MDA Appeals to FDA in Support of DMD Drug Eteplirsen

Dear MDA family and friends,

As part of MDA’s commitment to provide our DMD community with up-to-date research and advocacy news, we want to let you know that MDA today made another appeal to the U.S. Food and Drug Administration in support of eteplirsen, which, if approved, could help slow progression of the disease for some kids and adults living with Duchenne muscular dystrophy.

In a letter sent on May 10 to Janet Woodcock, M.D., Director of the Center for Drug Evaluation and Research at the FDA, MDA emphasized it believes the conditions under which the FDA can grant accelerated approval for eteplirsen appear to have been met.

The letter follows MDA’s written submissions to the FDA and oral testimony at the FDA advisory committee meeting in April in which it urged the FDA to apply the maximum possible regulatory flexibility in its review of the drug. Following testimony by MDA, sister organizations, clinical and scientific experts, and many individuals and families living with DMD who attested to the drug’s safety and effectiveness, in a close 7-6 vote, the committee recommended not granting accelerated approval for the drug.

In its letter, MDA affirmed the FDA’s regulatory oversight, but acknowledged that it may be appropriate for the FDA to go against the recommendation of the advisory committee in this circumstance. The FDA is expected to determine whether eteplirsen will be approved by May 26.

MDA’s active engagement in this process reflects our determination to advance safe and effective treatments on behalf of MDA families. The time is upon us, and we want to be active and overt in stating our supportive position on behalf of MDA families, based on the strong body of evidence.

Eteplirsen is under development for DMD by Sarepta Therapeutics, Inc., and is based on an exon skipping research technique first funded by MDA two decades ago. If approved, it would be the first drug on the market to treat the underlying cause of DMD and is expected to work for about 13 percent of DMD patients.

MDA has invested more than $200 million in DMD research to find treatments and cures, and more than 7,500 individuals with DMD have access to its nationwide network of Care Centers. We focus on DMD and dozens of related neuromuscular diseases in our portfolio as we work through our expansive research program to unlock breakthroughs that will accelerate treatments across diseases.

We know that for our families treatments can’t come quickly enough. That’s why all of us at MDA are working every day to find solutions now, and why we won’t stop until treatments are available for all the diseases in our program.


MDA Outreach