FDA panel unanimously backs first CAR T therapy

FDA's Oncologic Drugs Advisory Committee (ODAC) voted 10-0 in favor of approval of the chimeric antigen receptor T cell therapy tisagenlecleucel-T (CTL019) from Novartis AG (NYSE:NVS; SIX:NOVN) to treat pediatric and young adult relapsed or refractory B cell acute lymphoblastic leukemia (ALL). Tisagenlecleucel-T is the first CAR T product ODAC has reviewed.

Panel members were impressed with the 82.5% overall remission rate (ORR) observed in a pivotal Phase II trial of the therapy (95% CI: 70.9, 91.0). While they were concerned by the 47% rate of grade 3 or 4 cytokine release syndrome (CRS) seen in the 68-patient study, the two pediatric oncologists on the panel felt that most physicians would be able to manage the side effect...