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Albireo Announces Presentation of New Data in Paediatric and Adult Liver Diseases

  • New analyses of PEDFIC Phase 3 trials show that patients whose pruritus improves on Bylvay® (odevixibat) also experience improvements in sleep, quality of life, serum bile acids and hepatic parameters
  • Long-term data from PEDFIC trials of Bylvay show dramatic quality of life deficits in patients and families affected by PFIC
  • Studies show consistent and sustained efficacy for Bylvay across all PFIC types
  • Announces new SPARK Grants Program, accepting applications for projects to improve care for rare liver diseases, starting with PFIC

BOSTON — June 23, 2022 — Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, today announced the presentation of new data in pediatric and adult liver diseases at the European Association for the Study of the Liver (EASL) International Liver Congress™ 2022 being held in London. The presentations provide new analyses of the Phase 3 PEDFIC 1 and PEDFIC 2 trials of Bylvay® (odevixibat) in progressive familial intrahepatic cholestasis (PFIC), as well as data on pipeline ASBT/NTCP inhibitor compounds, including A2342 in Hepatitis B and D.

“With the largest data set ever compiled on children and families affected by PFIC, we’ve been able to quantify the significant impact of PFIC and illustrate the myriad ways that it dramatically harms patients’ and families’ quality of life,” said Jan Mattsson, Ph.D., Chief Scientific Officer and Head of R&D. “We’ve also shown that when patients with various types of PFIC find relief from their pruritus with Bylvay, they experience benefits across sleep, quality of life and hepatic parameters. Seeing these improvements across the board underscores the urgent need to assess and treat patients’ pruritus.”

Albireo also launched SPARK, a new grant-based program established to identify and drive improvements in the quality of care for rare liver diseases, beginning with PFIC in 2022. SPARK is designed to encourage new ideas with the potential to demonstrate best practices in patient-centered care. Healthcare professionals, advocates, and other PFIC experts are invited to submit applications at starting July 1st.

“PFIC data and research are important because they reinforce why we need to prioritize the treatment of patients’ pruritus,” said Francesca Lombardozzi, PFIC caregiver. “What I hear from families coping with PFIC is that pruritus impacts all aspects of their lives, most critically their sleep – leaving the entire family exhausted at times. I encourage families to advocate for effective pruritus treatment for their children as it has the potential to improve many aspects of their daily life.”

Bylvay PEDFIC 1 and PEDFIC 2 Data

The Company presented data on Bylvay in five posters. Bylvay is a potent, non-systemic once daily ileal bile acid transport inhibitor (IBATi) that is the only treatment approved in Europe for the treatment of all types of PFIC in patients aged 6 months or older and the only treatment approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of PFIC. The PEDFIC 1 trial was the first and largest, global, pivotal Phase 3 study conducted in PFIC, which evaluated the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids (sBAs) in a randomized, double-blind, placebo-controlled trial, and PEDFIC 2 is a long-term, open-label Phase 3 extension study. Key findings from the data include:

Long-Term Efficacy and Safety Data Show Bylvay Improves Quality of Life for Patients and Families Across PFIC Subtypes

  • Bylvay Responders Experience Improvements in Quality of Life Measures (poster #763). This analysis of pooled data from the PEDFIC studies reveals that all patients had large quality of life deficits at baseline across emotional functioning, social functioning, physical functioning and school functioning, with mean scores between 47 and 67 on the PedsQL. Patients who responded to Bylvay treatment, and particularly their families, experienced mean quality of life improvements that were sustained over time, while quality of life for patients who did not respond to Bylvay was largely unchanged for them and their families. Of 84 total patients in the pooled population, PedsQL data were available for 53 patients, and PEDsQL Family Impact (FI) data were available for 79 patients. Among patients with available QoL data, both responders and non-responders had impaired QoL at least some of the time at baseline. PEDsQL total scores showed improved QoL for responders vs non-responders over time up to week 72.


  • Bylvay Provides Durable Treatment Benefits (poster #1197). In this pooled analysis of data from the PEDFIC studies, Bylvay provided durable clinical treatment benefits in patients with PFIC who received treatment for at least 72 weeks, with mean improvements over time in sBA levels, pruritus, hepatic parameters and growth. Bylvay was generally well tolerated during ≥ 72 weeks of treatment. Among the subgroup of patients treated with Bylvay for ≥72 weeks, 96% had a TEAE and 52% experienced drug-related TEAEs. The most common TEAEs were pyrexia, upper respiratory tract infection, and diarrhea.


  • Improvements in Pruritus on Bylvay Lead to Improvements in Quality of Life, Hepatic Biomarkers, and Sleep (poster #865). Results of pooled analysis population of 82 patients treated with Bylvay including 44 pruritus responders of PEDFIC 1 and PEDFIC 2 show that patients across various types of PFIC who had a pruritus response with Bylvay experienced improvements in quality of life, sleep and hepatic biomarkers that were sustained for up to 72 weeks. From baseline to week 72, pruritus responders had significant quality of life improvements, as assessed by mean PEDsQL total scores (p=0.048) and PEDsQL FI total scores (p=0.007). Data also indicated that pruritus responders had significant improvements in several caregiver-reported sleep parameters (all P<0.001), including reductions in days with blood due to scratching, days needing help falling asleep, days needing soothing, and days sleeping with caregiver. Among all pruritus responders, 86% had any TEAE, none of which were drug related serious TEAEs.​Two patients experienced TEAEs that led to study discontinuation, which included splenomegaly, diarrhoea, jaundice, decreased weight, and/or hypophagia​.


  • Bylvay Treatment Impacts Hepatic Parameters, Growth, Sleep, and Biochemical Markers Across PFIC Types (poster #850). Patients with across various types of PFIC treated with Bylvay for up to 72 weeks, with some tracked up to 128 weeks, experienced reductions in autotaxin levels and increases in C4 levels, as well as variable changes in hepatic parameters, sleep characteristics and growth. 84 patients received Bylvay during the pooled analysis period. Patients of almost all PFIC types experienced mean improvements in caregiver-reported sleep parameters. Patients of most PFIC types generally had mean increases in height and/or weight Z scores with Bylvay treatment; patients with PFIC1 and PFIC3 experienced more variable changes in weight Z scores. Among all patients, 85% had any TEAE, which was similar across all PFIC typesand mild or moderate in severity​. All serious TEAEs were assessed as unrelated to study drug.


Evidence of Bylvay Efficacy, With and Without Concomitant UDCA

  • Total, Primary, and Secondary Serum Bile Acid Changes and Pruritus Improvement During Bylvay Treatment (Poster #847). A large proportion of patients in the PEDFIC 1 trial were on UDCA at baseline, but still had elevated sBAs and pruritus. This analysis assessed mean total, primary and secondary sBAs among Bylvay sBA responders, partial responders (≥ 30% reduction) and nonresponders, and showed that UDCA treatment resulted in higher sBAs at baseline, but it did not seem to affect sBA response to Bylvay. A sBA partial response was not associated with a mean decrease in pruritus with concomitant UDCA use and all sBA responder groups included patients for whom pruritus response did not correlate with sBA response.


Pipeline Data for A2342 and Other Investigational Compounds

Albireo is investigating several ASBT and NTCP inhibitor compounds. The apical sodium-dependent bile acid transporter (ASBT) and Na+ taurocholate co-transporting polypeptide (NTCP) play key roles in maintaining bile acid homeostasis, and their selective inhibition has shown efficacy in liver diseases. Key findings from the data presentations include:

  • The Orally Available Sodium/Taurocholate Co-Transporting Polypeptide Inhibitor A2342 Blocks Hepatitis B And D Entry In Vitro (Poster #1226). This preclinical study of A2342 supports its potential as the first orally bioavailable molecule able to inhibit Hepatitis B and D entry via NTCP in the nanomolar range.
  • Dual Ileal/Renal-Liver Bile Acid Transporter Inhibitors with Different Transporter Selectivity in Vitro Differentially Increase Faecal and Urinary Bile Acid Excretion in Organic Anion-Transporting Polypeptide 1a/1b Knockout Mice in Vivo (Poster #1238). This preclinical study showed that dual ASBT/NTCP inhibitors with different selectivity may have a role as part of precision medicine strategies to reduce the bile acid burden in forms of hepatobiliary disease.

Albireo hosted a company-sponsored symposium that will feature a case blended approach of idiopathic cholestasis and targeted next-generation sequencing panels.


Symposium: Idiopathic Cholestasis and Targeted Next-Generation Sequencing Panels: A Case Blended Approach

Expert panel: Prof. Patrick McKiernan, Birmingham Children's Hospital, NHS Foundation Trust, UK, Dr. Christoph Leiskau, Hannover Medical School, Germany, Dr. Angelo Di Giorgio, Hospital Papa Giovanni XXIII, Bergamo, Italy

Date & Time: Thursday, 23 June 23, 12:30-13:30 BST at ExCel London


Albireo Announces Presentation of New Data in Paediatric and Adult Liver Diseases

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Last Updated: 27-Jun-2022