Drug intelligence / Profile preview

efgartigimod + romiplostim

Development stage
Preclinical
Lead developer
argenx
Modality
Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics
Administration
Intravenous, Subcutaneous
01

Overview

Efgartigimod + romiplostim is a combination therapy involving two distinct agents used in the management of immune thrombocytopenic purpura (ITP), particularly in patients who are refractory to multiple prior treatments. Efgartigimod is a human IgG1-derived Fc fragment engineered for increased affinity to the neonatal Fc receptor (FcRn). By blocking FcRn, efgartigimod prevents IgG recycling and promotes targeted degradation of pathogenic IgG autoantibodies, thereby reducing platelet destruction[4][8][10]. Romiplostim is a peptibody that acts as an agonist of the thrombopoietin receptor (TPO-R or MPL) on megakaryocyte precursors, stimulating their proliferation and differentiation to increase platelet production[6]. The sequential or combined use of these drugs has been reported in case studies for multi-refractory ITP patients where standard therapies have failed, with evidence suggesting restoration of platelet response[1][2]. This combination leverages complementary mechanisms—reducing antibody-mediated platelet destruction while simultaneously enhancing platelet production.

02

Targets

TPOR (Thrombopoietin receptor)FCGRT (Neonatal crystallizable fragment receptor)

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