Drug

Carglumic acid

Established medicine

ClassCarbamoyl-phosphate synthase [ammonia], mitochondrial positive allosteric modulatorResearchOngoing clinical research

Also known as (2S)-2-(carbamoylamino)pentanedioic acid, (S)-2-ureidopentanedioic acid, Acide carglumique, Acidum carglumicum+10 more

(2S)-2-(carbamoylamino)pentanedioic acid, (S)-2-ureidopentanedioic acid, Acide carglumique, Acidum carglumicum, Carbaglu, Carbamino-L-glutamic acid, Carbamylglutamic acid, L-N-Carbamoylglutamic acid, N-Carbamoyl-L-Glutamic Acid, N-Carbamyl-L-glutamate, N-carbamyl L-glutamic acid, Ureidoglutaric acid, ácido carglúmico, Carglutamic acid.

RxNorm401713 ChEMBLCHEMBL1201780 ATCA16AA05UNII5L0HB4V1EW

CPS1

Primary target

Regulatory events

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).

Clinical trial2025-03-11Completed · ClinicalTrials.gov

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

Clinical trial2021-02-15Results posted · ClinicalTrials.gov

Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carbaglu® in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)

Clinical trial2019-03-19Completed · ClinicalTrials.gov

Approval: Ucedane (EMA)

Regulatory2017-06-23EMA

Approval: Carbaglu (EMA)

Regulatory2003-01-24EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Carglumic acid

Aliases & brands

(2S)-2-(carbamoylamino)pentanedioic acid(S)-2-ureidopentanedioic acidAcide carglumiqueAcidum carglumicumCarbagluCarbamino-L-glutamic acidCarbamylglutamic acidL-N-Carbamoylglutamic acidN-Carbamoyl-L-Glutamic AcidN-Carbamyl-L-glutamateN-carbamyl L-glutamic acidUreidoglutaric acidácido carglúmicoCarglutamic acid

RxNorm CUI

401713

ChEMBL ID

CHEMBL1201780

ATC codes

A16AA05

UNII

5L0HB4V1EW

Primary mechanism

Carbamoyl-phosphate synthase [ammonia], mitochondrial positive allosteric modulator

Regulatory jurisdictions

ema

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

Carbamoyl-phosphate synthase [ammonia], mitochondrial

CPS1 · P31327

POSITIVE ALLOSTERIC MODULATORCarbamoyl-phosphate synthase [ammonia], mitochondrial positive allosteric modulator

Regulatory timeline

2 events

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval

2003-01-24

Latest approval

2017-06-23

Authorities

EMA

Total events

emaEuropean Medicines Agency· 2 events

2017-06-23Approval

Approval: Ucedane (EMA)

Indication: Ucedane is indicated in treatment of: hyperammonaemia due to N-acetylglutamate synthase primary deficiency; Hyperammonaemia due to isovaleric acidaemia; Hyperammonaemia due to… Show full indication

Ucedane is indicated in treatment of: hyperammonaemia due to N-acetylglutamate synthase primary deficiency; Hyperammonaemia due to isovaleric acidaemia; Hyperammonaemia due to methymalonic acidaemia; Hyperammonaemia due to propionic acidaemia.

Evidence ↗

2003-01-24Approval

Approval: Carbaglu (EMA)

Indication: Carbaglu is indicated in treatment of: hyperammonaemia due to N-acetylglutamate-synthase primary deficiency; hyperammonaemia due to isovaleric acidaemia; hyperammonaemia due to… Show full indication

Carbaglu is indicated in treatment of: hyperammonaemia due to N-acetylglutamate-synthase primary deficiency; hyperammonaemia due to isovaleric acidaemia; hyperammonaemia due to methymalonic acidaemia; hyperammonaemia due to propionic acidaemia.

Evidence ↗