Testimony from MDA at FDA Advisory Committee Hearing for DMD Drug Eteplirsen

Today, MDA’s Executive Vice President & Chief Medical and Scientific Officer Dr. Valerie Cwik spoke during the FDA’s Advisory Committee hearing to review eteplirsen, under development by Sarepta Therapeutics for the treatment of some forms of Duchenne muscular dystrophy (DMD). 

Good afternoon.

I am Dr. Valerie Cwik, and I am pleased to be here today on behalf of the Muscular Dystrophy Association and the thousands of Duchenne families we represent. At the outset, I’d like to share MDA’s optimism that there will soon be treatment options to change the course of Duchenne muscular dystrophy—and that eteplirsen could be the first of what we hope will be many new treatments for MDA families.

As Chief Medical and Scientific Officer of MDA, and as a neurologist and former MDA Care Center director, I have worked with many families living with Duchenne. I am reminded that my 25 years of medical specialty in the neuromuscular diseases is about the same amount of time that the average person with Duchenne can expect to survive—and this is a reality that is unacceptable to MDA.

MDA has led the search for treatments and cures for Duchenne for more than half a century, and will continue to do so until there is a cure. Twenty years ago we funded foundational exon skipping research and follow on studies that led to the development of eteplirsen.  And while not a cure, the data indicates that the drug could slow disease progression.

Many leaders in the Duchenne research and clinical communities have voiced enthusiastic support for eteplirsen—and as a science- and evidence-based organization, their support carries great weight with us.

All of us at MDA—as well as our sister organizations, scientific community, families and supporters—have been working tirelessly to see a time like the present, a time when therapies could be more than just a hope for the future.  We are all here for those living with Duchenne, and the people who love them.

It is time that treatment options shift from being a goal to being reality.

While the decision of whether to approve a drug is ultimately a regulatory science determination for the FDA, given the support of Duchenne scientific and clinical leaders, the support of the families we serve, the urgent and unmet medical need, and the strong safety data, we urge you to strongly consider all of the tools available to the FDA to allow the earliest access to eteplirsen.

Thank you.