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.
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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’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).
Gottlieb Places Drug Pricing Out Front In First Speech To US FDA Staff
New FDA Commissioner tells employees that 'too many consumers are priced out of medicines they need' and that FDA must take meaningful steps to get lower cost alternatives to market.