Acer NDA for Urea Cycle Disorders

FDA has accepted for review an Acer Therapeutics NDA for Acer-001 (sodium phenylbutyrate) for treating patients with urea cycle disorders (UCDs). Acer says the submission is based on data from two bioequivalence trials in which Acer-001 showed similar relative bioavailability compared to the reference drug, Ucyclyd Pharma’s Buphenyl (sodium phenylbutyrate). The agency has assigned a user fee review action target date of 6/5/2022.

UCDs are caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream (hyperammonemia), Acer says. “Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits,” it says.