European Commission grants conditional approval to Ocaliva (obeticholic acid) for the treatment of primary biliary cholangitis.- Intercept Pharma.

Intercept Pharmaceuticals, Inc.announced that the European Commission has granted conditional approval for Ocaliva (obeticholic acid) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA. Ocaliva is a potent and selective agonist of the farnesoid X receptor (FXR), which is expressed at high levels in the liver and intestine and thought to be a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways.

The marketing authorization was based on efficacy and safety data derived from three randomized double-blind, placebo-controlled clinical trials evaluating the effect of Ocaliva on alkaline phosphatase (ALP) and bilirubin in patients with PBC. It was also supported by two clinical databases that include more than 10,000 patients from the Global PBC Study Group and UK-PBC Group, both independently confirming that achieving lower ALP and/or bilirubin levels is significantly correlated with increased transplant-free survival.

In the Phase III POISE study, nearly half of patients (46%) in the titration group treated with Ocaliva in combination with UDCA achieved the primary endpoint compared to 10% in the control group (placebo added to UDCA) (p<0.0001). Additionally, 77% of patients taking Ocaliva in combination with UDCA achieved a reduction of more than 15% in ALP at 12 months, compared to 29% taking UDCA alone.