Duchenne Muscular Dystrophy: Second Product Isn't The Charm
This article was originally published in The Pink Sheet Daily
FDA reviewers say patients treated with Sarepta's eteplirsen seemed to follow similar disease course as Duchenne muscular dystrophy natural history, casting doubts on product's approvability.
You may also be interested in...
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.
Biopsy results may be informative in early phase trials, but costs have limited their use in US clinical practice, raising questions about whether procedure should be required in drug development.
US FDA officials say they need knowledge of the disease to have the confidence to use accelerated approval.