Intercept Pharmaceuticals has withdrawn Ocaliva (obeticholic acid) from the U.S. market, and all clinical trials evaluating the drug have been placed on hold, the company announced September 11, 2024. The move follows a recent safety signal identified in an interim analysis of the POISE trial, revealing a higher-than-expected rate of serious liver injury and death among patients with non-cirrhotic primary biliary cholangitis (PBC).
Ocaliva, approved in 2016, was the first and only medication specifically designed to treat PBC, a chronic liver disease that slowly damages the bile ducts. Its withdrawal impacts the approximately 20,000 to 30,000 Americans diagnosed with PBC,manny of whom relied on Ocaliva to slow disease progression. The halt in clinical trials also affects ongoing research into potential new uses for obeticholic acid,including its investigation for nonalcoholic steatohepatitis (NASH). Intercept stated it is working with regulatory authorities to determine the best course of action and will provide updates as more information becomes available.
The POISE trial, initiated in February 2019, was designed to assess the efficacy and safety of Ocaliva in patients with early-stage PBC who had an inadequate response to ursodeoxycholic acid (UDCA), the standard of care. The interim analysis,conducted by an self-reliant Data Monitoring Committee,prompted the company to voluntarily withdraw the drug and pause all trials.
“Patient safety is our top priority,” Intercept CEO Mark pruzanski said in a press release. “We are taking these actions in close collaboration with the FDA and will share further information as it becomes available.”
The FDA has advised healthcare professionals to immediately stop prescribing Ocaliva and to monitor patients currently taking the medication for any signs of liver injury.Patients experiencing symptoms such as jaundice, abdominal pain, fatigue, or dark urine should seek immediate medical attention.
