Drug intelligence / Profile preview

romiplostim + antithymocyte globulin + cyclosporine a

Development stage
Unknown
Lead developer
Amgen
Modality
Recombinant Proteins and Enzymes, Antibody-Based Therapeutics, Small Molecules
Administration
Subcutaneous (romiplostim), Intravenous (antithymocyte Globulin), Oral (cyclosporine A)
01

Overview

This drug is a **combination therapy** consisting of **romiplostim**, **antithymocyte globulin (ATG)**, and **cyclosporine a**. It is used primarily for the treatment of **acquired aplastic anemia (AA)**, including severe and refractory forms. - **Romiplostim** is a peptibody (Fc-peptide fusion protein) acting as a thrombopoietin (TPO) receptor agonist (agonist of c-MPL, the TPO receptor), stimulating proliferation and differentiation of megakaryocytes to increase platelet production and promote trilineage hematopoiesis. - **Antithymocyte globulin** (usually from horses or rabbits) is a polyclonal antibody that targets T lymphocytes and provides immunosuppressive effects by depleting cytotoxic T cells, thereby reducing immune-mediated destruction of hematopoietic stem cells. - **Cyclosporine a** is a calcineurin inhibitor immunosuppressant that lowers the activity of the immune system, primarily by inhibiting T-lymphocyte activation. This combination is employed in both **first-line** and **rescue therapy** settings for patients with acquired AA, especially those refractory to standard immunosuppressive therapy (IST). The combination aims to enhance hematopoietic response rates (including trilineage-, erythroid-, neutrophil-, and platelet-responses) and achieve transfusion independence. It is also being investigated in both adult and pediatric populations and is associated with response rates exceeding 70% in several clinical studies[1][2].

Brand names
SandimmuneNeoral
Other names
cyclosporin acyclosporineATG
02

Targets

TPOR (Thrombopoietin receptor)CN (Calcineurin)

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