A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not Approved in Europe

Abstract

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.

Keywords: Duchenne muscular dystrophy; clinical trials; regulatory approval.