Last night, the U.S. Food and Drug Administration (FDA) approved Yescarta (axicabtagene ciloleucel), a T-cell therapy that utilizes chimeric antigen receptor (CAR) technology, for the treatment of some patients with relapsed/refractory aggressive B-cell non-Hodgkin lymphoma (NHL).
Yescarta becomes the second gene therapy approved by the FDA, and the first for these specific cases of NHL. The sanction comes nearly two months removed from the approval of Novartis’ Kymriah (tisagenlecleucel) for the treatment of B-cell precursor acute lymphoblastic leukemia (ALL).
The therapy was previously granted Priority Review and Breakthrough Therapy designations, and was also the recipient of FDA Orphan Drug designation.
"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 Scott Gottlieb, M.D. in a press release
. "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. We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms."
Yescarta, developed by Kite Pharma, is now indicated for use in adult patients after the failure of at least 2 other previous treatments. Among the lymphoma types for which the drug is approved are: diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma.
The gene therapy administers a customized treatment for each patient, using his or her own immune system to assist in the fight against lymphomas. Patients’ T-cells are collected and genetically modified to include a new gene that targets and terminates the lymphoma cells, and once the cells are effectively 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 Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER).
The safety and efficacy of Yescarta were confirmed in a multicenter trial of more than 100 adults, and the complete remission rate after treatment was 51%. Still, though, the drug has the potential to result in severe side effects, including life-threatening cytokine release syndrome (CRS), which can cause high fever and flu-like symptoms.
Because of the potential events, post-marketing observational studies are still required by the FDA to evaluate Yescarta’s long-term safety profile.
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