ARGINT PRIMARIE, Md., February 5, 2021 / PRNewswire / – Today, the U.S. Food and Drug Administration approved Breyanzi (lysocabtagene maraleucel), a cell-based gene therapy to treat adult patients with certain types of large B-cell lymphoma who did not respond or who relapsed after, at least two other types of systemic treatment. Breyanzi, a chimeric antigen receptor (CAR) T cell therapy, is the third FDA-approved gene therapy for certain types of non-Hodgkin’s lymphoma, including large diffuse B-cell lymphoma (DLBCL). Breyanzi is not indicated for the treatment of patients with primary lymphoma of the central nervous system.
Today’s approval is another milestone in the rapid progression of gene therapy, providing an additional treatment option for adults with certain cancers that affect the blood, bone marrow and lymph nodes, he said. Peter Marks, MD, Ph.D., director of the FDA Center for Biological Assessment and Research. „Gcell and gene therapies have evolved from promising concepts to practical cancer treatment regimens. ”
DLBCL is the most common type of non-Hodgkin’s lymphoma in adults. Non-Hodgkin’s lymphomas are cancers that start in certain cells of the immune system and can be either fast-growing (aggressive) or slow-growing. About 77,000 new cases of non-Hodgkin’s lymphoma are diagnosed in the United States each year, and DLBCL is about one in three newly diagnosed cases.
Each dose of Breyanzi is a personalized treatment created using the patient’s own T cells, a type of white blood cell, to help fight lymphoma. The patient’s T cells are collected and genetically modified to include a new gene that facilitates the targeting and destruction of lymphoma cells. Once the cells are modified, they are infused back into the patient.
The safety and efficacy of Breyanzi were established in a multicenter clinical study in more than 250 adults with refractory or recurrent large B-cell lymphoma. The complete remission rate after Breyanzi treatment was 54%.
Breyanzi treatment has the potential to cause severe side effects. The label bears a warning in the box for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T cells, causing high fever and flu-like symptoms and neurological toxicity. 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 usually occur within the first two weeks after treatment, but some side effects may occur later.
Due to the risk of CRS and neurological toxicities, Breyanzi is approved with a Risk Assessment and Mitigation Strategy (REMS) that includes elements to ensure safe use (ETASU). The FDA requires, among other things, that healthcare units that distribute Breyanzi be specially certified. As part of this certification, personnel involved in the prescribing, distribution or administration of Breyanzi should be trained to recognize and manage the risks of CRS and neurological toxicity. The REMS program specifies that patients should be informed of the signs and symptoms of CRS and neurological toxicity after infusion – and of the importance of prompt return to treatment if fever or other side effects occur after receiving Breyanzi treatment.
To further assess long-term safety, the FDA also requires the manufacturer to conduct an post-marketing observational study of patients treated with Breyanzi.
The FDA has awarded Breyanzi Orphan Drugs, Regenerative Medicine Advanced Therapy (RMAT) and the name Advanced Therapy. The RMAT designation program was created under 21St. Century Cures Act to facilitate the rapid development of regenerative medicine therapies for serious conditions. Breyanzi is the first FDA-licensed regenerative medicine therapy with RMAT designation. The designation of orphan drugs provides incentives to support and encourage the development of rare disease drugs. The Breyanzi application was reviewed using a coordinated, interagency approach, including both the Center for Biological Assessment and Research and the FDA’s Center of Oncological Excellence.
The FDA has granted Breyanzi approval to Juno Therapeutics Inc., a Bristol-Myers Squibb company.
Media contact: Monique Richards, 240-402-3014
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SOURCE US Food and Drug Administration
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