FDA Approves Yescarta (Axicabtagene Ciloleucel) for Adults with Certain Lymphomas

This is the second gene therapy approved in the US

Written By:Stacy Simon

The US Food and Drug Administration (FDA) has approved Yescarta (axicabtagene ciloleucel) to treat adults with certain types of large B-Cell lymphoma. Yescarta is the second gene therapy using CAR T-cells approved by the FDA, and the first for any type of non-Hodgkin lymphoma

CAR T-cell therapy is designed to help the body’s own immune system fight cancer. Each dose of Yescarta is made using a patient’s T-cells, a type of white blood cell that helps fight diseases in the body. The T-cells are collected from the patient and sent to a lab where they are changed in a way that programs them to find and kill the lymphoma cells. They are then given back to the patient through an infusion.

In August, the FDA approved the CAR T-cell therapy Kymriah (tisagenlecleucel) for certain children, adolescents, and young adults with a form of acute lymphoblastic leukemia (ALL).

The FDA approved Yescarta using breakthrough therapy and priority review designations, which are ways to speed up the evaluation of drugs to treat serious conditions. It was also granted orphan drug designation, which provides financial incentives to encourage the development of drugs for rare diseases.

Who can benefit from Yescarta?

Non-Hodgkin lymphoma is a cancer that starts in white blood cells called lymphocytes, which are part of the body’s immune system. Most adults in the US who have non-Hodgkin lymphoma have a type of B-cell lymphoma, named after the type of lymphocytes (B cells) where they start.

The FDA approved Yescarta to treat people with some types of large B-cell lymphoma after at least 2 other kinds of treatment failed. It’s for people with diffuse large B-cell lymphoma, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma and diffuse large B-cell lymphoma arising from follicular lymphoma. The approval is not for people with primary central nervous system lymphoma.

“The approval of Yescarta brings this innovative class of CAR-T cell therapies to an additional group of cancer patients with few other options – those adults with certain types of lymphoma that have not responded to previous treatments,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research.

The FDA based its approval on a clinical trial of more than 100 adults with large B-cell lymphoma that had not improved after treatment, or that had come back after treatment. Everyone in the trial received Yescarta and 51% had a complete remission, which means all signs and symptoms of the cancer disappeared.

Possible side effects

Yescarta comes with a boxed warning for a potentially life-threating syndrome called cytokine release syndrome (CRS), which causes high fever, flu-like symptoms, and potential for serious neurological changes. Other severe side effects include infection, low blood cell counts, and a weakened immune system. CRS and other serious side effects usually occur within the first week or two after the infusion, but some side effects may occur later.

Because of the risk for severe side effects, the FDA has put some extra safety requirements in place. Treatment with Yescarta will be available only at specially certified hospitals and clinics, and medical staff will get special training to manage CRS and serious neurological changes. Patients must be told about the possibility of these side effects, and how important it is to go back to the treatment center if they develop.

In addition, the FDA is requiring the drug’s manufacturer to conduct more studies on Yescarta after the approval. Yescarta is marketed by Kite Pharma, Inc.

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