Resmetirom: The First FDA-Approved Drug for Metabolic Dysfunction-Associated Steatohepatitis (MASH) with a

Fengshuo Zhang1, Yicong Meng1, Bohan Zhao1

  • 1Department of Pathogen Biology, School of Basic Medical Sciences, Henan University, Kaifeng, China.

Clinical Drug Investigation
|September 23, 2025
PubMed

Insights

Resmetirom, a new THR-β agonist, is the first FDA-approved drug for metabolic dysfunction-associated steatohepatitis (MASH). It effectively reduces liver fat and improves MASH markers, though challenges like side effects persist.

Area of Science:

  • Hepatology
  • Pharmacology
  • Endocrinology

Background:

  • Metabolic dysfunction-associated steatohepatitis (MASH) is a severe liver condition potentially leading to cirrhosis.
  • Current treatment options for MASH are limited, necessitating novel therapeutic approaches.

Purpose of the Study:

  • To review the discovery, mechanisms, clinical evaluation, and challenges of resmetirom for MASH treatment.
  • To discuss the role of resmetirom as a targeted therapy for MASH.

Main Methods:

  • Review of clinical trial data and scientific literature on resmetirom.
  • Analysis of resmetirom's mechanism of action as a thyroid hormone receptor-β (THR-β) agonist.

Main Results:

  • Resmetirom demonstrates significant improvements in MASH markers and liver fat content.
  • High selectivity for THR-β, predominantly expressed in the liver, is a key feature.
  • The drug is the first FDA-approved targeted therapy for MASH.

Conclusions:

  • Resmetirom represents a breakthrough in MASH treatment, offering a targeted therapeutic option.
  • Challenges including adverse reactions and variable efficacy require further investigation for optimal MASH precision therapy.

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