Fabry Disease – Pipeline Analysis 2019

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Global Fabry Disease – Pipeline Analysis 2019

Pages:

76

Table:

35

Figure:

17

Report ID:

UMHE19153

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Report Description
Table of content
Research Methodology

Report Description

Fabry Disease (also known as Anderson Fabry Disease) is a progressive X linked inherited genetic disorder of glycosphingolipid metabolism due to deficient or absent lysosomal α-galactosidase A activity. It is a devastating inborn error of metabolism with, particularly in the early stages, being played by cellular dysfunction and microvascular pathology being induced by lysosomal glycosphingolipid deposition. The absence or deficient activity of lysosomal exoglycohydrolase α-galactosidase A results in the progressive accumulation of globotriaosylceramide and related glycosphingolipids within lysosomes which are ubiquitous subcellular organelles.

The first clinical symptoms interfering with the child’s well-being and performance arise in childhood, typically between the ages of 3 and 10 years, and generally a few years later in girls than in boys. With age, progressive damage to vital organ systems develops in both genders leading to organ failure. End-stage renal disease and life-threatening cardiovascular or cerebrovascular complications limit life-expectancy. The clinical signs are multisystemic, heterogeneous, and progressive.

Split of Therapeutic Products

The biochemical diagnosis of Fabry Disease is established by measuring α-gal A activity in plasma or leukocytes taken from peripheral blood, cultured fibroblasts or using the samples extracted from the filter paper blood spots. The diagnosis of FD can arise from careful clinical and instrumental investigations, together with family history data and accurate interpretation of genetic and enzymatic analyses. Identification of a hemizygous GLA pathogenic variant by molecular genetic testing confirms the diagnosis in a male proband.

The therapeutic pipeline of Fabry Disease consists of approximately 9+ products in different stages of development. Currently, 2+ drugs are in Phase III development and major drugs are in pre-clinical stage.

Top Company Analysed

Some of the key players include Amicus Therapeutics; Evotec; Freeline; Greenovation Biotech; Idorsia Pharmaceuticals; Moderna; Pharming; Protalix Biotherapeutics; Resverlogix Corp; Sangamo Therapeutics and Sanofi Genzyme.

Scope of the study:

  • Provides an overview of therapeutic pipeline activity for Fabry Disease across the complete product development cycle including all clinical and non-clinical stages
  • The report comprises of detailed profiles of Fabry Disease therapeutic products with key coverage of developmental activities including licensing & collaboration deals, patents issued, designations, technologies and chemical information
  • Therapeutic assessment of the active pipeline products by stage, product type, molecule type, and route of administration
  • Detailed profiles of the dormant products have been included in the report

Reasons to buy:

  • The Fabry Disease pipeline presents the detailed profile of drugs. The analysis offered in the report is a combination of deep dive secondary research and input from Key Opinion Leader of the industry
  • The report presents a quick review of the current scenario regarding the drug development of the indication at one glance
  • The report covers in-depth analysis of prominent industry peers with a primary focus on company consolidation, designation, technology, agreements and patents regarding the therapy
  • Detailed examination on diagnosis, treatment and guidelines prevailing in the industry
  • Examination of industry attractiveness with the help of launch timelines
  • The study comprehensively covers the market across drugs in different phases of development
  • Extensive domain knowledge on therapy areas support the client in decision-making process regarding their therapeutic portfolio by identifying the reason behind the inactive or discontinued products

Customization Options:

The Fabry Disease pipeline analysis report can be customized to the country level or any other competitive segment. Besides this, UMI understands that you may have your own business need, hence we also provide fully customized solutions to clients.

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  1.1Objective of the Study
  1.2Secondary Research
  1.3Scope of the report:

 

  2.1Introduction 
  2.2Etiology  
  2.3Classification 
  2.4Signs and Symptoms
  2.5Pathogenesis 
   2.5.1Metabolic Defect
   2.5.2The Molecular Basis
   2.5.3Genotype/Phenotype Correlation
  2.6Diagnosis  
   2.6.1Biochemical Diagnosis:
    2.6.1.1Measurement of a-Gal Activity in Cultivated Fibroblasts, Plasma, or Leukocytes
    2.6.1.2Measurement of α-Gal A Activity in Blood Spot Filter Paper
    2.6.1.3GL-3 Levels
   2.6.2Molecular Diagnosis
   2.6.3Molecular Analysis
  2.7Treatment 
   2.7.1Clinical management of pediatric patients
   2.7.1Management of Fabry related pain in paediatric patients
    2.7.1.1Trigger management
    2.7.1.2Symptomatic therapy
     2.7.1.2.1Management of neuropathic pain
     2.7.1.2.2Management of psychological disturbances
   2.7.2Treatment Algorithm for the management of neuropathic pain in paediatric Fabry Disease patients
    2.7.2.1Enzyme replacement therapy
    2.7.2.2Recommendations for ERT initiation
   2.7.3Management in adults
    2.7.3.1Available Enzyme Replacement therapies
    2.7.3.2Initiation of enzyme replacement therapy
    2.7.3.3Expert panel recommendations for initiation of enzyme replacement therapy in adult patients with Fabry Disease
    2.7.3.4Adjunctive therapies
    2.7.3.5Non-ERT approaches for patients with specific mutations
   2.7.4Monitoring of adult patients with Fabry Disease

 

 3.1Pipeline Therapeutics at a Glance

 

 4.1Number of Products under Fabry Diseases
  5.1Comparative Analysis
  5.2Lucerastat: Idorsia Pharmaceuticals
   5.2.1Product Description
   5.2.2Research and Development
    5.2.2.1Clinical Studies
     5.2.2.1.1Phase III
     5.2.2.1.2Phase I
     5.2.2.1.3Results of Analysis
     5.2.2.1.4Results of Analysis II
   5.2.3Product Development Activities
    5.2.3.1Designation
    5.2.3.2Spin Off
  5.3Pegunigalsidase alfa: Protalix Biotherapeutics
   5.3.1Product Description
   5.3.2Research and Development
    5.3.2.1Clinical Studies
     5.3.2.1.1Phase III
     5.3.2.1.2Results of Analysis
     5.3.2.1.3Results of Analysis II
   5.3.3Product Development Activities
    5.3.3.1Designation
    5.3.3.2Agreement
    5.3.3.3Patent
    5.3.3.4Technology
  6.1Comparative Analysis
  6.2Venglustat: Sanofi Genzyme
   6.2.1Product Description
   6.2.2Research and Development
    6.2.2.1Clinical Studies
     6.2.2.1.1Phase II
   6.2.3Product Development Activities
    6.2.3.1Designation

 

  7.1Comparative Analysis
  7.2Apabetalone: Resverlogix Corp
   7.2.1Product Description
   7.2.2Research and Development
    7.2.2.1Clinical Studies
     7.2.2.1.1Phase II
  7.3Moss-aGal: Greenovation Biotech
   7.3.1Product Description
   7.3.2Research and Development
    7.3.2.1Clinical Studies
     7.3.2.1.1Phase I
     7.3.2.1.2Results of Analysis
   7.3.3Product Development Activities
    7.3.3.1Technology

 

  8.1Comparative Analysis
  8.2Fabry Gene Therapy: Amicus Therapeutics
   8.2.1Product Description
   8.2.2Product Development Activities
    8.2.2.1Agreement
  8.3FTL190: Freeline
   8.3.1Product Description
   8.3.2Research and Development
    8.3.2.1Pre-clinical Studies
   8.3.3Product Development Activities
    8.3.3.1Financing
  8.4mRNA-3630: Moderna
   8.4.1Product Description
  8.5PGN005 (α-galactosidase): Pharming
   8.5.1Product Description
  8.6ST-920: Sangamo Therapeutics
   8.6.1Product Description
   8.6.2Research and Development
    8.6.2.1Pre-clinical Studies
   8.6.3Product Development Activities
    8.6.3.1IND Acceptance

 

  9.1Assessment by Stage and Product Type
  9.2Assessment by Route of Administration
  9.3Assessment by Stage and Route of Administration
  9.4Assessment by Molecule Type
  9.5Assessment by Stage and Molecule Type

 

 10.1Clinical Products Approval Timelines

 

  12.1Comparative Analysis
  12.2Small Molecule Inhibitor: Evotec
   12.2.1Product Description
   12.2.2Product Development Activities
    12.2.2.1Collaboration

 

Research Methodology

Overview of pipeline development activities for Fabry Disease Pipeline Analysis of therapeutic drugs is not limited to drug description and development activities but also focuses on clinical and non-clinical results. It also includes designations, company consolidations & licensing deals, grants, technology and patent details. Therapeutic segmentation of products for Fabry Disease is done based on phase of development of the drugs. The report comprises of comparative pipeline therapeutics assessment along with detailed drug profile of pipeline products by development stage, therapy type, molecule type, and administration route across this indication. The report also consists of the launch timelines forecasted for the upcoming pipeline therapies. Additionally, Analyst Insight have also been focused upon to give a summary regarding the current market scenario. The detailed profiles of the dormant products have elucidated in the complete report with the relevant reasons for their dormancy.

Secondary Research

Detail secondary study was conducted to obtain the Fabry Disease Pipeline Analysis through company internal sources such as annual reports, performance presentations, press releases etc., and external sources including trade journals, news & articles, government publications, competitor publications, sector reports, regulatory bodies publications, safety standard organizations, third-party database and other creditable publications.

The secondary research on internal and external sources is being carried out to source qualitative and quantitative information relating to each market.

  • Company websites, annual reports, quarter reports, financial reports, broker reports, investor presentations and SEC filings
  • Industry trade journals and other literature
  • National government documents, statistical databases and market reports
  • News articles, press releases and web-casts specific to the companies operating in the market
  • Several databases for patents and clinical trials

Main objective of the Fabry Disease – Pipeline Analysis 2019

The current pipeline trends of the Fabry Disease are pinpointed in the study. Investors can gain strategic insights to base their discretion for investments from the qualitative and quantitative analysis performed in the study. Current and future pipeline trends would determine the overall attractiveness of the market, providing a platform for the industrial participant to exploit the untapped market to benefit as first mover advantage. The quantitative objectives for the report include:

  • An overview of therapeutic pipeline activity for Fabry Disease across the complete product development cycle including all clinical and non-clinical stages
  • Analyse the detailed profiles of Fabry Disease therapeutic products with key coverage of developmental activities including licensing & collaboration deals, patents issued, designations, technologies and chemical properties information
  • Therapeutic assessment of the active pipeline products by stage, product type, molecule type, and route of administration