Cholestatic Pruritus - Market Insight, Epidemiology And Market Forecast - 2034

Key Highlights:

• According to DelveInsight's estimates, there were approximately 98,000 total diagnosed prevalent cases of cholestatic pruritus in liver diseases in the US in 2024 and these cases are expected to increase during the forecast period (2025-2034).
• The cholestatic pruritus market is projected to see consistent growth, with a robust compound annual growth rate (CAGR) anticipated from 2025 to 2034. This expansion across the US will be driven by the introduction of innovative therapies, Linerixibat, Volixibat, and TH104, among others. Furthermore, the rising prevalence of cholestatic pruritus, driven by increased disease awareness, improved diagnostic capabilities, earlier detection of underlying liver disorders, and an aging population, will contribute to greater demand for effective treatments.
• According to DelveInsight's analysis, the cholestatic pruritus market in the US was valued at approximately USD 300 million in 2024. Over the forecast period from 2025 to 2034, this market is projected to grow at a CAGR of 23.4%.
• Currently, a range of treatments for managing cholestatic pruritus is available in the market. These include bile acid sequestrants (e.g., cholestyramine, colestipol, colesevelam), rifampicin, µ-opioid receptor antagonists (e.g., naltrexone), selective serotonin reuptake inhibitors (e.g., sertraline), as well as targeted therapies such as LIVMARLI (maralixibat) and BYLVAY (odevixibat) for pediatric cholestatic liver disorders.
• Patient adherence to cholestatic pruritus therapy is often poor due to limited tolerability, complex regimens, and insufficient supportive care, while incomplete understanding of its multifactorial pathophysiology further hampers development of effective targeted treatments, leaving symptoms inadequately controlled and quality of life impacted.
• GSK, Mirum Pharmaceuticals, and Tharimmune among others are progressing their assets through various clinical trial phases, driving innovation in the cholestatic pruritus market and creating significant growth opportunities.
• In June 2025, the US FDA accepted the NDA for GSK's linerixibat, an investigational IBAT inhibitor for cholestatic pruritus in PBC, with a PDUFA goal date of 24 March 2026.

DelveInsight's "Cholestatic Pruritus - Market Insights, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of cholestatic pruritus, historical and forecasted epidemiology, as well as the cholestatic pruritus market trends in the United States.

The cholestatic pruritus market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted US cholestatic pruritus market size from 2020 to 2034. The report also covers cholestatic pruritus treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Cholestatic Pruritus Understanding and Treatment Algorithm

Cholestatic pruritus overview

Cholestatic pruritus, a frequent and burdensome symptom of liver diseases marked by impaired bile secretion or bile flow obstruction, arises from diverse mechanisms including bile duct injury, reduced hepatic bile secretion, or intrahepatic and extrahepatic obstruction. Clinically significant yet often underrecognized, it commonly affects the palms and soles, worsens at night, and contributes to sleep disturbance, fatigue, and reduced quality of life, while current treatments remain inadequate. Although its pathophysiology is not fully understood, evidence suggests a multifactorial mechanism in which liver-derived pruritogens-such as bile acids, serotonin, progesterone derivatives, endogenous opioids, and lysophosphatidic acids-activate primary itch neurons in the skin, transmitting signals to the central nervous system.

Cholestatic pruritus diagnosis

Diagnosis of cholestatic pruritus relies on identifying itch in the context of established cholestasis while systematically excluding other potential causes. The broad differential-including dermatologic, systemic, metabolic, drug-induced, infectious, neoplastic, and psychiatric conditions-highlights the complexity of attributing pruritus solely to cholestasis and underscores the importance of a targeted yet comprehensive clinical evaluation.

Cholestatic pruritus treatment

Management of cholestatic pruritus requires a stepwise approach combining general measures, targeted pharmacologic therapy, and treatment of the underlying liver disease. First-line interventions include colestyramine, followed by rifampicin, µ-opioid receptor antagonists, and selective serotonin reuptake inhibitors for refractory cases. Severe, unresponsive pruritus may necessitate liver transplantation or specialized interventions, highlighting the complexity and unmet need in effective symptom control.

Cholestatic Pruritus Epidemiology

As the market is derived using a patient-based model, the cholestatic pruritus epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total diagnosed prevalent cases of liver diseases and total diagnosed prevalent cases of cholestatic pruritus in liver diseases in the US from 2020 to 2034.

• According to DelveInsight analysis, the US had an estimated 108,000 PBC cases in 2024, making it the most prevalent rare cholestatic liver disease, followed by roughly 30,000 PSC cases. The overall burden of PBC, PSC, PFIC, ALGS, and biliary atresia is expected to increase steadily from 2025 to 2034, reflecting growing recognition, earlier diagnosis, and the need for expanded therapeutic strategies.
• According to DelveInsight analysis, cholestatic pruritus posed a significant clinical burden across rare liver diseases in the US in 2024, with approximately 76,000 cases in PBC, followed by around 18,000 in PSC, more than 1,600 each in ALGS and biliary atresia, and roughly 650 in PFIC. While prevalence varies by disease, PBC accounts for the largest share due to its higher occurrence in adults, whereas PFIC, ALGS, and biliary atresia remain comparatively rare.

Cholestatic Pruritus Drug Chapters

The drug chapter segment of the cholestatic pruritus report encloses a detailed analysis of cholestatic pruritus-marketed drugs and pipeline drugs. It also helps understand the cholestatic pruritus clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Marketed Drugs

LIVMARLI (maralixibat): Mirum Pharmaceuticals

LIVMARLI (maralixibat) is an IBAT inhibitor that reduces reabsorption of bile salts, lowering serum bile acid levels, which is believed to alleviate pruritus in patients with ALGS and PFIC. LIVMARLI is approved in the US for treating cholestatic pruritus in patients with ALGS aged 3 months and older and in patients with PFIC aged 12 months and older. A Phase III clinical trial is ongoing to evaluate LIVMARLI in other ultra-rare cholestatic conditions, including biliary atresia and secondary sclerosing cholangitis, with enrollment expected to complete in 2026.

In the US, LIVMARLI regulatory milestones include FDA approvals for PFIC in March 2024 (supported by Phase III MARCH study), ALGS from three months of age in March 2023 (based on RISE study data), and a new tablet formulation for both ALGS and PFIC in April 2025. The initial ALGS approval was in September 2021 for patients aged 1 year and older. LIVMARLI has also received US FDA orphan drug and breakthrough therapy designations for ALGS and PFIC. In November 2024, Mirum Pharmaceuticals presented updated clinical data from the LIVMARLI program at the American Association for the Study of Liver Diseases (AASLD) annual Liver Meeting.

BYLVAY (odevixibat): Ipsen

BYLVAY (odevixibat) is an IBAT inhibitor approved in the US for the treatment of cholestatic pruritus in patients with PFIC aged 3 months and older and in patients with ALGS aged 12 months and older. Its efficacy may be limited in a subset of PFIC Type II patients with ABCB11 gene variants resulting in non-functional or absent BSEP protein. BYLVAY is also being evaluated in a Phase III trial for biliary atresia, with data readouts expected in 2026.

In the US, BYLVAY received FDA approval for PFIC-related pruritus in July 2021 and for ALGS-related pruritus in June 2023. The therapy has received FDA Orphan Drug Designations (ODD) for both PFIC and ALGS and was granted Priority Review for PFIC in 2021. Ipsen presented data at AASLD 2024 demonstrating sustained improvements in severe itch and serum bile acid levels in patients with PFIC and ALGS. In March 2023, Ipsen acquired Albireo Pharma, the developer of BYLVAY, to expand its rare liver disease portfolio.

Emerging Drugs

Linerixibat (GSK2330672): GSK

Linerixibat, an oral IBAT inhibitor, is under development for the treatment of cholestatic pruritus in PBC, a rare autoimmune liver disease. By inhibiting bile acid reuptake, it addresses a key underlying mechanism of pruritus. The drug is also being evaluated in a long-term Phase III study to assess safety and tolerability in patients with PBC.

Phase III GLISTEN trial results from May 2025 demonstrated rapid and sustained improvements in pruritus and sleep disturbance in PBC patients. The therapy received ODD from the FDA in 2019, and GSK presented data from the GLISTEN trial at AASLD 2024, highlighting its clinical potential in this patient population.

Volixibat: Mirum Pharmaceuticals

Volixibat is an oral, minimally absorbed IBAT inhibitor under investigation for adult cholestatic diseases, aiming to reduce systemic and hepatic bile acid levels and address disease-related complications. It is currently being evaluated in Phase IIb studies for cholestatic pruritus, including the VISTAS study in PSC and the VANTAGE study in PBC, with VISTAS completing enrollment in Q3 2025 and topline data expected in Q2 2026, and VANTAGE projected to complete enrollment in 2026. Interim results from these studies were presented in June 2024 and at AASLD 2024, demonstrating the drug's potential to improve pruritus in PBC and PSC.

Volixibat received US FDA ODD and Breakthrough Therapy Designation (BTD) for PBC in October 2024. The drug is being developed by Mirum Pharmaceuticals under exclusive global licenses from Shire International (later acquired by Takeda) and Sanofi-Aventis, covering development, commercialization, manufacturing, and sublicensing. Volixibat holds US composition of matter patent protection until 2027, with additional method-of-treatment and dosing patents pending that could extend coverage to 2042.

TH104: Tharimmune

TH-104 (formerly AV104) is a proprietary transmucosal buccal film embedding nalmefene, targeting µ-opioid and kappa opioid receptors to modulate pruritogenic pathways. Phase I results support Phase II readiness in the US, with positive FDA feedback and completed Clinical Chemistry, Manufacturing, and Controls (CMC). The Phase II study of TH-104 targets moderate-to-severe pruritus in PBC, referred to by the company as a hepatic impairment study, which is planned to be initiated in 2026, while also supporting a near-term NDA filing for prophylaxis against ultrapotent opioid exposure, with new patents extending protection until at least 2040.

Tharimmune presented Phase I data at AASLD's The Liver Meeting, showing a correlation between blood levels and symptom relief, with good tolerability and no unexpected adverse events. Under the Avior Patent License Agreement, Tharimmune holds exclusive worldwide rights to develop and commercialize TH-104, with obligations including USD 0.4M upfront, USD 0.15M quarterly fees, up to USD 24.25M in development milestones, additional sales-based milestones, and low to mid-single digit royalties.

Drug Class Insights

Current drug-based treatments for cholestatic pruritus follow a stepwise approach. First-line therapy is bile acid sequestrants like cholestyramine, which reduce enterohepatic bile salt recirculation. Second-line is rifampicin, a pregnane X receptor activator that lowers serum autotaxin and lysophosphatidic acid, with hepatotoxicity monitoring. Third-line are µ-opioid receptor antagonists such as naltrexone, which counter enhanced opioid signaling, and fourth-line are SSRIs like sertraline for central itch modulation. Except cholestyramine, these drugs are off-label but commonly used.

For refractory pruritus, advanced options include extracorporeal liver support (MARS), plasmapheresis, ultraviolet B phototherapy, and nasobiliary drainage. If all therapies fail and quality of life is severely impacted, liver transplantation may be required.

Market Outlook

Cholestatic pruritus arises from impaired bile flow and accumulation of pruritogens such as bile acids and lysophosphatidic acids, with symptoms influenced by circadian, hormonal, and environmental factors. Management involves addressing the underlying liver disease and symptomatic relief. Initial therapy includes bile acid sequestrants like cholestyramine and antihistamines for mild symptoms, while UDCA is used in PBC and ICP with limited pruritus relief. Patients unresponsive to first-line therapy may receive rifampicin, opioid antagonists, or SSRIs. Fibrates, including bezafibrate and PPAR agonists like seladelpar, have shown efficacy in reducing both liver injury and pruritus, though monitoring for adverse effects is necessary. IBAT inhibitors such as LIVMARLI and BYLVAY specifically target bile acid circulation, showing substantial benefit in pediatric cholestatic disorders, with age-specific US FDA approvals and additional efficacy limitations in certain PFIC subtypes.

Advanced interventions include UVB phototherapy, extracorporeal liver support, and liver transplantation for refractory cases. Observational data indicate conventional therapies remain widely used: bile acid-binding resins dominate PSC pruritus management, while antihistamines are most common in PBC, with limited use of rifampicin, sertraline, and other agents. Emerging therapies like linerixibat, volixibat, and TH-104 focus on targeting pruritus mechanisms. Linerixibat demonstrated rapid and sustained itch relief in the GLISTEN trial and has a PDUFA goal in March 2026. Volixibat showed significant pruritus reduction in Phase IIb VANTAGE (PBC) and VISTAS (PSC) studies, supporting its continued development with topline results expected in Q2 2026.

LIVMARLI was first approved in September 2021 for cholestatic pruritus in patients with ALGS aged 1 year and older, and in March 2024 for PFIC patients aged 5 years and older, with label expansion to include infants as young as 12 months. BYLVAY was first approved in July 2021 for PFIC patients aged 3 months and older, followed by approval in June 2023 for cholestatic pruritus in patients with ALGS aged 12 months and older.

Companies like GSK's Linerixibat, Mirum's Volixibat, and Tharimmune's TH104, among others are involved in the development of Phase III and Phase II drugs.

• The total market size of cholestatic pruritus in the US was approximately USD 300 million in 2024 and is projected to increase during the forecast period (2025-2034) due to the launch of emerging therapies.
• Estimates suggest that linerixibat is expected to generate approximately USD 840 million in the US by 2034.

Cholestatic Pruritus Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2020-2034.

Cholestatic Pruritus Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I. It also analyzes key players involved in developing targeted therapeutics.

Pipeline development activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for emerging therapies for cholestatic pruritus.

KOL Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on cholestatic pruritus evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Medical/scientific writers, Medical Professionals, Professors, Directors, and Others.

DelveInsight's analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the US. Centers like the New York Medical College Valhalla, US, Baylor Scott and White Medical Center, US, Johns Hopkins University School of Medicine, US, California Pacific Medical Center, US, Vanderbilt University Medical Center, US, among others, were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or cholestatic pruritus market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Physician's View

As per the KOLs from the US, cholestatic pruritus represents a multifactorial consequence of impaired bile flow, driven by the synergistic interplay of bile acids, lysophosphatidic acid, and bilirubin. No single mediator fully explains symptom severity, emphasizing the need for therapies that address the complex pathophysiology rather than isolated targets.

As per the KOLs from the US, cholestyramine remains the cornerstone first-line therapy for cholestatic pruritus, functioning through bile acid sequestration to alleviate symptoms. However, its inconsistent clinical efficacy and frequent gastrointestinal adverse effects limit broader tolerability, reflecting persistent unmet needs in effective symptom management.

As per the KOLs from the US, Before IBAT inhibitors, treatment options for pruritus were largely inadequate, often leaving patients to progress to liver transplantation. The primary therapeutic goal focused on improving quality of life, which is heavily impacted by pruritus. Although the patient population is small, the disease burden remains substantial. The introduction of IBAT inhibitors has fundamentally shifted the treatment paradigm, offering a targeted approach that significantly alleviates symptoms and improves patient outcomes.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Conjoint Analysis analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

To analyze the effectiveness of these therapies, have calculated their attributed analysis by giving them scores based on their ability to improve atrial and ventricular dimension/function and ability to regulate heart rate.

Further, the therapies' safety is evaluated wherein the adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials, which directly affects the safety of the molecule in the upcoming trials. It sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

? LIVMARLI

Mirum Access Plus Support Program

Approximately 94% of patients are approved by insurance, and 98% pay USD 10 or less per fill. LIVMARLI is covered by 100% of insurance plans as of January, 2023.

The Mirum Access Plus Savings Program allows eligible commercially insured patients to pay as little as USD 10 per fill (not available for patients using government-funded insurance). For patients without insurance, the Mirum Patient Assistance Program provides LIVMARLI at no cost for up to one calendar year, with an annual eligibility review.

The report provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenarios, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report:

• The report covers a segment of key events, an executive summary, and a descriptive overview of cholestatic pruritus, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines have been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the cholestatic pruritus market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the US drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the US cholestatic pruritus market.

Cholestatic Pruritus report insights

• Patient Population
• Therapeutic Approaches
• Cholestatic Pruritus Pipeline Analysis
• Cholestatic Pruritus Market Size and Trends
• Existing and Future Market Opportunity

Cholestatic Pruritus report key strengths

• 10 years Forecast
• The US Coverage
• Cholestatic Pruritus Epidemiology Segmentation
• Key Cross Competition
• Attribute analysis
• Drugs Uptake and Key Market Forecast Assumptions

Cholestatic Pruritus report assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Attribute Analysis)

Key Questions:

Market Insights

• What was the total market size of cholestatic pruritus, the market size of cholestatic pruritus by therapies, and market share (%) distribution in 2020, and what would it look like by 2034? What are the contributing factors for this growth?
• How will linerixibat affect the treatment paradigm of cholestatic pruritus?
• How will LIVMARLI compete with other upcoming products and marketed therapies?
• Which drug is going to be the largest contributor by 2034?
• What are the pricing variations among different geographies for approved and marketed therapies?
• How would future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights

• What are the disease risks, burdens, and unmet needs of cholestatic pruritus? What will be the growth opportunities across the US with respect to the patient population pertaining to cholestatic pruritus?
• What is the historical and forecasted cholestatic pruritus patient pool in the United States?
• Out of the countries mentioned above, which country would have the highest diagnosed prevalent cholestatic pruritus population during the forecast period (2025-2034)?
• What factors are contributing to the growth of cholestatic pruritus cases?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies

• What are the current options for the treatment of cholestatic pruritus? What are the current clinical and treatment guidelines for treating cholestatic pruritus?
• How many companies are developing therapies for the treatment of cholestatic pruritus?
• How many emerging therapies are in the mid-stage and late stage of development for treating cholestatic pruritus?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
• What is the cost burden of current treatment on the patient?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the accessibility issues of approved therapy in the US?
• What is the US historical and forecasted market of cholestatic pruritus?

Reasons to Buy:

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the cholestatic pruritus market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• The distribution of historical and current patient share is based on real-world prescription data in the US.
• Identifying upcoming solid players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• Highlights of Access and Reimbursement policies for cholestatic pruritus, barriers to accessibility of approved therapy, and patient assistance programs.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.