Substance / Medication

Carglumic acid

Overview

Active Ingredient
carglumic acid
RxNorm CUI
401713
Labeler: Navinta LLCUpdated: 2024-03-23T00:00:00.000ZFull label on DailyMed

Contraindications

When this intervention should not be used

None

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Clinical Trials

2 trials linked to this intervention

2
Total Trials
0
Recruiting
2
With Results

Research Evidence

Published studies and systematic reviews

Sort:
Long-term effectiveness of carglumic acid in patients with propionic acidemia (PA) and methylmalonic acidemia (MMA): a randomized clinical trial.
Alfadhel Majid, Nashabat Marwan, Saleh Mohammed et al. · Orphanet J Rare Dis · 2021
PMID: 34635114ObservationalFull text (PMC)
Carglumic acid enhances rapid ammonia detoxification in classical organic acidurias with a favourable risk-benefit profile: a retrospective observational study.
Valayannopoulos Vassili, Baruteau Julien, Delgado Maria Bueno et al. · Orphanet J Rare Dis · 2016
PMID: 27030250ObservationalFull text (PMC)
Challenges in optimizing sample preparation and LC-MS/MS conditions for the analysis of carglumic acid, an N-acetyl glutamate derivative in human plasma.
Sharma Primal, Shah Priyanka A, Sanyal Mallika et al. · Drug Test Anal · 2015
PMID: 25677217Observational
Case Report: Carglumic acid accelerates ammonia clearance in a neonate with methylmalonic acidemia.
Ma Qianli, Luo Yunfeng, Zhang Xu et al. · Front Pediatr · 2025
PMID: 41404430Case ReportFull text (PMC)
Carglumic acid as a treatment for persistent hyperammonemia in carnitine-acylcarnitine translocase deficiency: A case study.
Babazade Hanım, Zubarioglu Tanyel, Uygur Esma et al. · Mol Genet Metab Rep · 2025
PMID: 40092581Case ReportFull text (PMC)
[Sequential therapy with carglumic acid in three cases of organic acidemia crisis].
Chen Yan-Yan, Cheng Ting-Ting, Yao Jie et al. · Zhongguo Dang Dai Er Ke Za Zhi · 2025
PMID: 40695518Case ReportFull text (PMC)
Carglumic acid in methylmalonic acidemia: Use of breast milk as an alternative vehicle to water.
García-Díaz Héctor C, Parramón-Teixidó Carlos J, Clemente-Batista Susana et al. · J Clin Pharm Ther · 2022
PMID: 35633061Case Report
Carglumic acid (Carbaglu®) treatment in hyperammonemia post liver transplantation.
Arrieta Francisco, Belanger-Quintana Amaya, Gajate Luis et al. · Endocrinol Diabetes Nutr (Engl Ed) · 2020
PMID: 31447407Case Report
Carglumic Acid Contributes to a Favorable Clinical Course in a Case of Severe Propionic Acidemia.
Kido Jun, Matsumoto Shirou, Nakamura Kimitoshi · Case Rep Pediatr · 2020
PMID: 32231837Case ReportFull text (PMC)

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Carglumic acid (substance)
SNOMED CT
412295009
UMLS CUI
C1318649
RxNorm CUI
401713
Labeler
Navinta LLC

Clinical Data

This intervention maps to 1 entities in the Ltrl knowledge graph.

1
Conditions
0
Biomarkers
0
Specialists
0
Symptoms
2
Clinical Trials

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.