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Drugs. 2019 Oct;79(15):1697-1707. doi: 10.1007/s40265-019-01200-2.
Emicizumab (Hemlibra), a recombinant, humanized, bispecific monoclonal antibody, restores the function of missing activated factor VIII (FVIII) by bridging FIXa and FX to facilitate effective haemostasis in patients with haemophilia A. Subcutaneous emicizumab is approved in several countries, including in the USA and Japan, for the routine prophylaxis of bleeding episodes in patients with haemophilia A with or without FVIII inhibitors. It is also approved in the EU for the routine prophylaxis of bleeding episodes in patients with haemophilia A with inhibitors or severe haemophilia A without inhibitors. In phase III clinical trials, emicizumab prophylaxis significantly reduced annualized bleeding rates compared with no prophylaxis in adolescents and adults with haemophilia A with or without inhibitors, and prevented or substantially reduced bleeding in children with haemophilia A with or without inhibitors. Emicizumab was also associated with beneficial effects on health-related quality of life and health status, and was generally well tolerated. In view of its convenient route of administration and versatile dosage regimens (maintenance dose of once every 1, 2 or 4 weeks), emicizumab provides an effective and generally well-tolerated alternative to conventional FVIII replacement products for the prophylaxis of bleeding episodes in patients with haemophilia A, regardless of the presence or absence of inhibitors.
依库珠单抗（Hemlibra），一种重组的、人源化的、双特异性单克隆抗体，通过桥接 FIXa 和 FX 来恢复缺失的活化因子 VIII（FVIII）的功能，从而促进血友病 A 患者的有效止血。皮下注射依库珠单抗已在包括美国和日本在内的多个国家获得批准，用于血友病 A 患者伴或不伴 FVIII 抑制剂的常规预防出血发作。在欧盟，它也被批准用于预防血友病 A 患者伴或不伴抑制剂的出血发作，或用于预防血友病 A 患者伴或不伴抑制剂的严重出血发作。在 III 期临床试验中，与不预防相比，依库珠单抗预防可显著降低伴或不伴抑制剂的血友病 A 青少年和成人的年化出血率，并可预防或显著减少伴或不伴抑制剂的血友病 A 儿童的出血。依库珠单抗还对健康相关生活质量和健康状况具有有益影响，且一般耐受性良好。鉴于其方便的给药途径和多种剂量方案（维持剂量为每 1、2 或 4 周一次），依库珠单抗为血友病 A 患者的出血发作预防提供了一种有效且一般耐受性良好的替代传统 FVIII 替代产品的选择，无论是否存在抑制剂。
