by the FDA for the treatment of ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. This press release features multimedia. View the full release here: AMVUTTRA® (vutrisiran) product logo ATTR-CM is a devastating, rapidly progressive, and ultimately fatal disease estimated to affect approximately 150,000 people in the U.S. and over 300,000 people worldwide. There is no cure for ATTR-CM, and the deposition of misfolded transthyretin (TTR) fibrils causes irreversible damage over time which can lead to premature death. Today, most patients remain undiagnosed and untreated, and a significant portion of those who are treated with commonly used therapies experience disease progression. 'The FDA approval of AMVUTTRA for ATTR-CM marks a pivotal advancement for patients, providing a new and clinically differentiated treatment option that has been shown to improve outcomes, including cardiovascular mortality, and reduce progression for those living with this devastating disease,' said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. 'I would like to extend my deepest gratitude to the patients who participated in our clinical trials, their families and caregivers, the clinical researchers, regulators, and my colleagues at Alnylam who made this approval possible. Today represents a significant milestone in our nearly twenty years of partnership with the ATTR amyloidosis community, but we are not stopping here. We will continue to innovate for patients with ATTR amyloidosis so they can live longer, better, healthier lives.' AMVUTTRA is an RNAi therapeutic that works upstream to deliver rapid knockdown of TTR, addressing the disease at its source, with only four convenient subcutaneous doses per year. By rapidly knocking down TTR production, AMVUTTRA substantially decreases deposition of TTR fibrils, which form amyloid and cause irreversible cardiovascular damage and premature death in patients with ATTR-CM. 'This FDA approval provides an opportunity to further transform ATTR-CM treatment with a new mechanism of action. The HELIOS-B clinical trial found that vutrisiran allowed patients to live longer, experience fewer hospitalizations, and improve how they function and feel,' said Ronald Witteles, M.D., HELIOS-B Investigator, Professor of Medicine at Stanford University School of Medicine and Co-Director of the Stanford Amyloid Center. 'The trial enrolled patients who mirror the real-world population with this disease, and I am very encouraged by vutrisiran's ability to demonstrate meaningful clinical benefits across both cardiovascular outcomes and multiple measures of disease progression. This is a very exciting day for patients with this challenging disease.' This approval is based on the HELIOS-B Phase 3 clinical trial which evaluated AMVUTTRA for the treatment of ATTR-CM. The trial achieved statistical significance compared to placebo on all 10 pre-specified primary and secondary endpoints. The results were presented at the European Society of Cardiology Congress and simultaneously published in The New England Journal of Medicine. In the overall population, AMVUTTRA reduced the risk of all-cause mortality (ACM) and recurrent cardiovascular (CV) events by 28% during the double-blind treatment period of up to 36 months. Mortality in this population was significantly reduced by 36% through 42 months in a pre-specified secondary endpoint analysis which included up to 36 months of the double-blind period plus six months of open-label extension. In the monotherapy population, AMVUTTRA significantly reduced the risk of ACM and recurrent CV events by 33% in the double-blind period and significantly reduced the risk of mortality by 35% through 42 months. As compared to patients treated with placebo, patients treated with AMVUTTRA also experienced preservation of functional capacity and quality of life, as well as early improvements in biomarkers NT-proBNP and troponin I, which are predictive of cardiovascular outcomes. The safety and tolerability of AMVUTTRA are well-established, as demonstrated in the positive HELIOS-A clinical trial for AMVUTTRA in hATTR-PN which resulted in FDA approval in 2022 and over 5,000 patient-years exposure to-date, globally. In that study, the most common adverse reactions in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%). No new safety concerns were identified in the HELIOS-B clinical trial of patients with ATTR-CM. 'Despite recent advances, there remains a significant need for patients living with ATTR-CM and I've witnessed, firsthand, the impact that ATTR amyloidosis can have on families, including diminished quality of life and the loss of loved ones,' said Muriel Finkel, President of the Amyloidosis Support Groups. 'The availability of this groundbreaking treatment option is a significant moment for patients living with ATTR amyloidosis. It represents a beacon of hope for our community.' AMVUTTRA in hATTR-PN is covered by insurers for ~99% of patients with the majority paying $0 out-of-pocket. Similar broad coverage and out-of-pocket costs are expected in ATTR-CM given comparable payer dynamics and the clinical value demonstrated in the HELIOS-B clinical trial. Alnylam has a proven track record of working closely with payers to ensure broad patient access to our medicines, including pioneering innovative value-based agreements linked to clinical outcomes that ensure value and predictability for payers. Alnylam offers multiple support services and resources for patients prescribed its products through Alnylam Assist ®, the only in-house patient support program in ATTR-CM. Alnylam Assist ® is designed to provide one-on-one support for patients and their healthcare teams to help navigate the treatment journey, and includes a Quick Start program that provides eligible patients who encounter coverage delays or delays in treatment with an initial dose at no cost. In addition, Alnylam Assist ® offers support with insurance coverage, financial assistance, and disease and treatment education for a seamless start to the AMVUTTRA treatment experience. Physicians and patients can learn more about Alnylam's patient support services by visiting or calling 1-833-256-2748. Marketing authorization applications based on HELIOS-B data are currently under review by several global health agencies including the European Medicines Agency (EMA), the Brazilian Health Regulatory Agency (ANVISA), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025. Visit for more information. Investor Webcast Information Alnylam management will discuss the FDA approval of AMVUTTRA for ATTR-CM via webcast today at 6:00 pm ET. A live audio webcast of the call will be available on the Investors section of the Company's website at An archived webcast will be available on the Company's website approximately two hours after the event. Indications AMVUTTRA ® (vutrisiran) is indicated for the treatment of the: cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation 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. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse Reactions In a study of patients with hATTR-PN, 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%). In a study of patients with ATTR-CM, no new safety issues were identified. For additional information about AMVUTTRA, please see the full Prescribing Information. About AMVUTTRA® (vutrisiran) AMVUTTRA ® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection by a healthcare professional, 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 and is approved in the U.S. for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit About ATTR 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 – 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 – 300,000 people worldwide. 1-4 About RNAi 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 'a major scientific breakthrough that happens once every decade or so,' 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's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, 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. About Alnylam Pharmaceuticals Alnylam Pharmaceuticals (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's commercial RNAi therapeutic products are ONPATTRO ® (patisiran), AMVUTTRA ® (vutrisiran), GIVLAARI ® (givosiran), OXLUMO ® (lumasiran), and Leqvio ® (inclisiran), which is being developed and commercialized by Alnylam's 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 'Alnylam P5x25' 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 and engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn, Facebook, or Instagram. Alnylam Forward-Looking Statements 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's expectations, beliefs, goals, plans or prospects, including, without limitation, statements regarding Alnylam's expectations regarding the safety and efficacy of AMVUTTRA for the treatment of ATTR-CM and hATTR-PN; that FDA approval of AMVUTTRA for ATTR-CM represents a pivotal advancement for patients, providing a new and clinically differentiated treatment option for ATTR-CM; the ability of AMVUTTRA to improve outcomes and reduce progression for patients with ATTR-CM; Alnylam's ability to continue to innovate for patients with ATTR amyloidosis; the ability of AMVUTTRA or any of Alnylam's other products or product candidates to enable patients with ATTR amyloidosis to live longer, better and/or healthier lives; the potential for AMVUTRA to transform the treatment of ATTR-CM; the ability of AMVUTTRA to provide ATTR-CM patients with benefits comparable to the clinical benefits observed in the HELIOS-B clinical trial; Alnylam's expectations that AMVUTTRA in ATTR-CM will have coverage and out-of-pocket cost dynamics similar to AMVUTTRA's coverage and out-of-pocket cost dynamics in hATTR-PN; and the potential receipt and timing of any future marketing authorizations or the timing of any future regulatory submissions for vutrisiran, 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's ability to successfully execute on its ' Alnylam P 5 x25 ' goals; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam's product candidates; actions or advice of regulatory agencies and Alnylam's ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam's product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the 'Risk Factors' filed with Alnylam's 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam's subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing Alnylam's views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. 1 Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638. 2 Gertz MA. Am J Manag Care. 2017;23(7):S107-S112. 3 Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9. 4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31. CONTACT: Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom (Investors and Media) +1-617-682-4340 Josh Brodsky (Investors) +1-617-551-8276 SOURCE: Alnylam Pharmaceuticals, Inc. Copyright Business Wire 2025. PUB: 03/20/2025 05:54 PM/DISC: 03/20/2025 05:53 PM
