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Drugs. 2023 Nov;83(16):1551-1557. doi: 10.1007/s40265-023-01955-9.
Pozelimab (pozelimab-bbfg; VEOPOZ™) is a fully human immunoglobulin (Ig) G4 (i.e. IgG4 with a proline substitution to promote stabilization of the disulfide bonds between the two heavy chains) monoclonal antibody developed by Regeneron Pharmaceuticals Inc., to block the activity of complement factor 5 (C5) and prevent diseases mediated by the complement pathway. In August 2023, pozelimab received its first approval for the treatment of adults, and paediatric patients aged ≥ 1 year with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease, in the USA. It is the first US FDA-approved treatment for this disease. In the USA, pozelimab has been granted orphan drug designations for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) [both as a monotherapy and in combination with cemdisiran] and for the treatment of myasthenia gravis (in combination with cemdisiran). Pozelimab is also undergoing clinical development in several other countries worldwide for the treatment of CD55-deficient PLE, PNH and myasthenia gravis. This article summarizes the milestones in the development of pozelimab leading to this first approval for the treatment of adults, and paediatric patients aged ≥ 1 year with CD55-deficient PLE, also known as CHAPLE disease, in the USA.
波泽利单抗（pozelimab-bbfg；VEOPOZ™）是一种由再生元制药公司开发的完全人免疫球蛋白（Ig）G4（即 IgG4，脯氨酸取代以促进两条重链之间二硫键的稳定）单克隆抗体，用于阻断补体因子 5（C5）的活性并预防补体途径介导的疾病。2023 年 8 月，波泽利单抗在美国首次获批用于治疗成人和≥1 岁的 CD55 缺乏性蛋白丢失性肠病（PLE），也称为 CHAPLE 病的儿科患者。这是美国首个获得批准的该疾病治疗药物。在美国，波泽利单抗已被授予孤儿药资格，用于治疗阵发性夜间血红蛋白尿（PNH）[包括单药治疗和与 cemdisiran 联合治疗]和重症肌无力（与 cemdisiran 联合治疗）。波泽利单抗也在全球其他几个国家进行临床开发，用于治疗 CD55 缺乏性 PLE、PNH 和重症肌无力。本文总结了波泽利单抗开发过程中的重要里程碑，最终该药在美国获批用于治疗成人和≥1 岁的 CD55 缺乏性 PLE，也称为 CHAPLE 病的儿科患者。
