The pathogenesis of chronic idiopathic thrombocytopenic purpura (ITP) involves antibody-mediated platelet destruction and reduced platelet production. Stimulation of platelet production may be an effective treatment for this disorder.
Eltrombopag is an oral thrombopoietin-receptor agonist.
Eltrombopag is a small molecule agonist of the c-mpl (TpoR) receptor, which is the physiological target of the hormone thrombopoietin. Image source: Wikipedia, public domain.
A NEJM study from 2007 reported that eltrombopag increased platelet counts in a dose-dependent manner in patients with relapsed or refractory ITP.
Promacta® (eltrombopag) was approved by the FDA in November 2008 as the first oral medication to increase platelet production in ITP.
PROMACTA may cause hepatotoxicity. Because of the risk for hepatotoxicity and other risks, PROMACTA is available only through a restricted distribution program called PROMACTA Cares.
Eltrombopag for the Treatment of Chronic Idiopathic Thrombocytopenic Purpura. NEJM.
FDA approves Promacta® (eltrombopag), the first oral medication to increase platelet production for people with serious blood disorder. GSK.