Carbaglu approved for N-acetylglutamate synthase (NAGS) deficiency
CARBAGLU (carglumic acid) 200mg tablets by Orphan Europe
The FDA has approved Carbaglu (carglumic acid tablets, from Orphan Europe) for the treatment of the genetic disorder N-acetylglutamate synthase (NAGS) deficiency, which causes hyperammonemia. This approval was based on study results in 23 patients that demonstrated that Carbaglu reduced blood ammonia levels within 24 hours and normalized ammonia levels within three days. The majority of patients in the study appeared to maintain normal plasma ammonia levels with long-term Carbaglu treatment.