The US Food and Drug Administration (FDA) has given the nod to Yescarta (axicabtagene ciloleucel), the first cell-based gene therapy granted approval for the treatment of certain types of non-Hodgkin lymphoma (NHL) and only the second gene therapy to gain FDA approval.
The drug is indicated for the treatment of adult patients with certain kinds of large B-cell lymphoma who have relapsed or not responded to at least two other forms of treatment. The FDA decision was made following a multicenter clinical trial of more than 100 adults with refractory or relapsed large B-cell lymphoma, which reported a complete remission rate of 51 percent following treatment with Yescarta.
NHLs are cancers that begin in the immune system and can be either fast-growing or slow-growing. Around 72,000 new cases of NHL are diagnosed in the United States each year, of which one in three are diffuse large B-cell lymphoma. This particular form of NHL is the most common variety to develop in adults.
Scott Gottlieb, MD, FDA Commissioner, described the latest FDA announcement as a "milestone" in the development of new methods of treating 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," he asserted.
Each patient prescribed Yescarta has their own unique customized treatment, which is created using the patient's own immune system. T-cells, a variety of white blood cell, are genetically modified to include a new gene designed to target and kill the cancerous cells.
The drug carries a boxed warning or cytokine release syndrome (CRS), which can cause high fever, flu-like symptoms, and neurologic toxicities. Other side effects of the drug include serious infections, low blood cell counts , and a weakened immune system. The FDA approved Yescarta with a risk evaluation and mitigation strategy. Hospitals and clinics require special certification to dispense Yescarta.
Gilead's Kite Pharma was granted Priority Review, Breakthrough Therapy and Orphan Drug Designation for Yescarta. In August 2017, the FDA announced its first approval of a gene therapy for use in the United States. Kymriah (tisagenlecleucel) was grated approval for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia.
For more information on Yescarta, its indications and side effects, visit https://www.yescarta.com/