FDA set a target action date of March 27, 2021
Ide-cel is the first CAR T cell therapy accepted for regulatory review for multiple myeloma
Bristol Myers Squibb and bluebird bio, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review their Biologics License Application (BLA) for idecabtagene vicleucel (ide-cel; bb2121).
Idecabtagene vicleucel is an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of multiple myeloma. It will be indicated in patients who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021. The submission is based on results from the pivotal Phase 2 KarMMa study evaluating the efficacy and safety of ide-cel in 128 adults with heavily pre-treated and highly refractory multiple myeloma exposed to an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. Results from the study were shared during an oral presentation as part of the American Society of Clinical Oncology 2020 (ASCO20) Virtual Scientific Program.1
The KarMMa study demonstrated an ORR of 73% and median PFS of 8.6 mo; both increased with higher dose.
- ORR 50%, PFS not reported in the 150 × 106 group
- ORR 69%, PFS 5.8 months in the 300 × 106 group
- ORR 82%, PFS 11.3 months in the 450 × 106 group
All subgroups had an ORR ≥50%, including older and high-risk pts.
Most common any-grade (Gr) toxicities were cytopenias (97%) and cytokine release syndrome (CRS; 84%). CRS was mainly Gr 1/2; 5 pts (5%) had Gr 3, 1 had Gr 4, and 1 had Gr 5 (at 300 × 106). Neurotoxicity developed in 23 pts (18%); 4 (3%) Gr 3 and 0 Gr ≥4. Median peak CAR+ T cell expansion occurred at 11 d.
The authors concluded that Ide-cel demonstrated deep, durable responses in heavily pretreated RRMM pts.
References:
1. J Clin Oncol 38: 2020 (suppl; abstr 8503). DOI: 10.1200/JCO.2020.38.15_suppl.8503