IMPORTANT: HOW TO USE THIS INFORMATION: This is a summary and does NOT have all possible information about this product. This information does not assure that this product is safe, effective, or appropriate for you. This information is not individual medical advice and does not substitute for the advice of your health care professional. Always ask your health care professional for complete information about this product and your specific health needs.

NITISINONE - ORAL

(nye-TIS-i-none)

COMMON BRAND NAME(S): Orfadin

USES: Nitisinone is used to treat a certain inherited disorder (hereditary tyrosinemia type 1, also known as HT-1). HT-1 is usually discovered in infants and needs lifelong treatment. This condition is caused by a shortage of a certain natural substance that is needed to break down a nutrient (tyrosine) found in food. This effect causes a build-up of too much tyrosine and related substances in the liver. Nitisinone works by helping to prevent the formation and build-up of several toxic substances that cause damage to the liver, kidneys, and nervous system. This drug must be used along with a diet low in protein, tyrosine, and phenylalanine.

HOW TO USE: Take this medication by mouth on an empty stomach at least 1 hour before a meal, usually twice daily, or as directed by your doctor.

For children who are unable to swallow capsules, the capsules may be opened and the contents mixed in a small amount of water, formula, or applesauce immediately before use. Make sure the child swallows the entire dose.

The dosage is based on weight, medical condition, and response to treatment. The manufacturer recommends that patients should not take more than 2 milligrams per kilogram a day.

Take this medication regularly to get the most benefit from it. To help you remember, take it at the same times each day.

It is very important that patients taking this medication follow a special diet low in protein, tyrosine, and phenylalanine. Consult your doctor, pharmacist, or dietician for more details. (See also Drug Interactions and Precautions sections.)

Nitisinone can increase tyrosine levels and cause eye problems. Patients should have an eye test (slit-lamp examination) before using this medication so that later the doctor can see whether there have been changes. (See also Side Effects and Precautions sections.)