carglumic acid (carbaglu) Report issue

Small molecule Orphan Drug FDA Approved FDA First in Class Priority Review FDA
AACT ChEMBL DrugBank Drug Central Drugs@FDA KEGG MeSH PubChem

Active Ingredient History

NOW
  • Now
Carglumic acid is a Carbamoyl Phosphate Synthetase 1 (CPS 1) allosteric modulator. CPS1 is found in the mitochondria and is the first enzyme of the urea cycle, which converts ammonia into urea. Carglumic acid acts as a replacement for NAG in NAGS deficiency patients by activating CPS1 but it does not help to regulate the urea cycle. Carglumic acid under the trade name Carbaglu indicated as adjunctive therapy for the treatment of acute hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS). In addition, as maintenance therapy for the treatment of chronic hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS). This rare genetic disorder results in elevated blood levels of ammonia, which can eventually cross the blood–brain barrier and cause neurologic problems, cerebral edema, coma, and death.   NCATS

Chemistry

  • SMILES: NC(=O)N[C@@H](CCC(O)=O)C(O)=O
  • InChIKey: LCQLHJZYVOQKHU-VKHMYHEASA-N
  • Mol. Mass: 190.154
  • ALogP: -1.03
  • ChEMBL Molecule:
    carglumic acid  
More Chemistry

Pharmacology

Drug Pricing (per unit)

United States

$148.2793 - $822.3600
More Pricing Detail

Note: This drug pricing data is preliminary, incomplete, and may contain errors.

Alternative names

(2s)-2-(carbamoylamino) pentanedioic acid | (2s)-2-(carbamoylamino)pentanedioic acid | acide carglumique | acido carglumico | acidum carglumicum | carbaglu | carbamino-l-glutamic acid | carbamylglutamic acid | carglumic acid | l-n-carbamoylglutamic acid | n-carbamoyl-l-glutamic acid | n-carbamylglutamate | n-carbamyl-l-glutamate | oe-312 (laboratory-code-designation) | (s)-2-ureidopentanedioic acid | ureidoglutaric acid

Organizations (19)

Research & Development (14)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Marketing (5)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Indications (7)

Approved Indications (1)


 

Hyperammonemia

Experimental Indications - Clinical Trial Phases 1-4 (6)


 

Carbamoyl-Phosphate Synthase I Deficiency Disease (Phase 2)

Deficiency Diseases (Phase 2)

Metabolism, Inborn Errors (Phase 2/Phase 3)

Ornithine Carbamoyltransferase Deficiency Disease (Phase 2)

Propionic Acidemia (Phase 3)

Urea Cycle Disorders, Inborn (Phase 2)

Overview

  • Highest Phase: Phase 3
  • # of Trials: 6
  • # of Trial Organizations: 14

Trials by Phase

Trial Phase Start Date Organizations Indications

Feedback

Data collection and curation is an ongoing process for CDEK - if you notice any information here to be missing or incorrect, please let us know! When possible, please include a source URL (we verify all data prior to inclusion).

Report issue