Abecma Approved to Treat Multiple Myeloma

March 26, 2021 – The U.S. FDA has approved Abecma (idecabtagene vicleucel), manufactured by Bristol Myers Squibb and bluebird bio, Inc., to treat relapsed or refractory multiple myeloma (RRMM) in adults who have received at least four prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. It is the first chimeric antigen receptor T- cell (CAR-T) therapy to receive FDA approval to treat multiple myeloma.

Multiple myeloma is a cancer of the plasma cells – a type of white blood cell responsible for creating antibodies to fight infection. The cancerous plasma cells begin to produce an abnormal protein that can harm healthy tissues in the body. They also crowd out healthy cells and weaken the immune system by leaving it unable to properly fight off infections. Relapsed or refractory multiple myeloma is cancer that has either returned following treatment or that doesn’t respond to treatment. The prognosis for patients who have RRMM is often poor, with low response rates to treatment and low survival rates.

In a clinical trial, Abecma delivered an overall response rate of 72% with 28% of patients having a stringent complete response (sCR). The median time to response was 30 days, and the median duration of response was 11 months. For patients who had an sCR, median duration of response was 19 months.

Abecma is a one-time therapy created using the patient’s own immune cells. Healthcare professionals collect a donation of T cells from the patient, then send them to a facility that specializes in modifying the cells to seek out a protein produced by multiple myeloma cancer cells called BCMA, or B-cell maturation antigen. The modified T cells are then infused into the patient and will use the presence of BCMA to target and destroy cancer cells.

A black box warning cautions that treatment with Abecma carries a risk of cytokine release syndrome, neurologic toxicities, hemophagocytic lymphohistiocytosis, macrophage activation syndrome, and prolonged cytopenia with bleeding and infection. These reactions can be life-threatening and may be fatal. Abecma is available only through a Risk Evaluation and Mitigation Strategy (REMS) program.

Bristol Myers Squibb and bluebird bio plan to launch Abecma at a wholesale acquisition cost (WAC) of $419,500 per one-time treatment.