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Drugs. 2021 Apr;81(5):587-594. doi: 10.1007/s40265-021-01481-6.
Ravulizumab (Ultomiris), a humanized monoclonal antibody that inhibits complement protein C5, is indicated for the treatment of atypical haemolytic uraemic syndrome (aHUS) in several countries, including the USA and those of the EU. Ravulizumab has been re-engineered from eculizumab to extend its terminal elimination half-life, resulting in a more convenient maintenance dosage regimen of once every 4-8 weeks compared with once every 2-3 weeks for eculizumab. In single-arm phase 3 trials, ravulizumab resolved thrombotic microangiopathy in 54% and 78% of treatment-naïve adult and paediatric patients with aHUS, respectively, within 26 weeks. Ravulizumab was also effective in patients with postpartum aHUS and paediatric patients who responded to eculizumab and later switched to ravulizumab. Ravulizumab was generally well tolerated, with no unexpected safety events. The most common treatment-related adverse events with ravulizumab in treatment-naïve patients include headache, diarrhoea and vomiting. With its convenient once every 4-8 weeks maintenance regimen, ravulizumab is an important treatment option for aHUS in adult and paediatric patients.
拉维珠单抗（Ultomiris）是一种人源化单克隆抗体，可抑制补体蛋白 C5，已在包括美国和欧盟成员国在内的多个国家被批准用于治疗非典型溶血尿毒综合征（aHUS）。拉维珠单抗是从依库珠单抗重新设计而来，以延长其终末消除半衰期，与依库珠单抗每 2-3 周给药相比，其维持剂量方案更为方便，每 4-8 周给药一次。在单臂 3 期临床试验中，拉维珠单抗在 26 周内分别使 54%和 78%的初治成年和儿科 aHUS 患者的血栓性微血管病得到缓解。拉维珠单抗对产后 aHUS 患者和曾接受依库珠单抗治疗且后来转为拉维珠单抗治疗的儿科患者也有效。拉维珠单抗总体耐受性良好，无意外的安全事件。在初治患者中，拉维珠单抗最常见的治疗相关不良事件包括头痛、腹泻和呕吐。由于其方便的每 4-8 周维持治疗方案，拉维珠单抗是成年和儿科患者治疗 aHUS 的重要选择。
