FDA advisers recommend Intercept's potential liver disease blockbuster

Advisers for the US Food and Drug Administration have unanimously recommended accelerated approval for Intercept Pharmaceuticals' potential blockbuster obeticholic acid for a rare liver disease – just days after Gilead surprised the market by buying a rival liver drug company.

Speculation had been growing that Gilead would buy Intercept, which said the FDA's Gastrointestinal Drugs Advisory Committee voted 17 to 0 to recommend approval of obeticholic acid in primary biliary cholangitis.

But Gilead appears to have passed on Intercept, and instead opted to buy Nimbus Therapeutics in a deal worth up to $1.2 billion.

Obeticholic acid, which will have the brand name of Ocaliva in the US and EU should it make it to market, came top of a list of seven potential blockbuster drugs likely to be approved this year compiled by Thomson Reuters in February.

According to the Drugs to Watch 2016 report, Ocaliva could achieve sales in excess of $2.6 billion if approved in chronic liver diseases, of which primary biliary cholangitis is the biggest indication.

The FDA is not bound by the committee's guidance, but takes advice into consideration when reviewing investigational medicines. However the vote of confidence from the committee was enough to send Intercept's shares up by almost 6% in pre-market trading following the announcement.

If approved, Ocaliva will be the first new treatment for primary biliary cholangitis, previously known as primary biliary cirrhosis, for 20 years.

The FDA is due to make a regulatory decision on obeticholic acid by 29 May, after asking for more information from Intercept and extending the review period.

Intercept is seeking accelerated approval of Ocaliva for PBC in patients with an inadequate response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA), the only approved therapy for this disease.

While UDCA has a marked impact on clinical outcomes in PBC, a substantial percentage of UDCA-treated patients have a suboptimal response or are intolerant to treatment, leaving them at significantly increased risk of an adverse outcome.

The committee's recommendation is based on development data including the phase 3 POISE trial assessing safety and efficacy of Ocaliva in 216 PBC patients who had an inadequate therapeutic response to, or are unable to tolerate, UDCA.

Intercept's US filing included data from 432 PBC patients. It has also begun a phase 4 long-term outcomes trial to confirm the clinical benefit of Ocaliva in people living with primary biliary cholangitis.

The European Medicines Agency is also reviewing obeticholic acid. Intercept last year began a phase 3 trial of the drug in non-cirrhotic nonalcoholic steatohepatitis (NASH) with liver fibrosis.

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