Global Primary Sclerosing Cholangitis Therapeutics Market Growth, Share, Size, Trends and Forecast (2025 - 2031)
By Drug;
BTT1023, GS-9674, NGM282, OCA, Cenicriviroc, LUM001, DUR928, norUDCA, HTD1801, IDN-7314, and STP705By Treatment Type;
Pharmaceutical Therapy and Surgical InterventionBy Geography;
North America, Europe, Asia Pacific, Middle East & Africa, and Latin America - Report Timeline (2021 - 2031)Primary Sclerosing Cholangitis Therapeutics Market Overview
Primary Sclerosing Cholangitis Therapeutics Market (USD Million)
Primary Sclerosing Cholangitis Therapeutics Market was valued at USD 4,826.18 million in the year 2024. The size of this market is expected to increase to USD 6,350.93 million by the year 2031, while growing at a Compounded Annual Growth Rate (CAGR) of 4.0%.
Global Primary Sclerosing Cholangitis Therapeutics Market Growth, Share, Size, Trends and Forecast
*Market size in USD million
CAGR 4.0 %
Study Period | 2025 - 2031 |
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Base Year | 2024 |
CAGR (%) | 4.0 % |
Market Size (2024) | USD 4,826.18 Million |
Market Size (2031) | USD 6,350.93 Million |
Market Concentration | High |
Report Pages | 380 |
Major Players
- Intercept Pharmaceuticals, Inc
- Gilead Sciences, Inc
- Allergan plc
- Genfit SA
- Novartis AG
- Pfizer Inc
- Merck & Co., Inc
- Takeda Pharmaceutical Company Limited
- Bristol Myers Squibb
- F Hoffmann-La Roche Ltd
Market Concentration
Consolidated - Market dominated by 1 - 5 major players
Global Primary Sclerosing Cholangitis Therapeutics Market
Fragmented - Highly competitive market without dominant players
The Primary Sclerosing Cholangitis (PSC) Therapeutics Market is witnessing significant traction due to the increasing demand for effective treatments for this rare, chronic liver condition. The market is shaped by a growing awareness of autoimmune diseases and a consistent rise in research investments. An estimated 35% rise in clinical pipeline activities has further fueled development in this domain, reflecting the increasing interest in orphan drug development.
A surge in diagnosis rates has contributed to a 28% increase in treatment uptake, as healthcare systems improve their diagnostic capabilities. Technological advancements in biomarkers and imaging tools are enhancing early detection. Additionally, the adoption of combination therapies is rising, driven by their potential to delay disease progression and improve patient outcomes, leading to wider physician acceptance.
Therapeutic Advancements
The market is evolving with 42% of current clinical trials focusing on novel immunomodulators, indicating a paradigm shift towards targeted therapy approaches. Biologic drugs are gaining favor due to their capacity to modulate the immune system more precisely. The increasing prevalence of liver transplants as a last-resort treatment also emphasizes the urgent need for non-invasive alternatives.
Regulatory and R&D Landscape
Strong support from regulatory authorities for orphan drugs has led to a 31% rise in drug designations over recent years. This trend is catalyzing innovation by reducing approval timelines and encouraging investment. Collaborative efforts among academic institutions and biotech firms are significantly contributing to the pipeline robustness, fostering diversified treatment options.
Market Outlook
With a focus on curative treatments, the market is expected to see over 38% of pipeline therapies entering advanced trial phases soon. Companies are investing in precision medicine to provide individualized care solutions. The continued expansion of healthcare access and rising patient advocacy are expected to sustain long-term market momentum, ensuring the introduction of safer and more effective therapeutic options.
Primary Sclerosing Cholangitis Therapeutics Market Recent Developments
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In April 2022, Sirnaomics, a clinical-stage RNA therapeutics biopharmaceutical company, carried out a Phase I clinical trial analyzing the potential of STP707 to treat liver fibrosis in primary sclerosing cholangitis in the USA A systemic small interfering ribonucleic acid (siRNA) therapy STP707 comprises two siRNA oligonucleotides that act on TGF-β1 and COX-2 mRNA, respectively.
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In February 2022, Siranomics received the safe-to-proceed approval letter from the US Food and Drug Administration (FDA) for the Company's investigational new drug (IND) application for STP707, it's lead siRNA (small interfering RNA) drug candidate in Primary Sclerosing Cholangitis. The Company anticipates initiating a Phase I study in the second quarter of 2022, to evaluate the safety, tolerability, and pharmacokinetics of STP707, administered intravenously in healthy volunteers.
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In January 2022, Engitix built a Liver Disease Pipeline with €48M Series A round and a drug discovery partnership with the Milan-based Dompé Farmaceutici. The proceeds from Engitix’s Series A round will bankroll the development of the company’s tissue models for use in drug discovery. Moreover, the investment will fuel the preclinical development of Engitix’s drug pipeline, with a focus on treating liver diseases including primary sclerosing cholangitis and liver cancer. The first candidate is expected to reach phase I testing by 2025.
Primary Sclerosing Cholangitis Therapeutics Market Segment Analysis
In this report, the Global Primary Sclerosing Cholangitis Therapeutics Market has been segmented by Drug, Treatment Type and Geography.
Primary Sclerosing Cholangitis Therapeutics Market, Segmentation by Drug
The Primary Sclerosing Cholangitis Therapeutics Market has been segmented by Drug into BTT1023, GS-9674, NGM282, OCA, Cenicriviroc, LUM001, DUR928, norUDCA, HTD1801, IDN-7314, and STP705.
BTT1023
BTT1023 holds a promising share of approximately 12% in the PSC therapeutics pipeline, targeting VAP-1 inhibition to reduce inflammation and fibrosis in bile ducts. It is being evaluated for its ability to modulate immune cell trafficking and prevent disease progression.
GS-9674
GS-9674 contributes around 11% to the pipeline, functioning as an FXR agonist aimed at reducing bile acid toxicity and liver inflammation. It is under clinical investigation for its potential to improve liver enzyme profiles and halt fibrotic damage.
NGM282
NGM282 (aldafermin) represents approximately 10% of the market pipeline, known for its FGF19 analog activity that supports bile acid regulation and anti-inflammatory effects. Early-phase trials suggest improvement in liver histology and biomarker reduction.
OCA
OCA (obeticholic acid) accounts for nearly 9%, acting as a potent FXR agonist to address cholestasis and inflammation. It has shown potential in improving bile flow and is also being studied in related conditions like PBC and NASH.
Cenicriviroc
Cenicriviroc contributes close to 8%, targeting CCR2/CCR5 receptors to modulate immune-mediated fibrosis. It is being explored for its dual anti-inflammatory and anti-fibrotic properties in liver diseases including PSC.
LUM001
LUM001 (maralixibat) makes up about 7% of the pipeline, working as an IBAT inhibitor to reduce bile acid reabsorption and improve pruritus symptoms associated with cholestatic conditions.
DUR928
DUR928 accounts for roughly 7%, a novel epigenetic regulator with anti-inflammatory and anti-fibrotic mechanisms. It is gaining attention for its broad therapeutic potential across hepatic injury models.
norUDCA
norUDCA captures around 6% of the pipeline and is a side chain-shortened bile acid designed to enhance bile flow and reduce toxic bile acid accumulation. It is progressing through advanced clinical stages.
HTD1801
HTD1801 holds approximately 6%, offering dual benefits in liver metabolism and inflammation reduction. It combines berberine and ursodeoxycholic acid, and is under evaluation for PSC and metabolic liver disorders.
IDN-7314
IDN-7314 makes up nearly 5%, being studied as an FXR modulator to reduce bile acid synthesis and improve hepatic inflammation. It is showing potential in early-phase trials.
STP705
STP705 contributes the remaining 4%, a small interfering RNA (siRNA)-based therapy targeting TGF-β1 and COX-2 to combat liver fibrosis and inflammation. It is part of a novel class of gene-silencing therapeutics in liver disease pipelines.
Primary Sclerosing Cholangitis Therapeutics Market, Segmentation by Treatment Type
The Primary Sclerosing Cholangitis Therapeutics Market has been segmented by Treatment Type into Pharmaceutical Therapy, and Surgical Intervention.
Pharmaceutical Therapy
Pharmaceutical therapy dominates the PSC therapeutics market with a share of approximately 70%, driven by increasing clinical trials for anti-fibrotic, anti-inflammatory, and bile acid-modulating agents. Current drug candidates focus on symptom management, delaying disease progression, and reducing the risk of cholangiocarcinoma and liver failure.
Surgical Intervention
Surgical intervention accounts for around 30% of the market, typically reserved for advanced-stage patients. Procedures such as liver transplantation, biliary drainage, and endoscopic therapies are used to manage complications like dominant strictures and end-stage liver disease, often when pharmacological options are no longer effective.
Primary Sclerosing Cholangitis Therapeutics Market, Segmentation by Geography
In this report, the Primary Sclerosing Cholangitis Therapeutics Market has been segmented by Geography into five regions; North America, Europe, Asia Pacific, Middle East and Africa and Latin America.
Regions and Countries Analyzed in this Report
Global Primary Sclerosing Cholangitis Therapeutics Market Share (%), by Geographical Region
North America
North America dominates the PSC therapeutics market with a share of approximately 42%, attributed to a high incidence of inflammatory bowel diseases (IBD), advanced healthcare infrastructure, and strong presence of biopharmaceutical companies. The U.S. leads the region in terms of clinical research and drug pipeline activity.
Europe
Europe holds around 30% of the market, driven by increasing government funding for rare diseases, presence of specialized liver disease centers, and active participation in multinational clinical trials. Countries like Germany and the UK are key contributors to regional growth.
Asia Pacific
Asia Pacific accounts for roughly 15% of the market, with rising awareness of liver disorders, increasing investment in biotechnology, and expanding clinical trial networks. Countries like Japan, China, and South Korea are emerging markets in PSC research and treatment access.
Middle East and Africa
This region represents nearly 7% of the market, with gradual improvements in specialty care facilities and a growing focus on hepatobiliary disease management. Market penetration remains limited due to low disease awareness and healthcare access disparities.
Latin America
Latin America captures about 6% of the global market, supported by improvements in diagnostic capabilities and an increasing number of research collaborations in Brazil, Mexico, and Argentina. However, the availability of advanced therapies remains a challenge across the region.
Market Trends
This report provides an in depth analysis of various factors that impact the dynamics of Global Primary Sclerosing Cholangitis Therapeutics Market. These factors include; Market Drivers, Restraints and Opportunities Analysis.
Comprehensive Market Impact Matrix
This matrix outlines how core market forces—Drivers, Restraints, and Opportunities—affect key business dimensions including Growth, Competition, Customer Behavior, Regulation, and Innovation.
Market Forces ↓ / Impact Areas → | Market Growth Rate | Competitive Landscape | Customer Behavior | Regulatory Influence | Innovation Potential |
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Drivers | High impact (e.g., tech adoption, rising demand) | Encourages new entrants and fosters expansion | Increases usage and enhances demand elasticity | Often aligns with progressive policy trends | Fuels R&D initiatives and product development |
Restraints | Slows growth (e.g., high costs, supply chain issues) | Raises entry barriers and may drive market consolidation | Deters consumption due to friction or low awareness | Introduces compliance hurdles and regulatory risks | Limits innovation appetite and risk tolerance |
Opportunities | Unlocks new segments or untapped geographies | Creates white space for innovation and M&A | Opens new use cases and shifts consumer preferences | Policy shifts may offer strategic advantages | Sparks disruptive innovation and strategic alliances |
Drivers, Restraints and Opportunities Analysis
Drivers
- Increasing prevalence of liver diseases
- Advancements in medical research
- Growing healthcare expenditure globally
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Rising awareness about treatment options - Growing awareness about treatment options is a key driver fueling the global primary sclerosing cholangitis (PSC) therapeutics market. Increased patient education, improved physician engagement, and the dissemination of clinical guidelines are encouraging earlier diagnosis and proactive disease management. Patients and healthcare providers are now more informed about experimental therapies, supportive care strategies, and liver transplant protocols, leading to greater treatment uptake and enhanced disease monitoring.
This rising awareness is also driving demand for participation in clinical trials and investigational drug programs, especially as patients seek alternatives to manage this rare and complex liver disease. Advocacy groups and medical organizations are playing a critical role in disseminating reliable information and connecting patients to emerging therapies. As understanding of PSC grows, so does the momentum for research, investment, and innovation within the therapeutics landscape.
Restraints
- Limited understanding of disease mechanisms
- High cost of treatment
- Stringent regulatory requirements
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Availability of alternative therapies - The availability of alternative therapies remains a significant restraint in the global primary sclerosing cholangitis (PSC) therapeutics market. Although there is no approved curative treatment for PSC, patients often turn to off-label medications, symptomatic management, and lifestyle modifications to control disease progression and alleviate discomfort. These alternatives, while not disease-modifying, may reduce the perceived need for new or experimental therapeutics, particularly in cases where patients show slow disease progression or minimal symptoms.
Furthermore, the presence of non-pharmacological interventions such as endoscopic procedures, bile acid therapies, and liver transplantation reduces dependency on emerging drug candidates. This diversification in care strategies creates a fragmented treatment landscape, limiting the urgency and scale of therapeutic adoption. As long as these alternative approaches remain widely used and accessible, the uptake of novel therapies may face hesitancy from both patients and healthcare providers.
Opportunities
- Emerging markets in Asia-Pacific
- Development of novel therapeutic approaches
- Collaborations for clinical trials
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Personalized medicine and targeted therapies - The rise of personalized medicine and targeted therapies offers a promising opportunity in the global primary sclerosing cholangitis (PSC) therapeutics market. Advances in genomics, immune profiling, and biomarker research are enabling the development of customized treatments that align with individual disease characteristics. This approach is especially valuable in PSC, where disease progression and treatment responses vary significantly among patients, and current treatment options remain limited.
Pharmaceutical companies are increasingly focused on creating biologics and targeted small molecules that act on specific inflammatory and fibrotic pathways. These precision therapies can improve clinical outcomes while minimizing side effects, supporting the transition from generalized care to more effective, patient-specific strategies. As regulatory bodies show growing interest in personalized approaches, this trend is expected to drive both innovation and adoption in PSC treatment development.
Competitive Landscape Analysis
Key players in Global Primary Sclerosing Cholangitis Therapeutics Market include:
- Intercept Pharmaceuticals, Inc
- Gilead Sciences, Inc
- Allergan plc
- Genfit SA
- Novartis AG
- Pfizer Inc
- Merck & Co., Inc
- Takeda Pharmaceutical Company Limited
- Bristol Myers Squibb
- F Hoffmann-La Roche Ltd
In this report, the profile of each market player provides following information:
- Company Overview and Product Portfolio
- Market Share Analysis
- Key Developments
- Financial Overview
- Strategies
- Company SWOT Analysis
- Introduction
- Research Objectives and Assumptions
- Research Methodology
- Abbreviations
- Market Definition & Study Scope
- Executive Summary
- Market Snapshot, By Drug
- Market Snapshot, By Treatment Type
- Market Snapshot, By Region
- Primary Sclerosing Cholangitis Therapeutics Market Dynamics
- Drivers, Restraints and Opportunities
- Drivers
- Increasing prevalence of liver diseases
- Advancements in medical research
- Growing healthcare expenditure globally
- Rising awareness about treatment options
- Restraints
- Limited understanding of disease mechanisms
- High cost of treatment
- Stringent regulatory requirements
- Availability of alternative therapies
- Opportunities
- Emerging markets in Asia-Pacific
- Development of novel therapeutic approaches
- Collaborations for clinical trials
- Personalized medicine and targeted therapies
- Drivers
- PEST Analysis
- Political Analysis
- Economic Analysis
- Social Analysis
- Technological Analysis
- Porter's Analysis
- Bargaining Power of Suppliers
- Bargaining Power of Buyers
- Threat of Substitutes
- Threat of New Entrants
- Competitive Rivalry
- Drivers, Restraints and Opportunities
- Market Segmentation
- Primary Sclerosing Cholangitis Therapeutics Market, By Drug, 2021 - 2031 (USD Million)
- BTT1023
- GS-9674
- NGM282
- OCA
- Cenicriviroc
- LUM001
- DUR928
- norUDCA
- HTD1801
- IDN-7314
- STP705
- Primary Sclerosing Cholangitis Therapeutics Market, By Treatment Type, 2021 - 2031 (USD Million)
- Pharmaceutical Therapy
- Surgical Intervention
- Primary Sclerosing Cholangitis Therapeutics Market, By Geography, 2021 - 2031 (USD Million)
- North America
- United States
- Canada
- Europe
- Germany
- United Kingdom
- France
- Italy
- Spain
- Nordic
- Benelux
- Rest of Europe
- Asia Pacific
- Japan
- China
- India
- Australia & New Zealand
- South Korea
- ASEAN (Association of South East Asian Countries)
- Rest of Asia Pacific
- Middle East & Africa
- GCC
- Israel
- South Africa
- Rest of Middle East & Africa
- Latin America
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- North America
- Primary Sclerosing Cholangitis Therapeutics Market, By Drug, 2021 - 2031 (USD Million)
- Competitive Landscap
- Company Profiles
- Intercept Pharmaceuticals, Inc
- Gilead Sciences, Inc
- Allergan plc
- Genfit SA
- Novartis AG
- Pfizer Inc
- Merck & Co., Inc
- Takeda Pharmaceutical Company Limited
- Bristol Myers Squibb
- F Hoffmann-La Roche Ltd
- Company Profiles
- Analyst Views
- Future Outlook of the Market