On October 1, 2021, the FDA accelerated the approval of a new indication for brexucabtagene autoleucel (Tecartus; Kite Pharma), a CD19-directed CAR T-cell therapy, for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). The FDA granted brexucabtagene autoleucel breakthrough therapy and orphan drug designations for this indication.
This approval was based on the ZUMA-3 study, a single-arm multicenter clinical trial of adults with relapsed or refractory B-cell precursor ALL. Patients received a single infusion of brexucabtagene autoleucel after completion of lymphodepleting chemotherapy.
The efficacy measures were complete response (CR) within 3 months after receiving the CAR T-cell infusion, and the duration of the CR. Of the 54 patients evaluable for efficacy, 28 (52%; 95% confidence interval, 38-66) achieved CR within 3 months. At a median follow-up of 7.1 months, the median duration of CR was not reached, and the duration of CR was estimated to be >12 months for >50% of the patients.
The most common (≥20%) nonlaboratory side effects included fever, cytokine-release syndrome, hypotension, encephalopathy, tachycardias, nausea, chills, headache, fatigue, febrile neutropenia, diarrhea, musculoskeletal pain, hypoxia, rash, edema, tremor, infection with pathogen unspecified, constipation, decreased appetite, and vomiting.