All about the headline: FDA asks for more data to finalize AA
It's all about the headline:
1) FDA requests additional data to fill the remaining gaps to expedite Accelerated Approval (AA)
2) FDA requests additional data, prolonging the process and delaying AA because data was insufficient / incomplete.
They are light-years apart.
If #1 then absolutely supportive of SRPT.
If #2 then absolutely deterimental for SRPT.
People don't know which one it is yet. They fear #2 and hope for #1.
The FDA must appear to have exhaustively looked at all data points. The 72 week data is important to evaluate. AA is a big step and the FDA wants to make sure they covered all the bases. The market is reacting as if it's all about #2 above. While a bit of a delay is inevitable, AA will be the eventual outcome.
Agreed that 72-week data is important, and that AA is a big step & FDA wants all bases covered.
The market is definitely reacting as if it is #2.
I do not agree that a delay is inevitable. We simply do not know. It very well could be the case where the FDA told them:
"Gee, we could approve by December 2014, but if you can give us surrogate endpoint data in dystrophin levels, then we can approve by December 2013."
Since there was no real schedule on AA to begin with, I don't agree that there is any delay. This is merely the announcement about what is needed to grant AA.
The FDA spoke to McNary and others beforehand and told them what they were going to do. The parents still believe the meeting went excellent and they are very optimistic. I am sure the FDA said something that gave them the belief that approval was likely.