Obeticholic acid produces meaningful biochemical and clinical improvements in PBC cirrhosis patients

Results from an international Phase III study presented at the European Association for the Study of the Liver’s (EASL) International Liver Congress 2014 have shown obeticholic acid (OCA) given to patients suffering from primary biliary cirrhosis (PBC) who previously had an inadequate response to, or have been unable to tolerate ursodeoxycholic acid (UDCA), produced meaningful biochemical and clinical improvements. UDCA is the only therapy currently approved to treat PBC.

Under development by USA-based Intercept Pharmaceuticals (Nasdaq: ICPT), obeticholic acid at both a 10mg and a 5mg dose titrated to 10mg, met the trial's primary composite endpoint of achieving a serum alkaline phosphatase (ALP) activity of less than 1.67 times the upper limit of normal (ULN), a total bilirubin within normal limits, and at least a 15% decrease in ALP.

The proportion of patients meeting the primary endpoint was: 47% in the 10mg OCA group and 46% in the 5mg-10mg OCA group versus only 10% in the placebo group (both dose groups p<0.0001). In addition, both OCA dose groups met secondary endpoints of improvements in other liver function parameters, including gamma-glutamyl transferase (GGT), alanine aminotransferase (ALT) and total bilirubin.