Health Affairs August 29, 2023
On June 22, the Food and Drug Administration (FDA) granted accelerated approval to Elevidys, a new gene therapy for Duchenne Muscular Dystrophy (DMD). The decision was controversial for two reasons. First, the evidence submitted by the manufacturer, Sarepta, failed to show significant clinical benefit using the criteria agreed upon by Sarepta and the FDA. Second, Sarepta is conducting a larger trial and has promised to report results by the end of 2023. Many members of the FDA Advisory Panel thought that it would be prudent to wait a few months for the results of that trial.
The controversial decision and the process by which it was made has implications for many other therapies for rare diseases that are in the...
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