Albireo Pharma announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to A4250 for the treatment of biliary atresia. 

Currently, there is no FDA-approved treatment for biliary atresia, a rare pediatric liver disease. A4250 is a first-in-class ileal bile acid transporter (IBAT) inhibitor being developed for rare pediatric cholestatic hepatic diseases. 

Inhibition of IBAT, which plays a main role in enterohepatic circulation, may lead to decreased bile acids returning to the liver. A4250 works through highly potent and selective inhibition and exerts minimal systemic exposure as it acts locally in the small intestine.  

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A4250 has already received Orphan Drug designation for the treatment of progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome, and primary biliary cholangitis (PBC). It is currently in Phase 3 development for progressive familial intrahepatic cholestasis. Findings from an open-label Phase 2 study in pediatric patients with cholestatic liver disease and pruritus showed favorable tolerability as well as reductions in serum bile acids and pruritus in most patients. 

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