Albireo Enrolls First Patient in Phase 3 Clinical Trial of Odevixibat in Biliary Atresia - Seite 2

BOLD (NCT04336722) is a double-blind, randomized, placebo-controlled trial to evaluate the efficacy and safety of odevixibat in children who have biliary atresia and have undergone a Kasai procedure before age three months. Children in the treatment arm will receive odevixibat and escalate to 120 μg/kg orally once daily for 24 months. The 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, and secondary outcome measures include time to onset of any sentinel events, total bilirubin levels and serum bile acid levels. The trial will enroll approximately 200 patients at up to 75 sites globally.   

“We’re pleased to expand the evaluation of odevixibat into a second pivotal trial with the initiation of the precedent-setting BOLD trial for patients with biliary atresia, the most common rare pediatric liver disease,” said Ron Cooper, President and Chief Executive Officer of Albireo. “We look forward to continuing our work to realize the potential of odevixibat as a much-needed treatment option for patients across multiple cholestatic liver diseases with BOLD and our PEDFIC 1 Phase 3 trial of odevixibat in progressive familial intrahepatic cholestasis, which we continue to expect topline data from in mid-2020.”

In addition to the BOLD and PEDFIC 1 trials, Albireo is finalizing a third pivotal trial of odevixibat in Alagille syndrome and anticipates initiating the trial by the end of 2020. 

About Odevixibat
Odevixibat is a product candidate being developed to treat rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome. A highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), odevixibat has minimal systemic exposure and acts locally in the small intestine. Odevixibat is being evaluated in the PEDFIC 1 Phase 3 clinical trial in patients with PFIC (NCT03566238), as well as the BOLD Phase 3 clinical trial in patients with biliary atresia (NCT04336722).

The odevixibat PFIC program, or elements of it, have received fast track, rare pediatric disease and orphan drug designations in the United States. In addition, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to odevixibat for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. The European Medicines Agency (EMA) has granted odevixibat orphan designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. Its Pediatric Committee has agreed to Albireo’s odevixibat Pediatric Investigation Plan for PFIC. EMA also has granted orphan designation to odevixibat for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis.