The Wait for An Approved Drug for Duchenne Continues


The wait continues.  Sarepta Therapeutics announced this morning that the FDA will not complete the review of the eteplirsen new drug application by the PDUFA date.  Considering the thousands of lives that could be affected by a decision on eteplirsen, we understand and appreciate the care the FDA is taking in its review of this innovative drug. We are hopeful that the delay is a positive sign.  Today, the Duchenne community needs to remember that a delay is not a no.

CureDuchenne provided early funding to Sarepta for the development of eteplirsen, and we have worked together to reach this point in the years since. While we can certainly wait for the FDA’s decision, we cannot afford to lose another generation of boys to Duchenne because there weren’t treatment options. It is urgent that we get the right combination of approved treatments for our boys.

Every delay is taking precious time away from our boys. Time is not on the side of those with Duchenne.  We must enjoy every moment with our children and every hug.  Watch a video about the Procko family who’s son Evan, 12, is on the Sarepta trial is confronting the disease and living in the moment.

We continue to remain hopeful that we will have an approved treatment for all those with Duchenne soon.


Related Posts

Share This Page

Make an Impact

You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.