麻豆传媒

The FDA approved the first .

The approval of idecabtagene vicleucel (ide-cel, Abecma) pertains to patients who progressed on or did not respond to at least four prior lines of therapy, including an immunomodulator, a proteasome inhibitor, and an anti-CD38 antibody.

A form of chimeric antigen receptor (CAR) T-cell therapy, idecabtagene vicleucel is the first agent in the class to target B-cell maturation antigen (BCMA).

"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," Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research, said in the agency's announcement on Friday. "Today's approval provides a new treatment option for patients who have this uncommon type of cancer."

BCMA is expressed by almost all myeloma cells. Ide-cel binds to BCMA-expressing cells, leading to the cells' death.

Primary supporting data for the approval came from the phase II KarMMa trial involving 127 patients with relapsed/refractory myeloma, 100 of whom could be evaluated for response. The results showed an overall response rate of 72% and stringent complete responses (sCR) in 28% of patients. The median time to response was 30 days, and the median duration of response was 11 months, increasing to 19 months for patients who achieved sCR.

Low-grade cytokine release syndrome (CRS) occurred in 85% of patients, and grade ≥3 CRS occurred in 9% of patients. Neurotoxicity occurred in 28% of patients, reaching grade ≥3 severity in 4% of patients.

Hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS) occurred in 4% of patients, one of whom died of multiorgan HLH/MAS with CRS. A second patient died of bronchopulmonary aspergillosis with HLH/MAS as a contributing factor.

"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," principal investigator Nikhil C. Munshi, MD, of Dana-Farber Cancer Institute, said in a from drugmakers Bristol Myers Squibb and bluebird bio.

"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," Munshi said.

Because of the risk of CRS, neurotoxicity, and HLH/MAS, approval of ide-cel includes boxed warnings about the side effects and requires a risk evaluation and mitigation strategy.

Comment