On March 14, the U.S. Food and Drug Administration (FDA) approved resmetirom (Rezdiffra) for adult patients with noncirrhotic nonalcoholic steatohepatitis with moderate-to-advanced fibrosis, representing the first approved treatment option for liver scarring in this patient population. The drug—a thyroid hormone receptor beta agonist—was approved following positive results from multiple phase II and III trials, including MAESTRO-NASH (ClinicalTrials.gov identifier NCT03900429), a phase III study that analyzed the safety and efficacy of resmetirom in patients with noncirrhotic nonalcoholic steatohepatitis with moderate-to-advanced fibrosis. Patients were randomly assigned to receive either 80 mg (n = 298) or 100 mg of resmetirom (n = 296) or placebo (n = 294) once daily in addition to a diet and exercise regimen. The surrogate endpoint of the trial was the extent of liver inflammation and scarring. After a follow-up of 12 months, researchers discovered higher rates of NASH resolution and liver scarring improvement among the patients who received resmetirom at 80 mg (26% to 27% and 23%, respectively) and 100 mg (24% to 36% and 24% to 28%) compared with those who received placebo (9% to 13% and 13% to 15%). The researchers noted that the patients who received resmetirom experienced diarrhea and nausea; however, the drug is also known to cause drug-induced liver toxicity and gallbladder-related side effects and is contraindicated for those with decompensated cirrhosis. Resmetirom was approved under the accelerated approval pathway and received Breakthrough Therapy, Fast Track, and Priority Review designations. The researchers will continue to examine the clinical benefit of resmetirom through the trial’s endpoint of 54 months.
FDA Approves Resmetirom for Patients With Noncirrhotic Nonalcoholic Steatohepatitis and Moderate-to-Advanced Fibrosis
Conexiant
May 1, 2024