Dublin, June 07, 2024 (GLOBE NEWSWIRE) -- The "Niemann-Pick Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034" report has been added to ResearchAndMarkets.com's offering.
The 7 major Niemann-Pick disease markets are expected to exhibit a CAGR of 4.22% during 2023-2034.
Key Highlights
- 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.
Report Scope
This 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.
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
Competitive Landscape
This report also provides a detailed analysis of the current Niemann-Pick disease marketed drugs and late-stage pipeline drugs.
- Drug Overview
- Mechanism of Action
- Regulatory Status
- Clinical Trial Results
- Drug Uptake and Market Performance
Key Topics Covered
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
7.3 Epidemiology Scenario - United States
7.4 Epidemiology Scenario - Germany
7.5 Epidemiology Scenario - France
7.6 Epidemiology Scenario - United Kingdom
7.7 Epidemiology Scenario - Italy
7.8 Epidemiology Scenario - Spain
7.9 Epidemiology Scenario - Japan
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
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
12.1.3 AZ-3102 - Azafaros
12.1.4 Arimoclomol - Zevra Therapeutics
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
15.3 Market Scenario - United States
15.4 Market Scenario - Germany
15.5 Market Scenario - France
15.6 Market Scenario - United Kingdom
15.7 Market Scenario - Italy
15.8 Market Scenario - Spain
15.9 Market Scenario - Japan
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
For more information about this report visit https://www.researchandmarkets.com/r/mkf64z
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