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FDA panel backs Intercept's rare liver disease drug

Ocaliva recommended for accelerated approval as a primary biliary cholangitis treatment
FDA

Intercept Pharma has secured a positive FDA advisory committee vote for its rare liver disease therapy Ocaliva, considered by some to be a blockbuster-in-waiting.

The FDA's Gastrointestinal Drugs Advisory Committee voted unanimously (17 to 0) to recommend accelerated approval of Ocaliva (obeticholic acid) as a treatment for primary biliary cholangitis, a progressive disorder that will result in liver failure and death unless patients can get a liver transplant.

If approved, Ocaliva would become the first new therapy for PBC in almost two decades, according to Intercept, which has also filed for approval of the drug in Europe and is planning for a possible launch there before year-end.

The drug is intended for patients inadequately treated by or intolerant of standard first-line treatment ursodiol (ursodeoxycholic acid), the only approved therapy for PBC. The FDA is due to deliver a verdict on the US application by 29 May.

While Ocaliva is thought to represent a clinical advance in PBC, much of the excitement surrounding the commercial potential of the drug lies in its potential in other forms of fibrotic liver diseases, most notably non-alcoholic steatohepatitis (NASH) which affects up to 15 million people in the US and is predicted to become the leading indication of liver transplantation by 2020.

Intercept bagged a breakthrough designation from the FDA for Ocaliva in NASH patients in January 2015, kicking off a phase III programme last September with the start of the REGENERATE trial in non-cirrhotic NASH patients with advanced fibrosis.

In December the company started the phase II CONTROL trial, which will evaluate the effect of Ocaliva in combination with statin therapy on lipid metabolism in patients with NASH.

As a NASH therapy Ocaliva could face competition from Gilead Sciences' simtuzumab (in phase II), an earlier stage acetyl-CoA carboxylase (ACC) inhibitor candidate just acquired from Nimbus Therapeutics, as well as candidates from Shire, Genfit, Galectin, Roche/InterMune and Takeda.

Nevertheless, analysts at Wedbush Morgan have predicted that Ocaliva could eventually become a $2.2bn product.

Intercept owns worldwide rights to Ocaliva outside Japan and China, where it has out-licensed the drug to Sumitomo Dainippon Pharma.

Article by
Phil Taylor

11th April 2016

From: Research

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