At that point in time there was simply correspondence between sponsor and FDA about FDA's request for more data. Nothing material, related to WB data, revealed in those docs. It all comes down to whether or not there is statistically significant difference between baseline WB and 48 week. That's the bottom line.
This is really good news for Gavin. F D A hit men would say that this is not unusual in the hx of DMD. But with exon-51 boys? Probably not. More likely with Becker's DMD.
Baseline biopsies were taken just for this type of event, if approval of the NDA was delayed due to FDA requiring additional data. At least FDA's decision isn't being delayed until 2018.
Won't it all come down to how FDA interprets the WB data? Previously, they had issue with "enhanced" images, or was that with I.F.? I think J.W. wants to avoid the Type II error and needs confirmation for her ultimate ruling. Also, if there was a Differing of Professional Opinion within FDA, this will tip the scales one way or another. There has never been established minimum dystrophin levels to confer a clinical benefit, but significance above base line should be enough for the standard of "reasonably likely" to confer a clinical benefit, not "substantial evidence" of clinical benefit, as briefing docs demanded.
Did you carefully read Sarepta's P.R.? 'The Company plans to submit data from thirteen patient biopsy samples, at baseline and Week 48, to the FDA over the coming weeks to facilitate a prompt decision on the NDA by the Agency.' What about the "coming weeks" are you misinterpreting?
He's not the only one in the camp of a positive decision this week. Today, over 5,400 June 3 $30 call contracts have been sold/bought. Big bet on some positive news this week. The clock is counting down.