As reported on Healio, new findings presented at The Liver Meeting highlight the potential of resmetirom (Rezdiffra, Madrigal Pharmaceuticals) in managing metabolic dysfunction-associated steatohepatitis (MASH) with compensated cirrhosis—a population with few therapeutic options.
Key Outcomes
- Long-Term Efficacy: Patients receiving resmetirom for up to two years demonstrated significant reductions in liver stiffness and improvements across multiple noninvasive biomarkers.
- Portal Hypertension Risk: The treatment was associated with a meaningful decrease in predicted clinically significant portal hypertension (CSPH) and a shift toward lower Agile scores.
- Impact of Treatment Interruption: A temporary discontinuation—averaging 77 days—led to reversal of gains in liver biomarkers and imaging measures. While resuming therapy restored most improvements, recovery was incomplete in patients with very low platelet counts.
Study Details
The analysis drew from the MAESTRO-NAFLD-1 trial and its open-label extension, involving 122 adults with Child-Pugh A MASH cirrhosis. Participants had a mean age of 61 years, average BMI of 35 kg/m², and a high prevalence of type 2 diabetes (70%). Nearly half were at risk for CSPH at baseline, with platelet count and spleen volume strongly influencing risk stratification.
Clinical Perspective
“Patients with MASH cirrhosis face the greatest unmet need for pharmacologic therapy to prevent hepatic decompensation, yet no FDA-approved options exist,” noted Naim Alkhouri, MD, FAASLD, chief academic officer at Summit Clinical Research. While resmetirom is not currently approved for cirrhosis, its benefits in advanced fibrosis suggest promise for this challenging group.
Next Steps
Encouraging results from this cohort are being evaluated in the ongoing phase 3 MAESTRO-NASH-OUTCOMES trial. If confirmed, resmetirom could represent a breakthrough in reducing complications and improving outcomes for patients with MASH cirrhosis.
