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LGD-4665, also known as totrombopag, is an orally available small molecule agonist of the thrombopoietin receptor (TPO receptor, also known as c-Mpl or MPL). It acts as a TPO mimetic, stimulating megakaryopoiesis and increasing platelet production. Developed by Ligand Pharmaceuticals, LGD-4665 was investigated primarily for the treatment of thrombocytopenia, including immune thrombocytopenic purpura (ITP). Clinical studies demonstrated that it is a highly selective full agonist of the TPO receptor and can significantly increase platelet counts in both healthy volunteers and patients with ITP. The drug showed dose-proportional pharmacokinetics with a half-life of approximately 90 hours and was generally well tolerated in clinical trials[1][3][4][5][7].
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