CARBAGLU (carglumic acid)
Carbaglu (carglumic acid) is a carbamoyl phosphate synthetase 1 (CPS 1) activator indicated for use in pediatric and adult patients to treat hyperammonemia. It is used as adjunctive therapy to standard of care for **acute** hyperammonemia caused by N-acetylglutamate synthase (NAGS) deficiency, propionic acidemia (PA), or methylmalonic acidemia (MMA). It is also indicated for the **maintenance** treatment of chronic hyperammonemia specifically in patients with NAGS deficiency.
How CARBAGLU Works
Carglumic acid is a synthetic structural analogue of N-acetylglutamate (NAG), which is the essential allosteric activator of carbamoyl phosphate synthetase 1 (CPS 1). CPS 1 is the mitochondrial liver enzyme that catalyzes the initial step of the urea cycle. In patients with NAGS deficiency, or during acute metabolic decompensation in PA and MMA, endogenous NAG levels are insufficient. Carglumic acid mimics NAG to activate CPS 1, thereby restoring urea cycle function and facilitating the detoxification of ammonia into urea for renal excretion.
Details
- Status
- Prescription
- First Approved
- 2010-03-18
- Patent Cliff
- 2028
- Routes
- ORAL
- Dosage Forms
- TABLET, FOR SUSPENSION
CARBAGLU Approval History
What CARBAGLU Treats
4 indicationsCARBAGLU is approved for 4 conditions since its original approval in 2010. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Hyperammonemia
- N-Acetylglutamate Synthase Deficiency
- Propionic Acidemia
- Methylmalonic Acidemia
CARBAGLU Competitive Set
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Indication competitors
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Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
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Clinical Trial Registry
2 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT01599286 STO results posted | NCGC0008 | Ph 2 | completed | Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia |
| NCT02426775 CAMP | RC 13/116 | Ph 3 | completed | Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia |
Active Pipeline
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Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
CARBAGLU FDA Label Details
Indications & Usage
FDA Label (PDF)CARBAGLU is indicated for the treatment of Hyperammonemia; N-Acetylglutamate Synthase Deficiency; Propionic Acidemia; Methylmalonic Acidemia.
CARBAGLU Patents & Exclusivity
Exclusivity
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.