治疗CHAPLE病的新一代C5补体抑制药帕泽利单抗  

Pozelimab,a new generation of C5 complement inhibitors for CHAPLE disease

作　　者：张玄龄 陈超阳[1] 魏然 周颖[1,2] ZHANG Xuan-ling;CHEN Chao-yang;WEI Ran;ZHOU Ying(Department of Pharmacy,Peking University First Hospital,Beijing 100034,China;Drug Clinical Trial Facility,Peking University First Hospital,Beijing 100034,China)

机构地区：[1]北京大学第一医院药学部,北京100034 [2]北京大学第一医院药物临床试验机构,北京100034

出　　处：《中国临床药理学杂志》2024年第2期276-278,共3页The Chinese Journal of Clinical Pharmacology

摘　　要：2023年8月18日美国食品药品监督管理局(FDA)正式批准再生元公司的帕泽利单抗(商品名:Veopoz®)用于治疗1岁及以上儿童和成人患者的CD55缺陷型蛋白丢失性肠病(CHAPLE)。帕泽利单抗是一种重组单克隆抗体(免疫球蛋白G4型),通过结合末端补体蛋白C5,阻断C5裂解为C5a(过敏毒素)和C5b,阻止膜攻击复合物的形成,从而抑制末端补体活化。本文从帕泽利单抗的药理研究、药代动力学、临床研究、安全性等方面进行综述,以介绍该药的研究现状和已有成果。On August 18,2023,the U.S.Food and Drug Administration(FDA)officially approved Regeneron’s Pozelimab-bbfg(Veopoz®)for the treatment of complement factor H-related protein 5 deficiency type protein-losing enteropathy(CHAPLE)disease in children aged 1 and above,as well as adult patients.CHAPLE disease is a rare condition.Pazelimab monoclonal antibody is a recombinant monoclonal antibody(immunoglobulin G4 isotype).This antibody works by binding to the terminal complement protein C5,thereby preventing the cleavage of C5 into C5a(an anaphylatoxin)and C5b,and inhibit the activation of the terminal complement pathway.This article provides an overview of Pozelimab-bbfg,including its pharmacological research,pharmacokinetics,clinical studies,safety profile,and more,to introduce the current state of research and the achievements made with this drug.

关 键 词：帕泽利单抗 CD55缺陷型蛋白丢失性肠病 

分 类 号：R97[医药卫生—药品]