Orfadin Oral Suspension (Nitisinone)
Complete patient guide for uses, dosage, side effects, and safety information
Prescription RequiredQuick Facts
What Is Orfadin Oral Suspension Used For?
Orfadin Oral Suspension contains nitisinone, which is specifically designed to treat a rare genetic condition called hereditary tyrosinaemia type 1 (HT-1).
Primary Uses
Orfadin is used for the treatment of hereditary tyrosinaemia type 1 (HT-1), a genetic disease where the body cannot properly break down the amino acid tyrosine.
How It Works
In HT-1, the body cannot completely break down tyrosine, causing harmful substances to build up. Orfadin blocks the breakdown of tyrosine, preventing these harmful substances from forming. However, tyrosine remains in the body, so you must follow a special diet low in tyrosine and phenylalanine.
Good to know: Orfadin is not addictive.
How to Take Orfadin Oral Suspension
Orfadin oral suspension is taken directly in your mouth using the provided oral syringe without dilution. Never inject this medication or attach a needle to the syringe.
Dosage Instructions
The recommended starting dose is 1 mg/kg body weight per day, divided into 2 doses taken orally. Your doctor may change you to once daily dosing if you weigh more than 20 kg and have been taking it twice daily for at least 4 weeks. Take with food. If taking twice daily, take your first dose in the morning and second dose in the evening at about the same times each day. Your doctor will adjust your dose based on your response and test results.
What If You Miss a Dose?
If you forget to take a dose, do not take a double dose to make up for the missed one. This may increase your chance of side effects. If you’re unsure what to do, ask your doctor or pharmacist.
Important: Never take a double dose to make up for a missed dose – this increases the risk of side effects.
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 irritating sensation of skin
- 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
- inflammation in the eye
- inflammation in the eyelid
- sensitivity to light
- eye pain
- shortness of breath
- wheezing or difficulty breathing
- swelling of the face, lips, tongue or other parts of the body
- rash, itching or hives on the skin
Important Warnings
There are important safety considerations before starting Orfadin, including allergies, other medical conditions, and special dietary requirements.
Who Should Not Take Orfadin Oral Suspension
Do not use Orfadin if you are allergic to nitisinone or any ingredients listed. Do not use after the expiry date or if packaging is damaged.
Medical Conditions to Discuss
Tell your doctor if you have any other medical conditions or take any other medicines. Your doctor will need to monitor you regularly with blood and urine tests to check your dose and watch for side effects.
Pregnancy and Breastfeeding
Check with your doctor if you are pregnant or planning to become pregnant – they can discuss the risks and benefits. Do not breastfeed while taking Orfadin. Tell your doctor immediately if you become pregnant while taking this medicine.
Special Diet Required: You must stay on a special diet with low tyrosine and phenylalanine content as recommended by your doctor – this is essential for the medication to work safely.
Drug Interactions
Some medicines may interfere with Orfadin and affect how it works. Always tell your doctor about all medicines you’re taking.
- warfarin (blood thinner)
- phenytoin (epilepsy medicine)
- furosemide/frusemide (diuretic)
Alcohol & Driving
Be careful when driving or operating machinery until you know how Orfadin affects you.
Alcohol
No specific alcohol warnings mentioned in the source material.
Driving and Operating Machinery
Be careful driving or operating machinery until you know how Orfadin affects you. While Orfadin doesn’t typically cause dizziness or light-headedness, it can affect your vision if you develop certain side effects.
Storage
Keep unopened bottles in the refrigerator between 2°C – 8°C in the original box, stored upright. Do not freeze. After opening, store below 25°C or in the refrigerator and discard 2 months after opening. Write the discard date on the bottle label when first opened. Keep away from children in a locked cupboard at least 1.5 meters above ground.
Overdose Information
If you think you have taken too much Orfadin, seek urgent medical attention immediately. Phone 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
Your doctor will adjust your Orfadin dose based on your response and test results. Regular blood and urine tests will monitor how well the medication is working for you.
Yes, you should take Orfadin with food as directed in the instructions.
No, Orfadin is not addictive.
Do not stop taking Orfadin or lower the dose without checking with your doctor first. If you stop suddenly, your condition may worsen or you may have unwanted side effects.
Do not take a double dose to make up for the missed dose, as this may increase your chance of side effects. If you’re unsure what to do, ask your doctor or pharmacist.
Because Orfadin blocks tyrosine breakdown but tyrosine remains in your body, you must follow a special diet low in tyrosine and phenylalanine content. This is essential for the medication to work safely.
After opening, store the bottle below 25°C or in the refrigerator and discard it 2 months after opening. Write the discard date on the bottle label when you first open it.
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.