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Intercept Pharmaceuticals Withdraws Ocaliva from Shelves Following U.S. Regulator’s Request
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Key Takeaways
- Ocaliva, a farnesoid X receptor agonist, was withdrawn from the U.S. market due to FDA concerns about liver injury in patients.
- Initially approved in 2016 under an accelerated pathway, Ocaliva faced challenges in confirmatory trials due to recruitment difficulties.
Intercept Pharmaceuticals withdraws Ocaliva from the U.S. market following FDA concerns over safety and effectiveness for treating primary biliary cholangitis.
The withdrawal from shelves comes as another setback for Ocaliva after it was denied full approval from FDA last year.
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Intercept Pharmaceuticals announced Ocaliva’s withdrawal from the U.S. market per FDA regulators request. Ocaliva was declined full approval from FDA last year after the agency identified liver injury in patients administered the drug.
“We continue to believe the totality of clinical and real-world evidence supports Ocaliva’s use for appropriate patients, and we are proud of the contribution Ocaliva has made in advancing care for people living with primary biliary cholangitis (PBC). While our view of Ocaliva’s benefit-risk profile differs from FDA’s, we respect its request and have made this difficult decision to provide clear guidance for patients and prescribers,” said Vivek Devaraj, U.S. president at Intercept. “We remain committed to innovation in hepatology and to serving the needs of patients and physicians.”3
What is Ocaliva?
Ocaliva is a farnesoid X receptor (FXR) agonist indicated for the treatment of adult patients with PBC without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension either in combination with ursodeoxycholic acid (UDCA) with an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. This indication of Ocaliva is approved under accelerated approval based on a reduction in alkaline phosphatase (ALP). Improvements in survival or disease-related symptoms have yet to be established with continued approval for this indication becoming contingent upon verifications and descriptions of its clinical benefits in confirmatory trials.3
Ocaliva, which is chemically referred to as obeticholic acid, received FDA's accelerated approval back in 2016 with the indication to treat patients with primary biliary cholangitis, a rare disease resulting in inflammation of small bile ducts in the liver eventually destroying them.1 Under the accelerated pathway FDA mandates additional post-market trials verifying a drugs benefits, with the opportunity of FDA regulators requesting withdrawal from market if the collected data doesn’t reflect the drug’s effectiveness.
Ocaliva denied full FDA approval
Back in November of 2024, Ocaliva was denied full approval in the U.S. market, with FDA citing concerns surrounding the data of the treatment not clearly portraying the drug’s safety or effectives. Recruitment to Ocaliva’s confirmatory trial was halted, due to difficulties finding participants with PBC or other rare diseases, as around 200,000 cases are recorded within the U.S. annually.2 In June of 2024, Ocaliva also had its conditional authorization revoked from the European medicines regulators claiming the treatments benefits had yet to be confirmed.
Julia Wattacheril, associate professor at Columbia University Vagelos College of Physicians and Surgeons, touched on Ocaliva’s future last year when it did not receive full FDA approval, saying, “I think the future of the drug is now largely up to the applicant and potential patients who would enroll in new trials." 2
Sources
- Intercept Pharma pulls liver disease drug from US after regulator's request Reuters September 11, 2025 https://www.reuters.com/business/healthcare-pharmaceuticals/intercept-pharma-withdraws-liver-disease-drug-us-market-2025-09-11/
- US FDA declines full approval for Intercept's liver disease drug Reuters November 13, 2024 https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-declines-full-approval-intercepts-liver-disease-drug-2024-11-12/
- Intercept Announces Voluntary Withdrawal of OCALIVA® for Primary Biliary Cholangitis (PBC) from the US Market; US Clinical Trials Involving Obeticholic Acid Placed on Clinical Hold Intercept Pharmaceuticals September 11, 2025 https://www.globenewswire.com/news-release/2025/09/11/3148535/23024/en/Intercept-Announces-Voluntary-Withdrawal-of-OCALIVA-for-Primary-Biliary-Cholangitis-PBC-from-the-US-Market-US-Clinical-Trials-Involving-Obeticholic-Acid-Placed-on-Clinical-Hold.html
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