Albireo Pharma, Inc. announced the presentation of new data at the NASPGHAN Annual Meeting being held in Orlando, Florida from October 12-15, 2022. A new analysis of pooled data from the Phase 3 PEDFIC 1 and PEDFIC 2 trials demonstrates that Bylvay (odevixibat) reduces serum bile acids and pruritus in children with progressive familial intrahepatic cholestasis (PFIC) who have varying levels of baseline hepatic impairment. Importantly, the analysis shows that significantly more patients with mild hepatic impairment at baseline had a serum bile acid response, suggesting children may benefit from earlier treatment with Bylvay.

Additional data analyses presented at the meeting show that Bylvay reduced pruritus in patients regardless of their baseline pruritus scores. Further, pruritus reductions were associated with improvements in serum bile acids, hepatic parameters, growth, and disease aspects related to sleep, with greater reductions in pruritus associated, in general, with greater improvements across these additional measures. 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 large 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: 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.

Direct Link Between Sustained Level of Pruritus Reduction and Magnitude of Improvement in Other Disease Parameters: An analysis of pooled data from 77 patients treated with Bylvay showed that pruritus reductions were associated with improvements in serum bile acids, hepatic parameters, growth, and disease aspects related to sleep and, in general, patients who had larger-magnitude pruritus reductions had larger-magnitude improvements in other outcomes. The median percentage change from baseline to weeks 70–72 in serum bile acid level was –12% in patients who had pruritus reductions of <1, –54% in patients who had reductions of =1 to <2, –94% in patients who had reductions of =2 to <3,–96% in patients who had reductions of =3 to <4, and –94% in patients who achieved a pruritus score of 0 or 1. Bylvay was generally well tolerated regardless of the level of pruritus reduction. Evidence of Bylvay Efficacy in Patients with Lower Baseline Pruritus Severity: A pooled data analysis of 16 patients who had medium or lower pruritus severity at baseline showed that treatment with Bylvay was associated with improvement in serum bile acids, pruritus, hepatic parameters, growth, sleep, and quality of life (QoL), suggesting even patients with lower baseline pruritus severity may benefit from Bylvay treatment.

Treatment with Bylvay led to a serum bile acid response in 31% of patients; improvements in mean percentage of days needing help falling asleep, falling from baseline of 41% to 18%; needing soothing falling from 37% to 19%; and sleeping with a caregiver falling from 36% to 16%; but not in percentage of days with scratching associated with bleeding, which was 9% at baseline and 30% at weeks 61-72. Bylvay was generally well tolerated. Stability of Bylvay Mixed in Liquid for Pediatric Dosing: An in vitro study showed that when Bylvay is mixed in a range of liquids often used to administer medicines to young infants, it is stable and provides children with an adequate dose.

As the symptoms of PFIC often begin in infancy, when a child is not yet eating solid food, it is important that parents have an option to mix Bylvay in a liquid to give to their young children.