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Pompe Disease Market - Growth, Trends, and Forecasts (2020 - 2025)

  • ID: 5119894
  • Report
  • August 2020
  • Region: Global
  • 117 pages
  • Mordor Intelligence
until Sep 30th 2021


  • Amicus Therapeutics
  • Audentes Therapeutics
  • EpiVax, Inc
  • Genzyme Corporation
  • Oxyrane
  • Sangamo Therapeutics
The Pompe Disease Market is expected to register a CAGR of 4.5% during the forecast period. This is attributing to the launch of novel drugs and continued uptake of long-term therapies. Pompe disease is rare diseases classified as early infantile or late-onset majorly affecting skeletal muscle, heart defects, and breathing problems. As per the National Institute of Health, Pompe diseases affects 1 in 40,000 individuals in the United States. Thus, the launching of novel drugs worldwide will boost the demand for treatment of this disorder and further drive the revenue growth of the market. Moreover, increasing R&D expenditure globally will also promote working on novel therapeutics and treatment of pomp diseases as this disease most of the time leads to death if not treated. Furthermore, increasing development in the field of enzyme replacement therapies and gene therapy will also create a lucrative opportunity for the pompe disease market in the forecasted period as these are the therapies used in the treatment of this disorder. However, high treatment costs associated with the treatment of pompe diseases may hamper the market to a certain extent.

Key Market Trends

Enzyme Replacement Therapy in Pompe Disease is Estimated to Witness a Healthy Growth in Future.

Enzyme replacement therapy is estimated to witness healthy growth in the future. This therapy treatment is the only effective treatment wherein the enzyme acid alpha-glucosidase which is deficient in Pompe disease is given by injection. This will reduce and slows down the progressing of muscle wasting and heart problems. Moreover, a major company like Genozyme offers Myozyme, enzyme replacement therapy used to treatment for the pediatric population (less than 8 years), and another therapy offered by this Lumizyme which helps in treating the Pompe diseases patients above 8 years. These two therapies are the only long-term therapies used for the diagnosis of this disorder and adopted among healthcare professionals that will further promote the segment growth.

North America is Expected to Hold a Significant Share in the Market and Expected to do Same in the Forecast Period

North America is expected to hold a significant market share in the global Pompe Disease Market due to rising launches of novel drugs, the presence of a large number of pharmaceutical companies, growing research and development expenditure, rising patient preference associated with advanced therapies, product launches, and technological advancements. The United States owns the largest share of Pompe Disease Market in the North America region Furthermore, increasing awareness about rare disorders and increasing healthcare infrastructure and are also expected to fuel the Pompe Disease Market in this region.

Competitive Landscape

The Pompe Disease Market is moderately competitive and consists of several major players. Some of the companies which are currently dominating the market are Genzyme Corporation, Amicus Therapeutics, Valerion Therapeutics, Audentes Therapeutics, EpiVax, Inc, Oxyrane, and Sangamo Therapeutics.

Reasons to Purchase this report:
  • The market estimate (ME) sheet in Excel format
  • 3 months of analyst support
Note: Product cover images may vary from those shown


  • Amicus Therapeutics
  • Audentes Therapeutics
  • EpiVax, Inc
  • Genzyme Corporation
  • Oxyrane
  • Sangamo Therapeutics
1.1 Study Assumptions
1.2 Scope of the Study



4.1 Market Overview
4.2 Market Drivers
4.2.1 Launch of Novel Drugs
4.2.2 Continued Uptake of Long Term Therapies
4.3 Market Restraints
4.3.1 High Treatment Cost
4.4 Porter's Five Force Analysis
4.4.1 Threat of New Entrants
4.4.2 Bargaining Power of Buyers/Consumers
4.4.3 Bargaining Power of Suppliers
4.4.4 Threat of Substitute Products
4.4.5 Intensity of Competitive Rivalry

5.1 By Route of Administration
5.1.1 Oral
5.1.2 Parenteral
5.2 By Treatment Type
5.2.1 Substrate Reduction Therapy (SRT)
5.2.2 Enzyme Replacement Therapy (ERT)
5.2.3 Chaperone-Advanced Replacement Therapy (CART)
5.2.4 Supportive Care
5.3 By End User
5.3.1 Hospitals
5.3.2 Diagnostic Centers
5.3.3 Ambulatory Surgical Centers
5.3.4 Others
5.4 Geography
5.4.1 North America United States Canada Mexico
5.4.2 Europe Germany United Kingdom France Italy Spain Rest of Europe
5.4.3 Asia-Pacific China Japan India Australia South Korea Rest of Asia-Pacific
5.4.4 Middle-East and Africa GCC South Africa Rest of Middle-East and Africa
5.4.5 South America Brazil Argentina Rest of South America

6.1 Company Profiles
6.1.1 Genzyme Corporation
6.1.2 Amicus Therapeutics
6.1.3 Valerion Therapeutics
6.1.4 Audentes Therapeutics
6.1.5 EpiVax, Inc
6.1.6 Oxyrane
6.1.7 Sangamo Therapeutics

Note: Product cover images may vary from those shown

A selection of companies mentioned in this report includes:

  • Genzyme Corporation
  • Amicus Therapeutics
  • Valerion Therapeutics
  • Audentes Therapeutics
  • EpiVax, Inc
  • Oxyrane
  • Sangamo Therapeutics
Note: Product cover images may vary from those shown