Washington, DC, July 30, 2014 – In response to the 106,782 signatures collected through TheRacetoYes.Org petition urging the “FDA to Say Yes to Accelerated Approval for safe, effective therapies for children with Duchenne” muscular dystrophy, the White House issued a statement yesterday by Janet Woodcock, Director, FDA Center for Drug Evaluation and Research. The Race to Yes organizers expressed appreciation to the White House and FDA for the acknowledgement of the urgent need for these therapies, and remained cautiously hopeful that their children will receive the promising therapies before it is too late.
On behalf of the White House, Director Woodcock stated, “We are willing to explore the use of all potential pathways for the approval of drugs for Duchenne muscular dystrophy, including accelerated approval, as appropriate.” She went on to say that the FDA is “actively engaged with a number of drug companies focused on developing new drugs for Duchenne muscular dystrophy, including Sarepta Therapeutics, the company developing eteplirsen, an investigational new drug for Duchenne muscular dystrophy.” See the full response here.
The Race to Yes organizer Marissa Penrod thanked the FDA and Sarepta Therapeutics for acknowledging the urgent need to pursue accelerated approval, and stated, “There is a limited window where our children still have enough muscle for these therapies to help. Every day our children lose that muscle and the window of opportunity for these therapies to help this generation of children with Duchenne closes. For our children, time equals muscle.”
Fellow Race to Yes organizer Tracy Seckler expressed the ongoing concerns of the thousands of families facing Duchenne, “Our children are in a race for their lives, where every day counts. We call upon the drug companies and the FDA to put their money where their mouths are. Our children will be left behind until the regulators and the drug companies act to get these and future therapies across the finish line.”