江苏诺华(Novartis)(NVS.US)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。 Leqvio是一款"first-in-class"、靶向PCSK9mRNA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。 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 ...

Core Insights - AbbVie has received FDA accelerated approval for its antibody-drug conjugate, telisotuzumab vedotin (Emrelis), for treating certain patients with non-small cell lung cancer (NSCLC) [1][2] - Emrelis is the first FDA-approved therapy for adult patients with locally advanced or metastatic, non-squamous NSCLC with high c-Met protein overexpression who have received prior systemic therapy [2] - The approval is based on data from the phase II LUMINOSITY study, which demonstrated a 35% overall response rate in patients with high c-Met protein overexpression [5] Company Developments - Emrelis marks a significant milestone for AbbVie as it is the company's first internally developed solid tumor drug and its first solid tumor therapy approved for lung cancer [4] - AbbVie has expanded its oncology portfolio to five therapies, including Imbruvica, Venclexta, Epkinly, Elahere, and Emrelis [8] - The company is also developing another c-Met targeting ADC, Temab-A, for metastatic colorectal cancer and gastroesophageal cancer [10] Pipeline and Collaborations - AbbVie is conducting a phase III confirmatory study called TeliMET NSCLC-01 to convert the accelerated approval of Emrelis into a full approval [5] - The company has entered into a collaboration with ADARx Pharmaceuticals to develop small interfering RNA (siRNA) therapeutics across multiple disease areas [11] - AbbVie will make an upfront payment of $335 million to ADARx, with potential milestone payments reaching several billion dollars [13]