The U.S. Food and Drug Administration (FDA) approved idecabtagene vicleucel (ide-cel) for the treatment of adults with relapsed/refractory multiple myeloma (RRMM) following four or more previous treatments, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.
Ide-cel is the first B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor (CAR) T-cell therapy to be approved for this disease.
“CAR T-cell therapies have shown transformational potential for the treatment of hematologic malignancies, and we, with our partners at bluebird bio, are proud to bring the first CAR T-cell therapy to appropriate triple-class–exposed patients with RRMM, offering the chance for durable response,” said Samit Hirawat, MD, chief medical officer of Bristol Myers Squibb, the manufacturer of ide-cel, via a press release.
Approval is supported by results from the KarMMa trial, which enrolled 127 patients with triple-class RRMM. Of 100 patients who received the single-infusion of ide-cel, dosed at 300×106 to 460×106 CAR-positive T-cells, the overall response rate was 72%. A stringent complete response (CR) was achieved in 28% of this cohort (95% confidence interval, 19-38). Median time to response was 30 days, and median response duration was 11 months for the whole cohort and 19 months for those who achieved a CR.
“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 RRMM,” 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.”
The most common adverse events (AEs), occurring in more than 20% of patients, associated with ide-cel include cytokine release syndrome (CRS), infection, fatigue, musculoskeletal pain, hypogammaglobulinemia, diarrhea, nausea, encephalopathy, edema, pyrexia, cough, headache, and decreased appetite. CRS occurred in 85% of patients, with grade 3 or higher cases occurring in 9% of patients. Neurotoxicity occurred in 28% of patients, including grade 3 or higher events in 4%, and resolved in 92% of patients. Fatal AEs occurred in 6% of patients.