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.

Questions Answered

  • How will the size of the ITP population change through 2027?
  • What are key current therapies in the ITP market? What will be their commercial contribution to the ITP market in 2027?
  • What are the key drug targets emerging from basic and clinical research in ITP? Which emerging therapies do ITP experts consider most promising? How would new therapies influence management of ITP patients?
  • How are emerging ITP drugs being evaluated, and which are likely to launch by 2027? What commercial impact will they have on the ITP market?

Product Description

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.

Table of contents

  • Immune Thrombocytopenic Purpura - Landscape & Forecast - Disease Landscape & Forecast
    • Commercial Outlook
      • Key Findings
        • Regional Sales of Key Therapies to Treat Immune Thrombocytopenic Purpura: 2017 and 2027
        • Immune Thrombocytopenic Purpura SWOT Analysis
        • Actionable Recommendations in Immune Thrombocytopenic Purpura, According to Hematologists
      • Drivers and Constraints
        • What Factors Are Driving Sales in Immune Thrombocytopenic Purpura?
        • What Factors Are Constraining Sales in Immune Thrombocytopenic Purpura?
      • Drug-Class-Specific Trends
        • Spleen Tyrosine Kinase Inhibitors
        • Thrombopoietin-Receptor Agonists
        • Uptake Trends of Thrombopoietin Receptor Agonists
    • Forecast
      • Sales of Key Therapies in Immune Thrombocytopenic Purpura
    • Etiology and Pathophysiology
      • Disease Overview
        • Expert Insight
      • Etiology
        • Risk Factors Associated with Immune Thrombocytopenic Purpura
      • Pathophysiology
        • Pathophysiology of Immune Thrombocytopenic Purpura
      • Symptoms and Complications
        • Symptoms and Complications of Immune Thrombocytopenic Purpura
        • Association Between Thrombocytopenia and Bleeding
        • Key Differences Between Acute and Chronic Immune Thrombocytopenic Purpura
        • Expert Insight on Differences Between Pathophysiology of Chronic and Acute Immune Thrombocytopenic Purpura
      • Key Pathways and Drug Targets
      • Epidemiology
        • Key Findings
          • Epidemiology Populations
            • Disease Definition
            • Methods
            • Sources Used for Diagnosed Prevalence of Immune Thrombocytopenic Purpura
            • Number of Diagnosed Prevalent Cases of Immune Thrombocytopenic Purpura in the Major Pharmaceutical Markets by Subtype: 2017-2027
            • Sources Used for Drug-Treated Prevalence of Immune Thrombocytopenic Purpura
            • Number of Drug-Treated Prevalent Cases of Immune Thrombocytopenic Purpura in the Major Pharmaceutical Markets: 2017-2027
        • Current Treatment
          • Key Findings
            • Expert Insight
          • Diagnosis
            • Diagnostic Tests and Criteria
            • Treatment Providers and Referral Patterns
            • Expert Insight: Diagnosis of Immune Thrombocytopenic Purpura
          • Treatment Goals
            • Expert Insight on Treatment Goals
            • Key End Points Used in Clinical Trials for Immune Thrombocytopenic Purpura
            • Key Physician Insights on Clinical End Points
            • Expert Insight on Clinical End Points
          • Key Current Therapies
            • Overview
            • Mechanism of Action of Key Current Drug Classes Used for Immune Thrombocytopenic Purpura
            • Current Treatments Used for Immune Thrombocytopenic Purpura
            • Advantages and Disadvantages of Glucocorticoids
            • Expert Insight: Glucocorticoids
            • Clinical Trial Outcomes for Intravenous Immunoglobulins
            • Advantages and Disadvantages of Intravenous Immunoglobulins
            • Expert Insight: Intravenous Immunoglobulins
            • Clinical Trial Outcomes for Anti-RhD Immunoglobulins
            • Advantages and Disadvantages of Anti-RhD Immunoglobulins
            • Expert Insight: Anti-RhD Immunoglobulins
            • Expert Insight: Thrombopoietin-Receptor Agonists
            • Key Results from Select Clinical Trials Investigating Eltrombopag for the Treatment of Immune Thombocytopenic Purpura
            • Ongoing Clinical Development of Eltrombopag
            • Advantages and Disadvantages of Eltrombopag
            • Expert Insight: Eltrombopag
            • Key Results from Select Clinical Trials Investigating Romiplostim for the Treatment of Immune Thrombocytopenic Purpura
            • Ongoing Clinical Development of Romiplostim
            • Advantages and Disadvantages of Romiplostim
            • Expert Insight: Romiplostim
            • Clinical Trial Outcomes for Rituximab
            • Advantages and Disadvantages of Rituximab
            • Expert Insight: Rituximab
            • Advantages and Disadvantages of Splenectomy
            • Expert Insight on Splenectomy
          • Medical Practice
            • Overview
            • Treatment Guidelines
            • Immune Thrombocytopenic Purpura Treatment Guidelines
            • Factors Influencing Drug Selection in Immune Thrombocytopenic Purpura
            • Generalized Treatment Decision Tree for Immune Thrombocytopenic Purpura
        • Unmet Need Overview
          • Current and Future Attainment of Unmet Needs in Immune Thrombocytopenic Purpura
          • Top Unmet Needs in Immune Thrombocytopenic Purpura: Current and Future Attainment
          • Expert Insight: Unmet Need in Immune Thrombocytopenic Purpura
        • Emerging Therapies
          • Key Findings
            • Expert Insight
            • Pipeline Trends for Immune Thrombocytopenic Purpura
          • Key Emerging Therapies
            • Notable Developments Among Key Emerging Therapies for Immune Thrombocytopenic Purpura
            • Key Therapies in Development for Immune Thrombocytopenic Purpura
            • Estimated Launch Dates of Key Emerging Therapies for the Treatment of Immune Thrombocytopenic Purpura
            • Select Clinical Trial Results for Tavalisse in the Treatment of Immune Thrombocytopenic Purpura
            • Analysis of the Clinical Development Program for Tavalisse
            • Key Ongoing Clinical Trials of Tavalisse in the Treatment of Immune Thrombocytopenic Purpura
            • Expert Insight: Tavalisse
            • Expert Insight: Tavalisse
            • Expectations for Launch and Sales Opportunity of Tavalisse in Immune Thrombocytopenic Purpura
            • Select Clinical Trial Results for Avatrombopag in the Treatment of Immune Thrombocytopenic Purpura
            • Expert Insight: Avatrombopag
            • Expert Insight: Avatrombopag
            • Expectations for Launch and Sales Opportunity of Avatrombopag in Immune Thrombocytopenic Purpura
          • Early-Phase Pipeline Analysis
            • Notable Developments in the Early-Phase Pipeline for Immune Thrombocytopenic Purpura
            • Select Early-Phase Compounds in Development for Immune Thrombocytopenic Purpura
            • Expert Insight: Early-Phase Pipeline for Immune Thrombocytopenic Purpura
          • Patient Registries
            • Patient Registries for Immune Thrombocytopenic Purpura
            • Prominent Immune Thrombocytopenic Purpura Patient Organizations
          • Orphan Drug Designation
          • Access and Reimbursement Overview
            • Region-Specific Reimbursement Practices
              • Key Market Access Considerations in Immune Thrombocytopenic Purpura: United States
              • Key Market Access Considerations in Immune Thrombocytopenic Purpura: EU5
              • Looking for More?
          • Methodology
            • Bottom-Up Forecasting Overview
              • Patient Populations
              • Drug-Specific Assumptions
            • Bottom-Up Forecast Assumptions
              • General Sources of Data
              • Drug-Treatment Rate Assumptions in Immune Thrombocytopenic Purpura
              • Patient-Share Assumptions in Immune Thrombocytopenic Purpura
              • General Statements About Pricing
              • Sources for Drug Prices
              • 2017 Exchange Rates
              • Price Per Treated Day: 2017
              • Price per Treated Day: 2027
              • Dosing, Days of Therapy, and Compliance
              • Generic Erosion
              • Generic Erosion Assumptions
              • Biosimilar Erosion
              • Biosimilar Erosion Assumptions
              • Out-Year Forecasting
              • Emerging Therapy Prices
            • Primary Market Research
              • Experts Interviewed
          • Appendix
            • Key Abbreviations Related to Immune Thrombocytopenic Purpura
            • Brands, Marketers, and Generic Availability of Key Therapies for Immune Thrombocytopenic Purpura
            • Immune Thrombocytopenic Purpura Bibliography

        Author(s): Akash Saini, Ph.D.; Abey John, MPH

        Akash is a Principal Analyst in the China In-Depth team at Decision Resources Group. Since 2016, he has specialized in a range of indications including rare disease indications such as DMD, ALS, ITP, IPF, JIA, and retinitis pigmentosa. He has authored Disease Landscape & Forecast reports, Access & Reimbursement reports, and Treatment Algorithm reports based on primary market research and real-world evidence. Prior to joining DRG, Akash was a post-doctoral fellow at the University of Massachusetts Medical School. He has a Ph.D. from the International Centre for Genetic Engineering and Biotechnology, New Delhi.

        Abey John is a medical graduate with a Master’s in Public Health and has been associated with DRG since September 2015. He works with a global team of epidemiologists in performing systematic reviews of assigned diseases and prepare forecast models for clients. He also is involved in producing analyses for pharmaceutical drug developers on the descriptive epidemiology of major drug indications in mature and developing markets and have an overall experience of three years working in different healthcare sectors across the country.

        Prior to joining DRG, Abey had been working with Jhpiego (an affiliate of Johns Hopkins Medical University) in implementing Family Planning Health Programs in India with collaborating with the Government of India. He also has worked with a grassroots level NGO as a health team manager which worked for the benefit of the rural population living in the foothills of the Himalayas.