VS-01 Got Orphan Drug Designation To Treat Hyperammonemia

Release Date: 26-Sep-2021

US FDA has granted Orphan Drug designation to VS-01 to treat hyperammonemia in inborn errors of metabolism. VS-01 is a liposomal-based intraperitoneal fluid which acts as a clearance enhancer of toxic metabolites from the body, in particular ammonia.


In past, 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.


In patients with some genetic metabolic diseases, hyperammonemia results from a defective enzyme that inhibits the metabolic clearance of ammonia from the blood. This causes toxic accumulation of ammonia, leading to cerebral edema and severe neurologic impairment. Immediate treatment is required as patients may progress rapidly to death and coma.

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