Product Information for the User

Nitisinone MDK 2 mg Hard Capsules

Nitisinone MDK 5 mg Hard Capsules

Nitisinone MDK 10 mg Hard Capsules

nitisinone

Read this entire leaflet carefully before you start taking the medicine, as 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. Do not pass it on to others even if they have the same symptoms as you, as it may harm them.
• If you experience any side effects, talk to your doctor or pharmacist, even if they are not listed in this leaflet. See section 4.

1. What you need to know before taking Nitisinone MDK
2. How to take Nitisinone MDK
3. Possible side effects

1. Storage of Nitisinone MDK
2. Contents of the pack and additional information

The active ingredient of Nitisinone MDK is nitisinone. This medication is used for the treatment of a rare disease called hereditary tyrosinemia type 1 in adults, adolescents, and children (of any age group).

In this disease, your body cannot completely break down the amino acid tyrosine (amino acids are the fundamental elements of proteins), forming toxic substances. These toxic substances accumulate in your body. Nitisinone MDK blocks the breakdown of tyrosine, and toxic substances are not formed.

You must follow a special diet while taking this medication, because tyrosine will still be in your body. This diet is based on a low content of tyrosine and phenylalanine (another amino acid).

Do not take Nitisinone MDK

• 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 breastfeeding”).

Warnings and precautions

Consult your doctor or pharmacist before starting to take Nitisinone MDK,

• Your ophthalmologist will examine your eyes before treatment and regularly during treatment with nitisinone. If you notice redness of the eyes or any other eye effect, contact your doctor immediately for an eye examination. Eye problems (see section 4) may be a sign of inadequate dietary control.

Blood samples will be taken during treatment to control whether the treatment is adequate and to ensure that there are no blood-related side effects.

You will have regular liver checks because the disease affects the liver.

Your doctor should follow up every 6 months. If you experience any side effects, it is recommended to use shorter time intervals.

Other medicines and Nitisinone MDK

Inform your doctor or pharmacist if you are taking, have taken recently, or may need to take any other medicine.

Nitisinone MDK may interfere with the effect of other medicines, such as:

• Medicines for epilepsy (such as phenytoin)
• Blood-thinning medicines (such as warfarin)

Use of Nitisinone MDK with food and drink

If you start treatment taking it with food and drink, it is recommended to follow this regimen throughout the treatment.

Pregnancy and breastfeeding

The safety of this medicine has not been studied in pregnant women and breastfeeding women.

Consult your doctor if you plan to become pregnant. If you become pregnant, you must consult your doctor immediately.

Do not breastfeed while taking this medicine (see section “Do not take Nitisinone MDK ”).Driving and operating machines

The influence of this medicine on the ability to drive and use machines is small. However, if you experience side effects that affect vision, you should not drive or use machines until your vision has returned to normal (see section 4 “Possible side effects”).

Follow exactly the medication administration instructions provided by your doctor. If in doubt, ask your doctor or pharmacist.

The treatment with this medication should be initiated and supervised by a doctor experienced in the treatment of the disease (hereditary tyrosinemia type 1).

The recommended daily dose is 1 mg/kg of body weight administered orally. Your doctor will adjust the dose individually. It is recommended to administer the dose once a day.

However, due to limited data in patients with a body weight of <20

If you have trouble swallowing the capsules, you can open the capsules and mix the powder with a small amount of water or dietary supplement, before taking it.

If you take more Nitisinone MDK than you should

If you have taken more of this medication than you should, inform your doctor or pharmacist immediately.

If you forgot to take Nitisinone MDK

Do not take a double dose to compensate for the missed doses. If you have forgotten to take a dose, inform your doctor or pharmacist.

If you interrupt treatment with Nitisinone MDK

If you estimate that the medication's action is not adequate, inform your doctor. Do not change the dose or suspend treatment without first speaking with your doctor.

If you have any doubts about the use of this medication, ask your doctor or pharmacist.

Like all medications, this medication may produce adverse effects, although not everyone will experience them.

If you notice any eye-related adverse effects, inform your doctor immediately so that they can perform an ophthalmological examination. Treatment with nitisinona increases blood tyrosine levels, which can cause eye-related symptoms. Frequent eye-related adverse effects (affecting more than 1 in 10 people) due to higher tyrosine levels are eye inflammation (conjunctivitis), corneal opacity and inflammation (keratitis), light sensitivity (photophobia), and eye pain. Blepharitis, an inflammation of the eyelids, is a rare adverse effect (affecting up to 1 in 100 people).

Other Frequent Adverse Effects

• Decreased platelet count (thrombocytopenia) and white blood cell count (leucopenia), reduction of certain types of white blood cells (granulocytopenia).

Other Rare Adverse Effects

• Increased white blood cell count (leucocytosis),
• Itching (pruritus), skin inflammation (dermatitis exfoliativa), rash.

Reporting Adverse Effects

If you experience any type of adverse effect, consult your doctor, pharmacist, or nurse, even if it is a possible adverse effect not listed in this prospectus. You can also report them directly through the national notification system included in theAppendix V. By reporting adverse effects, you can contribute to providing more information about the safety of this medication.

Keep this medication out of the sight and reach of children.

Do not use this medication after the expiration date that appears on the box and the bottle after “EXP”. The expiration date is the last day of the month indicated.

Store in refrigerator (between 2 °C and 8 °C). Store in the original container to protect it from light. The medication can be stored for a period of 2 months, after the first opening of the container, at a temperature not exceeding 25 °C, after which it must be discarded.

Do not forget to note on the container the date when you removed it from the refrigerator.

Medicines should not be thrown down the drains or in the trash. Ask your pharmacist how to dispose of the containers and the medications that you no longer need. In this way, you will help to protect the environment.

Composition of Nitisinone MDK

• The active ingredient is nitisinone.

Nitisinone MDK 2 mg:Each capsule contains 2 mg of nitisinone.

Nitisinone MDK 5 mg:Each capsule contains 5 mg of nitisinone.

Nitisinone MDK 10 mg:Each capsule contains 10 mg of nitisinone.

• The other components (excipients) are:

Capule content: Maize pregelatinized starch

Capsule coating: Gelatin, titanium dioxide (E 171)

Printing: Iron oxide black (E 172), shellac

Appearance of the product and contents of the packaging

Nitisinone MDK capsules are white, opaque, hard, gelatin capsules measuring 15.7 mm in length and have "Nitisinone" and the dose "2 mg", "5 mg" or "10 mg" printed in black. The capsules contain a white or off-white powder.

The capsules are packaged in plastic bottles. Each bottle contains 60 capsules. Each carton contains a bottle.

Marketing Authorization Holder

MendeliKABS Europe Limited

The Light Box

111 Power Rd, Unit G.07, Chiswick

London, W4 5PY, United Kingdom

Manufacturer

Elara Pharmaservices Limited

Iron Farm

7 Grimes Gate, Diseworth

Leicestershire, DE74 2QD

United Kingdom

Last review date of this leaflet:

Other sources of information

The detailed information on this medicine is available on the European Medicines Agency website:http://www.ema.europa.eu/.