FDA Grants Priority Review to Application for Liso-Cel in Second-Line LBCL


The CD19-directed CAR T-cell therapy lisocabtagene maraleucel was granted priority review by the FDA following an application for its use in patients with relapsed or refractory large B-cell lymphoma receiving therapy in the second-line setting.

The FDA has granted priority review to a supplemental biologics license application for lisocabtagene maraleucel (liso-cel; Breyanzi) as treatment for second-line relapsed or refractory large B-cell lymphoma, according to a press release from the agent’s developer Bristol Myers Squibb.1

Data from the randomized phase 3 TRANSFORM trial (NCT03575351) supported the FDA’s decision, in which the CD19-directed CAR T-cell therapy resulted in a statistically significant improvement in event-free survival (EFS) vs standard-of-care (SOC) in this patient population.2

“[Liso-cel] as a differentiated CD19-directed CAR T-cell therapy has already proven to be an important treatment option for patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy and now has the potential to be a new standard of care for patients after failure of first-line therapy, offering significantly improved outcomes beyond the current mainstay of care,” Anne Kerber, senior vice president of Cell Therapy Development at Bristol Myers Squibb, said in a press release. “This acceptance from the FDA brings us one step closer to delivering a practice-changing treatment for primary refractory or relapsed large B-cell lymphoma, making [liso-cel] available to more patients in need, and underscores the advancements we’re making in cell therapy research to transform the lives of patients with difficult-to-treat blood cancers, including lymphoma.”

Results from the trial that were reported at the 2021 American Society of Hematology Annual Meeting & Exposition showed a median EFS of 10.1 months (95% CI, 6.1–not reached [NR]) in the liso-cel arm vs 2.3 months (95% CI, 2.2-4.3) among those treated with SOC (HR, 0.349; 95% CI, 0.229-0.530; P <.0001). Rates of EFS at 6 months were 63.3% (95% CI, 52.0%-74.7%) and 33.4% (95% CI, 23.0%-43.8%), respectively; corresponding rates at 12 months were 44.5% (95% CI, 29.4%-59.6%) and 23.7% (95% CI, 13.4%-34.1%).

The objective response rate with liso-cel was 86% (95% CI, 77.0%-92.3%) vs 48% (95% CI, 37.3%-58.5%) in the SOC arm. The rates of complete response were 66% (95% CI, 55.7%-75.8%) and 39% (95% CI, 29.1%-49.9%), respectively (P <.0001).

The median progression-free survival (PFS) in the liso-cel cohort was 14.8 months (95% CI, 6.6-NR) vs 5.7 months (95% CI, 3.9-9.4) in the SOC cohort (HR, 0.406; 95% CI, 0.250-0.659; P = .0001). Overall survival was not reached in either cohort, but there was a trend toward favorable outcomes in the experimental arm (HR, 0.509; 95% CI, 0.258-1.004; P = .0257).

At the data cut-off for the analysis, the median follow-up time for the liso-cel (n = 67) and SOC (n = 68) arms was 6.2 months. Those included in the analysis were patients who underwent leukapheresis followed by randomization, with the liso-cel arm receiving 100 x 106 CAR T cells and SOC having 3 cycles of salvage chemotherapy followed by high-dose chemotherapy plus autologous stem cell transplantation.

With liso-cel treatment, cytokine release syndrome (CRS) and neurological events (NEs) occurred in 49% and 12% of patients, respectively.

Previously, the FDA issued an approval to liso-cel in the treatment of adult patients with certain types of large B-cell lymphoma following 2 or more prior therapies.

The FDA set a Prescription Drug User Fee Act date for this application of June 24, 2022.


  1. U.S. Food and Drug Administration (FDA) accepts for priority review Bristol Myers Squibb’s supplemental biologics license application for Breyanzi (lisocabtagene maraleucel) as a second-line therapy for relapsed or refractory large B-cell lymphoma. News release. Bristol Myers Squibb. February 17, 2022. Accessed February 17, 2022. https://bit.ly/3sP2LYh
  2. Kamdar M, Solomon SR, Arnason J, et al. Lisocabtagene maraleucel (liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, versus standard of care (SOC) with salvage chemotherapy (CT) followed by autologous stem cell transplantation (ASCT) as second-line (2L) treatment in patients (Pts) with relapsed or refractory (R/R) large B lymphoma (LBCL): results from the randomized phase 3 transform study. Blood. 2021;138(suppl 1):91. doi:10.1182/blood-2021-147913
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