FDA New Drug and Biologic Approvals -- 2017 Year-in-Review

Mary L Windle, PharmD


January 11, 2018

In This Article

Yescarta (axicabtagene ciloleucel)


Adults with relapsed or refractory large B-cell lymphoma after ≥2 lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.


CD19-directed genetically modified autologous T-cell immunotherapy. A patient’s own T cells are reprogrammed with a transgene encoding a chimeric antigen receptor (CAR) to identify and eliminate CD19-expressing malignant and normal cells.


For autologous use only.

A single dose of axicabtagene ciloleucel is administered by IV infusion 2 days after completing lymphodepletion with conditioning chemotherapy.

Dosing of axicabtagene is based on the number of chimeric antigen receptor (CAR)-positive viable T cells.

Target dose is 2 x 106 CAR-positive viable T cells/kg body weight, not to exceed 2 x 108 CAR-positive viable T cells.

See the Certificate of Analysis for the actual number of CAR-positive T cells in the product.


Approval was based on results from the ZUMA-1 study (n=111), an open-label, multicenter trial. Following conditioning treatment (cyclophosphamide and fludarabine) for 3 days, 101 patients received axicabtagene ciloleucel. The ORR in the mITT analysis set of 101 patients was 82% (CR 54%, PR 28%). At a median follow-up of 8.7 months, 44% were in response and 39% were in CR. The median duration of response was 8.2 months overall and not reached for patients who achieved a CR. Median overall survival was not reached; 80% of patients remained alive at 6 months.


Locke FL, et al. CT019 - Primary results from ZUMA-1: a pivotal trial of axicabtagene ciloleucel (axicel; KTE-C19) in patients with refractory aggressive non-Hodgkin lymphoma (NHL). American Association for Cancer Research. Available at https://www.abstractsonline.com/pp8/#!/4292/presentation/12336. April 2, 2017; Accessed: November 14, 2017


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