Carglumic Acid

Summary

Carglumic Acid (CHEMBL1201780) is an approved small-molecule orphan drug (ATC A16AA05); indicated across 5 conditions including propionic acidemia and inborn disorder of amino acid metabolism.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A16AA05
• Indications: 5 conditions
• Clinical trials: 4
• Chemistry: 190.15 Da · C6H10N2O5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1201780
Name	Carglumic Acid
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	121396
ChEBI	CHEBI:71028
ATC	A16AA05
Molecular formula	C6H10N2O5
Molecular weight	190.15
InChIKey	LCQLHJZYVOQKHU-VKHMYHEASA-N

SMILES: C(CC(=O)O)[C@@H](C(=O)O)NC(=O)N

IUPAC name: (2S)-2-(carbamoylamino)pentanedioic acid

ChEBI definition: 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.

Pharmacological roles (ChEBI): orphan drug, carbamylphosphate synthetase I activator.

Also known as: Acide carglumique, Acido carglumico, Carbaglu, Carglumic acid, N-carbamyl-l-glutamic acid, NSC-760124, OE 312, OE 312 (LABORATORY CODE DESIGNATION), OE-312, Ucedane, CARGLUMIC ACID, SID144206097

Patent coverage: 479 distinct patent families (994 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

5 indications (4 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
propionic acidemia	4	MONDO:0011628	MONDO:0011628
inborn disorder of amino acid metabolism	4	MONDO:0004736	MONDO:0004736
inborn errors of metabolism	2	MONDO:0019052	MONDO:0019052

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 4.

Phase distribution

Phase	Trials
Not specified	2
PHASE3	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02426775	PHASE3	COMPLETED	Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
NCT01599286	PHASE2	COMPLETED	Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
NCT04176523	Not specified	RECRUITING	Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
NCT05040178	Not specified	RECRUITING	An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).