So let's take your main concern of whether or not the FDA can approve "two small trials (N= 25 and N= 33/36) that only ran for short times (AVATAR was high dose for only 7 weeks). Can they approve a drug that has been tested over 7 weeks?"
According to the FDA guidelines for Drug Development for Rare Diseases:
"The purpose of this guidance is to assist sponsors of drug and biological products for the treatment or prevention of rare diseases in conducting more efficient and successful drug development programs. Although the statutory requirements for marketing approval for drugs to treat rare and common diseases are the same and issues discussed in this guidance are encountered in other drug development programs, these issues are frequently more difficult to address in the context of a rare disease for which there is often limited medical and scientific knowledge, natural history data, and drug development experience."
"This draft guidance, when finalized, will represent the current thinking of the Food and Drug Administration (FDA or Agency) on this topic."
I provided the link above so that you can read their full "thinking" on this subject.
Clearly, the FDA recognizes that drug development for rare diseases is a very gray area that requires an open mind.
Therefore, their decision is not going to be made on a set of preconceived metrics that have been established from a larger n.
The FDA also states that the statutory requirements of safety and efficacy will be applied...but with flexibility.
Quote: "To A Degree, Flexibility Is Part of FDA Regulation Paradigm (21 CFR 314 (APPLICATIONS FOR FDA APPROVAL TO MARKET A NEW DRUG)
Regulations allow for flexibility and judgment in applying the standards, and the FDA has a solid record of appropriately applying regulatory flexibility.
Flexibility
“While the statutory standards apply to all drugs… the many kinds of drugs… and wide range of uses for those drugs demand flexibility in applying the standards.
Thus FDA is required to exercise its scientific judgment to determine the kind and quantity of data and information an applicant is required to provide for a particular drug to meet the statutory standards.” (21 CFR 314.105 Approval of an Application)
How Much Evidence Is Enough to Obtain FDA Approval?
FDA may consider “data from one adequate and well-controlled clinical investigation and confirmatory evidence” to constitute substantial evidence."
Hopefully, I've established the fact that the FDA is going to be very flexible when Anavex submits data from the two trials (P2 N=25 and P3 N=36...which totals 61 patients).
Furthermore, the two trials mentioned above are NOT the only data to prove safety. I'm assuming that Anavex will also submit a safety record that goes back seven years. The safety record is the #1 concern for the FDA before anything else.
Side note: I just noticed that the Doc posted his reservations about the adult Rett approval too. What he doesn't address is the 7-year safety record that Anavex will submit vs. other rare disease drug approvals. Again, safety is the #1 concern for the FDA before any drug is approved. (We got this!)
Lastly, the FDA will be meeting with the International Rett Syndrome Foundation on March 11th to discuss new drug approvals. What are the chances that the Rett Foundation will be promoting (demanding!) Anavex's drug for approval?
"The International Rett Syndrome Foundation and the Rett Syndrome Research Trust are hosting an Externally-Led Patient-Focused Drug Development meeting and invite you to attend. By signing up you will receive the link to a public meeting with the US Food and Drug Administration and other stakeholders including drug developers, where you will be able to share the impact of Rett syndrome on your child and family, your perspective on possible treatments, and how symptom improvements would affect your and your child’s quality of life. The FDA is especially interested in hearing from parents, caregivers, and family members from the United States."
One other note: Does anyone know if Anavex will be applying for drug approval in Australia and England with the same data? If so, that too may have some sway with the FDA as well.
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