Page last updated: 2024-11-07

carglumic acid

Description Research Excerpts Clinical Trials Roles Classes Pathways Study Profile Bioassays Related Drugs Related Conditions Protein Interactions Research Growth Market Indicators

Description

Carglumic acid, also known as N-carbamylglutamate, is a synthetic analogue of the natural amino acid glutamate. It is used to treat hyperammonemia type 1, a rare genetic disorder that prevents the body from properly breaking down ammonia. Carglumic acid works by acting as a substrate for the enzyme carbamoyl phosphate synthetase I (CPS I), which is deficient in patients with hyperammonemia type 1. This allows for the detoxification of ammonia in the liver and reduces the levels of ammonia in the blood. Carglumic acid is typically administered orally and is well-tolerated. It has been shown to be effective in reducing ammonia levels and improving clinical symptoms in patients with hyperammonemia type 1. Carglumic acid is a valuable treatment option for this serious disorder.'
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carglumic acid: carglumic acid could be helpful in lowering plasma ammonia levels over 400 micromol/L more rapidly [Medical Subject Headings (MeSH), National Library of Medicine, extracted Dec-2023]

carglumic acid : A urea that is the N-carbamoyl derivative of L-glutamic acid. An orphan drug used to treat a deficiency in the enzyme N-acetylglutamate synthase, which leads to acute hyperammonaemia. [Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Cross-References

ID SourceID
PubMed CID121396
CHEMBL ID1201780
CHEBI ID71028
SCHEMBL ID373546
MeSH IDM0520883

Synonyms (75)

Synonym
glutamic acid, n-carbamoyl-, l-
carglumic acid [inn]
carbamino-l-glutamic acid
carbaglu
l-glutamic acid, n-(aminocarbonyl)-
carbamylglutamic acid
l-n-carbamoylglutamic acid
n-carbamylglutamate
n-carbamoyl-l-glutamic acid
ureidoglutaric acid
D07130
carbaglu (tn)
carglumic acid (jan/usan/inn)
C05829
carglumic acid
n-carbamyl-l-glutamate ,
1188-38-1
n-carbamyl-l-glutamic acid
n-carbamoyl-l-glutamate
(2s)-2-(carbamoylamino)pentanedioic acid
(s)-2-ureidopentanedioic acid
DB06775
CHEMBL1201780
chebi:71028 ,
oe 312 (laboratory code designation)
nsc-760124
oe-312
oe 312
A804117
C-1805
cas-1188-38-1
tox21_112544
dtxsid7046706 ,
dtxcid5026706
carbamylglutamate
5l0hb4v1ew ,
carglumic acid [usan:inn]
nsc 760124
unii-5l0hb4v1ew
AKOS010384789
acide carglumique
acidum carglumicum
acido carglumico
S5301
carglumic acid [jan]
carglumic acid [ema epar]
carbamyl-l-glutamic acid
carglumic acid [vandf]
carglumic acid [mart.]
carglumic acid [usp-rs]
carglumic acid [usan]
carglumic acid [mi]
carglumic acid [who-dd]
carglumic acid [orange book]
NCGC00274073-01
tox21_112544_1
gtpl7458
SCHEMBL373546
n-carbamyl-glutamic acid
n-(aminocarbonyl)-l-glutamic acid(carglumic acid)
J-507608
carglumic acid, united states pharmacopeia (usp) reference standard
n-carbamyl-l-glutamic acid crystalline
CS-8208
BCP16753
Z756468010
HY-B0711
DS-17934
Q822884
EN300-122612
carglumic-acid
oe312
carglumic acid (mart.)
carglumic acid (usp-rs)
n-carbamylglutamate(ncg)

Research Excerpts

Overview

Carglumic acid is a structural analogue of human N-acetylglutamate. It has become an alternative therapeutic option for hyperammonaemia in organic acidaemias.

ExcerptReferenceRelevance
"Carglumic acid is a structural analogue of human N-acetylglutamate, which has become an alternative therapeutic option for hyperammonaemia in organic acidaemias such as isovaleric acidaemia, methylmalonic acidaemia and propionic acidaemia, and it has been suggested in other urea cycle disorders such as ornithine transcarbamylase deficiency and carbamoyl phosphate synthetase 1 deficiency."( First report of carglumic acid in a patient with citrullinemia type 1 (argininosuccinate synthetase deficiency).
Aksoy, B; Arslan, N; Häberle, J; Kose, E; Kuyum, P; Ozturk, Y, 2018
)
2.27

Bioavailability

ExcerptReferenceRelevance
"The ATP-binding cassette transporter P-glycoprotein (P-gp) is known to limit both brain penetration and oral bioavailability of many chemotherapy drugs."( A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
Ambudkar, SV; Brimacombe, KR; Chen, L; Gottesman, MM; Guha, R; Hall, MD; Klumpp-Thomas, C; Lee, OW; Lee, TD; Lusvarghi, S; Robey, RW; Shen, M; Tebase, BG, 2019
)
0.51

Dosage Studied

ExcerptRelevanceReference
" The patient was receiving a protein-restricted diet supplemented with L-carnitine at a dosage of 100 mg/kg/d during 2 consecutive months."( Outpatient treatment of propionic acidemia-associated hyperammonemia with N-carbamoyl-L-glutamate in an infant.
Aydin, A; Demirci, E; Soyucen, E, 2010
)
0.36
" coli K88 (10(8) CFU/mL), whereas pigs in the sham challenge groups (I and II) were orally dosed with an equal volume of water."( Dietary N-Carbamylglutamate Supplementation Boosts Intestinal Mucosal Immunity in Escherichia coli Challenged Piglets.
Huang, Z; Liu, H; Ma, X; Qiao, S; Yang, F; Zeng, X; Zhang, F, 2013
)
0.39
" If this observation is confirmed, future studies should aim to optimize the dosage and explore effects of the dosage requirements on other drugs and on protein tolerance."( Long-term continuous N-carbamylglutamate treatment in frequently decompensated propionic acidemia: a case report.
Carella, A; Di Mauro, AM; Fedele, S; Masciopinto, M; Melpignano, L; Ortolani, F; Papadia, F; Piccinno, E; Tummolo, A; Vendemiale, M, 2018
)
0.48
" The dosage and timing for NCG administration were evaluated for its effect on follicular development."( Effect of dietary N-carbamylglutamate on development of ovarian follicles via enhanced angiogenesis in the chicken.
Lin, X; Ma, Y; Mi, Y; Zeng, W; Zhang, C; Zhou, S, 2020
)
0.56
" Additional studies aimed at better defining a proper dosage for the chronic administration of carglumic acid and the clinical and biochemical characteristics of patients treated chronically are needed."( Long-term use of carglumic acid in methylmalonic aciduria, propionic aciduria and isovaleric aciduria in Italy: a qualitative survey.
Bettocchi, I; Biasucci, G; Bordugo, A; Burlina, A; Gasperini, S; La Spina, L; Maines, E; Meli, C; Menni, F; Paci, S; Procopio, E; Rossi, A; Rubert, L; Spada, M; Tubili, F; Tummolo, A, 2022
)
1.28
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Roles (2)

RoleDescription
orphan drugAny drug that has been developed specifically for treatment of a rare medical condition, the condition itself being known as an orphan disease.
carbamylphosphate synthetase I activatorAny compound that binds and activates the enzyme carbamylphosphate synthetase I.
[role information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Drug Classes (2)

ClassDescription
N-acyl-L-glutamic acidAny optically active N-acylglutamic acid having L-configuration.
ureas
[compound class information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Pathways (1)

PathwayProteinsCompounds
Biochemical pathways: part I0466

Protein Targets (1)

Potency Measurements

ProteinTaxonomyMeasurementAverage (µ)Min (ref.)Avg (ref.)Max (ref.)Bioassay(s)
GALC proteinHomo sapiens (human)Potency0.631028.183828.183828.1838AID1159614
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Bioassays (33)

Assay IDTitleYearJournalArticle
AID1347100qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for LAN-5 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347094qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for BT-37 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347093qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SK-N-MC cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347101qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for BT-12 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347091qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SJ-GBM2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347090qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for DAOY cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347106qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for control Hh wild type fibroblast cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1296008Cytotoxic Profiling of Annotated Libraries Using Quantitative High-Throughput Screening2020SLAS discovery : advancing life sciences R & D, 01, Volume: 25, Issue:1
Cytotoxic Profiling of Annotated and Diverse Chemical Libraries Using Quantitative High-Throughput Screening.
AID1347107qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh30 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1508630Primary qHTS for small molecule stabilizers of the endoplasmic reticulum resident proteome: Secreted ER Calcium Modulated Protein (SERCaMP) assay2021Cell reports, 04-27, Volume: 35, Issue:4
A target-agnostic screen identifies approved drugs to stabilize the endoplasmic reticulum-resident proteome.
AID1347102qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh18 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347095qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for NB-EBc1 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347082qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lassa (LASV) Arenavirus: LASV Primary Screen - GLuc reporter signal2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1347083qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lassa (LASV) Arenavirus: Viability assay - alamar blue signal for LASV Primary Screen2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1347425Rhodamine-PBP qHTS Assay for Modulators of WT P53-Induced Phosphatase 1 (WIP1)2019The Journal of biological chemistry, 11-15, Volume: 294, Issue:46
Physiologically relevant orthogonal assays for the discovery of small-molecule modulators of WIP1 phosphatase in high-throughput screens.
AID1347105qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for MG 63 (6-TG R) cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347096qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for U-2 OS cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347424RapidFire Mass Spectrometry qHTS Assay for Modulators of WT P53-Induced Phosphatase 1 (WIP1)2019The Journal of biological chemistry, 11-15, Volume: 294, Issue:46
Physiologically relevant orthogonal assays for the discovery of small-molecule modulators of WIP1 phosphatase in high-throughput screens.
AID1745845Primary qHTS for Inhibitors of ATXN expression
AID1347407qHTS to identify inhibitors of the type 1 interferon - major histocompatibility complex class I in skeletal muscle: primary screen against the NCATS Pharmaceutical Collection2020ACS chemical biology, 07-17, Volume: 15, Issue:7
High-Throughput Screening to Identify Inhibitors of the Type I Interferon-Major Histocompatibility Complex Class I Pathway in Skeletal Muscle.
AID1347092qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for A673 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347089qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for TC32 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID651635Viability Counterscreen for Primary qHTS for Inhibitors of ATXN expression
AID1347108qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh41 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347098qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SK-N-SH cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347097qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Saos-2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347103qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for OHS-50 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347104qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for RD cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347099qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for NB1643 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347154Primary screen GU AMC qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347086qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lymphocytic Choriomeningitis Arenaviruses (LCMV): LCMV Primary Screen - GLuc reporter signal2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1346987P-glycoprotein substrates identified in KB-8-5-11 adenocarcinoma cell line, qHTS therapeutic library screen2019Molecular pharmacology, 11, Volume: 96, Issue:5
A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
AID1346986P-glycoprotein substrates identified in KB-3-1 adenocarcinoma cell line, qHTS therapeutic library screen2019Molecular pharmacology, 11, Volume: 96, Issue:5
A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
[information is prepared from bioassay data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (125)

TimeframeStudies, This Drug (%)All Drugs %
pre-19906 (4.80)18.7374
1990's9 (7.20)18.2507
2000's15 (12.00)29.6817
2010's74 (59.20)24.3611
2020's21 (16.80)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Market Indicators

Research Demand Index: 50.96

According to the monthly volume, diversity, and competition of internet searches for this compound, as well the volume and growth of publications, there is estimated to be very strong demand-to-supply ratio for research on this compound.

MetricThis Compound (vs All)
Research Demand Index50.96 (24.57)
Research Supply Index5.00 (2.92)
Research Growth Index5.21 (4.65)
Search Engine Demand Index79.78 (26.88)
Search Engine Supply Index2.00 (0.95)

This Compound (50.96)

All Compounds (24.57)

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials18 (13.95%)5.53%
Reviews6 (4.65%)6.00%
Case Studies39 (30.23%)4.05%
Observational0 (0.00%)0.25%
Other66 (51.16%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]