A New Immunotherapy for a Common Lymphoma 

The FDA granted accelerated approval to a CAR T-cell therapy for certain adult patients with follicular lymphoma, a common type of non-Hodgkin lymphoma. 

The U.S. Food and Drug Administration (FDA) granted accelerated approval to axicabtagene ciloleucel (Yescarta) to treat adult patients with relapsed or refractory follicular lymphoma who previously underwent two or more lines of systemic therapy. Axicabtagene ciloleucel is a CAR T-cell therapy, which is a form of immunotherapy that involves extracting and genetically engineering a patient’s T cells and then reinfusing them back into the body to treat the cancer.   

The approval was based on a single-arm, open-label, multicenter trial among patients who had received prior treatment with two or more lines of systemic therapy. Prior to receiving treatment with axicabtagene ciloleucel, patients underwent chemotherapy to deplete their natural T-cells to keep them from interfering with the function of the genetically modified ones.   

There were 81 patients in the primary efficacy analysis group. Among this group, the objective response rate was 91 percent with a complete remission rate of 60 percent and a median time to response of one month. The median duration of response was not reached and the 1-year rate of continued remission was 76.2 percent. A total of 123 patients in the study underwent the T-cell extraction process and among this group, the objective response rate was 89 percent with a complete remission rate of 62 percent. 

Axicabtagene ciloleucel has the potential to cause cytokine release syndrome and neurological toxicities, as reported in a boxed warning in the prescribing information for this therapy. 

Follicular lymphoma gets its name from the follicle-like structures formed by the cancerous cells in the tissues they invade. It is slow-growing and ranks as the second most common form of non-Hodgkin lymphoma. According to federal statistics, in 2021 there were more than 81,000 new cases of non-Hodgkin lymphoma diagnosed in the U.S. and follicular lymphoma was estimated to account for roughly 10-20 percent of them. 

The FDA granted accelerated approval to axicabtagene ciloleucel on March 5, 2021. Accelerated approval means continued approval may be contingent upon a confirmatory trial.