1 Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.
2 Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium.
Nucleic Acid Ther. 2019 Feb;29(1):13-15. doi: 10.1089/nat.2018.0756. Epub 2018 Dec 11.
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.
依特司群于 2016 年 9 月获得批准，用于治疗符合条件的杜氏肌营养不良症（DMD）患者，这是美国食品和药物管理局（FDA）有史以来最具争议性的批准之一，如果不是最具争议性的批准之一。两年后，欧洲药品管理局（EMA）人用医药产品委员会（CHMP）对依特司群治疗持否定意见。他们早在 2018 年 5 月也表达过同样的看法，之后，开发依特司群的 Sarepta 公司提出上诉，启动了新的评估，包括涉及 DMD 专家和患者代表的科学咨询小组（SAG）会议。然而，重新评估后，CHMP 的意见仍然是否定的。在这篇评论中，我们概述了 FDA 和 EMA 的观点差异如何导致一种 DMD 疗法被 FDA 批准但未被 EMA 批准，反之亦然。
