Prescribing Information | Important Safety Information
Prescribing Information | Important Safety Information



Orfadin4U is a comprehensive patient support program for Orfadin (nitisinone) patients and their caregivers. We are here to support you at every step of your treatment journey. Our goal is to help you and your family live as normal a lifestyle as possible.

When you enroll in Orfadin4U, help is always just a phone call away. Through Orfadin4U, you may take advantage of the following services:

Direct-to-You Pharmacy Service

  • Convenient at-home prescription delivery
  • Refill management to ensure reliable on-time delivery
  • Phone and email reminders to keep you informed and on track

Co-Pay Assistance and Insurance Coverage Management for Eligible Patients

  • Benefits verification and reimbursement support
  • Information on financial assistance for co-pays and deductibles
  • Help with prior authorization and coverage appeals

Educational and Nutritional Information

  • Patient education from qualified healthcare professionals
  • Dietary and nutrition information
  • Resources to help you manage HT-1

Please be aware that Orfadin4U is the exclusive pharmacy services provider for the distribution of Orfadin in the U.S. You may contact an Orfadin4U specialist via:

  • Phone: 877-473-3179 (7am – 6pm CST; after-hours support is available 24/7)
  • Fax: 877-473-3049
  • Email:

Indication and Usage

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).

Important Safety Information

    • 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