GlaxoSmithKline (GSK) has announced results from a global Phase II study of its investigational small molecule oral platelet growth factor, eltrombopag. Data from this study demonstrated that treatment with eltrombopag resulted in a significant increase in platelet count compared to placebo across a variety of typically poorly responding ITP patients including those with prior splenectomy or those having a very low baseline platelet count.
At doses of 50 mg and 75 mg, 70 percent and 81 percent of patients respectively, achieved the primary clinical endpoint of having a platelet count greater than 50,000/ml after up to six weeks of dosing compared to 11 percent of patients on placebo. Based on these and previous clinical trial results, eltrombopag has the potential to be the first oral platelet growth factor for patients with thrombocytopenia, a disorder characterized by low platelet counts leaving patients at risk of episodes of spontaneous bruising, mucosal bleeding, and in severe cases intracranial hemorrhage.