Description and Brand Names

Drug information provided by: Micromedex

US Brand Name

  1. Orfadin


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.

This product is available in the following dosage forms:

  • Capsule

Before Using

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.


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.


Information about this nitisinone-oral-route
Pregnancy Category Explanation
All Trimesters C Animal 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.

Drug Interactions

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.

  • Cyclophosphamide

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

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.


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

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

  1. Bloated abdomen
  2. dark-colored urine
  3. dull, achy upper abdominal pain
  4. general feeling of tiredness or weakness
  5. headache
  6. light-colored stools
  7. loss of appetite
  8. unexplained weight loss
  9. vomiting
  10. yellow eyes or skin

Less common

  1. Black, tarry stools
  1. blindness
  2. blood in urine or stools
  3. blisters on skin
  4. bloody nose
  5. blurred vision
  6. change in color vision
  7. chest pain or discomfort
  8. chills
  9. cough
  10. darkening of urine
  11. decreased vision
  12. difficulty seeing at night
  13. dry or itching eyes
  14. dry skin
  15. excessive tearing from eyes
  16. eye pain
  17. fever
  18. fluid-filled skin blisters
  19. general feeling of discomfort or illness
  20. increased sensitivity of eyes to sunlight
  21. irritation or inflammation of the eye
  22. itching of the skin
  23. painful or difficult urination
  24. pinpoint red spots on skin
  25. rash with flat lesions or small raised lesions on the skin
  26. red, thickened, or scaly skin
  27. redness, pain, swelling of eye, eyelid, or inner lining of eyelid burning
  28. sensitivity to the sun
  29. shortness of breath
  30. skin thinness
  31. sore throat
  32. sores, ulcers, or white spots on lips or in mouth
  33. swollen and/or painful glands
  34. unusual bleeding or bruising
  35. unexplained nosebleed


  1. Agitation
  2. anxiety
  3. back pain
  4. bloody stools
  5. bluish color of fingernails, lips, skin, palms, or nail beds
  6. change in personality
  7. change in vision
  8. cold sweats
  9. coma
  10. confusion
  11. cool, pale skin
  12. cough producing mucus
  13. decreased urination
  14. diarrhea
  15. difficulty breathing
  16. dizziness
  17. drowsiness
  18. dry mouth
  19. earache
  20. fainting
  21. fast heartbeat
  22. feeling full in upper abdomen
  23. increase in heart rate
  24. increase in body movements
  25. increased hunger
  26. infection
  27. irregular, fast or slow, or shallow breathing
  28. irritability
  29. lightheadedness
  30. mood or mental changes
  31. nausea
  32. nervousness
  33. pain or swelling in arms or legs without any injury
  34. pale skin
  35. problems with walking or talking
  36. rapid breathing
  37. redness or swelling in ear
  38. seeing things that are not there
  39. shakiness
  40. skin rash found mostly on mucous membranes such as eyes and mouth
  41. stiff neck
  42. sunken eyes
  43. thirst
  44. tightness in chest
  45. vomiting of blood or material that looks like coffee grounds
  46. wheezing
  47. 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

  1. Absent, missed, or irregular menstrual periods
  2. burning feeling in chest or stomach
  3. hair loss
  4. indigestion
  5. sleepiness
  6. stomach upset
  7. stopping of menstrual bleeding
  8. tenderness in stomach area
  9. thinning of hair
  10. 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.