The U.S. Food and Drug Administration (FDA) has approved the gene-edited autologous chimeric antigen receptor (CAR) T-cell therapy lisocabtagene maraleucel (liso-cel) for adult patients whose disease has not responded to or has relapsed after at least two other systemic treatments. This approval covers diffuse large B-cell lymphoma (DLBCL); high-grade B-cell lymphoma; primary mediastinal large B-cell lymphoma; and grade 3b follicular lymphoma. Liso-cel is not indicated to treat primary central nervous system lymphoma.
The approval is based on results from the TRANSCEND-NHL-001 trial, which enrolled 269 adults with relapsed/refractory DLBCL. The complete remission rate was 54% among patients treated with liso-cel.
Liso-cel carries a boxed warning for cytokine release syndrome (CRS). Forty-two percent of trial participants experienced CRS, with 30% showing symptoms of neurotoxicity. The FDA is requiring health care facilities that dispense liso-cel for this indication to be certified and trained to recognize and manage the risks of CRS and neurologic toxicities.
In addition, the agency is requiring liso-cel's manufacturer, Bristol Myers Squibb's unit Juno Therapeutics, to conduct a post-marketing observational study to evaluate liso-cel's long-term safety.