A4250 Granted Rare Pediatric Disease Designation for Progressive Cholestasis

It is a highly potent and selective inhibitor of IBAT that works locally in the gut while exerting minimal systemic exposure.
It is a highly potent and selective inhibitor of IBAT that works locally in the gut while exerting minimal systemic exposure.

Albireo Pharma announced that the Food and Drug Administration (FDA) has granted rare pediatric disease designation to A4250 for the treatment of progressive familial intrahepatic cholestasis (PFIC)

PFIC can often lead to cirrhosis and liver failure within the first decade of life. A4250 is a first-in-class investigational ileal bile acid transporter (IBAT) being developed to treat the progressive, life-threatening hepatic disease for which there is no approved treatment. It is a highly potent and selective inhibitor of IBAT that works locally in the gut while exerting minimal systemic exposure. 

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The first patient has been enrolled in PEDFIC-1, a phase 3, single, randomized, double-blind, placebo-controlled trial designed to evaluate A4250 in 60 patients (aged 6 months–18 years) with PFIC subtype 1 or 2 who have elevated serum bile acid (sBA) levels and pruritus. Data from this trial and a subsequent open-label extension study will be submitted as part of drug approval applications for A4250 as a new treatment for patients with PFIC.

For more information visit AlbireoPharma.com.