Abecma has become the first cell-based gene therapy approved by the U.S. Food and Drug Administration for the treatment of multiple myeloma, an uncommon type of cancer.
The FDA granted approval of Abecma to Celgene Corp., affiliated to Bristol Myers Squibb (BMY). Abecma is being jointly developed and commercialized in the U.S. as part of an agreement between Bristol Myers Squibb and bluebird bio Inc. (BLUE).
In a statement, the FDA noted that Abecma (idecabtagene vicleucel) can now be used to treat adult patients with multiple myeloma who have not responded to, or whose disease has returned after, at least four prior lines or different types of therapy.
Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, said, "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."
Multiple myeloma is an uncommon type of blood cancer in which abnormal plasma cells build up in the bone marrow and form tumors in many bones of the body.
Abecma is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. The approval was given after the safety and efficacy of Abecma were established in a multicenter study of 127 patients with relapsed myeloma and refractory myeloma, who received at least three prior antimyeloma lines of therapy.
In the trial, 72% of patients partially or completely responded to the treatment. Of those studied, 28% of patients showed complete response or disappearance of all signs of multiple myeloma to Abecma, and 65% of this group remained in complete response to the treatment for at least 12 months.
The FDA noted that Abecma is being approved with a risk evaluation and mitigation strategy which includes elements to assure safe use.
The agency warned that treatment with Abecma may cause severe side effects, including cytokine release syndrome or CRS, neurologic toxicity, and prolonged cytopenia,among others, all of which can be fatal or life-threatening.
According to the agency, hospitals and their associated clinics that dispense Abecma are required to be specially certified. Staff involved in the prescribing, dispensing or administering of Abecma should be trained to recognize and manage CRS and nervous system toxicities and other side effects of Abecma.
The FDA is also requiring the manufacturer to conduct a post-marketing observational study involving patients treated with Abecma to further evaluate the long-term safety.
Abecma was granted Orphan Drug and Breakthrough Therapy designations by the FDA.
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