Pompe Disease Treatment Market Size in 7MM is expected to grow at a decent CAGR by 2034, estimates DelveInsight | Genzyme, Sanofi, Amicus Therapeutics, Actus Therapeutics, Valerion Therapeutics, Astellas Therapeutics, Roche, Lacerta Therapeutics

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 March 2025, Astellas Gene Therapies announced a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
• In March 2025, Amicus Therapeutics announced an open label extension study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject's country.
• In March 2025, Sanofi organized an open-lable study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD).
• 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 Genzyme, Sanofi, Amicus Therapeutics, Actus Therapeutics, Valerion Therapeutics, Astellas Therapeutics, Roche, Lacerta Therapeutics, and others.
• Promising Pompe disease Pipeline Therapies such as ACTUS-101, AT845, Cipaglucosidase alfa, Avalglucosidase alfa (GZ402666), SPK-3006, 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 Pompe Disease Live Birth Cases
• Total Pompe Disease Adult Prevalent Cases
• Total Pompe Disease Prevalent Cases
• Prevalence of Pompe Disease by Onset Types
• Pompe Disease Incidence of IOPD based on Clinical Phenotype

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 Dynamics
The market for Pompe disease therapies has garnered increasing interest due to advancements in understanding the condition and developing innovative treatments. Several factors have driven these changes. Firstly, heightened awareness and enhanced diagnostic tools have led to earlier and more accurate diagnoses, increasing the patient population. This has prompted pharmaceutical companies to invest in research and development to create new therapies, including enzyme replacement and gene therapies. Additionally, regulatory agencies have played a pivotal role in shaping the market. Accelerated approval processes and orphan drug designations have motivated pharmaceutical companies to pursue treatments for Pompe disease. These factors, along with incentives for rare disease research, have created a more competitive landscape.

Pompe Disease Therapies and Companies
• ACTUS-101: Asklepios Biopharmaceutical, Inc.
• AT845: Astellas Pharma Inc
• Cipaglucosidase alfa: Amicus Therapeutics
• Avalglucosidase alfa (GZ402666): Sanofi
• SPK-3006: Spark Therapeutics

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- Genzyme, Sanofi, Amicus Therapeutics, Actus Therapeutics, Valerion Therapeutics, Astellas Therapeutics, Roche, Lacerta Therapeutics, and others.
• Pompe disease Pipeline Therapies- ACTUS-101, AT845, Cipaglucosidase alfa, Avalglucosidase alfa (GZ402666), SPK-3006, and others.
• Pompe Disease Market Dynamics: Pompe Disease Market Drivers and Barriers
• Pompe Disease Market Access and Reimbursement, Unmet Needs and Future Perspectives

Table of Content
1. Key Insights
2. Pompe Disease Market Report Introduction
3. Pompe disease Market Overview at a Glance
4. Pompe Disease Methodology
5. Pompe Disease Executive Summary
6. Pompe Disease Background and Overview
7. Pompe Disease Diagnosis
8. Pompe Disease Treatment
9. Diagnostic and Treatment Guidelines for Pompe disease
10. Conclusion
11. Pompe Disease Epidemiology and Patient Population
12. Pompe Disease Patient Journey
13. Pompe Disease Approved Therapies
14. Pompe Disease Emerging Therapies
15. Pompe Disease Market Analysis
16. Pompe Disease KOL Views
17. Pompe Disease Market Barriers
18. Pompe Disease Market Drivers
19. Pompe Disease SWOT Analysis
20. Pompe Disease Unmet Needs
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
24. About DelveInsight

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This release was published on openPR.