药物基本信息
通用名：人凝血因子Ⅸ
临床用途：血友病B[凝血因子Ⅸ（FⅨ）缺陷症]2型
市场需求
B型血液病是一种罕见的伴性X染色体隐性遗传病，以凝血功能缺陷为特征，患者主要为男性，该病是由于患者体内编码凝血因子IX的基因发生突变，体内凝血因子IX活性显著降低或无活性，导致凝血功能下降，因此患者会经历反复发作的严重出血事件，而凝血因子IX对于正常的血液凝固至关重要。目前，B型血友病的标准护理疗法涉及定期
静脉输注血浆来源的或重组的凝血因子IX，来控制和预防出血发作。据全球B型血液病
联盟估计，在世界范围内大约有2.8万例B型血液病患者。
目前上市的凝血因子Ⅸ是从血浆中提取获得，具有病毒感染的风险，同时还受到血源的限制，随着基因工程技术的不断发展、成熟，重组凝血因子日渐取代了人凝血因子。除此以外，重组凝血因子经过结构修饰，如糖基化、融合蛋白、PEG修饰，可大幅延长作用时间，明显减少注射次数，长效凝血因子代替普通凝血因子是必然的趋势。
The basic information of drug
Common name: FIX
Clinical use: treatment of hemophilia B [coagulation factor Ⅸ (FIX) deficiency] II
Market demand
Blood disease B is a rare companion X chromosome recessive genetic disease, characterized by coagulation defects. The patients are mainly male, the disease is due to the patients` body code coagulation factor IX gene mutation. The body coagulation factor IX activity significantly is reduced or inactive, leading to decreased coagulation. So patients will experience recurrent episodes of severe bleeding, and coagulation factor IX is essential for normal blood clotting. Currently, standard care for hemophilia B involves periodic intravenous infusion of plasma-derived or recombinant coagulation factor IX to control and prevent bleeding episodes. According to the Global Type Blood Disease B Alliance, there are about 28,000 patients with hematological B diseases worldwide.
In the current, the coagulation factor Ⅸ is extracted from the plasma, with the risk of viral infection. At the same time, it is also limited by the blood sources. With the continuous development, mature of genetic engineering, recombinant coagulation factor increasingly replaces the human coagulation factor. In addition, the recombinant coagulation factor through structural modification, such as glycosylation, fusion protein, PEG modification, can significantly prolong the role of time and significantly reduce the number of injections. Long-acting coagulation factor instead of ordinary coagulation factor is an inevitable trend.
(血友病B2型患者经历反复发作的严重出血事件,图片来源健康经验网)
