What We Said to the FDA - Part 2

Posted by: Christine McSherry on June 17, 2013

Why we are here…

We are here to urge you to approve eteplirsen for those amenable to skipping exon 51, those with Duchenne. We are here as parents, but we also represent the Duchenne community – they have asked us to come here and they have supported our trip here. They want you to know that you have the community support behind you for this decision. We have also brought with us 180,000 electronic signatures. People from all over have signed this petition, urging you to approve eteplirsen. Please read the comments, many come from those who have lost someone to Duchenne, their stories are heartbreaking and deserving of your eyes.

In August 2011, 12 boys were lucky enough to get into the eteplirsen trial and no one knew what to expect – but now we realize, their lives have been changed forever. Fast forward to June 2013 – 2 years later, and I want you to think about that timeline – 2 years later, we know that the drug is working. Every single day, these parents, are seeing little miracles, miracles that we would have never expected to see after more than 2 years of living with Duchenne. We know the natural history of Duchenne, we know it well; we have several published papers on the natural history – today I will be referring to the data published in the 24 month longitudinal data by Mazzone. This paper describes the decline, and the ability to predict a decline in 113 patients. The trial was designed specifically to help identify inclusion criteria for ambulant patients in order to minimize risk of patients who may lose ambulation within the time of the trial.

I have listened to the company presentations, and I know that you have the data set – and it would be easy for you to point to something and tell me that I don’t know what you have in that data, but I am here to tell you that you are not the experts on Duchenne. These parents and myself live with this disorder every single day and have been for years, for me – 12 years. Some of us have more than one child with Duchenne. WE are the experts on the progression and timeline of this disorder. Our Duchenne community, as you know, are full of very savvy parents. We know what Duchenne is suppose to look like – and we know when we are seeing the the curve begin to change. We are here today to tell you that you have everything you need to approve this drug, so all the others diagnosed with Duchenne, whose caregivers are feeling helpless right now, whose lives are at stake – have a fighting chance.

While you are going to hear stories today about boys in the trial, those on eteplirsen, I am here to remind you that there are many who were not lucky enough to get into this trial…and that this trial, if granted accelerated approval, will allow industry to address others in the community. Those are the young men and women that I represent…those who have do not have a chance, those who will lose the ability to walk, feed themselves, and breathe on their own. They will succumb to an untimely death – most notably within the next 2 years. The same amount of time to produce the results you are about to see – this population can NOT wait another 2 years. If forced to go the more traditional route for approval, that will exceed 2 years. You have all the information you need, you have the support from the community and public you need.

You will see children being children in the next hour. But you must understand, this is not normal, not in Duchenne. Kids NEVER get better in Duchenne. Once they start on their decline, their is no stabilization, there are no outliers who can suddenly climb stairs, this does NOT happen. The drug is working, Jett needs it now and so do all those amenable.

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