Generic Name: nitisinone (nye-TIS-i-none)
Brand Name: Orfadin
Orfadin is used for:
Treating hereditary tyrosinemia type 1 (HT-1), a genetic metabolism disorder. Orfadin is used in combination with dietary restriction of tyrosine and phenylalanine.
Orfadin is a 4-hydroxyphenylpyruvate dioxygenase inhibitor. It works by blocking the breakdown of tyrosine. This helps to reduce the amount of toxic byproducts in the body produced by the breakdown of tyrosine.
Do NOT use Orfadin if:
- you are allergic to any ingredient in Orfadin
Contact your doctor or health care provider right away if any of these apply to you.
Before using Orfadin:
Some medical conditions may interact with Orfadin. Tell your doctor or pharmacist if you have any medical conditions, especially if any of the following apply to you:
- if you are pregnant, planning to become pregnant, or are breast-feeding
- if you are taking any prescription or nonprescription medicine, herbal preparation, or dietary supplement
- if you have allergies to medicines, foods, or other substances
- if you have any other metabolism disorders (eg, porphyria)
- If you have eye or vision problems, low white blood cell levels, or low platelet levels
Some MEDICINES MAY INTERACT with Orfadin. However, no specific interactions with Orfadin are known at this time.
This may not be a complete list of all interactions that may occur. Ask your health care provider if Orfadin may interact with other medicines that you take. Check with your health care provider before you start, stop, or change the dose of any medicine.
How to use Orfadin:
Use Orfadin as directed by your doctor. Check the label on the medicine for exact dosing instructions.
- Take Orfadin on an empty stomach at least 1 hour before or 2 hours after a meal.
- If you have difficulty swallowing the capsule, it may be opened and the contents sprinkled into a small amount of water, formula, or applesauce immediately before use. Do not store the mixture for use at a later time.
- If you miss a dose of Orfadin, take it as soon as possible. If it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not take 2 doses at once.
Ask your health care provider any questions you may have about how to use Orfadin.
Important safety information:
- It is important to follow the dietary plan provided by your doctor in order to avoid the risk of serious side effects from Orfadin.
- Do NOT change your dose or take more than the recommended dose without checking with your doctor.
- LAB TESTS, including platelet and white blood cell counts, vision tests, liver function tests, and blood phosphate or tyrosine levels, may be performed to monitor your progress or to check for side effects. Be sure to keep all doctor and lab appointments.
- PREGNANCY and BREAST-FEEDING: It is unknown if Orfadin can cause harm to the fetus. If you think you may be pregnant, discuss with your doctor the benefits and risks of using Orfadin during pregnancy. It is unknown if Orfadin is excreted in breast milk. If you are or will be breast-feeding while you are using Orfadin, check with your doctor or pharmacist to discuss the risks to your baby.
Possible side effects of Orfadin:
All medicines may cause side effects, but many people have no, or minor, side effects. When used in small doses, no COMMON side effects have been reported with this product. Seek medical attention right away if any of these SEVERE side effects occur:
Severe allergic reactions (rash; hives; itching; difficulty breathing; tightness in the chest; swelling of the mouth, face, lips, or tongue); black or bloody stools; blue skin or nails; changes in thinking or behavior; dark urine; eye problems (eg, redness, swelling, burning, vision changes, light sensitivity, pain); fever, chills, or persistent sore throat; pale stools; seizures; severe headache; skin lesions; sores on hands or feet; stomach pain; unusual bruising or bleeding; yellowing of the eyes or skin.
This is not a complete list of all side effects that may occur. If you have questions about side effects, contact your health care provider. Call your doctor for medical advice about side effects. To report side effects to the appropriate agency, please read the Guide to Reporting Problems to FDA.
If OVERDOSE is suspected:
Contact 1-800-222-1222 (the American Association of Poison Control Centers), your local poison control center, or emergency room immediately. Symptoms may include eye problems (eg, redness, swelling, burning, vision changes, light sensitivity, pain); skin lesions.Proper storage of Orfadin:
Store Orfadin in the refrigerator between 36 and 46 degrees F (2 and 8 degrees C). Keep Orfadin out of the reach of children and away from pets.
- If you have any questions about Orfadin, please talk with your doctor, pharmacist, or other health care provider.
- Orfadin is to be used only by the patient for whom it is prescribed. Do not share it with other people.
- If your symptoms do not improve or if they become worse, check with your doctor.
- Check with your pharmacist about how to dispose of unused medicine.
This information should not be used to decide whether or not to take Orfadin or any other medicine. Only your health care provider has the knowledge and training to decide which medicines are right for you. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a brief summary of general information about Orfadin. It does NOT include all information about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to Orfadin. This information is not specific medical advice and does not replace information you receive from your health care provider. You must talk with your healthcare provider for complete information about the risks and benefits of using Orfadin.
Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only your health care provider has the knowledge and training to decide which medicines are right for you. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a brief summary of general information about this medicine. It does NOT include all information about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not specific medical advice and does not replace information you receive from your health care provider. You must talk with your healthcare provider for complete information about the risks and benefits of using this medicine.