Precision Business Insights


Global Pompe Disease Treatment Market

: Industry Estimation, Dynamics, Regional Share, Trends, Competitor Analysis 2014-2018 and Forecast 2019-2025

Global Pompe Disease Treatment Market: By Treatment Type (Enzyme Replacement Therapy, Gene Therapy), By Age Group Type (infantile-Onset Pompe Disease, Late Onset Pompe Disease), and Geography (North America, Latin America, Europe, Asia Pacific and Middle East and Africa)


Global pompe disease treatment market report gives comprehensive outlook on pompe disease treatment across the globe with special emphasis on key regions such as North America, Europe, Asia Pacific, Latin America, and Middle East and Africa. The report gives historical, current, and future market sizes (US$ Mn) on the basis of treatment type, age group, and region. This report studies market dynamics elaborately to identify the current trends & drivers, future opportunities and possible challenges to the key stakeholders operating in the market. In addition, the report includes human demographics; regulatory scenario, and competition analysis with vividly illustrated the competition dashboard to assess the market competition. Moreover, PBI analyzed the market to better equip clients with possible investment opportunities across the regions (regional Investment Hot-Spots) and market unmet needs (Product Opportunities). Key stakeholders of the market include suppliers, manufacturers, marketers, policy makers, and healthcare service providers engaged in global pompe disease treatment products.


Pompe Disease Treatment Market Size


The global Pompe Disease Treatment market valued at US$ 981.1 Mn in 2018 and poised to grow at significant CAGR over 2019-2025 due to increase in prevalence of pompe disease around the globe. For instance, according to a research study, approximately 5000 to 10,000 people are affected with pompe disease around the world. Moreover, rise in R&D for the development of newer products, strong product pipeline, and raise in funding from public and private sectors are expected to propel the market over the forecast years.Increase in the R&D for the development of newer therapeutics is a major factor drive the global pompe disease treatment market over the forecast years. For instance, Sanofi Genzyme developing neoGAA, an enzyme replacement therapy molecule for the treatment of infantile and late onset pompe disease and the molecule is under phase 3 clinical trials. Moreover, raise in funding for R&D also boost the market over the forecast timeframe. For instance, in October 2015, Audentes Therapeutics announced that, company has raised $65 Million In Series C financing for development and commercialization of gene therapy products for patients with serious, rare diseases


North America Accounts for Larger Market Revenue Share in Global Pompe Disease Treatment Market

North America accounted for larger revenue share in global market by 2025 owing to increase in the prevalence of disease in USA and Canada. For instance, according to National Organization for Rare Disorders, pompe disease incidence is generally placed at approximately 1 in 40,000 births in the United States. Moreover, Europe pompe disease treatment market contributed highest growth rate in 2018 and expected to grow at 9.9% CAGR over the forecast years owing to increase in research and development on newer procedures, and favorable reimbursement policies in the region. For instance, in January 2013, Dutch Health Care Insurance Board advised Minister of Health to provide reimbursement for Enzyme Replacement Therapy for Pompe for a period of 3–4 years.


Competition Assessment

  • Valerion Therapeutics
  • Actus Therapeutics, Inc.
  • BioMarin Pharmaceutical
  • Audentes Therapeutics
  • Amicus Therapeutics
  • Sanofi Genozyme
  • Greenovation Biotech GmbH


Global pompe disease treatment market is surging owing to collaborations and product approvals by the market players. For instance, in 2018, Actus Therapeutics filed IND for AAV2/8-LSPhGAA with funding from the National Institutes of Health and initiated the clinical trials for the treatment of pompe disease.


Key Findings of the Report:


  • Global pompe disease treatment market expanding at significant CAGR over 2019 to 2025 owing to increase in the prevalence of pompe disease.
  • Based on treatment, enzyme replacement therapy accounted for larger market revenue share in 2018 and projected gain market revenue share over the forecast period
  • Players focusing on innovation of newer products and collaboration strategies to retain market position in global pompe disease treatment market


Detailed Segmentation


By Treatment Type

  • Enzyme Replacement Therapy
  • Myozyme/Lumizyme
  • ATB200/AT2221
  • GZ402666
  • VAL-1221
  • Gene Therapy


By Age Group

  • Infantile-Onset Pompe Disease
  • Classic Infantile
  • Non-Classic Infantile
  • Late Onset Pompe Disease



North America

  • US
  • Canada



  • Germany
  • France
  • UK
  • Italy
  • Spain
  • Russia
  • Poland
  • Rest of Europe



  • Japan
  • China
  • India
  • Australia & New Zealand
  • ASEAN (Includes Indonesia, Thailand, Vietnam, Philippines, Malaysia, and Others)
  • South Korea
  • Rest of Asia-Pacific


Latin America

  • Brazil
  • Mexico
  • Argentina
  • Rest of Latin America


Middle East and Africa (MEA)

  • Gulf Cooperation Council (GCC) Countries
  • Israel
  • South Africa
  • Rest of MEA


Key Features of the Report:


  • The report provides granular level information about the market size, regional market share, historic market (2014-2018) and forecast (2019-2028)
  • The report covers in-detail insights about the competitor’s overview, company share analysis, key market developments, and their key strategies
  • The report outlines drivers, restraints, unmet needs, and trends that are currently affecting the market
  • The report tracks recent innovations, key developments and startup’s details that are actively working in the market
  • The report provides plethora of information about market entry strategies, regulatory framework and reimbursement scenario
  • The report analyses the impact of socio-political environment through PESTLE Analysis and competition through Porter’s Five Force Analysis in addition to recent technology advancements and innovations in the market
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