Albireo granted rare pediatric disease designation for A4250

Albireo Pharma (Nasdaq: ALBO), a USA-based biotechnology company spun out from Anglo-Swedish drug major AstraZeneca in 2008,  announced on Tuesday that it has been granted Rare Pediatric disease designation by the FDA for A4250 for the treatment of progressive familial intrahepatic cholestasis (PFIC), a rare liver disease with no approved drug therapy.

“This designation affirms Albireo’s eligibility to apply for a rare pediatric disease priority review voucher upon submission of a new drug application for A4250 and highlights the serious, life-threatening manifestations of PFIC,” said Ron Cooper, president and chief executive of Albireo, adding: “A priority review voucher is a very valuable and important component of the incentives to develop products for rare, life-threatening diseases.”

These vouchers can also be traded on to other pharma companies, and have changed hands for hundreds of million dollars.