FDA removes barriers to CAR-T access in the US

The Risk Evaluation and Mitigation Strategies (REMS) for currently approved BCMA- and CD19-directed autologous chimeric antigen receptor (CAR) T cell immunotherapies have been removed. The Food and Drug Administration (FDA) has determined that a REMS is no longer necessary to ensure that the benefits of these CAR T cell immunotherapies outweigh their risks. It is hoped that the move minimise the burden on the healthcare delivery system of complying with the REMS. The move comes less than a week after Nicole Verdun, a director of the Office of Therapeutic Products at the Center for Biologics Evaluation and Research, and her deputy director Rachael Anatol were both put on administrative leave. Earlier this year, Peter Marks, the former Director of the FDA's Center for Biologics Evaluation and Research (CBER), resigned from his position. Changes to CAR-T product labelling The new ruling affects Abecma (idecabtagene vicleucel), Breyanzi (lisocabtagene maraleucel), Carvykti (ciltacabtagene autoleucel), Kymriah (tisagenlecleucel), Tecartus (brexucabtagene autoleucel), and Yescarta (axicabtagene ciloleucel). Because of the risks of cytokine release syndrome (CRS) and neurological toxicities, since their initial approvals until June 2025, the therapies were available through a restricted programme under a REMS. Now, the FDA has removed the requirement that hospitals and their associated clinics that dispense the above products are specially certified and have on-site, immediate access to tocilizumab. In addition, product labelling was updated to align with REMS elimination and streamline patient monitoring following product administration. Specifically, the FDA has removed the instructions to monitor patients for at least two weeks including daily monitoring for at least one week; to instruct patients to remain within proximity of a healthcare facility for at least two weeks; and to advise patients to avoid driving for two weeks following product administration. The intention is to help improve access to these products, particularly for patients who live in rural areas, while ensuring safe and effective administration to patients who need them. In a statement, the FDA concludes: “Continuous monitoring and assessment of the safety of all biological products, including the CAR T cell immunotherapies, is an FDA priority and we remain committed to informing the public when we learn new information about these products. “The elimination of the REMS for these products does not change FDA requirements for manufacturers to conduct post marketing observational safety studies to assess the risk of secondary malignancies and long-term safety with follow up of patients for 15 years after product administration.” Diana Spencer, Senior Digital Content Editor, DDW