Sapropterin Tarbis 100 mg soluble tablets EFG

Package leaflet: Information for the user

Introduction

Package leaflet: information for the user

Sapropterin Tarbis 100 mg dispersible tablets EFG

sapropterin dihydrochloride

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 or pharmacist.
• 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 or pharmacist, even if they are adverse effects not listed in this leaflet. See section 4.

Contents of the leaflet

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

1. What Sapropterin Tarbis is and what it is used for

Sapropterin contains the active substance sapropterin, which is a synthetic copy of a substance naturally present in the body called tetrahydrobiopterin (BH4). BH4 is required in the body to convert an amino acid called phenylalanine into another amino acid called tyrosine.

Sapropterin is used to treat hyperphenylalaninemia (HPA) or phenylketonuria (PKU) in patients of any age. HPA and PKU are caused by abnormally high levels of phenylalanine in the blood, which can be harmful. Sapropterin reduces these levels in some patients who respond to BH4 and may help increase the amount of phenylalanine that can be included in the diet.

This medicine is also used to treat an inherited disorder called BH4 deficiency in patients of any age, in which the body cannot produce sufficient amounts of BH4. Because BH4 levels are very low, the body cannot properly process phenylalanine, leading to increased levels of this amino acid, which can have harmful effects. By replacing the BH4 that the body cannot produce, sapropterin reduces the harmful excess of phenylalanine in the blood and increases tolerance to dietary phenylalanine.

2. What you need to know before starting Sapropterin Tarbis

Do not take Sapropterin Tarbis

If you are allergic to sapropterin or to any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Talk to your doctor or pharmacist before starting to take sapropterin, especially:

• if you are 65 years of age or older
• if you have kidney or liver problems
• if you are unwell. It is recommended to consult your doctor during illness, as blood phenylalanine levels may increase
• if you have a predisposition to seizures

While receiving treatment with sapropterin, your doctor will perform blood tests to monitor your levels of phenylalanine and tyrosine, and will decide whether to adjust your dose of sapropterin or your diet as necessary.

You must continue your dietary treatment as recommended by your doctor. Do not change your diet without informing your doctor. Even while taking sapropterin, if your blood phenylalanine levels are not well controlled, you may experience serious neurological problems. Your doctor must continue to monitor your blood phenylalanine levels regularly during treatment with sapropterin, to ensure they are neither too high nor too low.

Use of Sapropterin Tarbis with other medicines

Tell your doctor or pharmacist if you are taking, have recently taken, or might need to take any other medicines. In particular, you must inform your doctor if you are using:

• levodopa (used to treat Parkinson's disease)
• medicines for the treatment of cancer (e.g., methotrexate)
• medicines for the treatment of bacterial infections (e.g., trimethoprim)
• medicines that cause blood vessel dilation (e.g., glyceryl trinitrate [GTN], isosorbide dinitrate [ISDN], sodium nitroprusside [SNP], molsidomine, minoxidil).

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, think you might be pregnant, or are planning to become pregnant, consult your doctor or pharmacist before using this medicine.

If you are pregnant, your doctor will advise you on how to properly manage your phenylalanine levels. If these levels are not strictly controlled before or during pregnancy, it may be harmful to you and your baby. Your doctor will monitor dietary restriction of phenylalanine intake before and during pregnancy.

If a strict diet does not sufficiently reduce the amount of phenylalanine in your blood, your doctor will consider whether you should take this medicine.

You must not use this medicine during breastfeeding.

Driving and using machines

Sapropterin is not expected to affect your ability to drive or operate machinery.

Important information about some of the ingredients of Sapropterin Tarbis

This medicine contains less than 1 mmol of sodium (23 mg) per tablet; this is essentially "sodium-free".

3. How to take Sapropterin Tarbis

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

Dosage for PKU

The recommended starting dose of sapropterin in patients with PKU is 10 mg per kg of body weight. Take sapropterin once daily with a meal to enhance absorption, at the same time each day, preferably in the morning. Your doctor may adjust the dose, usually between 5 and 20 mg per kg of body weight per day, depending on your condition.

Dosage for BH4 deficiency

The recommended starting dose of sapropterin in patients with BH4 deficiency is 2 to 5 mg per kg of body weight. Take sapropterin with a meal to enhance absorption. Divide the total daily dose into 2 or 3 doses taken throughout the day. Your doctor may adjust the dose up to 20 mg per kg of body weight per day, depending on your condition.

The following table is an example of how to calculate the appropriate dose

Administration method

For patients with PKU, the total daily dose should be taken once daily at the same time each day, preferably in the morning.

For patients with BH4 deficiency, the total daily dose should be divided into 2 or 3 doses taken throughout the day.

Use in all patients

Place the prescribed tablets into a glass or cup of water, exactly as described below, and stir until they dissolve.

The tablets may take a few minutes to dissolve. To speed up dissolution, you may crush the tablets. Small particles may be visible in the solution, but these will not affect the effectiveness of the medicine. Drink the dissolved sapropterin solution with a meal within 15 to 20 minutes after preparation.

Do not ingest the desiccant capsule included in the bottle.

Use in patients with body weight above 20 kg

Place the tablets in a glass or cup (120 to 240 ml) of water and stir until they dissolve.

Use in children up to 20 kg body weight

The dose is based on body weight, which will change as the child grows. Your doctor will advise you on:

• the number of sapropterin tablets required for one dose
• the amount of water needed to mix one dose of sapropterin
• the volume of solution to administer to the child for the prescribed dose

The child should drink the sapropterin solution with a meal.

Administer the prescribed volume of solution to the child within 15 or 20 minutes after dissolving the tablets. If you cannot administer the dose within 15 or 20 minutes after dissolving the tablets, you must prepare a fresh solution, as the unused solution must not be used after 20 minutes have elapsed.

Materials required to prepare and administer the sapropterin dose to the child

• The number of sapropterin tablets required for one dose
• A dosing cup with graduated markings at 20, 40, 60, and 80 ml
• A glass or cup
• A clean spoon or stirring utensil
• An oral syringe (graduated in 1 ml increments; a 10 ml oral syringe for administering volumes ≤10 ml or a 20 ml oral syringe for administering volumes >10 ml)

Ask your doctor for the dosing cup to dissolve the tablets and the 10 ml or 20 ml oral syringe if you do not already have these materials.

Steps for preparing and administering the dose:

• Place the prescribed tablets into the dosing cup. Add the amount of water indicated by your doctor (e.g., your doctor may have instructed you to use 20 ml to dissolve one sapropterin tablet). Ensure the liquid level aligns with the corresponding graduation mark on the dosing cup as specified by your doctor. Stir with a clean spoon or utensil until the tablets dissolve.
• If your doctor has instructed you to administer only part of the solution, insert the tip of the oral syringe into the dosing cup. Slowly pull back the plunger to withdraw the volume specified by your doctor.
• Then transfer the solution to a glass or administration cup by slowly pushing the plunger until all the solution previously drawn into the oral dosing syringe is dispensed into the glass or cup (e.g., if your doctor instructed you to dissolve two sapropterin tablets in 40 ml of water and administer 30 ml to the child, you will need to use the 20 ml oral dosing syringe twice to withdraw the 30 ml of solution (e.g., 20 ml + 10 ml) and transfer it to the administration cup). Use a 10 ml oral syringe for volumes ≤10 ml or a 20 ml oral syringe for volumes >10 ml.
• If the infant is too young to drink from a glass or cup, you may administer the solution using the oral syringe. Withdraw the prescribed volume of prepared solution from the dosing cup and insert the tip of the oral syringe into the infant’s mouth. Direct the tip of the oral syringe toward one of the cheeks. Slowly push the plunger so that a small amount is released at a time, until the entire solution in the oral syringe has been administered.
• Discard any remaining unused solution. Remove the plunger from the body of the oral syringe. Wash both parts of the oral syringe and the dosing cup with warm water and allow them to air dry. Once the oral syringe is dry, reinsert the plunger into the syringe body. Store the oral syringe and dosing cup for next use.

If you take more Sapropterin Tarbis than you should

If you take more sapropterin than prescribed, you may experience adverse effects, which may include headache and dizziness. If you take more than the prescribed amount of sapropterin, 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 medication and the amount ingested.

If you forget to take Sapropterin Tarbis

Do not take a double dose to make up for missed doses. Take the next dose at your usual time.

If you stop taking Sapropterin Tarbis

Do not stop treatment with sapropterin without first discussing it with your doctor, as blood phenylalanine levels may increase.

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 can cause adverse effects, although not everyone experiences them.

A few cases of allergic reactions (such as skin rashes and severe reactions) have been reported. Their frequency is unknown (the frequency cannot be estimated from the available data).

If you have inflamed, red, and intensely itchy areas (urticaria), runny nose, rapid or irregular pulse, swelling of the tongue or throat, sneezing, wheezing, severe breathing difficulty, or dizziness, you may be experiencing a severe allergic reaction to the medicine. If you experience these symptoms, consult your doctor immediately.

Very common adverse effects (may affect more than 1 in 10 people):

Headache and runny nose.

Common adverse effects (may affect up to 1 in 10 people):

Sore throat, nasal congestion or blockage, cough, diarrhoea, vomiting, stomach pain, blood phenylalanine levels too low, indigestion, and general malaise (nausea) (see section 2: "Warnings and precautions").

Adverse effects frequency not known (cannot be estimated from the available data):

Gastritis (inflammation of the stomach lining), oesophagitis (inflammation of the oesophageal lining).

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 via the Spanish Pharmacovigilance System for Human Medicines: https://www.notificaram.es.

By reporting adverse effects, you can help provide more information on the safety of this medicine.

5. Storage of Sapropterin Tarbis

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 packaging following EXP. The expiry date refers to the last day of the month indicated.

This medicine does not require any special storage temperature.

Keep the bottle tightly closed to protect it from moisture.

Medicines must not be disposed of via wastewater or household waste. Dispose of unused medicines and their containers at the SIGRE collection point at your pharmacy. Ask your pharmacist how to properly dispose of medicines and containers you no longer need. This will help protect the environment.

6. Contents of the pack and other information

Composition of Sapropterin Tarbis

The active substance is sapropterin dihydrochloride.

Each tablet contains 100 mg of sapropterin dihydrochloride (equivalent to 77 mg of sapropterin).

The other components are

Mannitol (E421), ascorbic acid (E300), crospovidone (Type A), riboflavin (E101), colloidal anhydrous silica, stearic acid fumarate, and sodium.

Appearance of the product and contents of the pack

Round, speckled tablet, white to light yellow in colour, with an approximate diameter of 9.5 mm, marked with "I 1" on one side and smooth on the other.

Sapropterin Tarbis is available in child-resistant bottles containing 30 or 120 soluble tablets. Each bottle contains a small plastic desiccant tube (silica gel and oxygen absorber).

Only certain pack sizes may be marketed.

Marketing Authorization Holder

Tarbis Farma S.L.

Gran Vía Carlos III, 94

08028 Barcelona

Spain

Manufacturer responsible

Amarox Pharma B.V.

Rouboslaan 32

Voorschoten, 2252TR

The Netherlands

This medicinal product is authorized in the Member States of the European Economic Area under the following names:

Germany: Sapropterin Amarox 100 mg Tabletten zur Herstellung einer Lösung zum Einnehmen

The Netherlands: Sapropterine Amarox 100 mg oplosbare tabletten

Spain: Sapropterina Tarbis 100 mg comprimidos solubles EFG

Date of the most recent review of this leaflet: June 2024

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