- For immediate release:
The U.S. Food and Drug Administration has approved Abecma (idecabtagene vicleucel), a cell-based gene to respond to adult patients with multiple myeloma who have not responded, or whose disease has returned, at least four lines in advance (different types) of treatment. . Abecma is the first cell-based gene approved by the FDA for the treatment of multiple myeloma.
“The FDA remains committed to advancing modern treatment options for areas where patients are no longer needed,” said Peter Marks, MD, Ph.D., director of the FDA’s Center for Biological Assessment and Research. “While there is no cure for multiple myeloma, the long-term prognosis can vary depending on the person’s age and the stage of the condition at the time of diagnosis. Today ‘s agreement provides a new treatment option for patients with this rare type of cancer. ”
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. This disease keeps bone marrow from making enough healthy blood cells, which can lead to a low blood count. Myeloma can also damage the bones and kidneys and weaken the immune system. The exact cause of many myelomas 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 by 2020.
Abecma is a B-cell antigen maturation (BCMA) – genetically directed autologous antigen chimney antigen (CAR) T-cell therapy. Each dose of Abecma is a standard treatment created using the 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 modified to include a new gene that enables the targeting and killing of myeloma cells. As soon as the cells are modified, they are reintroduced to the patient.
The safety and efficacy of Abecma was established in a multicenter study of 127 patients with recurrent myeloma (myeloma who return after treatment was completed) and salvage myeloma (non-treatment-responsive myeloma), who received at least three layers of preoperative antimyeloma. . Approximately 88% of patients in the study group had received four or more episodes of antimyeloma therapy. Overall, 72% of patients partially or completely responded to the treatment. Of those surveyed, 28% of patients showed a full response – or all signs of multiple myeloma disappear – to Abecma, and 65% of this group remained a complete response to treatment for at least 12 years. months.
Treatment with Abecma has the potential to cause side effects. Boxing warning is on the label for, cytokine release syndrome (CRS), hemophagocytic lymphohistiocytosis / macrophage activation syndrome (HLH / MAS), neurologic toxicity, and prolonged, potentially fatal or life-threatening cytopenia. CRS and HLH / MAS are systemic responses to CAR-T cell activation and proliferation causing high fever and flu-like symptoms, and persistent cytopenia is a decrease in the number of specific blood cell types for an extended period of time. The most common side effects of Abecma include CRS, diarrhea, fatigue, muscle pain, and a weakened immune system. Side effects from treatment usually appear within the first week or two after treatment, but some side effects may occur later. Patients with multiple myeloma should speak to their healthcare professionals to determine if Abecma is an appropriate treatment for them.
Because of the risk of CRS and neurologic toxins, Abecma is licensed with a risk assessment and mitigation strategy that incorporates elements to ensure safe use. The FDA requires that hospitals and their associated clinics that dispense Abecma be specifically certified and that staff involved in prescribing, dispensing or administering Abecma be trained to administer CRS toxins. and identify nervous system and other side effects of Abecma. Patients must also be told about the possible side effects and the importance of a quick return to the treatment site if side effects occur after receiving Abecma.
To further assess long-term safety, the FDA is also asking the manufacturer to conduct a post-market observational study involving Abecma-treated patients.
Abecma has been awarded Drug Orphan Drug and Compensatory Injuries by the FDA. Orphan drug assignment provides an incentive to aid and encourage drug development for rare diseases. Breaking Breakthrough Therapy is a process designed to accelerate the development and review of drugs intended to treat a malignant condition and initial clinical evidence indicates that the drug may show significant improvement. on the available treatment of clinically important boundary (s). Breakthrough Therapy was assigned based on persistent responses observed in patients with recurrent and resuscitated myeloma.
Drugs approved under fast-track programs, such as Breakthrough Therapy specification, are maintained at the same levels of approval as all other FDA approvals.
Abecma was approved by the FDA for Celgene Corporation, a Bristol Myers Squibb company.
The FDA, an agency within the U.S. Department of Health and Human Services, protects public health by determining the safety, efficacy, and safety of human and veterinary drugs, vaccines, and other biological products for use. human, and medical devices. The organization is also responsible for the safety and security of our nation’s food supply, cosmetics, dietary products, products that emit electronic radiation, and for the regulation of tobacco products.
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