FDA Accepts Novartis, Kite Pharma’s Personalized Cancer Treatments for Review

The FDA has accepted for review the first two novel, personalized cancer treatments — known as chimeric antigen receptor T-cell therapies, or CAR-T — using immune system cells drawn from the blood and reprogrammed to target cancer cells.

Novartis was granted priority review for its CTL019 (tisagenlecleucel-T) treatment for younger patients with B-cell acute lymphoblastic leukemia. Also last week, Kite Pharma completed its rolling BLA for axicabtagene ciloleucel, previously known as KTE-C19, for non-Hodgkin lymphoma patients ineligible for stem cell transplants.

Novartis is basing its application in part on a global Phase II study, where 82 percent of 50 patients infused with CAR-T cells achieved complete remission or complete remission with incomplete blood count recovery after three months. Nearly half of the patients in the trial experienced grade 3 or 4 cytokine release syndrome, but there were no deaths. Novartis said it plans to submit an application with the European Medicines Agency later this year.

In December 2015, Kite’s axicabtagene received a Breakthrough Therapy designation in three types of lymphoma. If approved, Kite plans to commercially launch axicabtagene ciloleucel in 2017; an EMA application is also planned for this year.