FDA Approves Kite’s CAR-T Drug, Second Gene-Therapy in US

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This approval marks the second gene therapy to be approved by FDA and the first to be approved for certain types of non-Hodgkin lymphoma.

On Oct. 18, 2017, FDA approved Yescarta (axicabtagene ciloleucel), a cell-based gene therapy that is a chimeric antigen receptor (CAR)-T cell therapy, developed by the Santa Monica, CA-based biopharmaceutical company, Kite Pharma, a Gilead Company. The therapy was approved for treating certain types of large B-cell lymphoma, 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 (transformed follicular lymphoma). The therapy is not indicated for treating primary central nervous system lymphoma. This approval marks the second gene therapy to be approved by FDA in the United States and the first for certain types of non-Hodgkin lymphoma, according to the agency.

Each dose of the therapy is created using a patient’s own T-cells, which are collected and genetically modified to incorporate a gene that targets and eradicates lymphoma cells. The modified T-cells are infused back into the patient for treatment. Because treatment with this therapy can potentially cause severe side effects, FDA’s approval carries a boxed warning for cytokine release syndrome (CRS), a systemic response to the activation and proliferation of CAR-T cells that can cause high fever and flu-like symptoms. The boxed warning also includes a warning for neurologic toxicities.

Because of these risks, FDA is requiring hospitals and associated clinics that dispense the treatment to be specially certified, including training to recognize and manage CRS and nervous system toxicities. 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 therapy will be manufactured in Kite’s commercial manufacturing facility in El Segundo, CA, which the company opened in June 2016. The facility has an estimated capacity to produce up to 5,000 patient therapies per year.


“Engineered cell therapies like Yescarta represent the potential for a changing treatment paradigm for cancer patients,” said David Chang, MD, PhD, worldwide head of research and development and chief medical officer at Kite, in a company press release. “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.”

In August 2017, FDA approved the first gene therapy in the US, Novartis’ Kymriah (tisagenlecleucel), also a CAR-T therapy. Novartis’ product was approved for treating a form of acute lymphoblastic leukemia.

Source: FDA and Kite Pharma