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Nitisinone
  • Treatments
  • Hereditary Tyrosinemia Type 1

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 Sep 9, 2020. 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.

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 ...