Tagraxofusp

Tagraxofusp, sold under the brand name Elzonris, is an anti-cancer medication for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). It was approved for use in the United States in 2018.[3]

Tagraxofusp
Clinical data
Trade namesElzonris
Other namesDT388-IL3, SL-401, tagraxofusp-erzs
AHFS/Drugs.comMonograph
MedlinePlusa619022
License data
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Pharmacokinetic data
MetabolismProteases (expected)
Elimination half-life51 minutes
Identifiers
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC2553H4026N692O798S16
Molar mass57695.30 g·mol−1

The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[4]

Tagraxofusp is a fusion protein consisting of interleukin 3 (IL-3) fused to diphtheria toxin.[5][1] 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).

Society and culture

In July 2020, the European Medicines Agency (EMA) recommended the refusal of the marketing authorization for tagraxofusp.[6] 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.[6] 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.[6]

On 12 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).[6] Tagraxofusp was approved for medical use in the European Union in January 2021.[2]

References
  1. "Elzonris- tagraxofusp injection, solution". DailyMed. 9 June 2020. Retrieved 21 September 2020.
  2. "Elzonris EPAR". European Medicines Agency (EMA). 21 July 2020. Retrieved 25 January 2021.
  3. "FDA approves first treatment for rare blood disease" (Press release). U.S. Food and Drug Administration (FDA). December 21, 2018.
  4. New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.
  5. "Elzonris (tagraxofusp, SL-401)". Stemline Therapeutics.
  6. "Elzonris: Pending EC decision". European Medicines Agency (EMA). 24 July 2020. Retrieved 21 September 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
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