Orfadin Side Effects

Generic name: nitisinone

Medically reviewed by Drugs.com. Last updated on May 5, 2023.

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

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

Serious side effects of Orfadin

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
fast heartbeat
headache
irritability
seeing or hearing things that are not there
seizures
stiff neck
tightness in the chest
vomiting

Other side effects of Orfadin

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

Rare

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

For Healthcare Professionals

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

General

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.^[Ref]

Hepatic

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

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

Hematologic

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

Uncommon (0.1% to 1%): Anemia^[Ref]

Metabolic

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

Uncommon (0.1% to 1%): Dehydration, hypoglycemia^[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 (hepatic neoplasm [3.2%]; hepatic neoplasm, malignant [4.4%])

Uncommon (0.1% to 1%): Benign brain neoplasm; lymphoma, malignant^[Ref]

Dermatologic

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

Gastrointestinal

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