OpportunityAnalyzer: Pulmonary Arterial Hypertension – Opportunity Analysis and Forecasts to 2026

Table of Contents

1 Table of Contents

1.1 List of Tables

1.2 List of Figures

2 Executive Summary

2.1 PAH Market Will Exhibit Moderate Growth Between 2016 and 2026

2.2 Upfront and Sequential Dual and Triple Combination Therapies and Launch of Bardoxolone Methyl Will Fuel Market Growth

2.3 Bardoxolone Methyl Has the Potential to Become a Blockbuster Therapy and Fulfill an Unmet Need

2.4 Reformulation of Existing Drugs Will Continue to Dominate the PAH Pipeline

2.5 What Do Physicians Think?

3 Introduction

3.1 Catalyst

3.2 Related Reports

4 Disease Overview

4.1 Etiology and Pathophysiology

4.1.1 Etiology

4.1.2 Pathophysiology

4.2 Symptoms

4.3 Diagnosis

4.4 Prognosis

4.5 Quality of Life

5 Epidemiology

5.1 Disease Background

5.2 Risk Factors and Comorbidities

5.3 Global and Historical Trends

5.4 Forecast Methodology

5.4.1 Sources

5.4.2 Forecast Assumptions and Methods

5.5 Epidemiological Forecast for PAH (2016–2026) – Based on Registry Data

5.5.1 Diagnosed Incident Cases of PAH (Forecast Based on Registry Data)

5.5.2 Diagnosed Prevalent Cases of PAH (Forecast Based on Registry Data)

5.5.3 Diagnosed Prevalent Cases of PAH by NYHA Functional Classes (Forecast Based on Registry Data)

5.5.4 Comorbidities Among the Diagnosed Prevalent Cases of PAH (Forecast Based on Registry Data)

5.6 Epidemiological Forecast for PAH (2016–2026) – Adjusted for Underestimation of Registry Data

5.6.1 Diagnosed Incident Cases of PAH (Forecast Adjusted for Underestimation of Registry Data)

5.6.2 Age-Specific Diagnosed Incident Cases of PAH (Forecast Adjusted for Underestimation of Registry Data)

5.6.3 Sex-Specific Diagnosed Incident Cases of PAH (Forecast Adjusted for Underestimation of Registry Data)

5.6.4 Diagnosed Prevalent Cases of PAH (Forecast Adjusted for Underestimation of Registry Data)

5.6.5 Age-Specific Diagnosed Prevalent Cases of PAH (Forecast Adjusted for Underestimation of Registry Data)

5.6.6 Sex-Specific Diagnosed Prevalent Cases of PAH (Forecast Adjusted for Underestimation of Registry Data)

5.6.7 Diagnosed Prevalent Cases of PAH by NYHA Functional Class I–IV (Forecast Adjusted for Underestimation of Registry Data)

5.6.8 Comorbidities Among the Diagnosed Prevalent Cases of PAH (Forecast Adjusted for Underestimation of Registry Data)

5.7 Discussion

5.7.1 Epidemiological Forecast Insight

5.7.2 Limitations of Analysis

5.7.3 Strengths of Analysis

6 Current Treatment Options

6.1 Overview

6.2 Diagnosis and Treatment

6.3 Clinical Practice

6.4 Product Profiles – Endothelin Receptor Antagonists

6.4.1 Tracleer and Generics (bosentan)

6.4.2 Volibris/Letairis (ambrisentan)

6.4.3 Opsumit (macitentan)

6.5 Product Profiles – Phosphodiesterase Type 5 Inhibitors

6.5.1 Revatio and Generics (sildenafil citrate)

6.5.2 Adcirca (tadalafil)

6.6 Product Profiles – Soluble Guanylate Cyclase Stimulators

6.6.1 Adempas (riociguat)

6.7 Product Profiles – Prostacyclin Derivatives

6.7.1 Flolan and Generics (epoprostenol)

6.7.2 Veletri (epoprostenol)

6.7.3 Ventavis (iloprost)

6.7.4 Remodulin (treprostinil)

6.7.5 Tyvaso (treprostinil)

6.7.6 Orenitram (treprostinil)

6.8 Product Profiles – Prostacyclin IP Receptor Agonists

6.8.1 Uptravi (selexipag)

7 Unmet Needs and Opportunity Assessment

7.1 Overview

7.2 Novel Curative or Stabilizing Therapeutics

7.2.1 Unmet Need

7.2.2 Gap Analysis

7.2.3 Opportunity

7.3 Improved Efficacy

7.3.1 Unmet Need

7.3.2 Gap Analysis

7.3.3 Opportunity

7.4 Biomarkers and Assays to Tailor Treatment

7.4.1 Unmet Need

7.4.2 Gap Analysis

7.4.3 Opportunity

7.5 Drugs to Prevent Right Heart Failure

7.5.1 Unmet Need

7.5.2 Gap Analysis

7.5.3 Opportunity

7.6 Anti-inflammatory Therapies

7.6.1 Unmet Need

7.6.2 Gap Analysis

7.6.3 Opportunity

8 R&D Strategies

8.1 Overview

8.1.1 Reformulation of Existing PAH Drugs

8.1.2 Repurposing of Known Drugs from Other Therapy Areas

8.1.3 Novel Anti-inflammatory and Anti-proliferative Therapeutic Targets

8.1.4 Licensing Agreements and Acquisitions

8.2 Clinical Trials Design

8.2.1 Current Clinical Trial Design

8.2.2 Future Clinical Trial Design

9 Pipeline Assessment

9.1 Overview

9.2 Promising Drugs in Clinical Development

9.3 Promising Drugs in Clinical Development, Prostacyclin Derivatives

9.3.1 Trevyent (treprostinil diolamine)

9.3.2 Tysuberprost (esuberaprost sodium + treprostinil)

9.4 Promising Drugs in Clinical Development, Prostacyclin IP Receptor Agonists

9.4.1 Ralinepag

9.5 Promising Drugs in Clinical Development, Antioxidant Inflammation Modulators

9.5.1 Bardoxolone Methyl

9.6 Innovative Early-Stage Approaches

10 Pipeline Valuation Analysis

10.1 Clinical Benchmark of Key Pipeline Drugs

10.2 Commercial Benchmark of Key Pipeline Drugs

10.3 Competitive Assessment

10.4 Top-Line 10-Year Forecast

10.4.1 US

10.4.2 5EU

10.4.3 Japan

11 Appendix

11.1 Bibliography

11.2 Abbreviations

11.3 Methodology

11.3.1 Forecasting Methodology

11.3.2 Diagnosed Patients

11.3.3 Percent Drug-Treated Patients

11.3.4 Drugs Included in Each Therapeutic Class

11.3.5 Launch and Patent Expiry Dates

11.3.6 General Pricing Assumptions

11.3.7 Individual Drug Assumptions

11.3.8 Generic Erosion

11.3.9 Pricing of Pipeline Agents

11.4 Primary Research – KOLs Interviewed for This Report

11.5 Primary Research – Prescriber Survey

11.6 About the Authors

11.6.1 Analyst

11.6.2 Therapy Area Director

11.6.3 Epidemiologist

11.6.4 Director of Epidemiology

11.6.5 Global Director of Therapy Analysis and Epidemiology

11.6.6 Global Head of Healthcare

11.7 About GlobalData

11.8 Contact Us

11.9 Disclaimer

Table 1: Pulmonary Arterial Hypertension Key Metrics in the 7MM

Table 2: NYHA/WHO Functional Classification of PAH

Table 3: WHO Guidelines, Modified NYHA Functional Classes I–IV

Table 4: Risk Factors and Comorbidities for PAH

Table 5: 7MM, Diagnosed Incident Cases of PAH, Both Sexes, All Ages, N, Selected Years 2016–2026 (Based on Registry Data)

Table 6: 7MM, Diagnosed Prevalent Cases of PAH, Both Sexes, All Ages, N, Selected Years 2016–2026 (Forecast Based on Registry Data).

Table 7: 7MM, Diagnosed Incident Cases of PAH, Both Sexes, All Ages, N, Selected Years 2016–2026 (Forecast Adjusted for Underestimation of Registry Data)

Table 8: 7MM, Diagnosed Prevalent Cases of PAH, Both Sexes, All Ages, N, Selected Years 2016–2026 (Forecast Adjusted for Underestimation of Registry Data)

Table 9: Treatment Guidelines for PAH

Table 10: Most Prescribed Drugs for PAH by Class in the Global Markets, 2017

Table 11: Product Profile – Tracleer

Table 12: Tracleer SWOT Analysis, 2017

Table 13: Product Profile – Volibris/Letairis

Table 14: Volibris/Letairis SWOT Analysis, 2017

Table 15: Product Profile – Opsumit

Table 16: Opsumit SWOT Analysis, 2017

Table 17: Product Profile – Revatio

Table 18: Revatio SWOT Analysis, 2017

Table 19: Product Profile – Adcirca

Table 20: Adcirca SWOT Analysis, 2017

Table 21: Product Profile – Adempas

Table 22: Adempas SWOT Analysis, 2017

Table 23: Product Profile – Flolan

Table 24: Flolan SWOT Analysis, 2017

Table 25: Product Profile – Veletri

Table 26: Veletri SWOT Analysis, 2017

Table 27: Product Profile – Ventavis

Table 28: Ventavis SWOT Analysis, 2017

Table 29: Product Profile – Remodulin

Table 30: Remodulin SWOT Analysis, 2017

Table 31: Product Profile – Tyvaso

Table 32: Tyvaso SWOT Analysis, 2017

Table 33: Product Profile – Orenitram

Table 34: Orenitram SWOT Analysis, 2017

Table 35: Product Profile – Uptravi

Table 36: Uptravi SWOT Analysis, 2017

Table 37: Examples of Endpoints Used in the Pivotal Trials for Approved Treatments for PAH

Table 38: PAH – Late-Stage Clinical Pipeline, 2017

Table 39: Product Profile – Trevyent

Table 40: Trevyent SWOT Analysis, 2017

Table 41: Product Profile – Tysuberprost

Table 42: Tysuberprost SWOT Analysis, 2017

Table 43: Product Profile – Ralinepag

Table 44: Ralinepag SWOT Analysis, 2017

Table 45: Product Profile – Bardoxolone Methyl

Table 46: Bardoxolone Methyl SWOT Analysis, 2017

Table 47: Drugs in Development for PAH, 2016

Table 48: Clinical Benchmark of Key Pipeline Drugs – Prostacyclin Derivatives

Table 49: Clinical Benchmark of Key Pipeline Drugs – Prostacyclin IP Receptor Agonists

Table 50: Commercial Benchmark of Key Pipeline Drugs – Prostacyclin Derivatives

Table 51: Commercial Benchmark of Key Pipeline Drugs – Prostacyclin IP Receptor Agonists

Table 52: Key Events Impacting Sales for PAH, 2016–2026

Table 53: PAH Market – Global Drivers and Barriers, 2016–2026

Table 54: Key Historical and Projected Launch Dates for PAH

Table 55: Key Historical and Projected Patent Expiry Dates for PAH

Table 56: High-Prescribing Physicians (non-KOLs) Surveyed, By Country

Figure 1: Global Sales Forecast by Country for PAH in 2016 and 2026

Figure 2: Target Pathways of the Currently Marketed Drugs for PAH

Figure 3: Diagnostic Approach to PAH

Figure 4: 7MM, Age-Standardized Diagnosed Incidence of PAH (Cases per 100,000 Population), All Ages, by Sex, 2016 (Forecast Based on Registry Data)

Figure 5: 7MM, Age-Standardized Diagnosed Prevalence of PAH (%), All Ages, by Sex, 2016 (Forecast Based on Registry Data)

Figure 6: 7MM, Sources Used and Not Used for the Diagnosed Incident Cases of PAH and Diagnosed Prevalent Cases of PAH (for Forecast Based on Registry Data and Forecast Adjusted for Underestimation of Registry Data)

Figure 7: 7MM, Sources Used for the Diagnosed Prevalent Cases of PAH* by NYHA Functional Classes I–IV (for Forecast Based on Registry Data and Forecast Adjusted for Underestimation of Registry Data)

Figure 8: 7MM, Sources Used for the Diagnosed Prevalent Cases of PAH with CTD (for Forecast Based on Registry Data and Forecast Adjusted for Underestimation of Registry Data)

Figure 9: 7MM, Sources Used for the Diagnosed Prevalent Cases of PAH with SS (for Forecast Based on Registry Data and Forecast Adjusted for Underestimation of Registry Data)

Figure 10: 7MM, Sources Used for the Diagnosed Prevalent Cases of PAH with Diabetes (for Forecast Based on Registry Data and Forecast Adjusted for Underestimation of Registry Data)

Figure 11: 7MM, Sources Used for the Diagnosed Prevalent Cases of PAH with COPD (for Forecast Based on Registry Data and Forecast Adjusted for Underestimation of Registry Data)

Figure 12: 7MM, Sources Used for the Diagnosed Prevalent Cases of PAH with PT (for Forecast Based on Registry Data and Forecast Adjusted for Underestimation of Registry Data)

Figure 13: 7MM, Sources Used for the Diagnosed Prevalent Cases of PAH with CHD (for Forecast Based on Registry Data and Forecast Adjusted for Underestimation of Registry Data)

Figure 14: 7MM, Diagnosed Prevalent Cases of PAH, by NYHA Functional Class I–IV, Both Sexes, All Ages, N, 2016 (Forecast Based on Registry Data)

Figure 15: 7MM, Comorbidities Among the Diagnosed Prevalent Cases of PAH (N), All Ages, 2016 (Forecast Based on Registry Data)

Figure 16: 7MM, Age-Specific Diagnosed Incident Cases of PAH, Both Sexes, All Ages, N, 2016 (Forecast Adjusted for Underestimation of Registry Data)

Figure 17: 7MM, Sex-Specific Diagnosed Incident Cases of PAH, All Ages, N, 2016 (Forecast Adjusted for Underestimation of Registry Data)

Figure 18: 7MM, Age-Specific Diagnosed Prevalent Cases of PAH, Both Sexes, All Ages, N, 2016 (Forecast Adjusted for Underestimation of Registry Data)

Figure 19: 7MM, Sex-Specific Diagnosed Prevalent Cases of PAH, All Ages, 2016 (Forecast Adjusted for Underestimation of Registry Data)

Figure 20: 7MM, Diagnosed Prevalent Cases of PAH Categorized into NYHA Functional Class I–IV (N), Both Sexes, All Ages, 2016 (Forecast Adjusted for Underestimation of Registry Data)

Figure 21: 7MM, Comorbidities Among the Diagnosed Prevalent Cases of PAH (N), Both Sexes, All Ages, 2016 (Forecast Adjusted for Underestimation of Registry Data)

Figure 22: Unmet Need and Opportunity in PAH, 2017

Figure 23: Overview of the Development Pipeline in PAH

Figure 24: Competitive Assessment of the Marketed and Pipeline Prostacyclin Derivative Drugs Benchmarked Against the SOC, Remodulin

Figure 25: Competitive Assessment of the Marketed and Pipeline Prostacyclin IP Receptor Agonist Drugs Benchmarked Against the SOC, Uptravi

Figure 26: Global (7MM) Sales Forecast by Country for PAH in 2016 and 2026

Figure 27: Global Sales Forecast by Class for PAH in 2016 and 2026

Figure 28: Sales Forecast by Class for PAH in the US in 2016 and 2026