Pompe Disease Market to Grow Rapidly During the Forecast Period (2032) – DelveInsight | Key Companies – Amicus Therapeutics (AT-GAA), Aro Biotherapeutics (ABX1100), Maze Therapeutics (MZE001)

  • PublishedSeptember 17, 2022
Pompe Disease Market to Grow Rapidly During the Forecast Period (2032) - DelveInsight | Key Companies - Amicus Therapeutics (AT-GAA), Aro Biotherapeutics (ABX1100), Maze Therapeutics (MZE001)
Delveinsight Business Research LLP
As per DelveInsight’s assessment, globally, nearly 15+ key pharma and biotech companies are working in the Pompe Disease therapeutics landscape based on different Routes of Administration (RoA), Mechanism of Action (MoA), and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.

The Pompe Disease Market is expected to grow in the coming years owing to the increasing incidence and the expected launch of emerging therapies.

DelveInsight’s “Pompe Disease Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Pompe Disease Market size, share, and trends in the seven major market (7MM) (i.e., the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan).

The Pompe Disease market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market. 

Pompe Disease Market

Pompe Disease: An Overview

Pompe disease, also known as glycogen storage disease type II (GSD II) or “acid maltase deficiency,” is caused by the absence or deficiency of acid alpha-glucosidase (GAA), a lysosomal enzyme that is responsible for the cleavage of the α-1, 4- and α-1, 6-glycosidic bonds of glycogen to glucose.

Pompe disease comes at different ages and with different degrees of severity. The severity of the clinical presentations, the tissue involvement, and the age of onset generally correlate well with the nature of the mutation and the degree of residual enzyme activity. 

Pompe Disease Market Key Facts

• As per Delveinsight’s estimates, a total of 2,556+ Pompe disease cases were reported in 2017.

• Among the EU5 countries, Germany had the highest prevalent population of Pompe Disease with 816 cases, followed by France, which had a prevalence of 683 cases in 2017. On the other hand, Spain had the lowest prevalent population of 496 cases in 2017

• As per Delveinsight’s estimates, in 2017, the prevalent cases of Infantile-onset Pompe Disease and Late-onset Pompe Disease in Japan were found to be 150 and 920 respectively, and are expected to increase by 2032.

Pompe Disease Market

The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Pompe Disease market size by analyzing the impact of current and emerging therapies in the market. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies.

The report details the Pompe Disease market trend for each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, mechanism of action (MoA), competition with other therapies, brand value, and their impact on the market.

Pompe Disease Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of diagnosed patient pool and future trends.

Pompe Disease Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Pompe Disease market or expected to get launched during the study period. The analysis covers Pompe Disease market uptake by drugs, patient uptake by therapies, and sales of each drug. 

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Pompe Disease Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Key Developments in the Pompe Disease Therapeutics Market

• On September 15, 2022, Aro Biotherapeutics announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Drug (RPD) designation for ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in muscle, for the treatment of Pompe disease.

• On August 31, 2022, Maze Therapeutics announced that the US FDA had granted Orphan Drug Designation to MZE001, the company’s investigational product for treating Pompe disease. 

• In May 2021, Amicus Therapeutics announced the completion of a successful Type B Pre-Biologics License Application (BLA) meeting with the US FDA for AT-GAA (cipaglucosidase alfa co-administered with miglustat), for the treatment of Pompe disease.

Learn how the Pompe Disease Market will evolve in the coming years:

Pompe Disease Therapeutics Analysis

Companies across the globe are diligently working toward the development of novel treatment therapies with a considerable amount of success over the years.

There are approx. 15+ key companies which are developing therapies for Pompe Disease. Currently, Amicus Therapeutics is leading the Pompe Disease therapeutics market with its drug candidates in the most advanced stage of clinical development.

Some of the key companies in the Pompe Disease Therapeutic Market include

• Asklepios Biopharmaceutical

• Astellas Pharma Inc

• BioMarin Pharmaceutical

• Amicus Therapeutics

• Spark Therapeutics

• Maze Therapeutics

• Denali Therapeutics

• Rocket Pharmaceuticals

• Audentes Therapeutics

• Sanofi

• Actus

• Roche 

• Valerion 

And many others

Pompe Disease Emerging Drugs Covered in the report include:

• AT-GAA: Amicus Therapeutics

• SPK-3006: Spark Therapeutics

• Avalglucosidase 

• VAL-1221

• ACTUS-101

• AT845

And many more

Further product details are provided in the report. Download the sample Report to get more detailed insights at:

Table of Content (TOC)

1. Key Insights

2. Executive Summary 

3. Pompe Disease Competitive Intelligence Analysis

4. Pompe Disease Market Overview at a Glance

5. Pompe Disease Disease Background and Overview

6. Pompe Disease Patient Journey

7. Pompe Disease Epidemiology and Patient Population

8. Pompe Disease Treatment Algorithm, Current Treatment, and Medical Practices

9. Pompe Disease Unmet Needs

10. Key Endpoints of Pompe Disease Treatment

11. Pompe Disease Marketed Products

12. Pompe Disease Emerging Therapies

13. Pompe Disease Seven Major Market Analysis

14. Attribute Analysis

15. Pompe Disease Market Outlook (7 major markets)

16. Pompe Disease Access and Reimbursement Overview

17. KOL Views on the Pompe Disease Market.

18. Pompe Disease Market Drivers

19. Pompe Disease Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

Download Sample PDF Report –


About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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