FDA Approves First CAR T-Cell Therapy for rrCLL, rrSLL

The FDA has granted accelerated approval for lisocabtagene maraleucel (liso-cel), a CAR T-cell therapy, in certain adults with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Developed by Juno Therapeutics, the therapy, branded as Breyanzi, targets CD19 and is approved for patients who've undergone at least two prior lines of therapy, including specific inhibitors. This marks the first CAR T-cell therapy approved for this indication.

Lead investigator Tanya Siddiqi hailed the approval as a breakthrough, noting the shift from continuous therapy to a one-time personalized T-cell approach, potentially offering complete and lasting remission. Liso-cel was initially approved in 2021 for large B-cell lymphoma.

The approval was based on results from the TRANSCEND CLL 004 study, where 20% of CLL/SLL patients achieved a complete response post liso-cel infusion. The study, involving 89 participants, demonstrated a 45% overall response rate with a median duration of response of 35.3 months. The therapy exhibited a tolerable safety profile, with mostly low-grade cytokine release syndrome and neurologic events.

The approval of liso-cel represents a significant advancement in CLL/SLL treatment, providing hope for patients facing limited options and paving the way for further developments in CAR T-cell therapy.