LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label expansion of KALYDECO® (ivacaftor) for the treatment of infants down to 1 month of age with cystic fibrosis (CF) who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R..

伦敦--（商业新闻短讯）--Vertex Pharmaceuticals（纳斯达克：VRTX）今天宣布，欧盟委员会已批准扩大KALYDECO®（ivacaftor）的标签，用于治疗囊性纤维化（CF）1个月以下的婴儿，这些婴儿的囊性纤维化跨膜电导调节剂（CFTR）基因具有以下突变之一：R117H，G551D，G1244E，G1349D，G178R，G551S，S1251N，S1255P，S549N或S549R。。

“Today’s approval is an important milestone for the cystic fibrosis community. Treating CF early in life can potentially slow the progression of the disease, which is why it is so important to start treatment from a very young age,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex..

Vertex全球药物开发和医疗事务执行副总裁兼首席医疗官Carmen Bozic医学博士说：“今天的批准对于囊性纤维化社区来说是一个重要的里程碑。在生命早期治疗CF可能会减缓疾病的进展，这就是为什么从很小的时候就开始治疗如此重要。”。。

As a result of existing access agreements in Austria, Czech Republic, Denmark, Ireland, Norway, Sweden, and The Netherlands, eligible patients will have access to the expanded indication of KALYDECO® (ivacaftor) shortly following regulatory approval by the European Commission. Vertex will continue to work with reimbursement authorities across the European Union to ensure access for all other eligible patients.

根据奥地利、捷克共和国、丹麦、爱尔兰、挪威、瑞典和荷兰现有的准入协议，符合条件的患者将在欧盟委员会监管批准后不久获得扩大的KALYDECO®（ivacaftor）适应症。Vertex将继续与欧盟各地的报销机构合作，以确保所有其他符合条件的患者都能获得报销。

In the U.K., following MHRA approval at the end of 2023, and as a result of the existing reimbursement agreement between Vertex and the National Health Service, eligible infants ages 1 month and older in the U.K. have access to this expanded indication for KALYDECO® (ivacaftor)..

在英国，在2023年底MHRA批准后，由于Vertex与国家卫生服务局之间现有的报销协议，英国1个月及以上的合格婴儿可以使用KALYDECO®（ivacaftor）的扩展适应症。。

About Cystic Fibrosis

关于囊性纤维化

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. 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 CFTR gene.

囊性纤维化（CF）是一种罕见的缩短寿命的遗传疾病，影响全球92000多人。CF是一种进行性多器官疾病，会影响肺，肝，胰腺，胃肠道，鼻窦，汗腺和生殖道。CF是由CFTR基因的某些突变导致的CFTR蛋白缺陷和/或缺失引起的。

Children must inherit two defective 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 CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. 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.

儿童必须继承两个有缺陷的CFTR基因（每个父母一个）才能患有CF，这些突变可以通过基因测试来鉴定。尽管有许多不同类型的CFTR突变可导致该疾病，但绝大多数CF患者至少有一个F508del突变。CFTR突变通过导致CFTR蛋白缺陷或导致细胞表面CFTR蛋白短缺或缺失而导致CF。

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.

CFTR蛋白的功能缺陷和/或缺失会导致盐和水进出许多器官的细胞。在肺部，这会导致异常粘稠的粘液积聚，慢性肺部感染和进行性肺损伤，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..

中位死亡年龄在30多岁，但经过治疗，预计生存率正在提高。。

About KALYDECO® (ivacaftor)

关于KALYDECO®（ivacaftor）

In people with certain types of mutations in the CFTR gene, the CFTR protein at the cell surface does not function properly. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

在CFTR基因具有某些类型突变的人群中，细胞表面的CFTR蛋白功能不正常。伊瓦卡托被称为CFTR增强剂，是一种口服药物，旨在促进CFTR蛋白通过细胞膜运输盐和水的能力，从而有助于水合和清除气道粘液。

KALYDECO® (ivacaftor) was the first medicine to treat the underlying cause of cystic fibrosis in people with specific mutations in the CFTR gene..

KALYDECO®（ivacaftor）是第一种治疗CFTR基因特定突变患者囊性纤维化根本原因的药物。。

KALYDECO® (ivacaftor) is a prescription medicine for the treatment of people with CF aged at least 1 month and weighing at least 3 kg who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R..

KALYDECO®（ivacaftor）是一种处方药，用于治疗年龄至少1个月，体重至少3公斤的囊性纤维化患者，这些患者的囊性纤维化跨膜电导调节剂（CFTR）基因具有以下突变之一：R117H，G551D，G1244E，G1349D，G178R，G551S，S1251N，S1255P，S549N或S549R。。

For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.

有关完整的产品信息，请参阅www.ema.europa.eu上的产品特性摘要。

About Vertex

关于顶点

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases.

Vertex是一家全球生物技术公司，投资于科学创新，为患有严重疾病的人创造变革性药物。该公司已批准用于治疗多种慢性，缩短寿命的遗传疾病的根本原因的药物-囊性纤维化，镰状细胞病和输血依赖性β地中海贫血-并继续推进这些疾病的临床和研究计划。

Vertex 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 acute and neuropathic pain, APOL1-mediated kidney disease, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency..

Vertex还在其他严重疾病的一系列模式中拥有强大的临床研究治疗渠道，对因果人类生物学有深入的了解，包括急性和神经性疼痛，APOL1介导的肾脏疾病，常染色体显性多囊肾病，1型糖尿病，1型强直性肌营养不良和α-1抗胰蛋白酶缺乏症。。

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 14 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For.

Vertex成立于1989年，全球总部位于波士顿，国际总部位于伦敦。此外，该公司在北美、欧洲、澳大利亚、拉丁美洲和中东设有研发基地和商业办事处。Vertex一直被公认为行业最佳工作地点之一，包括连续14年入选《科学》杂志的最佳雇主名单，以及《财富》杂志的100家最佳工作公司之一。