I agree with you on your expectation for approval; but I rely more on the logic that all 15 of the patients in the earlier low dose trial obtain multiple benefits in several of the domains of inquiry, and therefore, I reason that the odds are very good that another trial of similar size will have at least a good number of similar responders, especially on a higher dose that worked better in the animal models.
My thought is that if all AVATAR shows is that 50% of the participants are good responders, since their is no drug that helps approved yet, then even a 20% to 50% favorable response rate (within proper safety margins) will lead to drug approval.
The Rett's parent community is very interested in getting a drug that may help. And the politics are going to be helpful, IMO.
The only real questions I have is whether (1) AVXL can get the approval on just AVATAR for adult patients, and (2) given the voucher's potential to accelerate the FDA's consideration of AVXLs AD indication, whether AVXL will wait to request approval until after they have the EXELLENCE results to pursue the Voucher.
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