Companies across the globe are diligently working toward the development of novel treatment therapies, with a considerable amount of success over the years. Currently, major pharma and biotech giants, such as Sanofi, Actus, Amicus, Roche, and others, are developing therapies that will enhance treatment outcomes in Pompe patients. Avalglucosidase alfa (Sanofi), AT-GAA (Amicus Therapeutics), and ACTUS-101 (Actus Therapeutics) are some of the most anticipated emerging therapies that are awaiting launch.
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, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The Pompe Disease market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Pompe Disease: An Overview
Pompe disease (PD), also known as glycogen storage disease type II (GSDII) 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.
The deficiency of the enzyme leads to the accumulation of glycogen in the lysosomes in numerous tissues, but clinical symptoms are primarily due to cardiac and skeletal muscle involvement. The disease is characterized by a wide variety of manifestations, ranging from severe infantile-onset muscle weakness, hypotonia, and hypertrophic cardiomyopathy to a relatively mild, slowly progressive skeletal muscle myopathy in adults.
Pompe Disease Market Key Facts
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As per Delveinsight’s estimates, a total of 12,790 Pompe disease cases were reported in 2020.
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Among the EU5 countries, Germany had the highest prevalent population of Pompe Disease, with 821 cases, followed by France, which had a prevalence of 689 cases in 2020. On the other hand, Spain had the lowest prevalent population of 505 cases in 2020.
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In 2020, the prevalent cases of Infantile-onset Pompe Disease and Late-onset Pompe Disease in Japan were found to be 151 and 926, respectively, and are expected to increase by 2030.
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The European Pompe Consortium (EPOC) consortium covered at least 1,250 Pompe patients of all ages from 9 European countries and hence deduced the prevalence of Pompe Disease in Europe. The prevalence was estimated to be about 1 in 283,000 (1,250/354 million inhabitants, given that all the patients are still alive), as per the findings.
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A study published by Hao et al. titled “The First-year Experience of Newborn Screening for Pompe Disease in California” screened almost half a million babies in its first year (2018–2019) and indicated a birth prevalence of 1 in 25,200 (IOPD and LOPD combined), which was within the range of previously reported prevalence.
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 pipeline therapies. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Pompe Disease market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Pompe Disease Epidemiology Assessment
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 epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and views of key opinion leaders.
The Report Covers the Pompe Disease Epidemiology Segmented as -
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Total Live Birth Cases of Pompe Disease in the 7MM (2019–2032)
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Total Adult Prevalent Cases of Pompe Disease in the 7MM (2019–2032)
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Total Prevalent Cases of Pompe Disease in the 7MM (2019–2032)
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Prevalence of Pompe Disease by Onset Types in the 7MM (2019–2032)
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Incidence of IOPD based on Clinical Phenotype in the 7MM (2019–2032)
Pompe Disease Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the rate of uptake of the potential drugs recently launched in the Pompe Disease market or expected to be launched during the study period. The analysis covers the 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.
Learn How the Pompe Disease Market Will Evolve and Grow by 2032 @
https://www.delveinsight.com/sample-request/pompe-disease-market
Pompe Disease Therapeutics Analysis
Several major pharma and biotech giants are developing therapies for Pompe Disease. Currently, Amicus Therapeutics is leading the therapeutics market with its Pompe Disease drug candidates in the most advanced stage of clinical development.
The Leading Companies in the Pompe Disease Therapeutics Market Include:
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Actus Therapeutics
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Amicus Therapeutics
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Asklepios Biopharmaceutical
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Astellas Pharma Inc
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Audentes Therapeutics
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BioMarin Pharmaceutical
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Denali Therapeutics
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M6P Therapeutics
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Maze Therapeutics
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Rocket Pharmaceuticals
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Sanofi
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Spark Therapeutics
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Valerion Therapeutics
And many others
Pompe Disease Therapies Covered in the Report Include:
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AAV2/8LSPhGAA: Asklepios Biopharmaceutical
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ACTUS-101: Actus Therapeutics
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AT-GAA: Amicus Therapeutics
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AT845: Astellas Therapeutics
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Avalglucosidase alfa: Sanofi
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M021: M6P Therapeutics
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MZE 001: Maze Therapeutics
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SPK-3006: Spark Therapeutics
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VAL-1221: Valerion Therapeutics
And many more
The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @
https://www.delveinsight.com/sample-request/pompe-disease-market
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 (In the US, EU5, and Japan)
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 Drugs and Latest Therapeutic Advances
13. Pompe Disease Seven Major Market Analysis
14. Attribute Analysis
15. Pompe Disease Market Outlook (In US, EU5, and Japan)
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 the Sample PDF to Learn More About the Key Offerings of the Report @
https://www.delveinsight.com/sample-request/pompe-disease-market
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About DelveInsight
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