The FDA has granted priority review to the biologics license application of idecabtagene vicleucel (ide-cel), for the treatment of adult patients with MM who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody.
Ide-cel, formerly known as bb2121, is an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T-cell therapy.
The FDA’s acceptance for regulatory review is based on results from the phase II KarMMa study, which were presented as part of EHA25 Virtual, the 25th Annual Congress of the European Hematology Association.
In this open-label, single-arm study of 128 patients with triple-class–exposed MM, ide-cel was administered at target doses of 150 to 450×106 cells/kg. Ninety-three patients (73%) responded to treatment with ide-cel, including 26% of patients who became negative for measurable residual disease. Median progression-free survival was 8.8 months, and the median duration of response was 10.7 months among all responders. The most common any-grade AEs included cytopenias (97%) and cytokine release syndrome (84%). Twenty-three patients (18%) developed neurotoxicity.
Ide-cel was granted breakthrough therapy designation by the FDA and has been granted PRIME (PRIority MEdicines) designation by the E.U.’s European Medicines Agency.