FDA Grants Priority Review to Ultragenyx BLA for UX111 Gene Therapy for Sanfilippo Syndrome Type A

Reuters01-30

FDA Grants Priority Review to Ultragenyx BLA for UX111 Gene Therapy for Sanfilippo Syndrome Type A

Ultragenyx Pharmaceutical Inc. has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for UX111 (rebisufligene etisparvovec), an AAV9 gene therapy intended to treat patients with Sanfilippo syndrome type A (MPS IIIA). The FDA granted Priority Review to the application in February 2025. A Prescription Drug User Fee Act (PDUFA) action date is expected to be assigned within a month of resubmission, with a review period of up to six months anticipated, and a PDUFA date projected for the third quarter of 2026. If approved, UX111 would become the first therapy available for Sanfilippo syndrome type A.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Ultragenyx Pharmaceutical Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9645894-en) on January 30, 2026, and is solely responsible for the information contained therein.

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/2607204545?lang=en_US&edition=fundamental","thumbnail":"","is_english":true,"pubTime":"2026-01-30 21:30","share_image_url":"https://static.laohu8.com/e9f99090a1c2ed51c021029395664489","id":"2607204545","market":"us","top_or_hot":-1,"title":"FDA Grants Priority Review to Ultragenyx BLA for UX111 Gene Therapy for Sanfilippo Syndrome Type A","media":"Reuters","content":"<html xmlns=\"http://www.w3.org/1999/xhtml\" xmlns:newsg2=\"http://iptc.org/std/nar/2006-10-01/\" xmlns:xhtml=\"http://www.w3.org/1999/xhtml\"><head><title>\n      FDA Grants Priority Review to Ultragenyx BLA for UX111 Gene Therapy for Sanfilippo Syndrome Type A\n    </title></head><body><div xmlns:xsd=\"http://www.w3.org/2001/XMLSchema\" xmlns:xsi=\"http://www.w3.org/2001/XMLSchema-instance\">\n<p>\n        <a href=\"https://laohu8.com/S/RARE\">Ultragenyx Pharmaceutical Inc</a>. has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for UX111 (rebisufligene etisparvovec), an AAV9 gene therapy intended to treat patients with Sanfilippo syndrome type A (MPS IIIA). The FDA granted Priority Review to the application in February 2025. A Prescription Drug User Fee Act (PDUFA) action date is expected to be assigned within a month of resubmission, with a review period of up to six months anticipated, and a PDUFA date projected for the third quarter of 2026. If approved, UX111 would become the first therapy available for Sanfilippo syndrome type A.\n      </p>\n</div><div xmlns:xsd=\"http://www.w3.org/2001/XMLSchema\" xmlns:xsi=\"http://www.w3.org/2001/XMLSchema-instance\">\n<p>\n<i>Disclaimer: <span>This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Ultragenyx Pharmaceutical Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9645894-en) on January 30, 2026, and is solely responsible for the information contained therein.</span></i>\n</p>\n</div></body></html>","source":null,"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 Grants Priority Review to Ultragenyx BLA for UX111 Gene Therapy for Sanfilippo Syndrome Type A</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 Grants Priority Review to Ultragenyx BLA for UX111 Gene Therapy for Sanfilippo Syndrome Type A\n</h2>\n\n<h4 class=\"meta\">\n\n\n<a class=\"head\" href=\"https://laohu8.com/wemedia/1032215980\">\n\n\n<div class=\"h-thumb\" style=\"background-image:url(https://community-static.tradeup.com/news/4567337cbdf294b657b1fa87c5488b48);background-size:cover;\"></div>\n\n<div class=\"h-content\">\n<p class=\"h-name\">Reuters </p>\n<p class=\"h-time\">2026-01-30 21:30</p>\n</div>\n\n</a>\n\n\n</h4>\n\n</header>\n<article>\n<html xmlns=\"http://www.w3.org/1999/xhtml\" xmlns:newsg2=\"http://iptc.org/std/nar/2006-10-01/\" xmlns:xhtml=\"http://www.w3.org/1999/xhtml\"><head><title>\n      FDA Grants Priority Review to Ultragenyx BLA for UX111 Gene Therapy for Sanfilippo Syndrome Type A\n    </title></head><body><div xmlns:xsd=\"http://www.w3.org/2001/XMLSchema\" xmlns:xsi=\"http://www.w3.org/2001/XMLSchema-instance\">\n<p>\n        <a href=\"https://laohu8.com/S/RARE\">Ultragenyx Pharmaceutical Inc</a>. has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for UX111 (rebisufligene etisparvovec), an AAV9 gene therapy intended to treat patients with Sanfilippo syndrome type A (MPS IIIA). The FDA granted Priority Review to the application in February 2025. A Prescription Drug User Fee Act (PDUFA) action date is expected to be assigned within a month of resubmission, with a review period of up to six months anticipated, and a PDUFA date projected for the third quarter of 2026. If approved, UX111 would become the first therapy available for Sanfilippo syndrome type A.\n      </p>\n</div><div xmlns:xsd=\"http://www.w3.org/2001/XMLSchema\" xmlns:xsi=\"http://www.w3.org/2001/XMLSchema-instance\">\n<p>\n<i>Disclaimer: <span>This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Ultragenyx Pharmaceutical Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9645894-en) on January 30, 2026, and is solely responsible for the information contained therein.</span></i>\n</p>\n</div></body></html>\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"NDAQ","symbol_name":"纳斯达克OMX交易所","start_time":0,"source_url":"https://api.refinitiv.com/data/news/v1/stories/urn:newsml:reuters.com:20260130:nNDL6P10gg:1","article_id":"2607204545","we_media_id":"1032215980","thumbnails":[],"rights":null,"url":"https://stock-news.laohu8.com/highlight/detail?id=2607204545","pubTimestamp":1769779830,"columns":[],"sourceInfo":null,"weMediaInfo":{"media_name":"Reuters","introduction":"Reuters.com brings you the latest news from around the world, covering breaking news in markets, business, politics, entertainment and technology","home_visible":1,"id":"1032215980","head_image":"https://community-static.tradeup.com/news/4567337cbdf294b657b1fa87c5488b48"},"summary":"Ultragenyx Pharmaceutical Inc. has resubmitted its Biologics License Application  to the U.S. Food and Drug Administration  for UX111 , an AAV9 gene therapy intended to treat patients with Sanfilippo syndrome type A . The FDA granted Priority Review to the application in February 2025. A Prescription Drug User Fee Act  action date is expected to be assigned within a month of resubmission, with a review period of up to six months anticipated, and a PDUFA date projected for the third quarter of 2026. If approved, UX111 would become the first therapy available for Sanfilippo syndrome type A.Disclaimer: This news brief was created by Public Technologies  using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Ultragenyx Pharmaceutical Inc. published the original content used to generate this news brief via Glo","collect":0,"end_time":0,"defaultTopTitle":"","property":[],"viewcount":null,"language":"en","relate_stocks":{"NDAQ":"纳斯达克OMX交易所","RARE":"Ultragenyx Pharmaceutical Inc","LU2210150020.SGD":"Natixis Thematics Subscription Economy R/A SGD","BK4588":"碎股","SG9999014005.SGD":"United Global Durable Equities Fund Acc SGD","SG9999014021.USD":"UNITED GLOBAL DURABLE EQUITIES (USDHDG) ACC","BK4585":"ETF&股票定投概念","BK4112":"金融交易所和数据","IE00BGHQF748.EUR":"GUINNESS GLOBAL MONEY MANAGERS \"C\" (EUR) ACC","IE00B64PRP62.GBP":"GUINNESS GLOBAL MONEY MANAGERS \"C\" (GBP) ACC","LU0314104364.USD":"MANULIFE GF AMERICAN GROWTH \"AA\" (USD) INC","SG9999014013.SGD":"United Global Durable Equities Fund Dis SGD","BK4139":"生物科技","LU2210149790.SGD":"Natixis Thematics Subscription Economy R/A SGD-H","SG9999014039.USD":"UNITED GLOBAL DURABLE EQUITIES (USDHDG) INC","LU2095319765.USD":"Natixis Thematics Subscription Economy R/A USD","IE00BKPKM429.USD":"NEUBERGER BERMAN GLOBAL SUSTAINABLE EQUITY \"A\" (USD) ACC","IE00B3QW5Z07.USD":"GUINNESS GLOBAL MONEY MANAGERS \"C\" (USD) ACC"},"translate_title":"FDA授予Ultragenyx BLA优先审查UX111基因疗法治疗A型Sanfilippo综合征","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"NDAQ":0.9,"RARE":0.9},"content_text":"FDA Grants Priority Review to Ultragenyx BLA for UX111 Gene Therapy for Sanfilippo Syndrome Type A\n    \n\nUltragenyx Pharmaceutical Inc. has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for UX111 (rebisufligene etisparvovec), an AAV9 gene therapy intended to treat patients with Sanfilippo syndrome type A (MPS IIIA). The FDA granted Priority Review to the application in February 2025. A Prescription Drug User Fee Act (PDUFA) action date is expected to be assigned within a month of resubmission, with a review period of up to six months anticipated, and a PDUFA date projected for the third quarter of 2026. If approved, UX111 would become the first therapy available for Sanfilippo syndrome type A.\n      \n\n\nDisclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Ultragenyx Pharmaceutical Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9645894-en) on January 30, 2026, and is solely responsible for the information contained therein.","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":"productRelease","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/2607204545","isCrawlerRequest":true}