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Tagraxofusp, sold under the brand name Elzonris, is an anti-cancer medication for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN).

Tagraxofusp is a fusion protein consisting of interleukin 3 (IL-3) fused to diphtheria toxin. The fusion protein readily kills cultured pDC by binding to their IL-3 receptors to thereby gain entrance to the cells and then blocking these cells' protein synthesis (due to its diphtheria toxin portion inhibiting eukaryotic elongation factor 2).

It was approved for use in the United States in 2018, and in the European Union in January 2021. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.

Society and culture

Legal status

In July 2020, the European Medicines Agency (EMA) recommended the refusal of the marketing authorization for tagraxofusp. The Agency was concerned that due to the design of the study and the small number of participants, it was not possible to be sure how effective the medicine was in treating blastic plasmacytoid dendritic cell neoplasm. In addition, the medicine could cause capillary leak syndrome (an unpredictable, potentially life-threatening side effect due to increased permeability of small blood vessels), which had led to some fatal outcomes.

In November 2020, the Committee for Medicinal Products for Human Use (CHMP) of the EMA adopted a positive opinion following a re-examination procedure, recommending the granting of a marketing authorization for the medicinal product Elzonris, intended for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). Tagraxofusp was approved for medical use in the European Union in January 2021.

References

1. "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.
2. ^ "Elzonris- tagraxofusp injection, solution". DailyMed. 9 June 2020. Retrieved 21 September 2020.
3. ^ "Elzonris EPAR". European Medicines Agency (EMA). 21 July 2020. Retrieved 25 January 2021.
4. "Elzonris Product information". Union Register of medicinal products. Retrieved 3 March 2023.
5. Hammond D, Pemmaraju N (June 2020). "Tagraxofusp for Blastic Plasmacytoid Dendritic Cell Neoplasm". Hematology/Oncology Clinics of North America. 34 (3): 565–574. doi:10.1016/j.hoc.2020.01.005. PMID 32336420. S2CID 216261672.
6. Lee SS, McCue D, Pemmaraju N (July 2020). "Tagraxofusp as treatment for patients with blastic plasmacytoid dendritic cell neoplasm". Expert Review of Anticancer Therapy. 20 (7): 543–550. doi:10.1080/14737140.2020.1776120. PMID 32460559. S2CID 218974611.
7. "Elzonris (tagraxofusp, SL-401)". Stemline Therapeutics.
8. "FDA approves first treatment for rare blood disease" (Press release). U.S. Food and Drug Administration (FDA). 21 December 2018.
9. New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.
10. ^ "Elzonris: Pending EC decision". European Medicines Agency (EMA). 24 July 2020. Retrieved 21 September 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.

External links

• "Tagraxofusp-erzs". NCI Drug Dictionary. National Cancer Institute.
• "Tagraxofusp-erzs". National Cancer Institute. 22 January 2019.

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