With the news from Saperta, I was wondering how this effects Prosensa/GSK? With all the trial information they have, why have they not tried for accelerated approval? Anyone have any thoughts or opinions on this, seems to me the first one to get 51 approved wins.
Their biopsy data from their original 12 boys was not double-blind, so they have not proven the surrogate marker in a blinded way as Sarepta has. They have not even released 24 week biopsy data - only the original 12 weeks from their original 12 boys, so there are questions as to why that hasn't happened.
Sarepta's study design for their phase II was much better done - designed to prove much more, much more quickly. They have a rich data set that Prosensa, at the time, did not design their study for. Plus, there is the toxicity issue...
In my book, the race is in the home stretch and Sarepta/AVI has solid lead.
But the finish line is approval, so now it's up to FDA decision subsequent to a Sarepta petition. If Sarepta wins race to first approval, one has to wonder if GSK will continue pouring money into Prosensa, especially if Sarepta PMO continues to show none of the safety issues seen with Prosensa PPMO.
Phase I/II Study of Prosensa 44 skip drug has changed the estimated primary completion date from August 2012 to March 2013. Is this change due to late recruiting or was there a problem with the drug? Does anyone have more info?