The U.S. Food and Drug Administration has approved significant label updates for Bristol Myers Squibb's two CAR T cell therapies, marking a pivotal step toward expanding patient access to these transformative treatments. The updates for Breyanzi (lisocabtagene maraleucel) and Abecma (idecabtagene vicleucel) reduce monitoring requirements and eliminate Risk Evaluation and Mitigation Strategy (REMS) programs that have historically limited treatment accessibility.
Reduced Monitoring Requirements Address Access Barriers
The FDA-approved changes directly target logistical barriers that have prevented many eligible patients from receiving CAR T cell therapy. Key modifications include reducing driving restrictions from 8 weeks to 2 weeks post-treatment and decreasing the requirement for patients to remain within proximity of healthcare facilities from 4 weeks to 2 weeks following infusion.
These prolonged requirements had posed significant burdens on healthcare delivery systems and patients, particularly those living far from certified cell therapy treatment centers. According to Bristol Myers Squibb, only about 2 in 10 eligible patients currently receive cell therapy treatment due to complex logistical and geographic barriers.
"CAR T cell therapy is a transformational, potentially life-saving option for patients living with blood cancers, and we are working to challenge current practices, assumptions and barriers that limit access," said Lynelle B. Hoch, president of Bristol Myers Squibb's Cell Therapy Organization.
REMS Program Removal Reflects Safety Confidence
The FDA's decision to remove REMS requirements from both product labels represents a significant regulatory milestone. REMS programs are typically required to help mitigate known or potential risks associated with new drugs or therapies. The FDA determined that established management guidelines and the extensive experience of the medical hematology/oncology community are sufficient to diagnose and manage risks of side effects, including cytokine release syndrome (CRS) and neurologic toxicities, without REMS for the class of CD19- and BCMA-directed autologous CAR T cell therapies.
This change is expected to accelerate cell therapy adoption in community center settings, further expanding geographic access to treatment.
Real-World Evidence Supports Safety Profile
The label updates reflect a growing body of clinical and real-world evidence supporting the favorable efficacy and safety profile of CAR T cell therapy. More than 30,000 patients have been treated with CAR T cell therapy to date, with recent studies, including an analysis Bristol Myers Squibb presented at the ASCO Annual Meeting, demonstrating that the vast majority of serious adverse events occur within the first two weeks of infusion.
In clinical trials of Breyanzi involving 702 patients with non-Hodgkin lymphoma, CRS occurred in 54% of patients, with Grade 3 or higher CRS in 3.2% of patients. The median time to onset was 5 days, and CRS resolved in 98% of patients with a median duration of 5 days.
For Abecma, among 349 patients receiving treatment for relapsed refractory multiple myeloma in the KarMMa and KarMMa-3 studies, CRS occurred in 89% of patients, including Grade 3 or higher CRS in 7% of patients. The median time-to-onset was 1 day, with a median duration of 5 days.
Expanding Treatment Center Network
Following the announcement, Bristol Myers Squibb will work with more than 150 treatment centers currently approved to administer Breyanzi and Abecma to implement the REMS program removal. The company is simultaneously focused on expanding the geographic footprint of cell therapy by adding community cancer centers nationwide to administer treatments closer to patients.
"Living with blood cancer is challenging, but patients and their loved ones still need to maintain jobs, take care of families, and plan for the future," said Sally Werner, chief executive officer of Cancer Support Community. "Today's announcement reduces some of the most onerous requirements that may have previously discouraged patients, particularly those who live far from a treatment center, from seeking the potentially transformational effects of cell therapy."
Therapeutic Indications and Safety Profile
Breyanzi is indicated for treating large B-cell lymphoma, chronic lymphocytic leukemia, small lymphocytic lymphoma, follicular lymphoma, and mantle cell lymphoma in various clinical settings. Abecma is approved for adult patients with relapsed or refractory multiple myeloma after two or more prior lines of therapy.
Both therapies carry boxed warnings for cytokine release syndrome, neurologic toxicities, and secondary hematological malignancies. The most common adverse reactions for Breyanzi in large B-cell lymphoma include fever, cytokine release syndrome, fatigue, musculoskeletal pain, and nausea. For Abecma, the most common nonlaboratory adverse reactions include pyrexia, CRS, hypogammaglobulinemia, infections, musculoskeletal pain, and fatigue.
Bristol Myers Squibb remains the only company with two approved CAR T cell therapies targeting distinct antigens, available in major markets worldwide. The company's vision encompasses treating hundreds of thousands of patients with cell therapy's transformational potential across blood cancers and new frontiers, including autoimmune diseases.
