治疗Duchenne型肌营养不良新药eteplirsen

Eteplirsen,a novel drug for the treatment of DMD

Eteplirsen是一种靶向51号外显子的磷酰吗啉寡核苷酸,通过诱导dystrophin基因51号外显子跳读,使其mRNA阅读框不被破坏,从而促进DMD患者dystrophin蛋白的生成,使致死型DMD转变为临床表型较轻的BMD。动物试验及临床研究显示,Eteplirsen具有良好的安全性和耐受性。2016年9月,美国FDA批准其上市。本文将对其药理作用、药动学、临床研究、安全性等进行综述。

Eteplirsen,a morpholino phosphorodiamidate oligonuclcotide,forces the exclusion of exon 51 from the mRNA of dystrophin protein,which induces dystrophin production. As a result,Duchenne muscular dystrophy（DMD）,a fatal disease,will turn into Becker muscular dystrophy（BMD）,a milder form of muscular dystrophy. In vitro assays and in vivo studies showed that eteplirsen is well tolerated and has mild side effects.Although under fire,eteplirsen has been approved by FDA in September 2016. We summarized and analyzed its pharmacological effect,pharmacokinetics,clinical use,and safety.