The FDA has approved a second chimeric antigen receptor (CAR) T-cell therapy for cancer: Kite Pharma's axicabtagene ciloleucel (Yescarta) for the treatment of certain types of refractory large B-cell lymphoma, the agency announced.
It's the first "CAR T" treatment for certain types of non-Hodgkin lymphoma, the FDA noted. In August, the agency approved Novartis' tisagenlecleucel (Kymriah) for the treatment of certain pediatric and young adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.
A company press release said the list price of Yescarta in the U.S. will be $373,000 but did not elaborate further.
The gene therapy involves modifying a patient's own T cells to include a new gene allowing them to target lymphoma cells.
Yescarta was specifically approved for adults with large B cell lymphoma who failed at least two other kinds of treatment; specific types include diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B cell lymphoma, high grade B cell lymphoma, and DLBCL arising from follicular lymphoma.
It is not indicated for treating patients with primary central nervous system lymphoma.
FDA said approval was based on a multi-center clinical trial of more than 100 adults with refractory or relapsed large B cell lymphoma. The complete remission rate after treatment with the therapy was 51%, the agency reported.
Yescarta will come with a boxed warning and a risk evaluation and mitigation strategy (REMS). The boxed warning will pertain to cytokine release syndrome and neurologic toxicities, both of which can be fatal or life-threatening. As part of the REMS, hospitals must be certified to dispense the drug, with staff required to be trained in identifying and managing these toxicities.
Other side effects include serious infections, low blood cell counts, and a weakened immune system. Side effects usually appear in the first week or two.
FDA is also requiring a post-marketing observational study.
Yescarta had been granted priority review, breakthrough therapy designation, and orphan drug designations.
Kite Pharma was recently purchased by Gilead.
In announcing the approval, FDA Commissioner Scott Gottlieb, MD, noted the agency will soon release a guidance to support the development of cell-based regenerative medicine.