Eteplirsen

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 25, 2017; expires September 25, 2020

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 on September 19, 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 has an effect on 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, but on October 7, 2016, 1 major insurance company, Anthem, announced that they will not reimburse eteplirsen treatment.

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