Pompe Disease: Opportunity Analysis and Forecasts to 2027

1 Table of Contents

1.1 List of Tables

1.2 List of Figures

2 Pompe Disease: Executive Summary

2.1 Growth of the Pompe Disease Market Is Expected from 2017–2027

2.2 High Unmet Need for Earlier Pompe Disease Diagnosis

2.3 Pipeline Drugs for Pompe Disease Treatment

2.4 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

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

5.4.3 Diagnosed Incident Cases of Pompe Disease – Based on International Registry Data

5.4.4 Diagnosed Incident Cases of Pompe Disease by Type – Based on International Registry Data

5.4.5 Diagnosed Incident Cases of LOPD by Severity – Based on International Registry Data

5.4.6 Diagnosed Incident Cases of IOPD by CRIM Status – Based on International Registry Data

5.4.7 Diagnosed Prevalent Cases of Pompe Disease – Based on International Registry Data

5.4.8 Diagnosed Prevalent Cases of Pompe Disease – Method-1

5.4.9 Diagnosed Prevalent Cases of Pompe Disease – Method-2

5.4.10 Forecast Adjusted for the Underestimation of International Registry Data

5.5 Epidemiological Forecast for Pompe Disease (2017–2027) – Based on International Registry Data

5.5.1 Diagnosed Incident Cases of Pompe Disease

5.5.2 Sex-Specific Diagnosed Incident Cases of Pompe Disease

5.5.3 Diagnosed Incident Cases of Pompe Disease by Type

5.5.4 Diagnosed Incident Cases of LOPD by Severity

5.5.5 Diagnosed Incident Cases of IOPD by CRIM Status

5.5.6 Diagnosed Prevalent Cases of Pompe Disease – Method-1

5.5.7 Diagnosed Prevalent Cases of Pompe Disease – Method-2

5.6 Epidemiological Forecast for Pompe Disease (2017–2027) – Adjusted for the Underestimation of International Registry Data

5.6.1 Diagnosed Incident Cases of Pompe Disease

5.6.2 Sex-Specific Diagnosed Incident Cases of Pompe Disease

5.6.3 Diagnosed Incident Cases of Pompe Disease by Type

5.6.4 Diagnosed Incident Cases of LOPD by Severity

5.6.5 Diagnosed Incident Cases of IOPD by CRIM Status

5.6.6 Diagnosed Prevalent Cases of Pompe Disease – Method-1

5.6.7 Diagnosed Prevalent Cases of Pompe Disease – Method-2

5.7 Discussion

5.7.1 Epidemiological Forecast Insight

5.7.2 Limitations of the Analysis

5.7.3 Strengths of the Analysis

6 Current Treatment Options

6.1 Overview

7 Unmet Needs and Opportunity Assessment

7.1 Overview

7.2 Earlier Diagnosis

7.3 Therapies with Alternative MoA

7.4 Treatments with Improved Efficacy

7.5 Treatment with a Less Invasive Route of Administration

8 R&D Strategies

8.1 Overview

8.1.1 Novel Enzyme Replacement Therapies

8.1.2 Gene Therapy

8.2 Clinical Trials Design

8.2.1 Clinical Trial Treatment Periods

9 Pipeline Assessment

9.1 Overview

9.2 Other Drugs in Development

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

10.4.4 Brazil

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

11.4 Primary Research – KOLs Interviewed for This Report

11.4.1 KOLs

11.5 About the Authors

11.5.1 Analyst

11.5.2 Managing Analyst

11.5.3 Therapy Area Director

11.5.4 Epidemiologist

11.5.5 Managing Epidemiologist

11.5.6 Global Director of Therapy Analysis and Epidemiology

11.5.7 Global Head and EVP of Healthcare Operations and Strategy

11.6 About GlobalData

11.7 Contact Us

11.8 Disclaimer

Table 1: Pompe Disease: Key Metrics in the 8MM 2017–2027

Table 2: Risk Factors for Pompe Disease

Table 3: Treatment Guidelines for Pompe Disease

Table 4: Comparison of Therapeutic Classes in Development for Pompe Disease, 2016–2027

Table 5: Drugs in Development for Pompe Disease, 2018

Table 6: Clinical Benchmark of Key Pipeline Drugs – PD Treatments

Table 7: Commercial Benchmark of Key Pipeline Drugs – Pompe Disease

Table 8: Key Events Impacting Sales for Pompe Disease, 2017–2027

Table 9: Pompe Disease Market – Drivers and Barriers, 2017–2027

Table 10: Key Historical and Projected Launch Dates for PD

Figure 1: Global Sales Forecast by Country for Pompe Disease in 2017 and 2027

Figure 2: Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against the SOC, Myozyme

Figure 3: Simultaneous Accumulation of Unfolded Proteins and Interventions

Figure 4: Classification of Pompe Disease

Figure 5: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of Pompe Disease

Figure 6: 8MM, Sources Used to Forecast the Diagnosed Prevalent Cases of Pompe Disease

Figure 7: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of Pompe Disease by Type

Figure 8: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of LOPD by Severity

Figure 9: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of IOPD by CRIM Status

Figure 10: 8MM, Diagnosed Incident Cases of Pompe Disease, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)

Figure 11: 8MM, Sex-Specific Diagnosed Incident Cases of Pompe Disease, N, All Ages, 2017 (Forecast Based on International Registry Data)

Figure 12: 8MM, Diagnosed Incident Cases of Pompe Disease by Type, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)

Figure 13: 8MM, Diagnosed Incident Cases of LOPD by Severity, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)

Figure 14: 8MM, Diagnosed Incident Cases of IOPD by CRIM Status, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)

Figure 15: 8MM, Diagnosed Prevalent Cases of Pompe Disease – Method-1, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)

Figure 16: 8MM, Diagnosed Prevalent Cases of Pompe Disease – Method-2, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)

Figure 17: 8MM, Diagnosed Incident Cases of Pompe Disease, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)

Figure 18: 8MM, Sex-Specific Diagnosed Incident Cases of Pompe Disease, N, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)

Figure 19: 8MM, Diagnosed Incident Cases of Pompe Disease by Type, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)

Figure 20: 8MM, Diagnosed Incident Cases of LOPD by Severity, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)

Figure 21: 8MM, Diagnosed Incident Cases of IOPD by CRIM Status, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)

Figure 22: 8MM, Diagnosed Prevalent Cases of Pompe Disease – Method-1, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)

Figure 23: 8MM, Diagnosed Prevalent Cases of Pompe Disease – Method-2, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)

Figure 24: Unmet Needs and Opportunities in Pompe Disease

Figure 25: Overview of the Development Pipeline in Pompe Disease

Figure 26: Key Phase III Trial for a Promising Novel ERT that GlobalData Expects be Licensed for Pompe Disease in the 8MM During the Forecast Period

Figure 27: Competitive Assessment of the Pipeline Drug Benchmarked Against the SOC, Myozyme

Figure 28: Global (8MM) Sales Forecast by Country for Pompe Disease in 2017 and 2027