Fabry Disease - Pipeline Analysis 2019
- UNI4065853
- 76 Pages
- June 2019
- Healthcare
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.
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 report:
• 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.
1.1 Objective of the Study 8
1.2 Secondary Research 8
1.3 Scope of the report: 8
2. DISEASE OVERVIEW 9
2.1 Introduction 9
2.2 Etiology 10
2.3 Classification 11
2.4 Signs and Symptoms 12
2.5 Pathogenesis 14
2.5.1 Metabolic Defect 14
2.5.2 The Molecular Basis 15
2.5.3 Genotype/Phenotype Correlation 18
2.6 Diagnosis 19
2.6.1 Biochemical Diagnosis: 19
2.6.1.1 Measurement of a-Gal Activity in Cultivated Fibroblasts, Plasma, or Leukocytes 19
2.6.1.2 Measurement of α-Gal A Activity in Blood Spot Filter Paper 20
2.6.1.3 GL-3 Levels 20
2.6.2 Molecular Diagnosis 21
2.6.3 Molecular Analysis 21
2.7 Treatment 23
2.7.1 Clinical management of pediatric patients 24
2.7.1 Management of Fabry related pain in paediatric patients 24
2.7.1.1 Trigger management 24
2.7.1.2 Symptomatic therapy 25
2.7.1.2.1 Management of neuropathic pain 25
2.7.1.2.2 Management of psychological disturbances 26
2.7.2 Treatment Algorithm for the management of neuropathic pain in paediatric Fabry Disease patients 27
2.7.2.1 Enzyme replacement therapy 28
2.7.2.2 Recommendations for ERT initiation 29
2.7.3 Management in adults 30
2.7.3.1 Available Enzyme Replacement therapies 31
2.7.3.2 Initiation of enzyme replacement therapy 31
2.7.3.3 Expert panel recommendations for initiation of enzyme replacement therapy in adult patients with Fabry Disease 31
2.7.3.4 Adjunctive therapies 33
2.7.3.5 Non-ERT approaches for patients with specific mutations 34
2.7.4 Monitoring of adult patients with Fabry Disease 35
3. PIPELINE THERAPEUTICS AT A GLANCE 38
3.1 Pipeline Therapeutics at a Glance 38
4. COMPARATIVE ANALYSIS 39
4.1 Number of Products under Fabry Diseases 39
5. LATE PHASE PRODUCTS (PHASE III) 40
5.1 Comparative Analysis 40
5.2 Lucerastat: Idorsia Pharmaceuticals 40
5.2.1 Product Description 40
5.2.2 Research and Development 41
5.2.2.1 Clinical Studies 41
5.2.2.1.1 Phase III 41
5.2.2.1.2 Phase I 41
5.2.2.1.3 Results of Analysis 41
5.2.2.1.4 Results of Analysis II 42
5.2.3 Product Development Activities 42
5.2.3.1 Designation 42
5.2.3.2 Spin Off 43
5.3 Pegunigalsidase alfa: Protalix Biotherapeutics 44
5.3.1 Product Description 44
5.3.2 Research and Development 44
5.3.2.1 Clinical Studies 44
5.3.2.1.1 Phase III 44
5.3.2.1.2 Results of Analysis 45
5.3.2.1.3 Results of Analysis II 46
5.3.3 Product Development Activities 47
5.3.3.1 Designation 47
5.3.3.2 Agreement 47
5.3.3.3 Patent 48
5.3.3.4 Technology 48
6. MID PHASE PRODUCTS (PHASE II) 50
6.1 Comparative Analysis 50
6.2 Venglustat: Sanofi Genzyme 50
6.2.1 Product Description 50
6.2.2 Research and Development 51
6.2.2.1 Clinical Studies 51
6.2.2.1.1 Phase II 51
6.2.3 Product Development Activities 51
6.2.3.1 Designation 51
7. EARLY STAGE PRODUCTS (PHASE I) 53
7.1 Comparative Analysis 53
7.2 Apabetalone: Resverlogix Corp 53
7.2.1 Product Description 53
7.2.2 Research and Development 54
7.2.2.1 Clinical Studies 54
7.2.2.1.1 Phase II 54
7.3 Moss-aGal: Greenovation Biotech 55
7.3.1 Product Description 55
7.3.2 Research and Development 55
7.3.2.1 Clinical Studies 55
7.3.2.1.1 Phase I 55
7.3.2.1.2 Results of Analysis 56
7.3.3 Product Development Activities 56
7.3.3.1 Technology 56
8. PRE-CLINICAL STAGE PRODUCTS 58
8.1 Comparative Analysis 58
8.2 Fabry Gene Therapy: Amicus Therapeutics 58
8.2.1 Product Description 58
8.2.2 Product Development Activities 59
8.2.2.1 Agreement 59
8.3 FTL190: Freeline 60
8.3.1 Product Description 60
8.3.2 Research and Development 60
8.3.2.1 Pre-clinical Studies 60
8.3.3 Product Development Activities 61
8.3.3.1 Financing 61
8.4 mRNA-3630: Moderna 62
8.4.1 Product Description 62
8.5 PGN005 (α-galactosidase): Pharming 63
8.5.1 Product Description 63
8.6 ST-920: Sangamo Therapeutics 63
8.6.1 Product Description 63
8.6.2 Research and Development 64
8.6.2.1 Pre-clinical Studies 64
8.6.3 Product Development Activities 64
8.6.3.1 IND Acceptance 64
9. THERAPEUTIC ASSESSMENT 65
9.1 Assessment by Stage and Product Type 65
9.2 Assessment by Route of Administration 66
9.3 Assessment by Stage and Route of Administration 67
9.4 Assessment by Molecule Type 68
9.5 Assessment by Stage and Molecule Type 69
10. APPROVAL TIMELINES 70
10.1 Clinical Products Approval Timelines 70
11. ANALYST INSIGHTS 71
12. INACTIVE PRODUCTS 72
12.1 Comparative Analysis 72
12.2 Small Molecule Inhibitor: Evotec 72
12.2.1 Product Description 72
12.2.2 Product Development Activities 72
12.2.2.1 Collaboration 72
Table 2: Clinical evaluation and monitoring of pediatric patients with Fabry Disease 21
Table 3: Clinical evaluation and monitoring of adult patients with Fabry Disease 22
Table 4: Recommendations for initiation of ERT in pediatric patients with FD 30
Table 5: Recommendations for initiation of ERT in adult male and female patients with classic or later-onset mutations, or GLA VUS. 31
Table 6: Adjunctive support for the management of adult patients with Fabry Disease 34
Table 7: Recommended assessments and schedule for monitoring organ involvement in adult patients with Fabry Disease 36
Table 8: Total Number of Products in Fabry Disease 39
Table 9: Late Stage Products (Phase III) 40
Table 10: Clinical Trial Description: Lucerastat 42
Table 11: General Description: Lucerastat 43
Table 12: Clinical Trial Description: Pegunigalsidase alfa 47
Table 13: General Description: Pegunigalsidase alfa 49
Table 14: Mid Stage Products (Phase II) 50
Table 15: Clinical Trial Description: Venglustat 51
Table 16: General Description: Venglustat 51
Table 17: Early Stage Products (Phase I) 53
Table 18: Clinical Trial Description: Apabetalone 54
Table 19: General Description: Apabetalone 54
Table 20: Clinical Trial Description: Moss-aGal 56
Table 21: General Description: Moss-aGal 57
Table 22: Pre-clinical Stage Products 58
Table 23: General Description: Fabry Gene Therapy 59
Table 24: General Description: FTL190 61
Table 25: General Description: mRNA-3630 62
Table 26: General Description: PGN005 63
Table 27: General Description: ST-920 64
Table 28: Assessment by Stage and Product Type 65
Table 29: Assessment by Route of Administration 66
Table 30: Assessment by Stage and Route of Administration 67
Table 31: Assessment by Molecule Type 68
Table 32: Assessment by Stage and Molecule Type 69
Table 33: Approval Timelines for Clinical Products 70
Table 34: Inactive Products 72
Table 35: General Description: Small Molecule Inhibitor 73
1.
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