Gilead ramping up vehicle T clinics after US FDA Yescarta approval

Ann Santiago
October 21, 2017

The Food and Drug Administration approved the gene therapy, to be sold under the name Yescarta, to treat adults with large B-cell lymphoma, a type of non-Hodgkin lymphoma, who have failed to respond to other treatments. Known as a chimeric antigen receptor (CAR) T cell therapy, Yescarta modifies a patient's white blood cells so they can attack and kill lymphoma.

Yescarta is the second CAR-T treatment approved by the FDA. To further evaluate long-term safety, FDA is also requiring that the manufacturer conduct a post-marketing observational study on patients treated with the therapy. The FDA warns that axicabtagene ciloleucel has a boxed warning for cytokine release syndrome, which causes a high fever, flu symptoms, and neurological toxicities.

The FDA has approved a second cancer treatment using gene therapy, and the first to target non-Hodgkins lymphomas.

The market capitalization of Gilead is now reeling at 104.65B based on its aggregate value over its current share price and the total amount of outstanding stocks. Commissioner Scott Gottlieb announced alongside the approval that FDA will soon issue a comprehensive agency policy clarifying how FDA's expedited programs apply to gene therapy products. To address the risk of CRS and neurologic toxicities, which 28% of patients experienced, the FDA approved axi-cel with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU).

Diffuse large B-cell lymphoma is the most common type of the disease in adults, with some 72,000 new patients diagnosed annually in the United States, according to the American Cancer Society, and some 20,000 will die from it this year. Other potential side effects include serious infections, low blood-cell counts, and a weakened immune system.

For more on what role pharmacists can play for patients with lymphoma, view the video below.

Arie Belldegrun, the Israeli-American founder of Kite Pharma, referred to the promising research when commenting on Gilead's purchase of Kite in August. "Now, thanks to this new therapy many patients are in remission for months".

Similar CAR-T therapies are being developed by Juno Therapeutics Inc., Bluebird Bio Inc., and others. In the United States alone, there are approximately 7,500 patients each year with refractory diffuse large B-cell lymphoma who are said to be eligible for vehicle T therapy.

The approval of Yescarta is supported by data from the ZUMA-1 pivotal trial.

Dr. Fred Locke, an oncologist and researcher at Moffitt Cancer Institute in Tampa, Florida, estimates that there are more than 10,000 adults who might benefit from these treatments, whereas the pediatric approval was initial geared for only about 600 patients a year. In a healthy body, the immune system can recognize abnormal, cancerous cells, but for people with cancer, it doesn't recognize that the cells are spreading.

The FDA decision opens the door for a new gene therapy to treat adults with aggressive lymphoma, noted Maloney.

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