NDAORALTABLET, FOR SUSPENSIONPriority Review
Approved
Mar 2010
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
3
Data completeness
5/8 — Partial
Priority Review
CARBAGLU (carglumic acid) by Recordati is carbamoyl phosphate synthetase 1 activators [moa]. First approved in 2010.
Drug data last refreshed 2h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
CARBAGLU (carglumic acid) is an oral small-molecule activator of carbamoyl phosphate synthetase 1 (CPS1), an enzyme critical to the urea cycle. It is indicated for the treatment of hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency, a rare genetic disorder. The drug works by directly activating CPS1, bypassing the need for N-acetylglutamate cofactor and restoring normal ammonia detoxification.
$9MPart D
LOE Approaching1.6 years to LOEModest market with very low claim volume and imminent LOE creates urgency for lifecycle management strategies and post-LOE transition planning.
Mechanism of Action
Carbamoyl Phosphate Synthetase 1 Activators
Pharmacologic Class:
Carbamoyl Phosphate Synthetase 1 Activator
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (3)
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Started Jun 2022
20 enrolled
HyperammonemiaMethylmalonic AcidemiaPropionic Acidemia
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Started Jan 2019
95 enrolled
Methylmalonic AcidemiaPropionic Acidemia
N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia
Started Aug 2008
52 enrolled
Inborn Errors of Metabolism
Career Relevance
CARBAGLU represents a niche orphan/ultra-rare disease asset with minimal linked job openings, reflecting its small patient population and modest market footprint. Career growth on this product is limited; roles focus on specialized rare disease expertise, compliance, and managed-care navigation rather than broad commercial scale.
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.