FDA Approves First CAR-T Cell Therapy for Multiple Myeloma

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Abecma is the first cell-based gene therapy approved for the treatment of patients with relapsed and refractory multiple myeloma who did not respond to at least four treatments, or whose disease returned after those four lines of therapy.

The Food and Drug Administration (FDA) approved the cell-based gene therapy Abecma (idecabtagene vicleucel) for the treatment of adults with multiple myeloma who did not respond to at least four lines of therapy or whose disease returned after the same number of therapies.

This is the first approval of a cell-based gene therapy by the FDA for the treatment of multiple myeloma, according to an FDA news release. Approximately 1.8% of new cancer cases in the U.S. in 2020 were associated with myeloma, although the exact cause of multiple myeloma is not known.

Abecma is a cell-based gene therapy that is customized with a patient’s own T cells. Once collected, a patient’s T cells are genetically modified with a gene that targets and kills myeloma cells. These cells are then infused back into the patient.

“CAR-T cell therapies have shown transformational potential for the treatment of hematologic malignancies, and we, with our partners at bluebird bio, are proud to bring the first CAR-T cell therapy to appropriate triple-class exposed patients with relapsed or refractory multiple myeloma, offering the chance for durable response,” said Dr. Samit Hirawat, chief medical officer of Bristol Myers Squibb, in a news release from the company.

“Our journey to today’s approval of Abecma started nearly a decade ago with pioneering research at bluebird bio and has been driven ever since by our mission to provide patients with multiple myeloma a new approach to fight this relentless disease,” said Nick Leschly, CEO of bluebird bio, in the company’s press release.

Abecma’s safety and efficacy were assessed in a study of 127 patients with relapsed and refractory myeloma who were previously treated with at least three lines of therapy. Most patients in this study (72%) either partially or completely responded to the treatment, of whom 28% showed complete response to Abecma. This effect persisted for at least 12 months in 65% of these patients.

“As a treating physician, I often work with patients with relapsed or refractory multiple myeloma who are in critical need of new therapies,” said Dr. Nikhil C. Munshi, associate director of The Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute in Boston, in the company release. “Now, with the approval of (Abecma) 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 FDA remains committed to advancing novel treatment options for areas of unmet patient need,” said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, in the agency’s release. “While there is no cure for multiple myeloma, the long-term outlook can vary based on the individual’s age and the stage of the condition at the time of diagnosis. Today’s approval provides a new treatment option for patients who have this uncommon type of cancer.”

Abecma has been shown to cause several side effects, the most common of which are infections, cytokine release syndrome, fatigue, a weakened immune system and musculoskeletal pain. These side effects can occur within one to two weeks after treatment, although some may occur later after treatment.

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