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Cancer

FDA Approves Label Updates for CAR T-Cell Therapies to Reduce Patient Monitoring Requirements and Remove REMS Programs

Strategic Insights

— 3 min read

The US FDA has approved label updates for lisocabtagene maraleucel (liso-cel; Breyanzi) in large B-cell lymphoma (LBCL) and other lymphomas, and idecabtagene vicleucel (ide-cel; Abecma) in multiple myeloma, to reduce certain patient monitoring requirements and remove the risk evaluation and mitigation strategy (REMS) programs. According to Bristol Myers Squibb, the developer of both agents, driving restrictions were reduced from 8 weeks to 2 weeks after treatment, and the requirement to stay within proximity of a health care facility following infusion was reduced from 4 weeks to 2 weeks. Despite the potential of cell therapy, approximately 2 in 10 eligible patients receive it due to complex logistical and geographic barriers affecting both patients and providers.

Key Takeaways:

  • The FDA has approved label updates for liso-cel and ide-cel to reduce patient monitoring requirements and remove REMS programs.
  • Driving restrictions were reduced from 8 weeks to 2 weeks after treatment for both liso-cel and ide-cel.
  • The requirement to stay within proximity of a health care facility following infusion was reduced from 4 weeks to 2 weeks for both agents.
  • Approximately 2 in 10 eligible patients receive cell therapy due to complex logistical and geographic barriers affecting both patients and providers.
  • The REMS requirement was removed because the FDA deemed the established management guidelines and extensive experience of the medical hematology/oncology community sufficient to diagnose and manage the risks of adverse effects (AEs) without the need for a REMS for CD19- and BCMA-directed autologous CAR T-cell therapies.
  • A retrospective analysis of clinical trial and real-world data from liso-cel revealed that the majority of AEs, such as cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and neurological events (NEs) were not severe and occurred 15 or fewer days after infusion.
  • The events that did occur after day 15 were low-grade and resolved with treatment.
  • In clinical trials, any grade CRS and NEs occurred in 54% and 31% of patients, and grade 3 or higher events occurred in 3% and 10% for liso-cel.
  • For ide-cel, the overall response rate was 72% (95% CI, 62%-81%), with a complete response rate of 28% (95% CI, 19%-38%) in the phase 2 KarMMa trial.
  • Liso-cel was evaluated and approved based on results from the phase 3 TRANSFORM trial, which showed that event-free survival (EFS) was significantly longer with liso-cel vs standard second-line therapy (HR, 0.34; 95% CI, 0.22-0.52; P<0.0001).
  • Ide-cel was evaluated and approved based on results from the phase 2 KarMMa trial.

Statistics:

  • 2 in 10 eligible patients receive cell therapy due to complex logistical and geographic barriers affecting both patients and providers.
  • In clinical trials, any grade CRS and NEs occurred in 54% and 31% of patients, and grade 3 or higher events occurred in 3% and 10% for liso-cel.
  • For ide-cel, the overall response rate was 72% (95% CI, 62%-81%), with a complete response rate of 28% (95% CI, 19%-38%) in the phase 2 KarMMa trial.
  • The median time from infusion to resolution, in clinical trials, was 9 days (range, 2-78) for CRS and 17 days (range, 2-151) with NEs for liso-cel.
  • In the CIBMTR registry, the median time from infusion to resolution was 7 days (range, 1-555) for CRS and 12 days (range, 2-104) for ICANS for liso-cel.

Sources:

1. U.S. Food and Drug Administration approves streamlined patient monitoring requirements and removal of REMS programs within Bristol Myers Squibb's cell therapy labels. News release. Bristol Myers Squibb. June 26, 2025. Accessed June 27, 2025.

2. Kamdar M, Shadman M, Ahmed S, et al. Optimizing post–chimeric antigen receptor (CAR) T cell monitoring: evidence across lisocabtagene maraleucel (liso-cel) pivotal clinical trials and real-world experience. J Clin Oncol. 2025;43(suppl 16):7026. doi:10.1200/JCO.2025.43.16_suppl.7026

3. FDA approves lisocabtagene maraleucel for second-line treatment of large B-cell lymphoma. FDA. June 27, 2022. Accessed June 27, 2025.

4. FDA approves idecabtagene vicleucel for multiple myeloma. FDA. March 29, 2021. Accessed June 27, 2025.

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