The Hepatoscope® from E-Scopics
Big news here from 08/27/2025: “FDA accepts proposal for reasonably likely surrogate endpoint for ‘MASH’ all-cause mortality or liver-related events.”
The key paragraph:
“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.”
The FDA Announcement
1. What Did the FDA Accept?
The FDA’s Center for Drug Evaluation and Research (CDER), Office of New Drugs, has accepted a Letter of Intent for the qualification of a biomarker as a “reasonably likely surrogate endpoint” specifically for clinical trials of non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) in adults with moderate-to-advanced liver fibrosis.
2. What’s the Biomarker?
The biomarker proposed is Liver Stiffness Measurement by Vibration-Controlled Transient Elastography (VCTE), a non-invasive test that assesses liver stiffness and correlates with fibrosis severity.
3. What Does It Predict?
According to the Letter of Intent, this biomarker is believed to predict the risk of all-cause mortality and liver-related clinical events in patients with MASH.
4. Why Is This Significant?
- Non-invasive alternative to liver biopsy: Traditionally, liver biopsy (histologic assessment) has been the gold standard for evaluating liver injury and fibrosis. However, biopsies are invasive, costly, and pose risks to patients.
- Practical benefits: Adopting a non-invasive biomarker could:
- Improve trial recruitment
- Enable trials to include more representative patient populations
- Help overcome the challenges of conducting adequately powered clinical studies
- Accelerate the development of new therapies for this serious liver condition.
5. Next Steps in the Process
This acceptance is the first stage in the Drug Development Tool qualification process. The applicant (sponsor) and FDA will now collaborate to develop a detailed qualification plan that outlines:
- Data requirements
- Validation steps
- Specific context of use for the biomarker.
Two Words: Biopsy Avoidance
The Net Net
The FDA has begun the qualification process for VCTE-measured liver stiffness as a potential reasonably likely surrogate endpoint in non-cirrhotic MASH drug trials. If successfully qualified, this non-invasive marker could transform how clinical outcomes—like mortality and liver complications—are predicted, reducing dependency on invasive biopsies and enhancing the efficiency and inclusivity of future clinical trials.