Skip to Content
UW Health SMPH
American Family Children's Hospital
SHARE TEXT

nitisinone

Pronunciation: nye TIS i none

Brand: Orfadin

What is the most important information I should know about nitisinone?

Multum calldoc

Call your doctor at once if you have vision problems, eye pain, eye redness or burning, easy bruising, unusual bleeding, itching, or jaundice (yellowing of the skin or eyes). Tell your doctor if your baby has a sudden change in behavior, ability, or development (sitting up, crawling, walking, talking, etc).

What is nitisinone?

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.

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.

Nitisinone may also be used for purposes not listed in this medication guide.

What should I discuss with my health care provider before taking nitisinone?

To make sure nitisinone is safe for you, tell your doctor if you have any medical conditions affecting your eyes.

Multum nopreg

FDA pregnancy category C. It is not known whether nitisinone will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant while using this medication.

Multum nobrfeed

It is not known whether nitisinone passes into breast milk or if it could harm a nursing baby. Tell your doctor if you are breast-feeding a baby.

How should I take nitisinone?

Follow all directions on your prescription label. Do not take this medicine in larger or smaller amounts or for longer than recommended.

Your doctor may occasionally change your dose to make sure you get the best results. Do not change your medication dose or schedule without your doctor's advice.

Multum icon26

Take nitisinone on an empty stomach, at least 1 hour before or 2 hours after a meal.

You may open the nitisinone capsule and sprinkle the medicine into a spoonful of water, baby formula, or applesauce to make swallowing easier. Use the mixture right away. Do not save for later use.

Nitisinone may be only part of a complete program of treatment that also includes a special diet. You must avoid certain foods for your treatment to be effective. Follow the diet plan created for you by your doctor or nutrition counselor.

High protein foods are high in tyrosine and phenylalanine. Since babies and children need protein to grow and develop, special foods have been developed to replace the high protein foods.

While using nitisinone, you may need frequent blood tests at your doctor's office. Your vision may also need to be checked.

Multum refig

Store in the refrigerator, do not freeze.

What happens if I miss a dose?

Take the missed dose as soon as you remember. Skip the missed dose if it is almost time for your next scheduled dose. Do not take extra medicine to make up the missed dose.

What happens if I overdose?

Multum emt

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while taking nitisinone?

Multum donot

While you are taking nitisinone, you must not eat foods that are high in tyrosine or phenylalanine, including:

  • chicken, beef, pork, liver, fish, processed meats (bologna, hot dogs, pepperoni, hard sausages), meat tenderizer, herring or other dried fish;
  • cheese, milk, sour cream, yogurt;
  • beer, wine, distilled liquor;
  • avocados, bananas, carrots, potatoes, spinach, tomatoes, raisins, red plums,
  • oatmeal, brown rice,
  • soy sauce, soy protein, tofu, bean curd, garbanzo beans, soy beans, and certain nuts or seeds; or
  • foods that contain an artificial sweetener called aspartame (NutraSweet)--diet soda, some foods labeled as "sugar-free" or "zero calorie."

There are other foods that may contain tyrosine or phenylalanine. Get familiar with the list of foods you must avoid to help control your condition.

What are the possible side effects of nitisinone?

Multum emt

Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Multum donot

Some side effects may result from not properly following your diet plan and consuming restricted foods or beverages.

Multum calldoc

Call your doctor at once if you have:

  • a sudden change in behavior, ability, or development (sitting up, crawling, walking, talking, etc);
  • easy bruising, unusual bleeding (nose, mouth, vagina, or rectum), purple or red pinpoint spots under your skin;
  • nausea, diarrhea, upper stomach pain, itching, loss of appetite, dark urine, clay-colored stools, jaundice (yellowing of the skin or eyes);
  • vision problems,
  • eye pain, redness or swelling, burning, white or yellow patches on your eyes; or
  • calluses, peeling, or hardened skin on the palms of your hands or the soles of your feet.

Common side effects may include:

  • increased sensitivity of your eyes to light;
  • dry skin; or
  • mild rash.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

What other drugs will affect nitisinone?

Other drugs may interact with nitisinone, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell each of your health care providers about all medicines you use now and any medicine you start or stop using.

Where can I get more information?

Your pharmacist can provide more information about nitisinone.


Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Every effort has been made to ensure that the information provided by Cerner Multum, Inc. ('Multum') is accurate, up-to-date, and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. Multum information has been compiled for use by healthcare practitioners and consumers in the United States and therefore Multum does not warrant that uses outside of the United States are appropriate, unless specifically indicated otherwise. Multum's drug information does not endorse drugs, diagnose patients or recommend therapy. Multum's drug information is an informational resource designed to assist licensed healthcare practitioners in caring for their patients and/or to serve consumers viewing this service as a supplement to, and not a substitute for, the expertise, skill, knowledge and judgment of healthcare practitioners. The absence of a warning for a given drug or drug combination in no way should be construed to indicate that the drug or drug combination is safe, effective or appropriate for any given patient. Multum does not assume any responsibility for any aspect of healthcare administered with the aid of information Multum provides. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the drugs you are taking, check with your doctor, nurse or pharmacist.

Copyright 1996-2014 Cerner Multum, Inc. Version: 2.01. Revision date: 3/27/2013.

Your use of the content provided in this service indicates that you have read, understood and agree to the End-User License Agreement, which can be accessed by clicking on this link.

This information does not replace the advice of a doctor. Healthwise, Incorporated disclaims any warranty or liability for your use of this information. Your use of this information means that you agree to the Terms of Use. How this information was developed to help you make better health decisions.

Healthwise, Healthwise for every health decision, and the Healthwise logo are trademarks of Healthwise, Incorporated.