The US Food and Drug Administration on Wednesday approved Yescarta (axicabtagene ciloleucel), a cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment. Yescarta, a chimeric antigen receptor (CAR) T cell therapy, is the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma (NHL).
The new drug’s maker, Kite Pharma, an industry leader in the emerging field of cell therapy founded by Israeli scientist Dr. Arieh Belldegrun, was acquired in late August by Gilead Sciences for $11.9 billion. Yescarta was developed by Prof. Zelig Ashchar of the Department of Immunology at the Weizmann Institute’s Faculty of Biology and Dr. Steven Rosenberg of the American National Cancer Institute.
Gilead gained 4% as soon as news broke about the FDA approval of the new lymphoma drug. As of Wednesday’s close in NY, shares of Gilead have gained close to 12% so far this year, according to CNBC.
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases. 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,” said FDA Commissioner Scott Gottlieb, M.D. “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. We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine. That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies. We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”
Each dose of Yescarta is a customized treatment created using a patient’s own immune system to help fight the lymphoma. The patient’s T-cells, a type of white blood cell, are collected and genetically modified to include a new gene that targets and kills the lymphoma cells. Once the cells are modified, they are infused back into the patient.
According to STAT News, the Yescarta therapy will cost $373,000 for a one-time dose, which will surely become a major issue of concern for insurance companies and public health officials. The Kymriah treatment, offered by Swiss multinational pharmaceutical company Novartis, costs $475,000 per patient, which the company defends by pointing to the treatment’s astonishing results (83% recovery after three months). In fact, Novartis proposed that Medicaid would only pay for the treatment if the patient responds within a month.
Gilead may not be able to offer the same deal, since its treatment targets a different form of cancer, and so far its success rate is only slightly above one-third.
Treatment with Yescarta has the potential to cause severe side effects: cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms; and neurologic toxicities, both of which can be fatal or life-threatening. Other side effects include serious infections, low blood cell counts and a weakened immune system.