FDA Approves First CAR T-Cell Therapy for ALL

October 06, 2021

The Food and Drug Administration recently approved brexucabtagene autoleucel (Tecartus, Kite Pharma) to treat adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) on Oct. 1. Brexucabtagene autoleucel, an autologous, anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, is the first FDA-approved CAR T-cell therapy for ALL.

Investigators evaluated the safety and efficacy of brexucabtagene autoleucel in the ZUMA-3 trial, a global, multicenter, single-arm, open-label study in which 65% of the evaluable patients (54) achieved complete remission (CR) or CR with incomplete hematological recovery (CRi) at a median actual follow-up of 12.3 months. Investigators estimated the duration of CR to exceed 12 months for more than half the patients. Among the 78 patients treated with brexucabtagene autoleucel at the target dose, Grade 3 or higher cytokine release syndrome and neurologic events occurred in 26% and 35% of patients, respectively.

Because of the risk of CRS and neurological toxicities, FDA approved brexucabtagene autoleucel with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use. The agency is also requiring Kite to conduct a post-marketing observational study involving patients treated with the therapy.

FDA previously approved brexucabtagene autoleucel to treat adult patients with relapsed or refractory mantle cell lymphoma. Outside of the United States, the therapy is currently under review in the European Union and United Kingdom for the treatment of adult patients with relapsed or refractory B-cell precursor ALL.