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

Linerixibat and Obeticholic Acid Drug Interaction Study in Healthy Subjects

Phase 1

Completed

Conditions: Cholestasis

Interventions: Drug: GSK2330672 (linerixibat)
Drug: Obeticholic acid

First Posted Date: 2019-08-12

Last Posted Date: 2020-07-24

Lead Sponsor: GlaxoSmithKline

Target Recruit Count: 19

Registration Number: NCT04053023

Locations: 🇬🇧
GSK Investigational Site, Cambridge, United Kingdom

Role of Obeticholic Acid in the Patients of NAFLD With Raised ALT

Not Applicable

Completed

Conditions: Non-Alcoholic Fatty Liver Disease

Interventions: Drug: Obeticholic acid

First Posted Date: 2019-02-11

Last Posted Date: 2022-05-17

Lead Sponsor: Sir Salimullah Medical College Mitford Hospital

Target Recruit Count: 70

Registration Number: NCT03836937

Locations: 🇧🇩
Department of Hepatology, Dhaka, Bangladesh

Effect of Obeticholic Acid on Transport of Bile Acids in PBC Examined by 11C-cholyl-sarcosine PET/CT

Early Phase 1

Completed

Conditions: Primary Biliary Cirrhosis

Interventions: Drug: Placebos
Drug: Obeticholic acid

First Posted Date: 2017-08-17

Last Posted Date: 2019-11-01

Lead Sponsor: University of Aarhus

Target Recruit Count: 8

Registration Number: NCT03253276

Locations: 🇩🇰
Susanne Keiding, Aarhus, Denmark

Combination Obeticholic Acid (OCA) and Statins for Monitoring of Lipids (CONTROL)

Phase 2

Completed

Conditions: Nonalcoholic Steatohepatitis

Interventions: Drug: Obeticholic Acid
Drug: Placebo
Drug: Atorvastatin

First Posted Date: 2015-12-17

Last Posted Date: 2018-06-04

Lead Sponsor: Intercept Pharmaceuticals

Target Recruit Count: 84

Registration Number: NCT02633956

Locations: 🇺🇸
Consultants for Clinical Research, Cincinnati, Ohio, United States
🇺🇸
University of Miamai, Schiff Center for Liver Diseases, Miami, Florida, United States
🇺🇸
Mercy Medical Center, Institute for Digestive Health & Liver Disease, Baltimore, Maryland, United States

and more 19 locations

Randomized Global Phase 3 Study to Evaluate the Impact on NASH With Fibrosis of Obeticholic Acid Treatment

Phase 3

Terminated

Conditions: Non Alcoholic Steatohepatitis (NASH)

Interventions: Drug: Obeticholic Acid
Drug: Placebo

First Posted Date: 2015-09-14

Last Posted Date: 2024-10-01

Lead Sponsor: Intercept Pharmaceuticals

Target Recruit Count: 2477

Registration Number: NCT02548351

Locations: 🇺🇸
The University of Chicago Medical Center, Chicago, Illinois, United States
🇺🇸
Johns Hopkins Hospital, Baltimore, Maryland, United States
🇺🇸
Beth Israel Deaconess Medical Center, Boston, Massachusetts, United States

and more 340 locations

Obeticholic Acid in Morbidly Obese Patients and Healthy Volunteers

Phase 1

Conditions: Obesity

Interventions: Drug: Obeticholic acid
Drug: Obeticholic acid placebo

First Posted Date: 2015-08-25

Last Posted Date: 2020-08-25

Lead Sponsor: Sahlgrenska University Hospital, Sweden

Target Recruit Count: 40

Registration Number: NCT02532335

Locations: 🇸🇪
Hanns-Ulrich Marschall, Göteborg, Sweden
🇸🇪
Sahlgrenska Academy, Göteborg, Sweden

Phase 2 Study of Obeticholic Acid for Lipodystrophy Patients

Phase 2

Completed

Conditions: Familial Partial Lipodystrophy

Interventions: Drug: Obeticholic Acid
Drug: Placebo

First Posted Date: 2015-04-29

Last Posted Date: 2024-08-27

Lead Sponsor: Abhimanyu Garg

Target Recruit Count: 10

Registration Number: NCT02430077

Locations: 🇺🇸
UT Southwestern Medical Center, Dallas, Texas, United States
🇺🇸
UT Southwestern Medical Center 5323 Harry Hines Blvd, Dallas, Texas, United States

Phase 2 Study on Effects of Obeticholic Acid (OCA) on Lipoprotein Metabolism in Participants With Primary Biliary Cirrhosis

Phase 2

Completed

Conditions: Primary Biliary Cirrhosis

Interventions: Drug: Obeticholic Acid

First Posted Date: 2013-05-31

Last Posted Date: 2022-08-24

Lead Sponsor: Intercept Pharmaceuticals

Target Recruit Count: 27

Registration Number: NCT01865812

Locations: 🇺🇸
McGuire DVAMC, Richmond, Virginia, United States
🇺🇸
Beth Israel Medical Center, New York, New York, United States
🇺🇸
Scripps Clinic, La Jolla, California, United States

and more 4 locations