FDA grants Albireo's A4250 orphan-drug designation for treatment of PFIC and PBC

Albireo AB (Gothenburg, Sweden; "Albireo"), a biopharmaceutical company specializing in gastroenterology, today announced that A4250 (an inhibitor of the ileal bile acid transporter, IBAT/ASBT) has been granted orphan-drug designation by the U.S Food and Drug Administration ("FDA") for the treatment of

• Progressive Familial Intrahepatic Cholestasis (PFIC)
• Primary Biliary Cirrhosis (PBC)

Orphan-drug designation is granted by the FDA to novel drugs that seek to treat a rare disease or condition and provides seven years of market exclusivity for the product upon regulatory approval. Albireo has previously received similar orphan drug designation for the same disease areas from the European Commission.

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.

"We are very pleased with the FDA´s recognition of our technology with A4250 as an orphan medicinal product for the treatment of these liver diseases," said Dr. Hans Graffner, Chief Medical officer at Albireo. "This designation is an important milestone in our efforts to provide a treatment for patients with cholestatic liver diseases; a disease entity often leading to severe symptoms, liver transplantation and with reduced survival. A4250 is an inhibitor of the bile acid transport mechanism and will decrease the toxic levels of bile acids in the liver cells. The approvals of the orphan drug designation for A4250 both in Europe and in the US provide evidence of the quality of the A4250 research and give us confidence to move forward with the development of A4250 in these liver diseases."