The therapy, Yescarta, is intended for adult patients with large B-cell lymphoma for whom the conventional types of treatment have failed.
'Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases,' said FDA commissioner Scott Gottlieb. 'Gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer.'
Large B-cell lymphoma is the most common form of non-Hodgkin's lymphoma, which account for four percent of cancers in the US. Currently, up to half of all patients with large B-cell lymphoma relapse or become resistant to treatments, which may include chemotherapy and stem cell transplants.
The new gene therapy, Yescarta, is a 'living drug' made from the patient's own immune cells. Like the first gene therapy for blood cancer, Kymriah (see BioNews 916), creating Yescarta requires extracting the patient's T-cells and modifying them to include a gene coding for chimeric antigen receptor (CAR). The so-called CAR-T cells are transfused back into the patient where they are better able to target and destroy cancer cells.
The drug was originally developed at the National Cancer Institute by a team led by Dr Steven Rosenberg. Yescarta was acquired by Kite Pharma in 2012, which was bought by pharma giant Gilead in August.
The FDA reported that 100 patients treated with Yescarta had a 51 percent complete remission rate in a multicentre clinical trial.
However, like other CAR-T therapies (see BioNews 917), the treatment carries the risk of life-threatening side effects, including high fever, low blood pressure, lung congestion and neurological problems. As a result the FDA requires hospitals and clinics to have special certification to provide the therapy.
'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,' said Gottlieb.
The New York Times reported the treatment takes about 17 days overall to be developed and available for a patient.
'I believe this treatment approach brings hope to many patients, but one thing to consider is cost,' said Dr Michaela Almgren of the University of South Carolina College of Pharmacy.
Gilead have priced Yescarta at $373,000 for a one-time dose.