FDA Flags Concerns Over Intercept’s Ocaliva Approval in Advisory Committee Briefing

Advisory Committee Meeting:
The FDA's Gastrointestinal Drugs Advisory Committee met on September 13, 2024, to discuss supplemental new drug application (sNDA) 207999 S-011 for Ocaliva (obeticholic acid) 5 mg titrated to 10 mg oral tablets.

Concerns Raised:
The FDA flagged concerns in the advisory committee briefing document regarding the efficacy and safety of Ocaliva for the treatment of adult patients with primary biliary cholangitis without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension.

Postmarketing Requirements:
The sNDA was submitted to fulfill the accelerated approval postmarketing requirements specified in the Ocaliva approval letter dated May 27, 2016.

Clinical Benefit:
The sNDA included data proposed to describe and verify clinical benefit for the indication, which was a point of discussion during the advisory committee meeting.

Previous Discussions:
There were previous discussions on Ocaliva's use for pre-cirrhotic liver fibrosis due to nonalcoholic steatohepatitis (NASH) in a May 19, 2023, meeting of the Gastrointestinal Drugs Advisory Committee.

Regulatory Process:
The FDA's advisory committees provide independent expert advice on regulatory issues, and their recommendations are non-binding but generally followed by the FDA.