General Information

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

• N-acetylglutamate synthase (NAGS) deficiency. Those with this lifelong disease lack a liver enzyme called NAGS. It normally helps break down ammonia. If the enzyme is not existing, it builds up in the blood and ammonia cannot be broken down;
• Some organic acidemias (isovaleric, propionic, and methylmalonic acidemia) where patients lack certain enzymes involved in protein metabolism

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

How does Carbaglu (Carglumic Acid) work?

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

 

Why use Carbaglu (Carglumic Acid)?

Carbaglu is a medication that contains Carglumic Acid as an active ingredient. This is available as dispersible tablets which mean that this can be dispersed or mixed in water. Carbaglu tablets for oral suspension have 200mg of Carglumic Acid. The Food and Drug Administration (FDA) approved this treatment.

How to use Carbaglu (Carglumic Acid)?

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

The initial daily dose for this medication should be 100mg per kilogram body weight, but up until 250mg/kg can be used if needed. The dose may be adjusted to maintain normal blood ammonia levels. The tablets should be dispersed in a small amount of water. Tablets can be easily be broken into two equal halves.

What are the side-effects of Carbaglu (Carglumic Acid)?

• Headache
• Vomiting
• Diarrhea
• Stomach pain
• Stuffy nose
• Ear infection
• Changes in sense of taste
• Fever
• Infection
• Flu symptoms
• Weight loss
• Sleepiness
• Skin rash

Call your doctor right away if these happen:

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

Warnings and Precautions

• Let your doctor know if you have any medical history, current treatments or any allergies.
• If you are pregnant or breastfeeding women, consult your doctor before taking this medication.
• Keep this away from the reach of children.