Home Medizin FDA genehmigt erstes Medikament für MASH

FDA genehmigt erstes Medikament für MASH

von NFI Redaktion

The US Food and Drug Administration (FDA) has approved Resmetirom (Rezdiffra, Madrigal Pharmaceuticals) as the first medication for the treatment of patients with metabolic dysfunction-associated steatohepatitis (MASH) and moderate to advanced liver fibrosis (consistent with disease stages F2 and F3), in conjunction with diet and exercise.

Resmetirom is a once-daily orally administered beta-selective agonist of the thyroid hormone receptor. The FDA granted the medication breakthrough therapy designation and priority review.

The approval is based on the Phase 3 MAESTRO-NASH study, where Resmetirom outperformed placebo in both 80mg and 100mg doses in healing non-alcoholic steatohepatitis (NASH) and improving liver fibrosis.

The study used the previous nomenclature of NASH and non-alcoholic fatty liver disease (NAFLD), which has now been changed to MASH and metabolic dysfunction-associated steatotic liver disease (MASLD) by an international consensus group.

The results were published online on February 7 in The New England Journal of Medicine.

„The approval of the first medication for NASH is a true milestone for healthcare providers, the research community, and most importantly, for patients living with this serious liver disease,“ said lead MAESTRO-NASH researcher Stephen Harrison, MD, Gastroenterologist and Hepatologist, and Chairman of Pinnacle Clinical Research and Summit Clinical Research, San Antonio, Texas, in a press release.

„Based on the robust efficacy and safety data identified in two large Phase 3 MAESTRO studies, I believe Rezdiffra will become the foundational therapy for patients with NASH with moderate to advanced liver fibrosis. It is crucial that we continue to investigate Resmetirom to confirm positive results,“ Harrison added.

Addressing an Unmet Need

MASH is a progressive liver disease and the leading cause of liver-related mortality. The disease affects an estimated 1.5 million adults in the United States, with approximately 525,000 suffering from significant fibrosis due to MASH. Until now, there has been no FDA-approved medication.

In the ongoing MAESTRO-NASH study, 996 adults with biopsy-confirmed NASH and significant fibrosis in stages 2–3 were randomly assigned to once-daily oral administration of Resmetirom (80mg or 100mg) or placebo.

The patients were observed for 52 weeks and evaluated for the two primary endpoints of NASH resolution (including a reduction of NAFLD activity score by ≥ 2 points) without worsening of fibrosis, and improvement (reduction) of fibrosis by at least one stage without worsening of NAFLD activity score.

Patients receiving Resmetirom showed significant improvement at both doses and both primary endpoints, as reported by Medscape Medical News.

After 52 weeks, 25.9% and 29.9% of patients in the 80mg and 100mg groups, respectively, achieved NASH regression without worsening fibrosis, compared to 9.7% under placebo.

For 24.2% and 25.9% of patients in the 80mg and 100mg groups, respectively, fibrosis improved by at least one stage without worsening NAFLD activity score, compared to 14.2% under placebo.

The study also achieved several secondary endpoints, including a statistically significant reduction in liver enzymes (alanine aminotransferase, aspartate aminotransferase, and gamma-glutamyl transferase) and low-density lipoprotein cholesterol compared to baseline with Resmetirom versus placebo.

Improvements in fibrosis biomarkers and relevant imaging tests were also observed in the Resmetirom treatment groups compared to placebo.

The most common adverse events included diarrhea and nausea, typically occurring at the beginning of treatment with mild to moderate severity. It was also reported itching, abdominal pain, vomiting, constipation, and dizziness.

Resmetirom is expected to be available for patients in the United States in April and distributed through a limited network of specialty pharmacies.

Complete prescribing information is available online. Liver biopsy is not required for diagnosis in the prescribing information.

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