Bristol Myers Squibb, bluebird bio announce submission of BLA for Myeloma Drug to USFDA

Princeton & Cambridge: Bristol Myers Squibb and bluebird bio, Inc. has announced the submission of their Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for idecabtagene vicleucel (ide-cel; bb2121), the companies' lead investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.

The submission is based on results from the pivotal Phase 2 KarMMa study. The KarMMa study evaluated the efficacy and safety of ide-cel in heavily pre-treated patients with relapsed and refractory multiple myeloma. Topline data from KarMMa, reported in December 2019, indicated the study met its primary endpoint of overall response rate, and the key secondary endpoint of complete response rate in this patient population treated with ide-cel. The safety results were consistent with those observed in the supportive Phase 1 CRB-401 study, which evaluated the preliminary safety and efficacy of ide-cel. Comprehensive results of the KarMMa study will be presented at a future medical meeting.

BCMA is a protein that is nearly universally expressed on cancer cells in multiple myeloma, making it an important potential target for the treatment of this aggressive blood cancer. Ide-cel is the first CAR T cell therapy submitted for regulatory approval to target this antigen and for multiple myeloma.

Ide-cel was granted Breakthrough Therapy Designation (BTD) by the FDA and PRIority MEdicines (PRIME) designation by the European Medicines Agency for relapsed and refractory multiple myeloma.

Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy.

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