Tisagenlecleucel

Tisagenlecleucel
Clinical data
Pronunciationtis" a jen" lek loo' se
/ˌtɪsədʒen'leklusel/
Trade namesKymriah
Other namesCTL019, CART-19, tisa-cel
AHFS/Drugs.comProfessional Drug Facts
MedlinePlusa617053
License data
Pregnancy
category
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Pharmacokinetic data
Elimination half-life16.8 days
Identifiers
CAS Number
DrugBank
UNII
KEGG

Tisagenlecleucel, sold under the brand name Kymriah, is a CAR T cells medication for the treatment of B-cell acute lymphoblastic leukemia (ALL) which uses the body's own T cells to fight cancer (adoptive cell transfer).[10][7]

The most common serious side effects are cytokine release syndrome (a potentially life-threatening condition that can cause fever, vomiting, shortness of breath, pain and low blood pressure) and decreases in platelets (components that help the blood to clot), hemoglobin (the protein found in red blood cells that carries oxygen around the body) or white blood cells including neutrophils and lymphocytes.[9]

T cells from a person with cancer are removed, genetically engineered to make a specific chimeric cell surface receptor with components from both a T-cell receptor and an antibody specific to a protein on the cancer cell, and transferred back to the person. The T cells are engineered to target a protein called CD19 that is common on B cells. A chimeric T cell receptor ("CAR-T") is expressed on the surface of the T cell.[medical citation needed]

The platform invented at the University of Pennsylvania was clinically developed by Novartis, including market authorization, and real world evidence.[11][12] In August 2017, it became the first FDA-approved treatment that included a gene therapy step in the United States.[10]

  1. ^ "Updates to the Prescribing Medicines in Pregnancy database". Therapeutic Goods Administration (TGA). 12 May 2022. Archived from the original on 3 April 2022. Retrieved 13 May 2022.
  2. ^ "FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 October 2023.
  3. ^ "KYMRIAH (Novartis Pharmaceuticals Australia Pty LTD) | Therapeutic Goods Administration (TGA)". Archived from the original on 2 January 2024. Retrieved 2 January 2024.
  4. ^ "KYMRIAH (Novartis Pharmaceuticals Australia Pty LTD) | Therapeutic Goods Administration (TGA)". Archived from the original on 2 January 2024. Retrieved 2 January 2024.
  5. ^ "KYMRIAH (Novartis Pharmaceuticals Australia Pty LTD) | Therapeutic Goods Administration (TGA)". Archived from the original on 2 January 2024. Retrieved 2 January 2024.
  6. ^ "Summary Basis of Decision (SBD) for Kymriah". Health Canada. 23 October 2014. Archived from the original on 31 May 2022. Retrieved 29 May 2022.
  7. ^ a b "Kymriah- tisagenlecleucel injection, suspension". DailyMed. 14 June 2019. Archived from the original on 22 October 2020. Retrieved 1 April 2020.
  8. ^ "Kymriah (tisagenlecleucel)". U.S. Food and Drug Administration. 7 July 2022. Archived from the original on 19 November 2022. Retrieved 19 November 2022.
  9. ^ a b Cite error: The named reference Kymriah EPAR was invoked but never defined (see the help page).
  10. ^ a b "FDA approval brings first gene therapy to the United States". U.S. Food & Drug Administration (FDA) (Press release). Archived from the original on 24 April 2019. Retrieved 31 August 2017.
  11. ^ Awasthi R, Maier HJ, Zhang J, Lim S (2023). "Kymriah® (Tisagenlecleucel) – an overview of the clinical development journey of the first approved CAR-T therapy". Human Vaccines & Immunotherapeutics. 19 (1). doi:10.1080/21645515.2023.2210046. PMC 10294746. PMID 37185251.
  12. ^ "BLA 125646 Tisagenlecleucel - Novartis Briefing document to FDA ODAC" (PDF). Food and Drug Administration. Archived (PDF) from the original on 16 July 2017. Retrieved 16 July 2017.