Treatment for Rare Genetic Diseases of Liver Granted Orphan Drug Designation

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Albireo AB announced that A4250 has been granted orphan-drug designation by the FDA for the treatment of progressive familial intrahepatic cholestasis (PFIC) and primary biliary cirrhosis (PBC). A4250 belongs to a class of inhibitors of the ileal bile acid transporter (IBAT, syn. apical sodium-dependent bile acid transporter ASBT).

Orphan designation was granted based on an extensive preclinical data package showing that A4250 has a high potency and a minimal systemic exposure. In addition, substantial benefits were shown in an animal model of cholestasis; the predominant feature of PFIC and PBC.

A4250 decreases the re-absorption of bile acids and will reduce the toxic levels of bile acids in the liver cells of patients with PFIC and PBC. In preclinical models of cholestasis, treatment with A4250 has been shown to be a very potent inhibitor of IBAT/ASBT with only minimal systemic exposure; thereby reducing the risk for systemic side effects and studies have shown that A4250 decreases the damage in the liver cells and the development of fibrosis/cirrhosis of the liver known to occur in PFIC and PBC.

For more information visit www.albireopharma.com.

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