Intercept Pharmaceuticals Withdraws Liver Disease Drug Ocaliva from US Market Following FDA Request

Intercept Pharmaceuticals has announced the withdrawal of its liver disease drug Ocaliva (obeticholic acid) from the US market, marking a significant setback for the company and its efforts to treat primary biliary cholangitis (PBC). The decision comes after years of regulatory challenges and follows a direct request from the Food and Drug Administration (FDA).

FDA Intervention and Clinical Hold

The FDA has taken decisive action by placing Intercept's trials of obeticholic acid on a clinical hold and requesting the voluntary withdrawal of Ocaliva from the market. This development affects the drug's accelerated approval status, which it had held since 2016 for the treatment of certain PBC patients.

Vivek Devaraj, Intercept's US president, stated, "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." The company maintains its commitment to hepatology innovation and patient care despite this setback.

Regulatory Struggles and Safety Concerns

Intercept's journey with Ocaliva has been fraught with challenges. In 2022, the FDA's Gastrointestinal Drug Advisory Committee voted against Intercept's data package, citing concerns over the drug's safety profile. The FDA's pre-meeting briefing documents highlighted potential risks, including an increased likelihood of liver transplantation or death for patients taking the medication.

Following the committee's decision, the FDA rejected Intercept's application for traditional approval but allowed the drug to remain available under its accelerated approval while post-marketing safety data was evaluated. However, the recent FDA request for withdrawal suggests that safety concerns have persisted.

Global Implications and Company Future

The US withdrawal of Ocaliva follows a similar action in Europe, where regulators revoked the drug's marketing authorization in late 2024. These setbacks have had a significant impact on Intercept Pharmaceuticals, which had previously aimed to market obeticholic acid for nonalcoholic steatohepatitis (NASH), now known as metabolic dysfunction-associated steatohepatitis (MASH).

In response to these challenges, Intercept sold itself to Italian drugmaker Alfasigma in 2023 for approximately $800 million. At the time of the acquisition, Ocaliva had generated $285.7 million in full-year 2022 sales as Intercept's sole product.

With Ocaliva's withdrawal, Intercept's pipeline now primarily consists of obeticholic acid formulations and a next-generation FXR agonist in phase 2a trials for severe alcohol-associated hepatitis. The company's future strategy and focus areas remain to be seen as it navigates this significant change in its product portfolio.