European agency approves plan for pediatric cholestatic medication

The Pediatric Committee, or PDCO, of the European Medicines Agency agreed to Albireo’s pediatric investigation plan for its lead product candidate, A4250, in pediatric patients with progressive familial intrahepatic cholestasis, according to a company press release.

“Because we are developing A4250 to treat a pediatric patient population, we are especially pleased that the PDCO ... has agreed to our pediatric investigation plan for A4250 in [progressive familial intrahepatic cholestasis],” Ron Cooper, president and CEO of Albireo, said in the release. “We believe A4250 has the potential with phase 3 success to make a significant difference in the lives of children suffering from this debilitating rare liver disease and their families. We plan to initiate our previously announced phase 3 trial of A4250 by year end.”

A4250 is a product candidate in development for progressive familial intrahepatic cholestasis and potentially other orphan pediatric cholestatic liver diseases. It is a highly potent and selective inhibitor of the ileal bile acid transport with minimal systematic exposure that acts locally in the gut.

The pediatric investigational plan for A4250 includes Albireo’s previously announced phase 3 clinical trial in patients aged 6 months to 18 years with progressive familial intrahepatic cholestasis and data from the completed phase 2 study in children with cholestatic liver disease.

After the phase 3 trial, Albireo will conduct a small clinical trial in neonates with progressive familial intrahepatic cholestasis. – by Talitha Bennett

Reference: www.albireopharma.com