Prospecto: information for the user

Sapropterina STADA 100 mg soluble tablets EFG

dihydrochloride of sapropterin

Read this prospectus carefully before starting to take this medicine, as it contains important information for you.

• Keep this prospectus, as you may need to read it again.
• If you have any doubts, consult your doctor or pharmacist.
• This medicine has been prescribed only for you, and you must not give it to other people even if they have the same symptoms as you, as it may harm them.
• If you experience any adverse effects, consult your doctor or pharmacist, even if they are not listed in this prospectus. See section 4.

Content of the prospectus

1. What Sapropterina Stada is and for what it is used
2. What you need to know before starting to take Sapropterina Stada
3. How to take Sapropterina Stada
4. Possible adverse effects

5Storage of Sapropterina Stada

6.Contents of the package and additional information

Sapropterina Stada contains the active ingredient sapropterin, which is a synthetic copy of a substance found in the body called tetrahydrobiopterin (BH4). BH4 is necessary 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 that 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 medication is also used to treat a hereditary disease called tetrahydrobiopterin deficiency in patients of any age, in which the body cannot produce enough BH4.

Because BH4 levels are very low, the body cannot use phenylalanine properly, and levels of this amino acid increase, having harmful effects. By substituting the BH4 that the body cannot produce, sapropterin reduces the damaging excess of phenylalanine in the blood and increases tolerance to phenylalanine in the diet.

Do not take Sapropterina Stada

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

Warnings and precautions

Consult your doctor or pharmacist before starting sapropterin, especially:

• If you are 65 years or older
• If you have kidney or liver problems
• If you are ill. It is recommended to consult with your doctor in case of illness since blood phenylalanine levels may increase
• If you have a predisposition to seizures

When receiving treatment with sapropterin, your doctor will perform blood tests to verify the content of phenylalanine and tyrosine and decide whether to adjust the sapropterin dose or diet as needed.

You must continue the dietary treatment according to your doctor's recommendations. Do not change your diet without informing your doctor. Even when taking sapropterin, if your blood phenylalanine levels are not well controlled, you may experience severe neurological problems. Your doctor must continue to monitor your blood phenylalanine levels frequently during sapropterin treatment,to ensure they are not too high or too low.

Use of Sapropterina Stada with other medications

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

• levodopa (to treat Parkinson's disease)
• medications for cancer treatment (e.g. methotrexate)
• medications for bacterial infection treatment (e.g. trimethoprim)
• medications 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, or think you may be pregnant, consult your doctor or pharmacist before using this medication.

If you are pregnant, your doctor will instruct you on how to control your phenylalanine levels properly. If they are not strictly controlled before pregnancy or when you become pregnant, it may be harmful to you and your baby. Your doctor will supervise the restriction of phenylalanine intake through diet before and during pregnancy.

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

You should not use this medication during breastfeeding.

Driving and operating machinery

Sapropterin is not expected to affect the ability to drive and operate machinery.

Important information about some of the components of Sapropterina Stada

This medication contains less than 1 mmol of sodium (23 mg) per tablet; it is essentially “sodium-free”.

Follow exactly the administration instructions of this medication as indicated by your doctor. In case of doubt, consult your doctor again.

Dose for PKU

The recommended initial dose of sapropterin in patients with PKU is 10 mg per kg of body weight. Take sapropterin once a day, with a meal to increase absorption, and at the same time every day, preferably in the morning. Your doctor may adjust the dose, generally between 5 and 20 mg daily per kg of weight, according to your condition.

Dose for BH4 deficiency

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

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

Administration form

For patients with PKU, the total daily dose is taken once a day and at the same time every day, preferably in the morning.

For patients with BH4 deficiency, the total daily dose is divided into 2 or 3 doses distributed throughout the day.

Use in all patients

Place the prescribed tablets in 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 dissolve the tablets faster, you can crush them. You may notice small particles in the solution that will not affect the efficacy of the medication. Drink the prepared sapropterin solution with a meal within 15 or 20 minutes of preparation.

Do not ingest the desiccant capsule included in the bottle.

Use in patients with a body weight over 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 of body weight

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

• the number of sapropterin tablets needed for a dose
• the amount of water needed to mix a dose of sapropterin
• the amount of solution to administer to the child for the prescribed dose The child should drink the sapropterin solution with a meal.

Administer the child the prescribed amount of solution within 15 or 20 minutes of preparation. If you cannot administer the dose to the child within 15 or 20 minutes of preparation, you will need to prepare a new solution, as unused solution should not be used after 20 minutes.

Materials needed to prepare and administer the child's sapropterin dose

• The number of sapropterin tablets needed for a dose
• A medication cup with markings corresponding to 20, 40, 60, and 80 ml
• A glass or cup
• A spoon or clean utensil to stir
• A oral syringe (graduated in 1 ml divisions) (a 10 ml syringe for administering volumes ≤10 ml or a 20 ml syringe for administering volumes >10 ml)

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

Steps for preparation and taking the dose:

• Place the prescribed tablets in the medication cup. Add the indicated amount of water to the medication cup (e.g., your doctor told you to use 20 ml to dissolve a 100 mg sapropterin tablet). Check that the amount of liquid aligns (with the corresponding marking on the medication cup) with the amount indicated by your doctor. Stir with the spoon or clean utensil until the tablets dissolve.
• If your doctor told you to administer only a part of the solution, insert the tip of the oral syringe into the medication cup. Pull the plunger back slowly to extract the indicated amount.
• Next, transfer the solution to a glass or cup dosing device by pushing the plunger slowly until all the previously extracted solution is in the oral syringe (e.g., if your doctor told you to dissolve two 100 mg sapropterin tablets in 40 ml of water and administer 30 ml to the child, you will need to use the 20 ml oral syringe twice to extract the 30 ml (e.g., 20 ml + 10 ml) of solution and transfer it to a glass or cup for administration). Use an oral syringe of 10 ml for administering volumes ≤10 ml or an oral syringe of 20 ml for administering volumes >10 ml.
• If the baby is too small to drink from a glass or cup, you can administer the solution using the oral syringe. Extract the prescribed volume of prepared solution from the dosing device and insert the tip of the oral syringe into the baby's mouth. Point the tip of the oral syringe towards one cheek. Push the plunger slowly, releasing a small amount at a time, until you administer all the solution contained in the oral syringe.
• Discard any remaining solution. Extract the plunger from the body of the oral syringe. Wash both parts of the oral syringe and the dosing device with warm water and let them air dry. When the oral syringe is dry, reinsert the plunger into the body of the oral syringe. Store the oral syringe and medication cup for the next use.

If you take more Sapropterin Stada than you should

If you take more sapropterin than prescribed, you may experience adverse effects that may include headache and dizziness. If you take more sapropterin than prescribed, inform your doctor or pharmacist immediately.

In case of overdose or accidental ingestion, consult your doctor or pharmacist immediately or call the Toxicological Information Service, phone: 91 562 04 20, indicating the medication and the amount ingested.

If you forget to take Sapropterin Stada

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

If you interrupt treatment with Sapropterin Stada

Do not interrupt treatment with sapropterin without previously discussing it with your doctor, as this may increase blood phenylalanine levels.

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

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

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), nasal discharge, rapid or irregular pulse, tongue or throat inflammation, sneezing, hissing while breathing, severe respiratory difficulty, or dizziness, you may be experiencing a severe allergic reaction to the medication. If you experience these symptoms, consult your doctor immediately.

Very Common Adverse Effects(may affect more than 1 in 10 people)

Headache and nasal discharge.

Common Adverse Effects(may affect up to 1 in 10 people)

Sore throat, congestion or nasal blockage, cough, diarrhea, vomiting, stomach pain, low phenylalanine levels in blood tests, indigestion, and general discomfort (nausea) (see section 2: "Warnings and Precautions").

Unknown Adverse Effects(cannot be estimated from the available data)

Gastritis (inflammation of the stomach lining), esophagitis (inflammation of the esophagus lining).

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 Spanish System for Pharmacovigilance of Medicines for Human Use:www.notificaRAM.es. 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 bottle and on the box after CAD. The expiration date is the last day of the month indicated.

This medication does not require special conditions for conservation.

Medications should not be thrown down the drains or in the trash. Dispose of the containers and medications you no longer need at the SIGRE collection point of the pharmacy.If in doubt, ask your pharmacist how to dispose of the containers and medications you no longer need. By doing so, you will help protect the environment.

Aspect of the product and packaging content

Sapropterin Stada 100 mg soluble tablets EFG, are 10 mm white to light yellow round tablets, with "C" marked on one side.

Sapropterin Stada 100 mg soluble tablets EFG is available in:

• HDPE bottles with desiccant and polyester coil closed with PP cap.

Packaging sizes: 30 and 120 tablets

• Aluminum/PVC/Aluminum/OPA blisters

Packaging sizes: 30 and 120 tablets

• Single-dose aluminum/PVC/aluminum/OPA blisters

Packaging sizes: 30x1 and 120x1 single-dose tablets

It may only be marketed in some packaging sizes.

Holder of the marketing authorization and responsible for manufacturing

Holder of the marketing authorization

Laboratorio STADA, S.L.

Frederic Mompou, 5

08960 Sant Just Desvern (Barcelona)

Spain

[email protected]

Responsible for manufacturing

STADA Arzneimittel AG,

Stadastrasse 2 - 18,

61118 Bad Vilbel,

Germany

or

Coripharma ehf.,

Reykjavikurvegur 78,

IS -220 Hafnarfjordur,

Iceland

or

Centrafarm Services B.V,

Van de Reijtstraat 31 E,

4814NE Breda,

Netherlands

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

DenmarkSapropterin STADA

GermanySapropterin AL 100 mg Tabletten zur Herstellung einer Lösung zum Einnehmen

IcelandSapropterin STADA 100 mg lausnartöflur

NetherlandsSapropterine diHCl CF 100 mg, oplosbare tabletten

NorwaySapropterin STADA 100 mg oppløslige tabletter

SpainSapropterina STADA 100 mg comprimidos solubles EFG

SwedenSapropterin STADA 100 mg lösliga tabletter

Last review date of this leaflet:December 2023

The detailed information of this medicine is available on the website of the Spanish Agency for Medicines and Medical Devices (AEMPS)http://www.aemps.gob.es/