NEW YORK – Bristol Myers Squibb and Bluebird Bio said on Wednesday that they submitted a biologics license application to the US Food and Drug Administration for their B-cell maturation antigen-directed CAR T-cell immunotherapy idecabtagene vicleucel.
The therapy is being developed as a treatment for adult patients with relapsed or refractory multiple myeloma, a cancer that occurs in the plasma cells.
The companies originally submitted a BLA for the CAR T-cell therapy to the FDA in March, but the agency requested additional information. In this new submission, the companies said they've addressed the agency's requests, and have provided additional details on the chemistry, manufacturing, and controls used in the pivotal Phase II KarMMa study. That trial is evaluating the efficacy and safety of idecabtagene vicleucel in relapsed and refractory multiple myeloma patients exposed to an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody.
Idecabtagene vicleucel demonstrated deep and durable responses in this patient population, with an overall response rate of 73 percent and a median progression-free survival of 8.6 months at a median follow up of 11.3 months. Additionally, only 5 percent of patients reported a grade 3 or higher adverse event due to treatment. CAR T cells were detected in 59 percent of patients at six months and in 36 percent of patients at 12 months.
BMS and Bluebird Bio have a co-development, co-promotion, and profit-sharing agreement for idecabtagene vicleucel. The European Medicines Agency is also evaluating the CAR T-cell therapy on an accelerated time frame.