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Pompe Disease Market by Therapy Type (Enzyme Replacement Therapy and Substrate Reduction Therapy), Molecule Type (Biologics and Small Molecules), Route of Administration (Oral and Parenteral), and Dosage Forms (Solid and Liquid) - Global Opportunity Analysis and Industry Forecast, 2019-2026
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Jul 2019 |
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Pompe disease is a disorder caused by the buildup of a complex sugar called glycogen in the body's cells. It is genetically inherited and the accumulation of glycogen in certain organs as well as tissues, especially muscles, impairs their ability to function normally. Pompe diseases affects approximately one individual in 40,000 in the U.S.; however, the incidence varies among different ethnic groups.
Increasing research activities preformed to find an effective treatment for the disease and the growth in the special regulatory drug designations for orphan drugs drive the market. However, inflated costs associated with the Pompe disease therapy restrain the market growth. Moreover, emerging immunotherapeutic approaches for Pompe disease offer lucrative opportunities for the expansion of the market.
The market is segmented on the basis of therapy type, molecule type, route of administration, dosage forms, and region. Based on therapy type, the market is divided into enzyme replacement therapy and substrate reduction therapy. Based on the molecule type, the market is classified into biologics and small molecules. Based on route of administration, the market is bifurcated into oral and parenteral. Based on the dosage forms, the market is categorized into solid and liquid. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.
Key players that operate in the market are Amicus Therapeutics, BioMarin Pharmaceutical, Genzyme, Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics.
Key Benefits
- The study provides an in-depth analysis of the global Pompe disease market with current trends and future estimations to elucidate imminent investment pockets.
- It presents a quantitative analysis from 2016 to 2023 to enable stakeholders to capitalize on prevailing market opportunities.
- Key players are profiled and their strategies are thoroughly analyzed, which predicts the competitive outlook of the market.
- By region, the market is analyzed across North America, Europe, Asia-Pacific, and LAMEA.
Pompe Disease Market Key Segments:
By Therapy Type
- Enzyme Replacement Therapy
- Substrate Reduction Therapy
By Molecule Type
- Biologics
- Small Molecules
By Route of Administration
- Oral
- Parenteral
By Dosage Forms
- Solid
- Liquid
By Region
- North America
- U.S.
- Canada
- Mexico
- Europe
- Germany
- UK
- France
- Spain
- Italy
- Rest of Europe
- Asia-Pacific
- Japan
- China
- India
- Australia
- Rest of Asia-Pacific
- LAMEA
- Brazil
- Saudi Arabia
- South Africa
- Argentina
- Rest of LAMEA
CHAPTER 1 INTRODUCTION
1.1. REPORT DESCRIPTION
1.2. KEY BENEFITS
1.3. KEY MARKET SEGMENTS
1.4. RESEARCH METHODOLOGY
1.4.1. Secondary research
1.4.2. Primary research
1.4.3. Analyst tools & models
CHAPTER 2 EXECUTIVE SUMMARY
2.1. CXO PERSPECTIVE
CHAPTER 3 MARKET OVERVIEW
3.1. MARKET DEFINITION AND SCOPE
3.2. KEY FINDINGS
3.2.1. Top investment pockets
3.2.2. Top winning strategies
3.3. MARKET SHARE ANALYSIS/TOP PLAYER POSITIONING, 2016
3.4. PORTER’S FIVE FORCES ANALYSIS
3.8. MARKET DYNAMICS
3.8.1. Drivers
3.8.2. Restraints
3.8.3. Opportunities
CHAPTER 4 GLOBAL POMPE DISEASE MARKET, BY THERAPY TYPE
4.1. OVERVIEW
4.1.1. Market size and forecast
4.2. ENZYME REPLACEMENT THERAPY
4.2.1. Key market trends
4.2.2. Growth factors and opportunities
4.2.3. Market size and forecast
4.3. SUBSTRATE REDUCTION THERAPY
4.3.1. Key market trends
4.3.2. Growth factors and opportunities
4.3.3. Market size and forecast
CHAPTER 5 GLOBAL POMPE DISEASE MARKET, BY MOLECULE TYPE
5.1. OVERVIEW
5.1.1. Market size and forecast
5.2. BIOLOGICS
5.2.1. Key market trends
5.2.2. Growth factors and opportunities
5.2.3. Market size and forecast
5.3. SMALL MOLECULES
5.3.1. Key market trends
5.3.2. Growth factors and opportunities
5.3.3. Market size and forecast
CHAPTER 6 GLOBAL POMPE DISEASE MARKET, BY ROUTE OF ADMINISTRATION
6.1. OVERVIEW
6.1.1. Market size and forecast
6.2. ORAL
6.2.1. Key market trends
6.2.2. Growth factors and opportunities
6.2.3. Market size and forecast
6.3. PARENTERAL
6.3.1. Key market trends
6.3.2. Growth factors and opportunities
6.3.3. Market size and forecast
CHAPTER 7 GLOBAL POMPE DISEASE MARKET, BY DOSAGE FORMS
7.1. OVERVIEW
7.1.1. Market size and forecast
7.2. SOLID
7.2.1. Key market trends
7.2.2. Growth factors and opportunities
7.2.3. Market size and forecast
7.3. LIQUID
7.3.1. Key market trends
7.3.2. Growth factors and opportunities
7.3.3. Market size and forecast
CHAPTER 8 GLOBAL POMPE DISEASE MARKET, BY REGION
8.1. OVERVIEW
8.2. NORTH AMERICA
8.2.1. Key market trends
8.2.2. Key growth factors and opportunities
8.2.3. Market size and forecast
8.2.3.1. U.S. market size and forecast
8.2.3.2. Canada market size and forecast
8.2.3.3. Mexico market size and forecast
8.2.4. North America Pompe Disease Market by Therapy Type
8.2.5. North America Pompe Disease Market by Molecule Type
8.2.6. North America Pompe Disease Market by Route of Administration
8.2.7. North America Pompe Disease Market by Dosage Forms
8.3. EUROPE
8.3.1. Key market trends
8.3.2. Key growth factors and opportunities
8.3.3. Market size and forecast
8.3.3.1. Germany market size and forecast
8.3.3.2. France market size and forecast
8.3.3.3. UK market size and forecast
8.3.3.4. Italy market size and forecast
8.3.3.8. Spain market size and forecast
8.3.3.6. Rest of Europe market size and forecast
8.3.4. Europe Pompe Disease Market by Therapy Type
8.3.5. Europe Pompe Disease Market by Molecule Type
8.3.6. Europe Pompe Disease Market by Route of Administration
8.3.7. Europe Pompe Disease Market by Dosage Forms
8.4. ASIA-PACIFIC
8.4.1. Key market trends
8.4.2. Key growth factors and opportunities
8.4.3. Market size and forecast
8.4.3.1. Japan market size and forecast
8.4.3.2. China market size and forecast
8.4.3.3. Australia market size and forecast
8.4.3.4. India market size and forecast
8.4.3.8. Rest of Asia-Pacific market size and forecast
8.4.4. Asia-Pacific Pompe Disease Market by Therapy Type
8.4.5. Asia-Pacific Pompe Disease Market by Molecule Type
8.4.6. Asia-Pacific Pompe Disease Market by Route of Administration
8.4.7. Asia-Pacific Pompe Disease Market by Dosage Forms
8.8. LAMEA
8.8.1. Key market trends
8.8.2. Key growth factors and opportunities
8.8.3. Market size and forecast
8.8.3.1. Brazil market size and forecast
8.8.3.2. Saudi Arabia market size and forecast
8.8.3.3. Argentina market size and forecast
8.8.3.4. South Africa market size and forecast
8.8.3.8. Rest of LAMEA market size and forecast
8.8.4. LAMEA Pompe Disease Market by Therapy Type
8.8.5. LAMEA Pompe Disease Market by Molecule Type
8.8.6. LAMEA Pompe Disease Market by Route of Administration
8.8.7. LAMEA Pompe Disease Market by Dosage Forms
CHAPTER 9 COMPANY PROFILES
9.1. AMICUS THERAPEUTICS
9.1.1. Operating business segments
9.1.2. Business performance
9.1.3. Key strategic moves and developments
9.2. BIOMARIN PHARMACEUTICAL
9.2.1. Operating business segments
9.2.2. Business performance
9.2.3. Key strategic moves and developments
9.3. GENZYME
9.3.1. Operating business segments
9.3.2. Business performance
9.3.3. Key strategic moves and developments
9.4. AUDENTES THERAPEUTICS
9.4.1. Operating business segments
9.4.2. Business performance
9.4.3. Key strategic moves and developments
9.8. EPIVAX
9.8.1. Operating business segments
9.8.2. Business performance
9.8.3. Key strategic moves and developments
9.6. OXYRANE
9.6.1. Operating business segments
9.6.2. Business performance
9.6.3. Key strategic moves and developments
9.7. SANGAMO BIOSCIENCES
9.7.1. Operating business segments
9.7.2. Business performance
9.7.3. Key strategic moves and developments
9.8. VALERION THERAPEUTICS
9.8.1. Operating business segments
9.8.2. Business performance
9.8.3. Key strategic moves and developments
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