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

Patients should restrict intake of natural proteins, all of which contain tyrosine.

The amino acid phenylalanine is converted to tyrosine in the body, thus the intake of phenylalanine also has to be restricted. To obtain sufficient amounts of all other amino acids, protein substitutes specially designed for hereditary tyrosinemia type 1 patients must be used. All other aspects of nutrition like energy, fat, vitamins, minerals should be adapted to the needs of the child to grow normally. In addition, regular blood tests are needed to monitor the blood levels of tyrosine and phenylalanine.

It's important to make sure that your child gets the right type of protein to help growth and development. So, infants with HT-1 will need to adhere to a special diet that includes specialty formula. As children with HT-1 mature, they must take protein supplements and keep to a special diet. Working with a dietitian can help you develop a diet that works best for your family.

Some foods to avoid include:

  • Meat
  • Fish
  • Cheese
  • Eggs
  • Milk
  • Nuts
  • Ordinary bread
  • Pastas

There are a lot of little- and no-protein foods that can be eaten, such as low-protein pasta, breads, pizza, fruit, vegetables and sweets. There are also low-protein food options that can be eaten in measured amounts, such as low-protein pasta, rice, cereals, peas, and sweet corn. Your doctor may refer to these foods a "protein exchanges."

Dietary treatment is life-long and requires a great deal of understanding and commitment from those affected, their parents and relatives, and their schools. Professional support and nutritional advice is often available in this respect.

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.
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