Udenafil Shows Potential in Treating Fontan-Associated Liver Disease

Fontan-associated liver disease (FALD) remains a critical long-term complication for patients whose survival depends on the Fontan procedure. The FUEL FALD substudy, which evaluated participants over a year of treatment, revealed that 95% of subjects entered the trial with elevated Enhanced Liver Fibrosis (ELF) scores. By the end of the study, 84% of these participants showed a measurable decrease in these scores, indicating a positive shift in fibrotic remodeling.

While ultrasound and magnetic resonance elastography did not show significant changes in overall liver stiffness, the researchers suggest that the drug may still influence the underlying fibrotic environment. Dr. Kurt Schumacher of University of Michigan Health C.S. Mott Children's Hospital, the study's lead author, noted that these results expand the current understanding of how to manage the long-term physiological toll of the Fontan circulation.

Mezzion Pharmaceuticals is now building on these findings as it progresses through the Phase 3 FUEL-2 trial. This larger, global study aims to secure regulatory approval for udenafil as the first therapy specifically indicated for the Fontan patient population. With no current standard of care for FALD, the company views this data as a vital step toward addressing a significant unmet medical need in pediatric and young adult cardiology.