Medicis Announces FDA Approval of Ammonul for Urea Cycle Disorder

The FDA has approved Medicis' Ammonul as an adjunctive therapy for the treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle.

The FDA granted Ammonul (sodium phenylacetate/sodium benzoate) orphan-drug status with seven years of exclusivity based on long-term compassionate patient use in patients with Urea Cycle Disorder (UCD). UCD is an inherited, inborn error of metabolism. Patients with UCD lack one of the key enzymes that comprise the urea cycle, which can result in the life-threatening condition of hyperammonemia, or dangerously heightened levels of ammonia in the bloodstream.

Ammonul is a hospital product administered intravenously as a rescue medication when a UCD patient progresses into a hyperammonemic crisis.