Orfadin 2 mg hard capsules
Spain
Table of Contents
Package leaflet: Information for the user
Introduction
Package leaflet: information for the user
Orfadin 2 mg hard capsules
Orfadin 5 mg hard capsules
Orfadin 10 mg hard capsules
Orfadin 20 mg hard capsules
nitisinone
Read the entire leaflet carefully before you start taking this medicine, because it contains important information for you.
- Keep this leaflet as you may need to read it again.
- If you have any questions, ask your doctor, pharmacist or nurse.
- This medicine has been prescribed for you only, and you should not give it to other people, even if they have the same symptoms as you, because it may harm them.
- If you experience any adverse effects, consult your doctor, pharmacist or nurse, even if they are adverse effects not listed in this leaflet. See section 4.
Contents of the leaflet
- What Orfadin is and what it is used for
- What you need to know before taking Orfadin
- How to take Orfadin
- Possible adverse effects
- How to store Orfadin
- Contents of the pack and other information
1. What Orfadin is and what it is used for
Orfadin contains the active substance nitisinone. Orfadin is used to treat:
- a rare disease called hereditary tyrosinemia type 1 in adults, adolescents, and children (of any age)
- a rare disease called alkaptonuria (AKU) in adults
In these diseases, your body cannot fully break down the amino acid tyrosine (amino acids are the building blocks of proteins), leading to the formation of toxic substances. These substances accumulate in your body. Orfadin blocks the breakdown of tyrosine, thereby preventing the formation of toxic substances.
For the treatment of hereditary tyrosinemia type 1, you must follow a special diet while taking this medicine, because tyrosine will still be present in your body. This diet is low in tyrosine and phenylalanine (another amino acid).
For the treatment of AKU, your doctor may advise you to follow a special diet.
2. What you need to know before starting Orfadin
Do not take Orfadin
- if you are allergic to nitisinone or to 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 starting to take Orfadin.
- An ophthalmologist will examine your eyes before starting treatment and regularly during treatment with nitisinone. If you notice redness of the eyes or any other eye-related symptoms, contact your doctor immediately for an ophthalmological examination. Eye problems (see section 4) may indicate inadequate dietary control.
During treatment, blood samples will be taken to monitor whether the treatment is appropriate and to ensure that there are no side effects causing blood abnormalities.
If you are receiving Orfadin for the treatment of hereditary tyrosinemia type 1, you will undergo periodic liver monitoring because the disease affects the liver.
Your doctor should monitor you every 6 months. If you experience any adverse reactions, shorter monitoring intervals are recommended.
Other medicines and Orfadin
Inform your doctor or pharmacist if you are taking, have recently taken, or might need to take any other medicines.
Orfadin may interfere with the effect of other medicines, such as:
- medicines for epilepsy (such as phenytoin)
- medicines to prevent blood clotting (such as warfarin)
Use of Orfadin with food
If you start treatment taking Orfadin with food, it is recommended to continue this regimen throughout the entire treatment.
Pregnancy and breast-feeding
The safety of this medicine has not been studied in pregnant or breast-feeding women.
Consult your doctor if you are planning to become pregnant. If you become pregnant, you must consult your doctor immediately.
Do not breast-feed while taking this medicine (see section “Do not take Orfadin”).
Driving and using machines
The effect of this medicine on the ability to drive and use machines is minor. However, if you experience any adverse effects affecting your vision, you should not drive or operate machinery until your vision returns to normal (see section 4 “Possible side effects”).
3. How to take Orfadin
Follow exactly the instructions for using this medicine given by your doctor. If you are unsure, consult your doctor or pharmacist.
For hereditary tyrosinemia type 1, treatment with this medicine should be initiated and supervised by a physician experienced in the management of this disease.
For hereditary tyrosinemia type 1, the recommended 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, because data are limited in patients with body weight < 20 kg, in this patient population the total daily dose should be divided into two doses per day.
For AKU, the recommended dose is 10 mg once daily.
If you have difficulty swallowing the capsules, you may open the capsules and mix the powder with a small amount of water or dietary supplement before taking it.
If you take more Orfadin than you should
If you have taken more of this medicine than you should, inform your doctor or pharmacist immediately.
If you forget to take Orfadin
Do not take a double dose to make up for forgotten doses. If you have missed a dose, inform your doctor or pharmacist.
If you stop taking Orfadin
If you feel that the medicine's effect is not adequate, inform your doctor. Do not change the dose or stop treatment without first speaking to your doctor.
If you have any doubts about how to use this medicine, ask your doctor or pharmacist.
4. Possible adverse effects
Like all medicines, this medicine can cause adverse effects, although not everyone experiences them.
If you notice any eye-related adverse effects, inform your doctor immediately so that an ophthalmological examination can be performed. Treatment with nitisinone increases blood levels of tyrosine, which may cause eye-related symptoms. In patients with hereditary tyrosinemia type 1, frequently reported ocular adverse effects (may affect more than 1 in 100 people) due to elevated tyrosine levels include eye inflammation (conjunctivitis), corneal clouding and inflammation (keratitis), light sensitivity (photophobia), and eye pain. Inflammation of the eyelids (blepharitis) is an uncommon adverse effect (may affect up to 1 in 100 people).
In patients with AKU, eye irritation (keratopathy) and eye pain are very commonly reported adverse effects (may affect more than 1 in 10 people).
The following is a list of other adverse effects reported in patients with hereditary tyrosinemia type 1:
Other common adverse effects
- Decrease in platelet count (thrombocytopenia) and white blood cells (leukopenia), reduction in certain types of white blood cells (granulocytopenia).
Other uncommon adverse effects
- Increase in white blood cell count (leukocytosis),
- Itching (pruritus), skin inflammation (exfoliative dermatitis), rash.
The following is a list of other adverse effects reported in patients with AKU:
Other common adverse effects
- Bronchitis,
- Pneumonia,
- Itching (pruritus), rash.
Reporting of adverse effects
If you experience any type of adverse effect, talk to your doctor, pharmacist, or nurse, even if it is a possible adverse effect not listed in this leaflet. You can also report them directly through the national reporting system included in Appendix V. By reporting adverse effects, you can help provide more information on the safety of this medicine.
5. Storage of Orfadin
Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date stated on the bottle and the carton after “EXP”. The expiry date refers to the last day of the month indicated.
Store in the refrigerator (between 2 °C and 8 °C).
The medicine may be stored for a single period of 2 months (for 2 mg capsules) or 3 months (for 5 mg, 10 mg and 20 mg capsules) at a temperature not exceeding 25 °C, after which it must be discarded.
Remember to write on the bottle the date when it was removed from the refrigerator.
Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines and containers you no longer need. This will help protect the environment.
6. Contents of the pack and further information
Composition of Orfadin
- The active substance is nitisinone.
Orfadin 2 mg: each capsule contains 2 mg of nitisinone.
Orfadin 5 mg: each capsule contains 5 mg of nitisinone.
Orfadin 10 mg: each capsule contains 10 mg of nitisinone.
Orfadin 20 mg: each capsule contains 20 mg of nitisinone.
- The other components (excipients) are:
Capsule contents: starch, pregelatinized (from maize).
Capsule shell: gelatin, titanium dioxide (E 171).
Printing ink: iron oxide (E 172), shellac, propylene glycol, ammonium hydroxide.
Appearance of the product and contents of the pack
The capsules are hard, white, opaque, gelatin capsules, printed with “NTBC” and the dose “2 mg”, “5 mg”, “10 mg” or “20 mg” in black. The capsule contains a powder which may be white or greyish.
The capsules are packed in plastic bottles with tamper-evident closures. Each bottle contains 60 capsules.
Marketing Authorisation Holder
Swedish Orphan Biovitrum International AB
SE-112 76 Stockholm
Sweden
Manufacturer
Apotek Produktion & Laboratorier AB
Prismavägen 2
SE-141 75 Kungens Kurva
Sweden
Date of the most recent revision of this leaflet:
Detailed information on this medicine is available on the European Medicines Agency website: http://www.ema.europa.eu/. Links to other websites on rare diseases and orphan medicines are also provided.