A second chimeric antigen receptor (CAR) T-cell therapy has gained FDA approval, this time for the treatment of large B-cell lymphoma in adults.

“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases,” FDA Commissioner Scott Gottlieb, MD, said in a statement. “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.”

Axicabtagene ciloleucel, to be marketed at Yescarta by Kite Pharma, is approved for use in adults with large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL). Patients must have failed at least two other treatments before axicabtagene ciloleucel can be tried.

Approval was based on ZUMA-1, a multicenter clinical trial of 101 adults with refractory or relapsed large B-cell lymphoma. Almost three-quarters (72%) of patients responded, including 51% who achieved complete remission.

CAR-T therapy can cause severe, life-threatening side effects, most notably cytokine release syndrome (CRS) and neurologic toxicities, for which axicabtagene ciloleucel will carry a boxed warning and will come with a risk evaluation and mitigation strategy (REMS), according to the FDA.

The list price for a single treatment of axicabtagene ciloleucel is $373,000, according to the manufacturer.

“We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine,” Dr. Gottlieb said in a statement. “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.”

Axicabtagene ciloleucel was developed by Kite Pharma, which was acquired recently by Gilead Sciences.


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