FDA Approves Updated Labels on CAR T-Cell Therapies, Eliminating REMS
FDA updates CAR T-cell therapy labels, easing monitoring requirements and expanding access for eligible patients in oncology.
US FDA
- The FDA has approved label updates for all currently approved autologous chimeric antigen receptor (CAR) T-cell therapies, significantly reducing patient monitoring requirements and eliminating their respective Risk Evaluation and Mitigation Strategy (REMS) programs.
- These changes reflect accumulating real-world evidence demonstrating the predictability and manageability of key toxicities within the first 2 weeks postinfusion, making treatment more accessible.
- The streamlined requirements aim to alleviate logistical and geographical barriers, expanding its reach to more eligible patients.
The FDA has announced label updates for approved autologous BCMA- and CD19-directed CAR T-cell therapies. These include:
- idecabtagene vicleucel (ide-cel; Abecma)
- lisocabtagene maraleucel (liso-cel; Breyanzi)
- ciltacabtagene autoleucel (cilta-cel; Carvytki)
- tisagenlecleucel (tisa-cel; Kymriah)
- brexucabtagene autoleucel (brexu-cel; Tecartus)
- axicabtagene ciloleucel (axi-cel; Yescarta)
These revisions streamline patient monitoring protocols and, crucially, eliminate the REMS programs previously associated with these agents.
The decision by the FDA stems from a growing body of clinical and real-world evidence that underscores the favorable efficacy and safety profiles of CAR T-cell therapies. While these therapies have demonstrated transformative potential in refractory hematologic malignancies, their complex administration and strict monitoring requirements have historically presented considerable logistical and geographical hurdles, leading to only about 2 in 10 eligible patients receiving this treatment.
Key changes across both CAR T-cell therapy labels include driving restrictions that are reduced from 8 weeks to 2 weeks posttreatment, and a requirement to stay within proximity of a certified healthcare facility following infusion reduced from 4 weeks to 2 weeks.
Additionally, the label change allows for the removal of the REMS requirement from each product label. REMS programs are typically mandated for drugs or therapies with known or potential serious risks to ensure the benefits outweigh the risks. However, the FDA has now determined that the extensive experience gained by the medical hematology/oncology community in diagnosing and managing these toxicities, combined with established clinical management guidelines, is sufficient to ensure patient safety without the need for a separate REMS program for the class of CD19- and BCMA-directed autologous CAR T-cell therapies.
This decision reflects a development of understanding regarding CAR T-cell associated toxicities. While cytokine release syndrome and neurologic toxicities remain critical considerations, the oncology community has developed robust protocols for their recognition, grading, and management, including the timely administration of supportive care, tocilizumab (Actemra), and corticosteroids.
Ultimately, the removal of REMS from the label is expected to simplify the administrative burden on treatment centers, thereby facilitating the expansion of CAR T-cell therapy into community cancer centers. This will hopefully allow more patients to receive treatment closer to home and reduce the need for extensive travel and prolonged stays away from their support networks.
