Research Update: Eteplirsen Approved!

Dear Friends,


Moments ago, history was made: The FDA approved Eteplirsen as the first drug to treat Duchenne muscular dystrophy. You can read the full press release from the FDA here. The head of the FDA’s Center for Drug Evaluation and Research herself is quoted directly:

“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research.

Eteplirsen – now known as Exondys 51 – was granted accelerated approval, which is part of a pathway to get patients with rare, life-threatening diseases access to safe and effective drugs on an accelerated basis. Full approval will be considered upon review of data from a confirmatory clinical trial.

This approval is a tremendous moment for the DMD community.  But this drug is not a cure, and it works only for the 13% of boys who have the specific mutation it targets. We have much work yet to do to expand the scope of this success to target ALL mutations and benefit ALL children effected.  But this hurdle is a monumental step.  We will get to what’s next in the days and weeks to come.  But today, take a moment and reflect on what we have all accomplished together.  And dare to be excited.

The success of our efforts here is testimony to how important our role in this process is – to how important YOUR role is.  Make no mistake – this accomplishment was possible because of the extraordinary efforts of our partners at Charley’s Fund.  The Race to Yes movement they co-founded played a critical role to ensure that the FDA followed the science and used flexibility appropriate for a rare, fatal disease with no other treatments.  They could not have accomplished this without us and we could not have accomplished it without you.  This is your success!

Thank you from all of us at Matt’s Promise for helping us to achieve this historic and unprecedented milestone.  It may well represent the most critical step yet in our fight to defeat Duchenne Muscular Dystrophy.

And congratulations to the entire DMD community on this tremendous first step toward treatments for all kids with Duchenne!

Randy Reiff

ResearchJ.T. Arbogast