Pompe Disease Treatment Market Will Size Observe Significant Surge during 2021 2030

In a recent published report, Kenneth Research has updated the market report for Pompe Disease Treatment Market for 2021 till 2030. Report further now discusses; the various strategies to be adopted or being adopted by the business players across the globe at various levels in the value chain. In the view of the global economic slowdown, we further estimated that China, India, Japan and South Korea to recover fastest amongst all the countries in the Asian market. Germany, France, Italy, Spain to take the worst hit and this hit is expected to be regain 25% by the end of 2021- Positive Growth in the economic demand and supply.  

U.S. Market recovers fast; In a release on May 4th 2021, the U.S. Bureau and Economic Analsysis and U.S. Census Bureau mentions the recovery in the U.S. International trade in March 2021. Exports in the country reached $200 billion, up by $12.4  billion in Feb 2021. Following the continuous incremental trend, imports tallied at $274.5 billion, picked up by $16.4 billion in Feb 2021. However, as COVID19 still haunts the economies across the globe, year-over-year (y-o-y) avergae exports in the U.S. declined by $7.0 billion from March 2020 till March 2021 whilest imports increased by $20.7 billion during the same time. This definitely shows how the market is trying to recover back and this will have a direct impact on the Healthcare/ICT/Chemical industries, creating a huge demand for Pompe Disease Treatment Market products.  

Pompe Disease Treatment Market Research 2018-2025, Global Size, Market Status, Top key players, and Forecasts

Recently, a significant growth in healthcare spending have been observed in the low- and middle-income nations. In the middle-income nations, healthcare spending rose by 6.3% a year between 2000 and 2017, while the economy rose by 5.9% a year. Additionally, health spending in low-income countries also observed a growth by 7.8% a year in the same period. The growth in the healthcare spending of these nations can be attributed to the increasing focus of the governments in these nations to meet the laid down targets of the Sustainable Development Goals (SDGs) of 2030. Such factors are anticipated to drive the growth of the Pompe Disease Treatment Market during the forecast period.

The Global Pompe Disease Treatment Market is expected to register a CAGR of 2.6% to reach USD 1,414.1 Million by 2025.Pompe disease is a genetic metabolic disorder that occurs in infants. Pompe disease is caused due to the mutations in the GAA gene responsible for producing the acid, alpha-glucosidase enzyme, that converts glycogen into a simple form. The absence or mutation in the GAA gene leads to the accumulation of glycogen and results in heart problems, muscle weakness, and liver damage that can lead to premature death in newborns.

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The key factors that drive the Pompe disease treatment market are increasing government initiatives and rising R&D by manufacturers. However, factors such as limited awareness about treatment methods and the high cost of therapy hamper market growth.

Market Dynamics

The government has been taking continuous initiatives for the development and manufacture of innovative products for the diagnosis and treatment of rare diseases such as Pompe disease. For instance, the Orphan Products Grants funded 18 new grants from a total of 92 grant applications in 2015, to support around 67 ongoing clinical studies that are related to rare diseases. Moreover, the governments of various countries such as Sweden, India, the Netherlands, and South Korea are focusing on approving guidelines for the reimbursement of patients undergoing enzyme replacement therapy (ERT) for the treatment of Pompe disease.

The State Government of Karnataka along with the Employee State Insurance Corporation provides free of cost ERT to patients with lysosomal storage disorders. Furthermore, the Union Ministry of Health and Family Welfare has been taking active initiatives for creating a fund of an amount of 13,940 USD million for the treatment of rare diseases, including lysosomal storage disorders. These growing government initiatives for promoting and advancing Pompe disease treatment solutions are expected to boost the growth of the global Pompe disease treatment market.


Pompe disease is a rare genetic condition, caused due to the mutation in the GAA gene, which is responsible for making an enzyme called acid alpha-glucosidase. The acid alpha-glucosidase is accountable for breaking glycogen, a complex form of sugar, into its simple form. The absence or mutation in the GAA gene leads to the accumulation of glycogen and damages the cells and tissues. There are three main classifications of Pompe disease, each differing based on age and severity. These classifications are—late-onset Pompe disease, non-classic infantile-onset Pompe disease, and classic infantile-onset Pompe disease.

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The treatment of Pompe disease is disease-specific and depends on the symptoms of the disease. The global Pompe disease treatment market on the basis of therapy has been segmented into, enzyme replacement therapy (ERT), gene therapy, and others. The enzyme replacement therapy (ERT) segment captures the maximum share of the total market as it is the only Food and Drug Administration (FDA) approved therapy for the treatment of Pompe diseases.

The Pompe disease treatment market has been segregated, on the basis of end user, into hospitals & clinics and research & academic institutes. The hospitals & clinics segment was valued at USD 860.8 million in 2018, and the research & academic institutes segment accounted for a value of USD 320.7 million in 2018 and are expected to exhibit a CAGR of 2.3% during the forecast period.

Regional Analysis

The global Pompe disease treatment market, based on region, has been classified as the Americas, Europe, Asia-Pacific, and the Middle East & Africa.The Americas accounted for the largest share in the global Pompe disease treatment market owing to the increase in the number of patients with Pompe disease. According to the National Organization for Rare Disorders, the estimated frequency of the occurrence Pompe disease is approximately 1 in 40,000 in the US.

Europe stands second in the global Pompe disease treatment market followed by Asia-Pacific. Europe accounts for the second-largest market share in the Pompe disease treatment market owing to factors such as the growing research and development initiatives, support by government for the healthcare sector, and improvement in reimbursement policies have enhanced the market growth.

Asia-Pacific is anticipated to be the fastest-growing region in the global Pompe disease treatment market during the forecast period. This growth is affected by factors such as rising healthcare expenditures, increasing awareness among patients regarding rare disorders, and improved healthcare.

The Middle East & Africa accounted for a relatively smaller market share in 2018 due to low per capita income and lack of adequate healthcare infrastructure support

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Key Players

The prominent players in the global Pompe disease treatment market are Amicus Therapeutics, Inc., Genzyme Corporation, Audentes Therapeutics, Oxyrane, Valerion Therapeutics, AVROBIO, Inc., and CENTOGENE AG.

The players operating in the global Pompe disease treatment market are focusing on product launches, along with expanding their global footprints by entering untapped markets. 

Market Segmentation

Global Pompe Disease Treatment Market, by Type

Late-Onset Pompe Disease

Classic Infantile-Onset Pompe Disease

Non-Classic Infantile-Onset Pompe Disease

Global Pompe Disease Treatment Market, by Therapy

Enzyme Replacement Therapy (ERT)

Gene Therapy

Global Pompe Disease Treatment Market, by End User

Hospitals & Clinics

Research & Academic Institutes

Global Pompe Disease Treatment   Market, by Region


North America



South America







Rest of Europe





Australia & New Zealand

Southeast Asia

Rest of Asia-Pacific

Middle East & Africa

Middle East


Available Additional Customizations


Sangamo BioSciences

Intended Audience

Drug Suppliers

Research and development (R&D) companies

Government research laboratories

Independent research laboratories

Government and independent regulatory authorities

Market research and consulting service providers

Medical research laboratories

Academic medical institutes and universities

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