Drug intelligence / Profile preview

VS-01-HAC

Development stage
Preclinical
Lead developer
Genfit
Modality
Small Molecules, Liposomes → Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intraperitoneal
01

Overview

VS-01-HAC is an investigational, liposomal-based peritoneal dialysis fluid containing citric acid, developed by Versantis (a subsidiary of Genfit), designed for the emergency treatment of acute hyperammonemic crisis associated with inborn errors of metabolism such as urea cycle disorders and organic acidemias. The drug leverages a proprietary scavenging liposomal technology to remove ammonia and other toxic metabolites from the body by intraperitoneal administration. This mechanism aims to rapidly reduce life-threatening ammonia levels in pediatric patients and support multi-organ function during metabolic crises. The formulation is being advanced as a first-line lifesaving therapy for acute hyperammonemia, with orphan drug designation granted for several rare indications including liver failure and hyperammonemia[1][4][6].

Other names
VS 01VS01VS-01

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