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Nitisinone

Generic name: nitisinone [ nye-TIS-i-none ]
Brand names: Nityr, Orfadin
Dosage forms: oral capsule (10 mg; 2 mg; 20 mg; 5 mg), oral suspension (4 mg/mL), oral tablet (10 mg; 2 mg; 5 mg)
Drug class: Miscellaneous metabolic agents

Medically reviewed by Drugs.com on Aug 30, 2023. Written by Cerner Multum.

What is nitisinone?

Nitisinone works by preventing the body from breaking down an amino acid called tyrosine and by keeping other toxic substances from building up and causing harm to your liver or kidneys.

Nitisinone is used to treat a rare genetic condition called hereditary tyrosinemia type 1 (HT-1). HT-1 is a metabolic disorder that occurs when the body does not produce enough of an enzyme that breaks down proteins from certain foods. This condition occurs most often in young babies.

Nitisinone may also be used for purposes not listed in this medication guide.

Nitisinone side effects

Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Some side effects may result from not properly following your diet plan and consuming restricted foods or beverages.

Nitisinone may cause serious side effects. Call your doctor at once if you have:

Common side effects of nitisinone may include:

The liquid form of nitisinone may contain glycerol and can cause headache, upset stomach, or diarrhea.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Warnings

Call your doctor at once if you have vision problems, eye pain, eye redness or burning, easy bruising, unusual bleeding, itching, or jaundice (yellowing of the skin or eyes). Tell your doctor if your baby has a sudden change in behavior, ability, or development (sitting up, crawling, walking, talking, etc).

Before taking this medicine

Tell your doctor if you have any medical conditions affecting your eyes.

Tell your doctor if you are pregnant or breastfeeding.

How should I take nitisinone?

Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose. Use the medicine exactly as directed.

Do not change your medication dose or schedule without your doctor's advice.

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

You may take nitisinone liquid with or without food.

You must avoid certain foods for your treatment to be effective. Follow the diet plan created for you by your doctor or nutrition counselor.

High protein foods are high in tyrosine and phenylalanine (including artificial sweeteners such as aspartame, Equal, or Nutrasweet). Since babies and children need protein to grow and develop, special foods have been developed to replace the high protein foods.

Shake the oral suspension (liquid) for at least 5 full seconds just before you measure a dose. Use the dosing syringe provided, or use a medicine dose-measuring device (not a kitchen spoon).

If you cannot swallow a capsule whole, open it and sprinkle the medicine into a spoonful of water, baby formula, or applesauce. Swallow the mixture right away without chewing. Do not save it for later use.

If you cannot swallow a tablet whole, crush the tablet and sprinkle the medicine into a spoonful of applesauce. Swallow the mixture right away without chewing. Do not save it for later use.

You may also dissolve a tablet in an oral syringe with water. Read and carefully follow any Instructions for Use provided with your medicine. Ask your doctor or pharmacist if you do not understand these instructions.

You may need frequent blood tests. Your vision may also need to be checked.

Store tablets at room temperature away from moisture, heat, and light. Keep the bottle tightly closed when not in use.

Store the capsules or liquid in the refrigerator, do not freeze.

After using nitisinone liquid for the first time, you may store the medicine at room temperature for up to 60 days.

What happens if I miss a dose?

Take the medicine as soon as you can, but skip the missed dose if it is almost time for your next dose. Do not take two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while taking nitisinone?

While you are taking nitisinone, you must not eat foods that are high in tyrosine or phenylalanine, including:

There are other foods that may contain tyrosine or phenylalanine. Get familiar with the list of foods you must avoid to help control your condition.

What other drugs will affect nitisinone?

Other drugs may affect nitisinone, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.