Less than two weeks following the closing of Gilead’s acquisition of the company, Kite Pharma’s CAR-T immunotherapeutic, axicabtagene ciloleucel, has gained approval from the U.S. Food and Drug Administration as the first CAR-T therapy for an adult indication. The approval, which came more than a month ahead of the expected FDA decision date, is for use of the new immunotherapeutic against relapsed or refractory large B-cell lymphoma in patients who had previously received two or more systemic therapies. Experts estimate there may be about 3500 people per year who qualify for the treatment, now named Yescarta, which Gilead is pricing at $373,000 — somewhat less than Novartis’ previously approved Kymriah CAR-T therapy, which was priced at $475,00 but with an associated “pay for performance” deal with the U.S. government. Gilead is not expected to offer any similar refunds for patients who do not respond to treatment.
Like Kymriah, Yescarta is an autologous CAR-T treatment involving the harvest, engineering, and return to the patient of their now-actived T cells — about a 17-day process. And like all of the CAR-T therapies to date, Yescarta is associated with significant and harsh side-effects. These include cytokine release syndrome in about 19% of those treated to date, and neurological events in 28% of treated patients. But unlike Kymriah, the Kite treatment appears to become more effective the longer it is in the body. Included in the approval was data for patients nine months after therapy; 72% of the 101 patients treated with the Kite CAR-T therapeutic showed a positive response, with 51% achieving a complete remission of their disease. Those rates were much higher than the results previously announced for the same patients, where six-month data had showed a 44% response rate, with 39% complete responses.
Novartis is also pursuing an adult NHL indication for Kymriah, which could come within months. Will Novartis offer a lower price for their treatment once they can also market Kymriah for the larger adult NHL indication? What will happen when other players in the CAR-T space, such as Bluebird and Juno, reach the market?