The first MASH drug therapy on the horizon: Current perspectives of resmetirom

• DOI: 10.1111/liv.15930
• Publication Date: 2024-04-05T12:58:43Z
• Authors: Salvatore Petta; Giovanni Targher; Stefano Romeo; Utpal B. Pajvani; Ming‐Hua Zheng; Alessio Aghemo; Luca V. C. Valenti
• Keywords: Steatohepatitis; Liver disease; Pharmacotherapy

Abstract The rising prevalence of metabolic dysfunction‐associated steatotic liver disease (MASLD) poses a significant global health challenge, affecting over 30% adults worldwide. MASLD is linked to increased mortality rates and substantial healthcare costs, primarily driven by its progression steatohepatitis (MASH), which can lead severe complications including cirrhosis hepatocellular carcinoma. Despite growing burden, effective pharmacotherapy for MASLD/MASH has been lacking until the recent conditional approval resmetirom FDA. Resmetirom, liver‐targeted thyroid hormone receptor‐β selective drug, shown promise in clinical trials treating non‐cirrhotic MASH with moderate advanced fibrosis. It demonstrated efficacy reducing hepatic fat content, improving histology (both resolution fibrosis improvement), ameliorating biomarkers damage without effects on body weight or glucose metabolism. Notably, also exhibits favourable circulating lipids, potentially cardiovascular risk patients. safety profile appears acceptable, gastrointestinal adverse events being most common, though generally mild moderate. However, long‐term surveillance warranted monitor potential risks related thyroid, gonadal, bone diseases. Clinical implementation faces challenges patient selection monitoring treatment response, will heavily rely non‐invasive tests assessment. Nonetheless, represents landmark breakthrough treatment, paving way future therapeutic strategies aiming mitigate multifaceted associated this complex disease.