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Eteplirsen由美国Sarepta Therapeutics公司开发,用于治疗杜氏肌营养不良症(Duchenne muscular dystrophy,DMD),商品名为Exondys 51。该药获得FDA罕见儿童疾病优先审查认证资格~([1]),并于2016年9月19日获批上市,成为第一个获批治疗DMD的药物。Exondys 51的有效成分是eteplirsen,其中文化学名称:[P-脱氧-P-(二甲氨基)](2’,3’-双脱氧-2’,3’-亚氨基-2’,3’-裂环)(2’a→5’)(C-m~5U-C-C-A-A-C-A-m~5U-C-A-AGG-A-A-G-A-m~5U-G-G-C-A-m~5U-m~5U-m~5U-C-m~5U-A-G)
Eteplirsen was developed by Sarepta Therapeutics in the United States for the treatment of Duchenne muscular dystrophy (DMD) under the trade name Exondys51. The drug has received the FDA FDA Rare Child Disease Pre-qualification Certification ([1]) and was approved for marketing on September 19, 2016, making it the first drug to be approved for the treatment of DMD. The active ingredient of Exondys 51 is eteplirsen, of which the chemical name is: [P-deoxy-P- (dimethylamino)] (2 ’, 3’-dideoxy-2’, 3’-imino- (2’a → 5 ’) (Cm ~ 5U-CCAACAm ~ 5U-CA-AGG-AAGAm ~ 5U-GGCAm ~ 5U-m ~ 5U-m ~ 5U-Cm ~ 5U-AG)