Where’s the Drug, FDA? The agency keeps delaying a therapy for muscular dystrophy.

http://www.wsj.com/articles/wheres-the-drug-fda-1467413266

The Food and Drug Administration is sitting on a therapy for Duchenne muscular dystrophy, and the agency may have days to waste but the boys don’t. Bureaucratic malpractice on a safe and effective treatment is corroding the agency’s scientific credibility and the public’s trust.

FDA in May delayed a decision on eteplirsen by Boston-based Sarepta Therapeutics. There is no treatment for Duchenne, a fatal disease that claims a boy’s ability to walk before organ failure in his 20s. Eteplirsen jumps over genetic code to produce a missing protein known as dystrophin.

Eight of 10 boys who seemed headed for wheelchairs still walk after four years of treatment; only one of 11 in a control group could walk. FDA reviewers say the drug doesn’t produce “enough” dystrophin or maybe the kids had motivated moms. Yes, the public pays for that analysis.

The agency last month asked Sarepta for dystrophin data from an ongoing trial. The results are likely to show that the treatment is delivering on its promise to pump out the protein, as dozens of experts, clinicians and scientists tried to tell the agency at an April meeting. A readout consistent with earlier findings would give Janet Woodcock, the drug evaluation center chief who can overrule her technical staff, ample reason to say yes.

An approval would have the added advantage of obeying the law. Legislation from 2012 allows FDA to sign off on a first-in-class drug that is “reasonably likely” to predict a clinical benefit based on small or innovative trials. FDA can pull the treatment if later studies fail. This process is called “accelerated approval,” though that is a dark joke to boys who lost walking or gripping abilities in the year since Sarepta filed an application.

Sarepta will soon start a required confirmation trial that deploys the same technology to treat boys with a different strain of Duchenne. Last week the company said that the investigation would last two years instead of one. The trial will be placebo-controlled, in which some patients receive saline. CONTINUE AT SITE

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