Megakaryocytes/platelets inducing agent being useful in treating diseases involving thrombopenia. Megakaryocytes/platelets inducing agent is an inducing agent for megakaryocytes and/or platelets from pluripotent stem cells.

Pipobroman (Vercyte) is an anti-cancer drug that probably acts as an alkylating agent. Pipobroman has well documented clinical activity in polycythemia vera (PV) and essential thrombocythemia (ET). Pipobroman allows, within 3 months, to attain a response in more than 90% of patients, without clinically relevant toxicities. The 10-years risk of thrombosis of patients treated with Pipobroman is about 15%, The anti-proliferative activity of Pipobroman on bone marrow megakaryocytes seems of particular value in lowering the occurrence of post-PV and post-ET MMM, whose risk is the lowest registered with available treatments. The 10-year risk of acute leukemia with Pipobroman is 5% in PVand 3% in ET, which is only slightly higher than that expected as a natural evolution of the disease. In conclusion, the use of Pipobroman is a definite alternative to hydroxyurea in patients with PV and ET at high risk of thrombosis.

Avatrombopag (YM477) is a new oral thrombopoietin (TPO) receptor agonist, activating TPO receptor and increasing megakaryocytic proliferation/differentiation and platelet production.

Avatrombopag, also known as AKR-501, YM477, AS 1670542 or E5501, is a novel orally-active thrombopoietin (TPO) receptor agonist. AKR-501 specifically targeted the TPO receptor and stimulated megakaryocytopoiesis throughout the development and maturation of megakaryocytes just as rhTPO did. AKR-501 may be useful in the treatment of patients with thrombocytopenia. Avatrombopag was first approved in 2018.

Totrombopag, also known LGD-4665, GSK2285921 and SB559448, is an oral thromobopoietin receptor agonist, is being developed as a new generation small molecule thrombopoietin (TPO) mimetic. It is a highly selective and potent agonist of the TPO receptor and therefore induces differentiation and proliferation of megakaryocytes. The pharmacologic characteristics indicate potential therapeutic use in thrombocytopenic patients with a variety of clinical etiologies.

Avatrombopag hydrochloride (AKR-501) is an orally active, nonpeptide agonist of the thrombopoietin (TPO) receptor with an EC 50 of 3.3 nM. It effectively mimics the biological activities of TPO, stimulating platelet production by activating the intracellular signaling system and facilitating the generation of platelets and megakaryocytes from hemopoietic precursor cells. Additionally, Avatrombopag hydrochloride serves as a substrate for cytochrome P450 (CYP) 2C9 and CYP3A.

Krds peptide is a synthetic tetrapeptide derived from lactotransferrin (residues 39 to 42) that suppresses binding of monoclonal antibody against glycoprotein IIb-IIIa on ADP-Stimulated platelets and megakaryocytes.