The FDA has given the go-ahead for a second chimeric antigen receptor (CAR) T cell therapy: axicabtagene ciloleucel (Yescarta), developed by Kite Pharma, which was recently acquired by Gilead Sciences, has been approved
to treat adult patients with diffuse large B-cell lymphoma (DLBCL) who have not responded to or who have relapsed after at least 2 other kinds of treatment. The most common type of non-Hodgkin lymphoma, DLBCL is diagnosed in an average of 72,000 new patients annually in the United States.
“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, MD, in a statement announcing the approval. “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.”
The approval follows a multicenter clinical trial that tested the safety and efficacy of axicabtagene ciloleucel in 100 adults with refractory or relapsed large B-cell lymphoma and observed a complete remission rate of 51%. The first
CAR T treatment to be approved—tisagenlecleucel (Kymriah), developed by Novartis—reported an 83% remission rate within 3 months of treatment in patients with B-cell precursor acute lymphoblastic leukemia.
Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement, “The approval of Yescarta brings this innovative class of CAR-T cell therapies to an additional group of cancer patients with few other options—those adults with certain types of lymphoma that have not responded to previous treatments.”
The adverse effect profile remains the same for this treatment as for tisagenlecleucel—the drug has a boxed warning for cytokine release syndrome and for neurologic toxicities, which can both be fatal or life-threatening. The FDA has linked a risk evaluation and mitigation strategy with the approval: hospitals and their associated clinics that dispense axicabtagene ciloleucel need to be specially certified. As part of that certification, staff involved in the prescribing, dispensing, or administering of Yescarta are required to be trained to recognize and manage CRS and nervous system toxicities. Also, patients must be informed of the potential serious side effects and of the importance of promptly returning to the treatment site if side effects develop.
Gilead has listed
axicabtagene ciloleucel at $373,000 in the United States, much cheaper than the launch price
of tisagenlecleucel at $475,000.