Lerodalcibep

LIB Therapeutics公司今日宣布，美国FDA已批准Lerochol（lerodalcibep）注射液上市，作为饮食和运动的辅助治疗，用于降低成人高胆固醇血症患者的低密度脂蛋白胆固醇（LDL-C），包括杂合子家族性高胆固醇血症（HeFH）患者。 Lerochol是一种创新第三代PCSK9抑制剂，旨在帮助患者实现并维持其LDL-C目标。该药物为患者自我给药，每月一次，单次小体积皮下注射，并具备延长的室温稳定性（最长可达3个月），使患者能够自由选择给药的时间和地点。从分子类型来说，Lerochol是一种小结合蛋白（SBP），其PCSK9靶向结合域为一种经过工程化设计的多肽，能够以亚纳摩尔级别的高亲和力与人类PCSK9蛋白结合。此外，通过与人血清白蛋白（HSA）的融合，Lerochol显著延长其血浆半衰期，可达12-15天。 Lerochol的批准基于全面的全球性3期临床试验项目LIBerate的数据。该项目纳入了超过2900名多样化患者，包括已患心血管疾病、未患心血管疾病但具有极高或高心血管风险的患者，以及杂合子和纯合子家族性高胆固醇血症患者。在这些关键性、支持注册的安慰剂对照试验中，Lerochol每月给药一次，最长达52周，且有超过2400名患者进入了为期72周的开放标签延长期试验。 在临床试验中，Lerochol在已患心血管疾病或具有极高或高心血管风险的患者中，实现了≥60%的持续LDL-C降低；在LDL-C升高更为严重的HeFH患者中，实现了≥50%的LDL-C降低。在整个LIBerate临床试验项目中，Lerochol总体耐受性良好，延长期研究中未报告与治疗相关的严重不良事件。 图片来源：123RF 心血管疾病（CVD）仍然是全球的首要死亡原因，研究已证实，降低LDL-C水平可带来更好的临床结局。尽管他汀类药物及其他降脂疗法已广泛应用，仍有数以百万计的心血管疾病患者或高风险人群（包括家族性高胆固醇血症患者）未能达到最新指南推荐的LDL-C目标。全球大约每200人中就有1人患有家族性高胆固醇血症，这是一种自出生即存在严重LDL-C升高、需要终身管理的遗传性疾病。 参考资料： [1] U.S. Food and Drug Administration Approves LIB Therapeutics’ LEROCHOL™ (lerodalcibep-liga) for Adults with Elevated LDL Cholesterol. Retrieved December 15, 2025, from https://www.businesswire.com/news/home/20251215907781/en/U.S.-Food-and-Drug-Administration-Approves-LIB-Therapeutics-LEROCHOL-lerodalcibep-liga-for-Adults-with-Elevated-LDL-Cholesterol 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

The FDA has green-lit LIB Therapeutics' third-generation PCSK9 inhibitor, Lerochol, which lowers LDL-C cholesterol and has a convenience edge over its PCSK9 predecessors, Amgen's Repatha and Regeneron and Sanofi's Praluent. LIB Therapeutics has scored an FDA approval for its cholesterol-lowering, third-generation PCSK9 inhibitor, lerodalcibep-liga.The injected treatment, which will carry the commercial name Lerochol, is approved to be used along with diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including those with heterozygous familial hypercholesterolemia (HeFH).Lerochol arrives on the market with a convenience edge over other PCSK9 drugs, as it is self-administered once monthly and doesn’t need refrigeration because it retains its stability for up to three months at room temperature. By comparison, Amgen’s Repatha and Sanofi and Regeneron’s Praluent are dosed between every two to four weeks, depending on patient needs, and have a shorter shelf life at room temperature.Lerochol is the first approved product for the privately held biotech, which is based in Cincinnati and was established in 2015 with assets gained from Bristol Myers Squibb. LIB expects to launch Lerochol in the second quarter of next year in a pre-filled syringe, with an upgraded autoinjector device coming before the end of 2026.“We founded LIB Therapeutics with a mission to bring an effective and more patient-friendly treatment option to the millions of patients who still need additional LDL-C reduction in order to achieve the lower targets recommended in recent guidelines,” Evan Stein, M.D., Ph.D., the founder, CEO and chief scientific officer of LIB, said in a release. “Lerochol was designed for patients who need life-long treatment to achieve and maintain the new lower LDL-C goals, especially those with cardiovascular disease and the millions with inherited high cholesterol.”The European Medicines Agency (EMA) is reviewing a marketing authorization application for Lerochol, with the company expecting approval there in June of 2026. Additional regulatory submissions in other markets worldwide are also under review, LIB said.The U.S. approval is based on data from a trial program which tested 2,900 patients who were already receiving cholesterol-lowering treatments. Dosed monthly for 52 weeks, Lerochol showed sustained LDL-C reductions of more than 60% in patients with cardiovascular disease (CVD) or those at risk of developing CVD. In those with HeFH, Lerochol showed sustained LDL-C reductions of more than 50%. Despite the availability of statins and other lipid-lowering therapies, millions of patients with or at risk of CVD, including those with familial hypercholesterolemia (FH), fail to achieve updated guideline-recommended LDL-C targets, LIB said.Approximately 1 in 200 people worldwide have FH, a genetic condition characterized by severely elevated LDL-C levels from birth that requires lifelong management. PCSK9 inhibitors offer the most effective and safe way of lowering LDL-C, though widespread adoption has been limited by the high cost of the drugs, plus access barriers.Existing PCSK9 inhibitors never came close to Wall Street’s high initial sales expectations in the tens of billions of dollars. In 2024, Repatha generated sales of $2.2 billion, while Praluent achieved revenue of $765 million. Both drugs reached the market a decade ago. Novartis reported 2024 sales of its PCSK9 drug Leqvio at $754 million. The treatment, which was approved in 2021, takes a unique approach as it targets and degrades the PCSK9 messenger RNA (mRNA) in the liver, stopping the protein from being made as opposed to blocking it.Merck is developing a PCSK9 pill, enlicitide, which has achieved LDL-C reductions that are similar to those observed with existing injectables in two phase 3 trials. Analysts from Leerink Partners wrote last month that Merck’s treatment provides a “significant advantage” over injected biologics, given its oral administration, room-temperature storage and likely lower pricing.