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