Summary

The 7 major Alagille syndrome markets reached a value of US$ 267.6 Million in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 435.6 Million by 2034, exhibiting a growth rate (CAGR) of 4.53% during 2024-2034.

The Alagille syndrome market has been comprehensively analyzed in IMARC's new report titled "Alagille Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Alagille syndrome is a rare genetic condition that affects multiple organ systems, including the liver, heart, kidneys, etc. The disorder is caused by mutations in the JAG1 or NOTCH2 genes, which are responsible for producing proteins involved in the development and maintenance of various organs in the body. The symptoms indicative of the ailment can vary widely but often include jaundice, itching, poor growth and weight gain, heart defects, kidney problems, etc. Infants with Alagille syndrome may have difficulty gaining weight and experience liver-related complications like cholestasis. In severe cases, the disease can lead to cirrhosis of the liver, liver failure, and other life-threatening complications. The diagnosis of Alagille syndrome typically involves a combination of clinical evaluation, medical history, and genetic testing. Physicians may perform a physical exam to look for characteristic features of the condition, such as facial abnormalities or liver enlargement. Blood tests are used to assess liver function and detect genetic mutations associated with Alagille syndrome. In some cases, various imaging tests, including ultrasound and magnetic resonance imaging, are utilized to evaluate the liver or other organs affected by the condition. Additionally, genetic testing can confirm a diagnosis of the ailment by identifying mutations in the JAG1 or NOTCH2 genes.

The increasing prevalence of rare genetic disorders and the rising unmet need for effective treatments to address the underlying genetic defect and improve patient outcomes are primarily driving the Alagille syndrome market. In addition to this, the escalating utilization of bile acid-binding resins, such as cholestyramine, to reduce itching caused by liver dysfunction in patients with Alagille syndrome is also creating a positive outlook for the market. These medications bind to bile acids present in the intestine and inhibit them from being reabsorbed into the bloodstream. Moreover, the widespread adoption of next-generation sequencing panels to diagnose Alagille syndrome since they can identify multiple genetic mutations simultaneously and provide a rapid and cost-effective method of genetic testing is further bolstering the market growth. Apart from this, several regulatory agencies are taking initiatives to expedite the approval of new therapeutics for rare diseases, including Alagille syndrome, through orphan drug status, fast-track designation, priority reviews, etc. This, in turn, is acting as another significant growth-inducing factor. Additionally, the ongoing development of various novel therapies for the treatment of Alagille syndrome, such as medications that target bile acid metabolism and gene therapies which aim to correct genetic defects, is expected to drive the Alagille syndrome market during the forecast period.

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

Alagille Syndrome: 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 Alagille syndrome 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 Alagille syndrome market?
What are the key regulatory events related to the Alagille syndrome market?
What is the structure of clinical trial landscape by status related to the Alagille syndrome market?
What is the structure of clinical trial landscape by phase related to the Alagille syndrome market?
What is the structure of clinical trial landscape by route of administration related to the Alagille syndrome 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 Alagille Syndrome - 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 Alagille Syndrome - 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 Alagille Syndrome - 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​)
7.2.6 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​)
7.3.6 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 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 Alagille Syndrome - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Alagille Syndrome - Unmet Needs

10 Alagille Syndrome - Key Endpoints of Treatment

11 Alagille Syndrome - Marketed Products
11.1 List of Alagille Syndrome Marketed Drugs Across the Top 7 Markets
11.1.1 Livmarli (Maralixibat) - Mirum 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 Alagille Syndrome - Pipeline Drugs
12.1 List of Alagille Syndrome Pipeline Drugs Across the Top 7 Markets
12.1.1 Odevixibat - Albireo Pharma
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 above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.

13. Alagille Syndrome - Attribute Analysis of Key Marketed and Pipeline Drugs

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

16 Alagille Syndrome - Recent Events and Inputs From Key Opinion Leaders

17 Alagille Syndrome Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Alagille Syndrome Market – Strategic Recommendations

19 Appendix