治疗Rett综合征的首个新药——Daybue
Daybue,the first new drug for the treatment of Rett syndrome
李妍 1郝晔2
作者信息
- 1. 山西省儿童医院、山西省妇幼保健院药剂科,山西太原 030013
- 2. 南京医科大学附属儿童医院药学部,江苏南京 210007
- 折叠
摘要
Daybue 口服溶液于2023年3月由美国食品药品监督管理局(FDA)正式批准用于2岁及以上的儿童和成人Rett综合征患者,是第1个也是唯一一个被批准用于治疗Rett综合征的药物.Daybue的主要活性成分是曲非奈肱(trofinetide),是类胰岛素生长因子Ⅰ(IGF-1)的氨基末端三肽的一种新型合成类似物.Daybue已被证明可以通过刺激突触成熟,克服Rett综合征患者病理生理学特征上的突触和神经元不成熟,减少潜在神经炎症以及减轻Rett综合征患者的主要症状.现对其作用机制、药效学、药代动力学、临床研究及安全性等作一介绍.
Abstract
Daybue oral solution has been approved by the U.S.Food and Drug Administration(FDA)in March 2023 for the treatment of Rett syndrome in adults and children aged two years and above,becoming the first and only drug approved for the treatment of Rett syndrome.The main active component of Daybue is trofinetide,a novel synthetic analogue of the amino terminal tripeptide of insulin-like growth factor Ⅰ(IGF-1).It can stimulate synaptic maturation,overcome synaptic and neuronal immaturity,and treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function.This paper introduces it's mechanism of action,pharmacodynamics,pharmacokinetics,clinical research and safety.
关键词
Daybue/曲非奈肱/Rett综合征Key words
Daybue/trofinetide/Rett syndrome引用本文复制引用
出版年
2024
国家科技期刊平台
NSTL
万方数据