Company Statement on FDA Advisory Committee Meeting

On September 13, 2024, the U.S. Food and Drug Administration's Gastrointestinal Drugs Advisory Committee met to review Intercept's supplemental New Drug Application for OCALIVA (obeticholic acid). This application seeks full approval for the treatment of primary biliary cholangitis (PBC), a rare and progressive liver disease that disproportionately affects women. The meeting included contributions from patients and healthcare providers, highlighting the importance of community support in the drug approval process. PBC leads to the accumulation of bile acids in the liver, causing inflammation and fibrosis, which can progress to severe complications such as cirrhosis, liver transplants, or even death if left untreated. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of October 15, 2024, for its decision on the application. The continued approval of OCALIVA for PBC treatment may hinge on the results of confirmatory trials that verify its clinical benefits. The advisory committee's discussions emphasized the need for ongoing monitoring of patients for potential adverse effects, including hepatic decompensation and failure, which have been reported in some cases. Intercept Pharmaceuticals expressed gratitude for the support from the PBC community and reiterated its commitment to collaborating with the FDA to ensure patient safety and efficacy of the treatment. The outcome of this meeting and the subsequent FDA decision will be crucial for patients relying on OCALIVA for managing their condition.