Eltrombopag
Eltrombopag, sold under the brand name Promacta among others, is a medication used to treat thrombocytopenia and severe aplastic anemia. Eltrombopag is sold under the brand name Revolade outside the US and is marketed by Novartis. It is a thrombopoietin receptor agonist. It is taken by mouth.
Eltrombopag was discovered as a result of research collaboration between GlaxoSmithKline and Ligand Pharmaceuticals and is transferred to Novartis Pharmaceuticals.
Eltrombopag was approved for medical use in the US in November 2008, and authorized in the European Union in March 2010.
Medical uses
Eltrombopag is indicated for the treatment of thrombocytopenia in people with persistent or chronic immune thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy; for the treatment of thrombocytopenia in people with chronic hepatitis C to allow the initiation and maintenance of interferon-based therapy; in combination with standard immunosuppressive therapy for the first-line treatment of people with severe aplastic anemia; and for the treatment of people with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy.Development
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. Animal studies confirmed that it increased platelet counts. In 73 healthy volunteers, higher doses of eltrombopag caused larger increases in the number of circulating platelets without tolerability problems.Clinical trials
Eltrombopag has been shown to be effective in two major clinical syndromes: idiopathic thrombocytopenic purpura and cirrhosis due to hepatitis C.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.