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Gaucher Disease Market
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The market for Gaucher disease was estimated at USD 980.0 million in 2021, and it is anticipated to grow at a CAGR of 8.5% to reach around USD 1,800.0 million in 2029. The rising prevalence of Gaucher disease, increasing awareness about Gaucher disease, and upsurge in the number of approved therapies for the treatment of Gaucher disease. Even it is the most prevalent lysosomal storage disorder, Gaucher disease is a rare disorder with an average prevalence of 1 in 100,000 individuals. However, some ethnic races such as Ashkenazi Jews are more inclined to this disorder. Ashkenazi Jews have an average disease prevalence of 1 in 100,000 individuals. However, the high cost related with the treatment of Gaucher’s disease may restrain the growth of this market during the forecast period.
| Attribute | Key Insights |
| Estimated Market Value (2021) | 980 USD Million |
| Forecasted Market Value (2028) | 1,800 USD Million |
| CAGR % 2023-2028 | 8.5%` |
| Leading Region | North America |
| Major Drivers | Increasing prevalence of Gaucher disease backed by the growing rate of inheritance of the disease, accessory initiatives by governments round the world
Gaucher disease market will be additional ascribed to the rising investments and expenditure for the R&D activities to incessantly refer additional possible solutions for the treatment. |
Disease Type Insights
The type 1 disease segment is expected to carry the largest market size by growing at a major CAGR over the forecast amount. The common symptoms of type 1 disease include embody spleen and liver enlargement, bone issues, and fatigue. the expansion of the phase will be related to the rising variety of individuals plagued by type-1 disease across the globe. As sourced from the National Gaucher Foundation, close to 95% of total cases area unit stricken by sort one Gaucher malady in western countries.
Some of the drugs received approvals recently for the Gaucher disease treatment. For instance, U.S. Food and Drug Administration (FDA) has approved multiple drugs in past several years for the treatment of disease. In 2012, Elelyso and in 2014, Cerdelga was permitted by FDA for type 1 Gaucher disease. In 2010, FDA approved Vpriv, an enzyme replacement therapy (ERT), for type 1 Gaucher disease. These drugs offer range of options based on their requests, in turn is anticipated to fuel the growth of the market.
Type 2 and 3 are rare types of this disease. Both these conditions are neuronopathic, causing CNS complications throughout the disease course. Infants with type 2 GD rarely survive over two years, due to lack of treatment and severity of disease progression.
Other rare types of the disease include perinatal lethal form and cardiac form. The perinatal lethal form is the most adverse type of the disease, leading to life-threatening complications in infants or fetuses. Cardiac form affects the heart, generally, causing calcification of heart valves.
Therapy Type Insights
The enzyme replacement therapy market is anticipated to grow at a substantial rate during the forecast period due to increasing cases of Gaucher disease worldwide. Around 10-20 cases per million are reported yearly in the U.S., however approximately 100 cases per million are reported in Europe and Asia.
However, ERT is predicted to lose shares in the coming years due to the high cost of therapy and troublesome dosing schedule. Furthermore, ERT is regularly used for type 1 and type 3 to address problems unrelated to CNS as replacement enzymes cannot enter blood brain barrier. The treatment landscape is observing a gradual shift towards substrate replacement therapy (SRT), controlled by Cerdelga and Zavesca.
Region Insights
North America is predicted to dominant the Gaucher disease market due to growing investigations for the development of new medication. In 2016, the human from National Institutes of Health researchers collaborated with National Institute of medical specialty Disorders and Stroke (NINDS) and therefore the National Center for Advancing travel Sciences (NCATS). Researchers known a brand new molecule that shows promising approach for treatment of the rare Gaucher disease.
Europe is predicted to be the second largest marketplace for Gaucher malady treatment thanks to the initiative taken by European organizations to unfold awareness concerning Gaucher malady among the population. for example, in 2014, European Gaucher Alliance launched Gaucher Awareness Day reaching to unfold awareness concerning this rare malady among the European population.
Key Companies Insights
Key players are aiming on their technological partnerships, collaborations, and merger & acquisitions strategies to attain a competitive edge and expand their product portfolio & business footprint. The ongoing Covid-19 pandemic hindered the healthcare systems across the globe and drove market players to innovate products for unexploited opportunities. Some of the key players operating in the global Gaucher disease market include:
- Genzyme Corporation (Sanofi)
- Pfizer, Inc.
- Shire Human Genetics Therapies, Inc.
- Actelion Pharmaceuticals Ltd. (Johnson & Johnson)
- AVROBIO, Inc.
- Bristol-Myers Squibb Company
- PerkinElmer Inc.
- Amicus Therapeutics, Inc.
- Moderna, Inc.
- Greenovation Biotech GmbH
- Biomarin
- Takeda Pharmaceutical Company Limited
- AstraZeneca PLC
- Eli Lilly and Company
- Other players
There are several drugs in pipeline of companies such as Lixte Biotechnology Holdings Inc., JCR Pharmaceuticals Co Ltd., Pharming Group NV, and Orphazyme ApS
Some of the Recent Developments:
- In 2017, Janssen acquired Actelion to add complementary therapeutics and promising late-stage pipeline candidates to its portfolio.
- In 2015, Protalix sold worldwide rights of Elelyso to Pfizer.
- In 2012, Protalix entered into a licensing deal with Pfizer in 2009 for clinical development and commercialization of Elelyso.
Segments
By Disease Type
- Type 1 Gaucher disease
- Type 2 Gaucher disease
- Type 3 Gaucher disease
By Therapy Type
- Enzyme Replacement Therapy (ERT)
- Substrate Reduction Therapy (SRT)
By End Users
- Hospitals
- Specialty Clinics
- Homecare
- Others
By Distribution Channel
- Hospitals
- Retail Pharmacies
- Online Pharmacies
By Geography
- North America
- S.
- Canada
- Mexico
- Europe
- K.
- Germany
- France
- Italy
- Spain
- Russia
- Asia-Pacific
- Japan
- China
- India
- Australia
- South Korea
- ASEAN
- Latin America
- Brazil
- Argentina
- Colombia
- MEA
- South Africa
- Saudi Arabia
- UAE
- Egypt
ToC
- Global Gaucher Disease Introduction and Market Overview
- Objectives of the Study
- Global Gaucher Disease Scope and Market Estimation
- Global Gaucher Disease Overall Market Size, Revenue (US$ Mn), Market CAGR (%), Market forecast (2023 – 2032)
- Global Gaucher Disease Revenue Share (%) and Growth Rate (Y-o-Y) from 2019 – 2032
- Market Segmentation
- Disease Type of Global Gaucher Disease
- Therapy Type of Global Gaucher Disease
- End User of Global Gaucher Disease
- Region of Global Gaucher Disease
- Executive Summary
- Market Factor Analysis
- Global Gaucher Disease Industry Trends under COVID-19 Outbreak
- Global COVID-19 Status Overview
- Influence of COVID-19 Outbreak on Global Gaucher Disease Industry Development
- Market Dynamics, By Region
- Drivers
- Limitations
- Opportunities
- Impact Analysis of Drivers and Restraints
- Ecosystem / Value Chain Analysis
- Raw Materials / Components Suppliers
- Manufacturers / Vendors
- Distributors
- Buyers / End-users
- Forward Integration & Backward Integration of Key Stakeholders
- Global Gaucher Disease – Pricing Trends Analysis & Average Selling Prices (ASPs)
- End-User / Customer Preferences & Consumer Surveys
- Technology Roadmap & Key Innovations /Developments
- Key Mandates & Regulatory Scenario Analysis
- Patents Landscape
- Manufacturing Cost Structure Share Analysis
- Key Mergers & Acquisitions, Expansions, JVs, Funding / VCs, etc.
- Porter’s Five Forces Analysis
- Bargaining Power of Suppliers
- Bargaining Power of Buyers
- Threat of Substitutes
- Threat of New Entrants
- Competitive Rivalry
- PEST Analysis
- Political Factors
- Economic Factors
- Social Factors
- Technological Factors
- SWOT Analysis
- Russia-Ukraine War Impacts Analysis
- ANSOFF Matrix
- Market Penetration Strategy
- Product Development Strategy
- Market Development Strategy
- Diversification Strategy
- Inflation Impacts Analysis
- Supply Side Impacts
- Demand Side Impacts
- Outlook in 2023 & Beyond
- Market Investment Feasibility Analysis
- Opportunity Map Analysis
- Market Investment Opportunity Analysis (Top Investment Pockets), By Segments & By Regions
- Global Gaucher Disease Industry Trends under COVID-19 Outbreak
- Global Gaucher Disease Estimates & Historical Trend Analysis (2019 – 2022)
- Global Gaucher Disease Estimates & Forecast Trend Analysis, by Disease Type
- Global Gaucher Disease Revenue (US$ Mn) Estimates and Forecasts, by Disease Type, 2019 to 2032
- Type 1 Gaucher disease
- Type 2 Gaucher disease
- Global Gaucher Disease Revenue (US$ Mn) Estimates and Forecasts, by Disease Type, 2019 to 2032
- Global Gaucher Disease Estimates & Forecast Trend Analysis, by Therapy Type
- Global Gaucher Disease Revenue (US$ Mn) Estimates and Forecasts, Therapy Type 2019 to 2032
- Enzyme Replacement Therapy (ERT)
- Substrate Reduction Therapy (SRT)
- Global Gaucher Disease Revenue (US$ Mn) Estimates and Forecasts, Therapy Type 2019 to 2032
- Global Gaucher Disease Estimates & Forecast Trend Analysis, by End Users
- Global Gaucher Disease Revenue (US$ Mn) Estimates and Forecasts, End Users 2019 to 2032
- Hospitals
- Specialty Clinics
- Homecare
- Others
- Global Gaucher Disease Revenue (US$ Mn) Estimates and Forecasts, End Users 2019 to 2032
- Global Gaucher Disease Estimates & Forecast Trend Analysis, by Region
- Global Antibody-Drug Conjugates Market Revenue (US$ Mn) Estimates and Forecasts, by Region, 2019 to 2032
- North America
- Europe
- Asia Pacific
- Middle East & Africa
- South America
- Global Antibody-Drug Conjugates Market Revenue (US$ Mn) Estimates and Forecasts, by Region, 2019 to 2032
- North America Gaucher Disease : Estimates & Forecast Trend Analysis
- North America Antibody-Drug Conjugates Market Assessments & Key Findings
- Gaucher Disease Introduction
- Gaucher Disease Size Estimates and Forecast (US$ Million) (2019 – 2031)
- By Therapy Type
- By Product Type
- By End User
- By Country
- The U.S.
- Canada
- Mexico
- North America Antibody-Drug Conjugates Market Assessments & Key Findings
- Europe Gaucher Disease : Estimates & Forecast Trend Analysis
- Europe Gaucher Disease Assessments & Key Findings
- Gaucher Disease Introduction
- Gaucher Disease Size Estimates and Forecast (US$ Million) (2019 – 2031)
- By Therapy Type
- By Product Type
- By End User
- By Country
- Germany
- K.
- France
- Italy
- Spain
- Russia
- Rest of Europe
- Europe Gaucher Disease Assessments & Key Findings
- Asia Pacific Gaucher Disease : Estimates & Forecast Trend Analysis
- Asia Pacific Market Assessments & Key Findings
- Gaucher Disease Introduction
- Gaucher Disease Size Estimates and Forecast (US$ Million) (2019 – 2031)
- By Therapy Type
- By Product Type
- By End User
- By Country
- China
- Japan
- India
- Australia
- South Korea
- ASEAN
- Rest of Asia Pacific
- Asia Pacific Market Assessments & Key Findings
- Middle East & Africa Gaucher Disease : Estimates & Forecast Trend Analysis
- Middle East & Africa Market Assessments & Key Findings
- Gaucher Disease Introduction
- Gaucher Disease Size Estimates and Forecast (US$ Million) (2019 – 2031)
- By Therapy Type
- By Product Type
- By End User
- By Country
- A.E.
- Saudi Arabia
- Egypt
- South Africa
- Rest of Middle East & Africa
- Middle East & Africa Market Assessments & Key Findings
- South America Gaucher Disease : Estimates & Forecast Trend Analysis
- South America Market Assessments & Key Findings
- Gaucher Disease Introduction
- Gaucher Disease Size Estimates and Forecast (US$ Million) (2019 – 2031)
- By Therapy Type
- By Product Type
- By End User
- By Country
- Brazil
- Argentina
- Colombia
- Rest of South America
- South America Market Assessments & Key Findings
- Competition Landscape
- Gaucher Disease Competition Index, By Leading Players
- Gaucher Disease Competition White Space Analysis, By Application / End-user
- Gaucher Disease Competition Heat Map Analysis, By Products / Services / Solutions
- Gaucher Disease Competition Regional Intensity Map Analysis, By Geographies Served
- Gaucher Disease Concentration & Company Market Shares (%) Analysis, 2022
- Gaucher Disease Competition Quadrant Analysis, By Leading Players / Innovators / Emerging Players / New Entrants
- Company Profiles
- Genzyme Corporation (Sanofi)
- Company Overview & Key Stats
- Financial Performance & KPIs
- Product / Service / Solutions Portfolio & Applications / End-uses
- Business Strategy & Recent Developments
- Genzyme Corporation (Sanofi)
* Similar details would be provided for all the players mentioned below
- Pfizer, Inc.
- Shire Human Genetics Therapies, Inc.
- Actelion Pharmaceuticals Ltd. (Johnson & Johnson)
- AVROBIO, Inc.
- Bristol-Myers Squibb Company
- PerkinElmer Inc.
- Amicus Therapeutics, Inc.
- Moderna, Inc.
- Greenovation Biotech GmbH
- Biomarin
- Takeda Pharmaceutical Company Limited
- AstraZeneca PLC
- Eli Lilly and Company
- Others
- Research Methodology
- External Sources / Databases
- Internal Proprietary Database
- Primary Research
- Secondary Research
- Assumptions
- Limitations
- Report FAQs
- Research Findings & Conclusion
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