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.

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  • Orfadin
  • More common
    Bloated abdomen
    dark-colored urine
    dull, achy upper abdominal pain
    general feeling of tiredness or weakness
    headache
    light-colored stools
    loss of appetite
    unexplained weight loss
    vomiting
    yellow eyes or skin
    Less common
    Black, tarry stools
    blindness
    blood in urine or stools
    blisters on skin
    bloody nose
    blurred vision
    change in color vision
    chest pain or discomfort
    chills
    cough
    darkening of urine
    decreased vision
    difficulty seeing at night
    dry or itching eyes
    dry skin
    excessive tearing from eyes
    eye pain
    fever
    fluid-filled skin blisters
    general feeling of discomfort or illness
    increased sensitivity of eyes to sunlight
    irritation or inflammation of the eye
    itching of the skin
    painful or difficult urination
    pinpoint red spots on skin
    rash with flat lesions or small raised lesions on the skin
    red, thickened, or scaly skin
    redness, pain, swelling of eye, eyelid, or inner lining of eyelid burning
    sensitivity to the sun
    shortness of breath
    skin thinness
    sore throat
    sores, ulcers, or white spots on lips or in mouth
    swollen and/or painful glands
    unusual bleeding or bruising
    unexplained nosebleed
    Rare
    Agitation
    anxiety
    back pain
    bloody stools
    bluish color of fingernails, lips, skin, palms, or nail beds
    change in personality
    change in vision
    cold sweats
    coma
    confusion
    cool, pale skin
    cough producing mucus
    decreased urination
    diarrhea
    difficulty breathing
    dizziness
    drowsiness
    dry mouth
    earache
    fainting
    fast heartbeat
    feeling full in upper abdomen
    increase in heart rate
    increase in body movements
    increased hunger
    infection
    irregular, fast or slow, or shallow breathing
    irritability
    lightheadedness
    mood or mental changes
    nausea
    nervousness
    pain or swelling in arms or legs without any injury
    pale skin
    problems with walking or talking
    rapid breathing
    redness or swelling in ear
    seeing things that are not there
    shakiness
    skin rash found mostly on mucous membranes such as eyes and mouth
    stiff neck
    sunken eyes
    thirst
    tightness in chest
    vomiting of blood or material that looks like coffee grounds
    wheezing
    wrinkled skin
    Some nitisinone side effects may not need any medical attention. As your body gets used to the medicine these side effects may disappear. Your health care professional may be able to help you prevent or reduce these side effects, but do check with them if any of the following side effects continue, or if you are concerned about them:

    Less common or rare
    Absent, missed, or irregular menstrual periods
    burning feeling in chest or stomach
    hair loss
    indigestion
    sleepiness
    stomach upset
    stopping of menstrual bleeding
    tenderness in stomach area
    thinning of hair
    tooth discoloration
  • (nitisinone) capsule