Liso-Cel Approved by FDA for CLL, SLL

The US Food and Drug Administration (FDA) has approved lisocabtagene maraleucel (liso-cel; Breyanzi^®), a chimeric antigen receptor (CAR) T-cell therapy, for adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), according to a press release by Bristol Myers Squibb, the developer of the drug.

The approval follows the FDA’s November 2023 statement on the potential risk of developing T-cell malignancies following CAR T-cell therapy. The FDA sent requests to drug manufacturers in January 2024 to update prescription labels with “Black Box” warnings on the six CAR-T therapies used to treat blood cancers.

Liso-cel was approved under accelerated approval based on response rate and duration of response data from the TRANSCEND CLL 004 trial. The phase I/II, open-label, single-arm study evaluated the safety and recommended dose of liso-cel in patients who have received at least two prior lines of therapy, including a Bruton tyrosine kinase inhibitor and a B-cell lymphoma 2 inhibitor.

The complete response (CR) rate was 20% (95% CI, 11.1-31.8). Among those who achieved a CR, the median duration of response was not reached (NR; 95% CI, 15 months-NR) at the time of data cutoff. Among all responders, the median duration of response was 35.3 months (95% CI, 12.4-NR).

Those who achieved a CR also had a measurable residual disease-negativity rate of 100% in the blood (95% CI, 75.3-100) and 92.3% in the bone marrow (95% CI, 64-99.8).

Occurrences of cytokine release syndrome (CRS) and neurologic events (NEs) were mostly low grade. Of 89 patients, 83% experienced any grade CRS, and 46% experienced any grade NEs.

“CLL and SLL are currently considered incurable diseases with few treatment options in the relapsed setting that can confer complete responses, something that has historically been associated with improved long-term outcomes,” said Tanya Siddiqi, MD, lead investigator of the TRANSCEND CLL 004 study and Associate Professor in the Division of Lymphoma at the City of Hope National Medical Center, in the press release.