Pompe Disease – Opportunity Assessment and Forecast to 2030

Pompe Disease (PD), also known as glycogen storage disease II, is a recessive genetic metabolic disorder caused by a deficiency in the enzyme α-glucosidase (GAA). GAA is normally responsible for hydrolyzing α-1, 4 and α-1, 6 linkages in various carbohydrates, including maltose, isomaltose, and glycogen. The absence or deficiency of GAA results in excessive storage of glycogen in cellular lysosomes. This inhibition can lead to cellular stress and injury. In PD, cardiac muscle tissue is severely affected alongside neurons in both the autonomic nervous system (ANS) and central nervous system (CNS).


GlobalData's Pompe Disease – Opportunity Assessment and Forecast combines data from the Pharma Intelligence Center with in-house analyst expertise to provide a competitive assessment of the disease marketplace.

Key Highlights

Report deliverables include a PowerPoint report and Excel-based forecast model

Forecasts include three countries

Forecasts cover three time points: base year, 5-year, and 10-year

Reasons to buy

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.

Develop business strategies by understanding the trends shaping and driving the global PD market.

Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global RVO market in the future.

Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.

Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.

Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

Companies mentioned

Genzyme, Sanofi, Amicus Therapeutics, AskBio, Spark Therpeutics, Astellas Gene Therapy

Table of Contents

1 Executive Summary

1.1 Executive Summary

2 Disease Overview

2.1 Definition of Pompe Disease

2.2 Pompe SWOT Analysis

2.3 Etiology and Pathophysiology

3 Epidemiology

3.1 Diagnosed Prevalent Cases of PD, 2020–2030

3.2 Diagnosed Prevalent Cases of PD by Specific Type, 2020

3.3 Sources and Methodology

3.4 Diagnosis of Pompe Disease – Treatable Population

4 Current Treatment Options

4.1 Treatment Paradigm

4.2 Marketed Products

4.3 Product Profile: Genzyme and Sanofi's Algucosidase alfa (Myozyme)

4.4 Product Profile: Genzyme and Sanofi's Algucosidase alfa (Lumizyme)

5 Unmet Needs and Opportunities

5.1 Unmet Needs in PD

5.2 Earlier Disease Diagnosis

5.3 Therapies that Restore GAA Expression in the CNS

5.4 Treatment with Improved Efficacy

5.5 Treatment with a Less Invasive Route of Administration

5.6 Reduced Cost annd Improved Criteria for Treatment Access

6 Pipeline Assessment

6.1 Pompe Disease: Pipeline Overview

6.2 Leading Pipeline Agents

6.3 Product Profile: avalglucosidase alfa

6.4 Product Profile: cipaglucosidase alfa

6.5 Product Profile: ACTUS-101

6.6 Product Profile: AVR-SPK-3006

6.7 Product Profile: AT-845

6.8 Pipeline Products – Review Designations

6.9 Pompe Disease: Clinical Trials Overview

7 R&D Strategies

7.1 Trends in Clinical Trial Design in Pompe Disease

7.2 Trends in Deal-Making in Pompe Disease

8 Market Outlook

8.1 Pompe Disease Market Growth

8.2 Pompe Disease Market Forecast

8.3 Market Drivers and Barriers

9 Appendix

9.1 Primary Research: KOL Information

9.2 About the Authors

10 Contact Us

List of Figures

Figure 1: Summary of Key Findings


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