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FDA Grants Albireo Pharma ODD

For lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor, for the treatment of Alagille syndrome

Albireo Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor, for the treatment of Alagille syndrome, a rare and life-threatening disease that affects the liver and has no approved pharmacologic treatment option.


A4250 also holds Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment of Alagille, and Orphan Drug designations from both the FDA and EMA for the treatment of progressive intrahepatic cholestasis (PFIC). The FDA grants Orphan Drug Designation to novel drugs that seek to treat a rare disease or condition and provides 7 years of market exclusivity for the product upon regulatory approval.

“We are very pleased with the FDA´s designation of A4250 as an orphan drug for the treatment of Alagille syndrome,” said Ron Cooper, president and chief executive officer of Albireo. “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. This further highlights the development potential of A4250.”

Alagille syndrome is a genetic disorder that can affect the liver, heart, skeleton, eyes and kidneys. Liver damage caused by cholestasis (bile flow blockage) is a major feature of the disease. Bile ducts may be narrow, malformed or fewer in number. As a result, bile builds up in the liver and causes scarring. 

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