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Overview: Carglumic acid is used to treat high ammonia levels in people with certain rare inherited conditions. It works by helping the body break down ammonia, which lowers ammonia levels. Common side effects include stomach pain, vomiting, diarrhea, fever, infections, and headache. Carglumic acid comes as a tablet that must be mixed in water before taking it.

Uses

What is carglumic acid used for?

Carglumic acid is commonly used to manage high ammonia levels (hyperammonemia) in people with certain rare inherited conditions. These conditions include N-acetylglutamate synthase (NAGS) deficiency, propionic acidemia (PA), and methylmalonic acidemia (MMA).

Carglumic acid may also be used for other conditions as determined by your health care provider.

How does carglumic acid work (mechanism of action)?

The urea cycle is an important chemical process that turns ammonia into a substance called urea. Urea is then cleared from the body in the urine (pee). In people with certain inherited disorders, the urea cycle does not work the way that it should, leading to high ammonia levels. 

Carglumic acid works by activating an enzyme that helps start the urea cycle. This helps to break down ammonia so that it can be cleared from the body, which reduces ammonia levels.

How is carglumic acid supplied (dosage forms)?

Brands and other names

  • Carbaglu

Dosage forms and strengths

  • 200 mg tablets for oral suspension

How should I store carglumic acid?

Carglumic acid should be stored in a refrigerator, between 36 F and 46 F (2 C and 8 C), in the original, unopened bottle. Once the bottle is opened, it should be stored at room temperature, between 59 F and 56 F (15 C and 30 C), for up to one month. Keep the bottle tightly closed when it is not in use to protect the medicine from moisture. Throw away the bottle and any remaining medicine after one month.

Side Effects

What are the most common side effects of carglumic acid?

The most common side effects of carglumic acid are listed below. These side effects may vary depending on the condition that is being treated. Tell your health care provider if you have any of these side effects that bother you.

  • Stomach pain, vomiting, or diarrhea
  • Fever
  • Swollen tonsils (tonsillitis)
  • Low red blood cell or hemoglobin levels (anemia)
  • Low levels of a type of white blood cell (neutropenia)
  • Infections, including ear infection, infection in the nose or throat, sore throat, or common cold symptoms
  • Headache
  • Changes in salt or electrolyte levels 
  • Low appetite
  • Low blood sugar
  • Tiredness or lack of energy
  • Change in brain function (encephalopathy)
  • Inflammation of the pancreas (pancreatitis)

There may be other side effects of carglumic acid that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of carglumic acid?

While less common, the most serious side effects of carglumic acid are described below, along with what to do if they happen.

Severe Allergic Reactions. Carglumic acid may cause allergic reactions, which can be serious. Stop taking carglumic acid and get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching, skin rash, or pale red bumps on the skin called hives
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Warnings & Precautions

Who should not use carglumic acid?

Allergies to Ingredients. People who are allergic to any of the following should not take carglumic acid.

  • Carbaglu
  • Carglumic acid
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in the specific carglumic acid products they stock.

What should I know about carglumic acid before using it?

Do not take carglumic acid unless it has been prescribed to you by a health care provider. Take it as prescribed. 

Do not share carglumic acid with other people, even if they have the same condition as you. It may harm them.

Keep carglumic acid out of the reach of children. Since the unopened bottle should be kept in a refrigerator, take special precautions to keep it away from children who also use the refrigerator.

Take carglumic acid right before a meal.

Carglumic acid tablets must be mixed in water before taking them. Do not swallow the tablets whole or crush them. Your medicine will come with “Instructions for Use.” Read these instructions carefully and ask your pharmacist or other health care provider if you are unsure how to prepare and take carglumic acid.

Depending on your dose, you may need to cut the carglumic acid tablet. Each tablet has lines that allow it to be cut into four equal parts. Ask your health care provider if you are unsure how to cut the tablet to get the right dose.

Keep all appointments, including for blood work, while you are taking carglumic acid.

What should I tell my health care provider before using carglumic acid?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them determine if carglumic acid is right for you.

In particular, make sure that you discuss any of the following.

Kidney Problems. Let your health care provider know if you have any kidney problems or have had kidney problems in the past.

Pregnancy. It is not known if or how carglumic acid could affect pregnancy or harm an unborn baby. Tell your health care provider if you are pregnant or plan to become pregnant. Your health care provider will let you know if you should take carglumic acid while you are pregnant or trying to get pregnant. If you become pregnant while taking carglumic acid, tell your health care provider. They can sign you up for a registry that collects safety information about the use of carglumic acid during pregnancy. 

Breastfeeding. It is not known if carglumic acid passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed. Your health care provider will let you know if you should take carglumic acid while breastfeeding.

Interactions

Who should not use carglumic acid?

Allergies to Ingredients. People who are allergic to any of the following should not take carglumic acid.

  • Carbaglu
  • Carglumic acid
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in the specific carglumic acid products they stock.

What should I know about carglumic acid before using it?

Do not take carglumic acid unless it has been prescribed to you by a health care provider. Take it as prescribed. 

Do not share carglumic acid with other people, even if they have the same condition as you. It may harm them.

Keep carglumic acid out of the reach of children. Since the unopened bottle should be kept in a refrigerator, take special precautions to keep it away from children who also use the refrigerator.

Take carglumic acid right before a meal.

Carglumic acid tablets must be mixed in water before taking them. Do not swallow the tablets whole or crush them. Your medicine will come with “Instructions for Use.” Read these instructions carefully and ask your pharmacist or other health care provider if you are unsure how to prepare and take carglumic acid.

Depending on your dose, you may need to cut the carglumic acid tablet. Each tablet has lines that allow it to be cut into four equal parts. Ask your health care provider if you are unsure how to cut the tablet to get the right dose.

Keep all appointments, including for blood work, while you are taking carglumic acid.

What should I tell my health care provider before using carglumic acid?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them determine if carglumic acid is right for you.

In particular, make sure that you discuss any of the following.

Kidney Problems. Let your health care provider know if you have any kidney problems or have had kidney problems in the past.

Pregnancy. It is not known if or how carglumic acid could affect pregnancy or harm an unborn baby. Tell your health care provider if you are pregnant or plan to become pregnant. Your health care provider will let you know if you should take carglumic acid while you are pregnant or trying to get pregnant. If you become pregnant while taking carglumic acid, tell your health care provider. They can sign you up for a registry that collects safety information about the use of carglumic acid during pregnancy. 

Breastfeeding. It is not known if carglumic acid passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed. Your health care provider will let you know if you should take carglumic acid while breastfeeding.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much carglumic acid?

If you or someone else has used too much carglumic acid, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of carglumic acid?

If you miss a dose, take it as soon as you remember. If it is almost time for your next dose, skip the missed dose and only take the next dose. Do not take double or extra doses.