A typically reliable method of securing U.S. approval for a cancer drug these days is to run a small, single-arm clinical trial in patients no longer responsive to all approved therapies. Eke out a modest benefit for patients from the study, and FDA is often willing to greenlight approval.
This is the path Karyopharm Therapeutics is pursuing with its experimental pill selinexor. The small biotech announced Monday evening that 25 percent of multiple myeloma patients responded to treatment with selinexor in a single-arm clinical trial. The median duration of response was 4.4 months.
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In response to the first paragraph I ask “What should they do?” Or to put it another way “The approach described in the first paragraph is bad because _____” and then the critic fills in the blank.