[8/27/2025] FDA’s Center for Drug Evaluation and Research, 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.
“This proposal represents an important step toward adopting non-invasive tests in drug development for MASH,” said Frank Anania, MD, director of the Division of Hepatology and Nutrition in CDER. “Such non-invasive studies could replace liver histology, which has been the primary way to assess liver injury and repair. However, obtaining histology requires liver biopsy, which is an invasive procedure.”
Evaluating new therapies for MASH without requiring repeated liver biopsies could help with trial recruitment, enable representative patient populations, help overcome difficulties in conducting adequately powered trials, and accelerate drug development for patients with this serious liver condition.
This acceptance represents the first step in the Drug Development Tool qualification process, where the company will work with FDA to develop a comprehensive qualification plan for data and validation requirements to support the specific context of use.
For more information:
Qualification Process for Drug Development Tools Guidance for Industry and FDA Staff
CDER & CBER Drug Development Tool Qualification Project Search
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Content current as of:
08/27/2025
