PathAI announced that the US Food and Drug Administration (FDA) has qualified AIM-MASH AI Assist through the Drug Development Tool (DDT) Biomarker Qualification Programme (BQP) for use within a context of use in metabolic dysfunction–associated steatohepatitis (MASH) clinical trials. AIM-MASH AI Assist (formerly known as AIM-NASH) is the first AI-powered pathology DDT to receive FDA qualification.

The qualification follows the European Medicines Agency (EMA) CHMP qualification of AIM-MASH AI Assist earlier this year, underscoring PathAI’s regulatory leadership across both US and European frameworks for AI-assisted histopathology in drug development.

This qualification signifies a major advancement in MASH clinical trials, offering a more efficient, standardised and reproducible approach to evaluating liver biopsies globally during enrollment and endpoint assessment. AIM-MASH AI Assist has the potential to accelerate the development of new treatments by providing more efficient and accurate assessments of biopsy-based patient response to drug candidates within and across MASH clinical trials.

The FDA's DDT programme enables biomarkers qualified for a specific context of use to enhance drug development. As per the FDA, drug developers may utilise this DDT for the qualified Context of Use (COU) in Investigational New Drug Applications (INDs), New Drug Applications (NDAs) and Biologics Licence Applications (BLAs) submissions, thereby eliminating the need to resubmit DDT information or undergo additional CDER re-review. This is aligned to support the needs of biopsy-based enrollment and endpoint assessment, including for accelerated approval in the US, and conditional approval in the EU, in MASH clinical trials. This first-in-class AI-based DDT allows a single pathologist to provide standardised and accurate MASH biopsy assessments at scale.

“Dual qualification by the EMA and the FDA gives sponsors a regulator-approved path to use AI-assisted histology for Phases 2 and 3 MASH clinical trial enrollment and endpoint assessment. For MASH trials worldwide, that means greater consistency, reproducibility and confidence in histologic endpoints,” said Naim Alkhouri, MD, Fellow, American Association for the Study of Liver Diseases (FAASLD).

The FDA qualification builds on an extensive analytical and clinical validation foundation referenced by EMA, which demonstrated non-inferiority to average individual pathologist reads for fibrosis and steatosis, superiority to individual expert manual reads for ballooning, inflammation and clinical trial enrollment and endpoint criteria, and superiority for reproducibility compared to manual reads across every precision metric tested, which is one of the key challenges in MASH biopsy scoring for primary endpoint assessment. Together, these regulatory milestones deliver an improved path for sponsors to operationalise AI-assisted histology across global MASH drug development programmes.

“We’re proud to have worked collaboratively with FDA, EMA, industry partners and advisers to achieve the first FDA and EMA qualification of an AI-based biomarker via their biomarker qualification programmes. Dual recognition from US and EU regulators underscores our commitment to rigorous validation and responsible deployment of AI to transform pathology and improve patient outcomes,” said Andy Beck, MD, PhD, Co-Founder and CEO, PathAI.