Progressive Familial Intrahepatic Cholestasis Treatment Market by Drug Type, Distribution Channel, End-Users

Description

List of Tables

[187 Pages Report] The Progressive Familial Intrahepatic Cholestasis Treatment Market size was estimated at USD 113.52 million in 2023 and expected to reach USD 118.62 million in 2024, at a CAGR 4.65% to reach USD 156.14 million by 2030.

Progressive familial intrahepatic cholestasis (PFIC) is a group of rare genetic liver disorders that occur due to defects in the biliary epithelial transporters, leading to impaired bile formation and flow and resulting in progressive liver disease. The severity and progression of the disease lead to liver failure if left untreated. Treatment strategies aim to manage symptoms, improve liver function, and prevent further damage. A growing awareness of PFIC among healthcare professionals and the public leads to earlier and more accurate diagnoses. Advanced genetic testing and molecular diagnostics have played a crucial role in identifying affected individuals, allowing for earlier intervention. Regulatory bodies such as the FDA and EMA have recognized the need for treatments targeting rare diseases, including PFIC. This has led to expedited review processes, grant programs, and other incentives for the development of therapies specifically aimed at rare conditions. The treatment of PFIC, precisely with novel therapies and liver transplantation, can be costly. This significantly burdens patients, families, and healthcare systems, potentially limiting access to necessary treatments. The high cost of treatment with novel therapies and liver transplantation poses a significant burden on patients, families, and healthcare systems, potentially limiting access to necessary treatments. Research and development in the field have led to innovative treatment options, including new pharmaceutical drugs and improved surgical techniques. This has expanded the treatment strategy portfolio, creating market growth opportunities.

KEY MARKET STATISTICS
Base Year [2023]	USD 113.52 million
Estimated Year [2024]	USD 118.62 million
Forecast Year [2030]	USD 156.14 million
CAGR (%)	4.65%

Regional Insights

The well-established healthcare infrastructure and increased healthcare expenditure support the growth of the PFIC treatment market in the Americas. Furthermore, the region benefits from active patient advocacy groups and government-supported health initiatives aimed at rare diseases, enhancing awareness and diagnosis rates. Regulatory incentives, including orphan drug designation, encourage pharmaceutical companies to invest in research and development, leading to a robust pipeline of therapeutic drugs. Canada and South American countries represent a growing market with increasing awareness and improving healthcare facilities for the treatment of rare diseases. The APAC region presents a diverse landscape for the PFIC treatment market, with countries including Japan and Australia leading in healthcare innovation and access to treatments. These countries have relatively high awareness of PFIC and its treatment options, supported by strong healthcare systems and policies favoring rare disease management. Europe is a leading region in terms of PFIC research and treatment, driven by favorable regulatory policies, extensive healthcare systems, and high levels of awareness. The European Medicines Agency (EMA) plays a crucial role in the fast-tracking approval of drugs for rare diseases, including PFIC. The region's unified approach to tackling rare diseases, exemplified by cross-border healthcare initiatives, significantly benefits PFIC patients. The Middle East and Africa, with advanced healthcare systems, have begun to recognize and address PFIC.

Market Insights

Market Dynamics

The market dynamics represent an ever-changing landscape of the Progressive Familial Intrahepatic Cholestasis Treatment Market by providing actionable insights into factors, including supply and demand levels. Accounting for these factors helps design strategies, make investments, and formulate developments to capitalize on future opportunities. In addition, these factors assist in avoiding potential pitfalls related to political, geographical, technical, social, and economic conditions, highlighting consumer behaviors and influencing manufacturing costs and purchasing decisions.

Market Drivers
- Increasing detection of progressive familial intrahepatic cholestasis with improvements in healthcare infrastructure
- Expanding role of patient advocacy groups to expand PFIC treatments

Market Restraints
- Access and affordability issues of PFIC treatments

Market Opportunities
- Advancements in PFIC research and diagnostic technologies
- Favorable government approvals for PFIC treatments

Market Challenges
- Complexity in PFIC disease management and treatment efficacy

Market Segmentation Analysis
- Drug Type: Rising usage of cholestyramine for reducing the level of bile acids in the liver and blood
- Distribution Channel: Preference for online pharmacies for continuous and convenient access to prescribed medications.
- End-Users: High potential for PFIC treatments in hospitals to manage severe complications of PFIC

Market Disruption Analysis
Porter's Five Forces Analysis
Value Chain & Critical Path Analysis
Pricing Analysis
Technology Analysis
Patent Analysis
Trade Analysis
Regulatory Framework Analysis

FPNV Positioning Matrix

The FPNV positioning matrix is essential in evaluating the market positioning of the vendors in the Progressive Familial Intrahepatic Cholestasis Treatment Market. This matrix offers a comprehensive assessment of vendors, examining critical metrics related to business strategy and product satisfaction. This in-depth assessment empowers users to make well-informed decisions aligned with their requirements. Based on the evaluation, the vendors are then categorized into four distinct quadrants representing varying levels of success, namely Forefront (F), Pathfinder (P), Niche (N), or Vital (V).

Market Share Analysis

The market share analysis is a comprehensive tool that provides an insightful and in-depth assessment of the current state of vendors in the Progressive Familial Intrahepatic Cholestasis Treatment Market. By meticulously comparing and analyzing vendor contributions, companies are offered a greater understanding of their performance and the challenges they face when competing for market share. These contributions include overall revenue, customer base, and other vital metrics. Additionally, this analysis provides valuable insights into the competitive nature of the sector, including factors such as accumulation, fragmentation dominance, and amalgamation traits observed over the base year period studied. With these illustrative details, vendors can make more informed decisions and devise effective strategies to gain a competitive edge in the market.

Recent Developments

FDA Endorses Livmarli for Pruritus Management in Progressive Familial Intrahepatic Cholestasis Patients

The U.S. Food and Drug Administration (FDA) has officially approved Mirum Pharmaceuticals' Livmarli oral solution in a significant advancement for patients with Progressive Familial Intrahepatic Cholestasis (PFIC), aimed at treating cholestatic pruritus in individuals aged five years and above. This decision is supported by extensive research efforts and a compelling accumulation of clinical data, demonstrating notable progress in several key health indicators, particularly in alleviating pruritus among affected children. [Published On: 2024-03-14]

Canadian Government Allocates CAD 20 Million to Enhance Health Outcomes for Children with Rare Diseases, Including Progressive Familial Intrahepatic Cholestasis

The Canadian government allocates an investment of CAD 20 million aimed at improving health outcomes. This funding initiative is part of a comprehensive strategy to address the challenges faced by children living with rare conditions, such as progressive familial intrahepatic cholestasis (PFIC), by enhancing access to specialized care, supporting innovative research, and fostering the development of new treatments. This investment highlights the government's commitment to elevating the standard of care for vulnerable populations, ensuring that children with rare diseases can look forward to a brighter, healthier future. [Published On: 2024-02-28]

French Government Awards Euro 4.9 Million Grant for Innovative Gene Therapy Development by Vivet Therapeutics"

The French Government, through its France Health Innovation Plan 2023, allocated a Euro 4.9 million (USD 5.3 million) grant to Vivet Therapeutics. This Paris-based biotech firm spearheads the development of VTX-806, a gene therapy aimed at combating cerebrotendinous xanthomatosis (CTX), the buildup of cholesterol in nerve cells. This initiative falls within the broader strategic framework of accelerating Biotherapies and Bioproduction in innovative therapies. [Published On: 2024-02-01]

Strategy Analysis & Recommendation

The strategic analysis is essential for organizations seeking a solid foothold in the global marketplace. Companies are better positioned to make informed decisions that align with their long-term aspirations by thoroughly evaluating their current standing in the Progressive Familial Intrahepatic Cholestasis Treatment Market. This critical assessment involves a thorough analysis of the organization's resources, capabilities, and overall performance to identify its core strengths and areas for improvement.

Key Company Profiles

The report delves into recent significant developments in the Progressive Familial Intrahepatic Cholestasis Treatment Market, highlighting leading vendors and their innovative profiles. These include AbbVie, Inc., Calliditas Therapeutics AB, CANbridge Pharmaceuticals Inc., Centurion Laboratories Private Limited, CymaBay Therapeutics, Inc., Enomark, Glenmark Pharmaceuticals Limited, Healthy Life Pharma Pvt. Ltd., Hebei Xingang Pharmaceutical Co., Ltd., Intercept Pharmaceuticals, Inc., Ipsen Pharma, Jackson Laboratories Pvt Ltd., Macleods Pharmaceuticals Ltd., Mirum Pharmaceuticals, Inc., Novartis AG, Par Pharmaceuticals, Inc. by Endo International PLC, Pfizer Inc., Sanofi S.A., SiNi Pharma Pvt. Ltd., Taj Pharmaceuticals Limited, Takeda Pharmaceutical Company Limited, Teva Pharmaceutical Industries Ltd., Viatris Inc., Vivet Therapeutics, Wellona Pharma, and Zoic Biotech Pvt. Ltd..

Market Segmentation & Coverage

This research report categorizes the Progressive Familial Intrahepatic Cholestasis Treatment Market to forecast the revenues and analyze trends in each of the following sub-markets:

Drug Type
- Cholestyramine
- Rifampicin
- Ursodeoxycholic Acid

Distribution Channel
- Hospital Pharmacies
- Online Pharmacies
- Retail Pharmacies

End-Users
- Ambulatory Surgical Centers
- Hospitals
- Specialty Clinics

Region
- Americas
  - Argentina
  - Brazil
  - Canada
  - Mexico
  - United States
    - California
    - Florida
    - Illinois
    - New York
    - Ohio
    - Pennsylvania
    - Texas
- Asia-Pacific
  - Australia
  - China
  - India
  - Indonesia
  - Japan
  - Malaysia
  - Philippines
  - Singapore
  - South Korea
  - Taiwan
  - Thailand
  - Vietnam

Europe, Middle East & Africa
- Denmark
- Egypt
- Finland
- France
- Germany
- Israel
- Italy
- Netherlands
- Nigeria
- Norway
- Poland
- Qatar
- Russia
- Saudi Arabia
- South Africa
- Spain
- Sweden
- Switzerland
- Turkey
- United Arab Emirates
- United Kingdom

Product Code: MRR-AD517FAAA5F2

1. Preface

1.1. Objectives of the Study
1.2. Market Segmentation & Coverage
1.3. Years Considered for the Study
1.4. Currency & Pricing
1.5. Language
1.6. Stakeholders

2. Research Methodology

2.1. Define: Research Objective
2.2. Determine: Research Design
2.3. Prepare: Research Instrument
2.4. Collect: Data Source
2.5. Analyze: Data Interpretation
2.6. Formulate: Data Verification
2.7. Publish: Research Report
2.8. Repeat: Report Update

3. Executive Summary

4. Market Overview

5. Market Insights

5.1. Market Dynamics
- 5.1.1. Drivers
  - 5.1.1.1. Increasing detection of progressive familial intrahepatic cholestasis with improvements in healthcare infrastructure
  - 5.1.1.2. Expanding role of patient advocacy groups to expand PFIC treatments
- 5.1.2. Restraints
  - 5.1.2.1. Access and affordability issues of PFIC treatments
- 5.1.3. Opportunities
  - 5.1.3.1. Advancements in PFIC research and diagnostic technologies
  - 5.1.3.2. Favorable government approvals for PFIC treatments
- 5.1.4. Challenges
  - 5.1.4.1. Complexity in PFIC disease management and treatment efficacy
5.2. Market Segmentation Analysis
- 5.2.1. Drug Type: Rising usage of cholestyramine for reducing the level of bile acids in the liver and blood
- 5.2.2. Distribution Channel: Preference for online pharmacies for continuous and convenient access to prescribed medications.
- 5.2.3. End-Users: High potential for PFIC treatments in hospitals to manage severe complications of PFIC
5.3. Market Trend Analysis
- 5.3.1. Rising approvals for PFIC treatment from regulatory bodies in the Americas
- 5.3.2. Favorable government support for advancing therapeutics for rare diseases and growing efforts to advance medical and healthcare services and solutions in the APAC region.
- 5.3.3. Increasing research activities to explore effective treatments and standards and the well-established pharmaceutical industry of the EMEA region
5.4. Cumulative Impact of High Inflation
5.5. Porter's Five Forces Analysis
- 5.5.1. Threat of New Entrants
- 5.5.2. Threat of Substitutes
- 5.5.3. Bargaining Power of Customers
- 5.5.4. Bargaining Power of Suppliers
- 5.5.5. Industry Rivalry
5.6. Value Chain & Critical Path Analysis
5.7. Regulatory Framework Analysis

6. Progressive Familial Intrahepatic Cholestasis Treatment Market, by Drug Type

6.1. Introduction
6.2. Cholestyramine
6.3. Rifampicin
6.4. Ursodeoxycholic Acid

7. Progressive Familial Intrahepatic Cholestasis Treatment Market, by Distribution Channel

7.1. Introduction
7.2. Hospital Pharmacies
7.3. Online Pharmacies
7.4. Retail Pharmacies

8. Progressive Familial Intrahepatic Cholestasis Treatment Market, by End-Users

8.1. Introduction
8.2. Ambulatory Surgical Centers
8.3. Hospitals
8.4. Specialty Clinics

9. Americas Progressive Familial Intrahepatic Cholestasis Treatment Market

9.1. Introduction
9.2. Argentina
9.3. Brazil
9.4. Canada
9.5. Mexico
9.6. United States

10. Asia-Pacific Progressive Familial Intrahepatic Cholestasis Treatment Market

10.1. Introduction
10.2. Australia
10.3. China
10.4. India
10.5. Indonesia
10.6. Japan
10.7. Malaysia
10.8. Philippines
10.9. Singapore
10.10. South Korea
10.11. Taiwan
10.12. Thailand
10.13. Vietnam

11. Europe, Middle East & Africa Progressive Familial Intrahepatic Cholestasis Treatment Market

11.1. Introduction
11.2. Denmark
11.3. Egypt
11.4. Finland
11.5. France
11.6. Germany
11.7. Israel
11.8. Italy
11.9. Netherlands
11.10. Nigeria
11.11. Norway
11.12. Poland
11.13. Qatar
11.14. Russia
11.15. Saudi Arabia
11.16. South Africa
11.17. Spain
11.18. Sweden
11.19. Switzerland
11.20. Turkey
11.21. United Arab Emirates
11.22. United Kingdom

12. Competitive Landscape

12.1. Market Share Analysis, 2023
12.2. FPNV Positioning Matrix, 2023
12.3. Competitive Scenario Analysis
- 12.3.1. FDA Endorses Livmarli for Pruritus Management in Progressive Familial Intrahepatic Cholestasis Patients
- 12.3.2. Canadian Government Allocates CAD 20 Million to Enhance Health Outcomes for Children with Rare Diseases, Including Progressive Familial Intrahepatic Cholestasis
- 12.3.3. French Government Awards Euro 4.9 Million Grant for Innovative Gene Therapy Development by Vivet Therapeutics"
- 12.3.4. Approval of Bylvay (odevixibat) by Health Canada for PFIC-Related Pruritus
- 12.3.5. CANbridge Pharmaceuticals Gains NMPA Approval for CAN108 NDA
- 12.3.6. UKRI Allocates GBP 14 Million to Advance Research on Progressive Familial Intrahepatic Cholestasis and Other Rare Diseases
- 12.3.7. Jadeite Medicines Advances Treatment for PFIC with Phase 3 Trial and Orphan Drug Designation in Japan
- 12.3.8. India Boosts Rare Diseases Treatment, Allocates INR 40 Crore with a Focus on PFIC and Enhanced Patient Care
- 12.3.9. Ipsen Enhances Rare Disease Offerings with Strategic Acquisition of Albireo Pharma

13. Competitive Portfolio

13.1. Key Company Profiles
13.2. Key Product Portfolio