The Jett Foundation Fighting Duchenne Muscular Dystrophy

Jett Foundation’s Statement on Sarepta Briefing Documents

January 16, 2016

Jett Foundation Statement in Response to FDA Briefing Documents

Christine McSherry, Executive Director, Jett Foundation.

proxy

Eteplirsen is a safe drug that in over 4+ years of testing has shown no adverse events, no danger to boys being treated. Congress has given FDA the tools to say “yes” to drugs developed to treat rare disease, especially where there is an unmet medical need, once they are shown to be safe.  We agree that further testing is necessary… but let’s start treating boys today.

We are deeply troubled by the FDA analysis, which appears to be sloppy and not grounded in science. It appears to us that the FDA analysts began with a conclusion, then looked for ways to justify their point of view.

For example, it is absurd to suggest that the boys in the original trial are all “outliers.” There is no doubt that eteplirsen has dramatically slowed the progression of the disease in these boys. Ask their mothers… ask their physical therapists… ask their doctors.

Further, we hope the FDA does not reject this drug because a group of researchers can’t agree on how and when to measure dystrophin, the protein largely missing in Duchenne boys.  We cannot afford to wait for that.”

Congress has directed the FDA to consider the views of patients in making its decisions. At next week’s Advisory Committee meeting, you will see a completely unified Duchenne community.  We are not grasping at straws.  We are not naive.  We know that eteplirsen is not perfect. But ask yourself:  Would you rather take the risk that a completely safe drug may not work as well as we had hoped, or wait and watch your child lose muscle every day as he progresses to a certain end.

To us, there is only benefit.

The risk is that FDA will ignore the experience of patients, families and physicians… the FDA should not be working to find excuses to say no, they should be looking for reasons to say yes.

We will see you in Washington next week.

For further information, please contact Christine McSherry at christine@jettfoundation.org.

 

 

 

Show Buttons
Hide Buttons