Update on Ipsen's Phase III BOLD Trial in Biliary Atresia

On July 24, 2026, Ipsen announced that the Phase III BOLD trial for biliary atresia did not meet its primary endpoint. The trial evaluated the efficacy of Bylvay (odevixibat) compared to a placebo in patients who had undergone a Kasai hepatoportoenterostomy. Biliary atresia, a rare liver disease in infants, remains a leading cause of pediatric liver transplants, often necessitating surgery or transplantation as no medical treatments exist.

The trial included 254 patients across 19 countries but showed no improvement in native liver survival. Despite the setback, the trial provided valuable data on this complex disease. Ipsen acknowledged the contributions of participants and emphasized the ongoing need for research in this area.

Bylvay, an ileal bile acid transport inhibitor, is currently approved for other liver conditions but has not shown efficacy in biliary atresia through this study. Further analysis of the trial's comprehensive data is anticipated to yield insights into disease biology and potential future treatments.