Gene Therapy, Yescarta (axicabtagene ciloleucel), Approved for B-Cell Lymphoma
THURSDAY, Oct. 19, 2017 --Yescarta (axicabtagene ciloleucel) has been approved by the U.S. Food and Drug Administration as the first form of gene therapy for a non-Hodgkin lymphoma (NHL) form of cancer.
Diffuse large B-cell lymphoma is the most common form of this immune system cancer in adults, the agency said in a news release. Some 72,000 cases of NHL are diagnosed each year in the United States, and the Diffuse large-B cell form represents about a third of these cases, the FDA added.
This is only the second form of gene therapy approved by the FDA. The technique uses cells from the patient's own immune system to create a custom treatment to help fight the cancer. In this case, the patient's white blood cells called T-cells are modified to include a new gene that targets and kills lymphoma cells, then re-infused back into the patient.
"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. "We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms."
Yescarta is approved for adults for whom at least two other types of treatment have failed. The therapy was evaluated in clinical studies involving more than 100 adults. Slightly more than half of people given the therapy ended up in "complete remission," the FDA said.
The therapy includes a boxed warning of a heightened risk of high fever, flu-like symptoms and life-threatening neurologic symptoms, the FDA said. Patients must be informed of these risks before receiving the therapy.
Other potential side effects include serious infections, low blood-cell counts and a weakened immune system.
Hospitals that provide the therapy will have to be specially certified, the FDA said.
The therapy is produced by Los Angeles-based Kite Pharma Inc., which as a condition of approval must conduct studies of the therapy's long-term effects, the agency said.
Visit the FDA to learn more.
Posted: October 2017