血友病B治疗药物情报分析

Intelligence Analysis of Treatments for Hemophilia B

血友病B(HB)是一种X染色体隐性遗传出血性疾病。过去几十年来,HB治疗取得了巨大进展。从凝血酶原复合物到血源性凝血因子提高了治疗安全性,从标准半衰期重组凝血因子到生物工程长效重组凝血因子改善了患者依从性和临床结局。非因子替代疗法不但能够通过皮下给药为患者减轻治疗负担,还避免了产生抑制物的风险,同时为已产生抑制物的患者提供了新的治疗方案。腺相关病毒载体基因治疗开启了HB的新篇章,目前已有一种基因治疗在美国获批上市。该文通过对欧美等发达国家及中国上市药物信息追踪,对在研药物研发管线及主要研究成果等信息进行挖掘,对HB治疗药物进行全视角、全周期情报解读,以期为医疗机构及相关研发单位提供参考。

Hemophilia B(HB)is a recessive congenital bleeding disorder with X-linked inheritance.The treatments for HB have made tremendous progress during past decades.Treatment paradigm shifting from prothrombin complex to plasma-derived coagulation factor IX(FIX)significantly improved the safety of patients.Bioengineered recombinant FIXs(rFIXs)with extended half-life compared to rFIXs ameliorat patient's compliance and clinical outcomes.Non-factor replacement therapies could reduce the burden of treatment through subcutaneous injection and avoid the risk of developing inhibitors to FIX.Meanwhile,these molecules could provide effective prophylaxis in the presence or absence of inhibitor.Genetically modified adeno-associated virus engineered to deliver FIX gene to the liver has heralded a new era of HB treatment.A comprehensive intelligence tracking of the treatments for HB,including currently marketed pharmaceuticals,pipelines of molecules under development and related research results is the focus of this paper.