FDA Approves BCMA-Directed Cell Therapy for Multiple Myeloma

The US Food and Drug Administration (FDA) has approved Abecma (idecabtagene vicleucel; ide-cel), the first ever in-class B-cell maturation antigen (BCMA)-directed personalized immune cell therapy delivered as a one-time infusion to treat triple-class exposed patients with multiple myeloma.

The approval, awarded to Bristol Myers Squibb and bluebird bio is indicated for adult patients with relapsed or refractory multiple myeloma after 4 or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.

The Treatment

The treatment will be available with a recommended dose range of 300 to 460 x 106 CAR-positive T cells.

Abecma works by recognizing and binding to BCMA, a protein that is almost universally expressed on cancer cells in multiple myeloma, leading to the death of BCMA-expressing cells. The treatment does come with Boxed Warning regarding Cytokine Release Syndrome (CRS), Neurologic Toxicities (NT), Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome (HLH/MAS), and Prolonged Cytopenia.

“In the KarMMa study, ide-cel elicited rapid responses in the majority of patients, and these deep and durable responses were observed in patients with triple-class exposed and refractory multiple myeloma,” said Nikhil C. Munshi, MD, Associate Director, The Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute, in a statement. “As a treating physician, I often work with patients with relapsed or refractory multiple myeloma who are in critical need of new therapies. Now, with the approval of ide-cel as the first anti-BCMA CAR T cell therapy, we are excited to finally be able to offer patients a new, effective personalized treatment option that is delivered through a single infusion.”

The Condition

Multiple myeloma is an incurable disease characterized by periods of remission and relapse in which the majority of patients experience relapses following initial therapies, while depth and duration of response, as well as survival outcomes decrease with each successive treatment.

Patients who have been exposed to all 3 major drug classes tend to demonstrate poor clinical outcomes with a response rate of 20-30%, a response duration of 2-4 months, and poor overall survival.