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:
- Enzyme Replacement Therapy (Acid Alpha-Glucosidase)
- Gene therapy (Adeno-associated virus)
- North America
- Rest of Europe
- Rest of Asia-Pacific
- Latin America
- 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.