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Nitisinone (Oral route)



Brand Names:

  • Orfadin

Dosage Forms:

  • Capsule



Gastrointestinal Agent

Uses of This Medicine:

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.

This medicine is available only with your or your child's 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:


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.


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

Older adults—

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.


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. When you are taking this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

  • Azilsartan
  • Celecoxib
  • Cyclophosphamide
  • Diclofenac
  • Fluvastatin
  • Fosphenytoin
  • Glipizide
  • Ibuprofen
  • Irbesartan
  • Losartan
  • Meloxicam
  • Naproxen
  • Nateglinide
  • Ospemifene
  • Phenytoin
  • Piroxicam
  • Rosiglitazone
  • Sulfamethoxazole
  • Tamoxifen
  • Tolbutamide
  • Torsemide
  • Voriconazole
  • Warfarin
  • Zileuton

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:

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 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 (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 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.


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 This Medicine:

While taking this medicine,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 this medicine is started.

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

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
Bloated abdomen
dark-colored urine
dull, achy upper abdominal pain
general feeling of tiredness or weakness
light-colored stools
loss of appetite
unexplained weight loss
yellow eyes or skin
Less common
Black, tarry stools
blood in urine or stools
blisters on skin
bloody nose
blurred vision
change in color vision
chest pain or discomfort
darkening of urine
decreased vision
difficulty seeing at night
dry or itching eyes
dry skin
excessive tearing from eyes
eye pain
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
back pain
bloody stools
bluish color of fingernails, lips, skin, palms, or nail beds
change in personality
change in vision
cold sweats
cool, pale skin
cough producing mucus
decreased urination
difficulty breathing
dry mouth
fast heartbeat
feeling full in upper abdomen
increase in heart rate
increase in body movements
increased hunger
irregular, fast or slow, or shallow breathing
mood or mental changes
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
skin rash found mostly on mucous membranes such as eyes and mouth
stiff neck
sunken eyes
tightness in chest
vomiting of blood or material that looks like coffee grounds
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
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.

Last Updated: 11/4/2014

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