carglumic acid
Orphan DrugFDA Approved, EMA Approved
Description
Carglumic acid is a synthetic structural analogue of N-acetylglutamate (NAG), which serves as an essential cofactor for the first enzyme in the urea cycle. It is used as adjunctive therapy for the treatment of acute hyperammonemia and maintenance therapy for chronic hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency. The drug can also be used in certain organic acidemias including methylmalonic acidemia.
Indications & Therapeutic Use
hyperammonemia due to N-acetylglutamate synthase deficiency, acute hyperammonemia in organic acidemias including methylmalonic acidemia
Linked Diseases:
Global Availability (8 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
carglumic acid
| Generic Name | carglumic acid |
| Brands | 1 brand available |
| Active Ingredient | carglumic acid |
| Drug Class | hyperammonemia due to N-acetylglutamate synthase deficiency |
| Manufacturer | Recordati Rare Diseases |
| Dosage Forms | Oral dispersible tablets, 200mg |
| Medical Code | A16AA05 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 14 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT00551200 |
| Countries | 8 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations9 Validated Nodes