Breaking: FDA Approves Second CAR-T Therapy for Cancer


The US Food and Drug Administration (FDA) has approved the second chimeric antigen receptor (CAR) T-cell therapy, indicated for certain types of non-Hodgkin lymphoma.

Axicabtagene ciloleucel (Yescarta, Kite Pharma) is a cell-based gene therapy approved for the treatment of adult patients with subtypes of large B-cell lymphoma who have not responded or who have relapsed after a minimum of two previous therapies, and are ineligible for autologous stem cell transplant. Lymphomas included in the designation are diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

The safety and efficacy of axicabtagene ciloleucel were shown in a multicenter clinical trial involving over 100 adult patients with refractory or relapsed large B-cell lymphoma. Complete remission rates after treatment were 51% for those taking the gene therapy.


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Axicabtagene ciloleucel carries a boxed warning for cytokine release syndrome. Other adverse events associated with the drug include serious infections, low blood cell counts, and a weakened immune system.

The price for axicabtagene ciloleucel is initially set at $373,000. The first gene therapy to receive FDA approval—tisagenlecleucel-T (Kymriah, Novartis)—entered the market on August 30 and was priced at $475,000.—Zachary Bessette