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., 2023–2032.
LAS VEGAS, Oct. 26, 2023 /PRNewswire/ — DelveInsight’s Pompe Disease Market Insights report includes a comprehensive understanding of current treatment practices, Pompe disease emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].
Key Takeaways from the Pompe Disease Market Report
As per DelveInsight analysis, the Pompe disease market is expected to grow positively at a significant CAGR during the study period (2019–2032).
As per DelveInsight analysis, the total diagnosed prevalent cases of Pompe disease in the 7MM were found to be approximately 13K cases in 2022, which are expected to increase by 2032.
Globally, leading Pompe disease companies such as Asklepios Biopharmaceutical, Inc., Astellas Pharma Inc, Amicus Therapeutics, Sanofi, Spark Therapeutics, Immusoft, Oxyrane, Maze Therapeutics, and others are developing novel Pompe disease drugs that can be available in the Pompe disease market in the coming years.
Some key therapies for Pompe disease treatment include ACTUS-101, AT845, Cipaglucosidase alfa, Avalglucosidase alfa (GZ402666), SPK-3006, and others.
Discover which therapies are expected to grab the major Pompe disease market share @ Pompe Disease Market Report
Pompe Disease Overview
Pompe disease, alternatively referred to as glycogen storage disease type II (GSD II) or acid maltase deficiency (AMD), is a rare autosomal recessive metabolic disorder resulting from a deficiency of the acid alpha-glucosidase (GAA) enzyme due to recessive mutations in the GAA gene. This disorder can manifest across all age groups, leading to a spectrum of phenotypes that can pose challenges in subtype classification. In general, two primary categories are recognized, primarily based on the timing of symptom onset and the presence or absence of cardiomyopathy.
Diagnosing Pompe disease typically involves a multi-faceted approach, which includes clinical evaluation, biochemical assessments to measure GAA enzyme activity in blood or tissue samples, and genetic testing. Electromyography (EMG) may also be employed as part of the diagnostic process. These assessments collectively form the cornerstone of the initial diagnostic workup. Notably, the functional test using skin fibroblasts to measure GAA activity is considered the gold standard due to its exceptional sensitivity.
Pompe Disease Epidemiology Segmentation
The Pompe disease epidemiology section provides insights into the historical and current Pompe disease patient pool and forecasted trends for individual seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders.
The Pompe disease market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:
Total Pompe Disease Diagnosed Prevalent Cases
Total Live Birth Cases of Pompe Disease
Total Adult Cases of Pompe Disease
Pompe Disease Type-specific Diagnosed Prevalent Cases
Pompe Disease Treated Cases
Pompe Disease Treatment Market
Pompe disease treatment is characterized by its disease-specific, symptomatic, and supportive nature. To provide effective care, a comprehensive approach is essential, involving a coordinated effort from a team of specialists with expertise in managing neuromuscular disorders. This interdisciplinary team may consist of pediatricians, internists, neurologists, orthopedists, cardiologists, dietitians, and other healthcare professionals, who collaboratively design and implement an individualized treatment plan for affected children. Genetic counseling plays a vital role in supporting affected individuals and their families, and it’s important to note that there are only two currently approved treatment regimens for this condition.
Sanofi’s NEXVIAZYME (avalglucosidase alfa-ngpt) has recently gained approval from the US FDA, marking a significant milestone in the treatment of late-onset Pompe disease. This innovative therapy is designed to target the mannose-6-phosphate (M6P) receptor, a critical pathway for enzyme replacement therapy. It effectively reduces glycogen accumulation in muscle cells, as demonstrated in successful Phase III clinical trials. Patients treated with NEXVIAZYME experienced remarkable improvements in respiratory function and walking distance, with a notable 2.9-point enhancement in forced vital capacity (FVC) percent predicted at Week 49, which was the primary endpoint of the study. The treatment involves a monotherapy enzyme replacement therapy (ERT) administered every two weeks. The recommended dosage is determined based on the patient’s body weight and is administered through a gradual intravenous infusion.
To know more about Pompe disease treatment, visit @ Pompe Disease Treatment Drugs
Key Pompe Disease Therapies and Companies
ACTUS-101: Asklepios Biopharmaceutical, Inc.
AT845: Astellas Pharma Inc
Cipaglucosidase alfa: Amicus Therapeutics
Avalglucosidase alfa (GZ402666): Sanofi
SPK-3006: Spark Therapeutics
Learn more about the FDA-approved drugs for Pompe disease @ Drugs for Pompe Disease Treatment
Pompe Disease Market Dynamics
The market for Pompe disease therapies has witnessed growing attention due to advancements in the understanding of this condition and the development of innovative treatments. Several factors have contributed to these changing dynamics. Firstly, increased awareness and improved diagnostic tools have led to earlier and more accurate diagnoses, expanding the patient population. This, in turn, has spurred pharmaceutical companies to invest in research and development to create novel therapies, including enzyme replacement and gene therapies. Additionally, regulatory agencies have played a crucial role in shaping the market. Expedited approval processes and orphan drug designations have encouraged pharmaceutical companies to pursue Pompe disease treatments. This, coupled with incentives for rare disease research, has fostered a more competitive landscape.
However, several factors are impeding the growth of the Pompe disease market. One major barrier is the high cost associated with developing and manufacturing therapies for rare diseases like Pompe. The small patient population and specialized nature of these treatments result in steep price tags, which can create challenges in terms of patient access and reimbursement. Another barrier is the complexity of the disease itself. Pompe disease exhibits considerable variability in its clinical presentation and progression, making it challenging to develop a one-size-fits-all treatment approach. Tailoring therapies to individual patients is a goal, but it poses technical and regulatory challenges.
Moreover, there’s the challenge of raising 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 hurdles can hinder the introduction of innovative therapies, as local approval processes and reimbursement policies may not be well-suited to accommodating rare diseases.
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Report Metrics |
Details |
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Study Period |
2019–2032 |
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Coverage |
7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] |
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Key Pompe Disease Companies |
Asklepios Biopharmaceutical, Inc., Astellas Pharma Inc, Amicus Therapeutics, Sanofi, Spark Therapeutics, Immusoft, Oxyrane, Maze Therapeutics, and others |
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Key Pompe Disease Therapies |
ACTUS-101, AT845, Cipaglucosidase alfa, Avalglucosidase alfa (GZ402666), SPK-3006, and others |
Scope of the Pompe Disease Market Report
Therapeutic Assessment: Pompe Disease current marketed and emerging therapies
Pompe Disease Market Dynamics: Conjoint Analysis of Emerging Pompe Disease Drugs
Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
Unmet Needs, KOL’s views, Analyst’s views, Pompe Disease Market Access and Reimbursement
Discover more about Pompe disease drugs in development @ Pompe Disease Clinical Trials
Table of Contents
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1. |
Pompe Disease Market Key Insights |
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2. |
Pompe Disease Market Report Introduction |
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3. |
Pompe Disease Market Overview at a Glance |
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4. |
Pompe Disease Market Executive Summary |
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5. |
Disease Background and Overview |
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6. |
Pompe Disease Treatment and Management |
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7. |
Pompe Disease Epidemiology and Patient Population |
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8. |
Patient Journey |
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9. |
Pompe Disease Marketed Drugs |
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10. |
Pompe Disease Emerging Drugs |
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11. |
Seven Major Pompe Disease Market Analysis |
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12. |
Pompe Disease Market Outlook |
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13. |
Potential of Current and Emerging Therapies |
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14. |
KOL Views |
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15. |
Unmet Needs |
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16. |
SWOT Analysis |
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17. |
Appendix |
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18. |
DelveInsight Capabilities |
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19. |
Disclaimer |
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20. |
About DelveInsight |
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SOURCE DelveInsight Business Research, LLP