FDA Approves First Cell-Based Gene Therapy for MM

On March 27, 2021, the FDA approved idecabtagene vicleucel (Abecma; Celgene), a cell-based gene therapy, for the treatment of patients with multiple myeloma (MM) who have not responded to, or whose disease has returned after, at least 4 prior lines of therapy.

The FDA said idecabtagene vicleucel, a BCMA-directed genetically modified autologous CAR-T therapy, is the first cell-based gene therapy it has approved for the treatment of MM.

“The FDA remains committed to advancing novel treatment options for areas of unmet patient need,” said Peter Marks, MD, PhD, Director of the FDA’s Center for Biologics Evaluation and Research, in a press release.

The approval was based on data from a multi-center study involving 127 patients with relapsed or refractory MM who received at least 3 prior anti-myeloma lines of therapy. About 88% of patients had received 4 or more prior lines of therapy. Overall, 72% of patients partially or completely responded to the treatment. Of those, 28% showed a complete response (CR), and 65% remained in CR for at least 12 months.

The most common severe side effects associated with idecabtagene vicleucel therapy include cytokine release syndrome, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, neurologic toxicity, and prolonged cytopenia. Other common side effects include infections, fatigue, musculoskeletal pain, and a weakened immune system.

“While there is no cure for [MM], 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,” Marks said.Emily Bader

Source: US Food and Drug Administration. FDA Approves First Cell-Based Gene Therapy for Adult Patients with Multiple Myeloma. March 27, 2021. Accessed March 29, 2021.

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