First CAR T-Cell Treatment Approved for Mantle Cell Lymphoma

The FDA granted accelerated approval to brexucabtagene autoleucel (formerly KTE-X19), a chimeric antigen receptor (CAR) T-cell therapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL). The approval marks the first CAR T-cell treatment approved for MCL.

The decision was based on ZUMA-2, a single-arm, multicenter, open-label trial of 60 adults with relapsed/refractory MCL, who were monitored for at least 6 months after first objective disease response. The objective response rate after a single infusion of brexucabtagene autoleucel was 87%, with 62% of patients achieving a complete response. Among patients evaluated for treatment safety, 18% experienced grade ≥3 cytokine release syndrome (CRS) and 37% experienced grade ≥3 neurologic toxicities.

Findings from ZUMA-2 were originally reported at the 2019 ASH Annual Meeting.

The treatment label carries a boxed warning for CRS. The most common adverse events associated with brexucabtagene autoleucel include serious infection, low blood cell counts, and a weakened immune system.

The treatment also was granted breakthrough therapy and orphan drug designations.

Sources: FDA press release, July 24, 2020; Gilead press release, July 24, 2020; ASH Clinical News, January 15, 2020.