Federal regulators have approved a game-changing immunotherapy cancer drug to treat patients with certain types of lymphoma that uses a patient’s own cells to boost the immune system to fight the disease.
The U.S. Food and Drug Administration’s approval of Yescarta (axicabtagene ciloleucel), is the second gene therapy approved by the agency and the first for certain types of non-Hodgkin lymphoma (NHL).
Yescarta – known as(CAR-T cell therapy – has been shown to be effective in treating adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment,
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases. In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer,” said FDA Commissioner Dr. Scott Gottlieb in announcing the agency’s action.
“This approval demonstrates the continued momentum of this promising new area of medicine and we’re committed to supporting and helping expedite the development of these products. We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine. That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies.”
Diffuse large B-cell lymphoma (DLBCL) is the most common type of NHL cancers that begin in certain cells of the immune system.
About 72,000 new cases of NHL are diagnosed in the U.S. each year.
Yescarta is a customized treatment developed to work with a patient’s own immune system to help fight the lymphoma. The patient’s T-cells, a type of white blood cell, are collected and genetically modified to include a new gene that targets and kills the lymphoma cells. Once the cells are modified, they are infused back into the patient.
“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 Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER).
The FDA approval came after a multicenter clinical trial of more than 100 adults with refractory or relapsed large B-cell lymphoma were found to benefit from it. About 51 percent of the patients treated with the new drug went into complete remission.
FDA officials noted that Yescarta can cause severe, and even life-threatening side effects. As are a result, Yescarta is being approved with a risk evaluation and mitigation strategy requiring that hospitals and their associated clinics that dispense the drug to be specially certified.
Also, patients must be informed of the potential serious side effects and of the importance of promptly returning to the treatment site if side effects develop.
In addition, the FDA is also requiring the manufacturer – Kite Pharma Inc. – to conduct a post-marketing observational study involving patients treated with Yescarta.
The FDA granted Yescarta Priority Review and Breakthrough Therapy designations, which fast-tracked its review and approval.
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