Health Guide

Nitisinone (By mouth)

Nitisinone (nye-TIS-i-none)

Treats a hereditary condition called tyrosinemia.

Brand Name(s):


There may be other brand names for this medicine.

When This Medicine Should Not Be Used:

Most people are able to use this medicine without serious problems. Talk to your doctor if you have any concerns.

How to Use This Medicine:

Capsule, Liquid

  • Take your medicine as directed. Your dose may need to be changed several times to find what works best for you.
  • Carefully follow your doctor's instructions about a special diet.
  • Capsule:
    • Take this medicine on an empty stomach, at least 1 hour before or 2 hours after a meal.
    • If you cannot swallow the capsule, you may open it and pour the medicine into a small amount of water, formula, or applesauce. Stir the mixture well and swallow it without chewing.
  • Oral liquid:
    • Take this medicine with or without food.
    • Allow the bottle to warm to room temperature for 30 to 60 minutes.
    • When used the first time, sake the bottle for at least 20 seconds until the solid cake at the bottom of the bottle is completely dissolved.
    • Measure the medicine with an oral syringe and use the bottle adaptor.
    • Shake the bottle for at least 5 seconds before measuring each dose to remove any particles at the bottom of the bottle.
  • Read and follow the patient instructions that come with this medicine. Talk to your doctor or pharmacist if you have any questions.
  • Missed dose: Take a dose as soon as you remember. If it is almost time for your next dose, wait until then and take a regular dose. Do not take extra medicine to make up for a missed dose.
    • Capsules: You may also store the capsules at room temperature for up to 45 days. Throw away any unused capsules after 45 days.
    • Oral liquid: Store the medicine in the refrigerator. Do not freeze. Once opened, you may store it at room temperature for up to 60 days. Throw away any unused medicine after 60 days.

Drugs and Foods to Avoid:

Ask your doctor or pharmacist before using any other medicine, including over-the-counter medicines, vitamins, and herbal products.

Warnings While Using This Medicine:

  • Tell your doctor if you are pregnant or breastfeeding, or if you have liver problems or eye problems.
  • This medicine may make you bleed, bruise, or get infections more easily. Take precautions to prevent illness and injury. Wash your hands often.
  • Your doctor will do lab tests at regular visits to check on the effects of this medicine. Keep all appointments. You may also need to have regular eye examinations.
  • Keep all medicine out of the reach of children. Never share your medicine with anyone.

Possible Side Effects While Using This Medicine:

Call your doctor right away if you notice any of these side effects:

  • Allergic reaction: Itching or hives, swelling in your face or hands, swelling or tingling in your mouth or throat, chest tightness, trouble breathing
  • Fever, chills, cough, sore throat, body aches
  • Problems with vision, or being more sensitive to light
  • Raised or thickened skin areas on the palms of your hands or soles of your feet
  • Unusual bleeding, bruising, or weakness

If you notice other side effects that you think are caused by this medicine, tell your doctor

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

Last Updated: 7/4/2017
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