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Global Gaucher’s Disease Treatment Market Share, Size, Growth, Forecast: By Indication Type: Type 1 Gaucher’s Disease, Type 2 Gaucher’s Disease, Type 3 Gaucher’s Disease; By Therapy Method: Enzyme Replacement Therapy, Substrate Reduction Therapy, Others; By Treatment Channel; Regional Analysis; Supplier Landscape; 2023-2031
Gaucher’s Disease Treatment Market Outlook
The global Gaucher’s disease treatment market size attained a value of about USD 1.55 billion in 2022. The market is expected to rise at a CAGR of 3.6% during the forecast period of 2023-2031 to attain a value of more than USD 2.1 billion by 2031.
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Gaucher’s Disease Insights: Pathophysiology, Diagnosis, and Treatment
Gaucher’s disease is an autosomal recessive disorder, mostly occurring due to genetic causes. When both the parents are the carriers of Gaucher’s mutated gene, it mostly gets inherited by the offspring .
Gaucher’s disease is due to the accumulation of certain fatty deposits in specific organs. The accumulation causes the enlargement of these organs and thus, affecting their normal function. Gaucher’s disease mainly affects the liver and spleen.
The fatty substances concentrate in bone tissue, which can eventually lead to bone weakening and bone fractures. The body’s clotting ability is also affected due to the build-up of fatty deposits in the bone marrow.
Majorly, there are three types of Gaucher’s disease, Type I, Type II, and Type III. The signs and symptoms vary according to the disease’s type. In some cases, patients experience minimal or very few symptoms. The most common symptoms are irregular abdominal pain, painful fractures, bone weakening, skeletal abnormalities, bruising, and blood disorders.
The people living in the Central European and Eastern Jewish ancestry are more susceptible to Gaucher’s disease. The diagnosis of Gaucher’s disease is made using blood tests, genetic analysis, and imaging tests. Magnetic resonance imaging and dual energy-ray absorptiometry are also used to assess the disease’s severity.
The therapeutic strategy depends on the type of patient, type of disease, and age of the patient. Oral use of medications, like MIglustat and Eliglustat, is the initial approach of healthcare providers. Enzyme replacement therapy, spleen removal, and bone marrow transplant are some surgical interventions for managing Gaucher’s disease.
According to the Gaucher’s disease treatment market research report, the market can be categorised into the following segments:
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Market Breakup by Indication Type
- Type 1 Gaucher’s Disease
- Type 2 Gaucher’s Disease
- Type 3 Gaucher’s Disease
Market Breakup by Therapy Method
- Enzyme Replacement Therapy
- Substrate Reduction Therapy
- Splenectomy
- Orthopaedic Surgery
- Gene Therapy
- Others
Market Breakup by Treatment Channel
- Public
- Private
Market Breakup by Region
- North America
- United States of America
- Canada
- Europe
- United Kingdom
- Germany
- France
- Italy
- Others
- Asia Pacific
- China
- Japan
- India
- ASEAN
- Australia
- Others
- Latin America
- Brazil
- Argentina
- Mexico
- Others
- Middle East and Africa
- Saudi Arabia
- United Arab Emirates
- Nigeria
- South Africa
- Others
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Gaucher’s Disease Treatment Market Scenario
The increase in the prevalence of the lysosomal disease is the key factor driving the growth of the market. Lysosomal disorders are inherited disorders caused by the abnormal accumulation of toxic chemicals in the body. Treatment strategies are required to eradicate the buildup of toxic substances, hence responsible for driving the market growth.
Gaucher’s disease treatment market is stimulated by the increased expenditure in the healthcare sector. The availability of raised funds by the government for research and development activities is also facilitating the market expansion. Gaucher’s syndrome susceptibility increase with age, and hence, the rising geriatric population can aid the growth of the market further.
Additional factors, such as the increasing adoption rate of early diagnosis and management and surging initiatives and policies by the government, are also anticipated to boost the market growth during the forecast period.
North America and Asia Pacific to be the Leading Regional Markets
The North American regional market is projected to witness a significant share in Gaucher’s disease treatment market owing to the increased prevalence, rising R&D activities, and technological advancements.
The Asia Pacific region is one of the fastest growing regions among the regional markets. This growth is assisted by the continuous development in the healthcare sector and the rising prevalence of Gaucher’s disease.
Therapeutic Landscape
The treatment for complete eradication of Gaucher’s disease is yet to be discovered. Current treatment interventions aim to alleviate the disease symptoms and improve the quality of living standards. The primary aim is to eliminate the accumulation of fat deposits and alter the defective enzyme responsible for causing Gaucher’s disease.
Currently Available Treatment Strategies: Oral Medications, Enzyme Replacement Therapy, and Surgical Interventions
The treatment for Gaucher’s disease depends on the type and severity of the disease. Daily monitoring needs to be done by the healthcare provider to assess disease progression and complications.
The initial treatment is carried out by incorporating medications that can be administered orally. The drugs act on the fatty deposits and hinder the production of fatty substances, thus helping manage Gaucher’s disease. Zavesca and Cerdelga are approved medicines for the treatment of this disease.
Enzyme replacement therapy is the most efficient therapy with plausible therapeutic applications. In this therapy, the defective enzyme is replaced with novel artificial enzymes. The intravenous route of administration is preferably used for taking these specific enzymes. This therapy needs to be performed after every two weeks.
The severe symptoms call for surgical or invasive therapeutic interventions. The two main approaches in the surgical process are spleen removal and bone marrow transplant. The replacement of damaged blood-forming cells with healthy cells is carried out in bone marrow transplants. Spleen removal is generally done when no other treatment options are left.
The novel treatments under the pipeline will help improve healthcare and the global economy. These new treatment strategies will majorly drive the growth of the global Gaucher’s disease treatment market.
Clinical Trial Landscape
Extensive research and development in the healthcare sector have benefitted in better understanding and awareness of the disease. The clinical trials will help discover novel therapeutic strategies for Gaucher’s disease.
Emerging Technologies: Promising Novel Therapies
The trials incorporating the innovations in Enzyme Replacement Therapy (ERT) are in the pipeline. Successful results will lead to the discovery of advanced ERT therapy that will be more efficient and economical.
Inculcation of advanced technologies in the diagnostic methods is anticipated to help in the early detection of disease. The novel neurodegeneration biomarkers are under evaluation, which seem promising in monitoring fatty deposits in the brain cells.
New oral medications are also in the developmental process and under clinical studies. Freeline Therapeutics, a leading key player, discovered a novel gene therapy product, FLT201, under Phase I/II clinical trial. The application of cell and gene therapy in Gaucher’s disease treatment is projected to have better prospects in the forecast period.
Implementing new technologies in healthcare has already provided enormous benefits to human health. Researchers’ tremendous efforts have anticipated a rapid increase in the approvals for intelligent therapies. Several ongoing clinical trials are emerging as a driving factor for market growth and development.
Competitive Landscape
The report gives an in-depth analysis of the key players involved in Gaucher’s disease treatment market, sponsors manufacturing the therapies, and putting them through trials to get FDA approvals. The companies included in the market are as follows:
- Genzyme (Sanofi S.A.)
- Amicus Therapeutics, Inc.
- Novartis AG
- Shire plc
- Actelion Pharmaceuticals Ltd.
- Protalix Bio Therapeutics Inc.
- Johnson & Johnson
- Takeda Pharmaceutical Company Limited
- Abbott Laboratories
- Aptalis Pharma Inc.
- GSK plc
Key Highlights of the Report
| REPORT FEATURES | DETAILS |
|---|---|
| Base Year | 2022 |
| Historical Period | 2016-2022 |
| Forecast Period | 2023-2031 |
| Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
|
| Breakup by Indication Type |
|
| Breakup by Therapy Method |
|
| Breakup by Treatment Channel |
|
| Breakup by Region |
|
| Market Dynamics |
|
| Supplier Landscape |
|
| Companies Covered |
|
*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR report.
1 Preface
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage – Key Segmentation and Scope
2 Research Methodology
3 Executive Summary
4 Gaucher’s Disease Overview
4.1 Guidelines and Stages
4.2 Pathophysiology
4.3 Screening and Diagnosis
4.4 Treatment Pathway
5 Patient Profile
5.1 Patient Profile Overview
5.2 Patient Psychology and Emotional Impact Factors
5.3 Risk Assessment and Treatment Success Rate
6 Current Scenario Evaluation and Regulatory Framework
6.1 Emerging Therapies and Clinical Trials
6.2 Patent Landscape
6.2.1 Patent Overview
6.2.1.1 Patent Status and Expiry
6.2.1.2 Timelines from Drug Development to Commercial Launch
6.2.1.3 New Drug Application
6.2.1.3.1 Documentation and Approval Process
6.3 Cost of Treatment
6.4 Regulatory Framework
6.4.1 Regulatory Overview
6.4.1.1 US FDA
6.4.1.2 EU EMA
6.4.1.3 INDIA CDSCO
6.4.1.4 JAPAN PMDA
6.4.1.5 Others
7 Challenges and Unmet Needs
7.1 Treatment Pathway Challenges
7.2 Compliance and Drop-Out Analysis
7.3 Awareness and Prevention Gaps
8 Global Gaucher’s Disease Treatment Market
8.1 Global Gaucher’s Disease Treatment Market Overview
8.2 Global Gaucher’s Disease Treatment Market Analysis
8.2.1 Market Overview
8.2.1.1 Global Gaucher’s Disease Treatment Market Historical Value (2016-2022)
8.2.1.2 Global Gaucher’s Disease Treatment Market Forecast Value (2023-2031)
8.3 Global Gaucher’s Disease Treatment Market by Indication Type
8.3.1 Market Overview
8.3.1.1 Type 1 Gaucher’s Disease
8.3.1.2 Type 2 Gaucher’s Disease
8.3.1.3 Type 3 Gaucher’s Disease
8.4 Global Gaucher’s Disease Treatment Market by Therapy Method
8.4.1 Market Overview
8.4.1.1 Enzyme Replacement Therapy
8.4.1.2 Substrate Reduction Therapy
8.4.1.3 Splenectomy
8.4.1.4 Orthopedic Surgery
8.4.1.5 Gene Therapy
8.4.1.6 Others
8.5 Global Gaucher’s Disease Treatment Market by Treatment Channel
8.5.1 Market Overview
8.5.1.1 Public
8.5.1.2 Private
8.6 Global Gaucher’s Disease Treatment Market by Region
8.6.1 Market Overview
8.6.1.1 North America
8.6.1.2 Europe
8.6.1.3 Asia Pacific
8.6.1.4 Middle East and Africa
8.6.1.5 Latin America
9 North America Gaucher’s Disease Treatment Market
9.1 Market Share by Country
9.2 United States of America
9.2.1 Historical Trend (2016-2022)
9.2.2 Forecast Trend (2023-2031)
9.3 Canada
9.3.1 Historical Trend (2016-2022)
9.3.2 Forecast Trend (2023-2031)
10 Europe Gaucher’s Disease Treatment Market
10.1 Market Share by Country
10.2 United Kingdom
10.2.1 Historical Trend (2016-2022)
10.2.2 Forecast Trend (2023-2031)
10.3 Germany
10.3.1 Historical Trend (2016-2022)
10.3.2 Forecast Trend (2023-2031)
10.4 France
10.4.1 Historical Trend (2016-2022)
10.4.2 Forecast Trend (2023-2031)
10.5 Italy
10.5.1 Historical Trend (2016-2022)
10.5.2 Forecast Trend (2023-2031)
10.6 Others
11 Asia Pacific Gaucher’s Disease Treatment Market
11.1 Market Share by Country
11.2 China
11.2.1 Historical Trend (2016-2022)
11.2.2 Forecast Trend (2023-2031)
11.3 Japan
11.3.1 Historical Trend (2016-2022)
11.3.2 Forecast Trend (2023-2031)
11.4 India
11.4.1 Historical Trend (2016-2022)
11.4.2 Forecast Trend (2023-2031)
11.5 ASEAN
11.5.1 Historical Trend (2016-2022)
11.5.2 Forecast Trend (2023-2031)
11.6 Australia
11.6.1 Historical Trend (2016-2022)
11.6.2 Forecast Trend (2023-2031)
11.7 Others
12 Latin America Gaucher’s Disease Treatment Market
12.1 Market Share by Country
12.2 Brazil
12.2.1 Historical Trend (2016-2022)
12.2.2 Forecast Trend (2023-2031)
12.3 Argentina
12.3.1 Historical Trend (2016-2022)
12.3.2 Forecast Trend (2023-2031)
12.4 Mexico
12.4.1 Historical Trend (2016-2022)
12.4.2 Forecast Trend (2023-2031)
12.5 Others
13 Middle East and Africa Gaucher’s Disease Treatment Market
13.1 Market Share by Country
13.2 Saudi Arabia
13.2.1 Historical Trend (2016-2022)
13.2.2 Forecast Trend (2023-2031)
13.3 United Arab Emirates
13.3.1 Historical Trend (2016-2022)
13.3.2 Forecast Trend (2023-2031)
13.4 Nigeria
13.4.1 Historical Trend (2016-2022)
13.4.2 Forecast Trend (2023-2031)
13.5 South Africa
13.5.1 Historical Trend (2016-2022)
13.5.2 Forecast Trend (2023-2031)
13.6 Others
14 Global Gaucher’s Disease Treatment Market Dynamics
14.1 Market Drivers and Constraints
14.2 SWOT Analysis
14.3 Porter’s Five Forces Model
14.4 Key Demand Indicators
14.5 Key Price Indicators
14.6 Industry Events, Initiatives, and Trends
14.7 Value Chain Analysis
15 Supplier Landscape
15.1 Genzyme (Sanofi S.A.)
15.1.1 Company Overview
15.1.2 Product Portfolio
15.1.3 Demographic Reach and Achievements
15.1.4 Mergers and Acquisitions
15.1.5 Certifications
15.2 Amicus Therapeutics, Inc.
15.2.1 Company Overview
15.2.2 Product Portfolio
15.2.3 Demographic Reach and Achievements
15.2.4 Mergers and Acquisitions
15.2.5 Certifications
15.3 Novartis AG
15.3.1 Company Overview
15.3.2 Product Portfolio
15.3.3 Demographic Reach and Achievements
15.3.4 Mergers and Acquisitions
15.3.5 Certifications
15.4 Shire plc
15.4.1 Company Overview
15.4.2 Product Portfolio
15.4.3 Demographic Reach and Achievements
15.4.4 Mergers and Acquisitions
15.4.5 Certifications
15.5 Actelion Pharmaceuticals Ltd.
15.5.1 Company Overview
15.5.2 Product Portfolio
15.5.3 Demographic Reach and Achievements
15.5.4 Mergers and Acquisitions
15.5.5 Certifications
15.6 Protalix Bio Therapeutics Inc.
15.6.1 Company Overview
15.6.2 Product Portfolio
15.6.3 Demographic Reach and Achievements
15.6.4 Mergers and Acquisitions
15.6.5 Certifications
15.7 Johnson & Johnson
15.7.1 Company Overview
15.7.2 Product Portfolio
15.7.3 Demographic Reach and Achievements
15.7.4 Mergers and Acquisitions
15.7.5 Certifications
15.8 Takeda Pharmaceutical Company Limited
15.8.1 Company Overview
15.8.2 Product Portfolio
15.8.3 Demographic Reach and Achievements
15.8.4 Mergers and Acquisitions
15.8.5 Certifications
15.9 Abbott Laboratories
15.9.1 Company Overview
15.9.2 Product Portfolio
15.9.3 Demographic Reach and Achievements
15.9.4 Mergers and Acquisitions
15.9.5 Certifications
15.10 Aptalis Pharma Inc.
15.10.1 Company Overview
15.10.2 Product Portfolio
15.10.3 Demographic Reach and Achievements
15.10.4 Mergers and Acquisitions
15.10.5 Certifications
15.11 GSK plc
15.11.1 Company Overview
15.11.2 Product Portfolio
15.11.3 Demographic Reach and Achievements
15.11.4 Mergers and Acquisitions
15.11.5 Certifications
16 Global Gaucher’s Disease Treatment Market- Drug Distribution Model (Additional Insight)
16.1 Overview
16.2 Potential Distributors
16.3 Key Parameters for Distribution Partner Assessment
17 Payment Methods (Additional Insight)
17.1 Government Funded
17.2 Private Insurance
17.3 Out-of-Pocket
*Additional insights provided are customisable as per client requirements.
Key Questions Answered in the Report
The market attained a value of about USD 1.55 billion in 2022, driven by the increased prevalence of the disease.
The market is likely to grow at a CAGR of 3.6% during the forecast period of 2023-2031 to attain a value of USD 2.1 billion by 2031.
Based on the indication type, the market is segmented into Type 2, 3, and 3 Gaucher’s disease.
Based on the treatment channels, the Gaucher’s disease treatment market is categorised into public and private.
The different regions in the market are North America, Europe, Asia Pacific, Latin America, and Middle East and Africa. North America is anticipated to hold a significant share in the market during the forecast period.
There is no effective treatment for complete eradication but different therapeutic strategies help alleviate symptoms. Enzyme replacement therapy, oral medications, and surgical procedures have effectively treated Gaucher’s disease.
Increasing prevalence of lysosomal disorders, technological advancements, increased research and developmental activities, and better awareness are the driving factors for the market growth.
The key players in the market include Genzyme (Sanofi S.A.), Amicus Therapeutics, Inc., Novartis AG, Shire plc, Actelion Pharmaceuticals Ltd., Protalix Bio Therapeutics Inc., Johnson & Johnson, Takeda Pharmaceutical Company Limited, Abbott Laboratories, Aptalis Pharma Inc., and GSK plc, among others.
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