ANONYMOUS: "Study Record Versions History of Changes for Study: NCT04168385 MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study (MERGE)", CLINICALTRIALS.GOV, 22 January 2020 (2020-01-22), pages 1 - 9, XP055977617, Retrieved from the Internet <URL:https://clinicaltrials.gov/ct2/history/NCT04168385?V_2=View#StudyPageTop> [retrieved on 20221103] (B1)

ANONYMOUS: "Study Record Versions History of Changes for Study: NCT04185363 An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)", CLINICAL TRIALS.GOV, 6 December 2019 (2019-12-06), clinical trials, pages 1 - 9, XP055977607, Retrieved from the Internet <URL:https://clinicaltrials.gov/ct2/history/NCT04185363?V_2=View#StudyPageTop> [retrieved on 20221103] (B1)

CLINICAL TRIALS.GOV: "Study Record Versions Contacts/Locations and Study Status History of Changes for Study: NCT03566238 This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC 1 or 2 (PEDFIC 1)", CLINICAL TRIALS .GOV, 8 February 2019 (2019-02-08), pages 1 - 16, XP055975866, Retrieved from the Internet <URL:https://clinicaltrials.gov/ct2/history/NCT03566238?V_18=View#StudyPageTop> [retrieved on 20221028] (B1)

CLINICAL TRIALS.GOV: "Study Record Versions History of Changes for Study: NCT03659916 Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC Types 1 & 2", CLINICAL TRIALS.GOV, 11 February 2019 (2019-02-11), pages 1 - 11, XP055975930, Retrieved from the Internet <URL:https://clinicaltrials.gov/ct2/history/NCT03659916?V_5=View#StudyPageTop> [retrieved on 20221028] (B1)

MALATACK JEFFREY ET AL: "Case RepoRt A Drug Regimen for Progressive Familial Cholestasis Type 2", PEDIATRICS, 1 January 2018 (2018-01-01), pages 1 - 5, XP055975631, Retrieved from the Internet <URL:https://publications.aap.org/pediatrics/article/141/1/e20163877/37702/A-Drug-Regimen-for-Progressive-Familial> [retrieved on 20221027], DOI: 10.1542/peds.2016-3877 (B1)

WO-A1-2011/137135 (B1)

MIRUM PHARMACEUTICALS: "Mirum Pharmaceuticals Presents New Data Demonstrating Durable Improvements in Clinical Outcome Measures in Patients with PFIC2 and Alagille Syndrome Treated with Maralixibat", 15 April 2019 (2019-04-15), XP055892012, Retrieved from the Internet <URL:https://www.prnewswire.com/news-releases/mirum-pharmaceuticals-presents-new-data-demonstrating-durable-improvements-in-clinical-outcome-measures-in-patients-with-pfic2-and-alagille-syndrome-treated-with-maralixibat-300831721.html> [retrieved on 20220215] (B1)

US-A1- 2013 108 573 (B1)

US-A1- 2016 193 277 (B1)

US-A1- 2017 210 717 (B1)

US-A1- 2017 368 085 (B1)

MELISSA PALMER ET AL: "A randomised, double-blind, placebo-controlled phase 1 study of the safety, tolerability and pharmacodynamics of volixibat in overweight and obese but otherwise healthy adults: implications for treatment of non-alcoholic steatohepatitis", BMC PHARMACOLOGY AND TOXICOLOGY, BIOMED CENTRAL LTD, LONDON, UK, vol. 19, no. 1, 16 March 2018 (2018-03-16), pages 1 - 13, XP021254505, DOI: 10.1186/S40360-018-0200-Y (B1)