UK’s NICE expands use of CAR-T cell therapy for blood cancer

The UK’s National Institute for Health and Care Excellence (NICE) has recommended additional uses for two CAR-T therapies from Gilead Sciences/Kite, as part of the Cancer Drugs Fund (CDF). Yescarta (axicabtagene ciloleucel: axi-cel) has been recommended by NICE for the treatment of eligible adult patients with diffuse large B cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL) that relapses within 12 months of completion of or is refractory to first-line treatment. The data supporting its use is based on primary results of the pivotal Phase III ZUMA-7 study. The 24-month event-free survival was 40.5% in the axi-cel arm and 16.3% in the standard-of-care (SOC) arm. The median progression free survival in the axi-cel arm was 14.7 months compared with 3.7 months for SOC. Gilead and Kite also recently announced that axi-cel has demonstrated a statistically significant improvement in overall survival (OS) for the initial treatment of relapsed/refractory DLBCL versus historical standard of care in a curative setting – the first and only treatment to do so in nearly 30 years. "This decision is a pivotal moment for expanding how CAR-T cell therapy is used to treat DLBCL – until now, these therapies have been reserved for use when patients have failed traditional standard of care and had few options remaining. With today’s announcement, we will now have the option to use it earlier for some patients, potentially creating a new pathway and standard of care," said Dr Sridhar Chaganti, Consultant Haematologist, Queen Elizabeth Hospital Birmingham. Tecartus recommended for ALL Kite’s second CAR-T cell therapy, Tecartus (brexucabtagene autoleucel; brexu-cel) is now available for the first time to treat eligible adult patients, 26 years of age and above, with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL). “The approval of this CAR T-cell therapy for adult patients with acute lymphoblastic leukaemia represents an important change for adult ALL patients. This treatment can achieve deep remissions, and significant survival in patients refractory to all other treatments. For example, CAR T-cell therapy may provide long term remission for ALL patients who relapse after allogeneic haematopoetic stem cell transplantation: it offers these patients real hope,” said Professor of Haematology and Stem Cell Transplantation, David Marks. “In addition, certain high risk patients who can’t achieve or maintain deep remissions, or who are unsuitable for alloHSCT, are now eligible for CA- T cells. In ALL, patients less than 26 years old have had the option of therapy with CAR-T cells for some time and this approval now ensures patients of all ages can access the latest scientific advances.” Data supporting the use of brexu-cel was observed in the pivotal ZUMA-3 single-arm trial. In the combined Phase I/II data set, 73% of the evaluable patients treated with brexu-cel achieved overall complete remission (with or without incomplete hematologic recovery), as determined by an independent revie