Intercept Pharma Stock Tumbles: FDA Panel Votes Against Intercept's Fatty Liver Drug, Dashing Its Dreams To Enter NASH Space

FDA's Gastrointestinal Drugs Advisory Committee voted 12 to 2—with two abstentions—that the benefits of Intercept Pharmaceutical Inc's ICPT Ocaliva 25 mg, also known as Obeticholic acid (OCA), don't outweigh the risks in nonalcoholic steatohepatitis (NASH) patients with stage 2 or 3 fibrosis.

Fifteen of 16 voting-eligible GIDAC members (with no abstentions) voted to defer approval until clinical outcome data from trial 747-303 are submitted and reviewed, at which time the traditional approval pathway could be considered.

While the FDA isn't beholden to the advice of its advisory committees, the regulator often follows its experts' lead. Intercept's NASH candidate Ocaliva is up for an approval decision by June 22.

The drug is already sold to treat primary biliary cholangitis, another liver disease. If Intercept snags approval, its med will become the first FDA-approved NASH therapy.

Responding to the FDA decision, Intercept CEO Jerry Durso said, "We are disappointed in the outcome of today's meeting."

"We continue to disagree with the FDA on certain characterizations of OCA's efficacy and safety in pre-cirrhotic fibrosis due to NASH and the role of non-invasive tests (NITs), as discussed in today's meeting," the statement added.

The FDA briefing document highlighted an increased risk of diabetes and liver injury from using OCA oral tablets to treat NASH.

In the briefing documents, the FDA reviewers raised concerns about the drug's risk-benefit profile. "During the course of our review, FDA identified modest benefits and serious risks of OCA for treatment of NASH," they wrote.

Price Action: ICPT shares are down 11.90% at $11.94 during the premarket session on the last check Monday.