Niemann-Pick Disease Type C Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034
The 7 major Niemann-Pick disease type C markets are expected to exhibit a CAGR of 16.9% during 2024-2034.
The Niemann-Pick disease type C market has been comprehensively analyzed in IMARC's new report titled "Niemann-Pick Disease Type C Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Niemann-Pick disease type C (NPC) refers to a rare and progressive genetic disorder that primarily affects the body's capability to metabolize cholesterol and other lipids within cells. It falls under the broader category of lysosomal storage disorders. The symptoms of the ailment can manifest in infancy, childhood, or even adulthood, making diagnosis and management complex. Some early signs may include delayed motor development, difficulty swallowing, and an enlarged liver or spleen. As the disease advances, individuals suffering from this condition might also experience impaired cognition, muscle stiffness, and loss of coordination. These symptoms arise due to the accumulation of lipids within the lysosomes of various organs, including the brain, liver, and lungs, leading to their dysfunction. The diagnosis of NPC is often challenging due to its variable presentation and the similarity of its indications to those of various other disorders. The healthcare provider may perform clinical evaluations, neurological assessments, imaging studies, and genetic testing to confirm the diagnosis.
The increasing cases of mutations in the NPC1 and NPC2 genes, resulting in impaired intracellular lipid transport and subsequent accumulation of lipids, mainly cholesterol and glycosphingolipids, within various organs, are primarily driving the Niemann-Pick disease type C market. In addition to this, the inflating utilization of supportive treatments, including anti-seizure medications and mobility aids, aimed at enhancing the quality of life for individuals suffering from the ailment is also creating a positive outlook for the market. Moreover, the widespread adoption of miglustat, a substrate reduction therapy that targets to impede the synthesis of lipids and slow disease progression, is further bolstering the market growth. Apart from this, the rising usage of physical, speech, and occupational therapies, since they assist in enhancing muscle tone, improving speech quality, and promoting fine motor skills, is acting as another significant growth-inducing factor. Additionally, the escalating application of cyclodextrin-based therapies to facilitate the removal of cholesterol accumulations from cells, thereby restoring a normal cellular environment, is also augmenting the market growth. Furthermore, the emerging popularity of gene therapy, a cutting-edge avenue involving the introduction of effective genetic sequences into cells to address or amend the defective genes underpinning the disorder, is expected to drive the Niemann-Pick disease type C market during the forecast period.
IMARC Group's new report provides an exhaustive analysis of the Niemann-Pick disease type C market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for Niemann-Pick disease type C and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Niemann-Pick disease type C market in any manner.
Time Period of the Study
Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034
Countries Covered
United States
Germany
France
United Kingdom
Italy
Spain
Japan
Analysis Covered Across Each Country
Historical, current, and future epidemiology scenario
Historical, current, and future performance of the Niemann-Pick disease type C market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Niemann-Pick disease type C market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current Niemann-Pick disease type C marketed drugs and late-stage pipeline drugs.
In-Market Drugs
Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance
Late-Stage Pipeline Drugs
Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance
*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.
Key Questions Answered in this Report:
Market Insights
How has the Niemann-Pick disease type C market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the Niemann-Pick disease type C market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the Niemann-Pick disease type C market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?
Epidemiology Insights
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease type C across the seven major markets?
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease type C by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease type C by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with Niemann-Pick disease type C across the seven major markets?
What is the size of the Niemann-Pick disease type C patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of Niemann-Pick disease type C?
What will be the growth rate of patients across the seven major markets?
Niemann-Pick Disease Type C: Current Treatment Scenario, Marketed Drugs and Emerging Therapies
What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for Niemann-Pick disease type C drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Niemann-Pick disease type C market?
What are the key regulatory events related to the Niemann-Pick disease type C market?
What is the structure of clinical trial landscape by status related to the Niemann-Pick disease type C market?
What is the structure of clinical trial landscape by phase related to the Niemann-Pick disease type C market?
What is the structure of clinical trial landscape by route of administration related to the Niemann-Pick disease type C market?
The Niemann-Pick disease type C market has been comprehensively analyzed in IMARC's new report titled "Niemann-Pick Disease Type C Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Niemann-Pick disease type C (NPC) refers to a rare and progressive genetic disorder that primarily affects the body's capability to metabolize cholesterol and other lipids within cells. It falls under the broader category of lysosomal storage disorders. The symptoms of the ailment can manifest in infancy, childhood, or even adulthood, making diagnosis and management complex. Some early signs may include delayed motor development, difficulty swallowing, and an enlarged liver or spleen. As the disease advances, individuals suffering from this condition might also experience impaired cognition, muscle stiffness, and loss of coordination. These symptoms arise due to the accumulation of lipids within the lysosomes of various organs, including the brain, liver, and lungs, leading to their dysfunction. The diagnosis of NPC is often challenging due to its variable presentation and the similarity of its indications to those of various other disorders. The healthcare provider may perform clinical evaluations, neurological assessments, imaging studies, and genetic testing to confirm the diagnosis.
The increasing cases of mutations in the NPC1 and NPC2 genes, resulting in impaired intracellular lipid transport and subsequent accumulation of lipids, mainly cholesterol and glycosphingolipids, within various organs, are primarily driving the Niemann-Pick disease type C market. In addition to this, the inflating utilization of supportive treatments, including anti-seizure medications and mobility aids, aimed at enhancing the quality of life for individuals suffering from the ailment is also creating a positive outlook for the market. Moreover, the widespread adoption of miglustat, a substrate reduction therapy that targets to impede the synthesis of lipids and slow disease progression, is further bolstering the market growth. Apart from this, the rising usage of physical, speech, and occupational therapies, since they assist in enhancing muscle tone, improving speech quality, and promoting fine motor skills, is acting as another significant growth-inducing factor. Additionally, the escalating application of cyclodextrin-based therapies to facilitate the removal of cholesterol accumulations from cells, thereby restoring a normal cellular environment, is also augmenting the market growth. Furthermore, the emerging popularity of gene therapy, a cutting-edge avenue involving the introduction of effective genetic sequences into cells to address or amend the defective genes underpinning the disorder, is expected to drive the Niemann-Pick disease type C market during the forecast period.
IMARC Group's new report provides an exhaustive analysis of the Niemann-Pick disease type C market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for Niemann-Pick disease type C and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Niemann-Pick disease type C market in any manner.
Time Period of the Study
Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034
Countries Covered
United States
Germany
France
United Kingdom
Italy
Spain
Japan
Analysis Covered Across Each Country
Historical, current, and future epidemiology scenario
Historical, current, and future performance of the Niemann-Pick disease type C market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Niemann-Pick disease type C market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current Niemann-Pick disease type C marketed drugs and late-stage pipeline drugs.
In-Market Drugs
Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance
Late-Stage Pipeline Drugs
Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance
*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.
Key Questions Answered in this Report:
Market Insights
How has the Niemann-Pick disease type C market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the Niemann-Pick disease type C market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the Niemann-Pick disease type C market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?
Epidemiology Insights
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease type C across the seven major markets?
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease type C by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease type C by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with Niemann-Pick disease type C across the seven major markets?
What is the size of the Niemann-Pick disease type C patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of Niemann-Pick disease type C?
What will be the growth rate of patients across the seven major markets?
Niemann-Pick Disease Type C: Current Treatment Scenario, Marketed Drugs and Emerging Therapies
What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for Niemann-Pick disease type C drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Niemann-Pick disease type C market?
What are the key regulatory events related to the Niemann-Pick disease type C market?
What is the structure of clinical trial landscape by status related to the Niemann-Pick disease type C market?
What is the structure of clinical trial landscape by phase related to the Niemann-Pick disease type C market?
What is the structure of clinical trial landscape by route of administration related to the Niemann-Pick disease type C market?
1 PREFACE
2 SCOPE AND METHODOLOGY
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology
3 EXECUTIVE SUMMARY
4 NIEMANN-PICK DISEASE TYPE C - INTRODUCTION
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence
5 NIEMANN-PICK DISEASE TYPE C - DISEASE OVERVIEW
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment
6 PATIENT JOURNEY
7 NIEMANN-PICK DISEASE TYPE C - EPIDEMIOLOGY AND PATIENT POPULATION
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (2018-2034)
7.2.4 Epidemiology by Gender (2018-2034)
7.2.5 Diagnosed Cases (2018-2034)
7.2.6 Patient Pool/Treated Cases (2018-2034)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (2018-2034)
7.3.4 Epidemiology by Gender (2018-2034)
7.3.5 Diagnosed Cases (2018-2034)
7.3.6 Patient Pool/Treated Cases (2018-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (2018-2034)
7.4.4 Epidemiology by Gender (2018-2034)
7.4.5 Diagnosed Cases (2018-2034)
7.4.6 Patient Pool/Treated Cases (2018-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (2018-2034)
7.5.4 Epidemiology by Gender (2018-2034)
7.5.5 Diagnosed Cases (2018-2034)
7.5.6 Patient Pool/Treated Cases (2018-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (2018-2034)
7.6.4 Epidemiology by Gender (2018-2034)
7.6.5 Diagnosed Cases (2018-2034)
7.6.6 Patient Pool/Treated Cases (2018-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (2018-2034)
7.7.4 Epidemiology by Gender (2018-2034)
7.7.5 Diagnosed Cases (2018-2034)
7.7.6 Patient Pool/Treated Cases (2018-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (2018-2034)
7.8.4 Epidemiology by Gender (2018-2034)
7.8.5 Diagnosed Cases (2018-2034)
7.8.6 Patient Pool/Treated Cases (2018-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (2018-2034)
7.9.4 Epidemiology by Gender (2018-2034)
7.9.5 Diagnosed Cases (2018-2034)
7.9.6 Patient Pool/Treated Cases (2018-2034)
8 NIEMANN-PICK DISEASE TYPE C - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 NIEMANN-PICK DISEASE TYPE C - UNMET NEEDS
10 NIEMANN-PICK DISEASE TYPE C - KEY ENDPOINTS OF TREATMENT
11 NIEMANN-PICK DISEASE TYPE C - MARKETED PRODUCTS
11.1 List of Niemann-Pick Disease Type C Marketed Drugs Across the Top 7 Markets
11.1.1 Zavesca (Miglustat) - Actelion Pharmaceuticals
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets
Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.
12 NIEMANN-PICK DISEASE TYPE C - PIPELINE DRUGS
12.1 List of Niemann-Pick Disease Type C Pipeline Drugs Across the Top 7 Markets
12.1.1 Trappsol Cyclo - Cyclo Therapeutics
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 VTS-270 - Mandos
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status
12.1.3 AZ-3102 - Azafaros
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status
12.1.4 Arimoclomol - Zevra Therapeutics
12.1.4.1 Drug Overview
12.1.4.2 Mechanism of Action
12.1.4.3 Clinical Trial Results
12.1.4.4 Safety and Efficacy
12.1.4.5 Regulatory Status
12.1.5 Adrabetadex - Mandos
12.1.5.1 Drug Overview
12.1.5.2 Mechanism of Action
12.1.5.3 Clinical Trial Results
12.1.5.4 Safety and Efficacy
12.1.5.5 Regulatory Status
Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.
13 NIEMANN-PICK DISEASE TYPE C - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS
14 NIEMANN-PICK DISEASE TYPE C – CLINICAL TRIAL LANDSCAPE
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events
15 NIEMANN-PICK DISEASE TYPE C - MARKET SCENARIO
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Niemann-Pick Disease Type C - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.3 Market Scenario - United States
15.3.1 Niemann-Pick Disease Type C - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Niemann-Pick Disease Type C - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapies (2024-2034)
15.4.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Niemann-Pick Disease Type C - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Niemann-Pick Disease Type C - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies (2024-2034)
15.6.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Niemann-Pick Disease Type C - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Niemann-Pick Disease Type C - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Niemann-Pick Disease Type C - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023)
15.9.2.2 Market Forecast by Therapies (2024-2034)
15.9.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
16 NIEMANN-PICK DISEASE TYPE C - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS
17 NIEMANN-PICK DISEASE TYPE C MARKET - SWOT ANALYSIS
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 NIEMANN-PICK DISEASE TYPE C MARKET – STRATEGIC RECOMMENDATIONS
19 APPENDIX
2 SCOPE AND METHODOLOGY
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology
3 EXECUTIVE SUMMARY
4 NIEMANN-PICK DISEASE TYPE C - INTRODUCTION
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence
5 NIEMANN-PICK DISEASE TYPE C - DISEASE OVERVIEW
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment
6 PATIENT JOURNEY
7 NIEMANN-PICK DISEASE TYPE C - EPIDEMIOLOGY AND PATIENT POPULATION
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (2018-2034)
7.2.4 Epidemiology by Gender (2018-2034)
7.2.5 Diagnosed Cases (2018-2034)
7.2.6 Patient Pool/Treated Cases (2018-2034)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (2018-2034)
7.3.4 Epidemiology by Gender (2018-2034)
7.3.5 Diagnosed Cases (2018-2034)
7.3.6 Patient Pool/Treated Cases (2018-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (2018-2034)
7.4.4 Epidemiology by Gender (2018-2034)
7.4.5 Diagnosed Cases (2018-2034)
7.4.6 Patient Pool/Treated Cases (2018-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (2018-2034)
7.5.4 Epidemiology by Gender (2018-2034)
7.5.5 Diagnosed Cases (2018-2034)
7.5.6 Patient Pool/Treated Cases (2018-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (2018-2034)
7.6.4 Epidemiology by Gender (2018-2034)
7.6.5 Diagnosed Cases (2018-2034)
7.6.6 Patient Pool/Treated Cases (2018-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (2018-2034)
7.7.4 Epidemiology by Gender (2018-2034)
7.7.5 Diagnosed Cases (2018-2034)
7.7.6 Patient Pool/Treated Cases (2018-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (2018-2034)
7.8.4 Epidemiology by Gender (2018-2034)
7.8.5 Diagnosed Cases (2018-2034)
7.8.6 Patient Pool/Treated Cases (2018-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (2018-2034)
7.9.4 Epidemiology by Gender (2018-2034)
7.9.5 Diagnosed Cases (2018-2034)
7.9.6 Patient Pool/Treated Cases (2018-2034)
8 NIEMANN-PICK DISEASE TYPE C - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 NIEMANN-PICK DISEASE TYPE C - UNMET NEEDS
10 NIEMANN-PICK DISEASE TYPE C - KEY ENDPOINTS OF TREATMENT
11 NIEMANN-PICK DISEASE TYPE C - MARKETED PRODUCTS
11.1 List of Niemann-Pick Disease Type C Marketed Drugs Across the Top 7 Markets
11.1.1 Zavesca (Miglustat) - Actelion Pharmaceuticals
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets
Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.
12 NIEMANN-PICK DISEASE TYPE C - PIPELINE DRUGS
12.1 List of Niemann-Pick Disease Type C Pipeline Drugs Across the Top 7 Markets
12.1.1 Trappsol Cyclo - Cyclo Therapeutics
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 VTS-270 - Mandos
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status
12.1.3 AZ-3102 - Azafaros
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status
12.1.4 Arimoclomol - Zevra Therapeutics
12.1.4.1 Drug Overview
12.1.4.2 Mechanism of Action
12.1.4.3 Clinical Trial Results
12.1.4.4 Safety and Efficacy
12.1.4.5 Regulatory Status
12.1.5 Adrabetadex - Mandos
12.1.5.1 Drug Overview
12.1.5.2 Mechanism of Action
12.1.5.3 Clinical Trial Results
12.1.5.4 Safety and Efficacy
12.1.5.5 Regulatory Status
Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.
13 NIEMANN-PICK DISEASE TYPE C - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS
14 NIEMANN-PICK DISEASE TYPE C – CLINICAL TRIAL LANDSCAPE
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events
15 NIEMANN-PICK DISEASE TYPE C - MARKET SCENARIO
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Niemann-Pick Disease Type C - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.3 Market Scenario - United States
15.3.1 Niemann-Pick Disease Type C - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Niemann-Pick Disease Type C - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapies (2024-2034)
15.4.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Niemann-Pick Disease Type C - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Niemann-Pick Disease Type C - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies (2024-2034)
15.6.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Niemann-Pick Disease Type C - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Niemann-Pick Disease Type C - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Niemann-Pick Disease Type C - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023)
15.9.2.2 Market Forecast by Therapies (2024-2034)
15.9.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
16 NIEMANN-PICK DISEASE TYPE C - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS
17 NIEMANN-PICK DISEASE TYPE C MARKET - SWOT ANALYSIS
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 NIEMANN-PICK DISEASE TYPE C MARKET – STRATEGIC RECOMMENDATIONS
19 APPENDIX
