Consumer Medicine Information
What is in this leaflet
This leaflet answers some common questions about ORFADIN hard capsules. It does not contain all the available information. It does not take the place of talking to your doctor or pharmacist.
All medicines have risks and benefits. Your doctor has weighed the risks of you taking ORFADIN against the benefits they expect it will have for you.
If you have any concerns about taking this medicine, ask your doctor or pharmacist.
Keep this leaflet with the medicine. You may need to read it again.
What ORFADIN is used for
ORFADIN contains a medicine called nitisinone which is used for the treatment of a disease called hereditary tyrosinaemia type 1 (HT-1). In this disease the body is unable to completely break down the amino acid tyrosine. Harmful substances will be formed and accumulated in the body.
ORFADIN blocks the breakdown of tyrosine and by doing so the harmful substances are not formed. However, tyrosine will remain in the body and therefore a special diet (with low tyrosine and phenylalanine content) must be followed when taking ORFADIN.
Nitisinone belongs to a group of medicines called 'other alimentary and metabolism products'.
Ask your doctor if you have any questions about why this medicine has been prescribed for you. Your doctor may have prescribed it for another reason.
ORFADIN is not addictive.
ORFADIN is available only with a doctor's prescription.
Before you take ORFADIN
When you must not take it
Do not take ORFADIN if you have an allergy to:
- any medicine containing nitisinone
- any of the ingredients listed at the end of this leaflet.
Some of the symptoms of an allergic reaction may include:
- 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
Do not breast-feed if you are taking this medicine.
Do not take this medicine after the expiry date printed on the pack or if the packaging is torn or shows signs of tampering. If it has expired or is damaged, return it to your pharmacist for disposal.
If you are not sure whether you should start taking this medicine, talk to your doctor.
Before you start to take it
Tell your doctor if you have allergies to any other medicines, foods, preservatives or dyes.
Tell your doctor about your diet regimen. Your doctor will advise you regarding a diet restricted in tyrosine and phenylalanine content.
Tell your doctor if you are pregnant or plan to become pregnant or are breastfeeding. Your doctor can discuss with you the risks and benefits involved.
If you have not told your doctor about any of the above, tell him/her before you start taking ORFADIN.
Taking other medicines
Tell your doctor or pharmacist if you are taking any other medicines, including any that you get without a prescription from your pharmacy, supermarket or health food shop.
Some medicines and ORFADIN may interfere with each other. These include:
- warfarin, a medicine used to thin your blood
- phenytoin, a medicine used to treat epilepsy
- furosemide (frusemide), a diuretics
These medicines may be affected by ORFADIN or may affect how well it works. You may need different amounts of your medicines, or you may need to take different medicines.
Your doctor and pharmacist have more information on medicines to be careful with or avoid while taking this medicine.
How to take ORFADIN
Treatment with ORFADIN should be initiated and supervised by a physician experienced in the treatment of HT-1 patients.
Follow all directions given to you by your doctor or pharmacist carefully. They may differ from the information contained in this leaflet.
If you do not understand the instructions on the box/bottle, ask your doctor or pharmacist for help.
How much to take
The recommended initial dose is 1 mg/kg body weight/day divided in 2 doses administered orally.
Your doctor may change your total dose from twice a day to once a day. You will need to have taken ORFADIN twice daily for at least 4 weeks before your doctor may consider changing your dose. You will need to be at least 20 kg for the ORFADIN to be used once a day.
The dose of ORFADIN will be adjusted individually for you by your doctor.
How to take it
Take ORFADIN with food.
Swallow the capsules whole with a full glass of water. If you have problems with swallowing the capsules, you may open the capsule and mix the powder with a small amount of water or formula diet just before you take it.
When to take it
The usual daily dose is 1 mg/kg body weight divided in morning and evening administrations.
Take your first dose in the morning.
Take your second dose in the evening.
Your doctors will advise you on the correct time to take ORFADIN when moving you to once daily dose.
Take your medicine at about the same time each day.
Taking it at the same time each day will have the best effect. It will also help you remember when to take it.
How long to take it
Continue taking ORFADIN for as long as your doctor tells you.
This medicine helps to control your condition, but does not cure it. It is important to keep taking your medicine even if you feel well.
If you forget to take it
Do not take a double dose to make up for the dose that you missed. This may increase the chance of you getting an unwanted side effect.
If you are not sure what to do, ask your doctor or pharmacist.
If you have trouble remembering to take your medicine, ask your pharmacist for some hints.
If you take too much (overdose)
Immediately telephone your doctor or the Poisons Information Centre (telephone 13 11 26) for advice, or go to Accident and Emergency at the nearest hospital, if you think that you or anyone else may have taken too much ORFADIN. Do this even if there are no signs of discomfort or poisoning. You may need urgent medical attention.
While you are using ORFADIN
Things you must do
It is very important that you stay on the special diet, with low tyrosine and phenylalanine content that your doctor recommended. If you do not understand the instructions on your diet, ask your doctor for help.
If you are about to be started on any new medicine, remind your doctor and pharmacist that you are taking ORFADIN.
Tell any other doctors, dentists, and pharmacists who treat you that you are taking ORFADIN. Remind them if any new medicines are about to be started.
If you are going to have surgery, tell the surgeon or anaesthetist that you are taking ORFADIN. It may affect other medicines used during surgery.
If you become pregnant while taking this medicine, tell your doctor immediately.
If you are about to have any blood tests, tell your doctor that you are taking this medicine. It may interfere with the results of some tests.
Keep all of your doctor’s appointments so that your progress can be checked.
- Your Doctor will test your blood to check if the amounts of ORFADIN given are enough and to make sure that there are no possible side effects causing blood disorders.
- Your doctor will also check your liver regularly as the disease can affect the liver.
- Your doctor will also check on your general development.
Things you must not do
Do not take ORFADIN to treat any other complaints unless your doctor tells you to.
Do not give ORFADIN to anyone else, even if they have the same condition as you.
Do not stop taking ORFADIN or lower the dosage without checking with your doctor. If you stop taking it suddenly, your condition may worsen or you may have unwanted side effects.
Things to be careful of
Be careful driving or operating machinery or doing other jobs that require you to be alert until you know how ORFADIN affects you.
If you feel light-headed, dizzy or faint when getting out of bed or standing up, get up slowly. Standing up slowly, especially when you get up from bed or chairs, will help your body get used to the change in position and blood pressure. If this problem continues or gets worse, talk to your doctor.
Tell your doctor or pharmacist as soon as possible if you do not feel well while you are taking ORFADIN.
This medicine helps most people with HT-1, but it may have unwanted side effects in a few people. All medicines can have side effects. Sometimes they are serious, most of the time they are not. You may need medical attention if you get some of the side effects.
Do not be alarmed by the following lists of side effects. You may not experience any of them.
Ask your doctor or pharmacist to answer any questions you may have.
Tell your doctor or pharmacist if you notice any of the following and they worry you:
- different eye symptoms as listed below:
– scarring or clouding of the cornea
– inflammation in the cornea
– inflammation in the eye
– inflammation in the eyelid
– sensitivity to light
– eye pain
- itching or irritating sensation of skin
- redness and peeling of skin
- abdominal pain
If you get red eyes or any other signs of effects on the eyes, contact your doctor immediately for an eye examination. Eye problems could be a sign of inadequate dietary control.
The above list includes the more common side effects of your medicine.
There may be other side effects that your doctor will carefully monitor by taking blood samples. These are:
- reduced number of platelets and white blood cells
- shortage of certain white blood cells (granulocytopenia)
Tell your doctor or pharmacist if you notice anything that is making you feel unwell.
Other side effects not listed above may also occur in some people.
Some of these side effects can only be found when your doctor does tests from time to time to check your progress.
After using ORFADIN
Keep your capsules in the bottle until it is time to take them. If you take the capsules out of the bottle they may not keep well.
ORFADIN capsules should be stored, in the box/bottle, in a refrigerator between 2°C – 8°C. Do not freeze. Alternatively, and for a period of 2 months only, ORFADIN capsules can also be stored in a cool dry place where the temperature stays below 25°C.
To help you remember, add the discard date in the space provided on the box/bottle when you stop refrigerating.
After this period, discard all ORFADIN capsules that have not been refrigerated. Do not store ORFADIN or any other medicine in the bathroom or near a sink. Do not leave it on a window sill or in the car, especially on hot or cold days.
Heat and dampness can destroy some medicines.
Keep it where children cannot reach it. A locked cupboard at least one-and-a-half metres above the ground is a good place to store medicines.
If your doctor tells you to stop taking this medicine or the expiry date has passed, ask your pharmacist what to do with any medicine that is left over.
What it looks like
ORFADIN capsules are white, opaque, hard gelatin capsules, imprinted with "NTBC" and the strength "2 mg", "5 mg" "10 mg" or "20mg" in black.
The capsules contain a white to off-white powder.
Each ORFADIN capsule contains either 2 mg, 5 mg, 10 mg, and 20 mg of nitisinone.
It also contains the following inactive ingredients:
- starch – pregelatinised maize
- titanium dioxide
- printing ink: Capsugel Ink 10A1 Black (Proprietary Ingredient Number 109522) or
- printing ink: Capsugel Ink 10A2 Black (Proprietary Ingredient Number 109521)
This medicine does not contain lactose, sucrose, gluten, tartrazine or any other azo dyes.
ORFADIN capsules are available in plastic bottles with child resistant closures.
Each bottle contains 60 capsules.
ORFADIN is supplied in Australia by:
A. Menarini Australia Pty. Ltd.
Level 8, 67 Albert Avenue
CHATSWOOD NSW 2067
Medical Information: 1800 644 542
Australian Registration Number:
ORFADIN 2 mg: AUST R 164163
ORFADIN 5 mg: AUST R 164173
ORFADIN 10 mg: AUST R 164174
ORFADIN 20 mg: AUST R 297735
ORFADIN® is a registered trademark of Swedish Orphan Biovitrum International AB, used under license by A. Menarini Australia Pty. Ltd.
This leaflet was revised October 2019.
For the most up to date version of this leaflet, please go to www.menarini.com.au/cmi
Published by MIMS December 2019