纯合子家族性高胆固醇血症治疗药物的临床研究进展  

Clinical research progress on therapeutic drugs for homozygous familial hypercholesterolemia

作　　者：任燕 汪璐 丁子文 郑岩 倪娜 邱云良 王俊龙 REN Yan;WANG Lu;DING Ziwen;ZHENG Yan;NI Na;QIU Yunliang;WANG Junlong(School of Chemistry and Chemical Engineering,Shanghai University of Engineering Science,Shanghai 201620,China;Clinical Research Center,China State Institute of Pharmaceutical Industry,Shanghai 200437,China;Shanghai Clinical Research Organization for Medicines,Shanghai 200437,China;National Shanghai Center for New Drug Safety Evaluation and Research,China State Institute of Pharmaceutical Industry,Shanghai 201203,China)

机构地区：[1]上海工程技术大学化学化工学院,上海201620 [2]中国医药工业研究总院药物临床研究中心,上海200437 [3]上海瀛科隆医药开发有限公司,上海200437 [4]中国医药工业研究总院国家上海新药安全评价研究中心,上海201203

出　　处：《世界临床药物》2022年第6期782-786,共5页World Clinical Drug

摘　　要：纯合子家族性高胆固醇血症(homozygous familial hypercholesterolaemia,HoFH)是一种严重且罕见的高胆固醇血症,其特征是低密度脂蛋白胆固醇水平显著升高,过早出现动脉粥样硬化性心血管疾病。目前已有依维苏单抗、米泊美生钠、洛美他派及前蛋白转化酶枯草溶菌素9抑制剂等药物被批准用于治疗HoFH,有效地解决了罕见病治疗药物缺乏的问题。现主要对治疗HoFH的药物临床研究进展作一简要概述。Homozygous familial hypercholesterolaemia(HoFH)is a severe and rare hypercholesterolemia,which is characterized by a significant increase in low density lipoprotein-cholesterol(LDL-C)and premature atherosclerotic cardiovascular disease.At present,evinacumab,mipomersen,lomitapid and proprotein convertase subtilisin/kexin type 9 inhibitors have been approved for the treatment of HoFH,which effectively solves the problem of the lack of drugs for rare diseases.The article briefly reviewed the clinical research progress on drugs for the treatment of HoFH.

关 键 词：纯合子家族性高胆固醇血症 罕见病 罕用药 

分 类 号：R972[医药卫生—药品]