Negative FDA AdCom vote on Ocaliva

The Gastrointestinal Drugs Advisory Committee (GIDAC) of the US Food and Drug Administration (FDA) met on Friday to discuss Intercept Pharmaceuticals’ supplemental New Drug Application (sNDA) for Ocaliva (obeticholic acid, OCA) for the treatment of primary biliary cholangitis (PBC) – a rare, progressive disease that disproportionally affects women.

The AdCom voted 10-1 (with three abstentions) that the farnesoid X receptor agonist did not have a favorable benefit-risk profile when used as a second-line treatment for eligible adults with PBC and no contraindications. As for the question of whether there is clinical benefit of obeticholic acid in the first place, the vote was 13-1 against approval.

One of those voting no to both questions was Daniel Gillen, a statistician from the University of California Irvine, who said it came down to not seeing a verifiable benefit and a reasonable question of harm.