FDA panel backs Emmaus' sickle cell therapy

FDA's Oncologic Drugs Advisory Committee voted 10-3 in favor of approval of Endari L-glutamine from Emmaus Life Sciences Inc. (Torrance, Calif.) to treat sickle cell disease (SCD). If FDA follows the panel's recommendation, it would mark the second approval ever for an SCD therapy. Endari would be the first new therapy approved for the Orphan disease in nearly two decades.

Endari's NDA is based on data from the Phase III GLUSCC09-01 trial, in which the compound met the primary endpoint by significantly reducing mean sickle cell crises from baseline to week 48 (3.2 vs. 3.9; p=0.005)...