So the trial that I’m describing and commenting on here is the publication by David Miklos and colleagues that came last year, so in fall of 2017, was published in blood, on 42 patients and that study specifically led the US Food and Drug Administration to grant a regulatory approval to chronic graph versus host disease, indication for ibrutinib for patients who failed one or more lines of therapy for this disease, so the second line therapy...
So the trial that I’m describing and commenting on here is the publication by David Miklos and colleagues that came last year, so in fall of 2017, was published in blood, on 42 patients and that study specifically led the US Food and Drug Administration to grant a regulatory approval to chronic graph versus host disease, indication for ibrutinib for patients who failed one or more lines of therapy for this disease, so the second line therapy.
People frequently ask, so how come an FDA approval was given to such a relatively small study, and the explanation is multiple. This is my interpretation; it’s a rare disease, and the FDA has established pathways for areas of prominent need, and this situation was a perfect storm again, because it’s a rare disease, it’s a life-threatening disease, it’s an unmet need, there’s an agent of promising new and novel mechanism of action, so all these things that came together, it’s an area where it’s impractical and frequently impossible to do large pivotal placebo-controlled trials.
So in that setting, 42 patients were tested using ibrutinib standard dose, and there are three main findings from this study. One, there is quite an impressive, high response rate with ibrutinib in these patients who failed frontline therapies, very difficult patient and frail patient population, with chronic GvHD, 68 percent overall response rate, there were some complete responses, it’s very impressive, and this is all by using the NIH response criteria.
