Immune 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 affects adults. For many years, immune-modulating therapies, such as corticosteroids, immunoglobulins (IVIG and anti-RhD), and rituximab, were the mainstays of treatment in ITP. However, the approval of thrombopoietin-receptor agonists (TPO–RAs), eltrombopag (Promacta) and romiplostim (Nplate), starting in 2008, revolutionized the treatment landscape of ITP by opening new and highly effective avenues of treatment other than immunomodulation. Despite the availability of many effective therapies, a significant proportion of patients exist who are either unresponsive or refractory to available treatments. The late-phase clinical pipeline of ITP comprises a spleen tyrosine kinase (Syk) inhibitor, Tavalisse, and a second-generation oral TPO–RA, avatrombopag, both of which are expected to launch in the next few years. The early-phase ITP pipeline includes mostly immunomodulating agents, such as inhibitors of CD20, CD40, Fc receptors, and Syk signaling. Although there are many available treatments and a sizable emerging therapy pipeline, the ITP landscape has ample scope and market opportunity for development of agents based on novel therapeutic approaches, particularly agents that can target the specific underlying causes of disease.
Niche & Rare Disease Landscape & Forecast: Comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.