There was an updated guidance for Accelerated Approval in...

There was an updated guidance for Accelerated Approval in December 2024. I'm sure another is on the way .

Confirmatory trials must be completed with due diligence.
38 FDA has interpreted the due
381diligence requirement to mean that sponsors must commit sufficient resources to conduct the
382trial(s) intended to verify the clinical benefit expeditiously so that a determination of whether the
383drug provides the expected clinical benefit can be made as soon as possible.
384
385When sponsors intend to seek accelerated approval, they should consult with FDA regarding
386postapproval confirmatory trial(s) early in the drug development program, and there should be
387agreement by FDA on the design and conduct of the confirmatory trial(s) to help ensure
388interpretable results. The protocol for a confirmatory trial should be developed and submitted to
389FDA as early as possible, and timelines for the trial should be specified; for example, timelines
390for enrollment and trial completion should be stipulated. The confirmatory trial’s design should
391ensure that the trial will be completed on timelines specified by FDA. FDA encourages sponsors
392to submit draft protocols to FDA during their development program to allow for sufficient time
393to review and discuss in preparation for submitting their final protocol.

394
395Discussions with FDA about using accelerated approval should come early in the development
396program, and confirmatory trial(s) should generally be underway at the time the marketing
397application is submitted. Except in limited circumstances, FDA intends to require that
398confirmatory trial(s) be underway prior to granting accelerated approval.


my take is , if you want an AA the FDA has to approve every part of the process BEFORE you start a confirmatory
trial and that the trial has to have commenced prior to lodging your BLA.


When I woke this morning from a blissful sleep, thx for asking, my mind was drawn to yesterday's announcement.

"The Company will await the final minutesfrom FDA in order to provide detailed feedback and timelines for potential filing".

Detailed feedback?? We don't , we being shareholders, get detailed feedback. Is this a free kick from Si?

Who needs to be provided detailed feedback? Who is going to fund the trial? Who has the resources to verify the clinical benefit expeditiously?
Tell me a partner would not insist on being involved in the planning of a confirmatory trial that had to be approved UPFRONT by the FDA .


388 interpretable results. The protocol for a confirmatory trial should be developed and submitted to
389 FDA as early as possible,



You can be in one of three camps

1. We won't qualify for an Accelerated Approval and therefore a confirmatory trial won't occur

2. We will qualify for an Accelerated Approval, design and conduct the trial ourselves, probably requiring a cash raise.

3. We partner with a Global Pharma who assists with the entire process, funds and conducts the trial, probably with an upfront cash injection.



If there is another option let me know.


This is way more exciting than waiting on an approval for Ryoncil, which was excruciating to say the least.


For those who bash Silviu, have a crack at Eric Rose too- he's the grand daddy of LVAD's and bought twice on market. The AA sought will cover
end stage LVAD patients- but a clever trial design may yield wider coverage of cardiac patients- globally.....................


Those minutes are due in the short term and they might just be the making of this company.

Pure speculation above, I read FDA guidance's as a fetish and I need more vegetables in my diet


Three options



Door number 3 thx - the one where Global Pharma helps up submit as early as possible


Reg