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Drugs. 2018 Feb;78(2):269-274. doi: 10.1007/s40265-018-0861-2.
Emicizumab-kxwh (Hemlibra) is a bispecific humanized monoclonal antibody that restores the function of missing activated FVIII by bridging activated FIX and FX to facilitate effective haemostasis in patients with haemophilia A. Subcutaneous emicizumab-kxwh is approved in the USA for use as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and paediatric patients with haemophilia A (congenital FVIII deficiency) with FVIII inhibitors. Subcutaneous emicizumab-kxwh is awaiting approval in several countries worldwide, including in the EU and Japan, and is undergoing phase 3 development in haemophilia A without FVIII inhibitors. This article summarizes the milestones in the development of emicizumab-kxwh leading to its first global approval for use as prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with haemophilia A.
依库珠单抗（Hemlibra）是一种双特异性人源化单克隆抗体，通过桥接激活的FIX 和 FX 来恢复缺失的活化 FVIII 的功能，从而促进血友病 A 患者的有效止血。皮下注射用依库珠单抗在美国获批用于常规预防，以预防或减少血友病 A（先天性 FVIII 缺乏症）伴 FVIII 抑制剂的成年和儿科患者出血发作的频率。皮下注射用依库珠单抗正在全球多个国家（包括欧盟和日本）等待批准，并在无 FVIII 抑制剂的血友病 A 患者中进行 3 期开发。本文总结了依库珠单抗开发过程中的重要里程碑，最终该药在全球首次获批用于预防或减少血友病 A 患者出血发作的频率。
