Introduction: Hereditary angioedema (HAE) with C1 esterase inhibitor deficiency (C1-INH-HAE) is a rare disease that manifests with cutaneous and/or submucosal swellings due to uncontrolled activation of the contact/kinin system. Attacks recur with unpredictable frequency and severity, laryngeal edema is potentially lethal, and the disease burden may severely disrupt patients’ lives.

Areas covered: This review provides an overview of lanadelumab, a human monoclonal antibody targeted against plasma kallikrein that was recently approved for prevention of symptoms in C1-INH-HAE.

Expert opinion: The phase III HELP Study demonstrated the efficacy of lanadelumab in reducing HAE attacks. These positive results are being further confirmed in the open-label extension study. This agent addresses some of the limitations of existing prophylactic options as tolerability issues, the need for intravenous administration and frequent dosing. Therefore, lanadelumab can profoundly improve the quality of life of patients with C1-INH-HAE.

简介：伴有C1酯酶抑制剂缺乏症（C1-INH-HAE）的遗传性血管性水肿（HAE）是一种罕见疾病，由于接触/激肽系统的激活失控而表现为皮肤和/或粘膜下肿胀。发作的频率和严重性无法预测，喉头水肿可能致命，疾病负担可能严重破坏患者的生命。

涵盖领域：本综述概述了lanadelumab，这是一种针对血浆激肽释放酶的人类单克隆抗体，最近被批准用于预防C1-INH-HAE中的症状。

专家意见：III期HELP研究证明了lanadelumab可以减少HAE发作。这些积极的结果正在开放标签扩展研究中得到进一步证实。该药物解决了现有预防措施的一些局限性，例如耐受性问题，静脉内给药的需要和频繁给药。因此，lanadelumab可以深刻改善C1-INH-HAE患者的生活质量。