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.

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

Related Reports

Pompe Disease Pipeline

Pompe Disease Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Pompe disease companies, including Immusoft, Asklepios Biopharmaceutical, Oxyrane, Maze Therapeutics, Amicus Therapeutics, Spark Therapeutics, among others.

Pompe Disease Epidemiology

Pompe Disease Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted Pompe disease epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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