K K Jain MD (Dr. Jain is a consultant in neurology and has no relevant financial relationships to disclose.)
Originally released October 7, 2016; last updated September 30, 2019; expires September 30, 2022

This article includes discussion of eteplirsen and AVI-4658. The foregoing terms may include synonyms, similar disorders, variations in usage, and abbreviations.

Historical note and terminology

Eteplirsen is an antisense-mediated exon skipping therapy for mutation suppression in Duchenne muscular dystrophy. Drisapersen, which has a similar mechanism of action, failed in phase 3 clinical trials, but eteplirsen was granted accelerated approval by the U.S. Food and Drug Administration in 2016 for treatment of some forms of Duchenne muscular dystrophy. Approval under an accelerated pathway can be based on adequate and well-controlled clinical trials showing that the drug affects a surrogate endpoint that is reasonably likely to predict clinical benefit to patients. This pathway provides earlier patient access to promising new drugs while the manufacturer conducts clinical trials to verify the predicted clinical benefit. Early-stage development of this drug started 20 years earlier and was supported by the Muscular Dystrophy Association. There is some controversy about the FDA s approval of this drug as some critics have used terms like “lowering the bar” to approve “an expensive placebo.” Eteplirsen initially costs $300,000 to $400,000 per year per patient. Several insurance companies have already approved coverage of eteplirsen. Two years after approval by the U.S. Food and Drug Administration, the Committee for Human Medicinal Products of the European Medicines Agency gave a negative opinion for eteplirsen treatment, and it was not approved in Europe (Aartsma-Rus and Goemans 2019).

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