Intercept's travails continue as it pulls NASH drug filing in EU

Already scrambling to refile its obeticholic acid (OCA) drug for non-alcoholic steatohepatitis (NASH) after the FDA rejected it last year, Intercept Pharma was hoping for more luck in Europe, but has now withdrawn its EU marketing application.

Intercept had been hoping to extend the review period in the EU to allow time for results from an ongoing to phase 3 trial to come in, now due in the first quarter of next year, but conceded defeat yesterday.

Now, the company will have to refile OCA in the EU, kicking off a new review period which will delay a possible launch of the drug, which is vying to become the first approved therapy for liver fibrosis in NASH patients. Shares in Intercept slipped around 15% after it announced the news.

Intercept first filed for approval of OCA in the EU and US in 2019, but the FDA finally turned down the US application in June 2020 after twice extending what should have been a swift six-month review of the drug.

The company said at the time that the US regulator decided biomarker data on liver fibrosis was not sufficient to support accelerated approval, and it would need to see NASH outcomes results with longer follow-up.

Intercept's former chief executive Mark Pruzanski criticised that stance, saying the company had been blindsided by the FDA as it had not indicated the drug wasn't approvable on the surrogate endpoint.

His replacement Jerry Durso said that the company is now "in the midst of producing the largest data package in the NASH field, which will include significantly more evidence regarding the benefit/risk of OCA in NASH."

OCA has also been found to cause severe itching in some patients, which has led some analysts to predict that Intercept may find it hard to build a market for the drug even if it is first to the NASH market.

"Intercept is in the process of generating additional efficacy and safety data from REGENERATE, including a full reread of month 18 biopsies using a new consensus biopsy reading approach…to support a potential refiling in the US," said the company.

It added that it will take a decision on refiling in Europe only after that new data comes in.

The active ingredient has already been approved as Ocaliva to treat rare disease primary biliary cholangitis (PBC), which brought in $93 million in third quarter sales, but approval in NASH will be required if the OCA franchise is ever to meet its once-promised blockbuster sales potential.

Along with REGENERATE, Intercept is also running the REVERSE trial in patients with compensated cirrhosis due to NASH, which could read out towards the end of this year.