Pompe Disease Treatment Market Size in the 7MM is expected to increase a significant CAGR by 2034, estimates DelveInsight | Asklepios Biopharmaceutical, Genzyme, Sanofi, GeneCradle Inc, Astellas Gene Therapies, Amicus Therapeutics, Spark Therapeutics, Inc

DelveInsight's "Pompe disease Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of the indication Pompe disease, historical and forecasted epidemiology as well as the Pompe disease market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

Discover which therapies are expected to grab the Pompe Disease Market Share @ Pompe Disease Market Outlook- https://www.delveinsight.com/sample-request/pompe-disease-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Key Takeaways from the Pompe Disease Market Report
• In February 2025, Aro Biotherapeutics announced that it has received FDA clearance for the Investigational New Drug Application (IND) for ABX1100. This novel therapy is currently being investigated in a Phase 1 study for late-onset Pompe disease (LOPD) patients.
• As per DelveInsight's estimates, in the year 2023, the total prevalent cases of Pompe disease were approximately 13,000 cases in the 7MM, which might rise by 2034 at a CAGR of XX%.
• EU4 and the UK, accounted for approximately 25% of the total prevalent cases of Pompe disease in the year 2023.
• In 2023, around 8.600 prevalent cases Pompe disease were diagnosed Pompe disease were reported in the United States out of which 98% cases were recorded in adult patients.
• Significant number of Pompe disease cases are infantile-onset, leading to high mortality within the first few months due to severe muscle weakness, poor muscle tone, and respiratory difficulties. This rapid progression can cause significant psychological distress for parents.
• In 2023, the incidence of the CRIM-positive phenotype in infantile-onset Pompe disease was notably higher in the United States, reaching approximately 80%. The CRIM-positive phenotype is characterized by the presence of residual enzyme activity, which can influence the disease's progression and response to treatment.
• The leading Pompe Disease Companies such as Asklepios Biopharmaceutical, Genzyme, Sanofi, GeneCradle Inc, Astellas Gene Therapies, Amicus Therapeutics, Spark Therapeutics, Inc., Asklepios Biopharmaceutical, and others
• Promising Pompe Disease Therapies such as ACTUS-101, MYOZYME (alglucosidase alfa), GC301, zocaglusagene nuzaparvovec, AT2221, SPK-3006, AAV2/8-LSPhGAA (ACTUS-101), and others.

Stay ahead in the Pompe Disease Therapeutics Market with DelveInsight's Strategic Report @ Pompe Disease Market Outlook- https://www.delveinsight.com/sample-request/pompe-disease-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Pompe Disease Epidemiology Segmentation in the 7MM
• Total Live Birth Cases of Pompe Disease in the 7MM [2020-2034].
• Total Adult Prevalent Cases of Pompe Disease in the 7MM [2020-2034].
• Total Prevalent Cases of Pompe Disease in the 7MM [2020-2034]
• Prevalence of Pompe Disease by Onset Types in the 7MM [2020-2034]
• Incidence of IOPD based on Clinical Phenotype in the 7MM

Download the report to understand which factors are driving Pompe Disease Epidemiology trends @ Pompe Disease Prevalence- https://www.delveinsight.com/sample-request/pompe-disease-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Pompe Disease Marketed Drugs
• MYOZYME (alglucosidase alfa): Genzyme/ Sanofi
Alglucosidase alfa is a recombinant form of human acid α-glucosidase and is produced in Chinese hamster cells (CHO) by recombinant DNA technology. MYOZYME will restore lysosomal GAA activity resulting in stabilisation or restoration of cardiac and skeletal muscle function (including respiratory muscles). Due to the blood-brain barrier effect and the enzyme's size, uptake of alglucosidase alfa in the central nervous system is unlikely.

• LUMIZYME (alglucosidase alfa): Genzyme/ Sanofi
Alglucosidase alfa provides an exogenous source of GAA. Binding to mannose-6-phosphate receptors on the cell surface has been shown to occur via carbohydrate groups on the GAA molecule, after which it is internalized and transported into lysosomes, where it undergoes proteolytic cleavage that results in increased enzymatic activity. It then exerts enzymatic activity in cleaving glycogen.

Pompe Disease Emerging Drugs
• AAV2/8-LSPhGAA (ACTUS-101): Asklepios Biopharmaceutical
ACTUS-101 is an adeno-associated virus (AAV) gene therapy that aims to address the deficiency of acid-alpha-glucosidase (GAA) in patients with Pompe disease. GAA deficiency leads to deleterious progressive accumulation of glycogen in organs and tissues, especially skeletal and cardiac muscle, leading to high morbidity and premature death. The current standard of care for patients with Pompe is enzyme replacement therapy (ERT).

• Avalglucosidase alfa: Sanofi
Avalglucosidase alfa, also known as Nexviazyme, is an enzyme replacement therapy (ERT) developed by Sanofi Genzyme to treat patients with late-onset Pompe disease (LOPD) or GAA deficiency. It's a recombinant form of acid alpha-glucosidase (GAA), a human enzyme that helps the body digest and absorb glycogen. Pompe disease is a genetic condition that occurs when GAA is deficient, causing a buildup of glycogen in the body's cells.

To learn more about Pompe Disease treatment guidelines, visit @ Pompe Disease Treatment Market Landscape- https://www.delveinsight.com/sample-request/pompe-disease-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Pompe disease Market Outlook
In 2023, the total market for Pompe disease therapies across the seven major markets (7MM) reached USD 18.4 million, with the US capturing about 70% of this market. The US market significantly outpaced the EU4 (Germany, Spain, Italy, France), the UK, and Japan. The United States represents the largest share of the Pompe disease market, accounting for a significant proportion compared to the EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.

Pompe Disease Therapies and Companies
• ACTUS-101: Asklepios Biopharmaceutical
• MYOZYME (alglucosidase alfa): Genzyme/ Sanofi
• GC301: GeneCradle Inc
• zocaglusagene nuzaparvovec: Astellas Gene Therapies
• AT2221: Amicus Therapeutics
• SPK-3006: Spark Therapeutics, Inc.
• AAV2/8-LSPhGAA (ACTUS-101): Asklepios Biopharmaceutical

Learn more about the FDA-approved drugs for Pompe Disease @ Drugs for Pompe Disease Treatment- https://www.delveinsight.com/sample-request/pompe-disease-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Scope of the Pompe Disease Market Report
• Coverage- 7MM
• Study Period- 2020-2034
• Pompe Disease Companies- Asklepios Biopharmaceutical, Genzyme, Sanofi, GeneCradle Inc, Astellas Gene Therapies, Amicus Therapeutics, Spark Therapeutics, Inc., Asklepios Biopharmaceutical, and others
• Pompe Disease Pipeline Therapies- ACTUS-101, MYOZYME (alglucosidase alfa), GC301, zocaglusagene nuzaparvovec, AT2221, SPK-3006, AAV2/8-LSPhGAA (ACTUS-101), and others.
• Pompe Disease Therapeutic Assessment: Pompe Disease current marketed and Pompe Disease emerging therapies
• Pompe Disease Market Dynamics: Pompe Disease market drivers and Pompe Disease market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Pompe Disease Unmet Needs, KOL's views, Analyst's views, Pompe Disease Market Access and Reimbursement

Discover more about Pompe Disease Drugs in development @ Pompe Disease Clinical Trials Assessment- https://www.delveinsight.com/sample-request/pompe-disease-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Table of Contents
1. Pompe Disease Market Report Introduction
2. Executive Summary for Pompe Disease
3. SWOT analysis of Pompe Disease
4. Pompe Disease Patient Share (%) Overview at a Glance
5. Pompe Disease Market Overview at a Glance
6. Pompe Disease Disease Background and Overview
7. Pompe Disease Epidemiology and Patient Population
8. Country-Specific Patient Population of Pompe Disease
9. Pompe Disease Current Treatment and Medical Practices
10. Pompe Disease Unmet Needs
11. Pompe Disease Emerging Therapies
12. Pompe Disease Market Outlook
13. Country-Wise Pompe Disease Market Analysis (2020-2034)
14. Pompe Disease Market Access and Reimbursement of Therapies
15. Pompe Disease Market Drivers
16. Pompe Disease Market Barriers
17. Pompe Disease Appendix
18. Pompe Disease Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

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