abew4me
I feel that our disagreement should be the focus of discussion so that we can clarify the strengths and weaknesses of the case for approval. There are some really talented professionals on this board that have the ability to discuss the issues in a way that the FDA may think about them. Much of that deep science stuff is beyond my expertise.
However, you seem to me to have an excellent handle on the subject matter. So I put it to you to make a case for your position if you can. As I said in my post, I can see a case for accelerated approval to get a drug into the hands of the waiting community. But, I think the key issue was raised by Duncan when he downgraded AVXL to a PT of $16.
Do we have a large enough size of clinical improvement, that is free of reasonable doubt and subjectivity issues, to support the FDA approving an NDA with just 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? I have similar questions with 12 Weeks in EXCELLENCE (is that long enough, do they need to show persistence of the benefit for approval?), and think they are going to need the OLE of 48 weeks data to help with long term safety and to further develop how the drug efficacy rolls out over longer times.
On the other hand, since this is a rare indication, with difficult to get participants in long Clinical trials, should the FDA grant approval on short, thin studies because the effect size is good and the drug should be allowed to be tested in the limited populatiion that need it under guidance of their doctors?
If you say they should file now and it will be approved, then make your argument that deals with the issues I just laid out, please. Or anyone, Doc or Investor, or anyone that can handle this question.
Yes we are doing very well with the latest top line data. But is it enough? Let the debate begin.
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