FDA Approves Expanded Use of BMS CAR-T Therapy in Follicular Lymphoma

The U.S. Food and Drug Administration (FDA) has expanded the approved use of Bristol Myers Squibb's CAR-T cell therapy Breyanzi (lisocabtagene maraleucel) to include treatment for patients with relapsed or refractory follicular lymphoma. This decision, announced on Wednesday, allows patients who have undergone at least two prior lines of systemic therapy to be treated with Breyanzi under the FDA’s accelerated approval pathway, which is based on response data. To maintain this approval, Bristol Myers Squibb (BMS) must verify Breyanzi’s clinical benefit in this indication through a Phase III confirmatory study.

Bryan Campbell, head of commercial cell therapy at BMS, described Breyanzi as a "cornerstone" of the company's cell therapy portfolio. He emphasized that the expanded approval offers patients with relapsed or refractory follicular lymphoma (FL) a significant treatment option with the potential for lasting remission through a one-time infusion.

Breyanzi is a CD19-directed CAR-T cell therapy administered intravenously. It works by targeting B cells, binding to the CD19 surface protein, which allows it to proliferate, trigger the secretion of proinflammatory cytokines, and induce cell death in cancer cells. Initially approved in February 2021 for treating relapsed or refractory large B-cell lymphoma, Breyanzi later received approvals for small lymphocytic leukemia and chronic lymphocytic leukemia in March 2024.

The recent label expansion was supported by data from the Phase II TRANSCEND FL study. This single-arm, open-label trial included over 210 patients with relapsed or refractory FL or marginal zone lymphoma. The study results demonstrated that treatment with Breyanzi led to an overall response rate of 95.7%, with a complete response rate of 73.4%. The response to treatment occurred rapidly, with a median onset within one month. At the time of analysis, the median duration of response had not yet been reached, with 80.9% of patients still showing a treatment response after 12 months.

The FDA has another decision pending for Breyanzi, scheduled for May 31, 2024, regarding its use in refractory mantle cell lymphoma.

As with all commercially available CAR-T therapies, Breyanzi includes a boxed warning for the risk of secondary hematological malignancies. In November 2023, the FDA announced that it had detected and was investigating cases of secondary cancers in patients treated with CAR-T therapies. By January 2024, more details were published, revealing the presence of the CAR transgene in three instances of these secondary cancers. Breyanzi’s boxed warning also highlights the risks of cytokine release syndrome and neurologic toxicities.

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