FDA Adcomm Endorses Intercept’s Obeticholic Acid in Unanimous Vote

Weighing the risks and benefits of Intercept Pharmaceuticals’ obeticholic acid, an FDA advisory committee voted unanimously 17-0 on the significance of alkaline phosphatase levels as evidence to justify an accelerated approval of the primary biliary cirrhosis candidate.

Members of the Gastrointestinal Drugs Advisory Committee expressed support for the use of alkaline phosphatase (ALP) as an adequate surrogate endpoint to forecast clinical benefit in early-stage primary biliary cirrhosis (PBC). Panelists also suggested bilirubin as a prospective additional primary endpoint.

Ninety percent of patients enrolled in the Phase 3 pivotal trial had early-stage PBC, prompting panelists to request more data to substantiate the use of OCA in patients with moderate to severe OCA. Some panelists deemed the data sufficient in proving a benefit for patients with moderately advanced PBC, but not advanced PBC.