Exondys 51’s Development: Was Placebo-Controlled Trial Possible?
Sarepta says US FDA said ‘no,’ but agency repeatedly urged the company to pursue the approach.
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User fee reauthorization negotiators from US FDA and industry divide into subgroups to tackle specific issues before moving to the broader agreement.
Sarepta will face a US FDA staff with which it does not have a controversial history, the Center for Biologics Evaluation and Research, likely avoiding the reviewers that evaluated its Duchenne muscular dystrophy treatment Exondys 51.
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.