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Fabry's Disease Pipeline Analysis 2019 (H1)

  • ID: 4801718
  • Report
  • 105 pages
  • Knowledge Sourcing Intelligence LLP
until Dec 31st 2019
1 of 4


  • Alexion
  • AMO Pharma
  • Greenovation Biotech GmbH
  • Pharming Group NV
  • Resverlogix Corp.
  • MORE
Fabry’s Disease Pipeline Analysis report covers more than 15S drugs currently in different phases of development. Fabry’s disease is a rare genetic disorder caused mostly in males than females due to the deficiency of the alpha-galactosidase A (a-Gal A) enzyme which causes a build-up of a type of fat, globotriaosylceramide (Gb3, or GL-3) in the body. It is also categorized as a type of lysosomal storage disorder. The disease affects skin, eyes, gastrointestinal system, kidney, heart, brain and nervous system among other body parts.

The report provides Fabry’s Disease treatment drugs by company, phases of development including products in early discovery stage and NDA filing, molecule type, route of administration and region. The report will help to evaluate the collaboration, in-licensing, and out-licensing opportunities, formulating business development strategies and tracking the activities of the key market players. Epidemiology, major drivers, restraints, and opportunities have been covered to provide an exhaustive picture of the market. The analysis presents in-depth information regarding the development, trends, and industry policies and regulations implemented to offer stakeholders a better understanding of the key factors affecting the overall market environment.

Various databases (for patents and Clinical Trials), studies and data published by industry associations, analyst reports, investor presentations, press releases and journals among others have been taken into consideration while conducting the secondary research.

Market intelligence is presented in the form of analysis, charts, and graphics to help the clients in gaining faster and efficient understanding of the market.

Major industry players profiled as part of the report are Biosidus, Sanofi, Amicus Therapeutics, Inc., Protalix and Neuraltus Pharmaceuticals, Inc.among others.

  • By Company
  • By Phase
  • By Molecule Type
  • By Region
  • By Route of Administration
Note: Product cover images may vary from those shown
2 of 4


  • Alexion
  • AMO Pharma
  • Greenovation Biotech GmbH
  • Pharming Group NV
  • Resverlogix Corp.
  • MORE
1. Introduction
1.1. Research Methodology
1.2. Research Scope
1.2.1. Analysis by Company
1.2.2. Analysis by Phase
1.2.3. By Molecule Type
1.2.4. By region

2. Disease Overview
2.1.1. Introduction
2.1.2. Classification
2.1.3. Symptoms
2.1.4. Causes
2.1.5. Diagnoses
2.1.6. Treatment
2.1.7. Epidemiology

3. Executive Summary

4. Market Dynamics
4.1. Drivers
4.2. Restraints
4.3. Opportunities

5. Pipeline Analysis/Outlook
5.1. Analysis by Company
5.2. Analysis by Phase
5.2.1. Phase of Development Introduction Drug Profiling
  • Drug Name
  • Generic Name
  • Synonyms
  • Company
  • Collaborator
  • Route of administration
  • Target
  • Mechanism of Action
  • Technology
  • Molecule type
  • CAS Number
  • Weight
  • Chemical Formula
  • IUPAC name
  • ATC code Strategic Developments Clinical trials Clinical trial results Patents Technology
5.2.2. Comparative Analysis for Trials by Phase (Pie, Bar graph)
5.3. By Molecule type
5.4. Analysis by Region

6. Company profiling
6.1. Protalix
6.1.1. Introduction
6.1.2. Financials
6.1.3. Products and Services
6.1.4. SWOT
6.1.5. Recent Development
6.2. Amicus Therapeutics, Inc.
6.2.1. Introduction
6.2.2. Financials
6.2.3. Products and Services
6.2.4. SWOT
6.2.5. Recent Development
6.3. Resverlogix Corp.
6.3.1. Introduction
6.3.2. Financials
6.3.3. Products and Services
6.3.4. SWOT
6.3.5. Recent Development
6.4. Idorsia Pharmaceuticals Ltd
6.4.1. Introduction
6.4.2. Financials
6.4.3. Products and Services
6.4.4. SWOT
6.4.5. Recent Development
6.5. AVROBIO, Inc
6.5.1. Introduction
6.5.2. Financials
6.5.3. Products and Services
6.5.4. SWOT
6.5.5. Recent Development
6.6. Greenovation Biotech GmbH
6.6.1. Introduction
6.6.2. Financials
6.6.3. Products and Services
6.6.4. SWOT
6.6.5. Recent Development
6.7. JCR Pharmaceuticals Co., Ltd.
6.7.1. Introduction
6.7.2. Financials
6.7.3. Products and Services
6.7.4. SWOT
6.7.5. Recent Development
6.8. Pharming Group NV
6.8.1. Introduction
6.8.2. Financials
6.8.3. Products and Services
6.8.4. SWOT
6.8.5. Recent Development
6.9. Sangamo Therapeutics, Inc.
6.9.1. Introduction
6.9.2. Financials
6.9.3. Products and Services
6.9.4. SWOT
6.9.5. Recent Development
6.10. Sanofi
6.10.1. Introduction
6.10.2. Financials
6.10.3. Products and Services
6.10.4. SWOT
6.10.5. Recent Development
6.11.1. Introduction
6.11.2. Financials
6.11.3. Products and Services
6.11.4. SWOT
6.11.5. Recent Development
6.12.1. Introduction
6.12.2. Financials
6.12.3. Products and Services
6.12.4. SWOT
6.12.5. Recent Development
6.13. Neuraltus Pharmaceuticals, Inc.
6.13.1. Introduction
6.13.2. Financials
6.13.3. Products and Services
6.13.4. SWOT
6.13.5. Recent Development
6.14. Alexion
6.14.1. Introduction
6.14.2. Financials
6.14.3. Products and Services
6.14.4. SWOT
6.14.5. Recent Development
6.15. Sigilon Therapeutics, Inc.
6.15.1. Introduction
6.15.2. Financials
6.15.3. Products and Services
6.15.4. SWOT
6.15.5. Recent Development
6.16. AMO Pharma
6.16.1. Introduction
6.16.2. Financials
6.16.3. Products and Services
6.16.4. SWOT
6.16.5. Recent Development
6.17. Oxyrane
6.17.1. Introduction
6.17.2. Financials
6.17.3. Products and Services
6.17.4. SWOT
6.17.5. Recent Development
Note: Product cover images may vary from those shown
3 of 4


4 of 4
  • Protalix
  • Amicus Therapeutics, Inc.
  • Resverlogix Corp.
  • Idorsia Pharmaceuticals Ltd
  • AVROBIO, Inc
  • Greenovation Biotech GmbH
  • JCR Pharmaceuticals Co., Ltd.
  • Pharming Group NV
  • Sangamo Therapeutics, Inc.
  • Sanofi
  • Neuraltus Pharmaceuticals, Inc.
  • Alexion
  • Sigilon Therapeutics, Inc.
  • AMO Pharma
  • Oxyrane
Note: Product cover images may vary from those shown
5 of 4

The research study is based on the publisher's proprietary research model. The process involves sourcing of information from various industry experts, key professionals, decision-makers and various other stakeholders along with the validation of the responses received from the vast in-house database and premium paid databases. Publicly available sources were also taken into consideration for the final market estimates and forecast for the segments and sub-segments.

The first phase of the research process is the identification of key industry leaders, experts, decision-makers and C-level executives and then conducting interviews and surveys to gain vital insights about the market. Simultaneously, extensive secondary research is done, and key secondary data is collected as part of this process. This secondary data is collected from various databases, industry journals, whitepapers, company annual reports, research publications, and other paid data sources and relevant literature. Company profiles which include strategies, new product developments, recent investments, major products and services, financials and business plans are sourced from the annual reports, SEC filings, industry associations to preserve the quality of the information being displayed. The findings from both the primary and the secondary research process combined go under further processing for market breakdown and forecasting.

The last phase is providing intelligence in the form of presentation, charts, graphics and other different formats helping the clients in a faster and efficient understanding of the market. Under this phase complete market engineering is involved which includes analyzing the collected data from different sources and existing proprietary datasets while using various data triangulation methods for market breakdown and forecasting. The publisher has developed their market forecasting methodology that, in addition to the main market values, also takes into account other macroeconomic factors (global or local) that might have some bearing on the market under study. Taking all the necessary data, a robust forecast is developed using an algorithm that involves both static (multivariate) as well as dynamic (time-series) regression analysis. Special care is taken to make sure their algorithm passes the necessary statistical tests, and the mean prediction for future years obtained is reliable with a very high confidence level. Following data validation, the analysts begin to write the report presenting insights with the help of charts, graphs, and tables.