Summary

The 7 major progressive familial intrahepatic cholestasis markets are expected to exhibit a CAGR of 4.68% during 2024-2034.

The progressive familial intrahepatic cholestasis market has been comprehensively analyzed in IMARC's new report titled "Progressive Familial Intrahepatic Cholestasis Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that affects the liver's ability to secrete bile, which can lead to progressive liver damage and, eventually, liver failure. There are three main types of PFIC, which are classified based on the specific genetic mutations that cause the condition: type 1, type 2, and type 3. Some of the common disease symptoms include persistent itching, yellowing of the skin and eyes (jaundice), poor weight gain or weight loss, and a swollen abdomen. Children with PFIC may also experience fatigue, weakness, and delayed growth and development. The diagnosis typically involves a combination of medical history, physical examination, and various laboratory tests. Initial testing includes blood investigations to evaluate liver function and imaging tests such as an ultrasound, MRI, or CT scan to examine the liver and bile ducts. If PFIC is suspected, a genetic test may be recommended to identify mutations in the genes known to cause PFIC. Additionally, a liver biopsy may be performed to assess the extent of liver damage and rule out other liver diseases.

The rising incidences of genetic mutations and the escalating demand for effective medications and therapies to treat such conditions are primarily driving the progressive familial intrahepatic cholestasis market. In addition to this, the increasing utilization of bile acid sequestrants, including cholestyramine and colestipol, to slow down the progression of the disease is creating a positive outlook for the market. Moreover, the rising usage of ursodeoxycholic acid (UDCA) to treat PFIC, as it can reduce bile acid levels and improve liver function, is further propelling the market growth. Besides this, several regulatory agencies across the globe are offering various incentives and assistance for developing novel treatments to combat rare diseases, such as PFIC. This, in turn, is acting as another significant growth-inducing factor. Additionally, the widespread adoption of biliary diversion surgery for patients with PFIC type 1 or type 2 who do not respond to medical management is also bolstering the market growth. Apart from this, numerous key players are making extensive investments in research activities to investigate the use of gene therapy in order to address the underlying genetic mutations that cause the disease. This, in turn, is expected to drive the progressive familial intrahepatic cholestasis market in the coming years.

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

Progressive Familial Intrahepatic Cholestasis: 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 progressive familial intrahepatic cholestasis 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 progressive familial intrahepatic cholestasis market?
What are the key regulatory events related to the progressive familial intrahepatic cholestasis market?
What is the structure of clinical trial landscape by status related to the progressive familial intrahepatic cholestasis market?
What is the structure of clinical trial landscape by phase related to the progressive familial intrahepatic cholestasis market?
What is the structure of clinical trial landscape by route of administration related to the progressive familial intrahepatic cholestasis 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 Progressive Familial Intrahepatic Cholestasis - 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 Progressive Familial Intrahepatic Cholestasis - 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 Progressive Familial Intrahepatic Cholestasis - 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 Progressive Familial Intrahepatic Cholestasis - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Progressive Familial Intrahepatic Cholestasis - Unmet Needs

10 Progressive Familial Intrahepatic Cholestasis - Key Endpoints of Treatment

11 Progressive Familial Intrahepatic Cholestasis - Marketed Products
11.1 List of Progressive Familial Intrahepatic Cholestasis Marketed Drugs Across the Top 7 Markets
11.1.1 Bylvay (Odevixibat) - Albireo Pharma
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 Progressive Familial Intrahepatic Cholestasis - Pipeline Drugs
12.1 List of Progressive Familial Intrahepatic Cholestasis Pipeline Drugs Across the Top 7 Markets
12.1.1 Maralixibat - Mirum Pharmaceuticals
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. Progressive Familial Intrahepatic Cholestasis - Attribute Analysis of Key Marketed and Pipeline Drugs

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

16 Progressive Familial Intrahepatic Cholestasis - Recent Events and Inputs From Key Opinion Leaders

17 Progressive Familial Intrahepatic Cholestasis Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Progressive Familial Intrahepatic Cholestasis Market – Strategic Recommendations

19 Appendix