About Gaucher Disease
Gaucher disease is a rare, inherited metabolic disorder caused by the deficiency of the enzyme glucocerebrosidase. This in turn causes the accumulation of certain detrimental lipids in the body, especially the glycolipid glucocerebroside. The lipids are accumulated mainly within the macrophages, blood cells, spleen, liver, and bone marrow. Approximately 34 genetic mutations have been identified that leads to Gaucher disease, out of which there are four most prominent mutations that lead to more than half of the Gaucher disease globally. Some people may have no symptoms of this disease, while some experience severe complications. Some of the clinical manifestations of this disease are hepatosplenomegaly, anemia, thrombocytopenia, and skeletal abnormalities.
TechNavio's analysts forecast the Global Gaucher Disease market to grow at a CAGR of 7.60 percent over the period 2014-2019.
Covered in this Report
This report covers the present scenario and the growth prospects of the Global Gaucher Disease market for the period 2015-2019. To calculate the market size, the report considers revenue from the sales of various drugs used in the therapy of Gaucher disease, which includes:
• Enzyme replacement therapy
• Substrate reduction therapy
Based on the type of molecule used for the treatment of Gaucher disease, the market is categorized into two segments:
• Biologics
• Small Molecules
Based on the route of administration adopted for the treatment of Gaucher disease, the market is categorized into two segments:
• Oral
• Parenteral
Based on the dosage forms of the drugs used to treat the disease, the Global Gaucher Disease market is categorized into two segments:
• Solid
• Liquid
TechNavio's report, Global Gaucher Disease Market 2015-2019, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the Americas, and the EMEA and APAC regions; it also covers the Global Gaucher Disease market landscape and its growth prospects in the coming years. The report also includes a discussion of the key vendors operating in this market.
Key Regions
• Americas
• APAC
• EMEA
Key Vendors
• Actelion Pharmaceuticals
• Genzyme
• Pfizer
• Shire
Other Prominent Vendors
• Amicus Therapeutics
• Belrose Pharma
• Bionaturis
• Biosidus
• ISU Abxis
• JCR Pharmaceuticals
• LIXTE Biotechnology
• Neuraltus Pharmaceuticals
• Protalix BioTherapeutics
• Teva Pharmaceuticals
• UCB
• Utrecht Holdings
• Zywie
Market Driver
• Special Provisions for Orphan Drugs
• For a full, detailed list, view our report
Market Challenge
• Limited Patient Population
• For a full, detailed list, view our report
Market Trend
• Regulatory Assistance in Emerging Nations
• For a full, detailed list, view our report
Key Questions Answered in this Report
• What will the market size be in 2019 and what will the growth rate be?
• What are the key market trends?
• What is driving this market?
• What are the challenges to market growth?
• Who are the key vendors in this market space?
• What are the market opportunities and threats faced by the key vendors?
• What are the strengths and weaknesses of the key vendors?
01. Executive Summary
02. List of Abbreviations
03. Scope of the Report
03.1 Market Overview
03.2 Product Offerings
04. Market Research Methodology
04.1 Market Research Process
04.2 Research Methodology
05. Introduction
06. Disease Overview
06.1 Understanding the Disease
06.1.1 Types of Gaucher Disease
06.1.2 Symptoms
06.1.3 Diagnosis
06.2 Etiology
06.3 Epidemiology
06.4 Clinical Manifestations
06.5 Management
06.5.1 Enzyme Replacement Therapy
06.5.2 Substrate Reduction Therapy
06.6 Economic Burden
07. Pipeline Analysis
08. Market Landscape
08.1 Market Overview
08.2 Market Size and Forecast
08.3 Five Forces Analysis
09. Market Segmentation by Type of Therapy
09.1 Enzyme Replacement Therapy
09.2 Substrate Reduction Therapy
10 Market Segmentation by Type of Molecule
10.1 Biologics
10.2 Small Molecules
11 Market Segmentation by Route of Administration
11.1 Oral
11.2 Parenteral
12 Market Segmentation by Dosage Form
12.1 Solid
12.2 Liquid
13 Geographical Segmentation
14 Buying Criteria
15 Market Growth Drivers
16 Drivers and their Impact
17 Market Challenges
18 Impact of Drivers and Challenges
19 Market Trends
20 Trends and their Impact
21 Vendor Landscape
21.1 Competitive Scenario
21.1.1 Key News
21.1.2 Mergers and Acquisitions
21.2 Market Share Analysis 2014
21.2.1 Genzyme
21.2.2 Shire
21.2.3 Actelion Pharmaceuticals
21.2.4 Pfizer
21.3 Other and Future Prominent Vendors
22 Key Vendor Analysis
22.1 Actelion Pharmaceuticals
22.1.1 Key Facts
22.1.2 Business Overview
22.1.3 Geographical Segmentation 2013
22.1.4 Business Strategy
22.1.5 Recent Developments
22.1.6 SWOT Analysis
22.2 Genzyme
22.2.1 Key Facts
22.2.2 Business Overview
22.2.3 Product Segmentation by Revenue 2013
22.2.4 Product Segmentation by Revenue 2012 and 2013
22.2.5 Geographical Segmentation
22.2.6 Business Strategy
22.2.7 Recent Developments
22.2.8 SWOT Analysis
22.3 Pfizer
22.3.1 Key Facts
22.3.2 Business Overview
22.3.3 Business Segmentation by Revenue 2013
22.3.4 Business Segmentation by Revenue 2012 and 2013
22.3.5 Geographical Segmentation by Revenue
22.3.6 Business Strategy
22.3.7 Key Developments
22.3.8 SWOT Analysis
22.4 Shire
22.4.1 Key Facts
22.4.2 Business Overview
22.4.3 Geographical Segmentation by Revenue 2013
22.4.4 Business Strategy
22.4.5 Recent Developments
22.4.6 SWOT Analysis
23 Other Reports in this Series
List of Exhibits:
Exhibit 1: Market Research Methodology
Exhibit 2: Snapshot of Gaucher Disease
Exhibit 3: Types of Gaucher Disease
Exhibit 4: Heredogram of Inheritance Pattern for Gaucher Disease
Exhibit 5: Clinical Manifestations of Gaucher Disease
Exhibit 6: Snapshot of Economic Burden of Gaucher Disease
Exhibit 7: Clinical Trials by Status (as of February 2015)
Exhibit 8: Clinical Trials by Phase (as of February 2015)
Exhibit 9: Snapshot of Global Gaucher Disease Market 2014
Exhibit 10: Global Gaucher Disease Market 2014-2019 (US$ million)
Exhibit 11: Drivers and Challenges of Global Gaucher Disease Market
Exhibit 12: Segmentation of Global Gaucher Disease Market by Type of Therapy
Exhibit 13: Segmentation of Global Gaucher Disease Market by Type of Therapy 2014
Exhibit 14: Segmentation of Global Gaucher Disease Market by Type of Molecule
Exhibit 15: Segmentation of Global Gaucher Disease Market by Type of Molecule 2014
Exhibit 16: Segmentation of Global Gaucher Disease Market by Route of Administration
Exhibit 17: Segmentation of Global Gaucher Disease Market by Route of Administration 2014
Exhibit 18: Segmentation of Global Gaucher Disease Market by Dosage Form
Exhibit 19: Segmentation of Global Gaucher Disease Market by Dosage Form 2014
Exhibit 20: Segmentation of Global Gaucher Disease Market by Geography 2014
Exhibit 21: Global Gaucher Disease Market Share Analysis 2014
Exhibit 22: Genzyme: Product Portfolio
Exhibit 23: Cerezyme: SWOT Analysis
Exhibit 24: Cerdelga: SWOT Analysis
Exhibit 25: Shire: Product Portfolio
Exhibit 26: Vpriv: SWOT Analysis
Exhibit 27: Actelion Pharmaceuticals: Product Portfolio
Exhibit 28: Zavesca: SWOT Analysis
Exhibit 29: Pfizer: Product Portfolio
Exhibit 30: Elelyso: SWOT Analysis
Exhibit 31: Actelion Pharmaceuticals: Geographical Segmentation by Revenue 2013
Exhibit 32: Genzyme: Product Segmentation by Revenue 2013
Exhibit 33: Genzyme: Product Segmentation by Revenue 2012 and 2013 (US$ million)
Exhibit 34: Genzyme: Geographical Segmentation
Exhibit 35: Pfizer: Business Segmentation by Revenue 2013
Exhibit 36: Pfizer: Business Segmentation by Revenue 2012 and 2013
Exhibit 37: Pfizer: Geographical Segmentation by Revenue 2013
Exhibit 38: Shire: Geographical Segmentation by Revenue 2013
