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Drug Evaluation

Odevixibat: an investigational inhibitor of the ileal bile acid transporter (IBAT) for the treatment of biliary atresia

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Pages 1143-1150 | Received 21 Apr 2022, Accepted 22 Nov 2022, Published online: 14 Dec 2022

References

  • Hartley JL, Davenport M, Kelly DA. Biliary atresia. Lancet. 2009;374:1704–1713.
  • Hopkins PC, Yazigi N, Nylund CM. Incidence of Biliary Atresia and Timing of Hepatoportoenterostomy in the United States. J Pediatr. 2017;187:253–257.
  • Chardot C, Buet C, Serinet MO, et al. Improving outcomes of biliary atresia: French national series 1986-2009. J Hepatol. 2013;58:1209–1217.
  • Lin JS, Chen SC, Lu CL, et al. Reduction of the ages at diagnosis and operation of biliary atresia in Taiwan: a 15-year population-based cohort study. World J Gastroenterol. 2015;21:13080–13086.
  • Wildhaber BE. Biliary atresia: 50 years after the first Kasai. ISRN Surg. 2012;2012:132089.
  • Kasai M, Suzuki S. A new operation for, ‘non-correctable’ biliary atresia: hepatic porto-enterostomy. Shuiyutsu. 1959;13:733–739.
  • Lykavieris P, Chardot C, Sokhn M, et al. Outcome in adulthood of biliary atresia: a study of 63 patients who survived for over 20 years with their native liver. Hepatology. 2005;41:366–371.
  • Adam R, Karam V, Cailliez V, et al. Annual Report of the European liver transplant registry (ELTR) – 50-year evolution of liver transplantation. Transpl Int. 2018;31:1293–1317.
  • de Ville de Goyet J, Baumann U, Karam V, et al. European liver transplant registry: donor and transplant surgery aspects of 16,641 liver transplantations in children. Hepatology. 2022;75:634–645.
  • Deo AK, Bandiera SM. Identification of human hepatic cytochrome p450 enzymes involved in the biotransformation of cholic and chenodeoxycholic acid. Drug Metab Dispos. 2008;36:1983–1991.
  • Hofmann AF, Hagey LR. Key discoveries in bile acid chemistry and biology and their clinical applications: history of the last eight decades. J Lipid Res. 2014;55:1553–1595.
  • Dawson PA, Lan T, Rao A. Bile acid transporters. J Lipid Res. 2009;50:2340–2357.
  • Otsuki M. Pathophysiological role of cholecystokinin in humans. J Gastroenterol Hepatol. 2000;15:71–83.
  • Chiang JY. Recent advances in understanding bile acid homeostasis. F1000 Res. 2017;6:2029.
  • Li T, Apte U. Bile acid metabolism and signaling in cholestasis, inflammation, and Cancer. Adv Pharmacol. 2015;74:263–302.
  • Gertzen CGW, Gohlke H, Häussinger D, et al. The many facets of bile acids in the physiology and pathophysiology of the human liver. Biol Chem. 2021;402:1047–1062.
  • Dawson PA, Karpen SJ. Intestinal transport and metabolism of bile acids. J Lipid Res. 2015;56:1085–1099.
  • Ørntoft NW, Munk OL, Frisch K, et al. Hepatobiliary transport kinetics of the conjugated bile acid tracer 11C-CSar quantified in healthy humans and patients by positron emission tomography. J Hepatol. 2017;67:321–327.
  • Neimark E, Chen F, Li X, et al. Bile acid–induced negative feedback regulation of the human ileal bile acid transporter. Hepatology. 2004;40:149–156.
  • Allen K, Jaeschke H, Copple BL. Bile acids induce inflammatory genes in hepatocytes: a novel mechanism of inflammation during obstructive cholestasis. Am J Pathol. 2011;178:175–186.
  • Jansen PLM, Ghallab A, Vartak N, et al. The ascending pathophysiology of cholestatic liver disease. Hepatology. 2017;65:722–738.
  • Muchova L, Vanova K, Zelenka J, et al. Bile acids decrease intracellular bilirubin levels in the cholestatic liver: implications for bile acid-mediated oxidative stress. J Cell Mol Med. 2011;15:1156–1165.
  • Vacca M, Degirolamo C, Massafra V, et al. Nuclear receptors in regenerating liver and hepatocellular carcinoma. Mol Cell Endocrinol. 2013;368:108–119.
  • Harpavat S, Hawthorne K, Shneider BL. Prognostic value of serum bile acids after achieving bile flow with the Kasai portoenterostomy in biliary atresia (abstract 339). Hepatology. 2020;72:1A–130A.
  • Wang KS, Tiao G, Bass LM, et al. Analysis of surgical interruption of the enterohepatic circulation as a treatment for pediatric cholestasis. Hepatology. 2017;65:1645–1654.
  • Schukfeh N, Metzelder ML, Petersen C, et al. Normalization of serum bile acids after partial external biliary diversion indicates an excellent long-term outcome in children with progressive familial intrahepatic cholestasis. J Pediatr Surg. 2012;47:501–505.
  • Verkade HJ, Thompson RJ, Arnell H, et al. Systematic review and meta-analysis: partial external biliary diversion in progressive familial intrahepatic cholestasis. J Pediatr Gastroenterol Nutr. 2020;71:176–183.
  • Loomes KM, Squires RH, Kelly D, et al. Maralixibat for the treatment of PFIC: long-term, IBAT inhibition in an open-label, phase 2 study. Hepatol Commun. 2022;6(9):2379–2390. 09/01; 2022/09
  • Khanna L, Camilleri M. Review article: elobixibat: a novel treatment for chronic constipation. Aliment Pharmacol Ther. 2021 ;53(2):234–242. 01/01; 2022/09
  • Hegade VS, Kendrick SFW, Dobbins RL, et al. Effect of ileal bile acid transporter inhibitor GSK2330672 on pruritus in primary biliary cholangitis: a double-blind, randomised, placebo-controlled, crossover, phase 2a study. Lancet. 2017;389(10074):1114–1123. 18–24 March 2017
  • Newsome PN, Palmer M, Freilich B, et al. Volixibat in adults with non-alcoholic steatohepatitis: 24-week interim analysis from a randomized, phase II study. J Hepatol. 2020 August;73(2):231.
  • Center for drug evaluation and research: integrated review of odevixibat, [Internet]. cited 2022 Jul 08, from https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/215498Orig1s000IntegratedR.pdf
  • Baghdasaryan A, Fuchs CD, Österreicher CH, 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;64:674–681.
  • Baumann U, Sturm E, Lacaille F, et al. Effects of odevixibat on pruritus and bile acids in children with cholestatic liver disease: phase 2 study. Clin Res Hepatol Gastroenterol. 2021;45:101751.
  • Baumann U, Sturm E, Lacaille F, et al. FRI-060-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:e411.
  • Thompson RJ, Horn P, Houwen RHJ, 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 (Poster: PO_1665) 2021. cited 2022 Jul 08 from https://www.postersessiononline.eu/173580348_eu/congresos/ILC2021/aula/-PO_1665_ILC2021.pdf
  • Gonzales E, Hardikar W, Stormon M, et al. Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study. Lancet. 2021;398:1581–1592.
  • Shneider BL, Spino C, Kamath BM, et al. Placebo-controlled randomized trial of an intestinal bile salt transport inhibitor for pruritus in alagille syndrome. Hepatology. 2018;2:1184–1198.
  • Chen HL, Wu SH, Hsu SH, et al. Jaundice revisited: recent advances in the diagnosis and treatment of inherited cholestatic liver diseases. J Biomed Sci. 2018;25: 75–018.
  • Hegyi P, Maléth J, Walters JR, et al. Guts and gall: bile acids in regulation of intestinal epithelial function in health and disease. Physiol Rev. 2018;98:1983–2023.

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