Pronunciation:

kar-GLOO-mik AS-id

Brand Names:

• Carbaglu

Dosage Forms:

• Tablet for Suspension

Uses of This Medicine:

Carglumic acid is used together with diet and other medicines to treat and prevent hyperammonemia (too much ammonia in the blood) caused by the lack of a liver enzyme called N-acetylglutamate synthase (NAGS). It is also used together with diet and other medicines to prevent hyperammonemia caused by certain genetic disorders called propionic acidemia (PA) or methylmalonic acidemia (MMA).

Ammonia is formed in the body when we digest protein. A person without the NAGS enzyme cannot process ammonia properly, and too much ammonia will collect in the blood. This will cause serious unwanted effects. Carglumic acid acts by replacing the NAGS enzyme and helps reduce the amount of ammonia in the blood.

This medicine is available only with your doctor's prescription.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of carglumic acid in children. Safety and efficacy have been established.

Older adults—

Appropriate studies have not been performed on the relationship of age to the effects of carglumic acid in the geriatric population. Safety and efficacy have not been established.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Proper Use of This Medicine:

Take this medicine exactly as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered. To do so may increase the chance of side effects.

It is important that you and your child follow any special diet instructions from your doctor, such as using a low-protein diet. If you have any questions about this, check with your doctor.

Do not swallow the tablet whole. Do not break, crush, or chew it. Also, it is best to take this medicine right away before meals or feedings.

To use:

• Dissolve the tablet in a small container or medicine cup with at least 2.5 milliliters (mL) or one-half teaspoonful of water. Shake the container gently to dissolve the tablet.
• Drink the mixture right away.
• Some small particles or specks may be left at the bottom of the container.
• Rinse the mixing container with water and drink the water right away to make sure you take all of the medicine.

To use with a nasogastric tube (NGT):

• Dissolve the tablet in a small container or medicine cup with at least 2.5 milliliters (mL) or one-half teaspoonful of water. Shake the container gently to dissolve the tablet.
• Pour the mixture into the tube right away.
• Some small particles or specks may be left at the bottom of the container or in the tubing.
• Rinse the mixing container with water and pour the water into the tube right away. This will clear the particles from the tube and make sure you have given all of the medicine.

To use with an oral syringe:

• Dissolve the tablet in a small container or medicine cup with at least 2.5 milliliters (mL) or one-half teaspoonful of water. Shake the container gently to dissolve the tablet.
• Use an oral syringe to measure the correct amount of mixture for the dose and give it right away. Throw away any unused medicine in the container.
• Some small particles or specks may be left in the oral syringe.
• Refill the oral syringe with 1 to 2 mL of water and give the water right away. This will clear the particles from the syringe and make sure you have given all of the medicine.

Dosing—

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• For oral dosage form (tablets):
  • For hyperammonemia caused by NAGS deficiency:
    • Adults and children—Dose is based on body weight and must be determined by your doctor. The starting dose is usually 100 to 250 milligrams (mg) per kilogram (kg) of body weight per day. The total dose will be divided and given into 2 to 4 doses per day. Your doctor may adjust your dose to 10 mg/kg to 100 mg/kg divided into 2 to 4 doses per day.
  • For hyperammonemia caused by PA or MMA:
    • Adults and children—Dose is based on body weight or body surface area and must be determined by your doctor.
      • Weighing more than 15 kilograms (kg)—3.3 grams (g) per square meter (m2), divided and given into 2 equal doses (12 hours apart) for at least 7 days.
      • Weighing less than or equal to 15 kg—150 milligrams (mg) per kilogram (kg) per day, divided and given into 2 equal doses (12 hours apart) for at least 7 days.

Missed dose—

If you miss a dose of this medicine, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

Storage—

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Before you open the bottle, store it in the refrigerator. Do not freeze.

After you open the bottle for the first time, store it at room temperature. Keep the bottle tightly closed, and away from heat, moisture, and direct light. Do not refrigerate the bottle. Write the date you used the first tablet on the bottle. Throw away any unused medicine 30 days after the date on the bottle.

Precautions While Using This Medicine:

It is very important that your doctor check your or your child's progress at regular visits to make sure that this medicine is working properly. Blood tests may be needed to check for unwanted effects.

Hyperammonemia (too much ammonia in the blood) is a life-threatening condition that requires immediate medical attention. Check with your doctor right away if you or your child have nausea or vomiting that continues, an increase in the frequency of seizures, loss of appetite, swelling of the face, tiredness and weakness, or yellow eyes or skin.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

Black, tarry stools

chills

cough

decreased weight

diarrhea

fever

general feeling of discomfort or illness

headache

hoarseness

increased sweating

joint pain

loss of appetite

lower back or side pain

muscle aches and pains

nausea

painful or difficult urination

pale skin

runny nose

shivering

sore throat

sweating

trouble sleeping

trouble breathing

ulcers, sores, or white spots in the mouth

unusual bleeding or bruising

unusual tiredness or weakness

vomiting

Less common

Agitation

anxiety

bloating

blurred vision

chest discomfort or pain

cold sweats

coma

confusion

cool, pale skin

darkened urine

decreased appetite

decreased awareness or responsiveness

depression

dizziness

drowsiness

faintness

hallucinations

indigestion

irritability

mood or mental changes

nervousness

nightmares

numbness, tingling, pain, or weakness in the hands or feet

pains in the stomach, side, or abdomen, possibly radiating to the back

seizures

severe sleepiness

shakiness

slurred speech

stiff neck

swelling of the feet or lower legs

trembling

weakness and heaviness of the legs

yellow eyes or skin

Incidence not known

Actions that are out of control

itching, rash

reddened skin

talking, feeling, and acting with excitement

Get emergency help immediately if any of the following symptoms of overdose occur:

Symptoms of overdose

Fast, pounding, or irregular heartbeat or pulse

increase in mucous from the lungs

restlessness

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Change in hearing

change in taste

congestion

ear drainage

earache or pain in the ear

lack or loss of strength

loss of taste

sleepiness or unusual drowsiness

stomach pain

stuffy or runny nose

swollen glands

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.