New Hope for Leukemia: FDA Approves Breyanzi, the First CAR T Therapy for CLL and SLL

The FDA has approved Breyanzi, a CAR T cell therapy, as the first treatment for relapsed or refractory chronic lymphocytic leukemia and small lymphocytic lymphoma. This one-time, personalized therapy has shown promising results, offering new hope to patients with limited options after failing traditional treatments.

Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has approved Breyanzi (lisocabtagene maraleucel; liso-cel), a CAR T cell therapy, marking a significant advancement in the treatment of chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). This approval positions Breyanzi as the first and only CAR T cell therapy for adults with relapsed or refractory CLL or SLL, offering new hope to patients who have exhausted other treatment options.

“CAR T cell therapies represent a transformative treatment option for patients with certain types of blood cancers. For years, attempts to bring other CAR T cell therapies to patients with relapsed or refractory CLL or SLL met challenges and found little success. With the approval of Breyanzi as the first CAR T for relapsed or refractory CLL or SLL, we are now able to offer these patients a personalized option, while further expanding access across the broadest array of B-cell malignancies, to address this critical unmet need” said Bryan Campbell, SVP, Head of Commercial, Cell Therapy, Bristol Myers Squibb.

A New Horizon for CLL and SLL Patients

Chronic lymphocytic leukemia and small lymphocytic lymphoma, two of the most common types of B-cell lymphoma, have traditionally been treated with targeted therapies, including Bruton tyrosine kinase (BTK) and B-cell lymphoma 2 (BCL-2) inhibitors. However, patients often relapse or become refractory after these treatments, with few options available that offer durable complete responses. Breyanzi's approval heralds a shift towards a one-time, personalized treatment approach, potentially offering patients a chance at complete and lasting remission.

“CLL and SLL are currently considered incurable diseases with few treatment options in the relapsed setting that can confer complete responses. The FDA approval of liso-cel in relapsed or refractory CLL and SLL after treatment with prior BTKi and BCL2i is a remarkable breakthrough, shifting the treatment paradigm from continuous therapy with sequential regimens to overcome drug resistance, to a one-time personalized T-cell based approach that has the potential to offer patients complete and lasting remission,” said Dr. Tanya Siddiqi, lead investigator and Division of Lymphoma, City of Hope National Medical Center.

The Science Behind Breyanzi

Breyanzi is a CD19-directed chimeric antigen receptor (CAR) T cell therapy. It involves reprogramming a patient's T cells to recognize and attack CLL or SLL cells. This therapy has demonstrated deep and durable responses in the TRANSCEND CLL 004 study, a first-of-its-kind multicenter trial evaluating a CAR T cell therapy in this patient population.

Remarkably, 20% of patients achieved a complete response, with a median duration of response not reached at the time of data cutoff. Furthermore, the study showed a high rate of minimal residual disease (MRD) negative status among those who achieved a complete response.

The approval of Breyanzi is not just a win for CLL and SLL patients but also signals a broader move towards employing CAR T cell therapies earlier in the treatment course for various cancers.

Addressing Safety Concerns

Despite its efficacy, CAR T cell therapies, including Breyanzi, carry risks such as cytokine release syndrome (CRS), neurologic toxicities, and secondary hematological malignancies. These concerns have been addressed through a boxed warning and the development of toxicity management guidelines.