FDA Approves First CAR T-Cell Therapy for CLL, SLL

The Food and Drug Administration granted accelerated approval on March 14 to the first chimeric antigen receptor (CAR) T-cell therapy for patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

Known as lisocabtagene maraleucel (liso-cel, marketed as Breyanzi by Bristol Myers Squibb), this CD19-directed therapy is approved for CLL or SLL patients who have undergone at least two prior lines of treatment, including a Bruton tyrosine kinase inhibitor and a B-cell lymphoma 2 inhibitor. FDA previously approved liso-cel to treat certain adult patients with large B-cell lymphoma, including diffuse large B-cell lymphoma, high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma and follicular lymphoma grade 3B. 

FDA based its approval on results from the phase 1/2 open-label, single-arm TRANSCEND CLL 004 study. In this study, patients treated with liso-cel showed a complete response rate (CR) of 20%, with a median duration of CR not reached at the data cutoff. Patients who achieved a CR also demonstrated high rates of minimal residual disease (MRD)-negative status, with MRD-negativity rates of 100% in the blood and 92.3% in the bone marrow.

Liso-cel comes with a boxed warning for cytokine release syndrome, neurologic toxicities and secondary hematological malignancies. The approval includes a risk evaluation and mitigation strategy (REMS) with elements to assure safe use. Additional information is available on FDA’s Cellular and Gene Therapy Products web page.