FDA Approves Axicabtagene Ciloleucel for Non-Hodgkin Lymphoma, Questions Remain About Cost and Toxicity

Share this content:
Despite high efficacy, the $373,000 list price from the drug’s manufacturer may preclude some patients from receiving axicabtagene ciloleucel.
Despite high efficacy, the $373,000 list price from the drug’s manufacturer may preclude some patients from receiving axicabtagene ciloleucel.

The US Food and Drug Administration (FDA) granted approval to axicabtagene ciloleucel, a chimeric antigen receptor (CAR)-T cell therapy for patients with some subtypes of relapsed or refractory non-Hodgkin lymphoma, including diffuse large B cell lymphoma (DLBCL).1

Approval was granted based on results from the pivotal phase 2 ZUMA-1 trial (ClinicalTrials.gov Identifier: NCT02348216), which showed a complete response rate of 39% among 101 treated patients at a median follow-up of 8.7 months.2 All included patients had relapsed or refractory DLBCL, primary mediastinal B cell lymphoma, or transformed follicular lymphoma.

The modified intention-to-treat (mITT) analysis showed an objective response rate of 82%, with an overall response duration of 8.2 months. Median overall survival was not reached and at 6 months 80% of patients were alive.

Three grade 5 adverse events were reported. The most common grade 3 or worse adverse events were neutropenia (66%), leukopenia (44%), and anemia (43%).

The high risk of treatment-related complications, which may arise from a lack of understanding of the evolutionary implications of CAR-T cell therapy, should be weighed against the high efficacy of the treatment. The FDA press release notes that axicabtagene ciloleucel “carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and for neurologic toxicities. Both CRS and neurologic toxicities can be fatal or life-threatening.”

The $373,000 list price from the drug's manufacturer may also preclude some patients from receiving axicabtagene ciloleucel.3

This FDA approval is the second for a gene therapy in the United States. The first, in August 2017, was for tisagenlecleucel, which treats pediatric patients with relapsed or refractory acute lymphoblastic leukemia.


  1. FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma [news release]. Silver Spring, MD: US Food and Drug Administration; October 18, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm581216.htm. Accessed October 18, 2017.
  2. Locke FL, Neelapu SS, Bartlett NL, et al. Primary results from ZUMA-1: a pivotal trial of axicabtagene ciloleucel (axicel; KTE-C19) in patients with refractory aggressive non-Hodgkin lymphoma (NHL). Cancer Res. 2017;77(suppl; abstr CT019). doi: 10.1158/1538-7445.AM2017-CT019
  3. Kite's Yescarta™ (axicabtagene ciloleucel) becomes first CAR T therapy approved by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy [news release]. Foster City, CA and Santa Monica, CA: BusinessWire; October 18, 2017. http://www.businesswire.com/news/home/20171018006639/en/Kite%E2%80%99s-Yescarta%E2%84%A2-Axicabtagene-Ciloleucel-CAR-Therapy-Approved. Accessed October 19, 2017.

Related Resources

You must be a registered member of Cancer Therapy Advisor to post a comment.

Sign Up for Free e-newsletters

Regimen and Drug Listings


Bone Cancer Regimens Drugs
Brain Cancer Regimens Drugs
Breast Cancer Regimens Drugs
Endocrine Cancer Regimens Drugs
Gastrointestinal Cancer Regimens Drugs
Gynecologic Cancer Regimens Drugs
Head and Neck Cancer Regimens Drugs
Hematologic Cancer Regimens Drugs
Lung Cancer Regimens Drugs
Other Cancers Regimens
Prostate Cancer Regimens Drugs
Rare Cancers Regimens
Renal Cell Carcinoma Regimens Drugs
Skin Cancer Regimens Drugs
Urologic Cancers Regimens Drugs