The US Food and Drug Administration (FDA) has approved Novartis’ Kymriah (tisagenlecleucel) suspension for intravenous infusion to treat patients up to 25 years of age with B-cell precursor acute lymphoblastic leukaemia (ALL) that is refractory or in second or later relapse.
ALL is a cancer of the lymphocytes, which is a type of white blood cell involved in the body's immune system.
Formerly CTL019, Kymriah is the company’s first chimeric antigen receptor T-cell (CAR-T) therapy to have received the approval.
It is a new immunocellular and one-time treatment that uses a patient's own T-cells to combat cancer.
Novartis oncology CEO Bruno Strigini said: “As a breakthrough immunocellular therapy for children and young adults who desperately need new options, Kymriah truly embodies our mission to discover new ways to improve patient outcomes and the way cancer is treated.”
The current FDA approval is based on the data obtained from the pivotal open-label, multicentre, single-arm Phase II ELIANA trial, which is the first paediatric global CAR-T cell therapy registration trial evaluating patients in 25 centres across the US, European Union (EU), Canada, Australia and Japan.
In addition, the US agency has approved a risk evaluation and mitigation strategy (REMS) for Kymriah that focuses on informing and educating healthcare professionals about the risks associated with the treatment.
In 2012, Novartis entered a global collaboration with the University of Pennsylvania (Penn) to further research, develop and market CAR-T cell therapies, including Kymriah, for the investigational treatment of cancers.
Later this year, the company intends to submit additional applications for Kymriah in the US and EU, including filings with the FDA and European Medicines Agency (EMA), for the treatment of adult patients with recurrence rate diffuse large B-cell lymphoma (DLBCL).
Image: A Wright's stained bone marrow aspirate smear of a patient with precursor B-cell acute lymphoblastic leukaemia. Photo: courtesy of VashiDonsk at English Wikipedia.