Eteplirsen was approved for the treatment of eligible patients with Duchenne muscular dystrophy (DMD) in September 2016 in one of the most, if not the most, controversial approvals ever made by the Food and Drug Administration of the United States. Two years later, the Committee for Human Medicinal Products (CHMP) of the European Medicines Agency gave a negative opinion for eteplirsen treatment. They had done so as well in May 2018, after which Sarepta (the company developing eteplirsen) appealed and a new evaluation was initiated, including a Scientific Advisory Group (SAG) meeting involving DMD experts and patient representatives. However, after reevaluation the opinion of the CHMP remained negative. In this commentary, we outline how differences in the perspective of FDA and EMA can lead to a DMD therapy being approved by FDA but not EMA, and vice versa.

中文翻译：

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Eteplirsen Saga的续集：Eteplirsen在美国获得批准，但在欧洲未获得批准。

2016年9月，Eteplirsen被批准用于治疗符合条件的Duchenne肌营养不良症（DMD）患者，这是美国食品与药物管理局（FDA）批准的最多（甚至不是最多）有争议的批准之一。两年后，欧洲药品管理局的人类药物产品委员会（CHMP）对eteplirsen治疗提出了负面意见。他们在2018年5月也这样做了，此后Sarepta（开发eteplirsen的公司）提出了上诉，并开始了新的评估，包括由DMD专家和患者代表参加的科学咨询小组（SAG）会议。但是，在重新评估后，CHMP的意见仍然是负面的。在这篇评论中，我们概述了FDA和EMA观点的差异如何导致DMD治疗获得FDA批准，但未获得EMA批准，