Progressive Familial Intrahepatic Cholestasis Treatment Market by Drug Type (Cholestyramine, Rifampicin, Ursodeoxycholic Acid), Distribution Channel (Hospital Pharmacies, Online Pharmacies, Retail Pharmacies), End-Users - Global Forecast 2024-2030
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.
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
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.
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.
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.
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
The prosthetics and orthotics market encompasses the design, manufacturing, and distribution of artificial limbs (prosthetic devices) and supportive or corrective medical braces (orthotic devices) designed to support, align, prevent, or correct deformities or to enhance the operation of movable parts of the body. Prosthetics are custom-made to fit the individual's unique needs, while orthotics can be customized or off-the-shelf. Increasing incidences of sports-related injuries and road accidents, along with a growing geriatric population and rising prevalence of osteosarcoma, drive market growth. Increasing adoption of braces, splints, and non-invasive treatments also fuels the market growth. High cost and lack of accessibility for orthotics & and prosthetics, complex designs, and discomfort related to prosthetics & orthotics pose a challenge to market growth. Rising product developments using advanced prosthetic manufacturing techniques make them more accessible and affordable. Moreover, developing ecosystems for start-ups manufacturing robotic prosthetic arms and exoskeletons also poses potential opportunities for the market growth.
Regional Insights
The prosthetics and orthotics market in North America is highly developed, with significant demand for advanced prosthetics and orthotic devices, driven by the growing elderly population, increasing prevalence of diabetes and obesity, and many amputations due to vascular diseases. The United States is among the major market share due to its robust healthcare infrastructure, R&D investments, and the presence of major industry players. In South America, the market for prosthetics and orthotics is expanding, and improving economic conditions and government efforts to enhance healthcare infrastructure are creating growth opportunities. In the Asia-Pacific (APAC) region, the prosthetics and orthotics market is rapidly growing due to its vast population, increasing healthcare expenditures, and growing awareness of available prosthetic and orthotic services. Countries such as China, Japan, India, and Australia are home to many local manufacturers producing low-cost devices, making it a competitive and diverse market. Europe is more advanced in terms of technology adoption in prosthetics and orthotics. The growing aging population and the subsequent rise in musculoskeletal disorders propel the European market. In the Middle East and Africa, there is increasing the potential for market growth due to economic development and a growing awareness of mobility aids. The region's focus on improving healthcare services and investments in training healthcare professionals in prosthetics and orthotics are important trends.
Market Insights- Market Dynamics
The market dynamics represent an ever-changing landscape of the Prosthetics & Orthotics 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 Incidences of Sports Injuries and Road Accidents
- Growing Geriatric Population and Rising Prevalence of Osteosarcoma
- Increasing Adoption of Braces and Splints and Non-Invasive Treatments
- Market Restraints
- High Cost and Lack of Accessibility for Orthotics & Prosthetics
- Market Opportunities
- Rising Product Developments Using Advanced Manufacturing Techniques in Prosthetics
- Developing Ecosystem for Start-ups Manufacturing Robotic Prosthetic Arms and Exoskeletons
- Market Challenges
- Complex Designs and Discomfort Related to Prosthetics & Orthotics
- Market Segmentation Analysis
- Type: Rising popularity for prosthetics to improve quality of life by providing movement and independence
- Application: Rising usage of prosthetics and orthotics in the hospitals to cater to a larger patient base with a wider variety of health concerns
- Market Disruption Analysis
- Porter’s Five Forces Analysis
- Value Chain & Critical Path Analysis
- Pricing Analysis
- Technology Analysis
- Patent Analysis
- Trade Analysis
- Regulatory Framework Analysis
The FPNV positioning matrix is essential in evaluating the market positioning of the vendors in the Prosthetics & Orthotics 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 Prosthetics & Orthotics 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- OrthoPediatrics acquires Boston Orthotics & Prosthetics
OrthoPediatrics Corp. acquired Boston Orthotics & Prosthetics to integrate an extensive range of specialized products, including innovative bracing, orthotic, and prosthetic technologies tailored for pediatric care. The transaction, completed with an initial cash disbursement of USD 22 million, signifies a landmark expansion of OrthoPediatrics' mission to fully support the complete spectrum of care for young patients with orthopedic conditions.
VIT-AP University inks pact to establish prosthetics and orthotics center
The VIT-AP Technology Business Incubation Foundation (VTBIF) and Buimerc India Foundation (BIF) have forged a valuable partnership through a Memorandum of Understanding aimed at establishing an innovative Centre for Prosthetics and Orthotics for Humans and Animals (POHA) at VIT-AP University. With a generous commitment of INR 52 lakh from BIF, the newly-formed POHA Research Centre embark on a transformative project entitled Design, Development & Fabrication of SSW Criteria-Based Trans-Tibial/Trans-Femoral Regenerative Effort Assistive Prosthesis for Youth/Elderly.
Spentys and Arize by HP partner to expand digital solutions to Orthotics and Prosthetics market in the US
Spentys teamed up with Arize by HP to empower healthcare providers in the United States with a cutting-edge, digital platform for creating patient-specific orthoses using 3D technologies. This collaboration brings Spentys's robust scanning, modeling, and 3D printing platform to a wider audience through Arize's expansive US sales network, promising a transformative impact on custom O&P device production.
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 Prosthetics & Orthotics 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 Prosthetics & Orthotics Market, highlighting leading vendors and their innovative profiles. These include B.Braun SE, Bauerfeind AG, Beast Prosthetics, Blatchford Limited, Boston Scientific Corporation, COAPT LLC, COVVI Ltd, Globus Medical Inc., Hanger, Inc., Howard Orthopedics, Inc., Human Technology, Inc., Johnson & Johnson Services, Inc., Merck & Co, Inc., Olympus Corporation, Ortho Europe, OrthoPediatrics Corp., Ossur HF, Ottobock SE & Co. KGaA, Shadow Robot Company, Shapeways Holdings, Inc., Smith & Nephew PLC, Steeper Inc., Stryker Corporation, Synergy Prosthetics, Inc., Ultraflex Systems, Inc., WillowWood Global LLC, and Zimmer Biomet Holdings, Inc..
Market Segmentation & Coverage
This research report categorizes the Prosthetics & Orthotics Market to forecast the revenues and analyze trends in each of the following sub-markets:- Type
- Orthotics
- Lower Limb
- Spinal
- Upper Limb
- Prosthetics
- Liners
- Lower Extremity
- Modular Components
- Sockets
- Upper Extremity
- Technology
- Conventional
- Electric Powered
- Hybrid Prosthetics
- Application
- Clinics
- Hospitals
- Rehabilitation Centers
- 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
Please Note: PDF & Excel + Online Access - 1 Year
- Americas
- Orthotics
- Market Drivers
- Americas
- Market Drivers
- 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"
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