A Bristol Myers Squibb cell therapy made from a patient’s own immune cells has won the FDA’s regulatory nod in multiple myeloma, giving the pharmaceutical giant its second approval of a so-called CAR-T treatment and the first of this type targeting one particular cancer protein.
The FDA’s Friday decision for the BMS drug, idecabtagene vicleucel, covers adults whose cancer has not responded to or has returned after at least four earlier treatments from other types of therapy. BMS, which developed the cell therapy in partnership with Cambridge, Massachusetts-based biotech company bluebird bio, will market the drug under the name “Abecma.”
Multiple myeloma is a blood cancer affecting the plasma cells in the bone marrow. These cancer cells form tumors in the bones of the body, keeping them from making enough healthy blood cells. Multiple myeloma is characterized by periods of remission and relapse. The National Cancer Institute estimates that multiple myeloma accounted for about 1.8% (32,000) of all new U.S. cancer cases last year.
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CAR-T therapies are made in a lengthy and expensive process that results in a therapy personalized to each patient. A patient’s immune cells are harvested and then engineered in a lab to better recognize cancer cells. Those cells are multiplied, then infused back into the patient. The lentiviral vector that is used to engineer the CAR T cells was developed by bluebird bio.
Last month, the FDA approved BMS’s Breyanzi, a CAR-T treatment for diffuse large B-cell lymphoma. That therapy is engineered to target the cancer protein CD19. Abecma is engineered to target a different cancer protein, B-cell maturation antigen (BCMA). Both drugs came to New York-based BMS via its $74 billion acquisition of Celgene.
Though the BMS therapy is the first CAR-T treatment approved to treat multiple myeloma, it’s not the first approved product that addresses BCMA. That distinction belongs to GlaxoSmithKline drug Blenrep, an antibody drug conjugate that the FDA approved last August. Amgen is also developing an antibody drug that targets BCMA. Other companies in the hunt with a BCMA CAR-T therapies include Johnson & Johnson and Novartis.
The FDA based its Abecma decision on a Phase 2 study enrolling 127 patients whose multiple myeloma relapsed or had not responded to at least three earlier lines of treatment. The main goal of the study was to show overall response to the treatment after 24 months. According to the results, 72% of patients showed a partial or complete response to treatment. In 28% of patients, the CAR-T therapy led to a complete response, meaning a disappearance of all signs of the cancer. Within this group of complete responders, 65% maintained that response to treatment for at least one year. Results of the clinical trial were published last month in the New England Journal of Medicine.
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Regulatory approval of Abecma requires BMS to develop a plan that informs physicians and patients about the treatment’s risks. In addition, the agency said that hospitals and clinics that provide Abecma must be certified, and their staff must be trained to recognize and manage the side effects of the treatment. The FDA is also requiring BMS to conduct a post-marketing observational study to further evaluate the long-term safety of Abecma. The approval of Breyanzi came with the same requirements.
Like the other CAR-T treatments that have reached the market, Abecma’s drug label carries a boxed warning that flags the therapy’s dangerous side effects. They include an immune response called cytokine release syndrome; hemophagocytic lymphohistiocytosis and macrophage activation syndrome (HLH/MAS), two potentially fatal syndromes in which immune system malfunction leads to inflammation and organ damage; brain toxicity; and cytopenia, which is a reduction in the number of mature blood cells. In addition to cytokine release syndrome, the most common side effects reported in clinical trials were infections, fatigue, muscle pain, and a weakened immune system.
Approval of Abecma comes later than BMS and bluebird had hoped. Last year, the FDA issued a refusal to file letter, which noted deficiencies in the application and asked the companies for more details on how the therapy is made. No additional clinical testing was required, and the companies resubmitted their application last July.
BMS and bluebird are sharing in the commercialization of Abecma. The therapy, which is intended to be a one-time treatment, carries a wholesale price, before any discounts or rebates, of $419,500.
Photo by Bristol Myers Squibb
