Kite Pharma Secures FDA Approval for CAR-T Immunotherapy

Nearly two months after the US Food and Drug Administration (FDA) approved the first CAR-T immunotherapy for a type of leukemia, the regulator has given the go-ahead to another member of this new class of drugs. Kite Pharma’s Yescarta (axicabtagene ciloleucel) has become the second CAR-T immunotherapy to be approved in the US, with an indication in treating adult patients with relapsed or refractory large B-cell lymphoma.

“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases,” said FDA Commissioner Dr. Scott Gottlieb. “In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer.”

The timing couldn’t be better for biopharmaceutical company Gilead, who announced in late August it would be acquiring Kite Pharma in a deal worth nearly $12 billion.

The results from Kite’s ZUMA-1 clinical trial were used to support approval of the cancer therapy. Of the 101 patients enrolled in the study, 72 percent responded to the CAR-T therapy, and 51 percent achieved complete remission.

However, 13 percent of study participants experienced cytokine release syndrome (CRS), a potentially life-threatening side effect of such immunotherapies. This prompted the FDA to mandate inclusion of a boxed warning on Yescarta to inform physicians and patients about the risk of CRS and associated neurologic toxicities.

“Engineered cell therapies like Yescarta represent the potential for a changing treatment paradigm for cancer patients,” said Dr. David Chang, Worldwide Head of Research and Development and Chief Medical Officer at Kite. “Together, Gilead and Kite will accelerate studies of CAR-T therapy in multiple blood cancers and advance other cell therapy approaches for solid tumors, with the goal of helping patients with diverse cancers benefit from this new era of personalized cancer therapy.”

The list price for one-time treatment with the personalized cancer therapy has been set at $373,000, according to a report from Reuters. While the two approved CAR-T immunotherapy drugs don’t share the same indications, the cost of Yescarta is over $100,000 less than Novartis’ Kymriah.

These high list prices come at a time when pharmaceutical companies have been under more pressure than ever to justify their pricing practises. While Novartis introduced value-based pricing for Kymriah, promising that patients would only have to pay for the therapy if they responded within in a set time, Gilead has yet to announce a similar plan.

CAR-T immunotherapy engineers a patient’s own immune cells – or occasionally donor cells – to recognize and destroy cancer tissue. Because this type of treatment has the potential to be curative, analysts are predicting that they could revolutionize the field of oncology.

“Immunotherapy is dramatically changing the way we approach blood cancer treatment, and we are hopeful that this therapy will ultimately be applicable to patients with other types of cancers as well,” said Dr. Louis J. DeGennaro, president and chief executive officer of The Leukemia and Lymphoma Society. “Today’s approval is a very significant advance for lymphoma patients and for the cancer community as a whole.”