Carglumic Acid
Carglumic acid is a synthetic analogue of N-acetylglutamate (NAG) used to treat hyperammonemia caused by N-acetylglutamate synthase (NAGS) deficiency. It works by activating carbamoyl phosphate synthetase I (CPS I), a key enzyme in the urea cycle that helps the body eliminate ammonia. This medication is typically prescribed for patients with rare genetic disorders affecting urea synthesis.
Carglumic acid treats hyperammonemia in NAGS deficiency by activating the urea cycle enzyme CPS I.
Key facts at a glance
- ATC
- A16AA05
- RxNorm
- 401713
- Generic
- Carglumic Acid
- Class
- atc-a16
- Brands
- Carbaglu
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- tablet
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Hyperammonemia caused by N-acetylglutamate synthase (NAGS) deficiency, a rare genetic condition where the body cannot properly process ammonia due to insufficient N-acetylglutamate production.
Carglumic acid is a structural analogue of N-acetylglutamate (NAG) that acts as an essential allosteric activator of carbamoyl phosphate synthetase I (CPS I), enabling the urea cycle to function properly and eliminate ammonia from the body.
This is a specialty medication for a rare metabolic disorder. Dosing is weight-based and should be monitored by a metabolic specialist. May be used short-term or long-term depending on the underlying condition. Regular monitoring of ammonia levels is required.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy