2 mg, 5 mg, 10 mg, 20 mg hard capsules
nitisinone
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 (in all ages)
- a rare disease called alkaptonuria (LFS) in adults.
In these diseases, your body can not break down the amino acid tyrosine completely ( amino acids are the building blocks of our proteins), which causes harmful substances to be formed. These substances accumulate in your body. Orfadin blocks the breakdown of tyrosine and therefore the harmful substances are not formed.
When treating hereditary tyrosinemia type 1, you must follow a special diet when taking this medicine as tyrosine will remain in your body. This particular diet has low levels of tyrosine and phenylalanine (another amino acid ).
When treating AKU, your doctor may advise you to follow a special diet.
What you need to know before you take Orfadin
Do not take Orfadin
- if you are allergic to nitisinone or any of the other ingredients of this medicine (listed in section 6).
Do not breastfeed while taking this medicine, see section “Pregnancy and breast-feeding”.
Warnings and cautions
Talk to your doctor or pharmacist before taking Orfadin.
- Your eyes are checked by an ophthalmologist before and regularly during treatment with nitisinone. If your eyes turn red or show other signs of exposure, contact your doctor immediately to have your eyes examined. Eye problems may be a sign of inadequate dietary control, see section 4.
During the treatment, blood tests will be taken so that your doctor can check that the treatment is adequate and to ensure that it does not have side effects that cause blood disorders.
If you are receiving Orfadin for the treatment of hereditary tyrosinemia type 1, your liver will be checked regularly as the disease affects the liver.
A follow-up examination should be done every 6 months by your doctor. If you experience side effects, shorter intervals between examinations are recommended.
Other medicines and Orfadin
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines.
Orfadin may affect the effectiveness of other medicines, for example:
- antiepileptic drugs (eg phenytoin )
- anticoagulants (eg warfarin ).
Orfadin with food
If you start treatment with Orfadin with food, you should continue with it throughout the treatment.
Pregnancy and breastfeeding
The safety of this medicine has not been studied in pregnant and lactating women.
Contact your doctor if you are planning to become pregnant. If you become pregnant, you should contact your doctor immediately.
Do not breastfeed while taking this medicine, see section “Do not take Orfadin”.
Driving and using machines
This medicine has less effect on the ability to drive and use machines. However, if you experience side effects that affect your vision, do not drive or use machines until your vision is normal again (see section 4 “Possible side effects”).
How to take Orfadin
Always take this medicine exactly as your doctor has told you. Ask your doctor or pharmacist if you are unsure.
For hereditary tyrosinemia type 1, treatment with this medicine should be initiated and supervised by a doctor who has experience in the treatment of the disease.
For hereditary tyrosinemia type 1, the recommended total daily dose is 1 mg/kg body weight. Your doctor will adjust the dose individually.
Dosage once daily is recommended. However, due to limited data in patients with body weight <20 kg, it is recommended that the total daily dose be divided into two daily doses for these patients.
For LFS, the recommended dose is 10 mg once daily.
If you have problems swallowing capsules, you can open them and mix the powder in a small amount of water or diet product in liquid form just before taking the medicine.
If you take more Orfadin than you should
If you have taken more medicine than you should, contact your doctor or pharmacist as soon as possible.
If you forget to take Orfadin
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 Orfadin
Contact your doctor if you feel that the effect of the medicine is too strong or weak. Do not change the dose or stop treatment without talking to your doctor.
If you have any further questions on the use of this product, ask your doctor, pharmacist, or nurse.
Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them.
If you notice any side effects that affect your eyes, you should consult your doctor immediately to have your eyes examined. Treatment with nitisinone leads to higher tyrosine levels in the blood which can cause eye-related symptoms. Common eye-related side effects (may affect more than 1 in 100 patients) caused by higher tyrosine levels in patients with hereditary tyrosinemia type 1 are inflammation of the eye ( conjunctivitis ), cloudiness, inflammation of the cornea ( keratitis ), photosensitivity ( photophobia ), and eye pain. Inflammation of the eyelid ( blepharitis ) is a less common side effect (may affect up to 1 in 100 people).
In patients with LFS, eye irritation ( keratopathy ) and eye pain are very commonly reported side effects (may affect more than 1 in 10 patients).
Other side effects reported in patients with hereditary tyrosinemia type 1 are listed below:
Other common side effects are
- Decreased platelet count ( thrombocytopenia ) and white blood cells (leukocytopenia), lack of certain white blood cells ( granulocytopenia ).
Other less common side effects are
- Increased white blood cell count ( leukocytosis ),
- itching ( pruritus ), dermatitis (exfoliative dermatitis ), rash.
Other side effects reported in patients with LFS are listed below:
Other common side effects are
- bronchitis
- pneumonia
- itching ( pruritus ), rash.
How to store Orfadin
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 can and carton after “EXP”. The expiration date is the last day of the specified month.
Store in a refrigerator (2 ° C‑ 8 ° C).
The product can be stored at temperatures up to 25 ° C for a single period of 2 months (for 2 mg capsules) or 3 months (for 5 mg, 10 mg, and 20 mg capsules), after which the product must be discarded.
Do not forget to write down the date on the jar when you take it out of the fridge.
Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. These measures will help to protect the environment.
Contents of the pack and other information
Content declaration
- The active substance is nitisinone.Orfadin 2 mg : Each capsule contains 2 mg nitisinone.Orfadin 5 mg : Each capsule contains 5 mg nitisinone.Orfadin 10 mg : Each capsule contains 10 mg of nitisinone.Orfadin 20 mg : Each capsule contains 20 mg of nitisinone.
- The other ingredients areCapsule contents:pregelatinized starch (from maize)Capsule shell:gelatinetitanium dioxide (E 171)Printing ink:iron oxide (E 172)shellackpropylene glycolammonium hydroxide
What the medicine looks like and contents of the pack
The capsules are white, opaque, hard, and made of gelatin marked with “NTBC” and the strength “2 mg”, “5 mg”, “10 mg” or “20 mg” in black. The capsules contain a white to off-white powder.
The capsules are packed in plastic jars with a guarantee-sealed seal. Each jar contains 60 capsules.
Marketing Authorisation Holder
Manufacturer
Apotek Produktion & Laboratorier AB
Prismavägen 2
SE-141 75 Kungens Kurva
Sweden