FDA Approves First Cell-Based Gene Therapy for Multiple Myeloma
Posted: Tuesday, March 30, 2021
On March 27, the U.S. Food and Drug Administration (FDA) granted approval to the cell-based gene therapy idecabtagene vicleucel (Abecma) to treat adults with multiple myeloma who have no response to, or whose disease has returned after, at least four different types of therapy.
The FDA approval was based on a multicenter study of 127 patients with relapsed and refractory multiple myeloma who received at least three prior therapies. About 88% of patients received four or more prior lines of antimyeloma treatment. Results showed 72% of patients partially or completely responded to the gene therapy. A complete response was observed in 28% of patients. Of the 28%, approximately 65% had a complete response for at least 12 months.
Several side effects classified as severe have been reported with idecabtagene vicleucel treatment. There is a boxed warning for cytokine-release syndrome, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, neurologic toxicity, and prolonged cytopenia.
For full information on treatment safety, visit fda.gov.