Nitisinone is a drug used to treat inherited type 1 tyrosinemia (HT-1), which is a condition in which the blood contains too much tyrosine. Nitisinone helps prevent the buildup of tyrosine in the blood.

Inherited type 1 tyrosinemia is caused by a lack of a substance the body needs to break down the amino acid tyrosine. This causes a buildup of tyrosine. HT-1 is commonly found in infants.

Nitisinone works by preventing the breakdown of tyrosine, as well as preventing the formation and accumulation of other toxic substances that can damage the liver, kidneys and nervous system. For optimal results, the use of this drug must also be accompanied by a diet low in protein, tyrosine and phenylalanine.

Please note, nitinone can not cure HT-1. Even so, the use of nitisinone accompanied by dietary adjustments can help slow the progression of the disease.

Nitisinone trademark: Orfadin

What is Nitisinone? 

Group Asam amino inhibitor
Category Prescription drug
Benefit Treating inherited tyrosinemia type 1 (HT-1)
Used by Adults and children
Nitisinone for pregnant and lactating women Category C :  Animal studies have shown an adverse effect on the fetus, but there have been no controlled studies in pregnant women.

Drugs should only be used if the expected benefit outweighs the risk to the fetus.

It is not known whether nitisinone is absorbed into breast milk or not. If you are breastfeeding, do not use this medicine without consulting your doctor first.

Drug form Capsules, tablets and syrup

Warnings Before Taking Nitisinone:

  • Do not take nitsinone if you have a history of allergies to this drug.
  • Tell your doctor if you are pregnant, breastfeeding or planning a pregnancy.
  • Tell your doctor if you have an eye problem, such as cataracts  or a corneal ulcer .
  • Do not consume foods and drinks that contain aspartame, or are high in tyrosine and phenylalanine, while using nitisinone.
  • Do regular health checks if you are taking nitisinone. This aims to detect any side effects that may occur.
  • Tell your doctor if you are taking any other medicines, including herbal medicines and supplements.
  • If a drug allergic reaction or overdose occurs after using nitisinone, see a doctor immediately.

Dosage and Instructions for Use Nitisinone 

The dose of nitisinone given to treat inherited tyrosinemia type 1 will be determined based on the form of the drug, health condition, age, weight (BB), and the patient's response to the drug. Here is the explanation:

Capsules or syrup

  • Adult: 0.5 mg/kg, 2 times a day. The maximum dose is 1-2 mg/kg per day.
  • Children: 0.5 mg/kg, 2 times a day. The maximum dose is 1 mg/kg, 2 times a day.


  • Adult: 0.5 mg/kg, 2 times a day. The maximum dose is 1 mg/kg, 2 times a day.
  • Children: 0.5 mg/kg, 2 times a day. The maximum dose is 1 mg/kg, 2 times a day.

How to Take Nitisinone Properly

Nitisinone should only be given by a doctor . Follow the doctor's recommendations and read the instructions for using the drug before taking nitsinone. Do not change the dosage or time of taking the drug without consulting your doctor first.

Take nitisinone in capsule form on an empty stomach, about 1 hour before eating or 2 hours after eating. For nitsinone tablets and syrup, the drug can be taken with or without food.

Consume nitinone regularly so you can get the maximum benefit from the drug. It is recommended to take nitisinone at the same time every day to help remember the schedule for taking the drug.

If you forget to take nitisinone, take the drug immediately if the break with the next schedule is not too close. If it's close, ignore it and don't double the dose.

Store the drug in a closed place at room temperature. Avoid direct sunlight and keep out of reach of children. Keep in mind, nitinone syrup that has been opened cannot be used again if it has been more than 60 days.

Nitisinone Interactions with Drugs and Other Substances 

When used with aspartame, tyrosine, or phenylalanine, nitisinone can cause interactions in the form of an increased risk of side effects.

Nitisinone Side Effects and Dangers

Taking nitsinone syrup may cause headaches, upset stomach, or diarrhea. Check with your doctor if the complaint does not improve. You also need to immediately check with your doctor if more serious side effects occur after taking nitisinone, such as:

  • Blindness , blurry eyes, red eyes, eyes that are more sensitive to light, or feel pain
  • Nosebleed , bruised, or bleeding gums
  • Passing urine or passing bloody stools
  • Unexplained fatigue and weakness
  • Loss of appetite
  • Chest pain
  • Abdominal pain
  • Hard to breathe
  • Headache
  • Fever
  • seizures

In addition, immediately go to the doctor if you experience a drug allergic reaction after taking nitsinone. Some of the symptoms that can arise are swelling of the eyelids and lips, a rash that is swollen and itchy, and difficulty breathing.

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