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Obeticholic acid

Not yet reviewed: Listed from FDA or ClinicalTrials.gov records; an editor has not checked it yetListed from FDA or ClinicalTrials.gov records; an editor has not checked it yet.

Also known as Ocaliva

INTERCEPT PHARMACEUTICALS, INC. · Compensated Cirrhosis; Non Alcoholic Steatohepatitis (NASH); Nonalcoholic Steatohepatitis; Primary Biliary Cirrhosis

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Key figures

  • Next catalyst

    None scheduled

    Only dates with a source are listed

  • Phase

    Approved

    Not recorded

  • Linked trials

    3

    From ClinicalTrials.gov

  • Latest FDA action

    Approved

    NDA207999,

Recalls, shortages, patents and EU status at a glance

Key information

Sponsor
INTERCEPT PHARMACEUTICALS, INC. Source: Company record for INTERCEPT PHARMACEUTICALS, INC. (opens in a new tab)
Indications
Compensated Cirrhosis; Non Alcoholic Steatohepatitis (NASH); Nonalcoholic Steatohepatitis; Primary Biliary Cirrhosis Source: Conditions from ClinicalTrials.gov (opens in a new tab)
Application
NDA 207999 · Priority review Source: Drugs@FDA via openFDA (opens in a new tab) · Action letter for NDA 207999 (opens in a new tab)
Designations
None recorded
Next catalyst
None scheduled

Development timeline

  1. Preclinical Completed
  2. Phase 1 Completed
  3. Phase 2 Completed
  4. Phase 3 CompletedFirst trial start 1 Jan 20123 linked trials
  5. Filed Completed
  6. Approved Current stageFirst approval 27 May 2016

Overview

Mechanism
Not recorded
Route
Not recorded
Modality
Not recorded
Specialties
Not recorded

Overview text is written by HealthTrade editors.

Key dates

No catalysts yet. Editors add each date together with its source.

FDA designations

No FDA designations are on record for Obeticholic acid. Editors add each one with its source.

Filed application

No filed application is on record for Obeticholic acid. Approvals are listed under FDA actions.

FDA advisory committees

No FDA advisory committee meetings are on record for Obeticholic acid.

Clinical trials

Trials linked to Obeticholic acid
TrialTitlePhaseStatusEnrollmentPrimary completionResults posted
NCT03439254ClinicalTrials.gov record for NCT03439254 (opens in a new tab)REVERSEStudy Evaluating the Efficacy and Safety of Obeticholic Acid in Subjects With Compensated Cirrhosis Due to Nonalcoholic SteatohepatitisPhase 3Completed919 (actual)8 Sep 2022 (actual) Yes First posted Results on ClinicalTrials.gov for NCT03439254 (opens in a new tab)

Posted results on ClinicalTrials.gov for REVERSE

  • DB Phase: Number of Participants Who Were Responders and Showed Improvement in Fibrosis by at Least 1 Stage Without Worsening of Nonalcoholic Steatohepatitis (NASH) (Participants)
    • DB: Placebo vs DB: OCA 10 Milligrams (mg): Response ratio(Mantel-Haenszel estimate) 1.13 (95% CI 0.71 to 1.79), p-value 0.6172, Cochran-Mantel-Haenszel, other
    • DB: Placebo vs DB: OCA 10 mg Titrated to OCA 25 mg: Response ratio(Mantel-Haenszel estimate) 1.20 (95% CI 0.77 to 1.89), p-value 0.4184, Cochran-Mantel-Haenszel, other

Serious adverse events (Up to 18 months for DB phase and Up to 1 year for OLE phase): DB: Placebo 43 of 312; DB: OCA 10 Milligrams (mg) 49 of 295; DB: OCA 10 mg Titrated to OCA 25 mg 46 of 309; OLE: OCA 10 mg (DB Placebo) 26 of 231; OLE: OCA 10 mg (DB OCA 10 mg) 50 of 221; OLE: Titrated to OCA 25 mg (DB OCA 10 mg Titrated to OCA 25 mg) 48 of 207

Source: ClinicalTrials.gov results for NCT03439254 (opens in a new tab)· imported

Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately.

NCT02548351ClinicalTrials.gov record for NCT02548351 (opens in a new tab)REGENERATERandomized Global Phase 3 Study to Evaluate the Impact on NASH With Fibrosis of Obeticholic Acid TreatmentPhase 3Terminated2,477 (actual)15 Sep 2023 (actual) Yes First posted Results on ClinicalTrials.gov for NCT02548351 (opens in a new tab)

Posted results on ClinicalTrials.gov for REGENERATE

  • Time to the First Adjudicated Event for Clinical Outcome Composite Endpoint: Percentage of Participants With an Event (Percentage of participants)
    • OCA 10 Milligrams (mg) vs Placebo: Hazard Ratio (HR) 0.814 (95% CI 0.635 to 1.043), p-value 0.1028, Log Rank, other
    • OCA 25 mg vs Placebo: Hazard Ratio (HR) 0.772 (95% CI 0.600 to 0.994), p-value 0.0444, Log Rank, other

Serious adverse events (Up to 7 years): OCA 10 Milligrams (mg) 273 of 825; OCA 25 mg 276 of 827; Placebo 248 of 825

Source: ClinicalTrials.gov results for NCT02548351 (opens in a new tab)· imported

Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately.

Publications listing REGENERATE (5), newest first

  1. Utility of pathologist panels for achieving consensus in NASH histologic scoring in clinical trials: Data from a phase 3 study.

    Hepatology communications · 2024 Jan 1

    PubMed 38126958 (opens in a new tab)DOI 10.1097/HC9.0000000000000325 (opens in a new tab)

  2. Obeticholic Acid Impact on Quality of Life in Patients With Nonalcoholic Steatohepatitis: REGENERATE 18-Month Interim Analysis.

    Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association · 2022 Sep

    PubMed 34274514 (opens in a new tab)DOI 10.1016/j.cgh.2021.07.020 (opens in a new tab)

  3. Non-invasive evaluation of response to obeticholic acid in patients with NASH: Results from the REGENERATE study.

    Journal of hepatology · 2022 Mar

    PubMed 34793868 (opens in a new tab)DOI 10.1016/j.jhep.2021.10.029 (opens in a new tab)

  4. Obeticholic acid for the treatment of non-alcoholic steatohepatitis: interim analysis from a multicentre, randomised, placebo-controlled phase 3 trial.

    Lancet (London, England) · 2019 Dec 14

    PubMed 31813633 (opens in a new tab)DOI 10.1016/S0140-6736(19)33041-7 (opens in a new tab)

  5. REGENERATE: Design of a pivotal, randomised, phase 3 study evaluating the safety and efficacy of obeticholic acid in patients with fibrosis due to nonalcoholic steatohepatitis.

    Contemporary clinical trials · 2019 Sep

    PubMed 31260793 (opens in a new tab)DOI 10.1016/j.cct.2019.06.017 (opens in a new tab)

Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies.

NCT01473524ClinicalTrials.gov record for NCT01473524 (opens in a new tab)POISEPhase 3 Study of Obeticholic Acid in Patients With Primary Biliary CirrhosisPhase 3Completed217 (actual)Dec 2013 (actual) Yes First posted Results on ClinicalTrials.gov for NCT01473524 (opens in a new tab)

Posted results on ClinicalTrials.gov for POISE

  • DB Phase: Composite Endpoint Alkaline Phosphatase (ALP) And Total Bilirubin, 10 mg OCA Versus Placebo (percentage of participants)
    • DB OCA 10 mg vs DB Placebo: Estimate not given, p-value <0.0001, Cochran-Mantel-Haenszel, superiority or other

Serious adverse events (DB Phase: Baseline up to 12 months (1 year). LTSE phase: Baseline (DB Month 12) up to 60 months (5 years).): DB 5-10 mg 11 of 70; DB OCA 10 mg 8 of 73; DB Placebo 3 of 73; LTSE OCA (DB OCA 5-10 mg) 30 of 63; LTSE OCA (DB 10 mg) 19 of 64; LTSE OCA (DB Placebo) 20 of 66; Overall LTSE OCA 69 of 193

Source: ClinicalTrials.gov results for NCT01473524 (opens in a new tab)· imported

Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately.

Publications listing POISE (6), newest first

  1. Long-Term Safety and Efficacy of Obeticholic Acid in Patients With Primary Biliary Cholangitis: Final Results of the POISE Long-Term Safety Extension.

    Alimentary pharmacology & therapeutics · 2026 Oct

    PubMed 42449190 (opens in a new tab)DOI 10.1111/apt.70832 (opens in a new tab)

  2. Obeticholic acid is associated with improvements in AST-to-platelet ratio index and GLOBE score in patients with primary biliary cholangitis.

    JHEP reports : innovation in hepatology · 2021 Feb

    PubMed 33319187 (opens in a new tab)DOI 10.1016/j.jhepr.2020.100191 (opens in a new tab)

  3. Long-Term Obeticholic Acid Therapy Improves Histological Endpoints in Patients With Primary Biliary Cholangitis.

    Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association · 2020 May

    PubMed 31606455 (opens in a new tab)DOI 10.1016/j.cgh.2019.09.050 (opens in a new tab)

  4. Long-term efficacy and safety of obeticholic acid for patients with primary biliary cholangitis: 3-year results of an international open-label extension study.

    The lancet. Gastroenterology & hepatology · 2019 Jun

    PubMed 30922873 (opens in a new tab)DOI 10.1016/S2468-1253(19)30094-9 (opens in a new tab)

  5. A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis.

    The New England journal of medicine · 2016 Aug 18

    PubMed 27532829 (opens in a new tab)DOI 10.1056/NEJMoa1509840 (opens in a new tab)

All 6 publications for NCT01473524

Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies.

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Posted results are shown as ClinicalTrials.gov prints them; results from company and journal sources are entered by editors and listed separately. Full result tables are on ClinicalTrials.gov.

Trial quality

About CT-VCA

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FDA actions

Original approvals and new-indication supplements for Obeticholic acid, newest first
ApplicationActionDateOutcomeReviewSource
NDA207999Action letter for NDA207999 (opens in a new tab) Original 1 Type 1 - New Molecular EntityApprovedpriorityDrugs@FDA via openFDA (opens in a new tab)

Data provided by the U.S. Food and Drug Administration.

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FDA drug shortages load here.

Orange Book patents load here.

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EU status (EMA)

RevokedConditional

Ocaliva (EMEA/H/C/004093)

Authorised
Therapeutic area
Liver Cirrhosis, Biliary
Marketing authorisation holder
Advanz Pharma Limited
Active substance (INN)
obeticholic acid
ATC code
A05AA04
Latest EMA decision
Withdrawn

Matched by active substance: EMA's INN or active substance equals this drug's name. EMA lists 1 other product with this substance, such as generics.

EMA page for Ocaliva (opens in a new tab)EMA medicine data (opens in a new tab)

Source: European Medicines Agency (EMA), medicines data download. Licence: EMA re-use with acknowledgement. EMA medicines report 2026-10-26.

Related news

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Sources

Record updated .

Dates come from company and FDA disclosures and can change. Nothing on this page is investment advice.