Ipsen's IBAT inhibitor Bylvay has failed a phase 3 trial in biliary atresia (BA), a rare and serious liver disease that affects babies and has limited treatment options. At the moment, the go-to treatment is a surgical procedure known as a Kasai hepatoportoenterostomy (HPE), which connects a loop of the small intestine directly to the liver surface to restore bile flow. "Biliary atresia remains the number one cause of liver transplantation in children, often before the age of 2," said lead BOLD investigator Dr Saul Karpen of the Stravitz-Sanyal Institute for Liver Disease and Metabolic Health at Virginia Commonwealth University in the US. "Research remains limited, leaving patients, families and clinicians with very few therapeutic options," he added. There have been other setbacks in Albireo's liver disease pipeline for Ipsen, which shelved ritivixibat, another IBAT inhibitor in development for primary sclerosing cholangitis (PSC), and NTCP inhibitor A2342 earlier this year.