Orfadin capsules (Nitisinone)
Complete patient guide for uses, dosage, side effects, and safety information
Prescription RequiredQuick Facts
What Is Orfadin capsules Used For?
Orfadin contains nitisinone, a medication specifically designed to treat a rare genetic condition that affects how your body processes certain amino acids.
Primary Uses
Orfadin is used to treat hereditary tyrosinaemia type 1 (HT-1), a genetic disease where the body cannot completely break down the amino acid tyrosine.
How It Works
In HT-1, harmful substances can form and build up in your body. Orfadin blocks the breakdown of tyrosine, preventing these harmful substances from forming. However, since tyrosine remains in the body, you must follow a special diet low in tyrosine and phenylalanine.
Good to know: Orfadin is not addictive and helps control your condition when used with proper diet management.
How to Take Orfadin capsules
Your doctor will determine the right dose for you and may adjust it based on your response and test results. Always take Orfadin exactly as prescribed.
Dosage Instructions
The recommended starting dose is 1 mg/kg body weight per day, divided into 2 doses. Take with food and swallow the capsule whole with a full glass of water. If you have trouble swallowing, you may open the capsule and mix the powder with a small amount of water or formula. For patients over 20 kg who have been taking twice daily doses for at least 4 weeks, your doctor may change you to once daily dosing.
What If You Miss a Dose?
If you forget a dose, do not take a double dose to make up for it. This may increase your chance of side effects. If you’re unsure what to do, ask your doctor or pharmacist.
Important: Never double dose if you miss a dose, and continue taking for as long as your doctor tells you – this medicine controls but does not cure your condition.
Available Tablet Strengths
Side Effects
Like all medications, this medicine can cause side effects. Most are mild and temporary, but some require medical attention.
Common Side Effects
These side effects occur relatively frequently and usually don’t require emergency care:
- rash
- itching or skin irritation
- redness and peeling of skin
- headache
- stomach pain
- constipation
Seek immediate medical attention if you experience:
- red eyes
- scarring or clouding of the cornea
- inflammation in the cornea, eye, or eyelid
- sensitivity to light
- eye pain
- shortness of breath
- wheezing or difficulty breathing
- swelling of face, lips, tongue or other body parts
- rash, itching or hives on skin (allergic reaction)
Important Warnings
Before taking Orfadin, it’s important to discuss your medical history and current medications with your doctor to ensure safe use.
Who Should Not Take Orfadin capsules
Do not use Orfadin if you are allergic to nitisinone or any ingredients in the capsules. Do not take if the packaging is damaged or past the expiry date.
Medical Conditions to Discuss
Tell your doctor about any other medical conditions you have and all medicines you take for other conditions. Regular monitoring is needed as the disease can affect your liver.
Pregnancy and Breastfeeding
Check with your doctor if you are pregnant or planning to become pregnant – they can discuss risks and benefits. Do not breastfeed while taking Orfadin. Tell your doctor immediately if you become pregnant while taking this medicine.
Eye Problems: Tell your doctor immediately if you experience red eyes, clouding or inflammation of the eyes, eyelid inflammation, light sensitivity, or eye pain – these could indicate inadequate dietary control.
Drug Interactions
Some medicines may interfere with Orfadin and affect how it works. Always tell your healthcare providers about all medications you’re taking.
- warfarin (blood thinner)
- phenytoin (epilepsy medicine)
- furosemide/frusemide (diuretic)
Alcohol & Driving
While Orfadin is not known to cause dizziness or lightheadedness, it can affect your vision if certain side effects develop.
Alcohol
No specific alcohol warnings are mentioned in the prescribing information.
Driving and Operating Machinery
Be careful when driving or operating machinery until you know how Orfadin affects you. The medication can affect your vision if you develop certain side effects, so monitor yourself carefully.
Storage
Keep capsules in the original bottle until time to take them. Store in refrigerator between 2°C – 8°C (do not freeze). Alternatively, capsules can be stored in a cool, dry place below 25°C for up to 2 months only. Keep out of reach of children in a locked cupboard at least 1.5 meters above ground. Do not store in bathroom or near sink, and avoid heat and dampness.
Overdose Information
If you think you have taken too much Orfadin, seek urgent medical attention immediately. Call the Poisons Information Centre (13 11 26), contact your doctor, or go to the nearest hospital Emergency Department, even if you feel fine.
Frequently Asked Questions
Orfadin helps control your condition but does not cure it. Your doctor will monitor your response through regular blood and urine tests and adjust your dose accordingly.
Yes, you should take Orfadin with food. Swallow the capsule whole with a full glass of water.
No, Orfadin is not addictive.
Do not stop taking Orfadin or lower the dose without checking with your doctor. If you stop suddenly, your condition may worsen or you may have unwanted side effects.
You must follow a special diet that is low in tyrosine and phenylalanine content as recommended by your doctor. This is very important while taking Orfadin.
Yes, if you have problems swallowing the capsules, you may open them and mix the powder with a small amount of water or formula just before taking it. Make sure you take the entire amount.
Store in the refrigerator between 2°C – 8°C (do not freeze). Alternatively, you can store in a cool dry place below 25°C for up to 2 months only.
Important Disclaimer
This information is intended for general educational purposes only and should not be considered medical advice. Always consult your healthcare provider or pharmacist before starting, stopping, or changing any medication.
If you think you may have a medical emergency, call your doctor or emergency services immediately.