Release Date: 03-Oct-2021
U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to lead product candidate VS-01 for the treatment of hyperammonemia (HA) in inborn errors of metabolism (IEM).
The CEO of a clinical-stage company ‘Versantis AG’ said that the new designation from the FDA continues to validate our approach and development of VS-01, which was shown to be safe and well tolerated in our Phase 1b clinical study in patients with decompensated liver cirrhosis.
The FDA Orphan Drug Designation Program gives orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the US and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.