Below is the meat and potatoes on FDA requirements
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Posted On: 11/16/2019 11:20:26 PM
Below is the meat and potatoes on FDA requirements for BTD approval, straight from the FDA’s website. I was thinking about NP’s numerous comments on mTNBC and BTD, specifically him saying that data from 5 patients could be enough to file for and receive BTD. I was specifically thinking about what leronlimab would have to demonstrate in these 5 mTNBC patients to receive BTD.
From what I’ve gathered, it appears CYDY will use the reduction in CTC, reduction in tumor size, and leronlimab’s excellent safety profile to file for the BTD designation (Of course, this is all pending proof of concept in the current mTNBC trial). Each of these 3 items meets the criteria as a “clinically significant endpoint”, as defined by the FDA below.
The results from the 1st mTNBC patient, more like generalities at this point because we weren’t given specific data, appears to point in the BTD direction as long as the subsequent patients show similar results. Let’s hope the recent PR will help fill the trial and get results needed to get the BTD process going.
From the FDA website:
In general, the preliminary clinical evidence should show a clear advantage over available therapy.
For purposes of Breakthrough Therapy designation, clinically significant endpoint generally refers to an endpoint that measures an effect on irreversible morbidity or mortality (IMM) or on symptoms that represent serious consequences of the disease. A clinically significant endpoint can also refer to findings that suggest an effect on IMM or serious symptoms, including:
-An effect on an established surrogate endpoint
-An effect on a surrogate endpoint or intermediate clinical endpoint considered reasonably likely to predict a clinical benefit (i.e., the accelerated approval standard)
-An effect on a pharmacodynamic biomarker(s) that does not meet criteria for an acceptable surrogate endpoint, but strongly suggests the potential for a clinically meaningful effect on the underlying disease
-A significantly improved safety profile compared to available therapy (e.g., less dose-limiting toxicity for an oncology agent), with evidence of similar efficacy
From what I’ve gathered, it appears CYDY will use the reduction in CTC, reduction in tumor size, and leronlimab’s excellent safety profile to file for the BTD designation (Of course, this is all pending proof of concept in the current mTNBC trial). Each of these 3 items meets the criteria as a “clinically significant endpoint”, as defined by the FDA below.
The results from the 1st mTNBC patient, more like generalities at this point because we weren’t given specific data, appears to point in the BTD direction as long as the subsequent patients show similar results. Let’s hope the recent PR will help fill the trial and get results needed to get the BTD process going.
From the FDA website:
In general, the preliminary clinical evidence should show a clear advantage over available therapy.
For purposes of Breakthrough Therapy designation, clinically significant endpoint generally refers to an endpoint that measures an effect on irreversible morbidity or mortality (IMM) or on symptoms that represent serious consequences of the disease. A clinically significant endpoint can also refer to findings that suggest an effect on IMM or serious symptoms, including:
-An effect on an established surrogate endpoint
-An effect on a surrogate endpoint or intermediate clinical endpoint considered reasonably likely to predict a clinical benefit (i.e., the accelerated approval standard)
-An effect on a pharmacodynamic biomarker(s) that does not meet criteria for an acceptable surrogate endpoint, but strongly suggests the potential for a clinically meaningful effect on the underlying disease
-A significantly improved safety profile compared to available therapy (e.g., less dose-limiting toxicity for an oncology agent), with evidence of similar efficacy
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