The global Pompe Disease market size reached USD 1,386.09 Million in 2021 and is expected to register a revenue CAGR of 3.9% during the forecast period, according to a latest analysis by Emergen Research.
The global Pompe Disease market size reached USD 1,386.09 Million in 2021 and is expected to register a revenue CAGR of 3.9% during the forecast period, according to a latest analysis by Emergen Research. The growing prevalence of lysosomal storage disorders and the availability of government funding for research & development activities are some of the key factors driving the growth of this market.
Pompe disease is a rare, inherited disorder caused by mutations in the GAA gene. This disease leads to the buildup of glycogen in muscles and other tissues, which can eventually lead to muscle weakness and respiratory problems. The most common symptom of Pompe disease is muscle weakness, which typically begins in infancy or early childhood. Other symptoms may include enlarged heart, difficulty breathing, feeding problems, and slowed growth. There is currently no cure for Pompe disease, but with early diagnosis and treatment, most people with the disease can expect to live a relatively normal life span. Treatment typically includes enzyme replacement therapy (ERT), which can help to improve muscle strength and function, and may also include other therapies such as physical therapy and occupational therapy.
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According to the Muscular Dystrophy Association (MDA), 20 states are currently tracking the disease, and more than 500 cases have been diagnosed in the United States. The MDA estimates that approximately one in every 40,000 people has Pompe disease.
Rapid scientific advances in research and development of Pompe disease treatments have been made over the past decade. The use of enzyme replacement therapy (ERT) and other therapeutic approaches has led to an improved understanding of this disease and a potential for its treatment. Despite these advances, however, there are still no FDA-approved treatments for Pompe disease, making it a challenge for patients and families to seek out and afford care.
Some Key Highlights From the Report
- The Chaperone Advanced Replacement Therapy (CHART) segment accounted for a moderate revenue share in 2021 due to the ongoing clinical trials of MyoCell-based therapy for the treatment of Pompe disease. MyoCell is an investigational autologous myoblasts transplantation program that uses a patient’s own muscle cells to repair and replace damaged tissue caused by Pompe disease. This approach may improve heart function and quality of life for patients with Pompe disease. MyoCell is currently being studied in an international, multicenter clinical trial (the CHART-1 trial). The first patient in the study was treated in September 2015, and the trial is expected to enroll a total of 18 patients. If the results of the CHART-1 trial are positive, it is possible that MyoCell-based therapy could become the new standard of care for Pompe disease.
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- The home care segment is expected to witness significant growth over the forecast period due to the preference for home-based treatments and the rising number of patients opting for self-care. Moreover, the development of novel therapies and the availability of advanced home care products are expected to drive market growth.
- The North American region is expected to witness significant growth over the forecast period due to the presence of a large number of pompe disease patients, the availability of specialized treatments, and the growing awareness about the disease. Other factors such as the availability of government funding and the presence of key market players are also expected to fuel market growth in the region.
- The Asia Pacific region is expected to witness significant growth over the forecast period due to the growing prevalence of pompe disease, the availability of government funding, and the increasing number of awareness programs. Other factors such as the launch of new products and the presence of key market players are also expected to fuel market growth in the region.
- Some major companies in the global market report include Sanofi S.A., Amicus Therapeutics, Inc., BioMarin Pharmaceutical, Valerion Therapeutics, Oxyrane, Genzyme Corporation, EpiVax, Inc., Bayer AG, Audentes Therapeutics, and Sangamo Therapeutics.
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Emergen Research has segmented global Pompe Disease market on the basis of type, treatment type, end-use and region:
- Type Outlook (Revenue, USD Million; 2019-2030)
- Infantile-Onset Pompe Disease (IOPD)
- Late-Onset Pompe Disease (LOPD)
- Treatment Type Outlook (Revenue, USD Million; 2019-2030)
- Enzyme Replacement Therapy (ERT)
- Chaperone Therapy
- Substrate Replacement Therapy (SRT)
- Others
- End-Use Outlook (Revenue, USD Million; 2019-2030)
- Hospitals
- Diagnostic Centers
- Others
- Regional Outlook (Revenue, USD Million; 2019-2030)
- North America
- U.S.
- Canada
- Mexico
- Europe
- Germany
- France
- U.K.
- Italy
- Spain
- Benelux
- Rest of Europe
- Asia Pacific
- China
- India
- Japan
- South Korea
- Rest of APAC
- Latin America
- Brazil
- Rest of LATAM
- Middle East & Africa
- Saudi Arabia
- U.A.E.
- South Africa
- Turkey
- Rest of MEA
- North America
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