FDA staff raises efficacy concerns over Intercept's liver disease drug

(Reuters) -The U.S. Food and Drug Administration's staff on Wednesday raised concerns over the efficacy of Intercept Pharmaceuticals' liver disease drug, which is awaiting the regulator's traditional approval.

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.

The comments come ahead of a meeting on Sept. 13, where independent experts will discuss and vote on the drug's confirmatory trial data.

Intercept, which holds the commercial rights to the drug, was acquired by Italy's pharmaceutical group Alfasigma for nearly $800 million last year.

(Reporting by Bhanvi Satija in Bengaluru; Editing by Shailesh Kuber)