治疗肌萎缩侧索硬化症新药Relyvrio  

Relyvrio,a new approved drug for the treatment of amyotrophic lateral sclerosis

作　　者：潘芳 曹静[2] PAN Fang;CAO Jing(Department of Pediatric Surgery,Children's Hospital of Nanjing Medical University,Nanjing 210008,Jiangsu Province,China;Departmenttof Pharmacy,Women's Hospital of Nanjing Medical University,Nanjing Maternity and Child Health Care Hospital,Nanjing 210004,Jiangsu Province,China)

机构地区：[1]南京医科大学附属儿童医院普儿科,江苏南京210008 [2]南京医科大学附属妇产医院、南京市妇幼保健院药剂科,江苏南京210004

出　　处：《中国临床药理学杂志》2023年第17期2552-2555,共4页The Chinese Journal of Clinical Pharmacology

摘　　要：苯丁酸钠和牛磺酸二醇(Relyvrio)的口服固定剂量联合制剂是Amylyx Pharmaceuticals正在开发的用于治疗神经退行性疾病的药物。2022年9月,该联合制剂获美国食品药品监督管理局(FDA)正式批准,用于治疗成人肌萎缩侧索硬化症(ALS)。该批准基于多中心Ⅱ期CENTAUR试验的结果,与安慰剂相比,Relyvrio可减缓ALS的进展。本文从作用机制、药效学、药代动力学、临床研究及安全性等方面对其做一介绍。An oral fixed-dose combination formulation of sodium phenylbutyrate and taurine diol(Relyvrio)is a drug being developed by Amylyx Pharmaceuticals for the treatment of neurodegenerative diseases.In September 2022,the combination formulation was officially approved by the U.S.Food and Drug Administration(FDA)for the treatment of adult amyotrophic lateral sclerosis(ALS).The approval was based on results from the multicenter phase 2 trial(CENTAUR),which showed that Relyvrio slowed the progression of ALS compared with placebo.This paper will introduce Relyviro from the aspects of mechanism of action,pharmacodynamics,pharmacokinetics,clinical research and safety.

关 键 词：Relyvrio 肌萎缩侧索硬化症 神经退行性疾病 苯丁酸钠 牛磺酸二醇 

分 类 号：R97[医药卫生—药品]