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The United States Food and Drug Administration has approved Abecma (idecabtagene vicleucel), a cellular gene therapy to treat adult patients with multiple myeloma who have not responded or whose disease has come back after at least four lines of treatment ( different types). . Abecma is the first FDA-approved cell gene therapy for the treatment of multiple myeloma.
“The FDA remains committed to advancing new treatment options for areas where patient needs are not being met,” said Peter Marks, MD, Ph.D., director of the Center for Clinical Assessment and Research. FDA biologics. “While there is no cure for multiple myeloma, the long-term outlook may vary depending on the age of the individual and the stage of the disease at the time of diagnosis. Today’s approval offers a new treatment option for patients with this rare type of cancer. ”
Multiple myeloma is a rare type of cancer of the blood in which abnormal plasma cells build up in the bone marrow and form tumors in many bones of the body. This disease prevents the bone marrow from making enough healthy blood cells, which can lead to low blood counts. Myeloma can also damage bones and kidneys and weaken the immune system. The exact cause of multiple myeloma is unknown. According to the National Cancer Institute, myeloma accounted for about 1.8% (32,000) of all new cancer cases in the United States in 2020.
Abecma is an autologous chimeric antigen receptor (CAR) T cell therapy directed by B cell maturing antigen (BCMA). Each dose of Abecma is a personalized treatment created using a patient’s own T cells, which are a type of white blood cell, to help fight myeloma. The patient’s T cells are collected and genetically engineered to include a new gene that facilitates targeting and destruction of myeloma cells. Once the cells are changed, they are reinjected into the patient.
The safety and efficacy of Abecma have been established in a multicenter study in 127 patients with relapsed myeloma (myeloma that comes back after treatment ends) and refractory myeloma (myeloma that does not respond to treatment), which have received at least three lines of anti-myeloma therapy. . About 88% of the patients in the study group had received at least four lines of anti-myeloma treatment. Overall, 72% of patients partially or completely responded to treatment. Of those studied, 28% of patients experienced a complete response – or all signs of multiple myeloma disappeared – to Abecma, and 65% of this group remained in complete response to treatment for at least 12 months.
Treatment with Abecma can cause serious side effects. The label carries a boxed warning for cytokine release syndrome (CRS), hemophagocytic lymphohistiocytosis / macrophage activation syndrome (HLH / MAS), neurological toxicity and prolonged cytopenia, all of which can be fatal or potentially fatal. . CRS and HLH / MAS are systemic responses to activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and prolonged cytopenia is a drop in the number of a certain type of blood cells for a long time. The most common side effects of Abecma include CRS, infections, fatigue, musculoskeletal pain, and a weakened immune system. Side effects from treatment usually appear within the first one to two weeks after treatment, but some side effects may occur later. Patients with multiple myeloma should consult their healthcare professionals to determine if Abecma is an appropriate treatment for them.
Due to the risk of CRS and neurological toxicities, Abecma is in the process of being approved with a risk assessment and mitigation strategy that includes elements to ensure safe use. The FDA requires that hospitals and associated clinics that dispense Abecma be specially certified and that personnel involved in prescribing, dispensing or administering Abecma be trained to recognize and manage CRS and nervous system toxicities and other side effects of Abecma. In addition, patients should be informed of the potential serious side effects and the importance of returning to the treatment site promptly if any side effects develop after receiving Abecma.
To further assess long-term safety, the FDA is also asking the manufacturer to conduct a post-market observational study involving patients treated with Abecma.
Abecma has obtained Orphan Drug and Breakthrough Therapy designation by the FDA. The orphan drug designation provides incentives to help and encourage drug development for rare diseases. The designation of breakthrough therapy is a process designed to accelerate the development and review of drugs to treat serious disease and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on one or more parameters. clinically significant. The breakthrough therapy designation was granted based on the sustained responses seen in patients with relapsed and refractory myeloma.
Drugs approved under accelerated programs, such as the Breakthrough Therapy designation, are subject to the same approval standards as all other FDA approvals.
The FDA granted Abecma approval to Celgene Corporation, a Bristol Myers Squibb company.
The FDA, an agency of the US Department of Health and Human Services, protects public health by ensuring the safety, efficacy, and safety of human and veterinary drugs, vaccines and other biologicals for human use, and medical devices . The agency is also responsible for the safety and security of the food supply, cosmetics, dietary supplements, products that emit electronic radiation, and the regulation of tobacco products.