Carglumic Acid (Oral)

Carglumic acid is a form of medication which is used to lower the amount of ammonia in the blood for people suffering from hyperammonemia.


Carglumic acid is a form of medication which is used to treat patients with hyperammonemia; a condition which arises when there is an excess of ammonia in the bloodstream. There is a particular enzyme which is usually produced by the liver. This enzyme is essential to the body as it is responsible for processing any excess amounts of nitrogen created during the digestion process. Nitrogen is created during the process used to metabolize any proteins which have been ingested. If the enzyme is not present, or is not present in significant enough quantities, nitrogen starts to build up in the body. This nitrogen takes the form of ammonia and is difficult for the body to excrete once it has been generated in this way.

Ammonia is an incredibly toxic substance when introduced into the bloodstream. It can be transported throughout the body very quickly and can begin to affect the function of many different organs. If left untreated, this ammonia can infiltrate bodily tissue and can lead to permanent and irreversible brain damage, can place patients into a coma and can even lead to death in extreme cases.

Carglumic acid is a man-made version of this enzyme. It acts as a substitute for the enzyme which is ordinarily produced in the liver of healthy people. It helps the body to break down nitrogen so that it does not build up in the form of ammonia.

Carglumic acid is not a cure for this condition. It will not stimulate the body to begin its own production of the enzyme, but instead it provides an artificial solution. For this reason, patients are often prescribed this medication as a long-term treatment option, and it is used in conjunction with other medications to manage this disorder potentially for the rest of a patient's life.

Conditions Treated

  • Hyperammonemia (excess ammonia in the blood)

Type of Medicine

  • Man-made enzyme

Side Effects

When prescribing a form of medication, doctors and pharmacists will weigh up the health benefits of a certain medication against any potential side effects it might cause. They will make a decision based on what they believe to be in the patient's best interests. In some cases, doctors will prescribe medications known to cause common side effects, because the benefits to the patient's health outweigh the adverse implications of any side effects.

Patients should take any professional medical advice seriously but should arrange to speak to their doctor or pharmacist if they have any concerns about side effects.

Carglumic acid does not provoke side effects in all patients. Of the side effects which are reported, the most common are as follows:

  • Vomiting
  • Nausea
  • Diarrhea
  • Abdominal cramps
  • Increased body temperature
  • Fever
  • Chills
  • Shivering
  • Sweating more than usual
  • Coughing
  • Sore throat
  • Hoarse/Lost voice
  • Tonsillitis
  • Unexplained weight loss
  • Loss of appetite
  • Headaches
  • Aches or pain in the joints, muscles or lower back
  • Difficulty passing urine
  • Unusually pale skin
  • Difficulty sleeping
  • Unexplained bruising
  • Unexplained tiredness or fatigue

If patients experience one or more of the symptoms above and believe that they might be attributed to carglumic acid, they should contact their doctor. There may be something which can be done to either limit the extent to which the side effects are experienced or to treat these new symptoms with a different form of medication.

In some cases, doctors may be able to lower the dose of carglumic acid prescribed. In some patients this could continue to treat the hyperammonemia whilst reducing the amount and severity of the side effects experienced. Doctors may not be able to alter the dose but will be able to discuss ways in which to manage or treat any side effects.

It is possible to consume too much carglumic acid, and the symptoms experienced in the case of an overdose will differ from the side effects associated with safe doses. Patients could seek urgent medical attention if they experience any of the following symptoms after taking significant doses of carglumic acid:

  • Pounding heartbeat
  • Irregular pulse
  • Large increase in the amount of mucus secreted from the lungs
  • Severe restlessness and agitation

Any combination of the symptoms above could be an indicator of an overdose of carglumic acid. Patients who develop any of these conditions whilst taking carglumic acid should seek immediate medical attention and should make sure that the medical professionals attending them are aware of the medication that they are taking.


Clinical experience has suggested that carglumic acid is safe to prescribe to both adults and children. It may even be used in the case of newborn babies. It is recommended to take the drug before eating food, or in the case of babies before they are fed.

The daily dose prescribed is usually calculated based on a patient's wait, and normally starts at an initial dose of 100mg/kg, but could be increased to anything up to 250mg/kg if necessary. Control of the condition may be established at even lower doses, and in these cases it would not be necessary to increase the initial dose.

Test doses are recommended in order to assess the responsiveness of the patient to this particular drug. Test doses should be administered, and then the ammonia plasma concentration should be measured regularly in order to monitor levels of ammonia in the blood. In most patients, these levels normalize just a few hours after the first dose is taken.

It is possible to expose the body to too much carglumic acid. Symptoms of overdoses of carglumic acid include:

  • Tachycardia
  • Excess sweating
  • Increase in body temperature
  • Fluid on the lungs
  • Agitation and restlessness


Whenever two or more types of medication are prescribed to be taken at the same time, there will always be a risk of contraindications between the different pharmaceuticals. Interactions between different drugs can affect the way in which the chemicals in the drugs function within the body, which can lead to one, both or all of the drugs working less effectively in tackling their respective medical condition. In other cases, the combination of two or more different types of medication can increase the possibility and/or severity of side effects, or lead to the patient developing completely new medical conditions with potentially harmful repercussions.

Not enough studies have been conducted with carglumic acid to prove any interactions with other pharmaceuticals. This does not, however, mean that this medication never interacts with other drugs. It is therefore even more important than usual for patients to keep a fully comprehensive and detailed account of all of the different medications and dietary supplements which they are taking at any one time. If a patient does start to feel unwell or starts to notice changes in the way in which their medication is functioning and they are taking carglumic acid in conjunction with other medications, they should consult with a healthcare professional as soon as possible. At this consultation it is important for the patient to bring a full list of all of their current and recent forms of medication in order to rule out any interactions between carglumic acid and other drugs.

It is not just prescription drugs which can interact with one another. Drugs which are purchased over the counter, or illegally, can also have a harmful effect when combined with prescription drugs. It is vital that patients keep a list of all of the pharmaceuticals they are taking, not just the prescription medication.


There has been very little research conducted into the long-term use of carglumic acid, as it is not a particularly common drug to prescribe. It is generally regarded as being safe and placing a minimal strain on the body; however, the effects of this medication long term have not been proven either way. It is therefore recommended that patients taking carglumic acid at regular intervals over a prolonged period of time should attend regular health checkups. The condition of liver and kidney function as well as the condition of the heart should be monitored to ensure no adverse long-term effects. Regular blood tests are also recommended as a precaution against other potential hematological problems.

No specific studies have been conducted into the safety of humans taking carglumic acid whilst pregnant. Studies in pregnant animals taking carglumic acid have shown a minor developmental toxicity associated with the drug. If you are pregnant, or plan on becoming pregnant, and have been prescribed carglumic acid, talk to your doctor about the specific details of your condition. There have been no links made between the consumption of carglumic acid and effects on fertility.

Similarly, only animals have been tested with regards to carglumic acid and breastfeeding. In the animal studies, small amounts of carglumic acid were present in the milk passed onto the young. This suggests that the same might happen in human cases, but it has yet to be proven. It is recommended not to breastfeed whilst taking carglumic acid as the effects it could have are unknown. If you are breastfeeding or plan to breastfeed whilst taking carglumic acid, speak to your doctor first to assess the benefits and risks involved.


As with all forms of medication, carglumic acid should be kept in the container in which it was supplied, with a tight seal kept at all times. It must be clearly labeled so as not to be confused with other medications or substances. Carglumic acid should be stored in a safe place, out of the reach of pets and children.

It should be kept away from extreme changes in temperature, should be kept out of direct sunlight and should not be stored near moisture. It is therefore not recommended to store this type of medication in kitchens or bathrooms. Carglumic acid should not be refrigerated.

Once the container has been opened, the medication inside is safe to use for 3 months. If not all tablets have been consumed after this date, patients are recommended to dispose of the tablets safely and gain a new prescription for the medication. The tablets should not be thrown into household garbage or disposed of down the toilet; instead, they should be returned to the doctor or pharmacist as part of a medicine 'take back program'. Medical professionals will then be able to ensure that the tablets are safely disposed of without risking harm to others.


Carglumic acid is an artificially constructed form of an enzyme which is usually found in the human body. Patients who suffer from hyperammonemia are usually missing this enzyme as their liver is unable to produce it naturally. This causes a dangerous buildup of nitrogen and, subsequently, ammonia in the bloodstream as the body is unable to break it down by itself. This condition can have seriously harmful effects on the body, as the bloodstream essentially begins to poison different sections of bodily tissue as the ammonia is transported around the body. If hyperammonemia is not diagnosed and treated, it can lead to severe and permanent brain damage, can place patients in a coma and can even be fatal if left completely untreated.

Carglumic acid is an artificial form of the enzyme required to break down nitrogen. It is prescribed in the form of tablets and provides the body with the ability to break down nitrogen in a way which will not cause harm to the rest of the body. It usually forms a significant part of a daily treatment plan which will enable patients with hyperammonemia to control and manage their condition throughout their life.

Last Reviewed:
December 22, 2017
Last Updated:
April 27, 2018