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Publications

Bylvay® (odevixibat)

Christine Clemson, Quanhong Ni, Patrick Horn. Correlations Between Patient- and Caregiver-reported Pruritus From PEDFIC 1: Evaluation of PRUCISION Data in Patients With Progressive Familial Intrahepatic Cholestasis. Value Health. 2022;25(7 suppl 1):S322.
Buket Dalgic, Henkjan J. Verkade, Alain Lachaux et al. Relationships Between Decreases in Serum Bile Acids, Pruritus, and Sleep Disturbance Scores With Up to 72 Weeks of Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis. Gastroenterology. 2022;162(7 suppl):S1295–S1296.
Chad Gwaltney, Stephanie Bean, Meredith Venerus et al. Development of the Patient- and Observer-reported PRUCISION Instruments to Assess Pruritus and Sleep Disturbance in Pediatric Patients With Cholestatic Liver Diseases. Adv Ther. 2022, in press.
Tassos Grammatikopoulos, Buket Dalgic, Girish Gupte et al. Efficacy and Safety Outcomes in Patients With Progressive Familial Intrahepatic Cholestasis Who Had an Odevixibat Dose Escalation: Pooled Results From the PEDFIC 1 and PEDFIC 2 Studies. J Pediatr Gastroenterol Nutr. 2022;74(S2 suppl 2):645–6.
Lorenzo D’Antiga, Girish Gupte, Richard J. Thompson et al. Changes in Hepatic Parameters, Growth, Sleep, and Biochemical Markers With Odevixibat Treatment Across Patients With Various Types of Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2022;77(suppl 1):S518–9.

Kathleen M. Loomes, Henkjan J. Verkade, Richard J. Thompson et al. Effects on Serum Bile Acids, Pruritus, and Safety With Up to 72 Weeks of Odevixibat Treatment: Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Abstract accepted at the Annual Meeting of the British Society of Pediatric Gastroenterology, Hepatology, and Nutrition (BSPGHAN). April 25–27, 2022; Birmingham, UK.

VIEW ABSTRACT (Adapted at PFIC Network 2022 (following submission, congress requested that this data be combined with that from McKiernan et al. AASLD 2021 and from Kamath et al. NASPGHAN 2021)

Henkjan J. Verkade, on behalf of study co-authors. Effects on Serum Bile Acids, Pruritus, and Safety With up to 72 Weeks of Odevixibat Treatment: Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Oral presentation at the PFIC Network Scientific Conference. April 29–30, 2022; Pittsburgh, PA, USA. (Adapted from original presentations at AASLD 2021 by Loomes et al. and by McKiernan et al. and at NASPGHAN 2021 by Kamath et al.)
Saul J. Karpen, Greg Wright, Phillip Stein, Patrick Horn. The BOLD Study: A RCT to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BA). Abstract accepted at the 2nd International and Interdisciplinary Congress on Biliary Atresia and Related Diseases (BARD). June 17–18, 2022; Bruges, Belgium.
Richard J. Thompson, Lorenzo D’Antiga, Emmanuel Gonzalès, et al. Odevixibat Therapy Improves Clinically Meaningful Endpoints in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials. Spanish-translated abstract accepted at the 28th Meeting of the Sociedad Española de Gastroenterología, Hepatología y Nutrición Pediátrica (SEGHNP). June 16–18, 2022: Palma de Mallorca, Spain.
Emmanuel Gonzalès, Patrick McKiernan, Buket Dalgic et al. Safety and Efficacy of Treatment With Odevixibat in Children With PFIC: Report of the PEDFIC 1 and PEDFIC 2 Studies. French-translated abstract accepted at the 43rd Meeting of the Groupe Francophone d'Hépatologie-Gastroentérologie et Nutrition Pédiatriques (GFHGNP). March 31–April 2, 2022; Bordeaux, France. (Adapted from accepted abstracts from AASLD 2021 by Gonzalès et al. and by McKiernan et al. and from NASPGHAN 2021 by Dalgic et al.)
Tassos Grammatikopoulos, Elke Lainka, Reha Artan et al. Efficacy and Safety of Odevixibat Therapy With Concomitant UDCA or Rifampicin in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials. Abstract accepted at die Jahrestagung der Gesellschaft für Pädiatrische Gastroenterologie und Ernährung (GPGE). May 11–14, 2022; Rostock, Germany.
Binita M. Kamath, Richard J. Thompson, Ulrich Baumann et al. Efficacy And Safety Of Odevixibat in Children With Progressive Familial Intrahepatic Cholestasis With Prior Partial External Biliary Diversion. Abstract accepted at die Jahrestagung der Gesellschaft für Pädiatrische Gastroenterologie und Ernährung (GPGE). May 11–14, 2022; Rostock, Germany.
Chad Gwaltney, Cristina Ivanescu, Lisa Karlsson et al. Validation of the PRUCISION Instruments in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis. Adv Ther. 2022, in press.
Henkjan J. Verkade, Folkert Kuipers, Quanhong N et al. Total, Primary, and Secondary Serum Bile Acid Concentrations in Patients With Progressive Familial Intrahepatic Cholestasis With Serum Bile Acid Response or Not With Odevixibat Treatment: Assessing the Contribution of Ursodeoxycholic Acid Concentration. J Pediatr Gastroenterol Nutr. 2022;74(S2 suppl 2):847–8.
Girish Gupte, Piotr Czubkowski, Florence Lacaille et al. Disease Burden and Natural History of Progressive Familial Intrahepatic Cholestasis: Baseline Clinical Characteristics Among Odevixibat-Treated Patients in the Phase 3 PEDFIC Studies. Gastroenterology. 2022;162(7 suppl):S1295.
Girish Gupte, Richard J. Thompson, Lorenzo D’Antiga et al. Analysis of Quality of Life, Hepatic Biochemical Markers, and Sleep in Patients With Progressive Familial Intrahepatic Cholestasis Who Had a Pruritus Response With Odevixibat Treatment. J Hepatol. 2022;77(suppl 1):S519. 
Patrick McKiernan, Ekkehard Sturm, Binita M. Kamath et al. Hepatic Parameters, Growth, and Sleep With Responders and Nonresponders to Odevixibat Treatment: Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Abstract accepted at the Annual Meeting of the British Society of Pediatric Gastroenterology, Hepatology, and Nutrition (BSPGHAN). April 25–27, 2022; Birmingham, UK.
Lorenzo D’Antiga, Girish Gupte, Richard J. Thompson et al. Changes in Hepatic Parameters, Growth, Sleep, and Biochemical Markers With Odevixibat Treatment Across Patients With Various Types of Progressive Familial Intrahepatic Cholestasis. J Pediatr Gastroenterol Nutr. 2022;74(S2 suppl 2):664–5.
Binita M. Kamath, Richard J. Thompson, Quanhong Ni et al. Efficacy and Safety of Odevixibat in Children With Progressive Familial Intrahepatic Cholestasis With Prior Partial External Biliary Diversion. Gastroenterology. 2022;162(7 suppl):S1296.
Cara L. Mack, Chad Gwaltney, Quanhong Ni et al. Improvements in Quality of Life in Odevixibat Responders and Nonresponders: An Analysis of Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies. J Hepatol. 2022;77(suppl 1):S517. 
Hasan Özen, Etienne Sokal, Florence Lacaille et al. Efficacy and Safety Outcomes With Odevixibat in Children With Progressive Familial Intrahepatic Cholestasis Due to Deficiencies in Multidrug Resistance Protein 3 (PFIC Type 3) or Myosin 5B (PFIC Type 6). Abstract accepted at the Annual Meeting of the British Society of Pediatric Gastroenterology, Hepatology, and Nutrition (BSPGHAN). April 25–27, 2022; Birmingham, UK.
Richard J. Thompson, Henrik Arnell, Reha Artan, et al. Odevixibat Treatment in Progressive Familial Intrahepatic Cholestasis: A Randomised, Placebo-controlled, Phase 3 Trial. Lancet Gastroenterol Hepatol. 2022;S2468-1253(22)00093-0 (online ahead of print).
Richard J. Thompson, Emmanuel Gonzalès, Reha Artan et al. Efficacy and Safety of Odevixibat Over 72 Weeks of Treatment in Patients With Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2022;77(suppl 1):S521.
Richard J. Thompson, Reha Artan, Lorenzo D’Antiga et al. Long-term Efficacy and Safety of Odevixibat, an Ileal Bile Acid Transporter Inhibitor in Children With Progressive Familial Intrahepatic Cholestasis: Interim Results From PEDFIC 2, an Open-label Phase 3 Trial. Abstract accepted at the Annual Meeting of the British Society of Pediatric Gastroenterology, Hepatology, and Nutrition (BSPGHAN). April 27–29, 2021; Virtual.
Johanna Ohlendorf, Imeke Goldschmidt, Norman Junge et al. Ileal Bile Acid Transporter Inhibition Reduces Post-transplant Diarrhea and Growth Failure in FIC1 Disease–A Case Report. Children (Basel). 2022;9(5):669. (Externally written)
Girish Gupte, Richard J. Thompson, Lorenzo D’Antiga et al. Analysis of Quality of Life, Hepatic Biochemical Markers, and Sleep in Patients With Progressive Familial Intrahepatic Cholestasis Who Had a Pruritus Response With Odevixibat Treatment. J Pediatr Gastroenterol Nutr. 2022;74(S2 suppl 2):647–9.
Richard J. Thompson, Reha Artan, Ulrich Baumann, et al. Odevixibat Effects on Cholestasis-Related Parameters: Analysis of Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Gastroenterology. 2022;162(7 suppl):S1296–S1297.
Richard J. Thompson, Pier Luigi Calvo, Winita Hardikar, et al. Long-term Treatment With Odevixibat Improves Multiple Sleep Parameters in Patients With Progressive Familial Intrahepatic Cholestasis: A Pooled Responder Analysis From the Phase 3 PEDFIC Studies. Gastroenterology. 2022;162(7 suppl):S1297.
Henkjan J. Verkade, Folkert Kuipers, Quanhong Ni et al. Total, Primary, and Secondary Serum Bile Acid Changes and Pruritus Improvement During Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2022;77(suppl 1):S517–8.
Richard J. Thompson, Lorenzo D’Antiga, Emmanuel Gonzalès, et al. Odevixibat Therapy Improves Clinically Meaningful Endpoints in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials. Gastroenterology. 2022;162(7 suppl):S1296.
Heo, Young-A. Odevixibat in progressive familial intrahepatic cholestasis: a profile of its use. Adis Drug Q&A – Drugs & Therapy Perspectives. 2022.
Richard Thompson, Reha Artan, Ulrich Baumann et al. Odevixibat Effects on Cholestasis-related Parameters: Analysis of Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2021;75(suppl 2):S687.
Emmanuel M. Gonzalès, Richard J. Thompson, Binita Kamath et al. Relationships Between Changes in Autotaxin, Pruritus, and Serum Bile Acids After Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis: Data From a Pooled Analysis. Hepatology. 2021;74(suppl 1):30A–1A.
Buket Dalgic, Henkjan Verkade, Alain Lachaux et al. Relationships Between Decreases in Serum Bile Acids, Pruritus, and Sleep Disturbance Scores With Up to 72 Weeks of Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis. J Pediatr Gastroenterol Nutr. 2021;73(suppl 1):S49–50.
Tassos Grammatikopoulos, Carrie Thompson, Nisreen Soufi et al. Long-term Safety and Tolerability of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children With Progressive Familial Intrahepatic Cholestasis: Results From the Phase 3 PEDFIC 1 and PEDFIC 2 Studies. J Pediatr Gastroenterol Nutr. 2021;72(suppl 1):781–2.
Richard J. Thompson, Lise Kjems, Winita Hardikar et al. Improved Quality of Life in Children With Progressive Familial Intrahepatic Cholestasis Following 24 Weeks of Treatment With Odevixibat, an Ileal Bile Acid Transporter Inhibitor: Results From the Phase 3 PEDFIC 1 Study. Value Health. 2021;24(5 suppl 1):S155.
Richard Thompson, Pier Luigi Calvo, Winita Hardikar et al. Long-term Treatment With Odevixibat Improves Multiple Sleep Parameters in Patients With Progressive Familial Intrahepatic Cholestasis: A Pooled Responder Analysis From the Phase 3 PEDFIC Studies. J Hepatol. 2021;75(suppl 2):S683–4.
Kathleen M. Loomes, Henkjan J. Verkade, Richard J. Thompson et al. Effects on Serum Bile Acids, Pruritus, and Safety With Up to 72 Weeks of Odevixibat Treatment: Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Hepatology. 2021;74(suppl 1):1159A–60A.
Ulrich Baumann, Ekkehard Sturm, Florence Lacaille, et al. Effects of Odevixibat on Pruritus and Bile Acids in Children With Cholestatic Liver Disease: Phase 2 Study. Clin Res Hepatol Gastroenterol. 2021;45(5):101751.
Tassos Grammatikopoulos, Elke Lainka, Reha Artan et al. Efficacy and Safety of Odevixibat Therapy With Concomitant UDCA or Rifampicin in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials. J Pediatr Gastroenterol Nutr. 2021;73(suppl 1):S53–4.
Chad Gwaltney, Cristina Ivanescu, Lisa Karlsson et al. Validation of the PRUCISION Caregiver-reported Pruritus Measure Using Data From the Phase 3, Randomised PEDFIC 1 Trial in Paediatric Patients With Progressive Familial Intrahepatic Cholestasis. J Pediatr Gastroenterol Nutr. 2021;72(suppl 1):814–15.
Girish Gupte, Piotr Czubkowski, Florence Lacaille et al. Disease Burden and Natural History of Progressive Familial Intrahepatic Cholestasis: Baseline Clinical Characteristics Among Odevixibat-Treated Patients in the Phase 3 PEDFIC Studies. J Pediatr Gastroenterol Nutr. 2021;73(suppl 1):S197–8.
Richard J. Thompson, Henkjan J. Verkade, Emmanuel Gonzalès et al. Long-term Treatment Benefits of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children With Progressive Familial Intrahepatic Cholestasis: Improvements in Total Bilirubin, Growth, and Sleep as Secondary and Exploratory Outcome Measures From the Phase 3 PEDFIC 1 and PEDFIC 2 Studies. J Pediatr Gastroenterol Nutr. 2021;72(suppl 1):819–20.
Richard Thompson, Lorenzo D’Antiga, Emmanuel Gonzalès et al. Odevixibat Therapy Improves Clinically Meaningful Endpoints in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials. J Hepatol. 2021;75(suppl 2):S688.
Patrick James McKiernan, Ekkehard Sturm, Binita Kamath et al. Hepatic Parameters, Growth, and Sleep With Responders and Nonresponders to Odevixibat Treatment: Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Hepatology. 2021;74(suppl 1):1164A.
Leonardo Ruiz-Casas, Sonia O’Hara, Claudia Mighiu, et al. Burden of Illness of Progressive Familial Intrahepatic Cholestasis in the US, UK, France, and Germany: Study Rationale and Protocol of the PICTURE Study. Expert Rev Pharmacoecon Outcomes Res. 2021;21(2):247–53.
Richard Thompson, Lorenzo D’Antiga, Binita M. Kamath et al. Rapid and Durable Effect of Odevixibat on Clinical and Biochemical Parameters of Cholestasis in Children With Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2021;75(suppl 2):S279–80.
Hasan Özen, Etienne Sokal, Florence Lacaille, Buket Dalgic, Quanhong Ni, Lise Kjems, Patrick Horn. Efficacy and Safety Outcomes With Odevixibat in Children With Progressive Familial Intrahepatic Cholestasis Due to Deficiencies in Multidrug Resistance Protein 3 (PFIC Type 3) or Myosin 5B (PFIC Type 6). Hepatology. 2021;74(suppl 1):1160A–1A.
Binita Kamath, Richard Thompson, Quanhong Ni et al. Efficacy And Safety Of Odevixibat In Children With Progressive Familial Intrahepatic Cholestasis With Prior Partial External Biliary Diversion. J Pediatr Gastroenterol Nutr. 2021;73(suppl 1):S204–6.
Philip Stein, Greg Wright, Lise Kjems et al. Drug-drug Interaction Study to Evaluate the Interaction of A4250 (Odevixibat) With Midazolam, a Sensitive CYP3A4 Substrate, and Itraconazole, a P-gp Inhibitor, in Healthy Adult Subjects. J Pediatr Gastroenterol Nutr. 2021;72(suppl 1):873–4.
Richard Thompson, Patrick Horn, Roderick H.J. Houwen et al. Substantial Clinical Benefits With Odevixibat Treatment Across Progressive Familial Intrahepatic Cholestasis Genetic Deficiencies: Subgroup Analysis of Serum Bile Acids, Pruritus, and Safety Using Pooled Data From the PEDFIC 1 and 2 Studies. J Hepatol. 2021;75(suppl 2):S684–5.
Henkjan J. Verkade, Folkert Kuipers, Qifeng Yu, Velichka Valcheva, Patrick Horn, Lise Kjems. Natural Variation of Serum Bile Acid Levels and Pruritus in Children With Progressive Familial Intrahepatic Cholestasis: Pretreatment Data From Patients in the Phase 3 PEDFIC 1 Study. Hepatology. 2021;74(suppl 1):1171A–2A.
Nadia Ovchinsky, Jessica Savage, Lise Kjems, Patrick Horn. The ASSERT Study: A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat In Patients With Alagille Syndrome. J Pediatr Gastroenterol Nutr. 2021;73(suppl 1):S191–2.
Henkjan Verkade, Folkert Kuipers, Quanhong Ni et al. Pretreatment Serum Bile Acid Parameters and Predictability of Response to Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children With Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2021;75(suppl 2):S688–9.
Saul Karpen, Greg Wright, Phillip Stein, Patrick Horn. The BOLD Study: A Randomized, Placebo-controlled Trial to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Infants With Biliary Atresia After Kasai Portoenterostomy. J Pediatr Gastroenterol Nutr 2020;71(suppl 1):S435–6.
Richard J. Thompson, Ulrich Baumann, Piotr Czubkowski et al. Efficacy and Safety of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2: Results From PEDFIC 1, a Randomized, Double-blind, Placebo-controlled Phase 3 Trial. AASLD 2020 Late-breaking Abstracts. Abstract LO4; page 9.
Christoph Slavetinsky, Ekkehard Sturm. Odevixibat and Partial External Biliary Diversion Showed Equal Improvement of Cholestasis in a Patient with Progressive Familial Intrahepatic Cholestasis. BMJ Case Rep. 2020;13(6):e234185.
Philip Stein, Greg Wright, Lise Kjems et al. Drug-drug Interaction Study to Evaluate the Interaction of A4250 (Odevixibat) With Itraconazole, a P-gp Inhibitor in Healthy Adult Subjects. J Pediatr Gastroenterol Nutr. 2020;71(suppl 1):S433–4.
Richard J. Thompson, Reha Artan, Lorenzo D’Antiga et al. Long-term Efficacy and Safety of Odevixibat, an Ileal Bile Acid Transporter Inhibitor in Children With Progressive Familial Intrahepatic Cholestasis: Interim Results From PEDFIC 2, an Open-label Phase 3 Trial. AASLD 2020 Late-breaking Abstracts. Abstract LP19; page 34–5.
Per-Göran Gillberg, Jan Mattsson, Kristina Torfgård et al. Clinical Pharmacology of Odevixibat, a Potent, Selective Ileal Bile Acid Transport Inhibitor with Minimal Systemic Exposure. J Pediatr Gastroenterol Nutr. 2019;69(suppl 2):S113.
Ulrich Baumann, Ekkehard Sturm, Florence Lacaille et al. Effects of the Ileal Bile Acid Transport Inhibitor A4250 on Pruritus and Serum Bile Acids in Patients With Biliary Atresia: Phase 2 Study Results. J Hepatol. 2019;70(1 suppl):e411.
Christoph Slavetinsky, Ekkehard Sturm. Impact of an Ileal Bile Acid Transporter Inhibitor Versus Partial External Biliary Diversion in Progressive Familial Intrahepatic Cholestasis – A Case Providing Direct Comparison of Medical and Surgical Therapies. J Pediatr Gastroenterol Nutr. 2019;68(suppl 1):892–3.
Ulrich Baumann, Ekkehard Sturm, Florence Lacaille et al. Effects of the Ileal Bile Acid Transport Inhibitor A4250 on Serum Bile Acids, Pruritus, and Sleep in Patients With Alagille Syndrome: Phase 2 Study Results. J Hepatol. 2019;70(1 suppl):e120. Z
Ulrich Baumann, Ekkehard Sturm, Florence Lacaille, Emmanuel Gonzalès, Henrik Arnell, Björn Fischler, Marianne Hørby Jørgensen, Richard J. Thompson, Jan P. Mattsson, Per-Göran Gillberg, Kristina Torfgård. Effects of the Ileal Bile Acid Transport Inhibitor A4250 on Pruritus, and Serum Bile Acids in Patients With Biliary Atresia: Phase 2 Study Results. J Pediatr Gastroenterol Nutr. 2019;68(suppl 1):767–8.
Ekkehard Sturm, Ulrich Baumann, Florence Lacaille, Emmanuel Gonzalès, Henrik Arnell, Björn Fischler, Marianne Hørby Jørgensen, Richard Thompson, Jan Mattsson, Mats Ekelund, Erik Lindström, Per-Göran Gillberg, Kristina Torfgård, Paresh Soni. The Ileal Bile Acid Transport Inhibitor A4250 Reduced Pruritus and Serum Bile Acid Levels in Children With Cholestatic Liver Disease and Pruritus: Final Results From a Multiple-dose, Open-label, Multinational Study. J Pediatr Gastroenterol Nutr. 2017;65(suppl 2):S168–9.
Emmanuel Gonzalès, Ekkehard Sturm, Ulrich Baumann et al. Correlation of Autotaxin Levels, Serum Bile Acids, and Pruritus in a Multiple-dose, Open label, Multinational Study of the Ileal Bile Acid Transport Inhibitor A4250. J Hepatol. 2018;68(suppl 1):S632.
Samer Al-Dury, Annika Wahlström, Staffan Wahlin et al. Pilot Study With IBAT Inhibitor A4250 for the Treatment of Cholestatic Pruritus in Primary Biliary Cholangitis. Sci Rep. 2018;8(1):6658.
Ekkehard Sturm, Ulrich Baumann, Florence Lacaille et al. The Ileal Bile Acid Transport Inhibitor A4250 Reduced Pruritus and Serum Bile Acid Levels in Children With Cholestatic Liver Disease and Pruritus: Final Results From a Multiple-dose, Open-label, Multinational Study. Hepatology. 2017;66(suppl 1):646A–7A.
Ulrich Baumann, Florence Lacaille, Ekkehard Sturm et al. The Ileal Bile Acid Transport Inhibitor A4250 Decreases Pruritus and Serum Bile Acids in Cholestatic Liver Diseases – An Ongoing Multiple-dose, Open-label, Multicentre Study. J Hepatol. 2017;66(1 suppl):S91.
Hans Graffner, Per-Göran Gillberg, Leif Rikner et al. The Ileal Bile Acid Transporter Inhibitor A4250 Decreases Serum Bile Acids by Interrupting the Enterohepatic Circulation. Aliment Pharmacol Ther. 2016;43(2):303–10.
Per-Göran Gillberg, Erik Lindström, Taishi Hashiguchi et al. Inhibition of the Ileal Bile Acid Transporter (IBAT) by A4250 Reduces Hepatic Damage in a Mouse Model of Non-alcoholic Steatohepatitis (NASH). Gastroenterology. 2016;150(4 suppl 1):S1091.
Anna Baghdasaryan, Claudia D. Fuchs, Christoph H. Österreicher et al. Inhibition of Intestinal Bile Acid Absorption Improves Cholestatic Liver and Bile Duct Injury in a Mouse Model of Sclerosing Cholangitis. J Hepatol. 2016;64(3):674–81.

Pediatric Cholestatic Liver Diseases

Claudia Mighiu, Sonia O'Hara, Enrico Ferri Grazzi et al. Impact of Progressive Familial Intrahepatic Cholestasis on Caregivers: Caregiver-Reported Outcomes From the Multinational PICTURE Study. Orphanet J Rare Dis. 2022;17(1):32.
Tracey Jones-Hughes, Jo Campbell, Louise Crathorne. Epidemiology and Burden of Progressive Familial Intrahepatic Cholestasis: A Systematic Review. Orphanet J Rare Dis. 2021;16(1):255.
Tracey Jones-Hughes, Joanna Campbell, Louise Crathorne et al. Epidemiology and Burden of Progressive Familial Intrahepatic Cholestasis: Systematic Review. J Hepatol. 2021;75(suppl 2):S685.
Velichka Valcheva, Tracey Jones-Hughes, Joanna Campbell et al. Epidemiology and Burden of Progressive Familial Intrahepatic Cholestasis: Systematic Review. Poster presented at the 56th Annual Meeting of the European Association for the Study of the Liver: The International Liver Congress. June 23–26, 2021; Virtual.
Henkjan J. Verkade, Richard J. Thompson, Henrik Arnell. Systematic Review and Meta-analysis: Partial External Biliary Diversion in Progressive Familial Intrahepatic Cholestasis. J Pediatr Gastroenterol Nutr. 2020;71(2):176–83.
Leonardo Ruiz, Claudia Mighiu, Alison Taylor et al. The PICTURE Burden of Illness Study: Quantifying the Socio-economic Burden of Progressive Familial Intrahepatic Cholestasis (PFIC) in the US, UK, France, and Germany. J Pediatr Gastroenterol Nutr. 2020;71(suppl 1):S100–1.
Saul J. Karpen, Deirdre Kelly, Cara Mack et al. Ileal Bile Acid Transporter Inhibition as an Anticholestatic Therapeutic Target in Biliary Atresia and Other Cholestatic Disorders. Hepatol Int. 2020;14(5):677–89.
Binita M. Kamath, Philip Stein, Roderick H.J. Houwen et al. Potential of Ileal Bile Acid Transporter Inhibition as a Therapeutic Target in Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis. Liver Int. 2020;40(8):1812–22.
Richard Thompson, Chad Gwaltney, Jean Paty et al. Development of Patient- and Observer-reported Outcome Measures for Paediatric Cholestatic Liver Diseases. J Pediatr Gastroenterol Nutr. 2019;69(suppl 2):S417–8.
Richard J. Thompson, Chad Gwaltney, Jean Paty et al. Development of Patient- and Observer-reported Outcome Measures for Paediatric Cholestatic Liver Diseases. J Pediatr Gastroenterol Nutr. 2019;68(suppl 1):905–6.
Richard J. Thompson, Chad Gwaltney, Jean Paty et al. Development of Patient- and Observer-reported Outcome Measures for Paediatric Cholestatic Liver Diseases. Poster presented at the 52nd Annual Meeting of the European Society for Pediatric Gastroenterology, Hepatology, and Nutrition. June 5–8, 2019; Glasgow, Scotland.
Kristina Torfgård, Chad Gwaltney, Jean Paty, Jan Mattsson, Paresh Soni. Symptoms and Daily Impacts Associated With Progressive Familial Intrahepatic Cholestasis and Other Pediatric Cholestatic Liver Diseases: A Qualitative Study With Patients and Caregivers. Pediatr Gastroenterol Nutr. 2018;67(suppl 1):S208–9.
Kristina Torfgård, Chad Gwaltney, Jean Paty, Jan P. Mattsson, Paresh N. Soni. Symptoms and Daily Impacts Associated With Progressive Familial Intrahepatic Cholestasis and Other Pediatric Cholestatic Liver Diseases: A Qualitative Study With Patients and Caregivers. J Pediatr Gastroenterol Nutr. 2018;66(suppl 2):813–4.
Henkjan J. Verkade, Richard Thompson, Henrik Arnell, Björn Fischler, Per-Göran Gillberg, Jan Mattsson, Kristina Torfgård, Erik Lindström, Paresh Soni. Systematic Literature Review of the Effect of Partial External Biliary Diversion Surgery on Clinical and Biochemical Outcomes in Progressive Familial Intrahepatic Cholestasis Patients. J Pediatr Gastroenterol Nutr. 2018;67(suppl 1):S209–10.
Henkjan Verkade, Richard J. Thompson, Henrik Arnell et al. Systematic Literature Review of the Effect of Partial External Biliary Diversion Surgery on Clinical and Biochemical Outcomes in Progressive Familial Intrahepatic Cholestasis Patients. J Pediatr Gastroenterol Nutr. 2018;66(suppl 2):818–9.

A2342

Britta Bonn, Ellen Strängberg, Ivana Uzelac et al. The Orally Available Sodium/Taurocholate Co-transporting Polypeptide Inhibitor A2342 Blocks Hepatitis B and D Entry In Vitro. J Hepatol. 2022;77(suppl 1):S843.
Erik Lindström, Per-Göran Gillberg, Ivana Uzelac, et al. Species-dependent Differences in Response to Sodium Taurocholate Cotransporting Polypeptide Inhibition: Translational Relevance to Human. Abstract accepted at the 26th Annual Meeting of the Falk Foundation: The International Bile Acid Meeting. July 8–9, 2022; Amsterdam, Netherlands.
Erik Lindström, Per-Göran Gillberg, Ivana Uzelac et al. Species-dependent Differences in Response to Sodium Taurocholate Cotransporting Polypeptide Inhibition: Translational Relevance to Human. Hepatology. 2021;74(suppl 1):519A–20A.
Erik Lindström, Per-Göran Gillberg, Ivana Uzelac et al. Preclinical Characterization of the Novel, Orally Bioavailable Hepatitis B Viral Entry Inhibitor A2342. Hepatology. 2021;74(suppl 1):512A–3A.

A3907

Peter Åkerblad, Erik Lindström, Jan Mattsson et al. A3907, a Novel Orally Available Inhibitor of the Apical Sodium-dependent Bile Acid Transporter, Improves Key Clinical Markers of Non-alcoholic Steatohepatitis in Obese Diet-induced and Biopsy-confirmed Mouse Models. J Hepatol. 2021;75(suppl 2):S597–8.
Peter Åkerblad, Erik Lindström, Jan Mattsson, et al. A3907, a Novel Orally Bioavailable Inhibitor of the Apical Sodium-dependent Bile Acid Transporter, Improves Liver Injury in a Mouse Model of Cholestatic Liver Disease. J Hepatol. 2021;75(suppl 2):S223–4.
Peter Åkerblad, Pål Lundin, Michael Feigh et al. A3907, a Novel Inhibitor of Bile Acid Transport in the Intestine and Kidney, Improves Markers of Metabolic and Hepatic Pathology, and Reduces Nonalcoholic Fatty Liver Disease Activity Score and Fibrosis Stage in a Diet-induced and Biopsy-confirmed Mouse Model of Nonalcoholic Steatohepatitis. Hepatology. 2020;72(suppl 1):S315–6.
Per-Göran Gillberg, Erik Lindström, Peter Åkerblad. Discovery and Characterization of Novel Bile Acid Transporter Inhibitors with Potential for Treatment of Liver Diseases. Hepatology. 2020;72(suppl 1):226A–7A.

Elobixibat

Mats Rudling, Michael Camilleri, Hans Graffner et al. Specific Inhibition of Bile Acid Transport Alters Plasma Lipids and GLP-1. BMC Cardiovasc Disord. 2015;15:75.
Banny S. Wong, Michael Camilleri. Elobixibat for the Treatment of Constipation. Expert Opin Investig Drugs. 2013;22(2):277–84.
Banny S. Wong, Michael Camilleri, Sanna McKinzie et al. Effects of A3309, an Ileal Bile Acid Transporter Inhibitor, on Colonic Transit and Symptoms in Females With Functional Constipation. Am J Gastroenterol. 2011;106(12):2154–64.
William D. Chey, Michael Camilleri, Lin Chang et al. A Randomized Placebo-controlled Phase IIb Trial of A3309, A Bile Acid Transporter Inhibitor, for Chronic Idiopathic Constipation. Am J Gastroenterol. 2011;106(10):1803–12.
Magnus Simrén, Antal Bajor, Per-Göran Gillberg. Randomised Clinical Trial: The Ileal Bile Acid Transporter Inhibitor A3309 vs. Placebo in Patients With Chronic Idiopathic Constipation – A Double-blind Study. Aliment Pharmacol Ther. 2011;34(1):41–50.
Per-Göran Gillberg, Mikael Dahlström, Ingemar Starke et al. The IBAT Inhibition by A3309 – A Potential Mechanism for the Treatment of Constipation. Gastroenterology. 2010;138(5 suppl 1):S224.

A3384

Richard N. Appleby, Antal Bajor, Per-Göran Gillberg et al. Effects of Conventional and a Novel Colonic-release Bile Acid Sequestrant, A3384, on Fibroblast Growth Factor 19 and Bile Acid Metabolism in Healthy Volunteers and Patients with Bile Acid Diarrhoea. United European Gastroenterol J. 2017;5(3):380–8.

Other Pipeline Products

Ellen Strängberg, Per-Göran Gillberg, Ivana Uzelac et al. 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. J Hepatol. 2022;77(suppl 1):S751–2.
Ellen Strängberg, Per-Göran Gillberg, Ivana Uzelac et al. Dual Ileal/Renal-Liver Bile Acid Transporter Inhibitors With Different Transporter Selectivity In Vitro Differentially Increase Faecal and Urinary Bile Acid Excretion in Oatp1a/1b Knockout Mice In Vivo. Abstract accepted at the 26th Annual Meeting of the Falk Foundation: The International Bile Acid Meeting. July 8–9, 2022; Amsterdam, Netherlands.