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Fabry disease also called as Anderson-Fabry disease and alpha-galactosidase-A deficiency is an X-linked, hereditary, lysosomal storage disease caused due to paucity of an enzyme α galactosidase A (an enzyme required to metabolize lipids, fat-like substances that include oils, waxes, and fatty acids). The mutated gene leads the formation of lipids to harmful levels in various part of body such as the autonomic nervous system, cardiovascular system, eyes, and kidneys. Some of the common symptoms of this multisystem disorder include chronic pain, acroparesthesia, gastrointestinal disturbances, characteristic skin lesions (angiokeratomata), progressive renal impairment, cardiomyopathy, and stroke.

Fabry Disease Treatment Market Drivers

In recent past, various regulatory bodies of key regions have approved novel medications for treatment of Fabry disease. Approval and launch of such novel medications in the region is expected to significantly support global Fabry disease treatment market growth over the forecast period.

For instance, in August 2018, Amicus Therapeutics, Inc., received the U.S. Food and Drug Administration (FDA) approval for its drug Galafold (migalastat). It is the first oral medication indicated for the treatment of adults with Fabry disease.

The presence of robust medication pipeline indicated for treatment of Fabry disease is also expected to be a major factor fueling growth of global Fabry disease treatment market over the forecast period. Table mentioned below represents list of some novel therapies in pipeline for Fabry disease treatment.


Furthermore, key players in the market are majorly investing in development of novel therapies for Fabry disease treatment, such strategic funding’s by various players is expected to drastically fuel global Fabry Disease treatment market growth over the forecast period. For instance, in February 2018, AvroBio Inc., closed US$ 60 million financing to support the phase 2 study of its novel gene therapy, AVR-RD-01 underdevelopment for the treatment of Fabry disease.

Fabry Disease Treatment Market Regional Analysis

Europe Fabry disease treatment market is expected to witness a drastic growth over the forecast period, owing to penetration of key players in key regions of Europe with their novel products for Fabry disease. For instance, Amicus Therapeutics Inc. launched Galafold (Migalastat) for treatment of Fabry disease in Italy (in March 2017) and in Spain (in January 2018)

In November 2018, JCR Pharmaceuticals Co., Ltd, a Japan-based company launched biosimilar for agalsidase beta for treatment of Fabry disease. Launch of biosimilars is expected to increase affordability of these therapies in key regions of Asia Pacific as biosimilars are quite cheaper than biologics, hence it is expected to boost Asia Pacific Fabry disease treatment market growth.

Fabry Disease Treatment Market Restraint

Fabry disease is a very rare disease affecting around 1 individual in 50,000 (according to the National Fabry Disease Foundation), the diagnosis of Fabry disease is confirmed by demonstrating an enzyme deficiency in males and by identifying the specific GLA gene mutation in males and females.

Hence, low diagnostic rate due to unavailability of such diagnostic procedures and trained professionals in emerging economies such as Africa, India, and China are expected to be major factor restraining global Fabry disease treatment market growth.

Fabry Disease Treatment Market Key Players

Key players operating in global Fabry disease treatment market include: Amicus Therapeutics Inc., Sanofi Genzyme (Sanofi S.A. company), Idorsia Pharmaceuticals Ltd., Protalix BioTherapeutics, Inc., AvroBio Inc., JCR Pharmaceuticals Co., Ltd., Resverlogix Corp, and Ozmosis Research Inc.

Fabry Disease Treatment Market Taxonomy:

The global Fabry disease treatment market is segmented on the basis of drugs, molecule type, route of administration, distribution channel, and region

By Drugs-

  • Approved Drugs
    • Agalsidase Beta (Fabrazyme/Replagal)
    • Migalastat (Galafold)
  • Pipeline Drugs

By Molecule Type-

  • Small Molecule
  • Large Molecule (Biologics)

By Route of Administration-

  • Oral
  • Intravenous

By Distribution Channel-

  • Hospitals Pharmacies
  • Retail Pharmacies
  • Online pharmacies

By Region-

  • North America
  • Latin America
  • Europe
  • Asia Pacific
  • Middle East
  • Africa

Research Methodology

Coherent Market Insights followsa comprehensive research methodology focused on providing the most precise market analysis. The company leverages a data triangulation model which helps company to gauge the market dynamics and provide accurate estimates. Key components of the research methodologies followed for all our market reports include:

  • Primary Research (Trade Surveys and Experts Interviews)
  • Desk Research
  • Proprietor Data Analytics Model

In addition to this, Coherent Market Insights has access to a wide range of the regional and global reputed paid data bases, which helps the company to figure out the regional and global market trends and dynamics. The company analyses the industry from the 360 Degree Perspective i.e. from the Supply Side and Demand Side which enables us to provide granular details of the entire ecosystem for each study. Finally, a Top-Down approach and Bottom-Up approach is followed to arrive at ultimate research findings.

Data Triangulation Methodology | Coherent Market Insights

Coherent Market Insights desk research is based on a principle set of research techniques:

  • National level desk research: It Includes research analysis of regional players, regional regulatory bodies, regional trade associations, and regional organization.
  • Multinational level desk research: The research team keeps a track of multinational players, global regulatory bodies, global trade associations, and global organization.

Coherent Market Insights has a large amount of in-house repository of industry database. This is leveraged as a burner for initiating a new research study. Key secondary sources include:

  • Governmental bodies, National and international social welfare institutions, and organizations creating economic policies among others.
  • Trade association, National and international media and trade press.
  • Company Annual reports, SEC filings, Corporate Presentations, press release, news, and specification sheet of manufacturers, system integrators, brick and mortar - distributors and retailers, and third party online commerce players.
  • Scientific journals, and other technical magazines and whitepapers.

Market Analysis | Coherent Market Insights

Preliminary Data Mining

The raw data is obtained through the secondary findings, in house repositories, and trade surveys. It is then filtered to ensure that the relevant information including industry dynamics, trends, and outlook is retained for further research process.

Data Standardization

Holistic approach is used to ensure that the granular and uncommon parameters are taken into consideration to ensure accurate results. The information from the paid databases are further combined to the raw data in order to standardize it.

Coherent Statistical model

We arrive at our final research findings through simulation models. Coherent Data Analytics Model is a statistical tool that helps company to forecast market estimates. Few of the parameters considered as a part of the statistical model include:

  • Micro-economic indicators
  • Macro-economic indicators
  • Environmental indicators
  • Socio-political indicators
  • Technology indicators

Data Processing

Once the findings are derived from the statistical model, large volume of data is process to confirm accurate research results. Data analytics and processing tools are adopted to process large chunk of collected informative data. In case, a client customizes the study during the process, the research finding till then are benchmarked, and the process for new research requirement is initiated again.

Data Validation

This is the most crucial stage of the research process. Primary Interviews are conducted to validate the data and analysis. This helps in achieving the following purposes:

  • It provides first-hand information on the market dynamics, outlook, and growth parameters.
  • Industry experts validates the estimates which helps the company to cement the on-going research study.
  • Primary research includes online surveys, face-to face interviews, and telephonic interviews.

The primary research is conducted with the ecosystem players including, but not limited to:

  • Raw Material Suppliers
  • Manufacturers
  • System Integrators
  • Distributors
  • End-users