治疗原发性高草酸尿症1型新药nedosiran的作用机制与临床评价  

Mechanism and clinical evaluation of a novel drug nedosiran for the treatment of primary hyperoxaluria type 1

作　　者：杜文林 罗春梅 吴小燕 张蓉 DU Wen-lin;LUO Chun-mei;WU Xiao-yan;ZHANG Rong(Department of Pharmacy,The Second Affiliated Hospital of Army Medical University,Chongqing 400037,China;National Drug Clinical Trail Institution,The Second Affiliated Hospital of Army Medical University,Chongqing 400037,China)

机构地区：[1]陆军军医大学第二附属医院药剂科,重庆400037 [2]陆军军医大学第二附属医院国家药物临床试验机构,重庆400037

出　　处：《中国新药杂志》2024年第19期2023-2027,共5页Chinese Journal of New Drugs

基　　金：重庆市临床药学重点专科建设项目(综合项目)(渝卫办发(2023)69号)。

摘　　要：nedosiran是诺和诺德制药公司(Novo Nordisk)研制的一种新型治疗原发性高草酸尿症1型的小干扰RNA药物,靶向作用于肝细胞中的乳酸脱氢酶A,降低乳酸脱氢酶的表达,有效减少草酸盐的产生并阻止草酸钙晶体沉积,从而降低尿草酸盐水平,于2023年9月29日获美国FDA批准上市,适用于降低9岁及以上儿童和成人的原发性高草酸尿1型患者尿草酸水平。现对nedosiran的用法用量、作用机制、药动学、临床评价、安全性等方面进行综述,以期为临床安全、合理用药提供理论参考。Nedosiran is a novel small interfering RNA drug developed by Novo Nordisk Pharmaceutical company for the treatment of primary hyperoxaluria type 1.It targets lactate dehydrogenase A in liver cells and reduces the expression of lactate dehydrogenase.Nedosiran effectively reduces oxalate production and prevents calcium oxalate crystal deposition,thereby reducing urinary oxalate levels.It was approved by the US FDA on September 29th,2023 and indicated to lower urinary oxalate levels in children at 9 years of age and adults with primary hyperoxaluria type 1.This article summarizes the use and dosage,mechanism of action,pharmacokinetics,clinical evaluation and safety of nedosiran to provide a theoretical reference for clinically safe and rational use of this drug.

关 键 词：nedosiran 原发性高草酸尿症1型 作用机制 临床评价 

分 类 号：R961[医药卫生—药理学]