A型血友病新药——Esperoct研究现状

Research status of Esperoct,a new hemophilia A drug

2019年2月美国食品药品监督管理局(FDA)批准Esperoct(turoctocog alfa pegol,N8-GP)用于A型血友病患者的常规预防以减少出血发作频率、按需治疗控制出血发作和围手术期出血管理。该药于2020年2月在美国上市销售。Esperoct是一种糖基聚乙二醇化形式的重组凝血因子Ⅷ。糖基聚乙二醇化技术能够延长重组凝血因子Ⅷ的循环半衰期,从而减少静脉给药次数,并降低出血发作频率。Esperoct的给药方案简单和固定,对于重度A型血友病患者中可提供有效的常规预防,且对治疗和控制出血事件和围手术期管理方面同样有效。

In February 2019,Esperoct(turoctocog alfa pegol,N8-GP)has been approved by the US Food and Drug Administration(FDA)for routine prevention of hemophilia A patients to reduce the frequency of bleeding episodes,to control bleeding episodes on demand,and to manage perioperative bleeding.Esperoct was marketed in the United States on February 2020.Esperoct is a recombinant coagulation factorⅧproduct in the form of glycosyl PEGylation.Glycosyl PEGylation can prolong the circulating half-life of recombinant coagulation factorⅧ,thus reducing the number of intravenous administration and the frequency of bleeding attack.The results show that Esperoct can provide effective routine prevention in patients with severe hemophilia A through a simple and fixed medicine application plan,and it is also effective in the treatment and controlment of bleeding events and perioperative management.