Drug intelligence / Profile preview

eltrombopag + rabbit anti-thymocyte globulin + cyclosporine A

Development stage
Phase 2
Lead developer
Novartis
Modality
Small Molecules, Antibody-Based Therapeutics
Administration
Oral, Intravenous
01

Overview

A combination therapy consisting of three agents: - **Eltrombopag**: an oral small molecule thrombopoietin receptor agonist that stimulates the myeloproliferative leukemia protein (MPL, a TPO receptor), promoting proliferation and differentiation of hematopoietic stem and progenitor cells to megakaryocytes, increasing platelet production. - **Rabbit anti-thymocyte globulin (ATG)**: a polyclonal immunoglobulin G (IgG) preparation generated by immunizing rabbits with human thymocytes, targeting multiple T-cell surface antigens, resulting in T-cell depletion primarily through complement-mediated cytotoxicity and apoptosis, used as an immunosuppressive agent. - **Cyclosporine A (CsA)**: a small molecule calcineurin inhibitor that suppresses T-cell mediated immune responses by inhibiting the transcription of interleukin-2. This combination is used for the first-line treatment of severe aplastic anemia (SAA) in patients who are not eligible for hematopoietic stem cell transplantation. The therapy aims to achieve both robust immunosuppression and stimulate residual hematopoiesis, improving hematologic responses over immunosuppressive therapy alone. Evidence from Japanese and international studies supports improved response rates and survival using eltrombopag in combination with rabbit-ATG and cyclosporine A in severe and non-severe aplastic anemia[1][3][5].

Other names
eltrombopag plus rabbit anti-thymocyte globulin and cyclosporine Aeltrombopag + rabbit-ATG + CsAeltrombopag + rabbit ATG + CsA
02

Targets

CN (Calcineurin)TPOR (Thrombopoietin receptor)MR1 (Thioesterase PNKD)CD3 (T-cell surface glycoprotein CD3)CD4 (T lymphocyte surface antigen)

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