Nitisinone Dipharma 10 mg hard capsules EFG

Package leaflet: Information for the user

Introduction

Package leaflet: information for the user

Nitisinona Dipharma 2 mg hard capsules EFG

Nitisinona Dipharma 5 mg hard capsules EFG

Nitisinona Dipharma 10 mg hard capsules EFG

Nitisinona Dipharma 20 mg hard capsules EFG

nitisinone

Read all of this leaflet carefully before you start taking this medicine, because it contains important information for you.

• Keep this leaflet. 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 other people, even if they have the same symptoms as you, because it may harm them.
• If you experience any side effects, talk to your doctor, pharmacist, or nurse, even if it is a side effect not listed in this leaflet. See section 4.

What is in this leaflet:

1. What Nitisinona Dipharma is and what it is used for
2. What you need to know before taking Nitisinona Dipharma
3. How to take Nitisinona Dipharma
4. Possible side effects
5. How to store Nitisinona Dipharma
6. Contents of the pack and other information

1. What Nitisinona Dipharma is and what it is used for

Nitisinona Dipharma contains the active substance nitisinone. Nitisinone is used to treat:

• a rare disease called hereditary tyrosinemia type 1 in adults, adolescents, and children (of any age group).
• 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. Nitisinone 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 based on low levels of 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 Nitisinone Dipharma

Do not take Nitisinone Dipharma

• 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 Nitisinone Dipharma.

• An ophthalmologist will examine your eyes before 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.

Blood samples will be taken during treatment to monitor whether the treatment is appropriate and to ensure that there are no side effects causing blood abnormalities.

If you are receiving Nitisinone Dipharma for the treatment of hereditary tyrosinemia type I, 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 Nitisinone Dipharma

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

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

• Medicines for epilepsy (like phenytoin)
• Medicines to prevent blood clots (like warfarin)

Taking Nitisinone Dipharma with food

If you start treatment taking it 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.

If you are pregnant or breast-feeding, think you may be pregnant, or are planning to become pregnant, consult your doctor or pharmacist before using this medicine. Do not breast-feed while taking this medicine (see section “Do not take Nitisinone Dipharma”).

Driving and using machines

The effect of this medicine on the ability to drive and use machines is minor. However, if you experience 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 Nitisinone Dipharma

Follow exactly the administration instructions for this medicine as indicated by your doctor. If in doubt, consult your doctor or pharmacist again.

For hereditary tyrosinemia type I, treatment with this medicine must be initiated and supervised by a physician experienced in the management of this disease.

For hereditary tyrosinemia type I, 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 it is recommended to divide the total daily dose into two daily doses.

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, apple juice, soft food such as apple puree, or dietary supplement, including an amino acid-based infant formula, before taking it.

If you take more Nitisinone Dipharma than you should

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

In case of overdose or accidental ingestion, contact your doctor or pharmacist immediately or call the Toxicology Information Service at telephone number: 91 562 04 20, stating the medicine and the amount ingested.

If you forget to take Nitisinone Dipharma

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

If you stop taking Nitisinone Dipharma

If you consider that the effect of the medicine is not adequate, inform your doctor. Do not change the dose or stop treatment without first speaking with your doctor.

If you have any further questions about the use of this medicine, ask your doctor or pharmacist.

4. Possible adverse effects

Like all medicines, this medicine may cause adverse effects, although not everyone will experience 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 ocular inflammation (conjunctivitis), corneal opacity 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).

Other adverse effects reported in patients with hereditary tyrosinemia type 1 are listed below:

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.
• Other adverse effects reported in patients with AKU are listed below:
• Other common adverse effects
  • bronchitis,
  • pneumonia,
  • itching (pruritus), rash.

Reporting of 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 leaflet. You may also report them directly via the Spanish Pharmacovigilance System for Human Medicines (www.notificaRAM.es). By reporting adverse effects, you can help provide more information on the safety of this medicine.

5. Storage of Nitisinone Dipharma

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

Do not use this medicine after the expiry date stated on the blister after "EXP" and on the bottle and carton after "CAD". The expiry date refers to the last day of the month indicated.

Store below 30 °C.

Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines and packaging that you no longer need. This will help protect the environment.

6. Contents of the pack and other information

Composition of Nitisinone Dipharma

• The active substance is nitisinone.

Nitisinone Dipharma 2 mg: each hard capsule contains 2 mg of nitisinone.

Nitisinone Dipharma 5 mg: each hard capsule contains 5 mg of nitisinone.

Nitisinone Dipharma 10 mg: each hard capsule contains 10 mg of nitisinone.

Nitisinone Dipharma 20 mg: each hard capsule contains 20 mg of nitisinone.

• The other components (excipients) are:

Contents of the hard capsule:

Pregelatinized starch (from corn)
Stearic acid

Capsule shell:

Gelatin
Titanium dioxide (E171)

Printing ink:

Shellac
Propylene glycol
Aluminium indigotine lacquer (E132)

Nature of the product and pack contents

The capsules are hard, white, opaque, made of gelatin, and marked with the "company logo" and the dose "2", "5", "10" or "20 mg" in dark blue ink. The capsule contains a powder which may be white or almost white.

Nitisinone Dipharma is available in plastic bottles with tamper-evident closure containing 60 capsules, and in OPA/Alu/PVC-Alu unit-dose perforated blisters containing 60 capsules.

Only certain pack sizes may be marketed.

Marketing Authorization Holder

Dipharma Arzneimittel GmbH
Offheimer Weg 33
65549 Limburg a.d. Lahn
Germany

Manufacturer

Doppel Farmaceutici S.r.l.
Via Volturno 48
20089 Quinto de Stampi – Rozzano (MI), Italy

For further information about this medicine, please contact the local representative of the Marketing Authorization Holder:

Aurovitas Spain, S.A.U.
Avda. de Burgos, 16-D
28036 Madrid
Spain
Tel.: 91 630 86 45

This medicine is authorized in the European Economic Area member states under the following names:

Germany Nitisinone Dipharma
Austria Nitisinon Dipharma
Belgium Nitisinone Dipharma
Denmark Nitisinone Dipharma
Slovakia Nitisinone Dipharma
Spain Nitisinona Dipharma
Finland Nitisinone Dipharma
France Nitisinone Dipharma
Ireland Nitisinone Dipharma
Italy Nitisinone Dipharma
Norway Nitisinone Dipharma
Netherlands Nitisinone Dipharma
Portugal Nitisinona Dipharma
United Kingdom Nitisinone Dipharma
Sweden Nitisinone Dipharma

Date of the most recent revision of this leaflet: May 2025.

Detailed information on this medicine is available on the website of the Spanish Agency of Medicines and Health Products (AEMPS) (http://www.aemps.gob.es/).