Vertex宣布欧盟委员会批准ALYFTREK®，一种用于治疗囊性纤维化的新每日一次CFTR调节剂-动脉网

Vertex Announces European Commission Approval of ALYFTREK ® , a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis

福泰制药等信源发布 2025-07-01 20:24

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可切换为仅中文

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- ALYFTREK

- 阿里夫特雷克

(deutivacaftor/tezacaftor/vanzacaftor) approved in the EU for people with CF 6 years and older with at least one non-class I mutation in the CFTR gene, making it the broadest label for this medicine in the world -

（deutivacaftor/tezacaftor/vanzacaftor）在欧盟获批用于治疗6岁及以上、CFTR基因中至少携带一个非I类突变的囊性纤维化患者，成为该药物在全球范围内最广泛的适应症标签。

- In head-to-head clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior on ppFEV

在头对头临床试验中，deutivacaftor/tezacaftor/vanzacaftor 在 ppFEV 上表现非劣效。

and superior compared to KAFTRIO

并且优于KAFTRIO

(ivacaftor/tezacaftor/elexacaftor) at reducing sweat chloride, demonstrating greater improvement in CFTR function -

(伊伐卡托/特扎卡托/埃莱卡托) 在减少汗液氯化物方面表现出更显著的CFTR功能改善 -

- Approximately 31,000 people with CF in the EU are now eligible for this new highly effective modulator therapy -

- 欧盟境内大约有31,000名CF患者现在有资格接受这种新的高效调节剂疗法 -

LONDON

伦敦

--(BUSINESS WIRE)--Jul. 1, 2025--

--（商业电报）--2025年7月1日--

Vertex Pharmaceuticals

顶点制药公司

(Nasdaq: VRTX) today announced that the

(Nasdaq: VRTX) 今天宣布

European Commission

欧盟委员会

has granted approval for ALYFTREK

已批准ALYFTREK

(deutivacaftor/tezacaftor/vanzacaftor)

(德卢卡福/特扎卡福/万扎卡福)

for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (

用于治疗6岁及以上患有囊性纤维化（CF）且在囊性纤维化跨膜电导调节因子（CFTR）中至少有一个非I类突变的患者。

CFTR

) gene.

) 基因。

“Thousands of people with CF across the EU may now benefit from this new, once-daily medicine, which has demonstrated further improvement in CFTR protein function versus KAFTRIO,” said

“欧盟成千上万名囊性纤维化患者现在可能受益于这种新的每日一次的药物，该药物在CFTR蛋白功能方面显示出比KAFTRIO更进一步的改善，”

Reshma Kewalramani

杰莎·凯瓦拉曼

, M.D., Chief Executive Officer and President of

医学博士，首席执行官兼总裁

Vertex

顶点

. “With this approval, we are one step closer to our ultimate goal of restoring normal levels of CFTR function in people living with CF.”

“随着这一批准，我们距离恢复囊性纤维化患者正常的CFTR功能的最终目标又近了一步。”

In two head-to-head pivotal clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior to KAFTRIO

在两项头对头的关键临床试验中，deutivacaftor/tezacaftor/vanzacaftor 不劣于 KAFTRIO。

(ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor on ppFEV

(ivacaftor/tezacaftor/elexacaftor) 联合 ivacaftor 对 ppFEV 的影响

and superior at reducing sweat chloride, demonstrating greater improvement in CFTR function.

并且在减少汗液氯化物方面表现优异，显示出 CFTR 功能的更大改善。

“CF care has been transformed by the advent of highly effective CFTR modulators, and I am very pleased that we now have a new treatment option to even better address this multi-systemic disease,” said Professor

“CF护理已经因高效CFTR调节剂的出现而发生了转变，我们现在有了一个更好的治疗选择来应对这种多系统疾病，我感到非常高兴，”教授说。

Marcus A

马库斯 A

. Mall, M.D., Professor and Chair of the

商城，医学博士，教授兼主任

Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine

儿科呼吸内科、免疫学和重症监护医学部

and Cystic Fibrosis Center at Charité Universitätsmedizin

Charité大学医院的囊性纤维化中心

Berlin

柏林

. “Deutivacaftor/tezacaftor/vanzacaftor has shown it can deliver greater reductions in sweat chloride compared to standard of care. By bringing more people closer to normal level of CFTR function, this new medicine has the potential to further improve outcomes for patients.”

“Deutivacaftor/tezacaftor/vanzacaftor 已显示出相较于标准治疗能够更大程度地减少汗液氯化物。通过使更多患者的 CFTR 功能接近正常水平，这种新药有潜力进一步改善患者的治疗效果。”

As a result of reimbursement agreements in

由于报销协议

Ireland

爱尔兰

and

和

Denmark

丹麦

and provisions for access in health care systems such as

以及医疗保健系统中的访问规定，例如

Germany

德国

, eligible patients in these countries will have access to deutivacaftor/tezacaftor/vanzacaftor shortly following regulatory approval by the

，这些国家的符合条件的患者将在获得监管机构批准后不久便可以使用deutivacaftor/tezacaftor/vanzacaftor。

European Commission

欧盟委员会

。

Vertex

顶点

will continue to work with reimbursement bodies across the

将继续与各地的报销机构合作

European Union

欧盟

member states to ensure access for all eligible patients as quickly as possible.

成员国应确保所有符合条件的患者尽快获得准入。

About Cystic Fibrosis

关于囊性纤维化

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in

囊性纤维化（CF）是一种罕见的、缩短寿命的遗传性疾病，影响着超过109,000人，其中包括94,000名患者。

North America

北美

，

Europe

欧洲

and

和

Australia

澳大利亚

. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the

CF 是一种进行性的多器官疾病，影响肺、肝、胰腺、胃肠道、鼻窦、汗腺和生殖道。CF 由 CFTR 蛋白缺陷和/或缺失引起，这是由于某些基因突变导致的。

CFTR

gene. Children must inherit two defective

基因。儿童必须继承两个有缺陷的

CFTR

genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of

基因——父母各一个——会导致CF，这些突变可以通过基因测试来识别。虽然有许多不同类型的

CFTR

囊性纤维化跨膜电导调节因子 (CFTR)

mutations that can cause the disease, the vast majority of people with CF have at least one

可能导致这种疾病的突变，绝大多数CF患者至少有一个

F508del

mutation.

突变。

CFTR

mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients.

突变通过导致CFTR蛋白功能缺陷或使细胞表面的CFTR蛋白缺乏或缺失，从而引发囊性纤维化（CF）。CFTR蛋白的功能缺陷和/或缺失会导致多个器官中的细胞盐分和水分流动不畅。在肺部，这会引起异常浓稠、粘稠的黏液积聚，慢性肺部感染以及逐渐加重的肺损伤，最终导致许多患者死亡。

The median age of death is in the 30s, but with treatment, projected survival is improving..

死亡的中位年龄在30多岁，但通过治疗，预计存活率正在提高。

Today

今天

Vertex

顶点

CF medicines are treating over 75,000 people with CF in more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.

囊性纤维化药物正在六大洲的60多个国家治疗超过75,000名囊性纤维化患者。这占符合CFTR调节剂治疗条件的已确诊患者的三分之二。

Sweat chloride is used to diagnose CF, which measures CFTR function. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of a .

汗液氯化物用于诊断囊性纤维化（CF），其测量CFTR功能。CF的诊断阈值为汗氯（SwCl）≥60 mmol/L，而30-59之间的水平表明可能存在CF，需要进一步检测以确诊。携带一个拷贝基因的人通常汗氯（SwCl）水平<30 mmol/L。

CFTR

囊性纤维化跨膜电导调节因子

gene mutation but do not have any manifestation of disease (carriers). At a population level, higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. SwCl levels below 60 mmol/L are associated with improved outcomes such as better and more stable lung function, fewer pulmonary exacerbations, better quality of life and improved survival.

基因突变但没有疾病表现（携带者）。在群体水平上，较高的SwCl水平与更严重的疾病相关。恢复CFTR功能可导致较低的SwCl水平。低于60 mmol/L的SwCl水平与改善的结果相关，例如更好的、更稳定的肺功能、较少的肺部恶化、更好的生活质量和提高的生存率。

Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for .

恢复SwCl水平低于30 mmol/L一直是最终的治疗目标。

Vertex

顶点

, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease.

，因为低于30 mmol/L的水平被认为是正常的，且是无症状的CF携带者的典型特征。

About ALYFTREK

关于ALYFTREK

(deutivacaftor/tezacaftor/vanzacaftor)

(德卢卡福/特扎卡福/万扎卡福)

In people with CF, mutations in the

在囊性纤维化（CF）患者中，基因突变

CFTR

囊性纤维化跨膜电导调节因子

gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane..

基因导致细胞表面CFTR蛋白通道的数量和/或功能减少。万扎卡夫托和特扎卡夫托旨在通过促进CFTR蛋白的加工和运输来增加细胞表面CFTR蛋白的数量。德替伐考托是一种增效剂，旨在增加递送至细胞表面的CFTR蛋白的通道开放概率，以改善盐和水在细胞膜上的流动。

ALYFTREK is approved in the EU for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (

ALYFTREK在欧盟被批准用于治疗6岁及以上至少带有一个非I类突变的囊性纤维化跨膜电导调节因子（CFTR）的囊性纤维化（CF）患者。

CFTR

囊性纤维化跨膜传导调节因子

) gene.

) 基因。

For complete product information, please see the Summary of Product Characteristics (SmPC) that can be found at

有关完整的产品信息，请参阅可在以下位置找到的产品特性概要 (SmPC)：

www.ema.europa.eu.

ALYFTREK is currently licensed in the

ALYFTREK 目前已获得许可在

U.S.

美国

, the

，这个

英国

and the

和

European Union

欧盟

and is under regulatory review in

并且正在接受监管审查

Canada

加拿大

，

Switzerland

瑞士

，

Australia and New Zealand

澳大利亚和新西兰

。

About

关于

Vertex

顶点

Vertex

顶点

is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas.

是一家全球生物技术公司，致力于投资科学创新，为患有严重疾病和病症的人们创造变革性的药物。该公司已获批的疗法包括囊性纤维化、镰状细胞病、依赖输血的β地中海贫血和急性疼痛，并继续在这些领域推进临床和研究项目。

。

Vertex

顶点

also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1..

此外，在其他严重疾病领域，该公司也拥有强大的临床研究治疗管道，涵盖多种治疗模式，这些疾病包括神经性疼痛、APOL1介导的肾病、IgA肾病、原发性膜性肾病、常染色体显性多囊肾病、1型糖尿病和1型肌强直性营养不良等，该公司对这些疾病的因果人类生物学有深入的认识。

Vertex

顶点

was founded in 1989 and has its global headquarters in

成立于1989年，其全球总部位于

Boston

波士顿

, with international headquarters in

，国际总部位于

London

伦敦

. Additionally, the company has research and development sites and commercial offices in

此外，该公司在

North America

北美

，

Europe

欧洲

，

Australia

澳大利亚

，

Latin America

拉丁美洲

and the

和

Middle East

中东

。

Vertex

顶点

is consistently recognized as one of the industry's top places to work, including 15 consecutive years on

连续15年被评为行业最佳工作场所之一，包括

Science magazine's

科学杂志的

Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit

顶级雇主名单和《财富》杂志最佳工作场所100强之一。如需了解公司最新动态并深入探索Vertex的创新历史，请访问

www.vrtx.com/en-global

www.vrtx.com/zh-global

。

Special Note Regarding Forward-Looking Statements

特别提示前瞻性陈述相关注意事项

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements made by

本新闻稿包含1995年《私人证券诉讼改革法案》（经修订）所定义的前瞻性陈述，包括但不限于以下声明

Reshma Kewalramani

杨舒玛·凯沃尔拉曼尼

, M.D., and

，医学博士，和

Professor Marcus A. Mall

马库斯·A·马尔教授

, M.D., in this press release and statements regarding the expectations for the potential benefits of ALYFTREK, expectations for the eligible patient population in the EU, expectations for patient access to deutivacaftor/tezacaftor/vanzacaftor shortly following regulatory approval by the

，医学博士，在本新闻稿中以及关于ALYFTREK潜在益处的预期、欧盟符合资格的患者群体的预期、以及在监管机构批准后不久患者将能够获取deutivacaftor/tezacaftor/vanzacaftor的预期声明中。

European Commission

欧盟委员会

, and Vertex’s continued work with reimbursement bodies across the EU member states to ensure access for all eligible patients as quickly as possible. While

，以及Vertex继续与欧盟成员国的报销机构合作，以确保所有符合条件的患者尽快获得（药物）使用。尽管

Vertex

顶点

believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements.

相信本新闻稿中包含的前瞻性陈述是准确的，但这些前瞻性陈述仅代表公司截至本新闻稿发布日期的观点，并且存在许多可能导致实际事件或结果与这些前瞻性陈述所表达或暗示的内容有重大差异的风险和不确定性。

Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading “Risk Factors” in .

这些风险和不确定性包括但不限于：公司开发项目的数据可能因安全性、有效性或其他原因而不支持其化合物的注册或进一步开发，以及“风险因素”标题下列出的其他风险。

Vertex

顶点

's annual report and in subsequent filings filed with the

的年度报告以及随后提交给

Securities and Exchange Commission

证券交易委员会

and available through the company's website at

并通过公司网站提供，网址为

www.vrtx.com

and

和