Intercept Voluntarily Withdraws Obeticholic Acid (Ocaliva) for PBC From US Market

Intercept Pharmaceuticals has announced its decision to voluntarily withdraw obeticholic acid (Ocaliva) from the US market for the treatment of primary biliary cholangitis (PBC).1

According to a September 11, 2025, release from the Company, the decision follows a request from the US Food and Drug Administration (FDA), and the Agency has placed a clinical hold on all Intercept clinical trials conducted under a US IND involving obeticholic acid.1

“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 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,” Vivek Devaraj, US President at Intercept, said in a statement.1 “We remain committed to innovation in hepatology and to serving the needs of patients and physicians.”

Obeticholic acid received FDA accelerated approval in 2016 for the treatment of PBC in adults with an inadequate response to or intolerance of ursodeoxycholic acid (UDCA). Since then, obeticholic acid has played a meaningful role in the treatment landscape for patients living with this rare disease.1

For many years, obeticholic acid was the sole second-line therapy in PBC and was the only option for patients who did not respond to or could not tolerate ADCA. In 2024, over the span of 2 months, the FDA granted accelerated approvals to both elafibranor (Iqirvo) and seladelpar (Livdelzi), making them the second and third approved second-line therapies in PBC, respectively.2.3

On November 12, 2024, the FDA issued a Complete Response Letter (CRL) to Intercept Pharmaceuticals’ supplemental New Drug Application for obeticholic acid (Ocaliva) for the treatment of PBC. In the CRL, the Agency informed Intercept that it was unable to approve the supplemental New Drug Application in its current form, consistent with the outcome of a Gastrointestinal Drugs Advisory Committee meeting held in September 2024 that yielded a negative opinion on the verification of obeticholic acid’s benefit on clinical outcomes in PBC as well as its benefit versus risk profile.4

At the time of the CRL, Intercept noted obeticholic acid would continue to be available for the treatment of appropriate patients living with PBC in the US under accelerated approval status.4

A month later, on December 12, 2024, the FDA announced the identification of cases of serious liver injury among patients being treated for PBC with obeticholic acid who did not have cirrhosis in an update to a previous FDA Drug Safety Communication in 2021 restricting the use of obeticholic acid in patients with PBC and advanced cirrhosis. Findings from this FDA review of postmarket clinical trial data for obeticholic acid showed an increased risk of liver injury in patients without cirrhosis.5

According to a release from the FDA, the Agency planned to notify health care professionals and patients of this new safety information and that frequent liver test monitoring is necessary to identify worsening liver function and ensure appropriate discontinuation of obeticholic acid, additionally detailing plans to continue to monitor the medicine’s safety and provide follow-up if additional information becomes available.5

As described in the latest release from Intercept, patients currently prescribed obeticholic acid for PBC treatment should consult their healthcare professionals before making any changes. The Company described plans to provide additional information to support healthcare professionals and patients as it works with FDA on the transition process.1

References

1. Intercept. 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. September 11, 2025. Accessed 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

2. Brooks, A. FDA Grants Accelerated Approval to Elafibranor (Iqirvo) for PBC. HCPLive. June 10, 2024. Accessed September 11, 2025. https://www.hcplive.com/view/fda-grants-accelerated-approval-to-elafibranor-iqirvo-for-pbc

3. Brooks, A. FDA Grants Accelerated Approval to Seladelpar (Livdelzi) for Primary Biliary Cholangitis. HCPLive. August 14, 2024. Accessed September 11, 2025. https://www.hcplive.com/view/fda-grants-accelerated-approval-to-seladelpar-livdelzi-for-primary-biliary-cholangitis

4. Brooks A. FDA Issues CRL to Obeticholic Acid (Ocaliva), Denies Full Approval for PBC. HCPLive. November 12, 2024. Accessed September 11, 2025. https://www.hcplive.com/view/fda-issues-crl-obeticholic-acid-ocaliva-denies-full-approval-pbc

5. Brooks A. FDA Finds Obeticholic Acid (Ocaliva) Linked to Serious Liver Injury in Noncirrhotic PBC. HCPLive. December 12, 2024. Accessed September 11, 2025. https://www.hcplive.com/view/fda-finds-obeticholic-acid-ocaliva-linked-serious-liver-injury-noncirrhotic-pbc