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PFIC Overview

Ron Cooper, President and CEO of Albireo Pharma gives an overview of progressive familial intrahepatic cholestasis (PFIC). As Mr. Cooper explains, PFIC is a rare genetic disorder that causes progressive, life-threatening liver disease due to an inability to properly bile acids from the liver. The most common manifestation of PFIC is pruritus, which often results in a severely diminished quality of life. In many cases, this disease leads to cirrhosis and liver failure within the first 10 years of life, and nearly all PFIC patients require treatment before the age of 40 years. The current treatments for PFIC are predominantly off-label drugs and surgery. Due to positive results of Albireo’s phase 3 trial for odevixibat, Mr. Cooper is optimistic that this drug will soon become the first treatment approved for PFIC. For more information about PFIC and other rare metabolic disorders, visit checkrare.com/diseases/metabolic-disorders/