The Jett Foundation Fighting Duchenne Muscular Dystrophy

Christine McSherry OPH Testimony: April 25, 2016

May 3, 2016

My name is Christine McSherry. You heard from me this morning when I presented the Patient Reported Outcomes Report. Now, I’d like to speak with you as a mother and an advocate.

My son Jett was diagnosed when he was five years old. Today he is 20. Jett took his last steps at the age of 13, despite being on 40 mg/kg of deflazacort, which is an extremely high dose. Last year, Jett was enrolled in the limited ambulation safety study for eteplirsen. In our view, he has stabilized and in some areas is regaining abilities he once lost.

But that is not what I am here to talk with you about today.

I want to be sure the panel understands what all of us are advocating for. We are asking that FDA approve a drug that has demonstrated consistent efficacy on multiple measures. We are asking that the agency utilize the flexibility and tools it has to approve a remarkably safe drug while pursuing further confirmatory trials. If, as a result of those trials, it becomes clear that eteplirsen isn’t working, we will stand behind the agency should it decide to remove it from the market. You see, we only want drugs that actually work.

We are not asking the FDA to “lower its standards” or grant the wishes of a desperate community. We are a community that is well informed, a community that funds and drives research, a community that writes draft guidance for drug development. We are here in large numbers because eteplirsen has met the safety and effectiveness standards for accelerated approval.
As a mother and an advocate I am surprised and disappointed by the briefing documents released in January and, even more so, by those released last week. What is clear from those documents is that the Division of Neurology is seeking to send a message to Sarepta, industry, and the rare disease community. And that message is: we will only accept a large randomized double-blind placebo controlled trial, no matter the severity, or the rarity of the disease.

We were very encouraged when FDA issued the DMD Draft Guidance which included historically controlled data as a potential pathway for approval. Now, to see FDA distancing itself so vehemently from that guidance is extremely disheartening. If FDA really wanted a large, placebo-controlled trial, why did the Neurology Division guide the company to start a single-arm study in the four to six year olds who would age into that study? There is virtually no one left who is drug naïve to enter into such a trial. We expect FDA to provide clear, viable regulatory pathways toward approval. The goal posts cannot be changed.

Twenty-five years ago, the FDA utilized accelerated approval to save a generation of young men dying from HIV/AIDS. Today, the agency can save another generation of young men dying from Duchenne. It is time for the Neurology Division to join the Oncology and Anti-Viral Divisions, among others, follow FDASIA, and to say yes to eteplirsen.

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