FDA approves Gilead's CAR-T therapy Yescarta for certain patients with large B-cell lymphoma

Gilead Sciences' recently acquired Kite unit announced Wednesday that the FDA granted approval to its CAR-T therapy Yescarta (axicabtagene ciloleucel) for certain patients with large B-cell lymphoma. The company noted that the list price of the CD19-directed genetically modified autologous T cell immunotherapy in the US will be $373 000.

Specifically, Kite noted that Yescarta is the first CAR-T therapy to be approved in the US for adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic treatment, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma and DLBCL arising from follicular lymphoma.

The FDA said that Yescarta is the second gene therapy approved in the US, following clearance of Novartis' CAR-T therapy Kymriah (tisagenlecleucel) in August for certain paediatric and young adult patients with acute lymphoblastic leukaemia. "Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases," remarked FDA Commissioner Scott Gottlieb. "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," Gottlieb said, adding "we will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine."

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According to the FDA, approval of Yescarta was based on findings from the ZUMA-1 study, which involved more than 100 adults with refractory or relapsed large B-cell lymphoma. Results showed that 72 percent of patients treated with a single infusion of Yescarta responded to therapy, including 51 percent of patients who achieved a complete remission. The regulator added that due to the risk of cytokine release syndrome and neurologic toxicities, the therapy is being approved with a risk evaluation and mitigation strategy.

Kite indicated that Yescarta will be manufactured at its facility in El Segundo, California, noting that during the pivotal trial for the therapy, a 99 percent manufacturing success rate was achieved with an average turnaround time of 17 days. The drugmaker noted that 16 centres will be immediately providing Yescarta to patients, with the company training another 30 sites out of a planned target of 70 to 90 centres across the US.

Commenting on the price of Yescarta, Gilead spokeswoman Amy Flood said "we are in ongoing and active discussions with payers, and we have communicated our openness to considering different solutions that improve patient access" Flood noted that the therapy's price was decided following extensive research with both government agencies that reimburse for drug costs and private insurers, and cancer centres.

A regulatory filing for Yescarta is currently under review by the European Medicines Agency, with a potential approval expected in the first half of 2018. Jefferies analyst Michael Yee estimates that the product could generate sales of up to $250 million by 2018. For related analysis, see ViewPoints: And so it begins- Gilead's CAR-T approval sets off the cell therapy race.

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