Immune (idiopathic) thrombocytopenic purpura (ITP) is a hematological autoimmune disorder characterized by low platelet count in the blood, which may lead to easy bruising and bleeding. ITP has an acute, generally self-limiting form that typically affects children and a chronic form that typically affects adults. Although the condition is primarily believed to be caused by autoantibodies against platelet-surface antigens, a growing consensus points to decreased platelet production from the bone marrow as an equal contributor to low platelet count in ITP. Immune-modulating therapies such as corticosteroids, immunoglobulins (IVIG and anti-RhD), and rituximab have remained the mainstays of treatment in ITP for many years. The approval of thrombopoietin receptor agonists (TPO-RAs) eltrombopag (Promacta) and romiplostim (Nplate) starting in 2008 has opened new avenues of treatment other than immunomodulation in ITP. However, the clinical pipeline in ITP comprises mostly immunomodulating agents, and only one drug, fostamatinib, a Syk inhibitor, is in late-phase development. Despite many available therapies, there remains a significant proportion of patients who are refractory to treatment. There is therefore ample scope and a market opportunity for development of agents based on novel therapeutic approaches, in particular agents that can target the specific underlying causes of disease in ITP.