Last reviewed · How we verify
Carbaglu (CARGLUMIC ACID)
Carbaglu works by activating the enzyme carbamoyl phosphate synthetase 1, which helps regulate ammonia levels in the body.
Carbaglu (Carglumic Acid) is a small molecule drug developed by Orphan Europe and currently owned by Recordati Rare. It works by activating carbamoyl phosphate synthetase 1, an enzyme involved in ammonia metabolism. Carbaglu is approved by the FDA to treat hyperammonemia associated with N-acetylglutamate synthase deficiency. The drug is off-patent and has multiple generic manufacturers. As a result, Carbaglu is commercially available in a generic form.
At a glance
| Generic name | CARGLUMIC ACID |
|---|---|
| Sponsor | Recordati |
| Drug class | Carbamoyl Phosphate Synthetase 1 Activator [EPC] |
| Target | Carbamoyl-phosphate synthase [ammonia], mitochondrial |
| Modality | Small molecule |
| Therapeutic area | Gastroenterology |
| Phase | FDA-approved |
| First approval | 2010 |
| Annual revenue | 200 |
Mechanism of action
Carglumic acid is synthetic structural analogue of N-acetylglutamate (NAG) which is produced from glutamate and acetyl-CoA in reaction catalyzed by N-acetylglutamate synthase (NAGS), mitochondrial liver enzyme. NAG acts as the essential allosteric activator of carbamoyl phosphate synthetase (CPS 1), mitochondrial liver enzyme which catalyzes the first reaction of the urea cycle. The urea cycle, whose role is the disposition of ammonia, includes series of biochemical reactions in the liver resulting in the conversion of ammonia into urea, which is then excreted through the urine. Carglumic acid acts as CPS1 activator, improves or restores the function of the urea cycle, and facilitates ammonia detoxification and urea production.
Approved indications
- Hyperammonemia associated with N-acetylglutamate synthase deficiency
Common side effects
- Vomiting
- Abdominal pain
- Pyrexia
- Tonsillitis
- Anemia
- Diarrhea
- Ear infection
- Infections
- Nasopharyngitis
- Hemoglobin decreased
- Headache
- Neutropenia
Key clinical trials
- An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
- Long-term Efficacy of Carglumic Acid in Organic Acidemia. (NA)
- Orphan Europe Carbaglu® Surveillance Protocol
- Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
- Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia (PHASE2)
- N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia (PHASE2,PHASE3)
- Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia (PHASE3)
- Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Carbaglu CI brief — competitive landscape report
- Carbaglu updates RSS · CI watch RSS
- Recordati portfolio CI