Carglumic Acid
CAS 1188-38-1 7 FDA-DMF suppliersSmall Molecule
Overview
Carglumic acid is an orphan drug for the treatment of hyperammonemia, due to N-acetylglutamate synthase deficiency. It works by activating the enzyme carbamoyl phosphate synthetase, which is a part of the urea cycle.
Patent & exclusivity (US)
Per the FDA Orange Book (3 approved US products), the latest listed US patent on Carglumic Acid expires — and exclusivity 22 Jan 2028. Generic API sourcing typically opens up once these lapse.
Therapeutic areas
Metabolic DisordersGenetic DisordersUrea Cycle Disorders
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