IBAT inhibitors (Ileal bile acid transporter Inhibitors) - Target Population, Competitive Landscape, and Market Forecast - 2034

Key Highlights:

• Ileal bile acid transporter (IBAT) inhibitors represent a promising class of therapies with demonstrated efficacy across a range of conditions, including primary sclerosing cholangitis (PSC), primary biliary cholangitis (PBC), metabolic dysfunction-associated steatohepatitis (MASH), chronic constipation, biliary atresia, and Alagille syndrome (ALGS). Their potential to address both rare genetic disorders and more prevalent hepatic and gastrointestinal diseases is increasingly being recognized.
• PSC is believed to arise from a genetic predisposition that, when coupled with environmental triggers, initiates complex immune-mediated processes. These interactions may drive lymphocyte infiltration, cholangiocyte injury, and progressive hepatic fibrosis. IBAT inhibitors offer a novel mechanistic approach that may help modulate disease progression in PSC and other cholestatic liver diseases.
• To date, two IBAT inhibitors have received regulatory approval. LIVMARLI has been approved by the FDA for treating cholestatic pruritus in patients with ALGS and PFIC, while BYLVAY/KAYFANDA has received similar approvals for pruritus associated with PFIC and ALGS. These approvals highlight the class's clinical utility in managing pruritus-a debilitating symptom in cholestatic diseases.
• For MASH, liver biopsy remains the only invasive and definitive diagnostic method capable of detecting disease severity and early-stage fibrosis, outperforming non-invasive tools like elastography in identifying early damage.
• The commercial potential of IBAT inhibitors is underscored by the 2024 U.S. market size of Volixibat, estimated at approximately USD 350 million. This growing interest is reflected in active development efforts by major pharmaceutical players, including GlaxoSmithKline, Mirum Pharmaceuticals, Ipsen Pharma, Albireo, Takeda, and others.
• Overall, IBAT inhibitors are positioned as a transformative therapeutic class with broad application across hepatic, metabolic, and autoimmune diseases. As ongoing clinical studies mature, a clearer therapeutic role will emerge, potentially establishing IBAT inhibitors as foundational treatments for several high-need indications.

DelveInsight's "IBAT inhibitors (Ileal bile acid transporter Inhibitors) - Target Population, Competitive Landscape, and Market Forecast - 2034" report delivers an in-depth understanding of the IBAT inhibitors, historical and competitive landscape as well as its market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The IBAT inhibitors market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM IBAT inhibitors market size from 2020 to 2034. The report also covers current IBAT inhibitors treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

IBAT inhibitors Understanding and Treatment Algorithm

IBAT inhibitors Overview

IBAT inhibitors are administered orally, with most being gut-restricted agents that exhibit minimal systemic absorption. Plasma concentrations often fall below the limit of quantification following single or multiple doses within the recommended range. However, systemically absorbed IBAT inhibitors are currently in development.

One potential limitation of gut-restricted IBAT inhibitors is reduced efficacy in cases of complete or near-complete biliary obstruction, where bile acids fail to reach the small intestine in significant amounts. This issue is particularly relevant in pediatric patients, where biliary atresia is the most common cause of cholestasis. To overcome this limitation, systemically active ASBT inhibitors have been developed. ASBTs are also expressed in the proximal renal tubules, where they play a role in reabsorbing small amounts of bile acids filtered by the glomerulus.

IBAT inhibitors Market Overview

In recent years, there has been a growing interest in the exploration of IBAT inhibitors for a variety of inflammatory disorders, encompassing cholestatic liver diseases, biliary atresia, chronic constipation, ALGS, PBC, MASH, and other indications.

IBAT inhibitors Epidemiology

The epidemiology chapter of IBAT inhibitors in the report provides historical as well as forecasted epidemiology segmented as total cases in selected indications for IBAT inhibitors, total eligible patient pool in selected indications for IBAT inhibitors, and total treated cases in selected indications for IBAT inhibitors in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), and the United Kingdom, and Japan from 2020 to 2034.

• The diagnosed prevalent cases of MASH in the 7MM in 2024 were nearly 16,106,000.
• In the US, about 88% of patients with ALGS are estimated to experience pruritus, with around 45% reported as severe.
• Among the EU4 and the UK, Germany accounted for the highest number of cases of PSC, followed by the UK and the lowest number of cases in Spain in 2024.
• Japan accounted for nearly 2,570,000 diagnosed prevalent cases of MASH in 2024.
• In 2024, there were about 9,400 males and 37,000 females were diagnosed with PBC in Japan.

IBAT Inhibitors Drug Chapters

The drug chapter segment of the IBAT inhibitors report encloses a detailed analysis of approved IBAT inhibitors, late-stage (Phase III and Phase II) IBAT inhibitors. It also helps understand the clinical trial details of IBAT inhibitors, 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 IBAT inhibitors

LIVMARLI (maralixibat chloride): Mirum Pharmaceuticals/Takeda

LIVMARLI is an orally administered, IBAT inhibitor approved by the US Food and Drug Administration for two pediatric cholestatic liver diseases, in both liquid and tablet formulations. It is approved for the treatment of cholestatic pruritus in patients with ALGS in the US three months of age and older and in Europe for patients two months of age and older. It is also approved in the US for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. LIVMARLI is currently being evaluated in the Phase III EXPAND study in additional settings of cholestatic pruritus, expected to complete enrollment in 2026.

• LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC2 (a subtype of PFIC) in the US and Orphan Drug designation (ODD) for ALGS and PFIC in the US and Europe. In Japan, the MHLW granted ODD for the expected indications of ALGS and PFIC in December 2022.
• In April 2025, Mirum Pharmaceuticals announced the US FDA approved a new tablet formulation of LIVMARLI for the treatment of ALGS and PFIC. The tablets are planned to be available in June 2025 through Mirum Access Plus.
• In November 2024, Mirum Pharmaceuticals announced the presentation of LIVMARLI data at the American Association for the Study of Liver Disease's (AASLD) Liver Meeting.

BYLVAY/KAYFANDA (odevixibat): Ipsen Pharma

BYLVAY is a potent, once-daily, non-systemic IBAT inhibitor that acts locally in the small intestine and has minimal systemic exposure. It is approved in the US for the treatment of pruritus in patients three months of age and older with PFIC, where it has orphan exclusivity. BYLVAY was first launched as a treatment option for patients with PFIC in the US in 2021, where it is supported by a program designed to assist with access to treatment and patient support. BYLVAY is also approved in the EU for the treatment of PFIC in patients aged six months or older. It has launched in over nine countries and has secured public reimbursement across several major markets including Germany, Italy, the UK, France, and Belgium.

• In September 2024, Ipsen announced that the European Commission has approved KAYFANDA under exceptional circumstances for the treatment of cholestatic pruritus in ALGS in patients aged 6 months or older.
• In June 2023, Ipsen announced that the US FDA has approved BYLVAY for the treatment of cholestatic pruritus in patients from 12 months of age with ALGS.

Emerging Drugs IBAT inhibitors

Volixibat: Mirum Pharmaceuticals

Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the IBAT. Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase IIb studies for PSC (VISTAS study), and PBC (VANTAGE study).

• As per Mirium's 2024 annual report, the volixibat VISTAS study in PSC is expected to complete enrollment in the second half of 2025 with topline data expected in 2026. Also, volixibat VANTAGE study in PBC is expected to complete enrollment in 2026.
• In November 2024, Mirum Pharmaceuticals presented volixibat data at the AASLD.

Linerixibat: GlaxoSmithKline (GSK)

Linerixibat is an IBAT inhibitor, a targeted oral agent with the potential to treat cholestatic pruritus (itch) associated with the rare autoimmune liver disease known as PBC. By inhibiting bile acid re-uptake, linerixibat aims to address a root cause of cholestatic pruritus. The US FDA and the European Medicines Agency (EMA) have granted ODD for linerixibat in the treatment of cholestatic pruritus associated with PBC.

• In November 2024, GSK announced positive headline results of GLISTEN, the ongoing global Phase III clinical trial evaluating linerixibat, an investigational targeted inhibitor of the IBAT, in adults with cholestatic pruritus (relentless itch) associated with PBC, a rare autoimmune liver disease.
• As per the Q4 report, linerixibat anticipated regulatory submission in the US, Europe, and China, in H1 2025 for treatment of cholestatic pruritus in PBC. The company also anticipates US regulatory decision and Japan regulatory submission in H2 2025. The regulatory decisions for Europe, Japan, and China are expected in 2026.

IBAT Inhibitors Market Outlook

The global IBAT inhibitors market is expected to witness substantial growth in the coming years, driven by the increasing prevalence of genetic diseases, robust clinical pipeline activity, and expanding regulatory approvals.

Several key players, including GlaxoSmithKline, Mirum Pharmaceuticals, Ipsen Pharma, Albireo, Takeda, and others, are involved in developing drugs for IBAT inhibitors such as PBC, PSC, ALGS, PFIC, and others, respectively. Overall, this is an exciting new class with great potential for development. The maturation of current studies over the next few years will lead to a better understanding of IBAT inhibitors and define their role in rare genetic disorders.

IBAT inhibitors Drugs Uptake

This section focuses on the uptake rate of potential approved and emerging IBAT inhibitors expected to be launched in the market during 2025-2034.

IBAT Inhibitors 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.

The presence of numerous drugs under different stages is expected to generate immense opportunity for IBAT inhibitors market growth over the forecast period.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for IBAT inhibitors therapies.

KOL Views

To keep up with current and future market trends, we take Industry Experts' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on IBAT inhibitors evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, drug uptake, along challenges related to accessibility.

DelveInsight's analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as the University of Pennsylvania, University of Colorado, University of Salerno, Hospital Papa Giovanni XXIII, and others.

Their opinion helps understand and validate current and emerging therapy treatment patterns or IBAT inhibitors market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT 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.

Market Access and Reimbursement

Reimbursement may be referred to as the negotiation of a price between a manufacturer and payer that allows the manufacturer access to the market. It is provided to reduce the high costs and make the essential drugs affordable. Health Technology Assessment (HTA) plays an important role in reimbursement decision-making and recommending the use of a drug. These recommendations vary widely throughout the seven major markets, even for the same drug.

In the US healthcare system, both Public and Private health insurance coverage are included. Also, Medicare and Medicaid are the largest government-funded programs in the US. The major healthcare programs including Medicare, Continuing Medical Education (CME) program, the Children's Health Insurance Program (CHIP), and the state and federal health insurance marketplaces are overseen by the Centers for Medicare & Medicaid Services (CMS). Other than these, Pharmacy Benefit Managers (PBMs), and third-party organizations that provide services, and educational programs to aid patients are also present.

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

Key Updates on IBAT Inhibitors

• In March 2025, Takeda announced that Japan's MHLW approved LIVMARLI Oral Solution 10 mg/mL for the treatment of pruritus associated with cholestasis in ALGS and PFIC.
• In November 2024, Ipsen announced data at the AASLD assessing the long-term efficacy and safety of patients treated with BYLVAY from two Phase III open-label extension studies in PFIC and ALGS.
• In October 2024, Mirum Pharmaceuticals that the FDA granted a breakthrough therapy designation (BTD) to volixibat, as a possible treatment for cholestatic pruritus in patients with PBC. Volixibat, also received FTD from the FDA in 2016 for the treatment of NASH in patients with liver fibrosis.
• The abstract list is not exhaustive, will be provided in the final report

Scope of the Report:

• The report covers a segment of key events, an executive summary, and a descriptive overview of the IBAT inhibitors, explaining its mechanism, and therapies (current and emerging).
• Comprehensive insight into the competitive landscape, and forecasts, the future growth potential of treatment rate, drug uptake, and drug information 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 landscape.
• A detailed review of the IBAT inhibitors market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis, expert insights/KOL views, and treatment preferences that help shape and drive the 7MM IBAT inhibitors market.

IBAT Inhibitors Report Insights

• IBAT Inhibitors Targeted Patient Pool
• Therapeutic Approaches
• IBAT Inhibitors Pipeline Analysis
• IBAT Inhibitors Market Size and Trends
• Existing and Future Market Opportunities

IBAT Inhibitors Report Key Strengths

• 10 years Forecast
• The 7MM Coverage
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

IBAT Inhibitors Report Assessment

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

Key Questions:

• What was the total market size of IBAT inhibitors, the market size by therapies, market share (%) distribution, and what would it look like in 2034? What are the contributing factors for this growth?
• Which drug is going to be the largest contributor in 2034?
• Which is the most lucrative market for IBAT inhibitors?
• What are the pricing variations among different geographies for approved therapies?
• How has the reimbursement landscape for IBAT inhibitors evolved since the first one was approved? Do patients have any access issues that are driven by reimbursement decisions?
• What are the risks, burdens, and unmet needs of treatment with IBAT inhibitors? What will be the growth opportunities across the 7MM for the patient population of IBAT inhibitors?
• What are the key factors hampering the growth of the market for IBAT inhibitors?
• What are the indications for which recent novel therapies and technologies have been developed to overcome the limitations of existing treatments?
• What key designations have been granted to the therapies for IBAT inhibitors?
• What is the cost burden of approved therapies on the patient?
• Patient acceptability in terms of preferred therapy options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies?

Reasons to buy:

• The report will help develop business strategies by understanding the latest trends and changing dynamics driving the IBAT inhibitors market.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain) the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of indication-wise current and emerging therapies under the conjoint analysis section to provide visibility around leading indications.
• Highlights of access and reimbursement policies of approved therapies, barriers to accessibility of expensive off-label therapies, 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.