After comparing data and patient testimonials, a U.S. Food and Drug Administration (FDA) advisory panel voted 8-5 against the approval of a new Duchenne muscular dystrophy (DMD) drug due to its failure to meet the minimum standards of the administration's process.
The Peripheral and Central Nervous System Drugs Advisory Committee determined that the drug did not meet standards due to a lack of "substantial evidence from adequate and well-controlled studies" that proved the drug can stimulate dystrophin production enough to be considered "reasonably likely to predict clinical benefit."
The panel's patient representative was 23-year-old Benjamin Dupree, a resident of Dallas with DMD that was very conflicted with the process.
"The study doesn't provide what I think is adequate evidence to support all of this testimony that I'm seeing in here," he said.
DMD is an X chromosome-linked genetic disorder that leads to the absence of the dystrophin protein that keeps muscles intact. It almost always affects boys and typically leaves patients wheelchair-bound due to the deterioration of muscles and motor functions. Although survival rates have increased in recent years, many patients die by their mid-20s.
The drug in question is Eteplirsen, tentatively branded Exondys 51, and was proposed for the treatment of DMD in patients with a specific mutation of the dystrophin gene - exon 51. This mutation in the exon 51 gene leads to a lack of dystrophin protein, a mutation that the drug is said to prevent by inhibiting gene translation, allowing muscle cells to produce dystrophin, although in a shortened form.
Numerous parents and patients showed up to the hearing, which ran for nearly 12 hours as presentations and anecdotal evidence were presented to push the positive effects of the drug. However, in the end, the FDA ruled in favor of the current scientific data.
"We come to you with sincere concerns, not because we take some perverse delight in keeping new medicines from those who urgently need them... but because it is our - we the FDA, and you, our advisory committee - collectively, it is our fundamental responsibility to ensure, as required by law, that the treatments we approve are effective," said William Dunn, director of the FDA's Office of Drug Evaluation I for the Division of Neurology Products.
