FDA reviewers express concerns about Emmaus' SCD data

In briefing documents released ahead of a May 24 meeting of FDA’s Oncologic Drugs Advisory Committee, FDA reviewers questioned the robustness of efficacy and magnitude of treatment seen in a Phase III study of Endari from Emmaus Life Sciences Inc. (Torrance, Calif.). The therapy is under FDA review to treat sickle cell disease (SCD), with a July 7 PDUFA date.

In their evaluation of the Phase III GLUSCC09-01 trial, the reviewers said data from the study were "insufficiently detailed" to assess why the premature withdrawal rate was higher among patients receiving Endari vs placebo (36.2% vs. 24.4%). The reviewers said all methods to account for missing data "had significant limitations." They also noted that the overall discontinuation rate of 31.9% was higher than the expected 25%...