Results from a phase III study presented at the 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 primary biliary cirrhosis.
Obeticholic acid at both a 10 mg dose and a 5 mg dose titrated to 10 mg, 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 10 mg OCA group and 46% in the 5-10 mg OCA group versus only 10% in the placebo group ( both dose groups p less than 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.
While UDCA has been the standard primary biliary cirrhosis therapy for the past 20 years, a significant percentage of patients fail to get an adequate response with this treatment, or are unable to tolerate it.
Primary biliary cirrhosis is a chronic disease that primarily occurs in women. It is characterised by the destruction of bile ducts in the liver, which in turn leads to liver scarring.
Long-term damage from primary biliary cirrhosis over the years can result in cirrhosis and liver failure.
Primary biliary cirrhosis affects around 30 people per million, with an estimated prevalence of 12,000 - 15,000 in the UK. It has been reported that primary biliary cirrhosis is more prevalent in some geographic areas, such as Northern Europe and Northern America.
OCA is a new bile acid analogue ( 6alpha-ethyl-chenodeoxycholic acid ) and first-in-class agonist of the nuclear receptor ( FXR ), which represents the central bile acid sensor in humans.
It is being studied in primary biliary cirrhosis, as well as non-alcoholic steatohepatitis ( NASH ) and other liver and intestinal diseases. ( Xagena )
Source: European Association for the Study of the Liver ( EASL ), 2014