Orfadin®
Brand Name: Orfadin®
Active Ingredient:   nitisinone
Strength(s): 2 mg, 5 mg, and 10 mg
Dosage Form(s):   Capsules
Company Name:    Swedish Orphan International, AB
Availability:         Prescription only
*Date Approved by FDA:   January 18, 2002
*Approval by FDA does not mean that the drug is available for consumers at this time.

What is Orfadin used for? 

Orfadin is used to treat hereditary tyrosinemia type 1, a rare pediatric disease causing progressive liver failure and liver cancer in young children. It is used along with a diet restricting the amino acids tyrosine and phenylalanine. 

Special Warnings with Orfadin:

Patients and their parents or caregivers should tell their doctor right away about: 

  • any unexplained eye symptoms (eye pain, redness, swelling or burning of the eyes) 
  • rash 
  • jaundice (yellowing of the skin and whites of the eyes, and dark color to the urine) 
  • excessive bleeding 

What should I tell my health care provider? 

  • Tell your health care provider if you are trying to become pregnant, are already pregnant, or are breast-feeding. 
  • Tell your health care provider if you are allergic to nitisinone (Orfadin) or to any of the other ingredients in Orfadin. 
  • Tell your health care provider about all the medicines you take, including prescription and non-prescription medicines, vitamins, and herbal supplements. 

What are some possible side effects of Orfadin? (This is NOT a complete list of side effects reported with Orfadin. Your health care provider can discuss with you a more complete list of side effects.)

The most common side effects of Orfadin were related to high tyrosine levels due to patients not eating the right foods as well as rare cases of a decrease in platelet and white blood cell counts. 

For more detailed information about Orfadin, ask your health care provider or pharmacist.

Link to Orfadin's label

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Posted: 11/22/02