Exagamglogene autotemcel

Exagamglogene autotemcel
Clinical data
Trade namesCasgevy
Other namesCTX001, exa-cel
AHFS/Drugs.comMonograph
MedlinePlusa624018
License data
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Identifiers
DrugBank
UNII
KEGG

Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease[4][6] and transfusion-dependent beta thalassemia.[4] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.[10]

The treatment was approved in the United Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023.[11][12][13] It was approved by the Kingdom of Bahrain's National Health Regulatory Authority (NHRA) to treat sickle-cell anemia and beta thalassemia on 2 December 2023.[14][15]. It was approved in the United States for the treatment of sickle cell disease in December 2023 and for the treatment of transfusion-dependent beta thalassemia in January 2024.[16][17][18]

Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA).[16] The most common side effects include low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile neutropenia (fever and low white blood cell count), headache, and itching.[16]

  1. ^ "Summary Basis of Decision for Casgevy". Drug and Health Products Portal. 8 February 2025. Retrieved 22 June 2025.
  2. ^ "Notice: Multiple additions to the Prescription Drug List (PDL) [2024-12-20]". Health Canada. 20 December 2024. Retrieved 21 December 2024.
  3. ^ "Casgevy product information". Health Canada. 11 October 2024. Retrieved 27 December 2024.
  4. ^ a b c "Summary of Product Characteristics". Medicines and Healthcare products Regulatory Agency (MHRA). 15 November 2023. Archived from the original (PDF) on 8 December 2023. Retrieved 9 December 2023.
  5. ^ "Casgevy 4–13 x 10Exp6 cells/mL dispersion for infusion". Electronic Medicines Compendium. 24 November 2023. Archived from the original on 9 December 2023. Retrieved 9 December 2023.
  6. ^ a b "Casgevy- exagamglogene autotemcel injection, suspension". DailyMed. 22 January 2024. Retrieved 3 March 2024.
  7. ^ "Casgevy". U.S. Food and Drug Administration (FDA). 8 December 2023. Archived from the original on 19 December 2023. Retrieved 8 December 2023. This article incorporates text from this source, which is in the public domain.
  8. ^ Cite error: The named reference Casgevy EPAR was invoked but never defined (see the help page).
  9. ^ "Casgevy product information". Union Register of medicinal products. 12 February 2024. Retrieved 19 February 2024.
  10. ^ Stein R (31 October 2023). "FDA advisers see no roadblocks for gene-editing treatment for sickle cell disease". NPR. Archived from the original on 4 December 2023. Retrieved 4 December 2023.
  11. ^ "MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia". Medicines and Healthcare products Regulatory Agency (MHRA) (Press release). 16 November 2023. Archived from the original on 25 November 2023. Retrieved 8 December 2023.
  12. ^ Sheridan C (November 2023). "The world's first CRISPR therapy is approved: who will receive it?". Nature Biotechnology. 42 (1): 3–4. doi:10.1038/d41587-023-00016-6. PMID 37989785. S2CID 265350318. Archived from the original on 4 December 2023. Retrieved 4 December 2023.
  13. ^ "Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, Casgevy (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia" (Press release). Vertex Pharmaceuticals. 16 November 2023. Archived from the original on 22 November 2023. Retrieved 9 December 2023 – via Business Wire.
  14. ^ Bahrain News Agency (2 December 2023). "CASGEVY receives Bahrain approval for treatment, marking it the second country in the world". BNA. Retrieved 25 July 2025.
  15. ^ Telegraph (8 August 2024). "Roll out of revolutionary gene-editing therapy is 'an historic moment". Telegraph. Retrieved 25 July 2025.
  16. ^ a b c "FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease". U.S. Food and Drug Administration (FDA). 8 December 2023. Archived from the original on 8 December 2023. Retrieved 8 December 2023. This article incorporates text from this source, which is in the public domain.
  17. ^ "Vertex and CRISPR Therapeutics Announce US FDA Approval of Casgevy (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease" (Press release). Vertex Pharmaceuticals. 8 December 2023. Archived from the original on 9 December 2023. Retrieved 9 December 2023 – via Business Wire.
  18. ^ Commissioner, Office of the (16 January 2024). "FDA Roundup: January 16, 2024". FDA. Archived from the original on 16 January 2024. Retrieved 19 January 2024.
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