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Generic Name: nitisinone (nye TIS i none)
Brand Name: Orfadin
Physician reviewed Orfadin patient information - includes Orfadin description, dosage and directions.
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Drug Information:
Orfadin 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. Orfadin 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. Orfadin may also be used for purposes not listed in this medication guide. Learn more

Orfadin Side Effects

Orfadin Side Effects

Note: This document contains side effect information about nitisinone. Some of the dosage forms listed on this page may not apply to the brand name Orfadin.

For the Consumer

Applies to nitisinone: oral capsule, oral suspension, oral tablet

Along with its needed effects, nitisinone (the active ingredient contained in Orfadin) may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur while taking nitisinone:

Less common

  • Black, tarry stools
  • bleeding gums
  • blindness
  • blood in the urine or stools
  • bloody nose
  • blurred vision
  • burning, dry, or itching eyes
  • change in color vision
  • chest pain or discomfort
  • chills
  • cough
  • decreased vision
  • difficulty seeing at night
  • excessive eye tearing
  • eye redness, irritation, or pain
  • fever
  • increased sensitivity of the eyes to sunlight
  • painful or difficult urination
  • pinpoint red spots on the skin
  • redness, pain, swelling of the eye, eyelid, or inner lining of the eyelid
  • sore throat
  • sores, ulcers, or white spots on the lips or in the mouth
  • swollen or painful glands
  • unusual bleeding or bruising
  • unusual tiredness or weakness


  • Agitation
  • back pain
  • bluish color of the fingernails, lips, skin, palms, or nail beds
  • confusion
  • cough with mucus
  • difficulty with breathing
  • dizziness
  • drowsiness
  • hallucinations
  • headache
  • irritability
  • seizures
  • stiff neck
  • tightness in the chest
  • vomiting

Some side effects of nitisinone may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

Less common

  • Dry skin
  • hair loss or thinning
  • itching or skin rash
  • red, swollen skin
  • scaly skin


  • Cold sweats
  • cool, pale skin
  • depression
  • fast heartbeat
  • headache
  • increased hunger
  • nausea
  • nightmares
  • seizures
  • shakiness
  • slurred speech
  • stomach pain

For Healthcare Professionals

Applies to nitisinone: oral capsule, oral suspension, oral tablet


The more commonly reported side effects have included elevated tyrosine levels, thrombocytopenia, leukopenia, conjunctivitis, corneal opacity, keratitis, photophobia, eye pain, blepharitis, cataracts, granulocytopenia, epistaxis, pruritus, exfoliative dermatitis, dry skin, maculopapular rash, and alopecia.


Common (1% to 10%): Liver failure (7%), porphyria (1%)

Frequency not reported: Hepatic function disorder, elevated hepatic enzymes, liver enlargement


Common (1% to 10%): Thrombocytopenia (3%), leucopenia (3%), granulocytopenia (1%), epistaxis (1%)

Uncommon (0.1% to 1%): Anemia


Very common (10% or more): Elevated tyrosine levels (10% or greater)

Uncommon (0.1% to 1%): Dehydration, hypoglycemia

Nervous system

Frequency not reported: Seizures, brain tumor, encephalopathy, headache, hyperkinesia, nervousness, somnolence


Common (1% to 10%): Conjunctivitis (2%), corneal opacity (2%), keratitis (2%), photophobia (2%), blepharitis (1%), eye pain (1%), cataracts (1%)


Common (1% to 10%): Hepatic neoplasm (hepatic neoplasm [3.2%]; hepatic neoplasm, malignant [4.4%])

Uncommon (0.1% to 1%): Benign brain neoplasm; lymphoma, malignant


Uncommon (0.1% to 1%): Pruritus (1%), exfoliative dermatitis (1%), dry skin (1%), maculopapular rash (1%), alopecia (1%)


Frequency not reported: Abdominal pain, diarrhea, enanthema, gastritis, gastroenteritis, gastrointestinal hemorrhage, melena, tooth discoloration


Uncommon (0.1% to 1%): Amenorrhea, hematuria


Frequency not reported: Pathological fracture


Frequency not reported: Death, cyanosis, thirst, infection, septicemia, otitis


Frequency not reported: Bronchitis, respiratory insufficiency, cyanosis

Editorial References and Review

Medically reviewed by BestRx Medical Team Last updated on 1/1/2020.

Source: Drugs.com Orfadin