Pompe Disease Market: Epidemiology, Therapies, Companies, DelveInsight | Genzyme, Sanofi, Amicus Therapeutics, Actus Therapeutics, Valerion Therapeutics, Astellas Therapeutics, Roche, Lacerta Therapeutics

Pompe Disease emerging therapies, such as MYOZYME (alglucosidase alfa), LUMIZYME, AAV2/8-LSPhGAA, Avalglucosidase alfa, and others, are expected to boost the Pompe Disease Market in the upcoming years.

DelveInsight has launched a new report on "Pompe Disease - Market Insights, Epidemiology, and Market Forecast-2034" that delivers an in-depth understanding of the Pompe Disease, historical and forecasted epidemiology as well as the Pompe Disease market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Discover about the Pompe Disease market report @ https://www.delveinsight.com/report-store/pompe-disease-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Some of the key facts of the Pompe Disease Market Report:

Pompe disease, or glycogen storage disease type II, is a rare inherited disorder caused by a deficiency of the enzyme acid alpha-glucosidase, which breaks down glycogen in lysosomes. Without this enzyme, glycogen builds up in muscles, causing progressive damage. In 2023, about 8,600 cases were reported in the U.S., with 98% occurring in adults. Infantile-onset cases are severe, often leading to early death from muscle weakness and respiratory problems, causing emotional distress for families.
In September 2023, the FDA approved this two-component therapy developed by Amicus Therapeutics. It is indicated for adults with LOPD who weigh at least 40 kg and are not responding adequately to existing enzyme replacement therapies. The approval was based on data from the Phase 3 PROPEL study, which demonstrated improvements in respiratory function and walking distance compared to prior treatments .
In 2023, around 80% of infantile-onset cases in the U.S. were CRIM-positive, meaning some enzyme activity remains, which affects disease progression and treatment response. The FDA has approved enzyme replacement therapies like MYOZYME, LUMIZYME, and POMBILITI for Pompe disease. However, the treatment pipeline is limited, with a few candidates in trials, including ACTUS-101 (Phase I/II) and Avalglucosidase alfa (Phase III). The disease's rarity makes patient recruitment difficult, slowing new drug development.
According to DelveInsight, there were about 13,000 prevalent Pompe disease cases across the 7 major markets (7MM) in 2023, with projections indicating growth at a CAGR of XX% by 2034. The EU4 countries and the UK together represented roughly 25% of these cases in 2023. In the same year, approximately 8,600 Pompe disease cases were reported in the United States, with adults accounting for 98% of these cases.
In February 2025, Aro Biotherapeutics announced that the FDA approved its Investigational New Drug Application (IND) for ABX1100. This new treatment is now entering a Phase 1 clinical trial targeting patients with late-onset Pompe disease (LOPD).
Key Pompe Disease companies such as Genzyme, Sanofi, Amicus Therapeutics, Actus Therapeutics, Valerion Therapeutics, Astellas Therapeutics, Roche, Lacerta Therapeutics, and others are evaluating new drugs for Pompe Disease to improve the treatment landscape.
Promising Pompe Disease therapies include MYOZYME (alglucosidase alfa), LUMIZYME, AAV2/8-LSPhGAA, Avalglucosidase alfa, and others.

Pompe Disease Overview

Pompe disease is a rare inherited metabolic disorder caused by mutations in the GAA gene, resulting in a deficiency of the enzyme acid alpha-glucosidase. This enzyme breaks down glycogen into glucose within lysosomes. When deficient, glycogen accumulates in cells, mainly muscles, causing progressive muscle weakness, breathing problems, and other serious issues. The disease has two main types: infantile-onset, appearing within the first months of life and often fatal if untreated, and late-onset, which develops later with a slower progression.

Diagnosis usually involves measuring acid alpha-glucosidase enzyme activity through blood or muscle tests, genetic testing for GAA mutations, and sometimes muscle biopsies. Early detection is vital, especially for the infantile-onset form, to manage the condition effectively.

Pompe Disease Market Outlook

In 2023, the Pompe disease therapy market across the seven major markets (7MM) reached USD 18.4 million, with the US capturing around 70% of this share, significantly outperforming the EU4 countries (Germany, Spain, Italy, France), the UK, and Japan.

The US holds the largest portion of the Pompe disease market compared to other regions. Within the EU4 and the UK, Germany led with a market size of approximately USD 94 million, followed by France, while one country had the smallest market size in 2023.

Asklepios Biopharmaceutical is developing AAV2/8-LSPhGAA, a gene therapy designed to treat Pompe disease by delivering the GAA gene to restore enzyme activity. Alongside Sanofi's Avalglucosidase alfa, a next-generation enzyme replacement therapy, these treatments show potential to meet unmet needs in Pompe disease care. Although early data for AAV2/8-LSPhGAA are promising, pivotal clinical trials are necessary to confirm its safety and effectiveness.

Discover how the Pompe Disease market is rising in the coming years @ https://www.delveinsight.com/sample-request/pompe-disease-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Pompe Disease Marketed Drugs
MYOZYME (alglucosidase alfa): Genzyme/ Sanofi
LUMIZYME (alglucosidase alfa): Genzyme/ Sanofi
Pompe Disease Emerging Drugs
AAV2/8-LSPhGAA (ACTUS-101): Asklepios Biopharmaceutical
Avalglucosidase alfa: Sanofi

Scope of the Pompe Disease Market Report
Study Period: 2020-2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Pompe Disease Companies: Genzyme, Sanofi, Amicus Therapeutics, Actus Therapeutics, Valerion Therapeutics, Astellas Therapeutics, Roche, Lacerta Therapeutics, and others
Key Pompe Disease Therapies: MYOZYME (alglucosidase alfa), LUMIZYME, AAV2/8-LSPhGAA, Avalglucosidase alfa, 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

To know what's more in our Pompe Disease report, visit https://www.delveinsight.com/report-store/pompe-disease-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Key benefits of the Pompe Disease Market Report:
Pompe Disease market report covers a descriptive overview and comprehensive insight of the Pompe Disease Epidemiology and Pompe Disease market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).
The Pompe Disease market report provides insights into the current and emerging therapies.
The Pompe Disease market report provides a global historical and forecasted market covering drug outreach in 7MM.
The Pompe Disease market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Pompe Disease market.

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Table of Contents

1. Report Introduction
2. Executive Summary
3. SWOT analysis
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. Unmet Needs
11. Pompe Disease Emerging Therapies
12. Pompe Disease Market Outlook
13. Country-Wise Pompe Disease Market Analysis (2020-2034)
14. Market Access and Reimbursement of Therapies
15. Market drivers
16. Market barriers
17. Appendix
18. Pompe Disease Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

Click here to read more about Pompe Disease Market Outlook 2034 https://www.delveinsight.com/report-store/pompe-disease-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Related Reports:

Pompe Disease Pipeline Insights, DelveInsight

"Pompe Disease Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Pompe Disease market. A detailed picture of the Pompe Disease pipeline landscape is provided, which includes the disease overview and Pompe Disease treatment guidelines.

Contact Info:

Kritika Rehani, Assistant Manager, Marketing & Branding
krehani@delveinsight.com
info@delveinsight.com
www.delveinsight.com
+14699457679

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.

This release was published on openPR.