Axicabtagene ciloleucel (Yescarta, Gilead Sciences) became the first chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of adults with relapsed or refractory large B-cell lymphoma who have undergone two or more lines of systemic therapy.
Non-Hodgkin lymphomas, which account for about 4% of all cancers in the United States, are malignancies that begin in certain cells of the immune system.
Gilead shares were up 1.2% at $81 in early trading.
Axicabtagene ciloleucel (Yescarta) has been approved to treat adult patients with diffuse large B-cell lymphoma who have not responded to or who have relapsed after at least 2 other kinds of treatment. A patient's T-cells are collected and genetically modified to include a new gene that targets and kill lymphoma cells. "That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies", Gottlieb said. He called the approval a "milestone".
"We believe this is only the beginning for vehicle T therapies", Arie Belldegrun, MD, FACS, founder of Kite, said in a statement.
Put simply, this is a type of product, which aims to help the immune system identify and target cancer cells. In CAR-T therapy for hematologic cancer, a patient's own T cells are engineered to seek and destroy cancer cells. After the T cells are altered, they are placed back in the patient.
The FDA approval is based on a multicenter clinical trial of more than 100 patients.
The NCI licensed the treatment to Kite Pharma, and once it was shown to cause complete remission in half the patients who tried it, Gilead bought Kite. As such, axicabtagene ciloleucel carries a boxed warning for cytokine release syndrome (CRS) and for neurologic toxicities.
Patient's immune systems can release a flurry of compounds called cytokines that cause high fever -sometimes deadly - and flu-like symptoms and nerve damage. Due to potential toxicities, the approval is subject to a risk evaluation and mitigation strategy to ensure patient safety. However, since the eligible patients are facing such a high risk of death, most seem to think it's worth the risk.
Noting the FDA's commitment to "supporting and helping expedite" the development of gene therapies, Gottlieb said, "we will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine". Several other companies also have CAR-T therapies in the works.
The therapy has been dubbed a "living drug" by researchers and is the second in a new class of such therapies to win the federal go-ahead.
Dr. Arie Belldegrun, founder of Kite, recognized "the FDA for their ability to embrace and support transformational new technologies that treat life-threatening illnesses". "We believe this is only the beginning for vehicle T therapies".
Gilead Sciences Inc.'s stock rose Thursday morning after the biotech company's Kite Pharma unit gained federal approval for its flagship cell-treatment therapy for adults with advanced lymphoma. This is the first gene therapy to be approved for non-Hodgkin lymphoma (NHL), according to the FDA.