A Letter to Our Community About the Eteplirsen Advisory Committee Outcome and Next Steps

Dear MDA family and friends,

At yesterday’s all-day FDA advisory committee meeting for the promising drug eteplirsen, MDA stood shoulder to shoulder with our sister groups, research and clinical experts, and most importantly you, our families. We couldn’t have been more proud to raise our voice on behalf of the thousands of Duchenne muscular dystrophy families we represent and serve.  

During our testimony, we expressed MDA’s optimism that this promising drug could change the course of Duchenne muscular dystrophy and be the first of what we hope will be many new treatments for MDA families. It was a very humbling and proud moment to have our longest-standing and steadfast partner, the International Association of Fire Fighters, join us in pledging its support. (To read and watch our full testimony, see: Testimony from MDA at FDA Advisory Committee Hearing for DMD Drug Eteplirsen.)

Yesterday’s committee meeting was an important step in the regulatory review process. While the panel members shared their perspectives and cast votes that were met with great disappointment by many, we are not disheartened. MDA remains determined to continue to do everything we can to help our families live longer and grow stronger.

What’s next in the evaluation process? The FDA will consider the committee’s recommendations as it prepares for a final ruling, expected to come in the next month. MDA remains enthusiastic that the supportive data, the widespread support of DMD researchers and clinicians, the critical unmet medical need, and your words will resonate powerfully with the FDA as it considers whether to grant drug approval.

MDA has been there from day one when we funded the work that identified the gene that causes Duchenne and when we funded the original exon skipping research that led to the drug eteplirsen. We are here for you today, stronger together, as we fight for strength, independence and life. 


MDA Outreach