Full Prescribing Information Important Safety Information

Questions?  Call 877-473-3179 (7:00 am — 6:00 pm CST) or email us.

the orfadin

standing together

Since 2002, Americans with hereditary tyrosinemia type 1 (HT-1) have been able to take Orfadin® (nitisinone) for their condition. That's over 150 patients to date who have been treated with Orfadin for HT-1. We proudly call them the Orfadin Generation. We've been with them all along, and we'll continue to support them every step of the way.

get info
about treatment options

get support
with Orfadin4U

get access
to Orfadin


Orfadin is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase indicated for use as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1).


Tyrosine levels can increase in the blood if you do not restrict tyrosine and phenylalanine in your diet while taking Orfadin. Too much tyrosine in the blood can cause serious eye problems or other complications.

Do not adjust your Orfadin dosage in order to lower the tyrosine levels in the blood.

A reduction in the number of white cells and platelets in the blood have been observed during treatment with Orfadin. Your platelet and white blood cell counts should be monitored regularly during Orfadin treatment.

The most common adverse reactions to taking Orfadin are liver cancer, liver failure, low platelets or white cells in the blood, and complaints related to the eyes, including conjunctivitis, corneal opacity, inflammation of the cornea, and extreme sensitivity to light.

Tell your physician promptly if you have unexplained eye symptoms, rash, jaundice (yellowing of the skin or whites of the eyes) or excessive bleeding.

Use Orfadin during pregnancy only if the potential benefit justifies the potential risk to the fetus.

Nursing women should discontinue either Orfadin or breast-feeding based on the recommendation of your healthcare professional.

Please see full Prescribing Information