FDA Advisory Committee Votes Against Ocaliva for Primary Biliary Cholangitis Due to Safety Concerns

FDA Advisory Committee Vote:
The Gastrointestinal Drugs Advisory Committee voted 13 to 1 against confirming the clinical benefits of Ocaliva (obeticholic acid) in primary biliary cholangitis (PBC), citing concerns for real possible harm.

Safety Concerns:
The FDA's analysis of post-marketing data showed unfavorable trends in liver transplantation and mortality associated with Ocaliva use.

Clinical Trials:
The confirmatory trial (747-302) failed to demonstrate efficacy on the primary composite endpoint of liver transplantation, death, and other liver-related outcomes.

Observational Study:
An observational cohort study did not demonstrate clinical benefit compared to PBC patients not treated with Ocaliva.

Regulatory Impact:
The negative advisory committee vote puts the future of Ocaliva's approval for PBC at risk, potentially leading to the withdrawal of its accelerated approval.

Background:
Ocaliva was granted accelerated approval in 2016 for PBC, but subsequent trials and real-world evidence have raised significant safety concerns.