OpportunityAnalyzer: Immune Thrombocytopenia (ITP) – Opportunity Analysis and Forecasts to 2025

1 Table of Contents

1.1 List of Tables

1.2 List of Figures

2 Introduction

2.1 Catalyst

2.2 Related Reports

2.3 Upcoming Related Reports

3 Disease Overview

3.1 Etiology and Pathophysiology

3.1.1 Etiology

3.1.2 Pathophysiology

3.2 Phases and Severity of ITP

3.3 Symptoms

3.4 Prognosis

3.5 Quality of Life

4 Epidemiology

4.1 Disease Background

4.2 Risk Factors and Comorbidities

4.3 Global Trends

4.3.1 US

4.3.2 5EU

4.3.3 Japan

4.4 Forecast Methodology

4.4.1 Sources Used Tables

4.4.2 Forecast Assumptions and Methods

4.4.3 Sources Not Used

4.5 Epidemiological Forecast for ITP (2015–2025)

4.5.1 Diagnosed Prevalent Cases of ITP

4.5.2 Age-Specific Diagnosed Prevalent Cases of ITP

4.5.3 Sex-Specific Diagnosed Prevalent Cases of ITP

4.5.4 Age-Standardized Diagnosed Prevalence of ITP

4.5.5 Diagnosed Incident Cases of ITP

4.5.6 Age-Specific Diagnosed Incident Cases of ITP

4.5.7 Sex-Specific Diagnosed Incident Cases of ITP

4.5.8 Age-Standardized Diagnosed Incidence of ITP

4.6 Discussion

4.6.1 Epidemiological Forecast Insight

4.6.2 Limitations of the Analysis

4.6.3 Strengths of the Analysis

5 Current Treatment Options

5.1 Overview

5.2 Diagnosis and Treatment

5.2.1 Diagnosis

5.2.2 Treatment Guidelines and Leading Prescribed Drugs

5.3 Clinical Practice

5.3.1 Decision on the Initiation of Treatment

5.3.2 First-Line Treatment

5.3.3 Second-Line Therapy

5.4 Product Profiles – Thrombopoietin Receptor Agonists

5.4.1 Nplate (Romiplostim)

5.4.2 Promacta/Revolade (Eltrombopag)

6 Unmet Needs Assessment and Opportunity Analysis

6.1 Overview

6.2 Safer and More Efficacious First-Line Therapy

6.2.1 Unmet Need

6.2.2 Gap Analysis

6.2.3 Opportunity

6.3 High Treatment Cost of Thrombopoietin Receptor Agonists

6.3.1 Unmet Need

6.3.2 Gap Analysis

6.3.3 Opportunity

6.4 Second-Line Therapeutic Options that Offer Long-Term Remission

6.4.1 Unmet Need

6.4.2 Gap Analysis

6.4.3 Opportunity

6.5 New Treatment for Multiple Drug Refractory Patients

6.5.1 Unmet Need

6.5.2 Gap Analysis

6.5.3 Opportunity

6.6 Difficulties in Clinical Trial Patient Recruitment

6.6.1 Unmet Need

6.6.2 Gap Analysis

6.6.3 Opportunity

7 Research and Development Strategies

7.1 Overview

7.1.1 Enlarging Eligible Patient Pool Through Label Expansion to Pediatric Patients

7.1.2 Targeting New Mechanisms of Action to Increase Competitiveness

7.2 Clinical Trial Design

7.2.1 Historical Lack of Consistency in Patient Selection Criteria

7.2.2 Inconsistent Timeframe for Endpoint Measurement and Study Duration

7.2.3 Selection of Suitable Comparator

7.2.4 Other Clinical Trial Requirements

8 Pipeline Assessment

8.1 Overview

8.2 Promising Drugs in Clinical Development

8.2.1 Avatrombopag

8.2.2 Fostamatinib (R-788)

8.3 Innovative Early-Stage Approaches

8.3.1 Overview

8.3.2 CD40/CD40L Antagonists

8.3.3 Anti-CD20 Monoclonal Antibodies

8.3.4 Thrombopoietin Receptor Agonists

8.3.5 Syk Inhibitors

8.3.6 Other Approaches

8.4 Biosimilars

8.5 Other Drugs in Development

9 Pipeline Valuation Analysis

9.1 Clinical Benchmark of Key Pipeline Drugs

9.2 Commercial Benchmark of Key Pipeline Drugs

9.3 Competitive Assessment

9.4 Top-Line 10-Year Forecast

9.4.1 US

9.4.2 5EU

9.4.3 Japan

10 Appendix

10.1 Bibliography

10.2 Abbreviations

10.3 Methodology

10.4 Forecasting Methodology

10.4.1 Diagnosed Immune Thrombocytopenia Patients

10.4.2 Percent Drug-Treated Patients

10.4.3 Drugs Included in Each Therapeutic Class

10.4.4 Launch and Patent Expiry Dates

10.4.5 General Pricing Assumptions

10.4.6 Individual Drug Assumptions

10.4.7 Generic Erosion

10.4.8 Pricing of Pipeline Agents

10.5 Primary Research – KOLs Interviewed for this Report

10.6 Primary Research – Prescriber Survey

10.7 About the Authors

10.7.1 Analyst

10.7.2 Therapy Area Directors

10.7.3 Epidemiologists

10.7.4 Epidemiology Reviewer

10.7.5 Director of Epidemiology

10.7.6 Global Director of Therapy Analysis and Epidemiology

10.8 About GlobalData

10.9 Contact Us

10.10 Disclaimer

Table 1: Secondary Causes of ITP

Table 2: Phases and Severity of ITP

Table 3: Symptoms of ITP

Table 4: Risk Factors and Comorbidities for ITP

Table 5: 7MM, Sources for Diagnosed Prevalence of Primary ITP

Table 6: 7MM, Sources for Diagnosed Prevalence of Secondary ITP

Table 7: 7MM, Sources for Diagnosed Incidence of Primary ITP

Table 8: 7MM, Sources for Diagnosed Incidence of Secondary ITP

Table 9: 7MM, Sources Not Used in the Epidemiological Analysis of ITP

Table 10: 7MM, Diagnosed Prevalent Cases of Primary ITP, All Ages, Men and Women, Select Years, 2015–2025

Table 11: 6MM, Diagnosed Prevalent Cases of Secondary ITP, All Ages, Men and Women, Select Years, 2015–2025

Table 12: 7MM, Age-Specific Diagnosed Prevalent Cases of Primary ITP, Men and Women, N (Row %), 2015

Table 13: 6MM, Age-Specific Diagnosed Prevalent Cases of Secondary ITP, Men and Women, N (Row %), 2015

Table 14: 7MM, Sex-Specific Diagnosed Prevalent Cases of Primary ITP, All Ages, N (Row %), 2015

Table 15: 6MM, Sex-Specific Diagnosed Prevalent Cases of Secondary ITP, All Ages, N, 2015

Table 16: 7MM, Diagnosed Incident Cases of Primary ITP, All Ages, Men and Women, Select Years, 2015–2025

Table 17: 6MM, Diagnosed Incident Cases of Secondary ITP, All Ages, Men and Women, Select Years, 2015–2025

Table 18: 7MM, Age-Specific Diagnosed Incident Cases of Primary ITP, Men and Women, N (Row %), 2015

Table 19: 6MM, Age-Specific Diagnosed Incident Cases of Secondary ITP, Men and Women, N (Row %), 2015

Table 20: 7MM, Sex-Specific Diagnosed Incident Cases of Primary ITP, All Ages, N (Row %), 2015

Table 21: 6MM, Sex-Specific Diagnosed Incident Cases of Secondary ITP, All Ages, N (Row %), 2015

Table 22: Non-Immunologic Causes of Thrombocytopenia

Table 23: Clinical Guidelines for ITP

Table 24: Leading Treatments for ITP, 2016

Table 25: Corticosteroid Therapy for First-Line ITP Treatment

Table 26: Efficacy of Rituxan in the Second-Line Setting

Table 27: Other Second-Line Options for ITP Treatment

Table 28: Product Profile – Nplate

Table 29: Efficacy of Nplate in Pivotal Trials

Table 30: Long-Term Efficacy of Nplate in Open-Label Studies

Table 31: Efficacy of Nplate Compared with SOC in Non-Splenectomized Patients

Table 32: Efficacy of Nplate in the NCT01444417 Trial

Table 33: Summary of AEs from the Two Pivotal Trials of Nplate

Table 34: Summary of AEs in the NCT01444417 Trial

Table 35: Nplate SWOT Analysis, 2016

Table 36: Product Profile – Promacta

Table 37: Efficacy of Promacta in the NCT00102739 Trial

Table 38: Efficacy of Promacta in the RAISE Trial

Table 39: Efficacy of Promacta in the PETIT Trial

Table 40: Age-Stratified Efficacy of Promacta in the PETIT Trial

Table 41: Efficacy of Promacta in the PETIT2 Trial

Table 42: Age-Stratified Efficacy of Promacta in the PETIT2 Trial

Table 43: Summary of AEs from the Pivotal Trials of Promacta in Adults

Table 44: Summary of AEs from the Pivotal Trials of Promacta in Children

Table 45: Promacta SWOT Analysis, 2016

Table 46: Unmet Need and Opportunity in ITP, 2016

Table 47: Phase III Pipeline Clinical Trial Design for ITP

Table 48: Key Late-Stage Pipeline Agents for ITP, 2016

Table 49: Product Profile – Avatrombopag

Table 50: Efficacy of Avatrombopag in the Amendment 02 Trial

Table 51: Efficacy of Avatrombopag in the NCT00441090 Trial

Table 52: Summary of AEs from Two Phase II Trials of Avatrombopag

Table 53: Avatrombopag SWOT Analysis, 2016

Table 54: Product Profile – Fostamatinib

Table 55: Efficacy of Fostamatinib in ITP

Table 56: Safety of Fostamatinib in ITP

Table 57: Fostamatinib SWOT Analysis, 2016

Table 58: Early-Stage Clinical and Preclinical ITP Pipeline

Table 59: Discontinued or Inactive ITP Pipeline

Table 60: Clinical Benchmark of Key Pipeline Drugs – ITP

Table 61: Commercial Benchmark of Key Pipeline Drugs – ITP

Table 62: Top-Line Sales Forecasts ($M) for ITP, 2015–2025

Table 63: Key Events Impacting Sales for ITP, 2015–2025

Table 64: ITP Market – Global Drivers and Barriers, 2015‒2025

Table 65: Key Historical and Projected Launch Dates for ITP Therapeutics

Table 66: Key Historical and Projected Patent Expiry Dates for ITP Therapeutics

Table 67: Average Adult Body Weight and Surface Area Across the 7MM

Table 68: High-Prescribing Physicians (non-KOLs) Surveyed, By Country

Figure 1: Diagnosed Prevalence Rate for ITP in the 7MM for All Ages, Men and Women, Cases per 100,000 Population, 2015

Figure 2: Diagnosed Incidence Rate for ITP in the 7MM for All Ages, Men and Women, Cases per 100,000 Population, 2015

Figure 3: Case Flow Map

Figure 4: 7MM, Diagnosed Prevalent Cases of Primary ITP Cases, All Ages, Men and Women, N, Select Years, 2015–2025

Figure 5: 6MM, Diagnosed Prevalent Cases of Secondary ITP Cases, All Ages, Men and Women, N, Select Years, 2015–2025

Figure 6: 7MM, Age-Specific Diagnosed Prevalent Cases of Primary ITP, Men and Women, N, 2015

Figure 7: 6MM, Age-Specific Diagnosed Prevalent Cases of Secondary ITP, Men and Women, N, 2015

Figure 8: 7MM, Sex-Specific Diagnosed Prevalent Cases of Primary ITP, All Ages, N, 2015

Figure 9: 6MM, Sex-Specific Diagnosed Prevalent Cases of Secondary ITP, All Ages, N, 2015

Figure 10: 7MM, Age-Standardized Diagnosed Prevalence of Primary ITP, All Ages, Cases per 100,000 population, 2015

Figure 11: 6MM, Age-Standardized Diagnosed Prevalence of Secondary ITP, All Ages, Cases per 100,000 population, 2015

Figure 12: 7MM, Diagnosed Incident Cases of Primary ITP, All Ages, Men and Women, N, Select Years, 2015–2025

Figure 13: 6MM, Diagnosed Incident Cases of Secondary ITP, All Ages, Men and Women, N, Select Years, 2015–2025

Figure 14: 7MM, Age-Specific Diagnosed Incident Cases of Primary ITP, Men and Women, N, 2015

Figure 15: 6MM, Age-Specific Diagnosed Incident Cases of Secondary ITP, Men and Women, N, 2015

Figure 16: 7MM, Sex-Specific Diagnosed Incident Cases of Primary ITP, All Ages, N, 2015

Figure 17: 6MM, Sex-Specific Diagnosed Incident Cases of Secondary ITP, All Ages, N, 2015

Figure 18: 7MM, Age-Standardized Diagnosed Incidence of Primary ITP, All Ages, Cases per 100,000 Population, 2015

Figure 19: 6MM, Age-Standardized Diagnosed Incidence of Secondary ITP, All Ages, Cases per 100,000 Population, 2015

Figure 20: Nplate’s Development in ITP

Figure 21: Development of Promacta in ITP

Figure 22: ITP – Phase III Pipeline, 2016

Figure 23: Avatrombopag’s Development in ITP

Figure 24: Fostamatinib’s Development in ITP

Figure 25: Competitive Assessment of Pipeline Agents in ITP, 2015–2025

Figure 26: Top-Line Sales for ITP by Region, 2015 and 2025

Figure 27: Top-Line Sales for ITP by Region, 2015–2025

Figure 28: Global Sales for ITP by Drug Class, 2015 and 2025

Figure 29: Sales for ITP by Drug Class in the US, 2015 and 2025

Figure 30: Sales for ITP by Drug Class in the 5EU, 2015 and 2025

Figure 31: Sales for ITP by Drug Class in the Japan, 2015 and 2025