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Drugs reference index «nitisinone»


nitisinone (Oral route)


Commonly used brand name(s):

In the U.S.

  • Orfadin

Available Dosage Forms:

  • Capsule

Therapeutic Class: Gastrointestinal Agent

Uses For nitisinone

Nitisinone is given along with a special diet to treat hereditary tyrosinemia, type 1. This disease is caused by too much tyrosine in the blood. It may cause damage to the liver, kidneys, eyes, skin, and nervous system. Treatment with nitisinone and diet may slow the disease, but it will not cure it.

nitisinone is available only with your or your child's doctor's prescription.

Before Using nitisinone

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 nitisinone, the following should be considered:


Tell your doctor if you have ever had any unusual or allergic reaction to nitisinone 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.


nitisinone has been tested in children and, in effective doses, has not been shown to cause specific problems.


Many medicines have not been studied specifically in older people. Therefore, it may not be known whether they work exactly the same way they do in younger adults or if they cause different side effects or problems in older people. There is no specific information comparing use of nitisinone in the elderly with use in other age groups.


Pregnancy CategoryExplanation
All TrimestersCAnimal studies have shown an adverse effect and there are no adequate studies in pregnant women OR no animal studies have been conducted and there are no adequate studies in pregnant women.

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.

Interactions with 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.

Interactions with Food/Tobacco/Alcohol

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 nitisinone

To use:

  • It is not known how nitisinone reacts with food. It is best to take it at least 1 hour before a meal.
  • For small children, you may open the capsule and put the medicine in a small amount of water, formula, or applesauce. Give the medicine as soon as it is mixed.


The dose of nitisinone 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 nitisinone. 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 (capsules):
    • For hereditary tyrosinemia, type 1:
      • Adults—The dose is based on body weight and will be determined by your doctor. Your doctor may increase the dose as needed.
      • Children—The dose is based on body weight and will be determined by your child's doctor. Your child's doctor may increase the dose as needed.

Missed Dose

If you miss a dose of nitisinone, 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.


Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.

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.

Precautions While Using nitisinone

While taking nitisinone,it is important that you or your child maintain a diet with restricted amounts of tyrosine and phenylalanine A nutritionist may be able to help you with the special diet needed to treat you. A nutritionist that has special training with children may help with a diet for your child.

Call your doctor right away for any redness, swelling, or burning of your or your child's eyes, an unusual rash, bleeding, or if your or your child's skin is yellow.

It is very important that the doctor check you or your child at regular visits to see how the medicine is working and increase the dose if needed. The doctor may test your your child's blood often.

A special examination of your or your child's eyes should be done before nitisinone is started.

Wearing sunglasses that block ultraviolet light is recommended. Nitisinone may cause sensitivity of the eyes to the sunlight.

nitisinone Side Effects

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
  • Bloated abdomen
  • dark-colored urine
  • dull, achy upper abdominal pain
  • general feeling of tiredness or weakness
  • headache
  • light-colored stools
  • loss of appetite
  • unexplained weight loss
  • vomiting
  • yellow eyes or skin
Less common
  • Black, tarry stools
  • blindness
  • blood in urine or stools
  • blisters on skin
  • bloody nose
  • blurred vision
  • change in color vision
  • chest pain or discomfort
  • chills
  • cough
  • darkening of urine
  • decreased vision
  • difficulty seeing at night
  • dry or itching eyes
  • dry skin
  • excessive tearing from eyes
  • eye pain
  • fever
  • fluid-filled skin blisters
  • general feeling of discomfort or illness
  • increased sensitivity of eyes to sunlight
  • irritation or inflammation of the eye
  • itching of the skin
  • painful or difficult urination
  • pinpoint red spots on skin
  • rash with flat lesions or small raised lesions on the skin
  • red, thickened, or scaly skin
  • redness, pain, swelling of eye, eyelid, or inner lining of eyelid burning
  • sensitivity to the sun
  • shortness of breath
  • skin thinness
  • sore throat
  • sores, ulcers, or white spots on lips or in mouth
  • swollen and/or painful glands
  • unusual bleeding or bruising
  • unexplained nosebleed
  • Agitation
  • anxiety
  • back pain
  • bloody stools
  • bluish color of fingernails, lips, skin, palms, or nail beds
  • change in personality
  • change in vision
  • cold sweats
  • coma
  • confusion
  • cool, pale skin
  • cough producing mucus
  • decreased urination
  • diarrhea
  • difficulty breathing
  • dizziness
  • drowsiness
  • dry mouth
  • earache
  • fainting
  • fast heartbeat
  • feeling full in upper abdomen
  • increase in heart rate
  • increase in body movements
  • increased hunger
  • infection
  • irregular, fast or slow, or shallow breathing
  • irritability
  • lightheadedness
  • mood or mental changes
  • nausea
  • nervousness
  • pain or swelling in arms or legs without any injury
  • pale skin
  • problems with walking or talking
  • rapid breathing
  • redness or swelling in ear
  • seeing things that are not there
  • shakiness
  • skin rash found mostly on mucous membranes such as eyes and mouth
  • stiff neck
  • sunken eyes
  • thirst
  • tightness in chest
  • vomiting of blood or material that looks like coffee grounds
  • wheezing
  • wrinkled skin

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:

Less common or rare
  • Absent, missed, or irregular menstrual periods
  • burning feeling in chest or stomach
  • hair loss
  • indigestion
  • sleepiness
  • stomach upset
  • stopping of menstrual bleeding
  • tenderness in stomach area
  • thinning of hair
  • tooth discoloration

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.

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  • nitisinone Concise Consumer Information (Cerner Multum)
  • Nitisinone MedFacts Consumer Leaflet (Wolters Kluwer)
  • Nitisinone Professional Patient Advice (Wolters Kluwer)
  • Orfadin Prescribing Information (FDA)

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