FDA Approves Breyanzi for Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

The FDA has granted accelerated approval of Breyanzi (lisocabtagene maraleucel; liso-cel), to treat adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). It is indicated for those who have received at least two prior lines of therapy.

CLL and SLL are among the most common types of B-cell lymphoma. Treatments primarily consist of targeted therapies including a Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor. However, patients often experience relapse or become refractory following early-line treatment with these therapies.

Tanya Siddiqi, M.D.

“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,” Tanya Siddiqi, M.D., lead investigator and associate professor, Division of Lymphoma, City of Hope National Medical Center, said in a press release.

Developed by Bristol Myers Squibb, Breyanzi is a CD19-directed CAR T cell therapy. It is already approved to treat adult patients with large B-cell lymphoma who have refractory disease or who have relapsed. Breyanzi is derived from a patient’s own T cells that have been engineered to target CD19 B cells. Breyanzi includes a box warning about about the risk of cytokine release syndrome, a large release of cytokines into the blood, which triggers overreaction of the immune system, and neurological toxicities. 

The list price for Breyanzi is $487,477.43. For full year 2023, Breyanzi generated revenue of $364 million.

The approval in CLL and SLL is based on data from phase 1/2 open-label, single-arm TRANSCEND CLL 004 study. In this study, 20% of 89 patients treated with Breyanzi achieved a complete response rate. Among all responders, median duration of response was 35.3 months. Patients treated with Breyanzi who achieved a complete response experienced a minimal residual disease negativity rate of 100% in the blood and 92.3% in the bone marrow. Cytokine release syndrome was experienced by 83% of patients, which was mostly low grade.

Breyanzi is also under FDA review to treat patients with relapsed or refractory follicular lymphoma with a Prescription Drug User Fee Act (PDUFA) date of May 23, 2024, and for patients with relapsed or refractory mantle cell lymphoma (MCL) with a PDUFA date of May 31, 2024. Applications for these indications are based on results from TRANSCEND FL and TRANSCEND NHL 001, in which Breyanzi demonstrated clinically meaningful benefit with deep and durable responses.