Albireo Pharma¬†has enrolled¬†first patient in global¬†Phase 3 trial¬†(BOLD) of odevixibat for biliary atresia, expanding the development of odevixibat to a second rare cholestatic liver disease indication.
Odevixibat selectively inhibits a protein called the ileal bile acid transporter (IBAT), which plays a key role in transporting bile from the liver to the colon.
200-subject trial’s primary efficacy endpoint is improvement in the proportion of patients who are alive and have not undergone a liver transplant after two years of treatment, compared to placebo.
Children in the treatment arm will receive odevixibat and escalate to 120 Œºg/kg orally once daily for 24 months.
In addition to the BOLD and PEDFIC 1 trials, Albireo is finalizing a third pivotal trial of odevixibat in Alagille syndrome and anticipates to initiate the trial by the end of 2020.