It’s old news by now…..Sarepta’s exon-skipping drug, Eteplirsen, is safe and shows efficacy. If we sift through all the details, all the facts, and all the distractions, we’re always left with the same basic, essential information – Eteplirsen is an appropriate candidate for Accelerated Approval. Duchenne is severe, rare and has no therapy. Duchenne is the perfect example of why Congress passed FDASIA, and allowed the FDA to move drugs to patients quickly when there are no other options and the drug is “reasonably likely” to predict clinical benefit.
We are sitting on the runway, safety checks complete, all systems go. We need this plane to take off, so it will clear the path for many other treatments that will be on the runway soon. But we are still waiting for clearance from the FDA. We are waiting for the only appropriate response – “This plane has been cleared for takeoff.”
This is the part of The Race to Yes that feels painfully like a marathon. It’s the part that takes the most patience, focus and persistence. And it’s the part on which The Race to Yes team is most focused now. The FDA has had enough time. We need action.
As a team that is over 100,000 strong, we led our community to this point, and we are not stopping. Please stay the course. There is a plan and it is not random or reactionary. It is specific and on purpose. We are communicating in no uncertain terms to the FDA and FDA Commissioner Hamburg that we are low on patience and our children are low on time.
The White House gave us 30 days to gather 100,000 signatures. We are challenging the FDA, the Commissioner herself, to give us a decision in 30 days. And we are making sure that every boy that can benefit from eteplirsen gets it as soon as possible and that the runway is cleared and expedited for all the boys who can benefit from follow on therapies.
The FAA needs to make sure we are safe to fly, but there would be no reason for the FAA to exist if the planes never took off. The same goes for the FDA. We have been grounded for far too long, and we must never lose sight of the fact that these folks work for us and for our kids. This plane is long overdue to be cleared for takeoff.
Please encourage your families, friends and supporters to keep checking back to The Race to Yes website or Facebook page. Will continue to raise our voices and ask for your support in doing so.