Table of Content

1. Key Insights

2. Report Introduction

3. Fabry Disease Market Overview at a Glance
3.1. Market Share (%) Distribution of Fabry Disease in 2019
3.2. Market Share (%) Distribution of Fabry Disease in 2032

4. Executive Summary of Fabry Disease

5. Disease Background and Overview
5.1. Introduction
5.2. Classification
5.3. Signs and Symptoms
5.4. Etiology
5.5. Inheritance Pattern
5.6. Genetics
5.7. Molecular Basis
5.8. Pathogenesis
5.9. Diagnosis
5.9.1. European Fabry Working Group (EFWG): Diagnostic criteria
5.9.2. Challenges in Diagnosis
5.10. Treatment and Management of Fabry Disease
5.10.1. Guidelines for the Management of Fabry Disease
5.10.2. Croatian Society: Recommendation for the treatment of Fabry Disease
5.10.3. Treatment Practices
5.10.4. Consensus recommendations for diagnosis, management, and treatment of Fabry disease in pediatric patients

6. Epidemiology and Patient Population
6.1. Key Findings
6.2. 7MM Total Prevalence of Fabry Disease
6.3. Epidemiology of Fabry Disease
6.4. The United States
6.4.1. Prevalence of Fabry Disease in the United States
6.4.2. Gender-specific cases of Fabry Disease in the United States
6.4.3. Phenotype-specific cases of Fabry Disease in the United States
6.4.4. Age-specific cases of Fabry Disease in the United States
6.5. EU5
6.5.1. Total Prevalence of Fabry Disease in EU5
6.5.2. Germany
6.5.2.1. Gender-specific cases of Fabry Disease in Germany
6.5.2.2. Phenotype-specific cases of Fabry Disease in Germany
6.5.2.3. Age-specific cases of Fabry Disease in Germany
6.5.3. France
6.5.3.1. Gender-specific cases of Fabry Disease in France
6.5.3.2. Phenotype-specific cases of Fabry Disease in France
6.5.3.3. Age-specific cases of Fabry Disease in France
6.5.4. Italy
6.5.4.1. Gender-specific cases of Fabry Disease in Italy
6.5.4.2. Phenotype-specific cases of Fabry Disease in Italy
6.5.4.3. Age-specific cases of Fabry Disease in Italy
6.5.5. Spain
6.5.5.1. Gender-specific cases of Fabry Disease in Spain
6.5.5.2. Phenotype-specific cases of Fabry Disease in Spain
6.5.5.3. Age-specific cases of Fabry Disease in Spain
6.5.6. The United Kingdom
6.5.6.1. Gender-specific cases of Fabry Disease in the United Kingdom
6.5.6.2. Phenotype-specific cases of Fabry Disease in the United Kingdom
6.5.6.3. Age-specific cases of Fabry Disease in the United Kingdom
6.5.7. Japan
6.5.7.1. Prevalence of Fabry Disease in Japan
6.5.7.2. Gender-specific cases of Fabry Disease in Japan
6.5.7.3. Phenotype-specific cases of Fabry Disease in Japan
6.5.7.4. Age-specific cases of Fabry Disease in Japan

7. Patient Journey

8. Marketed Therapies
8.1. Keycross Competition
8.2. Fabrazyme: Genzyme
8.2.1. Product Description
8.2.2. Regulatory Milestones
8.2.3. Other Developmental Activities
8.2.4. Safety and Efficacy
8.2.5. Product Profile
8.3. Replagal: Shire/Takeda
8.3.1. Drug Description
8.3.2. Regulatory Milestones
8.3.3. Other Developmental Activities
8.3.4. Safety and Efficacy
8.3.5. Product Profile
8.4. Galafold: Amicus Therapeutics
8.4.1. Drug Description
8.4.2. Regulatory Milestones
8.4.3. Other Developmental Activities
8.4.4. Safety and Efficacy
8.4.5. Product Profile

9. Emerging Therapies
9.1. Key Competitors: Emerging Therapies
9.2. PRX-102 (Pegunigalsidase Alfa): Protalix Biotherapeutics
9.2.1. Product Description
9.2.2. Other Development Activities
9.2.3. Clinical Development
9.2.4. Clinical Trials Information
9.2.5. Safety and Efficacy
9.2.6. Product Profile
9.2.7. Analyst Comment
9.3. Venglustat: Sanofi Genzyme
9.3.1. Product Description
9.3.2. Other Development Activities
9.3.3. Clinical Development
9.3.4. Clinical trials information
9.3.5. Product Profile
9.3.6. Analyst Comment
9.4. ST-920: Sangamo Therapeutics
9.4.1. Product Description
9.4.2. Other Development Activities
9.4.3. Clinical Development
9.4.4. Clinical trials information
9.4.5. Safety and Efficacy
9.4.6. Product Profile
9.4.7. Analyst Comments
9.5. FLT190: Freeline Therapeutics
9.5.1. Product Description
9.5.2. Other Development Activities
9.5.3. Clinical trials information
9.5.4. Safety and Efficacy
9.5.5. Product Profile
9.5.6. Analyst Comment?
9.6. 4D-310: 4D Molecular Therapeutics
9.6.1. Product Description
9.6.2. Other Development Activities
9.6.3. Clinical Development
9.6.4. Clinical trials information
9.6.5. Safety and Efficacy
9.6.6. Product Profile
9.6.7. Analyst Comment

10. Other Assets
10.1. Key Competitors
10.2. Lucerastat: Idorsia Pharmaceuticals
10.2.1. Product Description
10.2.2. Other Development Activities
10.2.3. Clinical Development
10.2.4. Clinical Trials Information
10.2.5. Safety and Efficacy
10.2.6. Product Profile
10.3. Moss-aGal: GREENOVATION BIOTECH GMBH
10.3.1. Product Description
10.3.2. Clinical Development
10.3.3. Clinical Trials Information
10.3.4. Safety and Efficacy
10.3.5. Product Profile

11. Discontinued Asset
11.1. AVR-RD-01: AVROBIO
11.1.1. Product Description
11.1.2. Other Development Activities
11.1.3. Clinical Development
11.1.4. Clinical trials information
11.1.5. Safety and Efficacy
11.1.6. Product Profile

12. Fabry Disease: Seven Major Market Analysis
12.1. Key Findings
12.2. Market Size of Fabry Disease in 7MM
12.3. Market Outlook
12.4. The United States Market Size
12.4.1. Total Market Size of Fabry Disease in the United States
12.4.2. Market Size of Fabry Disease by Therapies in the United States
12.5. Market Size of Fabry Disease in EU-5
12.5.1. Market Size of Fabry Disease by Therapies in Germany
12.5.2. Market Size of Fabry Disease by Therapies in France
12.5.3. Market Size of Fabry Disease by Therapies in Italy
12.5.4. Market Size of Fabry Disease by Therapies in Spain
12.5.5. Market Size of Fabry Disease by Therapies in the United Kingdom
12.6. Japan Market Size
12.6.1. Total Market Size of Fabry Disease in Japan
12.6.2. Market Size of Fabry Disease by Therapies in Japan

13. KOL Views

14. SWOT Analysis

15. Unmet Needs

16. Market Access

17. Appendix
17.1. Bibliography
17.2. Report Methodology

18. DelveInsight Capabilities

19. Disclaimer

20. About DelveInsight



List of Figures

Figure 1: Characteristics of Type 1 Classic and Type 2 Later-onset Fabry Disease
Figure 2: Symptoms of Fabry Disease in Adults, Childhood, and Adolescence
Figure 3: Role of a-galactosidase A in GL-3 Catabolism
Figure 4: Inheritance Pattern of Fabry Disease
Figure 5: ?-galactosidase A gene Mutations
Figure 6: Genetic and Molecular Basis of Fabry Disease
Figure 7: Diagnosis Algorithm for Fabry Disease
Figure 8: Treatment Algorithm
Figure 9: Treatment Algorithm for Fabry Disease in Pediatric Patients
Figure 10: Treatment Algorithm for Fabry Disease in Pediatric Patients
Figure 11: Total Prevalent Population of Fabry Disease in 7MM (2019–2032)
Figure 12: Prevalence of Fabry Disease in the United States (2019–2032)
Figure 13: Gender-specific cases of Fabry Disease in the United States (2019–2032)
Figure 14: Phenotype-specific cases of Fabry Disease in the United States (2019–2032)
Figure 15: Age-specific cases of Fabry Disease in the United States (2019–2032)
Figure 16: Total Prevalent Population of Fabry Disease in EU5 (2019–2032)
Figure 17: Gender-specific cases of Fabry Disease in Germany (2019–2032)
Figure 18: Phenotype-specific cases of Fabry Disease in Germany (2019–2032)
Figure 19: Age-specific cases of Fabry Disease in Germany (2019–2032)
Figure 20: Gender-specific cases of Fabry Disease in France (2019–2032)
Figure 21: Phenotype-specific cases of Fabry Disease in France (2019–2032)
Figure 22: Age-specific cases of Fabry Disease in France (2019–2032)
Figure 23: Gender-specific cases of Fabry Disease in Italy (2019–2032)
Figure 24: Phenotype-specific cases of Fabry Disease in Italy (2019–2032)
Figure 25: Age-specific cases of Fabry Disease in Italy (2019–2032)
Figure 26: Gender-specific cases of Fabry Disease in Spain (2019–2032)
Figure 27: Phenotype-specific cases of Fabry Disease in Spain (2019–2032)
Figure 28: Age-specific cases of Fabry Disease in Spain (2019–2032)
Figure 29: Gender-specific cases of Fabry Disease in the United Kingdom (2019–2032)
Figure 30: Phenotype-specific cases of Fabry Disease in the United Kingdom (2019–2032)
Figure 31: Age-specific cases of Fabry Disease in the United Kingdom (2019–2032)
Figure 32: Prevalence of Fabry Disease in Japan (2019–2032)
Figure 33: Gender-specific cases of Fabry Disease in Japan (2019–2032)
Figure 34: Phenotype-specific cases of Fabry Disease in Japan (2019–2032)
Figure 35: Age-specific cases of Fabry Disease in Japan (2019–2032)
Figure 36: Seven Major Market Size of Fabry Disease in USD Million (2019–2032)
Figure 37: Market Size of Fabry Disease in the United States in USD Million (2019–2032)
Figure 38: Market Size of Fabry Disease in the United States by Therapies (2019–2032)
Figure 39: EU5 Market Size of Fabry Disease in USD Million (2019–2032)
Figure 40: Market Size of Fabry Disease in Germany by Therapies (2019–2032)
Figure 41: Market Size of Fabry Disease in France by Therapies (2019–2032)
Figure 42: Market Size of Fabry Disease in Italy by Therapies (2019–2032)
Figure 43: Market Size of Fabry Disease in Spain by Therapies (2019–2032)
Figure 44: Market Size of Fabry Disease in the United Kingdom by Therapies (2019–2032)
Figure 45: Market Size of Fabry Disease in Japan in USD Million (2019–2032)
Figure 46: Market Size of Fabry Disease in Japan by Therapies (2019–2032)

List of Tables

Table 1: Summary of Fabry Disease, Epidemiology, and Key Events (2019–2032)
Table 2: Pathophysiologic Findings in Fabry Disease
Table 3: Proposed Assessments for Fabry Disease
Table 4: Clinical Evaluation and Monitoring of Adult Patients With Fabry Disease
Table 5: Clinical evaluation and Monitoring of Pediatric Patients With Fabry Disease
Table 6: Diagnostic criteria of European Fabry Working Group (EFWG)
Table 7: Gold-standard Methods to Diagnose Fabry Disease
Table 8: Fabry disease: Common Misdiagnoses
Table 9: Current Guidelines for Instituting Enzyme Replacement Therapy in Fabry Disease Patients
Table 10: Adjunctive Therapies and Preventive Measures
Table 11: Implications for Recommendations
Table 12: Treatment Goals for Fabry Disease Patients Associated With Other Conditions
Table 13: Effect of Enzyme Replacement Therapy on Signs and Symptoms of Fabry Disease
Table 14: Enzyme Replacement Therapy
Table 15: Recommended Analgesic Drugs for Supportive Treatment of Chronic Neuropathic Pain
Table 16: Recommended Analgesic Drugs for Supportive Treatment of Acute Pain in Fabry Disease
Table 17: Prevalence of Fabry Disease in the 7MM (2019–2032)
Table 18: Prevalence of Fabry Disease in the United States (2019–2032)
Table 19: Gender-specific cases of Fabry Disease in the United States (2019–2032)
Table 20: Phenotype-specific cases of Fabry Disease in the United States (2019–2032)
Table 21: Age-specific cases of Fabry Disease in the United States (2019–2032)
Table 22: Prevalence of Fabry Disease in EU5 (2019–2032)
Table 23: Gender-specific cases of Fabry Disease in Germany (2019–2032)
Table 24: Phenotype-specific cases of Fabry Disease in Germany (2019–2032)
Table 25: Age-specific cases of Fabry Disease in Germany (2019–2032)
Table 26: Gender-specific cases of Fabry Disease in France (2019–2032)
Table 27: Phenotype-specific cases of Fabry Disease in France (2019–2032)
Table 28: Age-specific cases of Fabry Disease in France (2019–2032)
Table 29: Gender-specific cases of Fabry Disease in Italy (2019–2032)
Table 30: Phenotype-specific cases of Fabry Disease in Italy (2019–2032)
Table 31: Age-specific cases of Fabry Disease in Italy (2019–2032)
Table 32: Gender-specific cases of Fabry Disease in Spain (2019–2032)
Table 33: Phenotype-specific cases of Fabry Disease in Spain (2019–2032)
Table 34: Age-specific cases of Fabry Disease in Spain (2019–2032)
Table 35: Gender-specific cases of Fabry Disease in the United Kingdom (2019–2032)
Table 36: Phenotype-specific cases of Fabry Disease in the United Kingdom (2019–2032)
Table 37: Age-specific cases of Fabry Disease in the United Kingdom (2019–2032)
Table 38: Prevalence of Fabry Disease in Japan (2019–2032)
Table 39: Gender-specific cases of Fabry Disease in Japan (2019–2032)
Table 40: Phenotype-specific cases of Fabry Disease in Japan (2019–2032)
Table 41: Age-specific cases of Fabry Disease in Japan (2019–2032)
Table 42: Patent Details: Migalastat
Table 43: Pegunigalsidase Alfa, Clinical Trial Description, 2022
Table 44: Venglustat, Clinical Trial Description, 2022
Table 45: ST-920, Clinical Trial Description, 2022
Table 46: FLT190, Clinical Trial Description, 2022
Table 47: 4D-310, Clinical Trial Description, 2022
Table 48: Lucerastat, Clinical Trial Description, 2022
Table 49: Moss-aGal, Clinical Trial Description, 2022
Table 50: AVR-RD-01 Clinical Trial Description, 2022
Table 51: Seven Major Market Size of Fabry Disease in USD Million (2019–2032)
Table 52: Market Size of Fabry Disease in the United States in USD Million (2019–2032)
Table 53: The US Market Size of Fabry Disease by Therapies in USD Million (2019–2032)
Table 54: EU5 Market Size of Fabry Disease in USD Million (2019–2032)
Table 55: Market Size of Fabry Disease by Therapies in Germany in USD Million (2019–2032)
Table 56: Market Size of Fabry Disease by Therapies in France in USD Million (2019–2032)
Table 57: Market Size of Fabry Disease by Therapies in Italy in USD Million (2019–2032)
Table 58: Market Size of Fabry Disease by Therapies in Spain in USD Million (2019–2032)
Table 59: Market Size of Fabry Disease by Therapies in the UK in USD Million (2019–2032)
Table 60: Market Size of Fabry Disease in Japan in USD Million (2019–2032)
Table 61: Market Size of Fabry Disease by Therapies in Japan in USD Million (2019–2032)
Table 62: Summary of Evaluation Committee?s Key Conclusions