Niemann-Pick Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034
The 7 major Niemann-Pick disease markets are expected to exhibit a CAGR of 4.22% during 2024-2034.
The Niemann-Pick disease market has been comprehensively analyzed in IMARC's new report titled "Niemann-Pick Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Niemann-Pick disease refers to a rare genetic disorder that affects the body's ability to metabolize fat and cholesterol. The condition results in the accumulation of lipids in abnormal amounts due to a deficiency or absence of an enzyme called acid sphingomyelinase (ASM), which is responsible for breaking down fatty substances. There are several types of Niemann-Pick disease, with varying severity and age of onset. Types A and B are the most common forms of this illness, which primarily affect the liver and spleen and may cause enlargement of these organs, anemia, and low platelet counts. Type C is a more severe form of the disease, affecting the liver, spleen and brain and causing developmental delays, seizures, difficulty with movement, and cognitive impairment. The diagnosis of Niemann-Pick disease is typically made after a comprehensive review of the patient's indications, medical history, and physical examination. The healthcare provider may also perform a blood or skin sample biopsy to measure the amount of sphingomyelinase in white blood cells and confirm disease diagnosis.
The increasing prevalence of autosomal recessive inherited disorders, that result in genetic mutations, causing a buildup of cholesterol and other fats within the body cells, is primarily driving the Niemann-Pick disease market. Furthermore, the widespread utilization of enzyme inhibitors, involving miglustat, which work by inhibiting the body from generating fatty substances and their abnormal accumulation in the organs, is also bolstering the market growth. Additionally, several government agencies and non-governmental organizations are funding health education programs to raise awareness about early diagnosis and available treatment options for Niemann-Pick disease. This, in turn, is creating a positive outlook for the market. Moreover, the inflating application of enzyme replacement therapy, since it aims to replace defective or deficient ASM enzymes, thereby minimizing fat accumulation, providing symptom relief, and enhancing overall well-being in patients, is further acting as another significant growth-inducing factor. Apart from this, the emerging popularity of gene therapy, that involves introducing a functional copy of the faulty gene into the body to treat this ailment, is expected to drive the Niemann-Pick disease market in the coming years.
IMARC Group's new report provides an exhaustive analysis of the Niemann-Pick disease 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 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 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 market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Niemann-Pick disease market
Reimbursement scenario in the market
In-market and pipeline drug
Competitive Landscape:
This report also provides a detailed analysis of the current Niemann-Pick disease 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 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 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 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 across the seven major markets?
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease by gender across the seven major markets?
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease by type across the seven major markets?
How many patients are diagnosed (2018-2034) with Niemann-Pick disease across the seven major markets?
What is the size of the Niemann-Pick disease 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?
What will be the growth rate of patients across the seven major markets?
Niemann-Pick Disease: 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 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 market?
What are the key regulatory events related to the Niemann-Pick disease market?
What is the structure of clinical trial landscape by status related to the Niemann-Pick disease market?
What is the structure of clinical trial landscape by phase related to the Niemann-Pick disease market?
What is the structure of clinical trial landscape by route of administration related to the Niemann-Pick disease market?
The Niemann-Pick disease market has been comprehensively analyzed in IMARC's new report titled "Niemann-Pick Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Niemann-Pick disease refers to a rare genetic disorder that affects the body's ability to metabolize fat and cholesterol. The condition results in the accumulation of lipids in abnormal amounts due to a deficiency or absence of an enzyme called acid sphingomyelinase (ASM), which is responsible for breaking down fatty substances. There are several types of Niemann-Pick disease, with varying severity and age of onset. Types A and B are the most common forms of this illness, which primarily affect the liver and spleen and may cause enlargement of these organs, anemia, and low platelet counts. Type C is a more severe form of the disease, affecting the liver, spleen and brain and causing developmental delays, seizures, difficulty with movement, and cognitive impairment. The diagnosis of Niemann-Pick disease is typically made after a comprehensive review of the patient's indications, medical history, and physical examination. The healthcare provider may also perform a blood or skin sample biopsy to measure the amount of sphingomyelinase in white blood cells and confirm disease diagnosis.
The increasing prevalence of autosomal recessive inherited disorders, that result in genetic mutations, causing a buildup of cholesterol and other fats within the body cells, is primarily driving the Niemann-Pick disease market. Furthermore, the widespread utilization of enzyme inhibitors, involving miglustat, which work by inhibiting the body from generating fatty substances and their abnormal accumulation in the organs, is also bolstering the market growth. Additionally, several government agencies and non-governmental organizations are funding health education programs to raise awareness about early diagnosis and available treatment options for Niemann-Pick disease. This, in turn, is creating a positive outlook for the market. Moreover, the inflating application of enzyme replacement therapy, since it aims to replace defective or deficient ASM enzymes, thereby minimizing fat accumulation, providing symptom relief, and enhancing overall well-being in patients, is further acting as another significant growth-inducing factor. Apart from this, the emerging popularity of gene therapy, that involves introducing a functional copy of the faulty gene into the body to treat this ailment, is expected to drive the Niemann-Pick disease market in the coming years.
IMARC Group's new report provides an exhaustive analysis of the Niemann-Pick disease 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 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 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 market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Niemann-Pick disease market
Reimbursement scenario in the market
In-market and pipeline drug
Competitive Landscape:
This report also provides a detailed analysis of the current Niemann-Pick disease 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 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 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 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 across the seven major markets?
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease by gender across the seven major markets?
What is the number of prevalent cases (2018-2034) of Niemann-Pick disease by type across the seven major markets?
How many patients are diagnosed (2018-2034) with Niemann-Pick disease across the seven major markets?
What is the size of the Niemann-Pick disease 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?
What will be the growth rate of patients across the seven major markets?
Niemann-Pick Disease: 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 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 market?
What are the key regulatory events related to the Niemann-Pick disease market?
What is the structure of clinical trial landscape by status related to the Niemann-Pick disease market?
What is the structure of clinical trial landscape by phase related to the Niemann-Pick disease market?
What is the structure of clinical trial landscape by route of administration related to the Niemann-Pick disease 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 - 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 - 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 - 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 Epidemiology by Type (2018-2034)
7.2.6 Diagnosed Cases (2018-2034)
7.2.7 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 Epidemiology by Type (2018-2034)
7.3.6 Diagnosed Cases (2018-2034)
7.3.7 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 Epidemiology by Type (2018-2034)
7.4.6 Diagnosed Cases (2018-2034)
7.4.7 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 Epidemiology by Type (2018-2034)
7.5.6 Diagnosed Cases (2018-2034)
7.5.7 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 Epidemiology by Type (2018-2034)
7.6.6 Diagnosed Cases (2018-2034)
7.6.7 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 Epidemiology by Type (2018-2034)
7.7.6 Diagnosed Cases (2018-2034)
7.7.7 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 Epidemiology by Type (2018-2034)
7.8.6 Diagnosed Cases (2018-2034)
7.8.7 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 Epidemiology by Type (2018-2034)
7.9.6 Diagnosed Cases (2018-2034)
7.9.7 Patient Pool/Treated Cases (2018-2034)
8 NIEMANN-PICK DISEASE - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 NIEMANN-PICK DISEASE - UNMET NEEDS
10 NIEMANN-PICK DISEASE - KEY ENDPOINTS OF TREATMENT
11 NIEMANN-PICK DISEASE - MARKETED PRODUCTS
11.1 List of Niemann-Pick Disease Marketed Drugs Across the Top 7 Markets
11.1.1 Xenpozyme (Olipudase alfa) - Sanofi
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
11.1.2 Zavesca (Miglustat) - Actelion Pharmaceuticals
11.1.2.1 Drug Overview
11.1.2.2 Mechanism of Action
11.1.2.3 Regulatory Status
11.1.2.4 Clinical Trial Results
11.1.2.5 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 - PIPELINE DRUGS
12.1 List of Niemann-Pick Disease 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
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 - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS
14. NIEMANN-PICK DISEASE – 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 - MARKET SCENARIO
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Niemann-Pick Disease - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Niemann-Pick Disease - 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 - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Niemann-Pick Disease - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Niemann-Pick Disease - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Niemann-Pick Disease - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Niemann-Pick Disease - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Niemann-Pick Disease - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Niemann-Pick Disease - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
16 NIEMANN-PICK DISEASE - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS
17 NIEMANN-PICK DISEASE MARKET - SWOT ANALYSIS
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 NIEMANN-PICK DISEASE 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 - 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 - 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 - 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 Epidemiology by Type (2018-2034)
7.2.6 Diagnosed Cases (2018-2034)
7.2.7 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 Epidemiology by Type (2018-2034)
7.3.6 Diagnosed Cases (2018-2034)
7.3.7 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 Epidemiology by Type (2018-2034)
7.4.6 Diagnosed Cases (2018-2034)
7.4.7 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 Epidemiology by Type (2018-2034)
7.5.6 Diagnosed Cases (2018-2034)
7.5.7 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 Epidemiology by Type (2018-2034)
7.6.6 Diagnosed Cases (2018-2034)
7.6.7 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 Epidemiology by Type (2018-2034)
7.7.6 Diagnosed Cases (2018-2034)
7.7.7 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 Epidemiology by Type (2018-2034)
7.8.6 Diagnosed Cases (2018-2034)
7.8.7 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 Epidemiology by Type (2018-2034)
7.9.6 Diagnosed Cases (2018-2034)
7.9.7 Patient Pool/Treated Cases (2018-2034)
8 NIEMANN-PICK DISEASE - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 NIEMANN-PICK DISEASE - UNMET NEEDS
10 NIEMANN-PICK DISEASE - KEY ENDPOINTS OF TREATMENT
11 NIEMANN-PICK DISEASE - MARKETED PRODUCTS
11.1 List of Niemann-Pick Disease Marketed Drugs Across the Top 7 Markets
11.1.1 Xenpozyme (Olipudase alfa) - Sanofi
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
11.1.2 Zavesca (Miglustat) - Actelion Pharmaceuticals
11.1.2.1 Drug Overview
11.1.2.2 Mechanism of Action
11.1.2.3 Regulatory Status
11.1.2.4 Clinical Trial Results
11.1.2.5 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 - PIPELINE DRUGS
12.1 List of Niemann-Pick Disease 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
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 - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS
14. NIEMANN-PICK DISEASE – 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 - MARKET SCENARIO
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Niemann-Pick Disease - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Niemann-Pick Disease - 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 - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Niemann-Pick Disease - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Niemann-Pick Disease - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Niemann-Pick Disease - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Niemann-Pick Disease - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Niemann-Pick Disease - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Niemann-Pick Disease - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Niemann-Pick Disease - 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 - Access and Reimbursement Overview
16 NIEMANN-PICK DISEASE - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS
17 NIEMANN-PICK DISEASE MARKET - SWOT ANALYSIS
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 NIEMANN-PICK DISEASE MARKET – STRATEGIC RECOMMENDATIONS
19 APPENDIX
