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Drugs. 2018 Oct;78(15):1639-1642. doi: 10.1007/s40265-018-0989-0.
Ablynx, a Sanofi Company, has developed the anti-von Willebrand factor Nanobody caplacizumab (Cablivi™) for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP). Based on positive results in phase II and III trials in patients with aTTP, caplacizumab was recently approved in the EU for the treatment of adults experiencing an episode of aTTP, in conjunction with plasma exchange and immunosuppression. This article summarizes the milestones in the development of caplacizumab leading to this first approval.
Ablynx，一家隶属于赛诺菲的公司，研发了抗血管性血友病因子纳米体 caplacizumab（Cablivi™）用于治疗获得性血栓性血小板减少性紫癜（aTTP）。基于在 aTTP 患者中进行的 II 期和 III 期临床试验的积极结果，caplacizumab 最近在欧盟获得批准，与血浆置换和免疫抑制联合用于治疗经历 aTTP 发作的成人患者。本文总结了 caplacizumab 开发过程中的重要里程碑，最终促成了该药的首次批准。
