Inclisiran sodium

The FDA cleared additional indications for Novartis’s cholesterol-lowering drug Leqvio (inclisiran) and Novo Nordisk's haemophilia prophylactic Alhemo (concizumab-mtci), broadening their use to wider patient populations.Leqvio gets go-ahead as montherapyNovartis's twice-yearly injectable siRNA drug Leqvio was approved by the FDA as monotherapy alongside diet and exercise to lower low-denisty lipoprotein cholesterol (LDL-C) in adults with hypercholesterolaemia, thereby eliminating the previous requirement for concurrent statin therapy.“With this new indication enabling Leqvio's use as monotherapy…we now have the potential to help even more patients achieve their LDL-C lowering goals earlier in their treatment journey," said Victor Bultó, president of Novartis US.The US regulator said Leqvio’s broader indication was approved based on strong LDL-C-lowering data from PCSK9-targeting therapies. Additional label updates include replacing "primary hyperlipidaemia" terminology with the more specific "hypercholesterolaemia" designation throughout the prescribing information.Novartis noted the decision also aligns with the 2025 American College of Cardiology/American Heart Association Clinical Practice Guidelines, recommending more aggressive treatment approaches for patients with acute coronary syndrome.Leqvio was discovered by Alnylam and co-developed with The Medicines Company, which Novartis acquired in 2020 for $9.7 billion. First approved in the EU for hypercholesterolaemia or mixed dyslipidaemia in 2020, the siRNA’s US approval faced initial delays owing to manufacturing issues, but was subsequently cleared by the FDA in 2021 for patients on maximally tolerated statin therapy.The Swiss drugmaker expects Leqvio not only to surpass $1 billion in sales this year, but potentially outperform consensus forecasts long term. For more, read – Spotlight On: Novartis' fastest growing drugs.Alhemo cleared for patients without inhibitorsThe FDA also okayed an expanded indication for Novo Nordisk's Alhemo (concizumab-mtci), extending its prophylactic use to adult and paediatric patients aged 12 and older with haemophilia A or B without inhibitors. The latest approval builds upon the tissue factor pathway (TFPI) inhibitor antagonist’s approval last year for patients with inhibitors after being rejected by the agency on the first attempt.Anna Windle, senior VP of clinical development, medical & regulatory affairs at Novo Nordisk, said the latest approval “marks a meaningful step forward for people with haemophilia A or B without inhibitors who are looking for a new prophylaxis treatment option."The label expansion was supported by the Phase III explorer8 study, wherein Alhemo achieved an 86% reduction in annualised bleeding rate for patients with haemophilia A and 79% reduction for those with haemophilia B versus no prophylaxis.Following a positive recommendation by the EU drug advisory body last week, the anti-TFPI monoclonal antibody is also on the verge of approval in Europe for the same indication.With the additional indication, Alhemo emerges as a potential rival to Pfizer’s once-weekly Hympavzi (marstacimab-hncq), approved last year for patients without inhibitors. Pfizer also seeks Hympavzi’s expansion to patients with inhibitors following a successful Phase III study.

诺华（Novartis）公司今日宣布，美国FDA已批准其每年两次给药的小干扰RNA（siRNA）疗法Leqvio（inclisiran）的扩展适应症申请，允许其作为单药，与饮食控制和运动联合使用，以降低成人高胆固醇血症患者的低密度脂蛋白胆固醇（LDL-C）水平。根据新闻稿，美国FDA根据该PCSK9靶向疗法降低LDL-C的积极数据，主动要求更新该药品的标签。更新后的药品标签去除了Leqvio必须与他汀类药物联合使用或在其基础上使用的限制。其他更新内容还包括将标签中“原发性高脂血症”一词统一修改为更具体的“高胆固醇血症”，以更准确地强调Leqvio在降低LDL-C方面的治疗作用。心血管疾病（CVD）影响全球数亿人，相关死亡人数超过癌症、慢性肺病和糖尿病的总和。据估计，通过解决导致或加重CVD的因素，或可预防约80%因CVD导致的过早死亡。动脉粥样硬化性心血管疾病（ASCVD）约占所有心血管死亡案例的85%。ASCVD是由动脉内壁斑块的形成和生长所引起，而动脉粥样硬化斑块主要由LDL-C组成，会随着时间的推移而积累。体内长期LDL-C水平的升高会增加个体心脏病发作或中风等心血管事件的风险。许多LDL-C水平升高的患者同时患有其他疾病，如高血压、肥胖或糖尿病，这显著增加了他们罹患ASCVD和心血管事件的风险。Leqvio是一款“first-in-class”、靶向PCSK9 mRNA的siRNA疗法，最初于2021年12月获得美国FDA批准，作为饮食和他汀类药物治疗的辅助药物，用于治疗患有原发性高脂血症的成年患者，包括杂合子型家族性高胆固醇血症（HeFH）患者，以降低其LDL-C的水平。PCSK9蛋白的作用是抑制低密度脂蛋白（LDL）受体的回收和再利用。因此，降低PCSK9蛋白的水平可以让更多LDL受体回到肝细胞表面，与更多LDL结合，将它们从血液中清除。新闻稿指出，它是首个能够降低LDL-C的siRNA疗法。去年8月，诺华宣布其V-MONO临床3期试验达到主要终点。Leqvio作为单药，可显著降低中、低风险ASCVD患者的LDL-C水平。分析显示，与安慰剂和活性对照药物相比，Leqvio单药在降低ASCVD患者的LDL-C水平上皆展现优效性，支持Leqvio作为ASCVD一级与二级预防药物。一级预防是指针对大众或个人，预防或延缓ASCVD发病的措施。二级预防则是指治疗已知、具有临床意义的ASCVD，以及预防或延缓疾病症状发作的措施。Leqvio须通过医疗专业人员皮下注射，初始剂量为一剂，三个月后再注射一剂，然后每六个月注射一剂。Leqvio每年两次给药的特性，可能有助于提升患者治疗依从性，这是胆固醇控制中常见的问题。Leqvio最初由Alnylam Pharmaceuticals公司开发，诺华后来获得了Leqvio开发、生产和商业化的全球权利。参考资料：[1] Novartis twice-yearly* Leqvio® (inclisiran) receives FDA approval for new indication enabling first-line use. Retrieved July 31, 2025 from https://www.prnewswire.com/news-releases/novartis-twice-yearly-leqvio-inclisiran-receives-fda-approval-for-new-indication-enabling-first-line-use-302519118.html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