Pompe Disease Market Size, Share, and Industry Report 2031

Segments - Pompe Disease Market by Type (Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease, and Non-Classic infantile-onset Pompe Disease), Therapy Type (Enzyme Replacement Therapy, Gene Therapy, and Others), and End User (Hospitals and Clinics and Research and Academic Institutes), and Regions (Asia Pacific, North America, Latin America, Europe, and Middle East & Africa) - Global Industry Analysis, Growth, Share, Size, Trends, and Forecast 2023 – 2031

Author : Raksha Sharma

Fact-checked by : Vineet Pandey

Editor : Vishal Golekar

Report Description

Pompe Disease Market Outlook 2031

The global Pompe disease market size was USD 1.43 Billion in 2022 and is anticipated to reach USD 2.02 Billion by 2031 expand at a substantial CAGR 3.9% during the forecast period, 2023–2031. The growth of the market is attributed to its ongoing use of long-term treatments, the introduction of new medicines, and the high cost of treatment.

Pompe disease is a hereditary condition in which the body's cells accumulate a complex substance called glycogen. The disorder is caused by a lack of an enzyme called acid alfa glucosidase (GAA), which helps the body break down complex sugars. Organs and tissues, particularly muscles, get clogged, leading them to break down. This disease is caused by mutations in the GAA gene, which aids in the breakdown of glycogen.

Pompe disease is divided into three categories. Classic infantile onset occurs within a few months of birth, non-classic infantile onset occurs around the age of one year, and late-onset occurs later in a child's life, maybe even into teen years or adulthood. As this is a hereditary disorder, those who develop it acquire it from one of their parents.

However, it is very uncommon for neither parent to exhibit any signs or symptoms. It is a rare condition. Pompe disease affects 1 out of every 40,000 people in the United States. It can affect both men and women from all ethnic backgrounds.

The enzymes in the blood are examined and counted when a blood sample is collected. Sleep studies, lung capacity tests, and electromyography are some of the other tests available (a test that measures how well the muscles work). The enzymes in the blood are examined and measured when a blood sample is collected. DNA testing is used to confirm the findings.

Other tests include taking of a complete patient and family history, breathing tests to measure lung capacity (pulmonary function tests), Electromyography (a test that measures how well the muscles work) and MRIs, Heart studies, including X-rays, electrocardiogram and echocardiogram, Sleep studies. All individuals with Pompe disease are eligible for enzyme replacement therapy (ERT).

Intravenously (through a vein in the patient), alglucosidase alfa is administered. It is a genetically modified enzyme that replicates the acid alpha glucosidase enzyme found naturally in the body. For people with Pompe disease, specialist teams (cardiologists, respiratory therapists, neurologists, and others) can treat symptoms and provide supportive care.

Inquire about the specifics of your situation with your physicians. Infants with Pompe disease will lead to death if they are not treated. Many patients with Pompe disease have respiratory (breathing) issues, cardiac issues, and muscular weakness.

Pompe Disease Market Trends, Drivers, Restraints, and Opportunities

Increasing prevalence of Pompe disease around the world is projected to boost the market growth during the forecast period.
Increasing government initiatives for developing and manufacturing innovative products for the diagnosis & treatment is anticipated to fuel the market growth during the forecast period.
Rising consumer awareness regarding the Pompe disease around the world and increasing healthcare expenditure are expected to drive the market growth in the coming years.
High cost of treatment is anticipated to restrict the market growth in the coming years.
Increased R&D investments to find new effective solutions for the diagnosis and treatment and technological advancements are expected to offer lucrative opportunities for the market players.

Scope of The Pompe Disease Market Report

The report on the global Pompe disease market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have also been included in the report.

Attributes	Details
Report Title	Pompe Disease Market - Global Industry Analysis, Growth, Share, Size, Trends, and Forecast
Base Year	2022
Historic Data	2016–2021
Forecast Period	2023–2031
Segmentation	Type (Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease, and Non-Classic infantile-onset Pompe Disease), Therapy Type (Enzyme Replacement Therapy, Gene Therapy, and Others), and End User (Hospitals and Clinics and Research and Academic Institutes)
Regional Scope	Asia Pacific, North America, Latin America, Europe, and Middle East & Africa
Report Coverage	Company Share, Market Analysis and Size, Competitive Landscape, Growth Factors, and Trends, and Revenue Forecast
Key Players Covered in the Report	Amicus Therapeutics; Sanofi; Audentes Therapeutics; Oxyrane; Valerion Therapeutics; AVROBIO, Inc.; and CENTOGENE AG.

Pompe Disease Market Segment Insights

Late-onset Pompe disease segment is expected to constitute a key share

Based on type, the Pompe disease market is segmented into late-onset Pompe disease, classic infantile-onset Pompe disease, and non-classic infantile-onset Pompe disease. The late-onset Pompe disease segment is expected to constitute a key share of the market during the forecast period as it is only long-term therapies available for the treatment of the disease.

Enzyme replacement therapy segment is expected to account for a key share

Based on therapy type, the Pompe disease market is segmented into enzyme replacement therapy (ERT), gene therapy, and others. The enzyme replacement therapy segment is expected to account for a key share of the market during the forecast period owing to strong preference for enzyme replacement therapy over alternative treatments.

Hospitals & clinics segment is expected to represent a key share

Based on end user, the Pompe disease market is segmented into hospitals & clinics and research & academic institutes. The hospitals & clinics segment is expected to represent a key share of the market during the forecast period owing to increase in number of private and government hospitals in emerging countries.

North America is anticipated to dominate the market

On the basis of regions, the Pompe disease market is classified as Asia Pacific, North America, Latin America, Europe, and Middle East & Africa. North America is anticipated to dominate the market during the forecast period. The regional market growth can be attributed to the presence of key payers and an increasing prevalence of Pompe disease patients in the region. Furthermore, the presence of a large number of hospitals in the region would aid market expansion throughout the forecast period.

On the other hand, Asia Pacific is expected to exhibit a rapid growth rate in the coming years due to rising healthcare expenditure, increased patient awareness of rare diseases, and the expansion of the healthcare industry in this region.

Segments

The global Pompe disease market has been segmented on the basis of

Type

Late-Onset Pompe Disease
Classic Infantile-Onset Pompe Disease
Non-Classic infantile-onset Pompe Disease

Therapy Type

Enzyme Replacement Therapy
Gene Therapy
Others

End User

Hospitals and Clinics
Research and Academic Institutes

Regions

Asia Pacific
North America
Latin America
Europe
Middle East & Africa

Key Players

Amicus Therapeutics, Inc.
Sanofi
Audentes Therapeutics
Oxyrane
Valerion Therapeutics
AVROBIO, Inc.
CENTOGENE AG

Competitive Landscape

Key players competing in the Pompe disease market are Amicus Therapeutics; Sanofi; Audentes Therapeutics; Oxyrane; Valerion Therapeutics; AVROBIO, Inc.; and CENTOGENE AG.

Some of the key business strategies employed by companies includes mergers, acquisitions, partnerships.

Pompe Disease Market Key Players

Table Of Content

1. Executive Summary
2. Assumptions and Acronyms Used
3. Research Methodology
4. Pompe Disease Market Overview
  4.1. Introduction
     4.1.1. Market Taxonomy
     4.1.2. Market Definition
  4.2. Macro-Economic Factors
     4.2.1. Industry Outlook
  4.3. Pompe Disease Market Dynamics
     4.3.1. Market Drivers
     4.3.2. Market Restraints
     4.3.3. Opportunity
     4.3.4. Market Trends
  4.4. Pompe Disease Market - Supply Chain
  4.5. Global Pompe Disease Market Forecast
     4.5.1. Pompe Disease Market Size (US$ Mn) and Y-o-Y Growth
     4.5.2. Pompe Disease Market Size (000â€™ Units) and Y-o-Y Growth
     4.5.3. Pompe Disease Market Absolute $ Opportunity
5. Global Pompe Disease Market Analysis and Forecast by Types
  5.1. Market Trends
  5.2. Introduction
     5.2.1. Basis Point Share (BPS) Analysis by Types
     5.2.2. Y-o-Y Growth Projections by Types
  5.3. Pompe Disease Market Size and Volume Forecast by Types
     5.3.1. Late-Onset Pompe Disease
     5.3.2. Classic Infantile-Onset Pompe Disease
     5.3.3. Non-Classic infantile-onset Pompe Disease
  5.4. Absolute $ Opportunity Assessment by Types
  5.5. Market Attractiveness/Growth Potential Analysis by Types
6. Global Pompe Disease Market Analysis and Forecast by End Users
  6.1. Market Trends
  6.2. Introduction
     6.2.1. Basis Point Share (BPS) Analysis by End Users
     6.2.2. Y-o-Y Growth Projections by End Users
  6.3. Pompe Disease Market Size and Volume Forecast by End Users
     6.3.1. Hospitals and Clinics
     6.3.2. Research and Academic Institutes
  6.4. Absolute $ Opportunity Assessment by End Users
  6.5. Market Attractiveness/Growth Potential Analysis by End Users
7. Global Pompe Disease Market Analysis and Forecast by Region
  7.1. Market Trends
  7.2. Introduction
     7.2.1. Basis Point Share (BPS) Analysis by Region
     7.2.2. Y-o-Y Growth Projections by Region
  7.3. Pompe Disease Market Size and Volume Forecast by Region
     7.3.1. North America
     7.3.2. Latin America
     7.3.3. Europe
     7.3.4. Asia Pacific
     7.3.5. Middle East and Africa (MEA)
  7.4. Absolute $ Opportunity Assessment by Region
  7.5. Market Attractiveness/Growth Potential Analysis by Region
  7.6. Global Pompe Disease Demand Share Forecast, 2019-2026
8. North America Pompe Disease Market Analysis and Forecast
  8.1. Introduction
     8.1.1. Basis Point Share (BPS) Analysis by Country
     8.1.2. Y-o-Y Growth Projections by Country
  8.2. North America Pompe Disease Market Size and Volume Forecast by Country
     8.2.1. U.S.
     8.2.2. Canada
  8.3. Absolute $ Opportunity Assessment by Country
  8.4. North America Pompe Disease Market Size and Volume Forecast by Types
     8.4.1. Late-Onset Pompe Disease
     8.4.2. Classic Infantile-Onset Pompe Disease
     8.4.3. Non-Classic infantile-onset Pompe Disease
  8.5. Basis Point Share (BPS) Analysis by Types
  8.6. Y-o-Y Growth Projections by Types
  8.7. North America Pompe Disease Market Size and Volume Forecast by End Users
     8.7.1. Hospitals and Clinics
     8.7.2. Research and Academic Institutes
  8.8. Basis Point Share (BPS) Analysis by End Users
  8.9. Y-o-Y Growth Projections by End Users
  8.10. Market Attractiveness/Growth Potential Analysis
     8.10.1. By Country
     8.10.2. By Product Type
     8.10.3. By Application
  8.11. North America Pompe Disease Demand Share Forecast, 2019-2026
9. Latin America Pompe Disease Market Analysis and Forecast
  9.1. Introduction
     9.1.1. Basis Point Share (BPS) Analysis by Country
     9.1.2. Y-o-Y Growth Projections by Country
     9.1.3. Latin America Average Pricing Analysis
  9.2. Latin America Pompe Disease Market Size and Volume Forecast by Country
      9.2.1. Brazil
      9.2.2. Mexico
      9.2.3. Rest of Latin America
   9.3. Absolute $ Opportunity Assessment by Country
  9.4. Latin America Pompe Disease Market Size and Volume Forecast by Types
     9.4.1. Late-Onset Pompe Disease
     9.4.2. Classic Infantile-Onset Pompe Disease
     9.4.3. Non-Classic infantile-onset Pompe Disease
  9.5. Basis Point Share (BPS) Analysis by Types
  9.6. Y-o-Y Growth Projections by Types
  9.7. Latin America Pompe Disease Market Size and Volume Forecast by End Users
     9.7.1. Hospitals and Clinics
     9.7.2. Research and Academic Institutes
  9.8. Basis Point Share (BPS) Analysis by End Users
  9.9. Y-o-Y Growth Projections by End Users
  9.10. Market Attractiveness/Growth Potential Analysis
     9.10.1. By Country
     9.10.2. By Product Type
     9.10.3. By Application
  9.11. Latin America Pompe Disease Demand Share Forecast, 2019-2026
10. Europe Pompe Disease Market Analysis and Forecast
  10.1. Introduction
     10.1.1. Basis Point Share (BPS) Analysis by Country
     10.1.2. Y-o-Y Growth Projections by Country
     10.1.3. Europe Average Pricing Analysis
  10.2. Europe Pompe Disease Market Size and Volume Forecast by Country
     10.2.1. Germany
     10.2.2. France
     10.2.3. Italy
     10.2.4. U.K.
     10.2.5. Spain
     10.2.6. Russia
     10.2.7. Rest of Europe
  10.3. Absolute $ Opportunity Assessment by Country
  10.4. Europe Pompe Disease Market Size and Volume Forecast by Types
     10.4.1. Late-Onset Pompe Disease
     10.4.2. Classic Infantile-Onset Pompe Disease
     10.4.3. Non-Classic infantile-onset Pompe Disease
  10.5. Basis Point Share (BPS) Analysis by Types
  10.6. Y-o-Y Growth Projections by Types
  10.7. Europe Pompe Disease Market Size and Volume Forecast by End Users
     10.7.1. Hospitals and Clinics
     10.7.2. Research and Academic Institutes
  10.8. Basis Point Share (BPS) Analysis by End Users
  10.9. Y-o-Y Growth Projections by End Users
  10.10. Market Attractiveness/Growth Potential Analysis
     10.10.1. By Country
     10.10.2. By Product Type
     10.10.3. By Application
  10.11. Europe Pompe Disease Demand Share Forecast, 2019-2026
11. Asia Pacific Pompe Disease Market Analysis and Forecast
  11.1. Introduction
     11.1.1. Basis Point Share (BPS) Analysis by Country
     11.1.2. Y-o-Y Growth Projections by Country
     11.1.3. Asia Pacific Average Pricing Analysis
  11.2. Asia Pacific Pompe Disease Market Size and Volume Forecast by Country
     11.2.1. China
     11.2.2. Japan
     11.2.3. South Korea
     11.2.4. India
     11.2.5. Australia
     11.2.6. Rest of Asia Pacific (APAC)
  11.3. Absolute $ Opportunity Assessment by Country
  11.4. Asia Pacific Pompe Disease Market Size and Volume Forecast by Types
     11.4.1. Late-Onset Pompe Disease
     11.4.2. Classic Infantile-Onset Pompe Disease
     11.4.3. Non-Classic infantile-onset Pompe Disease
  11.5. Basis Point Share (BPS) Analysis by Types
  11.6. Y-o-Y Growth Projections by Types
  11.7. Asia Pacific Pompe Disease Market Size and Volume Forecast by End Users
     11.7.1. Hospitals and Clinics
     11.7.2. Research and Academic Institutes
  11.8. Basis Point Share (BPS) Analysis by End Users
  11.9. Y-o-Y Growth Projections by End Users
  11.10. Market Attractiveness/Growth Potential Analysis
     11.10.1. By Country
     11.10.2. By Product Type
     11.10.3. By Application
  11.11. Asia Pacific Pompe Disease Demand Share Forecast, 2019-2026
12. Middle East & Africa Pompe Disease Market Analysis and Forecast
  12.1. Introduction
     12.1.1. Basis Point Share (BPS) Analysis by Country
     12.1.2. Y-o-Y Growth Projections by Country
     12.1.3. Middle East & Africa Average Pricing Analysis
  12.2. Middle East & Africa Pompe Disease Market Size and Volume Forecast by Country
     12.2.1. Saudi Arabia
     12.2.2. South Africa
     12.2.3. UAE
     12.2.4. Rest of Middle East & Africa (MEA)
  12.3. Absolute $ Opportunity Assessment by Country
  12.4. Middle East & Africa Pompe Disease Market Size and Volume Forecast by Types
     12.4.1. Late-Onset Pompe Disease
     12.4.2. Classic Infantile-Onset Pompe Disease
     12.4.3. Non-Classic infantile-onset Pompe Disease
  12.5. Basis Point Share (BPS) Analysis by Types
  12.6. Y-o-Y Growth Projections by Types
  12.7. Middle East & Africa Pompe Disease Market Size and Volume Forecast by End Users
     12.7.1. Hospitals and Clinics
     12.7.2. Research and Academic Institutes
  12.8. Basis Point Share (BPS) Analysis by End Users
  12.9. Y-o-Y Growth Projections by End Users
  12.10. Market Attractiveness/Growth Potential Analysis
     12.10.1. By Country
     12.10.2. By Product Type
     12.10.3. By Application
  12.11. Middle East & Africa Pompe Disease Demand Share Forecast, 2019-2026
13. Competition Landscape
  13.1. Global Pompe Disease Market: Market Share Analysis
  13.2. Pompe Disease Distributors and Customers
  13.3. Pompe Disease Market: Competitive Dashboard
  13.4. Company Profiles (Details: Overview, Financials, Developments, Strategy)
     13.4.1. Amicus Therapeutics, Inc.
     13.4.2. Sanofi
     13.4.3. Audentes Therapeutics
     13.4.4. Oxyrane
     13.4.5. Valerion Therapeutics
     13.4.6. AVROBIO, Inc.
     13.4.7. CENTOGENE AG