Intercept’s Ocaliva Continues Losing Streak as FDA Denies Full Approval

Product: Intercept Pharmaceuticals’ Ocaliva

Indication: Primary biliary cholangitis

After initially delaying its decision last month, the FDA on Tuesday declined to give Intercept Pharmaceuticals’ Ocaliva (obeticholic acid) full approval for primary biliary cholangitis (PBC).

Ocaliva, a farsenoid X receptor agonist, won the FDA’s accelerated approval for PBC in May 2016. In September 2024, however, the regulator’s Gastrointestinal Drugs Advisory Committee nearly unanimously voted against the oral drug, noting that Intercept had not provided enough data to support Ocaliva’s clinical benefit. In a briefing document released prior to the panel discussion, the FDA’s internal reviewers said that Ocaliva had “failed to demonstrate efficacy” in one of its confirmatory trials.

In its Complete Response Letter on Tuesday, the FDA indicated that it was continuing to review safety data for Ocaliva from one of its confirmatory studies. In the meantime, the drug will remain available to PBC patients under its accelerated approval status, according to Intercept.

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