December 27, 2022
Olpruva receives FDA approval for patients with some urea cycle disorders (UCD’s)
Acer Therapeutics Inc. and its collaboration partner, RELIEF THERAPEUTICS Holding SA, have announced that the U.S. Food and Drug Administration (FDA) has approved Olpruva™ (sodium phenylbutyrate) for oral suspension in the U.S. for the treatment of certain patients living with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).
Olpruva is used along with certain therapies, including changes in diet, for the long-term management of
adults and children weighing 44 pounds (20 kg) or more. It is not used to treat a rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment.