CLINICAL TRIALS PROFILE FOR OBETICHOLIC ACID

All Clinical Trials for OBETICHOLIC ACID

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00570765 ↗	Study of INT-747 as Monotherapy in Participants With Primary Biliary Cirrhosis (PBC)	Completed	Intercept Pharmaceuticals	Phase 2	2008-01-17	The primary hypothesis was that obeticholic acid (OCA) will cause a reduction in alkaline phosphatase levels in PBC participants, over a 12-week treatment period, as compared to placebo.
NCT01265498 ↗	The Farnesoid X Receptor (FXR) Ligand Obeticholic Acid in NASH Treatment Trial(FLINT)	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 2	2011-03-01	Administration of the farnesoid X receptor (FXR) ligand obeticholic acid (OCA) for 72 weeks to subjects with biopsy evidence of nonalcoholic steatohepatitis (NASH) will result in improvement in their liver disease as measured by changes in the nonalcoholic fatty liver disease (NAFLD) activity score (NAS).
NCT01473524 ↗	Phase 3 Study of Obeticholic Acid in Patients With Primary Biliary Cirrhosis	Completed	Intercept Pharmaceuticals	Phase 3	2012-01-01	The main objectives of the study were to assess the effects of Obeticholic Acid (OCA) on serum alkaline phosphatase (ALP) and total bilirubin, together as a composite endpoint and on safety in participants with primary biliary cirrhosis (PBC).
NCT01585025 ↗	Obeticholic Acid in Bile Acid Diarrhoea	Completed	Imperial College London	Phase 2	2012-04-01	The investigators propose to develop studies of obeticholic acid (OCA) in patients with bile acid diarrhoea. OCA is a semisynthetic bile acid, also known as 6αethylchenodeoxycholic acid or INT747,and is a potent farnesoid X receptor (FXR) agonist. Preliminary data suggests that patients with bile acid diarrhoea have impaired production of the ileal hormone Fibroblast Growth Factor 19 (FGF19). FGF19 is stimulated by FXR agonists, and regulates bile acid synthesis. This study is a pilot, proof-of-concept, open-label study to investigate whether OCA can stimulate FGF19 in bile acid diarrhoea patients to provide a safe and effective treatment.
NCT01625026 ↗	Obeticholic Acid in Bariatric and Gallstone Disease	Completed	Medical University of Vienna	Phase 2	2013-09-01	By binding to the nuclear receptor FXR, bile acids not only regulate their own turn-over but presumably also pivotal steps in cholesterol, triglyceride and glucose metabolism as shown in laboratory animals. Obeticholic acid (OCA) is a semisynthetic bile acid with very high affinity to FXR. In a pharmacodynamic study the effects of OCA on bile acid, lipid and glucose turn-over are studied in 20 morbidly obese and 20 gallstones patents, respectively, that are administered OCA at 25 mg/day in three weeks before bariatric (BS) or gallstone (GS) surgery where in addition to blood samples also biopsies are taken from the liver and in the case of BS, omental and subcutaneous adipose tissue and in case of GS, gallbladder bile.
NCT01625026 ↗	Obeticholic Acid in Bariatric and Gallstone Disease	Completed	Sahlgrenska University Hospital, Sweden	Phase 2	2013-09-01	By binding to the nuclear receptor FXR, bile acids not only regulate their own turn-over but presumably also pivotal steps in cholesterol, triglyceride and glucose metabolism as shown in laboratory animals. Obeticholic acid (OCA) is a semisynthetic bile acid with very high affinity to FXR. In a pharmacodynamic study the effects of OCA on bile acid, lipid and glucose turn-over are studied in 20 morbidly obese and 20 gallstones patents, respectively, that are administered OCA at 25 mg/day in three weeks before bariatric (BS) or gallstone (GS) surgery where in addition to blood samples also biopsies are taken from the liver and in the case of BS, omental and subcutaneous adipose tissue and in case of GS, gallbladder bile.
NCT01865812 ↗	Phase 2 Study on Effects of Obeticholic Acid (OCA) on Lipoprotein Metabolism in Subjects With Primary Biliary Cirrhosis	Completed	Intercept Pharmaceuticals	Phase 2	2013-11-01	The purpose of this study is to determine if obeticholic acid (OCA) has an effect on cholesterol levels in the blood in patients with PBC.

Clinical Trial Conditions for OBETICHOLIC ACID

Condition Name

Condition Name for OBETICHOLIC ACID
Intervention	Trials
Primary Biliary Cholangitis	7
Liver Cirrhosis, Biliary	4
Primary Biliary Cirrhosis	4
Healthy	3

Condition MeSH

Condition MeSH for OBETICHOLIC ACID
Intervention	Trials
Liver Cirrhosis, Biliary	14
Liver Cirrhosis	8
Fibrosis	8
Fatty Liver	7

Clinical Trial Locations for OBETICHOLIC ACID

Trials by Country

Trials by Country for OBETICHOLIC ACID
Location	Trials
United States	212
United Kingdom	31
Canada	27
Germany	23
Australia	17

Trials by US State

Trials by US State for OBETICHOLIC ACID
Location	Trials
Texas	13
Florida	11
California	11
Virginia	11
Ohio	9

Clinical Trial Progress for OBETICHOLIC ACID

Clinical Trial Phase

Clinical Trial Phase for OBETICHOLIC ACID
Clinical Trial Phase	Trials
PHASE3	3
PHASE2	2
Phase 4	3

Clinical Trial Status

Clinical Trial Status for OBETICHOLIC ACID
Clinical Trial Phase	Trials
Completed	15
Recruiting	12
Not yet recruiting	5

Clinical Trial Sponsors for OBETICHOLIC ACID

Sponsor Name

Sponsor Name for OBETICHOLIC ACID
Sponsor	Trials
Intercept Pharmaceuticals	19
GlaxoSmithKline	2
National Cancer Institute (NCI)	2

Sponsor Type

Sponsor Type for OBETICHOLIC ACID
Sponsor	Trials
Industry	26
Other	15
NIH	4

Last updated: April 27, 2026

Obeticholic Acid (OCA) Clinical Trials Update, Market Analysis, and Projection

Summary: Obeticholic acid (OCA) is an established option for primary biliary cholangitis (PBC) in the US and is in clinical-stage testing for additional cholestatic and metabolic indications. Commercial performance has been constrained by adverse-benefit profiles in some populations and by label-specific adoption patterns. Near-to-midterm growth hinges on (1) broader geographic access and payer uptake for approved indications, and (2) successful readouts in ongoing trials that target fibrosis, earlier disease stages, or complementary mechanisms (FXR agonism with refined safety management).

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What is the current approved footprint for obeticholic acid?

Indications and practical positioning

• Primary biliary cholangitis (PBC)
  • Approved for use in patients with PBC who have an inadequate response to ursodeoxycholic acid (UDCA) or intolerance to UDCA, and in some regions also for use with UDCA depending on local labeling.
  • OCA is an FXR agonist. Adoption typically depends on balancing cholestatic control against the risk of pruritus and dyslipidemia patterns seen in pivotal datasets.
• Cirrhosis and liver safety segmentation
  • OCA use is label-restricted in populations with decompensated cirrhosis and depends on liver status (Child-Pugh class) per region.
  • This segmentation materially affects treatable addressable population and creates a “line of therapy” effect even when efficacy is demonstrated.

Key labels that shape demand

• Payer and guideline behavior tends to favor patients with insufficient UDCA response and preserved liver function where tolerability management is feasible.
• Safety monitoring requirements (lipids, pruritus management, liver function thresholds) increase friction versus UDCA or generic off-label alternatives in some markets.

Source: FDA prescribing information for Ocaliva (obeticholic acid). [1]

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What do the recent and ongoing clinical trial updates indicate?

Core development theme

OCA development continues to test whether FXR agonism can deliver:

• Improved cholestasis markers and fibrosis-related endpoints
• Better benefit-risk in earlier stages or combination regimens
• Usable safety at real-world dosing frameworks

Clinical trials landscape by indication (programmatic view)

OCA’s development has historically targeted a cross-section of:

• Cholestatic liver disease (including PBC variants and related cholangiopathies)
• Nonalcoholic steatohepatitis and metabolic-associated liver disease (MAFLD/MASH) as a category-level hypothesis for FXR agonism
• Liver fibrosis endpoints and surrogate markers translating to clinical benefit

Interpreting the trial posture

Market-relevant implication:

• If trials show fibrosis endpoint improvement with manageable safety, it expands the addressable population beyond PBC to higher-volume fibrosis markets.
• If safety limits persist, incremental value concentrates in label expansion within cholestatic disease and in earlier PBC populations where treatment is still acceptable.

Source: FDA and clinical program materials for obeticholic acid. [1]

Note: This update is constrained to sources that explicitly describe trial status at the level necessary to support a clinical update. Where trial-level results are not directly evidenced in the provided sources, the clinical posture is summarized at the program level without specific readout dates or claims.

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How big is the addressable market for obeticholic acid today?

Commercial demand drivers

For PBC, OCA demand is a function of:

• Number of diagnosed PBC patients with inadequate response or intolerance to UDCA
• Willingness-to-treat in the FXR class despite pruritus and dyslipidemia risks
• Country-by-country reimbursement that can cap adoption if patient selection is strict

Commercial demand constraints

• Safety and tolerability drive discontinuation risk and dose modifications.
• Segmentation by cirrhosis status reduces the practical eligible pool in regions that treat fewer patients with advanced disease.
• Therapy competition in the broader cholestatic and fibrosis space can reduce conversion from baseline supportive care.

Market structure: PBC-first economics

OCA revenue and trajectory are expected to remain PBC-led in the near term given:

• Approval status and entrenched clinical usage pathways
• Established prescriber familiarity compared with investigational fibrosis/MASH programs

Source: Ocaliva prescribing information and approved indication framing. [1]

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What is the forward projection for growth and value capture?

Baseline projection logic

A practical 3-factor model for OCA’s mid-term outlook:

1. Patient pool expansion via label updates and earlier-stage use in PBC where supported by evidence and guidelines
2. Geographic reimbursement broadening and reduced prior-authorization friction
3. Pipeline outcome probability for fibrosis/MAFLD-related indications that, if positive with acceptable safety, moves OCA from PBC into higher-volume liver disease segments

Scenarios for 2026-2029 (directional)

Given the constraints described in labeling and the safety-managed adoption patterns, the directional outcomes are:

• Base case (PBC-centric growth):

  • Growth remains tied to incremental PBC diagnosis and improved payer access.
  • Uptake stays sensitive to pruritus management and lipid monitoring, keeping penetration growth moderate.

• Upside case (successful label expansion into fibrosis-relevant populations):

  • A positive clinical dataset with clear fibrosis-linked benefit and manageable safety extends use beyond strict PBC cohorts.
  • Uptake accelerates because fibrosis endpoints draw broader referral networks (hepatology and transplant-adjacent pathways).

• Downside case (pipeline safety or efficacy limits):

  • Market stays confined to approved PBC populations.
  • Adoption slows further as competitors offer alternatives with simpler dosing or better tolerability.

Source: FDA safety and dosing restrictions as reflected in Ocaliva prescribing information. [1]

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What is the competitive landscape affecting obeticholic acid?

Competition by segment

• PBC treatment options
  • UDCA remains foundational.
  • Other agents for refractory disease in some markets can pressure OCA positioning depending on reimbursement and clinician preference.
• Fibrosis/steatohepatitis space
  • Multiple mechanisms compete for attention and capital.
  • Without robust, clinically meaningful endpoint wins with tolerable safety, OCA’s broader expansion remains harder.

Implications for OCA strategy

• Prescriber adoption is likely to continue emphasizing:
  • Clear criteria (UDCA inadequate response or intolerance)
  • Liver function-based eligibility
  • Active management of pruritus and dyslipidemia

Source: Ocaliva prescribing information for warnings, liver-related restrictions, and monitoring expectations. [1]

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Key takeaways

• OCA demand is PBC-led and constrained by label-based liver status restrictions and tolerability management needs. [1]
• Clinical progress matters most if it supports expansion into fibrosis-relevant or earlier-stage PBC cohorts with durable benefit and manageable adverse events. [1]
• Mid-term value capture is likely to hinge on two levers: payer/geography adoption for approved PBC use and the probability that ongoing programs generate endpoint-relevant wins that justify broader uptake.

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FAQs

1) What pharmacologic mechanism drives obeticholic acid’s clinical use?

OCA is an FXR agonist used to modulate bile acid signaling in cholestatic liver disease. [1]

2) Why does labeling around liver status impact OCA sales?

Because eligibility and dosing are limited in patients with more advanced or decompensated liver disease, shrinking the treatable pool and increasing clinician friction. [1]

3) What safety profile features most affect real-world adoption?

Pruritus and lipid-related adverse effects and monitoring requirements drive patient selection, dose adjustments, and discontinuation risk. [1]

4) What is the most likely growth pathway for OCA?

Near to mid-term growth is most consistent with PBC-focused penetration and label-supported expansion, followed by possible upside from fibrosis- or metabolic-liver indication wins if supported by clinical evidence and safety. [1]

5) How should investors interpret pipeline risk for OCA?

Investors should treat pipeline outcomes as value drivers only when results show clinically meaningful endpoints and safety that supports broader prescribing beyond tightly selected PBC cohorts. [1]

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References

[1] U.S. Food and Drug Administration. Ocaliva (obeticholic acid) prescribing information. (Accessed via FDA label materials).