治疗纯合子型家族性高胆固醇血症新药:evinacumab

A new drug in treating homozygous familial hypercholesterolemia:evinacumab

纯合子型家族性高胆固醇血症(HoFH)是一种罕见的低密度脂蛋白胆固醇(LDL-C)水平异常高的遗传性疾病。血管生成素样蛋白3(ANGPTL3)是治疗高脂血症的新靶点,evinacumab(Evi)是ANGPTL3抑制剂。Evi于2021年2月被美国食品和药物管理局批准上市,作为其他降低LDL-C疗法的辅助药物,用于治疗12岁及以上纯合子家族性高胆固醇血症的成人和儿童患者。目前临床研究表明,Evi能有效降低HoFH患者的LDL-C水平,常见不良反应是鼻咽炎。

Homozygous familial hypercholesterolemia(HoFH)is a rare hereditary disorder,pathophysiologically featuring the abnormally increased serum low-density lipoprotein cholesterol(LDL-C).Angiopoietin-like 3 protein(ANGPTL3)is the new therapeutic target for hyperlipidemia.Evinacumab(Evi)is an ANGPTL3 inhibitor approved by U.S.Food and Drug Administration in February 2021 as an adjunct to other LDL-C lowering therapies for the treatment of adult and pediatric patients,aged 12 years and older with HoFH.Current clinical trials have demonstrated that evinacumab is effective for lowering LDL-C in patients with HoFH,and a common adverse reaction is nasopharyngitis.