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Nitisinone (By mouth)

Nitisinone (nye-TIS-i-none)

Treats a hereditary condition called tyrosinemia.

Brand Name(s):

Nityr, Orfadin

There may be other brand names for this medicine.

When This Medicine Should Not Be Used:

This medicine is not right for everyone. Do not use it if you had an allergic reaction to nitisinone.

How to Use This Medicine:

Capsule, Liquid, Tablet

  • 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 contents into a small amount of water, formula, or applesauce. Stir the mixture well and swallow it without chewing.
  • Oral liquid:
    • Allow the bottle to warm to room temperature for 30 to 60 minutes.
    • When used the first time, shake 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 adapter.
    • Shake the bottle for at least 5 seconds before measuring each dose to remove any particles at the bottom of the bottle.
  • Tablet:
    • 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 1 teaspoon of applesauce. If more than 1 tablet is needed, repeat the previous steps. Mix well.
      • Swallow this mixture right away. Add about 1 more teaspoon of applesauce to the same container, mix, and swallow right away to get the full dose of this medicine.
      • 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 2 tablets in the oral syringe. Add 5 mL of room temperature water.
      • Cap the syringe and let it sit for at least 60 minutes.
      • Turn the syringe up and down for at least 30 seconds.
      • Check the suspension in the syringe. The tablet should have broken down into small pieces and are evenly spread in the water. Take this mixture right away.
      • If the tablet has not fully dissolved, let it sit for another 10 minutes. Then, turn the syringe up and down for at least 30 seconds. Do not take this medicine if the tablet has still not completely dissolved.
      • If you can not take the mixture right away, you may store it at room temperature in the capped oral syringe, protected from direct sunlight for up to 24 hours.
      • 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.
  • 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: Store the medicine in the refrigerator.
    • 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.
    • Tablets: Store the medicine in its original container at room temperature. Keep the tablets and the medicine prepared in an oral syringe and with applesauce away from direct light. Throw away any mixture that is not used within 2 hours after mixing with applesauce or within 24 hours after mixing in the oral syringe.

Drugs and Foods to Avoid:

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

  • Some medicines can affect how nitisinone works. Tell your doctor if you are using adefovir, celecoxib, ganciclovir, methotrexate, phenytoin, tolbutamide, or warfarin.

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
  • Blurred vision or changes in vision, difficulty seeing at night, increased sensitivity of the eyes to sunlight, pain, redness, swelling, or burning of the eyes
  • Fever, chills, cough, sore throat, body aches
  • Problems in intelligence, short-term memory, learning ability, and attention
  • Raised or thickened skin areas on the palms of your hands or soles of your feet
  • Unusual bleeding, bruising, or weakness, nosebleed

If you notice these less serious side effects, talk with your doctor:

  • Diarrhea, stomach upset
  • Headache

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: 5/1/2020

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