FDA Accepts First Non-Invasive Surrogate Endpoint for MASH Clinical Trials
核心洞察
The FDA has accepted Echosens' Letter of Intent to qualify liver (搜索) stiffness measurement by FibroScan (搜索)® as the first non-invasive surrogate endpoint for MASH (搜索) clinical trials.
This milestone could replace invasive liver (搜索) biopsies for patient enrollment and treatment response assessment in MASH (搜索) drug development programs.
The acceptance is supported by over 5,600 peer-reviewed publications and endorsements from major pharmaceutical companies including Eli Lilly, Boehringer Ingelheim, and Novo Nordisk.
The U.S. Food and Drug Administration (搜索) has accepted Echosens' Letter of Intent to qualify Liver (搜索) Stiffness Measurement by Vibration-Controlled Transient Elastography (FibroScan (搜索)®) as a reasonably likely surrogate endpoint in clinical trials for metabolic dysfunction-associated steatohepatitis (搜索) (MASH (搜索)). This marks the first time the FDA is accepting the initiation of the qualification process of a non-invasive test as a reasonably likely surrogate endpoint in drug development for MASH—a progressive, advanced form of steatotic liver disease (搜索) that can lead to cirrhosis (搜索), liver cancer (搜索), transplantation, or death.
Regulatory Milestone for MASH Drug Development
Frank Anania, MD, director of the Division of Hepatology and Nutrition in the FDA's Center for Drug Evaluation and Research, stated, "This proposal represents an important step toward adopting non-invasive tests in drug development for MASH (搜索)." The acceptance opens a path for FibroScan (搜索)® to ultimately replace liver (搜索) biopsy as the primary endpoint for the interim analysis of trials designed for the accelerated approval of drugs for adults with non-cirrhotic MASH.
According to the Letter of Intent, LSM by VCTE (FibroScan (搜索)®) can predict the risk of all-cause mortality or liver (搜索)-related events in patients with MASH (搜索). This capability positions the technology as a viable alternative to the invasive liver biopsies currently used to enroll patients and monitor therapeutic response.
Clinical Advantages and Patient Impact
Replacing repeated liver (搜索) biopsies with FibroScan (搜索)® would ease a significant burden on patients. A FibroScan® exam takes just a few minutes, is painless, and assesses the liver comprehensively, providing a full picture of liver health that can be repeated as often as needed. This contrasts sharply with liver biopsies, which are known to be highly invasive, causing discomfort and safety concerns for patients.
Arun Sanyal, MD, Professor and Director at the Stravitz-Sanyal Institute for Liver (搜索) Disease and Metabolic Health at Virginia Commonwealth University, commented, "The acceptance of this Letter of Intent is a major milestone, built on years of clinical research and publications, together with the tireless efforts of numerous stakeholders across the field, with major implications for the field and is expected to accelerate drug development and also be easily translatable to clinical practice."
Strong Clinical Validation and Industry Support
The FDA's acceptance is driven by FibroScan (搜索)'s extensive clinical validation and its use in key pharmaceutical trials in liver (搜索) drug development, especially for MASH (搜索). The technology is supported by more than 5,600 peer-reviewed publications, including pivotal publications showing that changes in LSM by VCTE are associated with liver-related outcomes. These include studies by Gawrieh et al. in the Journal of Hepatology (2024), Lin et al. in JAMA (2024), and Mózes et al. in Lancet Gastroenterol Hepatol (2023).
Echosens' submission to the FDA included official letters of support from major pharmaceutical companies including Eli Lilly, Boehringer Ingelheim, and Novo Nordisk. FibroScan (搜索)® is already the reference point-of-care non-invasive test used by all key pharmaceutical companies and biotechs conducting MASH (搜索) trials.
Transformational Impact on Drug Development
Jay Patel, Senior Pharma Analyst at GlobalData (搜索), noted that "the announcement can have a transformational impact on future MASH (搜索) clinical trials, driving a shift away from the use of liver (搜索) biopsies in diagnosis and assessment of disease progression." He explained that biopsies complicate trial enrollment, driving up costs and causing unnecessary delays, creating demand for non-invasive alternatives.
For pharmaceutical companies, the acceptance of FibroScan (搜索)® as a reasonably likely surrogate endpoint has the potential to transform drug development in MASH (搜索) by improving trial recruitment and patient retention. This milestone could accelerate the path to new therapies while helping researchers design safer and more representative, efficient trials.
Professor Quentin Anstee, Pro-Vice-Chancellor, Faculty of Medical Sciences at Newcastle University, stated, "The FDA accepting this letter of intent is a critical first step to ultimately have non-invasive tests adopted as reasonably likely surrogate endpoints in MASH (搜索) clinical trials. It's a clear signal to all pharma sponsors to collect robust non-invasive biomarker data in their ongoing or future clinical trials in MASH."
Market Implications and Future Outlook
The acceptance strengthens Echosens' competitive position as the developer of the sole non-invasive test with surrogate endpoint status. Drug developers will likely be eager to adopt the technology to get their assets to market faster. The recent announcement by Eli Lilly that it would pursue a Phase III trial of tirzepatide and retratrutide (搜索) in metabolic dysfunction-associated steatotic liver disease (搜索) (MASLD (搜索)) with non-invasive recruitment demonstrates that major companies in the metabolic disease market are embracing this shift.
In addition to existing recommendations from all prominent guidelines and its preferred position in payer policies, the FDA's acceptance further strengthens FibroScan (搜索)'s position as the reference non-invasive test to prescribe and monitor MASH (搜索) treatments. The milestone also underscores Echosens' leadership and ongoing innovation, including recent advances with Guided VCTE in the latest generation of FibroScan® devices, delivering greater efficiency, automation, and standardization.