Versantis receives U.S. FDA Orphan Drug Designation for VS-01

Designation Awarded for Treatment of Hyperammonemia in Inborn Errors of Metabolism

Versantis AG, a clinical-stage company developing novel therapies for rare liver diseases and rare pediatric diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its lead product candidate VS-01 for the treatment of hyperammonemia (HA) in inborn errors of metabolism (IEM).

“This orphan drug designation from the FDA validates the scientific rationale of VS-01 in a new indication encompassing multiple genetic disorders where there is a vital need to remove the life-threatening build-up of ammonia,” said Meriam Kabbaj, Chief Operations Officer and Co-founder of Versantis. “VS-01 has now received U.S. FDA ODD for two main indications, hyperammonemia in inborn errors of metabolism and Acute-on-chronic Liver Failure (ACLF). Orphan drug incentives considerably strengthen the clinical development plan of our liposomal scavenging platform technology and provide a huge opportunity to soon address the growing burden of metabolic / liver diseases.”

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