Why is the FDA preventing access to a lifesaving drug?

Posted by: Christine McSherry on December 18, 2013

Duchenne is the most common lethal genetic disorder in children, one/3500 primarily boys are affected.  It is untreatable, incurable, 100% fatal, most affected die before their 20th birthday.

In August 2011, a clinical trial aimed at treating about 13% of the Duchenne population began. Forty-eight weeks later, 100% of the boys in the trial were producing dystrophin – the missing protein responsible for causing the disease.  With the most recent 96 week data, all participants had shown stabilization effects.  In August 2013, the company who makes the drug; Eteplirsen, Sarepta Therapeutics, was encouraged by the FDA to file a New Drug Application (NDA) which would allow access to this life saving access for children dying from Duchenne.

In early November, without any new data, without cause, the FDA reversed its previous guidance stating that a NDA application would be premature. This causes an undetermined time delay to access of the drug for thousands of children, causing untimely deaths.

All of the communications (documented in the attached ) with the FDA and the families have been positive, leading these families to believe that the agency would do everything possible to see that this drug received accelerated approval. Now, there is not even the hope that the company can file a new drug application.

Currently the community sits waiting for news. Parents of children with a fatal disease are facing a holiday under a cloud of confusion and ambiguity. It is not the science. This therapeutic agent is promising and has no side affects. The side affect from Duchenne is death. Why won’t the FDA allow access? We need media help and exposure. For more information contact Christine McSherry and Jenn McNary. See below for background stories and below for a timetable of events.

What is going on here?  Why did the FDA rescind its decision?  Is there more to the story?

Our children, my son – should NOT be serving the science – THE SCIENCE SHOULD BE SERVING MY SON.



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