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 10h ago · AI intelligence enriched 1mo ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
CARBAGLU (carglumic acid) is an oral small-molecule activator of carbamoyl phosphate synthetase 1 (CPS1) used to treat hyperammonemia and urea cycle disorders. It works by enhancing the first committed step of the urea cycle, reducing toxic ammonia levels in patients with CPS1 deficiency and related metabolic disorders.
$9.0MPart D
LOE Approaching1.2 years to LOENiche, low-volume specialty product with minimal commercial team expansion; LOE in ~1.3 years signals transition to generic support and lifecycle management focus.
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 offers niche career opportunities in rare-disease specialty pharma and precision medicine, with emphasis on MSL roles, KOL engagement, and patient support programs. However, the imminent LOE (1.3 years) makes this a short-term strategic position suitable for professionals seeking expertise in lifecycle transition, generic defense, and health economics in orphan indications.
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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.