1. Key Insights 2. Niemann–Pick Disease Type C (NPC) Market Overview at a Glance 2.1. Market Share (%) Distribution of Niemann–Pick Disease Type C (NPC) in 2017 2.2. Market Share (%) Distribution of Niemann–Pick Disease Type C (NPC) in 2028 3. Disease Background and Overview: Niemann–Pick Disease Type C (NPC) 3.1. Introduction 3.2. Classification of Niemann Pick Disease 3.3. Causes 3.4. Signs and Symptoms 3.4.1. Sign and symptoms on the basis of the age of onset 3.5. Inheritance patterns 3.6. Pathophysiology 3.7. Diagnosis 3.7.1. NPC Diagnostic Recommendations Expert Panel: Diagnostic Recommendations (2016) 3.7.2. NPC suspicion Index Tool 4. Epidemiology and Patient Population 4.1. Key Findings 4.2. 7MM Total Prevalent Patient Population of Niemann Pick Disease Type-C 4.3. 7MM Total Diagnosed Prevalent Patient Population of Niemann Pick Disease Type-C 5. Country Wise-Epidemiology of NPC 5.1. United States 5.1.1. KOL Insights 5.1.2. Total Prevalent Patient Population of NPC in the United States 5.1.3. Sub-type Specific Prevalence of NPC in the United States 5.1.4. Diagnosed Population of NPC in the United States 5.1.5. Diagnosed Prevalence of NPC based on Clinical Phenotypes 5.2. EU5 Countries 5.2.1. KOL Insights 5.3. Germany 5.3.1. Total Prevalent Patient Population of NPC in Germany 5.3.2. Sub-type Specific Prevalence of NPC in Germany 5.3.3. Diagnosed Population of NPC in Germany 5.3.4. Diagnosed Prevalence of NPC based on Clinical Phenotypes 5.4. France 5.4.1. Total Prevalent Patient Population of NPC in France 5.4.2. Sub-type Specific Prevalence of NPC in France 5.4.3. Diagnosed Population of NPC in France 5.4.4. Diagnosed Prevalence of NPC based on Clinical Phenotypes 5.5. Italy 5.5.1. Total Prevalent Patient Population of NPC in Italy 5.5.2. Sub-type Specific Prevalence of NPC in Italy 5.5.3. Diagnosed Population of NPC in Italy 5.5.4. Diagnosed Prevalence of NPC based on Clinical Phenotypes 5.6. Spain 5.6.1. Total Prevalent Patient Population of NPC in Spain 5.6.2. Sub-type Specific Prevalence of NPC in Spain 5.6.3. Diagnosed Population of NPC in Spain 5.6.4. Diagnosed Prevalence of NPC based on Clinical Phenotypes 5.7. United Kingdom 5.7.1. Total Prevalent Patient Population of NPC in the UK 5.7.2. Sub-type Specific Prevalence of NPC in the UK 5.7.3. Diagnosed Population of NPC in the United Kingdom 5.7.4. Diagnosed Prevalence of NPC based on Clinical Phenotypes 5.8. Japan 5.8.1. KOL Insights 5.8.2. Total Prevalent Patient Population of NPC in Japan 5.8.3. Sub-type Specific Prevalence of NPC in Japan 5.8.4. Diagnosed Population of NPC in Japan 5.8.5. Diagnosed Prevalence of NPC based on Clinical Phenotypes 6. Treatment Practices 6.1. Treatment Goals 6.2. Treatment Algorithm 6.3. Consensus clinical management guidelines for Niemann–Pick disease type C 7. Unmet Needs 8. Marketed drugs 8.1. Zavesca: Actelion Pharmaceuticals 8.1.1. Drug description 8.1.2. Regulatory milestones 8.1.3. Generic Availability 8.1.4. Clinical Development 8.1.5. Safety and efficacy 8.1.6. Product Profile 9. Emerging drugs 9.1. Key Cross Analysis 9.2. Arimoclomol: Orphazyme 9.2.1. Drug Description 9.2.2. Other Developmental Activities 9.2.3. Clinical Development 9.2.4. Safety and Efficacy 9.2.5. Product Profile 9.3. VTS-270: Vtesse (Mallinckrodt) 9.3.1. Drug Description 9.3.2. Other Developmental Activities 9.3.3. Clinical Development 9.3.4. Safety and Efficacy 9.3.5. Product Profile 9.4. Trappsol Cyclo (Cyclodextrin): CTD Holdings 9.4.1. Drug Description 9.4.2. Other Developmental Activities 9.4.3. Clinical Development 9.4.4. Safety and Efficacy 9.4.5. Product Profile 9.5. IB1001: IntraBio 9.5.1. Drug Description 9.5.2. Other Developmental Activities 9.5.3. Clinical Development 9.5.4. Product Profile 10. Niemann Pick Disease Type C: 7 Major Market Analysis 10.1. Key Findings 10.2. Total Market Size of Niemann Pick Disease Type C in 7MM 10.3. Market Size by Therapeutic Class in 7MM 10.4. Market Size by Therapies in 7MM 11. The United States: Market Outlook 11.1. United States Market Size 11.1.1. Total Market size of Niemann Pick Disease Type C [NPC] 11.1.2. Niemann Pick Disease Type C Market by Therapeutic Class 11.1.3. Market Size by Therapies 12. EU-5 Countries: Market Outlook 12.1. Germany 12.1.1. Total Market size of Niemann Pick Disease Type C 12.1.2. Niemann Pick Disease Type C Market by Therapeutic Class 12.1.3. Market Size by Therapies 12.2. France 12.2.1. Total Market size of Niemann Pick Disease Type C 12.2.2. Niemann Pick Disease Type C Market by Therapeutic Class 12.2.3. Market Size by Therapies 12.3. Italy 12.3.1. Total Market size of Niemann Pick Disease Type C 12.3.2. Niemann Pick Disease Type C Market by Therapeutic Class 12.3.3. Market Size by Therapies 12.4. Spain 12.4.1. Total Market size of Niemann Pick Disease Type C 12.4.2. Niemann Pick Disease Type C Market by Therapeutic Class 12.4.3. Market Size by Therapies 12.5. United Kingdom 12.5.1. Total Market size of Niemann Pick Disease Type C 12.5.2. Niemann Pick Disease Type C Market by Therapeutic Class 12.5.3. Market Size by Therapies 13. Japan: Market Outlook 13.1.1. Total Market size of Niemann Pick Disease Type C 13.1.2. Niemann Pick Disease Type C Market by Therapeutic Class 13.1.3. Market Size by Therapies 14. Market Drivers 15. Market Barriers 16. Appendix 16.1. Report Methodology 17. DelveInsight Capabilities 18. Disclaimer 19. About DelveInsight

Table 1: Types of Neiman-Pick Disease type, distinguish features, primary pathophysiology, and genetics issue. Table 2: Sign and symptom combination strongly suggestive of NPC Table 3: Cell biology diagnostic techniques in NPC Table 4: Primary and urinary diagnostic biomarkers in NPC Table 5: Clinical patient groups with an increased risk of Niemann–Pick Disease Type C Table 6: Diagnosis Table 7: Recommendations for diagnosis of patients identified by molecular genetic screening for NPC1 and NPC2 Table 8: NPC suspicion index tool: Signs and symptoms Table 9: Total Prevalent Patient Population of NPC in 7MM (2017-2028) Table 10: Total Diagnosed Prevalent Patient Population of NPC in the 7MM (2017-2028) Table 11: Total Prevalent Population of NPC in the United States (2017-2028) Table 12: Sub-type Specific Prevalence of NPC in the United States (2017-2028) Table 13: Diagnosed Population of NPC in the United States (2017-2028) Table 14: Diagnosed Prevalence of NPC based on Clinical Phenotypesin the United States (2017-2028) Table 15: Total Prevalent Population of NPC in Germany (2017-2028) Table 16: Sub-type Specific Prevalence of NPC in Germany (2017-2028) Table 17: Diagnosed Population of NPC in the United States (2017-2028) Table 18: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Germany (2017-2028) Table 19: Total Prevalent Population of NPC in France (2017-2028) Table 20: Sub-type Specific Prevalence of NPC in France (2017-2028) Table 21: Diagnosed Population of NPC in France (2017-2028) Table 22: Diagnosed Prevalence of NPC based on Clinical Phenotypes in France (2017-2028) Table 23: Total Prevalent Population of NPC in Italy (2017-2028) Table 24: Sub-type Specific Prevalence of NPC in Italy (2017-2028) Table 25: Diagnosed Population of NPC in Italy (2017-2028) Table 26: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Italy (2017-2028) Table 27: Total Prevalent Population of NPC in Spain (2017-2028) Table 28: Sub-type Specific Prevalence of NPC in Spain (2017-2028) Table 29: Diagnosed Population of NPC in Spain (2017-2028) Table 30: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Spain (2017-2028) Table 31: Total Prevalent Population of NPC in the UK (2017-2028) Table 32: Sub-type Specific Prevalence of NPC in the UK (2017-2028) Table 33: Diagnosed Population of NPC in the United Kingdom (2017-2028) Table 34: Diagnosed Prevalence of NPC based on Clinical Phenotypes in the UK (2017-2028) Table 35: Total Prevalent Population of NPC in Japan (2017-2028) Table 36: Sub-type Specific Prevalence of NPC in Japan (2017-2028) Table 37: Diagnosed Population of NPC in Japan (2017-2028) Table 38: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Japan (2017-2028) Table 39: Symptomatic Therapies for Niemann–Pick Disease type C Table 40: Zavesca, Clinical Trial Description, 2019 Table 41: Emerging Drugs Analysis Table 42: Arimoclomol, Clinical Trial Description, 2019 Table 43: VTS-270, Clinical Trial Description, 2019 Table 44: Trappsol Cyclo, Clinical Trial Description, 2019 Table 45: IB1001, Clinical Trial Description, 2019 Table 46: 7 Major Market Size of Niemann Pick Disease Type C in USD Million (2017-2028) Table 47: 7MM Market Size of NPC by Therapeutic Class in USD Million (2017-2028) Table 48 Market size of Niemann Pick Disease Type C by Therapies in 7MM, USD Million (2017-2028) Table 49: The US Market Niemann Pick Disease Type C in USD Million (2017-2028) Table 50: The U.S. NPC Market by Therapeutic Class in USD Million (2017-2028) Table 51: The US NPC market size by Therapies in USD Million (2017-2028) Table 52: Market Size of NPC in Germany, in USD Million (2017-2028) Table 53: Germany NPC Market by Therapeutic Class in USD Million (2017-2028) Table 54: Germany NPC market size by Therapies in USD Million (2017-2028) Table 55: Market Size of Niemann Pick Disease Type C in France, in USD Million (2017-2028) Table 56: France NPC Market by Therapeutic Class in USD Million (2017-2028) Table 57: France NPC market size by Therapies in USD Million (2017-2028) Table 58: Market Size of Niemann Pick Disease Type C in Italy, in USD Million (2017-2028) Table 59: Italy NPC Market by Therapeutic Class in USD Million (2017-2028) Table 60: Italy NPC market size by Therapies in USD Million (2017-2028) Table 61: Market Size of Niemann Pick Disease Type C in Spain, in USD Million (2017-2028) Table 62: Spain NPC Market by Therapeutic Class in USD Million (2017-2028) Table 63: Spain NPC market size by Therapies in USD Million (2017-2028) Table 64: Market Size of Niemann Pick Disease Type C in the UK, in USD Million (2017-2028) Table 65: The UK NPC Market by Therapeutic Class in USD Million (2017-2028) Table 66: Emerging Therapies market of Niemann Pick Disease Type C in the UK, in USD Million (2017-2028) Table 67: Market Size of Niemann Pick Disease Type C in Japan, in USD Million (2017-2028) Table 68: Japan Niemann Pick Disease Type C Market by Therapeutic Class in USD Million (2017-2028) Table 69: Japan NPC market size by Therapies in USD Million (2017-2028) Figure 1: Classification of Niemann Pick Disease Figure 2: Classification of Niemann–Pick Disease Type C Figure 3: Classification of signs and symptoms in NPC Figure 4: Manifestations of Niemann–Pick Disease Type C (NPC) by age group Figure 5: Schematic representation of Niemann–Pick Disease Type C Figure 6: Inheritance Pattern of Niemann–Pick Disease Type C (NPC) Figure 7: Pathophysiology of Niemann–Pick Disease Type C 1 Disease Figure 8: Pathophysiology of Niemann–Pick Disease Type C 2 Disease Figure 9: Diagnostic Algorithm for NPC Figure 10: Diagnostic Algorithm for Niemann–Pick Disease Type C Figure 11: Total Prevalent Patient Population of NPC in 7MM (2017-2028) Figure 12: Total Diagnosed Prevalent Patient Population of NPC in the 7MM (2017-2028) Figure 13: Prevalence of NPC in the United States (2017-2028) Figure 14: Sub-type Specific Prevalence of NPC in the United States (2017-2028) Figure 15: Diagnosed Population of NPC in the United States (2017-2028) Figure 16: Diagnosed Prevalence of NPC based on Clinical Phenotypes in the United States (2017-2028) Figure 17: Total Prevalent Population of NPC in Germany (2017-2028) Figure 18: Sub-type Specific Prevalence of NPC in Germany (2017-2028) Figure 19: Diagnosed Population of NPC in Germany (2017-2028) Figure 20: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Germany (2017-2028) Figure 21: Total Prevalent Population of NPC in France (2017-2028) Figure 22: Sub-type Specific Prevalence of NPC in France (2017-2028) Figure 23: Diagnosed Population of NPC in France (2017-2028) Figure 24: Diagnosed Prevalence of NPC based on Clinical Phenotypes in France (2017-2028) Figure 25: Total Prevalent Population of NPC in Italy (2017-2028) Figure 26: Sub-type Specific Prevalence of NPC in Italy (2017-2028) Figure 27: Diagnosed Population of NPC in Italy (2017-2028) Figure 28: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Italy (2017-2028) Figure 29: Total Prevalent Population of NPC in Spain (2017-2028) Figure 30: Sub-type Specific Prevalence of NPC in Spain (2017-2028) Figure 31: Diagnosed Population of NPC in Spain (2017-2028) Figure 32: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Spain (2017-2028) Figure 33: Total Prevalent Population of NPC in the UK (2017-2028) Figure 34: Sub-type Specific Prevalence of NPC in the UK (2017-2028) Figure 35: Diagnosed Population of NPC in the United Kingdom (2017-2028) Figure 36: Diagnosed Prevalence of NPC based on Clinical Phenotypes in the UK (2017-2028) Figure 37: Total Prevalent Population of NPC in Japan (2017-2028) Figure 38: Sub-type Specific Prevalence of NPC in Japan (2017-2028) Figure 39: Diagnosed Population of NPC in Japan (2017-2028) Figure 40: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Japan (2017-2028) Figure 41: Treatment Algorithm for Niemann–Pick Disease Type C (NPC) Figure 42: Unmet Needs for Niemann–Pick Disease Type C Figure 43: 7 Major Market Size of Niemann Pick Disease Type C in USD Million (2017-2028) Figure 44: 7MM Market Size of NPC by Therapeutic Class in USD Million (2017-2028) Figure 45: Market size of Niemann Pick Disease Type C by Therapies in 7MM, USD Million (2017-2028) Figure 46: Market Size of NPC in the United States, USD Millions (2017-2028) Figure 47: The U.S. NPC Market by Therapeutic Class in USD Million (2017-2028) Figure 48: The U.S. market size of NPC by therapies in USD Million (2017-2028) Figure 49: Market Size of NPC in Germany, USD Millions (2017-2028) Figure 50: Germany NPC Market by Therapeutic Class in USD Million (2017-2028) Figure 51: Germany NPC market size by Therapies in USD Million (2017-2028) Figure 52: Market Size of Niemann Pick Disease Type C in France, USD Millions (2017-2028) Figure 53: France NPC Market by Therapeutic Class in USD Million (2017-2028) Figure 54: France NPC market size by Therapies in USD Million (2017-2028) Figure 55: Market Size of Niemann Pick Disease Type C in Italy, USD Millions (2017-2028) Figure 56: Italy NPC Market by Therapeutic Class in USD Million (2017-2028) Figure 57: Italy NPC market size by Therapies in USD Million (2017-2028) Figure 58: Market Size of Niemann Pick Disease Type C in Spain, USD Millions (2017-2028) Figure 59: Spain NPC Market by Therapeutic Class in USD Million (2017-2028) Figure 60: Spain NPC market size by Therapies in USD Million (2017-2028) Figure 61: Market Size of Niemann Pick Disease Type C in the UK, USD Millions (2017-2028) Figure 62: The UK NPC Market by Therapeutic Class in USD Million (2017-2028) Figure 63: The UK NPC market size by Therapies in USD Million (2017-2028) Figure 64: Market Size of Niemann Pick Disease Type C in Japan, USD Millions (2017-2028) Figure 65: Japan NPC Market by Therapeutic Class in USD Million (2017-2028) Figure 66: Japan NPC market size by Therapies in USD Million (2017-2028) Figure 67: Market Drivers for Niemann–Pick Disease Type C Figure 68: Market Barriers for Niemann–Pick Type C Disease

1. Actelion Pharmaceuticals 2. Orphazyme 3. Vtesse (Mallinckrodt) 4. CTD Holdings 5. IntraBio And Others