Niemann-Pick Disease Type C (NPC) - Market Insights, Epidemiology and Market Forecast-2027

  • ID: 4564657
  • Drug Pipelines
  • 132 pages
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‘Niemann-Pick Disease Type C (NPC) - Market Insights, Epidemiology and Market Forecast-2027’ report delivers an in-depth understanding of the disease, historical & forecasted epidemiology as well as the market trends of Niemann-Pick Disease Type C (NPC) in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom), and Japan.

The Report provides the current treatment practices, emerging drugs having the potential to get launched in the forecast period, market share of the individual therapies, current and forecasted market size of Niemann-Pick Disease Type C (NPC) from 2016 to 2027 segmented by seven major markets. The Report also covers current treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market.

Geography Covered:
  • The United States
  • EU5 (Germany, France, Italy, Spain and the United Kingdom)
  • Japan
Study Period: 2016-2027

Niemann-Pick Disease Type C (NPC) - Disease Understanding and Treatment Algorithm

The Niemann-Pick Disease Type C (NPC) market report gives the thorough understanding of the Niemann-Pick Disease Type C (NPC) by including details such as disease definition, classification, symptoms, etiology, pathophysiology, diagnostic trends. It also provides treatment algorithms and treatment guidelines for Niemann-Pick Disease Type C (NPC) in the US, Europe, and Japan.

Niemann-Pick Disease Type C (NPC) Epidemiology

The Niemann-Pick Disease Type C (NPC) epidemiology division provide the insights about historical and current patient pool and forecasted trend for every 7 major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the diagnosed and treatable patient pool and their trends along with assumptions undertaken.

The disease epidemiology covered in the report is segmented by total prevalent patient population, diagnosed population, sub-types specific cases (NPC1 and NPC 2) and clinical distribution of NPC by phenotypes (early infantile, late infantile, juvenile and adult).

According to this research, the total prevalent population of NPC was found to be 2,099 in the year 2016 in 7MM.

Niemann-Pick Disease Type C (NPC) Drug Chapters

This segment of the Niemann-Pick Disease Type C (NPC) report encloses the detailed analysis of marketed drugs and late stage (Phase-III) pipeline drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

The drugs marketed for the treatment mainly includes Substrate Reduction Therapy - Zavesca [Miglustat; Actelion] and Supportive Therapies for the management of symptoms associated with NPC - Antiepileptics (Seizures); Tricyclic Antidepressants & CNS Stimulants (Cataplexy); Anticholinergics, Trihexyphenydil & GABA Derivatives (Dystonia & Tremor); atypical antipsychotics (Psychosis). Detailed chapters for all of these drugs, along with the upcoming therapies i.e., [VTS-270 (Mallinckrodt), Arimoclomol (Orphazyme), and Trappsol Cyclo (CTD Holdings, Inc.) have been covered in the report.

Niemann-Pick Disease Type C (NPC) Market Outlook

The Niemann-Pick Disease Type C (NPC) market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.

This segment gives a through detail of market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to this research, the global market of Niemann-Pick Disease Type C (NPC) was estimated to be USD 27.61 billion in 2016.

Niemann-Pick Disease Type C (NPC) Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2016-2027. The analysis covers market uptake by drugs; patient uptake by therapies and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

The market size of Niemann-Pick Disease Type C (NPC) includes the drug-uptake for individual therapies, which is further segmented on the basis of market size and market share by Line of therapy and Route of Administration.

Niemann-Pick Disease Type C (NPC) Report Insights
  • Patient Population
  • Therapeutic Approaches
  • Pipeline Analysis
  • Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies
Niemann-Pick Disease Type C (NPC) Report Key Strengths
  • 10 Year Forecast
  • 7MM Coverage
  • Epidemiology Segmentation
  • Drugs Uptake
  • Highly Analyzed Market
  • Key Cross Competition
Niemann-Pick Disease Type C (NPC) Report Assessment
  • Current Treatment Practices
  • Unmet Needs
  • Detailed Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers
Key Benefits:
  • This report will help to develop Business Strategies by understanding the trends shaping and driving the Niemann-Pick Disease Type C (NPC) market
  • Organize sales and marketing efforts by identifying the best opportunities for Niemann-Pick Disease Type C (NPC) market
  • To understand the future market competition in the Niemann-Pick Disease Type C (NPC) market
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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 2016
2.2. Market Share (%) Distribution of Niemann-Pick Disease Type C (NPC) in 2027

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. Inheritance patterns
3.5. Clinical Manifestations
3.6. Pathophysiology
3.7. Diagnosis
3.7.1. NP-C Diagnostic Recommendations Expert Panel: Diagnostic Recommendations (2016)
3.7.2. NP-C 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

5. Country Wise-Epidemiology of NPC
5.1. United States
5.1.1. Total Prevalent Patient Population of NPC in the United States
5.1.2. Sub-type Specific Prevalence of NPC in the United States
5.1.3. Diagnosed Population of NPC in the United States
5.1.4. Clinical Distribution of NPC by Phenotypes
5.2. EU5 Countries
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. Clinical Distribution of NPC by 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. Clinical Distribution of NPC by 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. Clinical Distribution of NPC by 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. Clinical Distribution of NPC by 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 UK
5.7.4. Clinical Distribution of NPC by Phenotypes
5.8. Japan
5.8.1. Total Prevalent Patient Population of NPC in Japan
5.8.2. Sub-type Specific Prevalence of NPC in Japan
5.8.3. Diagnosed Population of NPC in Japan
5.8.4. Clinical Distribution of NPC by Phenotypes

6. Treatment Practices
6.1. Treatment Goals
6.2. Treatment Algorithm
6.3. NP-C Guidelines Working Group: Treatment Recommendations

7. Unmet Needs

8. Marketed drugs
8.1. Zavesca: Actelion Pharmaceuticals Ltd
8.1.1. Drug description
8.1.2. Product profile
8.1.3. Regulatory milestones
8.1.4. Generic Availability
8.1.5. Safety and efficacy

9. Emerging drugs
9.1. Emerging Drugs Analysis
9.2. Arimoclomol: Orphazyme
9.2.1. Drug Description
9.2.2. Product Profile
9.2.3. Regulatory Milestones
9.2.4. Clinical Development
9.2.5. Safety and Efficacy
9.3. VTS-270: Vtesse Inc. (Mallinckrodt)
9.3.1. Drug Description
9.3.2. Product Profile
9.3.3. Regulatory Milestones
9.3.4. Clinical Development
9.3.5. Safety and Efficacy
9.4. Trappsol Cyclo (cyclodextrin): CTD Holdings, Inc.
9.4.1. Drug Description
9.4.2. Product Profile
9.4.3. Regulatory Milestones
9.4.4. Clinical Development
9.4.5. VTS-270 vs Trappsol Cyclo
9.4.6. Safety and Efficacy

10. Niemann Pick Disease Type C: 7 Major Market Analysis
10.1.1. Key Findings
10.1.2. Total Market Size of Niemann Pick Disease Type C in 7MM
10.1.3. Market Size by Therapeutic Class in 7MM
10.1.4. Market Size by Therapies in 7MM

11. Market Outlook by Country

12. The United States: Market Outlook
12.1. United States Market Size
12.1.1. Total Market size of Niemann Pick Disease Type C [NPC]
12.1.2. Niemann Pick Disease Type C Market by Therapeutic Class
12.1.3. Market Size by Therapies

13. EU-5 Countries: Market Outlook
13.1. Germany
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
13.2. France
13.2.1. Total Market size of Niemann Pick Disease Type C
13.2.2. Niemann Pick Disease Type C Market by Therapeutic Class
13.2.3. Market Size by Therapies
13.3. Italy
13.3.1. Total Market size of Niemann Pick Disease Type C
13.3.2. Niemann Pick Disease Type C Market by Therapeutic Class
13.3.3. Market Size by Therapies
13.4. Spain
13.4.1. Total Market size of Niemann Pick Disease Type C
13.4.2. Niemann Pick Disease Type C Market by Therapeutic Class
13.4.3. Market Size by Therapies
13.5. United Kingdom
13.5.1. Total Market size of Niemann Pick Disease Type C
13.5.2. Niemann Pick Disease Type C Market by Therapeutic Class
13.5.3. Market Size by Therapies

14. Japan: Market Outlook
14.1.1. Total Market size of Niemann Pick Disease Type C
14.1.2. Niemann Pick Disease Type C Market by Therapeutic Class
14.1.3. Market Size by Therapies

15. Market Drivers

16. Market Barriers

17. Appendix
17.1. Report Methodology

18. Capabilities

19. Disclaimer

20. About the Publisher

List of Tables
Table 1: Classification of signs and symptoms in NPC
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 (2016-2027)
Table 10: Total Prevalent Population of NPC in the United States (2016-2027)
Table 11: Sub-type Specific Prevalence of NPC in the United States (2016-2027)
Table 12: Diagnosed Population of NPC in the United States (2016-2027)
Table 13: Clinical distribution of NPC by Phenotypes of NPC in the United States (2016-2027)
Table 14: Total Prevalent Population of NPC in Germany (2016-2027)
Table 15: Sub-type Specific Prevalence of NPC in Germany (2016-2027)
Table 16: Diagnosed Population of NPC in Germany (2016-2027)
Table 17: Clinical distribution of NPC by Phenotypes in Germany (2016-2027)
Table 18: Total Prevalent Population of NPC in France (2016-2027)
Table 19: Sub-type Specific Prevalence of NPC in France (2016-2027)
Table 20: Diagnosed Population of NPC in France (2016-2027)
Table 21: Clinical distribution of NPC by Phenotypes in France (2016-2027)
Table 22: Total Prevalent Population of NPC in Italy (2016-2027)
Table 23: Sub-type Specific Prevalence of NPC in Italy (2016-2027)
Table 24: Diagnosed Population of NPC in Italy (2016-2027)
Table 25: Clinical distribution of NPC by Phenotypes in Italy (2016-2027)
Table 26: Total Prevalent Population of NPC in Spain (2016-2027)
Table 27: Sub-type Specific Prevalence of NPC in Spain (2016-2027)
Table 28: Diagnosed Population of NPC in Spain (2016-2027)
Table 29: Clinical distribution of NPC by Phenotypes in Spain (2016-2027)
Table 30: Total Prevalent Population of NPC in the UK (2016-2027)
Table 31: Sub-type Specific Prevalence of NPC in the UK (2016-2027)
Table 32: Diagnosed Population of NPC in the UK (2016-2027)
Table 33: Clinical distribution of NPC by Phenotypes in the UK (2016-2027)
Table 34: Total Prevalent Population of NPC in Japan (2016-2027)
Table 35: Sub-type Specific Prevalence of NPC in Japan (2016-2027)
Table 36: Diagnosed Population of NPC in Japan (2016-2027)
Table 37: Clinical distribution of NPC by Phenotypes in Japan (2016-2027)
Table 38: Symptomatic Therapies for Niemann-Pick Disease type C
Table 39: Recommended Miglustat dose alteration according to body surface area
Table 40: Recommendations for Symptomatic Therapies
Table 41: Emerging Drugs Analysis
Table 42: Arimoclomol, Clinical Trial Description, 2018
Table 43: Arimoclomol, Clinical Trial Description, 2018
Table 44: Trappsol Cyclo, Clinical Trial Description, 2018
Table 45: 7 Major Market Size of Niemann Pick Disease Type C in USD Million (2016-2027)
Table 46: 7MM Market Size of NPC by Therapeutic Class in USD Million (2016-2027)
Table 47 Market size of Niemann Pick Disease Type C by Therapies in 7MM, USD Million (2016-2027)
Table 48: The US Market Niemann Pick Disease Type C in USD Million (2016-2027)
Table 49: The U.S. NPC Market by Therapeutic Class in USD Million (2016-2027)
Table 50: The US NPC market size by Therapies in USD Million (2016-2027)
Table 51: Market Size of NPC in Germany, in USD Million (2016-2027)
Table 52: Germany NPC Market by Therapeutic Class in USD Million (2016-2027)
Table 53: Germany NPC market size by Therapies in USD Million (2016-2027)
Table 54: Market Size of Niemann Pick Disease Type C in France, in USD Million (2016-2027)
Table 55: France NPC Market by Therapeutic Class in USD Million (2016-2027)
Table 56: France NPC market size by Therapies in USD Million (2016-2027)
Table 57: Market Size of Niemann Pick Disease Type C in Italy, in USD Million (2016-2027)
Table 58: Italy NPC Market by Therapeutic Class in USD Million (2016-2027)
Table 59: Italy NPC market size by Therapies in USD Million (2016-2027)
Table 60: Market Size of Niemann Pick Disease Type C in Spain, in USD Million (2016-2027)
Table 61: Spain NPC Market by Therapeutic Class in USD Million (2016-2027)
Table 62: Spain NPC market size by Therapies in USD Million (2016-2027)
Table 63: Market Size of Niemann Pick Disease Type C in the UK, in USD Million (2016-2027)
Table 64: The UK NPC Market by Therapeutic Class in USD Million (2016-2027)
Table 65: Emerging Therapies market of Niemann Pick Disease Type C in the UK, in USD Million (2016-2027)
Table 66: Market Size of Niemann Pick Disease Type C in Japan, in USD Million (2016-2027)
Table 67: Japan Niemann Pick Disease Type C Market by Therapeutic Class in USD Million (2016-2027)
Table 68: Japan NPC market size by Therapies in USD Million (2016-2027)

List of Figures
Figure 1: Classification of Niemann Pick Disease
Figure 2: Classification of Niemann-Pick Disease Type C
Figure 3: Inheritance Pattern of Niemann-Pick Disease Type C (NPC)
Figure 4: Manifestations of Niemann-Pick Disease Type C (NP-C) by age group
Figure 5: Schematic representation of Niemann-Pick Disease Type C
Figure 6: Pathophysiology of Niemann-Pick Disease Type C 1 Disease
Figure 7: Pathophysiology of Niemann-Pick Disease Type C 2 Disease
Figure 8: Diagnostic Algorithm for Niemann-Pick Disease Type C
Figure 9: Total Prevalent Patient Population of NPC in 7MM (2016-2027)
Figure 10: Prevalence of NPC in the United States (2016-2027)
Figure 11: Sub-type Specific Prevalence of NPC in the United States (2016-2027)
Figure 12: Diagnosed Population of NPC in the United States (2016-2027)
Figure 13: Clinical distribution of NPC by Phenotypes in the United States (2016-2027)
Figure 14: Total Prevalent Population of NPC in Germany (2016-2027)
Figure 15: Sub-type Specific Prevalence of NPC in Germany (2016-2027)
Figure 16: Diagnosed Population of NPC in Germany (2016-2027)
Figure 17: Clinical distribution of NPC by Phenotypes in Germany (2016-2027)
Figure 18: Total Prevalent Population of NPC in France (2016-2027)
Figure 19: Sub-type Specific Prevalence of NPC in France (2016-2027)
Figure 20: Diagnosed Population of NPC in France (2016-2027)
Figure 21: Clinical distribution of NPC by Phenotypes in France (2016-2027)
Figure 22: Total Prevalent Population of NPC in Italy (2016-2027)
Figure 23: Sub-type Specific Prevalence of NPC in Italy (2016-2027)
Figure 24: Diagnosed Population of NPC in Italy (2016-2027)
Figure 25: Clinical distribution of NPC by Phenotypes in Italy (2016-2027)
Figure 26: Total Prevalent Population of NPC in Spain (2016-2027)
Figure 27: Sub-type Specific Prevalence of NPC in Spain (2016-2027)
Figure 28: Diagnosed Population of NPC in Spain (2016-2027)
Figure 29: Clinical distribution of NPC by Phenotypes in Spain (2016-2027)
Figure 30: Total Prevalent Population of NPC in the UK (2016-2027)
Figure 31: Sub-type Specific Prevalence of NPC in the UK (2016-2027)
Figure 32: Diagnosed Population of NPC in the UK (2016-2027)
Figure 33: Clinical distribution of NPC by Phenotypes in the UK (2016-2027)
Figure 34: Total Prevalent Population of NPC in Japan (2016-2027)
Figure 35: Sub-type Specific Prevalence of NPC in Japan (2016-2027)
Figure 36: Diagnosed Population of NPC in Japan (2016-2027)
Figure 37: Clinical distribution of NPC by Phenotypes in Japan (2016-2027)
Figure 38: Treatment Algorithm for Niemann-Pick Disease Type C (NPC)
Figure 39: Unmet Needs for Niemann-Pick Disease Type C (NPC)
Figure 40: 7 Major Market Size of Niemann Pick Disease Type C in USD Million (2016-2027)
Figure 41: 7MM Market Size of NPC by Therapeutic Class in USD Million (2016-2027)
Figure 42: Market size of Niemann Pick Disease Type C by Therapies in 7MM, USD Million (2016-2027)
Figure 43: Market Size of NPC in the United States, USD Millions (2016-2027)
Figure 44: The U.S. NPC Market by Therapeutic Class in USD Million (2016-2027)
Figure 45: The U.S. market size of NPC by therapies in USD Million (2016-2027)
Figure 46: Market Size of NPC in Germany, USD Millions (2016-2027)
Figure 47: Germany NPC Market by Therapeutic Class in USD Million (2016-2027)
Figure 48: Germany NPC market size by Therapies in USD Million (2016-2027)
Figure 49: Market Size of Niemann Pick Disease Type C in France, USD Millions (2016-2027)
Figure 50: France NPC Market by Therapeutic Class in USD Million (2016-2027)
Figure 51: France NPC market size by Therapies in USD Million (2016-2027)
Figure 52: Market Size of Niemann Pick Disease Type C in Italy, USD Millions (2016-2027)
Figure 53: Italy NPC Market by Therapeutic Class in USD Million (2016-2027)
Figure 54: Italy NPC market size by Therapies in USD Million (2016-2027)
Figure 55: Market Size of Niemann Pick Disease Type C in Spain, USD Millions (2016-2027)
Figure 56: Spain NPC Market by Therapeutic Class in USD Million (2016-2027)
Figure 57: Spain NPC market size by Therapies in USD Million (2016-2027)
Figure 58: Market Size of Niemann Pick Disease Type C in the UK, USD Millions (2016-2027)
Figure 59: The UK NPC Market by Therapeutic Class in USD Million (2016-2027)
Figure 60: The UK NPC market size by Therapies in USD Million (2016-2027)
Figure 61: Market Size of Niemann Pick Disease Type C in Japan, USD Millions (2016-2027)
Figure 62: Japan NPC Market by Therapeutic Class in USD Million (2016-2027)
Figure 63: Japan NPC market size by Therapies in USD Million (2016-2027)
Figure 64: Market Drivers for Niemann-Pick Disease Type C (NPC)
Figure 65: Market Barriers for Niemann–Pick Type C Disease
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  • Actelion Pharmaceuticals Ltd
  • Orphazyme
  • Vtesse Inc. (Mallinckrodt)
  • CTD Holdings, Inc.
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