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Drugs. 2024 May;84(5):599-606. doi: 10.1007/s40265-024-02009-4. Epub 2024 Mar 22.
Iptacopan (FABHALTA) is an oral complement Factor B inhibitor developed by Novartis Pharmaceuticals for the treatment of complement-mediated diseases. Acting upstream of complement 5 in the alternative pathway, iptacopan inhibits both terminal complement-mediated intravascular haemolysis and complement 3-mediated extravascular haemolysis. On 5 December 2023, iptacopan received approval in the USA for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH). This article summarizes the milestones in the development of iptacopan leading to this first approval for PNH.
依库珠单抗（FABHALTA）是一种由诺华制药公司开发的口服补体因子 B 抑制剂，用于治疗补体介导的疾病。依库珠单抗作用于旁路途径中的补体 5 上游，可抑制终末补体介导的血管内溶血和补体 3 介导的血管外溶血。2023 年 12 月 5 日，依库珠单抗获得美国批准，用于治疗阵发性夜间血红蛋白尿症（PNH）成人患者。本文总结了依库珠单抗开发过程中的重要里程碑，最终该药获得 PNH 的首个批准。
