FDA Defends Call For Placebo Study In uniQure Huntington's Gene Therapy Trial

Benzinga_recent_news01:11

An official from the U.S. Food and Drug Administration (FDA) defended the agency's request for a new placebo-controlled study for uniQure N.V.’s (NASDAQ:QURE) experimental gene therapy for Huntington'...

Source Link

Disclaimer: Investing carries risk. This is not financial advice. The above content should not be regarded as an offer, recommendation, or solicitation on acquiring or disposing of any financial products, any associated discussions, comments, or posts by author or other users should not be considered as such either. It is solely for general information purpose only, which does not consider your own investment objectives, financial situations or needs. TTM assumes no responsibility or warranty for the accuracy and completeness of the information, investors should do their own research and may seek professional advice before investing.

Comments

We need your insight to fill this gap

Leave a comment

{"i18n":{"language":"en_US"},"isChannel":false,"data":{"share":"https://ttm.financial/m/news/2617522930?lang=en_US&edition=fundamental","thumbnail":"https://cdn.benzinga.com/files/images/story/2026/03/05/gene-therapy.jpeg?optimize=medium&dpr=2&auto=webp&width=240","is_english":true,"pubTime":"2026-03-06 01:11","share_image_url":"https://static.laohu8.com/e9f99090a1c2ed51c021029395664489","id":"2617522930","market":"us","top_or_hot":-1,"title":"FDA Defends Call For Placebo Study In uniQure Huntington's Gene Therapy Trial","media":"Benzinga_recent_news","content":"<div>\n<p>An official from the U.S. Food and Drug Administration (FDA) defended the agency's request for a new placebo-controlled study for uniQure N.V.’s (NASDAQ:QURE) experimental gene therapy for Huntington'...</p>\n\n<a href=\"https://www.benzinga.com/news/fda/26/03/51074641/fda-defends-call-for-placebo-study-in-uniqure-huntingtons-gene-therapy-trial\">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 Defends Call For Placebo Study In uniQure Huntington's Gene Therapy Trial</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 Defends Call For Placebo Study In uniQure Huntington's Gene Therapy Trial\n</h2>\n\n<h4 class=\"meta\">\n\n\n2026-03-06 01:11 GMT+8&nbsp;&nbsp;&nbsp;<a href=https://www.benzinga.com/news/fda/26/03/51074641/fda-defends-call-for-placebo-study-in-uniqure-huntingtons-gene-therapy-trial><strong>Benzinga_recent_news</strong></a>\n\n\n</h4>\n\n</header>\n<article>\n<div>\n<p>An official from the U.S. Food and Drug Administration (FDA) defended the agency's request for a new placebo-controlled study for uniQure N.V.’s (NASDAQ:QURE) experimental gene therapy for Huntington'...</p>\n\n<a href=\"https://www.benzinga.com/news/fda/26/03/51074641/fda-defends-call-for-placebo-study-in-uniqure-huntingtons-gene-therapy-trial\">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":"BK4139","symbol_name":"生物科技","start_time":0,"source_url":"https://www.benzinga.com/news/fda/26/03/51074641/fda-defends-call-for-placebo-study-in-uniqure-huntingtons-gene-therapy-trial","article_id":"2617522930","we_media_id":null,"thumbnails":["https://cdn.benzinga.com/files/images/story/2026/03/05/gene-therapy.jpeg?optimize=medium&dpr=2&auto=webp&width=240"],"rights":{"source":"benzinga_recent_news","url":"https://www.benzinga.com/news/fda/26/03/51074641/fda-defends-call-for-placebo-study-in-uniqure-huntingtons-gene-therapy-trial","rn_cache_url":null,"directOrigin":true},"url":"https://stock-news.laohu8.com/highlight/detail?id=2617522930","pubTimestamp":1772730660,"columns":[],"sourceInfo":{"source_id":"benzinga_recent_news","name":"Benzinga"},"weMediaInfo":null,"summary":"FDA official says uniQure's Huntington's gene therapy trial lacked proof of slowing disease, calls for randomized placebo-controlled study.","collect":0,"end_time":0,"defaultTopTitle":"benzinga.com","property":[],"viewcount":null,"language":"en","relate_stocks":{"BK4139":"生物科技","LU0320765992.SGD":"FTIF - Franklin Biotechnology Discovery A Acc SGD","LU0889565916.HKD":"FRANKLIN BIOTECHNOLOGY DISCOVERY \"A\" (HKD) ACC","LU0109394709.USD":"富兰克林生物科技新领域基金A (acc)","QURE":"uniQure N.V."},"translate_title":"FDA为看涨期权在uniQure Huntington基因治疗试验中的安慰剂研究辩护","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"QURE":1},"content_text":"An official from the U.S. Food and Drug Administration (FDA) defended the agency's request for a new placebo-controlled study for uniQure N.V.’s (NASDAQ:QURE) experimental gene therapy for Huntington's disease.\nThe FDA official noted that the company's existing trial design failed to sufficiently prove the treatment slowed disease progression.\nOn Monday, the FDA advised against using Phase 1/2 study data as primary evidence for the marketing application of AMT-130, uniQure’s investigational gene therapy for Huntington's disease.\nThe FDA strongly recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study to proceed.\nuniQure plans to request a Type B meeting in the second quarter of 2026 for further discussions.\nThe FDA official told Reuters that the agency has historically required randomized, placebo-controlled trials when evaluating potential therapies for Huntington's disease.\nAccording to the official, comparisons against external patient datasets — such as those used by uniQure — are not considered adequate evidence of clinical benefit.\nUniQure previously reported that its study results showed a statistically significant slowing of disease progression over three years using a clinical rating scale.\nThe comparison was made against external datasets of Huntington's patients rather than against a randomized placebo group.\nCiting the FDA official, Reuters noted that this type of comparison was not acceptable for determining whether the therapy truly slowed disease progression.\nIn late 2024, uniQure had said it reached an agreement with the FDA on key components for an accelerated approval application. However, the official clarified that the agency never indicated the company's existing study would be sufficient for approval.\nDuring a conference call discussing financial results, Walid Abi-Saab, uniQure's chief medical officer, described the FDA as asking the company to \"superficially drill a hole on the skull\" as part of a placebo procedure.\nThe FDA official disputed that characterization. Instead, the agency suggested a randomized trial that could use a sham procedure involving a small scalp incision, according to the official.\nOn Wednesday, Andrew Nixon, a spokesperson for the Department of Health and Human Services, also criticized uniQure's description of the FDA request on social media, calling the claim false.\nQURE Price Action: uniQure shares were up 18.81% at $10.74 at the time of publication on Thursday. The stock is near its 52-week low of $7.76, according to Benzinga Pro data.\nImage via Shutterstock\nTo add Benzinga News as your preferred source on Google, click here.","kind":"highlight","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"0","news_tag":"","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,"tenantId":"TBGLOBAL","deviceId":"web-server-community-laohu8-v3","version":"4.36.2","shortVersion":"4.36.2","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/2617522930","isCrawlerRequest":true}