how accelerated approval works
first of all let me state that imo what we need to see is not necessarily an accelerated pathway or any approvals, but rather pd-1 elevation and big pharma attention. that is the end game here. that's the entire ball of wax, at the moment.
regarding "why was accelerated approval even established if you need a completed trial and a BLA"?
because otherwise
1) you would have to base the BLA on OUTCOMES. that alone could add years to a clinical trial vs surrogates.
2) accelerated approval allows the approval PROCESS to occur without waiting on the phase 3 trial, which would by itself add MANY more years to complete.
this easily cuts off 3 to 5 years.
meanwhile, PD-1 levels are not an officially defined endpoint for this trial. only officially defined endpoints, usually pre-approved by the FDA as sanctioned surrogates for accelerated approval, are adequate for seeking accelerated approval.
cytodyn could theoretically still pursue AA by using "tumor shrinkage" being deeper and/or lasting longer than SOC as a surrogate.
what timeline would you then be looking at?
1) finish the trial. this is 100% required. this trial is 12 months dosing after enrollment. so enrollment may fill lets say by the end of may. then 12 months dosing, which takes us to end of may 2027.
what we need to see is not necessarily an accelerated pathway or any approvals, but rather pd-1 elevation and big pharma attention. that is the end game here.
2) then the data cleanup and data lock must occur... that is a tedious process even for a world class CRO and can take months - they are working with the clinical sites on this.
that takes us to end of the summer 2027.
3) THEN the clinical section of the BLA can begin in earnest. The clinical section of a BLA takes 6 to 12 months. its literally tens of thousands of pages, even for a small trial. being a small trial and a world class CRO, lets say 6 to 8 months. that takes us into q1 of 2028 for BLA submission.
what we need to see is not necessarily an accelerated pathway or any approvals, but rather pd-1 elevation and big pharma attention. that is the end game here.
4) upon submission the FDA has 60 days to respond. this is the PRE-approval, just looking at the basics of your submission. they either accept or reject. rejection isn;t unusual. that means you go back and fix stuff. that can be quick n easy or can be brutal.
lets ignore that possibility though. the trial is straight forward the CRO is world class.
so we are now at the end of q1 or into q2 2028.
what we need to see is not necessarily an accelerated pathway or any approvals, but rather pd-1 elevation and big pharma attention. that is the end game here.
5) the final step is the actual BLA review which in this case is "up to 10 months" for standard review, and they usually take most or all of that time up. fast track would be 6 months instead of 10. i can't recall if they have mss-crc fast track, don't think do though. this takes us to the end of 2028.
that would be for an ACCELERATED approval.
what we need to see is not necessarily accelerated pathways, but rather pd-1 elevation and big pharma attention. that is the end game here.
first of all let me state that imo what we need to see is not necessarily an accelerated pathway or any approvals, but rather pd-1 elevation and big pharma attention. that is the end game here. that's the entire ball of wax, at the moment.
regarding "why was accelerated approval even established if you need a completed trial and a BLA"?
because otherwise
1) you would have to base the BLA on OUTCOMES. that alone could add years to a clinical trial vs surrogates.
2) accelerated approval allows the approval PROCESS to occur without waiting on the phase 3 trial, which would by itself add MANY more years to complete.
this easily cuts off 3 to 5 years.
meanwhile, PD-1 levels are not an officially defined endpoint for this trial. only officially defined endpoints, usually pre-approved by the FDA as sanctioned surrogates for accelerated approval, are adequate for seeking accelerated approval.
cytodyn could theoretically still pursue AA by using "tumor shrinkage" being deeper and/or lasting longer than SOC as a surrogate.
what timeline would you then be looking at?
1) finish the trial. this is 100% required. this trial is 12 months dosing after enrollment. so enrollment may fill lets say by the end of may. then 12 months dosing, which takes us to end of may 2027.
what we need to see is not necessarily an accelerated pathway or any approvals, but rather pd-1 elevation and big pharma attention. that is the end game here.
2) then the data cleanup and data lock must occur... that is a tedious process even for a world class CRO and can take months - they are working with the clinical sites on this.
that takes us to end of the summer 2027.
3) THEN the clinical section of the BLA can begin in earnest. The clinical section of a BLA takes 6 to 12 months. its literally tens of thousands of pages, even for a small trial. being a small trial and a world class CRO, lets say 6 to 8 months. that takes us into q1 of 2028 for BLA submission.
what we need to see is not necessarily an accelerated pathway or any approvals, but rather pd-1 elevation and big pharma attention. that is the end game here.
4) upon submission the FDA has 60 days to respond. this is the PRE-approval, just looking at the basics of your submission. they either accept or reject. rejection isn;t unusual. that means you go back and fix stuff. that can be quick n easy or can be brutal.
lets ignore that possibility though. the trial is straight forward the CRO is world class.
so we are now at the end of q1 or into q2 2028.
what we need to see is not necessarily an accelerated pathway or any approvals, but rather pd-1 elevation and big pharma attention. that is the end game here.
5) the final step is the actual BLA review which in this case is "up to 10 months" for standard review, and they usually take most or all of that time up. fast track would be 6 months instead of 10. i can't recall if they have mss-crc fast track, don't think do though. this takes us to the end of 2028.
that would be for an ACCELERATED approval.
what we need to see is not necessarily accelerated pathways, but rather pd-1 elevation and big pharma attention. that is the end game here.
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