FDA Approves Axi-cel in Relapsed/Refractory Follicular Lymphoma


Data from the phase 2 ZUMA-5 trial supported the approval of axicabtagene ciloleucel, a chimeric antigen receptor T-cell therapy, as a treatment for patients with follicular lymphoma in the third-line setting.

The FDA has granted accelerated approval to the chimeric antigen receptor (CAR) T-cell therapy axicabtagene ciloleucel (Yescarta; axi-cel) for the treatment of adults with relapsed/refractory follicular lymphoma after 2 or more lines of treatment, according to Kite, a Gilead Company, who is responsible for developing the cellular therapy.

The decision follows a breakthrough therapy designation and a priority review and marks the third indication for this CAR T-cell product that was originally approved in 2017.

“Once a follicular lymphoma patient’s disease relapses, the duration of response to care shortens with each round of therapy,” Caron A. Jacobson, MD, MMSc, Medical Director of the Immune Effector Cell Therapy Program at Dana-Farber Cancer Institute and Assistant Professor of Medicine at Harvard Medical School, said in a press release. “Additionally, for follicular [lymphoma] in the third line of therapy, the 5-year survival rate is only 20%, highlighting the urgent need for treatments that offer a real chance for durable remission.”

Data supporting the approval are from the multicenter, single-arm, phase 2 ZUMA-5 trial (NCT03105336), in which investigators sought to evaluate axi-cel in 151 patients with relapsed/refractory indolent non-Hodgkin lymphoma—either follicular or marginal zone lymphoma—following 2 or more lines of therapy.

Patients with relapsed/refractory follicular lymphoma (n = 81) responded to axi-cel at a rate of 91%, with 60% of patients achieving a complete remission. Patients with continued remission at 18 months comprised an estimated 74% of the study population; median response duration was not reached at 14.5 months.

In the safety analysis (n = 146), grade 3 or greater cytokine release syndrome and neurological events, adverse events of concern with CAR T-cell therapies, occurred in 8% and 21%, respectively.

“As we look to bring the hope of survival to more patients in need, today’s FDA decision represents a real step forward in our commitment in hematologic malignancies,” Christi Shaw, Chief Executive Officer of Kite, said in a press release. “Advancing CAR T[-cell] therapies for patients across lymphomas remains a cornerstone of our cell therapy development program, and we are excited about the potential of axi-cel for patients with indolent follicular lymphoma.”


U.S. FDA Approves Yescarta for Relapsed or Refractory Follicular Lymphoma After Two or More Lines of Systemic Therapy. News release. Kite, a Gilead Company. March 5, 2021. Accessed March 5, 2021. https://www.gilead.com/news-and-press/press-room/press-releases/2021/3/us-fda-approves-yescarta-for-relapsed-or-refractory-follicular-lymphoma-after-two-or-more-lines-of-systemic-therapy

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