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Eteplirsen Revisited? FDA Panel To Weigh Protocol Changes For Two Sarepta Drugs

Executive Summary

US agency’s advisory committees will consider whether ongoing trial of exon 45- and 53-skipping compounds for Duchenne muscular dystrophy should allow use of in-dwelling ports to aid infusion.

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Sarepta Trial Protocol Change Could Spur More IRB Referrals To US FDA

Investigational review boards might be more inclined to bring pediatric study design issues to FDA’s attention for public review following agency’s quick decision to allow in-dwelling ports for patients in Sarepta’s placebo-controlled ESSENCE trial.

Sarepta Protocol Changes Could Impact Future Duchenne Trials

If US FDA Commissioner Gottlieb allows use of in-dwelling ports in ESSENCE study, Office of Pediatric Therapeutics will assess whether the determination could serve as precedent for placebo-controlled trials of other exon-skipping compounds in Duchenne muscular dystrophy.

Sarepta’s ESSENCE Trial Gets FDA Panel Nod For Protocol Changes

US agency’s experts unanimously support allowing use of in-dwelling ports to aid infusions in trial of two Duchenne muscular dystrophy compounds, while also endorsing the two-year, double-blind, placebo-controlled design, which was influenced by the clinical experience with Exondys 51 (eteplirsen).

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