Drug intelligence / Profile preview

tagraxofusp

Development stage
Approved
Lead developer
Stemline Therapeutics
Modality
Bacterial Toxin Conjugates → Immunotoxins → Antibody Conjugates → Antibody-Based Therapeutics, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
Administration
Intravenous
01

Overview

Tagraxofusp is a CD123-directed cytotoxin and recombinant fusion protein composed of human interleukin-3 (IL-3) genetically fused to the catalytic and translocation domains of truncated diphtheria toxin. It is used for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare hematologic malignancy. Tagraxofusp binds with high affinity to the IL-3 receptor alpha chain (CD123) on target cells, is internalized via receptor-mediated endocytosis, and releases its diphtheria toxin component into the cytosol. The toxin irreversibly inhibits protein synthesis by inactivating elongation factor 2, leading to apoptosis of malignant cells. Tagraxofusp was first approved by the FDA in December 2018 as the first treatment for BPDCN and later received approval in Europe[2][4][5][7][8].

Brand names
Elzonris
Other names
tagraxofusp
02

Targets

IL3RA (Interleukin 3 Receptor)

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