Intercept Pharmaceuticals has voluntarily withdrawn its liver disease drug, Ocaliva, from the U.S. market in response to a request from the FDA.
Ocaliva, which received accelerated approval from the FDA in 2016, is a farnesoid X receptor agonist in tablet form. It treats the rare progressive liver disease primary biliary cholangitis (PBC) in adults who are intolerant to ursodeoxycholic acid (UDCA) or who had an inadequate response to treatment with UDCA.
Soon after Ocaliva received approval, the FDA issued a warning that excessive doses put patients at increased risk of liver injury. In May 2021, the FDA updated this warning to express that there was a risk even when patients were taking appropriate doses and subsequently restricted use in patients with advanced cirrhosis, due to the risk of liver failure. Most recently, in December 2024, the FDA identified cases of serious liver injury in patients who did not have cirrhosis of the liver. According the the FDA, the agency evaluated liver safety in Ocaliva’s postmarket clinical trial and found that the risk of both liver transplant and death were higher in patients receiving Ocaliva compared with those receiving placebo. Specifically, among patients with a lower risk of progression to liver transplant or death, 7 of 81 who received Ocaliva needed a liver transplant compared to 1 of 68 patients who received placebo.
The FDA has also placed all Intercept clinical trials under U.S. IND involving obeticholic acid (Ocaliva) on clinical hold.