Home » Healthcare » Pharmaceuticals » Global Pompe Disease Therapeutics Market

Global Pompe Disease Therapeutics Market – Growth, Future Prospects & Competitive Analysis, 2016-2024

Price: $4999

Published: | Report ID: 5989 | Report Format : PDF


Pompe disease, also known as lysosomal storage disorder, is an inherited, rare disease that is characterized by progressive muscle weakness and a respiratory disorder. Pompe disease usually occurs in 1 in 40,000 newborn babies, with the lowest rate observed in countries like Australia and New Zealand and a higher rate among African American populations. The major factorial entities supporting the growth in demand for Pompe disease treatment are its wide prevalence, the advancement of new genome sequencing, companion diagnostic kits, pharmacogenomics, research and development infrastructure, and regulatory agency initiatives.

Pompe disease is common in developed countries where disease awareness and diagnosis are made early on. New genome sequencing has been developed to a large extent, which will be helpful in the proper understanding of this disease at the fetal stage and at the genomic level. Companion diagnostic kits have been approved by the FDA, which will help in the positive growth of the Pompe disease therapeutics market. Recent advances in pharmacogenomics will aid in understanding the drug’s effect at the desired gene site and, thus, in developing drug delivery techniques such as enzyme replacement therapy, gene therapy, and so on. Big pharmaceutical players are investing in the research and development infrastructure in developing countries, which will bring the diagnosis and treatment of Pompe disease under their ambit. Regulatory agencies have created a special drug designation for the treatment of orphan diseases (Orphan Drug Act of 1983), which will be critical in the expansion of Pompe disease treatment. The limited number of Pompe disease patients would be a challenge in this scenario. Patient compliance during the treatment is a big hurdle, as the treatment demands a long-term commitment. The number of drugs in the pipeline is small as the available genomic data for a proper understanding of the disease is limited. Restraints regarding the Pompe disease therapeutic market are ambiguous reimbursement policies, biomarkers, and inaccurate process validation. Ambiguous reimbursement policies cause genetic discrimination in the cost coverage for rare diseases, which is the biggest hurdle for expenditure in the healthcare domain. The biomarkers used for the diagnosis of genetic diseases do not have the optimal structural configuration, which results in an inappropriate diagnosis of the disease. The process of process validation regarding the signaling pathways that are linked to Pompe disease needs to be explored, and the interpretation of the results in the diagnosis and treatment of Pompe disease is still a burden as the number of trained healthcare professionals is very small, which in turn acts as a hurdle in the growth of the Pompe disease therapeutics market.

The pipeline products for Pompe disease in phase 3 are AT-982 (Audentes Therapeutics), NeoGAA GZ402666 (Sanofi), and the phase 2 product is BMN 701 (Biomarin). The molecule in the preclinical study is MOSS-GAA (Greenovation Biopharmaceuticals). The other treatment areas that are employed for Pompe disease treatment are substrate reduction therapy, stem cell therapy, and protein chaperones.

The global Pompe disease therapeutics market is segmented on the following basis:

  • Therapeutics
  • Enzyme Replacement Therapy (Acid Alpha-Glucosidase)
  • Gene therapy (Adeno-associated virus)

By Geography

  • North America
    • U.S.
    • Canada
  • Europe
    • Germany
    • France
    • Italy
    • U.K.
    • Russia
    • Rest of Europe
  • Asia-Pacific
    • India
    • China
    • Japan
    • Rest of Asia-Pacific
  • Latin America
    • Brazil
    • Mexico
    • Rest of Latin America
  • Middle East and Africa
    • GCC Countries
    • South Africa
    • Rest of Middle East and Africa

Key players identified for the global Pompe disease therapeutics market include:

The major pharmaceutical players involved in enzyme replacement therapy are Sanofi (Neo GAA), Audentes Therapeutics (AT-982), Amicus Therapeutics (ATB200/AT2221), and Oxyrane OXY (2810). Manufacturers involved in gene therapy (adeno-associated virus) are Ceregene (110), Sangamo (Myozyme), and Genzyme.

This report offers the following:

  • an overview of the global markets for Pompe disease therapeutics
  • Market trends assessment for the period 2014-2024, including historical data for 2014 and 2015 as well as projections through 2024, with respective CAGRs from 2016 to 2024.
  • Qualitative assessment tools such as market drivers, challenges, and future prospects
  • Market competition examination tools such as market share analysis and fractal map evaluation
  • Focus on each level of market segmentation based on product approvals, launches, and current and anticipated market dynamics.
  • A general overview of the industry structure.
  • Company profiles highlight key information about the major players operating in the global Pompe disease therapeutics market.

Frequently Asked Questions: 

What is the size of Global Pompe Disease Therapeutics Market?

The market for Global Pompe Disease Therapeutics Market is expected to reach XX Bn in 2024.

What is the Global Pompe Disease Therapeutics Market CAGR

The Global Pompe Disease Therapeutics Market is expected to see significant CAGR growth over the coming years, at XX%.

What is the Forecast period considered for Global Pompe Disease Therapeutics Market?

The report is forecasted to 2016-2024.

What is the base year considered for Global Pompe Disease Therapeutics Market?

The base year of this report is 2015.

Intravitreal Injectable Market

Report ID: 7250

Pharmacogenomics Market

Report ID: 9486

Nosocomial Infection Treatment Market

Report ID: 14189

Anti-Cancer Drugs Market

Report ID: 39971

Vial Adaptors for Reconstitution Drug Market

Report ID: 15561

Congenital Disabilities Market

Report ID: 39845

Acquired Hemophilia Treatment Market

Report ID: 1124

Tyrosine Kinase Inhibitors (TKIs) Market

Report ID: 39696

Peptide Therapeutics Market

Report ID: 9142

Nootropics Market

Report ID: 14433

Fabry Disease Therapeutic Market

Report ID: 12266

Thioredoxin Market

Report ID: 39390

Purchase Options

Delivery Format: Excel.
Designed for the individual purchaser.
Users located at a single corporate site or regional office.
Allowed for unlimited sharing globally within one company.
Smallform of Sample request

Have a question?

User Profile

Don’t settle for less – trust Mitul to help you find the best solution.

Report delivery within 24 to 48 hours

– Other Info –

What people say?-

User Review

I am very impressed with the information in this report. The author clearly did their research when they came up with this product and it has already given me a lot of ideas.

Jana Schmidt
CEDAR CX Technologies

– Connect with us –


+91 6232 49 3207


24/7 Research Support


– Research Methodology –

Going beyond the basics: advanced techniques in research methodology

– Trusted By –

Pepshi, LG, Nestle
Motorola, Honeywell, Johnson and johnson
LG Chem, SIEMENS, Pfizer
Unilever, Samsonite, QIAGEN