9 August 2016 - Why won’t the FDA make a decision on a muscular dystrophy drug?

Government ineptitude is usually a punch line, but the dark reality is that bureaucratic malfeasance can carry a body count. The Food and Drug Administration is months late deciding whether to approve a drug for muscular dystrophy, and by now there’s only one explanation: Some of the agency’s staff have exploited FDA processes to evade accountability for an unprofessional review.

Ten of 12 boys with Duchenne muscular dystrophy walk after four years of treatment on eteplirsen, which produces the protein dystrophin. FDA has pushed off approval for the first-in-class treatment, asking for more time or even months-long delays blamed on snow. In June FDA asked the drug’s sponsor, Sarepta Therapeutics, for readouts from a continuing trial, which the company said it would provide shortly. Since then, radio silence from the FDA.

Read Wall Street Journal article