FDA Approves Idecabtagene Vicleucel as the First Cell-Based Gene Therapy for Adult Patients With Multiple Myeloma

March 30, 2021

On March 27, 2021, the U.S. Food and Drug Administration (FDA) approved (https://www.fda.gov/news-events/press-announcements/fda-approves-first-cell-based-gene-therapy-adult-patients-multiple-myeloma) idecabtagene vicleucel (Abecma), for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. This is the first FDA-approved cell-based gene therapy for multiple myeloma.

FDA Approves Idecabtagene Vicleucel as the First Cell-Based Gene Therapy for Adult Patients With Multiple Myeloma

Safety and efficacy were established in a multicenter study of 127 patients with relapsed or refractory myeloma who received at least three prior antimyeloma lines of therapy. About 88% of patients in the study group had received four or more prior lines of antimyeloma therapy. Overall, 72% of patients partially or completely responded to the treatment. Of those studied, 28% of patients showed complete response, and 65% of those sustained it for at least 12 months. 

The label (https://www.fda.gov/media/147055/download) carries a black-box warning for cytokine release syndrome (CRS), hemophagocytic lymphohistiocytosis or macrophage activation syndrome, neurologic toxicity, and prolonged cytopenia. The most common adverse events in the clinical trial included CRS, infections, fatigue, musculoskeletal pain, and hypogammaglobulinemia.

Because of the risk of CRS and neurologic toxicities, idecabtagene vicleucel is being approved with a risk evaluation and mitigation strategy that includes elements to ensure safe use. Of particular relevance to oncology nurses, FDA requires hospitals and clinics that dispense idecabtagene vicleucel to be specially certified and staff involved in its prescribing, dispensing, or administering to be trained to recognize and manage CRS, nervous system toxicities, and other adverse events. Also, patients must be informed of the potential adverse events and of the importance of promptly returning to the treatment site if adverse events develop after receiving idecabtagene vicleucel.

To further evaluate the long-term safety, the manufacturer must also conduct a post-marketing observational study and report the results to FDA.

FDA granted the application orphan drug (https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/orphan-drug-designation-disease-considerations) and breakthrough therapy (https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy) designations. A description of FDA expedited programs is in the Guidance for Industry—Expedited Programs for Serious Conditions—Drugs and Biologics (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics)

Healthcare professionals should report all serious adverse events they suspect are associated with the use of any medicine or device to FDA’s MedWatch Reporting System (https://www.accessdata.fda.gov/scripts/medwatch/index.cfm) or by calling 800-FDA-1088.  

For assistance with single-patient oncology investigational new drug applications, contact OCE’s Project Facilitate (https://www.fda.gov/about-fda/oncology-center-excellence/project-facilitate) at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov (mailto:OncProjectFacilitate@fda.hhs.gov)

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