Rezdiffra

The approval was based on data from the phase 3 MAESTRO-NASH study (ClinicalTrials.gov Identifier: NCT03900429), which evaluated the efficacy and safety of resmetirom in patients with biopsy-proven NASH and fibrosis who were on stable doses of medications for diabetes, dyslipidemia, and hypertension.

Study participants were randomly assigned to receive resmetirom 80mg (n=298), resmetirom 100mg (n=296), or placebo (n=294) once daily, in addition to lifestyle counseling on nutrition and exercise. The dual primary surrogate endpoints were NASH resolution with at least a 2-point reduction in nonalcoholic fatty liver disease (NAFLD) activity score, and with no worsening of fibrosis or at least a 1-stage improvement in fibrosis with no worsening of the NAFLD activity score after 52 weeks of treatment. 

Findings showed a total of 26% to 27% of patients treated with resmetirom 80mg, and 24% to 36% of patients treated with resmetirom 100mg achieved NASH resolution and no worsening of liver fibrosis compared with 9% to 13% of those who received placebo. The range of responses were based on readings from 2 pathologists. 

Moreover, 23% of patients treated with resmetirom 80mg and 24% to 28% of patients treated with resmetirom 100mg achieved at least a 1-stage improvement in liver fibrosis with no worsening of steatohepatitis compared with 13% to 15% of the placebo group. Greater reductions were also observed in average alanine transaminase (ALT) and aspartate transaminase (AST) in the resmetirom groups starting at month 3 through month 12 vs the placebo group.