In the latest twist surrounding Sarepta Therapeutics and its rare disease drug, the US Food and Drug Administration has asked the company to provide additional data to review, a move some see as a sign that the agency is looking for ways to approve the treatment. Shares in Sarepta, which disclosed the data request Monday night, jumped more than 25 percent Tuesday.
The request comes amid ongoing uncertainty over the fate of a medicine being developed to combat Duchenne muscular dystrophy, which destroys muscle fibers and eventually confines boys to wheelchairs before sending them to an early death. There are no treatments currently available, and the FDA recently rejected two other medicines, sparking a heated clash over the fate of the Sarepta drug.
As pressure mounts from parents and lawmakers, the agency has been sending mixed signals about its intentions. In recent months, agency medical reviewers repeatedly questioned the veracity of a 12-person clinical study and underlying trial data. But at the same time, a high-ranking FDA official recently — and very publicly — noted the dangers in failing to approve drugs for “devastating diseases.” And last month, the agency delayed making a decision.
About the Author Reprints
Never though I would see that day when an uninformed public opinion could affect a black-and-white scientific question. Obama’s America…?