The global Pompe disease market size is anticipated to expand at a substantial CAGR during the forecast period, 2021–2028. The growth of the market is attributed to its ongoing use of long-term treatments, the introduction of new medicines, and the high cost of treatment.
Pompe disease is a hereditary condition in which the body's cells accumulate a complex substance called glycogen. The disorder is caused by a lack of an enzyme called acid alfa glucosidase (GAA), which helps the body break down complex sugars. Organs and tissues, particularly muscles, get clogged, leading them to break down. This disease is caused by mutations in the GAA gene, which aids in the breakdown of glycogen. Pompe disease is divided into three categories. Classic infantile onset occurs within a few months of birth, non-classic infantile onset occurs around the age of one year, and late-onset occurs later in a child's life, maybe even into teen years or adulthood. As this is a hereditary disorder, those who develop it acquire it from one of their parents. However, it is very uncommon for neither parent to exhibit any signs or symptoms. It is a rare condition. Pompe disease affects 1 out of every 40,000 people in the United States. It can affect both men and women from all ethnic backgrounds.
The enzymes in the blood are examined and counted when a blood sample is collected. Sleep studies, lung capacity tests, and electromyography are some of the other tests available (a test that measures how well the muscles work). The enzymes in the blood are examined and measured when a blood sample is collected. DNA testing is used to confirm the findings. Other tests include taking of a complete patient and family history, breathing tests to measure lung capacity (pulmonary function tests), Electromyography (a test that measures how well the muscles work) and MRIs, Heart studies, including X-rays, electrocardiogram and echocardiogram, Sleep studies. All individuals with Pompe disease are eligible for enzyme replacement therapy (ERT). Intravenously (through a vein in the patient), alglucosidase alfa is administered. It is a genetically modified enzyme that replicates the acid alpha glucosidase enzyme found naturally in the body. For people with Pompe disease, specialist teams (cardiologists, respiratory therapists, neurologists, and others) can treat symptoms and provide supportive care. Inquire about the specifics of your situation with your physicians. Infants with Pompe disease will lead to death if they are not treated. Many patients with Pompe disease have respiratory (breathing) issues, cardiac issues, and muscular weakness.
Market Trends, Drivers, Restraints, and Opportunities
- Increasing prevalence of Pompe disease around the world is projected to boost the market growth during the forecast period.
- Increasing government initiatives for developing and manufacturing innovative products for the diagnosis & treatment is anticipated to fuel the market growth during the forecast period.
- Rising consumer awareness regarding the Pompe disease around the world and increasing healthcare expenditure are expected to drive the market growth in the coming years.
- High cost of treatment is anticipated to restrict the market growth in the coming years.
- Increased R&D investments to find new effective solutions for the diagnosis and treatment and technological advancements are expected to offer lucrative opportunities for the market players.
Scope of the Report
The report on the global Pompe disease market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have also been included in the report.
Pompe Disease Market - Global Industry Analysis, Growth, Share, Size, Trends, and Forecast
Type (Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease, and Non-Classic infantile-onset Pompe Disease), Therapy Type (Enzyme Replacement Therapy, Gene Therapy, and Others), and End User (Hospitals and Clinics and Research and Academic Institutes)
Asia Pacific, North America, Latin America, Europe, and Middle East & Africa
Company Share, Market Analysis and Size, Competitive Landscape, Growth Factors, and Trends, and Revenue Forecast
Key Players Covered in the Report
Amicus Therapeutics; Sanofi; Audentes Therapeutics; Oxyrane; Valerion Therapeutics; AVROBIO, Inc.; and CENTOGENE AG.
Market Segment Insights
Late-onset Pompe disease segment is expected to constitute a key share
Based on type, the Pompe disease market is segmented into late-onset Pompe disease, classic infantile-onset Pompe disease, and non-classic infantile-onset Pompe disease. The late-onset Pompe disease segment is expected to constitute a key share of the market during the forecast period as it is only long-term therapies available for the treatment of the disease.
Enzyme replacement therapy segment is expected to account for a key share
Based on therapy type, the Pompe disease market is segmented into enzyme replacement therapy (ERT), gene therapy, and others. The enzyme replacement therapy segment is expected to account for a key share of the market during the forecast period owing to strong preference for enzyme replacement therapy over alternative treatments.
Hospitals & clinics segment is expected to represent a key share
Based on end user, the Pompe disease market is segmented into hospitals & clinics and research & academic institutes. The hospitals & clinics segment is expected to represent a key share of the market during the forecast period owing to increase in number of private and government hospitals in emerging countries.
North America is anticipated to dominate the market
On the basis of regions, the Pompe disease market is classified as Asia Pacific, North America, Latin America, Europe, and Middle East & Africa. North America is anticipated to dominate the market during the forecast period. The regional market growth can be attributed to the presence of key payers and an increasing prevalence of Pompe disease patients in the region. Furthermore, the presence of a large number of hospitals in the region would aid market expansion throughout the forecast period.
On the other hand, Asia Pacific is expected to exhibit a rapid growth rate in the coming years due to rising healthcare expenditure, increased patient awareness of rare diseases, and the expansion of the healthcare industry in this region.
Segments Covered in the Report
The global Pompe disease market has been segmented on the basis of
- Late-Onset Pompe Disease
- Classic Infantile-Onset Pompe Disease
- Non-Classic infantile-onset Pompe Disease
- Enzyme Replacement Therapy
- Gene Therapy
- Hospitals and Clinics
- Research and Academic Institutes
- Asia Pacific
- North America
- Latin America
- Middle East & Africa
- Amicus Therapeutics, Inc.
- Audentes Therapeutics
- Valerion Therapeutics
- AVROBIO, Inc.
- CENTOGENE AG
Key players competing in the Pompe disease market are Amicus Therapeutics; Sanofi; Audentes Therapeutics; Oxyrane; Valerion Therapeutics; AVROBIO, Inc.; and CENTOGENE AG.
Some of the key business strategies employed by companies includes mergers, acquisitions, partnerships.