The approval marks the first B-cell maturation antigen-directed genetically modified autologous chimeric antigen receptor T-cell therapy for patients with relapsed or refractory multiple myeloma.
The FDA today announced the approval of idecabtagene vicleucel (ide-cel; sold as Abecma) for the treatment of patients with relapsed or refractory multiple myeloma after at least 4 prior lines of therapy. Ide-cel is the first BCMA (B-cell maturation antigen)-directed chimeric antigen receptor (CAR) T-cell therapy for patients with relapsed or refractory multiple myeloma.1
The approval is based on results from the phase 2 KarMMA trial, which included 128 patients with relapsed or refractory multiple myeloma who had been given at least 3 prior regimens, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.2
At a median follow-up of 13.2 months, 73% of patients receiving ide-cel had a response and 33% had a complete response or better. Median progression-free survival was 8.8 months (95% CI, 5.6-11.6). In addition, 26% of all patients treated tested negative for minimal residual disease (MRD). Of the patients who had a complete response or better, MRD-negative status was confirmed in 79%.
“In the KarMMa study, ide-celelicited rapid responses in the majority of patients, and these deep and durable responses were observed in patients with triple-class exposed and refractory multiple myeloma,” Nikhil C. Munshi, MD, Associate Director, The Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute, said in a statement.3 “As a treating physician, I often work with patients with relapsed or refractory multiple myeloma who are in critical need of new therapies. Now, with the approval of ide-cel as the first anti-BCMA CAR T cell therapy, we are excited to finally be able to offer patients a new, effective personalized treatment option that is delivered through a single infusion.”
Neutropenia, anemia, and thrombocytopenia were common toxic effects of treatment, affecting 91%, 70%, and 63% of patients, respectively. Cytokine release syndrome (CRS) was reported in 84% of patients, with 5% having grade 3 or higher events. No neurotoxic effects higher than grade 3 occurred, but 18% of patients had neurotoxic events.
Because of the risk of CRS and neurologic toxicity, ide-cel is being approved with a risk evaluation and mitigation strategy that requires hospitals and clinics dispensing the drug to be specially certified. Any staff involved in prescribing, dispensing, or administering the drug must be trained to recognize those toxicities and other potential side effects, and patients must be made aware of the potential serious side effects.
The label carries a boxed warning for CRS, hemophagocytic lymphohistiocytosis or macrophage activation syndrome, neurologic toxicity, and prolonged cytopenia. A post-marketing observational study is also required by the FDA to evaluate the long-term safety of ide-cel.
“The FDA remains committed to advancing novel treatment options for areas of unmet patient need,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “While there is no cure for multiple myeloma, the long-term outlook can vary based on the individual’s age and the stage of the condition at the time of diagnosis. Today’s approval provides a new treatment option for patients who have this uncommon type of cancer.”
1. FDA approves first cell-based gene therapy for adult patients with multiple myeloma. News release. FDA; March 27, 2021. Accessed March 27, 2021. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cell-based-gene-therapy-adult-patients-multiple-myeloma
2. Munshi NC, Anderson LD, Shah Nina, et al. Idecabtagene vicleucel in relapsed and refractory multiple myeloma. N Eng J Med. 2021;384(8):705-716. doi:10.1056/NEJMoa2024850
3. U.S. Food and Drug Administration approves Bristol Myers Squibb’s and bluebird bio’s Abecma (idecabtagene vicleucel), the first anti-BCMA CAR T cell therapy for relapsed or refractory multiple myeloma. News release. Bristol Myers Squibb; March 26, 2021. Accessed March 27, 2021. https://news.bms.com/news/corporate-financial/2021/U.S.-Food-and-Drug-Administration-Approves-Bristol-Myers-Squibbs-and-bluebird-bios-Abecma-idecabtagene-vicleucel-the-First-Anti-BCMA-CAR-T-Cell-Therapy-for-Relapsed-or-Refractory-Multiple-Myeloma/default.aspx