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Krabbe Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034

May 2024 | 128 pages | ID: KC460B15F8B1EN
IMARC Group

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The 7 major Krabbe disease markets reached a value of US$ 2.5 Billion in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 4.7 Billion by 2034, exhibiting a growth rate (CAGR) of 5.81% during 2024-2034.

The Krabbe disease market has been comprehensively analyzed in IMARC's new report titled "Krabbe Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Krabbe disease, also known as globoid cell leukodystrophy, refers to a progressive genetic disorder that affects the nervous system. The symptoms of the ailment typically appear in early infancy, although they can occur at any age. These include developmental delays, vision and hearing loss, muscle weakness and spasticity, seizures, etc. Affected individuals may also experience irritability, feeding difficulties, and difficulty swallowing. The severity and age of onset of indications vary depending on the specific mutations and the degree of enzyme deficiency. The diagnosis of Krabbe disease typically involves a combination of clinical evaluation, genetic testing, and imaging studies. A physical exam may reveal neurological symptoms, such as muscle weakness and spasticity, as well as developmental delays. Genetic testing can identify mutations in the galactosylceramide gene, which provides instructions for making galactocerebrosidase. Numerous imaging studies, like magnetic resonance imaging, can show abnormalities in the brain, including the loss of myelin and the presence of white matter changes. Blood and urine tests are also used to assess levels of galactocerebrosidase and other metabolites.

The increasing cases of genetic mutations that result in a deficiency or complete absence of the galactocerebrosidase enzyme, thereby leading to the accumulation of galactosylceramide in the nervous system, are primarily driving the Krabbe disease market. Besides this, the widespread adoption of hematopoietic stem cell transplantation (HSCT) to restore the missing enzyme and slow down the progression of the ailment is creating a positive outlook for the market. Furthermore, the inflating demand for enzyme replacement therapy, which involves the administration of a functioning galactocerebrosidase enzyme directly into the patient's bloodstream, is acting as another significant growth-inducing factor. Additionally, numerous key players are making extensive investments to develop drugs that target the accumulation of galactosylceramide and enhance the activity of other enzymes involved in myelin metabolism. This, in turn, is also augmenting the market growth. Moreover, the emerging popularity of electrophysiological testing to identify characteristic patterns of nerve damage caused by the accumulation of fatty substances in the nervous system and monitor disorder progression over time is expected to drive the Krabbe disease market in the coming years.

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

Krabbe 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 Krabbe 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 Krabbe disease market?
What are the key regulatory events related to the Krabbe disease market?
What is the structure of clinical trial landscape by status related to the Krabbe disease market?
What is the structure of clinical trial landscape by phase related to the Krabbe disease market?
What is the structure of clinical trial landscape by route of administration related to the Krabbe 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 KRABBE 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 KRABBE 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 KRABBE 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 KRABBE DISEASE - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES

8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 KRABBE DISEASE - UNMET NEEDS

10 KRABBE DISEASE - KEY ENDPOINTS OF TREATMENT

11 KRABBE DISEASE - MARKETED PRODUCTS

11.1 List of Krabbe Disease Marketed Drugs Across the Top 7 Markets
  11.1.1 Drug Name – Company Name
    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 complete list of marketed drugs has been provided in the report.

12 KRABBE DISEASE - PIPELINE DRUGS

12.1 List of Krabbe Disease Pipeline Drugs Across the Top 7 Markets
  12.1.1 FBX 101 - Forge Biologics
    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 PLX 200 - Polaryx Therapeutics
    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
Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report

13. KRABBE DISEASE - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS

14. KRABBE 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 KRABBE DISEASE - MARKET SCENARIO

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

16 KRABBE DISEASE - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS

17 KRABBE DISEASE MARKET - SWOT ANALYSIS

17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 KRABBE DISEASE MARKET – STRATEGIC RECOMMENDATIONS

19 APPENDIX


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