Bristol Myers Squibb’s CD19-directed CAR T cell therapy Breyanzi (lisocabtagene maraleucel) has been granted accelerated approval by the US Food and Drug Administration (FDA) to treat chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL).
The regulator’s decision specifically applies to adult patients with relapsed or refractory disease who have received at least two prior lines of therapy, including a Bruton tyrosine kinase inhibitor and a B-cell lymphoma 2 inhibitor.
CLL is one of the most common types of leukaemia in adults, with about 20,700 new cases of the disease expected to be diagnosed in the US this year.
CLL and SLL are essentially the same diseases that are treated in the same way but are named depending on the location of the patients’ cancer cells. In CLL, the cancer cells are present in the blood and bone marrow, and in SLL, they appear in the lymph nodes.
BMS noted that, while there are several treatments available for CLL and SLL, “there is a need for additional effective therapies” as there is no standard of care for relapsed or refractory disease following targeted therapies.
Breyanzi already holds approvals to treat certain cases of large B-cell lymphoma and is made from patients’ own T cells, which are collected and genetically re-engineered to become CAR T cells that are then delivered via infusion as a one-time treatment.
The FDA’s latest approval of the therapy was supported by positive results from the phase 1/2 TRANSCEND CLL 004 trial, which was the first pivotal multicentre trial to evaluate a CAR T cell therapy in patients with relapsed or refractory CLL or SLL, according to BMS.
Results showed that 20% of patients treated with Breyanzi achieved a complete response, with median duration of complete response not reached at the time of data cutoff.
Bryan Campbell, senior vice president, head of commercial, cell therapy, BMS, said: “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 personalised option, while further expanding access across the broadest array of B-cell malignancies, to address this critical unmet need.”