SILVER SPRING, Md., February 5, 2021 / PRNewswire / – Today, the US Food and Drug Administration has approved Breyanzi (lisocabtagene maraleucel), a cell-based gene therapy to treat adult patients with certain types of large B-cell lymphoma that have not responded or have had a recurrence after at least least two other types of systemic treatment. Breyanzi, a chimeric antigen receptor (CAR) therapy with T cells, is the third FDA-approved gene therapy for certain types of non-Hodgkin’s 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, providing an additional treatment option for adults with certain cancers that affect blood, bone marrow and lymph nodes,” he said. Peter Marks, MD, Ph.D., director of the FDA’s Center for Biological Research and Evaluation. “GEne and cell therapies have evolved from promising concepts to practical cancer treatment regimes. ”
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 (aggressive) or slow-growing. Approximately 77,000 new cases of non-Hodgkin’s lymphoma are diagnosed in the United States each year and DLBCL represents approximately one in three new cases diagnosed.
Each dose of Breyanzi is a personalized treatment created with 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 death of lymphoma cells. Once the cells are modified, they are infused back into the patient.
The safety and efficacy of Breyanzi has been established in a multicenter clinical trial with more than 250 adults with refractory or relapsed large B-cell lymphoma. The rate of complete remission after treatment with Breyanzi was 54%.
Treatment with Breyanzi has the potential to cause serious side effects. The labeling contains a boxed warning 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 toxicities. Both CRS and neurological events can be fatal. Other side effects include hypersensitivity reactions, severe infections, low blood cell counts and weakened immune systems. Side effects usually appear in the first 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 is demanding, among other things, that health facilities that dispense Breyanzi be specially certified. As part of this certification, personnel involved in the prescription, dispensing or administration of Breyanzi must be trained to recognize and manage the risks of CRS and neurological toxicities. The REMS program specifies that patients are informed of the signs and symptoms of CRS and neurological toxicities after the infusion – and the importance of returning immediately to the treatment site if they develop fever or other adverse reactions after receiving treatment with Breyanzi.
To further assess long-term safety, the FDA also requires the manufacturer to conduct an observational post-marketing study involving patients treated with Breyanzi.
The FDA has granted the orphan drug designations Breyanzi, advanced regenerative medicine therapy (RMAT) and innovative therapy. The RMAT designation program was created under the 21st Century Cures Act to help facilitate the rapid development of regenerative medicine therapies for serious illnesses. Breyanzi is the first regenerative medicine therapy with RMAT designation to be licensed by the FDA. The orphan drug designation provides incentives to assist and encourage the development of drugs for rare diseases. The Breyanzi app has been revised using a coordinated, inter-agency approach, including the FDA Center for Biological Research and Evaluation and the FDA Center for Excellence in Oncology.
The FDA 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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