the message seems clearer to me after rereading the pr with a one week break
when your expectations are grounded in reality, I am not sure how we could have reasonably expected any more than what this pr provided
the odds of FDA providing AA solely on to-date data set appears remote (you can criticize this all you want but appears to be the message to me)
having said that, the FDA has done just what woodcock once alluded to, which is the concept of "show us"
and the FDA appears to have told Srpt just how it can "show" the FDA, in a relatively short period of time, why its to date data is in fact worthy ... in essence, do it again
the other clear message I get is that with regards to take and measure dystrophin, the FDA and Srpt will agree on exactly how to do it ... IMO, this is the MOST important message in the pr
and in connection therewith, the 4th biopsy on the P2 boys seems megla important ... seems like the FDA told Srpt that if you do the 4th biop with our agreed upon methods, and it matches the 3 prior biops you did that we still question as to validity, then we are likely to accept the first 3 too
lastly, my take is that if the 4th biop on P2 boys is done and shows acceptable dystrophin restoration ala FDA accepted methods, and also the P2 144 week data continues to show the continued stability trend, that the AA filing is a lock
whether the AA actually gets approved in mid 2015 will additionally be dependent upon the new trial data that will become mostly available after the AA is submitted
make no mistake, it appears the AA submission will tie to 4th biop and 144 week data
IMO, this will be very difficult to bet against if those two data points are bullish
I generally agree with you, except:
1) AA filing will only depend on results of the 4th biop from the ongoing p2 study since this is the only new data requested by FDA for AA filing. There is an unlikely chance that the 144 week walk data is deteriorated dramatically which might prevent the AA filing. But both FDA and SRPT do not expect this even remotely.
2) A stability of 144 week and 168 week data will gain FDA approval.
3) The new studies will not be completed before the approval. FDA did not require/expect them for the approval.
Agree totally when i read it I thought 144 + 4th biopsy will get it done. Parents should have jumped on it the first time because they were bargaining from a weak position thanks to Hoffman. also agree on 168 deal will be done by then.