Approval extends treatment of Praluent to children aged 8 and older with heterozygous familial hypercholesterolemia (HeFH)

批准将Praluent的治疗扩展到8岁及以上患有杂合性家族性高胆固醇血症（HeFH）的儿童

TARRYTOWN, N.Y., March 11, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced the U.S. Food & Drug Administration (FDA) has extended the approval of Praluent® (alirocumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies to include pediatric patients aged 8 and older with heterozygous familial hypercholesterolemia (HeFH)..

纽约州塔里敦，2024年3月11日（环球通讯社）--Regeneron Pharmaceuticals，Inc.（纳斯达克：REGN）宣布，美国食品和药物管理局（FDA）已经延长了对Praluent®（alirocumab）的批准，将其作为饮食和其他低密度脂蛋白胆固醇（LDL-C）降低疗法的辅助手段，包括8岁及以上患有杂合性家族性高胆固醇血症（HeFH）的儿科患者。。

“Many children with heterozygous familial hypercholesterolemia (HeFH) are able to substantially improve their LDL-C (bad cholesterol) with currently available therapies. But for those children whose LDL-C remains dangerously high, this approval is an important milestone as it gives these children and their families an additional option to help reduce and manage their LDL-C levels much earlier in their lives,” said Mary P.

“许多患有杂合性家族性高胆固醇血症（HeFH）的儿童能够大大改善他们的LDL-C（坏胆固醇）目前可用的疗法。但对于那些低密度脂蛋白胆固醇（LDL-C）仍然处于危险高水平的儿童来说，这一批准是一个重要的里程碑，因为它为这些儿童及其家人提供了一个额外的选择，可以帮助他们在生命的早期降低和管理他们的LDL-C水平，”MaryP。

McGowan, M.D., Chief Medical Officer of the Family Heart Foundation..

McGowan，医学博士，家庭心脏基金会首席医疗官。。

Familial hypercholesterolemia (FH) is an inherited condition caused by mutations in one of several genes that control how the body processes cholesterol, which can lead to very high levels of LDL-C (bad cholesterol). FH can come in two forms: HeFH, which develops when one mutated gene is inherited from one parent; and homozygous familial hypercholesterolemia (HoFH), which develops when a mutated gene is inherited from both parents.

家族性高胆固醇血症（FH）是一种遗传性疾病，由控制身体如何处理胆固醇的几个基因之一突变引起，这可能导致非常高水平的LDL-C（坏胆固醇）。FH可以有两种形式：HeFH，当一个突变基因从一个亲本遗传时就会发生；和纯合子家族性高胆固醇血症（HoFH），当突变基因从父母双方遗传时会发生。

Praluent is approved to treat both children and adults with HeFH and adults with HoFH..

Praluent被批准用于治疗患有HeFH的儿童和成人以及患有HoFH的成人。。

The approval is based on a Phase 3, randomized multicenter trial evaluating pediatric patients aged 8 to 17 with HeFH, who had LDL-C levels of 130mg/dL or greater and were already being treated with lipid-lowering medications. Patients were randomized to receive Praluent (N=101) or placebo (N=52) every two or four weeks in two consecutive cohorts.

该批准基于一项3期随机多中心试验，该试验评估了8至17岁患有HeFH的儿科患者，这些患者的LDL-C水平为130mg/dL或更高，并且已经接受降脂药物治疗。患者被随机分配到两个连续队列中，每两周或四周接受一次Praluent（N=101）或安慰剂（N=52）。

Patients who received Praluent every four weeks had 31% lower LDL-C than placebo at 24 weeks (97.5% Confidence Interval: -45.0% to -17.9%; p<0.0001). Improvements in additional key lipid parameters were also observed. Results from the trial were recently published in the Journal of the American Medical Association Pediatrics..

每四周接受Praluent治疗的患者在24周时LDL-C比安慰剂低31%（97.5%置信区间：-45.0%至-17.9%；p＜0.0001）。还观察到其他关键脂质参数的改善。该试验的结果最近发表在《美国医学会儿科杂志》上。。

No new adverse reactions were identified in this trial, and the safety profile was consistent with the safety profile observed in adults with HeFH. Across Praluent trials in patients with primary hyperlipidemia (N=2,476), the most common adverse reactions (≥5%) more frequently observed with Praluent than placebo have been injection site reactions (7%), and influenza (6%) and diarrhea (5%)..

该试验未发现新的不良反应，安全性与成人HeFH观察到的安全性一致。在原发性高脂血症患者（N=2476）的Praluent试验中，Praluent比安慰剂更常见的不良反应（≥5%）是注射部位反应（7%），流感（6%）和腹泻（5%）。。

“The approval of Praluent for the treatment of high cholesterol was a historic landmark achievement, as it was the first approved therapy targeting the genetically-validated PCSK9 target for heart disease,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron, and a principal inventor of Praluent.

“Praluent治疗高胆固醇的批准是一项具有历史意义的成就，因为它是第一个针对经过基因验证的PCSK9靶点治疗心脏病的批准疗法，”医学博士、董事会联合主席、Regeneron总裁兼首席科学官兼Praluent的主要发明人GeorgeD.Yancopoulos说。

“Praluent has made a meaningful impact in the treatment of adults with familial hypercholesterolemia, and we are proud that our innovation will now be able to help appropriate children with the heterozygous form of this disease manage their dangerously high levels of LDL-C.”.

“Praluent在治疗家族性高胆固醇血症的成年人方面产生了有意义的影响，我们感到骄傲的是，我们的创新现在能够帮助患有这种疾病的杂合形式的适当儿童管理其危险的高水平LDL-C。”。

About the Praluent HeFH Pediatric Trial

关于Praluent HeFH儿科试验

The randomized multicenter Phase 3 trial consisted of a 24-week double-blind, placebo-controlled evaluating the efficacy and safety of Praluent in pediatric patients aged 8 to 17 years with HeFH (N=79). The primary endpoint was the percent change in LDL-C from baseline to week 24 in the Praluent and placebo treated patients.

这项随机多中心3期临床试验包括一项为期24周的双盲安慰剂对照试验，评估了Praluent对8至17岁HeFH患儿（N=79）的疗效和安全性。主要终点是服用安慰剂和安慰剂的患者从基线到第24周LDL-C的百分比变化。

At baseline, patients were on a low-fat diet and being treated with background lipid-lowering therapy. In the trial, patients were randomized 2:1 to receive Praluent or placebo every 2 or 4 weeks. The Praluent dose was based on body weight..

在基线时，患者接受低脂饮食并接受背景降脂治疗。在试验中，患者以2:1的比例随机分配，每2或4周接受一次Praluent或安慰剂。Praluent剂量基于体重。。

About Praluent

关于Praluent

Praluent inhibits the binding of PCSK9 to the LDL receptor and thereby increases the number of available LDL receptors on the surface of liver cells to clear LDL, which lowers LDL-C levels in the blood. Praluent was developed by Regeneron and Sanofi under a global collaboration agreement and invented by Regeneron using the company's proprietary VelocImmune® technology that yields optimized fully-human monoclonal antibodies..

Praluent抑制PCSK9与LDL受体的结合，从而增加肝细胞表面可用LDL受体的数量，以清除LDL，从而降低血液中的LDL-C水平。Praluent由Regeneron和赛诺菲根据全球合作协议开发，由Regeneron使用公司专有的VelocImmune®技术发明，该技术可产生优化的全人单克隆抗体。。

In the U.S., Praluent is currently indicated:

在美国，Praluent目前表示：

to reduce the risk of myocardial infarction, stroke, and unstable angina requiring hospitalization in adults with established cardiovascular disease

降低患有心血管疾病的成年人需要住院治疗的心肌梗塞，中风和不稳定型心绞痛的风险

as an adjunct to diet, alone or in combination with other low-density lipoprotein cholesterol (LDL-C) lowering therapies in adults with primary hyperlipidemia including HeFH to reduce LDL-C

作为饮食的辅助手段，单独或与其他低密度脂蛋白胆固醇（LDL-C）降低治疗成人原发性高脂血症（包括HeFH）以降低LDL-C

as an adjunct to other LDL-C-lowering therapies in adults with HoFH to reduce LDL-C

作为HoFH成人其他低密度脂蛋白胆固醇降低疗法的辅助手段，以降低低密度脂蛋白胆固醇

along with diet and other LDL-C lowering treatments in children aged 8 years and older with HeFH to reduce LDL-C

对8岁及以上患有HeFH的儿童进行饮食和其他低密度脂蛋白胆固醇降低治疗，以降低低密度脂蛋白胆固醇

In addition to the U.S., Praluent is approved in 60 countries, including the European Union, Japan, Canada, Switzerland and Brazil.

除美国外，Praluent在60个国家获得批准，包括欧盟、日本、加拿大、瑞士和巴西。

About Regeneron’s VelocImmune Technology

关于Regeneron的VelocImmune技术

Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W.

Regeneron的VelocImmune技术利用专有的基因工程小鼠平台，该平台具有基因人源化免疫系统，可产生优化的全人类抗体。Regeneron总裁兼首席科学官乔治·D·扬科波洛斯（GeorgeD.Yancopoulos）和他的导师弗雷德里克·沃克（FrederickW。

Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved fully human monoclonal antibodies currently available.

Alt在1985年，他们是第一个设想制造这种基因人性化小鼠的人，Regeneron花费了数十年的时间发明和开发VelocImmune和相关的VelociSuite®技术。Yancopoulos博士和他的团队使用VelocImmune技术创造了目前可用的所有原始的，FDA批准的完全人类单克隆抗体的很大一部分。

This includes Evkeeza® (evinacumab-dgnb), REGEN-COV® (casirivimab and imdevimab), Dupixent® (dupilumab), Libtayo® (cemiplimab-rwlc), Praluent, Kevzara® (sarilumab), Inmazeb® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz® (pozelimab-bbfg)..

这包括埃夫基扎® （evinacumab-dgnb），REGEN-COV® （casirivimab和imdevimab），Dupixent® （副本），Libtayo® （cemiplimab-rwlc），Praluent，Kevzara® （沙鲁单抗）、因马泽布® （阿托替单抗、马夫替单抗和奥迪维单抗ebgn）和Veopoz® （pozelimab bbfg）。。

IMPORTANT SAFETY INFORMATION AND INDICATIONS

重要安全信息和指示

INDICATIONS

适应症

PRALUENT is an injectable prescription medicine used:

PRALUENT是一种注射处方药，用于：

in adults with cardiovascular disease to reduce the risk of heart attack, stroke, and certain types of chest pain conditions (unstable angina) requiring hospitalization.

在患有心血管疾病的成年人中，可以降低心脏病发作，中风和某些需要住院治疗的胸痛（不稳定型心绞痛）的风险。

along with diet, alone or together with other cholesterol-lowering medicines in adults with high blood cholesterol levels called primary hyperlipidemia (including a type of high cholesterol called heterozygous familial hypercholesterolemia [HeFH]), to reduce low-density lipoprotein cholesterol (LDL-C) or bad cholesterol..

与饮食一起，单独或与其他降胆固醇药物一起用于高血胆固醇水平的成年人，称为原发性高脂血症（包括一种称为杂合性家族性高胆固醇血症（HeFH））的高胆固醇，以降低低密度脂蛋白胆固醇（LDL-C）或坏胆固醇。。

along with other LDL-lowering treatments in adults with a type of high cholesterol called homozygous familial hypercholesterolemia, who need additional lowering of LDL-C.

以及其他降低LDL的治疗方法，用于患有高胆固醇的成年人，称为纯合子家族性高胆固醇血症，需要进一步降低LDL-C。

along with diet and other LDL-C lowering treatments in children aged 8 years and older with HeFH to reduce LDL-C.

对8岁及以上患有HeFH的儿童进行饮食和其他降低LDL-C的治疗，以降低LDL-C。

It is not known if PRALUENT is safe and effective in children who are younger than 8 years of age or in children with other types of high cholesterol (hyperlipemias).

目前尚不清楚PRALUENT对8岁以下儿童或其他类型高胆固醇（高脂血症）儿童是否安全有效。

IMPORTANT SAFETY INFORMATION

重要安全信息

Do not use PRALUENT if you are allergic to alirocumab or to any of the ingredients in PRALUENT.

如果您对alirocumab或PRALUENT中的任何成分过敏，请不要使用PRALUENT。

Before you start using PRALUENT, tell your healthcare provider about all of your medical conditions, including allergies, and if you are pregnant or plan to become pregnant or if you are breastfeeding or plan to breastfeed.

在您开始使用PRALUENT之前，请告知您的医疗保健提供者您的所有医疗状况，包括过敏，以及您是否怀孕或计划怀孕，或者您是否正在母乳喂养或计划母乳喂养。

Tell your healthcare provider or pharmacist about any medicines you take, including prescription and over-the-counter medicines, vitamins, or herbal supplements.

告诉你的医疗保健提供者或药剂师你服用的任何药物，包括处方药和非处方药、维生素或草药补充剂。

PRALUENT can cause serious side effects, including allergic reactions that can be severe and require treatment in a hospital. Stop using PRALUENT and call your healthcare provider or go to the nearest hospital emergency room right away if you have any symptoms of an allergic reaction including a severe rash, redness, hives, severe itching, trouble breathing, or swelling of the face, lips, throat, or tongue..

PRALUENT会引起严重的副作用，包括可能严重的过敏反应，需要在医院治疗。如果您有任何过敏反应症状，包括严重皮疹、发红、荨麻疹、严重瘙痒、呼吸困难或面部、嘴唇、喉咙或舌头肿胀，请停止使用PRALUENT并致电您的医疗保健提供者或立即前往最近的医院急诊室。。

The common side effects of PRALUENT include: redness, itching, swelling, or pain/tenderness at the injection site, flu or flu-like symptoms, diarrhea, muscle pain, muscle spasms and bruising. Tell your healthcare provider if you have any side effect that bothers you or that does not go away.

PRALUENT的常见副作用包括：注射部位发红，瘙痒，肿胀或疼痛/压痛，流感或流感样症状，腹泻，肌肉疼痛，肌肉痉挛和瘀伤。告诉你的医疗保健提供者，如果你有任何副作用困扰你或不会消失。

Talk to your doctor about the right way to prepare and give yourself a PRALUENT injection and follow the “Instructions For Use” that comes with PRALUENT. In children aged 12 to 17 years, it is recommended that PRALUENT be given by or under the supervision of an adult. In children aged 8 to 11 years, PRALUENT should be given by a caregiver..

与你的医生谈论正确的方法来准备和给自己注射PRALUENT，并遵循PRALUENT附带的“使用说明”。对于12至17岁的儿童，建议由成年人或在成年人的监督下服用PRALUENT。对于8至11岁的儿童，应由护理人员给予PRALUENT。。

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

鼓励您向FDA报告处方药的负面副作用。访问www.fda.gov/medwatch或致电1-800-fda-1088。

Please click here for full Prescribing Information.

请点击此处获取完整的处方信息。

About Regeneron

关于Regeneron

Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led for over 35 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous FDA-approved treatments and product candidates in development, almost all of which were homegrown in our laboratories.

Regeneron是一家领先的生物技术公司，为患有严重疾病的人发明、开发和商业化改变生命的药物。由医生科学家创立并领导了35多年，我们反复且始终如一地将科学转化为医学的独特能力导致了许多FDA批准的治疗方法和候选产品的开发，几乎所有这些都是在我们的实验室中自主开发的。

Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, hematologic conditions, infectious diseases and rare diseases..

我们的药品和管道旨在帮助患有眼疾、过敏性和炎症性疾病、癌症、心血管和代谢疾病、血液病、传染病和罕见病的患者。。

Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, such as VelocImmune, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world..

Regeneron正在通过我们专有的VelociSuite®技术加速和改进传统药物开发过程，例如VelocImmune，它使用独特的基因人源化小鼠产生优化的全人类抗体和双特异性抗体，并通过雄心勃勃的研究计划，如Regeneron遗传学中心，这是世界上最大的遗传学测序工作之一。。

For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn.

有关更多信息，请访问www.Regeneron.com或在LinkedIn上关注Regeneron。

Forward-Looking Statements and Use of Digital Media

前瞻性声明和数字媒体的使用

This press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements.

本新闻稿包括前瞻性声明，涉及与未来事件和Regeneron Pharmaceuticals，Inc.（“Regeneron”或“公司”）未来业绩相关的风险和不确定性，实际事件或结果可能与这些前瞻性声明存在重大差异。

Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, the nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Products”) and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Product Candidates”) and research and clinical programs now underway or planned, including without limitation Praluent® (alirocumab) for the treatment of pediatric patients aged 8 to 17 with heterozygous familial hypercholesterolemia; uncertainty of the utilization, market acceptance, and commercial success of Regeneron’s Products and Regeneron’s Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the studies discussed or referenced in this press release, on any of the foregoing; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s Product Candidates and new indications for Regeneron’s Products; the ability of Regeneron’s collaborators, licensees, suppliers, or other third par.

诸如“预期”、“期望”、“打算”、“计划”、“相信”、“寻求”、“估计”等词语的变体以及类似的表达都旨在识别此类前瞻性陈述，尽管并非所有前瞻性陈述都包含这些识别词。这些声明涉及，这些风险和不确定性包括Regeneron和/或其合作者或被许可人（统称为“Regeneron产品”）销售或以其他方式商业化的产品的性质、时间、可能的成功和治疗应用以及由Regeneron和/或其合作者或被许可人（统称为“Regeneron的候选产品”）开发的候选产品，以及目前正在进行或计划进行的研究和临床计划，包括但不限于用于治疗8至17岁患有杂合性家族性高胆固醇血症的儿科患者的Praluent®（alirocumab）；Regeneron产品和Regeneron候选产品的利用、市场接受度和商业成功的不确定性，以及研究（无论是由Regeneron还是其他人进行的，无论是强制性的还是自愿的）对上述任何一项的影响，包括本新闻稿中讨论或引用的研究；Regeneron产品候选产品和Regeneron产品新适应症的可能监管批准和商业发布的可能性、时间和范围；Regeneron的合作者、被许可人、供应商或其他第三方的能力。

Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron's media and investor relations website (https://investor.regeneron.com) and its LinkedIn page (https://www.linkedin.com/company/regeneron-pharmaceuticals)..

Regeneron使用其媒体和投资者关系网站以及社交媒体发布有关公司的重要信息，包括可能被投资者视为重要的信息。有关Regeneron的财务和其他信息会定期发布，并可在Regeneron的媒体和投资者关系网站上访问(https://investor.regeneron.com)以及它的LinkedIn页面(https://www.linkedin.com/company/regeneron-pharmaceuticals)。。

Contacts:

联系人：

Media Relations

媒体关系

Mary Heather

玛丽·希瑟

Tel: +1 914-847-8650

电话：+1 914-847-8650

mary.heather@regeneron.com

mary.heather@regeneron.com

Investor Relations

投资者关系

Mark Hudson

哈德森

Tel: +1 914-847-3482

电话：+1 914-847-3482

mark.hudson@regeneron.com

mark.hudson@regeneron.com