Carglumic Acid 200mg Suppliers & Bulk Manufacturers

Available Forms: Dispersible Tablets

Available Strengths: 200mg

Reference Brands: Carbaglu (USA/EU)

Category: Orphan Drugs

Carglumic acid is a synthetic structural analogue of N-acetylglutamate, an essential activator of the enzyme carbamoyl phosphate synthetase I in the urea cycle. It helps lower elevated ammonia levels in the blood (hyperammonemia) caused by N-acetylglutamate synthase deficiency. The drug restores normal urea cycle activity, preventing neurological damage and other complications.

Product Description:

Carglumic acid is a synthetic analogue of N-acetylglutamate, the molecule that switches on carbamoyl phosphate synthetase 1 — the first enzyme of the urea cycle. Without that activator the cycle cannot start, ammonia accumulates, and hyperammonaemia damages the brain within hours. Carglumic acid substitutes for the missing activator and restarts the cycle.

It is used for hyperammonaemia caused by N-acetylglutamate synthase deficiency, where it is effectively curative and allows a normal protein diet, and for hyperammonaemia in the organic acidaemias — propionic, methylmalonic and isovaleric — where accumulating metabolites inhibit the same activator. In acute decompensation it works within hours, which matters because outcome depends on how long the brain is exposed to ammonia. The tablets are dispersed in water and given through the mouth or a nasogastric tube. It is generally well tolerated; vomiting, abdominal pain and fever are reported.

Sourcing notes. An orphan product with very small patient numbers and high cost, and one whose value depends on being physically present when a crisis occurs — metabolic units hold stock rather than ordering on demand. Emergency and named-patient supply routes matter more than tender pricing. Tablets must be dispersed, not swallowed whole.