Despite Pleas From Patients, FDA Panel Votes Against Drug For Muscular Dystrophy
The panel members said the trial was not well-controlled, noting that it only involved 12 patients without an adequate placebo control.
The New York Times:
Advisers To F.D.A. Vote Against Duchenne Muscular Dystrophy Drug
In a confrontation between the hopes of desperate patients and clinical trial data, advisers to the Food and Drug Administration voted on Monday not to recommend approval of what would become the first drug for Duchenne muscular dystrophy. The negative votes came despite impassioned pleas from patients, parents and doctors who insisted that the drug, called eteplirsen, was prolonging the ability of boys with the disease to walk well beyond when they would normally be in wheelchairs. (Pollack, 4/25)
The Wall Street Journal:
FDA Panel Votes Not To Recommend Approval For Muscular Dystrophy Drug
“I felt this wasn’t a well-controlled study,” said panel chairman G. Caleb Alexander, an associate epidemiology professor at the Johns Hopkins School of Public Health. The panel members who abstained said they were undecided because they were moved by public testimony from parents who believed their children had been helped by the drug. The decision followed decidedly negative comments from FDA reviewers who concluded that the study fell far short of producing enough evidence. (Burton, 4/25)
The Associated Press:
Muscular Dystrophy Drug Falls Short At High-Stakes Meeting
Federal health advisers voted against an experimental treatment for muscular dystrophy on Monday, the latest in a series of setbacks for the Sarepta Therapeutics' drug, which has become a rallying point for patients and families affected by the deadly disease. (Perrone, 4/25)
CQ Healthbeat:
Lawmakers Lobby For Rare Drug Approval, But Evidence Is Lacking
A Food and Drug Administration advisory committee on Monday heard arguments for the approval of a drug to treat Duchenne Muscular Dystrophy, a disease that affects young boys and currently has no approved treatment. The disease, which results in the loss of muscle use, affects one in 3500 boys. It is 100 percent fatal and most affected have a lifespan of fewer than 25 years. (Siddons, 4/25)
