- For immediate release:
The Food and Drug Administration has approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded, or whose disease has returned after at least four previous lines (different types) of therapy. Abecma is the first cell-based gene therapy approved by the FDA for the treatment of multiple myeloma.
“The FDA remains committed to moving forward with new treatment options for areas of unmet need for the patient,” said Peter Marks, MD, Ph.D., director of the FDA’s Center for Biological Research and Evaluation. “Although there is no cure for multiple myeloma, the long-term perspective can vary according to the individual’s age and the stage of the disease at the time of diagnosis. Today’s approval provides a new treatment option for patients who have this unusual type of cancer. ”
Multiple myeloma is an uncommon type of blood cancer in which abnormal plasma cells accumulate in the bone marrow and form tumors in many bones in the body. This disease prevents the bone marrow from producing enough healthy blood cells, which can result in low blood counts. Myeloma can also cause damage to 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 approximately 1.8% (32,000) of all new cancer cases in the United States in 2020.
Abecma is a B cell maturation antigen (BCMA), directed by a genetically modified chimeric antigen (CAR) receptor, T cell therapy. Each dose of Abecma is a personalized 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 facilitates the targeting and elimination of myeloma cells. Once the cells are modified, they are infused back into the patient.
The safety and efficacy of Abecma were established in a multicenter study of 127 patients with recurrent myeloma (myeloma that returns after treatment is completed) and refractory myeloma (myeloma that does not respond to treatment), who received at least three lines of antimeloma therapy previous ones. Approximately 88% of patients in the study group had received four or more previous lines of anti-myeloma therapy. Overall, 72% of patients responded partially or completely to treatment. Of those studied, 28% of patients showed complete response – or disappearance of all signs of multiple myeloma – 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 bears a boxed warning for cytokine release syndrome (CRS), haemophagocytic lymphohistiocytosis / macrophage activation syndrome (HLH / MAS), neurological toxicity and prolonged cytopenia, all of which can be fatal or life-threatening. CRS and HLH / MAS are systemic responses to the 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 cell over a long period of time. The most common side effects of Abecma include CRS, infections, fatigue, musculoskeletal pain and weakened immune systems. Side effects of treatment usually appear in the first 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 being approved with a risk assessment and mitigation strategy that includes elements to ensure safe use. The FDA is requiring that hospitals and their associated clinics that dispense Abecma be specially certified and employees involved in the prescription, dispensing or administration of Abecma be trained to recognize and manage CRS and nervous system toxicities and other side effects of Abecma. In addition, patients should be informed about the potential serious side effects and the importance of returning to the treatment site immediately if side effects occur after Abecma administration.
To further assess long-term safety, the FDA also requires the manufacturer to conduct an observational post-marketing study involving patients treated with Abecma.
Abecma received designations for Orphan Drugs and Revolutionary Therapy from the FDA. The orphan drug designation provides incentives to assist and encourage the development of drugs for rare diseases. The designation of innovative therapy is a process designed to accelerate the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug can demonstrate substantial improvement over available therapy at one point (s) (s) of clinically significant evaluation. The designation of innovative therapy was granted based on the sustained responses observed in patients with relapsing and refractory myeloma.
Drugs approved in accelerated programs, such as the Breakthrough Therapy designation, meet the same approval standards as all other FDA approvals.
The FDA has granted Abecma approval to Celgene Corporation, a Bristol Myers Squibb company.
The FDA, an agency of the United States Department of Health and Human Services, protects public health by ensuring the safety, efficacy and safety of human and veterinary drugs, vaccines and other biological products for human use and medical devices. The agency is also responsible for the security of the supply of food, cosmetics, dietary supplements, products that emit electronic radiation and for the regulation of tobacco products in our country.
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