Global immune thrombocytopenia therapy updates and outlook- Romiplostim primary results suggest improvements in platelets count with positive safety and efficaacy

Immune Thrombocytopenia is a subtype of bleeding disorders and results in low platelet count. Though the exact cause of ITP is still unclear, pathophysiology of the disease can be contributed by an increased destruction of platelets, a decreased production of platelets, or both. Therefore, there might be multiple causes and mechanisms that can contribute to the disease. ITP can affect patients at all ages, but pediatric patients are likely to recover spontaneously while adult patients have a higher chance to develop into a chronic condition.

It is estimated that the value of the ITP market in the 7MM in 2015 was $928M, including sales of the major branded and off-label drugs commonly prescribed for ITP patients across the 7MM. The US is the largest contributor to the 7MM, with $587M in 2015, representing 63.3% of the market share. The 5EU and Japan contributed $281M and $60M in 2015, respectively, to the major ITP markets.

ITP market to grow during in the first half of the forecast period (2015–2025); however, sales will start to decline in 2022 in the US and projects that total ITP sales in 2025 will grow to $985M at a modest overall Compound Annual Growth Rate (CAGR) of 0.6%.

Major drivers of the growth of the ITP market over the forecast period include the following:
• Increasing uptake and market penetration of marketed brands, Amgen’s Nplate (romiplostim) and Novartis’ Promacta/Revolade (eltrombopag), in both adult and pediatric ITP populations.
• Rising diagnosed prevalence of adult ITP, and hence a rising treated population.
• Launch of Rigel Pharmaceuticals’ fostamatinib, which will offer a new mechanism of action (MOA) for ITP treatment.

Companies are looking into drugs with novel MOA, which may offer hope to improve response rate and safety. It is identified that two promising approaches: spleen tyrosine kinase (Syk) inhibitors, including the Phase III fostamatinib, and CD40/CD40 ligand antagonists. As Rigel has managed to complete two pivotal Phase III trials for fostamatinib, it is expected that the drugs offering new MOAs to be essential for companies to successfully develop new ITP therapies moving forward.–Immune-Thrombocytopenia–ITP—-Opportunity-Analysis-and-Forecasts-to-2025

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