Vertex公司新的囊性纤维化三联疗法获准用于NHSNew cystic fibrosis triple from Vertex backed for NHS use

动脉网2025-07-15

Vertex Pharma has added another exon-skipping therapy for cystic fibrosis to its portfolio of drugs that can be prescribed by the NHS in England and Wales for patients with the genetic disorder.Vertex...

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disorder.Vertex Pharma 已将其用于治疗囊性纤维化的外显子跳跃疗法添加到其药物组合中，这些药物可以由英格兰和威尔士的 NHS 开具给患有这种遗传病的患者。It is an alternative to older twice-daily triplet Kaftrio  but can be used in a wider population of cystic fibrosis patients, with NICE estimating it will be an option for 89% of all cystic fibrosis patients in England.它是一种替代旧的每日两次三联药物Kaftrio的选择，但可以用于更广泛的囊性纤维化患者群体，NICE估计它将成为英格兰所有囊性纤维化患者中89%的可选方案。","collect":0,"end_time":0,"defaultTopTitle":"qq.com","property":[],"viewcount":null,"language":"zh","relate_stocks":{"LU2362541273.HKD":"WELLINGTON NEXT GENERATION GLOBAL EQUITY \"A\" (HKD) ACC","BK4023":"应用软件","SG9999001176.USD":"United Global Healthcare Acc USD","LU1585245621.USD":"EASTSPRING INV GLOBAL LOW VOLATILITY EQUITY FUND \"A\" (USD) ACC B","LU1430594728.SGD":"Eastspring Investments - Global Low Volatility Equity AS SGD","LU1035775433.USD":"AB SICAV I - AMERICAN GROWTH PORTFOLIO \"AD\" (USD) INC","LU0289739699.SGD":"AB INTERNATIONAL HEALTH CARE PORTFOLIO \"A\" (SGD) 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disorder.Vertex Pharma 已将其用于治疗囊性纤维化的外显子跳跃疗法添加到其药物组合中，这些药物可以由英格兰和威尔士的 NHS 开具给患有这种遗传病的患者。Reimbursement authority NICE has backed routine NHS use of Vertex's once-daily triple therapy Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) for all eligible individuals with cystic fibrosis aged six and over who have at least one F508del mutation or another responsive mutation in the CFTR gene, which is faulty in the disease..报销机构 NICE 支持在 NHS 中常规使用 Vertex 公司的每日一次三联疗法 Alyftrek（deutivacaftor/tezacaftor/vanzacaftor），适用于所有符合条件的六岁及以上囊性纤维化患者，这些患者至少携带一个 F508del 突变或 CFTR 基因中的其他响应突变，该基因在疾病中存在缺陷。Vertex's strategy inVertex的战略在于cystic fibrosis囊性纤维化has always been to progressively improve its therapies, based on compounds that modulate the activity of CFTR, to extend the range of mutations covered by the drugs and provide greater efficacy, and Alyftrek fits squarely into that mould.始终逐步改进其基于调节CFTR活性的化合物的疗法，扩大药物覆盖的突变范围并提供更大的疗效，而Alyftrek正好符合这一模式。It is an alternative to older twice-daily triplet Kaftrio (elexacaftor/tezacaftor/ivacaftor) but can be used in a wider population of cystic fibrosis patients, with NICE estimating it will be an option for 89% of all cystic fibrosis patients in England.它是一种替代旧的每日两次三联药物Kaftrio（依伐卡托/特卡托/伊伐卡托）的选择，但可以用于更广泛的囊性纤维化患者群体，NICE估计它将成为英格兰所有囊性纤维化患者中89%的可选方案。In trials, Alyftrek matched its predecessor at improving lung function scores but also improved on its ability to lower chloride in sweat, a biomarker for disease severity.在试验中，Alyftrek 在改善肺功能评分方面与其前代药物相当，但在降低汗液中的氯化物（疾病严重程度的生物标志物）能力上有所提升。In a statement, NICE said: 'Evidence suggests Alyftrek is as effective as Kaftrio, with similar costs, following a commercial agreement between NHS England and Vertex.'NICE 在一份声明中表示：“有证据表明，在 NHS 英格兰与 Vertex 达成商业协议后，Alyftrek 与 Kaftrio 的效果相当，且成本相似。”Alyftrek was approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in March based on the results of the SKYLINE 102 and SKYLINE 103 trials.Alyftrek于三月基于SKYLINE 102和SKYLINE 103试验的结果获得了药品和健康产品监管局（MHRA）的批准。The data revealed that the new triple was significantly more effective at achieving a sweat chloride level below 60 mmol/L, which is the threshold for a cystic fibrosis diagnosis, with 86% of patients on the new drug hitting that target at week 25 versus 77% of the Kaftrio group.数据显示，新的三联疗法在实现低于60 mmol/L的汗液氯化物水平（这是囊性纤维化的诊断阈值）方面显著更有效，使用新药的患者中有86%在第25周达到该目标，而使用Kaftrio的患者中这一比例为77%。NICE's decision has been welcomed by Prof Kevin Southern, chair of the UK Cystic Fibrosis Medical Association (CFMA), who said Alyftrek 'may offer an important alternative for some people with CF who are already treated with Kaftrio, or be an option for a small number of people who are not eligible for current therapies or have not been able to tolerate them.'.NICE的决定受到了英国囊性纤维化医学会（CFMA）主席凯文·索瑟恩教授的欢迎，他表示Alyftrek“可能为一些已经接受Kaftrio治疗的CF患者提供重要的替代方案，或者为少数不符合现有治疗条件或无法耐受现有治疗的患者提供选择。”Kaftrio made more than $10 billion in global sales last year, accounting for more than 90% of Vertex's revenues. So far the company has not divulged sales of Alyftrek, which was approved in the US, its first market, last December, and also got the green light in the EU in April.去年，Kaftrio的全球销售额超过100亿美元，占Vertex公司收入的90%以上。到目前为止，该公司尚未披露Alyftrek的销售情况，该药物于去年12月在美国（其首个市场）获批，并于今年4月在欧盟获得批准。In the EU, eligible patients in Ireland, Denmark, and Germany will be the first ones to access the new drug after the conclusion of reimbursement negotiations.在欧盟，爱尔兰、丹麦和德国的符合条件的患者将在报销谈判结束后成为最先获得这种新药的人群。Ludovic Fenaux, senior vice president of Vertex International, said the company is 'pleased to have reached this agreement with NHS England that recognises the value that this new medicine brings to CF patients, their families and society.'卢多维奇·费诺克斯，Vertex国际公司的高级副总裁表示，“公司很高兴能与英国国家医疗服务体系达成这项协议，该协议认可了这种新药为囊性纤维化患者、他们的家庭以及社会带来的价值。”Vertex is currently enrolling and dosing subjects in a phase 3 study of Alyftrek in children aged two to five years with cystic fibrosis in the hope of further broadening the drug's label.Vertex公司目前正在招募并给2至5岁患有囊性纤维化的儿童进行Alyftrek的三期临床试验，希望进一步拓宽该药物的适应症范围。","kind":"news","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"1","news_tag":"productRelease","news_rank":0,"isVideo":false,"video":null,"symbols":[],"gpt_button":1,"need_auth":false,"need_login_tip":false,"code":"91000000","status":"200"},"commentList":[],"isCommentEnd":true,"newsSizeData":{"likeSize":0,"commentSize":0,"repostSize":0,"favoriteSize":0,"likeStatus":false,"favoriteStatus":false},"APP":{"userAgent":"Mozilla/5.0 AppleWebKit/537.36 (KHTML, like Gecko; compatible; ClaudeBot/1.0; +claudebot@anthropic.com)","isDev":false,"isTTM":false,"tenantId":"TBCN","deviceId":"web-server-community-laohu8-v3","version":"4.43.1","shortVersion":"4.43.1","platform":"web","vendor":"web","appName":"laohu8","isIOS":false,"isAndroid":false,"isTiger":false,"isTHS":false,"isWeiXin":false,"isWeiXinMini":false,"isWeiBo":false,"isQQ":false,"isBaiduSwan":false,"isBaiduBox":false,"isDingTalk":false,"isToutiao":false,"isOnePlus":false,"isHuaWei":false,"isXiaomi":false,"isXiaomiWebView":false,"isOppo":false,"isVivo":false,"isSamsung":false,"isMobile":false},"href":"/m/news/2551193492","isCrawlerRequest":true}