The U.S. Food and Drug Administration granted a fast-track designation to Albireo Pharma Inc.'s A4250 to treat a rare and life-threatening liver disease.
A4250 received the designation to treat progressive familial intrahepatic cholestasis, which currently has no approved pharmacologic treatment option. The Boston-based biopharmaceutical company is investigating A4250 in a phase 3 study, named PEDFIC-1, as a treatment for this disease.
The fast-track designation facilitates development and expedites review of drugs to treat serious conditions and fill an unmet need.
The FDA has also granted the drug a rare pediatric disease designation allowing it to apply for a priority review voucher.
A4250 also received an orphan drug designation from the FDA for treating Alagille syndrome, a genetic disorder that can affect the liver, heart, skeleton, eyes and kidneys. The disease can result in liver damage caused by bile flow blockage.
The drug also has an orphan drug designation from the European Medicines Agency to treat Alagille, and orphan drug designations from both the FDA and EMA for the treatment of progressive intrahepatic cholestasis.
The FDA grants the orphan drug status 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.