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 expires on March 182017.
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