DelveInsight's 'Pompe Disease (PD) - Market Insights, Epidemiology and Market Forecast-2030' report delivers an in-depth understanding of the disease, historical &forecasted epidemiology as well as the market trends of PD in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom) and Japan.
The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Pompe Disease (PD) from 2017 to 2030 segmented by seven major markets. The Report also covers current treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market.
- The United States
- EU5 (Germany, France, Italy, Spain and the United Kingdom)
Study Period: 2017-2030
Pompe Disease (PD) - Disease Understanding and Treatment Algorithm
Pompe disease (PD), also known as glycogen storage disease type II (GSDII) or "acid maltase deficiency", is caused by the absence or deficiency of acid alpha-glucosidase (GAA), a lysosomal enzyme that is responsible for the cleavage of the α-1, 4- and α-1, 6-glycosidic bonds of glycogen to glucose. The disease is caused pathogenic variations in the acid alpha-glucosidase (GAA) gene. Close to 500 different GAA gene variations have been identified in families with this disorder.
The treatment of Pompe disease is disease-specific, symptomatic, and supportive. Treatment requires the coordinated efforts of a team of specialists with expertise in treating neuromuscular disorders. Pediatricians or internists, neurologists, orthopedists, cardiologists, dieticians, and other healthcare professionals may need to systematically and comprehensively plan an affect child's treatment. Enzyme replacement therapy is an approved treatment for all patients with Pompe disease. It involves the intravenous administration of recombinant human acid α-glucosidase. This treatment, manufactured by Genzyme, a Sanofi Corporation.
Pompe Disease Diagnosis
The diagnosis of Pompe disease often poses a diagnostic dilemma due to the rarity of the condition and the relatively nonspecific nature of the phenotypic features that may only lead to suspicion of Pompe disease. A chest x-ray and electrocardiogram (ECG) are valuable screening tests in the diagnostic algorithm for infantile Pompe disease and an echocardiogram is a valuable next step. Chest x-ray shows massive cardiomegaly. ECG shows a short PR interval as well as very tall QRS complexes. Among patients presenting with cardiomyopathy, an electromyogram (EMG) can be useful to document presymptomatic myopathy. Blood tests should include a serum creatine kinase (CK) examination. CK elevation is a sensitive although very nonspecific marker for Pompe disease.
Pompe Disease Treatment
It covers the details of conventional and current medical therapies available in the Pompe Disease market for the treatment of the condition. It also provides the country-wise treatment guidelines and algorithm across the United States, Europe and Japan.
The DelveInsight Pompe Disease market report gives a thorough understanding of PD by including details such as disease definition, symptoms, causes, pathophysiology, and diagnosis. It also provides PD treatment algorithms and treatment guidelines in the US, Europe, and Japan.
Pompe Disease Epidemiology
The Pompe Disease (PD) epidemiology division provide the insights about historical and current patient pool and forecasted trend for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total live birth cases of PD, Total Adult Prevalent cases of PD, Total Prevalent cases of PD, Comorbidities Prevalence of PD by Onset Types, Incidence of IOPD based on clinical phenotypes) scenario of Pompe Disease(PD) in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017-2030.
Pompe Disease Drug Chapters
This segment of the Pompe Disease report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details and the latest news and press releases.
The Pompe Disease (PD) drugs market is dominated by the use of Enzyme Replacement Therapies (ERT) along with the use of supportive therapies for the management of PD. Most patients require supportive therapy to address the symptoms of Pompe disease, which include respiratory and cardiac problems, physical disability, and difficulty swallowing. Although historically, supportive care had been the mainstay of treatment (thereby generating a huge amount of revenue), but the approval and subsequent launch of ERT in 2006 after decades of research, helped turn the treatment landscape upside down, prompting a new era in the treatment of this disease. This was the first disease-specific treatment for an inherited muscular disorder consisting of the intravenous administration of recombinant human GAA (Myozyme / Lumizyme).
Pompe Disease Marketed Drugs
Lumizyme/ Myozyme (alglucosidase alfa): Sanofi Genzyme
LUMIZYME (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency). It is an enzyme replacement therapy produced by recombinant DNA technology. It provides an exogenous source of acid α glucosidase (GAA), an essential lysosomal enzyme that is deficient or absent in patients with Pompe disease, leading to intralysosomal glycogen accumulation.
Pompe Disease Emerging Drugs
AT-GAA: Amicus Therapeutics
AT-GAA is an investigational therapy that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In Feb 2019, the US Food and Drug Administration (FDA) granted Amicus a Breakthrough Therapy Designation ("BTD") to AT-GAA for the treatment of late-onset Pompe disease
Pompe Disease Market Outlook
The Pompe Disease market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.
This segment gives a through detail of market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
According to DelveInsight, the market of Pompe Disease in 7MM is expected to undergo a major positive shift during the course of the study period (2017-2030).Among the 7MM, the United States accounts for the largest market size of PD, in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
This section includes a glimpse of the Pompe Disease market in 7MM.
The United States Market Outlook
This section provides the total Pompe Disease market size and market size by therapies in the United States.
EU-5 Countries: Market Outlook
The total Pompe Disease market size and market size by therapies in Germany, France, Italy, Spain and the United Kingdom is provided in this section.
Japan Market Outlook
The total Pompe Disease market size and market size by therapies in Japan is also mentioned.
Pompe Disease Drugs Uptake
This section focuses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2017-2030. The analysis covers market uptake by drugs; patient uptake by therapies and sales of each drug.
This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
Pompe Disease Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase II, and Phase III stage. It also analyses Pompe Disease key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for Pompe Disease emerging therapies.
Reimbursement Scenario in Pompe Disease
Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In report we take reimbursement into consideration to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business & price strategy.
To keep up with current market trends, we take KOLs and SME's opinion working in Pompe Disease domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Pompe Disease market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.
Competitive Intelligence Analysis
We perform Competitive & Market Intelligence analysis of the Pompe Disease Market by using various Competitive Intelligence tools that includes - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.
Scope of the Report:
- The report covers the descriptive overview of Pompe Disease, explaining its causes, signs and symptoms, pathophysiology and currently available therapies
- Comprehensive insight has been provided into the Pompe Disease epidemiology and treatment in the 7MM
- Additionally, an all-inclusive account of both the current and emerging therapies for Pompe Disease are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
- A detailed review of Pompe Disease market; historical and forecasted is included in the report, covering drug outreach in the 7MM
- The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Pompe Disease market
- In the coming years, Pompe Disease market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the Size of the market to enable the drug manufacturers to penetrate more into the market
- The companies and academics are working to assess challenges and seek opportunities that could influence Pompe Disease R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
- Major players are involved in developing therapies for Pompe Disease. Launch of emerging therapies, will significantly impact the Pompe Disease market
- A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Pompe Disease
- Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities
Pompe Disease Report Insights
- Patient Population
- Therapeutic Approaches
- Pipeline Analysis
- Market Size and Trends
- Market Opportunities
- Impact of upcoming Therapies
Pompe Disease Report Key Strengths
- 11 Year Forecast
- 7MM Coverage
- Epidemiology Segmentation
- Drugs Uptake
- Highly Analyzed Market
- Key Cross Competition
Pompe Disease Report Assessment
- Current Treatment Practices
- Unmet Needs
- Detailed Pipeline Product Profiles
- Market Attractiveness
- Market Drivers and Barriers
- What was the Pompe Disease Market share (%) distribution in 2017 and how it would look like in 2030?
- What would be the Pompe Disease total market Size as well as market Size by therapies across the 7MM during the forecast period (2020-2030)?
- What are the key findings pertaining to the market across 7MM and which country will have the largest Pompe Disease Market Size during the forecast period (2020-2030)?
- At what CAGR, the Pompe Disease market is expected to grow in 7MM during the study period (2017-2030)?
- What would be the Pompe Disease market outlook across the 7MM during the forecast period (2020-2030)?
- What would be the Pompe Disease market growth till 2030 and what will be the resultant market size in the year 2030?
- How would the market drivers, barriers and future opportunities affect the market dynamics and a subsequent analysis of the associated trends?
- What is the disease risk, burden and unmet needs of the Pompe Disease?
- What is the historical Pompe Disease patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan?
- What would be the forecasted patient pool of Pompe Disease in 7 major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan?
- What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Pompe Disease?
- Out of all 7MM countries, which country would have the highest Incident population of Pompe Disease during the forecast period (2020-2030)?
- At what CAGR the population is expected to grow in 7MM during the forecast period (2020-2030)?
Current Treatment Scenario, Marketed Drugs and Emerging Therapies:
- What are the current options for the treatment of Pompe Disease along with the approved therapy?
- What are the current treatment guidelines for the treatment of Pompe Disease (PD) in USA, Europe and Japan?
- What are the Pompe Disease marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy etc.?
- How many companies are developing therapies for the treatment of Pompe Disease?
- How many therapies are developed by each company for the treatment of Pompe Disease?
- How many emerging therapies are in mid stage, and late stage of development for the treatment of Pompe Disease?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Pompe Disease therapies?
- What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Pompe Disease and their status?
- What are the key designations that have been granted for the emerging therapies for Pompe Disease?
- What are the global historical and forecasted market of Pompe Disease?
Reasons to buy:
- The report will help in developing business strategies by understanding trends shaping and driving the Pompe Disease market
- To understand the future market competition in the Pompe Disease market and Insightful review of the key market drivers and barriers
- Organize sales and marketing efforts by identifying the best opportunities for Pompe Disease in US, Europe (Germany, Spain, Italy, France and United Kingdom) and Japan
- Identification of strong upcoming players in market will help in devising strategies that will help in getting ahead of competitors
- Organize sales and marketing efforts by identifying the best opportunities for Pompe Disease market
- To understand the future market competition in the Pompe Disease market
Table of Contents
1. Key Insights
2. Executive Summary of Pompe Disease (PD)
3. SWOT Analysis for Pompe Disease
4. Pompe Disease Market Overview at a Glance
- 4.1. Market Share (%) Distribution of Pompe Disease in 2017
- 4.2. Market Share (%) Distribution of Pompe Disease in 2030
5. Disease Background and Overview
- 5.1. Introduction
- 5.2. Types
- 5.3. Genetic defects
- 5.4. Clinical Manifestations
- 5.5. Causes
- 5.6. Signs and Symptoms
- 5.7. Pathogenesis
- 5.8. Diagnosis
6. Epidemiology and Patient Population
- 6.1. Key Findings
- 6.2. 7MM Total Prevalent Patient Population of Pompe Disease
7. Country Wise-Epidemiology of Pompe Disease
- 7.1. The United States
- 7.1.1. Assumptions and Rationale
- 7.1.2. Total Live Birth Cases of Pompe Disease in the US
- 7.1.3. Total Adult Prevalent Cases of Pompe Disease in the US
- 7.1.4. Total Prevalent Cases of Pompe Disease in the US
- 7.1.5. Prevalence of Pompe Disease by Onset Types in the US
- 7.1.6. Incidence of IOPD based on Clinical Phenotype in the US
- 7.2. EU5
- 7.2.1. Assumptions and Rationale
- 7.3. Germany
- 7.3.1. Total Live Birth Cases of Pompe Disease in Germany
- 7.3.2. Total Adult Prevalent Cases of Pompe Disease in Germany
- 7.3.3. Total Prevalent Cases of Pompe Disease in Germany
- 7.3.4. Prevalence of Pompe Disease by Onset Types in Germany
- 7.3.5. Incidence of IOPD based on Clinical Phenotype in Germany
- 7.4. France
- 7.4.1. Total Live Birth Cases of Pompe Disease in France
- 7.4.2. Total Adult Prevalent Cases of Pompe Disease in France
- 7.4.3. Total Prevalent Cases of Pompe Disease in France
- 7.4.4. Prevalence of Pompe Disease by Onset Types in France
- 7.4.5. Incidence of IOPD based on Clinical Phenotype in France
- 7.5. Italy
- 7.5.1. Total Live Birth Cases of Pompe Disease in Italy
- 7.5.2. Total Adult Prevalent Cases of Pompe Disease in Italy
- 7.5.3. Total Prevalent Cases of Pompe Disease in Italy
- 7.5.4. Prevalence of Pompe Disease by Onset Types in Italy
- 7.5.5. Incidence of IOPD based on Clinical Phenotype in Italy
- 7.6. Spain
- 7.6.1. Total Live Birth Cases of Pompe Disease in Spain
- 7.6.2. Total Adult Prevalent Cases of Pompe Disease in Spain
- 7.6.3. Total Prevalent Cases of Pompe Disease in Spain
- 7.6.4. Prevalence of Pompe Disease by Onset Types in Spain
- 7.6.5. Incidence of IOPD based on Clinical Phenotype in Spain
- 7.7. The United Kingdom
- 7.7.1. Total Live Birth Cases of Pompe Disease in the UK
- 7.7.2. Total Adult Prevalent Cases of Pompe Disease in the UK
- 7.7.3. Total Prevalent Cases of Pompe Disease in the UK
- 7.7.4. Prevalence of Pompe Disease by Onset Types in the UK
- 7.7.5. Incidence of IOPD based on Clinical Phenotype in the UK
- 7.8. Japan
- 7.8.1. Assumptions and Rationale
- 7.8.2. Total Live Birth Cases of Pompe Disease in Japan
- 7.8.3. Total Adult Prevalent Cases of Pompe Disease in Japan
- 7.8.4. Total Prevalent Cases of Pompe Disease in Japan
- 7.8.5. Prevalence of Pompe Disease by Onset Types in Japan
- 7.8.6. Incidence of IOPD based on Clinical Phenotype in Japan
8. Treatment and Management of Pompe Disease
- 8.1. Enzyme Replacement Therapy in Pompe Disease
9. Gene Therapy: What Future Holds
- 9.1. AAV Vectors
- 9.2. Gene therapy vs ERT
- 9.3. Challenges in Gene Therapy: Production & Immune Response
- 9.3.1. Production of Recombinant Adeno-Associated Vectors for Clinical Application
- 9.3.2. Immune-Responses to AAV Capsid and the Transgene, and Vector Re-Administration
10. Case Study
- 10.1. Pompe Disease: Presentation and Management of Early Onset Type with Perioperative Considerations
11. Patient Journey
12. Unmet Needs
13. Marketed Products
- 13.1. Lumizyme: Genzyme
- 13.1.1. Product Description
- 13.1.2. Regulatory Milestones
- 13.1.3. Safety and Efficacy
14. Emerging Therapies
- 14.1. Avalglucosidase alfa: Sanofi
- 14.1.1. Product Description
- 14.1.2. Clinical Development
- 14.1.3. Safety and Efficacy
- 14.2. AT-GAA: Amicus Therapeutics
- 14.2.1. Product Description
- 14.2.2. Other Developmental Activities
- 14.2.3. Regulatory Milestones
- 14.2.4. Clinical Development
- 14.2.5. Safety and Efficacy
- 14.3. ACTUS-101: Actus Therapeutics
- 14.3.1. Product Description
- 14.3.2. Other Developmental Activities
- 14.3.3. Regulatory Milestones
- 14.3.4. Clinical Development
- 14.4. VAL-1221: Valerion Therapeutics
- 14.4.1. Product Description
- 14.4.2. Product Development Activities
- 14.4.3. Clinical Development
- 14.4.4. Safety and Efficacy
- 14.5. AT845: Astellas Therapeutics
- 14.5.1. Product Description
- 14.5.2. Product Development Activities
- 14.5.3. Clinical Development
- 14.5.4. Pre-clinical Results
- 14.6. SPK-3006: Roche
- 14.6.1. Product Description
- 14.6.2. Product Development Activities
- 14.6.3. Clinical Development
- 14.6.4. Pre-clinical Results
15. Other Promising Candidates
- 15.1. AAV9: Lacerta Therapeutics
- 15.1.1. Product Description
- 15.1.2. Product Development Activities
- 15.1.3. Clinical Development
16. Pompe Disease: 7 Major Market Analysis
- 16.1. Key Findings
- 16.2. Market Size of Pompe Disease in 7MM
17. Market Outlook by Country
- 17.1. United States
- 17.1.1. The United States: Market Outlook
- 17.1.2. Total Market size of Pompe Disease (PD)
- 17.1.3. Pompe Disease Market Size by Therapies
- 17.2. EU5 Countries
- 17.2.1. EU-5 Countries: Market Outlook
- 17.2.2. Germany
- 17.2.3. France
- 17.2.4. Italy
- 17.2.5. Spain
- 17.2.6. United Kingdom
- 17.3. Japan
- 17.3.1. Japan: Market Outlook
- 17.3.2. Total Market size of Pompe Disease
- 17.3.3. Pompe Disease Market Size by Therapies
18. Market Drivers
19. Market Barriers
20. Health Economics of Pompe Disease
- 20.1. Health Economic Evidence of IOPD
- 20.1.1. Cost-Effectiveness of ERT in IOPD
- 20.2. Health Economic Evidence of LOPD
- 20.2.1. Cost-Effectiveness of ERT in LOPD
21. Market access and Reimbursement
- 21.1. Access to Medicines For Rare Diseases In The US
- 21.2. Access to Medicines for Rare Diseases in the EU5
- 21.2.1. Germany
- 21.2.2. France
- 21.2.3. Italy
- 21.2.4. Spain
- 21.2.5. UK
- 21.3. Orphan Drug Reimbursement in the US
- 21.4. Pricing and Reimbursement in the EU5 Countries
- 21.4.1. Germany
- 21.4.2. France
- 21.4.3. Italy
- 21.4.4. Spain
- 21.4.5. UK
- 21.5. Patient Access Schemes for Myozyme (Alglucosidase Alfa)
23. DelveInsight Capabilities
25. About DelveInsight