Intercept’s Ocaliva gains PBC thumbs in Europe, but with caveats

Five months after gaining FDA approval, Intercept Pharmaceuticals ($ICPT) has now been given the CHMP nod for its new liver drug Ocaliva (obeticholic acid) for primary biliary cirrhosis (PBC).

The New York-based biotech, which hopes this initial licence will be the start of bigger things in the future, has seen the EMA’s drug arm given a yes to its med, in combination with ursodeoxycholic acid (UDCA), in patients who have not responded adequately to UDCA--or on its own in those who are unable to tolerate treatment with UDCA.

The final decision will be made by the European Commission, a process that is typically a rubber stamp deal that should be done by the end of the year.

The recommendation comes with caveats, however, as the biotech has to give up results from two studies. “Until availability of full data, the CHMP will review the benefits and risks of Ocaliva annually to determine whether the conditional marketing authorisation can be maintained,” the group said in a statement.

“Although it is a rare disease, PBC remains one of the most common indications for liver transplant among women in Europe,” explained David Jones, Professor of Liver Immunology at Newcastle University and Consultant Hepatologist at Newcastle upon Tyne Hospitals Trust.

“There is substantial unmet need in this disease and a real urgency around the need for new therapies to help the many PBC patients who are either intolerant of the single existing approved therapy ursodeoxycholic acid or don't respond to it sufficiently to protect their livers and prevent the development of cirrhosis and the need for transplant.”

While happy with its PBC licences, it will be eyeing the potentially much bigger indication of non-alcoholic fatty liver disease, or NASH, as its main prize.

This disease space affects millions of Americans as the rate of obesity grows, and could be worth as much as $40 billion in the drug therapy stakes, given that there are no specific meds on the market for this condition. Intercept is not alone, and is racing a host of other biotechs and pharmas to be the first to market, but some have seen its PBC licence wins as a positive for its NASH hopes.

A range of blockbuster scenarios have been touted for the med at peak, with the highest set seen should it gain the fatty liver disease license. It has however seen setbacks in testing for NASH, including missing the mark in a Phase II a year ago, but it is planning to peek at some interim data from its latest test in 2019 to see whether it can go for an early FDA approval.

Intercept’s drug works as a semi-synthetic bile acid and activates the farnesoid X receptor (FXR), which controls the production of bile. By activating this receptor, Ocaliva is expected to reduce the production of bile in the liver, thus reducing the exposure of the liver to toxic levels of bile acids.