FDA's Rigid Approach Adds Uncertainty To uniQure's Accelerated Pathway For Huntington's Gene Therapy

Benzinga_recent_news11-05

The U.S. Food and Drug Administration (FDA) on Monday provided feedback to uniQure N.V. (NASDAQ:QURE) regarding its investigational gene therapy for Huntington's disease. uniQure believes that the FDA...

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(NASDAQ:QURE) regarding its investigational gene therapy for Huntington's disease.\nuniQure believes that the FDA...</p>\n\n<a href=\"https://www.benzinga.com/analyst-stock-ratings/downgrades/25/11/48633448/fdas-rigid-approach-adds-uncertainty-to-uniqures-accelerated-pathway-for-huntingtons-gene-therapy\">Source Link</a>\n\n</div>\n","source":"benzinga_recent_news","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>FDA's Rigid Approach Adds Uncertainty To uniQure's Accelerated Pathway For Huntington's Gene Therapy</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\">\nFDA's Rigid Approach Adds Uncertainty To uniQure's Accelerated Pathway For Huntington's Gene Therapy\n</h2>\n\n<h4 class=\"meta\">\n\n\n2025-11-05 03:25 GMT+8&nbsp;&nbsp;&nbsp;<a href=https://www.benzinga.com/analyst-stock-ratings/downgrades/25/11/48633448/fdas-rigid-approach-adds-uncertainty-to-uniqures-accelerated-pathway-for-huntingtons-gene-therapy><strong>Benzinga_recent_news</strong></a>\n\n\n</h4>\n\n</header>\n<article>\n<div>\n<p>The U.S. Food and Drug Administration (FDA) on Monday provided feedback to uniQure N.V. (NASDAQ:QURE) regarding its investigational gene therapy for Huntington's disease.\nuniQure believes that the FDA...</p>\n\n<a href=\"https://www.benzinga.com/analyst-stock-ratings/downgrades/25/11/48633448/fdas-rigid-approach-adds-uncertainty-to-uniqures-accelerated-pathway-for-huntingtons-gene-therapy\">Source Link</a>\n\n</div>\n\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"NGNE","symbol_name":"NEUROGENE INC","start_time":0,"source_url":"https://www.benzinga.com/analyst-stock-ratings/downgrades/25/11/48633448/fdas-rigid-approach-adds-uncertainty-to-uniqures-accelerated-pathway-for-huntingtons-gene-therapy","article_id":"2581763453","we_media_id":null,"thumbnails":["https://cdn.benzinga.com/files/images/story/2025/11/04/Concept-Of-Huntingtons-Disease-Write-On-.jpeg?optimize=medium&dpr=2&auto=webp&width=240"],"rights":{"source":"benzinga_recent_news","url":"https://www.benzinga.com/analyst-stock-ratings/downgrades/25/11/48633448/fdas-rigid-approach-adds-uncertainty-to-uniqures-accelerated-pathway-for-huntingtons-gene-therapy","rn_cache_url":null,"directOrigin":true},"url":"https://stock-news.laohu8.com/highlight/detail?id=2581763453","pubTimestamp":1762284300,"columns":[],"sourceInfo":{"source_id":"benzinga_recent_news","name":"Benzinga"},"weMediaInfo":null,"summary":"William Blair notes FDA's tougher stance on gene therapy programs as AMT-130 faces uncertainty in its accelerated approval timeline.","collect":0,"end_time":0,"defaultTopTitle":"benzinga.com","property":[],"viewcount":null,"language":"en","relate_stocks":{"NGNE":"NEUROGENE INC","LU0109394709.USD":"富兰克林生物科技新领域基金A (acc)","NVIW.SI":"NVDA 3xLongSG261006",".IXIC":"NASDAQ Composite","QLD":"2倍做多纳斯达克100指数ETF-ProShares","QURE":"uniQure N.V.","LU0889565916.HKD":"FRANKLIN BIOTECHNOLOGY DISCOVERY \"A\" (HKD) ACC","RCKT":"Rocket Pharmaceuticals, Inc.","PSQ":"做空纳斯达克100指数ETF-ProShares","CABA":"Cabaletta Bio","QID":"两倍做空纳斯达克指数ETF-ProShares","TSYW.SI":"TESLA 3xLongSG261006","USJW.SI":"ALPHAB 3xLongSG261006","BK4139":"生物科技","KYTX":"Kyverna Therapeutics","SQQQ":"纳指三倍做空ETF","USAW.SI":"AMZN 3xLongSG261006","LU0320765992.SGD":"FTIF - Franklin Biotechnology Discovery A Acc SGD","TQQQ":"纳指三倍做多ETF"},"translate_title":"FDA的僵化做法给uniQure亨廷顿基因治疗加速路径增加了不确定性","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"USJW.SI":0.6,"SQQQ":0.6,"KYTX":1,"TSYW.SI":0.6,"NVIW.SI":0.6,"QLD":0.6,"PSQ":0.6,"CABA":1,".IXIC":0.9,"USAW.SI":0.6,"RCKT":1,"QURE":1,"QID":0.6,"NQmain":0.6,"MNQmain":0.6,"NGNE":1,"TQQQ":0.6},"content_text":"The U.S. Food and Drug Administration (FDA) on Monday provided feedback to uniQure N.V. (NASDAQ:QURE) regarding its investigational gene therapy for Huntington's disease.\nuniQure believes that the FDA currently disagrees that the data from the Phase 1/2 studies of AMT-130, in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence in support of a BLA submission.\nAlso Read: FDA Outlines Process To Speed Up Rare Disease Therapy Approvals\nWilliam Blair highlighted that the key difference between this meeting and the company's prior meetings with the FDA is that Nicole Verdun was not present. Vinay Prasad was also not in the meeting.\nThe analyst downgraded uniQure from Outperform to Market Perform.\nInvestors should note that Verdun, the director of the FDA's Center for Biologics Evaluation and Research, was placed on administrative leave and escorted out of the agency.\nAnalyst Sami Corwin noted that the FDA raised no issues with the previously agreed pathway when the company submitted its final Statistical Analysis Plan after Dr. Verdun's exit.\nCorwin added that while management remains focused on securing an expedited path for AMT-130, the timing of its BLA submission is now uncertain.\nThe FDA's commentary on AMT-130 suggests that the agency is being more stringent in regulating gene and cell therapy programs for rare diseases.\nThe update could put negative pressure on other gene/cell therapy companies that previously aligned on single-arm trials, including Neurogene Inc. (NASDAQ:NGNE), Cabaletta Bio Inc. (NASDAQ:CABA), Kyverna Therapeutics Inc. (NASDAQ:KYTX), and Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT).\nHowever, since the agency's concern centers on the SAP not being submitted before the trial began, William Blair sees minimal read-through risk for the firms under its coverage.\nQURE Price Action: uniQure shares were down 10.89% at $30.55 at the time of publication on Tuesday, according to Benzinga Pro data.\nRead Next:\nPhoto by bangoland via Shutterstock","kind":"highlight","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"-1","news_tag":"policyRegulatory","news_rank":0,"symbols":[],"gpt_button":0,"need_auth":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":true,"deviceId":"web-server-community-laohu8-v3","version":"4.35.11","shortVersion":"4.35.11","platform":"web","vendor":"web","appName":"ttm","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/2581763453","isCrawlerRequest":true}