FDA Approval of Tisagenlecleucel

Abstract

Approval of the drug tisagenlecleucel (Kymriah; Novartis) by the US Food and Drug Administration (FDA) for the treatment of pediatric and young adult acute lymphoblastic leukemia (ALL) in August 2017 was a landmark in oncology. The science underlying chimeric antigen receptor T cells (CAR-T) heralds a new era of treatment, and the list price of $475 000 for the new drug (delivered as a one-time infusion) definitively shattered oncology drug pricing norms.¹