Ron Cooper, President and CEO of Albireo, discusses drug development at Albireo, which has a clinical trial in progressive familial intrahepatic cholestasis (PFIC), a rare genetic condition that affects the liver. PFIC 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.
