Abstract
The FDA has approved Bristol Myers Squibb's and bluebird bio's CAR-T cell therapy for treating the blood cancer multiple myeloma, the first cell-based gene therapy approved for this indication and the first B-cell maturation antigen (BCMA)-directed therapy.
Abecma (idecabtagene vicleucel) was approved for patients with multiple myeloma whose cancer has not responded to at least four types of therapy, or whose cancer had returned after previous therapies.
It is a highly personalized autologous therapy, which involves extraction of a patient's T-cells, modification of the T-cells to target the cancer, and re-infusion of these cells back into the patient.
Multiple myeloma affects the plasma cells, a kind of white blood cell that produces antibodies. While CAR-T cell therapies have shown good efficacy in blood cancers compared with solid tumors, until now they have only been approved to treat leukemia and lymphoma. This is the first such therapy to be approved for myeloma treatment and also the first in a new class of CAR T therapies targeting the BCMA protein rather than CD19.
The approval is based on results from a phase II trial, published in the New England Journal of Medicine earlier this month. In the trial, 73% of 128 patients had at least a partial response to the therapy, with 33% having a complete response or achieving remission.
The trial met its primary endpoint of 50% of patients having at least a partial response to Abecma, which is also known as ide-cel. Participants in the trial had a progression free survival time ranging from 5.6-11.6 months in the trial.