Pediatric Metabolic Dysfunction–Associated Steatotic Liver Disease Pharmacotherapy: Pathways to Regulatory Approval

Metabolic dysfunction–associated steatotic liver disease (MASLD) is now the most common chronic liver disease in children. Yet no pharmacotherapies are currently approved for pediatric use. Despite rapid advances in adult MASLD/metabolic dysfunction–associated steatohepatitis (MASH) drug development, translation/extrapolation to pediatric populations remains limited due to biologic differences, ethical considerations, feasibility challenges, and regulatory uncertainty. This session convenes regulatory leaders, pediatric clinical trial experts, and translational investigators to clarify pathways for pediatric MASLD drug development and approval. Through short, focused presentations and an interactive panel discussion, the presenters highlight strategies to align trial design, biomarker development, and regulatory expectations to accelerate safe and effective therapies for MASLD in children.