RARE's Cholesterol Drug Evkeeza Gains EC Approval for Expanded Use

Zacks01-07

Ultragenyx Pharmaceutical RARE announced that the European Commission (EC) has expanded the eligible patient population for one of its marketed products, Evkeeza (evinacumab), an ANGPTL3 inhibitor. ...

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It affects approximately 1,600 people in the EU and 1 in 300,000 globally. Please note that Evkeeza was initially approved in the EU in 2021 as an adjunct to diet and other LDL-C-lowering therapies to treat adult and adolescent patients aged 12 years and older with HoFH. The drug’s label was later expanded in 2023 to include the treatment of children aged 5-11 years old with HoFH. The drug is also approved in multiple other geographies for the HoFH indication like the United States, United Kingdom and Canada, among others.In the past three months, shares of Ultragenyx have plunged 20% compared with the industry’s 9.4% decline.More on Evkeeza’s Label Update for Younger HoFH Patients. Ultragenyx’s commercial portfolio comprises three other medicines — Mepsevii , Crysvita  and Dojolvi .","collect":0,"end_time":0,"defaultTopTitle":"yahoo.com","property":[],"viewcount":null,"language":"en","relate_stocks":{"VNDA":"万达生物制药","BK4201":"综合性石油与天然气企业","LU2468319806.SGD":"MANULIFE GF HEALTHCARE \"AA\" (SGDHDG) ACC","LU2362541273.HKD":"WELLINGTON NEXT GENERATION GLOBAL EQUITY \"A\" (HKD) ACC","LU0889565916.HKD":"FRANKLIN BIOTECHNOLOGY DISCOVERY \"A\" (HKD) ACC","LU2362541513.USD":"WELLINGTON NEXT GENERATION GLOBAL EQUITY \"A\" (USD) ACC","RARE":"Ultragenyx Pharmaceutical Inc","LU0114720955.EUR":"SUSTAINABLE GLOBAL HEALTH CARE \"A\" INC","LU1917777945.USD":"安联专题基金Cl AT Acc","BK4585":"ETF&股票定投概念","LU1974910355.USD":"Allianz Thematica Cl AMg DIS USD","LU0823416689.USD":"BNP PARIBAS HEALTH CARE INNOVATORS \"C\" (USD) ACC","BK4139":"生物科技","LU2089984988.USD":"MANULIFE GF HEALTHCARE \"AA\" (USD) ACC","EC":"哥伦比亚国家石油","LU0053666078.USD":"摩根大通基金-美国股票A（离岸）美元","BK4196":"保健护理服务","LU2106854487.HKD":"ALLIANZ THEMATICA \"AMG\" (HKD) INC","LU0289739699.SGD":"AB INTERNATIONAL HEALTH CARE PORTFOLIO \"A\" (SGD) ACC","LU0320765992.SGD":"FTIF - Franklin Biotechnology Discovery A Acc SGD","LU2023250504.SGD":"Allianz Thematica Cl AMg DIS H2-SGD","BK4588":"碎股","LU0109394709.USD":"富兰克林生物科技新领域基金A (acc)","LU2362540622.SGD":"WELLINGTON NEXT GENERATION GLOBAL EQUITY \"A\" (SGDHDG) ACC","LU2237438978.USD":"Amundi Funds US Pioneer A2 (C) USD","LU0058720904.USD":"联博国际健康护理基金A","REGN":"再生元制药公司","LU0882574055.USD":"富达全球健康医疗A ACC","CSTL":"Castle Biosciences, Inc."},"translate_title":"RARE的胆固醇药物Evkeeza获得EC批准扩大使用","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"VNDA":1,"REGN":1,"RARE":1,"CSTL":1,"EC":1},"content_text":"Ultragenyx Pharmaceutical RARE announced that the European Commission (EC) has expanded the eligible patient population for one of its marketed products, Evkeeza (evinacumab), an ANGPTL3 inhibitor. Per the latest approval, Evkeeza is now indicated as an adjunct to diet and other lipid-lowering therapies to treat children aged six months and older with homozygous familial hypercholesterolemia (HoFH). The EC’s approval makes Evkeeza the first HoFH medicine indicated for this pediatric population in the EU.\nHOFH is an inherited disease that causes dangerously high levels of low-density lipoprotein cholesterol (LDL-C), or bad cholesterol. It affects approximately 1,600 people in the EU and 1 in 300,000 globally. Please note that Evkeeza was initially approved in the EU in 2021 as an adjunct to diet and other LDL-C-lowering therapies to treat adult and adolescent patients aged 12 years and older with HoFH. The drug’s label was later expanded in 2023 to include the treatment of children aged 5-11 years old with HoFH. The drug is also approved in multiple other geographies for the HoFH indication like the United States, United Kingdom and Canada, among others.\nIn the past three months, shares of Ultragenyx have plunged 20% compared with the industry’s 9.4% decline.\nImage Source: Zacks Investment Research\nMore on Evkeeza’s Label Update for Younger HoFH Patients\nPlease note that the EC’s approval forEvkeeza to treat HoFH patients aged six months and older follows the positive recommendation received from its advisory committee in November 2024.\nEvkeeza's efficacy in pediatric patients aged six months to less than five years with HoFH has been predicted through model-based extrapolation. These analyses suggest that this age group is expected to experience a similar or greater reduction in LDL-C at week 24 compared to adults when given a 15 mg/kg dose of the drug, every 4 weeks. Additionally, supportive data for five HoFH patients aged 1 to 4 years who receive Evkeeza via compassionate use also showed a clinically meaningful LDL-C reduction consistent with 5 years or older pediatric patients in clinical studies. The safety profile for this younger age group is expected to align with that of older pediatric patients, with no new safety concerns identified.\nPlease note that Evkeeza was originally discovered and developed by Regeneron Pharmaceuticals REGN. In 2022, Ultragenyx signed a licensing agreement with Regeneron for Evkeeza to treat HoFH. Per the deal, RARE obtained the rights to develop, commercialize and distribute Evkeeza outside the United States. The regions include the European Economic Area. The collaboration with Regeneron for Evkeeza gives Ultragenyx a fourth-approved product that adds to the top line. However, REGN solely commercializes Evkeeza in the United States.\nRARE’s Other Marketed Products\nUltragenyx’s commercial portfolio comprises three other medicines — Mepsevii (vestronidase alfa), Crysvita (burosumab) and Dojolvi (UX007).\nMepsevii is approved for the treatment of children and adults with Mucopolysaccharidosis VII, also known as Sly syndrome.\nCrysvita, an antibody targeting fibroblast growth factor 23, is approved in the United States for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients. Crysvita received European conditional marketing authorization in February 2018 for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older, and adolescents with growing skeletons. Crysvita has also been approved for another indication, tumor-induced osteomalacia, in adults and pediatric patients aged two years and older by the FDA in June 2020.\nDojolvi was approved by the FDA in 2020 for the treatment of pediatric and adult patients for all forms of long-chain fatty acid oxidation disorders with a molecularly confirmed diagnosis. Dojolvi is the first FDA-approved therapy for these lifelong and life-threatening genetic disorders and is now available to patients in the United States.\n\nUltragenyx Pharmaceutical Inc. Price and Consensus\n\nUltragenyx Pharmaceutical Inc. price-consensus-chart | Ultragenyx Pharmaceutical Inc. Quote\n\nRARE’s Zacks Rank & Stocks to Consider\nUltragenyx currently carries a Zacks Rank #3 (Hold).\nSome better-ranked stocks from the sector are Castle Biosciences CSTL and Vanda Pharmaceuticals VNDA, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.\nIn the past 30 days, 2024 estimates for Castle Biosciences’ earnings per share have remained constant at 34 cents. During the same timeframe, loss per share estimates for 2025 have remained constant at $1.84. In three months, shares of Castle Biosciences have lost 2.2%.\nCSTL’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 172.72%.\nIn the past 30 days, estimates for Vanda Pharmaceuticals’ 2024 loss per share have remained constant at 32 cents. Estimates for 2025 loss per share have remained constant at 35 cents during the same timeframe. In three months, Vanda’s shares have gained 6.7%.\nVNDA has delivered a negative earnings surprise of 45.79% in the past four quarters.\nWant the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report\nRegeneron Pharmaceuticals, Inc. (REGN) : Free Stock Analysis Report\nVanda Pharmaceuticals Inc. (VNDA) : Free Stock Analysis Report\nUltragenyx Pharmaceutical Inc. (RARE) : Free Stock Analysis Report\nCastle Biosciences, Inc. (CSTL) : Free Stock Analysis Report\nTo read this article on Zacks.com click here.\nZacks Investment Research","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":1},"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":"4.30.2","shortVersion":"4.30.2","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/2501780966"}