CAMBRIDGE, Mass.

马萨诸塞州剑桥市

--(BUSINESS WIRE)--Apr. 28, 2025--

--（商业资讯）--2025年4月28日--

Alnylam Pharmaceuticals, Inc.

阿尔尼拉姆制药公司

(Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the

(Nasdaq: ALNY)，领先的RNA干扰（RNAi）治疗公司，今天宣布人类用药委员会（CHMP）

European Medicines Agency

欧洲药品管理局

(EMA) has adopted a positive opinion recommending approval of its RNAi therapeutic vutrisiran for the treatment of wild type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). ATTR-CM is a debilitating, rapidly progressive and potentially fatal disease for which there are limited treatment options.

欧洲药品管理局（EMA）已采纳积极意见，推荐批准其RNAi治疗药物vutrisiran用于治疗野生型或遗传性转甲状腺素蛋白淀粉样变性成年心肌病（ATTR-CM）患者。ATTR-CM是一种使人衰弱、进展迅速且可能致命的疾病，目前治疗选择有限。

Vutrisiran is currently approved in the .

Vutrisiran目前已在批准。

European Union

欧盟

(EU) under the brand name AMVUTTRA

(EU) 旗下品牌名为 AMVUTTRA

®

®

for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.

用于治疗成人1期或2期多发性神经病的遗传性转甲状腺素蛋白介导的（hATTR）淀粉样变性。

ATTR-CM is caused by the deposition of misfolded transthyretin (TTR) fibrils, which drive progressive and irreversible cardiovascular damage and premature death. Vutrisiran is an RNAi therapeutic that works upstream to reduce the production of TTR at its source, resulting in sustained TTR knockdown.

ATTR-CM 是由错误折叠的转甲状腺素蛋白 (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。Vutrisiran 是一种 RNAi 治疗药物，它在上游发挥作用，从源头减少 TTR 的产生，从而实现持续的 TTR 敲低。

In .

在。

Europe

欧洲

, 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. Vutrisiran has the potential to be the first and only RNAi therapeutic to receive

，它每三个月通过皮下注射给药一次，可以由医疗专业人员进行，也可以由患者或其护理人员自行给药，为治疗提供了灵活性。Vutrisiran 有潜力成为首个且唯一的 RNAi 治疗药物获得审批。

European Commission

欧盟委员会

approval for ATTR-CM, offering a clinically differentiated approach to the treatment of this disease.

批准用于ATTR-CM，为这种疾病的治疗提供了临床差异化的方案。

“This positive CHMP opinion marks another important milestone in our efforts to bring vutrisiran to people around the world living with ATTR amyloidosis with cardiomyopathy,” said

“这一积极的CHMP意见标志着我们在努力将vutrisiran带给全球患有ATTR淀粉样变心肌病的患者方面又迈出了重要的一步，”

Pushkal Garg

普什卡尔·加格

, M.D., Chief Medical Officer of

医学博士，首席医疗官

Alnylam

阿尔尼拉姆

. “In the HELIOS-B study, vutrisiran treatment resulted in rapid knockdown of TTR and led to improved survival, fewer hospitalizations and less disease progression in patients with ATTR-CM, nearly half of whom were on a TTR stabilizer. Combined with its quarterly dosing and well-established safety profile, we believe vutrisiran could offer an important new treatment option for patients in the EU.” .

“在HELIOS-B研究中，vutrisiran治疗导致TTR的快速减少，并改善了ATTR-CM患者的生存率，减少了住院次数和疾病进展，其中近一半的患者使用了TTR稳定剂。加上其每季度一次的给药方案和已确立的良好安全性，我们认为vutrisiran可为欧盟的患者提供一个重要且新的治疗选择。”

The CHMP opinion was based on positive results from the pivotal HELIOS-B Phase 3, randomized, double-blind, placebo-controlled multicenter global study, which met all 10 of its pre-specified primary and secondary endpoints across both the overall and monotherapy populations. The findings demonstrated the benefits of vutrisiran on outcomes of mortality and cardiovascular events, as well as functional capacity (6-minute walk test), health status and quality of life (Kansas City Cardiomyopathy Questionnaire), and heart failure symptoms and severity (NYHA class) in patients with ATTR-CM, with consistent effects across all patient subgroups, including those on a concomitant TTR stabilizer.

CHMP 的意见基于关键性 HELIOS-B 第 3 阶段随机、双盲、安慰剂对照的多中心全球研究的积极结果，该研究在其预先设定的 10 个主要和次要终点上均达到了预期目标，涵盖总体人群和单药治疗人群。研究结果表明，vutrisiran 在改善 ATTR-CM 患者的死亡率和心血管事件结果、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），以及心力衰竭症状和严重程度（NYHA 分级）方面具有显著益处，并且在所有患者亚组中均表现出一致的效果，包括同时使用 TTR 稳定剂的患者。

In HELIOS-B, rates of adverse events (AEs), serious AEs, severe AEs and AEs leading to study drug discontinuation were similar between the vutrisiran and placebo arms. The safety profile of vutrisiran is characterized by injection site reactions and increase in blood alkaline phosphatase and alanine transaminase.

在HELIOS-B中，vutrisiran组和安慰剂组之间的不良事件（AEs）、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。Vutrisiran的安全性特征表现为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

.

。

1

1

Vutrisiran was approved by the

Vutrisiran 已被批准

U.S. Food and Drug Administration

美国食品药品监督管理局

(FDA) on

（FDA）关于

20 March 2025

2025年3月20日

and the

和

Brazilian Health Regulatory Agency

巴西卫生监管局

(ANVISA) on

（ANVISA）在

31 March 2025

2025年3月31日

for the treatment of the cardiomyopathy of wild-type or hereditary ATTR-CM. It is currently under review by the

用于治疗野生型或遗传性ATTR-CM的心肌病。目前正在审查中，由

Japanese Pharmaceuticals

日本制药公司

and

和

Medical Devices Agency

医疗器械局

(PMDA).

（PMDA）。

Alnylam

阿尔尼拉姆

remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025 and beyond.

将继续按计划在2025年及以后进行vutrisiran的更多全球监管提交。

AMVUTTRA

AMVUTTRA

®

®

(vutrisiran) INDICATION AND IMPORTANT SAFETY INFORMATION

（vutrisiran）适应症和重要安全信息

Indication

指示

In

在

Europe

欧洲

and the

和

UK

英国

, vutrisiran is indicated for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.

，Vutrisiran 适用于治疗患有 1 期或 2 期多发性神经病的成人 hATTR 淀粉样变性患者。

Important Safety Information

重要安全信息

Reduced Serum Vitamin A Levels and Recommended Supplementation

血清维生素A水平降低及推荐补充

Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). .

Vutrisiran治疗会导致血清维生素A水平下降。建议服用vutrisiran的患者每天补充约2500至3000国际单位的维生素A，但不要超过这个范围。如果患者出现提示维生素A缺乏的眼部症状（如夜盲症），应转诊至眼科医生。

Adverse Reactions

不良反应

The most frequently occurring adverse reactions in patients treated with vutrisiran were pain in extremity and arthralgia. Other commonly reported adverse reactions with vutrisiran were dyspnoea, injection site reaction and increase in blood alkaline phosphatase.

接受Vutrisiran治疗的患者最常见的不良反应是四肢疼痛和关节痛。其他常见的不良反应包括呼吸困难、注射部位反应以及血液碱性磷酸酶升高。

For additional information about vutrisiran, please see the full

有关vutrisiran的更多信息，请参阅完整内容

Summary of Product Characteristics

产品特性总结

.

。

About AMVUTTRA

关于AMVUTTRA

®

®

(vutrisiran)

（vutrisiran）

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)，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)。

In .

在。

Europe

欧洲

, it is administered as a subcutaneous injection once every three months, either by a healthcare professional, or self-administered by patients or their caregivers. 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. .

，它每三个月通过皮下注射给药一次，可以由医疗专业人员进行注射，也可以由患者或其护理人员自行注射。Vutrisiran 也正在开发用于治疗伴有心肌病的转甲状腺素蛋白淀粉样变性（ATTR-CM），涵盖野生型和遗传型两种形式的疾病。

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) 是一种诊断不足、进展迅速、致残且致命的疾病，由错误折叠的转甲状腺素蛋白 (TTR) 引起，这些蛋白作为淀粉样沉积物在身体的各个部位（包括神经、心脏和胃肠道）积累。患者可能表现为多发性神经病、心肌病或两种疾病表现同时出现。

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），在没有TTR基因变异的情况下发生，全球估计有200,000至300,000人受到影响。

1-4

1-4

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）是一种天然的细胞基因沉默过程，代表了当今生物学和药物开发领域最有前途且进展最快的前沿之一。

5

5

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。

6

6

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。

5

5

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，有望改变遗传病和其他疾病患者的护理方式。

About

关于

Alnylam Pharmaceuticals

阿尼兰姆制药公司

Alnylam

阿尔尼拉姆

(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.

(Nasdaq: ALNY) 已将RNA干扰（RNAi）技术转化为一类全新的创新药物，有望改变那些患有未满足需求的罕见和常见疾病患者的生活。基于诺贝尔奖获奖科学，RNAi疗法代表了一种强大且经过临床验证的方法，能够带来变革性的药物。

Since its founding in 2002, .

自 2002 年成立以来，。

Alnylam

阿尔尼拉姆

has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality.

引领了RNAi革命，并继续致力于实现将科学可能性变为现实的大胆愿景。

Alnylam

阿尔尼拉姆

has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development.

拥有一个深入的在研药物管道，包括多个处于后期开发阶段的产品候选物。

Alnylam

阿尔尼拉姆

is executing on its “

正在执行其“

Alnylam P

阿尼拉姆制药公司

5

5

x25

x25

” 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.

“通过可持续创新和卓越的财务表现，提供在罕见病和常见病领域具有变革意义的药物，惠及全球患者，从而打造领先的生物技术企业形象。”

Alnylam

阿尔尼拉姆

is headquartered in

总部位于

Cambridge, MA.

马萨诸塞州剑桥市

Alnylam Forward-Looking Statements

Alnylam前瞻性声明

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, Alnylam’s expectations regarding the safety and efficacy of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy; the potential for vutrisiran to be the first and only RNAi therapeutic to receive .

本新闻稿包含1933年《证券法》第27A条和1934年《证券交易法》第21E条所指的前瞻性陈述。所有关于Alnylam的预期、信念、目标、计划或前景的非历史事实的陈述，包括但不限于Alnylam对vutrisiran治疗伴有心肌病的ATTR淀粉样变性的安全性和有效性的预期；vutrisiran有望成为首个且唯一的RNAi治疗药物。

European Commission

欧洲委员会

approval for ATTR-CM; the potential for vutrisiran to offer a clinically differentiated approach to treatment of ATTR-CM; the potential success of Alnylam’s efforts to bring vutrisiran to people around the world living with ATTR-CM; the potential for vutrisiran to offer an important new treatment option for patients in the EU; and the timing of global regulatory submissions for vutrisiran for ATTR-CM should be considered forward-looking statements.

治疗ATTR-CM的批准；vutrisiran有望提供一种临床差异化的ATTR-CM治疗方法；Alnylam将vutrisiran带给全球ATTR-CM患者的潜在成功；vutrisiran为欧盟患者提供一个重要新治疗选择的潜力；以及vutrisiran针对ATTR-CM的全球监管提交时间，均应视为前瞻性声明。

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 “.

实际结果和未来计划可能与这些前瞻性陈述所表明的内容存在重大差异，这是由于各种重要的风险、不确定性和其他因素，包括但不限于与Alnylam成功执行其“能力相关的风险和不确定性。

Alnylam P

Alnylam P

5

5

x25

x25

” strategy; Alnylam’s ability to successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates, including vutrisiran; actions or advice of regulatory agencies and Alnylam’s ability to obtain regulatory approval for its product candidates, including vutrisiran, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; and any delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; 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 .

“战略；Alnylam成功证明其候选产品有效性和安全性的能力；Alnylam候选产品的临床前和临床结果，包括vutrisiran；监管机构的行为或建议以及Alnylam为其候选产品（包括vutrisiran）获得监管批准的能力，以及获得有利的定价和报销；在全球范围内成功推出、营销和销售Alnylam已批准的产品；以及在Alnylam候选产品或其上市产品的生产和供应过程中出现的任何延迟、中断或失败；以及在Alnylam提交的2024年Form 10-K年度报告中“风险因素”部分更全面讨论的那些风险。”

Securities and Exchange Commission

证券交易委员会

(SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q and in its other

（美国证券交易委员会），可能会在Alnylam公司随后的10-Q季度报告及其其他文件中不时更新。

SEC

证券交易委员会

filings. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date.

备案。此外，任何前瞻性陈述仅代表Alnylam截至今天的观点，不应依赖其作为代表其在任何后续日期的观点。

Alnylam

阿尔尼拉姆

explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

明确声明不承担任何更新前瞻性陈述的义务，除非法律要求。

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商业电讯网

:

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Alnylam Pharmaceuticals, Inc.

Alnylam制药公司

Christine Regan Lindenboom

克里斯汀·里根·林登布姆

(Investors and Media)

（投资者和媒体）

+1-617-682-4340

+1-617-682-4340

Josh Brodsky

乔什·布罗德斯基

(Investors)

（投资者）

+1-617-551-8276

+1-617-551-8276

Emily Bunting

艾米丽·邦廷

(Media,

（媒体，

Europe

欧洲

)

)

+41 79 866 97 03

+41 79 866 97 03

Source:

源代码：

Alnylam Pharmaceuticals, Inc.

阿里拉姆制药公司