The U.S. Food and Drug Administration (FDA) approved axicabtagene ciloleucel (Yescarta) to treat 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.

Kite Pharma’s Yescarta, a chimeric antigen receptor (CAR) T cell therapy, is the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma (NHL).

Among the types of NHL the therapy is indicated for is diffuse large B-cell lymphoma (DLBCL), which is the most common type of NHL in adults. It is approved for use in adults with large B-cell lymphoma after at least two other kinds of treatment failed, including DLBCL, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma.

Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma.

Using CAR-T cells to attack cancer

The cell based therapy uses the same technology as Kymriah from Novartis, which is indicated for the childhood leukemia, ALL.

“With CAR T therapy, we are reengineering a patient’s own immune system to detect and kill cancer cells, and the results have been impressive,” said Frederick L. Locke, MD, ZUMA-1 Co-Lead Investigator and Vice Chair of the Department of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt Cancer Center in Tampa, Florida, in a statement.

The approval was based on the results of the ZUMA-1 trial, in which 101 patients received the treatment. Eight months later, 72 percent of patients saw their cancer shrink, including 51 percent who had no signs of cancer remaining.

“Many of the patients that received CAR T therapy had already relapsed several times with traditional treatments such as chemotherapy or hematopoietic stem cell transplant. Now, thanks to this new therapy many patients are in remission for months,” said Locke.  

There were four deaths in the study, three of them attributed to cytokine release syndrome (CRS). The therapy was approved with a black box warning for CRS, as well as neurologic toxicities. A Risk Evaluation and Mitigation Strategy (REMS) has been approved by the FDA for Yescarta.

Other adverse effects include serious infections, low blood cell counts and a weakened immune system.

Gilead Sciences, who acquired Kite Pharma in August, plans to price the treatment at $373,000 per patient. Novartis’ treatment for ALL is priced at $475,000 per patient. 

A big step forward for gene therapy

“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,” said FDA Commissioner Scott Gottlieb, M.D in a statement.

“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.”