Drug intelligence / Profile preview

ist + hetrombopag

Development stage
Unknown
Lead developer
Hengrui Pharmaceutical
Modality
Recombinant Proteins and Enzymes, Small Molecules
Administration
Oral, Intravenous
01

Overview

**ist + hetrombopag** refers to the combination of **immunosuppressive therapy (IST)**, typically consisting of antithymocyte globulin (ATG) and cyclosporine, with **hetrombopag**, an oral, small molecule, synthetic, nonpeptide thrombopoietin receptor agonist (TPO-RA). This combination is primarily used as a first-line regimen or for refractory cases in patients with **severe aplastic anemia (SAA)** who are ineligible for hematopoietic stem cell transplantation (HSCT). Hetrombopag binds to the transmembrane domain of the thrombopoietin (TPO) receptor (encoded by the MPL gene), stimulating platelet production and hematopoiesis. Clinical studies indicate that adding hetrombopag to IST increases hematologic response rates, shortens time to response, and improves transfusion independence compared with IST alone, without significant new safety concerns. The combination is also under investigation for other thrombocytopenic disorders[1][3][6].

Brand names
HengquThymoglobulinNeoralSandimmune
Other names
ist + hetrombopag
02

Targets

TPOR (Thrombopoietin receptor)

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