The only drug approved as a chronic treatment for urea cycle disorders requires high doses and has high sodium content, resulting in a risk of hypertension and reduced appetite. Hyperion Therapeutics Inc. believes its sodium-free HPN-100 can avoid these side effects with reduced dosing intensity while achieving similar efficacy.
Ammonia is produced in the gut with protein ingestion. As part of the urea cycle in healthy patients, ammonia is converted to urea in the liver and later excreted.
Patients with urea cycle disorder (UCD) lack one of the six enzymes involved in urea metabolism. This