Drug intelligence / Profile preview

atorvastatin + n-acetylcysteine + romiplostim

Development stage
Unknown
Lead developer
Amgen
Modality
Peptides, Small Molecules
Administration
Oral, Subcutaneous
01

Overview

Atorvastatin + N-acetylcysteine (NAC) + romiplostim is an investigational triple combination therapy being evaluated for the treatment of steroid-resistant or relapsed immune thrombocytopenia (ITP). The regimen combines three distinct pharmacological agents: **atorvastatin**, an HMG-CoA reductase inhibitor (statin) that enhances the quantity and function of bone marrow endothelial progenitor cells (EPCs); **N-acetylcysteine (NAC)**, an antioxidant and reactive oxygen species (ROS) scavenger that reduces oxidative stress within the bone marrow niche; and **romiplostim**, a thrombopoietin receptor agonist (TPO-RA) that directly stimulates megakaryopoiesis and platelet production. This combination is designed to address multiple pathophysiological mechanisms of ITP, including impaired platelet production and a dysfunctional bone marrow microenvironment, with the goal of inducing a sustained off-treatment response in adult patients who have failed corticosteroid therapy.

Other names
atorvastatin + NAC + romiplostimatorvastatin combined with N-acetyl-L-cysteine plus romiplostim
02

Targets

TPOR (Thrombopoietin receptor)HMGCR (3-hydroxy-3-methylglutaryl-coenzyme A reductase)

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