Intercept Pharma now has approval for its Ocaliva drug for primary biliary cholangitis (PBC) therapy Ocaliva on both sides of the Atlantic after getting a green light for the drug in the EU.
The European Medicines Agency (EMA) granted a conditional approval for Ocaliva (obeticholic acid) in combination with the only approved PBC therapy – ursodeoxycholic acid (UDCA) – in adult patients who can’t tolerate or don’t respond to UDCA on its own.
PBC is a rare and life-threatening disease that causes the gradual destruction of the small bile ducts in the liver, allowing bile to build up in the liver and cause damage. Ocaliva is a first-in-class drug that works by stimulating the farnesoid X receptor, reducing the production of bile.
Intercept launched Ocaliva in the US earlier this year – the first new drug for PBC in 20 years – and in both the US and EU full approval depends on the outcome of two ongoing clinical trials.
The company reported sales of just under $5m for the drug in the third quarter, and at the time the company’s commercial head Richard Kim said that it will take time to change prescribing behaviour as PBC is a slowly progressing disease.
“Despite the availability of UDCA, many patients have remained at significant risk of adverse outcomes with no alternative treatment option available,” said Frederik Nevens of University Hospitals Leuven & KU Leuven, who led the phase III POISE trial submitted in support of the marketing application.
“Ocaliva can now help fill an important unmet need for these patients,” he added.
Analysts at Credit Suisse predict that Ocaliva could make more than $300m as a PBC therapy in 2020, but they say a bigger payoff for Intercept will come if it can claim approval for the drug in non-alcoholic steatohepatitis (NASH). That will push the 2020 sales up above the $1bn level, they suggest.
It has been predicted that NASH and other forms of non-alcoholic fatty liver disease (NAFLD) could become a $35bn to $40bn market by 2025.
Other FXR agonists in clinical development include Novartis’ LJN 452 and Gilead Sciences/Phenex’ GS 9674, both of which are in phase II testing for PBC and NASH, and Allergan/Arkana’s AKN-083 in early development.