SILVER SPRING, Md., February 5, 2021 / PRNewswire / – Today the U.S. Food and Drug Administration approved Breyanzi (lisocabtagene maraleucel), a cell-based gene therapy for the treatment of adult patients with certain types of large B-cell lymphoma who have not responded to, or have relapsed after, at least two other types of systemic treatment. Breyanzi, a chimeric antigen receptor (CAR) T cell therapy, is the third gene therapy approved by the FDA for certain types of non-Hodgkin lymphoma, including diffuse large B-cell lymphoma (DLBCL). Breyanzi is not indicated for the treatment of patients with primary central nervous system lymphoma.
“Today’s approval represents another milestone in the rapidly advancing field of gene therapy by providing an additional treatment option for adults with certain types of cancer affecting the blood, bone marrow and lymph nodes,” said Peter Marks, MD, Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “GOne and cell therapies have evolved from promising concepts to practical cancer treatment regimens. ”
DLBCL is the most common type of non-Hodgkin lymphoma in adults. Non-Hodgkin lymphomas are cancers that start in certain cells of the immune system and can be fast-growing (aggressive) or slow-growing. About 77,000 new cases of non-Hodgkin’s lymphoma are diagnosed each year in the US, and DLBCL represents about one in three newly diagnosed cases.
Each dose of Breyanzi is a tailor-made treatment that uses the patient’s own T cells, a type of white blood cell, to fight lymphoma. The patient’s T cells are collected and genetically modified to include a new gene that facilitates targeting and killing of the lymphoma cells. Once the cells have been modified, they are reintroduced into the patient.
The safety and efficacy of Breyanzi were established in a multicentre clinical study in more than 250 adults with refractory or recurrent large B-cell lymphoma. The percentage of complete remission after treatment with Breyanzi was 54%.
Treatment with Breyanzi can cause serious side effects. The labeling includes a boxed warning of cytokine release syndrome (CRS), a systemic response to CAR T cell activation and proliferation that causes high fever and flu-like symptoms and neurological toxicities. Both CRS and neurological events can be life-threatening. Other side effects include hypersensitivity reactions, serious infections, low blood cell counts and a weakened immune system. Side effects generally occur within the first one to two weeks after treatment, but some side effects can occur later.
Due to the risk of CRS and neurological toxicities, Breyanzi is approved with a Risk Assessment and Risk Mitigation Strategy (REMS) that includes elements to ensure safe use (ETASU). Among other things, the FDA requires that healthcare facilities that provide Breyanzi be specially certified. As part of that certification, personnel involved in prescribing, dispensing or administering Breyanzi must be trained to recognize and manage the risks of CRS and neurological toxicities. The REMS program specifies that patients are informed about the signs and symptoms of CRS and neurological toxicity after infusion – and about the importance of returning to the treatment site immediately if they develop fever or other adverse reactions after treatment with Breyanzi.
To further evaluate long-term safety, the FDA also requires the manufacturer to conduct a post-marketing observational study in patients treated with Breyanzi.
The FDA has granted Breyanzi Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT), and Breakthrough Therapy designations. The RMAT designation program was created under 21st Century Cures Act to facilitate the rapid development of regenerative drug therapies intended for serious conditions. Breyanzi is the first RMAT-designated regenerative drug therapy to be approved by the FDA. Orphan designation provides incentives to support and encourage the development of medicines for rare diseases. The Breyanzi filing was reviewed using a coordinated approach across agencies, including both the Center for Biologics Evaluation and Research and the FDA’s Oncology Center of Excellence.
The FDA granted Breyanzi approval to Juno Therapeutics Inc., a Bristol-Myers Squibb company.
Media contact: Monique Richards240-402-3014
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SOURCE US Food and Drug Administration
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