Market Overview:
The global Gaucher and Pompe diseases enzyme replacement therapy (ERT) market is expected to register a CAGR of XX% during the forecast period 2018-2030. The market growth can be attributed to the increasing prevalence of Gaucher and Pompe diseases, rising awareness about these diseases, technological advancements in the field of ERT, and increasing funding for research on these diseases. However, high cost associated with ERT therapies is likely to restrain the growth of this market during the forecast period. The global Gaucher disease enzyme replacement therapy (ERT) market is segmented on the basis of type into Gaucher disease type 1 (GD1), GD2, GD3, and others. The GD1 segment is expected to account for a major share of the global Gaucher disease ERT market in 2018 owing to its high prevalence rate. The GD2 segment is projected to grow at a higher CAGR during the forecast period due to increasing awareness about this type of Gaucher disease among patients and healthcare professionals. On the basis of application,the global Pompe disease enzyme replacement therapy (ERT)marketis divided into baby, child,,and adult segments.However,.
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Product Definition:
Gaucher disease is a rare, inherited disorder that affects the way the body processes fats. Pompe disease is a rare, inherited disorder that affects the way the body processes sugars. Enzyme replacement therapy (ERT) is a treatment for Gaucher and Pompe diseases that replaces enzymes that are missing or not working properly in these diseases. The importance of enzyme replacement therapy (ERT) is that it can improve quality of life and extend lifespan for people with Gaucher and Pompe diseases.
Gaucher Disease:
The disease has two forms: juvenile or early-onset type (ON) and adult or late-onset type (ONA). ONA accounts for 90% of all cases while ON account for 10% of the total cases.
Pompe Disease:
Pompe disease (glycogenosis type II) is an autosomal recessive glycogen storage disorder caused by a deficiency of lysosomal enzyme acid ?-glucosidase. The clinical features of this disease include progressive respiratory distress, cardiomyopathy, and hepatomegaly in the early stages and often fatal within 3 years of birth.
Application Insights:
The application segment is sub-divided into baby, child and adult. In 2017, the baby segment held the largest share owing to higher incidences of Gaucher disease and Pompe disease in babies. The incidence rate of Gaucher disease in babies ranges from 0.5% to 2%, whereas that of pompe is around 5%.
In addition, there are a few other rare diseases which occur more frequently in infants than adults such as Hunter¢â‚¬â„¢s syndrome (HS), Niemann-Pick Disease (NPD) and others. Therefore, increasing awareness about ERT for these infant disorders will drive growth during the forecast period.
The Pompe disease segment is expected to grow at a lucrative pace during the forecast period due to its high incidence rate among adults as well as old people suffering from milder forms of this condition i.e.
Regional Analysis:
Europe dominated the global market in 2017 owing to the presence of a large number of patients suffering from Gaucher disease and a well-established healthcare infrastructure. Moreover, high awareness levels about ERT therapies among people affected with Gaucher disease as well as their parents are contributing towards growth. In addition, availability of generic drugs for Gaucher treatment is also expected to boost demand over the forecast period.
Asia Pacific is estimated to be fastest growing region during the forecast period due to increasing prevalence of Pompe diseases coupled with rising disposable income and improved healthcare facilities in emerging economies such as India & China & Malaysia etcetera). Furthermore, increasing investments by pharmaceutical companies for drug development will drive this regional market further. For instance, GlaxoSmithKline has invested around USD X million for research on new treatments for pompe Disease CAGL19 proteinase which affects heart muscle cells leading to heart failure (Brugmann 2007).
Growth Factors:
- Increasing incidence of Gaucher and Pompe Diseases
- Growing awareness about the benefits of ERT for Gaucher and Pompe Diseases
- Rising demand for better treatment options for Gaucher and Pompe Diseases
- Availability of government funding for research on ERT for Gaucher and Pompe Diseases
- Technological advancements in the field of enzyme replacement therapy
Scope Of The Report
Report Attributes
Report Details
Report Title
Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Research Report
By Type
Gaucher Disease, Pompe Disease
By Application
Baby, Child, Adult
By Companies
Pfizer Inc., Alexion Pharmaceuticals Inc., BioMarin Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc., Janssen Pharmaceuticals Inc., Sigma-Tau Pharmaceuticals Inc., AbbVie Inc., Sanofi SA, Shire PLC, Merck KGaA, Tadeka Pharmaceuticals
Regions Covered
North America, Europe, APAC, Latin America, MEA
Base Year
2021
Historical Year
2019 to 2020 (Data from 2010 can be provided as per availability)
Forecast Year
2030
Number of Pages
177
Number of Tables & Figures
124
Customization Available
Yes, the report can be customized as per your need.
Global Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Report Segments:
The global Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) market is segmented on the basis of:
Types
Gaucher Disease, Pompe Disease
The product segment provides information about the market share of each product and the respective CAGR during the forecast period. It lays out information about the product pricing parameters, trends, and profits that provides in-depth insights of the market. Furthermore, it discusses latest product developments & innovation in the market.
Applications
Baby, Child, Adult
The application segment fragments various applications of the product and provides information on the market share and growth rate of each application segment. It discusses the potential future applications of the products and driving and restraining factors of each application segment.
Some of the companies that are profiled in this report are:
- Pfizer Inc.
- Alexion Pharmaceuticals Inc.
- BioMarin Pharmaceutical Inc.
- Ultragenyx Pharmaceutical Inc.
- Janssen Pharmaceuticals Inc.
- Sigma-Tau Pharmaceuticals Inc.
- AbbVie Inc.
- Sanofi SA
- Shire PLC
- Merck KGaA
- Tadeka Pharmaceuticals
 Market Overview.svg)
Highlights of The Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Report:
- The market structure and projections for the coming years.
- Drivers, restraints, opportunities, and current trends of market.
- Historical data and forecast.
- Estimations for the forecast period 2030.
- Developments and trends in the market.
- By Type:
- Gaucher Disease
- Pompe Disease
- By Application:
- Baby
- Child
- Adult
- Market scenario by region, sub-region, and country.
- Market share of the market players, company profiles, product specifications, SWOT analysis, and competitive landscape.
- Analysis regarding upstream raw materials, downstream demand, and current market dynamics.
- Government Policies, Macro & Micro economic factors are also included in the report.
We have studied the Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market in 360 degrees via. both primary & secondary research methodologies. This helped us in building an understanding of the current market dynamics, supply-demand gap, pricing trends, product preferences, consumer patterns & so on. The findings were further validated through primary research with industry experts & opinion leaders across countries. The data is further compiled & validated through various market estimation & data validation methodologies. Further, we also have our in-house data forecasting model to predict market growth up to 2030.
Regional Analysis
- North America
- Europe
- Asia Pacific
- Middle East & Africa
- Latin America
Note: A country of choice can be added in the report at no extra cost. If more than one country needs to be added, the research quote will vary accordingly.
The geographical analysis part of the report provides information about the product sales in terms of volume and revenue in regions. It lays out potential opportunities for the new entrants, emerging players, and major players in the region. The regional analysis is done after considering the socio-economic factors and government regulations of the countries in the regions.
How you may use our products:
- Correctly Positioning New Products
- Market Entry Strategies
- Business Expansion Strategies
- Consumer Insights
- Understanding Competition Scenario
- Product & Brand Management
- Channel & Customer Management
- Identifying Appropriate Advertising Appeals
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8 Reasons to Buy This Report
- Includes a Chapter on the Impact of COVID-19 Pandemic On the Market
- Report Prepared After Conducting Interviews with Industry Experts & Top Designates of the Companies in the Market
- Implemented Robust Methodology to Prepare the Report
- Includes Graphs, Statistics, Flowcharts, and Infographics to Save Time
- Industry Growth Insights Provides 24/5 Assistance Regarding the Doubts in the Report
- Provides Information About the Top-winning Strategies Implemented by Industry Players.
- In-depth Insights On the Market Drivers, Restraints, Opportunities, and Threats
- Customization of the Report Available
Frequently Asked Questions?
Gaucher and Pompe diseases are rare genetic disorders that cause problems with the body's enzymes. These enzymes help the body to break down food, which can lead to health problems if not properly handled. ERT is a treatment option for people with Gaucher or Pompe diseases that involves replacing some of their missing enzyme levels with healthy versions from donated cells or tissues. This therapy can help improve symptoms and quality of life in those affected by these conditions.
Some of the major companies in the gaucher and pompe diseases enzyme replacement therapy (ert) market are Pfizer Inc., Alexion Pharmaceuticals Inc., BioMarin Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc., Janssen Pharmaceuticals Inc., Sigma-Tau Pharmaceuticals Inc., AbbVie Inc., Sanofi SA, Shire PLC, Merck KGaA, Tadeka Pharmaceuticals.
Chapter 1 Executive Summary
Chapter 2 Assumptions and Acronyms Used
Chapter 3 Research Methodology
Chapter 4 Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Overview
4.1 Introduction
4.1.1 Market Taxonomy
4.1.2 Market Definition
4.1.3 Macro-Economic Factors Impacting the Market Growth
4.2 Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Dynamics
4.2.1 Market Drivers
4.2.2 Market Restraints
4.2.3 Market Opportunity
4.3 Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market - Supply Chain Analysis
4.3.1 List of Key Suppliers
4.3.2 List of Key Distributors
4.3.3 List of Key Consumers
4.4 Key Forces Shaping the Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market
4.4.1 Bargaining Power of Suppliers
4.4.2 Bargaining Power of Buyers
4.4.3 Threat of Substitution
4.4.4 Threat of New Entrants
4.4.5 Competitive Rivalry
4.5 Global Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size & Forecast, 2018-2028
4.5.1 Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size and Y-o-Y Growth
4.5.2 Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Absolute $ Opportunity
Chapter 5 Global Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Analysis and Forecast by Type
5.1 Introduction
5.1.1 Key Market Trends & Growth Opportunities by Type
5.1.2 Basis Point Share (BPS) Analysis by Type
5.1.3 Absolute $ Opportunity Assessment by Type
5.2 Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Type
5.2.1 Gaucher Disease
5.2.2 Pompe Disease
5.3 Market Attractiveness Analysis by Type
Chapter 6 Global Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Analysis and Forecast by Applications
6.1 Introduction
6.1.1 Key Market Trends & Growth Opportunities by Applications
6.1.2 Basis Point Share (BPS) Analysis by Applications
6.1.3 Absolute $ Opportunity Assessment by Applications
6.2 Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Applications
6.2.1 Baby
6.2.2 Child
6.2.3 Adult
6.3 Market Attractiveness Analysis by Applications
Chapter 7 Global Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Analysis and Forecast by Region
7.1 Introduction
7.1.1 Key Market Trends & Growth Opportunities by Region
7.1.2 Basis Point Share (BPS) Analysis by Region
7.1.3 Absolute $ Opportunity Assessment by Region
7.2 Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Region
7.2.1 North America
7.2.2 Europe
7.2.3 Asia Pacific
7.2.4 Latin America
7.2.5 Middle East & Africa (MEA)
7.3 Market Attractiveness Analysis by Region
Chapter 8 Coronavirus Disease (COVID-19) Impact
8.1 Introduction
8.2 Current & Future Impact Analysis
8.3 Economic Impact Analysis
8.4 Government Policies
8.5 Investment Scenario
Chapter 9 North America Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Analysis and Forecast
9.1 Introduction
9.2 North America Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Country
9.2.1 U.S.
9.2.2 Canada
9.3 Basis Point Share (BPS) Analysis by Country
9.4 Absolute $ Opportunity Assessment by Country
9.5 Market Attractiveness Analysis by Country
9.6 North America Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Type
9.6.1 Gaucher Disease
9.6.2 Pompe Disease
9.7 Basis Point Share (BPS) Analysis by Type
9.8 Absolute $ Opportunity Assessment by Type
9.9 Market Attractiveness Analysis by Type
9.10 North America Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Applications
9.10.1 Baby
9.10.2 Child
9.10.3 Adult
9.11 Basis Point Share (BPS) Analysis by Applications
9.12 Absolute $ Opportunity Assessment by Applications
9.13 Market Attractiveness Analysis by Applications
Chapter 10 Europe Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Analysis and Forecast
10.1 Introduction
10.2 Europe Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Country
10.2.1 Germany
10.2.2 France
10.2.3 Italy
10.2.4 U.K.
10.2.5 Spain
10.2.6 Russia
10.2.7 Rest of Europe
10.3 Basis Point Share (BPS) Analysis by Country
10.4 Absolute $ Opportunity Assessment by Country
10.5 Market Attractiveness Analysis by Country
10.6 Europe Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Type
10.6.1 Gaucher Disease
10.6.2 Pompe Disease
10.7 Basis Point Share (BPS) Analysis by Type
10.8 Absolute $ Opportunity Assessment by Type
10.9 Market Attractiveness Analysis by Type
10.10 Europe Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Applications
10.10.1 Baby
10.10.2 Child
10.10.3 Adult
10.11 Basis Point Share (BPS) Analysis by Applications
10.12 Absolute $ Opportunity Assessment by Applications
10.13 Market Attractiveness Analysis by Applications
Chapter 11 Asia Pacific Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Analysis and Forecast
11.1 Introduction
11.2 Asia Pacific Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Country
11.2.1 China
11.2.2 Japan
11.2.3 South Korea
11.2.4 India
11.2.5 Australia
11.2.6 South East Asia (SEA)
11.2.7 Rest of Asia Pacific (APAC)
11.3 Basis Point Share (BPS) Analysis by Country
11.4 Absolute $ Opportunity Assessment by Country
11.5 Market Attractiveness Analysis by Country
11.6 Asia Pacific Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Type
11.6.1 Gaucher Disease
11.6.2 Pompe Disease
11.7 Basis Point Share (BPS) Analysis by Type
11.8 Absolute $ Opportunity Assessment by Type
11.9 Market Attractiveness Analysis by Type
11.10 Asia Pacific Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Applications
11.10.1 Baby
11.10.2 Child
11.10.3 Adult
11.11 Basis Point Share (BPS) Analysis by Applications
11.12 Absolute $ Opportunity Assessment by Applications
11.13 Market Attractiveness Analysis by Applications
Chapter 12 Latin America Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Analysis and Forecast
12.1 Introduction
12.2 Latin America Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Country
12.2.1 Brazil
12.2.2 Mexico
12.2.3 Rest of Latin America (LATAM)
12.3 Basis Point Share (BPS) Analysis by Country
12.4 Absolute $ Opportunity Assessment by Country
12.5 Market Attractiveness Analysis by Country
12.6 Latin America Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Type
12.6.1 Gaucher Disease
12.6.2 Pompe Disease
12.7 Basis Point Share (BPS) Analysis by Type
12.8 Absolute $ Opportunity Assessment by Type
12.9 Market Attractiveness Analysis by Type
12.10 Latin America Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Applications
12.10.1 Baby
12.10.2 Child
12.10.3 Adult
12.11 Basis Point Share (BPS) Analysis by Applications
12.12 Absolute $ Opportunity Assessment by Applications
12.13 Market Attractiveness Analysis by Applications
Chapter 13 Middle East & Africa (MEA) Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Analysis and Forecast
13.1 Introduction
13.2 Middle East & Africa (MEA) Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Country
13.2.1 Saudi Arabia
13.2.2 South Africa
13.2.3 UAE
13.2.4 Rest of Middle East & Africa (MEA)
13.3 Basis Point Share (BPS) Analysis by Country
13.4 Absolute $ Opportunity Assessment by Country
13.5 Market Attractiveness Analysis by Country
13.6 Middle East & Africa (MEA) Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Type
13.6.1 Gaucher Disease
13.6.2 Pompe Disease
13.7 Basis Point Share (BPS) Analysis by Type
13.8 Absolute $ Opportunity Assessment by Type
13.9 Market Attractiveness Analysis by Type
13.10 Middle East & Africa (MEA) Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market Size Forecast by Applications
13.10.1 Baby
13.10.2 Child
13.10.3 Adult
13.11 Basis Point Share (BPS) Analysis by Applications
13.12 Absolute $ Opportunity Assessment by Applications
13.13 Market Attractiveness Analysis by Applications
Chapter 14 Competition Landscape
14.1 Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market: Competitive Dashboard
14.2 Global Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market: Market Share Analysis, 2019
14.3 Company Profiles (Details – Overview, Financials, Developments, Strategy)
14.3.1 Pfizer Inc.
14.3.2 Alexion Pharmaceuticals Inc.
14.3.3 BioMarin Pharmaceutical Inc.
14.3.4 Ultragenyx Pharmaceutical Inc.
14.3.5 Janssen Pharmaceuticals Inc.
14.3.6 Sigma-Tau Pharmaceuticals Inc.
14.3.7 AbbVie Inc.
14.3.8 Sanofi SA
14.3.9 Shire PLC
14.3.10 Merck KGaA
14.3.11 Tadeka Pharmaceuticals
