Lamb Yvette N
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Drugs. 2025 Feb;85(2):263-269. doi: 10.1007/s40265-024-02130-4. Epub 2024 Dec 24.
Marstacimab (marstacimab-hncq; HYMPAVZI™) is a subcutaneously administered human monoclonal immunoglobulin G1 antibody against tissue factor pathway inhibitor (TFPI) that is being developed by Pfizer for the treatment of hemophilia A and B. Marstacimab received its first approval on 11 October 2024 in the USA. It was approved for use as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors. Marstacimab has since been approved on 18 Nov in the EU for the treatment of adults and adolescents with severe hemophilia A or B without inhibitors. This article summarizes the milestones in the development of marstacimab leading to this first approval for hemophilia.
马司他单抗（marstacimab-hncq；商品名：HYMPAVZI™）是一种皮下注射的抗组织因子途径抑制剂（TFPI）的人源单克隆免疫球蛋白G1抗体，由辉瑞公司研发用于治疗A型和B型血友病。马司他单抗于2024年10月11日在美国首次获批。它被批准用于12岁及以上无凝血因子VIII抑制剂的A型血友病或无凝血因子IX抑制剂的B型血友病成年和儿科患者，作为常规预防措施以预防或减少出血事件的发生频率。此后，马司他单抗于11月18日在欧盟获批，用于治疗无抑制剂的重度A型或B型血友病成人和青少年。本文总结了马司他单抗在血友病首次获批之前的研发历程中的重要节点。
