Bristol Myers Squibb (NYSE: BMY) and bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has approved Abecma (idecabtagene vicleucel; ide-cel) as the first B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy 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. Abecma is a personalized immune cell therapy approved as a one-time infusion with a recommended dose range of 300 to 460 x 106 CAR-positive T cells.1 As an anti-BCMA CAR T cell therapy, Abecma recognizes and binds to BCMA, a protein that is nearly universally expressed on cancer cells in multiple myeloma, leading to the death of BCMA-expressing cells.2 Please see the Important Safety Information section below, including Boxed WARNINGS for Abecma regarding Cytokine Release Syndrome (CRS), Neurologic Toxicities (NT), Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome (HLH/MAS), and Prolonged Cytopenia.
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(Photo: Business Wire)
(Photo: Business Wire)
“CAR T cell therapies have shown transformational potential for the treatment of hematologic malignancies, and we, with our partners at bluebird bio, are proud to bring the first CAR T cell therapy to appropriate triple-class exposed patients with relapsed or refractory multiple myeloma, offering the chance for durable response,” said Samit Hirawat, M.D., chief medical officer, Bristol Myers Squibb. “Bristol Myers Squibb is now the only company with two approved CAR T cell therapies with distinct targets of CD19 and BCMA. As our second FDA-approved CAR T cell therapy, Abecma underscores our commitment to deliver on the promise of cell therapies for patients who are battling aggressive and advanced blood cancers with limited effective treatment options.”
“Our journey to today’s approval of Abecma started nearly a decade ago with pioneering research at bluebird bio and has been driven ever since by our mission to provide patients with multiple myeloma a new approach to fight this relentless disease. This achievement would not have been possible without all of the patients, caregivers, investigators and healthcare staff who participated in our clinical studies, as well as the tremendous collaboration with the FDA,” said Nick Leschly, chief bluebird, bluebird bio. “Today’s announcement represents an important milestone for bluebird bio, marking both our first approved treatment in oncology and our first approved treatment in the United States.”
Despite advances in treatment, multiple myeloma remains an incurable disease characterized by periods of remission and relapse.3 Most patients experience relapse following initial therapies, and depth and duration of response as well as survival outcomes decrease with each successive treatment.4-9 Patients with relapsed or refractory multiple myeloma that have been exposed to all three major drug classes (triple-class exposed), including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, tend to demonstrate poor clinical outcomes with very low response rates (20% to 30%), short duration of response (2 to 4 months) and poor survival.5,10,11,12
“In the KarMMa study, ide-celelicited 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,” said Nikhil C. Munshi, M.D., Associate Director, The Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute, Boston, Massachusetts. “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-celas 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.”
A network has been created to support rapid and dependable manufacturing of Abecma and ensure capacity to accommodate patient demand. Abecma will be manufactured for each individual patient using the patient’s own T cells at Bristol Myers Squibb’s state-of-the-art cellular immunotherapy manufacturing facility in Summit, New Jersey. The lentiviral vector, which is used to engineer the CAR T cells, was developed by bluebird bio. Abecma patients, caregivers and physician teams can access relevant information, manufacturing updates and patient and caregiver support through Cell Therapy 360, a digital service platform provided to optimize the Abecma patient and physician treatment experience. Various programs and resources will also be offered to help address the needs of patients and caregivers and provide support that allows for access to therapies, including Abecma. Due to the specialized nature of administering cell therapy, Abecma will be available at certified treatment centers throughout the country. A Risk Evaluation and Mitigation Strategy (REMS) program will be implemented at certified centers to support appropriate use of Abecma including training on the management of cytokine release syndrome and neurologic toxicities.
Abecma is being jointly developed and commercialized in the U.S. as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio.