FDA Accepts Liver Stiffness Measurement as Surrogate Endpoint,Accelerating MASH Drug Development
Washington D.C. – In a landmark decision poised to dramatically accelerate the development of treatments for metabolic-associated steatohepatitis (MASH), formerly known as NASH, the Food and Drug Administration (FDA) has accepted liver stiffness measurement (LSM) by Vibration Controlled Transient Elastography (VCTE) as a surrogate endpoint for clinical benefit. This change, announced recently, streamlines the pathway for drug approvals in MASH, a condition affecting millions wiht no currently approved pharmaceutical therapies.
Previously, MASH drug trials required lengthy and costly studies tracking histological improvements – examining liver tissue biopsies – to demonstrate efficacy. The FDA’s acceptance of LSM by VCTE, a non-invasive method of assessing liver fibrosis, significantly reduces the time and resources needed for clinical trials, potentially bringing effective treatments to patients faster. This decision impacts pharmaceutical companies currently developing MASH therapies, and also the estimated 1.5 million Americans and millions more worldwide affected by the disease.The acceptance of LSM by VCTE builds on growing evidence demonstrating its correlation with long-term clinical outcomes, including liver-related events and mortality. Several key trials are already utilizing the endpoint, including Madrigal Pharmaceuticals’ SYNCHRONY-Outcomes trial of efruxifermin and 89bio’s ENLIGHTEN-Cirrhosis trial of pegozafermin. Additionally, LSM by VCTE is being employed in alcohol-related liver disease (ALD) trials, such as GSK’s Phase II STARLIGHT trial of GSK4532990 and Altimmune’s Phase II RESTORE trial of pemvidutide.
According to Jay Patel, a Pharma analyst with GlobalData, the FDA’s decision ”consolidates Echosens’ position as the leading non-invasive diagnostic manufacturer in the MASH space and opened the door for faster approval of a wider range of drugs to meet the unmet need in this growing disease.” Echosens recently launched its Guided VCTE technology, further streamlining the assessment process.
The shift is expected to encourage increased investment and innovation in MASH drug development, offering hope for patients facing a condition that can progress to cirrhosis, liver failure, and the need for transplantation.