Albireo Enrolls First Patient in Late-Stage Study of Treatment for Life-Threatening Pediatric Liver Disorder

Rare Daily Staff

Albireo Pharma said it enrolled the first patient in a late-stage clinical trial of its lead product candidate A4250, an experimental therapy for the treatment of patients with progressive familial intrahepatic cholestasis, a rare and deadly liver disease.

PFIC is estimated to affect between one in every 50,000 to 100,000 children born worldwide and causes progressive, life-threatening liver disease. Moderate to severe pruritus is a common and problem of PFIC that can severely diminish quality of life. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all patients with PFIC require treatment before age 30. There are currently no approved pharmacological treatment options for the condition.

A4250 is a first-in-class product candidate being developed to treat rare pediatric cholestatic liver diseases and is in late-stage development in its initial indication of PFIC. It is a highly potent and selective inhibitor of the ileal bile acid transporter.

The late-stage program includes a single randomized, double-blind, placebo-controlled clinical trial designed to evaluate A4250 in 60 patients, ages 6 months to 18 years, with PFIC (subtype 1 or 2), elevated serum bile acid levels and pruritus, and an open-label extension study to assess long-term safety and durability of response. Patients in the double-blind trial will receive one of two doses or a placebo once daily for 24 weeks.

The primary endpoint for the U.S. Food and Drug Administration evaluation will be an assessment of change in pruritus. The European Medicines Agency evaluation will use serum bile acid responder rate as the primary endpoint.

A4250 has received orphan drug designation for PFIC in the United States and European Union and has been granted access to the EMA’s Priority Medicines (PRIME) program for the treatment of PFIC.

“Our team is committed to working expeditiously to activate clinical trial sites around the world, complete the study and seek regulatory approval as soon as possible,” said Ron Cooper, president and CEO of Albireo.

May 16, 2018
Photo: Ron Cooper, president and CEO of Albireo