The Food and Drug Administration (FDA) has granted Orphan Drug designation to VS-01 for the treatment of hyperammonemia in inborn errors of metabolism.
In patients with certain genetic metabolic diseases, hyperammonemia results from a defective enzyme that inhibits the metabolic clearance of ammonia from the blood. This leads to the toxic accumulation of ammonia, leading to cerebral edema and severe neurologic impairment. Immediate treatment is critical as patients may progress rapidly to coma and death.
VS-01 is a novel liposomal-based intraperitoneal fluid that acts as a clearance enhancer of toxic metabolites from the body, in particular ammonia. “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, COO and Co-founder of Versantis.”
Previously, the FDA granted Orphan Drug designation to VS-01 for the treatment of acute-on-chronic liver failure, along with Rare Pediatric Diseases designation for the acute treatment of urea cycle disorder.
Versantis receives US FDA Orphan Drug designation for VS-01. News release. Versantis AG. Accessed September 7, 2021. https://www.businesswire.com/news/home/20210907005194/en/Versantis-Receives-U.S.-FDA-Orphan-Drug-Designation-for-VS-01.