Carglumic acid for the treatment of hyperammonemia.

Carglumic acid is an orphan drug that helps in converting ammonia into urea in patients with a rare genetic disorder caused by NAGS (N-acetylglutamate synthase) deficiency. It is used as adjunctive therapy for the treatment of acute and maintenance therapy of chronic hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS).

Carglumic acid Dose in Adults

For the treatment of Acute hyperammonemia:

100 to 250 mg/kg/day given orally in two or four divided doses.

For the treatment of Chronic hyperammonemia:

100 mg/kg/day or less based on the plasma ammonia levels given orally in two or four divided doses.

Carglumic acid Dose in Childrens

For the treatment of Acute hyperammonemia:

Infants, Children, and Adolescents:
- 100 to 250 mg/kg/day given orally in two or four divided doses.

Use in treatment of Chronic hyperammonemia:

Infants, Children, and Adolescents:
- 100 mg/kg/day or less given orally in two or four divided doses.

Pregnancy Risk Factor C

It has not been tested in pregnant women. It should be continued throughout pregnancy.

Carglumic acid use during breastfeeding:

It is unknown if breastmilk can be excreted. It is not advised to breastfeed during maternal therapy with carglumic.

Carglumic acid Dose in Renal Disease:

It has not been studied in patients with renal disease.
The manufacturer has not recommended any dose adjustment in patients with renal impairment.

Carglumic acid Dose in Liver Disease:

It has not been studied in patients with liver disease.
The manufacturer has not recommended any dose adjustment in patients with hepatic impairment.

Common Side Effects Of Carglumic Acid Include:

Central nervous system:
- Headache
Gastrointestinal:
- Vomiting
- Abdominal pain
- Diarrhea
Hematologic & oncologic:
- Anemia
- Decreased hemoglobin
Infection:
- Infection
Otic:
- Otic infection
Respiratory:
- Tonsillitis
- Nasopharyngitis
Miscellaneous:
- Fever

Less Common Side Effects Of Carglumic Acid Include:

Central nervous system:
- Drowsiness
Dermatologic:
- Hyperhidrosis
- Skin rash
Endocrine & metabolic:
- Weight loss
Gastrointestinal:
- Anorexia
- Dysgeusia
Infection:
- Influenza
Neuromuscular & skeletal:
- Weakness
Respiratory:
- Pneumonia

Contraindication to Carglumic Acid include:

Allergy reactions to any component of the drug or the drug itself
Breastfeeding

Warnings and Precautions

An experienced physician
- Acute symptomatic hyperammonemia can be life-threatening and should be treated with carglumic Acid by an experienced physician.
Management of nutrition:
- To reverse protein catabolism, it is important to eat a lot of protein during acute episodes.

Monitor:

Plasma ammonia
Clinical features of hyperammonemia like lethargy, ataxia, confusion, vomiting, seizures, and impaired memory.

How to administer carglumic acid:

It should be administered immediately before meals.
The drug should be dispersed in water before the administration and should not be mixed with any other foods or liquids (other than water).
The oral tablets should not be crushed or swallowed whole.
It should be dispersed in water and administered immediately. Do not dissolve the drug completely in water.
When it is administered via a syringe or a nasogastric tube, the required volume should be drawn and administered.
An extra volume of 1-2 ml water (if given via a syringe) or more (if administered via a nasogastric tube) should be then administered

Mechanism of action of Carglumic Acid (carbaglu):

Carglumic acid is used as a substitute for patients suffering from N-acetylglutamate deficiency (NAG).
N-acetylglutamate synase (NAGS), a mitochondrial enzyme, produces N-acetylglutamate.
It is responsible for converting ammonia to urea in step one of the urea cycle.

It isMetabolizedThe intestinal flora converts carbon dioxide to carbon dioxide.

It has been ahalf-life eliminationThis ranges between 4.3 and 9.5 hours. Time to get therepeak plasma concentrationIt takes approximately 3 hours. It is excreted mainly via feces.