FDA批准Qfitlia™（fitusiran），首个用于治疗A型或B型血友病的siRNA（RNAi疗法）-动脉网

FDA Approves Qfitlia™ (fitusiran), the First siRNA (RNAi Therapeutic) for the Treatment of Hemophilia A or B

阿里拉姆制药等信源发布 2025-03-28 04:50

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CAMBRIDGE, Mass.

马萨诸塞州剑桥市

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

--（商业资讯）--2025年3月28日--

Alnylam Pharmaceuticals, Inc.

Alnylam制药公司

(Nasdaq: ALNY), the leading RNAi therapeutics company, today highlighted the significance of the

(Nasdaq: ALNY)，领先的RNAi治疗公司，今天强调了这一重要意义。

U.S.

美国

Food and Drug Administration’s (FDA) approval of Qfitlia™ (fitusiran), the sixth

美国食品药品监督管理局 (FDA) 批准了 Qfitlia™（fitusiran），这是第六个...

Alnylam

阿尔尼拉姆

-discovered RNAi therapeutic approved in the

-发现的RNAi治疗药物获批在

U.S.

美国

, and the first and only therapeutic to lower antithrombin (AT), a protein that inhibits blood clotting, with the goal of promoting thrombin generation to rebalance hemostasis and prevent bleeds. Qfitlia is indicated in the

，并且是首个也是唯一一个降低抗凝血酶（AT）的治疗药物，抗凝血酶是一种抑制血液凝固的蛋白质，其目的是促进凝血酶生成，以重新平衡止血并预防出血。Qfitlia适用于

U.S.

美国

for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B, with or without factor VIII or IX inhibitors (neutralizing antibodies).

用于常规预防，以预防或减少12岁及以上伴有或不伴有因子VIII或IX抑制剂（中和抗体）的A型或B型血友病成人和儿童患者的出血发作频率。

Alnylam

阿尼拉姆

scientists discovered Qfitlia, published the first clinical data in the

科学家发现了Qfitlia，公布了首个临床数据在

New England Journal of Medicine

新英格兰医学杂志

in 2017 showing reduced bleeding rates in hemophilia patients, and initiated the Phase 3 development program. In 2014, Sanofi obtained global rights to co-develop and co-commercialize Qfitlia under a license and collaboration agreement. The agreement was amended in 2018, with Sanofi obtaining global development and commercialization rights to Qfitlia, and .

2017年显示出在血友病患者中减少了出血率，并启动了第3阶段的开发计划。2014年，赛诺菲根据许可和合作协议获得了全球共同开发和共同商业化Qfitlia的权利。该协议于2018年进行了修订，赛诺菲获得了Qfitlia的全球开发和商业化权利。

Alnylam

阿尔尼拉姆

becoming eligible to receive tiered royalties of 15 to 30 percent on global net sales.

有资格在全球净销售额上获得15%到30%的分层版税。

Qfitlia is the sixth

Qfitlia 是第六

Alnylam

阿尔尼拉姆

-discovered medicine using its RNAi therapeutic platform to be approved to date. The approval marks the completion of the “products” goal in Alnylam’s

- 迄今为止，利用其RNAi治疗平台发现的药物已获批准。这一批准标志着Alnylam公司“产品”目标的完成。

x25

strategy, a set of corporate goals for 2025. Qfitlia has the potential to benefit the estimated one million people living with hemophilia A and B around the world.

战略，一组 2025 年企业目标。Qfitlia 有望造福全球约一百万名 A 型和 B 型血友病患者。

“Today’s approval of Qfitlia is an important moment for

“今天对Qfitlia的批准是一个重要的时刻对于

Alnylam

阿尔尼拉姆

and our RNAi therapeutics platform, and for the patients living with hemophilia, who now have access to what we believe will be a transformative medicine,” said

“我们的RNAi治疗平台，以及现在能够获得我们认为将是一种变革性药物的血友病患者，”

Yvonne Greenstreet

伊冯·格林斯特里特

, MBChB, Chief Executive Officer of

，医学学士，首席执行官

Alnylam

阿尔尼拉姆

. “Clinical studies showed that by lowering antithrombin, Qfitlia was able to prophylactically reduce annualized bleeding rates by 90%. Importantly, based on its unique mechanism of action, Qfitlia has demonstrated efficacy in patients with hemophilia A or B, with or without inhibitors, with a subcutaneous injection regimen once every two months.

“临床研究表明，通过降低抗凝血酶，Qfitlia 能够预防性地将年出血率降低 90%。重要的是，基于其独特的机制，Qfitlia 已在 A 型或 B 型血友病患者中显示出疗效，无论是否具有抑制物，且每两个月进行一次皮下注射。”

We are excited about the potential of Qfitlia to be an important non-factor option for patients with this burdensome disease.” .

我们对Qfitlia成为这种负担性疾病的患者的重要非因子选项的潜力感到兴奋。”

Qfitlia is indicated in the

Qfitlia 适用于

U.S.

美国

to treat hemophilia A or B for adults and pediatric patients 12 years of age and older with or without inhibiting antibodies to factor VIII (hemophilia A) or factor IX (hemophilia B). Regulatory submissions for Qfitlia have also been completed in

用于治疗12岁及以上成人和儿童患者的A型或B型血友病，无论是否具有针对凝血因子VIII（A型血友病）或凝血因子IX（B型血友病）的抑制性抗体。Qfitlia的监管提交也已完成。

China

中国

and

和

Brazil

巴西

。

About RNAi Therapeutics

关于RNAi疗法

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.

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。它的发现被誉为“每十年左右才会出现一次的重大科学突破”，并因其重要性而荣获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自然生物学过程RNAi，一类被称为RNAi治疗药物的新药已经成为现实。小干扰RNA（siRNA）是介导RNAi的分子，也是Alnylam公司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的遗传前体——从而阻止这些蛋白的生成，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，有望改变对遗传病和其他疾病患者的治疗。

About

关于

Alnylam Pharmaceuticals

Alnylam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，有望改变那些患有罕见和普遍疾病但需求未得到满足的患者的生活。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，能够产生变革性的药物。

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. Alnylam’s commercial RNAi therapeutic products include ONPATTRO

引领了RNAi革命，并继续致力于实现将科学可能性转化为现实的大胆愿景。Alnylam的商业RNAi治疗产品包括ONPATTRO。

(patisiran), AMVUTTRA

(patisiran)，AMVUTTRA

(vutrisiran), GIVLAARI

(vutrisiran)，GIVLAARI

(givosiran), and OXLUMO

(givosiran)，以及OXLUMO

(lumasiran), which are being developed and commercialized by

（lumasiran），这些药物正在被开发和商业化

Alnylam

阿尔尼拉姆

, and Leqvio

，以及Leqvio

(inclisiran) and Qfitlia

(inclisiran) 和 Qfitlia

™

商标符号

(fitusiran), which are being developed and commercialized by Alnylam’s partners, Novartis and Sanofi, respectively.

（fitusiran），它们分别由Alnylam的合作伙伴诺华和赛诺菲开发和商业化。

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药物。

Alnylam

阿尔尼拉姆

is executing on its “

正在执行其“

Alnylam P

阿尼拉姆 P

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。

For more information about our people, science and pipeline, please visit

如需更多关于我们的人才、科学和研发管线的信息，请访问

www.alnylam.com

and engage with us on X (formerly Twitter) at

并在X（前身为Twitter）上与我们互动

@Alnylam

, or on

，或者在

领英

，

Facebook

脸书

, or

，或者

Instagram

。

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, statements regarding Qfitlia’s potential to benefit people living with hemophilia A and B around the world, Qfitlia’s potential to be a transformational medicine, and Qfitlia’s potential to be an important non-factor option for patients with hemophilia; and the potential for .

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性事实的陈述，包括但不限于：Qfitlia有望为全球患有A型和B型血友病的人群带来益处，Qfitlia有潜力成为一款变革性药物，Qfitlia可能成为血友病患者重要的非因子选择；以及其潜在可能性。

Alnylam

阿尔尼拉姆

to receive royalties on global net sales of Qfitlia, 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: Sanofi’s ability to obtain marketing authorizations for Qfitlia in countries outside the .

就Qfitlia的全球净销售额收取特许权使用费，应视为前瞻性声明。实际结果和未来计划可能与这些前瞻性声明所表明的内容存在重大差异，原因包括各种重要的风险、不确定性和其他因素，特别是与赛诺菲在其他国家获得Qfitlia上市授权能力相关的风险和不确定性。

U.S.

美国

and thereafter to launch Qfitlia and to obtain adoption and reimbursement for Qfitlia; Alnylam’s ability to successfully execute on its “

此后推出Qfitlia并获得Qfitlia的采用和报销；Alnylam成功执行其“

Alnylam P

阿尼兰P

x25

” strategy; 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, including Sanofi; the outcome of litigation; the risk of future government investigations and substantial changes in governmental polices, regulations, funding and enforcement; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2024 Annual Report on Form 10-K filed with the .

“战略；Alnylam发现和开发新型候选药物及递送方法并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；监管机构的行为或建议以及Alnylam为其候选产品获得并维持监管批准的能力，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的已批准产品；Alnylam候选产品或其上市产品的生产与供应中的延迟、中断或失败；获取、维护和保护知识产权；Alnylam通过在运营方面进行纪律严明的投资以管理其增长和运营开支，并在未来实现财务自给自足的能力；Alnylam维持战略性业务合作的能力；Alnylam对第三方在某些产品的开发和商业化方面的依赖，包括赛诺菲；诉讼的结果；未来政府调查的风险和政府政策、法规、资金和执法的重大变化；以及意外支出；还包括在Alnylam提交的2024年Form 10-K年度报告中详细讨论的“风险因素”中更全面提及的风险和不确定性。”

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 other filings that

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

Alnylam

阿尔尼拉姆

makes with the

与...一起制作

SEC

证券交易委员会

. 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.

此外，任何前瞻性声明仅代表Alnylam截至今日的观点，不应被视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.

明确否认任何义务，除非法律要求，否则不会更新任何前瞻性声明。

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