Pharmaceutical

Albireo Pharma (Nasdaq:ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor, for the treatment of progressive familial intrahepatic cholestasis (PFIC), a rare and life-threatening …

Albireo Pharma (Nasdaq:ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor, for the treatment of progressive familial intrahepatic cholestasis (PFIC), a rare and life-threatening liver disease with no approved pharmacologic treatment option.

As quoted in the press release:

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

Albireo recently announced the enrollment of the first patient in PEDFIC-1, a single, randomized, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate A4250 in 60 patients aged 6 months to 18 years with PFIC (subtype 1 or 2) who have elevated serum bile acid (sBA) 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.

Click here to read the full press release.

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