Posted: Wednesday, March 2, 2022
On February 28, the U.S. Food and Drug Administration (FDA) announced that it has approved ciltacabtagene autoleucel (Carvykti) for the treatment of adults with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. Ciltacabtagene autoleucel is a chimeric antigen receptor T-cell therapy that has two B-cell maturation antigen-targeting single domain antibodies.
The approval is based on data from the CARTITUDE-1 study. In this study of 97 patients with relapsed or refractory multiple myeloma, 98% responded to a one-time treatment with ciltacabtagene autoleucel. Notably, 78% of the patients achieving this level of response (n = 76) experienced a stringent complete response. At a median of 18 months of follow-up, the median duration of response was 21.8 months.
Ciltacabtagene autoleucel is available only through a restricted program under a Risk Evaluation and Mitigation Strategy. The safety information for this agent includes a Boxed Warning regarding cytokine-release syndrome, immune effector cell–associated neurotoxicity syndrome, parkinsonism and Guillain-Barré syndrome, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, and prolonged and/or recurrent cytopenias. Its longer-term efficacy and safety profile are currently being assessed.