阵发性睡眠性血红蛋白尿治疗药物—Pegcetacoplan

A novel drug for Paroxysmal nocturnal hemoglobinuria—Pegcetacoplan

Pegcetacoplan是C3靶向性抑制剂,特异性地与补体C3和C3b结合,用于治疗补体介导的相关疾病。2021年5月美国食品药品管理局(FDA)批准C3靶向抑制剂Pegcetacoplan上市,用于治疗成人阵发性睡眠性血红蛋白尿(PNH)。PNH是一种非常罕见的、获得性、非恶性血液疾病。本文对Pegcetacoplan的基本信息、药理作用及作用机制、药物代谢动力学、临床疗效、安全性、用法用量、药物最新进展等方面进行综述,旨在为临床应用提供参考。

Pegcetacoplan is a C3 targeting inhibitor that specifically binds to complement C3 and C3b and is used to treat complement mediated diseases.In May 2021,the US Food and Drug Administration(FDA)approved Pegcetacoplan,for the treatment of adult paroxysmal nocturnal hemogloinuria(PNH)as a novel treatment for the rare and life-threatening blood disorder.This paper reviewed the basic information,pharmacological action and mechanism,pharmacokinetics,clinical efficacy,safety,usage and dosage,and the latest progress of Pegcetacoplan,aiming to provide reference for its clinical application.