Tagraxofusp Explained

Pronounce:
Tradename:	Elzonris
Dailymedid:	Tagraxofusp
Routes Of Administration:	Intravenous
Atc Prefix:	L01
Atc Suffix:	XX67
Legal Us:	Rx-only
Legal Eu:	Rx-only
Legal Eu Comment:	^[1]
Metabolism:	Proteases (expected)
Elimination Half-Life:	51 minutes
Cas Number:	2055491-00-2
Drugbank:	DB14731
Unii:	8ZHS5657EH
Kegg:	D11358
Synonyms:	DT388-IL3, SL-401, tagraxofusp-erzs
C:	2553
H:	4026
N:	692
O:	798
S:	16

Tagraxofusp, sold under the brand name Elzonris, is an anti-cancer medication for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN).^{[2] [3]}

Tagraxofusp is a fusion protein consisting of interleukin 3 (IL-3) fused to diphtheria toxin.^{[4] [5]} 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,^[6] 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.^[7]

Society and culture

Legal status

In July 2020, the European Medicines Agency (EMA) recommended the refusal of the marketing authorization for tagraxofusp.^[8] 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.^[9]

External links

• Web site: Tagraxofusp-erzs . NCI Drug Dictionary . National Cancer Institute .
• Web site: Tagraxofusp-erzs . National Cancer Institute . 22 January 2019 .

Notes and References

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