FDA Approves Olpruva for Urea Cycle Disorders

The FDA has approved Acer Therapeutics and Relief Therapeutics’ Olpruva (sodium phenylbutyrate) for oral suspension in treating urea cycle disorders, rare genetic conditions that cause excess ammonia to build up in the blood.

The drug is specifically approved for treatment of patients with deficiencies of the enzymes carbamylphosphate synthetase, ornithine transcarbamylase or argininosuccinic acid synthetase.

The companies supported the New Drug Application with preclinical and clinical data showing bioequivalence to the reference listed drug Buphenyl powder, which is approved as adjunctive therapy for patients with urea cycle disorders with the same enzyme deficiencies.