Drug intelligence / Profile preview

danazol + romiplostim

Development stage
Phase 2
Lead developer
Amgen
Modality
Small Molecules, Recombinant Proteins and Enzymes
Administration
Oral, Subcutaneous
01

Overview

Danazol + romiplostim is a combination therapy consisting of danazol, a synthetic steroid with antigonadotropic and antiestrogenic properties, and romiplostim, a thrombopoietin receptor agonist. This combination has been investigated as a salvage treatment for patients with immune thrombocytopenia (ITP) who are refractory to other therapies such as eltrombopag. Danazol works primarily by reducing platelet destruction through immunosuppressive effects, while romiplostim stimulates platelet production by activating the thrombopoietin receptor (MPL), mimicking endogenous TPO activity. The rationale for combining these agents is to leverage their complementary mechanisms—danazol modulating the immune environment and reducing antibody-mediated platelet destruction, and romiplostim increasing platelet production—to achieve better control of severe or refractory ITP[1][4]. Clinical studies suggest that this combination may be effective in heavily pretreated or resistant ITP cases[1][4].

02

Targets

AR (Adrenergic receptors)TPOR (Thrombopoietin receptor)

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