Albireo enrolls first patient in phase 3 trial for pediatric cholestasis treatment

Albireo enrolled the first patient in a phase 3 clinical trial for its lead product candidate A4250, an ileal bile acid transporter inhibitor designed to treat pediatric patients with progressive familial intrahepatic cholestasis, according to a press release.

Ron Cooper

“Pediatric cholestatic liver disease is a very serious condition, particularly PFIC, which has high morbidity and mortality,” Ron Cooper, president and CEO of Albireo, told Healio Gastroenterology and Liver Disease. “These children are suffering from horrible pruritus and the prognosis for reduction is very poor.”

The primary endpoint for this phase 3 randomized control trial for the U.S. FDA is reduction of serum bile acids, which serves as the secondary endpoint for the European Medicines Agency. For Europe, the primary endpoint is the reduction of pruritus, which serves as the secondary endpoint in the U.S.

The trial will include 60 patients aged between 6 months and 18 years with PFIC subtype 1 or 2, elevated serum bile acid levels and pruritus.

The U.S. and European Union approved an orphan drug designation for A4250 in the treatment of PFIC and the EMA granted access to its Priority Medicines (PRIME) program for the treatment of PFIC.

“A4250 represents an opportunity to provide the first pharmacological treatment for this terrible disease,” Cooper said. “We believe that if the outcomes of this phase 3 trial are successful, we could provide a really important treatment option.” – by Talitha Bennett

Reference: www.albireopharma.com

Disclosure: Cooper is an employee of Albireo.