General Information

Nitisinone is used to treat a certain inherited disorder called hereditary tyrosinemia type 1 (HT-1). It is usually discovered in infants and requires lifelong treatment. HT-1 is caused by a shortage of a certain natural substance that is needed to break down a nutrient found in food. This effect causes a build-up of too much tyrosine. It may also cause a related substance in the liver. Nitisinone is used along with a diet low in protein, tyrosine, and phenylalanine.

How does Nitisinone work?

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

Why use Nitisinone?

Nitisinone is an inhibitor of the tyrosine catabolism. This helps prevent some substances from forming that may cause damage to your nervous system. Nitisinone occurs as white to yellowish-white crystalline powder. This is supplied as a tablet that contains Nitisinone as an active ingredient. Each tablet is available in the strengths of 2 mg, 5 mg, and 10 mg. The Food and Drug Administration (FDA) has already approved this medication.

How to use Nitisinone?

Nitisinone is to be taken by mouth as directed. This is usually taken twice a day. You may take each dose with or without a meal. Swallow the tablet as a whole with a full glass of water. Do not break or chew it. Don’t forget to take it at the same time each day.

The recommended dosage is based on your medical condition, age, and response to it. Do not take more or less than the prescription. You must take this drug exactly as it is given. Let your doctor know if your condition does not improve or if it gets worse.

What are the side effects of Nitisinone?

  • Hair loss
  • Skin redness and peeling in large areas
  • Nosebleed
  • Itching
  • A red rash
  • Eye pain
  • Eye sensitivity to light
  • Clouding of the cornea

If these side effects persist or worsen, call your doctor right away.

Warnings and Precautions

  • Do not use this drug if you are allergic to it. Call your doctor if there are any signs of an allergic reaction occur.
  • Tell your doctor if you have a medical history of eye problems. Tell as well if you have current medications (vitamins or herbal supplements).
  • Consult your doctor if you are pregnant or breastfeeding women.
  • Keep this at room temperature far from moisture or heat.