PFIC1

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Completed 1

New drug shows promise for rare childhood liver disease

Disease control Completed

This study tested a drug called odevixibat (A4250) in 62 children with progressive familial intrahepatic cholestasis (PFIC) types 1 and 2, a rare liver disease that causes severe itching and bile buildup. The drug works by blocking the reabsorption of bile acids in the gut. The g…

Matched conditions: PFIC1

Phase: PHASE3 • Sponsor: Albireo • Aim: Disease control

Last updated Jun 27, 2026 08:00 UTC

PFIC1

Get alerted when new PFIC1 trials appear

New drug shows promise for rare childhood liver disease