SILVER SPRING, Md., February 5, 2021 / PRNewswire / – Today, the U.S. Food and Drug Administration approved Breyanzi (maraleucel lysocabtagene), a cell-based gene therapy to treat adult patients with certain types of large B-cell lymphoma that have not responded or have relapsed afterwards, 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 B-cell diffuse lymphoma (DLBCL). . Breyanzi is not indicated for the treatment of patients with primary lymphoma of the central nervous system.
“Today’s approval represents another milestone in the field of gene therapy that is progressing rapidly as it provides an additional treatment option for adults with certain types of cancer that affect the blood, bone marrow and lymph nodes, ”he said. Peter Marks, MD, Ph.D., director of the FDA’s Center for Biological Assessment and Research. “Ggene and cell 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 fast-growing (aggressive) or slow-growing. Approximately 77,000 new cases of non-Hodgkin’s lymphoma are diagnosed each year in the U.S. and DLBCL accounts for approximately 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 killing of lymphoma cells. Once the cells have been modified, they are infused back into the patient.
The safety and efficacy of Breyanzi were established in a multicenter clinical trial of more than 250 adults with large refractory or relapsed B cell lymphoma. The rate of complete remission after treatment with Breyanzi was 54%.
Breyanzi treatment can cause serious side effects. The label contains a box warning about 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 toxicities. neurological. Both CRS and neurological events can be life-threatening. Other side effects include hypersensitivity reactions, severe infections, low blood cell count, and a weakened immune system. Side effects usually appear in the first two to two weeks after treatment, but some side effects may occur later.
Due to the risk of CRS and neurological toxicities, Breyanzi is being approved with a Risk Assessment and Mitigation Strategy (REMS) that includes elements to ensure safe use (ETASU). The FDA requires, among other things, that the sanitary facilities that dispense Breyanzi be specially certified. As part of this certification, staff involved in the prescription, dispensing, or administration of Breyanzi should be trained to recognize and manage the risks of CRS and neurological toxicity. The REMS program specifies that patients will be informed of the signs and symptoms of CRS and neurological toxicities after infusion and the importance of returning quickly to the site of treatment if they develop fever or other adverse reactions after receiving Breyanzi treatment.
To further evaluate long-term safety, the FDA also requires the manufacturer to conduct a post-marketing observational study with patients treated with Breyanzi.
The FDA awarded Breyanzi orphan drug designations, Advanced Regenerative Medicine Therapy (RMAT) and Advanced Therapy. The RMAT designation program was created under 21c Century Cures Act to help facilitate the rapid development of regenerative medicine therapies for severe conditions. Breyanzi is the first FDA-licensed regenerative medicine therapy with RMAT designation. The designation of orphan drugs provides incentives to help and encourage the development of drugs for rare diseases. The Breyanzi application was reviewed through a coordinated, cross-agency approach, which included both the Biological Assessment and Research Center and the FDA Center for Oncological Excellence.
The FDA granted Breyanzi approval to Juno Therapeutics Inc., a Bristol-Myers Squibb company.
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SOURCES US Food and Drug Administration
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