Albireo Presents Data on Greater Efficacy in PFIC with Earlier Bylvay Treatment at NASPGHAN - Seite 2

PFIC is a rare genetic disorder that causes progressive, life-threatening liver disease. Patients with PFIC have impaired bile flow, or cholestasis, and the resulting bile build-up in liver cells causes liver disease and symptoms, such as intense itching, poor sleep, delayed growth, and diminished quality of life. The harmful impacts of the disease extend to parents and caregivers, as the 2022 multinational PICTURE study revealed that PFIC negatively affects caregivers’ quality of life, relationships, and career prospects.

New Analyses of the PEDFIC 1 and PEDFIC 2 Trials
The global PEDFIC trials represent the largest studies ever completed in children with PFIC. New pooled data analyses from PEDFIC 1, a randomized, double-blind, placebo-controlled Phase 3 trial that evaluated the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids (sBAs) in children with PFIC, and PEDFIC 2, a long-term, open-label Phase 3 extension study, are being shared at NASPGHAN in an oral presentation and two poster presentations. An additional poster presents data showing that Bylvay may be mixed in liquids to give to the youngest patients:

Benefit to Treating Earlier with Bylvay

Oral Presentation: Hepatic Impairment Classifications at Baseline in Responders to Odevixibat Therapy in Children with Progressive Familial Intrahepatic Cholestasis
Lead Author: Dr. Lorenzo D’Antiga, Department of Paediatric Hepatology, Gastroenterology, and Transplantation, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy
Session Title: Concurrent Session V – Potent P’s in Hepatology: A daily double
Date & Time: Saturday, October 15, 2:00pm – 3:30pm ET

Pooled data analysis showed that treatment with Bylvay is associated with improvements in serum bile acids and pruritus and suggests greater efficacy when treatment with Bylvay occurs earlier in the disease. Significantly more patients with mild hepatic impairment at baseline, according to Child-Pugh scores, had a serum bile acid response than patients with more severe hepatic impairment at baseline. Among patients with mild and moderate hepatic impairment at baseline, 45% and 24% were Bylvay responders, respectively (p value=0.039). No patients had Child-Pugh scores of severe hepatic impairment at baseline. Across baseline hepatic impairment levels, pruritus response rates were comparable and Bylvay was generally well tolerated.