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"Prediction is very difficult, especially if it's about the future." Nils Bohr Leading pharmaceutical companies invest billions of dollars each year in research and development with little or no guarantee of a return on their investment. With the average cost of developing a new drug... More
  • GSK: Promacta – Immune Thrombocytopenia Medication 0 comments
    Oct 11, 2013 7:14 AM | about stocks: GSK, LGND

    GlaxoSmithKline's (GSK) Promacta (eltrombopag) is an orally delivered small molecule agonist of the c-mpl (TpoR) receptor, which is the physiological target of the hormone thrombopoietin.

    Eltrombopag is used to treat low blood platelet counts in adults with chronic immune (idiopathic) thrombocytopenia (ITP), when either corticosteroids, immunoglobulins or surgery to remove the spleen have not worked well enough.

    The Eltrombopag molecule was discovered as a result of research collaboration between GlaxoSmithKline and Ligand Pharmaceuticals (LGND). It is currently marketed by GSK under the brand names Promacta in the US and Revolade in Europe.

    Designated an orphan drug in the USA and European Union, since the chronic ITP population is under 200,000 in both the US and Europe. Promacta was approved by the FDA in November 2008.

    Treatment Regimen

    Promacta is supplied as a tablet designed for oral administration. The recommended initial dose of the drug is 50 mg once daily except in patients who are of East Asian ancestry or who have moderate to severe hepatic impairment. In this population, and for patients with moderate or severe hepatic impairment, the recommended initial dose of Promacta is 25 mg once daily.

    Promacta should be adjusted to achieve and maintain a platelet count >50 x 109/L as necessary to reduce the risk for bleeding. The dosing of Promacta should not exceed 75 mg daily.

    Clinical Efficacy

    In preclinical studies, Eltrombopag was shown to interact selectively with the thrombopoeitin receptor, leading to activation of the JAK-STAT signaling pathway and increased proliferation and differentiation of megakaryocytes. Animal studies confirmed that administration could increase platelet counts.

    In Phase 1 clinical studies, in 73 healthy volunteers, higher doses of eltrombopag caused larger increases in the number of circulating platelets without tolerability problems.

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

    After 6 weeks of therapy in a phase 3 trial, eltrombopag 50mg/day was associated with a significantly higher response rate than placebo in adult patients with chronic ITP.

    Side Effects

    Eltrombopag side effects may include, but are not limited to, hemorrhage, nausea, vomiting, menorrhagia, myalgia, paresthesia and cataract.

    Systematic (IUPAC) Name

    3'-{(2Z)-2-[1-(3,4-dimethylphenyl)-3-methyl-5-oxo-1,5-dihydro-4H-pyrazol-4-ylidene]hydrazino}-2'-hydroxy-3-biphenylcarboxylic acid

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