A federal advisory panel voted Monday that a drug from Sarepta Therapeutics was not effective for treating Duchenne muscular dystrophy, a rare and fatal muscle-wasting disease. About 13,000 children, mostly boys, are afflicted.
The vote came after a daylong session punctuated by emotional pleas from dozens of parents and their children, some of whom appeared in wheelchairs, to describe how the Sarepta drug, called eteplirsen, made a substantial difference in clinical trials. Their testimony was balanced by presentations from US Food and Drug Administration staff who took a dim view of the Sarepta trial data.
The panel vote, however, is not the last word. The agency must now determine by May 26 whether to follow the recommendation. […]
The fate of the Sarepta drug has been closely watched as a litmus test for an intensifying struggle between the FDA and patient groups that want the agency to take a more expansive view toward approving medicines for unmet medical needs. In this instance, patient advocates hope the FDA will use the accelerated approval process to endorse eteplirsen. This approach relies on a substitute outcome in a clinical trial to suggest a drug may have, but not does not guarantee, a benefit. […]
The only outright opposition to approval came from Laura Gottschalk, a senior fellow at the National Center for Health Research, a nonprofit think tank. “Unfortunately, the data do not meet a scientific standard of evidence of effectiveness,” she told the panel. “These boys and their families deserve better.”
To see original article, click here.
To view Dr. Laura Gottschalk’s testimony, click here.