By next Thursday, the FDA will make a decision on approval for eteplirsen.
Whether the FDA’s answer on eteplirsen is “YES,”or “YES, BUT…,” or “NO,” the race is far from over.
If eteplirsen is approved, we will celebrate this historic milestone of the first ever therapy for DMD. For the 87% who cannot benefit from skipping exon 51, we WILL make sure lessons learned through this process will expedite therapies for the even rarer exons. Likewise, because exon-skipping is only one of the many treatments in the pipeline, we need the lessons learned through this process applied to all potential therapies for DMD.
If the answer on this therapy is “YES, BUT….” or the unthinkable “NO”, we will have even more work to do and even more urgency to do it.
It has taken an unprecedented effort from our community to get this far. 100,000+ signatures on a White House petition. Letters signed by one-fourth of the U.S. House of Representatives, and another letter signed by one-fourth of the U.S. Senate. 1,000 patients, families, caregivers, scientists, and clinicians in attendance at the FDA Advisory Committee meeting.
And we are just getting started.
May 26th is an important day.
But it’s not the end.
It’s another beginning, and we WILL need your help.
We will ask you to STAY THE COURSE.
We will ask you to REACH OUT TO YOUR ELECTED LEADERS.
We will ask you to ENGAGE ON SOCIAL MEDIA.
We will ask you to CONTINUE CHANGING THE WAY THE FDA LOOKS AT RARE DISEASES.
TOGETHER we will do whatever it takes to get safe, effective treatments across the finish line for 100% of DMD patients.
Stay tuned for more in the coming days.