进行性骨化性纤维发育不良治疗药物——帕罗伐汀  

A new drug for fibrodysplasia ossification progressive:palovarotene

作　　者：王欣雨 叶红梅 封宇飞 WANG Xin-yu;YE Hong-mei;FENG Yu-fei(School of Life Sciences and Biopharmaceutics,Shenyang Pharmaceutical University,Shenyang 110016,China;Clinical Trial Institute,Peking University People's Hospital,Beijing 100044,China)

机构地区：[1]沈阳药科大学生命科学与生物制药学院,沈阳110016 [2]北京大学人民医院临床试验机构,北京100044

出　　处：《临床药物治疗杂志》2024年第5期23-26,38,共5页Clinical Medication Journal

摘　　要：帕罗伐汀是由法国益普生生物制药公司开发的一种可口服的选择性视黄酸受体(RAR)激动剂,用于治疗8岁以上的女性儿童、10岁以上的男性儿童和成年进行性骨化性纤维发育不良(FOP)患者。2023年8月16日,FDA批准帕罗伐汀上市。本文就帕罗伐汀的基本信息、作用机制、药动学、临床疗效、安全性等研发动态进行综述,以期为临床合理用药提供参考。Palovarotene is an orally available selective retinoic acid receptor(RAR)agonist developed by Ipsen Biophar⁃maceuticals Company of France for the treatment of children(female children over 8 years of age and male children over 10 years of age)and adults with fibrodysplasia ossification progressiva(FOP).On August 16,2023,the U.S.FDA approved Pal⁃ovarotene for marketing.In this paper,the pharmacological action,mechanism,pharmacokinetics,clinical efficacy,and safety of palovarotene were reviewed to provide some guidance for clinical rational drug use.

关 键 词：帕罗伐汀 进行性骨化性纤维发育不良 临床评价 

分 类 号：R977.9[医药卫生—药品]