A U.S. Food & Drug Administration (FDA) advisory panel voted not to support the accelerated approval of a drug aimed at helping boys with Duchenne muscular dystrophy (DMD).
According to Muscular Dystrophy News Today, the panel did not recommend a drug called Eteplirsen, manufactured by Cambridge, Mass.-based Sarepta Therapeutics.
The panel’s decision not to support the drug puts the medication’s accelerated approval status in jeopardy, since the FDA typically relies heavily on advisory panel recommendations.
Muscular Dystrophy News Today said Eteplirsen “would be the first drug approved for treating DMD that addresses the disease’s genetic cause.”
The panel reportedly rejected the drug because it did not believe Sarepta had proven its efficiency in a clinical trial of 12 patients, though some parents of children with DMD defended the drug’s efficiency as they addressed the FDA.
In an accelerated approval process, the FDA can approve a drug aimed at treating a serious medical condition before the drug has actually proved it delivers a clinical benefit. In the case of an accelerated approval, the FDA says it instead considers a “surrogate endpoint,” such as a laboratory measurement, radiographic image or other positive indication, to grant approval to a much-needed drug, thus shortening the FDA approval process.