Drug intelligence / Profile preview

carglumic acid

Development stage: Approved
Lead developer: Recordati Rare Diseases
Modality: Small Molecules
Administration: Oral

Overview

Carglumic acid is a synthetic structural analog of N-acetylglutamate (NAG) used to treat acute and chronic hyperammonemia in patients with N-acetylglutamate synthase (NAGS) deficiency, as well as for hyperammonemia due to propionic acidemia and methylmalonic acidemia. It acts by replacing the function of NAG as an essential allosteric activator of carbamoyl phosphate synthetase 1 (CPS1), the first enzyme in the urea cycle, thereby facilitating ammonia detoxification. Carglumic acid is administered orally and was approved by the FDA in March 2010. The drug is available under brand names such as Carbaglu and Ucedane.

Brand names: CarbagluUcedane
Other names: (S)-2-ureidopentanedioic acid

Targets

CPS1 (Carbamoyl phosphate synthetase I)

Beyond the preview

Go deeper on carglumic acid.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect source evidence and development news to your research questions.

Publications
News
Analysis

Bring the full picture into focus.

See how Gosset can support your research on carglumic acid.

Explore the full profile