Ipsen has announced that its Bylvay drug has failed to meet the primary endpoint in a major Phase 3 clinical trial targeting biliary atresia in young children.
The French pharmaceutical company revealed on July 24, 2026, that the Phase 3 BOLD trial evaluating Bylvay, also known as odevixibat, did not demonstrate improvement in native liver survival versus placebo.
The trial was testing Bylvay in patients living with biliary atresia who had already undergone a Kasai hepatoportoenterostomy surgical procedure.
Ipsen noted that current topline data is in line with the well-established safety profile of odevixibat in its approved indications, offering some limited reassurance despite the disappointing result.
The BOLD trial is a randomised, double-blind, placebo-controlled study and stands as the largest trial ever conducted evaluating disease modification specifically in biliary atresia patients.
The trial enrolled 254 patients across 19 countries, all of whom had undergone Kasai hepatoportoenterostomy surgery within the first 90 days of life, receiving either odevixibat or placebo once daily for up to 104 weeks.
Biliary atresia is a rare and life-threatening pediatric liver disease and the leading cause of liver transplantation in infants, often before a child reaches the age of two.
With no approved medical therapies currently available for the condition, treatment options remain limited to surgery or transplantation, making the failed trial a significant blow to an already thin pipeline.
Bylvay is already approved in other rare pediatric liver diseases, including Progressive Familial Intrahepatic Cholestasis and Alagille syndrome, and posted 180 million euros in sales in 2025.
The failure represents a further setback for Ipsen’s Albireo portfolio, acquired in a $952 million deal that originally centred on the already-approved Bylvay as its primary commercial asset.
Ipsen also picked up other potential liver medicines through the Albireo acquisition, including ritivixibat and A2342, though neither compound is currently being actively developed by the company.
The BOLD trial result narrows the treatment landscape for biliary atresia patients even further, leaving liver transplantation as the primary long-term option for children diagnosed with the condition.