Approval of idecabtagene vicleucel by the U.S. Food and Drug Administration

The U.S. Food and Drug Administration (FDA) has announced the approval of idecabtagene vicleucel (ide-cel) for the treatment of adults with relapsed/refractory multiple myeloma (MM) following four or more previous lines of therapy. Ide-cel is the first B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor (CAR) T-cell therapy to be approved for this indication.

This approval is based on the work done during the phase II KarMMa trial (NCT03361748) in which 33% of patients achieved a complete response or better, and an overall response rate of 73% was achieved. The safety profile of ide-cel is in line with other CAR T-cell therapies, with low-grade neurotoxicity and cytokine release syndrome being common adverse events. Further information on the results of the KarMMa trial will be published on the Multiple Myeloma Hub soon.

Ide-cel is given as a one-off dose of 300−460 × 10⁶ CAR T-cells. The mode of action of ide-cel is to bind to MM cells that express BCMA, which leads to the death of these cells. This CAR T-cell therapy is indicated for patients who have received treatment with an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody and have become refractory to them.

This announcement follows the priority review of the Biologics License Application (BLA) for ide-cel granted in September 2020.