Ultragenyx BLA Resubmission Puts UX111 And RARE Valuation In Focus

Simply Wall St.02-02

Ultragenyx Pharmaceutical resubmitted its Biologics License Application to the FDA for UX111, a gene therapy candidate for Sanfilippo syndrome type A. The filing includes long term clinical data and ...

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/2608388415?lang=en_US&edition=fundamental","thumbnail":"https://images.simplywall.st/asset/industry/6111000-choice2-main-header/1585186599552","is_english":true,"pubTime":"2026-02-02 01:21","share_image_url":"https://static.laohu8.com/e9f99090a1c2ed51c021029395664489","id":"2608388415","market":"sg","top_or_hot":-1,"title":"Ultragenyx BLA Resubmission Puts UX111 And RARE Valuation In Focus","media":"Simply Wall St.","content":"<div>\n<p>Ultragenyx Pharmaceutical resubmitted its Biologics License Application to the FDA for UX111, a gene therapy candidate for Sanfilippo syndrome type A. The filing includes long term clinical data and ...</p>\n\n<a href=\"https://simplywall.st/stocks/us/pharmaceuticals-biotech/nasdaq-rare/ultragenyx-pharmaceutical/news/ultragenyx-bla-resubmission-puts-ux111-and-rare-valuation-in\">Source Link</a>\n\n</div>\n","source":"simplywall_highlight","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>Ultragenyx BLA Resubmission Puts UX111 And RARE Valuation In Focus</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\">\nUltragenyx BLA Resubmission Puts UX111 And RARE Valuation In Focus\n</h2>\n\n<h4 class=\"meta\">\n\n\n2026-02-02 01:21 GMT+8&nbsp;&nbsp;&nbsp;<a href=https://simplywall.st/stocks/us/pharmaceuticals-biotech/nasdaq-rare/ultragenyx-pharmaceutical/news/ultragenyx-bla-resubmission-puts-ux111-and-rare-valuation-in><strong>Simply Wall St.</strong></a>\n\n\n</h4>\n\n</header>\n<article>\n<div>\n<p>Ultragenyx Pharmaceutical resubmitted its Biologics License Application to the FDA for UX111, a gene therapy candidate for Sanfilippo syndrome type A. The filing includes long term clinical data and ...</p>\n\n<a href=\"https://simplywall.st/stocks/us/pharmaceuticals-biotech/nasdaq-rare/ultragenyx-pharmaceutical/news/ultragenyx-bla-resubmission-puts-ux111-and-rare-valuation-in\">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":"RARE","symbol_name":"Ultragenyx Pharmaceutical Inc","start_time":0,"source_url":"https://simplywall.st/stocks/us/pharmaceuticals-biotech/nasdaq-rare/ultragenyx-pharmaceutical/news/ultragenyx-bla-resubmission-puts-ux111-and-rare-valuation-in","article_id":"2608388415","we_media_id":null,"thumbnails":["https://images.simplywall.st/asset/industry/6111000-choice2-main-header/1585186599552"],"rights":{"source":"simplywall_highlight","url":"https://simplywall.st/stocks/us/pharmaceuticals-biotech/nasdaq-rare/ultragenyx-pharmaceutical/news/ultragenyx-bla-resubmission-puts-ux111-and-rare-valuation-in","rn_cache_url":null,"directOrigin":true},"url":"https://stock-news.laohu8.com/highlight/detail?id=2608388415","pubTimestamp":1769966469,"columns":[],"sourceInfo":{"source_id":"simplywall_highlight","name":"Simply Wall St."},"weMediaInfo":null,"summary":"Ultragenyx Pharmaceutical resubmitted its Biologics License Application to the FDA for UX111, a gene therapy candidate for Sanfilippo syndrome type A.\nThe filing includes long term clinical data and responses to earlier regulatory concerns about the therapy.\nIf accepted, UX111 could become the first approved treatment specifically targeting this rare and serious condition.\n\nUltragenyx Pharmaceutical (NasdaqGS:RARE), trading at $24.07 as of the last close, is moving ahead with a key...","collect":0,"end_time":0,"defaultTopTitle":"simplywall.st","property":[],"viewcount":null,"language":"en","relate_stocks":{"RARE":"Ultragenyx Pharmaceutical Inc","BK4139":"生物科技"},"translate_title":"Ultragenyx BLA重新提交使UX111和稀有估值成为焦点","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"RARE":1.5},"content_text":"Ultragenyx Pharmaceutical resubmitted its Biologics License Application to the FDA for UX111, a gene therapy candidate for Sanfilippo syndrome type A. The filing includes long term clinical data and responses to earlier regulatory concerns about the therapy. If accepted, UX111 could become the first approved treatment specifically targeting this rare and serious condition.  Ultragenyx Pharmaceutical (NasdaqGS:RARE), trading at $24.07 as of the last close, is moving ahead with a key regulatory step for its rare disease pipeline. The stock has seen a 44.1% decline over the past year and a 47.4% decline over three years, reflecting the risks and uncertainty that can come with a focus on complex gene therapies. The UX111 resubmission puts fresh attention on how regulators assess gene therapies for ultra rare conditions and how Ultragenyx manages scientific and regulatory hurdles. For investors, the next updates from the FDA process, along with any additional clinical disclosures, will be important markers for understanding how this program could influence the overall story for NasdaqGS:RARE. Stay updated on the most important news stories for Ultragenyx Pharmaceutical by adding it to your watchlist or portfolio. Alternatively, explore our Community to discover new perspectives on Ultragenyx Pharmaceutical. NasdaqGS:RARE Earnings & Revenue Growth as at Feb 2026 How Ultragenyx Pharmaceutical stacks up against its biggest competitors AdvertisementQuick Assessment  ✅ Price vs Analyst Target: At US$24.07 versus a US$64.00 analyst target, the price sits about 62% below consensus. ✅ Simply Wall St Valuation: The shares are described as trading 92.7% below the estimated fair value, flagging a large valuation gap. ✅ Recent Momentum: A 30 day return of roughly 2.0% shows modest positive short term momentum into the resubmission.  Check out Simply Wall St's\nin depth valuation analysis for Ultragenyx Pharmaceutical. Key Considerations  📊 The FDA resubmission, backed by long term UX111 data, keeps a potential first in disease gene therapy in play for the Ultragenyx story. 📊 Watch for FDA acceptance milestones, labeling questions and any new safety or efficacy data that could shift how UX111 is viewed in the portfolio. ⚠️ With two identified risk flags including a short cash runway and share price volatility, funding needs and dilution risk stay front of mind if timelines extend.  Dig Deeper For the full picture including more risks and rewards, check out the\ncomplete Ultragenyx Pharmaceutical analysis.  This article by Simply Wall St is general in nature. We provide commentary based on historical data\n    and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your\n    financial situation. We aim to bring you long-term focused analysis driven by fundamental data.\n    Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material.\n    Simply Wall St has no position in any stocks mentioned.New: AI Stock Screener & AlertsOur new AI Stock Screener scans the market every day to uncover opportunities.• Dividend Powerhouses (3%+ Yield)• Undervalued Small Caps with Insider Buying• High growth Tech and AI CompaniesOr build your own from over 50 metrics.Explore Now for FreeHave feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team@simplywallst.com","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":"","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/2608388415","isCrawlerRequest":true}