Health Guide

Nitisinone (Oral route)



Brand Names:

  • Nityr
  • Orfadin

Dosage Forms:

  • Tablet
  • Capsule
  • Suspension



Gastrointestinal Agent

Uses of This Medicine:

Nitisinone is used to treat hereditary tyrosinemia type 1, which is too much tyrosine in the blood. It helps to prevent high levels of tyrosine in the blood. Treatment with nitisinone and diet changes may slow the progression of the disease, but will not cure it.

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:


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.


Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of nitisinone in children.

Older adults—

Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of nitisinone in the elderly. However, elderly patients are more likely to have age-related liver, kidney, or heart problems, which may require caution in the dose for patients receiving nitisinone tablets.


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.

  • 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.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

  • Conjunctivitis (eye inflammation) or
  • Eye pain or
  • Leukopenia (low white blood cells) or
  • Photophobia (eye sensitivity to light) or
  • Thrombocytopenia (low platelets in the blood)—Use with caution. May make these conditions worse.

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.

Carefully follow your doctor's instructions about a special diet.

This medicine should come with a Medication Guide. Read and follow these instructions carefully. Ask your doctor if you have any questions.

To use the capsule:

  • Take the capsule on an empty stomach at least 1 hour before or 2 hours after a meal.
  • If needed, 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.

To use the suspension:

  • Take the suspension with or without food.
  • Allow the bottle to warm to room temperature for 30 to 60 minutes.
  • Shake the bottle for at least 20 seconds until the solid cake at the bottom of the bottle is completely dissolved.
  • Insert the bottle adaptor and measure the medicine with an oral syringe.
  • Shake the bottle for at least 5 seconds before measuring each dose to remove any particles at the bottom of the bottle.

To use the tablet:

  • Take it with or without food.
  • If you cannot swallow the tablet, you can dissolve it in water and take the solution using an oral syringe. It can also be crushed and mixed with applesauce. Do not mix it with any other food or liquid.
    • To prepare for an applesauce mixture:
      • Clean your hands well before preparing the medicine.
      • Place the tablet onto a teaspoon and a second teaspoon on top of the tablet. Press and rotate the second teaspoon until the tablet is crushed into a fine powder. Crush only 1 tablet at a time.
      • Carefully place the powder into the applesauce. If more than 1 tablet is needed, repeat the previous steps. Mix well.
      • Swallow this mixture right away.
      • If you can not take the mixture right away, you may store it within 2 hours after adding applesauce.
    • To prepare for an oral syringe:
      • Clean your hands well before preparing the medicine.
      • Use only 1 or 2 tablets in an oral syringe at a time.
      • For 1 tablet: Place the tablet in the oral syringe. Add 2.6 milliliters (mL) of room temperature water and pull the plunger back to the 3 mL mark. For 2 tablets: Place the tablets in the oral syringe. Add 5 mL of room temperature water.
      • Turn the syringe up and down for at least 1 minute. Let it sit for at least 20 minutes.
      • Turn it up and down again for at least 1 minute. Let it sit for another 30 minutes, then turn it up and down again for at least 1 minute.
      • Check the mixture in the syringe. The tablet should have broken down into small pieces and are evenly spread in the water.
      • Use the mixture within 2 hours after adding water.
      • To make sure the full dose is given, refill the syringe with water. Use 2 mL of water and shake the syringe well for 10 seconds, then give the mixture.


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 forms (capsules, suspension, or tablets):
    • For hereditary tyrosinemia type 1:
      • Adults—Dose is based on body weight and must be determined by your doctor. The starting dose is usually 0.5 milligram (mg) per kilogram (kg) of body weight 2 times a day. Your doctor may adjust the dose as needed. However, the dose is usually not more than 1 mg per kg of body weight 2 times a day.
      • Children—Dose is based on body weight and must be determined by your doctor. The starting dose is usually 0.5 mg per kg of body weight 2 times a day. Your doctor may adjust the dose as needed. However, the dose is usually not more than 1 mg per kg of body weight 2 times a day.

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.


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.

Store in the refrigerator. Do not freeze.

You may also store the capsules at room temperature for up to 45 days. Throw away any unused capsules after 45 days.

Once opened, you may store the bottle of oral liquid at room temperature for up to 60 days. Throw away any unused medicine after 60 days.

Store the tablets in its original container at room temperature. Keep the tablets and the medicine prepared in an oral syringe and with the applesauce away from direct light. Throw away any mixture that is not used within 2 hours after mixing.

Precautions While Using This Medicine:

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

Check with your doctor right away if blurred vision, difficulty seeing at night, or any other vision change occurs during or after treatment. Your doctor may want your eyes be checked by an ophthalmologist (eye doctor).

Nitisinone can temporarily lower the number of white blood cells in your blood, increasing the chance of getting an infection. It can also lower the number of platelets, which are necessary for proper blood clotting. Avoid people with infections and check with your doctor if you have symptoms of an infection, such as a fever or chills, a cough or hoarseness, lower back or side pain, or painful or difficult urination. Call your doctor if you have any unusual bleeding or bruising, black, tarry stools, blood in the urine or stools, or pinpoint red spots on your skin.

The oral suspension contains glycerol and may cause diarrhea, an upset stomach, or headaches. Let your doctor know if you have these symptoms.

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:

Less common
Black, tarry stools
bleeding gums
blood in the urine or stools
bloody nose
blurred vision
burning, dry, or itching eyes
change in color vision
chest pain or discomfort
decreased vision
difficulty seeing at night
excessive eye tearing
eye redness, irritation, or pain
increased sensitivity of the eyes to sunlight
painful or difficult urination
pinpoint red spots on the skin
redness, pain, swelling of the eye, eyelid, or inner lining of the eyelid
sore throat
sores, ulcers, or white spots on the lips or in the mouth
swollen or painful glands
unusual bleeding or bruising
unusual tiredness or weakness
back pain
bluish color of the fingernails, lips, skin, palms, or nail beds
cough with mucus
difficulty with breathing
stiff neck
tightness in the chest

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
Dry skin
hair loss or thinning
itching or skin rash
red, swollen skin
scaly skin
Abdominal or stomach pain
cold sweats
cool, pale skin
fast heartbeat
increased hunger
slurred speech

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: 9/4/2017
The information provided herein should not be used during any medical emergency or for the diagnosis or treatment of any medical condition. A licensed medical professional should be consulted for diagnosis and treatment of any and all medical conditions. Call 911 for all medical emergencies. Links to other sites are provided for information only -- they do not constitute endorsements of those other sites.

All rights reserved