Fabry Disease - Competitive Analysis 2019
- UNI4065854
- 95 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.
There are 2+ products approved for the treatment of Fabry Disease. 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:
• The report provides the marketed drugs information including its approval details, development activities and details of patent expiry
• 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 competitive report 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 approval 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 dormancy of the products
Customization Options:
The Fabry Disease competitive 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 10
1.2 Secondary Research 10
1.3 Scope of the report: 10
2. DISEASE OVERVIEW 11
2.1 Introduction 11
2.2 Etiology 12
2.3 Classification 13
2.4 Signs and Symptoms 14
2.4.1 The early and progressive clinical symptoms include: 14
2.5 Pathogenesis 15
2.5.1 Metabolic Defect 15
2.5.2 The Molecular Basis 17
2.5.3 Genotype/Phenotype Correlation 20
2.6 Diagnosis 21
2.6.1 Biochemical Diagnosis: 21
2.6.1.1 Measurement of a-Gal Activity in Cultivated Fibroblasts, Plasma, or Leukocytes 21
2.6.1.2 Measurement of α-Gal A Activity in Blood Spot Filter Paper 22
2.6.1.3 GL-3 Levels 22
2.6.2 Molecular Diagnosis 22
2.6.3 Molecular Analysis 23
2.7 Treatment 25
2.7.1 Clinical management of pediatric patients 25
2.7.2 Management of Fabry related pain in pediatric patients 26
2.7.2.1 Trigger management 26
2.7.2.2 Symptomatic therapy 26
2.7.2.3 Management of neuropathic pain 26
2.7.2.4 Management of psychological disturbances 27
2.7.3 Treatment Algorithm for the management of neuropathic pain in paediatric Fabry Disease patients 28
2.7.3.1 Enzyme replacement therapy 29
2.7.3.2 Recommendations for ERT initiation 30
2.7.4 Management in adults 31
2.7.4.1 Available Enzyme Replacement therapies 32
2.7.4.2 Initiation of enzyme replacement therapy 32
2.7.4.3 Expert panel recommendations for initiation of enzyme replacement therapy in adult patients with Fabry Disease 32
2.7.4.4 Adjunctive therapies 34
2.7.4.5 Non-ERT approaches for patients with specific mutations 36
2.7.5 Monitoring of adult patients with Fabry Disease 36
3. PRODUCTS UNDER DEVELOPMENT BY TECHNOLOGY 39
4. PRODUCTS UNDER DEVELOPMENT BY DESIGNATION 40
4.1 Comparative Analysis 40
5. TOTAL NUMBER OF MARKETED AND EMERGING PRODUCTS 41
5.1 Comparative Analysis 41
6. MARKETED PRODUCTS 42
6.1 Galafold: Amicus Therapeutics 42
6.1.1 Product Description 42
6.1.1.1 Dosage and Administration: 42
6.1.2 Regulatory Milestones 43
6.1.2.1 Approval 43
6.1.3 Research and Development 43
6.1.3.1 Clinical Studies 43
6.1.3.1.1 Clinical Studies 43
6.1.3.1.2 Results of Analysis 44
6.1.4 Product Development Activities 46
6.1.4.1 Positive Recommendation 46
6.1.4.2 Designation 46
6.1.4.3 Patents 46
6.1.4.4 Licensing 47
6.1.4.4.1 GlaxoSmithKline 47
6.1.4.4.2 Mt. Sinai School of Medicine 47
6.2 Replagal: Shire 49
6.2.1 Product Description 49
6.2.1.1 Dosage and Administration: 49
6.2.2 Regulatory Milestones 49
6.2.2.1 Approval 49
6.2.3 Research and Development 49
6.2.3.1 Clinical Studies 49
6.2.4 Product Development Activities 51
6.2.4.1 BLA Withdrawn 51
6.2.4.2 Business Expansion 51
6.2.4.3 Acquisition 51
6.2.4.4 Designation 51
6.3 Fabrazyme: Sanofi Genzyme 52
6.3.1 Product Description 52
6.3.1.1 Dosage and Administration: 53
6.3.2 Regulatory Milestones 53
6.3.2.1 Approval 53
6.3.3 Research and Development 53
6.3.3.1 Clinical Studies 53
6.3.4 Product Development Activities 56
6.3.4.1 Designation 56
7. PIPELINE THERAPEUTICS AT A GLANCE 57
7.1 General Overview 57
8. COMPARATIVE ANALYSIS 58
8.1 General Overview 58
9. LATE PHASE PRODUCTS (PHASE III) 59
9.1 Comparative Analysis 59
9.2 Lucerastat: Idorsia Pharmaceuticals 59
9.2.1 Product Description 59
9.2.2 Research and Development 60
9.2.2.1 Clinical Studies 60
9.2.2.1.1 Phase III 60
9.2.2.1.2 Phase I 60
9.2.2.1.3 Results of Analysis 60
9.2.2.1.4 Results of Analysis II 61
9.2.3 Product Development Activities 62
9.2.3.1 Designation 62
9.2.3.2 Spin Off 62
9.3 Pegunigalsidase alfa: Protalix Biotherapeutics 63
9.3.1 Product Description 63
9.3.2 Research and Development 63
9.3.2.1 Clinical Studies 63
9.3.2.1.1 Phase III 63
9.3.2.1.2 Results of Analysis 64
9.3.2.1.3 Results of Analysis II 65
9.3.3 Product Development Activities 66
9.3.3.1 Designation 66
9.3.3.2 Agreement 66
9.3.3.3 Patent 67
9.3.3.4 Technology 67
10. MID PHASE PRODUCTS (PHASE II) 69
10.1 Comparative Analysis 69
10.2 Venglustat: Sanofi Genzyme 69
10.2.1 Product Description 69
10.2.2 Research and Development 70
10.2.2.1 Clinical Studies 70
10.2.2.1.1 Phase II 70
10.2.3 Product Development Activities 70
10.2.3.1 Designation 70
11. EARLY STAGE PRODUCTS (PHASE I) 72
11.1 Comparative Analysis 72
11.2 Apabetalone: Resverlogix Corp 72
11.2.1 Product Description 72
11.2.2 Research and Development 73
11.2.2.1 Clinical Studies 73
11.2.2.1.1 Phase II 73
11.3 Moss-aGal: Greenovation Biotech 74
11.3.1 Product Description 74
11.3.2 Research and Development 75
11.3.2.1 Clinical Studies 75
11.3.2.1.1 Phase I 75
11.3.2.1.2 Results of Analysis 75
11.3.3 Product Development Activities 76
11.3.3.1 Technology 76
12. PRE-CLINICAL STAGE PRODUCTS 77
12.1 Comparative Analysis 77
12.2 Fabry Gene Therapy: Amicus Therapeutics 77
12.2.1 Product Description 77
12.2.2 Product Development Activities 78
12.2.2.1 Agreement 78
12.3 FTL190: Freeline 79
12.3.1 Product Description 79
12.3.2 Research and Development 79
12.3.2.1 Pre-clinical Studies 79
12.3.3 Product Development Activities 80
12.3.3.1 Financing 80
12.4 mRNA-3630: Moderna 81
12.4.1 Product Description 81
12.5 PGN005 (α-galactosidase): Pharming 81
12.5.1 Product Description 81
12.6 ST-920: Sangamo Therapeutics 82
12.6.1 Product Description 82
12.6.2 Research and Development 82
12.6.2.1 Pre-clinical Studies 82
12.6.3 Product Development Activities 82
12.6.3.1 IND Acceptance 82
13. THERAPEUTIC ASSESSMENT 84
13.1 Assessment by Stage and Product Type 84
13.2 Assessment by Route of Administration 85
13.3 Assessment by Stage and Route of Administration 86
13.4 Assessment by Molecule Type 87
13.5 Assessment by Stage and Molecule Type 88
14. APPROVAL TIMELINES 89
14.1 Approval Timeline for Clinical Products 89
15. ANALYST INSIGHTS 90
16. INACTIVE PRODUCTS 91
16.1 Comparative Analysis 91
16.2 Small Molecule Inhibitor: Evotec 91
16.2.1 Product Description 91
16.2.2 Product Development Activities 91
16.2.2.1 Collaboration 91
Table 2: Clinical evaluation and monitoring of pediatric patients with Fabry Disease 23
Table 3: Clinical evaluation and monitoring of adult patients with Fabry Disease 24
Table 4: Recommendations for initiation of ERT in pediatric patients with FD 31
Table 5: Recommendations for initiation of ERT in adult male and female patients with classic or later-onset mutations, or GLA VUS. 32
Table 6: Adjunctive support for the management of adult patients with Fabry Disease 35
Table 7: Recommended assessments and schedule for monitoring organ involvement in adult patients with Fabry Disease 37
Table 8: Products under development by Technology 39
Table 9: Products under development by Designation 40
Table 10: Total Number of Marketed and Emerging Products 41
Table 11: Marketed Products 42
Table 12: Clinical Trial Description: Migalastat 45
Table 13: General Description: Migalastat 48
Table 14: Clinical Trial Description: Replagal 51
Table 15: General Description: Replagal 52
Table 16: Reduction of GL-3 Inclusions to Normal or Near Normal Levels (0 Score) in the Capillary Endothelium of the Kidney, Heart, and Skin 54
Table 17: Clinical Trial Description: Fabrazyme 55
Table 18: General Description: Fabrazyme 56
Table 19: Total Number of Products in Fabry Disease 58
Table 20: Late Stage Products (Phase III) 59
Table 21: Clinical Trial Description: Lucerastat 61
Table 22: General Description: Lucerastat 62
Table 23: Clinical Trial Description: Pegunigalsidase alfa 66
Table 24: General Description: Pegunigalsidase alfa 68
Table 25: Mid Stage Products (Phase II) 69
Table 26: Clinical Trial Description: Venglustat 70
Table 27: General Description: Venglustat 70
Table 28: Early Stage Products (Phase I) 72
Table 29: Clinical Trial Description: Apabetalone 73
Table 30: General Description: Apabetalone 74
Table 31: Clinical Trial Description: Moss-aGal 75
Table 32: General Description: Moss-aGal 76
Table 33: Pre-clinical Stage Products 77
Table 34: General Description: Fabry Gene Therapy 78
Table 35: General Description: FTL190 80
Table 36: General Description: mRNA-3630 81
Table 37: General Description: PGN005 81
Table 38: General Description: ST-920 83
Table 39: Assessment by Stage and Product Type 84
Table 40: Assessment by Route of Administration 85
Table 41: Assessment by Stage and Route of Administration 86
Table 42: Assessment by Molecule Type 87
Table 43: Assessment by Stage and Molecule Type 88
Table 44: Approval Timelines for Clinical Products 89
Table 45: Inactive Products 91
Table 46: General Description: Small Molecule Inhibitor 92
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