Vutrisiran Sodium(Vutrisiran Sodium) - 药物靶点：TTR_在研适应症：转甲状腺素蛋白淀粉样变性心肌病，转甲状腺素蛋白介导的淀粉样变性，遗传性转甲状腺素蛋白淀粉样变性_专利_临床

概要

基本信息

药物类型

siRNA

别名

Vutrisiran、AD-65492、ALN-65492

+ [4]

靶点

TTR

作用方式

抑制剂

作用机制

TTR抑制剂(转甲状腺素蛋白抑制剂)

治疗领域

神经系统疾病遗传病与畸形内分泌与代谢疾病+ [2]

在研适应症

转甲状腺素蛋白淀粉样变性心肌病转甲状腺素蛋白介导的淀粉样变性遗传性转甲状腺素蛋白淀粉样变性+ [3]

非在研适应症-

原研机构

Alnylam Pharmaceuticals, Inc.

在研机构

Alnylam Pharmaceuticals, Inc.Medison Pharma Australia Pty Ltd.Alnylam Netherlands BV

+ [2]

非在研机构-

权益机构

Alnylam Pharmaceuticals, Inc.

Blackstone Life Sciences, LLC

Genzyme Corp.

+ [1]

最高研发阶段批准上市

首次获批日期

美国 (2022-06-13),

遗传性转甲状腺素蛋白淀粉样变性

最高研发阶段(中国)-

特殊审评快速通道 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (韩国)、孤儿药 (澳大利亚)、孤儿药 (英国)、孤儿药 (日本)

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结构/序列

使用我们的RNA技术数据为新药研发加速。

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Sequence Code 30032027

来源: *****

Sequence Code 30033233

来源: *****

关联

5

项与 Vutrisiran Sodium 相关的临床试验

CTIS2024-518343-38-00

/ Recruiting临床3期

An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy) - ALN-TTRSC02-007

开始日期2025-02-21

申办/合作机构

Alnylam Pharmaceuticals, Inc.

NCT06679946

/ Enrolling by invitation临床3期

An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.

开始日期2024-12-03

申办/合作机构

Alnylam Pharmaceuticals, Inc.

NCT04153149

/ Active, not recruiting临床3期

HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR amyloidosis with cardiomyopathy.

开始日期2019-11-26

申办/合作机构

Alnylam Pharmaceuticals, Inc.

100 项与 Vutrisiran Sodium 相关的临床结果

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100 项与 Vutrisiran Sodium 相关的转化医学

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100 项与 Vutrisiran Sodium 相关的专利（医药）

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71

项与 Vutrisiran Sodium 相关的文献（医药）

2025-12-01·Current Cardiology Reports

Transthyretin Cardiac Amyloidosis: Current and Emerging Therapies

Review

作者: Kesari, Aditya ; Shah, Keyur B ; Li, Pengyang ; Badrish, Narotham ; Patel, Aditi G M

Abstract:

Purpose of Review:

In this article, we describe current and newer TTR stabilizers, TTR silencers which include small interfering RNA agents (siRNA), antisense oligonucleotides (ASO) and CRISPR-Cas9 gene editing, and TTR depleters, which investigates the use of monoclonal antibodies to remove amyloid fibril deposits for patients with advanced disease.

Recent Findings:

Once thought to be a rare and fatal condition, increased recognition, improved non-invasive diagnostic tools, and the explosive development of novel therapies, has transformed the landscape of transthyretin amyloid cardiomyopathy (ATTR-CM). Advances in cardiac imaging with respect to echocardiography, cardiac magnetic resonance imaging (CMR), and radionuclide bone scintigraphy has increased the diagnosis of ATTR-CM over the last twenty years. Ongoing clinical trials are evaluating several novel therapies at several mechanistic targets in the transthyretin (TTR) amyloidogenesis cascade, including the recently published findings from the study of vutrisiran, a siRNA agent.

Summary:

Our review provides a comprehensive summary of current and emerging therapies for ATTR-CM. While these are promising, disease-modifying treatments, reaching vulnerable populations early in the disease course should be a focus for future studies and interventions.

2025-12-01·BIOCHEMICAL PHARMACOLOGY

Intracellular fatty acid levels differentially impact target silencing by FDA-approved siRNA drugs

Article

作者: Jin, Jing ; Tawfik, Sherouk M ; Zhong, Xiao-Bo ; Giang Nguyen, Le Tra

With seven approved and many more anticipated over the next decade, small interfering RNA (siRNA) therapeutics have emerged as an innovative class of nucleic acid-based drugs, offering potential treatments for both rare and common diseases. A substantial portion of the patient population affected by these diseases may also present with obesity and metabolic-associated fatty liver disease (MAFLD), conditions characterized by excessive accumulation of hepatic free fatty acids (FFAs). However, the impact of intracellular FFA levels on siRNA drug efficacy has not been fully determined. In this study, hepatic HepG2 and HepaRG cells were treated with varying concentrations of oleic and palmitic acids to simulate a microcellular environment with elevated FFA levels. Efficacy in the reduction of targets at both the mRNA and protein levels was determined for three selected Food and Drug Administration (FDA)-approved siRNA drugs, patisiran, vutrisiran, and inclisiran. Our findings demonstrate strong evidence that elevated intracellular FFA levels significantly alter the efficacy of the FDA-approved siRNA drugs, impacting both mRNA and protein target reduction and highlighting a previously underexplored factor which could impact clinical outcomes. Understanding the impact on siRNA efficacy is critical for optimizing the therapeutic potential of siRNA-based treatments for patients with FFA diseases, such as obesity and MAFLD.

2025-12-01·MOLECULAR BIOLOGY REPORTS

Transthyretin as a therapeutic target: the future of disease-modifying therapies for Alzheimer’s disease

Review

作者: Shaw, Swetaleena ; Aran, Khadga Raj ; Porel, Pratyush

BACKGROUND:

Alzheimer's disease (AD) is the most common neurodegenerative disease for causing memory deficits and primarily characterized by extracellular deposition of amyloid-β (Aβ) plaques, intracellular neurofibrillary tangles (NFTs), and hyperphosphorylation of tau protein, all are pathological hallmarks for AD. Transthyretin (TTR) is a highly conserved homo-tetrameric protein, primarily synthesized in liver and choroid plexus, and most importantly involved in transport of T₃-T₄ hormones and retinol.

OBJECTIVES:

This review explores the dual role of TTR, with a greater emphasis on its neuroprotective action, particularly in AD.

METHODS:

Based on the available literature, TTR's potential as a biomarker in the central nervous system (CNS), focusing its role in stabilizing Aβ aggregation and the senile plaque formation during neurodegeneration. Additionally, TTR's dual roles, in neurodegeneration and neuroprotection are studied, emphasizing its potential for improving AD diagnosis and treatment strategies.

RESULTS:

Recent research has revealed that TTR is gradually showcasing its promise in neuroprotection and neuronal viability in AD by binding with Aβ and mitigating its neurotoxic effects. Current preclinical and clinical studies also support that TTR is actively involved in maintaining the blood-brain barrier (BBB) integrity and maintain neurotransmitter balance, all of which offer significant therapeutic promise through TTR stabilizers, such as Tafamidis, Acoramidis, and Vutrisiran, highlighting their potential in AD treatment CONCLUSION: This review concludes that TTR plays bidirectional role and gaining interest as a potential biomarker, though several challenges must be addressed before it can be established a novel therapeutic target in AD management in the modern era of drug discovery.

466

项与 Vutrisiran Sodium 相关的新闻（医药）

2025-09-04

·PRNewswire

Late Breaking Clinical Research Coming to Heart Failure Society of America Annual Scientific Meeting September 26-29

WASHINGTON, Sept. 4, 2025 /PRNewswire/ -- The Heart Failure Society of America (HFSA) is pleased to announce the Late Breaking Clinical Research that will be presented at its Annual Scientific Meeting (ASM), September 26-29, at the at the Minneapolis Convention Center in Minneapolis, MN. Late breaking clinical research to be featured during two plenary sessions includes results and updates from these major randomized trials: Continue Reading Heart Failure Society of America Annual Scientific Meeting 2025 takes place September 26-29 in Minneapolis, MN. Sunday, September 28 Plenary Session | 9:00 AM – 10:30 AM Late Breaking Clinical Research 1: Devices and Cardiomyopathies Integra-D Trial Effects of a Novel Cardiac Contractility Modulation-Defibrillator (CCM-D) on NYHA Functional Class in ICD-Indicated Patients: Post-Implant Results from the Integra-D Trial FUTURE-HF Development of an Individualized Congestion Score using a Novel Implantable Inferior Vena Cava Sensor: the FUTURE-HF Trial Portfolio DETECT-HF Development and Training of a Voice-Based Algorithm for Heart Failure Monitoring: Performance results from 599 Patients over 360,000 Patient-Days FOREST-HCM Safety and Efficacy of Aficamten in Patients with Nonobstructive Hypertrophic Cardiomyopathy: a 96-week analysis from FOREST-HCM MAPLE-HCM Divergent Effect of Aficamten Versus Metoprolol on Exercise Performance in Obstructive Hypertrophic Cardiomyopathy: A Prespecified Analysis of MAPLE-HCM ATTRibute-CM Acoramidis Reduces Cumulative Cardiovascular Outcomes Within the First Month of Treatment in Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM HELIOS-B Reduction in Gastrointestinal Events in ATTR-CM Patients Treated with Vutrisiran Compared with Placebo: Analysis from HELIOS-B Monday, September 29 Plenary Session | 9:00 AM – 10:30 AM Late Breaking Clinical Research 2: Novel Therapies VICTOR Trial Vericiguat Efficacy and Safety Across Baseline Background Therapy in Contemporary Ambulatory Patients with HFrEF Enrolled in the VICTOR trial Tolerability and Safety of Vericiguat in Chronic Heart Failure with Reduced Ejection Fraction SEISMiC C Safety and Efficacy of Intravenous Administration of Istaroxime for 48 hours in Patients with SCAI C Cardiogenic Shock due to Acute Heart Failure SUMMIT Trial Effects of Tirzepatide in Obesity-Related HFpEF by Sex: A Prespecified Secondary Analysis from the SUMMIT Trial GARDEN TIMI-74 GDF-15 Neutralization in Patients with Heart Failure: Primary Results of the GARDEN-TIMI 74 Trial Danicamtiv Trial Danicamtiv Restores Ventricular and Atrial Function in Patients with Dilated Cardiomyopathy Caused by Genetic Variants that Depress Sarcomere Function Late Breaking Clinical Research showcases novel results of major randomized trials and rapidly evolving research. Due to the quantity of remarkable trials submitted, HFSA will also feature late breaking research through Rapid Fire Oral Sessions. These Rapid Fire sessions are fast-paced oral presentations spotlighting key data from late breaking clinical research. Sessions take place on Sunday and Monday with Q&A and new topics each day. Sunday September 28 | Rapid Fire 1 | 10:45 AM - 11:45 AM Phase 1 Study of Daratumumab-Hyaluronidase for Reduction of Circulating Antibodies in Patients with High Allosensitization Awaiting Heart Transplantation Safety of Sodium-Glucose Cotransporter 2 Inhibitors in Post-Heart Transplant Patients: A Multi-Center Retrospective Cohort Study Long Term Outcomes after LVAD Weaning for Myocardial Recovery: Results from the VAD Wean Recovery Network First-In-Human Implantation of a Next-Generation Membrane-Based LVAD Long Term Trajectory of Glomerular Filtration Rate Post-LAVD and the Impact on Clinical Outcomes: An analysis of the MOMEMTUM 3 Study Increased LVEF at Six Months Following LVAD Implant is Associated with Improved Long-Term Outcomes - A MOMENTUM-3 Analysis Screening for advanced heart failure in stable outpatients (The SAINTS study) Sunday, September 28 | Rapid Fire 2: | 2:15 PM - 3:15 PM Clinical Outcomes for Heart Failure at High Risk of Hyperkalemia according to Optimization of Agents Targeting the Renin-Angiotensin-Aldosterone System Intravenous Ferric Carboxymaltose in Patients With Ischemic Versus Non-Ischemic Etiology of Heart Failure and Iron Deficiency Residual Risk of Hyperkalemia Among Patients with Heart Failure Treated with SGLT2i and ARNI Instead of ACEI/ARB High-Dose IV Loop Diuretic Therapy and Ototoxicity Risk in Patients with Acute Heart Failure: Insights from the FASTR Trial Expanded Results from a Guideline-Directed Medical Therapy Clinic Optimized Heart Failure Therapy in Patients at Risk of Cardiac Wasting in Patients with Advanced Cancer: Results from the EMPATICC trial Supervised Exercise Training Improves Outcomes in Heart Failure: Evidence from the PACT-HF 2×2 Factorial Trial Sunday, September 28 | Rapid Fire 3 | 4:15 PM - 5:15 PM Cardiac Contractility Modulation Reduces Mortality and Heart Failure Hospitalizations: A Matched Comparison of Patients Receiving and Not Receiving CCM Derived from a Real-World Dataset PROACTIVE-HF: 2-year outcomes, stratified by LVEF Outcomes of Poor Clinical Responders After Transcatheter Aortic Valve Replacement in Older Patients Invasive Hemodynamic Risk Stratification and Transcatheter Edge-to-Edge Tricuspid Repair: One-Year Results from TRILUMINATE Pivotal Trial Relationship between changes in seated pulmonary artery pressure, blood pressure, heart rate, and weight prior to heart failure hospitalization A Randomized Trial of a Remote, Digital Intervention Targeting Heart Failure Medical Therapy Machine-Learning Estimation of Pulmonary Capillary Wedge and Right Atrial Pressures With a Non-invasive Multisensor Device Deep learning assisted prediction for occurrence of pacing-induced cardiomyopathy in the atrio-ventricular block Monday, September 29 | Rapid Fire 4 | 12:00 PM – 1:00 PM Effect of vericiguat, compared with placebo, across baseline risk categories in chronic heart failure with reduced ejection fraction in the VICTOR Trial Effect of Vericiguat on Heart Failure Hospitalisation Events in Ambulatory Patients with Heart Failure and Reduced Ejection Fraction: VICTOR Trial Prespecified Analysis Effect of Vericiguat on Mortality in Ambulatory Patients with Heart Failure and Reduced Ejection Fraction: VICTOR Trial Prespecified Analysis Regional Variation in Outcomes and Response to Vericiguat in Heart Failure with Reduced Ejection Fraction: The VICTOR Trial Effect of the Soluble Guanylate Cyclase Stimulator Vericiguat on Health Status in the Phase 3 VICTOR Trial The Effect of Vericiguat on Mode of Death in Patients with Heart Failure with Reduced Ejection Fraction in the VICTOR Trial Rapidity of benefit of simultaneous initiation of finerenone and empagliflozin in people with chronic kidney disease and type 2 diabetes: the CONFIDENCE trial Dapagliflozin in Patients with Heart Failure: A Meta-Analysis of DAPA-HF, DELIVER, and DAPA ACT HF-TIMI 68 The meeting kicks off in just three weeks. A full schedule at a glance is available online, as well as information on the science and research and networking available at the meeting. Learn more about the meeting and register at hfsa.org/asm2025. About the Heart Failure Society of America The Heart Failure Society of America, Inc. (HFSA) represents the first organized effort by heart failure experts from the Americas to provide a forum for all those interested in heart function, heart failure, and congestive heart failure (CHF) research and patient care. The mission of HFSA is to provide a platform to improve and expand heart failure care through collaboration, education, innovation, research, and advocacy. HFSA members include physicians, scientists, nurses, nurse practitioners, pharmacists, trainees, other healthcare workers and patients. For more information, visit hfsa.org. Media Contact: Laura Poko, 301-798-4493, ext. 226, [email protected] SOURCE Heart Failure Society of America WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床结果临床3期临床1期

2025-09-02

·EndPoints

Watch: What it takes to build a new biotech in 2025, with John Maraganore and Clive Meanwell

If anybody knows the trials and tribulations of the biotech market, it’s John Maraganore and Clive Meanwell . Both the former longtime Alnylam CEO and former Medicines Company executive have been in the industry for decades. They opened up a new class of medicines with RNAi therapies like Amvuttra and Leqvio. Following their work at those companies, they’ve taken their expertise to City Therapeutics, Metsera, multiple investment firms and other biotechs. Now, they’re coming together for a third act as co-CEOs of Corsera Health, a cardiovascular disease prevention biotech that they’ve built with $50 million from themselves and a few friends. They came out of stealth on Tuesday , and say they will be in the clinic before 2026. They’ve also got an AI cardiovascular health prediction tool in the works. Today on Post-Hoc Live, John and Clive joined Executive Editor Drew Armstrong and me to chat about Corsera, the shifting biotech business model, how drug development can be done more efficiently, and how the industry is adjusting to all the headwinds of 2025. You can watch a replay of the show on YouTube , or listen on Apple Podcasts or Spotify . Thanks for tuning in! — Kyle LaHucik, Biotech Correspondent

2025-08-29

·FiercePharma

Pfizer to discontinue low-dose ATTR drug Vyndaqel in US ahead of 2028 patent loss

Pfizer plans to discontinue Vyndaqel, leaving the high-dose Vyndamax as the only tafamidis brand on the U.S. market. Pfizer is taking a curtain call for Vyndaqel, the low-dose version of its blockbuster tafamidis franchise for the rare heart disease transthyretin amyloid cardiomyopathy (ATTR-CM).Pfizer will discontinue Vyndaqel in the U.S. by the end of 2025, multiple amyloidosis patient groups alerted their members on social media earlier this month.The move will leave the high-dose Vyndamax as the only tafamidis brand on the market.“This decision was made to enhance patient-centered care and convenience as Vyndamax offers the same active ingredient and clinical benefits as Vyndaqel, but in a single capsule taken once daily, making it easier for patients to take their prescription,” according to the posts, which appear to be relaying a message from Pfizer.A Pfizer spokesperson confirmed the decision to Fierce Pharma, saying the move will simplify the experience for patients and providers and avoid confusion.“This change was made in consultation with experts in the field and patient advocates who agreed that a single-pill option is easier for patients to manage,” the Pfizer spokesperson said. “Pfizer is committed to ensuring a seamless transition to Vyndamax by working closely with healthcare providers, payers, patients, and other stakeholders.”The drug’s price will remain the same, the spokesperson said. The two tafamidis formulations were approved simultaneously by the FDA in 2019 to treat ATTR-CM. While Vyndaqel is given in four 20-mg capsules daily, Vyndamax comes as one 61-mg capsule per day.Since its launch, the tafamidis family has been a major growth driver for Pfizer. Last year, the franchise delivered 65% sales growth to reach $5.45 billion in annual revenues.The Vyndaqel market withdrawal is not a surprise. At the time of the FDA approval, Pfizer told Fierce Pharma that the company would aim to transition all patients to the more convenient Vyndamax once sufficient supply becomes available. Today, fewer than 20% of tafamidis scripts in the U.S. are for Vyndaqel, according to a Jefferies analysis of IQVIA data. The majority of patients in the U.S. are currently being treated with Vyndamax, including new patients, the Pfizer spokesperson told Fierce Pharma. Pfizer is pulling the trigger on the full transition now as Vyndaqel is expected to lose patent protection in 2028. An early switch could lead to slower-than-expected erosion of the tafamidis franchise following Vyndaqel’s generic entry, Jefferies analysts observed in a Thursday note.“By the time Vyndaqel generics enter the market, clinical use of the Vyndaqel formulation may already be minimal, reducing the relevance of generics,” the analysts said.For its part, Vyndamax currently has U.S. patent protection for its crystalline form until 2035, although that polymorph patent is being challenged, the Jefferies team noted.Pfizer is shoring up support for Vyndamax as competition intensifies in ATTR-CM.The FDA in November approved BridgeBio’s rival TTR stabilizer Attruby. The initial launch of Alnylam’s RNA interference therapy Amvuttra also went significantly better than Wall Street had expected.About 1,400 ATTR-CM patients have started treatment with Amvuttra since its FDA nod in late March, translating to about $150 million in revenue in the second quarter, Alnylam shared in its earnings update in July.Meanwhile, Pfizer’s Vyndaqel family grew at 21% year over year in the second quarter, with sales reaching $1.62 billion during the period. Between the three treatments, Vyndamax and Attruby are taken orally once daily and twice daily, respectively, while Amvuttra is injected subcutaneously once every three months by a healthcare professional.Alnylam has also noted some combination use of Amvuttra and Vyndaqel, although the company doesn’t expect it to be a widespread practice because of potential payer challenges. That could change, however, when Vyndaqel generics enter the market in 2028.

上市批准

100 项与 Vutrisiran Sodium 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11916 D11917	-	-	DB16699

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
转甲状腺素蛋白淀粉样变性心肌病	美国	Alnylam Pharmaceuticals, Inc.	2025-03-20
转甲状腺素蛋白介导的淀粉样变性	澳大利亚	Medison Pharma Australia Pty Ltd.	2024-06-21
遗传性转甲状腺素蛋白淀粉样变性	美国	Alnylam Pharmaceuticals, Inc.	2022-06-13

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
转甲状腺素蛋白相关家族性淀粉样多神经病变	申请上市	欧盟	Alnylam Netherlands BV	2025-04-25
老年性心脏淀粉样变性	临床3期	美国	Alnylam Pharmaceuticals, Inc.	2019-11-26
老年性心脏淀粉样变性	临床3期	日本	Alnylam Pharmaceuticals, Inc.	2019-11-26
老年性心脏淀粉样变性	临床3期	阿根廷	Alnylam Pharmaceuticals, Inc.	2019-11-26
老年性心脏淀粉样变性	临床3期	澳大利亚	Alnylam Pharmaceuticals, Inc.	2019-11-26
老年性心脏淀粉样变性	临床3期	奥地利	Alnylam Pharmaceuticals, Inc.	2019-11-26
老年性心脏淀粉样变性	临床3期	比利时	Alnylam Pharmaceuticals, Inc.	2019-11-26
老年性心脏淀粉样变性	临床3期	加拿大	Alnylam Pharmaceuticals, Inc.	2019-11-26
老年性心脏淀粉样变性	临床3期	克罗地亚	Alnylam Pharmaceuticals, Inc.	2019-11-26
老年性心脏淀粉样变性	临床3期	捷克	Alnylam Pharmaceuticals, Inc.	2019-11-26

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04153149 (HELIOS-B, Pubmed \| J Am Coll Cardiol)	临床3期	转甲状腺素蛋白淀粉样变性心肌病 NT-proBNP \| troponin I	655	Vutrisiran	鹹醖築夢繭構積繭願窪(憲願繭遞糧鏇鹽憲築窪) = 鹹簾艱顧醖選遞衊艱淵構糧窪顧鏇製窪鑰鹹構 (夢淵衊淵醖簾憲鏇廠鬱, -25.0 ~ 10.0)	积极	2025-08-01
				Placebo	鹹醖築夢繭構積繭願窪(憲願繭遞糧鏇鹽憲築窪) = 顧構齋襯夢選鹹製製網構糧窪顧鏇製窪鑰鹹構 (夢淵衊淵醖簾憲鏇廠鬱, -6.3 ~ 41.2)
NCT04153149 (HELIOS-B, Pubmed \| BusinessWire) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	655	Vutrisiran 25 mg	襯艱網鑰顧鏇蓋積齋憲(淵餘構鏇鬱廠築願壓製): HR = 0.64 (95% CI, 0.46 ~ 0.88) 更多	积极	2025-05-01
				Placebo
NCT04153149 (HELIOS-B, Pubmed) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	654	Vutrisiran 25 mg	鬱齋顧積齋簾願壓範鑰(鑰齋襯製鑰夢構遞觸壓) = 鹹糧繭衊齋鏇願夢鹹願鏇網觸鹽壓願蓋糧顧糧 (膚鏇鏇衊選淵獵夢襯糧 ) 更多	积极	2025-03-17
				Placebo	-
NCT04153149 (HELIOS-B, BusinessWire) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病 N-terminal pro-B-type Natriuretic peptide \| Cardiac Troponin I	655	Vutrisiran 25mg	襯顧簾繭遞選簾餘積遞(艱壓醖觸築積構糧醖衊) = 簾夢醖蓋艱鹽觸網餘鏇襯範鏇醖築夢鏇艱願鬱 (顧築壓繭夢遞築網鑰製 ) 更多	积极	2024-09-29
NCT04153149 (HELIOS-B, Pubmed \| N Engl J Med) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	655	Vutrisiran 25 mg	壓壓簾衊築衊鏇糧齋衊(壓廠鹽繭繭鹽願醖壓憲): HR = 0.72 (95% CI, 0.56 ~ 0.93), P-Value = 0.01 更多	积极	2024-08-30
				Placebo
NCT04153149 (HELIOS-B, Company_Website) 人工标引	临床3期	转甲状腺素蛋白淀粉样变性心肌病	655	Vutrisiran (overall population)	鑰餘選齋簾夢淵獵鑰顧(觸襯遞餘製積觸網遞壓) = Rates of adverse events (AEs), serious AEs and AEs leading to study drug discontinuation were similar between the vutrisiran and placebo arms. No AEs were seen ≥3% more frequently in the vutrisiran arm compared to the placebo arm. 鑰廠獵膚願襯鏇顧蓋艱 (齋積憲築積齋齋醖壓網 )	积极	2024-06-24
				Vutrisiran (not receiving tafamidis at baseline)
NCT03759379 (HELIOS-A, Pubmed)	临床3期	家族性淀粉样神经病	164	Vutrisiran	鏇蓋壓遞淵鹹築襯憲鏇(夢積繭醖衊憲餘壓願顧) = 窪構鬱觸窪願鑰齋築窪願網築網築艱壓餘鹽鏇 (窪選遞糧糧鹹選範構夢 )	-	2023-07-31
NCT01960348 \| NCT03759379 (APOLLO, ANA2022)	临床3期	多发性神经病 \| 淀粉样变性 NfL	-	Patisiran	網鏇觸襯蓋鹽衊鹽壓積(醖觸艱蓋顧獵選繭繭鹽) = 製窪淵憲醖獵願窪襯膚遞齋製鹽鏇繭願鏇顧膚 (衊觸膚膚窪醖鹹構糧獵 )	积极	2022-09-14
				Vutrisiran	網鏇觸襯蓋鹽衊鹽壓積(醖觸艱蓋顧獵選繭繭鹽) = 醖淵糧觸襯窪壓膚鏇簾遞齋製鹽鏇繭願鏇顧膚 (衊觸膚膚窪醖鹹構糧獵 )
NCT03759379 (HELIOS-A, Corporate Publications) 人工标引	临床3期	心肌疾病	199	vutrisiran	繭憲淵衊鏇齋膚齋製獵(夢憲觸遞糧獵衊艱願廠) = 衊鏇獵齋夢醖獵窪膚齋膚範製遞鹹選淵顧鬱繭 (製鏇餘衊鑰鹹鹹憲齋窪 )	积极	2022-05-23
				Placebo	繭憲淵衊鏇齋膚齋製獵(夢憲觸遞糧獵衊艱願廠) = 鑰遞顧鏇衊觸鬱壓鹽糧膚範製遞鹹選淵顧鬱繭 (製鏇餘衊鑰鹹鹹憲齋窪 )
NCT03759379 (HELIOS-A, Corporate Publications) 人工标引	临床3期	淀粉样神经病	199	Vutrisiran	廠糧積艱鬱艱繭遞餘鏇(願選構夢遞壓膚網廠餘) = 製鹽壓鑰襯廠積範窪鑰窪繭築艱鹹網範鑰鹹願 (鹹壓範鹹廠淵淵範鏇鑰 ) 更多	积极	2022-01-21
				Placebo	廠糧積艱鬱艱繭遞餘鏇(願選構夢遞壓膚網廠餘) = 遞醖壓壓淵蓋觸鑰憲窪窪繭築艱鹹網範鑰鹹願 (鹹壓範鹹廠淵淵範鏇鑰 ) 更多