Albireo Pharma announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to A4250 for the treatment of Alagille syndrome, a rare and life-threatening liver disease. This genetic disorder is characterized by liver damage caused by cholestasis with symptoms including jaundice, poor weight gain and growth, and severe pruritus; signs and symptoms usually become evident in infancy or early childhood. 

Currently, there is no approved treatment for Alagille syndrome. A4250 is a first-in-class ileal bile acid transporter (IBAT) inhibitor, that is being developed for rare pediatric cholestatic liver diseases. It acts locally in the gut as a highly potent and selective inhibitor of IBAT with minimal systemic exposure. 

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A Phase 3 trial, PEDFIC-1, is currently being conducted to investigate the safety and efficacy of low and high doses of A4250 in children with progressive intrahepatic cholestasis (PFIC). “While we are focused on executing our PEDFIC-1 Phase 3 clinical trial of A4250 in PFIC, there is extremely high unmet need across numerous rare cholestatic liver diseases, including Alagille, ” said Ron Cooper, President and Chief Executive Officer of Albireo. “This further highlights the development potential of A4250.”

Alagille syndrome affects approximately 1 in 70,000 newborns.

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