FDA Approves Updated Label of Axi-Cel Based on ZUMA-1 Findings


The label for axicabtagene ciloleucel now includes prophylactic corticosteroids use in all approved indications.

The FDA has approved a label update for axicabtagene ciloleucel (axi-cel; Yescarta) to include prophylactic corticosteroids use in all approved indications. This update is based on the safety management cohort of the ZUMA-1 (NCT02348216) trial, according to a press release from Kite, a Gilead Comopany.1

With this updated label, axi-cel is the only chimeric antigen receptor (CAR) T-cell therapy with label information that helps physicians manage treatment side effects.

Axicabtagene ciloleucel is a CD-19–directed, genetically modified autologous T cell therapy used in the treatment of adults with relapsed or refractory large B-cell lymphoma (LBCL) after two or more lines of systemic therapy and with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.

The ZUMA-1 trial’s safety management cohort, Cohort 6, investigated cytokine release syndrome (CRS) and neurologic events in patients with added prophylactic corticosteroids and earlier treatments of corticosteroid and tocilizumab. CRS and neurologic events were the primary endpoints of this cohort.2

Cohort 6 (n = 39) showed no grade 3 or worse CRS compared with 13% seen in cohorts 1/2 (n = 108). However, cohort 6 did experience grade 3 or worse neurologic events in 13% of patients, and one patient experienced grade 5 neurologic events after data cutoff. Grade 3 or higher neurologic events in cohorts 1/2 expressed in 31% of patients. Cohort 6 had a median CRS time to onset of 5 days (range 1-15) and a neurotoxicity median to onset of 6 days (range 1-274).

Data also showed that 68% of cohort 6 patients didn’t have any CRS or neurologic events events within 72 hours of axicabtagene ciloleucel infusion. It was also found that cohort 6 received fewer median steroid doses than patients in cohorts 1/2 to manage CRS and neurologic events.

“These new data will enable doctors to more easily and confidently manage treatment for patients,” said Frank Neumann, MD, PhD, Kite’s global head of Clinical Development. “Since the first approval of Yescarta, Kite has worked closely with physicians to optimize all aspects of CAR T-cell therapy to enable as many patients as possible to have the chance to benefit from this treatment. Our responsibility includes research to expand into new diseases and earlier lines of treatment, but also continuously improving the efficacy and safety of our existing CAR T therapies.”

Axi-cel is under review in the US and European Union as an earlier treatment option for adult patients with second-line relapsed or refractory LBCL.


1. U.S. FDA approves new label update for car t-cell therapy yescarta® showing prophylactic steroid use improves management of cytokine release syndrome. Press release. Gilead; January 31, 20202. Accessed February 1, 2022. https://bit.ly/3s9Bwri

2. Oluwole OO, Bouabdallah K, Muñoz J, et al. Prophylactic corticosteroid use in patients receiving axicabtagene ciloleucel for large B-cell lymphoma. Br J Haematol. 2021;194(4):690-700. doi:10.1111/bjh.17527

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