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Albireo gets FDA rare pediatric disease designation for rare liver disease treatment A4250

PBR Staff Writer Published 13 June 2018

Albireo Pharma has secured rare pediatric disease designation from the US Food and Drug Administration for its product candidate, A4250, to treat progressive familial intrahepatic cholestasis (PFIC).

PFIC is a rare and life-threatening liver disease with no approved pharmacologic treatment option.

A4250 is an ileal bile acid transporter (IBAT) inhibitor, which is being developed for the treatment of rare pediatric cholestatic liver diseases.

The first-in-class product candidate is said to have minimal systemic exposure and acts locally in the gut.

Recently, the company recruited first patient in single, randomized, double-blind and placebo-controlled phase 3 clinical trial called PEDFIC-1.

The trial has been designed to assess A4250 in 60 patients aged six months to 18 years with PFIC (subtype 1 or 2) who have elevated serum bile acid (sBA) levels and pruritus.

The results from the trial will be used to file drug approval applications for A4250 in the US and EU to treat patients with PFIC.

In 2012, the company also secured orphan drug designation for A4250 to treat PFIC in the US and EU. In 2016, the firm secured access to the EMA’s PRIority MEdicines (PRIME) program to treat PFIC.

According to the company, the PFIC is estimated to affect between one in every 50,000 to 100,000 children born across the globe, and causes progressive and life-threatening liver disease.

Albireo president and CEO Ron Cooper said: “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.

“A priority review voucher is a very valuable and important component of the incentives to develop products for rare, life-threatening diseases.”

Based in Boston of Massachusetts, Albireo Pharma is engaged in the development of novel bile acid modulators for the treatment of orphan pediatric liver diseases, as well as other liver and gastrointestinal diseases and disorders.