Lisocabtagene maraleucel receives U.S. FDA approval for adult patients with R/R MCL

On May 30, 2024, it was announced that the U.S. Food and Drug Administration (FDA) had granted approval to lisocabtagene maraleucel (liso-cel), a CD19-directed chimeric antigen receptor T-cell therapy, for the treatment of adult patients with relapsed/refractory mantle cell lymphoma after ≥2 prior lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor. This approval was based on positive results from the phase I TRANSCEND NHL trial (NCT02631044).

The TRANSCEND NHL trial

TRANSCEND NHL is a multicentre, open-label, single-arm phase I study investigating the safety and efficacy of liso-cel in patients with relapsed/refractory B-cell non-Hodgkin lymphoma, including mantle cell lymphoma. Among the evaluable patients with mantle cell lymphoma (n = 68):

• The overall response rate was 85.3% (95% CI, 74.6-92.7).
• The complete response rate was 67.6% (95% CI, 55.2-78.5).
• Durable and rapid responses were observed:

• • median time to response was 1 month (range, 0.7-3.0 months);
  • at a median follow-up of 22.2 months, the duration of response was 13.3 months (95% CI, 6.0-23.3);
  • at 12 months, 51.4% of responders remained in ongoing response; and
  • at 18 months, 38.8% of responders remained in ongoing response.

• The most commonly reported adverse reactions in ≥30% of patients were:
  • cytokine release syndrome;
  • fatigue;
  • musculoskeletal pain;
  • encephalopathy;
  • neutrophil count decrease;
  • white blood cell decrease; and
  • platelet count decrease.

Liso-cel comes with a boxed warning for cytokine release syndrome, neurologic toxicities, and secondary hematologic malignancies.

The U.S. FDA has recommended a liso-cel dose of 90−110 × 10⁶ viable chimeric antigen receptor T-cells.

1. Business Wire. Bristol Myers Squibb’s CAR T cell therapy Breyanzi approved by the U.S. Food and Drug Administration for relapsed or refractory mantle cell lymphoma. https://www.businesswire.com/news/home/20240523741069/en/U.S.-Food-and-Drug-Administration-Approves-Bristol-Myers-Squibb%E2%80%99s-Breyanzi-as-a-New-CAR-T-Cell-Therapy-for-Relapsed-or-Refractory-Mantle-Cell-Lymphoma. Published May 30, 2024. Accessed May 31, 2024.