Wheely on Tour in Hamburg

Andi Weiland | wheelmap.org (CC by)

Last month the Food and Drug Administration (FDA) approved the first treatment for Duchenne Muscular Dystrophy, a horrible disease that weakens muscles and eventually confines sufferers to wheelchairs. Patients and parents celebrated the FDA approval – at an FDA meeting this year one sufferer pleaded: “please don’t let me die early.”

But The Economist raise concerns about what the decision means for the future of drug discovery in America. The problem is that Serapta Therpeutics, the firm behind the drug, did not meet the usual standard for approval to market it. Worse still, staff at the FDA’s drug-evaluation division are apparently sceptical about the efficacy of Exondys 51, arguing that the clinical evidence available involved a flawed experiment on just 12 patients. While the FDA have asked Serapta to conduct further efficacy trials, the division director Janet Woodcock has overruled these concerns.

Read the full article from the Economist here