[6][7][8] Eltrombopag was approved by the US Food and Drug Administration (FDA) in November 2008, for the treatment of thrombocytopenia in people with chronic immune (idiopathic) thrombocytopenic purpura who have had an insufficient response to corticosteroids, immunoglobulin therapy, or splenectomy.

[12] In preclinical studies, the compound was shown to interact selectively with the thrombopoietin receptor, leading to activation of the JAK-STAT signaling pathway and increased proliferation and differentiation of megakaryocytes.

[13] Eltrombopag has been shown to be effective in two major clinical syndromes: idiopathic thrombocytopenic purpura (ITP)[14] and cirrhosis due to hepatitis C (in which low platelet counts may be a contraindication for interferon treatment).

[15] After six weeks of therapy in a phase III trial, eltrombopag 50 mg/day was associated with a significantly higher response rate than placebo in adult patients with chronic idiopathic thrombocytopenic purpura (ITP).

[21] Eltrombopag has been shown to target ELAVL1/HuR-RNA interactions affecting gene expression, iron metabolism, and glycoprotein hormones, alpha polypeptide (CGA) levels.