FDA panel rejects Sarepta drug to treat Duchenne muscular dystrophy
Aaron S. Kesselheim, associate professor at Harvard Medical School, is quoted.
The Boston Globe
By Robert Weisman
An influential advisory committee narrowly declined to recommend US sale of an experimental treatment for Duchenne muscular dystrophy Monday, disappointing hundreds of patient advocates who argued for approval of the medicine.
The panel of medical experts concluded in a 7-6 vote that a clinical study by Cambridge biotech Sarepta Therapeutics Inc. failed to meet the Food and Drug Administration’s standard for accelerated approval. In a separate 7-3 vote, with three members abstaining, the committee determined that Sarepta’s clinical trial was poorly designed and didn’t prove the drug candidate was effective in treating the fatal muscle-wasting disease.
Coming at the end of a 12-hour meeting marked by emotionally-charged testimony from boys with Duchenne and family members, the nonbinding votes leave the fate of Sarepta’s drug, called Eteplirsen, in the hands of the FDA. The agency, which is set to rule on the drug application by May 26, historically has relied heavily on its advisory committees for guidance.
Summing up a widely held view on the panel, committee member Aaron S. Kesselheim, associate professor at Harvard Medical School in Boston, said “the studies provided by [Sarepta] were not adequate and well controlled.” But he acknowledged that it remains an “open question” whether Eteplirsen produces a clinical benefit to patients who take it.