首个雷特综合征治疗药物trofinetide  

Trofinetide:the first drug for the treatment of Rett syndrome

作　　者：陈静 罗颖 陈寅波 刘相奎 CHEN Jing;LUO Ying;CHEN Yin-bo;LIU Xiang-kui(China State Institute of Pharmaceutical Industry Co.,Ltd.,Shanghai 201203,China)

机构地区：[1]中国医药工业研究总院有限公司,上海201203

出　　处：《中国新药杂志》2024年第17期1756-1760,共5页Chinese Journal of New Drugs

摘　　要：trofinetide是胰岛素样生长因子1(insulin like growth factor-1,IGF-1)的氨基末端三肽甘氨酸-脯氨酸-谷氨酸的合成类似物,于2023年3月10日被美国FDA批准上市,用于治疗2岁及以上成人和儿童患者的Rett综合征。trofinetide是第一种被批准用于治疗Rett综合征的药物,本文对其作用机制、药效学、药动学、临床评价、安全性进行综述。Trofinetide is a synthetic analog of glycine⁃proline⁃glutamate which is the N⁃terminal tripeptide derivative of insulin like growth factor⁃1(IGF⁃1).It was approved in march 2023 in the USA for the treatment of Rett syndrome in adult and pediatric patients 2 years of age and older.Trofinetide is the first drug approved for the treatment of Rett syndrome,and this article reviews the mechanism of action,pharmacodynamics,pharmacokinetics,clinical evaluation,and safety of trofinetide.

关 键 词：trofinetide RETT综合征 胰岛素样生长因子1 

分 类 号：R971[医药卫生—药品]