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Help is on the way (maybe)

Letter to the Editor

The Food and Drug Administration’s decision to give an experimental drug (eteplirsen) for Duchenne Muscular Dystrophy another chance is giving parents hope that they might be able to save their children from the worst effects of the fatal disease.

The FDA previously had stalled approval of eteplirsen partly over concerns about its safety and effectiveness. The drug has been tested in only 12 boys, but it appears to be working with no side effects.

FDA spokeswoman Sandy Walsh said : “The FDA is fully committed to make safe and effective drugs available for patients with Duchenne muscular dystrophy as soon as possible, and is actively engaged with all drug companies developing new drugs for Duchenne muscular dystrophy.”

On the surface this sounds encouraging, but in reality it is likely more spin.

Cambridge-based Sarepta Therapeutics officially will file a new drug application with the FDA later this year. If the FDA gives the application a priority review and ultimately approves it, eteplirsen could be commercially available in the second half of 2015, which probably means sometime in 2016 (maybe).

It would be nice if our Bay State Senators, Congressmen, and Governor put pressure on the FDA to cut through the red tape, and make this happen now. The families of those suffering deserve at least that much.

 

Mark Veilleux

Danvers, MA





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