Carglumic acid
Carglumic acid is an orphan drug, marketed by Orphan Europe under the trade name Carbaglu. Carglumic acid is used for the treatment of hyperammonaemia in patients with N-acetylglutamate synthase deficiency. The initial daily dose ranges from 100 to 250 mg/kg, adjusted thereafter to maintain normal plasma levels of ammonia.
The US FDA approved it for treatment of hyperammonaemia on March 18, 2010. Orphan Drug exclusivity expired on March 18, 2017.Adverse effects
The most common adverse effects include vomiting, abdominal pain, fever, and tonsillitis.
