Replies to post #318216 on Anavex Life Sciences Corp (AVXL)

[Investor2014]

First there is a process at the FDA/TGA/EMA to schedule a review, which may have been already initiated by Anavex or more likely is about to be.

Then Anavex will meet with the FDA for their guidance e.g. is the data compelling enough to apply for an Expedited Programs approval or is the discussion on the endpoints of a P3 trial and its design.

If the former then simply because first the data must be organised and submitted to the FDA, which is something that in many instances amounts to even thousands pages, this can take some time to prepare. Maybe the Anavex ex-FDA folks have already done a good amount of that work, but they will need to understand the meeting guidance before completing the submission.

Expedited Programs for Serious Conditions – Drugs and Biologics

I would say below applies equally to Rett and PDD/PD.

2. Where There Is Available Therapy
When available therapy exists for a condition, a new treatment generally would be considered to
address an unmet medical need if the treatment:

? Has an effect on a serious outcome of the condition that is not known to be influenced by
available therapy (e.g., progressive disability or disease progression when the available
therapy has shown an effect on symptoms, but has not shown an effect on progressive
disability or disease progression)

? Has an improved effect on a serious outcome(s) of the condition compared with available
therapy (e.g., superiority of the new drug to available therapy when either used alone or
in combination with available therapy (i.e., as demonstrated in an add-on study))

? Has an effect on a serious outcome of the condition in patients who are unable to tolerate
or failed to respond to available therapy

? Can be used effectively with other critical agents that cannot be combined with available
therapy

? Provides efficacy comparable to those of available therapy, while (1) avoiding serious
toxicity that occurs with available therapy, (2) avoiding less serious toxicity that is
common and causes discontinuation of treatment of a serious condition, or (3) reducing
the potential for harmful drug interactions

? Provides safety and efficacy comparable to those of available therapy but has a
documented benefit, such as improved compliance, that is expected to lead to an
improvement in serious outcomes

? Addresses an emerging or anticipated public health need, such as a drug shortage

As a side note regarding the much debated Biogen Aduhelm Accelerated Approval, below is worth keeping in mind for blarcamesine and AD:

As discussed in sections VII and III.B., FDA recognizes, as a general matter, that it is preferable to have more than one treatment approved under the accelerated approval provisions because of the possibility that clinical benefit may not be verified in postapproval confirmatory trials. FDA will therefore consider products as addressing an unmet medical need if the only approved treatments were granted accelerated approval based on a surrogate endpoint or an intermediate clinical endpoint and clinical benefit has not been verified by postapproval studies.

With Fast Track, Breakthrough Therapy and Priority Review, but not Accelerated Approval the FDA timeline for response is within 60 calendar days of receipt of the request. Only the Priority Review designation if granted reduces the review of the marketing application from 10 months to 6 months. Note that regardless of designation the FDA must process the marketing application, which takes 6 - 10 months. The reviews, guidance and meetings prior to that is expedited compared instances with no special designation.

It is all in the linked doc and needs to be read carefully.

So hence at least 6+ months to a year+.

Just about a year would be amazing for approval, but we must be prepared that P3 trials are a distinct possibility. Of course a P3 trial also has the preparation for submitting data and then 6 - 10 months worth of FDA wranglings.