Albireo Pharma announces orphan drug designation for A4250

Albireo Pharma announced the FDA has granted orphan drug designation to lead product candidate A4250, an ileal bile acid transporter, or IBAT, inhibitor, for the treatment of biliary atresia, a rare and life-threatening liver disease with no approved pharmacologic treatment option. A4250 now holds orphan drug designations from both the FDA and EMA for the treatment of progressive familial intrahepatic cholestasis, or PFIC, Alagille syndrome, biliary atresia and primary biliary cholangitis. 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.

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