Why Eteplirsen is a real breakthrough- don't underestimate Pat and the parents
For the last 10 years, or so, the only treatment available for DMD boys have been things like steroids and over the counter pills like CoQ10.
They are mildly effective, if at all. More than anything, those treatments give the parents and boys a feeling that they are doing 'something' to try and combat the relentless decline of muscles and loss of ambulation.
Eteplirsen is the most significant breakthrough therapy ever developed for these families. It is based on new and effective science, is safe, and is showing clinical benefits.
Until now, parents have been living on the 'hope' for a treatment or cure. This drug is the first treatment that moves beyond 'hope.'
There will be a groundswell from parents lobbying the FDA to make this drug available ASAP. They will not be content waiting another 2 years for a phase III as they watch their boys decline.
I believe the odds of an accelerated approval are much higher than people believe. Pat has gotten sophisticated at navigating the FDA and she will be a real force in using the power of the parents and politicians to pressure the FDA for early approval.
If that happens, the boys and families will benefit greatly. And SRPT will be worth several billion dollars.
Absolutely. If anyone can make a good case for early approval, that is Dr. Mendell. None of the rest has as much power. It will be difficult to get this through the FDA unless the 48 week data is very strong.