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Orfadin Side Effects

Generic Name: nitisinone

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

Rare

  • 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

Rare

For Healthcare Professionals

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

General

The most common reported side effects included hepatic neoplasm, liver failure, thrombocytopenia, leucopenia, visual system complaints (conjunctivitis, corneal opacity, keratitis, photophobia).[Ref]

Dermatologic

Uncommon (0.1% to 1%): Pruritis (1%), exfoliative dermatitis (1%), dry skin (1%), maculopapular rash (1%), alopecia (1%)[Ref]

Endocrine

Frequency not reported: Hypoglycemia[Ref]

Gastrointestinal

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

Genitourinary

Frequency not reported: Amenorrhea[Ref]

Hematologic

Common (1% to 10%): Thrombocytopenia (3%), leucopenia (3%), granulocytopenia (1%), porphyria (1%), epistaxis (1%)[Ref]

Hepatic

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

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

Metabolic

Frequency not reported: High plasma tyrosine levels, dehydration[Ref]

Musculoskeletal

Frequency not reported: Pathological fracture[Ref]

Nervous system

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

Ocular

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

Oncologic

Common (1% to 10%): Hepatic neoplasm (8%)[Ref]

Other

Frequency not reported: Death, cyanosis, thirst, infection, septicemia, otitis[Ref]

Respiratory

Frequency not reported: Bronchitis, respiratory insufficiency[Ref]

References

1. "Product Information. Orfadin (nitisinone)." Orphan Pharmaceuticals USA, Inc, Nashville, TN.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Some side effects may not be reported. You may report them to the FDA.

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