Work from Birmingham led by Dr Gideon Hirschfield has contributed to FDA approval on April 7th 2016 of the first new drug approved for use in the liver disease primary biliary cirrhosis (PBC) in 20 years.
PBC is a rare liver disease that primarily results from autoimmune destruction of the bile ducts that transport bile acids out of the liver, resulting in cholestasis. It is primarily a disease of women, afflicting approximately one in 1,000 women over the age of 40. In Europe, the disease accounts for approximately half of liver transplants due to cholestatic diseases and 6% of all liver transplants.
Data from UK-PBC played a key role in convincing the FDA committee that there is significant unmet need in PBC and thus a need for new drugs.