Medications and Drugs
Brand Names: Orfadin
Generic Name: nitisinone (Pronunciation: ni TIZ i none)
What is nitisinone (Orfadin)?
Nitisinone is used to treat a rare genetic condition called hereditary tyrosinemia type 1 (HT-1). HT-1 is a metabolic disorder that occurs when the body does not produce enough of an enzyme that breaks down proteins from certain foods. This condition occurs most often in young babies. The symptoms of HT-1 include unusual bleeding and signs of liver failure, such as dark urine, jaundice (yellowing of the skin or eyes), sudden stomach pain, nausea, vomiting, diarrhea, and fatigue.
Nitisinone works by preventing the body from breaking down an amino acid called tyrosine and by keeping other toxic substances from building up and causing harm to your liver or kidneys.
What are the possible side effects of nitisinone (Orfadin)?
Stop using nitisinone and get emergency medical help if you have any of these serious side side effects. Some of these side effects can be caused by the medication or by your diet:
Other, less serious side effects may be more likely to occur. Continue using nitisinone and talk with your doctor if you have any of these side effects:
Side effects other than those listed here may also occur. Talk to your doctor about any side effect that seems unusual or that is especially bothersome. You may report side effects to FDA at 1-800-FDA-1088.
What is the most important information I should know about nitisinone (Orfadin)?
Nitisinone is only part of a complete program of treatment that also includes a special diet. It is very important to follow the diet plan created for you by your doctor or nutrition counselor. You should become very familiar with the list of foods you must avoid to help control your condition.
You may experience side effects that are caused by nitisinone. Similar side effects may also be caused by your diet. Call your doctor immediately if you have jaundice (yellowing of the skin or eyes), unusual bleeding, vision changes, or eye redness, pain, burning, or sensitivity to light.
Report Problems to the Food and Drug Administration
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit the FDA MedWatch website or call 1-800-FDA-1088.
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