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Resmetirom: Duke Research Leads to First FDA-Approved Drug for MASH Liver Disease

• Resmetirom, a novel drug developed with Duke University research, has gained FDA approval as the first treatment for metabolic dysfunction-associated steatohepatitis (MASH). • The drug targets thyroid hormone pathways to stimulate fat breakdown in the liver, addressing a critical need for the estimated 115 million people worldwide affected by MASH. • While only 30% of trial participants benefited after one year and the treatment costs $50,000 annually, resmetirom marks a significant advancement, with hopes for expanded treatment options and reduced costs through further research. • Research indicates that interest in MASH is growing, with resmetirom and similar drugs potentially working alongside other therapies to combat the disease and alleviate the health burden associated with advanced liver and cardiovascular complications.

Duke University professor Paul Yen's research has culminated in the FDA approval of resmetirom, the first drug to treat metabolic dysfunction-associated steatohepatitis (MASH), a severe form of nonalcoholic liver disease. This condition, affecting an estimated 115 million people globally, is characterized by damaging fat buildup in the liver and is often linked to obesity.
Yen's work, initiated at Duke-NUS Medical School in 2009, focused on metabolism research and thyroid hormone activity. His team's microarray studies revealed significant changes in a thyroid hormone gene in patients with fatty liver disease. Further research demonstrated that thyroid hormones stimulate autophagy, a crucial cellular process for fat breakdown in the liver. Collaboration with Christopher Newgard highlighted that these hormones were defective in patients with nonalcoholic liver disease.

Thyroid Hormone Mimetic

Resmetirom, developed by Madrigal Pharmaceuticals, is an oral drug that mimics thyroid hormone action, targeting the liver directly. Yen emphasized the potential health economic benefits of early intervention, stating, "If we can attack the disease earlier, maybe we can prevent people getting more severe disease, and then that will actually alleviate some of the health burdens."

Clinical Trial Outcomes and Future Directions

While Yen noted that only 30% of trial participants experienced benefits after one year of resmetirom treatment, and the annual cost is approximately $50,000, he remains optimistic. He anticipates that ongoing research will lead to a broader range of treatments, reduced costs, and more options for patients. Yen also observed a growing interest in MASH within the endocrinology field, signaling a shift towards greater research and therapeutic development in this area.

The Growing Interest in MASH

"I would say, five years ago, if we went to the Endocrine Society, we might be one of two people that even had anything related to MASH," Yen said. "At the last meeting I went to, there were five symposia dedicated to this topic."
Yen envisions resmetirom and similar drugs working in conjunction with other pharmacological therapies. He acknowledged the need for further research to assess the drug's benefits at various disease stages but expressed satisfaction in contributing to a significant advancement in the fight against MASH.
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Reference News

[1]
Duke professor's research leads to first drug for type of liver disease - The Chronicle
dukechronicle.com · Sep 6, 2024

The FDA approved resmetirom, a drug for metabolic dysfunction-associated steatohepatitis (MASH), developed by Duke profe...

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