The Jett Foundation Fighting Duchenne Muscular Dystrophy

We Must Get Up Again

January 26, 2016

imgres-1Like our children, when we fall, when we spontaneously collapse, when our legs are taken out from under us by something we never saw coming, we must get up again. We know it wont be easy, but we have to do it.

Last week’s storm and the subsequent postponing of the Advisory Committee Meeting for eteplirsen was a spontaneous collapse for our entire community. Like many of us, I never saw it coming, but just like Jett always did and always does, no matter the obstacle, I must get up again.

Our conversations with FDA have indicated that eteplirsen’s ADCOM will be rescheduled within 30 days.

We hope that you will, once again, take the time out of your life, out of your family’s life to be there.

I know our community will be ready. In fact, we will be more ready than we were before. We will take advantage of this delay to ensure that every allegation, each inexplicable falsehood included in the FDA briefing documents is scrutinized and demolished by our patients, our families and our DMD researchers and clinicians.

Our Open Public Hearing (OPH) will be impactful, intelligent, data driven, and balanced by passion. It will surprise the FDA. We will surprise the Division of Neurology reviewers when we strike back with our clinicians and experts and tell them they are unequivocally wrong in their analysis. It will, in fact, rattle them to their core, make them doubt their own analysis, and more importantly, make the advisory committee panel doubt FDA’s analysis.

We will clarify the data that they seem to have misunderstood. Our clinicians and experts will show and explain what the data really says and clear up the misinterpretations in the analysis.  We will explain the truth of the data and make the advisory panel understand that this is a safe and effective drug that our community desperately needs. As experts have testified, it would be a travesty if FDA does not approve this drug.

This will not be an OPH full of sad stories, rather this will be an OPH that shows the Duchenne community at it’s informed, educated, eyes wide-open best. We will bring our A-game, an A-game that will put the neurology division on its heels and leave the advisory committee panel with no choice but to endorse eteplirsen’s accelerated approval.

We will call our representatives in the House and the Senate, and urge them to sign onto letters asking FDA to use the tools congress provided in the FDA Safety and Innovation Act (FDASIA), as congress intended, to allow a safe and effective drug – even with a small clinical trial – to win accelerated approval.

We will call our local media outlets and tell our story, about our families and our children and how we will be converging upon the FDA in support of this drug.

And we will ask that FDA release the date of the new advisory committee meeting for eteplirsen. In order to begin the enormous logistical challenge all over again, in order to book the flights and the hotel rooms, to get just as many families to Silver Spring, Maryland as possible, we need to know the date and we need to know it now.

Without the new ADCOM date, our children’s fates are still up in the air. Without that date, we don’t know when the drug will be approved and we are still driving blind through the blizzard that left many of us stranded on Friday.

Help us persuade FDA to release the date of this new ADCOM as soon as possible. Help get this thing over the finish line. Once we have the date, please help by showing up again—by putting your lives on hold for a day to support what could be the first ever approved drug to treat Duchenne.

It is a lot to ask, I know, but together we can do this.

I will see you soon in Washington.


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