Drug development for urea cycle disorders

Urea cycle disorder is an inherited metabolic disorder. The human body excretes the protein metabolite ammonia from the body through the urea cycle, and when this cycle is blocked, ammonia will accumulate in the blood. When the concentration of ammonia in the blood is high enough, it will cause brain damage, coma or death. The incidence of neonatal urea cycle disorders is 1 in 10,000.
      Not long ago, the final clinical trial of HPN-100, an innovative drug used by Hyperion Therapeutics for urea cycle disorders in the United States, obtained positive results. This research result provided a guarantee that the drug was approved by the FDA in September 2011. Christine Nash, vice president of Hyperion Therapeutics, estimates that the drug has a market prospect of $200 million. In addition, Medicis Pharmaceuticals has an FDA-approved adjuvant drug Ammonul for acute hyperammonemia and related encephalopathy in patients with urea cycle-related enzyme defects.