The Jett Foundation Fighting Duchenne Muscular Dystrophy

Shock and Awe

January 20, 2016

When I started reading through the FDA Briefing Documents posted in advance of this week’s eteplirsen Advisory Committee meeting in Washington, I was surprised. Was the FDA looking at the same trial results Sarepta has released publicly? Were they really dismissing data that shows the drug has slowed the progression of Duchenne in the boys enrolled in the trial as demonstrated in the 6-minute walk test? Were they really questioning the dystrophin data because they can’t agree on how to measure?

Then, over the weekend, I moved from surprise to shock. The more I reviewed the documents, the angrier I got. I began to hear from researchers, clinicians and scientists… people much smarter than me… who were questioning the FDA’s methodology, science, and expertise in writing the Briefing Document. They don’t understand where some of their questions and conclusions came from.

I heard from families whose boys are enrolled in the original trial who think the FDA must be talking about a different trial than the one they’ve experienced. They are walking beyond the 6mwt, they are walking through malls, the grocery store, the airport, and all day in their schools, walking longer than they could before. They can see other changes in the boys, and while the the 6-minute walk test has shown overall efficacy and benefit, it doesn’t seem to pick up some of the more meaningful things that these boys are experiencing, those that parents, teachers, physical therapists and others are seeing everyday.

One can only assume that some in the FDA – and the Neurology Division specifically – are afraid. They must be worried that, if this drug is approved, the Neurology Division will have to change their ways, they will have to follow congress’ mandate in FDASIA and give accelerated approval to safe seemingly efficacious drugs that treat rare disease on the basis of very small trials followed-up by confirmatory studies to ensure the safety and efficacy of the treatments. They must be concerned that regulatory science is moving beyond the large, placebo-based clinical trials they’ve become reliant on to a new way of evaluating treatments, using methods they are unfamiliar with.

That seems to be the only explanation for the reverse-engineering we see in the Briefing Documents.

And it is shocking.

But since Friday, my shock has turned to awe.

I shouldn’t be surprised that the Duchenne community – the families coming to Washington this week for the Advisory Committee and those fighting just as hard from home – is not deterred. I heard from multiple families, multiple clinicians and researchers, and multiple members of the public who experienced the same feelings I did when reading the Briefing Documents… and every one said that these documents only make us more determined to fight.

We believe in eteplirsen. We are not fooling ourselves. We are not working for the company. But we understand the data. The clinicians and researchers and scientific advisors that we rely on in our community understand the data. We do not expect special treatment because our boys have a life limiting disease, we do not want the FDA to approve this drug based off sympathy, but we do expect the FDA to interpret the data correctly and use the tools they have to provide access to a potential treatment. We are supported in our data interpretation by dozens of researchers and clinicians. And what we see is clear: eteplirsen works.

Once again, the community is fighting back. You are contacting your U.S. Representative and urging him/her to sign-on to the Fitzpatrick-Keating-King letter to FDA; you are contacting local media to tell your story using the Race-to-Yes Media Kit; you are traveling to Washington to testify to anyone who will listen. You are pushing the petition.

I continue to be in awe of your commitment, your devotion, your willingness to do what it takes to save our boys.

Keep fighting.

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