Global Fabry Disease Drug Market

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  • Type: Rare Disease Area
  • Published : March 2021
  • Region: Worldwide
  • Country: Other
  • Report ID : 121

  • Format: PPT, PDF

Global Fabry Disease Drug Market – Size, Share, Impact, Growth, Trends, and Forecasts, North America, Asia-Pacific, US (2019 – 2028)

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Report Overview

The Global Fabry disease drugs market size stood at USD 2.9 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Fabry disease is a rare X-linked lysosomal storage disorder with a deficiency of alpha-galactosidase enzyme resulting in progressive organ dysfunction. This disease is caused by an abnormal build-up of a specific fatty matter called globotriaosylceramide in multiple tissues of the body including eyes, skin, kidney, gastrointestinal system, brain, heart, and central nervous system

Market Drivers                                                 

Growing demand for messenger RNA as a treatment modality for Fabry disease is boosting the market growth. The rising adoption of the treatment is mostly witnessed in the countries that have higher ratio of patients diagnosed with Fabry disease. For instance, according to the estimates provided by Census Bureau’s population, in the U.S. around 3,800 males are suffering from Fabry disease. Hence, the adoption of these novel treatments to mitigate or remove the disease is witnessing a significant market growth over the forecast period However, lack of awareness about advancements with respect to treatments for Fabry disease and limitations in adopting new technologies in certain countries owing to inadequate financial support from the respective government is hampering the adoption of the novel treatment and may hinder the market in the upcoming years

1. Global Fabry disease Drugs Market Overview….……………
A. Market Size
2. Market Growth Drivers………………………………………
A. Rise in Global Prevalence of Fabry disease
B. Growing R&D Investments and Reimbursement Policies
3. Major Types of Treatment for Fabry disease….…………
A. Enzyme Replacement Therapy,
B. Chaperone Treatment,
C. Substrate Reduction Therapy,
D. Others
4. Fabry disease Drugs Market Segmentation….………………
A. Drug class
I. Velaglucerase Alfa,
II. Taliglucerase Alfa,
III. Agalsidase beta,
IV. Laronidase,
V. Imiglucerase
VI. 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. Fabry 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 Fabry disease Drugs Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Drug Class:

Based on treatment type, the global Fabry disease treatment market has been categorized into enzyme replacement therapy (ERT), chaperone treatment, substrate reduction therapy (SRT), and others. The Standard of care for disease management is enzyme replacement therapy. Although, both the ERTs, Sanofi’s Fabrazyme and Shire’s Replagal, have been approved in Europe, only Fabrazyme has been approved in the U.S. The focus of current clinical trials is to improve safety and efficacy profile of ERTs and introduction of novel oral therapies that can eliminate the need for intravenous infusions. Recently Amicus Therapeutics’ Galafold has been approved as the first oral chaperone therapy for the treatment of adult patients in the U.S., Canada, EU, Japan, Australia, Israel, and South Korea

By Distribution Channel:

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

By Region:

North America was the most prominent regional market in 2019, followed by Europe. Higher adoption of novel therapies, better healthcare facilities, and favourable reimbursement policies are playing a vital role in the growth of the market in the region

Coverage of expensive medications such as Fabrazyme by health insurance programs and favourable governmental healthcare policies are also prompting drug companies to increase R&D investments in the field of rare diseases

Asia Pacific presents immense growth opportunities for pharmaceutical companies due to increasing healthcare expenditure and improving infrastructure. The region is likely to experience the fastest growth during the forecast period, followed by Latin America, primarily due to large populations in emerging countries

Major Players

Some of the key players operating in the market are Sanofi S.A.; Shire Plc.; Amicus Therapeutics Inc.; ISU Abxis Co Ltd.; JCR Pharmaceuticals Co Ltd.; Protalix Biotherapeutics Inc.; Idorsia Pharmaceuticals Ltd.; Avrobio Inc.; Greenovation Biotech GmbH; Moderna Therapeutics Inc.; and Green Cross Pharma Pte Ltd. The players are engaged in strategic planning, development of novel therapies, and deals to accomplish widespread growth and obtain the leading position in the market

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”.

Our analysis methodology involves three critical stages:

Stage I: Market Data Collection

Primary Interviews: We have developed a network of experts, freelancers and researchers across countries through which we engage with local experts to gather key data points and assumptions around each market. We also engage regularly with some of the best market research agencies such as Atheneum, GuidePoint, GLG, etc. to conduct surveys and interviews, and build intelligence. We have language translators as a part of our team, who between them can cover 30+ languages allowing us to extract better local insights.

Secondary Data Collection: We have developed strong expertise and experience in secondary data collection methods for developing unique data sets and research material. We gather data from multiple reliable sources to maintain the high level of accuracy and consistency. The market data is analysed and forecasted using appropriate statistical and coherent models. The report offers an overall analysis of the market size, growth, market share as well as segment-level analysis of the specific market. Our report includes precise, to the point information related to the overall market, competition, growth drivers, challenges, regulatory updates, and competition.

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Stage II: Market Data Analysis and Statistical Model

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.

Presentation & Reporting: The market research report is presented in different forms such as charts by using a scientific approach for easy understanding. Historic, current and future analysis is provided for each market in terms of both value and volume. The size of the market is interpreted in the US Dollar value and the respective unit, based on the product, for volume consumptions.
The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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