Summary

The 7 major Wolman disease markets are expected to exhibit a CAGR of 8.29% during 2024-2034.

The Wolman disease market has been comprehensively analyzed in IMARC's new report titled "Wolman Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Wolman disease refers to a rare genetic disorder that affects the body's ability to break down certain fats, called lipids. This condition is caused by a deficiency of the lysosomal acid lipase enzyme, which is responsible for breaking down lipids in the body. As a result, lipids may accumulate abnormally in the organs and tissues of the body, manifesting a variety of symptoms. The most common indications of this disorder include bloating or swelling of the stomach, enlargement of the spleen and liver, low muscle tone, scarring of the liver, etc. Individuals suffering from Wolman disease may also experience persistent and often forceful vomiting, foul-smelling, frequent diarrhea, fatty stools, growth failure, malnutrition, etc. The diagnosis of this ailment relies on a comprehensive clinical examination, a complete patient history, and specialized tests that show lysosomal lipase acid enzyme absence or insufficient activity in particular cells and tissues of the body. The healthcare provider may also perform several imaging studies, such as a CT scan, MRI, X-ray, etc., to identify enlargement of the person's spleen or liver, enlarged lymph nodes, and calcification of the adrenal glands.

The rising cases of gene mutations that affect the breakdown or metabolism of certain fats in the body are primarily driving the Wolman disease market. Besides this, the increasing prevalence of inherited metabolic disorders that lead to the buildup of fatty substances in specific organs is acting as another significant growth-inducing factor. Furthermore, the widespread adoption of recombinant enzyme therapies, including sebelipase alfa, for treating the condition, since they can help to break down fats and control them from accumulating in the body's cells, is also creating a positive outlook for the market. Apart from this, the inflating utilization of hematopoietic stem cell transplantation to enable the growth of healthy cells with sufficient levels of lysosomal acid lipase that can dramatically improve existing symptoms is further augmenting the market growth. Moreover, the emerging popularity of gene therapy, in which the defective gene is permanently replaced with a normal gene to promote the production of active enzymes and inhibit the progression and development of the ailment, is expected to drive the Wolman disease market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Wolman 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 Wolman 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 Wolman 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 Wolman disease market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Wolman disease market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current Wolman 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 Wolman 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 Wolman disease market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the Wolman 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 Wolman disease across the seven major markets?
What is the number of prevalent cases (2018-2034) of Wolman disease by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Wolman disease by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with Wolman disease across the seven major markets?
What is the size of the Wolman 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 Wolman disease?
What will be the growth rate of patients across the seven major markets?

Wolman 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 Wolman 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 Wolman disease market?
What are the key regulatory events related to the Wolman disease market?
What is the structure of clinical trial landscape by status related to the Wolman disease market?
What is the structure of clinical trial landscape by phase related to the Wolman disease market?
What is the structure of clinical trial landscape by route of administration related to the Wolman disease market?

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Table of Contents

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 Wolman 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 Wolman 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 Wolman 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 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 Wolman Disease - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Wolman Disease - Unmet Needs

10 Wolman Disease - Key Endpoints of Treatment

11 Wolman Disease - Marketed Products
11.1 List of Wolman Disease Marketed Drugs Across the Top 7 Markets
11.1.1 Kanuma (Sebelipase alfa) - Alexion AstraZeneca Rare Disease
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 Wolman Disease - Pipeline Drugs
12.1 List of Wolman Disease Pipeline Drugs Across the Top 7 Markets
12.1.1 Drug Name – Company Name
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

Kindly note that the complete list of pipeline drugs has been provided in the report.

13. Wolman Disease - Attribute Analysis of Key Marketed and Pipeline Drugs

14. Wolman 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 Wolman Disease - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Wolman Disease - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Wolman 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 Wolman Disease - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Wolman 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 Wolman Disease - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Wolman Disease - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Wolman 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 Wolman Disease - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Wolman Disease - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Wolman 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 Wolman Disease - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Wolman Disease - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Wolman 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 Wolman Disease - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Wolman Disease - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Wolman 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 Wolman Disease - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Wolman Disease - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Wolman 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 Wolman Disease - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Wolman Disease - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Wolman 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 Wolman Disease - Access and Reimbursement Overview

16 Wolman Disease - Recent Events and Inputs From Key Opinion Leaders

17 Wolman Disease Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Wolman Disease Market – Strategic Recommendations

19 Appendix