The 7 major pompe disease markets reached a value of USD 1,336.1 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 2,240.4 Million by 2035, exhibiting a growth rate (CAGR) of 4.81% during 2025-2035.
Report Attribute
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Key Statistics
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Base Year | 2024 |
Forecast Years | 2025-2035 |
Historical Years |
2019-2024
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Market Size in 2024
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USD 1,336.1 Million |
Market Forecast in 2035
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USD 2,240.4 Million |
Market Growth Rate (2025-2035)
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4.81% |
The pompe disease market has been comprehensively analyzed in IMARC's new report titled "Pompe Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Pompe disease, also known as glycogen storage disease type II, is a rare, autosomal recessive metabolic disorder caused by mutations in the GAA gene, which encodes the enzyme acid alpha-glucosidase. This enzyme is responsible for breaking down glycogen within lysosomes. The deficiency of acid alpha-glucosidase leads to excess glycogen accumulation in muscle tissues, primarily affecting skeletal, cardiac, and respiratory muscles. The disorder manifests in different forms based on the age of onset and severity: infantile-onset Pompe disease (IOPD), late-onset Pompe disease (LOPD), and juvenile-onset Pompe disease. IOPD, the most severe form, presents within the first few months of life with cardiomyopathy, hypotonia, and respiratory failure, often leading to mortality if untreated. LOPD develops later in life, causing progressive muscle weakness, fatigue, and respiratory complications that severely impact mobility and quality of life. Diagnosing Pompe disease involves a combination of enzyme activity assays, genetic testing, and muscle biopsies, while newborn screening programs are increasingly being implemented in several countries to enable early detection and timely intervention.
The rising diagnosis and awareness levels of Pompe disease are major growth drivers for the market, in addition to the growing application of enzyme replacement therapy (ERT) as the main treatment option. Myozyme and Lumizyme (alglucosidase alfa) currently dominate the market, which are ERTs used to replace the deficient acid alpha-glucosidase enzyme, thus lowering glycogen accumulation. But ERT mandates lifetime intravenous infusions and has limitations with immune responses, variability in response, and reactions on infusion. In an effort to overcome the drawbacks, therapies are under way, such as cipaglucosidase alfa, an engineered enzyme of higher specificity and stability, and chaperone therapy, designed to augment the action of an enzyme by rescuing misfolded proteins. Gene therapy entrants are under investigation as potentially single-dosing curative entities, which promises to transform management of Pompe disease. In addition, strategic collaborations among pharmaceutical companies and biotech organizations are speeding up research and development, which results in a rich treatment pipeline. With ongoing innovations in diagnostics, newborn screening programs, and new therapies, the Pompe disease market is likely to experience impressive growth during the forecast period.
IMARC Group's new report provides an exhaustive analysis of the pompe disease market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for pompe disease and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the pompe disease market in any manner.
Nexviazyme (avalglucosidase alfa-ngpt) is an enzyme replacement therapy for patients aged one year and older with late-onset Pompe disease. It targets the mannose-6-phosphate receptor to enhance glycogen clearance in muscle cells, improving respiratory function and mobility.
Myozyme (alglucosidase alfa) is an enzyme replacement therapy for pompe disease, one of the rare genetic conditions characterized by a deficiency of acid alpha-glucosidase. Infusion bi-weekly helps to reduce glycogen accumulation in muscles, resulting in improved motor and respiratory functions in such patients. It would deliver the therapy by intravenous infusion of alglucosidase alfa.
ABX1100 is a putative therapy for late-onset Pompe disease based on directly inhibiting glycogen synthesis through the delivery of GYS1-specific siRNA into muscle tissues. It achieved a well-tolerated Phase 1 study, demonstrating a significant reduction in levels of GYS1 mRNA and protein.
Time Period of the Study
Countries Covered
Analysis Covered Across Each Country
This report also provides a detailed analysis of the current pompe disease marketed drugs and late-stage pipeline drugs.
In-Market Drugs
Late-Stage Pipeline Drugs
Drugs | Company Name |
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Nexviazyme (avalglucosidase alfa-ngpt) | Sanofi |
Myozyme (alglucosidase alfa) | Sanofi |
ABX 1100 | Aro Biotherapeutics |
Imlifidase | Hansa Biopharma |
DNL 952 | Denali Therapeutics Inc |
*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.
Market Insights
Epidemiology Insights
Pompe Disease: Current Treatment Scenario, Marketed Drugs and Emerging Therapies