Ron Cooper, President and CEO of Albireo, provides and overview of Cholestatic liver diseases. Cholestasis is a chronic condition resulting from an impairment of the biliary system, that provokes a decrease or interruption of bile secretion from the liver to the intestine, leading to the degeneration of liver tissues, chronic inflammation and the formation of scar tissue (fibrosis). The consequences are serious, and if not treated cholestasis will invariably progress towards cirrhosis, hepatic insufficiency, and ultimately liver transplantation.
Albiero has a phase three trial for progressive familial intrahepatic cholestasis type (PFIC) is a rare condition that affects the liver. PFIC is a rare genetic disorder that is estimated to affect between one in every 50,000 to 100,000 children born worldwide and causes progressive, life-threatening liver disease.
The most prominent and problematic ongoing manifestation of PFIC is pruritus, which often results in a severely diminished quality of life. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all children with PFIC will require treatment before age 30. There are no medicines currently approved for PFIC, only surgical options – including a procedure known as Partial External Biliary Diversion (PEBD) or liver transplantation. These options carry substantial risk of post-surgical complications, as well as psychological and social issues.