Axicabtagene ciloleucel

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Axicabtagene ciloleucel
Clinical data
Trade namesYescarta
Other namesKTE-C19, Axi-cel
AHFS/Drugs.comProfessional Drug Facts
License data
Routes of
Intravenous injection
ATC code
Legal status
Legal status

Axicabtagene ciloleucel, sold under the brand name Yescarta, is a medication used for the treatment for large B-cell lymphoma that has failed conventional treatment.[6] T cells are removed from a person with lymphoma and genetically engineered to produce a specific T-cell receptor. The resulting chimeric antigen receptor T cells (CAR-Ts) that react to the cancer are then given back to the person to populate the bone marrow.[7] Axicabtagene treatment carries a risk for cytokine release syndrome (CRS) and neurological toxicities.[7]

Due to CD19 being a pan-B cell marker,[8] the T-cells that are engineered to target CD19 receptors on the cancerous B cells[7] also influence normal B cells, except some plasma cells.[9]

Side effects

Because treatment with axicabtagene carries a risk of cytokine release syndrome and neurological toxicities, the FDA has mandated that hospitals be certified for its use prior to treatment of any patients.[7]


It was developed by California-based Kite Pharma.[10]

Axicabtagene ciloleucel was awarded U.S. Food and Drug Administration (FDA) breakthrough therapy designation on 18 October 2017, for diffuse large B-cell lymphoma, transformed follicular lymphoma, and primary mediastinal B-cell lymphoma.[11] It also received priority review and orphan drug designation.[7]

Based on the ZUMA-1 trial, Kite submitted a biologics license application for axicabtagene in March 2017, for the treatment of non-Hodgkin lymphoma.[12]

The FDA granted approval on 18 October 2017, for the second-line treatment of diffuse large B-cell lymphoma.[7][13][5]

On 1 April 2022, the FDA approved axicabtagene ciloleucel for adults with large B-cell lymphoma (LBCL) that is refractory to first-line chemoimmunotherapy or relapses within twelve months of first-line chemoimmunotherapy.[14] It is not indicated for the treatment of patients with primary central nervous system lymphoma.[14]

Approval was based on ZUMA-7, a randomized, open-label, multicenter trial in adults with primary refractory LBCL or relapse within twelve months following completion of first-line therapy.[14] Participants had not yet received treatment for relapsed or refractory lymphoma and were potential candidates for autologous hematopoietic stem cell transplantation (HSCT).[14] A total of 359 participants were randomized 1:1 to receive a single infusion of axicabtagene ciloleucel following fludarabine and cyclophosphamide lymphodepleting chemotherapy or to receive second-line standard therapy, consisting of two or three cycles of chemoimmunotherapy followed by high-dose therapy and autologous HSCT in participants who attained complete remission or partial remission.[14]


  1. ^ a b "T Cells - Axicabtagene ciloleucel, cryopreserved - T - Yescarta (axicabtagene ciloleucel) Suspension for Intravenous Infusion". Therapeutic Goods Administration (TGA). Retrieved 16 September 2020.
  2. ^ "Updates to the Prescribing Medicines in Pregnancy database". Therapeutic Goods Administration (TGA). 12 May 2022. Retrieved 13 May 2022.
  3. ^ "Summary Basis of Decision (SBD) for Yescarta". Health Canada. 23 October 2014. Retrieved 29 May 2022.
  4. ^ "Yescarta- axicabtagene ciloleucel suspension". DailyMed. 31 January 2022. Retrieved 4 April 2022.
  5. ^ a b "Yescarta (axicabtagene ciloleucel)". U.S. Food and Drug Administration (FDA). 18 October 2017. Retrieved 1 April 2020.
  6. ^ Axicabtagene Ciloleucel (Yescarta) for B-Cell Lymphoma Med Lett Drugs Ther. 2018 Jul 16;60(1551):e122-123
  7. ^ a b c d e f "FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma". U.S. Food and Drug Administration (Press release). Retrieved 20 October 2017. Public Domain This article incorporates text from this source, which is in the public domain.
  8. ^ Wang K, Wei G, Liu D (November 2012). "CD19: a biomarker for B cell development, lymphoma diagnosis and therapy". Experimental Hematology & Oncology. 1 (1): 36. doi:10.1186/2162-3619-1-36. PMC 3520838. PMID 23210908.
  9. ^ Halliley JL, Tipton CM, Liesveld J, Rosenberg AF, Darce J, Gregoretti IV, et al. (July 2015). "Long-Lived Plasma Cells Are Contained within the CD19(-)CD38(hi)CD138(+) Subset in Human Bone Marrow". Immunity. 43 (1): 132–45. doi:10.1016/j.immuni.2015.06.016. PMC 4680845. PMID 26187412.
  10. ^ "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". Gilead (Press release). Retrieved 20 October 2017.
  11. ^ "Kite to Present Two Plenary Presentations from the ZUMA-1 Pivotal Trial of Axicabtagene Ciloleucel at the 2017 American Association of Cancer Research Annual Meeting". Archived from the original on 21 June 2017. Retrieved 9 May 2017.
  12. ^ "Kite Completes Submission of U.S. Biologics License Application (BLA) for Axicabtagene Ciloleucel as the First CAR-T Therapy for the Treatment of Patients With Aggressive Non-Hodgkin Lymphoma (NHL) March 2017". Archived from the original on 25 April 2017. Retrieved 9 May 2017.
  13. ^ F.D.A. Approves Second Gene-Altering Treatment for Cancer 2017
  14. ^ a b c d e "FDA approves axicabtagene ciloleucel for second-line treatment of large B-cell lymphoma". U.S. Food and Drug Administration (FDA). 1 April 2022. Retrieved 4 April 2022. Public Domain This article incorporates text from this source, which is in the public domain.

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