CTL 019 is filed at EMA for the treatment of young people with r/r B-cell acute lymphoblastic leukemia and for adults with r/r diffuse large B-cell lymphoma.- Novartis

Novartis announced that the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for CTL 019 (tisagenlecleucel) for two indications. The application is for the treatment of children and young adults with relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (ALL) and for adult patients with r/r diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant (ASCT).

CTL 019 is a novel immunocellular therapy and a one-time treatment that uses a patient's own T cells to fight cancer. There has been a dire need for innovative therapies to treat pediatric and young adult patients with r/r B-cell ALL and adult patients with r/r DLBCL, who have few options and historically poor outcomes. DLBCL is the most common subtype of non-Hodgkin lymphoma (NHL), accounting for 40% of all NHL cases globally. If left untreated, r/r DLBCL has a life expectancy of three to four months. In Europe, ALL accounts for approximately 80% of leukemia cases among children. Less than 10% of patients with relapsed or refractory ALL survive five years.

CTL 019 is an innovative immunocellular therapy that is a one-time treatment. CTL 019 uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular expansion and persistence. In 2012, Novartis and the University of Pennsylvania (Penn) entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including CTL 019, for the investigational treatment of cancers.