Yes harvett - I also think that a really important takeaway from...

Yes harvett - I also think that a really important takeaway from ACCELERATE was this bit, that you alluded to. Interesting and new, could be a valid description.

"....support innovative regulatory approaches to obtain an indication." Indication just means a particular disease.


A bit further down in this post...... is the header to the ACCELERATE news release NOTE especially the attendance of the FDA at this meeting. A photograph and name of the FDA person in attendance, is shown on the ACCELERATE web page also. That is great to see

So mention of anything to do with any new drug applications and FDA views ....post ACCELERATE, is validated by the FDA attendance.

I think though harvett - the prompt speed of any paxalisib approval, in DIPG may already be covered by the previous grant of a  Rare Pediatric Disease Designation and Orphan Designation by the US FDA for DIPG in August 2020 (plus of course the voucher). "Fast" is embedded in these grants, I understand....although not a Fast Track designation, such as paxalisib in GBM.

I had never heard of ACCELERATE until now -  but check their web page. It is like of world rep team selection  - of regulators, expects, best of the best in their field. No wonder the FDA were in attendance.

Paxalisib perfectly matches the summary of criteria presented by ACCELARATE - at least in the brain penetrative PI3K field.

"interesting, and new "...........To better explain - remember, my post whereby I suggested this DIPG data may reveal 60 patients with a medium OS of 16 months (verse say 7 months without treatment).

Now this ACCELERATE news release (FDA seemingly endorsed it) -  means the forthcoming Kazia DIPG data, does NOT have to be so absolute, so clear cut (as the I described).  So you see, these words are new and important from ACCELEATE ......."support innovative regulatory approaches to obtain an indication "

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Above may be a bit confusing already -  but what about the data for GBM AGILE, with now suggestions of FDA accepting innovative approaches.  Remember Dr Wen in a presentation post that high drama GBM AGILE news - Dr Wen saying paxalisib does provide a signal in GBM disease. I don't think the FDA will walk away, or turn down a very high profile PI3K Clinical trial NDA .........if the GBM data is endorsed to go forward by GBM AGILE itself.

The large amount of more recent positive research in PI3K combination therapy, may be one innovate option in new drug applications. (ie what ACCELERATE are referring to)  - given what is well documented now, and how PI3K works in other drug combinations.

Suggest watch carefully the words associated with release of this paxalisib DIPG, GBM  data. (all of the paxalisib in monotherapy data, in fact ).....from these worlds best doctors. Data releases are not coming from the minnow Kazia - but the worlds best hospitals.

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Paediatric Strategy Forum for Medicinal Product Development of PI3K, mTOR, AKT and GSK3β Inhibitors in children and adolescents



ACCELERATE in collaboration with the European Medicines Agency

With participation of the Food and Drug Administration