The reviewers, in briefing documents published on the FDA’s website, said the confirmatory trial for the drug did not provide evidence that it was effective in patients with primary biliary cholangitis (PBC).
Intercept’s oral drug, Ocaliva, received an accelerated approval from the FDA in 2016 as a treatment for PBC, a rare chronic condition in which the body mistakes bile ducts in the liver as foreign objects and tries to destroy their lining.
In 2021, the U.S. health regulator restricted use of the drug to PBC patients who did not have advanced cirrhosis or severe scarring of the liver.
Intercept is now hoping to convert the accelerated nod, which comes with the caveat of a confirmatory trial, into a traditional approval.=
Staff advisers said that an analysis of Ocaliva’s confirmatory trial did not show that it was effective in PBC patients with or without advanced cirrhosis.