New hope for kids with rare liver disease: long-term drug shows promise
Disease control
Completed
This study looked at the long-term safety and effectiveness of a drug called A4250 (odevixibat) in 116 children with a rare liver disease called progressive familial intrahepatic cholestasis (PFIC). The goal was to see if the drug could lower bile acid levels and reduce itching o…
Phase: PHASE3 • Sponsor: Albireo, an Ipsen Company • Aim: Disease control
Last updated May 17, 2026 10:42 UTC