Pompe Disease Therapeutic Market, by Therapy Type (Gene Therapy, Enzyme Replacement Therapy (ERT), and Chaperone-Advanced Replacement Therapy (CHART)), by Administrational Route (Oral and Parenteral), and by Patient Type (Infants and Adults) - Global Industry Insights, Trends, Outlook and Opportunity Analysis 2018-2026

  • To Be Published : Sep 2018 |
  • Format : CMI Insight PPT Format CMI Insight PDF Format

Pompe disease is an inherited Lysosomal Storage Disorder (LSD) caused by a deficiency of enzyme acid named alpha-glucosidase (GAA). This causes the glycogen to accumulate in different kinds of tissues, primarily into smooth muscles, skeletal muscles and cardiac muscle. It leads to muscle weakness, respiratory problems, and may also affects the liver and heart. Pompe disease is inherited as an autosomal recessive genetic trait. There are three major types of pompe disease, which differ in severity and appear at different age, these types are known as classic infantile-onset, which starts within a few months of birth, non-classic infantile-onset Pompe disease- usually appears by age 1, and late-onset type of Pompe disease that may shows the symptoms later in childhood, adolescence, or adulthood. Pompe disease therapeutic market can exhibit immense growth potential due to presence of limited number of manufacturers, which can significantly enhance and expand their business potential and scope in this market.

Pompe Disease Therapeutic Market Taxonomy:

By Therapy Type:

  • Gene Therapy
  • Enzyme Replacement Therapy (ERT)
  • Chaperone-Advanced Replacement Therapy (CHART)

By Administrational Route:

  • Oral
  • Parenteral

By Patient Type:

  • Infants
  • Adults

Increasing government initiatives is expected to augment growth of the pompe disease therapeutic market

Considering the public health problems, government is taking continuous initiatives in promoting and advancing the development of innovative products for the prevention and diagnosis or treatment of rare diseases or conditions such as Pompe disease. For instance, in 2015, Orphan Product Grants Program funded 18 new grant awards from 92 grant applications and provided funding and support to around 67 ongoing clinical study projects related to rare disease. Furthermore, initiatives from the governments of economies such as Sweden, the Netherlands, and South Korea that focus on approving guidelines for the reimbursement of individuals undergoing enzyme replacement therapy (ERT) are also expected to boost growth of the pompe disease therapeutic market in near future.

Increasing prevalence of pompe disease in many economies is expected to have a lucrative opportunities for pharmaceutical manufacturers to come with a tailored solution for diagnosis and treatment of disease. For instance, according to United Pompe Foundation (UPF), 2017, the estimated frequency occurrence of Pompe disease may vary among different ethnic groups and nationalities: in Holland: 1 in 40,000 (Adults: 1 in 57,000; Infantile: 1 in 138,000), Southern China and Taiwan: 1 in 50,000 births, African-Americans: 1 in 14,000 births and Caucasian: 1 in 100,000 suffer from pompe disease. Gene therapy and enzyme replacement therapy may be highly effective at reducing clinical manifestations of this rare disease. Currently, there are two commercialized products in enzyme replacement therapy, which are Myozyme for the treatment of infants and children with Pompe disease and Lumizyme for late-onset non-infantile Pompe disease. Furthermore, various pipeline products in phase 3 (NeoGAA GZ402666 (Sanofi), AT-982 (Audentes therapeutics)) and in phase 2 (BMN 701(Biomarin)) clinical trial are also expected to create a meteoric growth in this market during the forecast period. However, the high cost associated with the treatment and long duration of the treatment are the factors restraining growth of the pompe disease therapeutic market. For instance, according to a research paper published by Harvard School, in 2016, Lumizyme accounted for US$ 100,000 per year for children and US$ 300,000 per year for adult treatments.

Increasing investments in research and development by manufacturers is expected to propel the market growth

Manufacturers are focusing on finding new solutions by expanding their pipeline to cater the demand for pompe disease therapeutics market. For instance, in September, 2017, Avrobio Inc. expanded their products pipeline with gene therapy to treat pompe disease by utilizing a proprietary lysosomal targeting sequence to deliver high levels of enzyme to lysosomes. Furthermore, in October 2017, Amicus Therapeutics received orphan drug designation to ATB200/AT2221 from the U.S. FDA for the treatment of Pompe disease. This novel treatment paradigm consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P) to enhance uptake, co-administered with AT2221, a pharmacological chaperone.

Key players operating in the pompe disease therapeutic market include Amicus Therapeutics, Inc., Valerion Therapeutics, LLC, Sanofi S.A., Oxyrane, and Sangamo Therapeutics, Inc.

Research Methodology

Coherent Market Insights followsa comprehensive research methodology focused on providing the most precise market analysis. The company leverages a data triangulation model which helps company to gauge the market dynamics and provide accurate estimates. Key components of the research methodologies followed for all our market reports include:

  • Primary Research (Trade Surveys and Experts Interviews)
  • Desk Research
  • Proprietor Data Analytics Model

In addition to this, Coherent Market Insights has access to a wide range of the regional and global reputed paid data bases, which helps the company to figure out the regional and global market trends and dynamics. The company analyses the industry from the 360 Degree Perspective i.e. from the Supply Side and Demand Side which enables us to provide granular details of the entire ecosystem for each study. Finally, a Top-Down approach and Bottom-Up approach is followed to arrive at ultimate research findings.


Smiley face

Coherent Market Insights desk research is based on a principle set of research techniques:

  • National level desk research: It Includes research analysis of regional players, regional regulatory bodies, regional trade associations, and regional organization.
  • Multinational level desk research: The research team keeps a track of multinational players, global regulatory bodies, global trade associations, and global organization.

Coherent Market Insights has a large amount of in-house repository of industry database. This is leveraged as a burner for initiating a new research study. Key secondary sources include:

  • Governmental bodies, National and international social welfare institutions, and organizations creating economic policies among others.
  • Trade association, National and international media and trade press.
  • Company Annual reports, SEC filings, Corporate Presentations, press release, news, and specification sheet of manufacturers, system integrators, brick and mortar - distributors and retailers, and third party online commerce players.
  • Scientific journals, and other technical magazines and whitepapers.
Smiley face

Preliminary Data Mining

The raw data is obtained through the secondary findings, in house repositories, and trade surveys. It is then filtered to ensure that the relevant information including industry dynamics, trends, and outlook is retained for further research process.

Data Standardization:

Holistic approach is used to ensure that the granular and uncommon parameters are taken into consideration to ensure accurate results. The information from the paid databases are further combined to the raw data in order to standardize it.

Coherent Statistical model

We arrive at our final research findings through simulation models. Coherent Data Analytics Model is a statistical tool that helps company to forecast market estimates. Few of the parameters considered as a part of the statistical model include:

  • Micro-economic indicators
  • Macro-economic indicators
  • Environmental indicators
  • Socio-political indicators
  • Technology indicators

Data Processing

Once the findings are derived from the statistical model, large volume of data is process to confirm accurate research results. Data analytics and processing tools are adopted to process large chunk of collected informative data. In case, a client customizes the study during the process, the research finding till then are benchmarked, and the process for new research requirement is initiated again.

Data Validation

This is the most crucial stage of the research process. Primary Interviews are conducted to validate the data and analysis. This helps in achieving the following purposes:

  • It provides first-hand information on the market dynamics, outlook, and growth parameters.
  • Industry experts validates the estimates which helps the company to cement the on-going research study.
  • Primary research includes online surveys, face-to face interviews, and telephonic interviews.

The primary research is conducted with the ecosystem players including, but not limited to:

  • Raw Material Suppliers
  • Manufacturers
  • System Integrators
  • Distributors
  • End-users

Choose License Type

"How Can I Help You" ?

Finding it difficult to find the research that would cater to your business demands? Give us a chance to help you. One of our Research Consultants will connect to provide a customized solution.

[email protected]

Key Benefits

  • Custom Research Solution

    Talk to our research consultant to design an exclusive report as per your research needs.

  • Customer Fulfilment

    We aim to fulfil client's research demands with tailored research solutions.

  • Quick Turnaround Time Reports

    We aim to provide research studies in quickest turnaround time and in a much cost effective manner.

  • 360-degree Approach

    We cover each industry from supply and demand side with an aim to provide a most holistic research study.

  • Assured Quality

    We strive to provide most accurate and reliable research findings in our research reports.