June 12, 2018
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Albireo receives rare pediatric disease designation for A4250

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Albireo Pharma Inc. received a rare pediatric disease designation from the FDA for its lead product candidate A4250, an ileal bile acid transporter inhibitor designed for the treatment of progressive familial intrahepatic cholestasis, according to a company press release.

Albireo recently enrolled the first patient in a phase 3 clinical trial for A4250. The primary endpoint of the trial in the U.S. is a reduction of serum bile acids, and the secondary endpoint is the reduction of pruritus.

“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 [progressive familial intrahepatic cholestasis (PFIC)],” Ron Cooper, president and CEO of Albireo, said in the release. “A priority review voucher is a very valuable and important component of the incentives to develop products for rare, life-threatening diseases.”

The company noted that A4250 already received an orphan drug designation in the U.S. and Europe in 2012 and Albireo received access in 2016 to the European Medicines Agency’s Priority Medicines (PRIME) program for the treatment of PFIC.

Reference: www.albireopharma.com