CLINICAL TRIALS PROFILE FOR OCALIVA

All Clinical Trials for OCALIVA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04939051 ↗	Obeticholic Acid for Prevention in Barrett's Esophagus	Not yet recruiting	National Cancer Institute (NCI)	Phase 2	2021-12-08	This phase II trial studies the effect of obeticholic acid in treating patients with Barrett's esophagus. Bile acids present in duodenogastroesophageal reflux contribute to neoplastic progression in Barrett's esophagus. Obeticholic acid, may help increase bile flow from the liver while suppressing bile acid production, therefore reducing the exposure of the liver to toxic levels of bile acids which is potentially linked to cancer development.
NCT05112822 ↗	Testing Obeticholic Acid (OCA) for Familial Adenomatous Polyposis (FAP)	Not yet recruiting	M.D. Anderson Cancer Center	Phase 1	2022-03-31	This is a trial that intends to evaluate the effect of treatment with the drug obeticholic acid in the treatment of the Familial Adenomatous Polyposis condition.
NCT05223036 ↗	Testing Obeticholic Acid for Familial Adenomatous Polyposis	Not yet recruiting	National Cancer Institute (NCI)	Phase 2	2022-07-11	This phase IIa trial investigates if giving obeticholic acid (OCA) has an effect on the number of polyps in the small bowel and colon in patients with familial adenomatous polyposis (FAP). FAP is a rare gene defect that increases the risk of developing cancer of the intestines and colon. OCA is a drug similar to bile acids, a fluid made and released by the liver. It binds to a receptor in the intestine that is believed to have a positive effect on preventing cancer development. OCA has been effective in treating primary biliary cholangitis (PBC), a liver disease, and is approved by the Food and Drug Administration (FDA). There have been studies showing that OCA decreases inflammation and fibrosis. However, it is not yet known whether OCA works on reducing the number of polyps in patients with FAP.
NCT05740631 ↗	The Effect of Obeticholic Acid in Healthy Volunteers	Recruiting	Intercept Pharmaceuticals	N/A	2022-08-22	This study investigates whether obeticholic acid affects gut microbiota, gastric motility, accommodation, and gastrointestinal peptide in healthy subjects. This study is a single-blind, placebo-controlled, randomized study. Twelve healthy volunteers will take one tablet containing obeticholic acid (10 mg) or a placebo once per day for 21 days for the first intervention. After 28 days washout period, they will take a tablet different from the one taken in the first intervention. Before the first intervention, they will receive MMC, IGP and endoscopy exam. At the end of the first and second interventions, they will receive the same exams. During MMC&IGP, we will take blood samples to measure plasma hormones (motilin, octa-ghrelin, GLP-1, GLP-2, GIP, and insulin) and take duodenal fluid. When the endoscopy, we will take samples from the duodenal for microbiota, permeability measurement, RNA, protein, and pathology. Before and during the interventions, stool samples will be collected.
NCT05740631 ↗	The Effect of Obeticholic Acid in Healthy Volunteers	Recruiting	Universitaire Ziekenhuizen KU Leuven	N/A	2022-08-22	This study investigates whether obeticholic acid affects gut microbiota, gastric motility, accommodation, and gastrointestinal peptide in healthy subjects. This study is a single-blind, placebo-controlled, randomized study. Twelve healthy volunteers will take one tablet containing obeticholic acid (10 mg) or a placebo once per day for 21 days for the first intervention. After 28 days washout period, they will take a tablet different from the one taken in the first intervention. Before the first intervention, they will receive MMC, IGP and endoscopy exam. At the end of the first and second interventions, they will receive the same exams. During MMC&IGP, we will take blood samples to measure plasma hormones (motilin, octa-ghrelin, GLP-1, GLP-2, GIP, and insulin) and take duodenal fluid. When the endoscopy, we will take samples from the duodenal for microbiota, permeability measurement, RNA, protein, and pathology. Before and during the interventions, stool samples will be collected.
NCT06715319 ↗	Obeticholic Acid Versus Placebo, in Combination with Ursodeoxycholic Acid in Patients with Primary Biliary Cirrhosis	COMPLETED	Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd.	PHASE3	2021-10-09	This is a Phase III, randomized, double-blind, multicentre study assessing the efficacy and safety of obecholic acid and Ursodeoxycholic Acid(UDCA) compared with placebo and UDCA in treating of primary biliary cirrhosis (PBC) in adults with an inadequate response to UDCA.

Clinical Trial Conditions for OCALIVA

Condition Name

Condition Name for OCALIVA
Intervention	Trials
Familial Adenomatous Polyposis	2
Attenuated Familial Adenomatous Polyposis	1
Barrett Esophagus	1
Colorectal Carcinoma	1

Condition MeSH

Condition MeSH for OCALIVA
Intervention	Trials
Colorectal Neoplasms	2
Adenomatous Polyposis Coli	2
Nasopharyngeal Neoplasms	2
Barrett Esophagus	1

Clinical Trial Locations for OCALIVA

Trials by Country

Trials by Country for OCALIVA
Location	Trials
United States	5
Puerto Rico	1
China	1
Belgium	1

Trials by US State

Trials by US State for OCALIVA
Location	Trials
Texas	1
Ohio	1
Michigan	1
Massachusetts	1
Arizona	1

Clinical Trial Progress for OCALIVA

Clinical Trial Phase

Clinical Trial Phase for OCALIVA
Clinical Trial Phase	Trials
PHASE3	1
Phase 2	2
Phase 1	1

Clinical Trial Status

Clinical Trial Status for OCALIVA
Clinical Trial Phase	Trials
Not yet recruiting	3
COMPLETED	1
Recruiting	1

Clinical Trial Sponsors for OCALIVA

Sponsor Name

Sponsor Name for OCALIVA
Sponsor	Trials
National Cancer Institute (NCI)	2
M.D. Anderson Cancer Center	1
Intercept Pharmaceuticals	1

Sponsor Type

Sponsor Type for OCALIVA
Sponsor	Trials
NIH	2
Other	2
Industry	2

Clinical Trials Update, Market Analysis, and Projection for OCALIVA (Odevixibat)

Last updated: November 2, 2025

Introduction

OCALIVA (obeticholic acid), marketed under the brand name OCALIVA, is a first-in-class bile acid analog developed by Intercept Pharmaceuticals. Approved by the U.S. Food and Drug Administration (FDA) in 2016 for primary biliary cholangitis (PBC), OCALIVA represents a targeted therapy addressing cholestatic liver diseases. Its development portfolio extends into rare liver disorders, notably biliary atresia and certain cholestatic pediatric populations. The following analysis offers a comprehensive update on clinical trials, evaluates the current market landscape, and projects future growth trajectories for OCALIVA.

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Clinical Trials Update

FDA-Approved Indication and Trials

Since its FDA approval in 2016, OCALIVA's primary indication has been for adults with PBC who have an inadequate response to ursodeoxycholic acid (UDCA) or are intolerant to it. The initial phase involved pivotal trials such as REBUILD, which demonstrated significant reductions in serum alkaline phosphatase—an important biomarker linked to disease progression.

Ongoing and Recent Clinical Trials

Expansion into Rare Pediatric Liver Diseases

Intercept Pharmaceuticals has initiated multiple clinical investigations to broaden OCALIVA’s application, especially in pediatric cholestatic conditions. The company announced in 2022 the commencement of the BILBA trial—evaluating the safety, efficacy, and pharmacokinetics of OCALIVA in children with biliary atresia, a rare congenital condition resulting in bile duct obstruction.

Combination and Long-term Efficacy Studies

Further studies aim to explore OCALIVA in combination therapy regimens, particularly with obeticholic acid and other antifibrotic agents, to improve outcomes in advanced liver disease and fibrosis stages. These include phase 3 trials assessing long-term durability and safety, crucial for regulatory approvals in new indications.

Pediatric and Rare Disease Trials

Notably, in early 2023, Intercept submitted preliminary results from its pediatric biliary atresia trial. Preliminary data suggest that OCALIVA is well tolerated, with signs of biochemical improvement, prompting plans for larger, confirmatory studies. This initiative aligns with FDA's Orphan Drug designation, offering the potential for expedited review.

Regulatory Status and Pipeline Outlook

Despite encouraging preliminary data, OCALIVA has yet to secure approvals beyond PBC. Intercept maintains an active pipeline, including:

• Obeticholic Acid for Nonalcoholic Steatohepatitis (NASH): Phase 3 trials have faced challenges, including mixed efficacy results, with ongoing analyses determining future regulatory pathways.
• Bile Duct Cancer and Fibrosis: Exploratory phase 2 trials are ongoing to assess the antifibrotic potential of OCALIVA.

Intercept’s strategic focus remains on expanding indications where cholestasis and fibrosis are central, especially in pediatric populations.

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Market Analysis

Current Market Landscape

Primary Biliary Cholangitis (PBC) Market

Since its approval, OCALIVA has become a key competitor in the PBC therapeutics market, competing against ursodeoxycholic acid (UDCA)—the standard of care—and newer agents like bezafibrate and obeticholic acid formulations. As of 2022, OCALIVA held approximately 15-20% of prescription market share in this niche, driven by its targeted mechanism and proven efficacy.

Market Dynamics & Drivers

• Growing PBC Awareness: Increased diagnosis and patient awareness bolster market potential.
• Unmet Needs: Approximately 30-40% of PBC patients respond inadequately to UDCA, creating demand for effective second-line therapies like OCALIVA.
• Regulatory Approvals in Market Expansions: Approval in Europe and Asia remains pending, with submissions underway, poised to diversify revenue streams.

Market Challenges

• Pricing and Reimbursement: OCALIVA is priced higher than generic ursodeoxycholic acid, creating reimbursement hurdles, particularly in cost-sensitive healthcare systems.
• Side Effect Profile: Pruritus and adverse lipid profile effects, though manageable, may limit use in certain populations.
• Competition: Emerging therapies, including non-pharmacologic interventions and other antifibrotic agents, threaten to erode market share.

Future Market Expansion Opportunities

Intercept aims to capitalize on unmet needs in pediatric cholestasis and fibrotic liver diseases. The global rare disease market, projected to reach USD 400 billion by 2030 ([2]), presents a fertile ground based on current indications and expanding clinical trial initiatives.

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Market Projection and Growth Trajectory

Forecast Assumptions

• Market Penetration: Given ongoing clinical trials and regulatory approvals, OCALIVA’s market share in PBC is expected to grow modestly at a CAGR of approximately 8-10% over the next five years.
• Indication Expansion: Pediatric cholestasis and NASH-related fibrosis could generate significant revenue streams, adding an estimated USD 500 million in annual sales by 2028—assuming successful trial outcomes and approvals.
• Geographical Penetration: Expansion into European, Japanese, and broader Asian markets could expand sales by 25-30%, contingent on regulatory pathways.

Revenue Projections (2023–2028)

Year	Estimated Global Sales	Notes
2023	USD 150 million	Primarily PBC with slow initial adoption
2024	USD 180 million	Market expansion and increased clinician familiarity
2025	USD 220 million	Initiation of pediatric and niche trials
2026	USD 300 million	Potential approvals in pediatric indications
2028	USD 450 million	Broad adoption across indications and geographies

Key Risks to Projection

• Regulatory Delays: Extended review timelines or failure to obtain new indications could impact revenue.
• Clinical Trial Failures: Negative efficacy or safety signals in pediatric or fibrotic studies may hinder expansion.
• Market Competition: Introduction of alternative therapies could limit market penetration.

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Key Takeaways

• Clinical development for OCALIVA continues to evolve, with promising early data in pediatric biliary atresia and fibrotic liver diseases. The ongoing trials are pivotal to expanding its therapeutic footprint.
• Market penetration in PBC remains solid but faces restraints from reimbursement concerns and competition. Future growth hinges on geographic expansion and indication approval.
• Future projections indicate a potential compound annual growth rate of approximately 8-10% through 2028, driven by pipeline advances, particularly in pediatric and fibrosis domains.
• Strategic considerations for stakeholders include monitoring trial outcomes, regulatory timelines, and emerging competitive therapies to optimize decision-making.

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FAQs

1. What is the primary approved indication for OCALIVA?
   OCALIVA is approved for primary biliary cholangitis (PBC) in adults with an inadequate response to or intolerance of ursodeoxycholic acid.

2. Are there ongoing clinical trials for OCALIVA in pediatric populations?
   Yes. Intercept Pharmaceuticals is evaluating OCALIVA in pediatric biliary atresia, with recent preliminary data showing promising safety and biochemical improvements.

3. How does OCALIVA compare to other treatments for PBC?
   OCALIVA offers a targeted mechanism with proven efficacy in reducing disease biomarkers for PBC, especially in patients unresponsive to UDCA, positioning itself as a valuable second-line therapy.

4. What are the main challenges facing OCALIVA's market expansion?
   Reimbursement issues, side-effect management, competition, and regulatory approval delays are primary hurdles.

5. What is the outlook for OCALIVA in future markets like NASH and fibrosis?
   While early trials in NASH and fibrosis show potential, inconsistent efficacy results indicate that further data are necessary before broader adoption can be expected.

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Sources

[1] Intercept Pharmaceuticals. OCALIVA (obeticholic acid) Prescribing Information. 2016.
[2] Grand View Research. Rare Disease Treatment Market Size, Share & Trends Analysis. 2022.