Albireo Wins FDA Approval for Drug to Treat Rare Liver Disease

Boston biotech Albireo Pharma has won FDA approval for Bylvay (odevixibat), the first drug approved for the treatment of a rare liver disease called progressive familial intrahepatic cholestasis or PFIC. PFIC is a rare and devastating disorder affecting young children that causes progressive, life-threatening liver disease. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life. Until now, there have been no approved drugs for PFIC. Only surgical options and liver transplantation have been available, and without them, most PFIC patients do not survive past the age of 30. There are approximately 15,000 with PFIC worldwide (excluding China and India).

“Until now invasive surgery was the only approved treatment option. With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” said Emily Ventura, leader of PFIC Advocacy and Resource Network and mother to a PFIC patient. “As a community, we experience extreme challenges and diminished quality of life for children and families with PFIC. Managing the symptoms can be extremely difficult — the burden is unimaginable with our kids suffering physically, emotionally and developmentally.” (Source: Albireo Pharma Website, 20 July 2021)