The expected launch of various emerging therapies along with various approved therapies shall fuel the growth of the Pompe disease market during the forecast period, i.e., 2023–2034.
DelveInsight’s “Pompe Disease Market Insights, Epidemiology, and Market Forecast 2034” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Pompe Disease market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The Pompe Disease market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2020 to 2034. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Key highlights from the Pompe Disease Market Report:
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As per DelveInsight analysis, the Pompe disease market is expected to grow positively at a significant CAGR during the study period (2020–2034).
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Key pompe disease companies working in the pompe disease market are Genzyme, Sanofi, Amicus Therapeutics, Actus Therapeutics, Valerion Therapeutics, Astellas Therapeutics, Roche, Lacerta Therapeutics, and others.
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Some key therapies for Pompe disease treatment include ACTUS-101, AT845, Cipaglucosidase alfa, Avalglucosidase alfa (GZ402666), SPK-3006, and others.
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Pompe disease, also known as glycogen storage disease type II, is a rare inherited metabolic disorder caused by a deficiency of the enzyme acid alpha-glucosidase, which is essential for breaking down glycogen into glucose within lysosomes. When this enzyme is deficient, glycogen accumulates in various tissues, particularly muscle cells, leading to progressive damage.
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In 2023, approximately 8,600 prevalent cases of Pompe disease were reported in the United States, with 98% of cases diagnosed in adult patients. A significant portion of Pompe disease cases are infantile-onset, which often results in high mortality within the first few months due to severe muscle weakness, poor muscle tone, and respiratory issues. This rapid progression causes considerable psychological distress for families.
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In the United States, the incidence of the CRIM-positive phenotype in infantile-onset Pompe disease was notably higher in 2023, reaching around 80%. The CRIM-positive phenotype is characterized by residual enzyme activity, which can influence both the disease’s progression and its response to treatment.
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Several enzyme replacement therapies for Pompe disease have been approved by the US FDA, including MYOZYME (alglucosidase alfa) and LUMIZYME (alglucosidase alfa) by Genzyme/Sanofi, and POMBILITI (cipaglucosidase alfa-atga) by Amicus Therapeutics.
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The Pompe disease treatment pipeline is still limited, with only a few candidates advancing through clinical trials. Notable candidates include AAV2/8-LSPhGAA (ACTUS-101) by Asklepios Biopharmaceutical in Phase I/II, and Avalglucosidase alfa by Sanofi, currently in Phase III. The rarity of Pompe disease presents challenges in patient recruitment for clinical trials, which in turn delays the development of new treatments.
Read detailed insights on Pompe Disease market outlook 2034 @ https://www.delveinsight.com/report-store/acute-respiratory-distress-syndrome-Pompe Disease-market
Pompe Disease Overview
Pompe disease is a rare, inherited metabolic disorder caused by mutations in the GAA gene, leading to a deficiency in the enzyme acid alpha-glucosidase. This enzyme is crucial for breaking down glycogen into glucose within lysosomes. When the enzyme is deficient, glycogen accumulates in cells, particularly in muscles, resulting in progressive muscle weakness, respiratory issues, and other severe complications. Pompe disease has two primary forms: infantile-onset, which appears within the first few months of life and often leads to early mortality if untreated, and late-onset, which can develop from childhood to adulthood and progresses more slowly.
Diagnosis of Pompe disease typically involves enzyme assays to assess acid alpha-glucosidase activity in blood or muscle tissue, genetic testing to confirm GAA mutations, and muscle biopsies. Early detection is crucial for effective management, especially in the case of infantile-onset Pompe disease.
The main treatment for Pompe disease is enzyme replacement therapy (ERT), which replenishes the deficient enzyme. MYOZYME and LUMIZYME (both alglucosidase alfa), developed by Sanofi/Genzyme, have been the standard ERT treatments for many years. More recently, POMBILITI (cipaglucosidase alfa-atga), developed by Amicus Therapeutics, was approved as an alternative ERT. In addition, researchers are investigating gene therapy approaches, such as AAV2/8-LSPhGAA by Asklepios Biopharmaceutical, as potential future treatments.
Supportive care is also essential and typically includes respiratory management, physical therapy, and nutritional support to address the muscle weakness and respiratory difficulties common in Pompe disease. These interventions help preserve mobility, improve breathing, and manage complications.
Pompe Disease Epidemiology Insights
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According to DelveInsight’s estimates, the total number of prevalent Pompe disease cases in the 7MM (United States, EU4, and Japan) was approximately 13,000 in 2023, with projections showing an increase by 2034 at a compound annual growth rate (CAGR) of XX%.
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In 2023, EU4 and the UK accounted for approximately 25% of the total prevalent Pompe disease cases. In the United States alone, around 8,600 cases were diagnosed, with 98% of these cases reported in adult patients.
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A significant portion of Pompe disease cases are infantile-onset, which often leads to high mortality within the first few months due to severe muscle weakness, poor muscle tone, and respiratory difficulties. This rapid progression can cause considerable emotional distress for families.
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In 2023, the incidence of the CRIM-positive phenotype in infantile-onset Pompe disease was notably higher in the United States, reaching around 80%. The CRIM-positive phenotype, characterized by residual enzyme activity, can impact disease progression and influence the response to treatment.
Pompe Disease Epidemiology Segmentation:
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Total Pompe Disease Diagnosed Prevalent Cases
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Total Live Birth Cases of Pompe Disease
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Total Adult Cases of Pompe Disease
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Pompe Disease Type-specific Diagnosed Prevalent Cases
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Pompe Disease Treated Cases
Pompe Disease Treatment Market
In 2023, the total market for Pompe disease therapies across the seven major markets (7MM) was valued at USD 18.4 million, with the United States capturing approximately 70% of this market share. The US market significantly outpaced the EU4 (Germany, Spain, Italy, France), the UK, and Japan.
Within the EU4 and the UK, Germany held the largest market share, accounting for around USD 94 million, while France had the second-largest market, with [XX] representing the smallest market size in 2023.
Asklepios Biopharmaceutical is developing AAV2/8-LSPhGAA, a gene therapy candidate that aims to treat Pompe disease by delivering the GAA gene to restore enzyme function. Alongside Avalglucosidase alfa by Sanofi, which represents the next generation of enzyme replacement therapy (ERT), these candidates have the potential to address significant unmet needs in Pompe disease treatment. While early-stage results for AAV2/8-LSPhGAA are promising, pivotal trials will be crucial to confirm its safety and efficacy in humans.
Pompe Disease Therapies and Key Companies
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ACTUS-101: Asklepios Biopharmaceutical, Inc.
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AT845: Astellas Pharma Inc
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Cipaglucosidase alfa: Amicus Therapeutics
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Avalglucosidase alfa (GZ402666): Sanofi
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SPK-3006: Spark Therapeutics
Learn How the Pompe Disease Market Will Evolve and Grow by 2034 @ https://www.delveinsight.com/sample-request/acute-respiratory-distress-syndrome-Pompe Disease-market
Pompe Disease Market Dynamics
The market for Pompe disease therapies has garnered increasing interest due to advancements in understanding the condition and developing innovative treatments. Several factors have driven these changes. Firstly, heightened awareness and enhanced diagnostic tools have led to earlier and more accurate diagnoses, increasing the patient population. This has prompted pharmaceutical companies to invest in research and development to create new therapies, including enzyme replacement and gene therapies. Additionally, regulatory agencies have played a pivotal role in shaping the market. Accelerated approval processes and orphan drug designations have motivated pharmaceutical companies to pursue treatments for Pompe disease. These factors, along with incentives for rare disease research, have created a more competitive landscape.
However, several challenges hinder the growth of the Pompe disease market. One significant obstacle is the high cost of developing and manufacturing therapies for rare diseases like Pompe. The limited patient population and specialized nature of these treatments result in high prices, which can complicate patient access and reimbursement. Another challenge is the disease’s complexity. Pompe disease shows considerable variability in its clinical presentation and progression, making it difficult to develop a universal treatment approach. Customizing therapies for individual patients is a goal, but it presents technical and regulatory challenges.
Furthermore, there is a need to raise awareness not only among the general public but also within the medical community. Many cases of Pompe disease are initially misdiagnosed due to its rarity, delaying appropriate treatment. In some regions, regulatory barriers can impede the introduction of innovative therapies, as local approval processes and reimbursement policies may not be well-suited to accommodating rare diseases.
Leading Players in the Pompe Disease Therapeutics Market Include:
Pompe Disease Companies working in the market are Genzyme, Sanofi, Amicus Therapeutics, Actus Therapeutics, Valerion Therapeutics, Astellas Therapeutics, Roche, Lacerta Therapeutics, and others.
Pompe Disease Report Covers the In-depth Assessment of the Emerging Pompe Disease Drugs & Key Companies. Download the Pompe Disease Market Sample Report to Learn More @ https://www.delveinsight.com/report-store/acute-respiratory-distress-syndrome-Pompe Disease-market
Table of Contents
1. Key Insights
2. Executive Summary
3. Pompe Disease Competitive Intelligence Analysis
4. Pompe Disease Market Overview at a Glance
5. Pompe Disease Disease Background and Overview
6. Pompe Disease Patient Journey
7. Pompe Disease Epidemiology and Patient Population (In the US, EU5, and Japan)
8. Pompe Disease Treatment Algorithm, Current Treatment, and Medical Practices
9. Pompe Disease Unmet Needs
10. Key Endpoints of Pompe Disease Treatment
11. Pompe Disease Marketed Products
12. Pompe Disease Emerging Drugs and Latest Therapeutic Advances
13. Pompe Disease Seven Major Market Analysis
14. Attribute Analysis
15. Pompe Disease Market Outlook (In US, EU5, and Japan)
16. Pompe Disease Access and Reimbursement Overview
17. KOL Views on the Pompe Disease Market
18. Pompe Disease Market Drivers
19. Pompe Disease Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
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