Sanofi Plans Regulatory Submission For Investigational Drug For Rare Disease Despite Mixed Data From Pivotal Trials

Benzinga_recent_news02-02 21:30

On Monday, Sanofi SA (NASDAQ:SNY) shared topline data from results from the LEAP2MONO phase 3 study of once daily oral venglustat versus intravenous enzyme replacement therapy (ERT) every two weeks in...

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Additional analyses of the data are ongoing.\nA second phase 3 study, the CARAT study, evaluating the effect of venglustat on left cardiac ventricular mass index in men and women with Fabry disease, is ongoing.\nVenglustat was well tolerated overall with no new safety signals compared with previous studies.\nSanofi currently markets Fabrazyme, an ERT for Fabry disease, and Cerezyme and Cerdelga for Gaucher disease, an ERT and oral therapy, respectively, in markets around the world.\nIn January 2026, the FDA approved an expanded label for Cerezyme to include non-central nervous system (CNS) manifestations of GD3, building on its approval in the US for GD1.\nThe LEAP2MONO study is ongoing, and results from its open-label phase will be presented in the future when available.\nPrice Action: SNY stock is up 1.28% at $47.64 during the premarket session at the last check on Monday, according to Benzinga Pro data.\nPhoto by HJBC via Shutterstock","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":"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,"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/2608641048","isCrawlerRequest":true}