carglumic acid

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carglumic acid

(car-gloo-mik as-id) ,


(trade name)


Therapeutic: hyperammonemia treatments
Pharmacologic: enzyme activators
Pregnancy Category: C


Treatment (acute/maintenance) of hyperammonemia due to deficiency of hepatic enzyme N-acetylglutamate synthase (NAGS). During acute hyperammonemia, additional treatments should be used including alternate pathway medications, hemodialysis, and protein restriction.


Serves as an analogue of N-acetyl glutamate (NAG), the natural activator of cabamoyl phosphate synthetase 1 (CPS 1) in the liver. CPS 1 helps to convert ammonia into urea. Carglumic acid replaces NAG in NAGS deficiency patients by activating CPS 1.

Therapeutic effects

↓ ammonia levels.


Absorption: Some absorption follows oral administration; bioavailability unknown.
Distribution: .
Metabolism and Excretion: Some metabolism by intestinal bacteria; 9% excreted unchanged in urine, 60% excreted unchanged in feces. Metabolic end product (CO2) eliminated via lungs.
Half-life: 5.6 hr.

Time/action profile (↓ ammonia levels)

POwithin 24 hr3 daysunknown


Contraindicated in: Lactation: Breastfeeding not recommended.
Use Cautiously in: Obstetric: Due to risk of neurologic complications in NAGS patients, carglumic acid must be used throughout pregnancy.

Adverse Reactions/Side Effects

Central nervous system

  • headache (most frequent)
  • drowsiness
  • weakness

Ear, Eye, Nose, Throat

  • nasopharyngitis (most frequent)


  • abdominal pain (most frequent)
  • diarrhea (most frequent)
  • vomiting (most frequent)
  • abnormal taste


  • rash


  • anemia (most frequent)


  • ↓ weight


  • infections (most frequent)
  • fever (most frequent)


Drug-Drug interaction

None noted.


Oral (Adults) 100–250 mg/kg/day initially, given in 2–4 divided doses immediately before meals/feedings, adjusted to maintain normal plasma ammonia levels based on age. Dose should be rounded to the nearest 100 mg.
Oral (Children) 100–250 mg/kg/day initially, given in 2–4 divided doses immediately before meals/feedings.


Tablets: 200 mg

Nursing implications

Nursing assessment

  • Assess for hyperammonemia (neurological status, plasma ammonia levels) during therapy.
  • Lab Test Considerations: Monitor plasma ammonia levels periodically during therapy. Maintain within normal range for age via individual dose adjustment.
    • May cause ↓ hemoglobin.

Potential Nursing Diagnoses

Activity intolerance


  • Concommitant administration of other ammonia-lowering therapies is recommended.
  • Oral: Disperse each 250 mg tablet in at least 2.5 mL of water immediately before use and take immediately; do not crush or swallow whole. Tablets may not dissolve completely in water; undissolved particles may remain in mixing container. Rinse mixing container with additional volumes of water to ensure complete delivery of dose; swallow immediately. Administer via oral syringe or nasogastric tube.
    • Refrigerate before opening. After first opening of container, do not refrigerate. Keep container tightly closed to protect from moisture. Write date of opening on tablet container; discard 1 mo after first opening. Do not use after expiration date stated on container.

Patient/Family Teaching

  • Instruct patient to take carglumic acid as directed.
  • Advise patient that during acute hyperammonemic episodes, protein restriction and hypercaloric intake is recommended to block ammonia generating catabolic pathways. When plasma ammonia levels have normalized, protein intake can usually be ↑ with goal of unrestricted protein intake.
  • Advise female patients to notify health care professional if pregnancy is planned or suspected or if breastfeeding Due to risk of neurologic complications in NAGS patients, carglumic acid must be used throughout pregnancy.

Evaluation/Desired Outcomes

  • Normalization of plasma ammonia levels.
References in periodicals archive ?
Role of carglumic acid in the treatment of acute hyperammonemia due to N-acetylglutamate synthase deficiency.
announces the successful development of a new, fast dissolving formulation of Carglumic acid tablets stable at room temperature.
Carglumic acid is a key therapeutic tool in the management of rare, life threatening inborn metabolic disorders affecting the urea cycle; as such it was a natural candidate for the Company's products portfolio.
also announces the filing of a patent application claiming new fast dissolving and stable tablet formulations of Carglumic acid, together with the proprietary technology developed to improve the product stability, without modifying the pharmaceutical form the patients are familiar to.
Carglumic acid formulation is the fourth Dipharma product approaching the market: Diterin (sapropterin dihydrochloride 100 mg tabs) has already been approved in South Korea and Russia for the treatment of hyperphenylalaninemia (HPA) due to phenylketonuria (PKU), Miglustat (miglustat 100 mg caps) was submitted in the USA through an abbreviated new drug application (ANDA) in 2016 for the treatment of Gaucher disease and Disanit[R] (nitisinone capsules) was submitted in EU in the last quarter of 2016 for the treatment of hereditary tyrosinemia type I.
The three endocrine products are carglumic acid (Carbaglu; C) for treatment of hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS); tesamorelin (Egrifta; X) a growth hormone--releasing factor analog for reduction of excess abdominal fat in HIV-infected patients; and velaglucerase alfa (VPRIV; B) given for long-term enzyme replacement in patients with Gaucher disease.