Intercept Pharmaceuticals has voluntary withdrawn Ocaliva (obeticholic acid), intented for primary biliary cholangitis (PBC), from the U.S. market, according to a recently published press release.
Ocaliva had played a role in managing the disease since its accelerated approval in 2016 for patients unresponsive to or intolerant of ursodeoxycholic acid (UDCA). However, the lack of clear survival benefit and growing safety concerns ultimately outweighed its continued use.
The decision comes after a request from the U.S. Food and Drug Administration (FDA), which reviewed recent postmarketing clinical trial data revealing cases of serious liver injury among patients with PBC treated with Ocaliva who did not have cirrhosis. Ocaliva was already contraindicated in patients with advanced cirrhosis in 2021, due to a known risk of hepatic decompensation.
Additionally, 20 cases submitted to the FDA between May 2021 and September 2024 described liver-related events, such as transplants or deaths, in patients on Ocaliva. In at least three of these cases, the medication was inappropriately continued despite clear evidence of liver disease progression.
Furthermore, in a postmarketing clinical trial, patients with noncirrhotic PBC taking Ocaliva showed significantly higher rates of liver transplant and death compared to those receiving placebo. Of 81 patients treated with Ocaliva, seven required liver transplants and four died, compared to one transplant and one death among 68 placebo recipients.
“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,” said Vivek Devaraj, U.S. president at Intercept. “We remain committed to innovation in hepatology and to serving the needs of patients and physicians.”
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