Drug intelligence / Profile preview

eltrombopag + recombinant human thrombopoietin

Development stage
Preclinical
Lead developer
Novartis
Modality
Recombinant Proteins and Enzymes, Small Molecules
Administration
Oral, Subcutaneous
01

Overview

Eltrombopag + recombinant human thrombopoietin is a combination therapy consisting of two agents that stimulate platelet production via the thrombopoietin receptor pathway. Eltrombopag is an orally bioavailable small molecule agonist of the thrombopoietin receptor (also known as c-Mpl), while recombinant human thrombopoietin (rhTPO) is a biologically engineered protein that mimics endogenous TPO to activate the same receptor. This combination has been investigated for its synergistic effects in increasing platelet counts in conditions such as severe aplastic anemia and corticosteroid-resistant or relapsed immune thrombocytopenia (ITP). Clinical studies indicate that this regimen can rapidly improve hematological response rates with minimal toxic effects, particularly in patients who are refractory to standard therapies[2][3][1]. Both components act by stimulating megakaryocyte proliferation and differentiation, thereby enhancing platelet production.

Other names
eltrombopag plus recombinant human thrombopoietineltrombopag + rhTPOeltrombopag and rhTPO
02

Targets

TPOR (Thrombopoietin receptor)

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