Drug design, development and therapy

Resmetirom as a treatment update for fatty liver disease linked to metabolism problems

Updated

Abstract

Metabolic dysfunction associated steatotic liver disease (MASLD) affects nearly one-third of adults worldwide.

  • Resmetirom is the first therapy to show both resolution of metabolic dysfunction associated steatohepatitis () and improvement in fibrosis.
  • This liver-specific drug mimics the action of thyroid hormone T3, leading to reduced fat within the liver.
  • Treatment is generally safe and well tolerated, with current guidelines recommending it for patients with non-cirrhotic fibrotic MASH.
  • Rapid adoption of non-invasive assessments for treatment eligibility has been observed in real-world data.
  • Early improvements in liver stiffness have been noted, with treatment response appearing independent of weight-loss therapy.

Simplified

Key numbers

29.9%
Resolution Rate
Observed in patients receiving 100 mg of resmetirom after 52 weeks.
−37.3%
Hepatic Fat Reduction
Compared to −8.5% in the placebo group after 36 weeks.
March 2024
Approval Timeline
Resmetirom received accelerated approval from the .

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Funding

Competing interests

LvK Travel expenses from EASL and Falk Foundation. MM declares consultant honorary from Boehringer Ingelheim, Ipsen, Madrigal Pharmaceuticals; Speaker honorarium from AbbVie, AstraZeneca, Daiichi Sankyo, Gilead Sciences, GSK, Ipsen and NovoNordisk; Travel support: Abbvie, Boehringer Ingelheim, Gilead Sciences, Ipsen, Mirum and NovoNordisk. NA has received grant/research support from 89bio, Akero Therapeutics, Arbutus Biopharma, AstraZeneca, BioAge, Boehringer Ingelheim, Bristol Myers Squibb, Corcept Therapeutics, Galectin Therapeutics, Genentech, Gilead Sciences, Hepagene Therapeutics, Intercept Pharmaceuticals, Inventiva Pharma, Ionis Pharmaceuticals, Ipsen, Lilly, Madrigal Pharmaceuticals, Merck, NGM Biopharmaceuticals, NorthSea Therapeutics, Novo Nordisk, Perspectum, Pfizer, PharmaIN, Poxel, Regeneron, Viking Therapeutics, and Zydus Pharmaceuticals; reports speaker’s fees from AbbVie, AstraZeneca, Echosens, Gilead Sciences, Ipsen, Madrigal Pharmaceuticals, Novo Nordisk, and Perspectum; and reports consulting for 89bio, AbbVie, Akero, Boehringer Ingelheim, Echosens, Fibronostics, Gilead Sciences, HistoIndex, Intercept Pharmaceuticals, Ipsen, LiverRight, Madrigal Pharmaceuticals, NorthSea Therapeutics, Novo Nordisk, Perspectum, Pfizer, Regeneron, and Sonicy Incytes. JMS declares consultant honorary from Akero, Alentis, Alexion, Altimmune, Astra Zeneca, 89Bio, Bionorica, Boehringer Ingelheim, Gilead Sciences, GSK, HistoIndex, Ipsen, Inventiva Pharma, Madrigal Pharmaceuticals, Kríya Therapeutics, Lilly, eTherapeutics, Merck, MSD Sharp & Dohme GmbH, Nordic Bioscience, Northsea Therapeutics, Novartis, Novo Nordisk, Pfizer, Roche, Sanofi, Siemens Healthineers, Summit Clinical, and Vantage Biosciences Research; speaker honorarium from AbbVie, Boehringer Ingelheim, Gilead Sciences, Ipsen Novo Nordisk, Madrigal Pharmaceuticals, Worldwide Clinical Trials, Stockholder options: Hepta Bio. WPB received speakers fees for Eli Lilly, is part of the advisory board of Novo Nordisk and participates in trials of 89BIO, Boehringer Ingelheim, Novo Nordisk, and Inventiva Pharma. The authors report no other conflicts of interest in this work.
PubMed

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