Albireo enrolls 1st patient in Phase 3 pediatric liver disease med trial

Albireo Pharma announced the first patient has been enrolled in PEDFIC-1, a Phase 3 clinical trial of lead product candidate A4250, an ileal bile acid transporter inhibitor being studied for the treatment of patients with progressive familial intrahepatic cholestasis. 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 problematic clinical presentation 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 PFIC. The Phase 3 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, 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 a 40 or 120 mug/kg oral dose of A4250 or placebo once daily for 24 weeks. The primary endpoint for the US Food and Drug Administration evaluation will be an assessment of change in pruritus, and the primary endpoint for the European Medicines Agency evaluation will be sBA responder rate.

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