CARGLUMIC ACID · NOVITIUM PHARMA
Carglumic acid is a synthetic structural analogue of N-acetylglutamate (NAG) indicated for the treatment of acute and chronic hyperammonemia in pediatric and adult patients with N-acetylglutamate synthase (NAGS) deficiency. It is used as an adjunctive therapy to standard of care during acute hyperammonemic episodes and as long-term maintenance therapy to prevent ammonia toxicity by restoring urea cycle function.
How CARGLUMIC ACID Works
Carglumic acid acts as a replacement for N-acetylglutamate (NAG) by functioning as a synthetic structural analogue. NAG is the essential allosteric activator of carbamoyl phosphate synthetase 1 (CPS 1), the mitochondrial liver enzyme that catalyzes the first and rate-limiting step of the urea cycle. In patients with NAGS deficiency, the lack of endogenous NAG prevents the conversion of ammonia to urea. Carglumic acid activates CPS 1, thereby restoring the detoxification of ammonia into urea, which is then excreted in the urine.
Details
- Status
- Prescription
- First Approved
- 2010-03-18
- Routes
- ORAL
- Dosage Forms
- TABLET, FOR SUSPENSION
CARGLUMIC ACID Approval History
What CARGLUMIC ACID Treats
2 indicationsCARGLUMIC ACID is approved for 2 conditions since its original approval in 2021. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Hyperammonemia
- N-Acetylglutamate Synthase Deficiency
CARGLUMIC ACID 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
1 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| 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
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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.
CARGLUMIC ACID FDA Label Details
Indications & Usage
Carglumic acid tablets for oral suspension is a carbamoyl phosphate synthetase 1 (CPS 1) activator indicated in pediatric and adult patients as: Adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency. Maintenance therapy for the treatment of chronic hyperammonemia due to NAGS deficiency. 1.1 Acute and Chronic Hyperammonemia due to N-acetylglutamate Synthase (NAGS) Deficiency Carglumic acid tablets for oral suspension is indicated in adult and pediatric patients as: Adjunctive therapy to standard of care for the treat...
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Full clinical data, patents, trials, and competitive landscape for carglumic acid.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.