It's now widely believed that as each day goes on and we don't get a PR from Sarepta, it's likely the Dystrophin data is at worse "mixed" and at best "great". Of course, ones definition of great varies depending on whether you're a bull or a bear. For me, "great" is a stat sig difference from baseline approximating 1% of normal though the 1% is not as important as the stat sig increase.
So we can reasonably say that given it's now been nearly 5 weeks since SRPT got the request for the D data, they obviously have it and we haven't gotten a PR. But what we don't know is what the FDA thinks of the "mixed" or "great" data. A bear would say, unless it's 3% of normal or greater, they will CRL.
Additionally, you have some bulls saying that the FDA will not approve till ESSENCE is either enrolling or dosing. In this case, ESSENCE should begin enrolling shortly (expected in July as of now but that is always subject to change) but will not dose for 2 months after it starts screening, according to a Boston Don article.
So this is what I think. Given it's very likely Sarepta has the D data and then, by inference, likely FDA has the data by now they have a variety of choices they can make.
D data too low and/or not stat sig. Nothing Sarepta can show them can make up for this. FDA will CRL. They have no reason to wait weeks or months to do this. It will come "promptly".
D data acceptable. Will grant #AA. This could take time just to finalize the label but also should come "promptly" unless they waiting for ESSENCE to enroll OR dose.
My point is, as we get into mid/late July, the odds of a CRL become increasingly small. In my opinion, the only reason the FDA sits on the decision post examining the D data is to approve pending ESSENCE enrollment or dosing. There is virtually no reason to sit on the decision if the D data is not acceptable for approval as there is nothing else Sarepta can offer the FDA at this time to change their opinion. Politically, the FDA wants this dec
you're correct but even if it starts there is guarantee if or when it will fully enroll and further, when a company is granted AA they have a hard deadline to finish the confirmatory trial and submit results, usually 4-7 years depending on the circumstance.