4 October 2021 - - The US Food and Drug Administration has granted approval for Tecartus (brexucabtagene autoleucel) for the treatment of adult patients (18 years and older) with relapsed or refractory B-cell precursor acute lymphoblastic leukemia, US-based biopharmaceutical company Gilead's (NASDAQ: GILD) Kite business said.

Following FDA Breakthrough Therapy Designation and a priority review, Tecartus is the first and only chimeric antigen receptor T-cell therapy approved for adults (18 years and older) with ALL.

There is a high unmet need, as half of this patient population will relapse, and median overall survival is only approximately eight months with current standard-of-care treatments. Patients can access Tecartus through 109 authorized treatment centers across the US.

The approval is based on results from ZUMA-3, a global, multicenter, single-arm, open-label study in which 65% of the evaluable patients achieved complete remission or CR with incomplete hematological recovery at a median actual follow-up of 12.3 months.

The duration of CR was estimated to exceed 12 months for more than half the patients. Among efficacy-evaluable patients, median duration of remission was 13.6 months.

Among the patients treated with Tecartus at the target dose, Grade 3 or higher cytokine release syndrome and neurologic events occurred in 26% and 35% of patients, respectively, and were generally well-managed.

Adults with relapsed or refractory ALL often undergo multiple treatments including chemotherapy, targeted therapy and stem cell transplant.

CAR T-cell therapy works differently, by harnessing a patient's own immune system to fight cancer. With CAR T, the patient's blood is drawn and the T cells are separated.

Then the T cells are genetically engineered with a specific receptor that enables them to identify and attack cancer cells, and put back into the patient's body.

Tecartus is also currently under review in the European Union and United Kingdom for the treatment of adult patients with relapsed or refractory B-cell precursor ALL.

The Tecartus US Prescribing Information has a BOXED WARNING for the risks of CRS and neurologic toxicities, and Tecartus is approved with a Risk Evaluation and Mitigation Strategy due to these risks; see below for Important Safety Information.

Further efficacy results from the ZUMA-3 trial have been published and presented in scientific forums. Published Phase 1 data showed 32% of responders were still in remission at the median follow-up of 22.1 months.

In Phase 2 data presented at the 2021 American Society of Clinical Oncology annual meeting, investigators reported that among treated patients, 31% of these patients were in ongoing response at a median follow-up of 16.4 months.

97% of responders had deep molecular remission, with undetectable minimal residual disease, and median OS among all responders has not yet been reached.

A safety analysis, reported in the Lancet, showed among all patients who experienced a neurologic event, 94% of CRS events and 88% of neurologic events were resolved.

ZUMA-3 is an international multicenter, registrational Phase 1/2 study in adult patients (≥18 years old) with ALL whose disease is refractory to or has relapsed following first standard systemic therapy with remission of 12 months or less, after two or more lines of systemic therapy or at least 100 days after allogeneic stem cell transplantation.

Seventy-one patients were enrolled (and leukapheresed) in the study, and the primary endpoint was overall complete remission rate (OCR, equaling complete remission plus complete remission with incomplete hematologic recovery) as determined by an independent review.

ALL is an aggressive type of blood cancer that can also involve the lymph nodes, spleen, liver, central nervous system and other organs.

Approximately 1,000 adults are treated annually for relapsed or refractory ALL. B-cell precursor ALL is the most common form, accounting for approximately 75% of cases, and treatment is typically associated with inferior outcomes compared with other types of ALL.

Survival rates remain very poor in adult patients with relapsed or refractory ALL, with median OS at less than eight months.

Tecartus is an autologous, anti-CD19 CAR T-cell therapy. Tecartus uses the XLP manufacturing process that includes T cell enrichment, a necessary step in certain B-cell malignancies in which circulating lymphoblasts are a common feature.

Tecartus is also being evaluated in pediatric ALL, where its use is investigational, and its safety and efficacy have not been established.

Kite, a Gilead company, is a global biopharmaceutical company based in Santa Monica, California, with commercial manufacturing operations in North America and Europe.

Kite's singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company.

Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.