BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for investigational once-daily vanzacaftor/tezacaftor/deutivacaftor triple combination therapy (vanza triple) for people living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene responsive to the vanza triple.

波士顿--（商业新闻短讯）--Vertex Pharmaceuticals Incorporated（Nasdaq:VRTX）今天宣布，美国食品和药物管理局（FDA）已接受其新药申请（NDA），用于研究每日一次的vanzacaftor/tezacaftor/deutivacaftor三联疗法（vanza triple），用于治疗6岁及以上囊性纤维化（CF）患者，这些患者至少有一个F508del突变或囊性纤维化跨膜电导调节剂（CFTR）基因中对vanza triple有反应的另一个反应突变。

Vertex used a priority review voucher for this submission reducing the review time from 10 months to 6 months, resulting in a Prescription Drug User Fee Act (PDUFA) target action date of January 2, 2025..

Vertex为此提交使用了优先审查凭证，将审查时间从10个月减少到6个月，从而制定了《处方药用户费用法案》（PDUFA），目标行动日期为2025年1月2日。。

“The FDA acceptance of our vanza triple application and the MAA validation by the EMA represent important milestones in the decades-long development of CFTR modulators and another example of our track record of serial innovation in CF,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex.

Vertex执行副总裁兼首席监管和质量官Nia Tatsis博士说：“FDA接受我们的vanza triple应用程序和EMA的MAA验证代表了CFTR调制器数十年发展的重要里程碑，也是我们CF系列创新记录的另一个例子。”。

“Vanzacaftor raises the high bar set by TRIKAFTA® and gives more people with CF the chance to get to levels of sweat chloride below the diagnostic threshold for CF, and even to levels of sweat chloride seen in those without CF.”.

“Vanzacaftor提高了TRIKAFTA®设定的高标准，使更多CF患者有机会达到低于CF诊断阈值的汗液氯化物水平，甚至达到无CF患者的汗液氯化物水平。”。

Vertex also received validation of its Marketing Authorization Application (MAA) submission by the European Medicines Agency (EMA) in the EU for patients ages 6 years and older. The company has also submitted in Canada, Australia, Switzerland and the U.K.

Vertex还收到了欧盟欧洲药品管理局（EMA）针对6岁及以上患者提交的上市授权申请（MAA）的验证。该公司还在加拿大、澳大利亚、瑞士和英国提交了。

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。。

Today Vertex CF medicines are treating over 65,000 people with CF across 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.

今天，Vertex CF药物正在治疗六大洲60个国家的65000多名CF患者。这代表了有资格接受CFTR调节剂治疗的CF患者的2/3。

Diagnosis of CF is often made by genetic testing and is confirmed by testing sweat chloride (SwCl), which measures CFTR protein dysfunction. 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.

CF的诊断通常通过基因检测进行，并通过检测汗液氯化物（SwCl）来证实，该汗液氯化物可测量CFTR蛋白功能障碍。CF的诊断阈值为SwCl≥60 mmol/L，而30-59之间的水平表明CF是可能的，可能需要更多的测试来诊断CF。

A SwCl level of <30 mmol/L is seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). 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.

携带一份CFTR基因突变但没有任何疾病表现（携带者）的人的SwCl水平低于30 mmol/L。较高水平的SwCl与更严重的疾病有关。恢复CFTR功能会导致SwCl水平降低。低于60 mmol/L的SwCl水平与改善的结果相关，例如更好和更稳定的肺功能，更少的肺恶化，更好的生活质量和改善的生存率。

Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease..

长期以来，将SwCl水平恢复到30 mmol/L以下一直是Vertex的最终治疗目标，因为低于30 mmol/L的水平被认为是正常的，并且是没有疾病的CF携带者的典型特征。。

About vanzacaftor/tezacaftor/deutivacaftor (the “vanza triple”)

关于vanzacaftor/tezacaftor/deutivacaftor（“vanza三联”）

In people with CF, mutations in the CFTR 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.

在CF患者中，CFTR基因的突变导致细胞表面CFTR蛋白通道的数量和/或功能降低。Vanzacaftor和tezacaftor旨在通过促进CFTR蛋白的加工和运输来增加细胞表面CFTR蛋白的量。

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..

Deutivacaftor是一种增强剂，旨在增加CFTR蛋白传递到细胞表面的通道开放概率，从而改善盐和水在细胞膜上的流动。。

Investigational vanzacaftor/tezacaftor/deutivacaftor was granted Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration for the treatment of cystic fibrosis.

研究性vanzacaftor/tezacaftor/deutivacaftor被美国食品和药物管理局授予快速通道和孤儿药物名称，用于治疗囊性纤维化。

The vanza triple will be subject to a meaningfully lower single-digit royalty obligation, compared to the rate payable on Vertex’s current CF portfolio.

与Vertex当前CF投资组合的应付利率相比，vanza triple将承担一位数的版税义务。

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（囊性纤维化、镰状细胞病和输血依赖性β地中海贫血）的根本原因的药物，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, IgA nephropathy, 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，IgA肾病，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。