Global Gaucher Disease Drugs Market


  • Type: Rare Disease Area
  • Published : March 2021
  • Region: Worldwide
  • Country: Other
  • Report ID : 122

  • Format: PPT, PDF

Global Gaucher Disease Drugs Market – Size, Share, Trends, Growth And Forecast (2019-2028)


Report Overview

The Global Gaucher disease drugs market size stood at USD 2.5 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Gaucher disease is an inherited disorder resulting build-up of certain fatty substance in certain organ, mainly in liver, bone marrow and spleen. It is the most common lysosomal storage disorders. Due to the low level of glucocerebrosidase an enzyme that breaks down the fatty chemical in the body known as glucocerebroside. Gaucher cells are normal scavenger cells known as macrophages that become full of unprocessed glucocerebroside. These unprocessed Gaucher cells accumulate primarily in the sleep, liver and bone marrow and causes serious orang dysfunction and inflammation. Each pregnant female has a 1 in 4 chance that the baby will be born with the disease. People with Gaucher disease carrier status do not have signs or symptoms. Symptoms of Gaucher disease include skeletal abnormalities, blood disorders and abdominal complaints

Market Drivers                                                 

The market is likely to exhibit modest growth during the forecast period. Rising incidence of the disease and increasing efforts for the identification of more GD patients are among the primary growth stimulants for the market

Lack of drugs in the late-stage pipeline and the rising cost of treatment are limiting the market from realizing its utmost potential. There remains a high unmet need for the development of improved treatments that combat neuronopathic complications at reduced costs and convenient administration schedules. Nevertheless, increasing investments in research and development activities related to rare diseases are projected to shape the future of the market

1. Global Gaucher’s disease Drugs Market Overview….…………………..…………….
A. Market Size
2. Market Growth Drivers……………………………………………………………………….
A. Rise in Global Prevalence of Gaucher’s disease
B. Growing R&D Investments and Reimbursement Policies
3. Major Types of Gaucher’s disease….……………………………….…………………….
A. Type 1
B. Type 2
C. Type 3
4. Gaucher’s disease Drugs Market Segmentation….……………………….…….……..
A. Drug Class
I. Enzyme Replacement Therapy
II. Substrate Replacement Therapy
III. Others
B. Geography
I. North America Market Analysis
II. Europe Market Analysis
III. Asia-Pacific Market Analysis
IV. Middle East and Africa Market Analysis
C. Distribution Channel
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
IV. Others
5. Gaucher’s disease Drugs Market Share…………..…………………………..…………
A. Market Analysis, Insights and Forecast – By Revenue
6. Competitive Landscape…………………………….……………………………………….
A. Major Players
B. Products in Pipeline
7. Key Company Profiles…………….………………………………………..……………….
A. Amicus Therapeutics Inc. Product & Services, Strategies & Financials
B. Sanofi Company Product & Services, Strategies & Financials
C. Shire PLC Company, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape…………………….……..……….…
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth……………………………………………………………
A. New Trends and Development of Gaucher’s disease Drugs Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Drug Class:

Enzyme replacement therapy (ERT) dominated the Gaucher disease drugs market in 2017. ERT will remain the more preferred therapy through 2025, which can be backed by strong commercial performances of Cerezyme, Vpriv, and Elelyso

However, ERT is expected to lose shares over the coming years owing to the high cost of therapy and inconvenient dosing schedule. Additionally, ERT is commonly used for type 1 and type 3 to address issues unrelated to CNS as replacement enzymes cannot penetrate blood brain barrier. The treatment landscape is witnessing a gradual shift towards substrate replacement therapy (SRT), led by Cerdelga and Zavesca

By Distribution Channel:

Based on the distribution channel, the market has been segregated into hospitals, retail, online and other pharmacies

By Region:

The U.S. commanded the largest share among the seven major markets for Gaucher disease treatment in 2017. The U.S. is anticipated to retain its position until 2025. Rising awareness of treatments, increasing access to treatment, and growing patient pool are among the key factors driving the regional market

EU5 countries such as Germany, France, and Italy are the key markets for Cerezyme. Japan is anticipated to exhibit the fastest growth due to rising disposable income, increasing disease awareness, and high unmet clinical needs of patients

Major Players

Some of the key players operating in the market are Sanofi Genzyme, Shire, Pfizer, and Johnson & Johnson. Sanofi Genzyme dominated the market with a nearly 65.0% share in 2017. The company will continue to hold prominence in the global arena over the coming years, due to strong sales of Cerezyme and Cerdelga


Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision making for business stakeholders. Our focused approach to develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

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Stage I: Market Data Collection

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Market Trends: We generally look at macro parameter and micro indicators. The macro parameters includes changes in government policies, demand and supply of the market, government intervention programs and major market share. The micro indicators GDP growth, market size, market volume etc. We also understand nuances specific to each country like US, Canada, India, Germany, etc. and have worked across 60+ countries and hence no only understand global trends, but how these differ by country, how payment models, market structure, cultural parameters, etc. differ in each country.

Market Sizing and Analysis: Our expert data analytics team has created various market forecast models by employing the top-down approach i.e. start with large overall market and segment different areas and bottom-up approach i.e. start with population and epidemiology and roll up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape and policy and regulatory scenario and the quantitative analysis covers different market estimates and forecasts.

Data Triangulation & Validation:

Data triangulation of various sources and results of the research are carried out by benchmarking with reliable sources such as industry statistics, statistical databases and the company level averages etc.

We make sure to finalize the numbers in alignment to the market research. Firstly, our internal experts ensure thorough validation and checking to ensure the accurate and precise analysis and then validation is also done using multiple data analysis model.  Two level validation is done and entails finalization of report scope and to way of representation pattern.

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analysed and synthesized. A second series of interviews are done if necessary to check and validate. The future opportunities are analysed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results are then, interpreted by considering following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
  • Analyzing current needs and determining penetration to estimate market

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Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

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The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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