Carglumic Acid


Where is Carglumic Acid used?

Carglumic Acid is indicated for the treatment of hyperammonaemia (high blood levels of ammonia). It occurs in patients with the following metabolic diseases:

  • Particular organic acidemias (propionic, isovaleric, and methylmalonic acidemia) where patients don’t have enough certain enzymes involved in protein metabolism.
  • N-acetylglutamate synthase (NAGS) deficiency. People with this illness lack a liver enzyme called NAGS. It usually helps break down ammonia. If the enzyme does not exist, it builds up in the blood and ammonia cannot be broken down.

This medication was designated as an ‘orphan medicine’ because this is used in rare diseases. The number of patients with this condition is low, they are considered ‘rare’.

Carglumic Acid is available as dispersible tablets which means that this can be dispersed or mixed in water. This is for oral suspension that has 200 mg of Carglumic Acid. The Food and Drug Administration (FDA) approved this treatment.

How does Carglumic Acid work?

It is toxic in the body especially the brain when ammonia builds up in the blood. Carglumic Acid is very similar in structure to N-acetylglutamate, which activates an enzyme that breaks down ammonia. This medicine, as a result, helps break down ammonia. It also reduces ammonia blood levels and its toxic effects.

How is Carglumic Acid taken?

This treatment should be started by a doctor who has experience in treating such metabolic diseases.

The first daily dosage for this medication must be 100 mg per kilogram body weight, but up until 250 mg/kg could be used if necessary. The dose can be adjusted to keep normal blood ammonia levels. The medicine must be dispersed in a small amount of water. Tablets can be easily be broken into two halves.

What are the possible side effects of Carglumic Acid?

  • Headache
  • Skin rash
  • Vomiting
  • Diarrhea
  • Weight loss
  • Stomach pain
  • Fever
  • Flu symptoms
  • Stuffy nose
  • Sleepiness

Call your doctor right away if these happen:

  • Low red blood cells (anemia) –unusual tiredness, pale skin, shortness of breath, cold hands and feet
  • Signs of an ear infection –ear pain, fever, full feeling, drainage from the ear, or trouble hearing
  • Low white blood cell counts –cough, fever, sore throat, skin sores, trouble breathing, or mouth sores

What are the precautions in taking Carglumic Acid?

  • Let your doctor know if you have any medical history, current treatments, or any allergies.
  • If you are a pregnant or breastfeeding woman, consult your doctor before taking this medication.
  • Your doctor must check your progress at regular visits to verify that this medication is functioning properly. Blood tests may be needed to check for unwanted effects.
  • Prevent from eating foods that are high in protein when you start taking Carglumic Acid. Follow the instructions of your doctor about any other restrictions on beverages, food, or activity. If you skip a meal, do not take your dose of this medicine. Wait until your next meal.
  • Your blood will need to be tested frequently. A build-up of ammonia in the blood may rapidly cause brain injury or death. Each person with a urea cycle disorder must stay under the care of a doctor.