Anavex Life Sciences Corp (AVXL)

[Anshu2]

I choose my words carefully. See below what I said. There is no legal (as in, there is no law by Congress) and neither is there any FORMAL guidance (meaning, there is no written guidance document from FDA). Feel free to correct me if you find either, based on your DD. Instructions to specific companies is neither.

Specifically, when BMRN filed for AA of its Prosensa's DMD drug in 2016 --- it was asked exactly that question during a CC, i.e, how do you expect an AA when you don't have any confirmatory trials started? The CEO/CMO of BMRN answered after some pause -- that FDA doesn't have any such requirement. BMRN's drug was finally rejected but for clinical efficacy and safety reasons (though, yes, lack of confirmatory studies may have played a role too).

Part of the reason for lack of such requirement is that only the FDA review can truly determine the right design for such a confirmatory trial.

There is no requirement (legal or even formal guidance). It’s just an implicit preference.