NICE Recommends Expanded and Earlier Use of Kite CAR T-Cell Therapy to Treat Certain Types of Blood Cancer

Gilead Sciences Ltd. and Kite, a Gilead company, today announced that the National Institute for Health and Care Excellence (NICE) has recommended additional uses for its two CAR T-cell therapies as options for the treatment of certain blood cancers, within the Cancer Drugs Fund (CDF). As the leading cell therapy company, Kite has two CAR T-cell therapies now available on the NHS for four types of blood cancer.

Axicabtagene ciloleucel; axi-cel, has been recommended by NICE as an option 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 3 ZUMA-7 study. The primary endpoint was event-free survival (EFS); the 24-month EFS was 40.5% in the axi-cel arm and 16.3% in the standard-of-care (SOC) arm.¹ The median progression free survival (PFS) in the axi-cel arm was 14.7 months compared with 3.7 months (95%) in the SOC arm.¹ In addition, Gilead and Kite 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.

In Zuma-7, adverse events of grade 3 or higher occurred in 155 of 170 patients (91%) who received axi-cel and in 140 of 168 patients (83%) who received SOC. One grade 5 adverse event was considered to be related to axi-cel (hepatitis B virus reactivation).

"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.

In addition, Kite’s second CAR T-cell therapy, Tecartus^® (brexucabtagene autoleucel; brexu-cel) is now available as an option 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 CAR 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 1/2 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 review. Adverse events of grade 3 or higher occurred in 76% of patients who received brexu-cel. Two grade 5 events were considered to be related to brexu-cel (brain herniation and septic shock).

“From the first clinical trials, we have been committed to ensuring patients are able to access these innovative cell therapies and today’s decision for blood cancer patients comes just five years since we worked with NICE and NHS England to make England the first country in Europe to access CAR T-cell therapy for DLBCL,” said Dr Véronique Walsh, Vice President and General Manager, Gilead Sciences UK & Ireland. “This latest journey, however, has been long and complex and NHS patients are now accessing these new indications after their peers in other countries. Industry, NHS England and NICE must continue to work together and collaborate to ensure that the process is accelerated and improved for the new waves of innovative cancer medicines, and that there is equitable access to such treatments for all eligible patients.”

CAR T-cell therapies are individually made starting from a patient’s own white blood cells, called T-cells. The cells are removed through a process similar to donating blood platelets or plasma and sent to Kite’s specialised manufacturing facilities where they are engineered to target the patient’s cancer, expanded, and then returned to the hospital for infusion back into the patient.