Orfadin

Orfadin helps stop the body from breaking down an amino acid called tyrosine and prevents other harmful substances from building up and damaging your liver or kidneys.

Orfadin is used to treat a rare genetic condition known as hereditary tyrosinemia type 1 (HT-1). HT-1 is a metabolic disorder that happens when the body does not make enough of an enzyme needed to break down proteins from certain foods. This condition usually occurs in very young babies.

Orfadin may also be used for reasons not mentioned in this medication guide.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose. Use the medicine exactly as directed.

Do not change your medication dose or schedule without your doctor's advice.

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

You may take Orfadin liquid with or without food.

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 (including artificial sweeteners such as aspartame, Equal, or Nutrasweet). Since babies and children need protein to grow and develop, special foods have been developed to replace the high protein foods.

Shake the oral suspension (liquid) for at least 5 full seconds just before you measure a dose. Use the dosing syringe provided, or use a medicine dose-measuring device (not a kitchen spoon).

If you cannot swallow a capsule whole, open it and sprinkle the medicine into a spoonful of water, baby formula, or applesauce. Swallow the mixture right away without chewing. Do not save it for later use.

If you cannot swallow a tablet whole, crush the tablet and sprinkle the medicine into a spoonful of applesauce. Swallow the mixture right away without chewing. Do not save it for later use.

You may also dissolve a tablet in an oral syringe with water. Read and carefully follow any Instructions for Use provided with your medicine. Ask your doctor or pharmacist if you do not understand these instructions.

You may need frequent blood tests. Your vision may also need to be checked.

Store tablets at room temperature away from moisture, heat, and light. Keep the bottle tightly closed when not in use.

Store the capsules or liquid in the refrigerator, do not freeze.

After using Orfadin liquid for the first time, you may store the medicine at room temperature for up to 60 days.

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

Tell your doctor if you have any medical conditions affecting your eyes.

Tell your doctor if you are pregnant or breastfeeding.

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

Some side effects may happen if you do not follow your diet plan properly and eat restricted foods or drinks.

Orfadin may cause serious side effects. Contact your doctor immediately if you experience:

• a sudden change in behavior, ability, or development (such as sitting up, crawling, walking, talking, etc);
• easy bruising, unusual bleeding (from your nose, mouth, vagina, or rectum), or 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 your skin or eyes);
• vision problems;
• eye pain, redness or swelling, burning, or 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 of Orfadin may include:

• changes in vision, such as increased sensitivity to light;
• eye pain, redness, or puffiness;
• nosebleeds;
• heightened sensitivity of your eyes to light;
• rash or itching;
• dry or peeling skin; or
• thinning hair.

The liquid form of nitisinone may contain glycerol and can cause headache, upset stomach, or diarrhea.

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

Take the medicine as soon as you can, but skip the missed dose if it is nearly time for your next dose. Do not take two doses at once.

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

While you are taking Orfadin, 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--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.

Other drugs may affect Orfadin, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you begin or stop using.