Priority review for ide-cel granted by the FDA

It has been announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application for idecabtagene vicleucel (ide-cel, bb2121) for use in adult patients with triple-class refractory multiple myeloma (MM). The Prescription Drug User Fee Act date has been set for March 27, 2021.¹

This decision comes after the results of the KarMMa phase II trial (NCT03361748). The MM Hub spoke to Nikhil Munshi, Dana-Farber Cancer Institute, Boston, US, about recent updates from this trial in a podcast, which can be found below.

The KarMMa trial included 128 patients with triple-class refractory MM and had a median of 13.3 months follow-up. Within this trial, the primary endpoint was overall response rate (ORR), and complete response (CR) was a secondary endpoint. The target dose was 450 × 10⁶ cells infused. At this dose, ORR was 82%, with close to 40% of patients achieving CR. In terms of adverse events, the most common were Grade 3 neutropenia in 89% of patients, and any Grade cytokine release syndrome in 84% of patients. There were five deaths (4%) during the trial, two from disease progression and three associated with adverse events.²

Ide-cel is an anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell immunotherapy. Its indication, triple-class refractory MM, describes patients who are refractory to a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. Ide-cel uses 4-1BB as a costimulatory domain and CD3ζ as a T-cell activation domain.

The FDA previously granted ide-cel a breakthrough therapy designation, and the European Medicines Agency also granted priority medicines (PRIME) designation for the treatment of patients with relapsed/refractory MM.