"Yescarta, a chimeric antigen receptor (CAR) T cell therapy, is the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma", the FDA said in a statement. Each dose of axicabtagene ciloleucel is a customized treatment created using a patient's own immune system to help fight the lymphoma.
Yescarta, formerly KTE-C19, is a CD19-directed genetically modified autologous T cell immunotherapy that will be manufactured specifically for each individual patient.
"Yescarta is approved for use in adult patients with large B-cell lymphoma after at least two other kinds of treatment failed", the FDA said.
This includes diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL), high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma (transformed follicular lymphoma, or TFL).
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".
A second new personalized treatment for cancer has won approval from the Food and Drug Administration - a clear sign that such treatments will become more widely available for patients with no other options.
With other players such as Bluebird Bio also developing CAR-T therapies, it seems the age of cancer cell therapy is upon us. "We must also recognize the FDA for their ability to embrace and support transformational new technologies that treat life-threatening illnesses". After the cells are modified, they are infused back into the patient, where they patrol for years. Novartis' (NVS) Kymriah, which is meant for treatment of children afflicted with B-cell acute lymphoblastic leukemia, was the first drug in this class to be approved in August. The company says 44% of patients were still responding after six months, with 39% of these in complete remission. The therapy is still working in most study participants, so the average duration of its effects isn't known yet.
Yescarta has a boxed warning regarding the risks of cytokine release syndrome (CRS) and neurological toxicities.
Because of these risks, the FDA has approved Yescarta with a risk evaluation and mitigation strategy (REMS), which includes a requirement that hospitals and clinics be specially certified before dispensing the therapy.
The new treatment, Yescarta (axicabtagene ciloleucel), is for a kind of blood cancer called large B-cell lymphoma. Serious adverse reactions occurred in 52% of patients and included CRS, neurologic toxicity, prolonged cytopenias (including neutropenia, thrombocytopenia, and anemia), and serious infections.
Israeli-American oncologist Arie Belldegrun, founder of Kite Pharma. "Together, Gilead and Kite will accelerate studies of vehicle T therapy in multiple blood cancers and advance other cell therapy approaches for solid tumors, with the goal of helping patients with diverse cancers benefit from this new era of personalized cancer therapy". The manufacturer aims to train up to 90 centers about the drug.
Gilead, which just recently took over Kit Pharma, said the Yescarta list price is $373,000.
For more on what role pharmacists can play for patients with lymphoma, view the video below.