FDA Grants Rare Pediatric Disease Designation to IBAT Inhibitor for Treatment for PFIC

The US Food and Drug Administration (FDA) has granted rare pediatric disease designation to Albiero Pharma, Inc.’s A4250 for the treatment of progressive familial intrahepatic cholestasis (PFIC), a rare liver disease for which no approved treatment is currently available.

“The designation affirms Albiero’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,” Albiero’s president and chief executive officer, Ron Cooper, said in a recent statement. “A priority review voucher is a very valuable and important component of the incentives to develop products for rare, life-threatening diseases.”

A4250 is an ileal bile acid transporter (IBAT) inhibitor. When flow of bile from the liver is hindered or stopped, bile acids will build up in the liver; these elevated levels of bile acid have been linked with severe pruritus in patients with cholestatic liver diseases. The IBAT works to mediate the update of bile acids from the small intestine to the liver, according to Albiero. A4250 inhibits the IBAT which works to reduce the bile acids returning to the liver.

Recently, the first patient has been enrolled in a single, randomized, double-blind, placebo-controlled phase 3 trial dubbed PEDFIC-1, which will assess the drug in 60 patients with PCIF (subtype 1 or 2) between the ages of 6 months and 18 years who have high serum bile acid (sBA) levels as well as pruritus.

If results of the trial come back as positive, these data, along with data from an open-label extension study will form the basis of drug approval applications for the drug in the United States and the European Union (EU) for treatment of PFIC patients, according to Albiero.

Previous to the rare pediatric disease designation, in 2012, A4250 received orphan drug designation for the treatment of PFIC in the United States and the EU. Furthermore, in 2016, the pharmaceutical company was granted access to the European Medicines Agency’s PRIority Medicines (PRIME) program for the treatment of PFIC.

Estimated to affect 1 in every 50,000 to 100,000 children worldwide, PFIC causes progressive, life-threatening liver disease. To date, there are no treatment options available to patients with PFIC that have received approval.