Published : Nov-2018
No. of Pages : 86
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
1.1 List of Tables
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
1.2 List of Figures
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
