Patient leaflet - Nitisinone MDK (previously Nitisinone MendeliKABS)
1. What Nitisinone MDK is and what it is used for
The active ingredient of Nitisinone MDK is nitisinone. This medicine is used for treatment of a rare disease called hereditary tyrosinemia type 1 in adults, adolescents and children (in any age range).
In this disease your body is unable to completely break down the amino acid tyrosine (amino acids are building blocks of our proteins), forming harmful substances. These substances are accumulated in your body. Nitisinone MDK blocks the breakdown of tyrosine and the harmful substances are not formed.
You must follow a special diet while you are taking this medicine, because tyrosine will remain in your body. This special diet is based on low tyrosine and phenylalanine (another amino acid) content.
2. What you need to know before you take Nitisinone MDK
Do not take Nitisinone MDK:
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– if you are allergic to nitisinone or any of the other ingredients of this medicine (listed in section 6).
Do not breast-feed while taking this medicine, see section “Pregnancy and breast-feeding”.
Warnings and precautions
Talk to your doctor or pharmacist before taking Nitisinone MDK.
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– Your eyes will be checked by an ophthalmologist before and regularly during nitisinone treatment. 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 (see section 4).
During the treatment, blood samples will be drawn in order for your doctor to check whether the treatment is adequate and to make sure that there are no possible side effects causing blood disorders. Your liver will be checked at regular intervals because the disease affects the liver.
Follow-up by your doctor should be performed every 6 months. If you experience any side effects, shorter intervals are recommended.
Other medicines and Nitisinone MDK
Tell your doctor or pharmacist if you are taking, have recently taken or might take any other medicines.
Nitisinone MDK may interfere with the effect of other medicines, such as:
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– Medicines for epilepsy (such as phenytoin)
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– Medicines against blood clotting (such as warfarin)
Nitisinone MDK with food and drink
If you start Nitisinone MDK treatment by taking it with food, it is recommended that you carry on taking it with food throughout your course of treatment.
Pregnancy and breast-feeding
The safety of this medicine has not been studied in pregnant and breast-feeding women. Please contact your doctor if you plan to become pregnant. If you become pregnant you should contact your doctor immediately.
Do not breast-feed while taking this medicine, see section “Do not take Nitisinone MDK”.
Driving and using machines
This medicine has minor influence on the ability to drive and use machines. However, if you experience side effects affecting your vision you should not drive or use machines until your vision is back to normal (see section 4 “Possible side effects”).
3. How to take Nitisinone MDK
Always take this medicine exactly as your doctor has told you. Check with your doctor or pharmacist if you are not sure.
Treatment with this medicine should be started and supervised by a doctor experienced in the treatment of the disease (hereditary tyrosinemia type 1).
The recommended total daily dose is 1 mg/kg body weight administered orally. Your doctor will adjust the dose individually. It is recommended to administer the dose once daily. However, due to the limited data in patients with body weight <20 kg, it is recommended to divide the total daily dose into two daily administrations in this patient population.
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.
If you take more Nitisinone MDK than you should
If you have taken more of this medicine than you should, contact your doctor or pharmacist as soon as possible.
If you forget to take Nitisinone MDK
Do not take a double dose to make up for a forgotten dose. If you forget to take a dose, contact your doctor or pharmacist.
If you stop taking Nitisinone MDK
If you have the impression that the medicine is not working properly, talk to your doctor. Do not change the dose or stop the treatment without talking to your doctor.
If you have any further questions on the use of this medicine, ask your doctor, pharmacist or nurse.
4. Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them.
If you notice any side effects relating to the eyes, talk to your doctor immediately to have an eye examination. Treatment with nitisinone leads to higher levels of tyrosine in the blood which can cause eye related symptoms. Common eye related side effects (may affect more than 1 in 10 people) caused by higher tyrosine levels are inflammation in the eye (conjunctivitis), opacity and inflammation in the cornea (keratitis), sensitivity to light (photophobia) and eye pain. Inflammation of the eyelid (blepharitis) is an uncommon side effect (may affect up to 1 in 100 people).
Other common side effects
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– Reduced number of platelets (thrombocytopenia) and white blood cells (leukopenia), shortage of certain white blood cells (granulocytopenia).
Other uncommon side effects
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– increased number of white blood cells (leucocytosis),
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– itching (pruritus), skin inflammation (exfoliative dermatitis), rash.
Reporting of side effects
If you get any side effects, talk to your doctor, pharmacist or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the national reporting system listed in Appendix V. By reporting side effects you can help provide more information on the safety of this medicine.
5. How to store Nitisinone MDK
Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date which is stated on the carton and the bottle after “EXP”. The expiry date refers to the last day of that month.
Store in a refrigerator (2°C – 8°C). Store in the original bottle in order to protect from light.
The medicine can be stored for a period of 2 months, after first opening the bottle, at a temperature not above 25°C, after which it must be discarded.
Do not forget to mark the date on the bottle, when removed from the refrigerator.
Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.
6. Contents of the pack and other information
What Nitisinone MDK contains
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– The active substance is nitisinone.
Nitisinone MDK 2 mg: Each capsule contains 2 mg nitisinone.
Nitisinone MDK 5 mg: Each capsule contains 5 mg nitisinone.
Nitisinone MDK 10 mg: Each capsule contains 10 mg nitisinone.
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– The other ingredients are:
What Nitisinone MDK looks like and contents of the pack
Nitisinone MDK capsules are 15.7 mm long, white, opaque, hard gelatin capsules, imprinted with “Nitisinone” and the strength “2 mg”, “5 mg” or “10 mg”, in black. The capsule contains a white to off-white powder.
The capsules are packaged in plastic bottles. Each bottle contains 60 capsules. Each carton pack contains one bottle.
Marketing Authorisation Holder
MendeliKABS Europe Limited
Unit 3D, North Point House
North Point Business Park
New Mallow Road
Cork, T23 AT2P, Ireland
Manufacturer
Elara Pharmaservices Europe Limited
239 Blanchardstown Corporate Park
Ballycoolin, Dublin
D15 KV21, Ireland
This leaflet was last revised in
Other sources of information
Detailed information on this medicine is available on the European Medicines Agency web site:.
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