The U.S. Food and Drug Administration granted the rare pediatric disease designation to an Albireo Pharma, Inc. medicine to treat a type of liver disease.
The Boston-based biopharmaceutical company is investigating A4250 as a treatment of progressive familial intrahepatic cholestasis — a genetic disorder with no approved pharmacologic treatment option.
The U.S. FDA grants the tag to therapies that treat disorders primarily affecting children from birth to 18 years old and below 200,000 people in the country.
Albireo said it is eligible to apply for rare pediatric disease priority review voucher upon submission of a new drug application for A4250.
The company said that if its phase 3 trial of A4250 in patients with the disease is successful, the data will be used for securing drug approval in the U.S. and EU for treating progressive familial intrahepatic cholestasis.
Albireo said A4250 received the orphan drug designation for the same use in the U.S. and EU in 2012.
