Obeticholic acid

Generic Name: Obeticholic acid

Brand Names: Ocaliva

Drug Type: Small Molecule

Chemical Formula: C_₂₆H_₄₄O_₄

CAS Number: 459789-99-2

Unique Ingredient Identifier: 0462Z4S4OZ

Background: Primary biliary cirrhosis, or PBC, is a progressive and chronic condition that leads to hepatic injury often resulting in end-stage liver failure that requires liver transplantation.

Obeticholic acid is a farnesoid-X receptor (FXR) agonist used to treat this condition, possibly allowing for increased survival. In 2016, it was granted approval to treat primary biliary cholangitis in combination with ursodeoxycholic acid, which was previously the mainstay treatment for this condition. In May 2021, the FDA updated its prescribing information to contraindicate the use of obeticholic acid in patients with PBC and advanced cirrhosis (e.g. those with portal hypertension or hepatic decompensation) due to a risk of liver failure, in some cases requiring liver transplantation.

Obeticholic acid is currently being considered for FDA approval to treat fibrosis caused by non-alcoholic liver steatohepatitis (NASH). The NDA from Intercept Pharmaceuticals was approved in November 2019 and obeticholic acid is expected to be granted full approval for this indication in 2020.

Indication: 用于治疗原发性胆汁性胆管炎(PBC)或用于治疗原发性胆汁性肝硬化（PBC）。

Associated Conditions: Primary Biliary Cholangitis

Clinical Trials

Obeticholic Acid Versus Placebo, in Combination with Ursodeoxycholic Acid in Patients with Primary Biliary Cirrhosis

Phase 3

Completed

Conditions: PBC

Interventions: Drug: UDCA
Drug: OCA
Drug: Placebo

First Posted Date: 2024-12-04

Last Posted Date: 2024-12-04

Lead Sponsor: Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd.

Target Recruit Count: 100

Registration Number: NCT06715319

Locations: 🇨🇳
The First Hospital of Jilin University, Changchun, Jilin, China

Study to Assess Efficacy, Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Obeticholic Acid (OCA) Compared to Placebo in Pediatric Participants With Biliary Atresia, Post-hepatoportoenterostomy

Phase 2

Recruiting

Conditions: Biliary Atresia

Interventions: Drug: Matching Placebo
Drug: OCA

First Posted Date: 2023-11-07

Last Posted Date: 2024-11-20

Lead Sponsor: Intercept Pharmaceuticals

Target Recruit Count: 144

Registration Number: NCT06121375

Locations: 🇦🇺
Queensland Childrens Hospital, South Brisbane, Queensland, Australia
🇦🇺
Women's and Children's Hospital, North Adelaide, South Australia, Australia
🇨🇦
Stollery Children's Hospital, Edmonton, Alberta, Canada

and more 6 locations

The Effect of Obeticholic Acid in Healthy Volunteers

Not Applicable

Recruiting

Conditions: Healthy

Interventions: Drug: Ocaliva
Drug: Placebo

First Posted Date: 2023-02-23

Last Posted Date: 2023-02-23

Lead Sponsor: Universitaire Ziekenhuizen KU Leuven

Target Recruit Count: 12

Registration Number: NCT05740631

Locations: 🇧🇪
KU Leuven, Leuven, Vlaams-Brabant, Belgium

Comparative Study Between Obeticholic Acid Versus Vitamin E in Patients with Non-alcoholic Steatohepatitis

Phase 2

Active, not recruiting

Conditions: Non-alcoholic Steatohepatitis

Interventions: Drug: Obeticholic Acid Oral Tablet
Drug: Vit E

First Posted Date: 2022-10-10

Last Posted Date: 2025-01-10

Lead Sponsor: Tanta University

Target Recruit Count: 59

Registration Number: NCT05573204

Locations: 🇪🇬
Department of Tropical Medicine and Infectious Diseases, Faculty of Medicine,Tanta University, Tanta, Egypt

Testing Obeticholic Acid for Familial Adenomatous Polyposis

Phase 2

Recruiting

Conditions: Attenuated Familial Adenomatous Polyposis
Familial Adenomatous Polyposis
Colorectal Carcinoma
Duodenal Carcinoma

Interventions: Procedure: Biopsy Procedure
Procedure: Biospecimen Collection
Procedure: Gastrointestinal Endoscopy
Biological: Obeticholic Acid
Drug: Placebo Administration
Other: Questionnaire Administration

First Posted Date: 2022-02-03

Last Posted Date: 2025-05-16

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 80

Registration Number: NCT05223036

Locations: 🇺🇸
Mayo Clinic in Arizona, Scottsdale, Arizona, United States
🇺🇸
University of Kansas Cancer Center, Kansas City, Kansas, United States
🇺🇸
Dana-Farber Cancer Institute, Boston, Massachusetts, United States

and more 4 locations

Linerixibat and Obeticholic Acid Drug Interaction Study in Healthy Adult Participants

Phase 1

Completed

Conditions: Pruritus

Interventions: Drug: Obeticholic acid
Drug: Linerixibat

First Posted Date: 2021-11-24

Last Posted Date: 2022-11-08

Lead Sponsor: GlaxoSmithKline

Target Recruit Count: 52

Registration Number: NCT05133830

Locations: 🇺🇸
GSK Investigational Site, Baltimore, Maryland, United States

Study of Obeticholic Acid（OCA） Combination With Ursodeoxycholic Acid (UDCA) in Patients With Primay Biliary Cirrhosis (PBC)

Phase 3

Conditions: Primary Biliary Cirrhosis

Interventions: Drug: Obeticholic Acid Tablets
Drug: UDCA
Drug: Placebo

First Posted Date: 2021-07-09

Last Posted Date: 2021-07-26

Lead Sponsor: Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Target Recruit Count: 120

Registration Number: NCT04956328

Locations: 🇨🇳
The first hospital of Jilin University, Changchun, Jilin, China

Obeticholic Acid for Prevention in Barrett's Esophagus

Phase 2

Recruiting

Conditions: Barrett Esophagus
Esophageal Adenocarcinoma

Interventions: Procedure: Biospecimen Collection
Procedure: Esophageal Biopsy
Procedure: Esophagogastroduodenoscopy
Procedure: Liver Ultrasonographic Elastography
Drug: Placebo Administration
Biological: Obeticholic Acid
Other: Questionnaire Administration

First Posted Date: 2021-06-25

Last Posted Date: 2025-05-04

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 30

Registration Number: NCT04939051

Locations: 🇺🇸
Case Western Reserve University, Cleveland, Ohio, United States
🇺🇸
University of Kansas Cancer Center, Kansas City, Kansas, United States
🇺🇸
University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan, United States

and more 5 locations

Study of OCA in Combination with BZF Evaluating Efficacy, Safety, and Tolerability in Participants with PBC

Phase 2

Active, not recruiting

Conditions: Primary Biliary Cholangitis

Interventions: Drug: Obeticholic acid
Drug: OCA
Drug: Bezafibrate 200 MG
Drug: OCA Placebo
Drug: Bezafibrate 200 mg Placebo
Drug: Bezafibrate
Drug: Bezafibrate 400 MG
Drug: Bezafibrate 400 mg Placebo

First Posted Date: 2020-10-20

Last Posted Date: 2024-11-04

Lead Sponsor: Intercept Pharmaceuticals

Target Recruit Count: 72

Registration Number: NCT04594694

Locations: 🇬🇷
Department of Medicine and Research Laboratory of Internal Medicine, University Hospital of Larissa, Larissa, Greece
🇫🇷
Hôpital Henri Mondor, Créteil, France
🇩🇪
Medizinische Hochschule Hannover, Hannover, Germany

and more 30 locations

Prospective, Multicenter Cohort Study on Primary Biliary Cholangitis

Recruiting

Conditions: PBC
Primary Biliary Cholangitis

Interventions: Drug: UDCA
Drug: Ocaliva

First Posted Date: 2019-09-03

Last Posted Date: 2023-06-07

Lead Sponsor: University of Leipzig

Target Recruit Count: 1200

Registration Number: NCT04076527

Locations: 🇩🇪
Charité-Campus Virchow-Klinikum, Berlin, Germany
🇩🇪
University Hospital Aachen, Aachen, Germany
🇩🇪
Charité - Campus Benjamin Franklin, Berlin, Germany

and more 41 locations

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