On March 27, 2021, the FDA approved idecabtagene vicleucel (Abecma; Bristol Myers Squibb/Bluebird Bio), a B-cell maturation antigen–directed, genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy, for the treatment of adults with multiple myeloma whose disease did not respond to, or recurred, after ≥4 lines of therapy. Idecabtagene vicleucel is the first cell-based gene therapy approved by the FDA for the treatment of multiple myeloma. The FDA granted idecabtagene vicleucel orphan drug and breakthrough therapy designations.
“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,” said Peter Marks, MD, PhD, Director of the FDA’s Center for Biologics Evaluation and Research.
The FDA approved idecabtagene vicleucel based on results of a multicenter study of 127 patients with relapsed or refractory multiple myeloma after ≥3 lines of therapy. Approximately 88% of the patients had received ≥4 lines of therapy.
Overall, 72% of patients had a response to the study drug, including 28% complete responses. Of the patients with complete responses, 65% had a complete response lasting ≥12 months.
The most common adverse reactions associated with idecabtagene vicleucel include cytokine release syndrome (CRS), infections, fatigue, musculoskeletal pain, and a weakened immune system. Adverse reactions usually appear within the first 1 or 2 weeks after treatment, but some may occur later.
Severe adverse reactions associated with idecabtagene vicleucel include CRS, hemophagocytic lymphohistiocytosis or macrophage activation syndrome, neurologic adverse events, and prolonged cytopenia, all of which can be fatal or life-threatening. Because of such serious adverse reactions, idecabtagene vicleucel is only available through a REMS program. The FDA is also requiring the manufacturer to conduct a postmarketing observational study to validate the long-term safety of the drug.