治疗神经纤维瘤病新药--丝裂原活化蛋白激酶抑制剂Selumetinib

A New Drug in Treatment of Neurofibromatosis: Mitogen-Activated Protein Kinase Inhibitors Selumetinib

Selumetinib(商品名:Koselugo)是阿斯利康和默沙东公司共同研发的丝裂原活化蛋白激酶抑制剂,用于治疗2岁及以上患有神经纤维瘤病1型(NF1)和有症状的、无法手术的丛状神经纤维瘤(PN)的儿童患者。2020年4月10日,Selumetinib被美国食品药品监督管理局(FDA)批准成为首个治疗NF1和PN儿童患者的口服药物。本文主要介绍Selumetinib的药理作用机制、药效学、药动学、临床试验和安全性等最新研究进展,为临床用药提供参考。

Selumetinib(trade name:Koselugo)is a mitogen-activated protein kinase inhibitor being developed by AstraZeneca and Merck,for the treatment of children aged 2 years or older with neurofibromatosis type 1(NF1)and children with symptomatic,inoperable plexus neurofibromatosis(PN).On April 10,2020,it was approved by the US Food and Drug Administration(FDA)as the first oral drug for children with NF1 and PN.This paper mainly introduces the pharmacological action mechanism,pharmacodynamics,pharmacokinetics,clinical trials and safety of selumetinib,and provides references for clinical medication.