福泰制药新一代囊性纤维化疗法申报上市

医药魔方07-03

7月2日，福泰制药（Vertex）宣布新一代三联疗法（vanzacaftor/tezacaftor/deutivacaftor，简称vanza三联疗法）用于治疗囊性纤维化（CF）的新药申请（NDA）获FDA受理并被纳入优先审评名单，PDUFA日期为2025年1月2日。此次NDA主要是基于3项III期研究的积极结果。RIDGELINE 105研究该研究是一项单臂、开放标签临床试验（n=78），评估了...

网页链接

免责声明：本文观点仅代表作者个人观点，不构成本平台的投资建议，本平台不对文章信息准确性、完整性和及时性做出任何保证，亦不对因使用或信赖文章信息引发的任何损失承担责任。

精彩评论

我们需要你的真知灼见来填补这片空白

发表看法

{"i18n":{"language":"zh_CN"},"isChannel":false,"data":{"share":"https://www.laohu8.com/m/news/2448197380?lang=zh_CN&edition=full","thumbnail":"","is_english":false,"pubTime":"2024-07-03 05:31","share_image_url":"https://static.laohu8.com/9a95c1376e76363c1401fee7d3717173","id":"2448197380","market":"us","top_or_hot":-1,"title":"福泰制药新一代囊性纤维化疗法申报上市","media":"医药魔方","content":"<div>\n<p>7月2日，福泰制药（Vertex）宣布新一代三联疗法（vanzacaftor/tezacaftor/deutivacaftor，简称vanza三联疗法）用于治疗囊性纤维化（CF）的新药申请（NDA）获FDA受理并被纳入优先审评名单，PDUFA日期为2025年1月2日。此次NDA主要是基于3项III期研究的积极结果。RIDGELINE 105研究该研究是一项单臂、开放标签临床试验（n=78），评估了...</p>\n\n<a href=\"http://gu.qq.com/resources/shy/news/detail-v2/index.html#/?id=nesSN202407030533369ef739ed&s=b\">网页链接</a>\n\n</div>\n","source":"tencent","html":"<!DOCTYPE html>\n<html>\n<head>\n<meta http-equiv=\"Content-Type\" content=\"text/html; charset=utf-8\" />\n<meta name=\"viewport\" content=\"width=device-width,initial-scale=1.0,minimum-scale=1.0,maximum-scale=1.0,user-scalable=no\"/>\n<meta name=\"format-detection\" content=\"telephone=no,email=no,address=no\" />\n<title>福泰制药新一代囊性纤维化疗法申报上市</title>\n<style type=\"text/css\">\na,abbr,acronym,address,applet,article,aside,audio,b,big,blockquote,body,canvas,caption,center,cite,code,dd,del,details,dfn,div,dl,dt,\nem,embed,fieldset,figcaption,figure,footer,form,h1,h2,h3,h4,h5,h6,header,hgroup,html,i,iframe,img,ins,kbd,label,legend,li,mark,menu,nav,\nobject,ol,output,p,pre,q,ruby,s,samp,section,small,span,strike,strong,sub,summary,sup,table,tbody,td,tfoot,th,thead,time,tr,tt,u,ul,var,video{ font:inherit;margin:0;padding:0;vertical-align:baseline;border:0 }\nbody{ font-size:16px; line-height:1.5; color:#999; background:transparent; }\n.wrapper{ overflow:hidden;word-break:break-all;padding:10px; }\nh1,h2{ font-weight:normal; line-height:1.35; margin-bottom:.6em; }\nh3,h4,h5,h6{ line-height:1.35; margin-bottom:1em; }\nh1{ font-size:24px; }\nh2{ font-size:20px; }\nh3{ font-size:18px; }\nh4{ font-size:16px; }\nh5{ font-size:14px; }\nh6{ font-size:12px; }\np,ul,ol,blockquote,dl,table{ margin:1.2em 0; }\nul,ol{ margin-left:2em; }\nul{ list-style:disc; }\nol{ list-style:decimal; }\nli,li p{ margin:10px 0;}\nimg{ max-width:100%;display:block;margin:0 auto 1em; }\nblockquote{ color:#B5B2B1; border-left:3px solid #aaa; padding:1em; }\nstrong,b{font-weight:bold;}\nem,i{font-style:italic;}\ntable{ width:100%;border-collapse:collapse;border-spacing:1px;margin:1em 0;font-size:.9em; }\nth,td{ padding:5px;text-align:left;border:1px solid #aaa; }\nth{ font-weight:bold;background:#5d5d5d; }\n.symbol-link{font-weight:bold;}\n/* header{ border-bottom:1px solid #494756; } */\n.title{ margin:0 0 8px;line-height:1.3;color:#ddd; }\n.meta {color:#5e5c6d;font-size:13px;margin:0 0 .5em; }\na{text-decoration:none; color:#2a4b87;}\n.meta .head { display: inline-block; overflow: hidden}\n.head .h-thumb { width: 30px; height: 30px; margin: 0; padding: 0; border-radius: 50%; float: left;}\n.head .h-content { margin: 0; padding: 0 0 0 9px; float: left;}\n.head .h-name {font-size: 13px; color: #eee; margin: 0;}\n.head .h-time {font-size: 11px; color: #7E829C; margin: 0;line-height: 11px;}\n.small {font-size: 12.5px; display: inline-block; transform: scale(0.9); -webkit-transform: scale(0.9); transform-origin: left; -webkit-transform-origin: left;}\n.smaller {font-size: 12.5px; display: inline-block; transform: scale(0.8); -webkit-transform: scale(0.8); transform-origin: left; -webkit-transform-origin: left;}\n.bt-text {font-size: 12px;margin: 1.5em 0 0 0}\n.bt-text p {margin: 0}\n</style>\n</head>\n<body>\n<div class=\"wrapper\">\n<header>\n<h2 class=\"title\">\n福泰制药新一代囊性纤维化疗法申报上市\n</h2>\n\n<h4 class=\"meta\">\n\n\n2024-07-03 05:31 北京时间&nbsp;&nbsp;&nbsp;<a href=http://gu.qq.com/resources/shy/news/detail-v2/index.html#/?id=nesSN202407030533369ef739ed&s=b><strong>医药魔方</strong></a>\n\n\n</h4>\n\n</header>\n<article>\n<div>\n<p>7月2日，福泰制药（Vertex）宣布新一代三联疗法（vanzacaftor/tezacaftor/deutivacaftor，简称vanza三联疗法）用于治疗囊性纤维化（CF）的新药申请（NDA）获FDA受理并被纳入优先审评名单，PDUFA日期为2025年1月2日。此次NDA主要是基于3项III期研究的积极结果。RIDGELINE 105研究该研究是一项单臂、开放标签临床试验（n=78），评估了...</p>\n\n<a href=\"http://gu.qq.com/resources/shy/news/detail-v2/index.html#/?id=nesSN202407030533369ef739ed&s=b\">网页链接</a>\n\n</div>\n\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"LU0889565916.HKD","symbol_name":"FRANKLIN BIOTECHNOLOGY DISCOVERY \"A\" (HKD) ACC","start_time":0,"source_url":"http://gu.qq.com/resources/shy/news/detail-v2/index.html#/?id=nesSN202407030533369ef739ed&s=b","article_id":"2448197380","we_media_id":null,"thumbnails":[],"rights":{"source":"tencent","url":"http://gu.qq.com/resources/shy/news/detail-v2/index.html#/?id=nesSN202407030533369ef739ed&s=b","rn_cache_url":null,"customStyle":"body{padding-top:10px;}#news_title{font-weight:bold;#titleStyle#;}#news_description span{font-size:12px;#descriptionStyle#;}.footer-note{#statement#}","selectors":".mod-LoadTzbdNews, body","filters":".relate-stock, .hot-list, .recom-box, .wx-sou","directOrigin":true},"url":"https://stock-news.laohu8.com/highlight/detail?id=2448197380","pubTimestamp":1719955886,"sourceInfo":{"source_id":"tencent","name":"腾讯"},"weMediaInfo":null,"summary":"结果显示，vanza三联疗法在6-11岁CF患者与12岁及以上CF患者中的安全性相似。结果显示，在ppFEV1主要终点方面，vanza三联疗法非劣效于Trikafta。患者的中位死亡年龄为30多岁，但若得到恰当治疗，其生存率可得到提高。","collect":0,"end_time":0,"defaultTopTitle":"qq.com","property":[],"viewcount":null,"language":"zh","relate_stocks":{"LU0889565916.HKD":"FRANKLIN BIOTECHNOLOGY DISCOVERY \"A\" (HKD) ACC","LU2362541513.USD":"WELLINGTON NEXT GENERATION GLOBAL EQUITY \"A\" (USD) ACC","BK4532":"文艺复兴科技持仓","IE00B894F039.SGD":"Legg Mason ClearBridge - US Aggressive Growth A Acc SGD-H","LU0234570918.USD":"高盛全球核心股票组合Acc Close","VRTX":"福泰制药","BK4567":"ESG概念","LU0417517546.SGD":"Allianz US Equity Cl AT Acc SGD","IE00B19Z3581.USD":"Legg Mason ClearBridge - Value A Acc USD","BK4585":"ETF&股票定投概念","LU0079474960.USD":"联博美国增长基金A","BK4139":"生物科技","IE0009355771.USD":"骏利亨德森环球生命科技A Acc","BK4533":"AQR资本管理(全球第二大对冲基金)","LU0320765992.SGD":"FTIF - Franklin Biotechnology Discovery A Acc SGD","LU0289739699.SGD":"AB INTERNATIONAL HEALTH CARE PORTFOLIO \"A\" (SGD) ACC","IE00B19Z3B42.SGD":"Legg Mason ClearBridge - Value A Acc SGD","IE00B2B36J28.USD":"JANUS HENDERSON GLOBAL LIFE SCIENCES \"I1\" (USD) INC","IE00BJT1NW94.SGD":"JANUS HENDERSON GLOBAL LIFE SCIENCES \"A2\" (SGDHDG) ACC","LU0109394709.USD":"富兰克林生物科技新领域基金A (acc)","LU2362540622.SGD":"WELLINGTON NEXT GENERATION GLOBAL EQUITY \"A\" (SGDHDG) ACC","IE00B19Z9P08.USD":"LEGG MASON CLEARBRIDGE US AGGRESSIVE GROWTH \"A\" (USD) INC","LU0058720904.USD":"联博国际健康护理基金A","BK4588":"碎股","LU2237438978.USD":"Amundi Funds US Pioneer A2 (C) USD","IE00B19Z9Z06.USD":"Legg Mason ClearBridge - US Aggressive Growth A Acc USD","LU1430594728.SGD":"Eastspring Investments - Global Low Volatility Equity AS SGD","LU0256863811.USD":"ALLIANZ US EQUITY \"A\" INC","IE0002270589.USD":"LEGG MASON CLEARBRIDGE VALUE \"A\" (USD) INC","LU0061475181.USD":"THREADNEEDLE (LUX) AMERICAN \"AU\" (USD) ACC","IE0002141913.USD":"JANUS HENDERSON GLOBAL LIFE SCIENCES \"I2\" (USD) ACC","IE00BJJMRZ35.SGD":"JANUS HENDERSON GLOBAL LIFE SCIENCES \"I2\" (SGDHDG) ACC","IE00BFTCPJ56.SGD":"Janus Henderson Global Life Sciences A Acc SGD","LU2362541273.HKD":"WELLINGTON NEXT GENERATION GLOBAL EQUITY \"A\" (HKD) ACC","LU1585245621.USD":"EASTSPRING INV GLOBAL LOW VOLATILITY EQUITY FUND \"A\" (USD) ACC B"},"translate_title":"Futai Pharmaceutical's new generation of cystic fibrosis therapy declared for marketing","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"VRTX":1},"content_text":"7月2日，福泰制药（Vertex）宣布新一代三联疗法（vanzacaftor/tezacaftor/deutivacaftor，简称vanza三联疗法）用于治疗囊性纤维化（CF）的新药申请（NDA）获FDA受理并被纳入优先审评名单，PDUFA日期为2025年1月2日。此次NDA主要是基于3项III期研究的积极结果。RIDGELINE 105研究该研究是一项单臂、开放标签临床试验（n=78），评估了vanza三联疗法治疗6-11岁CF患者的长期疗效和安全性。研究的主要终点为安全性。结果显示，vanza三联疗法在6-11岁CF患者与12岁及以上CF患者中的安全性相似。此外，治疗24周，95%患者的汗液氯化物（SwCl）水平低于60mmol/L，53%患者的SwCl水平低于30mmol/L。SKYLINE 102研究该研究是一项随机、双盲、阳性药物对照临床试验（n=398），评估了vanza三联疗法对比Trikafta（elexacaftor/tezacaftor/ivacaftor）治疗①存在F508del纯合子突变或杂合子突变且存在门控功能或残余功能突变或②不存在F508del突变且至少对一种囊性纤维化跨膜传导调节因子（CFTR）突变有反应的12岁及以上CF患者的疗效和安全性。研究的主要终点为预期一秒用力呼气量百分比（ppFEV1）相较于基线的绝对变化值。结果显示，在ppFEV1主要终点方面，vanza三联疗法非劣效于Trikafta。此外，多项关键次要终点达到了优效性标准。SKYLINE 103研究该研究是一项随机、双盲、阳性药物对照临床试验（n=573），评估了vanza三联疗法对比Trikafta（elexacaftor/tezacaftor/ivacaftor）治疗①存在F508del纯合子突变或杂合子突变且存在门控功能或残余功能突变或②不存在F508del突变且至少对一种囊性纤维化跨膜传导调节因子（CFTR）突变有反应的12岁及以上CF患者的疗效和安全性。研究的主要终点为ppFEV1相较于基线的绝对变化值。结果显示，在ppFEV1主要终点方面，vanza三联疗法非劣效于Trikafta。此外，多项关键次要终点达到了优效性标准。CF是一种罕见的、进行性、缩短寿命的遗传病，累及肺、肝、胰腺、胃肠道、鼻窦、汗腺和生殖道。全球有超过9.2万例CF患者。CF由CFTR基因中的某些突变导致CFTR蛋白功能缺陷和/或细胞表面CFTR蛋白缺失而引起。儿童必须遗传两个有缺陷的CFTR基因（父母双方各一个）才会患病，这些突变可以通过基因检测识别。虽然有许多不同类型的CFTR突变均可导致CF，但绝大多数CF患者至少存在一个F508del突变。CFTR蛋白功能缺陷和/或CFTR蛋白缺失会导致盐和水在许多器官的细胞中流入和流出过程不畅。若这种情况在肺部发生，会导致肺部粘液聚集、慢性肺部感染和进行性肺损伤，最终导致患者死亡。患者的中位死亡年龄为30多岁，但若得到恰当治疗，其生存率可得到提高。Vanza三联疗法中的vanzacaftor和tezacaftor旨在通过促进CFTR蛋白的加工和运输，增加细胞表面CFTR蛋白数量，而deutivacaftor是旨在增加送达细胞表面的CFTR蛋白的通道开放概率，以改善细胞膜上盐和水的流动。Copyright © 2024 PHARMCUBE. All Rights Reserved.欢迎转发分享及合理引用，引用时请在显要位置标明文章来源；如需转载，请给微信公众号后台留言或发送消息，并注明公众号名称及ID。免责申明：本微信文章中的信息仅供一般参考之用，不可直接作为决策内容，医药魔方不对任何主体因使用本文内容而导致的任何损失承担责任。","kind":null,"is_publish_news":true,"is_publish_highlight":false,"is_publish_live":null,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"0","news_tag":"","news_rank":0,"symbols":[],"gpt_button":1},"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,"deviceId":"web-server-community-laohu8-v3","version":"4.25.1","shortVersion":"4.25.1","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/2448197380"}