FDA backs proposal on surrogate endpoint for MASH trials

The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER), Office of New Drugs has accepted a Letter of Intent for the qualification of liver stiffness measurement by vibration-controlled transient elastography as a reasonably likely surrogate endpoint for clinical trials in adults with non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate-to-advanced liver fibrosis (scarring).

According to the Letter of Intent, the biomarker can predict risk of all-cause mortality or liver-related events in patients with MASH. MASH is a severe form of metabolic-associated fatty liver disease that develops when fat buildup in the liver causes inflammation and scarring. MASH is a progressive disease that can lead to cirrhosis (severe liver scarring), hepatic decompensation (worsening of liver function), liver cancer, liver transplantation, or death.

The proposed biomarker offers a non-invasive method for assessing liver stiffness; correlates with liver fibrosis severity; may predict clinical outcomes; and may provide a safer, more accessible approach for monitoring disease progression and treatment response.