Kite Pharma, acquired by Gilead Sciences in October 2016 for $11.9 billion, has announced that the FDA has granted approval to Yescarta™ (axicabtagene ciloleucel), a CAR T (chimeric antigen receptor T cell) cancer therapy. CAR T therapy is a breakthrough in hematologic cancer treatment in which a patient’s own T cells are engineered to seek and destroy cancer cells.
Yescarta has been approved for the treatment of adult patients with relapsed or refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL), after two or more lines of systemic therapy. DLBCL is the most common aggressive non-Hodgkin lymphoma (NHL), accounting for three out of every five cases. In the United States each year, there are approximately 7,500 patients with refractory DLBCL who are eligible for CAR T therapy.
Historically, when treated with the current standard of care, patients with refractory large B-cell lymphoma had a median overall survival of approximately six months, with only seven percent attaining a complete response. Currently, patients with large B-cell lymphoma in second or later lines of therapy have poor outcomes and greater unmet need, since nearly half of them either do not respond or relapse shortly after transplant.
“With CAR T therapy, we are reengineering a patient’s own immune system to detect and kill cancer cells, and the results have been impressive,” said Frederick L. Locke, MD, ZUMA-1 Co-Lead Investigator and Vice Chair of the Department of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt Cancer Center in Tampa, Florida. “Many of the patients that received CAR T therapy had already relapsed several times with traditional treatments such as chemotherapy or hematopoietic stem cell transplant. Now, thanks to this new therapy many patients are in remission for months.”
“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. “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.”
Yescarta has a Boxed Warning in its product label regarding the risks of cytokine release syndrome (CRS) and neurologic toxicities. A Risk Evaluation and Mitigation Strategy (REMS) has been approved by the FDA for Yescarta. The REMS program will inform and educate healthcare professionals about the risks associated with Yescarta therapy. Training and certification on the REMS program will be an integral part of the final authorization for centers offering Yescarta.
Yescarta will be manufactured in Kite’s state-of-the-art commercial manufacturing facility in El Segundo, California. In the ZUMA-1 pivotal trial, Kite demonstrated a 99 percent manufacturing success rate with a median manufacturing turnaround time of 17 days, which is important to patients given the potential for rapid disease progression in this population.
In 2017, Kite established a multi-disciplinary field team focused on providing education and logistics training for centers. Upon Yescarta’s approval, this team will provide final site certification to 16 centers, enabling them to make Yescarta available to appropriate patients. This support is designed to assure the safe and effective use of Yescarta for patients and physicians. Kite is actively working to train more than 30 additional centers with an eventual target of 70 to 90 centers across the United States
The list price of Yescarta in the United States is $373,000. Yescarta has been granted Priority Medicines (PRIME) regulatory support for DLBCL in the European Union. A Marketing Authorization Application (MAA) for axicabtagene ciloleucel is currently under review with the European Medicines Agency (EMA) and potential approval is expected in the first half of 2018.