Patisiran脂质体ONPATTRO发展及临床应用

Progress and clinical applications of the ONPATTRO~?,a type of siRNA lipid complex injection

获得诺贝尔奖的RNA干扰技术无疑是令人兴奋的重大发现,但RNA类药物存在诸如稳定性差、难以靶向递送等问题限制了其应用。美国Alnylam制药和赛诺菲共同开发的新药patisiran脂质复合物注射液于2018年获批上市治疗遗传性转甲状腺素蛋白淀粉样病变,成为首个siRNA药物的成功临床应用。本文从该产品的处方、作用机制、临床试验及市场等方面进行分析,对ONPATTRO的开发及药效做一简要综述。

The RNA interference technology that won the Nobel Prize is undoubtedly an exciting major discovery,but the shortage of RNA drugs such as poor stability and difficult target delivery limits their clinical application.The new drug"patisiran lipid complex injection"jointly developed by Alnylam Pharmaceuticals and Sanofi was approved by FDA in 2018 for the treatment of hereditary transthyretin amyloidosis and became the first successful clinical application of siRNA drug.This article introduces and analyzes Onpattro from four aspects:formulation,technology,clinical advantages and market conditions,and makes a brief review of the development alone with clinical applications of this first-in-market siRNA liposome injection.