FDA advisers unconvinced by Intercept's liver disease drug benefits, safety

By Bhanvi Satija and Sruthi Narasimha Chari

(Reuters) -Independent advisers to the U.S. Food and Drug Administration said on Friday that the available data from Intercept's liver disease drug does not clearly prove its effectiveness or remove doubts about its safety, putting into question the treatment's future in the U.S. market.

Intercept's Ocaliva received FDA's accelerated approval in 2016 to treat patients with primary biliary cholangitis, a rare disease that causes inflammation of small bile ducts in the liver and can eventually destroy them.

Under the accelerated pathway, the FDA mandates additional trials that verify the drug's benefits. A failure to do so could lead the agency to withdraw the drug from the market.

Several advisers said that the benefits of Ocaliva were inconclusive and that there was evidence of patients with advanced liver scarring that could be harmed by the drug, echoing concerns raised by FDA staff earlier this week.

In 2021, the FDA restricted Ocaliva's use in patients with less liver scarring due to safety issues. Intercept also sought the FDA's approval twice for the use of Ocaliva in the treatment of nonalcoholic steatohepatitis, or NASH, but received rejections both times.

"There might be a benefit, perhaps a slight benefit, but it's not clearly or convincingly a verified benefit," said FDA's Frank Anania, a non-voting panel member.

The panel voted 13-1 against the available data, and 10 panelists voted against benefits of Ocaliva outweighing its risks.

Advisers acknowledged the challenges of collecting real world data for a rare condition but said that data from an additional observational study of the drug could not be relied upon.

Panelist Almut Winterstein said the question for FDA would be "whether there is enough promise for Oca(liva) to remain on the market."

The FDA is set to make its decision on a traditional approval for the drug by Oct. 15. It usually follows the panel's advice but is not obligated to do so.

(Reporting by Bhanvi Satija and Sruthi Narasimha Chari in Bengaluru; Editing by Alan Barona)