The U.S. Food and Drug Administration (FDA) has approved idecabtagene vicleucel (ide-cel) as the first B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T-cell therapy for the treatment of adult patients with relapsed or refractory multiple myeloma (MM) who have received four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.
Ide-cel, a personalized immune cell therapy, has been approved as a one-time infusion with a recommended dose range of 300 to 460×106 CAR-positive T cells. This decision is based on data from the phase II KarMMa trial, which enrolled 127 patients with relapsed or refractory MM. Among the efficacy-evaluable population of 100 patients, the overall response rate was 72%, with 28% achieving a stringent complete response (sCR). The median time to response was 30 days (range = 15 – 88 days) and the median duration of response was 11 months for all patients who responded and 19 months for the 28 patients who achieved an sCR.
“In the KarMMa study, ide-cel elicited 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,” said Nikhil C. Munshi, MD, associate director of the Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute. “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.”
Serious adverse events (AEs) occurred in 67% of patients, and the most common (occurring in ≥5% of patients) included cytokine release syndrome (CRS), general physical health deterioration, pneumonia, infections, viral infections, sepsis, and febrile neutropenia. The most common grade 3 or higher AEs were febrile neutropenia and infections. Six percent of patients experienced fatal AEs.
This indication carries boxed warnings for CRS, neurologic toxicities, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, and prolonged cytopenia.