Posted by Michael Wonder on 13 Jun 2018
FDA grants rare paediatric disease designation to A4250; Albireo eligible to apply for priority review voucher
12 June 2018 - Albireo Pharma a clinical-stage orphan paediatric liver disease company developing novel bile acid modulators, today announced the U.S. FDA has granted rare paediatric disease designation to lead product candidate A4250, an ileal bile acid transporter inhibitor, for the treatment of progressive familial intrahepatic cholestasis, a rare and life-threatening liver disease with no approved pharmacologic treatment option.
Albireo recently announced the enrolment of the first patient in PEDFIC-1, a single, randomised, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate A4250 in 60 patients aged 6 months to 18 years with progressive familial intrahepatic cholestasis (PFIC) (subtype 1 or 2) who have elevated serum bile acid levels and pruritus. If successful, data from the Phase 3 trial and an open-label extension study are expected to form the basis of the drug approval applications for A4250 in the U.S. and E.U. for the treatment of patients with PFIC.
