Resmetirom (brand name Rezdiffra) is the first FDA-approved medication for treating metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as nonalcoholic fatty liver disease (NAFLD), in adults with moderate to advanced liver fibrosis. This drug offers new hope for individuals with MASLD who have progressed to moderate stages of fibrosis known as F2 or F3.

 

Mechanism of Action:

Resmetirom is a liver-directed, selective thyroid hormone receptor-beta (THR-β) agonist. It works by:

• Stimulating fat breakdown in the liver

• Reducing lipotoxicity

• Reducing liver cell damage

• Decreasing liver inflammation and fibrosis

 

Key Clinical Findings:

• The MAESTRO-NASH phase 3 trial demonstrated significant efficacy

• NASH resolution with no worsening of fibrosis

• Fibrosis improvement

• Reduction in liver fat content

 

Approved Use:

Resmetirom is indicated for adults with MASH and moderate to advanced liver fibrosis (F2-F3), in conjunction with lifestyle changes.

 

Ongoing Research for Pediatric Use:

While currently approved for adults 18 and older, research is ongoing to evaluate resmetirom's safety and efficacy in children and adolescents. Some notable pediatric studies on ClinicalTrials.gov include:

 

MAESTRO-NASH-PEDS (NCT05722418): A phase 3, randomized, double-blind, placebo-controlled study in children and adolescents aged 8-17 years with biopsy-confirmed NASH and liver fibrosis.

 

MAESTRO-NAFLD-OBE (NCT04929210): A phase 2 study evaluating resmetirom in adolescents aged 12-17 years with presumed NAFLD/NASH and obesity.

These studies aim to assess the drug's impact on liver fat reduction, NASH resolution, and fibrosis improvement in pediatric populations.

 

Importance of Lifestyle Changes:

While resmetirom offers a promising treatment option, it's important to remember that lifestyle modifications remain the cornerstone of MASLD management. A healthy diet and regular exercise are essential components of any treatment plan, regardless of medication use.

 

For the latest information on ongoing clinical trials and potential future indications, including pediatric use, regularly check ClinicalTrials.gov and consult with healthcare professionals specializing in liver diseases.