Press Release: Race to Yes Optimistic About Path Forward for Duchenne Children

Urges FDA to Move Quickly with Final Approval of Eteplirsen

WASHINGTON, DC, April 21, 2014 – The Race to Yes campaign today lauded drug maker Sarepta on its plans to move forward immediately to seek FDA approval of the first drug to successfully treat Duchenne muscular dystrophy, the world’s leading genetic killer of children.

“We have been waiting for this for a long time,” said Tracy Seckler, whose son, Charley, has Duchenne. “This is good news that gives our children a fighting chance. But it’s only one lap in a long race and we have a long way to go to get to the finish line. Too much time has already been lost, and in that time, boys have continued to get sicker and die.

“We know eteplirsen is safe and works. The children who have been treated for more than two years are clear evidence that the drug stabilizes lung function and walking ability for the group amenable to this treatment.”

After initial encouragement from the FDA, Sarepta was ready to go to an accelerated approval process when the FDA unexpectedly said it needed more time to consider next steps. On April 8, The Race to Yes wrote FDA Commissioner Margaret Hamburg and urged her to quickly restart the eteplirsen approval process that has been facing agency delays for nearly 18 months.

Sarepta today cited recent consultations with the FDA as the basis for announcing it was moving forward with the new testing and the goal of obtaining quick approval.

The letter to Hamburg came after 106,000 persons signed a White House petition last month asking it to get involved with the FDA. The campaign has met with FDA officials, and in February hosted a congressional briefing where leading scientists from around the world said the drug has shown it works, and has no side effects.

Jenn McNary’s two sons, Max and Austin, both have Duchenne. Max is doing well as part of the ongoing eteplirsen study while Austin is not getting the drug. His health is declining. She promised the campaign would continue its advocacy for boys with Duchenne.

“We will be carefully following this study and the process leading to what should be an accelerated approval by the FDA,” McNary said. “Every step forward that takes place here means additional progress for other potential drugs that will stop and eventually cure Duchenne.”

“We are in a race to save the lives of boys around the world, who have not had much hope for the future. The FDA must be more sensitive to those patients’ needs, and held accountable for delays in the process,” McNary said.