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Review

Resmetirom

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In: LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012.
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Review

Resmetirom

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Excerpt

Resmetirom is a thyroid hormone receptor beta (THR-β) agonist used in conjunction with diet and exercise in the therapy of nonalcoholic steatohepatitis (NASH) with moderate-to-severe fibrosis. Resmetirom therapy is associated with mild and transient serum aminotransferase elevations during the first month of therapy and with rare instances of acute liver injury which can be severe, but which reverses on drug discontinuation.

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References

    1. FDA. Resmetirom, Integrated Review. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2024/217785Orig1s000I...(FDA Summary of the data on resmetirom safety and efficacy submitted in support of the application for approval as therapy of nonalcoholic steatohepatitis (NASH) mentions that one probable and one possible case of drug induced liver disease arose among the 1333 patients included in two prospective trials used in the safety assessment; the probable case was in a 60 year old woman who developed liver test abnormalities 57 days after starting resmetirom [ALT 236 U/L, Alk P not done, bilirubin 0.4 mg/dL], which were slow to improve after stopping but rose from normal to high levels within a month of restarting resmetirom [ALT 3226 U/L, Alk P 140 U/L, bilirubin 10.9 mg/dL], which again fell to normal within 2 months of stopping without corticosteroid therapy).
    1. Kelly MJ, Pietranico-Cole S, Larigan JD, Haynes NE, Reynolds CH, Scott N, Vermeulen J, et al. Discovery of 2-[3,5-dichloro-4-(5-isopropyl-6-oxo-1,6-dihydropyridazin-3-yloxy)phenyl]-3,5-dioxo-2,3,4,5-tetrahydro[1,2,4]triazine-6-carbonitrile (MGL-3196), a highly selective thyroid hormone receptor β agonist in clinical trials for the treatment of dyslipidemia. J Med Chem. 2014; 57: 3912-23.(Description of development of thyroid hormone [TH] mimetic chemical structures that had selective affinity for the β receptors [THR-β] which are found predominantly on hepatocytes, the optimal molecule [MGL-3196: resmetirom] showed 84% of the relative activity of T3 against the β receptor but only 49% of that against the α receptor, and in animal models it had cholesterol and triglyceride lowering activity with no cardiac toxicity or effect on the central thyroid axis). - PubMed
    1. Harrison SA, Bashir MR, Guy CD, Zhou R, Moylan CA, Frias JP, Alkhouri N, et al. Resmetirom (MGL-3196) for the treatment of non-alcoholic steatohepatitis: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet. 2019; 394: 2012-2024.(Among 125 adults with biopsy confirmed non-cirrhotic NASH treated with resmetirom [60-80 mg daily] or placebo once daily for 36 weeks, MRI estimated liver fat fraction decreased more with resmetirom as did liver biopsy fat, inflammation and ballooning, and serum ALT and AST levels, while adverse events were similar in both groups except for symptoms of diarrhea and nausea; no mention of hepatotoxicity or liver injury with jaundice). - PubMed
    1. Harrison SA, Bashir M, Moussa SE, McCarty K, Pablo Frias J, Taub R, Alkhouri N. Effects of resmetirom on noninvasive endpoints in a 36-week phase 2 active treatment extension study in patients with NASH. Hepatol Commun. 2021; 5: 573-588.(Among 125 patients with NASH participating in a 36-week randomized placebo-controlled trial, 31 were enrolled in an open-label extension study for another 36 weeks which demonstrated a further decline in the liver fat fraction as well as improvements in serum aminotransferase levels and lipids, with no weight loss and no treatment related severe adverse events). - PMC - PubMed
    1. Karim G, Bansal MB. Resmetirom: an orally administered, small molecule, liver-directed, β-selective THR agonist for the treatment of non-alcoholic fatty liver disease and non-alcoholic steatohepatitis. touchREV Endocrinol. 2023; 19: 60-70.(Review of the mechanism of action and clinical effects of resmetirom, a relatively selective thyroid hormone receptor beta agonist, which decreases hepatic fat and lipotoxicity, and holds promise as therapy of NASH). - PMC - PubMed

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