Dr Treagus after the FDA meeting said that we "have a pathway...

Dr Treagus after the FDA meeting said that we "have a pathway towards a New Drug Application for Rett syndrome"

So even though a normal pathways would be 2 different large scale - phase 3 trials I would hazard a guess and say that NEU secured just one phase 3 trial with higher dosage. One of the pre conditions of this would have been to do a small trial albeit amongst kids younger than 16 ( contrary to what others are saying). Once the tolerability is established, FDA will accept the submission for a single phase 3 trial and agree to provide approval for new drug.

They have yet to reach an agreement in efficacy measures with FDA and I would think that NEU would gain a bit of benefit as a first in line purely because of their organization of trial and collection of addendum data. NEU will propose MBA as one of the measure and FDA will invite comments. Who better to comment then same old Novartis and other trial stakeholders primarily Dr Liz who is also a stakeholder with NEU's trial.

All in all it will be a phenomenal year when the trial begins next year and expect to read a lot in the media and realization of the actual stock value.