The last couple of months have been a whirlwind of activity with cell and gene therapy. One of the most pivotal moments happened on June 27, 2025, when the FDA announced the elimination of the Risk Evaluation and Mitigation Strategy (REMS) program for the following CAR-T therapies: Abecma (idecabtagene vicleucel), Breyanzi (lisocabtagene maraleucel), Carvykti (ciltacabtagene autoleucel), Kymriah (tisagenlecleucel), Tecartus (brexucabtagene autoleucel), and Yescarta (axicabtagene ciloleucel).
REMS is a safety program that the FDA can require for certain medications with serious safety concerns to help ensure the benefits of the medication outweigh its risks. The FDA has since determined that a REMS is no longer necessary to ensure that the benefits of the autologous CAR-T cell immunotherapies outweigh their risks. These products will continue to be subject to safety monitoring through adverse event reporting requirements. Per the FDA announcement, 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 fifteen (15) years after product administration. Facilities will still be required to go through the certification process with each of the manufacturers.
The elimination of REMS for the CAR-T therapies will result in the following changes:
Removes the requirements that hospitals and their associated clinics that dispense these therapies must be specially certified and have on-site, immediate access to tocilizumab.
Streamlined post-treatment monitoring guidelines:
Daily monitoring for approximately one week, then continued monitoring through two weeks post-infusion (down from four).
Patients will be required to stay near the treatment center for two weeks (down from four).
Patients are to avoid driving for two weeks (down from eight).
According to the Multiple Myeloma Research Foundation, only about 20% of eligible myeloma patients receive CAR-T therapy, largely due to where they live. Patients in rural or underserved areas often face long-distance travel and financial hurdles that put CAR-T out of reach. This sentiment has been voiced by manufacturers such as Bristol Myers Squibb (BMS) who shared only about 2 in 10 eligible patients receive Breyanzi and Abecma, due to the confluence of complex logistical and geographic barriers affecting patients and providers.
In gene therapy news, the FDA has requested a change to Skysona (elivaldogene autotemcel), a lentiviral autologous hematopoietic stem cell (HSC)-based gene therapy, initially approved in 2022 in boys ages 4-17 with early, active cerebral adrenoleukodystrophy (CALD). Myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) were known serious risks for approximately 4% of the patients who received Skysona. Recent reports have shown the risk of MDS and AML to be 15% and time to diagnosis of hematologic malignancy ranges from 14 months to 10 years after Skysona administration. Due to updated information, the FDA has required labeling changes and black box warnings to include new safety information on the increased risk of hematologic malignancy. The revised Indications and Usage restricts the indication to patients without an available human leukocyte antigen (HLA)-matched allogeneic hematopoietic stem cell (allo-HSC) donor. Therefore, Skysona should only be used in CALD patients without suitable alternative treatment options, given the increased risk of hematologic malignancy.
Article by Kathy Clark, RN, BSN, CMCN, RIT, Vice President, Director of Managed Care. For more information about how this may affect your plan, please contact your Summit ReSources care specialist. The following sources were used as reference material for this article:
FDA Removes REMS Requirements for CAR T. https://themmrf.org/mmrf-blogs/fda-removes-rems-requirements-for-car-t/. Accessed 8/13/2025
FDA Eliminates Risk Evaluation and Mitigation Strategies (REMS) for Autologous Chimeric Antigen Receptor CAR T cell Immunotherapies. https://www.fda.gov/news-events/press-announcements/fda-eliminates-risk-evaluation-and-mitigation-strategies-rems-autologous-chimeric-antigen-receptor. Accessed 8/12/2025.
Warkentin PI; Foundation for the Accreditation of Cellular Therapy. Voluntary accreditation of cellular therapies: Foundation for the Accreditation of Cellular Therapy (FACT). Cytotherapy. 2003;5(4):299-305. doi: 10.1080/14653240310002298. PMID: 12944235. Accessed 8/13/2025.