Alnylam Pharmaceuticals, Inc.(Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that the

Alnylam Pharmaceuticals, Inc.(Nasdaq: ALNY)，领先的RNA干扰（RNAi）治疗公司，今天宣布

European Commission

欧盟委员会

(EC) has granted approval for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) as an additional indication for its orphan RNAi therapeutic AMVUTTRA

欧洲委员会（EC）已批准将野生型或遗传性转甲状腺素蛋白淀粉样变性（ATTR-CM）成年心肌病患者作为其孤儿RNAi治疗药物AMVUTTRA的额外适应症。

(vutrisiran). The approval broadens the indication for AMVUTTRA, which now becomes the first and only RNAi therapeutic approved by the EC for the treatment of the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. .

（vutrisiran）。此次批准扩大了AMVUTTRA的适应症，使其成为首个也是唯一一个获欧洲委员会（EC）批准的RNAi疗法，用于治疗转甲状腺素蛋白淀粉样变性（ATTR）的心肌病表现及遗传性转甲状腺素蛋白介导的淀粉样变性（hATTR）的多发性神经病表现的成年患者。

“Estimates show approximately 100,000 people are affected by ATTR amyloidosis across

“估计显示，全球约有10万人受到ATTR淀粉样变性的影响，

Europe

欧洲

, most with cardiomyopathy, so this approval marks a critical step toward addressing this underserved patient population,” said

”，其中大多数患有心肌病，因此这项批准标志着在解决这一服务不足的患者群体方面迈出了关键一步，”

Pushkal Garg

普什卡尔· garg

, M.D., Chief Medical Officer at

医学博士，首席医疗官 tại

Alnylam

阿尔尼拉姆

. “AMVUTTRA is supported by a well-established efficacy and safety profile, with over 6,000 patient-years of global experience in the treatment of hATTR with polyneuropathy. By delivering rapid and sustained knockdown of TTR through convenient, quarterly dosing, it offers a clinically differentiated approach with the potential to transform outcomes for patients living with this debilitating and potentially fatal disease.

“AMVUTTRA 拥有明确的有效性和安全性数据支持，在治疗伴有多种神经病变的遗传性转甲状腺素蛋白淀粉样变性（hATTR）方面，已有超过6000个患者年的全球经验。通过便捷的每季度一次给药，该药物能够快速且持续地降低TTR水平，提供了一种具有临床差异化的治疗方案，有望改变患有这种致残且可能致命疾病患者的预后。”

We now look forward to securing access to AMVUTTRA for eligible patients across the EU as quickly as possible.” .

我们期待尽快为欧盟各地的符合条件的患者提供AMVUTTRA的使用机会。

The EC

欧洲委员会

decision is based on positive results from the pivotal HELIOS-B Phase 3 study – a randomized, double-blind, placebo-controlled, multicenter, global trial that enrolled a diverse group of patients reflective of the contemporary ATTR-CM population, including those receiving substantial concurrent use of available standard-of-care therapies such as tafamidis and SGLT2 inhibitors.

决策基于关键的HELIOS-B第三阶段研究的积极结果——这是一项随机、双盲、安慰剂对照、多中心、全球性试验，招募了反映当代ATTR-CM人群多样性的患者群体，包括那些大量同时使用现有标准治疗疗法（如tafamidis和SGLT2抑制剂）的患者。

AMVUTTRA met all 10 pre-specified primary and secondary endpoints across both the overall and monotherapy populations. These included statistically significant reductions in all-cause mortality and recurrent cardiovascular events, as well as significant improvements in functional capacity (6-minute walk test), health status and quality of life (Kansas City Cardiomyopathy Questionnaire), and heart failure symptoms and severity (NYHA class).

AMVUTTRA 在整体人群和单药治疗人群中均达到了所有 10 项预先设定的主要和次要终点。这些终点包括全因死亡率和心血管事件复发的统计学显著降低，以及功能能力（6 分钟步行测试）、健康状况和生活质量（堪萨斯城心肌病问卷）和心力衰竭症状及严重程度（NYHA 分级）的显著改善。

In the overall population, AMVUTTRA achieved a 28% reduction in the primary composite of all-cause mortality and recurrent cardiovascular events as compared to placebo. 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.

在总体人群中，与安慰剂相比，AMVUTTRA 在全因死亡率和复发性心血管事件的主要综合指标上实现了 28% 的降低。在此人群的预设次要终点分析中，包括长达 36 个月的双盲期和 6 个月的开放标签延长期，死亡率在 42 个月内显著降低了 36%。

In HELIOS-B, rates of adverse events (AEs), serious AEs, severe AEs and AEs leading to study drug discontinuation were similar between the AMVUTTRA and placebo arms. Adverse drug reactions of AMVUTTRA include injection site reactions and increase in blood alkaline phosphatase and alanine transaminase.

在HELIOS-B中，AMVUTTRA组和安慰剂组之间的不良事件（AE）、严重不良事件、重度不良事件以及导致研究药物停用的不良事件发生率相似。AMVUTTRA的药物不良反应包括注射部位反应以及血液碱性磷酸酶和丙氨酸转氨酶升高。

Detailed results from the HELIOS-B study were published in .

HELIOS-B 研究的详细结果发表在 。

The New England Journal of Medicine

新英格兰医学杂志

“The HELIOS-B findings provide compelling evidence to support the use of vutrisiran as a first-line treatment option for patients with ATTR-CM,” said

“HELIOS-B 研究结果为将 vutrisiran 作为 ATTR-CM 患者的一线治疗选择提供了有力证据，”

Marianna Fontana

玛丽安娜·丰塔纳

, M.D., Ph.D., HELIOS-B investigator, Professor of Cardiology,

医学博士，哲学博士，HELIOS-B 研究员，心脏病学教授，

University College London

伦敦大学学院

, National Amyloidosis Center,

，国家淀粉样变性中心，

Royal Free Hospital

皇家自由医院

London

伦敦

. “As a physician, it is a privilege to see the true impact on patients in the clinic. The trial enrolled a broad population reflective of real-world clinical practice, and that’s what makes the results so meaningful. This is a milestone for patients, who now have a new treatment option that has the potential to significantly improve outcomes of this disease.” .

“作为一名医生，能在诊所中亲眼看到对患者的真实影响是一种荣幸。该试验招募了反映真实世界临床实践的广泛人群，这使得研究结果具有深远的意义。这对患者而言是一个里程碑，因为他们现在拥有了一种有望显著改善这种疾病预后的新治疗选择。”

ATTR-CM is caused by the deposition of misfolded TTR fibrils, which drive progressive and irreversible cardiovascular damage and premature death. AMVUTTRA is an RNAi therapeutic that works upstream by delivering sustained knockdown of disease-causing TTR at its source. In the EU, it is administered as a subcutaneous injection once every three months, either by a healthcare professional, or self-administered by patients or their caregivers, offering flexibility in treatment delivery. .

ATTR-CM 是由错误折叠的 TTR 原纤维沉积引起的，这些原纤维导致进行性和不可逆的心血管损伤以及过早死亡。AMVUTTRA 是一种 RNAi 疗法，通过在源头持续抑制致病性 TTR 的表达来发挥作用。在欧盟，该药物每三个月通过皮下注射给药一次，可由医疗专业人员进行注射，或由患者本人或其护理人员自行注射，从而为治疗提供了灵活性。

“Amyloidosis is a serious and progressive disease that significantly impacts not only patients’ physical health, but also their quality of life and independence. I am thrilled by the news of a new therapy for people in the EU living with ATTR-CM who often face delayed diagnosis. Having a new treatment option available marks a welcome development for the amyloidosis community,” said Giovanni d’Alessio, President of the .

“Amyloidosis 是一种严重且进展性的疾病，不仅显著影响患者的身体健康，还影响他们的生活质量和独立性。得知欧盟地区患有ATTR-CM的人们有了新的疗法，我感到非常激动，这些人常常面临诊断延迟的问题。拥有一个新的治疗选择标志着淀粉样变性患者群体迎来了可喜的发展，”Giovanni d’Alessio（某组织的主席）表示。

Amyloidosis Alliance

淀粉样变病联盟

, the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on the maintenance of the EU Orphan Designation for AMVUTTRA in ATTR amyloidosis.

欧洲药品管理局的孤儿药产品委员会（COMP）对AMVUTTRA在ATTR淀粉样变性中的欧盟孤儿药资格维持发表了积极意见。

AMVUTTRA was approved in

AMVUTTRA 已获批在

March 2025

2025年3月

by the

通过

U.S. Food and Drug Administration

美国食品药品监督管理局

(FDA) and the

（FDA）和

Brazilian Health Regulatory Agency

巴西卫生监管局

(ANVISA) for the treatment of the cardiomyopathy of wild-type or hereditary ATTR amyloidosis in adults.

(ANVISA) 用于治疗成人野生型或遗传性ATTR淀粉样变性的心肌病。

About AMVUTTRA

关于AMVUTTRA

AMVUTTRA

AMVUTTRA

(vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of variant and wild‑type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, vutrisiran 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) 是一种 RNAi 治疗药物，能够快速减少变异型和野生型转甲状腺素蛋白 (TTR)，从而针对转甲状腺素蛋白 (ATTR) 淀粉样变性的根本原因。vutrisiran 通过皮下注射每季度给药一次，已在超过 15 个国家获批并上市，用于治疗成人遗传性转甲状腺素蛋白介导的淀粉样变性多发性神经病 (hATTR-PN)。

for the treatment of wild-type or hereditary ATTR amyloidosis in adult patients with cardiomyopathy (ATTR-CM). In the EU, AMVUTTRA is administered once every three months, either by a healthcare professional or through self-administration by patients or their caregivers.

用于治疗野生型或遗传性ATTR淀粉样变性伴心肌病（ATTR-CM）的成年患者。在欧盟，AMVUTTRA每三个月给药一次，可以由医疗专业人员进行，或者由患者或其护理人员自行给药。

About ATTR

关于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.

转甲状腺素蛋白淀粉样变性（ATTR）是一种因错误折叠的转甲状腺素蛋白（TTR）在身体各部位（包括神经、心脏和胃肠道）堆积形成淀粉样沉积物而导致的诊断不足、进展迅速、致残且致命的疾病。患者可能表现为多发性神经病、心肌病或两种症状并存。

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..

ATTR有两种不同的形式——遗传性ATTR（hATTR），由TTR基因变异引起，全球约有50,000人受到影响；以及野生型ATTR（wtATTR），它在没有TTR基因变异的情况下发生，全球约有200,000至300,000人受到影响。

About RNAi

关于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.

RNAi（RNA干扰）是一种天然的基因沉默细胞过程，代表了当今生物学和药物开发中最有前途且进展最快的前沿领域之一。

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.

它的发现被誉为“每隔十年左右才会出现一次的重大科学突破”，并因此获得了2006年诺贝尔生理学或医学奖。

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..

通过利用我们细胞中发生的天然生物过程RNAi，一类被称为RNAi治疗药物的新药现已成为现实。小干扰RNA（siRNA）是介导RNAi的分子，也是Alnylam的RNAi治疗平台的组成部分，它通过有效沉默信使RNA（mRNA）——编码致病或疾病通路蛋白质的遗传前体——从而阻止这些蛋白质的生成，在当今药物的上游发挥作用。

This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

这是一项革命性的方法，有潜力改变遗传病和其他疾病患者的护理方式。

AboutAlnylam Pharmaceuticals

关于Alnylam Pharmaceuticals

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.

Alnylam(Nasdaq: ALNY) 已引领将RNA干扰（RNAi）转化为一类全新创新药物的开发，这些药物有潜力改变那些患有罕见和普遍但需求未被满足的疾病患者的生活。基于诺贝尔奖获奖科学成果，RNAi疗法代表了一种强大且经过临床验证的方法，能够产生变革性的药物。